Is autologous hematopoietic stem cell transplantation (AHSCT) a safe and effective treatment for multiple sclerosis (MS)?

Updated: Oct 08, 2019
  • Author: Christopher Luzzio, MD; Chief Editor: Jasvinder Chawla, MD, MBA  more...
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Autologous hematopoietic stem cell transplantation (AHSCT) may be effective for slowing the course of MS and for repairing damage to the nervous system.

One retrospective study evaluated 281 patients with MS who underwent AHSCT between 1995 and 2006. Almost half of the patients (46%) remained free from neurological progression for 5 years after transplant. However, 8 deaths (2.8%; 95% CI, 1.0%-4.9%) were reported within 100 days of transplant and were considered transplant-related mortality.

It is important to note that patients with a relapsing form of MS fared better than those with progressive MS. This suggests that AHSCT may not necessarily treat a neurodegenerative component of the disease that is operative in the later stages of the disease. [119]

In a trial of 110 patients with relapsing-remitting disease, stem cell therapy helped control MS. After 1 year, just one stem cell recipient had experienced a relapse, versus 39 relapses in drug therapy recipients. Additionally, during a mean follow-up of 3 years, treatment failure occurred in just 6% of stem cell patients, compared with 60% of those on drug therapy. [120]

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