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Meta-Analysis
. 2022 Oct;66(4):462-470.
doi: 10.1002/mus.27682. Epub 2022 Aug 6.

Prognostic factors, disease course, and treatment efficacy in Duchenne muscular dystrophy: A systematic review and meta-analysis

Fabio J Weber #  1 , Tsogyal D Latshang #  1   2 , Manuel R Blum  3   4 , Malcolm Kohler  1   5 , Maria M Wertli  3   6
Affiliations
Meta-Analysis

Prognostic factors, disease course, and treatment efficacy in Duchenne muscular dystrophy: A systematic review and meta-analysis

Fabio J Weber et al. Muscle Nerve. 2022 Oct.

Abstract

Introduction/aims: Prognostic factors in Duchenne muscular dystrophy (DMD) predict the disease course and may help individualize patient care. The aim was to summarize the evidence on prognostic factors that may support treatment decisions.

Methods: We searched six databases for prospective studies that each included ≥50 DMD patients with a minimum follow-up of 1 y. Primary outcomes were age at loss of ambulation (LoA), pulmonary function (forced vital capacity percent of predicted, FVC%p), and heart failure.

Results: Out of 5074 references, 59 studies were analyzed. Corticosteroid use was associated with a delayed LoA (pooled effect hazard ratio [HR] 0.42, 95% confidence interval [CI] 0.23-0.75, I2 94%), better pulmonary function tests (higher peak FVC%, prolonged time with FVC%p > 50%, and reduced need for assisted ventilation) and delayed cardiomyopathy. Longer corticosteroid treatment was associated with later LoA (>1 y compared to <1 y; pooled HR: 0.50, 95% CI 0.27-0.90) and early treatment start (aged <5 y) may be associated with early cardiomyopathy and higher fracture risk. Genotype appeared to be an independent driver of LoA in some studies. Higher baseline physical function tests (e.g., 6-minute walk test) were associated with delayed LoA. Left ventricular dysfunction and FVC <1 L increased and the use of angiotensin-converting enzyme (ACE) inhibitors reduced the risk of heart failure and death. Fusion surgery in scoliosis may potentially preserve pulmonary function.

Discussion: Prognostic factors that may inform clinical decisions include age at corticosteroid treatment initiation and treatment duration, ACE-inhibitor use, baseline physical function tests, pulmonary function, and cardiac dysfunction.

Keywords: Duchenne muscular dystrophy; Morbus Duchenne; mortality; outcome; prognostic factor.

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Conflict of interest statement

None of the authors has any conflict of interest to disclose.

Figures

FIGURE 1
FIGURE 1
Study flow
FIGURE 2
FIGURE 2
Influence of corticosteroid treatment and dosing regimen on loss of ambulation main plot (Bello 43 ).
FIGURE 3
FIGURE 3
Sensitivity analysis of influence of corticosteroid treatment and dosing regimen on loss of ambulation. Sensitivity analysis excluding multiple arms in individual studies (Kim late corticosteroid start, Bello and Zhang deflazacort arm).
See this image and copyright information in PMC

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