Evidence map›Paper›PMID 39945205›Full record

ArticleMuscle & nerve2025

Longitudinal Outcomes Among Patients With Duchenne Muscular Dystrophy: A Canadian Retrospective Population-Based Study.

Christina Qian, Alexa C Klimchak, Shelagh M Szabo, Katherine L Gooch, Roxana Dragan, Heather J Prior, Jean K Mah

Abstract read
In one paragraph

Article in Muscle & nerve, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed, 1 pooled it
–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

3 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Article
  3. 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

7 authors.

Christina QianBroadstreet Health Economics & Outcomes Research, Vancouver, British Columbia, Canada.
Alexa C KlimchakSarepta Therapeutics Inc., Cambridge, Massachusetts, USA.
Shelagh M SzaboBroadstreet Health Economics & Outcomes Research, Vancouver, British Columbia, Canada.
Katherine L GoochSarepta Therapeutics Inc., Cambridge, Massachusetts, USA.
Roxana DraganUniversity of Manitoba, Winnipeg, Manitoba, Canada.
Heather J PriorUniversity of Manitoba, Winnipeg, Manitoba, Canada.
Jean K MahDepartment of Pediatrics, Cumming School of Medicine, University of Calgary, Calgary, Alberta, Canada.

Funding

Sarepta Therapeutics Inc.
6 · The paper itself

Abstract

aimsThere are few long-term studies evaluating clinical outcomes and mortality among individuals with Duchenne muscular dystrophy (DMD); particularly using longitudinal health administrative claims data, reflecting populations managed in typical clinical practice. This study aimed to characterize DMD outcomes via a population-based database.

methodsPatients with DMD, diagnosed between 01/1979 and 03/2020 at ≤ 10 years of age, were identified using the Manitoba Population Research Data Repository housed at the Manitoba Centre for Health Policy. De-identified longitudinal administrative data from 1998 to 2020 were used to retrospectively assess frequencies and age at first observation of key DMD outcomes including scoliosis, cardiovascular-related complications, severe respiratory-related morbidities, and mortality. Survival analyses using Kaplan-Meier curves were used to describe attrition and estimate probability of patients remaining observation-free by age.

resultsThis study included 198 patients with median (IQR) follow-up of 9.6 (6.6-15.5) years. Corticosteroid use was observed in 26%, with a mean (SD) percentage of days covered of 31% (39%) from initiation to end of follow-up. Scoliosis observations were captured in 18% (median[IQR] age 12 [11-15] years at first observation), severe respiratory-related morbidities in 20% (14[6.5-18] years), and cardiovascular-related complications in 32% of the cohort (12.5[2-20.5] years). Mortality was observed in 14% of the cohort. Kaplan-Meier curves estimated 15% mortality by age 20 years and 20% by 25 years. DISCUSSION: In a population-based data set with decades of follow-up, these data provide longitudinal observations of the substantial burden of DMD, and insight into contemporary estimates of mortality and treatment patterns in Canada.

Indexed as

Muscular Dystrophy, DuchenneAdolescentCanadaChildChild, PreschoolFemaleHumansKaplan-Meier EstimateLongitudinal StudiesMaleManitobaRetrospective StudiesScoliosisadministrative dataDuchenne muscular dystrophylong‐term outcomesmortalitypopulation‐based study

Identifiers

PMID39945205
PMCPMC12060602

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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.