Treatment for postpolio syndrome.

BACKGROUND: Postpolio syndrome (PPS) may affect survivors of paralytic poliomyelitis and is characterised by a complex of neuromuscular symptoms leading to a decline in physical functioning. The effectiveness of pharmacological treatment and rehabilitation management in PPS is not yet established. This is an update of a review first published in 2011. OBJECTIVES: To systematically review the evidence from randomised and quasi-randomised controlled trials for the effect of any pharmacological or non-pharmacological treatment for PPS compared to placebo, usual care or no treatment.  SEARCH METHODS: We searched the following databases on 21 July 2014: Cochrane Neuromuscular Disease Group Specialized Register, the Cochrane Central Register of Controlled Trials (CENTRAL), MEDLINE, EMBASE, PsycINFO and CINAHL Plus. We also checked reference lists of all relevant articles, searched the Database of Abstracts of Reviews of Effects (DARE), the Health Technology Assessment (HTA) Database and trial registers and contacted investigators known to be involved in research in this area. SELECTION CRITERIA: Randomised and quasi-randomised trials of any form of pharmacological or non-pharmacological treatment for people with PPS. The primary outcome was self perceived activity limitations and secondary outcomes were muscle strength, muscle endurance, fatigue, pain and adverse events. DATA COLLECTION AND ANALYSIS: We used standard methodological procedures expected by The Cochrane Collaboration. MAIN RESULTS: We included 10 pharmacological (modafinil, intravenous immunoglobulin (IVIg), pyridostigmine, lamotrigine, amantadine, prednisone) and three non-pharmacological (muscle strengthening, rehabilitation in a warm climate (that is temperature ± 25°C, dry and sunny) and a cold climate (that is temperature ± 0°C, rainy or snowy), static magnetic fields) studies with a total of 675 participants with PPS in this review. None of the included studies were completely free from any risk of bias, the most prevalent risk of bias being lack of blinding.There was moderate- and low-quality evidence that IVIg has no beneficial effect on activity limitations in the short term and long term, respectively, and inconsistency in the evidence for effectiveness on muscle strength. IVIg caused minor adverse events in a substantial proportion of the participants. Results of one trial provided very low-quality evidence that lamotrigine might be effective in reducing pain and fatigue, resulting in fewer activity limitations without generating adverse events. Data from two single trials suggested that muscle strengthening of thumb muscles (very low-quality evidence) and static magnetic fields (moderate-quality evidence) are safe and beneficial for improving muscle strength and pain, respectively, with unknown effects on activity limitations. Finally, there was evidence varying from very low quality to high quality that modafinil, pyridostigmine, amantadine, prednisone and rehabilitation in a warm or cold climate are not beneficial in PPS. AUTHORS' CONCLUSIONS: Due to insufficient good-quality data and lack of randomised studies, it was impossible to draw definite conclusions about the effectiveness of interventions for PPS. Results indicated that IVIg, lamotrigine, muscle strengthening exercises and static magnetic fields may be beneficial but need further investigation to clarify whether any real and meaningful effect exists.

Safety, walking ability, and satisfaction outcomes of the NEURO TRONIC stance-control knee-ankle-foot orthosis (SCKAFO): A comparative evaluation to the E-MAG active SCKAFO.

BACKGROUND: Stance control knee-ankle-foot orthoses (SCKAFOs) ensure knee stability by locking during stance while allowing knee flexion during swing. Differences in function of the knee joints and building principles between devices may affect their effectiveness. OBJECTIVE: To investigate the preliminary effectiveness of a NEURO TRONIC on safety outcomes, net energy cost (EC), and user experiences in individuals already using an E-MAG Active SCKAFO. STUDY DESIGN: Prospective uncontrolled intervention study. METHODS: A convenience sample of 10 subjects with flaccid lower extremity muscle weakness, including the quadriceps, due to neuromuscular disorders already using an E-MAG Active SCKAFO were provided with a newly fabricated NEURO TRONIC SCKAFO. Outcomes included knee joint locking failures and unlocking failures (ULFs) (i.e., percentage of steps the knee joint failed to lock/unlock) when walking under challenging conditions on an instrumented treadmill while wearing a safety harness; net EC (J/kg per meter) assessed with a 6-min walk test at comfortable speed; 3D gait kinematics and kinetics; and patient-reported outcomes. RESULTS: No differences between devices were found for knee joint locking failures (both devices 0%) and ULFs (9.9% for the NEURO TRONIC vs. 13.9% for the E-MAG Active SCKAFO). The mean (standard deviation) net EC with the NEURO TRONIC SCKAFO was 8.2% (from 3.68 [0.81] to 3.38 [0.75] J/kg per meter, p = 0.123) lower, although not significantly, compared with that with the E-MAG Active SCKAFO. Significant improvements with the NEURO TRONIC SCKAFO were found for ankle power ( p = 0.003), perceived walking effort ( p = 0.014), and reported falls ( p = 0.034). CONCLUSION: Both the NEURO TRONIC SCKAFO and the E-MAG Active SCKAFO were safe in terms of knee joint locking, while ULFs were frequent with both devices. The net EC with the NEURO TRONIC SCKAFO decreased, although not significantly, by 8.2%, likely due to insufficient power. Perceived walking effort was in favor of the NEURO TRONIC SCKAFO.

A Mobile Health App to Support Home-Based Aerobic Exercise in Neuromuscular Diseases: Usability Study.

BACKGROUND: Home-based aerobic exercise in people with neuromuscular diseases (NMDs) has benefits compared to exercise in the hospital or a rehabilitation center because traveling is often cumbersome due to mobility limitations, and societal costs are lower. Barriers to home-based aerobic exercise include reduced possibilities for monitoring and lack of motivation. To overcome these and other barriers, we developed a mobile health app: Keep on training with ReVi (hereafter referred to as ReVi). OBJECTIVE: We aimed to determine the usability of the ReVi app. METHODS: Patients followed a 4-month, polarized, home-based aerobic exercise program on a cycle or rowing ergometer, with 2 low-intensity sessions and 1 high-intensity session per week supported by the ReVi app. The app collected training data, including heart rate and ratings of perceived exertion, provided real-time feedback on reaching target intensity zones, and enabled monitoring via an online dashboard. Physiotherapists instructed patients on how to use the ReVi app and supervised them during their training program. Patients and physiotherapists separately evaluated usability with self-developed questionnaires, including 9 questions on a 5-point Likert scale, covering the usability elements efficiency, effectiveness, and satisfaction. RESULTS: Twenty-nine ambulatory adult patients (n=19 women; mean age 50.4, SD 14.2 years) with 11 different slowly progressive NMDs participated. Both patients and physiotherapists (n=10) reported that the app, in terms of its efficiency, was easy to use and had a rapid learning curve. Sixteen patients (55%) experienced 1 or more technical issue(s) during the course of the exercise program. In the context of effectiveness, 23 patients (81%) indicated that the app motivated them to complete the program and that it helped them to exercise within the target intensity zones. Most patients (n=19, 70%) and physiotherapists (n=6, 60%) were satisfied with the use of the app. The median attendance rate was 88% (IQR 63%-98%), with 76% (IQR 69%-82%) of time spent within the target intensity zones. Four adverse events were reported, 3 of which were resolved without discontinuation of the exercise program. CONCLUSIONS: The usability of the ReVi app was high, despite the technical issues that occurred. Further development of the app to resolve these issues is warranted before broader implementation into clinical practice.

Measurement Properties of Aerobic Capacity Measures in Neuromuscular Diseases: A Systematic Review.

OBJECTIVE: To systematically evaluate the measurement properties of aerobic capacity measures in neuromuscular diseases. DATA SOURCES: MEDLINE, EMBASE, SportDiscus and Web of Science Conference Proceedings Citation Index - Science were systematically searched from inception until 30 June 2021. STUDY SELECTION AND DATA EXTRACTION: Screening, data extraction, risk of bias assessment and quality assessment were performed by 2 independent researchers. Studies were included if they evaluated measurement properties of aerobic capacity measures in adults with neuromuscular diseases. Risk of bias was assessed using the COnsensus based Standards for the selection of health status Measurement INstruments (COSMIN) checklist. Results were pooled and the quality of the evidence was determined using a modified Grading of Recommendations, Assessment, Development and Evaluations (GRADE) approach. DATA SYNTHESIS: Nine studies including 187 participants were included in this review. Low quality of evidence was found for sufficient content validity of peak oxygen consumption through maximal exercise testing. Criterion validity of 4 out of 7 different measures to predict peak oxygen consumption was sufficient; however, quality of evidence was low or very low for all measures. No studies were found evaluating reliability or responsiveness. CONCLUSION: There was a lack of high-quality studies with sufficiently large sample sizes that evaluated the measurement properties of aerobic capacity measures in neuromuscular diseases.

Treatment for postpolio syndrome.

BACKGROUND: Postpolio syndrome (PPS) may affect survivors of paralytic poliomyelitis and is characterised by a complex of neuromuscular symptoms leading to a decline in physical functioning. The effectiveness of pharmacological treatment and rehabilitation management in PPS is not yet established. OBJECTIVES: To review systematically the effects of any treatment for PPS compared to placebo, usual care or no treatment.   SEARCH STRATEGY: We searched the following databases on 1 October 2010: Cochrane Neuromuscular Disease Group Specialized Register, the Cochrane Central Register of Controlled Trials, MEDLINE, EMBASE, PsycINFO and CINAHL Plus from inception to September 2010. SELECTION CRITERIA: Randomised and quasi-randomised trials of any form of pharmacological or non-pharmacological treatment for people with PPS. The primary outcome was self-perceived activity limitations and secondary outcomes were muscle strength, muscle endurance, fatigue, pain and adverse events. DATA COLLECTION AND ANALYSIS: Two authors independently selected eligible studies, assessed risk of bias and extracted data. MAIN RESULTS: Nine pharmacological (modafinil, intravenous immunoglobulin, pyridostigmine, lamotrigine, amantadine, prednisone) and three non-pharmacological (muscle strengthening, rehabilitation in a warm climate (i.e. temperature ± 25°C, dry and sunny) and a cold climate (i.e. temperature ± 0°C, rainy or snowy), static magnetic fields) studies were included in this review. None of the included studies was completely free from any risk of bias and the most prevalent risk of bias was lack of blinding.There is moderate quality evidence that intravenous immunoglobulin has no beneficial effect on activity limitations and there is inconsistency in the evidence for effectiveness on muscle strength and pain. Results of one trial provide very low quality evidence that lamotrigine might be effective in reducing pain and fatigue, resulting in fewer activity limitations. Data from two single trials suggest that muscle strengthening of thumb muscles (very low quality evidence) and static magnetic fields (moderate quality evidence) are beneficial for improving muscle strength and pain, respectively, with unknown effects on activity limitations. Finally, there is evidence varying from very low quality to high quality that modafinil, pyridostigmine, amantadine, prednisone and rehabilitation in a warm or cold climate are not beneficial in PPS. AUTHORS' CONCLUSIONS: Due to insufficient good quality data and lack of randomised studies it is impossible to draw definite conclusions on the effectiveness of interventions for PPS. Results indicate that IVIG, lamotrigine, muscle strengthening exercises and static magnetic fields may be beneficial but need further investigation.

Specialised orthotic care to improve functioning in adults with neuromuscular disorders: protocol of a prospective randomised open-label blinded end-point study.

INTRODUCTION: People suffering from leg muscle weakness caused by neuromuscular disorders (NMDs) are often provided with leg orthoses to reduce walking problems such as increased walking effort, diminished walking speed, reduced balance and falls. However, evidence for the effectiveness of leg orthoses to improve walking in this patient group is limited and there is an absence of standardised practice in orthotic prescription. In 2012 a Dutch multidisciplinary guideline was developed aimed to standardise the orthotic treatment process in NMD. Although application of the guideline in expert centres (specialised orthotic care) seems beneficial regarding clinical effectiveness, larger studies are necessary to confirm results and investigate cost-effectiveness. Therefore, this study aims to examine the effectiveness and cost-effectiveness of specialised orthotic care compared with usual orthotic care in adults with slowly progressive NMD. METHODS AND ANALYSIS: A prospective randomised open-label blinded end-point study will be performed, in which 70 adults with slowly progressive NMD are randomly assigned to specialised orthotic care (intervention) or usual orthotic care (control). Outcome measures are assessed at baseline and at 3 and 6 months follow-up. The primary endpoints are gross walking energy cost (J/kg/m) assessed during a 6 min walk test and achievement of personal goals, measured with the Goal Attainment Scale. Secondary endpoints include walking speed, gait biomechanics, stability, physical functioning, falls and fear of falling, perceived fatigue and satisfaction. For the economic evaluation, societal costs and health-related quality of life will be assessed using cost questionnaires and the 5-Level version of EuroQol 5 Dimension, retrospectively. ETHICS AND DISSEMINATION: The study is registered in the Dutch trial register (NL 7511) and the protocol has been approved by the Medical Ethics Committee of the Academic Medical Center in Amsterdam. Results will be presented at national and international scientific conferences and disseminated through peer-reviewed journals and media aimed at a broad audience including patients.