Autologous Hematopoietic Stem Cell Transplantation for “Rare” Solid Malignant Diseases in Children. Experience of the N.N. Blokhin National Medical Research Center of Oncology
Abstract
Introduction: One of the key stages of therapy for children with “rare” solid malignant neoplasms (MN), improving the prognosis for the underlying disease, is autologous hematopoietic stem cell transplantation (auto-HSCT). In world practice, this therapy is carried out for children with common diseases such as malignant neoplasms (MN), however, there is a small cohort of patients with “rare” diseases, with which not all centers have experience in carrying out HSCT in children.
Methodology: At the Research Institute of Pediatric Oncology and Hematology (RI DOiG) of the N.N. Blokhin National Medical Research Center of Oncology, auto-HSCT was performed in 12 patients over 5 years (median age 7.6 years, range 1.3-17.2). The median follow-up was 37.5 months (interquartile range 5.1-78.3). Indications included retinoblastoma (9 patients, RB), pleuropulmonary blastoma (1 patient, PPB), sialoblastoma (1 patient, SB), hepatoma (1 patient, HB). M:F= 7:5. Stem cell source: PBSC – 100%. Conditioning regimens: PPB – Treo/Mel, HB – Eto/Carbo, SB/RB – thiotepa-based regimens. The median CD34+ cell dose was 5.27×10 6/kg (2.4 – 15.1).
Results: The patients successfully underwent the HSCT stage. In the early stages after HSCT, complications were noted: toxicoderma up to grade 2-3, oropharyngeal mucositis up to grade 1-3, neutropenic enterocolitis grade 1-2, infections, hepato- and nephrotoxicity. These complications were stopped. All patients engrafted. The median ANC recovery was 12 (9.7–16.2) days. No significant toxicity was recorded. There were 3 fatal outcomes in patients with RB associated with relapse of the underlying disease.
Conclusion: Due to the low frequency of occurrence of these diseases in medical practice, as well as late treatment/referral to specialized medical centers, there is no standard therapy and experience of working with these patients in the early and late stages of HSCT. Each patient with a “rare” disease requires an individual approach to management at the stage of HSCT.
How to Cite
Aliev TZ, Machneva EB, Kostareva I, Sergeenko K, Smirnova D, Burlaka N, et al. Autologous Hematopoietic Stem Cell Transplantation for “Rare” Solid Malignant Diseases in Children. Experience of the N.N. Blokhin National Medical Research Center of Oncology. OncoDaily Med J. 2025. doi:10.69690/ODMJ-018-0425-1713