Evidence map›Paper›PMID 38914938›Full record

ArticleBMC medical research methodology2024

Framework for personalized prediction of treatment response in relapsing-remitting multiple sclerosis: a replication study in independent data.

Anna Maria Sakr, Ulrich Mansmann, Joachim Havla, Begum Irmak Ön, Begum Irmak Ön

Abstract read
In one paragraph

Article in BMC medical research methodology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

4 citing papers in PubMed.

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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

5 authors.

Anna Maria SakrInstitute for Medical Information Processing, Biometry and Epidemiology (IBE), Faculty of Medicine, LMU Munich, Marchioninistrasse 15, Munich, 81377, Germany. anna.sakr@ibe.med.uni-muenchen.de.ORCID 0000-0001-7827-6419
Ulrich MansmannInstitute for Medical Information Processing, Biometry and Epidemiology (IBE), Faculty of Medicine, LMU Munich, Marchioninistrasse 15, Munich, 81377, Germany.ORCID 0000-0002-9955-8906
Joachim HavlaInstitute of Clinical Neuroimmunology, University Hospital, LMU Munich, Marchioninistrasse 15, Munich, 81377, Germany.ORCID 0000-0002-4386-1340
Begum Irmak ÖnInstitute for Medical Information Processing, Biometry and Epidemiology (IBE), Faculty of Medicine, LMU Munich, Marchioninistrasse 15, Munich, 81377, Germany.ORCID 0000-0002-9008-0141
Begum Irmak ÖnInstitute for Medical Information Processing, Biometry and Epidemiology (IBE), Faculty of Medicine, LMU Munich, Marchioninistrasse 15, Munich, 81377, Germany.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundIndividualizing and optimizing treatment of relapsing-remitting multiple sclerosis patients is a challenging problem, which would benefit from a clinically valid decision support. Stühler et al. presented black box models for this aim which were developed and internally evaluated in a German registry but lacked external validation.

methodsIn patients from the French OFSEP registry, we independently built and validated models predicting being free of relapse and free of confirmed disability progression (CDP), following the methodological roadmap and predictors reported by Stühler. Hierarchical Bayesian models were fit to predict the outcomes under 6 disease-modifying treatments given the individual disease course up to the moment of treatment change. Data was temporally split on 2017, and models were developed in patients treated earlier (n = 5517). Calibration curves, discrimination, mean squared error (MSE) and relative percentage of root MSE (RMSE%) were assessed by external validation of models in more-recent patients (n = 3768). Non-Bayesian fixed-effects GLMs were also applied and their outcomes were compared to these of the Bayesian ones. For both, we modelled the number of on-therapy relapses with a negative binomial distribution, and CDP occurrence with a binomial distribution.

resultsThe performance of our temporally-validated relapse model (MSE: 0.326, C-Index: 0.639) is potentially superior to that of Stühler's (MSE: 0.784, C-index: 0.608). Calibration plots revealed miscalibration. Our CDP model (MSE: 0.072, C-Index: 0.777) was also better than its counterpart (MSE: 0.131, C-index: 0.554). Results from non-Bayesian fixed-effects GLM models were similar to the Bayesian ones.

conclusionsThe relapse and CDP models rebuilt and externally validated in independent data could compare and strengthen the credibility of the Stühler models. Their model-building strategy was replicable.

Indexed as

Bayes TheoremMultiple Sclerosis, Relapsing-RemittingPrecision MedicineAdultDisease ProgressionFemaleHumansMaleMiddle AgedRecurrenceRegistriesTreatment OutcomeClinical prediction ruleMultiple sclerosisPrognosisRegistry dataRelapsing-RemittingReproducibility of results

Identifiers

PMID38914938
PMCPMC11194862

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.