Results of a new study comparing autologous haematopoietic stem cell transplantation (AHSCT) to various highly efficacious disease-modifying treatments (DMTs) reveal it to be superior to fingolimod and comparable with ocrelizumab and natalizumab therapy in the prevention of relapses.
In AHSCT, patients’ stem cells are harvested and reinfused after high-dose chemotherapy, in an attempt to reset the immune system. From observational, single-arm cohorts, autologous haematopoietic stem cell transplantation (AHSCT) is known to be very effective in MS patients with highly active disease . In the absence of direct, randomised, comparative trials, the presented study compared the effectiveness of AHSCT with fingolimod, ocrelizumab, and natalizumab therapy by emulating a series of pairwise trials . Patient data was collected in 6 centres specialised in AHSCT from 5 different countries, and was paired with patients from the MSBase registry (msbase.org) using fingolimod, ocrelizumab, or natalizumab. The AHSCT and pharmaceutical groups were matched using a propensity score that was based on demographic factors, previous relapses, time since diagnosis, and most effective prior therapy. The main endpoints were annualised relapse rate (ARR), freedom from relapse, and Expanded Disability Status Scale (EDSS) change. Prof. Tomas Kalincik (University of Melbourne, Australia) presented the results.
Matched participants had a mean of >0.9 relapses in the prior year and a mean EDSS of 3–4. Compared with 612 fingolimod users, 120 matched AHSCT participants experienced less relapses (mean ARR 0.20 vs 0.11), relapse risk (HR 0.55; 95% CI 0.37–0.91). The risk of EDSS worsening was not significantly different (HR 0.49; 95% CI 0.16–1.54) and AHSCT patients were more likely to experience disability improvement (HR 2.62; 95% CI 1.46–4.72).
No significant differences were detected between 91 AHSCT and 303 ocrelizumab-treated participants: ARR 0.10 versus 0.08, relapse risk (HR 0.85; 95% CI 0.46–1.56), EDSS worsening (HR 0.41; 95% CI 0.09–1.90), and EDSS improvement (HR 2.31; 95% CI 0.63–8.48). Compared with 606 natalizumab users, 116 AHSCT participants had similar results in ARR (0.12 vs 0.09), relapse risk (HR 0.78; 95% CI 0.40–1.52), and EDSS worsening (HR 0.50; 95% CI 0.09–2.61). AHSCT was however associated with a higher rate of EDSS improvement (HR 1.82; 95% CI 1.19–2.78), despite the fact that natalizumab is known for the reduction of disability in clinical trials.
- Willison AG, et al. J Neurol. 2022;269(7): 3937–3958.
- Kalincik T, et al. Comparative effectiveness of autologous haematopoietic stem cell transplantation vs. fingolimod, ocrelizumab and natalizumab in relapsing-remitting MS. Abstract O019, ECTRIMS 2022, 26–28 October, Amsterdam, the Netherlands.
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Table of Contents: ECTRIMS 2022
Letter from the Editor
ECTRIMS 2022 Highlights Podcast
Diagnosis and Prediction of Disease Course
A case for including optic nerve lesions in the McDonald criteria
Cerebrospinal fluid kappa-free light chains for MS diagnosis
Early, non-disabling relapses increase disability accumulation
Physical impairment is present before perceived MS onset
Chronic active MS lesions respond poorly to anti-CD20 antibodies
Treatment: Trials & Strategies
Dimethyl fumarate reduces the risk of a first clinical event in RIS
How and when to make a timely switch to high-efficacy DMT
Comparing real-world effectiveness of DMTs
Study fails to show non-inferiority of rituximab to ocrelizumab
Autologous haematopoietic stem cell transplantation versus DMTs
Stem cell transplantation not superior to natalizumab in progressive MS
Efficacy of DMTs fades away in secondary progressive MS
Smartphone tapping can help detect progressive MS
Early treatment with DMT effective in paediatric-onset MS
Fingolimod in paediatric MS: results of up to 6 years
Switching treatment after initial platform injectable DMT: real-world data
Pregnancy and infant outcomes in women receiving ocrelizumab
New safety data of anti-CD20 mAbs around pregnancy
MS activity and pregnancy outcomes after long-term use of natalizumab
Ravulizumab significantly reduced relapses in AQP4+ NMOSD
NMOSD patients are cognitively impaired regardless of serostatus
Evidence-based consensus on pregnancy in NMOSD
COVID-19 and MS: lessons learned thus far
Ocrelizumab and fingolimod increase the risk of COVID-19 and of worse outcomes
Humoral and cellular immune responses after SARS-CoV-2 vaccination
Re-myelination strategies in MS still pose many unanswered questions
MS associated with a broader Epstein-Barr virus specific T-cell receptor repertoire
Cognitive rehab and mindfulness reduce cognitive complaints in MS
Update on estimated PML risk related to fingolimod