Stem-cell transplant appears to provide long-lasting benefit for people with multiple sclerosis

Reuters Health – 20/01/2021 – Autologous hematopoietic stem cell transplant (aHSCT) appears to provide lasting benefits for people with multiple sclerosis (MS), according to a retrospective study from Italy.

“Intense immunosuppression followed by aHSCT is highly efficacious in patients with aggressive MS who have failed currently available disease-modifying therapies (DMTs),” Dr. Matilde Inglese, of the University of Genoa, told Reuters Health by email.

“aHSCT is not only efficacious in suppressing disease activity but also in preventing long-term disability and inducing disability improvement. This is a very important milestone in MS treatment,” said Dr. Inglese.

In the journal Neurology, the researchers report outcomes out to 10 years in 210 patients with MS who underwent aHSCT between 1997 and 2019. The cohort included 122 patients with relapsing-remitting MS (RRMS), 86 with secondary progressive MS and two with primary progressive MS.

Overall, 80% of patients had no worsening of their MS disability five years after the transplant. At 10 years, 66% still had not experienced a worsening of disability.

In the RRMS group, 86% and 71% of patients had no worsening of their disability at five and 10 years, respectively. Patients with progressive MS also benefited from aHSCT, with 71% and 57% free of worsening disability at five and 10 years, respectively.

The researchers note that the optimal intensity of the conditioning regimen for the treatment of MS “remains an open question.”

In this cohort, use of the high-intensity BEAM+ATG conditioning protocol was independently associated with “more pronounced suppression of clinical relapses and MRI inflammatory activity, allowing complete disease control in a higher proportion of patients.”

The BEAM+ATG regimen, however, was associated with a higher mortality rate. Three patients (1.4%) died within 100 days of transplant and all had been transplanted with the BEAM+ATG conditioning regimen. There were no transplant-related deaths after 2007.

“As for other available high-efficacy DMTs for MS, early treatment is crucial. Specifically, aHSCT should be the treatment choice in patients with aggressive relapsing-remitting MS soon after the failure of an available high-efficacy DMT,” Dr. Inglese told Reuters Health.

The study was supported by the Italian Multiple Sclerosis Foundation and the Elena Pecci Research Fund.

SOURCE: https://bit.ly/3p4xfCz Neurology, online January 20, 2021.

By Megan Brooks