Forms to be released

1 | P a g e Spring 2020 Release Highlights

Spring (May 8th) 2020 Release Highlights

Forms to be released: • 2402 R5 - Pre-Transplant Essential Data: Disease Classification • 2014 R4 - Myelodysplastic Syndrome Pre-Infusion • 2114 R4 - Myelodysplastic Syndrome Post-Infusion • 2057* R1 - Myeloproliferative Neoplasms Pre-Infusion • 2157* R1 - Myeloproliferative Neoplasms Post-Infusion • 2400£ R7 - Pre-Transplant Essential Data • 2500 R4 - Recipient Eligibility • 2149* R1 - Respiratory Virus Post-Infusion • 4000£ R6 - Pre-Cellular Therapy Essential Data • 2450£ R5 - Post-Transplant Essential Data • 2900£ R4 - Recipient Death Data • 2532 R2 - BMT CTN 1702 Enrollment • 2533 R2 - BMT CTN 1702 Donor Testing • 2534 R2 - BMT CTN 1702 Monthly Update • 2536 R2 - BMT CTN 1702 Off Study

£ Changes related to COVID-19 *indicates new form For information on high level changes to the forms (excluding BMT-CTN 1702 forms), select from the list below. The BMT-CTN 1702 Release Highlights is provided separately, on an additional document.

Table of Contents Pre-Transplant Essential Data (2402) Summary ..................................................................................................... 2

Myelodysplastic Syndrome Pre-Infusion (2014) Summary ..................................................................................... 7

Myelodysplastic Syndrome Post-Infusion (2114) Summary ................................................................................. 10

Myeloproliferative Neoplasms Pre-Infusion (2057) Highlights ............................................................................. 13

Myeloproliferative Neoplasms Post-Infusion (2157) Highlights ........................................................................... 15

Pre-Transplant Essential Data (2400) Summary ................................................................................................... 17

Recipient Eligibility (2500) Summary ................................................................................................................... 18

Respiratory Virus Post-Infusion (2149) Highlights ................................................................................................ 19

Pre-Cellular Therapy Essential Data (4000) Summary .......................................................................................... 20

Post-Transplant Essential Data (2450) Summary .................................................................................................. 21

Recipient Death Data (2900) Summary ................................................................................................................ 22


Pre-Transplant Essential Data (2402) Summary

Primary Disease for HCT / Cellular Therapy - Split the combined MDS / MPN primary disease option under Q2 into two categories, one for

MDS and another for MPN

Acute Myelogenous Leukemia (AML) - Updated questions 26, 53, and 80 from “FLT3 – D835 point mutation” to “FLT – TKD (point

mutations in D835 or deletions of codon I836)”

Myelodysplastic Syndrome (MDS) - MDS classifications under Q179 include the following, which have been updated to align with

2016 WHO criteria: o “Refractory cytopenia with unilineage dysplasia (RCUD)” updated to “Myelodysplastic

syndrome with single lineage dysplasia (MDS-SLD)” o Removed “Refractory anemia with ringed sideroblasts (RARS)” and added two options:

Myelodyplastic syndrome with ring sideroblasts with single lineage dysplasia (MDS-RS-SLD)

Myelodyplastic syndrome with ring sideroblasts with multilineage dysplasia (MDS-RS-MLD)

o “Refractory anemia with excess blasts-1 (RAEB-1)” updated to “MDS with excess blasts-1 (MDS-EB-1)”

o “Refractory anemia with excess blasts-2 (RAEB-2)” updated to “MDS with excess blasts-2 (MDS-EB-2)”

o “Refractory cytopenia with multilineage dysplasia (RCMD)” updated to “Myelodysplastic syndrome with multilineage dysplasia (MDS-MLD)”

o “Childhood myelodysyplastic syndrome (Refractory cytopenia of childhood (RCC))” updated to “Refractory cytopenia of childhood”

o “Myelodysplastic syndrome with isolated del(5q) o Myelodysplastic syndrome (MDS), unclassifiable

Expanded unclassifiable options in new Q180 o Overlap syndromes remain under MDS section including:

Chronic myelomonocytic leukemia (CMMoL) Juvenile myelomonocytic leukemia (JMML) Atypical chronic myeloid leukemia (aCML), BCR-ABL1 MDS / MPN with ring sideroblasts and thrombocytosis (MDS / MPN–RS–T) Myelodysplastic syndrome/ myeloproliferative neoplasm, unclassifiable

- Moved the MPN classifications in Q179 to the new MPN section in Q260 (updated to align with 2016 WHO criteria):


o Chronic neutrophilic leukemia o Chronic eosinophilic leukemia, not otherwise specified o Essential thrombocythemia o Polycythemia vera (PCV) o Primary myelofibrosis, removed text “(includes chronic idiopathic myelofibrosis (CIMF),

angiogenic myeloid metaplasia (AMM), myelofibrosis/sclerosis with myeloid metaplasia (MMM), idiopathic myelofibrosis)” to match 2016 WHO criteria exactly

o Mastocytosis, removed single “Mastocytosis” option Added three new options for “Cutaneous mastocytosis (CM)”, “Systemic

mastocytosis”, and “Mast cell sarcoma (MCS)” • Added options under “Systemic mastocytosis” for “Indolent systemic

mastocytosis (ISM”, “Smoldering systemic mastocytosis SSM”, “Systemic mastocytosis with an associated hematologic neoplasm (SM-AHN)”, “Aggressive systemic mastocytosis (ASM)”, and “Mast cell leukemia (MCL)”

o Myeloproliferative neoplasm (MPN), unclassifiable o Myeloid / lymphoid neoplasms with PDGFRA rearrangement o Myeloid / lymphoid neoplasms with PDGFRB rearrangement o Myeloid / lymphoid neoplasms with FGFR1 rearrangement o Myeloid / lymphoid neoplasms with PCM1-JAK2

- Added Q181 to capture if documentation was submitted to CIBMTR (pathology reported used for diagnosis) for “Myelodysplastic syndrome (MDS, unclassifiable)” & “Myelodysplastic syndrome / myeloproliferative neoplasm, unclassifiable” recipients

- Updated list of predisposing conditions in Q184 o “Aplastic Anemia” and “Fanconi Anemia” remains o Removed “Bloom syndrome” and “Down syndrome” o Added “DDX41-association familial MDS”, “Diamond-Blackfan Anemia”, “GATA2

deficiency”, “Li-Fraumeni Syndrome”, “Paroxysmal nocturnal hemoglobinuria”, “RUNX1 deficiency”, “SAMD9 or SAMD9L-associated familial MDS”, “Shwachman-Diamond Syndrome”, and “Telomere biology disorder” If “Paroxysmal nocturnal hemoglobinuria” recipients are placed on the CRF

track, the Aplastic Anemia pre-infusion disease insert will also be required for completion, in addition to the MDS pre-infusion disease insert

If “Fanconi anemia” recipients are placed on the CRF track, the Fanconi Anemia pre-infusion disease insert will also be required for completion, in addition to the MDS pre-infusion disease insert.

Laboratory Studies at diagnosis of MDS - Added Q186 to capture “Date CBC drawn” - Added questions 191 and 192 previously on the Pre-Infusion F2014 for blast % in the peripheral

blood (removed questions 22 &23 from F2014) - Karyotyping and FISH broken down into separate questions (202 and 210)


o Added questions to collect “sample source”, blood or BM (203 and 211) o Cytogenetic abnormality list is now a “check all that apply” (207 and 215) o Added questions to capture if documentation was submitted to CIBMTR (209 and 217)

- Updated MDS subtypes after transformation in Q219 in alignment with 2016 WHO criteria

Laboratory studies at Last Evaluation Prior to the start of the preparative regimen / infusion - Added Q223 to capture “Date CBC drawn” - Added questions 228 and 229 previously on the Pre-Infusion F2014 for blast % in the peripheral

blood (removed questions 130 & 131 from F2014) - Karyotyping and FISH are broken down into separate questions (239 and 247)

o Added questions to collect “sample source”, blood or BM (240 and 248) o Cytogenetic abnormality list is now a “check all that apply” (244 and 252) o Added questions to capture if documentation was submitted to CIBMTR (246 and 254)

Status at transplantation / infusion - Updated disease status criteria alignment with 2018 IWG criteria (will be clarified in manual)

o Question 257 added to specify transfusion dependence for recipients in “Hematologic improvement” with erythroid cell line (please note, “High transfusion burden” option will be disabled and removed from the form the next time the 2402 is revised. Q258 will also be disabled and removed from the form the next time the 2402 is

revised.

Myeloproliferative neoplasms (MPN) (new section) - Added Q262 to ask if documentation (pathology report used for diagnosis) was submitted for

subtype “Myeloproliferative Neoplasms (MPN), unclassifiable” - Does not include “Was the disease MPN therapy related?” (not relevant to MPN) - Does not include “Did the recipient have a predisposing condition?” (not relevant to MPN)

Assessment at diagnosis - New sub-section to capture constitutional symptoms in Q263

Laboratory Studies at diagnosis of MPN - Added Q264 to capture “Date CBC drawn” - Added questions 269 and 270 to capture blast % in the peripheral blood (removed questions 22

&23 from F2014) - Added questions to capture driver mutation results and if documentation was submitted to

CIBMTR in questions 279- 289 o JAK2: JAK2 V617F & JAK2 Exon 12 o CALR: CALR type 1, CALR type 2, and not defined o MPL o CSF3R

- Karyotyping and FISH broken down into separate questions (291 and 299) o Added questions to collect “sample source”, blood or BM (292 and 300)


o Cytogenetic abnormality list is a “check all that apply” & updated to include only relevant MPN abnormalities Monosomy:

• Removed -13 and -20 Trisomy:

• Removed: +19 • Added: +9

Translocation: • Removed: t(1;3), t(2;11), t(3;3), t(3;21), t(11;16) • Added: t(1;any), t(3q21;any), t(12p11.2;any), t(11q23;any)

Deletion: • Removed: del(3q / 3q, del(9q) / 9q

Inversion: • Added: dup(1)

o Added questions to capture if documentation was submitted to CIBMTR (298 and 306) - Updated MPN subtypes after transformation in Q308 to include only “Post-essential

thrombocythemic myelofibrosis”, “Post-polycythemic myelofibrosis”, and “Transformed to AML”

Assessment at last evaluation prior to the start of the preparative regimen/ infusion - New sub-section capturing transfusion dependence, constitutional symptoms, splenomegaly,

and hepatomegaly (questions 311-320)

Laboratory studies at Last Evaluation Prior to the start of the preparative regimen / infusion - Added Q321 to capture “Date CBC drawn” - Added questions 326 and 327 to capture blast % in the peripheral blood (removed questions

130 &131 from F2014) - Added questions to capture driver mutation results and the results and if documentation was

submitted to CIBMTR in questions 336- 346 o JAK2: JAK2 V617F & JAK2 Exon 12 o CALR: CALR type 1, CALR type 2, and not defined o MPL o CSF3R

- Karyotyping and FISH broken down into separate questions (348 and 356) o Added questions to collect “sample source”, blood or BM (349 and 357) o Cytogenetic abnormality list is a “check all that apply” & updated to include only

relevant MPN abnormalities Monosomy:

• Removed -13 and -20 Trisomy:

• Removed: +19 • Added: +9


Translocation: • Removed: t(1;3), t(2;11), t(3;3), t(3;21), t(11;16) • Added: t(1;any), t(3q21;any), t(12p11.2;any), t(11q23;any)

Deletion: • Removed: del(3q / 3q, del(9q) / 9q

Inversion: • Added: dup(1)

o Added questions to capture if documentation was submitted to CIBMTR (355 and 363)

Status at transplantation / infusion - Disease status criteria in Q364 updated to 2018 IWG criteria (will be clarified in manual)

o New questions 365- 367 to capture “anemia response”, “spleen response’, and “symptom response” for those recipients in “Clinical improvement”

o Added questions 369- 372 to capture “cytogenetic response” and “molecular response”


Myelodysplastic Syndrome Pre-Infusion (2014) Summary

Key Fields - Updated “CIBMTR Recipient ID” to 'CIBMTR Research ID' in key fields - Changed question text from “Date of HCT for which this form is being completed” to “Event

Date” - Removed “HCT Type” and “Product Type” fields

o Removed these fields so forms can be used as part of the cellular therapy form series

Subsequent Transplant or Cellular Therapy - Updated “Is this a report of a second or subsequent transplant or cellular therapy for the same

disease” field to an actual question (Q1) o Allows for easier querying

Disease Assessment at Diagnosis - Removed “MDS subtype” previous Q2 as this is already collected on the Disease Classification

F2402 - Previous Q3 “Was the disease MDS therapy related” and moved to the F2402 in Q182

o Child questions 2 – 7 remain on the F2014 Updated to level 1 Only to be enabled if the disease was reported as therapy related on the F2402

o Under “therapy for prior disease”, added options “Immune therapy’ and “Cellular therapy” and updated to “check all that apply”

- Removed previous Q12 “Did the recipient have a predisposing condition” as this is already captured on the Disease Classification F2402.

- Updated previous Q15 “Did the recipient receive any RBC transfusions at the time of diagnosis” to “Specify transfusion dependence at diagnosis” in Q8

- Updated splenomegaly and hepatomegaly questions to capture the method of assessment and size (questions 10 – 17)

o Questions on constitutional symptoms, splenomegaly, and hepatomegaly only to be answered for recipients with overlap syndromes

Diagnostic Studies (Measured Prior to Any Disease Treatment) - Previous questions 22 – 24 to capture “Blasts in blood” moved to the Disease Classification

F2402 in questions 191 – 192 o “Date CBC drawn” captured in Q186 on F2402

- Added Q18 to capture “Date CBC with differential drawn” - Added CBC differential questions: Bands, Metamyelocytes, Myelocytes, Lymphocytes, Basophils,

Eosinophils (questions 21 – 34) - Questions added to specify the MF grade in questions 38 – 39 - Molecular tests


o No longer asking for each result of positive, negative, not done. Several positive results can be reported as the questions have been updated to a multiple (multiple contains questions 43 – 46)

o Numerous molecular markers added including: BCOR, BCORL1, CBL, CUX1, DNMT3A, FLT3, GATA2, IDH1, IDH2, KRAS, NF1, NPM1, NRAS, PHF6, PPM1D, PTPN11, SETBP1, SF3B1, SRSF2, STAG2, TET2, U2AF1, WT1, ZRSR2

o Added questions 45 – 46 to capture the amino acid change in p. format

IPSS-R Prognosis score - New section to capture the maximum IPSS-R score ever achieved (questions 48 – 81)

o When it was documented o Clinical and laboratory assessments used to determine the maximum IPSS-R score

Pre-Infusion Therapy - Added “Date CBC with differential drawn” in Q83 and removed “date sample collected” from

“WBC”, “Hemoglobin”, “Platelets”, and “Neutrophils” as these should all be from the same CBC - Added CBC differential questions 88 - 101: Bands, Metamyelocytes, Myelocytes, Lymphocytes,

Monocytes, Basophils, Eosinophils - Added questions 102 – 103 to capture “blasts in blood” - Cytogenetic Results (questions 113 – 131)

o Karyotyping and FISH are broken down into separate questions o Added questions to collect “sample source”, blood or BM o New fields added for ISCN (International System for Human Cytogenetic Nomenclature)

Will not be enabled yet, work still in progress o Cytogenetic abnormality list updated to a “check all that apply” o Added questions to indicate if documentation was submitted

- Under “Line of Therapy” o Added Q137 to capture the reason therapy stopped o Systemic drugs updated to “check all that apply” in Q139

Added “Luspatercept” and “Venetoclax” Removed “Bendamustine”, “Ruxolitinib”, “Thalidomide”, and “Tyrosine kinase

inhibitor” o Split out supportive treatment into separate questions 141 – 142 o Added Q143 to capture if a cellular therapy was received o Added questions 144 – 145 to capture blinded randomized trial o Split out “splenic radiation”, “splenectomy”, and “other therapy” into their own

questions 146 – 150 - Best response HI option updated to align with the IWG criteria

o Question 152 added to specify transfusion dependence: non transfused or low.


Transformation - This section has been removed due to duplication. Transformation between diagnosis and the

start of the prep is already captured on the Disease Classification F2402 in questions 218 – 222.

Laboratory Studies at Last Evaluation Prior to the Start of the Preparative Regimen / Infusion

- Added Q157 to capture “Date CBC with differential drawn” - Added CBC differential questions 160 -171: Bands, Metamyelocytes, Myelocytes, Monocytes,

Basophils, Eosinophils - Previous questions 130 – 132 “Blasts in blood” moved to the Disease Classification F2402 in

questions 228 – 229 - Questions 175 – 176 added to specify the MF grade - Molecular tests

o No longer asking for each result of positive, negative, not done. Several positive results can be reported as the questions have been updated to a multiple (multiple contains questions 180 – 183)

o Numerous molecular markers added including: BCOR, BCORL1, CBL, CUX1, DNMT3A, FLT2, GATA2, IDH1, IDH2, KRAS, NF1, NPM1, NRAS, PHF6, PPM1D, PTPN11, SETBP1, SF3B1, SRSF2, STAG2, TET2, U2AF1, WT1, ZRSR2

o Added questions 182 – 183 to capture the amino acid change in p. format o Added Q184 to clarify if documentation was submitted

- Under flow results, if disease is detected, added a question to capture % in Q189 and Q193

Disease Assessment at Last Evaluation Prior to the Start of the Preparative Regimen / Infusion

- Add Q194 to capture transfusion dependence - Updated splenomegaly and hepatomegaly questions to capture the method of assessment and

size (questions 196 – 203) o Constitutional symptoms, splenomegaly, and hepatomegaly questions only to be

answered for overlap syndromes - Added questions 204 – 208 to capture “Iron overload”, including how the diagnosis was made

and therapy given - Removed disease status prior to prep in previous questions 157 - 161 as this is already captured

on the F2402 in questions 255 – 259.


Myelodysplastic Syndrome Post-Infusion (2114) Summary

Key Fields - Updated “CIBMTR Recipient ID” to “CIBMTR Research ID” in key fields - Changed question text from “Date of HCT for which this form is being completed” to “Event

date” - Removed “HCT Type” and “Product Type” fields

o Removed these fields so forms can be used as part of the cellular therapy form series

Disease Assessment at the Time of Best Response to HCT or Cellular Therapy - Best response in questions 1 -3 updated to align with 2018 IWG criteria, added questions to

capture transfusion dependence and major/ minor response - Mirrors the post-infusion AML F2110 format

o Added questions to capture splenomegaly and hepatomegaly at time of best response in questions 6 – 17 (only to be answered for overlap syndromes)

o Molecular tests Several positive results can be reported as the questions have been updated to

a multiple (multiple contains questions 21-24) Numerous molecular markers added, same list as seen on the Pre-Infusion form Added questions 23- 24 to capture the amino acid change in p. format Added question 26 to clarify if documentation was submitted to CIBMTR

o Added questions 31 & 36 to capture % disease detected in the flow results for both blood and BM

o Added questions 38 – 58 to capture raw cytogenetic results, sample source, abnormalities identified, and if documentation was submitted to CIBMTR

o Added questions 63- 64 to capture Myelofibrosis grading by WHO o Added questions 66 – 67 to capture if extramedullary disease indicative of AML was

detected o Added questions 68 – 72 to capture if disease was assessed by any other assessment

Post HCT / Post-Infusion Therapy - Mirrors the post-infusion AML F2110, section added to capture post-HCT/ post-infusion therapy

o Questions added to capture therapy given (questions 73 – 87) Including start date, stop date, reason stopped, therapy given Also including options for cellular therapy, blinded randomized trial, and other

therapy o Multiple lines of therapy can be reported

Disease detection since the date of last report - “Disease Relapse or Progression post-HCT” section updated to mirror the post-transplant AML

F2110


- Added questions 88 – 99 to capture splenomegaly and hepatomegaly - Molecular tests (questions 100 – 108) to capture raw results

o Several positive results can be reported as the questions are a multiple (multiple contains questions 103 – 106)

o Numerous molecular markers added, same list as seen on the Pre-Infusion form o Added questions to capture amino acid change in p. format in questions 105 – 106 o Added Q108 to clarify if documentation was submitted to CIBMTR

- Added questions 109 – 117 to capture raw flow cytometry results - Added questions 118 – 136 to capture raw cytogenetic results - Added questions 137 – 143 to capture raw BM examination results - Added questions 144 – 147 to capture if extramedullary disease indicative of AML detected - Added questions 148 – 151 to capture if disease was detected by any other assessment - Added questions 152 – 171 to capture intervention given for minimal residual disease,

persistent disease, relapsed disease, decreased/ loss of chimerism, or progression to AML since the date of last report

o Under Q153 “specify reason for which intervention was given”, added options for “decreased/loss of chimerism” and “Progression to AML”

o Added questions for date therapy started, stopped, and reason stopped o Treatment includes options for cellular therapy, subsequent HCT, accelerated

withdrawal of immunosuppression, blinded randomized trial, and other therapy

Laboratory studies at the time of evaluation for this reporting period - Section removed, BM examination questions moved to “Best response”, “Disease Detection

Since the Date of Last Report”, and “Disease status at the Time of Evaluation for this Reporting Period” sections

Disease status at the time of evaluation for this reporting period - Added Q172 to clarify if the current disease status reflects the disease detected in this reporting

period section, also mirroring the post-transplant AML F2110 o Questions 173 - 182 to capture splenomegaly and hepatomegaly o Questions 183 - 190 added to capture positive molecular results

Numerous molecular markers added, same list as seen on the Pre-Infusion form Added questions 188 - 189 to capture amino acid change in p. format Added Q190 to clarify if documentation was submitted to CIBMTR

o Added questions 191 – 199 to capture if the disease status was assessed via flow cytometry, and to specify % percent disease detected in blood and BM

o Added questions 200 - 209 to capture cytogenetic results for both FISH and karyotyping Includes Q209 to capture documentation submitted to CIBMTR

o Added questions 219 – 224 to capture BM examination results o Added questions 225 – 228 to capture if extramedullary disease indicative of AML

detected o Added questions 229 – 232 to capture if disease assessed by any other assessment


- Disease status in questions 233 – 236 updated to align with 2018 IWG criteria, added questions to capture transfusion dependence


Myeloproliferative Neoplasms Pre-Infusion (2057) Highlights *NOTE: new form, highlights do not include all fields Myelofibrosis CMS Study Pre-HCT Supplemental form retired and all questions incorporated into 2057

Disease Assessment at diagnosis - Includes questions to capture transfusion dependence, splenomegaly, and hepatomegaly

Diagnostic Studies (Measured Prior to Any Disease Treatment) - Contains questions to capture CBC differential counts such as Bands, Metamyelocytes,

Myelocytes, Lymphocytes, Basophils, and Eosinophils - Includes questions 31 – 32 to capture Myelofibrosis grading WHO classification - Captures positive molecular markers including the amino acid change in p. format in questions

33 – 40

DIPSS Prognosis Score - Captures the maximum DIPSS score, when the maximum DIPSS score was documented, and the

clinical & laboratory assessments used to determine the maximum DIPSS score in questions 41 – 54

Pre-HCT / Pre-Infusion Therapy - Contains questions to capture CBC differential counts such as Bands, Metamyelocytes,

Myelocytes, Lymphocytes, Basophils, and Eosinophils - Includes questions to capture blasts in blood, constitutional symptoms, and tests for driver

mutations - Captures positive molecular markers including the amino acid change in p. format in questions

98 – 162 o The appropriate number of repeat question sets will enable based on the total number

of positive molecular markers reported in Q100. - Cytogenetic results captured in questions 163 – 181, contains only those abnormalities

applicable to MPN subtypes - Under “Line of Therapy” in questions 182 – 201, to capture only treatments applicable to MPN

subtypes o Best response in questions 202 – 210 in alignment with 2018 IWG criteria

Laboratory Studies at Last Evaluation Prior to the Start of the Preparative Regimen / Infusion

- Contains questions to capture CBC differential counts such as Bands, Metamyelocytes, Myelocytes, Basophils, and Eosinophils

- Includes questions 231 – 232 to capture Myelofibrosis grading WHO classification - Captures positive molecular markers including the amino acid change in p. format in questions

233 – 240 - Includes flow cytometry results in questions 241 – 249


- Contains questions on total serum ferritin & CD34+ cells in questions 250 – 254

Disease Assessment at Last Evaluation Prior to the Start of the Preparative Regimen / Infusion

- Includes Q255 to capture pulmonary hypertension - Includes Q256 to capture portal hypertension - Contains questions 257 – 261 to capture iron overload

o How the diagnosis was made, and therapy given


Myeloproliferative Neoplasms Post-Infusion (2157) Highlights *NOTE: new form, highlights do not include all fields

Myelofibrosis CMS Study Post-HCT Supplemental form retired and all questions incorporated into 2157

Mirrors the post-infusion AML F2110 format

Disease Assessment at the Time of Best Response to HCT or Cellular Therapy - Best response in questions 1 – 4 updated to align with 2018 IWG criteria

o Includes Clinical Improvement (CI) option followed by “anemia response”, “spleen response’, and “symptom response”

- Contains splenomegaly and hepatomegaly questions 7 – 18 - Includes results on driver mutations in questions 19 – 31 - Captures positive molecular markers in questions 32 – 40 - Includes flow cytometry results in questions 41 – 51 - Contains cytogenetic results in questions 52 - 72 - Captures BM examination results in questions 73 – 79 - Includes questions 80 - 81 to capture if extramedullary disease indicative of AML detected - Questions 82 – 86 capture disease assessed by any other assessment

Post HCT / Post-Infusion Therapy - Questions 87 – 104 capture therapy given specific to MPN recipients

o Including start date, stop date, reason stopped, therapy given

Disease Detection Since the Date of Last Report - Contains splenomegaly & hepatomegaly questions 105 – 116 - Includes driver mutations results in questions 117 – 129 - Captures positive molecular markers in questions 130 – 138 - Includes flow cytometry results in questions 139 – 147 - Contains cytogenetic results in questions 148 – 166 - Captures BM examination results in questions 167 - 173 - Includes questions 174 – 177 to capture if extramedullary disease indicative of AML detected - Questions 178 – 181 capture disease detected by any other assessment - Captures intervention given for relapsed, persistent, or progressive disease including detection

of minimal residual disease or decreased/ loss of donor chimerism

Disease status at the Time of Evaluation for this Reporting Period - Contains splenomegaly & hepatomegaly questions 204 – 213 - Includes driver mutations results in questions 214 – 225 - Captures positive molecular markers in questions 226 – 233 - Includes flow cytometry results in questions 234 – 242 - Contains cytogenetic results in questions 243 – 261 - Captures BM examination results in questions 262 - 267


- Includes questions 268 – 271 to capture if extramedullary disease indicative of AML detected - Questions 272– 275 capture disease assessed by any other assessment - Disease status in questions 276 – 284 updated to align with the 2018 IWG criteria

o Includes Clinical Improvement (CI) option followed by “anemia response”, “spleen response’, and “symptom response”

o Added questions to capture cytogenetic response and molecular response


Pre-Transplant Essential Data (2400) Summary

Recipient Information - Zip Code in Q11 will continue to be required for USA residents, however it is now also be

optional for residents of Canada

Comorbid Conditions - Added questions 88 - 90 to capture COVID-19 (SARS-CoV-2) infection prior to the start of the

preparative regimen / infusion

Prior Exposure: Potential Study Eligibility - Moved prior Q1 on the Recipient Eligibility F2500 R3 to the Pre-TED F2400 R7, now Q147


Recipient Eligibility (2500) Summary

Recipient Exposure (removed section from F2500 R3)

- Moved prior Q1 on the Recipient Eligibility F2500 R3 to the Pre-TED F2400 R7, now Q147


Respiratory Virus Post-Infusion (2149) Highlights *New Form

The 2149 should only be completed for those recipients with COVID-19 (SARS-CoV-2) infection post-infusion.

Key Fields - Two Visits, “Initial” and “Follow-up”

Infection Diagnosis - Questions 1 – 10 capture diagnostic information

Hematologic Findings - Questions 11 – 24 capture hematologic findings

Therapy - Questions 25 – 39 capture therapy received - Q40 captures status of the infection


Pre-Cellular Therapy Essential Data (4000) Summary

Comorbid Conditions - Added questions 111 - 113 to capture COVID-19 (SARS-CoV-2) infection prior to the start of the preparative

regimen / infusion


Post-Transplant Essential Data (2450) Summary Survival

- Added option for COVID-19 (SARS-CoV-2) as both a primary and contributing cause of death in questions 3 and 5

Infection (new section) - Added questions 50 and 51 to capture if the recipient developed COVID-19 (SARS-CoV-2) since

the date of last report


Recipient Death Data (2900) Summary Recipient Death

- Added option for COVID-19 (SARS-CoV-2) as both a primary and contributing cause of death in questions 4 and 6

1 | P a g e BMT CTN – 1702 Release Highlights

BMT – CTN 1702 Release Highlights: Spring (May 8th) 2020

Forms to be released: • 2532 R2 - BMT CTN 1702 Enrollment • 2533 R2 - BMT CTN 1702 Donor Testing • 2534 R2 - BMT CTN 1702 Monthly Update • 2536 R2 - BMT CTN 1702 Off Study

For information on high level changes to the BMT-CTN 1702 forms, select from the list below. The Release Highlights for all other forms in the Spring 2020 Release are provided separately, on an additional document.

Table of Contents BMT-CTN 1702 Enrollment (2532) Summary .......................................................................................................... 2

BMT-CTN 1702 Donor Testing (2533) Summary ..................................................................................................... 2

BMT-CTN 1702 Monthly Update (F2534) Summary ................................................................................................ 2

BMT-CTN 1702 Off Study (2536) Summary ............................................................................................................ 3


BMT-CTN 1702 Enrollment (2532) Summary STUDY INCLUSION CRITERIA

a. Study Inclusion Criteria – Addition Expanded “Study Inclusion Criteria” (Q1-4) so each criterion is asked as a separate question. Rationale: Assist in study monitoring and confirmation that each criterion is met.

STUDY EXCLUSION CRITERIA a. Study Exclusion Criteria – Addition

Expanded “Study Exclusion Criteria” (Q5-6) so each criterion is asked as a separate question. Rationale: Assist in study monitoring and confirmation that each criterion is not met.

RECIPIENT STATUS a. Functional Status – Addition

Added questions 17-21 to capture the functional status (Karnofksy / Lanksy), including date of assessment. Rationale: Compare status between enrollment, evaluability, and transplant (if the patient proceeds to HCT).

b. Primary Disease for HCT – Revised Added option, now Q22, for “Acute leukemia of ambiguous lineage and other myeloid neoplasms”. Rationale: Provide a disease option for additional AML sub-types.

c. Disease Status – Addition Added option, now Q27 and 29, for “Currently undergoing induction treatment” under AML / ALL and HL / NHL. Rationale: Provide a disease status option for patients still having therapy.

BMT-CTN 1702 Donor Testing (2533) Summary DONOR TESTING

a. Related Relationship – Addition Added “full sibling”, “unspecified aunt”, “unspecified uncle”, and “unspecified cousin” to the related donor relationship list, Q1. Rationale: “Full sibling” was missed as an option on the initial form revision. “Unspecified aunt”, “unspecified uncle”, and “unspecified cousin” added for those centers that do not collect maternal / paternal data for extended family members.

b. Donor Identifier – Addition Added donor identifier field, Q4. Rationale: Allow data managers to differentiate between donors.

c. HLA Allele Match – Addition Added “< 4/8” as an option for HLA allele match, now Q12. Rationale: Provide a field for those donors tested with a match lower than 4/8, e.g. family members.

BMT-CTN 1702 Monthly Update (F2534) Summary CLINICAL STATUS

a. Functional Status– Addition Added Q4-5, “date functional status assessed”. Rationale: Include a date of assessment for functional status each time form submitted, in the same way the Enrollment Form (2532) was updated.


QUALITY OF LIFE SUBSTUDY (NEW SECTION) a. Enrollment Questions – Revised

Quality of Life Substudy enrollment questions, 32-38, asked at the time a donor has been selected, rather than after the patient has proceeded to HCT. Were previously Q30-36 on R1 and question text language slightly revised. Rationale: By the time the final donor is selected, substudy eligibility criteria are likely to be known, and by confirming substudy eligibility prior to transplant, it will assist in avoiding missed baseline quality of life surveys.

b. Quality of Life Survey Administration – Addition Added questions 39-46, quality of life survey administration, to capture method of administration, date survey provided and completed, and reason(s) not administered (as applicable). Rationale: Aid in tracking of baseline survey administration and in the event a baseline survey is missed, to collect reason(s) why the survey was not completed.

BMT-CTN 1702 Off Study (2536) Summary OFF STUDY

a. Taken off primary study – Addition Added questions 1-5 to capture if the subject was taken off of the primary study after enrollment but BEFORE being made evaluable & the reason if so, only to be answered if the subject status is not evaluable. Rationale: To capture reason for subjects who exit the study before they are declared evaluable.

Forms to be released

Documents

Transcript of Forms to be released