Hematopoietic stem cell transplant (HCT) treatment of myelodysplastic syndromes (MDS) resulted in a lower risk of death if performed early after diagnosis or among patients with high-risk disease, according to results of an observational study published in Leukemia.

HCT is the only potentially curative treatment for MDS. However, the disease primarily occurs among older patients in whom myoablative conditioning regimens are associated with an increased risk of mortality. The aim of this study was to evaluate mortality after transplant in a real-world population of patients with MDS.

The prospective, observational study included 290 patients with advanced MDS aged 60 to 75 years from the MDS Transplant-Associated Outcomes study who were determined to be fit to undergo HCT. All patients had disease that warranted consideration of HCT, including patients with intermediate- to high-risk disease or those with standard risk and severe cytopenia. The primary endpoint was overall survival (OS).

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At baseline, the median age was 69 years, and the majority of patients had intermediate-risk disease (78%) and good cytogenetic risk (53%). Mutations in TP53, JAK2, or in the RAS pathway were either not present or unknown in most patients.

HCT was performed in 113 patients (39%) with a median time to HCT of 5 months (range, 1-58 months).

The median OS of the entire cohort was 29 months (95% CI, 19-39.5 months), which translated to a 3-year OS of 46%. Overall, there was no difference in the risk of death between the cohort who underwent HCT and those who did not (hazard ratio [HR], 0.75; 95% CI, 0.52-1.09; P =.13), in a multivariate analysis. However, landmark analyses showed a survival benefit with HCT at 5 (P =.04) and 9 months (P =.01), but not at 12 months or 24 months.

HCT performed earlier, within 5 months from study entry, was significantly associated with a lower risk of death than transplants performed later (HR, 0.53; 95% CI, 0.33-0.83; P =.006). HCTs performed after 5 months did not result in a survival benefit between the HCT and non-HCT cohorts.

Transplant also lowered the risk of death among patients with adverse-risk disease compared with patients with standard-risk MDS with severe cytopenia (HR, 0.57; 95% CI, 0.37-0.88; P =.01).

Other characteristics associated with a survival benefit with HCT included patients with poor cytogenetic risk, an Eastern Cooperative Oncology Group performance status of 1, and patients of male sex.

The authors concluded that these data showed that “there were also significant benefits for those receiving HCT within 5 months and for those with adverse disease risk factors as compared to standard risk with severe cytopenias.”


Abel GA, Kim HT, Hantel A, et al. Fit older adults with advanced myelodysplastic syndromes: who is most likely to benefit from transplant? Leukemia. Published November 17, 2020. doi:10.1038/s41375-020-01092-2