New Study Adds to Case for Stem-Cell Transplants as MS Therapy

Stem cell transplantation as a treatment for multiple sclerosis appears to hold up over the long-term.

In a study published Monday in JAMA Neurology, researchers looked at observational data from 281 patients at hospitals across Europe and North America who underwent autologous hemopoietic stem cell transpant (AHST) between 1995 and 2006. They found nearly half (46%) of MS patients who underwent the transplant were alive and had no disease progression after five years.

Paolo A. Muraro, MD, the study’s lead author, said the long-term results are an important addition to the growing canon of research on AHST for the treatment of MS.

“We think it’s important to do long-term studies because that’s what really matters to people with MS,” said Muraro, of the Imperial College of London, in a JAMA audio supplement to the study. “An intervention really should be measured in the long term because short-term outcomes do not always predict long-term outcomes.”

Muraro explained that because it was based on retroactive data, it yielded a number of potentially useful insights.

“This observational study that we published has very broad unselective criteria from multiple centers, thus it reflects the practice at the time, but also gives a lot more information on variables that could not be really assessed in a more controlled trial setting,” he said.

For instance, the study suggested that younger patients fared best when given the stem cell transplant, as did patients who underwent more moderate chemotherapy treatments, rather than high-intensity treatment. Furthermore, patients who had relapsing MS saw a much higher rate of progression-free survival at five years—73%. Those whose disease was progressing at the time of transplantation had only a 33% progression-free survival rate at five years.

The study was not a controlled study, thus there’s no ability to compare the results of patients treatment with AHST to those treated by other therapies. However, Muraro said the evidence appears strong enough to warrant further, large-scale study.

One negative data point in the study was its high mortality rate. Of the nearly 300 patients, eight died within 100 days of treatment.

“I think in many ways what this study demonstrated is sort of the process that went on in terms of what were the best patients to be selected (for transplantation),” said Michael K. Racke, MD, of Ohio State University, in the JAMA audio supplement.

As evidence of the successful refinement of patient selection, Racke noted that three other recent studies, covering more recent transplantations, have consistently shown that about 70% of patients had no evidence of disease activity after five years.

Racke said the study should give neurologists more peace of mind.

“If people are going to refer patients for stem cell transplants I think they want to be fairly confident that the patients are going to have… reduced risk of mortality,” he said.

Muraro’s study was published online Monday (February 20) in JAMA Neurology. The study is titled “Long-Term Outcomes After Autologous Stem Cell Transplantation for Multiple Sclerosis.”

Related Coverage:

Phase 3 Trial Reports Ozanimod Cuts Multiple Sclerosis Relapse Rate

Stem Cell Therapy May Halt Multiple Sclerosis

Drug May Slow Secondary Progressive Multiple Sclerosis