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Study Sets Guidelines for Stem Cell Transplants in Older Patients with Myelodysplastic Syndromes

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Key Points

  • In the study, patients with higher-risk myelodysplastic syndrome who received reduced-intensity conditioning allogeneic stem cell transplantation had an average life expectancy of 36 months, better the 28 months for those receiving non-transplant therapies.
  • Patients in the lower-risk groups who underwent transplant lived an average of 38 months after treatment, less than the 77 months for those who were treated without transplant.
  • The study provides the first statistically based guidelines for determining whether stem cell transplant is appropriate for older patients with myelodysplastic syndromes.

A new study by an international team of scientists provides the first statistically based guidelines for determining whether a stem cell transplant is appropriate for older patients with myelodysplastic syndromes (MDS), the most common blood disorders in people over 60 years of age, and frequently a precursor for leukemia.

Using mathematical models to analyze hundreds of MDS cases from around the world, the researchers found reduced-intensity conditioning allogeneic hematopoietic stem cell transplants are advisable for patients aged 60 to 70 who have higher-risk forms of MDS that are likely to turn into leukemia in the near future. For patients with lower-risk MDS, nontransplant treatments are preferable, the model indicates.

The research was published online in the Journal of Clinical Oncology.

Questions about Transplantation in Older Patients

“Our study helps inform older MDS patients and their doctors whether a stem cell transplant is preferred or whether it makes more sense to pursue other options,” said John Koreth, MBBS, DPhil, medical oncologist in the Division of Hematologic Malignancies at Dana-Farber Cancer Institute, who is the study’s lead author and co-principal investigator. “Until now, there haven’t been statistically quantified guidelines for making these decisions for older patients, who are most impacted by the disease.”

The only potentially curative treatment for MDS is allogeneic stem cell transplant, which can, in principle, be used for patients with any stage of MDS. But because even reduced-intensity transplants are fatal in a significant minority of cases, there has been some uncertainty over the use of transplantation for older patients with MDS.

Study Details

“It hasn’t been clear for which older patient groups the benefits of transplant outweigh the risks,” Dr. Koreth said. To find out, researchers collected data on 514 patients, aged 60 to 70, who were newly diagnosed with MDS. Patients were stratified by International Prognostic Scoring System (IPSS) risk. For both lower- and higher-risk groups, the researchers built separate mathematical models to compare treatment outcomes in patients who received reduced-intensity allogeneic stem cell transplants with outcomes in patients who received nontransplant therapies. They analyzed not only length of survival but also the quality of life of patients in those groups.

Patients in the lower-risk groups (low/intermediate-1 IPSS MDS) who underwent transplant lived an average of 38 months after treatment, less than the 77 months for those who were treated without transplant. For patients in the higher-risk groups (ntermediate-2/high IPSS MDS), by contrast, average life expectancy was 36 months for those receiving transplants, better than the 28 months for those receiving nontransplant therapies. Adjusting for patients’ quality of life did not change the conclusions regarding the relative merits of the treatments.

“The clear result is that, on balance, reduced-intensity stem cell transplantation offers a survival benefit for patients with higher-risk MDS, but not for those with lower-risk disease,” Dr. Koreth said. “The findings should offer useful guidance for older patients with MDS on deciding the best course of treatment.”

Dr. Koreth is a Leukemia and Lymphoma Society Scholar in Clinical Research. Dr. Cutler is supported by the Stem Cell Cyclists of the Pan Mass Challenge.

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.


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