No Disease Activity in Two-Thirds of Patients With Relapsing-Remitting MS a Decade After Stem Cell Therapy

More than half of patients with relapsing-remitting multiple sclerosis (MS) who underwent autologous hematopoietic stem cell transplantation (aHSCT) experienced improved disability, and nearly two-thirds showed no evidence of disease activity at 10 years, according to results from an observational study published in the Journal of Neurology, Neurosurgery, and Psychiatry.

“Our study corroborates the results observed in the only randomized controlled trial [of aHSCT for relapsing-remitting MS] conducted to date,” wrote corresponding author Joachim Burman, MD, PhD, Uppsala University, Uppsala, Sweden, and study coauthors. “We believe that aHSCT could benefit a greater number of MS patients and should be included as a standard of care for highly active MS.”

The study analyzed data for 174 patients with relapsing-remitting MS in Sweden who were treated with aHSCT between 2004 and 2020. Among patients, the median age at aHSCT was 31 years, the median disease duration was 3.4 years, and 64% were women. Patients had received a median 2 disease-modifying treatments before aHSCT, and almost 2 dozen patients were untreated. At nearly 3 years after aHSCT, 11% of patients received a disease-modifying drug.

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Some 73% of patients had no evidence of disease activity at 5 years after aHSCT, and 65% had no disease activity at 10 years after aHSCT, according to the study. Of 149 patients who had disability at baseline, 54% improved, 37% remained stable, and 9% worsened at their last follow-up appointment.

Adverse events were manageable, researchers reported. With 3 months of aHSCT, the average number of grade 3 adverse events per patient was 1.7, and the average number of grade 4 adverse events per patient was 0.06. The most common adverse event was febrile neutropenia, which affected 68% of patients. There was no evidence of treatment-related mortality.

“In summary,” researchers wrote, “our findings demonstrate that aHSCT for relapsing-remitting MS is feasible within regular health care and can be performed without compromising safety.”

References

Silfverberg T, Zjukovskaja C, Ljungman P, et al. Haematopoietic stem cell transplantation for treatment of relapsing-remitting multiple sclerosis in Sweden: an observational cohort study. J Neurol Neurosurg Psychiatry. Published online September 25, 2023. doi:10.1136/jnnp-2023-331864

Stem cell therapy can safely slow progression of relapsing-remitting MS. News release. BMJ; September 25, 2023. Accessed October 16, 2023.