Mixed methods evaluation of a self-management group programme for patients with neuromuscular disease and chronic fatigue.

OBJECTIVE: To obtain insight into experiences of patients with a neuromuscular disease and chronic fatigue and their healthcare professionals regarding content and delivery of a multidisciplinary outpatient self-management group programme to improve social participation. This will inform future implementation. DESIGN: A mixed method study alongside a randomised controlled trial. SETTING: University hospital, rehabilitation centre and community health centre. PARTICIPANTS: 29 patients with a neuromuscular disease and chronic fatigue and 13 healthcare professionals participated in this mixed methods study. INTERVENTION: Multidisciplinary group programme, called Energetic, consisted of a 4 months intervention with weekly meetings and covered four modules: (1) individually tailored aerobic exercise training; (2) education about aerobic exercise; (3) self-management training in applying energy conservation strategies and (4) implementation and relapse prevention in daily life. MAIN MEASURES: Quantitative data were collected by a questionnaire measuring patients' (n=25, all completed the programme) satisfaction with the perceived results, content and delivery of the programme. Qualitative data were collected by individual and focus group interviews to gain insight in the experiences of patients (n=18), next of kin (n=2) and healthcare professionals (n=13) with facilitators and barriers to programme implementation. RESULTS: Patients were satisfied with the number and length of the sessions, the different modules and the therapists. Analysis of the interviews led to five themes: (1) the combination of modules makes a complete picture, (2) the programme is physically and mentally intensive, (3) the group setting is valuable, (4) small variations in delivery occur in different settings, (5) therapists are coaches. Suggestions for programme improvement include a combination of face to face and e-health, enhancement of therapists' skills in guiding group interventions and inclusion of more booster sessions to evaluate and maintain self-management competencies. CONCLUSIONS: The Energetic programme could be implemented in different healthcare settings and group settings, and a combination of modules proved to be a facilitator for improving self-management. TRIAL REGISTRATION NUMBER: NCT02208687.

Changes in severity and impact of drooling after submandibular gland botulinum neurotoxin A injections in children with neurodevelopmental disabilities.

AIMS: To examine changes in objective and subjective drooling severity measures after submandibular botulinum neurotoxin A injection in children with neurodevelopmental disabilities, explore their relationship, and evaluate if clinically relevant responses relate to changes in the impact of drooling. METHOD: This longitudinal, observational cohort study involved 160 children (92 males, 68 females; 3-17y, mean 9y 1mo, SD 3y 6mo) treated between 2000 and 2012 at the Radboud University Medical Center, Nijmegen, the Netherlands. Repeated measures analysis of variance was used to compare the 5-minute Drooling Quotient (DQ5) and Visual Analogue Scale (VAS) for drooling severity pretreatment and posttreatment, and Pearson's rho to assess their association. A parent questionnaire was used to assess drooling impact in responders (defined as ≥50% reduction in DQ5 and/or ≥2 SD reduction in VAS for drooling severity 8wks postintervention) and non-responders. RESULTS: One hundred and twelve children (70%) were responders. Their mean VAS for drooling severity and DQ5 scores were significantly lower 32 weeks postintervention compared to baseline. At baseline, the VAS for drooling severity-DQ5 relationship was 'weak' (rs =0.15, p=0.060), whereas it was 'fair' at 8 weeks (rs =0.43, p=0.000) and 32 weeks (rs =0.30, p=0.000). For responders, a significant change was found regarding the impact of drooling on daily care and social interactions at 8 weeks after intervention; most of these effects were maintained at 32 weeks. INTERPRETATION: A clinically relevant response based on a combination of objective and subjective measures of drooling severity was accompanied by positive changes regarding the impact of drooling on daily care and social interactions. WHAT THIS PAPER ADDS: Botulinum neurotoxin A injection into the submandibular glands reduced drooling severity in 70% of children with neurodevelopmental disabilities. Objective (5-minute Drooling Quotient) and subjective (Visual Analogue Scale for drooling severity) measures correlated 8 and 32 weeks after treatment. Objective and subjective measures complemented each other when changes in drooling severity were assessed. Reduced drooling severity was accompanied by positive changes with regard to the impact of drooling.

Strength training and aerobic exercise training for muscle disease.

BACKGROUND: Strength training or aerobic exercise programmes, or both, might optimise muscle and cardiorespiratory function and prevent additional disuse atrophy and deconditioning in people with a muscle disease. This is an update of a review first published in 2004 and last updated in 2013. We undertook an update to incorporate new evidence in this active area of research. OBJECTIVES: To assess the effects (benefits and harms) of strength training and aerobic exercise training in people with a muscle disease. SEARCH METHODS: We searched Cochrane Neuromuscular's Specialised Register, CENTRAL, MEDLINE, Embase, and CINAHL in November 2018 and clinical trials registries in December 2018. SELECTION CRITERIA: Randomised controlled trials (RCTs), quasi-RCTs or cross-over RCTs comparing strength or aerobic exercise training, or both lasting at least six weeks, to no training in people with a well-described muscle disease diagnosis. DATA COLLECTION AND ANALYSIS: We used standard methodological procedures expected by Cochrane. MAIN RESULTS: We included 14 trials of aerobic exercise, strength training, or both, with an exercise duration of eight to 52 weeks, which included 428 participants with facioscapulohumeral muscular dystrophy (FSHD), dermatomyositis, polymyositis, mitochondrial myopathy, Duchenne muscular dystrophy (DMD), or myotonic dystrophy. Risk of bias was variable, as blinding of participants was not possible, some trials did not blind outcome assessors, and some did not use an intention-to-treat analysis. Strength training compared to no training (3 trials) For participants with FSHD (35 participants), there was low-certainty evidence of little or no effect on dynamic strength of elbow flexors (MD 1.2 kgF, 95% CI -0.2 to 2.6), on isometric strength of elbow flexors (MD 0.5 kgF, 95% CI -0.7 to 1.8), and ankle dorsiflexors (MD 0.4 kgF, 95% CI -2.4 to 3.2), and on dynamic strength of ankle dorsiflexors (MD -0.4 kgF, 95% CI -2.3 to 1.4). For participants with myotonic dystrophy type 1 (35 participants), there was very low-certainty evidence of a slight improvement in isometric wrist extensor strength (MD 8.0 N, 95% CI 0.7 to 15.3) and of little or no effect on hand grip force (MD 6.0 N, 95% CI -6.7 to 18.7), pinch grip force (MD 1.0 N, 95% CI -3.3 to 5.3) and isometric wrist flexor force (MD 7.0 N, 95% CI -3.4 to 17.4). Aerobic exercise training compared to no training (5 trials) For participants with DMD there was very low-certainty evidence regarding the number of leg revolutions (MD 14.0, 95% CI -89.0 to 117.0; 23 participants) or arm revolutions (MD 34.8, 95% CI -68.2 to 137.8; 23 participants), during an assisted six-minute cycle test, and very low-certainty evidence regarding muscle strength (MD 1.7, 95% CI -1.9 to 5.3; 15 participants). For participants with FSHD, there was low-certainty evidence of improvement in aerobic capacity (MD 1.1 L/min, 95% CI 0.4 to 1.8, 38 participants) and of little or no effect on knee extension strength (MD 0.1 kg, 95% CI -0.7 to 0.9, 52 participants). For participants with dermatomyositis and polymyositis (14 participants), there was very low-certainty evidence regarding aerobic capacity (MD 14.6, 95% CI -1.0 to 30.2). Combined aerobic exercise and strength training compared to no training (6 trials) For participants with juvenile dermatomyositis (26 participants) there was low-certainty evidence of an improvement in knee extensor strength on the right (MD 36.0 N, 95% CI 25.0 to 47.1) and left (MD 17 N 95% CI 0.5 to 33.5), but low-certainty evidence of little or no effect on maximum force of hip flexors on the right (MD -9.0 N, 95% CI -22.4 to 4.4) or left (MD 6.0 N, 95% CI -6.6 to 18.6). This trial also provided low-certainty evidence of a slight decrease of aerobic capacity (MD -1.2 min, 95% CI -1.6 to 0.9). For participants with dermatomyositis and polymyositis (21 participants), we found very low-certainty evidence for slight increases in muscle strength as measured by dynamic strength of knee extensors on the right (MD 2.5 kg, 95% CI 1.8 to 3.3) and on the left (MD 2.7 kg, 95% CI 2.0 to 3.4) and no clear effect in isometric muscle strength of eight different muscles (MD 1.0, 95% CI -1.1 to 3.1). There was very low-certainty evidence that there may be an increase in aerobic capacity, as measured with time to exhaustion in an incremental cycle test (17.5 min, 95% CI 8.0 to 27.0) and power performed at VO2 max (maximal oxygen uptake) (18 W, 95% CI 15.0 to 21.0). For participants with mitochondrial myopathy (18 participants), we found very low-certainty evidence regarding shoulder muscle (MD -5.0 kg, 95% CI -14.7 to 4.7), pectoralis major muscle (MD 6.4 kg, 95% CI -2.9 to 15.7), and anterior arm muscle strength (MD 7.3 kg, 95% CI -2.9 to 17.5). We found very low-certainty evidence regarding aerobic capacity, as measured with mean time cycled (MD 23.7 min, 95% CI 2.6 to 44.8) and mean distance cycled until exhaustion (MD 9.7 km, 95% CI 1.5 to 17.9). One trial in myotonic dystrophy type 1 (35 participants) did not provide data on muscle strength or aerobic capacity following combined training. In this trial, muscle strength deteriorated in one person and one person had worse daytime sleepiness (very low-certainty evidence). For participants with FSHD (16 participants), we found very low-certainty evidence regarding muscle strength, aerobic capacity and VO2 peak; the results were very imprecise. Most trials reported no adverse events other than muscle soreness or joint complaints (low- to very low-certainty evidence). AUTHORS' CONCLUSIONS: The evidence regarding strength training and aerobic exercise interventions remains uncertain. Evidence suggests that strength training alone may have little or no effect, and that aerobic exercise training alone may lead to a possible improvement in aerobic capacity, but only for participants with FSHD. For combined aerobic exercise and strength training, there may be slight increases in muscle strength and aerobic capacity for people with dermatomyositis and polymyositis, and a slight decrease in aerobic capacity and increase in muscle strength for people with juvenile dermatomyositis. More research with robust methodology and greater numbers of participants is still required.

Interventions for preventing falls in people after stroke.

BACKGROUND: Falls are one of the most common complications after stroke, with a reported incidence ranging between 7% in the first week and 73% in the first year post stroke. This is an updated version of the original Cochrane Review published in 2013. OBJECTIVES: To evaluate the effectiveness of interventions aimed at preventing falls in people after stroke. Our primary objective was to determine the effect of interventions on the rate of falls (number of falls per person-year) and the number of fallers. Our secondary objectives were to determine the effects of interventions aimed at preventing falls on 1) the number of fall-related fractures; 2) the number of fall-related hospital admissions; 3) near-fall events; 4) economic evaluation; 5) quality of life; and 6) adverse effects of the interventions. SEARCH METHODS: We searched the trials registers of the Cochrane Stroke Group (September 2018) and the Cochrane Bone, Joint and Muscle Trauma Group (October 2018); the Cochrane Central Register of Controlled Trials (CENTRAL; 2018, Issue 9) in the Cochrane Library; MEDLINE (1950 to September 2018); Embase (1980 to September 2018); CINAHL (1982 to September 2018); PsycINFO (1806 to August 2018); AMED (1985 to December 2017); and PEDro (September 2018). We also searched trials registers and checked reference lists. SELECTION CRITERIA: Randomised controlled trials of interventions where the primary or secondary aim was to prevent falls in people after stroke. DATA COLLECTION AND ANALYSIS: Two review authors (SD and WS) independently selected studies for inclusion, assessed trial quality and risk of bias, and extracted data. We resolved disagreements through discussion, and contacted study authors for additional information where required. We used a rate ratio and 95% confidence interval (CI) to compare the rate of falls (e.g. falls per person-year) between intervention and control groups. For risk of falling we used a risk ratio and 95% CI based on the number of people falling (fallers) in each group. We pooled results where appropriate and applied GRADE to assess the quality of the evidence. MAIN RESULTS: We included 14 studies (of which six have been published since the first version of this review in 2013), with a total of 1358 participants. We found studies that investigated exercises, predischarge home visits for hospitalised patients, the provision of single lens distance vision glasses instead of multifocal glasses, a servo-assistive rollator and non-invasive brain stimulation for preventing falls.Exercise compared to control for preventing falls in people after strokeThe pooled result of eight studies showed that exercise may reduce the rate of falls but we are uncertain about this result (rate ratio 0.72, 95% CI 0.54 to 0.94, 765 participants, low-quality evidence). Sensitivity analysis for single exercise interventions, omitting studies using multiple/multifactorial interventions, also found that exercise may reduce the rate of falls (rate ratio 0.66, 95% CI 0.50 to 0.87, 626 participants). Sensitivity analysis for the effect in the chronic phase post stroke resulted in little or no difference in rate of falls (rate ratio 0.58, 95% CI 0.31 to 1.12, 205 participants). A sensitivity analysis including only studies with low risk of bias found little or no difference in rate of falls (rate ratio 0.88, 95% CI 0.65 to 1.20, 462 participants). Methodological limitations mean that we have very low confidence in the results of these sensitivity analyses.For the outcome of number of fallers, we are very uncertain of the effect of exercises compared to the control condition, based on the pooled result of 10 studies (risk ratio 1.03, 95% CI 0.90 to 1.19, 969 participants, very low quality evidence). The same sensitivity analyses as described above gives us very low certainty that there are little or no differences in number of fallers (single interventions: risk ratio 1.09, 95% CI 0.93 to 1.28, 796 participants; chronic phase post stroke: risk ratio 0.94, 95% CI 0.73 to 1.22, 375 participants; low risk of bias studies: risk ratio 0.96, 95% CI 0.77 to 1.21, 462 participants).Other interventions for preventing falls in people after strokeWe are very uncertain whether interventions other than exercise reduce the rate of falls or number of fallers. We identified very low certainty evidence when investigating the effect of predischarge home visits (rate ratio 0.85, 95% CI 0.43 to 1.69; risk ratio 1.48, 95% CI 0.71 to 3.09; 85 participants), provision of single lens distance glasses to regular wearers of multifocal glasses (rate ratio 1.08, 95% CI 0.52 to 2.25; risk ratio 0.74, 95% CI 0.47 to 1.18; 46 participants) and a servo-assistive rollator (rate ratio 0.44, 95% CI 0.16 to 1.21; risk ratio 0.44, 95% CI 0.16 to 1.22; 42 participants).Finally, transcranial direct current stimulation (tDCS) was used in one study to examine the effect on falls post stroke. We have low certainty that active tDCS may reduce the number of fallers compared to sham tDCS (risk ratio 0.30, 95% CI 0.14 to 0.63; 60 participants). AUTHORS' CONCLUSIONS: At present there exists very little evidence about interventions other than exercises to reduce falling post stroke. Low to very low quality evidence exists that this population benefits from exercises to prevent falls, but not to reduce number of fallers.Fall research does not in general or consistently follow methodological gold standards, especially with regard to fall definition and time post stroke. More well-reported, adequately-powered research should further establish the value of exercises in reducing falling, in particular per phase, post stroke.

Validity and reproducibility of the Functional Gait Assessment in persons after stroke.

OBJECTIVE:: To evaluate construct validity and reproducibility of the Functional Gait Assessment (FGA) for measuring walking balance capacity in persons after stroke. DESIGN:: Cross-sectional study. SETTING:: Inpatient and outpatient rehabilitation center. SUBJECTS:: Fifty-two persons post-stroke (median (25% and 75% percentiles)) time post-stroke 6 (5-10) weeks) with independent walking ability (mean gait speed 1.1 ± .4 m/s). METHODS:: Subjects completed a standardized FGA twice within one to eight days by the same investigator. Validity was evaluated by testing hypotheses on the association with two timed walking tests, Berg Balance Scale, and the mobility domain of the Stroke Impact Scale using correlation coefficients ( r), and with Functional Ambulation Categories using the Kruskal-Wallis test. Reproducibility of FGA scores was assessed with intraclass correlation coefficient and standard error of measurement. RESULTS:: Subjects scored a median of 22 out of 30 points at the first FGA. Moderate to high significant correlations ( r .61-.83) and significant differences in FGA median scores between the Functional Ambulation Categories were found. Eight hypotheses (80%) could be confirmed. Inter-rater, intra-rater, and test-retest reliability of the total scores were excellent. The standard error of measurement and minimal detectable change were 2 and 6 points, respectively. No relevant ceiling effect was observed. CONCLUSION:: The FGA demonstrated good measurement properties in persons after stroke and yielded no ceiling effect in contrast to other capacity measures. In clinical practice, a measurement error of 6 points should be taken into account in interpreting changes in walking balance.

Hand-Use-at-Home Questionnaire: validity and reliability in children with neonatal brachial plexus palsy or unilateral cerebral palsy.

OBJECTIVE: To investigate construct validity and test-retest reliability of the parent-rated Hand-Use-at-Home questionnaire (HUH) in children with neonatal brachial plexus palsy or unilateral cerebral palsy. DESIGN AND SUBJECTS: For this cross-sectional study, children with neonatal brachial plexus palsy or unilateral cerebral palsy, aged 3-10 years, were eligible. MAIN MEASURES: The HUH, Pediatric Outcome Data Collection Instrument Upper Extremity Scale (neonatal brachial plexus palsy only), and Children's Hand-Use Experience Questionnaire (unilateral cerebral palsy only) were completed. The HUH was completed twice in subgroups of both diagnoses. Lesion-extent (indication of involved nerve rootlets in neonatal brachial plexus palsy as confirmed during clinical observation and/or nerve surgery) and Manual Ability Classification System levels (unilateral cerebral palsy) were obtained from the medical records. Spearman correlation coefficients between the HUH and all clinical variables, agreement, standard error of measurement, smallest detectable change and intra-class correlation were calculated. RESULTS: A total of 260 patients participated (neonatal brachial plexus palsy: 181), of which 56 completed the second HUH (neonatal brachial plexus palsy: 16). Median age was 6.9 years for children with neonatal brachial plexus palsy, 116 had C5-C6 lesions. Median age for children with unilateral cerebral palsy was 6.4 years, 33 had Manual Ability Classification System Level II. The HUH correlated moderately with lesion-extent ( rs =-0.5), Pediatric Outcome Data Collection Instrument Upper Extremity Scale ( rs = 0.6) and Children's Hand-Use Experience Questionnaire ( rs = 0.5) but weakly with Manual Ability Classification System levels ( rs = -0.4). Test-retest reliability was excellent (intra-class correlation2,1 = 0.89, standard error of measurement = 0.599 and smallest detectable change = 1.66 logits) and agreement was good (mean difference HUH1 - HUH2 = 0.06 logits). CONCLUSION: The HUH showed good construct validity and test-retest reliability in children with neonatal brachial plexus palsy or unilateral cerebral palsy.

Definition dependent properties of the cortical silent period in upper-extremity muscles, a methodological study.

BACKGROUND: To explore if stimulus-response (S-R) characteristics of the silent period (SP) after transcranial magnetic stimulation (TMS) are affected by changing the SP definition and by changing data presentation in healthy individuals. This information would be clinically relevant to predict motor recovery in patients with stroke using stimulus-response curves. METHODS: Different landmarks to define the SP onset and offset were used to construct S-R curves from the biceps brachii (BB) and abductor digiti minimi (ADM) muscles in 15 healthy participants using rectified versus non-rectified surface electromyography (EMG). A non-linear mixed model fit to a sigmoid Boltzmann function described the S-R characteristics. Differences between S-R characteristics were compared using paired sample t-tests. The Bonferroni correction was used to adjust for multiple testing. RESULTS: For the BB, no differences in S-R characteristics were observed between different SP onset and offset markers, while there was no influence of data presentation either. For the ADM, no differences were observed between different SP onset markers, whereas both the SP offset marker "the first return of any EMG-activity" and presenting non-rectified data showed lower active motor thresholds and less steep slopes. CONCLUSIONS: The use of different landmarks to define the SP offset as well as data presentation affect SP S-R characteristics of the ADM in healthy individuals.

The effectiveness of an augmented cognitive behavioural intervention for post-stroke depression with or without anxiety (PSDA): the Restore4Stroke-PSDA trial.

BACKGROUND: Post-Stroke Depression with or without Anxiety (PSDA) is a common disorder in the chronic phase of stroke. Neuropsychiatric problems, such as PSDA, have a negative impact on social reintegration and quality of life. Currently, there is no evidence-based treatment available for reducing PSDA symptoms. In the recent literature on depression in the general population it has been shown that depression complaints can diminish by cognitive behavioural therapy (CBT). In the current study, the effectiveness of augmented, activation-based and individually tailored CBT on the reduction of depression and anxiety will be investigated in patients with PSDA. Additionally, the effects on various secondary outcome measures, such as quality of life, goal attainment and societal participation will be evaluated. This study is embedded in a consortium of 4 interrelated studies on quality of life after stroke (Restore4Stroke). METHODS/DESIGN: A multi-centre, assessor-blind, randomized controlled trial is conducted. A sample of 106 PSDA patients, as assessed with the Hospital Anxiety and Depression Scale (HADS depression subscale >7), will be recruited and randomly allocated to either an experimental or a control group. The experimental intervention consists of an augmented CBT intervention. The intervention is based on CBT principles of recognizing, registering, and altering negative thoughts and cognitions so that mood, and emotional symptoms are improved. CBT is augmented with direct in-vivo activation offered by occupational or movement therapists. Patients in the control group will receive a computerized cognitive training intervention. Outcomes will be assessed at baseline, immediately post intervention, and at 6 and 12 months follow up. DISCUSSION: This study is the first randomized clinical trial that evaluates the (maintenance of) effects of augmented CBT on post-stroke depression with or without anxiety symptoms. Together with three other projects, the Restore4Stroke PSDA trial will provide novel information about the (treatment of) emotional problems and quality of life after stroke. TRIAL REGISTRATION NUMBER: Dutch Trial Register NTR2999.

Physical training in boys with Duchenne Muscular Dystrophy: the protocol of the No Use is Disuse study.

BACKGROUND: "Use it or lose it" is a well known saying which is applicable to boys with Duchenne Muscular Dystrophy (DMD). Besides the direct effects of the muscular dystrophy, the increasing effort to perform activities, the fear of falling and the use of personal aids indirectly impair leg and arm functions as a result of disuse. Physical training could oppose this secondary physical deterioration. The No Use is Disuse (NUD) study is the first study in human subjects with DMD that will examine whether a low-intensity physical training is beneficial in terms of preservation of muscle endurance and functional abilities. The study consists of two training intervention studies: study 1 "Dynamic leg and arm training for ambulant and recently wheelchair-dependent boys with DMD and, study 2 "Functional training with arm support for boys with DMD who have been confined to a wheelchair for several years". This paper describes the hypotheses and methods of the NUD study. METHODS: Study 1 is an explorative randomized controlled trial with multiple baseline measurements. Thirty boys with a DNA-established diagnosis of DMD will be included. The intervention consists of a six-months physical training during which boys train their legs and arms with active and/or assisted cycling training equipment. The primary study outcomes are muscle endurance and functional abilities, assessed with a Six-Minute Bicycle Test and the Motor Function Measure. Study 2 has a within-group repeated measurements design and will include ten boys with DMD who have already been confined to a wheelchair for several years. The six-months physical training program consists of 1) a computer-assisted training and 2) a functional training with an arm support. The primary study outcome is functional abilities of the upper extremity, assessed with the Action Research Arm Test. DISCUSSION: The NUD study will fill part of the gap in the current knowledge about the possible effects of training in boys with DMD and will increase insight into what type of exercise should be recommended to boys with DMD. The study will finish at the end of 2010 and results are expected in 2011. TRIAL REGISTRATION: The Netherlands National Trial Register1631.