Noninvasive ventilation use by patients enrolled in VITALITY-ALS

Area of Science:

• Neurology
• Pulmonology
• Clinical Trials

Background:

• Noninvasive ventilation (NIV) is a critical intervention for managing respiratory insufficiency in amyotrophic lateral sclerosis (ALS).
• Understanding prescribing patterns and patient compliance with NIV is essential for optimizing care in ALS clinical trials.

Purpose of the Study:

• To evaluate the prescribing practices and patient compliance of noninvasive ventilation (NIV) during the VITALITY-ALS clinical trial.
• To identify reasons for NIV prescription and assess its utilization in relation to respiratory function decline.

Main Methods:

• The VITALITY-ALS trial randomized 565 patients with slow vital capacity (SVC) ≥70% to placebo or tirasemtiv.
• Physicians prescribed NIV without restriction; reasons, initiation dates, and compliance (hours/day) were recorded.
• Slow vital capacity (SVC) and ALSFRS-R were assessed throughout the 56-week follow-up.

Main Results:

• Of 565 patients, 195 (34.5%) were prescribed NIV, with high compliance rates (≥2h/24h: 78.5%, ≥4h/24h: 71.3%).
• Common reasons for NIV prescription included declining vital capacity, respiratory, and sleep symptoms.
• Only 68.2% of patients experiencing an SVC decline below 50% were prescribed NIV, with similar rates in North America and Europe.

Conclusions:

• NIV underutilization was observed in ALS patients during the VITALITY-ALS trial, even with declining respiratory function.
• Prescribing patterns showed regional differences between North America and Europe.
• The potential underuse of NIV may affect survival outcomes in ALS patients, including those in clinical trials.