Author(s): Pietrusz A.; Quinlivan R.; Desikan M.; Trucco F.; Muntoni F.; Manzur A.; Guglieri M.; Hilton-Jones D.; Burke G.; Hammans S.; Davies N.
Source: Neuromuscular Disorders; Oct 2019; vol. 29
Publication Date: Oct 2019
Publication Type(s): Conference Abstract
Abstract:Several years of extensive data collection of natural history in children with Duchenne muscular dystrophy (DMD) has resulted in better knowledge and understanding of the role of corticosteroids in patients’ treatment and management. Despite currently published standards of care in the UK as well as, pulmonary function data from the cohort of 397 patients both suggesting beneficial role of corticosteroids in preservation of respiratory function and effectively slowing down the disease progression, there is limited evidence regarding corticosteroid use in adults with DMD. A retrospective multi-site clinical audit will be performed to assess the effects of corticosteroid treatment on respiratory function in adults with DMD after loss of ambulation. We will present data from non-ambulant adult patients and compare longitudinal outcomes for patients where corticosteroids are continued after loss of ambulation vs the effect of stopping corticosteroids. Preliminary data on the use of corticosteroids in adults with DMD after loss of ambulation in the UK will be presented and discussed.
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