Low-dose everolimus as an effective therapy for unresectable giant cardiac rhabdomyoma in a preterm infant: a case report

Abstract Background Cardiac Rhabdomyomas (CRs) are the most common cardiac tumors in children and often regress spontaneously, however, large lesions in neonates may cause hemodynamic compromise. Surgical resection is standard for symptomatic tumors, but complete excision may be challenging in cases of diffuse myocardial infiltration or multifocal lesions. Everolimus has emerged as a potential therapeutic option when surgery is not feasible. Case presentation A preterm infant with a giant left ventricular rhabdomyoma causing significant mitral regurgitation and clinical deterioration was unable to undergo complete surgical resection due to diffuse myocardial infiltration. Low-dose everolimus (0.25 mg twice weekly) was initiated, resulting in rapid clinical improvement and remarkable tumor regression from 3.79 × 2.0 cm to 2.7 × 0.7 cm over six weeks. Subsequent echocardiography tests confirmed improved left ventricular function and reduced mitral valve insufficiency Conclusions This case demonstrates that low-dose everolimus may be an effective therapeutic option for symptomatic neonatal CRs when total surgical resection is not feasible, offering a promising alternative for high-risk or unresectable cardiac tumors.