Comparative effectiveness of disease-modifying therapies for highly active relapsing-remitting multiple sclerosis despite previous treatment – a systematic review and network meta-analysis

Abstract Background Comparative assessments of all available disease-modifying therapies (DMTs) in patients with highly active relapsing-remitting multiple sclerosis (RRMS) are lacking, even though some of these DMTs are restricted to this MS subpopulation. We therefore aimed to compare DMTs in pati...

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Main Authors: Michael Köhler, Friedemann Paul, Kirsten Janke, Sibylle Sturtz, Daniela Preukschat, Sabine Ostlender, Michaela Florina Kerekes, Thomas Kaiser
Format: Article
Language:English
Published: BMC 2025-08-01
Series:BMC Neurology
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Online Access:https://doi.org/10.1186/s12883-025-04338-7
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author Michael Köhler
Friedemann Paul
Kirsten Janke
Sibylle Sturtz
Daniela Preukschat
Sabine Ostlender
Michaela Florina Kerekes
Thomas Kaiser
author_facet Michael Köhler
Friedemann Paul
Kirsten Janke
Sibylle Sturtz
Daniela Preukschat
Sabine Ostlender
Michaela Florina Kerekes
Thomas Kaiser
author_sort Michael Köhler
collection DOAJ
description Abstract Background Comparative assessments of all available disease-modifying therapies (DMTs) in patients with highly active relapsing-remitting multiple sclerosis (RRMS) are lacking, even though some of these DMTs are restricted to this MS subpopulation. We therefore aimed to compare DMTs in patients with highly active RRMS using re-analyses of individual patient data (IPD) provided by study sponsors. Methods We searched for randomised controlled trials (RCTs) that included adult patients with RRMS and directly compared alemtuzumab, cladribine, dimethyl fumarate, fingolimod, natalizumab, ocrelizumab, ofatumumab, ozanimod, ponesimod and teriflunomide, or compared these DMTs with other drugs or placebo. Re-analyses of IPD for subpopulations of patients with high disease activity despite previous DMT were included in network meta-analyses (NMAs). As there is no widely accepted definition of high disease activity in RRMS, criteria were chosen to cover as wide a range of definitions as possible, while being sufficiently similar across studies. Results We identified 14 relevant RCTs, including only 3 head-to-head comparisons of DMTs, and no relevant studies on natalizumab. All studies were pivotal studies for approval. The available re-analyses of IPD did not allow comprehensive NMAs. The main reasons for this were the overall paucity of RCTs, especially head-to-head comparisons, and a high risk of bias. In addition, data on patient-relevant outcomes and long-term follow-up (> 2 years) were lacking. Conclusion Based on the largest possible evidence base, including previously unpublished data, our systematic review shows substantial evidence gaps for DMTs in highly active RRMS. This indicates a need for further research beyond regulatory requirements. Trial registration Clinical trial number: not applicable.
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spelling doaj-art-98fa709ba3af40e098d52dd0bdf6c15f2025-08-20T03:05:09ZengBMCBMC Neurology1471-23772025-08-0125111510.1186/s12883-025-04338-7Comparative effectiveness of disease-modifying therapies for highly active relapsing-remitting multiple sclerosis despite previous treatment – a systematic review and network meta-analysisMichael Köhler0Friedemann Paul1Kirsten Janke2Sibylle Sturtz3Daniela Preukschat4Sabine Ostlender5Michaela Florina Kerekes6Thomas Kaiser7Institute for Quality and Efficiency in Health Care (IQWiG)Experimental and Clinical Research Center (ECRC), Charité– Universitätsmedizin BerlinInstitute for Quality and Efficiency in Health Care (IQWiG)Institute for Quality and Efficiency in Health Care (IQWiG)Institute for Quality and Efficiency in Health Care (IQWiG)Institute for Quality and Efficiency in Health Care (IQWiG)Institute for Quality and Efficiency in Health Care (IQWiG)Institute for Quality and Efficiency in Health Care (IQWiG)Abstract Background Comparative assessments of all available disease-modifying therapies (DMTs) in patients with highly active relapsing-remitting multiple sclerosis (RRMS) are lacking, even though some of these DMTs are restricted to this MS subpopulation. We therefore aimed to compare DMTs in patients with highly active RRMS using re-analyses of individual patient data (IPD) provided by study sponsors. Methods We searched for randomised controlled trials (RCTs) that included adult patients with RRMS and directly compared alemtuzumab, cladribine, dimethyl fumarate, fingolimod, natalizumab, ocrelizumab, ofatumumab, ozanimod, ponesimod and teriflunomide, or compared these DMTs with other drugs or placebo. Re-analyses of IPD for subpopulations of patients with high disease activity despite previous DMT were included in network meta-analyses (NMAs). As there is no widely accepted definition of high disease activity in RRMS, criteria were chosen to cover as wide a range of definitions as possible, while being sufficiently similar across studies. Results We identified 14 relevant RCTs, including only 3 head-to-head comparisons of DMTs, and no relevant studies on natalizumab. All studies were pivotal studies for approval. The available re-analyses of IPD did not allow comprehensive NMAs. The main reasons for this were the overall paucity of RCTs, especially head-to-head comparisons, and a high risk of bias. In addition, data on patient-relevant outcomes and long-term follow-up (> 2 years) were lacking. Conclusion Based on the largest possible evidence base, including previously unpublished data, our systematic review shows substantial evidence gaps for DMTs in highly active RRMS. This indicates a need for further research beyond regulatory requirements. Trial registration Clinical trial number: not applicable.https://doi.org/10.1186/s12883-025-04338-7Multiple sclerosisHigh disease activityData availabilitySystematic review
spellingShingle Michael Köhler
Friedemann Paul
Kirsten Janke
Sibylle Sturtz
Daniela Preukschat
Sabine Ostlender
Michaela Florina Kerekes
Thomas Kaiser
Comparative effectiveness of disease-modifying therapies for highly active relapsing-remitting multiple sclerosis despite previous treatment – a systematic review and network meta-analysis
BMC Neurology
Multiple sclerosis
High disease activity
Data availability
Systematic review
title Comparative effectiveness of disease-modifying therapies for highly active relapsing-remitting multiple sclerosis despite previous treatment – a systematic review and network meta-analysis
title_full Comparative effectiveness of disease-modifying therapies for highly active relapsing-remitting multiple sclerosis despite previous treatment – a systematic review and network meta-analysis
title_fullStr Comparative effectiveness of disease-modifying therapies for highly active relapsing-remitting multiple sclerosis despite previous treatment – a systematic review and network meta-analysis
title_full_unstemmed Comparative effectiveness of disease-modifying therapies for highly active relapsing-remitting multiple sclerosis despite previous treatment – a systematic review and network meta-analysis
title_short Comparative effectiveness of disease-modifying therapies for highly active relapsing-remitting multiple sclerosis despite previous treatment – a systematic review and network meta-analysis
title_sort comparative effectiveness of disease modifying therapies for highly active relapsing remitting multiple sclerosis despite previous treatment a systematic review and network meta analysis
topic Multiple sclerosis
High disease activity
Data availability
Systematic review
url https://doi.org/10.1186/s12883-025-04338-7
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