New Recommendations Open Door a Bit to Stem Cell Transplant for MS

The procedure might work best in patients who have had multiple sclerosis (MS) for less than 10 years, the report said.

Patients younger than 50 years of age with a shorter duration of multiple sclerosis might be considered candidates for an autologous hematopoietic stem cell transplant (AHSCT).

Earlier this week, the National Medical Advisory Committee of the National Multiple Sclerosis Society published recommendations in JAMA Neurology about this issue based on current research.

However, the committee also said further research is needed and said some issues remain unresolved. The committee also said the procedure should only be performed at centers with substantial experience and expertise.

The committee concluded that AHSCT may be a useful treatment option for patients with relapsing MS who demonstrate substantial breakthrough disease activity despite treatment with high-efficacy disease-modifying therapy (DMT) or who have contraindications to high-efficacy DMTs.

They also said that AHSCT might work optimally in people who have had the disease for less than 10 years.

The recommendations note that previous research studies have concluded that the likelihood of benefit from AHSCT is much smaller for patients with progressive MS without recent disease activity. In addition, patients who are older and have greater disability have a greater risk for serious complications or death associated with the procedure.

Unanswered questions include how long disease duration should be; how much disease activity an appropriate candidate should have; if clinical activity should be required; and whether a patient presenting with very active disease should turn to AHSCT as a first-line therapy, among others.

Only a few centers offer the adequate amount of expertise and quality assurance oversight to perform AHSCT. In addition to needing accreditation, centers performing AHSCT for MS should have transplant teams that include not only hematologist-oncologists with extensive experience in AHSCT, but also neurologists with expertise in MS diagnosis and treatment.

The authors also noted that it is appropriate to weigh the costs between AHSCT and drug therapy when considering treatment options. The estimated cost for AHSCT is approximately $150,000 for 1-time treatment. DMTs cost about $80,000 or more a year.

A version of this story originally published on NeurologyLive. For more, see here.


Miller AE. Chitnis T, Cohen BA, Costello K, Sicotte NL, Stacom R. Autologous hematopoietic stem cell transplant in multiple sclerosis. JAMA Neurol. Published online October 26, 2020. doi: 10.1001/jamaneurol.2020.4025

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