In September 2023, a paper from Sweden was published in the Journal of Neurology Neurosurgery and Psychiatry (JNNP) (Haematopoietic stem cell transplantation for treatment of relapsing-remitting multiple sclerosis in Sweden: an observational cohort study | Journal of Neurology, Neurosurgery & Psychiatry (bmj.com), presenting a retrospective analysis of the use of autologous haematopoietic stem cell transplantation (aHSCT) for the treatment of relapsing-remitting multiple sclerosis (RRMS). Here is more on the results of the Swedish study:
Based on studies available so far, aHSCT is considered an effective measure, especially for highly active MS courses, but is not yet integrated into most national clinical guidelines. Therefore, the aim of the Swedish study was to evaluate the efficacy and safety of implementing aHSCT in routine healthcare.
Stem cell therapy in MS also a big topic in Germany
The analysis included 174 RRMS patients who were treated with aHSCT in Sweden before January 1, 2020. Procedure-related safety was assessed by analyzing data from the period of 100 days after aHSCT. To anticipate this – fortunately, there were no deaths related to the treatment, the safety profile was rated as acceptable by the authors. Regarding the effect, at a mean follow-up time of 5.5 years, 73% of the included patients showed no signs of disease activity. In 54% of patients, an improvement in disability was observed, 37% remained stable, and 9% continued to show deterioration. Given these results, the authors recommend classifying the procedure as standard treatment for patients with highly active RRMS.
Thus, the Swedish study underscores the value of aHSCT for patients with highly active multiple sclerosis, especially since the method now also has an acceptable safety profile. This is also a major issue in Germany – similar analyses are planned and a nationwide initiative of MS centers has been launched to ensure the financing of aHSCT for selected patients. A task force aHSCT has been established by the Competence Network MS (KKNMS), which has already agreed on criteria for the use of aHSCT and will also take care of future financing. For those who want to know more about the current status and which criteria have been defined for the use of aHSCT, the position paper of the task force and the corresponding patient information on the KKNMS homepage are recommended (https://www.kompetenznetz-multiplesklerose.de/).
aHSTC and Ocrelizumab similarly effective
Despite all the euphoria, one must accept that although aHSCT is probably one of the most effective immunotherapies, it does not represent regenerative therapy. This means that younger patients (< 50 years) with still preserved walking ability (EDSS 6) and high disease activity with a short duration of disease (< 10 years) will benefit from the therapy.
In addition, it should also be mentioned that a study was published in JAMA Neurology this summer, which compared the efficacy of aHSCT in highly active MS patients with the efficacy of highly effective MS drugs using a statistical method based on data from the MS Base Register (https://pubmed.ncbi.nlm.nih.gov/37437240/). This study was able to show that aHSCT was significantly more effective in preventing relapses and improving disability than Fingolimod, but only small differences were found when compared with Natalizumab. There was no difference in effectiveness when comparing aHSCT patients with patients who received Ocrelizumab.
Of course, one can criticize the method of the MS Base evaluations – but the study indicates that an overemphasis of aHSCT is certainly out of place and that a consistent treatment with highly effective MS therapies is a sensible strategy for the time being – especially also against the background of good drug safety relative to efficacy so far.
- Latest post on MS and stem cell therapy on MS-Docblog: Neuronal stem cell therapy in MS
- Latest post on MS and stem cell therapy on amsel.de: MS drugs and stem cell therapy similarly effective?
