09 October 2023

Autologous stem cell treatment is effective in slowing progression of multiple sclerosis, according to a new Swedish study into its use in routine healthcare.

There is growing evidence that autologous haematopoietic stem cell transplantation (aHSCT) is suitable for treating relapsing-remitting MS, the researchers say.

Because aHSCT has yet to be included in most national clinical guidelines, a team of researchers from Uppsala University wanted to assess its safety and effectiveness when used in routine healthcare. Their findings are published in the Journal of Neurology Neurosurgery & Psychiatry.

They identified 231 patients with relapsing-remitting MS, of whom 174 had been treated with aHSCT before 2020. Their average age when treated was 31 and 64% were women.

They analysed data collected from the Swedish MS registry and safety was assessed by scrutinising the patients’ electronic medical records for the 100 days following the procedure.

On average, patients had had their disease for more than three years and had received an average of two lots of disease modifying drugs before aHSCT, although 23 had not had any treatment.

20 patients (11%) were given a disease modifying drug after a median of three years after undergoing aHSCT. Five years after transplant, there was no evidence of disease activity in 73% of those treated, and in 65% after 10 years.

Of the 149 MS patients with some disability to begin with, 54% (80) improved, 37% (55) remained stable, and 9% (14) got worse.

The annualised rate of MS relapse was 1.7 in the year before aHSCT and 0.035 during the monitoring period (an average of 5.5 years). In other words, a patient had, on average, 1.7 relapses per year before aHSCT treatment, and one relapse every 30th year after aHSCT treatment.

Five patients needed intensive care and 61 developed a bacterial infection within 100 days of treatment, with febrile neutropenia being the most common side effect, affecting 68% of patients.

As this is an observational study, with no comparative group, no firm conclusions can be made. However, the researchers write: “Our findings demonstrate that aHSCT is feasible within regular healthcare and can be performed without compromising safety.

“Our study corroborates the results observed in the only randomised controlled trial conducted to date. 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.”


Silfverberg T, Zjukovskaja C, Ljungman P, Nahimi A, Ahlstrand E, Dreimane A, Einarsdottir S, Fagius J, Iacobaeus E, Hägglund H, Lange N, Lenhoff S, Lycke J, Mellergård J, Piehl F, Svenningsson A, Tolf A, Cherif H, Carlson K, Burman J. (2023) “Haematopoietic stem cell transplantation for treatment of relapsing-remitting multiple sclerosis in Sweden: an observational cohort study.” Journal of Neurology Neurosurgery & Psychiatry, doi 10.1136/jnnp-2023-331864

Link: https://jnnp.bmj.com/content/early/2023/08/14/jnnp-2023-331864.long

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