Post on 17-Aug-2020
The role of medical therapy in the management of
desmoid tumor
Andrea Marrari, MDVisiting Research Fellow
Center for Sarcoma and Bone Oncology
Dana-Farber Cancer Institute
Sporadic tumors are rare (600-1200 cases per year in the US)
Patients with familial adenomatous polyposis have an increased risk of developing desmoid tumor
Unpredictable clinical behaviour
Desmoid tumor
Desmoids can be found in a wide variety of locations
10% head and neck
22% scapular girdle
10% upper extremity
25% abdominal wall
5% intra-abdominal
5% pelvic girdle
23% lower extremity
Surgery is the primary treatment for DT
Survival is virtually unaffected by
extra-abdominal DT
Some anatomic sites may become
life-threatening:mesentery and head
and neck
Medical therapy for DT
Who to treat
What to use
How long
Toxicity
Treatment strategy
Resectable Unresectable
Stable
Progressing Surgery
Surgery Observation
Medical therapy+/- surgery
Observation
Medical therapy+/- surgery
Active drugs
Anti-estrogens
Nonsteroidal anti-inflammatory drugs
Chemotherapy
Molecular targeted therapy
Interferons
Tamoxifen
Tamoxifen 20 mg/day for 2 years
Courtesy of Gronchi A., INT
Anti-inflammatory drugs
Nishida Y. et al, JCO 2010
Combination strategy
Low dose chemotherapy
Methotrexate and vinblastine
Methotrexate and vinorelbine
Vinorelbine
Methotrexate and vinorelbine
Methotrexate and vinorelbine for 26 cycles
Courtesy of Gronchi A., INT
Conventional chemotherapy
Adriamycin
Adriamycin and dacarbazine
Liposomal doxorubicin
Liposomal doxorubicin
Liposomal doxorubicin after 9 months
Bertagnolli M.M. et al, Eur J Cancer 2006
Molecular targeted therapy
Molecular targeted therapy
Cell proliferationAngiogenesisMetabolism
Motility…
Ligand
Receptor
STOP
Intracellular space
Extracellular space
Adapted from Krause D.S. et al, NEJM 2005
Molecular targeted therapy
Imatinib: ABL, KIT and PDGFR
Sunitinib: KIT, PDGFR , VEGFR and RET
Sorafenib: KIT, PDGFR, VEGFR, RET and RAF
Imatinib
Post 14 months8.7 cm
Baseline13.9 cm
Heinrich M.C. et al, JCO 2006
Imatinib400 mg twice daily
Molecular targeted therapy
Imatinib: ABL, KIT and PDGFR
Sunitinib: KIT, PDGFR , VEGFR and RET
Sorafenib: KIT, PDGFR, VEGFR, RET and RAF
Molecular targeted therapy
Imatinib: ABL, KIT and PDGFR
Sunitinib: KIT, PDGFR , VEGFR and RET
Sorafenib: KIT, PDGFR, VEGFR, RET and RAF
Sorafenib
Gounder M.M. et al, ASCO 2010
Sorafenib400 mg twice daily
Post 11 monthsBaseline
Multidisciplinary assessment and treatment planning is critical
Treatment should be tailored according to patients characteristics and tumor behaviour
Chemotherapy is effective in disease control
Conclusion
GRAZIE!
Milan Cathedral, 1368-1965