Clinical use of multipotent mesenchymal stem cells in children after allogeneic bone marrow transplantation: the experience of the N.N. Blokhin National Medical Research Center of Oncology

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Introduction. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is one of the most important curative and consolidating treatment options for hematologic malignancies, immunologic diseases, and solid tumors. Nevertheless, its use may be limited due to life-threatening post-transplant complications, such as acute graft-versus-host disease (aGVHD), chronic graft-versus-host disease (cGVHD), and poor graft function. The standard treatment for these complications includes the administration of immunosuppressive drugs. However, their side effects and limited effectiveness necessitate the continued search for additional treatments, among which the use of mesenchymal stem cells (MSCs) may prove highly effective. Materials and methods. In this retrospective/prospective study conducted from 2021 to 2025, we enrolled 25 patients aged from 10 months to 16 years (with a median age of 11 years) with refractory aGVHD, cGVHD, and/or poor graft function after allo-HSCT. MSCs isolated from the bone marrow of allogeneic donors and cultured to passages 2–3 were administered intraosseously/intravenously or through a nasojejunal tube every 1–2 weeks. The primary endpoint was clinical response to therapy. Results. The median cell product dose was 20 (4–69) × 106 cells/kg recipient body weight. A response to MSC therapy was achieved in 15 (60.0%) out of 25 patients: in 8/9 (88.9%) patients with isolated poor graft function, in 4/6 patients who had both poor graft function and GVHD, and in 3/10 patients with isolated aGVHD/cGVHD. No adverse events associated with MSC administration were observed in any patient. Conclusion. The use of allogeneic MSCs in pediatric patients with refractory complications of allo-HSCT is a safe and potentially effective method. Our results are consistent with published data and warrant prospective randomized studies with a structured design and more precise response criteria.

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