High-dose chemotherapy followed by autologous hematopoietic stem cell transplantation in children with poor-prognosis germ cell tumors: a single-center experience

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Background. Germ cell tumors (GCT) in children represent a group of chemosensitive malignant neoplasms. However, for a small subset of patients with refractory, relapsed, or disseminated GCT, the prognosis remains extremely poor, with overall survival rates potentially not exceeding 45%. High-dose chemotherapy (HDCT) followed by autologous hematopoietic stem cell transplantation (auto-HSCT) is considered a treatment strategy with the potential to improve outcomes in this patient population. Objective – to analyze the experience of HDCT with auto-HSCT in children with poor-prognosis GCT, with assessment of the efficacy and safety of this therapeutic approach. Materials and methods. This single-center retrospective study enrolled 20 children (9 boys and 11 girls) with relapsed (n = 9), refractory (n = 3), and disseminated (n = 8) forms of GCT who received HDCT followed by auto-HSCT. The median age is 2 (1–18) years. All patients had a graft of adequate cellularity collected prior to transplantation: the median CD34+ cell dose was 3.6 (range 2.0–8.1) ×106/kg body weight. A tandem HDCT regimen was administered to 14 patients (first course: carboplatin 1200 mg/m2, etoposide 1500 mg/m2, second course: etoposide 1500 mg/m2 and thiotepa 900 mg/m2). Six patients received a single-course HDCT regimen (etoposide 1500 mg/m2 and thiotepa 900 mg/m2). The median follow-up was 27 (range 1–61) months. Results. All patients achieved hematopoietic recovery following auto-HSCT. Febrile neutropenia developed in 11 patients (55.0%) during the early post-transplant period, with a mean duration of fever of 4 (range 0–6) days. Oropharyngeal mucositis and gastrointestinal mucositis were observed in all 20 patients (100.0%), and dermatological toxicity was noted in 16 patients (80.0%). In the majority of cases, the severity of toxic and infectious complications did not exceed grade 2. At a median follow-up of 2.7 years, overall survival was 84.0%, event-free survival was 78.0%, and relapse-free survival was 81.0%. Disease progression after auto-HSCT was documented in 3 patients, 2 of whom remain alive. Two patients died: one due to disease progression, and one due to an infectious episode at the place of residence 6 months after the completion of therapy. Conclusion. HDCT with auto-HSCT in children with poor-prognosis GCT may be considered an effective therapeutic approach with an acceptable toxicity profile.

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