Treatment of generalized infantile myofibromatosis with sorafenib and imatinib: A case report

Behzad Bidadi; Andrea Watson; Brenda Weigel; Andre Oliveira; Justin Kirkham; Carola Arndt

doi:10.1002/pbc.28288

Treatment of generalized infantile myofibromatosis with sorafenib and imatinib: A case report

Behzad Bidadi, Andrea Watson, Brenda Weigel, Andre Oliveira, Justin Kirkham, Carola Arndt

Pediatrics - Hematology

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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)
Article number	e28288
Journal	Pediatric Blood and Cancer
Volume	67
Issue number	6
DOIs	https://doi.org/10.1002/pbc.28288
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

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title = "Treatment of generalized infantile myofibromatosis with sorafenib and imatinib: A case report",

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AB - 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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Treatment of generalized infantile myofibromatosis with sorafenib and imatinib: A case report

Abstract

Bibliographical note

Keywords

UN SDGs

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