Limited evidence supports many hematopoietic stem cell transplant guidelines

Reuters Health - 19/10/2021 - Guidelines for hematopoietic stem cell transplants often are not supported by randomized clinical trials published in peer-reviewed journals, a new study finds.

Researchers examined how often there was evidence from randomized trials published in peer-reviewed journals to support recommendations laid out by the American Society for Transplantation and Cellular Therapy (ASTCT) 2020 guidelines for indications with standard of care alone or combined with clinical evidence. They focused on research published for both allogenic and autologous transplant.

In total, the study examined 103 ASTCT recommendations for allogenic and autologous transplant. For allogenic transplant, the researchers identified 43 recommendations with indications for standard of care alone and 27 recommendations that also included clinical evidence. For autologous transplant, they found 23 indications based on standard of care alone and 18 combined with clinical evidence. Overall, there were four randomized clinical trials for allogenic transplant and 24 randomized clinical trials for autologous transplant. These trials corresponded with 3 standard of care indications and 1 clinical evidence indication for allogenic transplant, and 11 standard of care and 6 clinical evidence indications for autologous transplant, according to the results in JAMA Internal Medicine.

"Bone marrow transplant is interesting in that likely there are some uses that are valuable, but at the same time, we may use this tool in other settings where there is net harm," said senior study author Dr. Vinayak Prasad of the department of epidemiology and biostatistics at the University of California, San Francisco. Without these trials, there is no good way to tell these situations apart, Dr. Prasad said by email. "Although I knew that some places had weak evidence, I was surprised by just how many recommended uses of bone marrow transplant were supported by weak evidence at best," Dr. Prasad said.

In literature published since 2016, researchers found 299 observational or nonrandomized studies of allogenic transplant, including 208 (70%) single-arm studies. Researchers also found 156 observational or nonrandomized studies of autologous transplant, including 87 (56%) single-arm studies. Since 2016, there have been no randomized clinical trials for allogenic transplant and only four for autologous transplant, the analysis found.

While ethical and feasibility issues may preclude randomized clinical trials in some instances, it is also problematic to offer patients an unproven and aggressive intervention on the basis of poor predicted outcomes, the study team writes. It's possible that physicians might underestimate the burden or harms of treatment or related complications, the researchers also note. In addition, clinicians may mistakenly equate feasibility or response rates with better survival options or cure rates, the study team points out.

Doctors and patients should have honest conversations about what is known and not known about bone marrow transplant for the condition being treated, Dr. Prasad said. Beyond this, physicians should tell patients if they're recommending the procedure because it's been proven to improve survival, or if it's just something that's been done for a long time. "People should embark on a bone marrow transplant only after knowing all the options and getting all the facts," Dr. Prasad said. "Doctors and researchers should develop a robust research agenda to sort out which of these uses of bone marrow transplant save lives and which may not."

SOURCE: https://bit.ly/3AUOhbl JAMA Network Open, online September 7, 2021.

By Lisa Rapaport



Posted on 19 October 202127 October 2021