Evidence map›Paper›PMID 27682227›Full record

Trial reportMultiple sclerosis (Houndmills, Basingstoke, England)2017

Baseline EDSS proportions in MS clinical trials affect the overall outcome and power: A cautionary note.

Guoqiao Wang, Gary R Cutter, Stacey S Cofield, Fred Lublin, Jerry S Wolinsky, Tarah Gustafson, Stephen Krieger, Amber Salter

Abstract readClinical Trial, Phase IIIMulticenter StudyRandomized Controlled Trial
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Multiple sclerosis (Houndmills, Basingstoke, England), 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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

2 citing papers in PubMed.

  1. Effectiveness of Ocrelizumab in Primary Progressive Multiple Sclerosis: a Multicenter, Retrospective, Real-world Study (OPPORTUNITY).Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2023
    Article
  2. Article
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

8 authors.

Guoqiao WangDivision of Biostatistics, Washington University in St. Louis, St. Louis, MO, USA.
Gary R CutterDepartment of Biostatistics, The University of Alabama at Birmingham, Birmingham, AL, USA.
Stacey S CofieldDepartment of Biostatistics, The University of Alabama at Birmingham, Birmingham, AL, USA.
Fred LublinIcahn School of Medicine at Mount Sinai, New York City, NY, USA.
Jerry S WolinskyMcGovern Medical School, The University of Texas Health Science Center at Houston, Houston, TX, USA.
Tarah GustafsonIcahn School of Medicine at Mount Sinai, New York City, NY, USA.
Stephen KriegerIcahn School of Medicine at Mount Sinai, New York City, NY, USA.
Amber SalterDivision of Biostatistics, Washington University in St. Louis, St. Louis, MO, USA.

Funding

Extension of Combination Therapy for Multiple Sclerosis (eCombiRx)U01NS045719 · NINDS · MOUNT SINAI SCHOOL OF MEDICINE OF NYU · PI LUBLIN, FRED D · 2003 to 2012
$44.7M
NINDS NIH HHS U01 NS045719
6 · The paper itself

Abstract

backgroundIn randomized clinical trials, when treatments do not work equally effectively across stratifications of participants, observed event rates may differ from those hypothesized leading to deviations in estimated power.

objectivesTo investigate the effect of distributions of baseline Expanded Disability Status Scale (EDSS) proportions in relapsing-remitting multiple sclerosis (RRMS) on the trial outcome, confirmed disability progression rate (CDPR), and power.

methodsWe reported CDPRs in the CombiRx trial by baseline EDSS and by groups (1st (0, 1), 2nd (1.5, 2), 3rd (2.5, 3), and 4th (⩾3.5)) and investigated the effect of different combinations of baseline EDSS proportions on the trial outcome and power.

resultsThere were 244 (25.4%) participants in the 1st group, 368 (38.4%) in the 2nd group, 223 (23.3%) in the 3rd group, and 124 (12.9%) in the 4th group with CDPRs of 40.1%, 13.9%, 11.2%, and 16.9%, respectively. Both CDPR and power increased when the proportion of the 1st group increased in hypothetical trials with equal sample sizes in each arm, and a 10% increase in the 1st group led to a 5% increase in power.

conclusionVarious baseline EDSS proportions yielded different CDPRs and power, suggesting caution in interpretation of treatment effects across trials that enrolled participants with different proportions of baseline EDSS.

Indexed as

Disability EvaluationResearch DesignAdolescentAdultData Interpretation, StatisticalDisease ProgressionDouble-Blind MethodDrug Therapy, CombinationEndpoint DeterminationFemaleGlatiramer AcetateHumansImmunosuppressive AgentsInterferon beta-1aMaleMiddle AgedGlatiramer AcetateImmunosuppressive AgentsInterferon beta-1aconfirmed disability progression rateExpanded Disability Status ScaleMultiple sclerosisrelapsing-remitting multiple sclerosis

Identifiers

PMID27682227
PMCPMC5344761

What OpenQuestion holds

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Registered trials

None linked

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.