Divisions of Hematology, Oncology, and Blood and Marrow Transplant,
Cardiology.
Pediatrics. 2019 Jun;143(6). doi: 10.1542/peds.2018-2495. Epub 2019 May 14.
Cardiac rhabdomyoma is the most common neonatal cardiac tumor and is typically associated with tuberous sclerosis complex (TSC). Although these tumors may naturally regress, some patients require surgical resection because of cardiac instability. If not fully resected, patients may also require medical therapy to improve their hemodynamics. Everolimus, a mammalian target of rapamycin inhibitor, has shown promise in reducing rhabdomyoma in patients with TSC, but the drug's impact in patients without TSC has not been reported. Monitoring of tumor response has typically been limited to echocardiograms, which is not ideal given inherent difficulties in three-dimensional measurements. We report a case of sporadic cardiac rhabdomyoma in a neonate treated with everolimus resulting in tumor regression as documented by cardiac MRI. While on everolimus, our patient had an increased incidence of a preexisting arrhythmia, which resolved with planned cessation of therapy, suggesting that close monitoring is imperative in patients with arrhythmia.
心脏横纹肌瘤是最常见的新生儿心脏肿瘤,通常与结节性硬化症复合征(TSC)相关。尽管这些肿瘤可能会自然消退,但由于心脏不稳定,一些患者需要手术切除。如果不能完全切除,患者可能还需要药物治疗以改善其血液动力学。雷帕霉素哺乳动物靶蛋白抑制剂依维莫司在减少 TSC 患者的横纹肌瘤方面显示出良好的效果,但该药物在无 TSC 患者中的作用尚未报道。肿瘤反应的监测通常仅限于超声心动图,但由于三维测量存在固有困难,这种方法并不理想。我们报告了一例散发性心脏横纹肌瘤新生儿接受依维莫司治疗的病例,心脏 MRI 证实肿瘤消退。在接受依维莫司治疗期间,我们的患者出现了先前存在的心律失常的发生率增加,通过计划停止治疗得到解决,这表明心律失常患者需要密切监测。