Individualized Prediction of Changes in 6-Minute Walk Distance for Patients with Duchenne Muscular Dystrophy
| العنوان: | Individualized Prediction of Changes in 6-Minute Walk Distance for Patients with Duchenne Muscular Dystrophy |
|---|---|
| المؤلفون: | Elyse Swallow, Collaborative Trajectory Analysis, Marleen Van den Hauwe, James Signorovitch, Jinlin Song, Nathalie Goemans |
| المصدر: | PLoS ONE PLoS ONE, Vol 11, Iss 10, p e0164684 (2016) |
| بيانات النشر: | Public Library of Sciene, 2016. |
| سنة النشر: | 2016 |
| مصطلحات موضوعية: | Male, Heredity, Supine position, Physiology, Genetic Linkage, Epidemiology, Duchenne muscular dystrophy, Walking, Duchenne Muscular Dystrophy, Biochemistry, Muscular Dystrophies, Standard deviation, Dystrophin, Mathematical and Statistical Techniques, 0302 clinical medicine, Medicine and Health Sciences, Biomechanics, Child, Multidisciplinary, Organic Compounds, Age Factors, Prognosis, Explained variation, Chemistry, Neurology, X-Linked Traits, Sex Linkage, Child, Preschool, 030220 oncology & carcinogenesis, Physical Sciences, Ambulatory, Medicine, Steroids, Statistics (Mathematics), Research Article, medicine.medical_specialty, Drug Research and Development, Adolescent, Genotype, Science, Research and Analysis Methods, Natural history of disease, 03 medical and health sciences, Diagnostic Medicine, Genetics, medicine, Humans, Clinical Trials, Statistical Methods, Clinical Genetics, Pharmacology, Biological Locomotion, business.industry, Organic Chemistry, Chemical Compounds, Biology and Life Sciences, Proteins, Repeated measures design, Models, Theoretical, medicine.disease, Muscular Dystrophy, Duchenne, Clinical trial, Cytoskeletal Proteins, Natural History of Disease, Exercise Test, Physical therapy, Clinical Medicine, business, Mathematics, 030217 neurology & neurosurgery, Follow-Up Studies, Forecasting |
| الوصف: | BackgroundDeficits in ambulatory function progress at heterogeneous rates among individuals with Duchenne muscular dystrophy (DMD). The resulting inherent variability in ambulatory outcomes has complicated the design of drug efficacy trials and clouded the interpretation of trial results. We developed a prediction model for 1-year change in the six minute walk distance (6MWD) among DMD patients, and compared its predictive value to that of commonly used prognostic factors (age, baseline 6MWD, and steroid use).MethodsNatural history data were collected from DMD patients at routine follow up visits approximately every 6 months over the course of 2-5 years. Assessments included ambulatory function and steroid use. The annualized change in 6MWD (Δ6MWD) was studied between all pairs of visits separated by 8-16 months. Prediction models were developed using multivariable regression for repeated measures, and evaluated using cross-validation.ResultsAmong n = 191 follow-up intervals (n = 39 boys), mean starting age was 9.4 years, mean starting 6MWD was 351.8 meters, and 75% had received steroids for at least one year. Over the subsequent 8-16 months, mean Δ6MWD was -37.0 meters with a standard deviation (SD) of 93.7 meters. Predictions based on a composite of age, baseline 6MWD, and steroid use explained 28% of variation in Δ6MWD (R2 = 0.28, residual SD = 79.4 meters). A broadened prognostic model, adding timed 10-meter walk/run, 4-stair climb, and rise from supine, as well as height and weight, significantly improved prediction, explaining 59% of variation in Δ6MWD after cross-validation (R2 = 0.59, residual SD = 59.7 meters).ConclusionsA prognostic model incorporating timed function tests significantly improved prediction of 1-year changes in 6MWD. Explained variation was more than doubled compared to predictions based only on age, baseline 6MWD, and steroid use. There is significant potential for composite prognostic models to inform DMD clinical trials and clinical practice. |
| وصف الملف: | Electronic-eCollection; application/pdf |
| اللغة: | English |
| الوصول الحر: | https://explore.openaire.eu/search/publication?articleId=doi_dedup___::549f2e150970d8e439ec6a09f9e507a6Test http://dx.plos.org/10.1371/journal.pone.0164684Test |
| حقوق: | OPEN |
| رقم الانضمام: | edsair.doi.dedup.....549f2e150970d8e439ec6a09f9e507a6 |
| قاعدة البيانات: | OpenAIRE |
| الوصف غير متاح. |