Transplant or No Transplant for Older, Fit Patients With MDS?

Hematopoietic cell transplantation (HCT) with reduced-intensity conditioning improved overall survival (OS) in older patients with advanced myelodysplastic syndromes (MDS), compared with OS in patients who did not undergo transplant, according to a non-randomized report from the Myelodysplastic Dysplastic Syndromes Transplant-Associated Outcomes (MDS-TAO) study.

The results, which were presented at the 2018 ASH Annual Meeting by Gregory A. Abel, MD, MPH, from Dana-Farber Cancer Institute in Boston, suggest that transplant should be considered for older, fit patients.

This observational study included patients between the ages of 60 and 75 with advanced MDS or chronic monomyelocytic leukemia who presented at Dana-Farber. Patients who were considered eligible for HCT, met the study inclusion criteria (int-2/high disease per the International Prognostic Scoring System [IPSS], poor-risk cytogenetics, or the severe cytopenia/platelet or red blood cell transfusion–dependence), and were willing to undergo transplant were enrolled in the trial.

Between May 2011 and May 2018, 290 patients were enrolled. 24 patients had fewer than 90 days of follow-up and were excluded from the final analysis.

The median age among the remaining 266 patients was 69 (range = 60-75 years), and most had favorable-risk cytogenetics (53%). Most patients had either int-1 or int-2 disease (44% and 36%, respectively).

A total of 102 participants underwent transplant (; range not reported) and 164 underwent treatment without HCT. The researchers found that patients who had higher-risk disease (int-2/high on the IPSS) at baseline were more likely to undergo HCT (65% vs. 35%; p=0.0003), after adjusting for age, gender, Eastern Cooperative Oncology Group (ECOG) performance status, IPSS stage and cytogenetics, and year of trial consent.

The study used a landmark analysis at five months to estimate OS among patients who were alive for at least five months and received HCT, versus patients who did not (landmark cohort).

During the study observation period, 45 patients in the HCT group and 98 in the non-HCT group had died, leaving a final cohort of 123 surviving patients.

The median follow-up among survivors was 31 months (range = 1.9-84 months), and the median follow-up for patients alive and not yet transplanted (n=66) was 24 months (range = 1.9-82 months).

In a multivariable analysis, patients who underwent transplant had a significantly lower risk of death (hazard ratio [HR] for OS = 0.63; 95% CI 0.42-0.96; p=0.03). The investigators evaluated other patient characteristics (disease stage, ECOG score, age, gender, year of consentand found that only poor-risk cytogenetics were associated with worse OS (HR=1.86; 95% CI 1.15-3.00; p=0.006);

“In this [study] of fit elderly patients with advanced MDS, a treatment strategy that included HCT was associated with better OS,” the authors concluded. The study’s findings are limited by the lack of a randomized design, the potential for selection bias, and the researchers noted that subgroup analyses to evaluate whether molecular characteristics or other disease factors affect survival outcomes are ongoing.

The authors report with H3 Biosciences, Celgene, Arog, AbbVie, Agios, Novartis, ARIAD, Blueprint Medicines, GlycoMimetics, Millennium, Prometheus, and Miltenyi Biotech.


Abel GA, Kim HT, Steensma DP, et al. Survival after hematopoietic stem cell transplantation in an older, fit cohort of patients with advanced myelodysplastic syndromes: the MDS-TAO study. Abstract #972. Presented at the 2018 ASH Annual Meeting, December 3, 2018; San Diego, CA.