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Disease-modifying treatment and disability progression in subclasses of patients with primary progressive MS: results
Johannes Lorscheider1,2, Alessio Signori3, Suvitha Subramaniam4
1Department of Neurology, University Hospital Basel, Basel, Switzerland johannes.lorscheider@usb.ch.
Background:
Effectiveness of disease-modifying treatment (DMT) in people affected by primary progressive multiple sclerosis (PPMS) is limited. Whether specific subgroups may benefit more from DMT in a real-world setting remains unclear. Our aim was to investigate the potential effect of DMT on disability worsening among patients with PPMS stratified by different disability trajectories.
Methods:
Within the framework of the Big MS Data network, we merged data from the Observatoire Français de la Sclérose en Plaques, the Swedish and Italian MS registries, and MSBase. We identified patients with PPMS that started DMT or were never treated during the observed period. Subpopulations with comparable baseline characteristics were selected by propensity score matching. Disability outcomes were analysed in time-to-recurrent event analyses, which were repeated in subclasses with different disability trajectories determined by latent class mixed models.
Results:
Of the 3243 included patients, we matched 739 treated and 1330 untreated patients with a median follow-up of 3 years after pairwise censoring. No difference in the risk of confirmed disability worsening (CDW) was observed between the groups in the fully matched dataset (HR 1.11, 95% CI 0.97 to 1.23, p=0.127). However, we found a lower risk for CDW among the class of treated patients with an aggressive disability trajectory (n=360, HR 0.68, 95% CI 0.50 to 0.92, p=0.014).
Conclusions:
In line with previous studies, our data suggest that DMT does not ameliorate disability worsening in PPMS, in general. However, we observed a beneficial effect of DMT on disability worsening in patients with aggressive predicted disability trajectories.
Insights
Disease-modifying treatments (DMTs) show limited effectiveness for primary progressive multiple sclerosis (PPMS). However, DMTs may reduce disability worsening in PPMS patients with aggressive disease trajectories.
Area of Science:
- Neurology
- Clinical Research
- Epidemiology
Background:
- Effectiveness of disease-modifying treatments (DMTs) for primary progressive multiple sclerosis (PPMS) is generally limited.
- Identifying subgroups of PPMS patients who may benefit from DMTs in real-world settings is crucial.
Purpose of the Study:
- To investigate the effect of DMTs on disability worsening in PPMS patients.
- To stratify PPMS patients by disability trajectories to identify potential treatment benefits.
Main Methods:
- Utilized the Big MS Data network, merging data from multiple international MS registries.
- Applied propensity score matching to create comparable groups of treated and untreated PPMS patients.
- Analyzed disability outcomes using time-to-recurrent event analyses within subgroups defined by latent class mixed models.
Main Results:
- No significant difference in confirmed disability worsening (CDW) was observed between treated and untreated PPMS patients overall.
- A reduced risk of CDW was found in treated PPMS patients with an aggressive disability trajectory (HR 0.68, p=0.014).
Conclusions:
- DMTs do not generally ameliorate disability worsening in PPMS, consistent with prior research.
- A subset of PPMS patients, specifically those with aggressive predicted disability trajectories, may benefit from DMTs regarding disability worsening.
