December 20, 2019
By Marilynn Larkin
NEW YORK (Reuters Health) - A study of hematopoietic stem cell transplantation (HCT) for adults with myelodysplastic syndrome (MDS) showed that patients 65 and older had comparable outcomes to those ages 55 to 64, leading the authors to recommend that the US Centers for Medicare and Medicaid Services cover HCT.
"We have demonstrated the safety of HCT in older patients," Dr. Ehab Atallah of the Medical College of Wisconsin in Milwaukee told Reuters Health by email. "There was no difference in outcomes based on age alone. Outcomes were affected by other factors such as disease stage, other medical problems, and type of transplant. In addition, the study gave and continues to give access to HCT for older patients with MDS."
"Medicare is reviewing data from our study to evaluate the safety of performing HCT in older patients," he said, "and waiting for the results of the BMT CTN 1102 of Allo vs. Hypomethylating/Best Supportive Care in MDS (http://bit.ly/2Zcv83K) to evaluate efficacy compared to other therapies."
"Based on that information," he said, "Medicare will make a final decision."
The study included 688 patients age 65 or older who underwent HCT for MDS; their outcomes were compared with 592 patients ages 55 to 64. The median follow-up was 47 months.
As reported in JAMA Oncology, other than age, there were no differences in patient and disease characteristics between the groups.
On univariate analysis, the three-year non-relapse mortality rate was 28% for the 65 years and older group versus 25% for those ages 55 to 64. The three-year overall survival was 37% for those 65 and older versus 42% for those 55 to 64.
On multivariable analysis, after adjustment for excess risk of mortality in the older group, age had no significant association with overall survival (HR, 1.09) or non-relapse mortality (HR, 1.19).
Further, subsets of the 65 years or older population (i.e., 70 years or older versus 65-69) were also compared in the multivariable models but were not significantly different for any outcomes.
However, a Hematopoietic Cell Transplantation-specific Comorbidity Index (HCT-CI8) score of 4 or higher was independently associated with worse outcomes for overall survival, disease-free survival, non-relapse mortality and graft-versus-host disease, according to the authors.
"These results indicate that chronologic age alone, which is likely a surrogate for other risk factors associated with aging, may not be an appropriate selection factor for HCT," they note. "Physiologic age and functional status, as measured by the HCT-CI, are more relevant indicators of fitness for HCT."
Notably, since approval of Coverage with Evidence (CED) studies such as this one, the number of transplants in patients with MDS older than 65 years has quadrupled, from 96 in 2010 to 361 in 2014, providing access through the CED mechanism, the authors state. "Clearly, insurance coverage was a factor limiting access to HCT in this population."
Dr. Atallah said, "Clinicians should be aware of the safety and feasibility of HCT in older patients, and should refer fit older patients to an HCT center for evaluation."
Dr. Uwe Platzbecker of the University of Leipzig Medical Center in Germany, coauthor of a related editorial, told Reuters Health that in addition to finding that age was not the main variable determining the outcome of HCT, "another lesson learned is that the number of patients transplanted for MDS ages 70 years and older increased greatly during the trial, because reimbursement was covered by Medicare."
"This shows that transplantation is a sensible approach in higher risk MDS also in elderly patients, provided they are otherwise healthy," he said by email. "It also shows that transplanters and patients in the US have been waiting for such a trial - and reimbursement - and are ready to go along that path in the future."
"The trial will also help to convince healthcare authorities around the world deciding over financial coverage for allogeneic transplantation in MDS to extend reimbursement for allogeneic transplantation, regardless of age," he concluded.
SOURCE: http://bit.ly/2PJ5OiI and http://bit.ly/34J3Nam JAMA Oncology, online December 12, 2019.
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