The following article features coverage from the American Society of Hematology 2020 meeting. Click here to read more of Hematology Advisor‘s conference coverage.

Allogeneic hematopoietic cell transplantation (HSCT) should be offered to all patients with high-risk MDS who are aged 50 to 75 years and for whom a suitable donor can be identified, according to a new trial presented at the virtual 62nd American Society of Hematology (ASH) Annual Meeting and Exposition.

This conclusion was based on findings of a multicenter, biologic assignment trial in older persons with high-risk MDS conducted by the Blood and Marrow Transplant Clinical Trials Network (BMT CTN 1102, Identifier: NCT02016781) and presented by Corey Cutler, MD, MPH, FRCPC, of the Dana-Farber Cancer Institute in Boston, Massachusetts.

The study aimed to define the benefit of HSCT over non-HSCT therapy in this patient population because HSCT, which is the only curative treatment in MDS, is infrequently offered to older patients.

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“Recently, [a] cohort analysis demonstrated no survival disadvantage for individuals greater than 65 years of age who underwent transplantation in comparison for those aged 55 to 64,” said Dr Cutler, “[and another] cohort study done here at Dana Farber demonstrated a survival advantage for transplantation amongst among subjects with higher risk MDS.”

Eligible participants were aged 50 to 75 years with higher-risk de novo MDS (IPSS Intermediate-2 [Int-2] or High) and were candidates for reduced-intensity conditioning (RIC) allogeneic HSCT. All patients were initially assigned to the no donor arm. High-resolution HLA typing of eligible family members and a search of the unrelated donor registries (up to 90 days) was conducted for each participant. When a suitable donor (8/8 HLA-matched) was found, the participant was reassigned to the Donor arm. The primary outcome compared between the arms was 3-year overall survival (OS).

Overall, 384 participants were enrolled, 260 in the Donor arm and 124 in the No Donor arm, at 34 centers. Patient and disease characteristics were well balanced across the study arms. For surviving patients, the median follow-up duration was 34.2 months in the donor arm and 26.9 months in the no donor arm. 

The adjusted 3-year OS was significantly higher in the Donor arm (47.9%; 95% CI, 41.3-54.1) than in the No Donor arm (26.6%; 95% CI, 18.4-35.6; P =.0001; absolute difference, 21.3%; 95% CI, 10.2-31.8) in the intent-to-treat analysis. A benefit in 3-year leukemia-free survival (LFS) was also observed (Donor arm LFS, 35.8%; 95% CI, 29.8-41.8 vs No Donor arm, 20.6%; 95% CI, 13.3-29.1; P =.003). These results remained consistent when excluding patients assigned to the No Donor arm who died or withdrew before the 90 days ended (OS, 48.0% vs 28.1%; P =.0004).

The noncompliance rate for the trial was 26.3%. In an as-treated analysis, comparison of the HSCT yielded a significant advantage in 3-year OS over No HSCT (47.4% vs 16.0%, P <.0001) and LFS (39.3% vs. 10.9%; P <.0001) for participants who underwent HSCT, and for 25 patients who underwent alternative donor reduced intensity transplant, 3-year OS and LFS were both 58.5%, highlighting the potential value of alternative donor transplants in this high-risk population.

“[A]mong patients with higher risk MDS aged 50 to 75 years, finding a suitable donor for transplantation is associated with a marked survival advantage, even in those above the age of 65. Accordingly, transplantation should be included as an integral part of MDS management, with early referral to a transplant center for evaluation, and to begin a donor search,” concluded Dr Cutler.

Disclosures: Some authors have declared affiliations with or received funding from the pharmaceutical industry. Please refer to the original study for a full list of disclosures.

Read more of Hematology Advisor’s coverage of the ASH 2020 meeting by visiting the conference page.


Nakamura R, Saber W, Martens MJ, et al.  A multi-center biologic assignment trial 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. Presented at: American Society of Hematology (ASH) 62nd Annual Meeting and Exposition; December 5-8, 2020. Abstract 75.