Safety and feasibility of tandem high-dose chemotherapy followed by autologous hematopoietic stem cell transplantation in children with solid malignant neoplasms: a single-center experience

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Background. Tandem high-dose chemotherapy (HDCT) followed by autologous hematopoietic stem cell transplantation (auto-HSCT) is an intensification strategy for consolidation therapy in children with certain high-risk solid malignant neoplasms (SMN). Despite evidence supporting the efficacy of this approach, its tolerability, technical feasibility, and toxicity profile in the pediatric population remain subjects of ongoing investigation. Objective – to analyze the tolerability, toxicity, and technical feasibility of tandem HDCT with auto-HSCT in children with high-risk SMN, including a comparative assessment against single auto-HSCT. Materials and methods. A retrospective-prospective single-center study was conducted, enrolling 115 pediatric patients (median age 48 (range – 7–215) months) with high-risk SMN who underwent HDCT with auto-HSCT. The study group comprised 20 patients who received tandem HDCT with auto-HSCT: 14 with germ cell tumors, 4 with neuroblastoma, 1 with hepatoblastoma, and 1 with sialoblastoma. The historical control group (n = 95) included 74 patients with neuroblastoma, 16 with Ewing sarcoma, and 5 with germ cell tumors, all of whom received single auto-HSCT. A graft of satisfactory quality was successfully collected from all patients in the study group. All patients received a conditioning regimen according to the underlying disease protocol, performance status, and baseline characteristics. Therapy toxicity, outcomes, and transplant-related mortality (TRM) were evaluated. Results. Hematopoietic recovery was achieved in all patients in the study group after both transplantation stages, the median time to neutrophil engraftment was 10.5 and 11.5 days after auto-HSCT №1 and auto-HSCT №2, respectively. Grade III–IV toxic complications were recorded in 40.0% of patients after auto-HSCT №1 and in 90.0% after auto-HSCT №2 (p = 0.001), which was comparable to the historical control group (81.1%). TRM in the study group was 0%. At the time of last follow-up, 17 (85.0%) patients in the study group were alive, of whom 16 (80.0%) were in remission; in the historical control group, 74 (77.9%) patients were alive, of whom 70 (73.7%) were in remission. Conclusion. Tandem HDCT with auto-HSCT is a technically feasible consolidation strategy in children with high-risk SMN, with an acceptable tolerability and toxicity profile. The absence of TRM and a manageable toxicity profile comparable to single auto-HSCT support the expansion of indications for this approach. Further multicenter studies are needed to evaluate long-term outcomes and optimize patient selection criteria.

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