Douglas McKim | University of Ottawa | Université d'Ottawa (original) (raw)

Papers by Douglas McKim

Chest, 2003

PURPOSE: Despite limited research and large cost differences, rollator-style walkers and oxygen a... more PURPOSE: Despite limited research and large cost differences, rollator-style walkers and oxygen are both used to improve the ability to walk in people with mildly hypoxemia (PO 2 55-65 mmHg) and exertional desaturation. The purpose of this trial was to compare rollator-style walkers to oxygen on the six minute walk test. METHODS: A single-blind randomized controlled crossover trial was used to assess the endpoints of distance walked, oxygen saturation, perceived exertion, and speed on the six-minute walk test. Open-and closed-ended questions were used to elicit the subject's values and feelings towards the interventions. RESULTS: Eleven participants were recruited from a tertiary rehabilitation centre from May to December 2001. The patients mean age (± standard deviation) was 67.5 ± 16.6 years, FEV 1.0 (percent predicted) was 26.1 ± 6.8 %, and PaO 2 was 61.2 ± 2.3 mmHg. There were no significant period by treatment interactions, or period effects on any outcome. There was no significant difference between walkers and oxygen on distance or speed walked, but oxygen outperformed walkers on final oxygen saturation and perceived exertion. The mean difference between interventions was 22.2 (95% CI:-76.7 to +32.4) metres for distance, 7.8 (5.0 to 10.6) percent for oxygen saturation, 1.3 (95% CI 0.1 to2.4) points for exertional desaturation, and 0.2 (95% CI-0.13 to 0.08) metres per second for speed. Oxygen (51.2 (95% CI 18.6 to 84.0) metres), but not walkers (31.4 (95% CI-3.4 to 66.2) metres), improved distance walked compared to unassisted walks. Participants indicated that both interventions improved their walking but more preferred the walker. CONCLUSIONS: Despite not correcting exertional desaturation or reducing perceived exertion, walkers still resulted in similar distance walked compared to oxygen.

Digital health, 2022

IntroductionFamily caregivers play an important role supporting the day-to-day needs of ventilato... more IntroductionFamily 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.MethodsA 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.ResultsIn 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.DiscussionOur 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.

Canadian journal of respiratory, critical care, and sleep medicine, May 4, 2021

capacity (FVC; 43.7 vs. 49.1% in the ventilated group and the control group, respectively) and FE... more capacity (FVC; 43.7 vs. 49.1% in the ventilated group and the control group, respectively) and FEV 1 (28.2 vs. 28.5%). At year 0, the ventilated group had significantly greater declines in FVC (-3.6 8 9.2 vs. +0.8 8 8.9%, p = 0.03) and in FEV 1 (-3.0 8 6.7 vs. +2.6 8 4.4, p ! 0.0001). At year +1, the decreases in FVC (-2.1 8 10.0 vs.-2.2 8 9.9%) and in FEV 1 (-2.2 8 6.7 vs.-2.3 8 6.2%) were similar in both groups. Conclusions: These data show that NPPV is associated with stabilization of the decrease in lung function in patients with advanced CF.

Muscle & Nerve, Dec 21, 2021

IntroductionLaryngospasm is an involuntary, sustained closure of sphincter musculature that leads... more IntroductionLaryngospasm 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.MethodsThe 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.ResultsLaryngospasm 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.DiscussionDespite 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.

Paediatrics and Child Health, Jun 14, 2017

No studies have explored the experiences of Canadian mechanical ventilator-assisted adolescents (... more No studies have explored the experiences of Canadian mechanical ventilator-assisted adolescents (VAAs) living at home as they transition from paediatric to adult health providers. A better understanding of the needs of this growing population is essential to provide transition services responsive to VAAs and caregiver-identified needs. Methods: We conducted semistructured telephone interviews with adolescents and family caregivers who had recently initiated or completed transition to adult care recruited from three Canadian university-affiliated paediatric home ventilation programs. We analyzed transcripts using a theoretical framework for understanding facilitators and barriers to transition. Results: We interviewed 18 individuals representing 14 episodes of paediatric to adult transition. Participants identified early planning, written informational materials and joint paediatric-adult provider-family transition meetings as facilitators of care transition to adult services and providers. Barriers included insufficient information, limited access to interprofessional (nursing and allied health) providers and reduced funding or health services. Barriers resulted in service disruption and a sense of 'medical homelessness'. While most families related a positive transition to a new 'medical home', families caring for VAAs with moderate-to-severe cognitive and/or physical dependence more commonly reported transition difficulties. Conclusions: Important opportunities exist to enable improvements in the transition experiences of VAAs and their family caregivers. To maximize service continuity during paediatric to adult transition, future research should focus on transition navigator roles, interprofessional health outreach and the needs of families caring for VAAs with cognitive and physical deficits.

The Lancet Respiratory Medicine, Apr 1, 2022

The life-limiting complications of Duchenne muscular dystrophy (DMD) include loss of lung functio... more 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.

ERJ Open Research, 2017

The aim of the present study was to determine whether lung volume recruitment (LVR) acutely incre... more 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 cmH 2 O. 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 cmH 2 O versus 109±10 mL•cmH 2 O −1 , p<0.001). Immediately after LVR, Crs increased by 39.5±9.8% to 50±7 mL•cmH 2 O −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. @ERSpublications Acute changes following lung volume recruitment http://ow.ly/2dqE308g3oG Cite this article as: Molgat-Seon Y, Hannan LM, Dominelli PB, et al. Lung volume recruitment acutely increases respiratory system compliance in individuals with severe respiratory muscle weakness.

American Journal of Respiratory and Critical Care Medicine, Sep 1, 2005

Restricting oxygen administration to those who benefit is desirable. Objective: To determine the ... more Restricting oxygen administration to those who benefit is desirable. Objective: To determine the impact of alternative strategies for assessing eligibility for domiciliary oxygen on funded oxygen use, quality of life, and costs. Methods: We randomized applicants for domiciliary oxygen therapy to an assessment system that relied on data collected by oxygen providers at the time of application and judgments by Home Oxygen Program personnel (conventional assessment) or to a system of data collection by a respiratory therapist that included, in patients unstable at the time of initial assessment, a repeat assessment after 2 months of stability (alternative assessment). Measurements and Main Results: A total of 276 applicants were allocated to the conventional arm and 270 to the alternative assessment. In the year after application, oxygen use was lower in the alternative arm with no between-group differences in mortality, quality of life, or resource use in the community. Although alternative assessment applicants had on average higher assessment costs by 155perapplicant,thesecostsweremorethanoffsetbydecreasedHomeOxygenProgramcostsof155 per applicant, these costs were more than offset by decreased Home Oxygen Program costs of 155perapplicant,thesecostsweremorethanoffsetbydecreasedHomeOxygenProgramcostsof596 per applicant using Canadian cost weights. The comparable U.S. dollar figures were 309and309 and 309and432, respectively, and the difference in cost between strategies was therefore smaller using U.S. cost weights. Conclusions: Reassessment of applicants for domiciliary oxygen after several months of stability identifies an appreciable portion of initially eligible patients who are no longer eligible, thus reducing program costs to public funders without adverse consequences on quality of life, mortality, or other resource use.

The European respiratory journal, 2005

The original chronic respiratory questionnaire (CRQ), one of the most widely used measures of hea... more The original chronic respiratory questionnaire (CRQ), one of the most widely used measures of health-related quality of life (HRQL) in chronic respiratory disease (CRD), is traditionally interviewer administered (IA) and includes an individualised dyspnoea domain. The present authors studied the impact of self-administered (SA) and standardised dyspnoea questions on CRQ measurement properties. In a factorial design multicentre trial, 177 patients with CRD (mean age 67.7 yrs; mean forced expiratory volume in one second per cent predicted 44.6%) were randomised to CRQ-IA (n586) or CRQ-SA (n591), and to initially complete the standardised or individualised items before and after respiratory rehabilitation. While maintaining validity, the CRQ-SA proved more responsive to changes in HRQL than the CRQ-IA in all domains. Compared with the standardised dyspnoea domain, the individualised dyspnoea domain indicated greater responsiveness. The correlations of baseline scores and change scores with other HRQL instruments indicated good validity of the CRQ-SA. In conclusion, self-administration and standardisation of the chronic respiratory questionnaire maintains validity and responsiveness relative to the interviewer-administered chronic respiratory questionnaire. These results challenge the assumption that interviewer-administered questionnaires are superior to self-administered questionnaires in older patients with chronic respiratory disease.

Pediatric Pulmonology, 2020

Gene therapy is an attractive approach being intensively studied to prevent muscle deterioration ... more 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.

Introduction The care of patients with amyotrophic lateral sclerosis (ALS) is a very challenging ... more Introduction The care of patients with amyotrophic lateral sclerosis (ALS) is a very challenging but truly rewarding role for respiratory specialists. ALS is an incurable neurodegenerative disease affecting both upper and lower motor neurons, resulting in progressive skeletal muscle weakness. At onset, it generally affects predominantly either limb or bulbar muscles. Rarely, its first presentation can be respiratory failure secondary to early respiratory muscle involvement.

PLOS ONE, 2019

Background Population trends of disease prevalence and incidence over time measure burden of dise... more 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.

Respiratory Care, 2016

BACKGROUND: Noninvasive ventilation (NIV) is commonly used to provide ventilatory support for ind... more BACKGROUND: Noninvasive ventilation (NIV) is commonly used to provide ventilatory support for individuals with amyotrophic lateral sclerosis (ALS). Once 24-h ventilation is required, the decision between invasive tracheostomy ventilation and palliation is often faced. This study describes the use and outcomes of daytime mouthpiece ventilation added to nighttime mask ventilation for continuous NIV in subjects with ALS as an effective alternative. METHODS: This was a retrospective study of 39 subjects with ALS using daytime mouthpiece ventilation over a 17-y period. RESULTS: Thirty-one subjects were successful with mouthpiece ventilation, 2 were excluded, 2 stopped because of lack of motivation, and 4 with bulbar subscores of the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (b-ALSFRS-R) between 0 and 3 physically failed to use it consistently. No subject in the successful group had a b-ALSFRS-R score of <6. Thirty of the successful subjects were able to generate a maximum insufflation capacity ؊ vital capacity difference with lung volume recruitment. The median (range) survival to tracheostomy or death from initiation of nocturnal NIV and mouthpiece ventilation were 648 (176-2,188) and 286 (41-1,769) d, respectively. Peak cough flow with lung-volume recruitment >180 L/min at initiation of mouthpiece ventilation was associated with a longer survival (637 ؎ 468 vs 240 ؎ 158 d (P ‫؍‬ .01). CONCLUSIONS: Mouthpiece ventilation provides effective ventilation and prolonged survival for individuals with ALS requiring full-time ventilatory support and maintaining adequate bulbar function.

Cmaj Canadian Medical Association Journal Journal De L Association Medicale Canadienne, Feb 15, 1992

Canadian respiratory journal : journal of the Canadian Thoracic Society

No national Canadian data define resource requirements and care delivery for ventilator-assisted ... more No national Canadian data define resource requirements and care delivery for ventilator-assisted individuals (VAIs) requiring long-term institutional care. Such data will assist in planning health care services to this population. To describe institutional and patient characteristics, prevalence, equipment used, care elements and admission barriers for VAIs requiring long-term institutional care. Centres were identified from a national inventory and snowball referrals. The survey weblink was provided from December 2012 to April 2013. Weekly reminders were sent for six weeks. The response rate was 84% (54 of 64), with 44 adult and 10 pediatric centres providing data for 428 VAIs (301 invasive ventilation; 127 noninvasive ventilation [NIV]), equivalent to 1.3 VAIs per 100,000 population. An additional 106 VAIs were on wait lists in 18 centres. More VAIs with progressive neuromuscular disease received invasive ventilation than NIV (P<0.001); more VAIs with chronic obstructive pulmon...

Canadian respiratory journal : journal of the Canadian Thoracic Society

Various terms, including 'prolonged mechanical ventilation' (PMV) and 'long-term mech... more Various terms, including 'prolonged mechanical ventilation' (PMV) and 'long-term mechanical ventilation' (LTMV), are used interchangeably to distinguish patient cohorts requiring ventilation, making comparisons and timing of clinical decision making problematic. To develop expert, consensus-based criteria associated with care transitions to distinguish cohorts of ventilated patients. A four-round (R), web-based Delphi study with consensus defined as >70% was performed. In R1, participants listed, using free text, criteria perceived to should and should not define seven transitions. Transitions comprised: T1 - acute ventilation to PMV; T2 - PMV to LTMV; T3 - PMV or LTMV to acute ventilation (reverse transition); T4 - institutional to community care; T5 - no ventilation to requiring LTMV; T6 - pediatric to adult LTMV; and T7 - active treatment to end-of-life care. Subsequent Rs sought consensus. Experts from intensive care (n=14), long-term care (n=14) and home vent...

Chest, 2003

PURPOSE: Despite limited research and large cost differences, rollator-style walkers and oxygen a... more PURPOSE: Despite limited research and large cost differences, rollator-style walkers and oxygen are both used to improve the ability to walk in people with mildly hypoxemia (PO 2 55-65 mmHg) and exertional desaturation. The purpose of this trial was to compare rollator-style walkers to oxygen on the six minute walk test. METHODS: A single-blind randomized controlled crossover trial was used to assess the endpoints of distance walked, oxygen saturation, perceived exertion, and speed on the six-minute walk test. Open-and closed-ended questions were used to elicit the subject's values and feelings towards the interventions. RESULTS: Eleven participants were recruited from a tertiary rehabilitation centre from May to December 2001. The patients mean age (± standard deviation) was 67.5 ± 16.6 years, FEV 1.0 (percent predicted) was 26.1 ± 6.8 %, and PaO 2 was 61.2 ± 2.3 mmHg. There were no significant period by treatment interactions, or period effects on any outcome. There was no significant difference between walkers and oxygen on distance or speed walked, but oxygen outperformed walkers on final oxygen saturation and perceived exertion. The mean difference between interventions was 22.2 (95% CI:-76.7 to +32.4) metres for distance, 7.8 (5.0 to 10.6) percent for oxygen saturation, 1.3 (95% CI 0.1 to2.4) points for exertional desaturation, and 0.2 (95% CI-0.13 to 0.08) metres per second for speed. Oxygen (51.2 (95% CI 18.6 to 84.0) metres), but not walkers (31.4 (95% CI-3.4 to 66.2) metres), improved distance walked compared to unassisted walks. Participants indicated that both interventions improved their walking but more preferred the walker. CONCLUSIONS: Despite not correcting exertional desaturation or reducing perceived exertion, walkers still resulted in similar distance walked compared to oxygen.

Digital health, 2022

IntroductionFamily caregivers play an important role supporting the day-to-day needs of ventilato... more IntroductionFamily 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.MethodsA 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.ResultsIn 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.DiscussionOur 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.

Canadian journal of respiratory, critical care, and sleep medicine, May 4, 2021

capacity (FVC; 43.7 vs. 49.1% in the ventilated group and the control group, respectively) and FE... more capacity (FVC; 43.7 vs. 49.1% in the ventilated group and the control group, respectively) and FEV 1 (28.2 vs. 28.5%). At year 0, the ventilated group had significantly greater declines in FVC (-3.6 8 9.2 vs. +0.8 8 8.9%, p = 0.03) and in FEV 1 (-3.0 8 6.7 vs. +2.6 8 4.4, p ! 0.0001). At year +1, the decreases in FVC (-2.1 8 10.0 vs.-2.2 8 9.9%) and in FEV 1 (-2.2 8 6.7 vs.-2.3 8 6.2%) were similar in both groups. Conclusions: These data show that NPPV is associated with stabilization of the decrease in lung function in patients with advanced CF.

Muscle & Nerve, Dec 21, 2021

IntroductionLaryngospasm is an involuntary, sustained closure of sphincter musculature that leads... more IntroductionLaryngospasm 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.MethodsThe 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.ResultsLaryngospasm 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.DiscussionDespite 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.

Paediatrics and Child Health, Jun 14, 2017

No studies have explored the experiences of Canadian mechanical ventilator-assisted adolescents (... more No studies have explored the experiences of Canadian mechanical ventilator-assisted adolescents (VAAs) living at home as they transition from paediatric to adult health providers. A better understanding of the needs of this growing population is essential to provide transition services responsive to VAAs and caregiver-identified needs. Methods: We conducted semistructured telephone interviews with adolescents and family caregivers who had recently initiated or completed transition to adult care recruited from three Canadian university-affiliated paediatric home ventilation programs. We analyzed transcripts using a theoretical framework for understanding facilitators and barriers to transition. Results: We interviewed 18 individuals representing 14 episodes of paediatric to adult transition. Participants identified early planning, written informational materials and joint paediatric-adult provider-family transition meetings as facilitators of care transition to adult services and providers. Barriers included insufficient information, limited access to interprofessional (nursing and allied health) providers and reduced funding or health services. Barriers resulted in service disruption and a sense of 'medical homelessness'. While most families related a positive transition to a new 'medical home', families caring for VAAs with moderate-to-severe cognitive and/or physical dependence more commonly reported transition difficulties. Conclusions: Important opportunities exist to enable improvements in the transition experiences of VAAs and their family caregivers. To maximize service continuity during paediatric to adult transition, future research should focus on transition navigator roles, interprofessional health outreach and the needs of families caring for VAAs with cognitive and physical deficits.

The Lancet Respiratory Medicine, Apr 1, 2022

The life-limiting complications of Duchenne muscular dystrophy (DMD) include loss of lung functio... more 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.

ERJ Open Research, 2017

The aim of the present study was to determine whether lung volume recruitment (LVR) acutely incre... more 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 cmH 2 O. 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 cmH 2 O versus 109±10 mL•cmH 2 O −1 , p<0.001). Immediately after LVR, Crs increased by 39.5±9.8% to 50±7 mL•cmH 2 O −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. @ERSpublications Acute changes following lung volume recruitment http://ow.ly/2dqE308g3oG Cite this article as: Molgat-Seon Y, Hannan LM, Dominelli PB, et al. Lung volume recruitment acutely increases respiratory system compliance in individuals with severe respiratory muscle weakness.

American Journal of Respiratory and Critical Care Medicine, Sep 1, 2005

Restricting oxygen administration to those who benefit is desirable. Objective: To determine the ... more Restricting oxygen administration to those who benefit is desirable. Objective: To determine the impact of alternative strategies for assessing eligibility for domiciliary oxygen on funded oxygen use, quality of life, and costs. Methods: We randomized applicants for domiciliary oxygen therapy to an assessment system that relied on data collected by oxygen providers at the time of application and judgments by Home Oxygen Program personnel (conventional assessment) or to a system of data collection by a respiratory therapist that included, in patients unstable at the time of initial assessment, a repeat assessment after 2 months of stability (alternative assessment). Measurements and Main Results: A total of 276 applicants were allocated to the conventional arm and 270 to the alternative assessment. In the year after application, oxygen use was lower in the alternative arm with no between-group differences in mortality, quality of life, or resource use in the community. Although alternative assessment applicants had on average higher assessment costs by 155perapplicant,thesecostsweremorethanoffsetbydecreasedHomeOxygenProgramcostsof155 per applicant, these costs were more than offset by decreased Home Oxygen Program costs of 155perapplicant,thesecostsweremorethanoffsetbydecreasedHomeOxygenProgramcostsof596 per applicant using Canadian cost weights. The comparable U.S. dollar figures were 309and309 and 309and432, respectively, and the difference in cost between strategies was therefore smaller using U.S. cost weights. Conclusions: Reassessment of applicants for domiciliary oxygen after several months of stability identifies an appreciable portion of initially eligible patients who are no longer eligible, thus reducing program costs to public funders without adverse consequences on quality of life, mortality, or other resource use.

The European respiratory journal, 2005

The original chronic respiratory questionnaire (CRQ), one of the most widely used measures of hea... more The original chronic respiratory questionnaire (CRQ), one of the most widely used measures of health-related quality of life (HRQL) in chronic respiratory disease (CRD), is traditionally interviewer administered (IA) and includes an individualised dyspnoea domain. The present authors studied the impact of self-administered (SA) and standardised dyspnoea questions on CRQ measurement properties. In a factorial design multicentre trial, 177 patients with CRD (mean age 67.7 yrs; mean forced expiratory volume in one second per cent predicted 44.6%) were randomised to CRQ-IA (n586) or CRQ-SA (n591), and to initially complete the standardised or individualised items before and after respiratory rehabilitation. While maintaining validity, the CRQ-SA proved more responsive to changes in HRQL than the CRQ-IA in all domains. Compared with the standardised dyspnoea domain, the individualised dyspnoea domain indicated greater responsiveness. The correlations of baseline scores and change scores with other HRQL instruments indicated good validity of the CRQ-SA. In conclusion, self-administration and standardisation of the chronic respiratory questionnaire maintains validity and responsiveness relative to the interviewer-administered chronic respiratory questionnaire. These results challenge the assumption that interviewer-administered questionnaires are superior to self-administered questionnaires in older patients with chronic respiratory disease.

Pediatric Pulmonology, 2020

Gene therapy is an attractive approach being intensively studied to prevent muscle deterioration ... more 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.

Introduction The care of patients with amyotrophic lateral sclerosis (ALS) is a very challenging ... more Introduction The care of patients with amyotrophic lateral sclerosis (ALS) is a very challenging but truly rewarding role for respiratory specialists. ALS is an incurable neurodegenerative disease affecting both upper and lower motor neurons, resulting in progressive skeletal muscle weakness. At onset, it generally affects predominantly either limb or bulbar muscles. Rarely, its first presentation can be respiratory failure secondary to early respiratory muscle involvement.

PLOS ONE, 2019

Background Population trends of disease prevalence and incidence over time measure burden of dise... more 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.

Respiratory Care, 2016

BACKGROUND: Noninvasive ventilation (NIV) is commonly used to provide ventilatory support for ind... more BACKGROUND: Noninvasive ventilation (NIV) is commonly used to provide ventilatory support for individuals with amyotrophic lateral sclerosis (ALS). Once 24-h ventilation is required, the decision between invasive tracheostomy ventilation and palliation is often faced. This study describes the use and outcomes of daytime mouthpiece ventilation added to nighttime mask ventilation for continuous NIV in subjects with ALS as an effective alternative. METHODS: This was a retrospective study of 39 subjects with ALS using daytime mouthpiece ventilation over a 17-y period. RESULTS: Thirty-one subjects were successful with mouthpiece ventilation, 2 were excluded, 2 stopped because of lack of motivation, and 4 with bulbar subscores of the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (b-ALSFRS-R) between 0 and 3 physically failed to use it consistently. No subject in the successful group had a b-ALSFRS-R score of <6. Thirty of the successful subjects were able to generate a maximum insufflation capacity ؊ vital capacity difference with lung volume recruitment. The median (range) survival to tracheostomy or death from initiation of nocturnal NIV and mouthpiece ventilation were 648 (176-2,188) and 286 (41-1,769) d, respectively. Peak cough flow with lung-volume recruitment >180 L/min at initiation of mouthpiece ventilation was associated with a longer survival (637 ؎ 468 vs 240 ؎ 158 d (P ‫؍‬ .01). CONCLUSIONS: Mouthpiece ventilation provides effective ventilation and prolonged survival for individuals with ALS requiring full-time ventilatory support and maintaining adequate bulbar function.

Cmaj Canadian Medical Association Journal Journal De L Association Medicale Canadienne, Feb 15, 1992

Canadian respiratory journal : journal of the Canadian Thoracic Society

No national Canadian data define resource requirements and care delivery for ventilator-assisted ... more No national Canadian data define resource requirements and care delivery for ventilator-assisted individuals (VAIs) requiring long-term institutional care. Such data will assist in planning health care services to this population. To describe institutional and patient characteristics, prevalence, equipment used, care elements and admission barriers for VAIs requiring long-term institutional care. Centres were identified from a national inventory and snowball referrals. The survey weblink was provided from December 2012 to April 2013. Weekly reminders were sent for six weeks. The response rate was 84% (54 of 64), with 44 adult and 10 pediatric centres providing data for 428 VAIs (301 invasive ventilation; 127 noninvasive ventilation [NIV]), equivalent to 1.3 VAIs per 100,000 population. An additional 106 VAIs were on wait lists in 18 centres. More VAIs with progressive neuromuscular disease received invasive ventilation than NIV (P<0.001); more VAIs with chronic obstructive pulmon...

Canadian respiratory journal : journal of the Canadian Thoracic Society

Various terms, including 'prolonged mechanical ventilation' (PMV) and 'long-term mech... more Various terms, including 'prolonged mechanical ventilation' (PMV) and 'long-term mechanical ventilation' (LTMV), are used interchangeably to distinguish patient cohorts requiring ventilation, making comparisons and timing of clinical decision making problematic. To develop expert, consensus-based criteria associated with care transitions to distinguish cohorts of ventilated patients. A four-round (R), web-based Delphi study with consensus defined as >70% was performed. In R1, participants listed, using free text, criteria perceived to should and should not define seven transitions. Transitions comprised: T1 - acute ventilation to PMV; T2 - PMV to LTMV; T3 - PMV or LTMV to acute ventilation (reverse transition); T4 - institutional to community care; T5 - no ventilation to requiring LTMV; T6 - pediatric to adult LTMV; and T7 - active treatment to end-of-life care. Subsequent Rs sought consensus. Experts from intensive care (n=14), long-term care (n=14) and home vent...