Nightime NIV Initiation in Amyotrophic Lateral Sclerosis in an Outpatient Setting

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

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.

Study Overview

• Status: Completed
• Conditions: Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Procedure: Nightime NIV initiation

Detailed Description

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.

• Study Type: Interventional
• Enrollment (Actual): 50
• Phase: Not Applicable

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: inpatients group; 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; In-hospital vs home
Experimental: outpatients group; 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; In-hospital vs home

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Fondazione Salvatore Maugeri
• Collaborators: Fondazione Don Carlo Gnocchi Onlus
• Investigators: Principal Investigator: Enrica Bertella, MD, Fondazione Salvatore Maugeri

Study record dates

Study Major Dates

• Study Start: March 1, 2011
• Primary Completion (Actual): March 1, 2013
• Study Completion (Actual): March 1, 2014

Study Registration Dates

• First Submitted: April 27, 2016
• First Submitted That Met QC Criteria: April 28, 2016
• First Posted (Estimate): May 3, 2016

Study Record Updates

• Last Update Posted (Estimate): May 4, 2016
• Last Update Submitted That Met QC Criteria: May 3, 2016
• Last Verified: April 1, 2016

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Sclerosis; Motor Neuron Disease; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: CTS 02/2011

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO