New treatment approaches have increased the number of older patients with myelodysplastic syndromes (MDS) eligible for a stem cell transplant. Now, Dana-Farber research has identified those that are most likely to benefit from one.
In a prospective study involving patients age 60 to 75 with advanced MDS, investigators found that participants at high risk of having their disease worsen fared better with transplant than with other therapies. The same was true for those who underwent a transplant within five months of enrolling in the trial. The results, published in the journal Leukemia, will help older patients and their physicians weigh options for treatment going forward.
“Older people have a higher risk of developing MDS; the median age of diagnosis is 70,” says study first author Gregory Abel, MD, MPH, director of the Older Adult Hematologic Malignancy Program at Dana-Farber. “The only therapy known to cure MDS is stem cell transplantation, but the high-dose chemotherapy or radiation therapy traditionally used to prepare patients for transplant were too risky for many older patients.
“The development of reduced-intensity conditioning regimens opened the possibility of transplant for more older patients with MDS, but it hasn’t been clear how the benefits and risks of transplant apply to them,” he continues. “Our study sought to answer those questions.”
A ‘significant survival benefit’
The study, conducted over a seven-year period, enrolled 290 patients, 175 of whom had adverse disease risk likely to advance to leukemia in a relatively short time — and 115 had standard risk. One hundred thirteen underwent a stem cell transplant after a median of five months, and the patients’ outcomes were tracked for a median of more than three years.
Analysis of the data showed that transplant was associated with a significant survival benefit for patients with adverse disease risk, especially if they received the transplant within five months of being referred to the study.
“Our findings suggest that timely transplant for older adults with MDS who are eligible for transplant because they’re at high risk for worsening disease likely improves survival,” Abel says.
The senior author of the study is Corey Cutler, MD, MPH, medical director of the Adult Stem Cell Transplantation Program and director of Clinical Research, Stem Cell Transplantation. Dana-Farber co-authors are Haesook Kim, PhD, Andrew Hantel, MD, Richard Stone, MD, Vincent Ho, MD, Martha Wadleigh, MD, Daniel DeAngelo, MD, PhD, John Koreth, MBBS, DPhil, Joseph H. Antin, MD, and Robert J. Soiffer, MD.
In Jan 2014 I met Drs. Cutler and Steensma. My MDS had gone from somewhat less than benign to full blown AML. I returned to Dan-Farber in April and was included in a clinical trial which deviated from the standard heavy doses of chemo to what was affectionately called “purple koolaid,” at least by the nurses who saw it administered and followed my progress. After a month of the treatment I was off for a month and then on June 4 the stem cell transplant took place. Ninety five days later I left Boston with a new lease on life. Now, nearly seven years later, I am still here. I have been back to Dana Farber every year since, except during Covid, to thank the staffing nurses and Dr. Cutler and Dr. Steensma. I, and I assume many other transplant patients, feel that we can never adequately express our thoughts, feelings, and gratitude to everyone at Dana-Farber. You went from a team to part of our family. Thank you