Study Finds Significant Survival Advantage for Patients With MDS Who Have HLA-Matched Donor for HCT

The abstract, presented at the 2020 American Society of Hematology Annual Meeting and Exposition, found favorable overall survival and leukemia-free survival for these patients.

Compared with patients without a donor, older patients with intermediate-2 (Int-2) or high International Prognostic Scoring System (IPSS) risk de novo myelodysplastic syndrome (MDS) who are candidates for reduced-intensity conditioning hematopoietic cell transplantation (HCT) and have a human leukocyte antigen (HLA)-matched donor have a significant survival advantage, shows a study abstract presented at the 2020 American Society of Hematology Annual Meeting and Exposition.

Allogeneic HCT remains the only curative option for patients with MDS; however, “while transplantation outcomes among selected older patients with MDS are similar to younger patients with MDS, early transplantation for older patients is infrequently offered since the relative benefits of HCT over non-HCT therapy have not been well defined in this patient group,” wrote the abstract authors, who collected data on nearly 400 patients in an attempt to determine the benefit.

The multicenter biologic assignment trial included 260 patients with a donor and 124 without, and after median follow-ups of 34.2 and 26.9 months , respectively, for patients with and without a donor, data showed that patients in the donor arm had significantly improved overall survival, with 47.9% of patients in the donor arm alive at 3 years compared with 26.6% patients in the no-donor arm.

The patients who had a matched donor also saw significantly greater leukemia-free survival at 3 years compared with patients with no donor (35.8% vs 20.6%).

The benefit was consistent across all subgroups, including both patients of Medicare age and younger, leading the researchers to argue that HCT should be offered to all patients between the ages of 50 and 75 years who have Int-2 and high IPSS risk MDS for whom a matched donor can be identified.

All patients were recruited form 34 centers between January 2014 and November 2018, and the 2 cohorts were well balanced for age, gender, Karnofsky performance status, IPSS risk, MDS disease duration, and responsiveness to hypomethylating therapy.

Throughout the trial, there was an overall noncompliance rate of 26.3%, with reasoning including use of myeloablative conditioning or failure to proceed to reduced-intensity conditioning transplant in the donor cohort and the use of alternative donors in the no-donor cohort.


Nakamura R, Saber W, Martens M, et al. A multi-center biologic assessment study comparing reduced intensity allogeneic hematopoietic cell transplantation to hypomethylating therapy or best supportive care in patients aged 50-75 with advanced myelodysplastic syndrome: blood and marrow transplant clinical trials network study 11-2. Presented at: 62nd American Society of Hematology Annual Meeting and Exposition; December 5-8, 2020. Abstract 75.