Diaphragm Pacing in motor neurone disease/ Amyotrophic Lateral Sclerosis

Conditions

Motor neurone disease Nervous System Diseases Motor neuron disease

McDermott CJ, Bradburn MJ, Maguire C, Cooper CL, Baird WO, Baxter SK, Cohen J, Cantrill H, Dixon S, Ackroyd R, Baudouin S, Bentley A, Berrisford R, Bianchi S, Bourke SC, Darlison R, Ealing J, Elliott M, Fitzgerald P, Galloway S, Hamdalla H, Hanemann CO, Hughes P, Imam I, Karat D, Leek R, Maynard N, Orrell RW, Sarela A, Stradling J, Talbot K, Taylor L, Turner M, Simonds AK, Williams T, Wedzicha W, Young C, Shaw PJ.

2016-06-0112 citations

10.3310/hta20450

Protocol for diaphragm pacing in patients with respiratory muscle weakness due to motor neurone disease (DiPALS): a randomised controlled trial.

McDermott CJ, Maguire C, Cooper CL, Ackroyd R, Baird WO, Baudouin S, Bentley A, Bianchi S, Bourke S, Bradburn MJ, Dixon S, Ealing J, Galloway S, Karat D, Maynard N, Morrison K, Mustfa N, Stradling J, Talbot K, Williams T, Shaw PJ.

2012-08-166 citations

10.1186/1471-2377-12-74

Interventions

54 participants to be recruited to each of the 2 arms. Arm 1 = Standard care alone (NIV), Arm 2 = Standard care (NIV) plus DP NIV arm patients will attend clinic for initiation of NIV with a possib

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Age 18 or older 2. Familial or sporadic MND/ALS diagnosed as laboratory-supported probable, probable, or definite according to the World Federation of Neurology El Escorial criteria 3. Stabilised on Riluzole therapy 4. Respiratory insufficiency as determined by one or more of: 4.1. Forced Vital Capacity (FVC) less than 75% predicted/Sniff Nasal Inspiratory Pressure (SNIP) less than 40 cmH2O 4.2. Supine vital capacity (VC) less than 75% of sitting or standing VC 4.3. Partial pressure of carbon dioxide in the blood (PaCO2) > 6kPa (daytime) 4.4. Significant overnight oxygen (O2) desaturation (>5% of night with Sp02 50% predicted or sniff nasal inspiratory pressure (SNIP) > 30 cmH2O in patients unable to perform FVC (bulbar patients)

Exclusion criteria

Exclusion criteria: 1. Prior NIV prescription 2. Pre-existing implanted electrical device such as pacemaker or cardiac defibrillator 3. Underlying cardiac, pulmonary diseases or other disorders that would affect pulmonary tests independently of MND/ALS or would increase the risk of general anaesthesia 4. Current pregnancy or breastfeeding 5. Significant decision making incapacity preventing informed consent by the subject due to a major mental disorder such as major depression or schizophrenia or dementia 6. Marked obesity affecting surgical access to diaphragm or significant scoliosis/ chest wall deformity 7. The involvement in any respiratory trial that can influence the safety or outcome measures of this study within three months of the planned implantation of the device or during the year of follow up 8. Pre-existing diaphragm abnormality such as a hiatus hernia or paraoesophageal hernia of abdominal contents ascending into the thoracic cavity

Design outcomes

Primary

Measure	Time frame
The primary objective of this trial will be to evaluate the effect of Diaphragm Pacing (DP) on survival over the study duration in patients with MND/ amyotrophic lateral sclerosis (ALS) with respiratory muscle weakness.	—

Secondary

Measure	Time frame
To evaluate the effect of DP on: Efficacy endpoints 1. Quality adjusted life years (QALYs) as calculated by combining EQ-5D and mortality data 2. Quality of life: sleep apnoea quality of life index (SAQLI), and SF-36 3. Quality of life of the main carer of the patient (Caregiver Burden Inventory) For each efficacy endpoint, the treatment effect will be assessed by analysing the difference between groups over the 12-month follow-up period, and the difference at 12 months. Safety endpoints Safety (adverse events) and tolerability (patient withdrawal from treatment)	—

Measure

Time frame

To evaluate the effect of DP on: Efficacy endpoints 1. Quality adjusted life years (QALYs) as calculated by combining EQ-5D and mortality data 2. Quality of life: sleep apnoea quality of life index (SAQLI), and SF-36 3. Quality of life of the main carer of the patient (Caregiver Burden Inventory) For each efficacy endpoint, the treatment effect will be assessed by analysing the difference between groups over the 12-month follow-up period, and the difference at 12 months. Safety endpoints Safety (adverse events) and tolerability (patient withdrawal from treatment)

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Countries

United Kingdom

Outcome results

None listed