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.
Original language | English (US) |
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Article number | e28288 |
Journal | Pediatric Blood and Cancer |
Volume | 67 |
Issue number | 6 |
DOIs | |
State | Published - Jun 1 2020 |
Bibliographical note
Funding Information:This work was supported by the Mayo Clinic Children's Research Center.
Publisher Copyright:
© 2020 Wiley Periodicals, Inc.
Keywords
- imatinib
- infantile myofibromatosis
- soft tissue tumor
- sorafenib
- targeted therapy