- People with primary or secondary progressive MS who were treated with autologous haematopoietic stem cell transplantation (AHSCT) were compared to those treated with the disease-modifying therapy (DMT) natalizumab over four years.
- There was no difference between the two treatments in terms of disability progression or relapse rate.
- This research found that AHSCT does not prevent or reverse disability progression in people living with progressive MS and does not reduce the rate of relapses.
Evaluating AHSCT for progressive MS: is it the right choice?
Autologous haematopoietic stem cell transplantation (AHSCT) is a powerful treatment that works by resetting the immune system to reduce inflammation in the brain and spinal cord.
It begins with collecting and storing a person’s stem cells, which can grow into new blood and immune cells. Chemotherapy is then used to remove the person’s current immune system, and their stored stem cells are returned to “restart” the immune system. The goal is to replace the harmful immune cells attacking the body with healthier ones.
Few disease-modifying therapies (DMTs) are effective for treating progressive MS, creating a pressing need for better treatments. AHSCT is effective in relapsing-remitting MS with high relapse activity. However, research so far has not established its benefits for progressive MS.
Additionally, AHSCT carries significant risks, so careful consideration is essential before using it as a treatment option.
What did the researchers do?
MS Australia-supported researchers, including Professor Tomas Kalincik, Dr Sifat Sharmin, Dr Izanne Roos (The University of Melbourne, VIC), Dr Jennifer Massey (St Vincent’s Hospital, NSW) along with international colleagues, investigated disability progression and relapse rates in people with primary or secondary progressive MS. The study included 39 people treated with AHSCT and 65 treated with natalizumab.
The data used in this study came from individuals who had either been treated at specialised AHSCT centres in Australia, the UK, Canada, Sweden, Norway or the Czech Republic, or who were registered with the MSBase registry.
To ensure a fair comparison, participants were matched based on their age, gender, disability status, number of relapses, how long they had MS, most effective previous therapy, and their country.
At the start of the study, disability levels in both treatment groups ranged from being able to walk without help or rest for 200m to needing a wheelchair (measured using the Expanded Disability Status Scale, or EDSS).
The study followed participants for four years after starting either AHSCT or natalizumab. Since natalizumab is not effective in controlling disability in progressive MS without relapse activity, it served as a comparison treatment for this study.
What did the researchers find?
Published in the Journal of Neurology, Neurosurgery and Psychiatry, the researchers found that people treated with either AHSCT or natalizumab both experienced worsening disability over time, with no difference between the two treatments. Both groups showed little improvement in disability, and the chances of improvement were similar. Both treatment groups also experienced similar numbers of relapses.
For people treated with AHSCT, most experienced side effects from the treatment. These included febrile neutropenia (fever caused by low levels of a type of white blood cell called neutrophils), serum sickness (a reaction to foreign proteins in the blood) or requiring intensive care. After treatment, some experienced infections caused by a reactivation of viruses.
For people treated with natalizumab, there was not enough information recorded about side effects for the researchers to analyse. There were four deaths among all participants in the study, one in the AHSCT group and three in the natalizumab group, however none were directly related to the treatments.
What does this mean for people with MS?
AHSCT is a very intensive treatment that comes with serious risks, so it is important to carefully evaluate its potential benefits before considering it for a person living with MS.
Natalizumab, a DMT used for treating relapsing-remitting MS, is already known to have a limited impact on progressive MS. This study found that AHSCT has similar outcomes to natalizumab in terms of slowing disability progression and reducing relapses in people with progressive MS. Based on this research, AHSCT is not considered an effective treatment in people with progressive MS who have few relapses.
Anyone who has received AHSCT or who is considering treatment with AHSCT is encouraged to contribute their information to the MS AHSCT Registry. More information can be found here.
