Applying Propensity Score Methods in Clinical Research in Neurology

Austin, Peter C.; Yu, Amy; Vyas, Manav V.; Kapral, Moira K.

doi:10.1212/wnl.0000000000012777

Cited by 56 publications

(43 citation statements)

References 59 publications

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“…The obtained propensity scores were subsequently entered in an Average Treatment effect on the Treated weighting model to provide a balanced sample of patients except for their respective treatment. Due to differences in sample sizes, we preferred an optimal full matching approach as to avoid a selection of patients to remain unmatched 16. As previously described,16 17 the balance between the two groups was assessed by comparing the standardised mean differences of the covariates before and after propensity score adjustment.…”

Section: Methodsmentioning

confidence: 99%

“…Due to differences in sample sizes, we preferred an optimal full matching approach as to avoid a selection of patients to remain unmatched 16. As previously described,16 17 the balance between the two groups was assessed by comparing the standardised mean differences of the covariates before and after propensity score adjustment. Using a model of optimal full matching, we achieved standardised mean differences for the selected covariates below 0.1 indicating adequate balance of the two groups (online supplemental figure 1).…”

Section: Methodsmentioning

confidence: 99%

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Eculizumab versus rituximab in generalised myasthenia gravis

Nelke

Schroeter

Stascheit

et al. 2022

J Neurol Neurosurg Psychiatry

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ObjectiveMyasthenia gravis (MG) is the most common autoimmune disorder affecting the neuromuscular junction. However, evidence shaping treatment decisions, particularly for treatment-refractory cases, is sparse. Both rituximab and eculizumab may be considered as therapeutic options for refractory MG after insufficient symptom control by standard immunosuppressive therapies.MethodsIn this retrospective observational study, we included 57 rituximab-treated and 20 eculizumab-treated patients with MG to compare the efficacy of treatment agents in generalised, therapy-refractory anti-acetylcholine receptor antibody (anti-AChR-ab)-mediated MG with an observation period of 24 months. Change in the quantitative myasthenia gravis (QMG) score was defined as the primary outcome parameter. Differences between groups were determined in an optimal full propensity score matching model.ResultsBoth groups were comparable in terms of clinical and demographic characteristics. Eculizumab was associated with a better outcome compared with rituximab, as measured by the change of the QMG score at 12 and 24 months of treatment. Minimal manifestation of disease was more frequently achieved in eculizumab-treated patients than rituximab-treated patients at 12 and 24 months after baseline. However, the risk of myasthenic crisis (MC) was not ameliorated in either group.InterpretationThis retrospective, observational study provides the first real-world evidence supporting the use of eculizumab for the treatment of refractory, anti-AChR-ab positive MG. Nonetheless, the risk of MC remained high and prompts the need for intensified monitoring and further research effort aimed at this vulnerable patient cohort.

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Section: Methodsmentioning

confidence: 99%

Section: Methodsmentioning

confidence: 99%

Eculizumab versus rituximab in generalised myasthenia gravis

Nelke

Schroeter

Stascheit

et al. 2022

J Neurol Neurosurg Psychiatry

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“…Propensity score matching (PSM), a reliable method of decreasing potential bias in large cohorts with multiple confounders, was conducted with a 2:1 matching of the logit of the propensity score using the nearest neighbor (greedy type) matching and 0.2 caliper width 10. Matching was performed without replacement, and unpaired patients not meeting the matching criteria were excluded.…”

Section: Methodsmentioning

confidence: 99%

Endovascular thrombectomy beyond 24 hours from ischemic stroke onset: a propensity score matched cohort study

Dhillon

Butt

Podlasek

et al. 2022

J NeuroIntervent Surg

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BackgroundThe safety and functional outcome of endovascular thrombectomy (EVT) in the very late (VL; >24 hours) time window from ischemic stroke onset remains undetermined.MethodsUsing data from a national stroke registry, we used propensity score matched (PSM) individual level data of patients who underwent EVT, selected with CT perfusion or non-contrast CT/CT angiography, between October 2015 and March 2020. Functional and safety outcomes were assessed in both late (6–24 hours) and VL time windows. Subgroup analysis was performed of imaging selection modality in the VL time window.ResultsWe included 1150 patients (late window: 1046 (208 after PSM); VL window: 104 (104 after PSM)). Compared with EVT treatment initiation between 6 and 24 hours, patients treated in the VL window had similar modified Rankin Scale (mRS) scores at discharge (ordinal shift; common OR=1.08, 95% CI 0.69 to 1.47, p=0.70). No significant differences in achieving good functional outcome (mRS ≤2 at discharge; 28.8% (VL) vs 29.3% (late), OR=0.97, 95% CI 0.58 to 1.64, p=0.93), successful reperfusion (modified Thrombolysis in Cerebral Infarction score of 2b–3) (p=0.77), or safety outcomes of symptomatic intracranial hemorrhage (p=0.43) and inhospital mortality (p=0.23) were demonstrated. In the VL window, there was no significant difference in functional outcome among patients selected with perfusion versus those selected without perfusion imaging (common OR=1.38, 95% CI 0.81 to 1.76, p=0.18).ConclusionIn this real world study, EVT beyond 24 hours from stroke onset or last known well appeared to be feasible, with comparable safety and functional outcomes to EVT initiation between 6 and 24 hours. Randomized trials assessing the efficacy of EVT in the VL window are warranted, but may only be feasible with a large international collaborative approach.

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“…Optimal use of these invaluable tools for comparative effectiveness research in MS relies on their most appropriate implementation. While guidelines on PS methods have been published in neurology 7 and in other disease areas, [74][75][76] reaching the MS research community with recommendations tailored to the field was urgently needed. These guidelines provide the necessary practical tools to ensure continuous improvements in the quality of MS research.…”

Section: Discussionmentioning

confidence: 99%

Recommendations for the use of propensity score methods in multiple sclerosis research

Simoneau¹,

Pellegrini²,

Debray

et al. 2022

Mult Scler

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Background: With many disease-modifying therapies currently approved for the management of multiple sclerosis, there is a growing need to evaluate the comparative effectiveness and safety of those therapies from real-world data sources. Propensity score methods have recently gained popularity in multiple sclerosis research to generate real-world evidence. Recent evidence suggests, however, that the conduct and reporting of propensity score analyses are often suboptimal in multiple sclerosis studies. Objectives: To provide practical guidance to clinicians and researchers on the use of propensity score methods within the context of multiple sclerosis research. Methods: We summarize recommendations on the use of propensity score matching and weighting based on the current methodological literature, and provide examples of good practice. Results: Step-by-step recommendations are presented, starting with covariate selection and propensity score estimation, followed by guidance on the assessment of covariate balance and implementation of propensity score matching and weighting. Finally, we focus on treatment effect estimation and sensitivity analyses. Conclusion: This comprehensive set of recommendations highlights key elements that require careful attention when using propensity score methods.

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Applying Propensity Score Methods in Clinical Research in Neurology

Cited by 56 publications

References 59 publications

Eculizumab versus rituximab in generalised myasthenia gravis

Eculizumab versus rituximab in generalised myasthenia gravis

Endovascular thrombectomy beyond 24 hours from ischemic stroke onset: a propensity score matched cohort study

Recommendations for the use of propensity score methods in multiple sclerosis research

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