Abstract:
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Disease progression in children with neuromuscular disorder is frequently assessed by the 6-min walk test, which classically constitutes the clinical primary outcome in the present therapeutic trials. However, assessing the muscle function in non-ambulatory patients constitutes a major challenge for therapeutic trials in such patients for who other outcome measures must be proposed, particularly on the upper limb.Upper limb evaluation may be assessed either by direct muscular strength measurement, or by clinical scale, by questionnaire, or by clinical test that still have to be standardized and validated. The aim of the ULENAP study (Upper Limb Evaluation in Non-Ambulatory Patients) is to consider these different approaches on a group of non-ambulatory patients with neuromuscular disorders. In each patient, strength of pinch, grip, hand flexion and extension is performed on both limbs. In addition, limb function is evaluated through a hand function questionnaire, motor function measurement (MFM), taping, and a recently developed device to measure the ability of patients to hit two targets with fingers, the MoviPlate.We present the feasibility and reproducibility of theses different approaches, and the correlations that may be observed between strength and functional outcome measures in 55 patients (among them 30 non ambulatory Duchenne boys) and 47 healthy children. Among the various tests, three of them seem particularly interesting, even in very weak patients presenting strong contractures: the grip and pinch strength assessment methods and the MoviPlate. Reliability of these methods is excellent in the patients as well as in the control subjects. No ceiling and floor effects could be detected. Moreover, various non linear relationships between strength and function assessment methods could be observed. This study demonstrates that a reliable upper limb evaluation in weak patients is feasible and robust when adapted tools are used.This project is supported by AFM (Association Franse contre les Myopathies) and by ADNA (Advanced Diagnostics for New Therapeutic Approaches), a program dedicated to personalized medicine, coordinated by Institut Meux and supported by research and innovation aid from the French public agency, OSEO.
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