Objective  To analyze the clinical characteristics, treatment, and outcomes of children with primary vaginal malignancies.

  Methods  We conducted a retrospective analysis on clinical and pathological data of all children with pathologically confirmed primary vaginal malignancies treated at Peking Union Medical College Hospital between January 1980 and March 2015. The clinical characteristics, therapeutic methods, treatment effect, and outcomes of the children were summarized.

  Results  Twenty-five patients (median age, 11 months; range, 8 months to 12 years) were identified, including 16 (64%) endodermal sinus tumor (EST), 7 (28%) embryonal rhabdomyosarcoma (ERMS), and 2 (8%) clear-cell adenocarcinoma (CCA). Bleeding or blood-tinged discharge was the most common clinical presentation (76%), followed by a protruding mass (24%). All patients were performed conservative surgery. Only one patient received radiotherapy. Childhood genital malignancies were generally sensitive to chemotherapy.According to the histological type and high risk factors, different chemotherapy regimens were administrated after surgery, such as PEB (cisplatin, etoposide, bleomycin), PVB (cisplatin, vincristine, bleomycin), IVA (ifosfamide, vincristine, actinomycin), or VAC (vincristine, actinomycin, cyclophosphamide). The patients were followed up for a median of 44 months (range, 6 months to 18 years). Twenty-two patients (88%) remained alive. Three patients (12%) died of disease progression (1 ERMS, and 2 CCA). Recurrence and death occurred mostly in the first two years after treatment.

  Conclusions  Vaginal primary malignancies are extremely rare in children, among which EST is the most common type, followed by ERMS and CCA. EST and ERMS at the vagina are sensitive to chemotherapy, therefore the main therapeutic method is combined chemotherapy, and the prognosis is good. CCA is not sensitive to chemotherapy, thus the main therapeutic methods are surgery and radiotherapy, and the prognosis is poor. Early recognition of symptoms and early treatment is the key to improve survival.