Koeks, ZaïdaBladen, Catherine LSalgado, Davidvan Zwet, ErikPogoryelova, OksanaMcMacken, GraceMonges, SoledadFoncuberta, Maria EKekou, KyriakiKosma, KonstantinaDawkins, HughLamont, LeanneBellgard, Matthew IRoy, Anna JChamova, TeodoraGuergueltcheva, VelinaChan, SopheliaKorngut, LawrenceCampbell, CraigDai, YiWang, JenBarišić, NinaBrabec, PetrLähdetie, JaanaWalter, Maggie CSchreiber-Katz, OliviaKarcagi, VeronikaGarami, MartaHerczegfalvi, AgnesViswanathan, VenkatarmanBayat, FarhadBuccella, FilippoFerlini, AlessandraKimura, Envan den Bergen, Janneke CRodrigues, MiriamRoxburgh, RichardLusakowska, AnnaKostera-Pruszczyk, AnnaSantos, RosárioNeagu, ElenaArtemieva, SvetlanaRasic, Vedrana MilicVojinovic, DinaPosada De la Paz, ManuelBloetzer, ClemensKlein, AndreaDíaz-Manera, JordiGallardo, EduardKaraduman, A AyşeOznur, TuncaTopaloğlu, HalukEl Sherif, RashaStringer, AngelaShatillo, Andriy VMartin, Ann SPeay, Holly LKirschner, JanFlanigan, Kevin MStraub, VolkerBushby, KateBéroud, ChristopheVerschuuren, Jan JLochmüller, Hanns2023-03-062023-03-062017J Neuromuscul Dis. 2017;4(4):293-306.2214-3599http://hdl.handle.net/20.500.12105/15573Background: Recent short-term clinical trials in patients with Duchenne Muscular Dystrophy (DMD) have indicated greater disease variability in terms of progression than expected. In addition, as average life-expectancy increases, reliable data is required on clinical progression in the older DMD population. Objective: To determine the effects of corticosteroids on major clinical outcomes of DMD in a large multinational cohort of genetically confirmed DMD patients. Methods: In this cross-sectional study we analysed clinical data from 5345 genetically confirmed DMD patients from 31 countries held within the TREAT-NMD global DMD database. For analysis patients were categorised by corticosteroid background and further stratified by age. Results: Loss of ambulation in non-steroid treated patients was 10 years and in corticosteroid treated patients 13 years old (p = 0.0001). Corticosteroid treated patients were less likely to need scoliosis surgery (p < 0.001) or ventilatory support (p < 0.001) and there was a mild cardioprotective effect of corticosteroids in the patient population aged 20 years and older (p = 0.0035). Patients with a single deletion of exon 45 showed an increased survival in contrast to other single exon deletions. Conclusions: This study provides data on clinical outcomes of DMD across many healthcare settings and including a sizeable cohort of older patients. Our data confirm the benefits of corticosteroid treatment on ambulation, need for scoliosis surgery, ventilation and, to a lesser extent, cardiomyopathy. This study underlines the importance of data collection via patient registries and the critical role of multi-centre collaboration in the rare disease field.engVoRhttp://creativecommons.org/licenses/by-nd/4.0/Duchenne muscular dystrophyDMDNeuromuscular diseasesTREAT-NMDAdolescentAdrenal Cortex HormonesAdultChildChild, PreschoolCross-Sectional StudiesDatabases as TopicHumansInfantInfant, NewbornMaleMuscular Dystrophy, DuchenneTreatment OutcomeYoung AdultAttribution-NoDerivatives 4.0 Internacional291255044429310.3233/JND-170280Journal of neuromuscular diseasesopen access