Nightime NIV Initiation in Amyotrophic Lateral Sclerosis in an Outpatient Setting

Study Overview
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Brief Summary
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In patients with amyotrophic lateral sclerosis (ALS), non-invasive mechanical ventilation (NIV) is usually initiated in an in-hospital regime. The investigators evaluated if NIV initiated in an outpatient setting can be as effective as regards patients' adherence. The investigators also evaluated factors predicting NIV adherence and disease progression.

Detailed Description
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All patients with a definite ALS diagnosis, aged > 18 years, referred to the ALS outpatient clinics of the Fondazione Salvatore Maugeri Institute of Lumezzane (Brescia) and Istituto Don Gnocchi Onlus (Milano), Italy, for respiratory functional assessments for the purpose of early initiation of NIV were considered. Patients who were in clinically stable condition and without chest infections during the previous 3 months were considered eligible for the study. Between March 2011 and March 2014, during an outpatient visit, the patients were randomized 1:1 to two groups for NIV initiation: outpatient vs. inpatient protocol. In both groups a diurnal NIV initiation trial was performed using pressure-support ventilators in spontaneous/timed mode with a preset tidal volume (300 ml/kg) and a fixed back-up respiratory rate (12 breaths/min). The trial included: choice of the mask, setting of ventilator pressure, at least 2 hours of NIV under supervision. Then, the patients underwent the nighttime NIV initiation trial for a maximum of 20 nights according to the outpatient vs. inpatient protocol. NIV trial was stopped when: 1) patients used NIV > 4 hours/night for 3 consecutive nights, 2) patients and caregivers were able to manage with ventilator, 3) patients failed after 10 consecutive educational sessions. At enrollment (T0), the end of NIV initiation trial (T1) and after 3 months from the end of the trial (T2) respiratory function tests, blood gas analysis, and sleep study were performed. At T1 the investigators assessed NIV acceptance (as a minimum of 3 consecutive days >4 h/night), and dyspnea symptoms by VAS (day/night), staff and patients' satisfaction. At T2: NIV adherence (>120 h/month) and patients' satisfaction.

Official Title
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Nightime Noninvasive Mechanical Ventilation Initiation in Amyotrophic Lateral Sclerosis in an Outpatient Setting. A Non-inferiority Study.

Conditions
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Amyotrophic Lateral Sclerosis

Intervention / Treatment
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* Procedure: Nightime NIV initiation


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: consecutive patients definite ALS diagnosis aged > 18 years in clinically stable condition referred to the ALS outpatient clinics of the Fondazione Salvatore Maugeri Institute of Lumezzane (Brescia) and the Istituto Don Gnocchi Onlus (Milano), Italy for respiratory functional assessment for the purpose of early initiation of NIV no chest infections during the previous 3 months. Exclusion Criteria: cognitive impairment refusal to participate severe comorbidities and contraindications to NIV (arrhythmias, cardiac failure, history of pneumothorax) distance from hospital > 40 km, travel problems to attend the outpatient clinic

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: inpatients group<br>In-hospital nightime NIV initiation. For the inpatients Group, NIV was initiated in the respiratory wards of the two hospitals and continued during night for a minimum of 4 hours/night. Inpatients had a 24-h availability of health staff care. During the night, they had nurses and physicians available on-hand. | Procedure: Nightime NIV initiation<br>* In-hospital vs home<br>|
| Experimental: outpatients group<br>Home nightime NIV initiation. For the outpatients group, diurnal NIV was initiated during a scheduled visit in a hospital dedicated room(at least 4 hours/day of care), the trial proceeded at home during the night with a personal caregiver. A minimum of 4 hours/night was required. No support during the night was provided to these patients. | Procedure: Nightime NIV initiation<br>* In-hospital vs home<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Adherence as measured by the use of ventilator (hours/month) | Evaluation after 3 months from the end of NIV initiation trial (T2) | At 3 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Changes in respiratory function as assessed by spirometry | Evaluation at 3 months from the end of NIV initiation trial (T2) | At 3 months | 
| Changes in dyspnea symptoms as assessed by Visual Analogue Scale | Evaluation at 3 months from the end of NIV initiation trial (T2) | At 3 months | 
| Patient's questionnaire of satisfaction | Evaluation at the end of NIV initiation trial (T1) | At 20 days | 
| Changes in respiratory function as assessed by Maximal inspiratory pressure/Maximal expiratory pressure | Evaluation at 3 months from the end of NIV initiation trial (T2) | At 3 months | 
| Changes in respiratory function as assessed by blood gas analysis | Evaluation at 3 months from the end of NIV initiation trial (T2) | At 3 months |