High-dose chemotherapy with autologous hemopoietic stem cell transplantation in children with retinoblastoma. Literature review and case series

Abstract

Retinoblastoma (RB) is the most common malignant eye tumor in children accounting for 10-15% of all cancer cases in infants. In spite of generally good standard treatment results there is still a subgroup of high-risk patients with by unfavorable prognosis, which warrants the search for additional treatment strategies. These children may benefit from treatment intensification in form of high-dose consolidation with subsequent autologous hemopoietic stem cell therapy (auto-HSCT). We here describe a case series illustrating N. N. Blokhin Cancer Center experience in this area. Materials and methods In 2021 a total of 3 children with high-risk RB were treated in N. N. Blokhin Cancer Center receiving highdose regimen consisting of carboplatin (total dose of 1250 mg/m2), etoposide (1750 mg/m2), and cyclophos-phamide (6000 mg/m2) with subsequent auto-HSCT. Results All 3 patients engrafted in day +11, +14, and +17 after auto-HSCT. The early post-transplant period was complicated by oral mucositis, neutropenic enterocolitis, and febrile neutropenia. One patient had a single episode of seizures due to hyponatremia. With a median follow-up of 6 (4-10) months two patients are alive and disease-free, one died due to disease progression. Conclusions The high-dose consolidation with subsequent auto-HSCT is a fea-sible option for high-risk RB patients. It is characterized by acceptable toxicity and is a potentially effective for disease control. In order to further evaluate the long-term results, there is a need for a larger prospective patient cohort. The multicenter study design may be beneficial in these settings.