Merck Pharmaceuticals, North Wales, Pennsylvania.
Division of Pediatric Hematology/Oncology, Department of Pediatric and Adolescent Medicine, Mayo Clinic, Rochester, Minnesota.
Pediatr Blood Cancer. 2020 Jun;67(6):e28288. doi: 10.1002/pbc.28288. Epub 2020 Apr 19.
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.
婴儿肌纤维瘤病（IM）的特征是在婴儿期出现孤立的肌肉骨骼结节，但也可以表现为多发性病变伴内脏受累。多中心伴内脏受累的 IM 死亡率高，治疗尚无共识。我们报告了一例多中心 IM 伴肺受累的患者，该患者在几种化疗方案后进展，并随后对索拉非尼和伊马替尼有完全反应。本报告描述了索拉非尼和伊马替尼治疗广泛 IM 的新用途，以及持续使用酪氨酸激酶抑制剂治疗以维持缓解的作用。
