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Lookup NU author(s): Dr Marianela Schiava, Dr John Bourke, Meredith JamesORCiD, Dr Maha Elseed, Dr Monika Malinova, Jassi Michell-Sodhi, Dionne Moat, Dr Liz Ghimenton, Dr Anna Mayhew, Karen Wong, Mark Richardson, Professor Giorgio TascaORCiD, Gail Eglon, Catherine TurnerORCiD, Emma HeslopORCiD, Professor Volker StraubORCiD, Professor Michela GuglieriORCiD
This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License (CC BY-NC-ND).
© 2024 The Authors. European Journal of Neurology published by John Wiley & Sons Ltd on behalf of European Academy of Neurology.Background and purpose: The transition to adult services, and subsequent glucocorticoid management, is critical in adults with Duchenne muscular dystrophy. This study aims (1) to describe treatment, functional abilities, respiratory and cardiac status during transition to adulthood and adult stages; and (2) to explore the association between glucocorticoid treatment after loss of ambulation (LOA) and late-stage clinical outcomes. Methods: This was a retrospective single-centre study on individuals with Duchenne muscular dystrophy (≥16 years old) between 1986 and 2022. Logistic regression, Cox proportional hazards models and survival analyses were conducted utilizing data from clinical records. Results: In all, 112 individuals were included. Mean age was 23.4 ± 5.2 years and mean follow-up was 18.5 ± 5.5 years. At last assessment, 47.2% were on glucocorticoids; the mean dose of prednisone was 0.38 ± 0.13 mg/kg/day and of deflazacort 0.43 ± 0.16 mg/kg/day. At age 16 years, motor function limitations included using a manual wheelchair (89.7%), standing (87.9%), transferring from a wheelchair (86.2%) and turning in bed (53.4%); 77.5% had a peak cough flow <270 L/min, 53.3% a forced vital capacity percentage of predicted <50% and 40.3% a left ventricular ejection fraction <50%. Glucocorticoids after LOA reduced the risk and delayed the time to difficulties balancing in the wheelchair, loss of hand to mouth function, forced vital capacity percentage of predicted <30% and forced vital capacity <1 L and were associated with lower frequency of left ventricular ejection fraction <50%, without differences between prednisone and deflazacort. Glucocorticoid dose did not differ by functional, respiratory or cardiac status. Conclusion: Glucocorticoids after LOA preserve late-stage functional abilities, respiratory and cardiac function. It is suggested using functional abilities, respiratory and cardiac status at transition stages for adult services planning.
Author(s): Schiava M, Lofra RM, Bourke JP, Diaz-Manera J, James MK, Elseed MA, Malinova M, Michel-Sodhi J, Moat D, Ghimenton E, Mccallum M, Diaz CFB, Mayhew A, Wong K, Richardson M, Tasca G, Eglon G, Eagle M, Turner C, Heslop E, Straub V, Bettolo CM, Guglieri M
Publication type: Article
Publication status: Published
Journal: European Journal of Neurology
Year: 2024
Pages: epub ahead of print
Online publication date: 31/03/2024
Acceptance date: 14/02/2024
Date deposited: 18/04/2024
ISSN (print): 1351-5101
ISSN (electronic): 1468-1331
Publisher: John Wiley and Sons Inc
URL: https://doi.org/10.1111/ene.16267
DOI: 10.1111/ene.16267
PubMed id: 38556893
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