How to optimize outcome of patients undergoing HLA-matched related haematopoietic stem cell transplantation in acquired and inherited bone marrow failure syndromes.
Stefano GiardinoFilomena PierriMaura FaraciPublished in: British journal of haematology (2023)
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.
Keyphrases
- stem cell transplantation
- high dose
- low dose
- bone marrow
- patients undergoing
- hematopoietic stem cell
- mesenchymal stem cells
- young adults
- end stage renal disease
- endothelial cells
- early onset
- allogeneic hematopoietic stem cell transplantation
- ejection fraction
- peritoneal dialysis
- prognostic factors
- drug induced
- social media
- healthcare
- acute lymphoblastic leukemia
- replacement therapy
- induced pluripotent stem cells