The Effect of Long-Term Non-Invasive Ventilation on Tracheostomy-Free Survival and Hospitalizations in Types 2 and 3 Spinal Muscular Atrophy Patients

: Background: The impact of Long-Term Non-Invasive Ventilation (LT-NIV) on patient-relevant outcomes has yet to be clearly established in types 2 and 3 Spinal Muscular Atrophy (SMA). Objectives: The current study aimed to assess LT-NIV effect(s) on tracheostomy-free survival and the need for hospitalization. Its secondary aim was to identify patient characteristics that can be considered risk factors for an unsatisfactory response to treatment. Design: This study is a retrospective analysis of long-term outcomes in types 2 and 3 SMA patients receiving home LT-NIV. Methods:Patients and interventions: Eighteen types 2 and 3 SMA patients who were receiving home LT-NIV between 1 January 1990 and 31 March 2025 were included. Primary study endpoint: The endpoint comprised a composite of tracheostomy-free survival time and hospitalization rate. Results: Twelve patients (66.7%) had a positive response to LT-NIV (group A); the response was unsatisfactory in the remaining 6 (33.3%) (group B). Tracheostomy-free survival time was significantly reduced in the latter compared to the former [66 (range: 2-172) vs. 280 (range: 67-407) months; p = 0.010] and the hospitalization rate was significantly higher [0.35 (range: 0.06-1.44) vs. 0.07 (range: 0.00-0.18) episodes/yr; p = 0.007]. A correlation was found between an unsatisfactory response to LT-NIV and treatment initiation following an episode of acute respiratory failure (OR: 7.90; 95% CI, 0.99-123.2; p = 0.051). Conclusions: LT-NIV has a positive impact on tracheostomy-free survival and hospitalizations in types 2 and 3 SMA patients. The risk of an unsatisfactory response appears to be higher when the treatment is initiated in clinically unstable patients.