A blood test and basic information about the medical status of patients with myelodysplastic syndrome (MDS) can predict stem cell transplant success and guide doctors to choose the most effective course of pretransplant therapy.1

Researchers from the Dana-Farber Cancer Institute, Brigham and Women’s Hospital, and the Center for International Blood and Marrow Transplant Research found that by genetically profiling patient blood cells and factoring in patient age amongst other factors, they can predict the likelihood of stem cell transplant success and recommend the intensity of pretransplant chemotherapy and/or radiation therapy for individual patients. The study included blood samples from 1514 patients with MDS, from age 6 months to older than 70 years.

Patients with MDS produce insufficient amounts of healthy blood cells. “Although donor stem cell transplantation is the only curative therapy for MDS, many patients die after transplantation, largely due to relapse of the disease or complications relating to the transplant itself,” said R. Coleman Lindsley, MD, PhD, at Dana-Farber and the study’s lead researcher.

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The most influential factor determining transplant success was a mutation in the TP53 gene that decreased survival times and caused relapse, regardless of whether the patient received standard conditioning therapy or reduced intensity conditioning therapy. Patients 40 years or older without TP53 mutations, but with mutations in the RAS pathway or in the JAK2 gene also had shorter survival times, but only relapsed if they received reduced intensity conditioning.

“Improving our ability to identify patients who are the most likely to have a relapse or to experience life-threatening complications from a transplant could lead to better pretransplant therapies and strategies for preventing relapse,” said Lindsley. Indeed, doctors at Dana-Farber are working on strategies to treat patients with TP53 mutations and suggest that higher intensity pretransplant conditioning be considered for patients with mutations in the RAS pathway or the JAK2 gene.


1. Lindsley RC, Saber W, Brenton G, et al. Prognostic mutations in myelodysplastic syndrome after stem-cell transplantation. N Engl J Med. 2017 Feb 9. doi: 10.1056/NEJMoa1611604 [Epub ahead of print]