Final analysis of the pilot trial of diaphragm pacing in amyotrophic lateral sclerosis with long-term follow-up: diaphragm pacing positively affects diaphragm respiration

Onders, Raymond P.; Elmo, MaryJo; Kaplan, Cindy; Katirji, Bashar; Schilz, Robert

doi:10.1016/j.amjsurg.2013.08.039

Cited by 49 publications

(31 citation statements)

References 8 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…Successful cases have also been reported with patients with congenital hypoventilation [174,175,176] syndrome and other neurological diseases [177]. Some data originally suggested a possible benefit of phrenic nerve pacing in patients with ALS [178,179], but two recent, prospective randomized trials evaluating the effect of this intervention in this setting had to be prematurely stopped because of a decrease in survival in the treatment arms [180,181]. In all cases, evaluation for phrenic pacing should be performed in specialized centers with experience in the technique.…”

Section: Therapeutic Managementmentioning

confidence: 99%

Diaphragm Dysfunction: Diagnostic Approaches and Management Strategies

Dubé

Dres

2016

JCM

157

109

View full text Add to dashboard Cite

The diaphragm is the main inspiratory muscle, and its dysfunction can lead to significant adverse clinical consequences. The aim of this review is to provide clinicians with an overview of the main causes of uni- and bi-lateral diaphragm dysfunction, explore the clinical and physiological consequences of the disease on lung function, exercise physiology and sleep and review the available diagnostic tools used in the evaluation of diaphragm function. A particular emphasis is placed on the clinical significance of diaphragm weakness in the intensive care unit setting and the use of ultrasound to evaluate diaphragmatic action.

show abstract

Section: Therapeutic Managementmentioning

confidence: 99%

Diaphragm Dysfunction: Diagnostic Approaches and Management Strategies

Dubé

Dres

2016

JCM

157

109

View full text Add to dashboard Cite

show abstract

“…There are a few case reports of this, although the functional consequence of this is unknown should it occur. 29 Improved compliance of the lungs and prevention of basal collapse; restoration of the co-ordination of breathing, lost as a result of upper motor neurone dysfunction; and increased tidal volumes are other potential as yet unproven mechanisms. Unhelpfully, we can be no more certain as to why DP may be harmful.…”

Section: Understanding the Resultsmentioning

confidence: 99%

DiPALS: Diaphragm Pacing in patients with Amyotrophic Lateral Sclerosis – a randomised controlled trial

McDermott

Bradburn

Maguire

et al. 2016

Health Technol Assess

View full text Add to dashboard Cite

BackgroundAmyotrophic lateral sclerosis (ALS) is a neurodegenerative disease resulting in death, usually from respiratory failure, within 2–3 years of symptom onset. Non-invasive ventilation (NIV) is a treatment that when given to patients in respiratory failure leads to improved survival and quality of life. Diaphragm pacing (DP), using the NeuRx/4®diaphragm pacing system (DPS)™ (Synapse Biomedical, Oberlin, OH, USA), is a new technique that may offer additional or alternative benefits to patients with ALS who are in respiratory failure.ObjectiveThe Diaphragm Pacing in patients with Amyotrophic Lateral Sclerosis (DiPALS) trial evaluated the effect of DP on survival over the study duration in patients with ALS with respiratory failure.DesignThe DiPALS trial was a multicentre, parallel-group, open-label, randomised controlled trial incorporating health economic analyses and a qualitative longitudinal substudy.ParticipantsEligible participants had a diagnosis of ALS (ALS laboratory-supported probable, clinically probable or clinically definite according to the World Federation of Neurology revised El Escorial criteria), had been stabilised on riluzole for 30 days, were aged ≥ 18 years and were in respiratory failure. We planned to recruit 108 patients from seven UK-based specialist ALS or respiratory centres. Allocation was performed using 1 : 1 non-deterministic minimisation.InterventionsParticipants were randomised to either standard care (NIV alone) or standard care (NIV) plus DP using the NeuRX/4 DPS.Main outcome measuresThe primary outcome was overall survival, defined as the time from randomisation to death from any cause. Secondary outcomes were patient quality of life [assessed by European Quality of Life-5 Dimensions, three levels (EQ-5D-3L), Short Form questionnaire-36 items and Sleep Apnoea Quality of Life Index questionnaire]; carer quality of life (EQ-5D-3L and Caregiver Burden Inventory); cost–utility analysis and health-care resource use; tolerability and adverse events. Acceptability and attitudes to DP were assessed in a qualitative substudy.ResultsIn total, 74 participants were randomised into the trial and analysed, 37 participants to NIV plus pacing and 37 to standard care, before the Data Monitoring and Ethics Committee advised initial suspension of recruitment (December 2013) and subsequent discontinuation of pacing (on safety grounds) in all patients (June 2014). Follow-up assessments continued until the planned end of the study in December 2014. The median survival (interquartile range) was 22.5 months (lower quartile 11.8 months; upper quartile not reached) in the NIV arm and 11.0 months (6.7 to 17.0 months) in the NIV plus pacing arm, with an adjusted hazard ratio of 2.27 (95% confidence interval 1.22 to 4.25;p = 0.01).ConclusionsDiaphragmatic pacing should not be used as a routine treatment for patients with ALS in respiratory failure.Future workIt may be that certain population subgroups benefit from DP. We are unable to explain the mechanism behind the excess mortality in the pacing arm, something the small trial size cannot help address. Future research should investigate the mechanism by which harm or benefit occurs further.Trial registrationCurrent Controlled Trials ISRCTN53817913.FundingThis project was funded by the National Institute for Health Research Health Technology Assessment programme and will be published in full inHealth Technology Assessment; Vol. 20, No. 45. See the HTA programme website for further project information. Additional funding was provided by the Motor Neurone Disease Association of England, Wales and Northern Ireland.

show abstract

“…Diaphragm pacing is a newer, alternative means of supporting ventilation. Its effectiveness in prolonging survival in ALS, however, is unclear at this time (Gonzalez-Bermejo et al, 2012; Mahajan, Bach, Saporito, & Perez, 2012; Onders, Elmo, Kaplan, Katirji, & Schilz, 2014) and is the subject of ongoing research (McDermott et al, 2012). Additional preventive interventions include influenza and pneumococcal vaccination that are recommended for all people with ALS.…”

Section: Symptom Management As Als Progresses: Medications and Assistmentioning

confidence: 99%

Comprehensive rehabilitative care across the spectrum of amyotrophic lateral sclerosis

Paganoni

Karam

Joyce

et al. 2015

NRE

View full text Add to dashboard Cite

Background Amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease) is a neurodegenerative disease that results in progressive muscle weakness and wasting. There is no known cure and the disease is uniformly fatal. Purpose This review discusses current concepts in ALS care, from breaking the diagnosis to end-of-life care. People with ALS have several multidisciplinary needs due to a complex and dynamic disease process. They benefit from rehabilitation interventions that are individualized and have the goal of optimizing independence, function, and safety. These strategies also help minimize symptomatic burden and maximize quality of life. Conclusion Patient-centered, multidisciplinary care has a significant impact on the life of people with ALS and is the current standard of care for this patient population.

show abstract

Final analysis of the pilot trial of diaphragm pacing in amyotrophic lateral sclerosis with long-term follow-up: diaphragm pacing positively affects diaphragm respiration

Cited by 49 publications

References 8 publications

Diaphragm Dysfunction: Diagnostic Approaches and Management Strategies

Diaphragm Dysfunction: Diagnostic Approaches and Management Strategies

DiPALS: Diaphragm Pacing in patients with Amyotrophic Lateral Sclerosis – a randomised controlled trial

Comprehensive rehabilitative care across the spectrum of amyotrophic lateral sclerosis

Contact Info

Product

Resources

About