Browse by author
Lookup NU author(s): Professor Volker StraubORCiD
This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License (CC BY-NC-ND).
BACKGROUND AND OBJECTIVES: Development of a prognostic score for time to loss of ambulation (LoA) may help guide treatment of patients with Duchenne muscular dystrophy (DMD). Information from clinical data sources was used to create a prognostic score for LoA for use in practice. METHODS: Data used included 4 natural history databases and 3 clinical trial placebo arms. Inclusion criteria were as follows: (1) 6-minute walk distance ≥75 m; (2) a subsequent visit with an outcome assessment that identified LoA; (3) current corticosteroid use; and (4) available data on prognostic factors. Classification and regression tree models for time to LoA were used to classify patients into 5 risk groups (group 5 = highest risk), prioritizing the easily obtained rise from floor (RFF) and 10-m walk/run (10MWR) times as predictors. The prognostic score was validated in the Cooperative International Neuromuscular Research Group data. RESULTS: Data from 608 boys were analyzed (mean age 9.1 years). Kaplan-Meier curves for time to LoA for each risk group were well separated, describing meaningful differences in time to LoA. Median time to LoA for risk group 1 was not reached during a median of 2.0 years of follow-up. Median times to LoA for risk groups 2-5 were 4.4 (95% CI 4.0-inf), 3.0 (2.1-4.1), 2.0 (1.6-2.1), and 0.9 (0.8-1.6) years, respectively. These risk groups showed concordant times to LoA among 226 patients in the validation data and provided finer separation of patients across different levels of LoA risk than thresholds based on individual functional tests. DISCUSSION: Patients with DMD were classified into 5 risk groups for LoA using a simple, easy-to-use prognostic score based on RFF and 10MWR thresholds. The prognostic score replicated well in validation data and performed better than previously proposed classifications used in practice. Incorporating the prognostic score for time to LoA into a clinical visit may allow clinicians to counsel patients on trial eligibility, need for mobility devices, home adjustments, financial accommodations, and psychosocial needs. The prognostic score may also be used in trials to inform inclusion criteria and stratification. Study limitations included limited data for prediction of LoA risk beyond 4 years.
Author(s): McDonald CM, Signorovitch J, Goemans NM, Mercuri E, Gordish-Dressman H, Vandenborne K, Sajeev G, Ward SJ, Fillbrunn M, Frean M, Servais L, Niks EH, Straub V, De Groot IJM, Muntoni F
Publication type: Article
Publication status: Published
Journal: Neurology
Year: 2026
Volume: 106
Issue: 12
Print publication date: 23/06/2026
Online publication date: 26/05/2026
Acceptance date: 30/01/2026
Date deposited: 23/07/2026
ISSN (print): 0028-3878
ISSN (electronic): 1526-632X
Publisher: American Academy of Neurology
URL: https://doi.org/10.1212/WNL.0000000000214705
DOI: 10.1212/WNL.0000000000214705
Data Access Statement: All relevant aggregate data are reported within the article and the Supporting Information files. This study uses third-party data sources accessed by the cTAP, through data use agreements with the relevant data holders. Individual-level data for 2 of these data sources (PRO-DMD-01 and tadalafil DMD trial) are available on Vivli, a public repository, and can be accessed through requests to the data holders through Vivli. Other data sources used are not available on public repositories and may be available through data use agreements with the individual data holders. Requests for individual patient data may be directed toward the individual institutions/organizations that have collected, curated, and/or hold these patient data. These organizations will consider data requests according to their own data sharing policies and governance.
PubMed id: 42190145
Altmetrics provided by Altmetric
