Autologous hematopoietic stem cell transplantation (AHSCT) is a topic of intense interest among patients. To explain briefly again: Autologous hematopoietic stem cell transplantation (AHSCT) is not about replacement or repair of nerve tissue. This possibility of stem cell therapy is still “in the future”. AHSCT is a method for “resetting” the immune system. The method is considered by experts to be the currently maximum possible anti-inflammatory therapy for MS and is primarily a therapy option for (young) patients with a high inflammatory load. Those who would like more detailed information are once again recommended a DocBlog text from 2019 on Autologous hematopoietic stem cell transplantation.An interesting contribution on this exciting therapy option was presented at the ECTRIMS Meeting 2022 by Tomas Kalincik (Melbourne) on behalf of the MS Base Study Group. I had already introduced the MS Base database in earlier articles. This is “real world” data from several tens of thousands of MS patients, which are often used for the statistical method of propensity score matching. With this method, patients can be identified from the large MS Base data pool who are similar in many parameters but have received different therapies.
For the current analysis, patients who have received an AHSCT were matched with patients from the MS Base register who are treated with highly effective MS therapies (Fingolimod, Ocrelizumab, Natalizumab). The aim of the analysis was to compare the effectiveness of AHSCT with that of highly effective therapies, specifically in terms of reducing relapses and controlling disabilities. In addition, adverse events and treatment-related mortality after AHSCT were evaluated.
The matched pairs had a high mean disease activity of > 0.9 relapses in the past 12 months and a mean EDSS value of 3 -4. Compared to Fingolimod (n = 612), fewer relapses occurred with AHSCT (n = 120) (annual relapse rate: mean ± SD 0.20 ± 0.43 vs. 0.11 ± 0.36) with a similar risk of deterioration of EDSS and higher probability of improvement of disability. Ocrelizumab (n= 303) and AHSCT (n=91) showed a similar annual relapse rate (0.10 ± 0.39 vs. 0.08 ± 0.33), EDSS deterioration and EDSS improvement. Also, with Natalizumab (n = 606) and AHSCT (n = 116), the annual relapse rates were only marginally different (0.12 ± 0.37 vs. 0.09 ± 0.30). With regard to the deterioration of EDSS, both groups were similar, but an improvement in EDSS was observed more frequently after AHSCT compared to Natalizumab.
The authors therefore conclude that AHSCT is superior to Fingolimod in patients with highly active MS with moderate disability, but is comparable to Ocrelizumab and Natalizumab in preventing relapses. No new aspects regarding the side effect profile of AHSCT were revealed, one therapy-associated death after AHSCT was recorded.
Overall, the contribution was controversially discussed as it contradicts the general perception of AHSCT being a therapy option superior to all other measures. In principle, one might be able to justify the results with the methodological problems of a retrospective statistical analysis. On the other hand, the data suggests that AHSCT is not a panacea and that the possibilities of highly effective MS therapies should be consistently considered before use.
Incidentally, a poster from colleagues in Antwerp fits the theme, who analyzed the experiences of their center with patients requesting AHSCT. The analysis shows that most patients who demand AHSCT do not meet the indication for it because there is no longer any clinical or MRI disease activity. The colleagues therefore conclude that much more professional patient education on this important topic must take place and that education should not be left solely to the numerous available emotional case reports.
