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Autologous hematopoietic stem cell transplantation (aHSCT) demonstrated long-term effectiveness in treating aggressive multiple sclerosis (MS), a small retrospective study showed.

Among 71 patients with aggressive MS who received aHSCT, no new relapses were recorded over a follow-up period that ranged from 8 months to 20 years, reported Francis Brunet, MD, of the University of Ottawa in Canada, at ACTRIMS Forum 2022, the annual meeting of the Americas Committee for Treatment and Research in Multiple Sclerosis.

Pre-transplant, the average annualized relapse rate was 1.39 per year. "We didn't see any patient who had a new relapse after the transplant," Brunet observed. "This is really significant in terms of efficacy."

The goal of aHSCT is to reset the immune system, Brunet said. "aHSCT is increasingly used to treat patients with aggressive multiple sclerosis in our center in Ottawa and elsewhere," he noted.

The observational study included 71 MS patients who had a mean age at transplant of 32.5 and mean time from diagnosis to transplant of 5.6 years. A total of 41 participants (58%) were women. Most of the sample (85%) had relapsing-remitting MS, and 15% had secondary progressive disease.

The median baseline Expanded Disability Status Scale () score was 4 (range 1.5-6.5). Nearly all participants received one or more disease-modifying treatments before the transplant.

Prior to aHSCT, EDSS scores were increasing, signaling worsening disability over time. After aHSCT, the rate of change in EDSS was slightly negative: for many patients, disability remained stable or improved minimally after the transplant.

Disability outcomes were better among those with lower baseline EDSS scores. Of 30 people with EDSS scores of 4 or lower, 62% had an improvement in disability, mostly mild, after transplant. People with a baseline EDSS score of 4 or lower were two times as likely to improve following aHSCT compared with people whose baseline EDSS score was over 4 (95% CI 1.3-3.2).

"Proper patient selection is important, with better results seen in patients who have acquired less disability at the time of aHSCT," Brunet pointed out.

Only 33 people had MRI data available; their scans showed no new lesions after transplant.

Three patients died: one from sepsis and liver disease 27 days after transplant, one from pneumonia 67 months after transplant, and one from sudden death 18 years after transplant. "Mortality -- 4.2% -- is similar to other reported studies," Brunet said.

Limitations to the study included missing data from some patients over the follow-up period, he noted. In addition, the analysis did not assess possible long-term autoimmune, metabolic, cardiovascular, or cancer effects after the transplant.

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