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Stem cell transplant shows promise for MS outcomes

Stem cell transplant shows promise for MS outcomes

Nearly half of a group of people with multiple sclerosis who were treated with autologous haematopoietic stem cell transplantation (AHSCT) survived for at least five years following the transplant without disease progression, UK-led research has found. Authors of the large observational study,* published online in JAMA Neurology, have now called for full randomised controlled trials of AHSCT in the treatment of multiple sclerosis (MS).

Researchers from Imperial College London and colleagues said that AHSCT has already been investigated as a possible treatment for aggressive MS, but they wanted to examine more closely its long-term effects on the course of the disease – which they said affects more than 2.3 million people worldwide.

To do so they analysed data covering 281 people, from 13 countries, who had aggressive forms of MS that had failed to respond to standard therapies, and who had undergone AHSCT between 1995 and 2006. They looked at both MS progression-free survival and overall survival.

Among these patients there were eight deaths reported with 100 days of AHSCT that were considered to be related to the transplant; as MS is not life-threatening, treatment-related deaths would be a major concern. However, the study authors suggested that this 2.8% death rate probably reflected early experience with AHSCT, as they analysed data relating only to patients who had transplants up until the end of 2006. They pointed out that a separate analysis, of European data, found that treatment-related death rates fell dramatically over time as patient selection improved, from 7.3% in transplants for MS performed from 1995-2000 (inclusive) to 1.3% in transplants for MS performed from 2001-2007 (inclusive).

They also reported that five years after AHSCT, MS progression-free survival was 46%; factors associated with better outcomes included younger age; a relapsing form of MS; use of prior immunotherapies; and lower neurological disability scores.

They acknowledged that their study had some limitations, such as its partially retrospective nature, but concluded: “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.”

They added: “Furthermore, our results raise the question whether AHSCT may attenuate the progression of disability in patients with progressive forms of MS, a possibility that is more plausible in patients with MRI evidence of central nervous system inflammatory activity before transplant and that could be addressed in a randomised trial of AHSCT controlled against standard care.”

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