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- W2891802010 abstract "Prospective studies with standardized evaluations provide one of the strongest methodologies for studying the progression of neuromuscular diseases and constructing clinical development plan to evaluate new therapies. Drop-out is a major methodological problem of longitudinal studies. It may represent an attrition bias and lead to erroneous conclusions if participants who stay in a study differ from those who dropped out. Here, we present drop-out rate and reasons for dropping-out in prospective studies in neuromuscular diseases and we study predictive factors. We focused on 4 prospective and longitudinal natural history studies conducted or coordinated in our center in three neuromuscular diseases: Duchenne muscular dystrophy (DMD), spinal muscular atrophy (SMA) and myotubular myopathy (MTM). We studied factors previously shown as predictors of attrition such as socio-demographic variables including age at enrollment, gender, patient's native language as compared to center's one, in- or outpatient. We also studied factors such as the clinical status of the patient (ambulatory, respiratory and feeding status), the study design (duration of the study, visit frequency, number of assessment) and the existence of concomitant therapeutic clinical trials or market approved treatments. On 31st December 2017, the median duration of the studies were 4.0 years [2.6 - 6.2]. The probability of drop-out occurrence during the first year was 11.3% (12.3%, 10.8% and 9.9 % in SMA, DMD and MTM patients respectively). Among the eighty-two patients who dropped out over 188 enrolled, 54% withdrew to be enrolled in a therapeutic study, 7% because of death and 24% withdrew for different reasons (i.e. lack of motivation, distance from the center or fatigue). The understanding of dropping out and precise estimation of its rates allows better sample size estimation of natural history studies and may help identifying patients at risk of dropping out at early stage of the study." @default.
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- W2891802010 date "2018-10-01" @default.
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- W2891802010 title "REGISTRIES AND CARE OF NEUROMUSCULAR DISORDERS" @default.
- W2891802010 doi "https://doi.org/10.1016/j.nmd.2018.06.330" @default.
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