How to optimize outcome of patients undergoing HLA‐matched related haematopoietic stem cell transplantation in acquired and inherited bone marrow failure syndromes

Abstract

Up‐front allogeneic haematopoietic stem cell transplantation (allo‐HSCT) after a reduced intensity conditioning regimen is the standard treatment in children with acquired severe aplastic anaemia (aSAA) and inherited bone marrow failure syndromes (iBMFs) in the presence of a healthy matched related donor (MRD). The paper by Alsultan et al. report the safety and efficacy of MRD HSCT conditioned with low‐dose cyclophosphamide, fludarabine and thymoglobulin in both aSAA and non‐Fanconi iBMFs, strengthening the concept of the pivotal role of immunosuppressive approach in allo‐HSCT for specific subgroups of non‐malignant diseases requiring a reduced risk of toxicities, offering the opportunity to discuss the essential points for achieving patients' long‐term survival after MRD HSCT in BMF.Commentary on: Alsultan et al. Human leucocyte antigen‐matched related haematopoietic stem cell transplantation using low‐dose cyclophosphamide, fludarabine and thymoglobulin in children with severe aplastic anaemia. Br J Haematol 2023 (Online ahead of print). doi: 10.1111/bjh.19004.

HLA‐haploidentical stem cell transplantation in children with inherited bone marrow failure syndromes: A retrospective analysis on behalf of EBMT severe aplastic Anemia and pediatric diseases working parties

“…Upfront allo-SCT still represents the only curative option for patients with I-BMFs. 29 Haplo-SCT is the main alternative for patients without a healthy matched related or unrelated donor, and TCD mitigates the otherwise high risk of developing severe GvHD. However, historically haplo-SCT with TCD was burdened with a higher incidence of engraftment failure and a slow achievement of immune reconstitution, but the evolution in both ex vivo and in vivo TCD has changed the history of haplo-SCT in both malignant [30][31][32] and non-malignant diseases.…”