Personalized orthotic care to improve functioning in patients with neuromuscular diseases

BACKGROUND People with neuromuscular diseases (NMD) often experience mobility limitations due to impaired leg muscle strength. Walking effort is usually increased and speed diminished with increased risk of falling. To reduce mobility problems leg orthoses are provided. However, there is large variety in types of orthoses that are applied and they are often insufficiently matched to the complex health needs of NMD patients. Failure to achieve functional success obviously limits the cost-effectiveness of leg orthoses. Therefore, in 2012, a guideline was developed to provide leg orthoses matched to the patients’ individual health needs. Application of such personalized leg orthoses shows promising results with respect to improving functioning when compared to standard care. However, the effectiveness and the cost-effectiveness of this personalized approach are unknown. OBJECTIVE To assess the effectiveness and cost-effectiveness of personalized orthotic care to improve functioning in patients with NMD. HYPOTHESIS Personalized orthotic care is more effective and cost-effective when compared to standard care. STUDY DESIGN A prospective randomized open label blinded end-point study with an economic evaluation. STUDY POPULATION AND SAMPLE SIZE Patients with NMD and quadriceps and/or calf muscle weakness, between 18 and 75 years, experiencing walking problems with increased walking effort for which a leg orthosis is considered. According to pilot data, we anticipate a 0.30 J/kg/m reduction in walking effort for standard care and 0.90 J/kg/m for personalized orthotic care. Based on an intention-to-treat analysis, alpha of 0.05, power of 80%, and considering a 15% drop-out, 70 patients need to be included (35 per treatment group) to detect this difference of 0.60 J/kg/m. This sample size also allows detection of 1 point difference in person-centered treatment goal scores. TYPE OF DEVICE/CARE The intervention group will receive orthotic care in two expert centers (AMC Amsterdam and Maartenskliniek Nijmegen) with facilities for advanced gait analysis and experienced professionals jointly working with orthotists to provide high quality, often custom made orthoses. Orthoses are provided according to the Guideline for leg orthoses of the Dutch Society of Rehabilitation Medicine, which was developed in a ZonMw-funded project by a multidisciplinary group of experts. The guideline complies with the ‘Process description medical devices’, and covers the entire process of care delivery including algorithm-based protocols to match the orthotic design to the individual needs and health problems. USUAL CARE /COMPARISON The control group receives standard care, which will be delivered at 6 general rehabilitation centers throughout the Netherlands. Standard care consists of the provision of orthopedic shoes, prefabricated and custom-made ankle foot orthoses (all designs and materials) and prefabricated and custom-made knee-ankle-foot orthoses (all designs and materials). OUTCOME MEASURES The two primary outcome measures to evaluate the clinical effectiveness will be change in walking effort (in J/kg/m) from baseline to 6 months post treatment, as measured by the 6-minute walk test and change in achievement of person-centered treatment goals, measured with the Goal Attainment Scale (GAS). The GAS is chosen as additional primary outcome to capture the orthotic goals relevant to the individual patient. Secondary outcomes will be change in: walking speed (6-minute walk test); stability during walking (10-point NRS); quality of life (EQ-5D-5L); physical functioning (SF36-physical functioning subscale); falling and fear of falling (FES-1); gait pattern (3D gait analysis); and satisfaction (D-Quest). Furthermore, rejection rate and adverse events will be assessed. Outcome measures will be assessed at baseline and at 3 months and 6 months post treatment. ECONOMIC EVALUATION The economic evaluation is conducted from a societal perspective. It will be carried out according to the Dutch guidelines for pharmaco-economic research and will include a cost-effectiveness and cost-utility analysis. Resource use will be available from the clinical report form, complemented with questionnaires to collect cost data (including healthcare costs, informal care, medication and patient- and productivity-related costs) at baseline and 3 and 6 months post treatment. Unit costs will be based on available sources (guideline prices, literature) or local costings. Utilities will be based on the EQ5D-5L. Multiple imputation, bootstrapping and seemingly unrelated regression analyses will be used in the analysis. TIME SCHEDULE Total duration of the study is 36 months. Prior to the study start, medical ethics approval will be obtained; 0-3 months: training of the researcher and setting up logistics; 3-24 months: recruitment of patients and baseline assessments; 9-33 months: follow-up assessments; 33-36 months: data analysis, interpretation and reporting.