Grace Totoe MBChB SBB

Hematopoietic Stem Cell

Therapy

CME August 2012

Accra-Ghana

Hematopoietic Stem Cells

Cells capable of self renewal

and differentiation into all

blood cell lineages

Objectives

Historical overview

Sources

Indications

Complications

Historical Overview

The search for stem cells began in the aftermath

of the bombings in Hiroshima and Nagasaki in 1945

Survivors who died later developed myelodysplastic syndromes with recurrent infections bleeding complications and severe anemia

In 1949 Jacobson et al demonstrated that mice given doses of whole body X-irradiation developed the same radiation syndromes

Historical Overview

At a minimal lethal dose the mice died from hematopoietic failure approximately two weeks after radiation exposure

In 1951 Lorenzo et al using inbred strains of mice showed that whole-body-irradiated mice could be rescued from otherwise fatal hematopoietic failure by injection of suspensions of cells from blood-forming organs such as the bone marrow

Historical Overview

In 1956 three laboratories demonstrated that the injected bone marrow cells directly regenerated the blood-forming system

Hence the advent of hematopoietic (progenitor) stem cell (HPC) transplant

Sources of HPC

Collection ndash Peripheral blood by apheresis

ndash Bone marrow

ndash Umbilical cord blood

Donor ndash Primed mobilized with granulocyte colony

stimulating factor (G-CSF) to increase number of HPC in the peripheral blood for about 3-4 days before collection on Day 5

ndash Medications such as mozobil can be given to increase HPCs in the peripheral blood

NIHgov

Hematopoietic and stromal cell differentiation

Important Terminology

Autologous transplant- ldquotransplantrdquo using a personrsquos own

HPCs

Allogeneic transplant-The infusion of HPCs from another

individual into the patient Allogeneic cells may be from

related or unrelated

Syngeneic transplant- The infusion of HPCs from a twin

into the other twin

Xenogeneic transplant- using HPC from a non human

source or from human cells that have had ex vivo contact

with live non human animal cells

Important Terminology

CD 34+ cells- cell surface glycoprotein that functions as

an adhesion molecule Used to identify hematopoietic

stem cells

Engraftment- sign of cell recovery after transplant An

absolute neutrophil count gt500mm3 in 3 consecutive

days or platelet count 20-50K

GVHD-Post transplant complication characterized by skin liver and GI complications Occurs when the donor cells are recognized by the recipient cells as foreign and immunologically rejects the donor cells ndashmediated by T-lymphocytes

Important Terminology

What occurs prior to stem cell

collection

The routine workup of the donor includes a

history CBC BMP Red blood type and

screening for RBC allo antibodies chest x-ray

and electrocardiogram (EKG)

Infectious disease screening for hepatitis B and

C HIV Human T-cell lymphoma virus syphilis

and other viral diseases such as

cytomegalovirus herpes and West Nile

When and how are stem cells stored

Stored in cellular therapy labs under regulated

and monitored temperatures according to

standards set up by governing agencies- FDA

FACT AABB etc

If the collected stem cells are to be frozen or

stored for a longer period they are

cryopreserved with dimethyl sulphoxide (DMSO)

When are the stem cells given to the

patient

Stem cells are infused after the patient has undergone

the necessary treatment- chemotherapy radiation or

both- conditioning This is to reduce the tumor burden

and suppress the recipientrsquos immune system in order to

allow engraftment of stem cells

Conditioning can be myeloblative non-

myeloablative or reduced intensity-classification

is based on the duration of cytopenia and the

requirement for stem cell support

Bacigalupo et al Defining the intensity of conditioning regimens Working definitions Biol Blood Marrow Transplant 2009 December 15(12) 1628ndash1633l

Hematopoietic Stem Cells

Cells capable of self renewal

and differentiation into all

blood cell lineages

Objectives

Historical overview

Sources

Indications

Complications

Historical Overview

The search for stem cells began in the aftermath

of the bombings in Hiroshima and Nagasaki in 1945

Survivors who died later developed myelodysplastic syndromes with recurrent infections bleeding complications and severe anemia

In 1949 Jacobson et al demonstrated that mice given doses of whole body X-irradiation developed the same radiation syndromes

Historical Overview

At a minimal lethal dose the mice died from hematopoietic failure approximately two weeks after radiation exposure

In 1951 Lorenzo et al using inbred strains of mice showed that whole-body-irradiated mice could be rescued from otherwise fatal hematopoietic failure by injection of suspensions of cells from blood-forming organs such as the bone marrow

Historical Overview

In 1956 three laboratories demonstrated that the injected bone marrow cells directly regenerated the blood-forming system

Hence the advent of hematopoietic (progenitor) stem cell (HPC) transplant

Sources of HPC

Collection ndash Peripheral blood by apheresis

ndash Bone marrow

ndash Umbilical cord blood

Donor ndash Primed mobilized with granulocyte colony

stimulating factor (G-CSF) to increase number of HPC in the peripheral blood for about 3-4 days before collection on Day 5

ndash Medications such as mozobil can be given to increase HPCs in the peripheral blood

NIHgov

Hematopoietic and stromal cell differentiation

Important Terminology

Autologous transplant- ldquotransplantrdquo using a personrsquos own

HPCs

Allogeneic transplant-The infusion of HPCs from another

individual into the patient Allogeneic cells may be from

related or unrelated

Syngeneic transplant- The infusion of HPCs from a twin

into the other twin

Xenogeneic transplant- using HPC from a non human

source or from human cells that have had ex vivo contact

with live non human animal cells

Important Terminology

CD 34+ cells- cell surface glycoprotein that functions as

an adhesion molecule Used to identify hematopoietic

stem cells

Engraftment- sign of cell recovery after transplant An

absolute neutrophil count gt500mm3 in 3 consecutive

days or platelet count 20-50K

GVHD-Post transplant complication characterized by skin liver and GI complications Occurs when the donor cells are recognized by the recipient cells as foreign and immunologically rejects the donor cells ndashmediated by T-lymphocytes

Important Terminology

What occurs prior to stem cell

collection

The routine workup of the donor includes a

history CBC BMP Red blood type and

screening for RBC allo antibodies chest x-ray

and electrocardiogram (EKG)

Infectious disease screening for hepatitis B and

C HIV Human T-cell lymphoma virus syphilis

and other viral diseases such as

cytomegalovirus herpes and West Nile

When and how are stem cells stored

Stored in cellular therapy labs under regulated

and monitored temperatures according to

standards set up by governing agencies- FDA

FACT AABB etc

If the collected stem cells are to be frozen or

stored for a longer period they are

cryopreserved with dimethyl sulphoxide (DMSO)

When are the stem cells given to the

patient

Stem cells are infused after the patient has undergone

the necessary treatment- chemotherapy radiation or

both- conditioning This is to reduce the tumor burden

and suppress the recipientrsquos immune system in order to

allow engraftment of stem cells

Conditioning can be myeloblative non-

myeloablative or reduced intensity-classification

is based on the duration of cytopenia and the

requirement for stem cell support

Bacigalupo et al Defining the intensity of conditioning regimens Working definitions Biol Blood Marrow Transplant 2009 December 15(12) 1628ndash1633l

Objectives

Historical overview

Sources

Indications

Complications

Historical Overview

The search for stem cells began in the aftermath

of the bombings in Hiroshima and Nagasaki in 1945

Survivors who died later developed myelodysplastic syndromes with recurrent infections bleeding complications and severe anemia

In 1949 Jacobson et al demonstrated that mice given doses of whole body X-irradiation developed the same radiation syndromes

Historical Overview

At a minimal lethal dose the mice died from hematopoietic failure approximately two weeks after radiation exposure

In 1951 Lorenzo et al using inbred strains of mice showed that whole-body-irradiated mice could be rescued from otherwise fatal hematopoietic failure by injection of suspensions of cells from blood-forming organs such as the bone marrow

Historical Overview

In 1956 three laboratories demonstrated that the injected bone marrow cells directly regenerated the blood-forming system

Hence the advent of hematopoietic (progenitor) stem cell (HPC) transplant

Sources of HPC

Collection ndash Peripheral blood by apheresis

ndash Bone marrow

ndash Umbilical cord blood

Donor ndash Primed mobilized with granulocyte colony

stimulating factor (G-CSF) to increase number of HPC in the peripheral blood for about 3-4 days before collection on Day 5

ndash Medications such as mozobil can be given to increase HPCs in the peripheral blood

NIHgov

Hematopoietic and stromal cell differentiation

Important Terminology

Autologous transplant- ldquotransplantrdquo using a personrsquos own

HPCs

Allogeneic transplant-The infusion of HPCs from another

individual into the patient Allogeneic cells may be from

related or unrelated

Syngeneic transplant- The infusion of HPCs from a twin

into the other twin

Xenogeneic transplant- using HPC from a non human

source or from human cells that have had ex vivo contact

with live non human animal cells

Important Terminology

CD 34+ cells- cell surface glycoprotein that functions as

an adhesion molecule Used to identify hematopoietic

stem cells

Engraftment- sign of cell recovery after transplant An

absolute neutrophil count gt500mm3 in 3 consecutive

days or platelet count 20-50K

GVHD-Post transplant complication characterized by skin liver and GI complications Occurs when the donor cells are recognized by the recipient cells as foreign and immunologically rejects the donor cells ndashmediated by T-lymphocytes

Important Terminology

What occurs prior to stem cell

collection

The routine workup of the donor includes a

history CBC BMP Red blood type and

screening for RBC allo antibodies chest x-ray

and electrocardiogram (EKG)

Infectious disease screening for hepatitis B and

C HIV Human T-cell lymphoma virus syphilis

and other viral diseases such as

cytomegalovirus herpes and West Nile

When and how are stem cells stored

Stored in cellular therapy labs under regulated

and monitored temperatures according to

standards set up by governing agencies- FDA

FACT AABB etc

If the collected stem cells are to be frozen or

stored for a longer period they are

cryopreserved with dimethyl sulphoxide (DMSO)

When are the stem cells given to the

patient

Stem cells are infused after the patient has undergone

the necessary treatment- chemotherapy radiation or

both- conditioning This is to reduce the tumor burden

and suppress the recipientrsquos immune system in order to

allow engraftment of stem cells

Conditioning can be myeloblative non-

myeloablative or reduced intensity-classification

is based on the duration of cytopenia and the

requirement for stem cell support

Bacigalupo et al Defining the intensity of conditioning regimens Working definitions Biol Blood Marrow Transplant 2009 December 15(12) 1628ndash1633l

Historical Overview

The search for stem cells began in the aftermath

of the bombings in Hiroshima and Nagasaki in 1945

Survivors who died later developed myelodysplastic syndromes with recurrent infections bleeding complications and severe anemia

In 1949 Jacobson et al demonstrated that mice given doses of whole body X-irradiation developed the same radiation syndromes

Historical Overview

At a minimal lethal dose the mice died from hematopoietic failure approximately two weeks after radiation exposure

In 1951 Lorenzo et al using inbred strains of mice showed that whole-body-irradiated mice could be rescued from otherwise fatal hematopoietic failure by injection of suspensions of cells from blood-forming organs such as the bone marrow

Historical Overview

In 1956 three laboratories demonstrated that the injected bone marrow cells directly regenerated the blood-forming system

Hence the advent of hematopoietic (progenitor) stem cell (HPC) transplant

Sources of HPC

Collection ndash Peripheral blood by apheresis

ndash Bone marrow

ndash Umbilical cord blood

Donor ndash Primed mobilized with granulocyte colony

stimulating factor (G-CSF) to increase number of HPC in the peripheral blood for about 3-4 days before collection on Day 5

ndash Medications such as mozobil can be given to increase HPCs in the peripheral blood

NIHgov

Hematopoietic and stromal cell differentiation

Important Terminology

Autologous transplant- ldquotransplantrdquo using a personrsquos own

HPCs

Allogeneic transplant-The infusion of HPCs from another

individual into the patient Allogeneic cells may be from

related or unrelated

Syngeneic transplant- The infusion of HPCs from a twin

into the other twin

Xenogeneic transplant- using HPC from a non human

source or from human cells that have had ex vivo contact

with live non human animal cells

Important Terminology

CD 34+ cells- cell surface glycoprotein that functions as

an adhesion molecule Used to identify hematopoietic

stem cells

Engraftment- sign of cell recovery after transplant An

absolute neutrophil count gt500mm3 in 3 consecutive

days or platelet count 20-50K

GVHD-Post transplant complication characterized by skin liver and GI complications Occurs when the donor cells are recognized by the recipient cells as foreign and immunologically rejects the donor cells ndashmediated by T-lymphocytes

Important Terminology

What occurs prior to stem cell

collection

The routine workup of the donor includes a

history CBC BMP Red blood type and

screening for RBC allo antibodies chest x-ray

and electrocardiogram (EKG)

Infectious disease screening for hepatitis B and

C HIV Human T-cell lymphoma virus syphilis

and other viral diseases such as

cytomegalovirus herpes and West Nile

When and how are stem cells stored

Stored in cellular therapy labs under regulated

and monitored temperatures according to

standards set up by governing agencies- FDA

FACT AABB etc

If the collected stem cells are to be frozen or

stored for a longer period they are

cryopreserved with dimethyl sulphoxide (DMSO)

When are the stem cells given to the

patient

Stem cells are infused after the patient has undergone

the necessary treatment- chemotherapy radiation or

both- conditioning This is to reduce the tumor burden

and suppress the recipientrsquos immune system in order to

allow engraftment of stem cells

Conditioning can be myeloblative non-

myeloablative or reduced intensity-classification

is based on the duration of cytopenia and the

requirement for stem cell support

Bacigalupo et al Defining the intensity of conditioning regimens Working definitions Biol Blood Marrow Transplant 2009 December 15(12) 1628ndash1633l

Historical Overview

At a minimal lethal dose the mice died from hematopoietic failure approximately two weeks after radiation exposure

In 1951 Lorenzo et al using inbred strains of mice showed that whole-body-irradiated mice could be rescued from otherwise fatal hematopoietic failure by injection of suspensions of cells from blood-forming organs such as the bone marrow

Historical Overview

In 1956 three laboratories demonstrated that the injected bone marrow cells directly regenerated the blood-forming system

Hence the advent of hematopoietic (progenitor) stem cell (HPC) transplant

Sources of HPC

Collection ndash Peripheral blood by apheresis

ndash Bone marrow

ndash Umbilical cord blood

Donor ndash Primed mobilized with granulocyte colony

stimulating factor (G-CSF) to increase number of HPC in the peripheral blood for about 3-4 days before collection on Day 5

ndash Medications such as mozobil can be given to increase HPCs in the peripheral blood

NIHgov

Hematopoietic and stromal cell differentiation

Important Terminology

Autologous transplant- ldquotransplantrdquo using a personrsquos own

HPCs

Allogeneic transplant-The infusion of HPCs from another

individual into the patient Allogeneic cells may be from

related or unrelated

Syngeneic transplant- The infusion of HPCs from a twin

into the other twin

Xenogeneic transplant- using HPC from a non human

source or from human cells that have had ex vivo contact

with live non human animal cells

Important Terminology

CD 34+ cells- cell surface glycoprotein that functions as

an adhesion molecule Used to identify hematopoietic

stem cells

Engraftment- sign of cell recovery after transplant An

absolute neutrophil count gt500mm3 in 3 consecutive

days or platelet count 20-50K

GVHD-Post transplant complication characterized by skin liver and GI complications Occurs when the donor cells are recognized by the recipient cells as foreign and immunologically rejects the donor cells ndashmediated by T-lymphocytes

Important Terminology

What occurs prior to stem cell

collection

The routine workup of the donor includes a

history CBC BMP Red blood type and

screening for RBC allo antibodies chest x-ray

and electrocardiogram (EKG)

Infectious disease screening for hepatitis B and

C HIV Human T-cell lymphoma virus syphilis

and other viral diseases such as

cytomegalovirus herpes and West Nile

When and how are stem cells stored

Stored in cellular therapy labs under regulated

and monitored temperatures according to

standards set up by governing agencies- FDA

FACT AABB etc

If the collected stem cells are to be frozen or

stored for a longer period they are

cryopreserved with dimethyl sulphoxide (DMSO)

When are the stem cells given to the

patient

Stem cells are infused after the patient has undergone

the necessary treatment- chemotherapy radiation or

both- conditioning This is to reduce the tumor burden

and suppress the recipientrsquos immune system in order to

allow engraftment of stem cells

Conditioning can be myeloblative non-

myeloablative or reduced intensity-classification

is based on the duration of cytopenia and the

requirement for stem cell support

Bacigalupo et al Defining the intensity of conditioning regimens Working definitions Biol Blood Marrow Transplant 2009 December 15(12) 1628ndash1633l

Historical Overview

In 1956 three laboratories demonstrated that the injected bone marrow cells directly regenerated the blood-forming system

Hence the advent of hematopoietic (progenitor) stem cell (HPC) transplant

Sources of HPC

Collection ndash Peripheral blood by apheresis

ndash Bone marrow

ndash Umbilical cord blood

Donor ndash Primed mobilized with granulocyte colony

stimulating factor (G-CSF) to increase number of HPC in the peripheral blood for about 3-4 days before collection on Day 5

ndash Medications such as mozobil can be given to increase HPCs in the peripheral blood

NIHgov

Hematopoietic and stromal cell differentiation

Important Terminology

Autologous transplant- ldquotransplantrdquo using a personrsquos own

HPCs

Allogeneic transplant-The infusion of HPCs from another

individual into the patient Allogeneic cells may be from

related or unrelated

Syngeneic transplant- The infusion of HPCs from a twin

into the other twin

Xenogeneic transplant- using HPC from a non human

source or from human cells that have had ex vivo contact

with live non human animal cells

Important Terminology

CD 34+ cells- cell surface glycoprotein that functions as

an adhesion molecule Used to identify hematopoietic

stem cells

Engraftment- sign of cell recovery after transplant An

absolute neutrophil count gt500mm3 in 3 consecutive

days or platelet count 20-50K

GVHD-Post transplant complication characterized by skin liver and GI complications Occurs when the donor cells are recognized by the recipient cells as foreign and immunologically rejects the donor cells ndashmediated by T-lymphocytes

Important Terminology

What occurs prior to stem cell

collection

The routine workup of the donor includes a

history CBC BMP Red blood type and

screening for RBC allo antibodies chest x-ray

and electrocardiogram (EKG)

Infectious disease screening for hepatitis B and

C HIV Human T-cell lymphoma virus syphilis

and other viral diseases such as

cytomegalovirus herpes and West Nile

When and how are stem cells stored

Stored in cellular therapy labs under regulated

and monitored temperatures according to

standards set up by governing agencies- FDA

FACT AABB etc

If the collected stem cells are to be frozen or

stored for a longer period they are

cryopreserved with dimethyl sulphoxide (DMSO)

When are the stem cells given to the

patient

Stem cells are infused after the patient has undergone

the necessary treatment- chemotherapy radiation or

both- conditioning This is to reduce the tumor burden

and suppress the recipientrsquos immune system in order to

allow engraftment of stem cells

Conditioning can be myeloblative non-

myeloablative or reduced intensity-classification

is based on the duration of cytopenia and the

requirement for stem cell support

Bacigalupo et al Defining the intensity of conditioning regimens Working definitions Biol Blood Marrow Transplant 2009 December 15(12) 1628ndash1633l

Sources of HPC

Collection ndash Peripheral blood by apheresis

ndash Bone marrow

ndash Umbilical cord blood

Donor ndash Primed mobilized with granulocyte colony

stimulating factor (G-CSF) to increase number of HPC in the peripheral blood for about 3-4 days before collection on Day 5

ndash Medications such as mozobil can be given to increase HPCs in the peripheral blood

NIHgov

Hematopoietic and stromal cell differentiation

Important Terminology

Autologous transplant- ldquotransplantrdquo using a personrsquos own

HPCs

Allogeneic transplant-The infusion of HPCs from another

individual into the patient Allogeneic cells may be from

related or unrelated

Syngeneic transplant- The infusion of HPCs from a twin

into the other twin

Xenogeneic transplant- using HPC from a non human

source or from human cells that have had ex vivo contact

with live non human animal cells

Important Terminology

CD 34+ cells- cell surface glycoprotein that functions as

an adhesion molecule Used to identify hematopoietic

stem cells

Engraftment- sign of cell recovery after transplant An

absolute neutrophil count gt500mm3 in 3 consecutive

days or platelet count 20-50K

GVHD-Post transplant complication characterized by skin liver and GI complications Occurs when the donor cells are recognized by the recipient cells as foreign and immunologically rejects the donor cells ndashmediated by T-lymphocytes

Important Terminology

What occurs prior to stem cell

collection

The routine workup of the donor includes a

history CBC BMP Red blood type and

screening for RBC allo antibodies chest x-ray

and electrocardiogram (EKG)

Infectious disease screening for hepatitis B and

C HIV Human T-cell lymphoma virus syphilis

and other viral diseases such as

cytomegalovirus herpes and West Nile

When and how are stem cells stored

Stored in cellular therapy labs under regulated

and monitored temperatures according to

standards set up by governing agencies- FDA

FACT AABB etc

If the collected stem cells are to be frozen or

stored for a longer period they are

cryopreserved with dimethyl sulphoxide (DMSO)

When are the stem cells given to the

patient

Stem cells are infused after the patient has undergone

the necessary treatment- chemotherapy radiation or

both- conditioning This is to reduce the tumor burden

and suppress the recipientrsquos immune system in order to

allow engraftment of stem cells

Conditioning can be myeloblative non-

myeloablative or reduced intensity-classification

is based on the duration of cytopenia and the

requirement for stem cell support

Bacigalupo et al Defining the intensity of conditioning regimens Working definitions Biol Blood Marrow Transplant 2009 December 15(12) 1628ndash1633l

NIHgov

Hematopoietic and stromal cell differentiation

Important Terminology

Autologous transplant- ldquotransplantrdquo using a personrsquos own

HPCs

Allogeneic transplant-The infusion of HPCs from another

individual into the patient Allogeneic cells may be from

related or unrelated

Syngeneic transplant- The infusion of HPCs from a twin

into the other twin

Xenogeneic transplant- using HPC from a non human

source or from human cells that have had ex vivo contact

with live non human animal cells

Important Terminology

CD 34+ cells- cell surface glycoprotein that functions as

an adhesion molecule Used to identify hematopoietic

stem cells

Engraftment- sign of cell recovery after transplant An

absolute neutrophil count gt500mm3 in 3 consecutive

days or platelet count 20-50K

GVHD-Post transplant complication characterized by skin liver and GI complications Occurs when the donor cells are recognized by the recipient cells as foreign and immunologically rejects the donor cells ndashmediated by T-lymphocytes

Important Terminology

What occurs prior to stem cell

collection

The routine workup of the donor includes a

history CBC BMP Red blood type and

screening for RBC allo antibodies chest x-ray

and electrocardiogram (EKG)

Infectious disease screening for hepatitis B and

C HIV Human T-cell lymphoma virus syphilis

and other viral diseases such as

cytomegalovirus herpes and West Nile

When and how are stem cells stored

Stored in cellular therapy labs under regulated

and monitored temperatures according to

standards set up by governing agencies- FDA

FACT AABB etc

If the collected stem cells are to be frozen or

stored for a longer period they are

cryopreserved with dimethyl sulphoxide (DMSO)

When are the stem cells given to the

patient

Stem cells are infused after the patient has undergone

the necessary treatment- chemotherapy radiation or

both- conditioning This is to reduce the tumor burden

and suppress the recipientrsquos immune system in order to

allow engraftment of stem cells

Conditioning can be myeloblative non-

myeloablative or reduced intensity-classification

is based on the duration of cytopenia and the

requirement for stem cell support

Bacigalupo et al Defining the intensity of conditioning regimens Working definitions Biol Blood Marrow Transplant 2009 December 15(12) 1628ndash1633l

Important Terminology

Autologous transplant- ldquotransplantrdquo using a personrsquos own

HPCs

Allogeneic transplant-The infusion of HPCs from another

individual into the patient Allogeneic cells may be from

related or unrelated

Syngeneic transplant- The infusion of HPCs from a twin

into the other twin

Xenogeneic transplant- using HPC from a non human

source or from human cells that have had ex vivo contact

with live non human animal cells

Important Terminology

CD 34+ cells- cell surface glycoprotein that functions as

an adhesion molecule Used to identify hematopoietic

stem cells

Engraftment- sign of cell recovery after transplant An

absolute neutrophil count gt500mm3 in 3 consecutive

days or platelet count 20-50K

GVHD-Post transplant complication characterized by skin liver and GI complications Occurs when the donor cells are recognized by the recipient cells as foreign and immunologically rejects the donor cells ndashmediated by T-lymphocytes

Important Terminology

What occurs prior to stem cell

collection

The routine workup of the donor includes a

history CBC BMP Red blood type and

screening for RBC allo antibodies chest x-ray

and electrocardiogram (EKG)

Infectious disease screening for hepatitis B and

C HIV Human T-cell lymphoma virus syphilis

and other viral diseases such as

cytomegalovirus herpes and West Nile

When and how are stem cells stored

Stored in cellular therapy labs under regulated

and monitored temperatures according to

standards set up by governing agencies- FDA

FACT AABB etc

If the collected stem cells are to be frozen or

stored for a longer period they are

cryopreserved with dimethyl sulphoxide (DMSO)

When are the stem cells given to the

patient

Stem cells are infused after the patient has undergone

the necessary treatment- chemotherapy radiation or

both- conditioning This is to reduce the tumor burden

and suppress the recipientrsquos immune system in order to

allow engraftment of stem cells

Conditioning can be myeloblative non-

myeloablative or reduced intensity-classification

is based on the duration of cytopenia and the

requirement for stem cell support

Bacigalupo et al Defining the intensity of conditioning regimens Working definitions Biol Blood Marrow Transplant 2009 December 15(12) 1628ndash1633l

Important Terminology

CD 34+ cells- cell surface glycoprotein that functions as

an adhesion molecule Used to identify hematopoietic

stem cells

Engraftment- sign of cell recovery after transplant An

absolute neutrophil count gt500mm3 in 3 consecutive

days or platelet count 20-50K

GVHD-Post transplant complication characterized by skin liver and GI complications Occurs when the donor cells are recognized by the recipient cells as foreign and immunologically rejects the donor cells ndashmediated by T-lymphocytes

Important Terminology

What occurs prior to stem cell

collection

The routine workup of the donor includes a

history CBC BMP Red blood type and

screening for RBC allo antibodies chest x-ray

and electrocardiogram (EKG)

Infectious disease screening for hepatitis B and

C HIV Human T-cell lymphoma virus syphilis

and other viral diseases such as

cytomegalovirus herpes and West Nile

When and how are stem cells stored

Stored in cellular therapy labs under regulated

and monitored temperatures according to

standards set up by governing agencies- FDA

FACT AABB etc

If the collected stem cells are to be frozen or

stored for a longer period they are

cryopreserved with dimethyl sulphoxide (DMSO)

When are the stem cells given to the

patient

Stem cells are infused after the patient has undergone

the necessary treatment- chemotherapy radiation or

both- conditioning This is to reduce the tumor burden

and suppress the recipientrsquos immune system in order to

allow engraftment of stem cells

Conditioning can be myeloblative non-

myeloablative or reduced intensity-classification

is based on the duration of cytopenia and the

requirement for stem cell support

Bacigalupo et al Defining the intensity of conditioning regimens Working definitions Biol Blood Marrow Transplant 2009 December 15(12) 1628ndash1633l

GVHD-Post transplant complication characterized by skin liver and GI complications Occurs when the donor cells are recognized by the recipient cells as foreign and immunologically rejects the donor cells ndashmediated by T-lymphocytes

Important Terminology

What occurs prior to stem cell

collection

The routine workup of the donor includes a

history CBC BMP Red blood type and

screening for RBC allo antibodies chest x-ray

and electrocardiogram (EKG)

Infectious disease screening for hepatitis B and

C HIV Human T-cell lymphoma virus syphilis

and other viral diseases such as

cytomegalovirus herpes and West Nile

When and how are stem cells stored

Stored in cellular therapy labs under regulated

and monitored temperatures according to

standards set up by governing agencies- FDA

FACT AABB etc

If the collected stem cells are to be frozen or

stored for a longer period they are

cryopreserved with dimethyl sulphoxide (DMSO)

When are the stem cells given to the

patient

Stem cells are infused after the patient has undergone

the necessary treatment- chemotherapy radiation or

both- conditioning This is to reduce the tumor burden

and suppress the recipientrsquos immune system in order to

allow engraftment of stem cells

Conditioning can be myeloblative non-

myeloablative or reduced intensity-classification

is based on the duration of cytopenia and the

requirement for stem cell support

Bacigalupo et al Defining the intensity of conditioning regimens Working definitions Biol Blood Marrow Transplant 2009 December 15(12) 1628ndash1633l

What occurs prior to stem cell

collection

The routine workup of the donor includes a

history CBC BMP Red blood type and

screening for RBC allo antibodies chest x-ray

and electrocardiogram (EKG)

Infectious disease screening for hepatitis B and

C HIV Human T-cell lymphoma virus syphilis

and other viral diseases such as

cytomegalovirus herpes and West Nile

When and how are stem cells stored

Stored in cellular therapy labs under regulated

and monitored temperatures according to

standards set up by governing agencies- FDA

FACT AABB etc

If the collected stem cells are to be frozen or

stored for a longer period they are

cryopreserved with dimethyl sulphoxide (DMSO)

When are the stem cells given to the

patient

Stem cells are infused after the patient has undergone

the necessary treatment- chemotherapy radiation or

both- conditioning This is to reduce the tumor burden

and suppress the recipientrsquos immune system in order to

allow engraftment of stem cells

Conditioning can be myeloblative non-

myeloablative or reduced intensity-classification

is based on the duration of cytopenia and the

requirement for stem cell support

Bacigalupo et al Defining the intensity of conditioning regimens Working definitions Biol Blood Marrow Transplant 2009 December 15(12) 1628ndash1633l

When and how are stem cells stored

Stored in cellular therapy labs under regulated

and monitored temperatures according to

standards set up by governing agencies- FDA

FACT AABB etc

If the collected stem cells are to be frozen or

stored for a longer period they are

cryopreserved with dimethyl sulphoxide (DMSO)

When are the stem cells given to the

patient

Stem cells are infused after the patient has undergone

the necessary treatment- chemotherapy radiation or

both- conditioning This is to reduce the tumor burden

and suppress the recipientrsquos immune system in order to

allow engraftment of stem cells

Conditioning can be myeloblative non-

myeloablative or reduced intensity-classification

is based on the duration of cytopenia and the

requirement for stem cell support

Bacigalupo et al Defining the intensity of conditioning regimens Working definitions Biol Blood Marrow Transplant 2009 December 15(12) 1628ndash1633l

When are the stem cells given to the

patient

Stem cells are infused after the patient has undergone

the necessary treatment- chemotherapy radiation or

both- conditioning This is to reduce the tumor burden

and suppress the recipientrsquos immune system in order to

allow engraftment of stem cells

Conditioning can be myeloblative non-

myeloablative or reduced intensity-classification

is based on the duration of cytopenia and the

requirement for stem cell support

Bacigalupo et al Defining the intensity of conditioning regimens Working definitions Biol Blood Marrow Transplant 2009 December 15(12) 1628ndash1633l

How are stem cells infused

Stem cells are infused similar to a regular blood transfusion

They may be infused on the same day of collection or stored in a cellular therapy lab and infused later

Current Uses and Outcomes of Hematopoietic

Stem Cell Transplantation

2011

Summary Slides

Worldwide

SUM-WW11_1ppt

Center for International Blood and Marrow

Transplant Research-CIBMTR

Collaborates with the global scientific community to advance hematopoietic cell transplantation and cellular therapy research worldwide

Has a combined research program with the National Marrow Donor Program (NMDP) and the Medical College of Wisconsin US

Location of Centers participating

in the CIBMTR

2011

SUM-WW11_2ppt

Indications for Hematopoietic Stem Cell

Transplants in the United States 2009

SUM-WW11_8ppt

Num

ber

of

Tra

nspla

nts

0

500

1000

1500

2000

2500

3000

3500

4000

4500

5000

5500

Multiple

Myeloma

NHL AML HD ALL MDSMPD Aplastic

Anemia

CML Other

Leuk

Non-

Malig

Disease

Other

Cancer

Allogeneic (Total N=7012)

Autologous (Total N=9778)

Congenital immune deficiencies Severe combined immunodeficiency disease

Wiskott-Aldrich syndromes

Marrow failure syndromes

Severe aplastic anemia

Fanconirsquos anemia

Diamond- Blackfan anemia

( Congenital hypoplastic anemia)

Inborn disorders of metabolism Mucopolysaccharidoses eg Hunter or Hurler syndrome

Leukodystrophies eg adrenoleukodystrophy

Glycoprotein disorders eg fucosidosis mannosidosis

Lysosomal disorders eg Gaucher Pompe Neimann-Pick disease

Osteropetrosis

Osteogenesis imperfecta

Hemoglobinopathies Thalasemia

Sickle cell disease

Other Indications for HPCs Stem Cell Transplant

Solid tumors Breast cancer

Ovarian cancer

Renal cell carcinoma

Testicular cancer

Childhood solid tumors Wilms tumor

Neuroblastoma

AABB Technical

Manual 17th Edition

Tra

nspla

nts

SUM11_41ppt

Transplant Activity in the US

1980-2010

0

2000

4000

6000

8000

10000

12000

14000

80 81 82 83 84 85 86 87 88 89 90 91 92 93 94 95 96 97 98 99 00 01 02 0304 05 06 07 08 09 10

Autologous

Related Donor

Unrelated Donor

SUM-WW11_4ppt

Autologous Stem Cell Sources

by Recipient Age

2000-2009

Age 20 yrs Age gt20 yrs

Tra

nspla

nts

0

20

40

60

80

100

2000-2004 2005-2009 2000-2004 2005-2009

Bone Marrow (BM)Peripheral Blood (PB)BM + PB

SUM-WW11_3ppt

Allogeneic Stem Cell Sources

by Recipient Age

2000-2009

Age 20 yrs Age gt20 yrs

Tra

nspla

nts

0

20

40

60

80

100

2000-2004 2005-2009 2000-2004 2005-2009

Bone Marrow (BM)

Peripheral Blood (PB)

Cord Blood (CB)

One-year survival after myeloablative conditioning for acute leukemias

in any remission phase

CML or MDS age lt50 years by year of transplant and graft source

1988-2009

One-Y

ear

Surv

ival

SUM11_40ppt

0

20

40

60

80

100

1988-

90

91 92 93 94 95 96 97 98 99 00 01 02 03 04 05 06 07 08 09

HLA-matched sibling

Unrelated Donor

SUM-WW11_14ppt

100-day Mortality after

Autologous Transplants

2008-2009

Mort

ality

0

10

20

30

40

50

Acute Leukemia Non-Hodgkin

Lymphoma

Hodgkin

Disease

Multiple

Myeloma

Early Disease

IntermediateAdvance DiseaseSensitive

ResistantUnknown

Other

0

20

40

60

80

100

AML ALL CML MDSMPS Aplastic

Anemia

Immune

Deficiency

SUM-WW11_16ppt

100-day Mortality after

Unrelated Donor Transplants

2008-2009

Early Disease

Intermediate Disease

Advanced Disease

Chronic Phase

Accelerated Phase

Blast Phase

Other

Mort

ality

Causes of Death

after Transplants

performed in

2008-2009

Autologous

Infection (8)

Other (16)

Organ Failure (2)

New Malignancy (1)

Primary Disease (73)

Unrelated Donor

Infection (16)

Other (29) Organ

Failure (6)

Primary Disease (33)

New Malignancy (1)

GVHD (15)

SUM-WW11_17ppt

HLA-identical Sibling

Infection (12)

Other (21)

Primary Disease (47)

GVHD (14)

Organ Failure (4)

New Malignancy (1)

In summary

After more than 50 years of research and

clinical use hematopoietic stem cells

have become the best-studied stem cells

with several indications

There is still active research ongoing to

expand its usehellip

1 Cellular Therapy AABB 16th Edition

2 AABB Techical Manual 17th edition

3 WWWNIHGOV

4 WWWCIBMTRORG

5 Jacobsen LO Marks EK Gaston EO Zirkle RE Effect of spleen protection on

mortality following X-irradiation J Lab Clin Med Vol 34 19491538ndash1543

6 Lorenz E Uphoff ED Reid TR Shelton E Modification of acute irradiation injury in

mice and guinea pigs by bone marrow injection Radiology Vol 58 1951863ndash877

7 Ford CE Hamerton JL Barnes DWH Loutit JF Cytological identification of

radiation-chimaeras Nature Vol 177 1956452ndash454

8 Nowell PC Cole LJ Habermyer JG Roan PL Growth and continued function of

rat marrow cells in x-irradiated mice Cancer Res Vol 16 1956258ndash261

9 Weissman IL Translating stem and progenitor cell biology to the clinic barriers

and opportunities Science Vol 287 20001442ndash1446

10Manz MG Akashi K Weissman IL Biology of Hematopoietic Stem and Progenitor

Cells In Appelbaum FR ed Thomas Hematopoietic Cell Transplantation Third

ed Malden MA Blackwell Publishing 2004

11Bacigalupo et al Defining the intensity of conditioning regimens Working

definitions Biol Blood Marrow Transplant 2009 December 15(12) 1628ndash1633l

REFERENCES

Current Uses and Outcomes of Hematopoietic

Stem Cell Transplantation

2011

Summary Slides

Worldwide

SUM-WW11_1ppt

Center for International Blood and Marrow

Transplant Research-CIBMTR

Collaborates with the global scientific community to advance hematopoietic cell transplantation and cellular therapy research worldwide

Has a combined research program with the National Marrow Donor Program (NMDP) and the Medical College of Wisconsin US

Location of Centers participating

in the CIBMTR

2011

SUM-WW11_2ppt

Indications for Hematopoietic Stem Cell

Transplants in the United States 2009

SUM-WW11_8ppt

Num

ber

of

Tra

nspla

nts

0

500

1000

1500

2000

2500

3000

3500

4000

4500

5000

5500

Multiple

Myeloma

NHL AML HD ALL MDSMPD Aplastic

Anemia

CML Other

Leuk

Non-

Malig

Disease

Other

Cancer

Allogeneic (Total N=7012)

Autologous (Total N=9778)

Congenital immune deficiencies Severe combined immunodeficiency disease

Wiskott-Aldrich syndromes

Marrow failure syndromes

Severe aplastic anemia

Fanconirsquos anemia

Diamond- Blackfan anemia

( Congenital hypoplastic anemia)

Inborn disorders of metabolism Mucopolysaccharidoses eg Hunter or Hurler syndrome

Leukodystrophies eg adrenoleukodystrophy

Glycoprotein disorders eg fucosidosis mannosidosis

Lysosomal disorders eg Gaucher Pompe Neimann-Pick disease

Osteropetrosis

Osteogenesis imperfecta

Hemoglobinopathies Thalasemia

Sickle cell disease

Other Indications for HPCs Stem Cell Transplant

Solid tumors Breast cancer

Ovarian cancer

Renal cell carcinoma

Testicular cancer

Childhood solid tumors Wilms tumor

Neuroblastoma

AABB Technical

Manual 17th Edition

Tra

nspla

nts

SUM11_41ppt

Transplant Activity in the US

1980-2010

0

2000

4000

6000

8000

10000

12000

14000

80 81 82 83 84 85 86 87 88 89 90 91 92 93 94 95 96 97 98 99 00 01 02 0304 05 06 07 08 09 10

Autologous

Related Donor

Unrelated Donor

SUM-WW11_4ppt

Autologous Stem Cell Sources

by Recipient Age

2000-2009

Age 20 yrs Age gt20 yrs

Tra

nspla

nts

0

20

40

60

80

100

2000-2004 2005-2009 2000-2004 2005-2009

Bone Marrow (BM)Peripheral Blood (PB)BM + PB

SUM-WW11_3ppt

Allogeneic Stem Cell Sources

by Recipient Age

2000-2009

Age 20 yrs Age gt20 yrs

Tra

nspla

nts

0

20

40

60

80

100

2000-2004 2005-2009 2000-2004 2005-2009

Bone Marrow (BM)

Peripheral Blood (PB)

Cord Blood (CB)

One-year survival after myeloablative conditioning for acute leukemias

in any remission phase

CML or MDS age lt50 years by year of transplant and graft source

1988-2009

One-Y

ear

Surv

ival

SUM11_40ppt

0

20

40

60

80

100

1988-

90

91 92 93 94 95 96 97 98 99 00 01 02 03 04 05 06 07 08 09

HLA-matched sibling

Unrelated Donor

SUM-WW11_14ppt

100-day Mortality after

Autologous Transplants

2008-2009

Mort

ality

0

10

20

30

40

50

Acute Leukemia Non-Hodgkin

Lymphoma

Hodgkin

Disease

Multiple

Myeloma

Early Disease

IntermediateAdvance DiseaseSensitive

ResistantUnknown

Other

0

20

40

60

80

100

AML ALL CML MDSMPS Aplastic

Anemia

Immune

Deficiency

SUM-WW11_16ppt

100-day Mortality after

Unrelated Donor Transplants

2008-2009

Early Disease

Intermediate Disease

Advanced Disease

Chronic Phase

Accelerated Phase

Blast Phase

Other

Mort

ality

Causes of Death

after Transplants

performed in

2008-2009

Autologous

Infection (8)

Other (16)

Organ Failure (2)

New Malignancy (1)

Primary Disease (73)

Unrelated Donor

Infection (16)

Other (29) Organ

Failure (6)

Primary Disease (33)

New Malignancy (1)

GVHD (15)

SUM-WW11_17ppt

HLA-identical Sibling

Infection (12)

Other (21)

Primary Disease (47)

GVHD (14)

Organ Failure (4)

New Malignancy (1)

In summary

After more than 50 years of research and

clinical use hematopoietic stem cells

have become the best-studied stem cells

with several indications

There is still active research ongoing to

expand its usehellip

1 Cellular Therapy AABB 16th Edition

2 AABB Techical Manual 17th edition

3 WWWNIHGOV

4 WWWCIBMTRORG

5 Jacobsen LO Marks EK Gaston EO Zirkle RE Effect of spleen protection on

mortality following X-irradiation J Lab Clin Med Vol 34 19491538ndash1543

6 Lorenz E Uphoff ED Reid TR Shelton E Modification of acute irradiation injury in

mice and guinea pigs by bone marrow injection Radiology Vol 58 1951863ndash877

7 Ford CE Hamerton JL Barnes DWH Loutit JF Cytological identification of

radiation-chimaeras Nature Vol 177 1956452ndash454

8 Nowell PC Cole LJ Habermyer JG Roan PL Growth and continued function of

rat marrow cells in x-irradiated mice Cancer Res Vol 16 1956258ndash261

9 Weissman IL Translating stem and progenitor cell biology to the clinic barriers

and opportunities Science Vol 287 20001442ndash1446

10Manz MG Akashi K Weissman IL Biology of Hematopoietic Stem and Progenitor

Cells In Appelbaum FR ed Thomas Hematopoietic Cell Transplantation Third

ed Malden MA Blackwell Publishing 2004

11Bacigalupo et al Defining the intensity of conditioning regimens Working

definitions Biol Blood Marrow Transplant 2009 December 15(12) 1628ndash1633l

REFERENCES

Center for International Blood and Marrow

Transplant Research-CIBMTR

Collaborates with the global scientific community to advance hematopoietic cell transplantation and cellular therapy research worldwide

Has a combined research program with the National Marrow Donor Program (NMDP) and the Medical College of Wisconsin US

Location of Centers participating

in the CIBMTR

2011

SUM-WW11_2ppt

Indications for Hematopoietic Stem Cell

Transplants in the United States 2009

SUM-WW11_8ppt

Num

ber

of

Tra

nspla

nts

0

500

1000

1500

2000

2500

3000

3500

4000

4500

5000

5500

Multiple

Myeloma

NHL AML HD ALL MDSMPD Aplastic

Anemia

CML Other

Leuk

Non-

Malig

Disease

Other

Cancer

Allogeneic (Total N=7012)

Autologous (Total N=9778)

Congenital immune deficiencies Severe combined immunodeficiency disease

Wiskott-Aldrich syndromes

Marrow failure syndromes

Severe aplastic anemia

Fanconirsquos anemia

Diamond- Blackfan anemia

( Congenital hypoplastic anemia)

Inborn disorders of metabolism Mucopolysaccharidoses eg Hunter or Hurler syndrome

Leukodystrophies eg adrenoleukodystrophy

Glycoprotein disorders eg fucosidosis mannosidosis

Lysosomal disorders eg Gaucher Pompe Neimann-Pick disease

Osteropetrosis

Osteogenesis imperfecta

Hemoglobinopathies Thalasemia

Sickle cell disease

Other Indications for HPCs Stem Cell Transplant

Solid tumors Breast cancer

Ovarian cancer

Renal cell carcinoma

Testicular cancer

Childhood solid tumors Wilms tumor

Neuroblastoma

AABB Technical

Manual 17th Edition

Tra

nspla

nts

SUM11_41ppt

Transplant Activity in the US

1980-2010

0

2000

4000

6000

8000

10000

12000

14000

80 81 82 83 84 85 86 87 88 89 90 91 92 93 94 95 96 97 98 99 00 01 02 0304 05 06 07 08 09 10

Autologous

Related Donor

Unrelated Donor

SUM-WW11_4ppt

Autologous Stem Cell Sources

by Recipient Age

2000-2009

Age 20 yrs Age gt20 yrs

Tra

nspla

nts

0

20

40

60

80

100

2000-2004 2005-2009 2000-2004 2005-2009

Bone Marrow (BM)Peripheral Blood (PB)BM + PB

SUM-WW11_3ppt

Allogeneic Stem Cell Sources

by Recipient Age

2000-2009

Age 20 yrs Age gt20 yrs

Tra

nspla

nts

0

20

40

60

80

100

2000-2004 2005-2009 2000-2004 2005-2009

Bone Marrow (BM)

Peripheral Blood (PB)

Cord Blood (CB)

One-year survival after myeloablative conditioning for acute leukemias

in any remission phase

CML or MDS age lt50 years by year of transplant and graft source

1988-2009

One-Y

ear

Surv

ival

SUM11_40ppt

0

20

40

60

80

100

1988-

90

91 92 93 94 95 96 97 98 99 00 01 02 03 04 05 06 07 08 09

HLA-matched sibling

Unrelated Donor

SUM-WW11_14ppt

100-day Mortality after

Autologous Transplants

2008-2009

Mort

ality

0

10

20

30

40

50

Acute Leukemia Non-Hodgkin

Lymphoma

Hodgkin

Disease

Multiple

Myeloma

Early Disease

IntermediateAdvance DiseaseSensitive

ResistantUnknown

Other

0

20

40

60

80

100

AML ALL CML MDSMPS Aplastic

Anemia

Immune

Deficiency

SUM-WW11_16ppt

100-day Mortality after

Unrelated Donor Transplants

2008-2009

Early Disease

Intermediate Disease

Advanced Disease

Chronic Phase

Accelerated Phase

Blast Phase

Other

Mort

ality

Causes of Death

after Transplants

performed in

2008-2009

Autologous

Infection (8)

Other (16)

Organ Failure (2)

New Malignancy (1)

Primary Disease (73)

Unrelated Donor

Infection (16)

Other (29) Organ

Failure (6)

Primary Disease (33)

New Malignancy (1)

GVHD (15)

SUM-WW11_17ppt

HLA-identical Sibling

Infection (12)

Other (21)

Primary Disease (47)

GVHD (14)

Organ Failure (4)

New Malignancy (1)

In summary

After more than 50 years of research and

clinical use hematopoietic stem cells

have become the best-studied stem cells

with several indications

There is still active research ongoing to

expand its usehellip

1 Cellular Therapy AABB 16th Edition

2 AABB Techical Manual 17th edition

3 WWWNIHGOV

4 WWWCIBMTRORG

5 Jacobsen LO Marks EK Gaston EO Zirkle RE Effect of spleen protection on

mortality following X-irradiation J Lab Clin Med Vol 34 19491538ndash1543

6 Lorenz E Uphoff ED Reid TR Shelton E Modification of acute irradiation injury in

mice and guinea pigs by bone marrow injection Radiology Vol 58 1951863ndash877

7 Ford CE Hamerton JL Barnes DWH Loutit JF Cytological identification of

radiation-chimaeras Nature Vol 177 1956452ndash454

8 Nowell PC Cole LJ Habermyer JG Roan PL Growth and continued function of

rat marrow cells in x-irradiated mice Cancer Res Vol 16 1956258ndash261

9 Weissman IL Translating stem and progenitor cell biology to the clinic barriers

and opportunities Science Vol 287 20001442ndash1446

10Manz MG Akashi K Weissman IL Biology of Hematopoietic Stem and Progenitor

Cells In Appelbaum FR ed Thomas Hematopoietic Cell Transplantation Third

ed Malden MA Blackwell Publishing 2004

11Bacigalupo et al Defining the intensity of conditioning regimens Working

definitions Biol Blood Marrow Transplant 2009 December 15(12) 1628ndash1633l

REFERENCES

Location of Centers participating

in the CIBMTR

2011

SUM-WW11_2ppt

Indications for Hematopoietic Stem Cell

Transplants in the United States 2009

SUM-WW11_8ppt

Num

ber

of

Tra

nspla

nts

0

500

1000

1500

2000

2500

3000

3500

4000

4500

5000

5500

Multiple

Myeloma

NHL AML HD ALL MDSMPD Aplastic

Anemia

CML Other

Leuk

Non-

Malig

Disease

Other

Cancer

Allogeneic (Total N=7012)

Autologous (Total N=9778)

Congenital immune deficiencies Severe combined immunodeficiency disease

Wiskott-Aldrich syndromes

Marrow failure syndromes

Severe aplastic anemia

Fanconirsquos anemia

Diamond- Blackfan anemia

( Congenital hypoplastic anemia)

Inborn disorders of metabolism Mucopolysaccharidoses eg Hunter or Hurler syndrome

Leukodystrophies eg adrenoleukodystrophy

Glycoprotein disorders eg fucosidosis mannosidosis

Lysosomal disorders eg Gaucher Pompe Neimann-Pick disease

Osteropetrosis

Osteogenesis imperfecta

Hemoglobinopathies Thalasemia

Sickle cell disease

Other Indications for HPCs Stem Cell Transplant

Solid tumors Breast cancer

Ovarian cancer

Renal cell carcinoma

Testicular cancer

Childhood solid tumors Wilms tumor

Neuroblastoma

AABB Technical

Manual 17th Edition

Tra

nspla

nts

SUM11_41ppt

Transplant Activity in the US

1980-2010

0

2000

4000

6000

8000

10000

12000

14000

80 81 82 83 84 85 86 87 88 89 90 91 92 93 94 95 96 97 98 99 00 01 02 0304 05 06 07 08 09 10

Autologous

Related Donor

Unrelated Donor

SUM-WW11_4ppt

Autologous Stem Cell Sources

by Recipient Age

2000-2009

Age 20 yrs Age gt20 yrs

Tra

nspla

nts

0

20

40

60

80

100

2000-2004 2005-2009 2000-2004 2005-2009

Bone Marrow (BM)Peripheral Blood (PB)BM + PB

SUM-WW11_3ppt

Allogeneic Stem Cell Sources

by Recipient Age

2000-2009

Age 20 yrs Age gt20 yrs

Tra

nspla

nts

0

20

40

60

80

100

2000-2004 2005-2009 2000-2004 2005-2009

Bone Marrow (BM)

Peripheral Blood (PB)

Cord Blood (CB)

One-year survival after myeloablative conditioning for acute leukemias

in any remission phase

CML or MDS age lt50 years by year of transplant and graft source

1988-2009

One-Y

ear

Surv

ival

SUM11_40ppt

0

20

40

60

80

100

1988-

90

91 92 93 94 95 96 97 98 99 00 01 02 03 04 05 06 07 08 09

HLA-matched sibling

Unrelated Donor

SUM-WW11_14ppt

100-day Mortality after

Autologous Transplants

2008-2009

Mort

ality

0

10

20

30

40

50

Acute Leukemia Non-Hodgkin

Lymphoma

Hodgkin

Disease

Multiple

Myeloma

Early Disease

IntermediateAdvance DiseaseSensitive

ResistantUnknown

Other

0

20

40

60

80

100

AML ALL CML MDSMPS Aplastic

Anemia

Immune

Deficiency

SUM-WW11_16ppt

100-day Mortality after

Unrelated Donor Transplants

2008-2009

Early Disease

Intermediate Disease

Advanced Disease

Chronic Phase

Accelerated Phase

Blast Phase

Other

Mort

ality

Causes of Death

after Transplants

performed in

2008-2009

Autologous

Infection (8)

Other (16)

Organ Failure (2)

New Malignancy (1)

Primary Disease (73)

Unrelated Donor

Infection (16)

Other (29) Organ

Failure (6)

Primary Disease (33)

New Malignancy (1)

GVHD (15)

SUM-WW11_17ppt

HLA-identical Sibling

Infection (12)

Other (21)

Primary Disease (47)

GVHD (14)

Organ Failure (4)

New Malignancy (1)

In summary

After more than 50 years of research and

clinical use hematopoietic stem cells

have become the best-studied stem cells

with several indications

There is still active research ongoing to

expand its usehellip

1 Cellular Therapy AABB 16th Edition

2 AABB Techical Manual 17th edition

3 WWWNIHGOV

4 WWWCIBMTRORG

5 Jacobsen LO Marks EK Gaston EO Zirkle RE Effect of spleen protection on

mortality following X-irradiation J Lab Clin Med Vol 34 19491538ndash1543

6 Lorenz E Uphoff ED Reid TR Shelton E Modification of acute irradiation injury in

mice and guinea pigs by bone marrow injection Radiology Vol 58 1951863ndash877

7 Ford CE Hamerton JL Barnes DWH Loutit JF Cytological identification of

radiation-chimaeras Nature Vol 177 1956452ndash454

8 Nowell PC Cole LJ Habermyer JG Roan PL Growth and continued function of

rat marrow cells in x-irradiated mice Cancer Res Vol 16 1956258ndash261

9 Weissman IL Translating stem and progenitor cell biology to the clinic barriers

and opportunities Science Vol 287 20001442ndash1446

10Manz MG Akashi K Weissman IL Biology of Hematopoietic Stem and Progenitor

Cells In Appelbaum FR ed Thomas Hematopoietic Cell Transplantation Third

ed Malden MA Blackwell Publishing 2004

11Bacigalupo et al Defining the intensity of conditioning regimens Working

definitions Biol Blood Marrow Transplant 2009 December 15(12) 1628ndash1633l

REFERENCES

Indications for Hematopoietic Stem Cell

Transplants in the United States 2009

SUM-WW11_8ppt

Num

ber

of

Tra

nspla

nts

0

500

1000

1500

2000

2500

3000

3500

4000

4500

5000

5500

Multiple

Myeloma

NHL AML HD ALL MDSMPD Aplastic

Anemia

CML Other

Leuk

Non-

Malig

Disease

Other

Cancer

Allogeneic (Total N=7012)

Autologous (Total N=9778)

Congenital immune deficiencies Severe combined immunodeficiency disease

Wiskott-Aldrich syndromes

Marrow failure syndromes

Severe aplastic anemia

Fanconirsquos anemia

Diamond- Blackfan anemia

( Congenital hypoplastic anemia)

Inborn disorders of metabolism Mucopolysaccharidoses eg Hunter or Hurler syndrome

Leukodystrophies eg adrenoleukodystrophy

Glycoprotein disorders eg fucosidosis mannosidosis

Lysosomal disorders eg Gaucher Pompe Neimann-Pick disease

Osteropetrosis

Osteogenesis imperfecta

Hemoglobinopathies Thalasemia

Sickle cell disease

Other Indications for HPCs Stem Cell Transplant

Solid tumors Breast cancer

Ovarian cancer

Renal cell carcinoma

Testicular cancer

Childhood solid tumors Wilms tumor

Neuroblastoma

AABB Technical

Manual 17th Edition

Tra

nspla

nts

SUM11_41ppt

Transplant Activity in the US

1980-2010

0

2000

4000

6000

8000

10000

12000

14000

80 81 82 83 84 85 86 87 88 89 90 91 92 93 94 95 96 97 98 99 00 01 02 0304 05 06 07 08 09 10

Autologous

Related Donor

Unrelated Donor

SUM-WW11_4ppt

Autologous Stem Cell Sources

by Recipient Age

2000-2009

Age 20 yrs Age gt20 yrs

Tra

nspla

nts

0

20

40

60

80

100

2000-2004 2005-2009 2000-2004 2005-2009

Bone Marrow (BM)Peripheral Blood (PB)BM + PB

SUM-WW11_3ppt

Allogeneic Stem Cell Sources

by Recipient Age

2000-2009

Age 20 yrs Age gt20 yrs

Tra

nspla

nts

0

20

40

60

80

100

2000-2004 2005-2009 2000-2004 2005-2009

Bone Marrow (BM)

Peripheral Blood (PB)

Cord Blood (CB)

One-year survival after myeloablative conditioning for acute leukemias

in any remission phase

CML or MDS age lt50 years by year of transplant and graft source

1988-2009

One-Y

ear

Surv

ival

SUM11_40ppt

0

20

40

60

80

100

1988-

90

91 92 93 94 95 96 97 98 99 00 01 02 03 04 05 06 07 08 09

HLA-matched sibling

Unrelated Donor

SUM-WW11_14ppt

100-day Mortality after

Autologous Transplants

2008-2009

Mort

ality

0

10

20

30

40

50

Acute Leukemia Non-Hodgkin

Lymphoma

Hodgkin

Disease

Multiple

Myeloma

Early Disease

IntermediateAdvance DiseaseSensitive

ResistantUnknown

Other

0

20

40

60

80

100

AML ALL CML MDSMPS Aplastic

Anemia

Immune

Deficiency

SUM-WW11_16ppt

100-day Mortality after

Unrelated Donor Transplants

2008-2009

Early Disease

Intermediate Disease

Advanced Disease

Chronic Phase

Accelerated Phase

Blast Phase

Other

Mort

ality

Causes of Death

after Transplants

performed in

2008-2009

Autologous

Infection (8)

Other (16)

Organ Failure (2)

New Malignancy (1)

Primary Disease (73)

Unrelated Donor

Infection (16)

Other (29) Organ

Failure (6)

Primary Disease (33)

New Malignancy (1)

GVHD (15)

SUM-WW11_17ppt

HLA-identical Sibling

Infection (12)

Other (21)

Primary Disease (47)

GVHD (14)

Organ Failure (4)

New Malignancy (1)

In summary

After more than 50 years of research and

clinical use hematopoietic stem cells

have become the best-studied stem cells

with several indications

There is still active research ongoing to

expand its usehellip

1 Cellular Therapy AABB 16th Edition

2 AABB Techical Manual 17th edition

3 WWWNIHGOV

4 WWWCIBMTRORG

5 Jacobsen LO Marks EK Gaston EO Zirkle RE Effect of spleen protection on

mortality following X-irradiation J Lab Clin Med Vol 34 19491538ndash1543

6 Lorenz E Uphoff ED Reid TR Shelton E Modification of acute irradiation injury in

mice and guinea pigs by bone marrow injection Radiology Vol 58 1951863ndash877

7 Ford CE Hamerton JL Barnes DWH Loutit JF Cytological identification of

radiation-chimaeras Nature Vol 177 1956452ndash454

8 Nowell PC Cole LJ Habermyer JG Roan PL Growth and continued function of

rat marrow cells in x-irradiated mice Cancer Res Vol 16 1956258ndash261

9 Weissman IL Translating stem and progenitor cell biology to the clinic barriers

and opportunities Science Vol 287 20001442ndash1446

10Manz MG Akashi K Weissman IL Biology of Hematopoietic Stem and Progenitor

Cells In Appelbaum FR ed Thomas Hematopoietic Cell Transplantation Third

ed Malden MA Blackwell Publishing 2004

11Bacigalupo et al Defining the intensity of conditioning regimens Working

definitions Biol Blood Marrow Transplant 2009 December 15(12) 1628ndash1633l

REFERENCES

Congenital immune deficiencies Severe combined immunodeficiency disease

Wiskott-Aldrich syndromes

Marrow failure syndromes

Severe aplastic anemia

Fanconirsquos anemia

Diamond- Blackfan anemia

( Congenital hypoplastic anemia)

Inborn disorders of metabolism Mucopolysaccharidoses eg Hunter or Hurler syndrome

Leukodystrophies eg adrenoleukodystrophy

Glycoprotein disorders eg fucosidosis mannosidosis

Lysosomal disorders eg Gaucher Pompe Neimann-Pick disease

Osteropetrosis

Osteogenesis imperfecta

Hemoglobinopathies Thalasemia

Sickle cell disease

Other Indications for HPCs Stem Cell Transplant

Solid tumors Breast cancer

Ovarian cancer

Renal cell carcinoma

Testicular cancer

Childhood solid tumors Wilms tumor

Neuroblastoma

AABB Technical

Manual 17th Edition

Tra

nspla

nts

SUM11_41ppt

Transplant Activity in the US

1980-2010

0

2000

4000

6000

8000

10000

12000

14000

80 81 82 83 84 85 86 87 88 89 90 91 92 93 94 95 96 97 98 99 00 01 02 0304 05 06 07 08 09 10

Autologous

Related Donor

Unrelated Donor

SUM-WW11_4ppt

Autologous Stem Cell Sources

by Recipient Age

2000-2009

Age 20 yrs Age gt20 yrs

Tra

nspla

nts

0

20

40

60

80

100

2000-2004 2005-2009 2000-2004 2005-2009

Bone Marrow (BM)Peripheral Blood (PB)BM + PB

SUM-WW11_3ppt

Allogeneic Stem Cell Sources

by Recipient Age

2000-2009

Age 20 yrs Age gt20 yrs

Tra

nspla

nts

0

20

40

60

80

100

2000-2004 2005-2009 2000-2004 2005-2009

Bone Marrow (BM)

Peripheral Blood (PB)

Cord Blood (CB)

One-year survival after myeloablative conditioning for acute leukemias

in any remission phase

CML or MDS age lt50 years by year of transplant and graft source

1988-2009

One-Y

ear

Surv

ival

SUM11_40ppt

0

20

40

60

80

100

1988-

90

91 92 93 94 95 96 97 98 99 00 01 02 03 04 05 06 07 08 09

HLA-matched sibling

Unrelated Donor

SUM-WW11_14ppt

100-day Mortality after

Autologous Transplants

2008-2009

Mort

ality

0

10

20

30

40

50

Acute Leukemia Non-Hodgkin

Lymphoma

Hodgkin

Disease

Multiple

Myeloma

Early Disease

IntermediateAdvance DiseaseSensitive

ResistantUnknown

Other

0

20

40

60

80

100

AML ALL CML MDSMPS Aplastic

Anemia

Immune

Deficiency

SUM-WW11_16ppt

100-day Mortality after

Unrelated Donor Transplants

2008-2009

Early Disease

Intermediate Disease

Advanced Disease

Chronic Phase

Accelerated Phase

Blast Phase

Other

Mort

ality

Causes of Death

after Transplants

performed in

2008-2009

Autologous

Infection (8)

Other (16)

Organ Failure (2)

New Malignancy (1)

Primary Disease (73)

Unrelated Donor

Infection (16)

Other (29) Organ

Failure (6)

Primary Disease (33)

New Malignancy (1)

GVHD (15)

SUM-WW11_17ppt

HLA-identical Sibling

Infection (12)

Other (21)

Primary Disease (47)

GVHD (14)

Organ Failure (4)

New Malignancy (1)

In summary

After more than 50 years of research and

clinical use hematopoietic stem cells

have become the best-studied stem cells

with several indications

There is still active research ongoing to

expand its usehellip

1 Cellular Therapy AABB 16th Edition

2 AABB Techical Manual 17th edition

3 WWWNIHGOV

4 WWWCIBMTRORG

5 Jacobsen LO Marks EK Gaston EO Zirkle RE Effect of spleen protection on

mortality following X-irradiation J Lab Clin Med Vol 34 19491538ndash1543

6 Lorenz E Uphoff ED Reid TR Shelton E Modification of acute irradiation injury in

mice and guinea pigs by bone marrow injection Radiology Vol 58 1951863ndash877

7 Ford CE Hamerton JL Barnes DWH Loutit JF Cytological identification of

radiation-chimaeras Nature Vol 177 1956452ndash454

8 Nowell PC Cole LJ Habermyer JG Roan PL Growth and continued function of

rat marrow cells in x-irradiated mice Cancer Res Vol 16 1956258ndash261

9 Weissman IL Translating stem and progenitor cell biology to the clinic barriers

and opportunities Science Vol 287 20001442ndash1446

10Manz MG Akashi K Weissman IL Biology of Hematopoietic Stem and Progenitor

Cells In Appelbaum FR ed Thomas Hematopoietic Cell Transplantation Third

ed Malden MA Blackwell Publishing 2004

11Bacigalupo et al Defining the intensity of conditioning regimens Working

definitions Biol Blood Marrow Transplant 2009 December 15(12) 1628ndash1633l

REFERENCES

Tra

nspla

nts

SUM11_41ppt

Transplant Activity in the US

1980-2010

0

2000

4000

6000

8000

10000

12000

14000

80 81 82 83 84 85 86 87 88 89 90 91 92 93 94 95 96 97 98 99 00 01 02 0304 05 06 07 08 09 10

Autologous

Related Donor

Unrelated Donor

SUM-WW11_4ppt

Autologous Stem Cell Sources

by Recipient Age

2000-2009

Age 20 yrs Age gt20 yrs

Tra

nspla

nts

0

20

40

60

80

100

2000-2004 2005-2009 2000-2004 2005-2009

Bone Marrow (BM)Peripheral Blood (PB)BM + PB

SUM-WW11_3ppt

Allogeneic Stem Cell Sources

by Recipient Age

2000-2009

Age 20 yrs Age gt20 yrs

Tra

nspla

nts

0

20

40

60

80

100

2000-2004 2005-2009 2000-2004 2005-2009

Bone Marrow (BM)

Peripheral Blood (PB)

Cord Blood (CB)

One-year survival after myeloablative conditioning for acute leukemias

in any remission phase

CML or MDS age lt50 years by year of transplant and graft source

1988-2009

One-Y

ear

Surv

ival

SUM11_40ppt

0

20

40

60

80

100

1988-

90

91 92 93 94 95 96 97 98 99 00 01 02 03 04 05 06 07 08 09

HLA-matched sibling

Unrelated Donor

SUM-WW11_14ppt

100-day Mortality after

Autologous Transplants

2008-2009

Mort

ality

0

10

20

30

40

50

Acute Leukemia Non-Hodgkin

Lymphoma

Hodgkin

Disease

Multiple

Myeloma

Early Disease

IntermediateAdvance DiseaseSensitive

ResistantUnknown

Other

0

20

40

60

80

100

AML ALL CML MDSMPS Aplastic

Anemia

Immune

Deficiency

SUM-WW11_16ppt

100-day Mortality after

Unrelated Donor Transplants

2008-2009

Early Disease

Intermediate Disease

Advanced Disease

Chronic Phase

Accelerated Phase

Blast Phase

Other

Mort

ality

Causes of Death

after Transplants

performed in

2008-2009

Autologous

Infection (8)

Other (16)

Organ Failure (2)

New Malignancy (1)

Primary Disease (73)

Unrelated Donor

Infection (16)

Other (29) Organ

Failure (6)

Primary Disease (33)

New Malignancy (1)

GVHD (15)

SUM-WW11_17ppt

HLA-identical Sibling

Infection (12)

Other (21)

Primary Disease (47)

GVHD (14)

Organ Failure (4)

New Malignancy (1)

In summary

After more than 50 years of research and

clinical use hematopoietic stem cells

have become the best-studied stem cells

with several indications

There is still active research ongoing to

expand its usehellip

1 Cellular Therapy AABB 16th Edition

2 AABB Techical Manual 17th edition

3 WWWNIHGOV

4 WWWCIBMTRORG

5 Jacobsen LO Marks EK Gaston EO Zirkle RE Effect of spleen protection on

mortality following X-irradiation J Lab Clin Med Vol 34 19491538ndash1543

6 Lorenz E Uphoff ED Reid TR Shelton E Modification of acute irradiation injury in

mice and guinea pigs by bone marrow injection Radiology Vol 58 1951863ndash877

7 Ford CE Hamerton JL Barnes DWH Loutit JF Cytological identification of

radiation-chimaeras Nature Vol 177 1956452ndash454

8 Nowell PC Cole LJ Habermyer JG Roan PL Growth and continued function of

rat marrow cells in x-irradiated mice Cancer Res Vol 16 1956258ndash261

9 Weissman IL Translating stem and progenitor cell biology to the clinic barriers

and opportunities Science Vol 287 20001442ndash1446

10Manz MG Akashi K Weissman IL Biology of Hematopoietic Stem and Progenitor

Cells In Appelbaum FR ed Thomas Hematopoietic Cell Transplantation Third

ed Malden MA Blackwell Publishing 2004

11Bacigalupo et al Defining the intensity of conditioning regimens Working

definitions Biol Blood Marrow Transplant 2009 December 15(12) 1628ndash1633l

REFERENCES

SUM-WW11_4ppt

Autologous Stem Cell Sources

by Recipient Age

2000-2009

Age 20 yrs Age gt20 yrs

Tra

nspla

nts

0

20

40

60

80

100

2000-2004 2005-2009 2000-2004 2005-2009

Bone Marrow (BM)Peripheral Blood (PB)BM + PB

SUM-WW11_3ppt

Allogeneic Stem Cell Sources

by Recipient Age

2000-2009

Age 20 yrs Age gt20 yrs

Tra

nspla

nts

0

20

40

60

80

100

2000-2004 2005-2009 2000-2004 2005-2009

Bone Marrow (BM)

Peripheral Blood (PB)

Cord Blood (CB)

One-year survival after myeloablative conditioning for acute leukemias

in any remission phase

CML or MDS age lt50 years by year of transplant and graft source

1988-2009

One-Y

ear

Surv

ival

SUM11_40ppt

0

20

40

60

80

100

1988-

90

91 92 93 94 95 96 97 98 99 00 01 02 03 04 05 06 07 08 09

HLA-matched sibling

Unrelated Donor

SUM-WW11_14ppt

100-day Mortality after

Autologous Transplants

2008-2009

Mort

ality

0

10

20

30

40

50

Acute Leukemia Non-Hodgkin

Lymphoma

Hodgkin

Disease

Multiple

Myeloma

Early Disease

IntermediateAdvance DiseaseSensitive

ResistantUnknown

Other

0

20

40

60

80

100

AML ALL CML MDSMPS Aplastic

Anemia

Immune

Deficiency

SUM-WW11_16ppt

100-day Mortality after

Unrelated Donor Transplants

2008-2009

Early Disease

Intermediate Disease

Advanced Disease

Chronic Phase

Accelerated Phase

Blast Phase

Other

Mort

ality

Causes of Death

after Transplants

performed in

2008-2009

Autologous

Infection (8)

Other (16)

Organ Failure (2)

New Malignancy (1)

Primary Disease (73)

Unrelated Donor

Infection (16)

Other (29) Organ

Failure (6)

Primary Disease (33)

New Malignancy (1)

GVHD (15)

SUM-WW11_17ppt

HLA-identical Sibling

Infection (12)

Other (21)

Primary Disease (47)

GVHD (14)

Organ Failure (4)

New Malignancy (1)

In summary

After more than 50 years of research and

clinical use hematopoietic stem cells

have become the best-studied stem cells

with several indications

There is still active research ongoing to

expand its usehellip

1 Cellular Therapy AABB 16th Edition

2 AABB Techical Manual 17th edition

3 WWWNIHGOV

4 WWWCIBMTRORG

5 Jacobsen LO Marks EK Gaston EO Zirkle RE Effect of spleen protection on

mortality following X-irradiation J Lab Clin Med Vol 34 19491538ndash1543

6 Lorenz E Uphoff ED Reid TR Shelton E Modification of acute irradiation injury in

mice and guinea pigs by bone marrow injection Radiology Vol 58 1951863ndash877

7 Ford CE Hamerton JL Barnes DWH Loutit JF Cytological identification of

radiation-chimaeras Nature Vol 177 1956452ndash454

8 Nowell PC Cole LJ Habermyer JG Roan PL Growth and continued function of

rat marrow cells in x-irradiated mice Cancer Res Vol 16 1956258ndash261

9 Weissman IL Translating stem and progenitor cell biology to the clinic barriers

and opportunities Science Vol 287 20001442ndash1446

10Manz MG Akashi K Weissman IL Biology of Hematopoietic Stem and Progenitor

Cells In Appelbaum FR ed Thomas Hematopoietic Cell Transplantation Third

ed Malden MA Blackwell Publishing 2004

11Bacigalupo et al Defining the intensity of conditioning regimens Working

definitions Biol Blood Marrow Transplant 2009 December 15(12) 1628ndash1633l

REFERENCES

SUM-WW11_3ppt

Allogeneic Stem Cell Sources

by Recipient Age

2000-2009

Age 20 yrs Age gt20 yrs

Tra

nspla

nts

0

20

40

60

80

100

2000-2004 2005-2009 2000-2004 2005-2009

Bone Marrow (BM)

Peripheral Blood (PB)

Cord Blood (CB)

One-year survival after myeloablative conditioning for acute leukemias

in any remission phase

CML or MDS age lt50 years by year of transplant and graft source

1988-2009

One-Y

ear

Surv

ival

SUM11_40ppt

0

20

40

60

80

100

1988-

90

91 92 93 94 95 96 97 98 99 00 01 02 03 04 05 06 07 08 09

HLA-matched sibling

Unrelated Donor

SUM-WW11_14ppt

100-day Mortality after

Autologous Transplants

2008-2009

Mort

ality

0

10

20

30

40

50

Acute Leukemia Non-Hodgkin

Lymphoma

Hodgkin

Disease

Multiple

Myeloma

Early Disease

IntermediateAdvance DiseaseSensitive

ResistantUnknown

Other

0

20

40

60

80

100

AML ALL CML MDSMPS Aplastic

Anemia

Immune

Deficiency

SUM-WW11_16ppt

100-day Mortality after

Unrelated Donor Transplants

2008-2009

Early Disease

Intermediate Disease

Advanced Disease

Chronic Phase

Accelerated Phase

Blast Phase

Other

Mort

ality

Causes of Death

after Transplants

performed in

2008-2009

Autologous

Infection (8)

Other (16)

Organ Failure (2)

New Malignancy (1)

Primary Disease (73)

Unrelated Donor

Infection (16)

Other (29) Organ

Failure (6)

Primary Disease (33)

New Malignancy (1)

GVHD (15)

SUM-WW11_17ppt

HLA-identical Sibling

Infection (12)

Other (21)

Primary Disease (47)

GVHD (14)

Organ Failure (4)

New Malignancy (1)

In summary

After more than 50 years of research and

clinical use hematopoietic stem cells

have become the best-studied stem cells

with several indications

There is still active research ongoing to

expand its usehellip

1 Cellular Therapy AABB 16th Edition

2 AABB Techical Manual 17th edition

3 WWWNIHGOV

4 WWWCIBMTRORG

5 Jacobsen LO Marks EK Gaston EO Zirkle RE Effect of spleen protection on

mortality following X-irradiation J Lab Clin Med Vol 34 19491538ndash1543

6 Lorenz E Uphoff ED Reid TR Shelton E Modification of acute irradiation injury in

mice and guinea pigs by bone marrow injection Radiology Vol 58 1951863ndash877

7 Ford CE Hamerton JL Barnes DWH Loutit JF Cytological identification of

radiation-chimaeras Nature Vol 177 1956452ndash454

8 Nowell PC Cole LJ Habermyer JG Roan PL Growth and continued function of

rat marrow cells in x-irradiated mice Cancer Res Vol 16 1956258ndash261

9 Weissman IL Translating stem and progenitor cell biology to the clinic barriers

and opportunities Science Vol 287 20001442ndash1446

10Manz MG Akashi K Weissman IL Biology of Hematopoietic Stem and Progenitor

Cells In Appelbaum FR ed Thomas Hematopoietic Cell Transplantation Third

ed Malden MA Blackwell Publishing 2004

11Bacigalupo et al Defining the intensity of conditioning regimens Working

definitions Biol Blood Marrow Transplant 2009 December 15(12) 1628ndash1633l

REFERENCES

One-year survival after myeloablative conditioning for acute leukemias

in any remission phase

CML or MDS age lt50 years by year of transplant and graft source

1988-2009

One-Y

ear

Surv

ival

SUM11_40ppt

0

20

40

60

80

100

1988-

90

91 92 93 94 95 96 97 98 99 00 01 02 03 04 05 06 07 08 09

HLA-matched sibling

Unrelated Donor

SUM-WW11_14ppt

100-day Mortality after

Autologous Transplants

2008-2009

Mort

ality

0

10

20

30

40

50

Acute Leukemia Non-Hodgkin

Lymphoma

Hodgkin

Disease

Multiple

Myeloma

Early Disease

IntermediateAdvance DiseaseSensitive

ResistantUnknown

Other

0

20

40

60

80

100

AML ALL CML MDSMPS Aplastic

Anemia

Immune

Deficiency

SUM-WW11_16ppt

100-day Mortality after

Unrelated Donor Transplants

2008-2009

Early Disease

Intermediate Disease

Advanced Disease

Chronic Phase

Accelerated Phase

Blast Phase

Other

Mort

ality

Causes of Death

after Transplants

performed in

2008-2009

Autologous

Infection (8)

Other (16)

Organ Failure (2)

New Malignancy (1)

Primary Disease (73)

Unrelated Donor

Infection (16)

Other (29) Organ

Failure (6)

Primary Disease (33)

New Malignancy (1)

GVHD (15)

SUM-WW11_17ppt

HLA-identical Sibling

Infection (12)

Other (21)

Primary Disease (47)

GVHD (14)

Organ Failure (4)

New Malignancy (1)

In summary

After more than 50 years of research and

clinical use hematopoietic stem cells

have become the best-studied stem cells

with several indications

There is still active research ongoing to

expand its usehellip

1 Cellular Therapy AABB 16th Edition

2 AABB Techical Manual 17th edition

3 WWWNIHGOV

4 WWWCIBMTRORG

5 Jacobsen LO Marks EK Gaston EO Zirkle RE Effect of spleen protection on

mortality following X-irradiation J Lab Clin Med Vol 34 19491538ndash1543

6 Lorenz E Uphoff ED Reid TR Shelton E Modification of acute irradiation injury in

mice and guinea pigs by bone marrow injection Radiology Vol 58 1951863ndash877

7 Ford CE Hamerton JL Barnes DWH Loutit JF Cytological identification of

radiation-chimaeras Nature Vol 177 1956452ndash454

8 Nowell PC Cole LJ Habermyer JG Roan PL Growth and continued function of

rat marrow cells in x-irradiated mice Cancer Res Vol 16 1956258ndash261

9 Weissman IL Translating stem and progenitor cell biology to the clinic barriers

and opportunities Science Vol 287 20001442ndash1446

10Manz MG Akashi K Weissman IL Biology of Hematopoietic Stem and Progenitor

Cells In Appelbaum FR ed Thomas Hematopoietic Cell Transplantation Third

ed Malden MA Blackwell Publishing 2004

11Bacigalupo et al Defining the intensity of conditioning regimens Working

definitions Biol Blood Marrow Transplant 2009 December 15(12) 1628ndash1633l

REFERENCES

SUM-WW11_14ppt

100-day Mortality after

Autologous Transplants

2008-2009

Mort

ality

0

10

20

30

40

50

Acute Leukemia Non-Hodgkin

Lymphoma

Hodgkin

Disease

Multiple

Myeloma

Early Disease

IntermediateAdvance DiseaseSensitive

ResistantUnknown

Other

0

20

40

60

80

100

AML ALL CML MDSMPS Aplastic

Anemia

Immune

Deficiency

SUM-WW11_16ppt

100-day Mortality after

Unrelated Donor Transplants

2008-2009

Early Disease

Intermediate Disease

Advanced Disease

Chronic Phase

Accelerated Phase

Blast Phase

Other

Mort

ality

Causes of Death

after Transplants

performed in

2008-2009

Autologous

Infection (8)

Other (16)

Organ Failure (2)

New Malignancy (1)

Primary Disease (73)

Unrelated Donor

Infection (16)

Other (29) Organ

Failure (6)

Primary Disease (33)

New Malignancy (1)

GVHD (15)

SUM-WW11_17ppt

HLA-identical Sibling

Infection (12)

Other (21)

Primary Disease (47)

GVHD (14)

Organ Failure (4)

New Malignancy (1)

In summary

After more than 50 years of research and

clinical use hematopoietic stem cells

have become the best-studied stem cells

with several indications

There is still active research ongoing to

expand its usehellip

1 Cellular Therapy AABB 16th Edition

2 AABB Techical Manual 17th edition

3 WWWNIHGOV

4 WWWCIBMTRORG

5 Jacobsen LO Marks EK Gaston EO Zirkle RE Effect of spleen protection on

mortality following X-irradiation J Lab Clin Med Vol 34 19491538ndash1543

6 Lorenz E Uphoff ED Reid TR Shelton E Modification of acute irradiation injury in

mice and guinea pigs by bone marrow injection Radiology Vol 58 1951863ndash877

7 Ford CE Hamerton JL Barnes DWH Loutit JF Cytological identification of

radiation-chimaeras Nature Vol 177 1956452ndash454

8 Nowell PC Cole LJ Habermyer JG Roan PL Growth and continued function of

rat marrow cells in x-irradiated mice Cancer Res Vol 16 1956258ndash261

9 Weissman IL Translating stem and progenitor cell biology to the clinic barriers

and opportunities Science Vol 287 20001442ndash1446

10Manz MG Akashi K Weissman IL Biology of Hematopoietic Stem and Progenitor

Cells In Appelbaum FR ed Thomas Hematopoietic Cell Transplantation Third

ed Malden MA Blackwell Publishing 2004

11Bacigalupo et al Defining the intensity of conditioning regimens Working

definitions Biol Blood Marrow Transplant 2009 December 15(12) 1628ndash1633l

REFERENCES

0

20

40

60

80

100

AML ALL CML MDSMPS Aplastic

Anemia

Immune

Deficiency

SUM-WW11_16ppt

100-day Mortality after

Unrelated Donor Transplants

2008-2009

Early Disease

Intermediate Disease

Advanced Disease

Chronic Phase

Accelerated Phase

Blast Phase

Other

Mort

ality

Causes of Death

after Transplants

performed in

2008-2009

Autologous

Infection (8)

Other (16)

Organ Failure (2)

New Malignancy (1)

Primary Disease (73)

Unrelated Donor

Infection (16)

Other (29) Organ

Failure (6)

Primary Disease (33)

New Malignancy (1)

GVHD (15)

SUM-WW11_17ppt

HLA-identical Sibling

Infection (12)

Other (21)

Primary Disease (47)

GVHD (14)

Organ Failure (4)

New Malignancy (1)

In summary

After more than 50 years of research and

clinical use hematopoietic stem cells

have become the best-studied stem cells

with several indications

There is still active research ongoing to

expand its usehellip

1 Cellular Therapy AABB 16th Edition

2 AABB Techical Manual 17th edition

3 WWWNIHGOV

4 WWWCIBMTRORG

5 Jacobsen LO Marks EK Gaston EO Zirkle RE Effect of spleen protection on

mortality following X-irradiation J Lab Clin Med Vol 34 19491538ndash1543

6 Lorenz E Uphoff ED Reid TR Shelton E Modification of acute irradiation injury in

mice and guinea pigs by bone marrow injection Radiology Vol 58 1951863ndash877

7 Ford CE Hamerton JL Barnes DWH Loutit JF Cytological identification of

radiation-chimaeras Nature Vol 177 1956452ndash454

8 Nowell PC Cole LJ Habermyer JG Roan PL Growth and continued function of

rat marrow cells in x-irradiated mice Cancer Res Vol 16 1956258ndash261

9 Weissman IL Translating stem and progenitor cell biology to the clinic barriers

and opportunities Science Vol 287 20001442ndash1446

10Manz MG Akashi K Weissman IL Biology of Hematopoietic Stem and Progenitor

Cells In Appelbaum FR ed Thomas Hematopoietic Cell Transplantation Third

ed Malden MA Blackwell Publishing 2004

11Bacigalupo et al Defining the intensity of conditioning regimens Working

definitions Biol Blood Marrow Transplant 2009 December 15(12) 1628ndash1633l

REFERENCES

Causes of Death

after Transplants

performed in

2008-2009

Autologous

Infection (8)

Other (16)

Organ Failure (2)

New Malignancy (1)

Primary Disease (73)

Unrelated Donor

Infection (16)

Other (29) Organ

Failure (6)

Primary Disease (33)

New Malignancy (1)

GVHD (15)

SUM-WW11_17ppt

HLA-identical Sibling

Infection (12)

Other (21)

Primary Disease (47)

GVHD (14)

Organ Failure (4)

New Malignancy (1)

In summary

After more than 50 years of research and

clinical use hematopoietic stem cells

have become the best-studied stem cells

with several indications

There is still active research ongoing to

expand its usehellip

1 Cellular Therapy AABB 16th Edition

2 AABB Techical Manual 17th edition

3 WWWNIHGOV

4 WWWCIBMTRORG

5 Jacobsen LO Marks EK Gaston EO Zirkle RE Effect of spleen protection on

mortality following X-irradiation J Lab Clin Med Vol 34 19491538ndash1543

6 Lorenz E Uphoff ED Reid TR Shelton E Modification of acute irradiation injury in

mice and guinea pigs by bone marrow injection Radiology Vol 58 1951863ndash877

7 Ford CE Hamerton JL Barnes DWH Loutit JF Cytological identification of

radiation-chimaeras Nature Vol 177 1956452ndash454

8 Nowell PC Cole LJ Habermyer JG Roan PL Growth and continued function of

rat marrow cells in x-irradiated mice Cancer Res Vol 16 1956258ndash261

9 Weissman IL Translating stem and progenitor cell biology to the clinic barriers

and opportunities Science Vol 287 20001442ndash1446

10Manz MG Akashi K Weissman IL Biology of Hematopoietic Stem and Progenitor

Cells In Appelbaum FR ed Thomas Hematopoietic Cell Transplantation Third

ed Malden MA Blackwell Publishing 2004

11Bacigalupo et al Defining the intensity of conditioning regimens Working

definitions Biol Blood Marrow Transplant 2009 December 15(12) 1628ndash1633l

REFERENCES

In summary

After more than 50 years of research and

clinical use hematopoietic stem cells

have become the best-studied stem cells

with several indications

There is still active research ongoing to

expand its usehellip

1 Cellular Therapy AABB 16th Edition

2 AABB Techical Manual 17th edition

3 WWWNIHGOV

4 WWWCIBMTRORG

5 Jacobsen LO Marks EK Gaston EO Zirkle RE Effect of spleen protection on

mortality following X-irradiation J Lab Clin Med Vol 34 19491538ndash1543

6 Lorenz E Uphoff ED Reid TR Shelton E Modification of acute irradiation injury in

mice and guinea pigs by bone marrow injection Radiology Vol 58 1951863ndash877

7 Ford CE Hamerton JL Barnes DWH Loutit JF Cytological identification of

radiation-chimaeras Nature Vol 177 1956452ndash454

8 Nowell PC Cole LJ Habermyer JG Roan PL Growth and continued function of

rat marrow cells in x-irradiated mice Cancer Res Vol 16 1956258ndash261

9 Weissman IL Translating stem and progenitor cell biology to the clinic barriers

and opportunities Science Vol 287 20001442ndash1446

10Manz MG Akashi K Weissman IL Biology of Hematopoietic Stem and Progenitor

Cells In Appelbaum FR ed Thomas Hematopoietic Cell Transplantation Third

ed Malden MA Blackwell Publishing 2004

11Bacigalupo et al Defining the intensity of conditioning regimens Working

definitions Biol Blood Marrow Transplant 2009 December 15(12) 1628ndash1633l

REFERENCES

1 Cellular Therapy AABB 16th Edition

2 AABB Techical Manual 17th edition

3 WWWNIHGOV

4 WWWCIBMTRORG

5 Jacobsen LO Marks EK Gaston EO Zirkle RE Effect of spleen protection on

mortality following X-irradiation J Lab Clin Med Vol 34 19491538ndash1543

6 Lorenz E Uphoff ED Reid TR Shelton E Modification of acute irradiation injury in

mice and guinea pigs by bone marrow injection Radiology Vol 58 1951863ndash877

7 Ford CE Hamerton JL Barnes DWH Loutit JF Cytological identification of

radiation-chimaeras Nature Vol 177 1956452ndash454

8 Nowell PC Cole LJ Habermyer JG Roan PL Growth and continued function of

rat marrow cells in x-irradiated mice Cancer Res Vol 16 1956258ndash261

9 Weissman IL Translating stem and progenitor cell biology to the clinic barriers

and opportunities Science Vol 287 20001442ndash1446

10Manz MG Akashi K Weissman IL Biology of Hematopoietic Stem and Progenitor

Cells In Appelbaum FR ed Thomas Hematopoietic Cell Transplantation Third

ed Malden MA Blackwell Publishing 2004

11Bacigalupo et al Defining the intensity of conditioning regimens Working

definitions Biol Blood Marrow Transplant 2009 December 15(12) 1628ndash1633l

REFERENCES

Acknowledgements

Thanks to faculty of Post Graduate

Medical Training Program

Thanks to the Africa Partners Medical

Thanks to all for attending the conference