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A phase II study of imatinib mesylate in children with refractory or relapsed solid tumors: A Children's Oncology Group study
Author(s) -
Bond Mason,
Bernstein Mark L.,
Pappo Alberto,
Schultz Kirk R.,
Krailo Mark,
Blaney Susan M.,
Adamson Peter C.
Publication year - 2008
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.21132
Subject(s) - medicine , imatinib mesylate , sarcoma , imatinib , osteosarcoma , neuroblastoma , synovial sarcoma , refractory (planetary science) , oncology , platelet derived growth factor receptor , surgical oncology , pathology , receptor , physics , myeloid leukemia , growth factor , biology , astrobiology , genetics , cell culture
Background Imatinib mesylate is a small molecule inhibitor of certain tyrosine kinases, most notably the chimeric bcr‐abl fusion protein found in CML. It also inhibits KIT and PDGF receptor tyrosine kinases in vitro. Ewing sarcoma, osteosarcoma, neuroblastoma, desmoplastic small round cell, and synovial sarcomas often overexpress KIT or the PDGF receptor. A phase II study of imatinib in children and young adults with select solid tumors was performed. Procedure Patients less than 30 years of age with refractory or recurrent Ewing sarcoma, osteosarcoma, neuroblastoma, desmoplastic small round cell, synovial sarcomas or GIST were eligible. Imatinib was administered daily for 28 day courses at a dose of 440 mg/m 2 /day. Responses were assessed according to Response Evaluation Criteria in Solid Tumor (RECIST). Results Seventy eligible patients, 48 male and 22 female, were enrolled and 59 were evaluable for response. Only one partial response was seen among 24 patients with Ewing sarcoma. There were no other objective responses. Hemorrhagic pleural effusions occurred in seven patients with pulmonary lesions, four of whom had progressive disease at the time of the hemorrhage. Intratumoral bleeding was reported in three additional patients. Conclusion Imatinib as a single agent at a dose of 440 mg/m 2 /day demonstrated little or no activity as a single agent in children with relapsed or refractory Ewing sarcoma, osteosarcoma, neuroblastoma, or desmoplastic small round cell tumors. Pediatr Blood Cancer 2008;50:254–258. © 2007 Wiley‐Liss, Inc.