A Randomized Controlled Clinical Trial of Lung Volume Recruitment in Adults with Neuromuscular Disease.

Rationale: Clinical care guidelines advise that lung volume recruitment (LVR) be performed routinely by people with neuromuscular disease (NMD) to maintain lung and chest wall flexibility and slow lung function decline. However, the evidence base is limited, and no randomized controlled trials of regular LVR in adults have been published. Objectives: To evaluate the effect of regular LVR on respiratory function and quality of life in adults with NMD. Methods: A randomized controlled trial with assessor blinding was conducted between September 2015 and May 2019. People (>14 years old) with NMD and vital capacity <80% predicted were eligible, stratified by disease subgroup (amyotrophic lateral sclerosis/motor neuron disease or other NMDs), and randomized to 3 months of twice-daily LVR or breathing exercises. The primary outcome was change in maximum insufflation capacity (MIC) from baseline to 3 months, analyzed using a linear mixed model approach. Results: Seventy-six participants (47% woman; median age, 57 [31-68] years; mean baseline vital capacity, 40 ± 18% predicted) were randomized (LVR, n = 37). Seventy-three participants completed the study. There was a statistically significant difference in MIC between groups (linear model interaction effect P = 0.002, observed mean difference, 0.19 [0.00-0.39] L). MIC increased by 0.13 (0.01-0.25) L in the LVR group, predominantly within the first month. No interaction or treatment effects were observed in secondary outcomes of lung volumes, respiratory system compliance, and quality of life. No adverse events were reported. Conclusions: Regular LVR increased MIC in a sample of LVR-naive participants with NMD. We found no direct evidence that regular LVR modifies respiratory mechanics or slows the rate of lung volume decline. The implications of increasing MIC are unclear, and the change in MIC may represent practice. Prospective long-term clinical cohorts with comprehensive follow-up, objective LVR use, and clinically meaningful outcome data are needed. Clinical trial registered with anzctr.org.au (ACTRN12615000565549).

Respiratory Management of Patients With Neuromuscular Weakness: An American College of Chest Physicians Clinical Practice Guideline and Expert Panel Report.

BACKGROUND: Respiratory failure is a significant concern in neuromuscular diseases (NMDs). This CHEST guideline examines the literature on the respiratory management of patients with NMD to provide evidence-based recommendations. STUDY DESIGN AND METHODS: An expert panel conducted a systematic review addressing the respiratory management of NMD and applied the Grading of Recommendations, Assessment, Development, and Evaluations approach for assessing the certainty of the evidence and formulating and grading recommendations. A modified Delphi technique was used to reach a consensus on the recommendations. RESULTS: Based on 128 studies, the panel generated 15 graded recommendations, one good practice statement, and one consensus-based statement. INTERPRETATION: Evidence of best practices for respiratory management in NMD is limited and is based primarily on observational data in amyotrophic lateral sclerosis. The panel found that pulmonary function testing every 6 months may be beneficial and may be used to initiate noninvasive ventilation (NIV) when clinically indicated. An individualized approach to NIV settings may benefit patients with chronic respiratory failure and sleep-disordered breathing related to NMD. When resources allow, polysomnography or overnight oximetry can help to guide the initiation of NIV. The panel provided guidelines for mouthpiece ventilation, transition to home mechanical ventilation, salivary secretion management, and airway clearance therapies. The guideline panel emphasizes that NMD pathologic characteristics represent a diverse group of disorders with differing rates of decline in lung function. The clinician's role is to add evaluation at the bedside to shared decision-making with patients and families, including respect for patient preferences and treatment goals, considerations of quality of life, and appropriate use of available resources in decision-making.

Understanding the experiences of lung volume recruitment among boys with Duchenne muscular dystrophy: A multicenter qualitative study.

BACKGROUND: Despite recommendations for regular lung volume recruitment (LVR) use in clinical practice guidelines for children with neuromuscular disease, adherence to LVR is poor. We aimed to describe the experience of LVR by boys with Duchenne muscular dystrophy (DMD), their families, and healthcare providers (HCPs), as well as to identify the barriers and facilitators to LVR use. METHODS: This multicenter, qualitative study evaluated boys with DMD (n = 11) who used twice-daily LVR as part of a randomized controlled trial, as well as their parents (n = 11), and HCPs involved in the clinical use of LVR (n = 9). Semistructured interviews were conducted to identify participants' understanding of LVR therapy and their beliefs, barriers and facilitators to its use. Thematic analysis was conducted using an inductive approach. A subanalysis compared adherent and nonadherent children. RESULTS: Seven themes were identified related to participants' beliefs and experiences with LVR: emotional impact, adaptation to LVR, perceived benefits of LVR, routine, family engagement, clinical resources, and equipment-related factors. Strategies to improve adherence were also identified, including education, reinforcement and demonstration of LVR benefit, as well as clinician support. There were no thematic differences between adherent and nonadherent children. DISCUSSION: Despite the benefits of LVR and positive experiences with it by many families, there remain barriers to adherence to treatment. HCPs need to balance the need for early introduction to give families time to adapt to LVR while ensuring that the benefit of LVR outweighs the burden. Clinician support is important for family engagement.

The experience of family caregivers of ventilator-assisted individuals who participated in a pilot web-based peer support program: A qualitative study.

Introduction: Family caregivers play an important role supporting the day-to-day needs of ventilator-assisted individuals (VAIs) living at home. Peer-to-peer communication can help support these caregivers and help them sustain caregiving in the community. Online peer-support has been suggested as a way to help meet caregivers' support needs. Methods: A qualitative descriptive approach was used to elicit the perspectives of support received from caregivers who participated in a pilot web-based peer support program from October to December 2018. Data were collected through the transcripts of weekly online peer-to-peer group chats. Data were analyzed using an integration of thematic and framework analysis. Results: In total, eight caregivers and five peer mentors participated in the pilot. All five mentors and four of the caregivers participated in the weekly chats. We identified three themes, a) The experience of caregivers is characterized by unique challenges related to the complexity of VAI care including technology; b) Mentors and caregiver participants reciprocally share support; c) Despite hardships, there are things that make caregiving easier and joyful. Discussion: Our results add to the growing body of evidence pointing to the importance of online communities for supporting vulnerable caregivers. The reciprocal element of peer support, where trained mentors and untrained participants both benefit from support, can help sustain peer-support interventions. Despite the challenges of providing care to a VAI, there are facilitators that may help ease the caregiving experience and caregivers can benefit from ongoing support that is tailored to their needs along the caregiving trajectory.

Cardiorespiratory management of Duchenne muscular dystrophy: emerging therapies, neuromuscular genetics, and new clinical challenges.

The life-limiting complications of Duchenne muscular dystrophy (DMD) include loss of lung function and progressive cardiomyopathy; when patients are treated with assisted ventilation, cardiac function becomes the main determinant of survival. Therapy for DMD is changing rapidly, with the emergence of new genetic and molecular therapeutic options, the proliferation of which has fostered the perception that DMD is a potentially curable disease. However, data for respiratory and cardiac outcomes are scarce and available evidence is not uniformly positive. Patients who share a dystrophin (DMD) genotype can have highly divergent cardiorespiratory phenotypes; genetic modifiers of DMD gene expression are a probable cause of respiratory and cardiac phenotypic variability and discordance. In this Personal View, we provide an overview of new and emerging DMD therapies, highlighting the limitations of current research and considering strategies to incorporate cardiorespiratory assessments into clinical trials. We explore how genetic modifiers could be used to predict cardiorespiratory natural history and how manipulation of such modifiers might represent a promising therapeutic strategy. Finally, we examine the changing role of respiratory physicians, cardiologists, and intensive care clinicians on the frontline of a challenging new clinical landscape.

Laryngospasm in amyotrophic lateral sclerosis.

INTRODUCTION: Laryngospasm is an involuntary, sustained closure of sphincter musculature that leads to an unpleasant subjective experience of dyspnea and choking. It is an underreported symptom in amyotrophic lateral sclerosis (ALS). In this study we aimed to better characterize the prevalence and clinical characteristics of laryngospasm in ALS patients. METHODS: The medical records of 571 patients with ALS followed between 2008 and 2018 were searched for evidence of laryngospasm. A total of 23 patients with laryngospasm were identified and the data related to patient and laryngospasm characteristics were extracted. RESULTS: Laryngospasm was reported in 4% of ALS patients. Females comprised 57% of patients and their mean age was 63.4 years. Laryngospasm frequently manifested in patients with moderate bulbar dysfunction and seemed independent of respiratory function. Among laryngospasm patients, 26% were cigarette smokers and 13% had a history of gastroesophageal reflux. The most common reported trigger was excessive saliva irritating the vocal cords (35%) followed by eating a meal (17%). There was significant variation in laryngospasm frequency (up to 5 per hour) and duration (seconds to minutes). Most patients could not identify an effective coping mechanism, although 13% reported that drinking water was effective. DISCUSSION: Despite its low prevalence in ALS, laryngospasm should be included in the symptom inquiry. The present findings may improve patient care through increased recognition of the clinical features of laryngospasm in ALS patients, identifying a link between laryngospasm and moderate bulbar dysfunction, and highlighting trigger avoidance as a management strategy. Additional research is required to understand the pathophysiology and optimal treatment.

Objective measurement of lung volume recruitment therapy: laboratory and clinical validation.

Lung volume recruitment manoeuvres are often prescribed to maintain respiratory health in neuromuscular disease. Unfortunately, no current system accurately records delivered dose. This study determined the performance characteristics of a novel, objective, manual lung volume recruitment bag counter ('the counter') with bench and healthy volunteer testing, as well as in individuals with neuromuscular disease. We undertook (1) bench test determination of activation threshold, (2) bench and healthy volunteer fidelity testing during simulated patient interface leak and different pressure compressions and (3) comparisons with self-report in individuals with neuromuscular disease. The data are reported as summary statistics, compression counts, percentage of recorded versus delivered compressions and concordance (Cohen's kappa (K) and absolute agreement). RESULTS: Minimum counter activation pressure under conditions of zero leak was 1.9±0.4 cm H2O. No difference was observed between the number of repetitions delivered and recorded during high airway pressure condition. Interface leak approximating 25% resulted in underestimation of repetition counts, and once the leak was at 50% or beyond, the counter recorded no activity. Faster sampling frequency collected data with more fidelity. Counter data agreed with diary self-report during community trials (16 participants, 960 participant days, 77% agreement, Cohen's Κ=0.66 and p<0.001). Disagreement typically favoured more diary reported (18%) than counter (5%) sessions. CONCLUSIONS: The performance characteristics of a new lung volume recruitment counter have been established in both laboratory and community settings. Objective usage and dosage data should accelerate new knowledge development and better translation of lung volume recruitment therapy into policy and practice.

The effect of emerging molecular and genetic therapies on cardiopulmonary disease in Duchenne muscular dystrophy.

Gene therapy is an attractive approach being intensively studied to prevent muscle deterioration in patients with Duchenne muscular dystrophy. While clinical trials are only in early stages, initial reports are promising for its effects on ambulation. Cardiopulmonary failure, however, is the most common cause of mortality in Duchenne muscular dystrophy (DMD) patients, and little is known regarding the prospects for gene therapy on alleviating DMD-associated cardiomyopathy and respiratory failure. Here we review current knowledge regarding effects of gene therapy on DMD cardiomyopathy and discuss respiratory endpoints that should be considered as outcome measures in future clinical trials.

Healthcare utilization and costs of pediatric home mechanical ventilation in Canada.

BACKGROUND: Children using home mechanical ventilation (HMV) live at home with better quality of life, despite financial burden for their family. Previous studies of healthcare utilization and costs have not considered public and private expenditures, including family caregiver time. Our objective was to examine public and private healthcare utilization and costs for children using HMV, and variables associated with highest costs. METHODS: Longitudinal, prospective, observational cost analysis study (2012-2014) collecting data on public and private (out-of-pocket, third-party insurance, and caregiving) costs every 2 weeks for 6 months using the Ambulatory Home Care Record. Functional Independence Measure (FIM), WeeFIM, and Caregiving Impact Scale (CIS) were measured at baseline and study completion. Regression modeling examined a priori selected variables associated with monthly costs using Andersen and Newman's framework for healthcare utilization, relevant literature, and clinical expertise. Data are reported in 2015 Canadian dollars ($1CAD = $0.78USD). RESULTS: Forty two children and their caregivers were enrolled. Overall median (interquartile range) monthly healthcare cost was $12 131 ($8159-$15 958) comprising $9929 (89%) family caregiving hours, $996 (9%) publicly funded, and $252 (2%) out-of-pocket (<1% third-party insurance) costs. With higher FIM score (lower dependency), median costs were reduced by 4.5% (95% confidence interval: 8.3%-0.5%), adjusted for age, sex, tracheostomy, and daily ventilation duration. Note: since the three cost categories did not sum to the total statistically derived median cost, the percentage of each category used the sum of median public + caregiver lost time + private out-of-pocket + third-party insurance as the denominator. CONCLUSIONS: For HMV children, most healthcare costs were due to family caregiving costs. More dependent children incur highest costs. The financial burden to family caregivers is substantial and needs to considered in future policy decisions related to pediatric HMV.

The Oral Secretion Scale and Prognostic Factors for Survival in Subjects With Amyotrophic Lateral Sclerosis.

BACKGROUND: Noninvasive ventilation (NIV) can be tolerated in patients with amyotrophic lateral sclerosis (ALS), unless bulbar impairment becomes severe. Excessive oral secretions may result in NIV intolerance and insufficient ventilation. OBJECTIVE: To assess the reliability of the Oral Secretion Scale (OSS) for predicting the tolerance of NIV, when to initiate hospice or transition to tracheostomy, and prognostic factors for survival of users of NIV. METHODS: A validated OSS was developed to measure oral secretions in correlation with the ability to swallow saliva and clear the upper airway: OSS score of 4 = normal (automatic swallow); OSS score of 3 = infrequent secretions (automatic swallow decreased); OSS score of 2 = occasional drooling and/or pooling (conscious swallow required); OSS score of 1 = severe, frequent drooling and/or pooling (conscious swallow difficult); OSS score of 0 = most severe, constant drooling/pooling (conscious swallow impossible). A total of 137 subjects were followed up prospectively during ongoing patient visits from NIV initiation until death or tracheostomy. Survival was calculated by using Kaplan-Meier analysis. OSS scores when NIV became intolerable were determined. Uni- and/or multivariate Cox-regression analyses showed prognostic factors that affect survival. RESULTS: The median months of survival from NIV initiation were the following: 11 (95% CI 7.3-14.0), 5 (95% CI 3.1-6.1), and 1 (95% CI 1.1-1.5) stratified by OSS scores of 4, 3-2, and 1, respectively; and 21 (95% CI 8.6-33.2), 8 (95% CI 3.4-11.5), 6 (95% CI 4.2-8.2), and 2 (95% CI 1.5-2.7) stratified by 24, 17-23, 4-16, and <4 h/d of NIV use, respectively. Survival was significantly (P < .001) longer with an OSS score of 4 than an OSS score of 1 at NIV initiation; and significantly (P < .001) longer when NIV used 24 h/d than <24 h/d. In the subjects unable to tolerate NIV, ≥80% had OSS score of 1 or 0. Univariate and multivariate analyses hazard ratio 4.91, 95% CI 2.98-8.09, P < .001, and hazard ratio 4.60, 95% CI 2.66-7.96, P < .001), respectively, showed hours per day of NIV use was a significant factor associated with survival. CONCLUSIONS: The subjects with an OSS score of 4 tolerated NIV and survived significantly longer than subjects with an OSS score of <4. An OSS score of 1 signaled NIV intolerance and the need for hospice or transition to planned tracheostomy. Use of OSS can help guide NIV management decisions.

Trends in incidence, prevalence, and mortality of neuromuscular disease in Ontario, Canada: A population-based retrospective cohort study (2003-2014).

BACKGROUND: Population trends of disease prevalence and incidence over time measure burden of disease and inform healthcare planning. Neuromuscular disorders (NMD) affect muscle and nerve function with varying degrees of severity and disease progression. OBJECTIVE: Using health administrative databases we described trends in incidence, prevalence, and mortality of adults and children with NMD. We also explored place of death and use of palliative care. METHODS: Population-based (Ontario, Canada) cohort study (2003 to 2014) of adults and children with NMD identified using International Classification of Disease and health insurance billing codes within administrative health databases. RESULTS: Adult disease prevalence increased on average per year by 8% (95% confidence interval (CI) 6% to 10%, P <.001), with the largest increase in adults18-39 years. Childhood disease prevalence increased by 10% (95% CI 8% to 11%, P <.0001) per year, with the largest increase in children 0 to 5 years. Prevalence increased across all diagnoses except amyotrophic lateral sclerosis and spinal muscular atrophy for adults and all diagnoses for children. Adult incidence decreased by 3% (95% CI -4% to -2%, P <.0001) but incidence remained stable in children. Death occurred in 34,336 (18.5%) adults; 21,236 (61.8%) of whom received palliative care. Death occurred in 1,009 (5.6%) children; 507 (50.2%) of whom received palliative care. Mortality decreased over time in adults (odds ratio (OR) 0.86, 95% CI 0.86-0.87, P <.0001) and children (OR 0.79, 95% CI 0.76-0.82, P <.0001). Use of palliative care over time increased for adults (OR 1.18, 95% CI 1.09 to 1.28, P <.0001) and children (OR 1.22, 95% CI 1.20 to 1.23, P <.0001). CONCLUSIONS: In both adults and children, NMD prevalence is rising and mortality rates are declining. In adults incidence is decreasing while in children it remains stable. This confirms on a population-based level the increased survival of children and adults with NMD.

Development of a Web-Based Peer Support Program for Family Caregivers of Ventilator-Assisted Individuals Living in the Community: Protocol for a Pilot Randomized Controlled Trial.

BACKGROUND: Across Europe, Canada, Australia, and the United States, the prevalence of home mechanical ventilation (HMV) prevalence is 6.6-12.9 per 100,000. At-home ventilator-assisted individuals (VAIs) are often vulnerable and highly comorbid, requiring complex care. In Canada, most VAI care is provided by family, leading to poor health-related quality of life and increased caregiver burden. No supportive interventions or peer support programs are tailored to VAI caregivers. Owing to the financial, geographic, and time limitations, Web-based support delivery may especially meet VAI family caregiver needs. We have developed a peer mentor training and Web-based peer support program for VAI caregivers including information-sharing, peer-to-peer communication, and peer mentorship. OBJECTIVE: Study Stage 1 aims to (1) evaluate the face and content validity of the peer mentor training program and (2) investigate participant satisfaction. Study Stage 2 aims to evaluate (1) the feasibility of participant recruitment and Web-based program delivery; (2) acceptability, usability, and satisfactoriness; (3) experiences of caregivers and peer mentors with the Web-based peer support program; and (4) effect of the Web-based peer support program on caregiver health outcomes. METHODS: Study Stage 1: We will train 7 caregivers to act as peer mentors for the Web-based peer support program trial; they will complete questionnaires rating the utility of individual training sessions and the training program overall. Study Stage 2: We will recruit 30 caregiver peers for a pilot randomized controlled trial of the 12-week Web-based peer support program using a waitlist control; the program includes private chat, a public discussion forum, and weekly moderated chats. Caregiver peers will be randomized to the intervention or waitlist control group using a 1:1 ratio using Randomize.net. Both groups will complete pre- and postintervention health outcome questionnaires (ie, caregiving impact, mastery, coping, personal gain, positive affect, and depression). Caregiver peers in the intervention arm will only complete a program evaluation and will be invited to participate in an interview to provide insight into their experience. Peer mentors will be invited to participate in a Web-based focus group to provide insight into their experience as mentors. We will judge the feasibility per the number of recruitment and program delivery goals met, use analysis of covariance to compare health outcomes between intervention and control groups, and analyze qualitative data thematically. RESULTS: Peer mentor training was completed with 5 caregivers in July 2018. To date, 2 caregivers have beta-tested the website, and the Web-based peer support program trial will commence in September 2018. Results are expected by early 2019. CONCLUSIONS: This study will result in the production and initial evaluation of a rigorously developed, evidence- and stakeholder-informed Web-based peer training and peer support program for caregivers of VAIs residing at home. INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID): PRR1-10.2196/11827.

Monitoring Cough Effectiveness and Use of Airway Clearance Strategies: A Canadian and UK Survey.

BACKGROUND: Regular monitoring combined with early and appropriate use of airway clearance can reduce unplanned hospital admissions for patients with neuromuscular disease (NMD) and spinal cord injury (SCI). We aimed to describe and compare knowledge of guidelines, monitoring of cough effectiveness, clinician prescription/provision of airway clearance strategies, and service provision constraints in the United Kingdom and Canada. METHODS: This was a cross-sectional survey of clinicians affiliated with NMD and SCI clinics in Canada, 2016 attendees at the Home Mechanical Ventilation Conference in the United Kingdom, and United Kingdom physiotherapist networks. RESULTS: We received 155 surveys (92 from Canada; 63 from the United Kingdom). More UK respondents (76%) were aware of airway clearance guidelines than Canadian (56%) respondents (P = .02). Routine assessment of cough effectiveness was reported by more UK respondents (59%) than Canadian (42%) respondents (P = .044). Cough peak flow (CPF) was the most common method used in both countries, although it was more commonly used in the UK (96%) than in Canada (81%, P = .02). Fewer Canadian respondents reported using CPF before initiation of airway clearance (81% vs 94%, P = .046), and fewer Canadian respondents showed results to patients for technique feedback (76% vs 97%, P = .007). Similar participant numbers reported using CPF after initiation to ensure adequate technique (73% vs 72%, P = .92). Mechanical insufflation-exsufflation (MI-E) + lung volume recruitment (LVR) + manually assisted cough when CPF ≤ 270 L/min was most routinely recommended (41% overall). Monotherapy was infrequent (LVR 15%, manually assisted cough 7%, and MI-E 4%). More Canadians identified constraints on service provision, specifically insufficient public funding for equipment (68% vs 39%, P = .002) and inadequate community workers' knowledge (56% vs 34%, P = .002). Funding for community support was a common constraint in both countries (49% vs 42%). CONCLUSIONS: The somewhat variable cough effectiveness monitoring and airway clearance practices identified in this survey confirm the need for further work on knowledge translation related to guideline recommendations and the need to address common constraints to optimal service delivery.

Respiratory health service utilization of children with neuromuscular disease.

OBJECTIVES: To quantify health service utilization including assessment, monitoring, and treatment of respiratory complications of children with neuromuscular disease (NMD), identifying practice variation and adherence to guideline recommendations at a population level. METHODS: North American population-based cohort study (2003-2015) of children with NMD using hospital diagnostic and physician billing codes within health administrative databases. RESULTS: We identified 18 163 children with NMD. Mean (SD) age was 7.8 (5.6) years with 40% ≤5; 45% were female. Most common diagnoses were cerebral palsy (50%) and spina bifida (16%); 8% had muscular dystrophy. From fiscal years 2003-2014, 15 600 (86%) children went to an emergency department on average 3.5 times every 3 years; 6575 (36%) for respiratory reasons. 8788 (48%) were admitted to hospital with 2190 (12%) for respiratory reasons and 2451 (13%) required intensive care. Respiratory specialist outpatient visits occurred for 2226 (12%) children on average 6.5 visits every 3 years; 723 (4%) had in-hospital respiratory specialist consultation. Pulmonary function testing was conducted in 3194 (18%) children on average 2.4 times every 3 years; sleep studies in 1389 (8%). CONCLUSION: In this population-based study of children with NMD, healthcare utilization for respiratory complications was considerable. Frequency of respiratory specialist consultation, monitoring of respiratory function and sleep disordered breathing was variable but on average reflected professional society recommendations. Children with NMD are frequent ED users suggesting a need to improve community and social supports. We did not detect reduced access to respiratory monitoring or specialist consultation in adolescents transitioning to adult services.

Patterns of healthcare utilisation for respiratory complications of adults with neuromuscular disease: a population study.

Our objective was to quantify health service utilisation including monitoring and treatment of respiratory complications for adults with neuromuscular disease (NMD), identifying practice variation and adherence to guideline recommendations at a population level.We conducted a population-based longitudinal cohort study (2003-2015) of adults with NMD using hospital diagnostic and health insurance billing codes within administrative health databases.We identified 185 586 adults with NMD. Mean age 52 years, 59% female. 41 173 (22%) went to an emergency department for respiratory complications on average 1.6 times every 3 years; 14 947 (8%) individuals were admitted to hospital 1.4 times every 3 years. Outpatient respiratory specialist visits occurred for 64 084 (35%) with four visits every 3 years, although substantial variation in visit frequency was found. 157 285 (85%) went to the emergency department (all-cause) almost 4 times every 3 years, 100 052 (54%) were admitted to hospital. Individuals with amyotrophic lateral sclerosis/motor neurone disease (ALS/MND) had more emergency department visits compared with other types of NMD (p<0.0001).One-third of adults with NMD received respiratory specialist care at a frequency recommended by professional guidelines, although substantial variation exists. Emergent healthcare utilisation was substantial, emphasising the burden of NMD on the healthcare system and urgent need to improve community and social supports, particularly for ALS/MND patients.

Healthcare utilisation and costs of home mechanical ventilation.

BACKGROUND: Individuals using home mechanical ventilation (HMV) frequently choose to live at home for quality of life, despite financial burden. Previous studies of healthcare utilisation and costs do not consider public and private expenditures, including caregiver time. OBJECTIVES: To determine public and private healthcare utilisation and costs for HMV users living at home in two Canadian provinces, and examine factors associated with higher costs. METHODS: Longitudinal, prospective observational cost analysis study (April 2012 to August 2015) collecting data on public and private (out-of-pocket, third-party insurance, caregiving) costs every 2 weeks for 6 months using the Ambulatory and Home Care Record. Functional Independence Measure (FIM) was used at baseline and study completion. Regression models examined variables associated with total monthly costs selected a priori using Andersen and Newman's framework for healthcare utilisation, relevant literature, and clinical expertise. Data are reported in 2015 Canadian dollars ($C1=US$0.78=£0.51=€0.71). RESULTS: We enrolled 134 HMV users; 95 with family caregivers. Overall median (IQR) monthly healthcare cost was $5275 ($2291-$10 181) with $2410 (58%) publicly funded; $1609 (39%) family caregiving; and $141 (3%) out-of-pocket (<1% third-party insurance). Median healthcare costs were $8733 ($5868-$15 274) for those invasively ventilated and $3925 ($1212-$7390) for non-invasive ventilation. Variables associated with highest monthly costs were amyotrophic lateral sclerosis (1.88, 95% CI 1.09 to 3.26, P<0.03) and lower FIM quintiles (higher dependency) (up to 6.98, 95% CI 3.88 to 12.55, P<0.0001) adjusting for age, sex, tracheostomy and ventilation duration. CONCLUSIONS: For HMV users, most healthcare costs were publicly supported or associated with family caregiving. Highest costs were incurred by the most dependent users. Understanding healthcare costs for HMV users will inform policy decisions to optimise resource allocation, helping individuals live at home while minimising caregiver burden.

Lung volume recruitment acutely increases respiratory system compliance in individuals with severe respiratory muscle weakness.

The aim of the present study was to determine whether lung volume recruitment (LVR) acutely increases respiratory system compliance (Crs) in individuals with severe respiratory muscle weakness (RMW). Individuals with RMW resulting from neuromuscular disease or quadriplegia (n=12) and healthy controls (n=12) underwent pulmonary function testing and the measurement of Crs at baseline, immediately after, 1 h after and 2 h after a single standardised session of LVR. The LVR session involved 10 consecutive supramaximal lung inflations with a manual resuscitation bag to the highest tolerable mouth pressure or a maximum of 50 cmH2O. Each LVR inflation was followed by brief breath-hold and a maximal expiration to residual volume. At baseline, individuals with RMW had lower Crs than controls (37±5 cmH2O versus 109±10 mL·cmH2O-1, p<0.001). Immediately after LVR, Crs increased by 39.5±9.8% to 50±7 mL·cmH2O-1 in individuals with RMW (p<0.05), while no significant change occurred in controls (p=0.23). At 1 h and 2 h post-treatment, there were no within-group differences in Crs compared to baseline (all p>0.05). LVR had no significant effect on measures of pulmonary function at any time point in either group (all p>0.05). During inflations, mean arterial pressure decreased significantly relative to baseline by 10.4±2.8 mmHg and 17.3±3.0 mmHg in individuals with RMW and controls, respectively (both p<0.05). LVR acutely increases Crs in individuals with RMW. However, the high airway pressures during inflations cause reductions in mean arterial pressure that should be considered when applying this technique.

Cough augmentation techniques for extubation or weaning critically ill patients from mechanical ventilation.

BACKGROUND: There are various reasons why weaning and extubation failure occur, but ineffective cough and secretion retention can play a significant role. Cough augmentation techniques, such as lung volume recruitment or manually- and mechanically-assisted cough, are used to prevent and manage respiratory complications associated with chronic conditions, particularly neuromuscular disease, and may improve short- and long-term outcomes for people with acute respiratory failure. However, the role of cough augmentation to facilitate extubation and prevent post-extubation respiratory failure is unclear. OBJECTIVES: Our primary objective was to determine extubation success using cough augmentation techniques compared to no cough augmentation for critically-ill adults and children with acute respiratory failure admitted to a high-intensity care setting capable of managing mechanically-ventilated people (such as an intensive care unit, specialized weaning centre, respiratory intermediate care unit, or high-dependency unit).Secondary objectives were to determine the effect of cough augmentation techniques on reintubation, weaning success, mechanical ventilation and weaning duration, length of stay (high-intensity care setting and hospital), pneumonia, tracheostomy placement and tracheostomy decannulation, and mortality (high-intensity care setting, hospital, and after hospital discharge). We evaluated harms associated with use of cough augmentation techniques when applied via an artificial airway (or non-invasive mask once extubated/decannulated), including haemodynamic compromise, arrhythmias, pneumothorax, haemoptysis, and mucus plugging requiring airway change and the type of person (such as those with neuromuscular disorders or weakness and spinal cord injury) for whom these techniques may be efficacious. SEARCH METHODS: We searched the Cochrane Central Register of Controlled Trials (CENTRAL; Issue 4, 2016), MEDLINE (OvidSP) (1946 to April 2016), Embase (OvidSP) (1980 to April 2016), CINAHL (EBSCOhost) (1982 to April 2016), and ISI Web of Science and Conference Proceedings. We searched the PROSPERO and Joanna Briggs Institute databases, websites of relevant professional societies, and conference abstracts from five professional society annual congresses (2011 to 2015). We did not impose language or other restrictions. We performed a citation search using PubMed and examined reference lists of relevant studies and reviews. We contacted corresponding authors for details of additional published or unpublished work. We searched for unpublished studies and ongoing trials on the International Clinical Trials Registry Platform (apps.who.int/trialsearch) (April 2016). SELECTION CRITERIA: We included randomized and quasi-randomized controlled trials that evaluated cough augmentation compared to a control group without this intervention. We included non-randomized studies for assessment of harms. We included studies of adults and of children aged four weeks or older, receiving invasive mechanical ventilation in a high-intensity care setting. DATA COLLECTION AND ANALYSIS: Two review authors independently screened titles and abstracts identified by our search methods. Two review authors independently evaluated full-text versions, independently extracted data and assessed risks of bias. MAIN RESULTS: We screened 2686 citations and included two trials enrolling 95 participants and one cohort study enrolling 17 participants. We assessed one randomized controlled trial as being at unclear risk of bias, and the other at high risk of bias; we assessed the non-randomized study as being at high risk of bias. We were unable to pool data due to the small number of studies meeting our inclusion criteria and therefore present narrative results rather than meta-analyses. One trial of 75 participants reported that extubation success (defined as no need for reintubation within 48 hours) was higher in the mechanical insufflation-exsufflation (MI-E) group (82.9% versus 52.5%, P < 0.05) (risk ratio (RR) 1.58, 95% confidence interval (CI) 1.13 to 2.20, very low-quality evidence). No study reported weaning success or reintubation as distinct from extubation success. One trial reported a statistically significant reduction in mechanical ventilation duration favouring MI-E (mean difference -6.1 days, 95% CI -8.4 to -3.8, very low-quality evidence). One trial reported mortality, with no participant dying in either study group. Adverse events (reported by two trials) included one participant receiving the MI-E protocol experiencing haemodynamic compromise. Nine (22.5%) of the control group compared to two (6%) MI-E participants experienced secretion encumbrance with severe hypoxaemia requiring reintubation (RR 0.25, 95% CI 0.06 to 1.10). In the lung volume recruitment trial, one participant experienced an elevated blood pressure for more than 30 minutes. No participant experienced new-onset arrhythmias, heart rate increased by more than 25%, or a pneumothorax.For outcomes assessed using GRADE, we based our downgrading decisions on unclear risk of bias, inability to assess consistency or publication bias, and uncertainty about the estimate of effect due to the limited number of studies contributing outcome data. AUTHORS' CONCLUSIONS: The overall quality of evidence on the efficacy of cough augmentation techniques for critically-ill people is very low. Cough augmentation techniques when used in mechanically-ventilated critically-ill people appear to result in few adverse events.