The Role of Chemotherapy in Pediatric Myoepithelial Carcinoma: A Systematic Review of the Literature

Myoepithelial carcinoma (MEC) in pediatric patients is a rare and aggressive malignancy characterized by heterogeneous morphology and variable molecular features. The optimal role of chemotherapy remains unclear. We conducted a systematic review according to PRISMA 2020 guidelines to evaluate chemotherapy in pediatric and young-adult patients with extra-salivary MEC. Twenty-one studies including 40 patients were analyzed. Chemotherapy response was evaluable in 19 patients, with partial responses in 57.9%, stable disease in 15.8%, and progressive disease in 26.3%; no complete responses were observed with chemotherapy alone. Comparable activity was found between TREP-based (ICpE) and sarcoma-like regimens, with no evidence of superiority of one regimen over the other, but supporting ifosfamide-based combinations as a therapeutic backbone. Radiotherapy and complete surgical resection were associated with improved local control. At a median follow-up of 21 months, 35% of patients were alive with no evidence of disease, while 37.5% had died of disease. Patients with EWSR1 rearrangements showed relatively favorable outcomes. Overall, chemotherapy demonstrates limited efficacy, and a multimodal approach remains essential. These findings highlight the need for international guidelines within the EXPeRT group to standardize management and improve outcomes.