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Treatment of generalized infantile myofibromatosis with sorafenib and imatinib: A case report
Author(s) -
Bidadi Behzad,
Watson Andrea,
Weigel Brenda,
Oliveira Andre,
Kirkham Justin,
Arndt Carola
Publication year - 2020
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.28288
Subject(s) - medicine , sorafenib , imatinib , tyrosine kinase inhibitor , tyrosine kinase , oncology , cancer , receptor , myeloid leukemia , hepatocellular carcinoma
Abstract Infantile myofibromatosis (IM) is characterized by solitary musculoskeletal nodules presenting during infancy but can manifest as multiple lesions with visceral involvement. Multicentric IM with visceral involvement carries a high risk of mortality and there is no consensus on treatment. We present a case of a patient with multicentric IM and pulmonary involvement who progressed on several chemotherapeutic regimens and subsequently had a complete response to sorafenib and later imatinib. This report describes the novel use of sorafenib and imatinib to treat generalized IM and the role of continued tyrosine kinase inhibitor therapy to maintain remission.

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