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Updated: Jul 20, 2025

Use of Hematopoietic Stem Cell Transplantation to Assess the Origin of Myelodysplastic Syndrome
Published on: October 3, 2018
[Allogeneic hematopoietic stem cell transplantation for myelodysplastic syndromes]
Viviane Gournay1, Marie Robin1
1AP-HP, université de Paris Cité, hôpital Saint-Louis, Paris, France.
Abstract:
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is one of the treatment options for myelodysplastic syndromes (MDS). This treatment is indicated as first-line treatment for high-risk MDS according to the IPSS and R-IPSS classifications and improves overall survival and progression-free survival. However, allo-HSCT is not indicated in first intention for low-risk MDS. It can be discussed in case of cytopenias needing transfusions, poor evolution under other treatment, or in case of poor prognosis molecular anomaly. Allo-HSCT is a treatment that can be complicated by early or late toxicities (graft versus host disease, infections, chemotherapy toxicity…). The decision to do an allo-HSCT is based on the benefit/risk ratio between the risk of progression from MDS to myeloid leukemia and the risk of transplant related mortality, which increases with the patient's age and comorbidities. The indication of a cytoreductive treatment before allo-HSCT depends on the blasts count, and on the delay before the allograft. The use of reduced intensity conditioning regimen and alternative donors such as haploidentical donors, expanded the indications for allo-HSCT. Relapse remains one of the main causes of mortality after allo-HSCT. Some genetic mutations and karyotype anomalies increase the risk of post-transplant relapse. Preventive treatments for relapse are currently being studied. Treatments such as azacytidine, donor lymphocytes infusions or targeted therapies can be used, prophylactically or preemptively.
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