Long-term outcomes following stem cell transplant for multiple sclerosis published in JAMA


A new study published online by JAMA Neurology examines the long-term outcomes of patients with aggressive forms of multiple sclerosis who failed to respond to standard therapies and who underwent autologous hematopoietic stem cell transplantation using their own stem cells.

Autologous haematopoietic stem cell transplantation (AHSCT) has been investigated as a treatment for aggressive multiple sclerosis, the rationale for which is immune reconstitution. It is important to examine the course of multiple sclerosis after AHSCT over the long term.

The study, by Paolo A Muraro of Imperial College, London, UK, and coauthors, included data from 13 countries on 281 patients who underwent AHSCT between 1995 and 2006. Primary outcomes examined by the study were multiple sclerosis progression-free survival and overall survival.

Eight deaths (2.8%) were reported within 100 days of transplant and were considered transplant-related. The authors suggest the 2.8% death rate in the current study likely reflects the early experience with AHSCT because only transplants performed through 2006 were included.

Additionally, multiple sclerosis progression-free survival was 46% at five years after AHSCT, with younger age, a relapsing form of multiple sclerosis, use of fewer prior immunotherapies and lower neurological disability scores associated with better outcomes, according to the report.

The authors note some study limitations.

“In this large observational study of patients with MS treated with AHSCT, almost half of them remained free from neurological progression for five years after transplant. … The results support the rationale for further randomised clinical trials of AHSCT for the treatment of MS,” the article concludes.