Case report: Slipped capital femoral epiphysis: a rare adverse event associated with FGFR tyrosine kinase inhibitor therapy in a child

Meziane Brizini, Tina Drimes, Cathy Bourne, Jessica Streilein, Annie Drapeau, Jens Wrogemann, Lori Anne Archer, Marc Del Bigio, M. Vanan
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Abstract

We report a case of slipped capital femoral epiphysis (SCFE), an on target skeletal toxicity of a pan-FGFR TKI inhibitor, erdafitinib. A 13-year-old boy was diagnosed to have an optic pathway/hypothalamic glioma with signs of increased intracranial pressure and obstructive hydrocephalus requiring placement of ventriculo-peritoneal (VP) shunt. Sequencing of the tumor showed FGFR1-tyrosine kinase domain internal tandem duplication (FGFR1-KD-ITD). He developed hypothalamic obesity with rapid weight gain and BMI >30. At 12 weeks of treatment with erdafitinib, he developed persistent knee pain. X-ray of the right hip showed SCFE. Erdafitinib was discontinued, and he underwent surgical pinning of the right hip. MRI at discontinuation of erdafitinib showed a 30% decrease in the size of the tumor, which has remained stable at 6 months follow-up. Our experience and literature review suggest that pediatric patients who are treated with pan-FGFR TKIs should be regularly monitored for skeletal side effects.
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病例报告:儿童股骨头骺滑脱:与表皮生长因子受体酪氨酸激酶抑制剂治疗相关的罕见不良事件
我们报告了一例股骨骨骺滑脱(SCFE)病例,这是一种泛表皮生长因子受体 TKI 抑制剂厄达非替尼的目标骨骼毒性。一名13岁男孩被诊断患有视通路/下丘脑胶质瘤,并伴有颅内压增高和梗阻性脑积水,需要进行脑室-腹膜(VP)分流术。肿瘤测序结果显示为表皮生长因子受体1-酪氨酸激酶结构域内部串联重复(FGFR1-KD-ITD)。他出现了下丘脑肥胖症,体重迅速增加,体重指数(BMI)大于 30。在接受厄达非替尼治疗12周后,他出现了持续性膝关节疼痛。右髋部X光片显示他患有SCFE。他停用了厄达菲尼,并接受了右髋关节置钉手术。停用厄达非尼后的核磁共振成像显示肿瘤缩小了30%,随访6个月后肿瘤仍保持稳定。我们的经验和文献综述表明,接受泛FGFR TKIs治疗的儿童患者应定期监测骨骼副作用。
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