Non‐invasive and tracheostomy invasive ventilation in amyotrophic lateral sclerosis: Utilization and survival rates in a cohort study over 12 years in Germany

Abstract

Background and purpose The aim of this study was to investigate utilization rates, treatment pathways and survival prognosis in patients with amyotrophic lateral sclerosis (ALS) undergoing non‐invasive (NIV) and tracheostomy invasive ventilation (TIV) in a real‐world setting. Methods A prospective cohort study using a single‐centre register of 2702 ALS patients (2007 to 2019) was conducted. Utilization of NIV/TIV and survival data were analysed in three cohorts: (i) non‐NIV; (ii) NIV (NIV without subsequent TIV); and (iii) TIV (including TIV preceded by NIV). Results A total of 1720 patients with available data were identified, 72.0% of whom (n = 1238) did not receive ventilation therapy. NIV was performed in 20.8% of patients (n = 358). TIV was performed in 9.5% of patients (n = 164), encompassing both primary TIV (7.2%, n = 124) and TIV with preceding NIV (2.3%, n = 40). TIV was more often utilized without previous NIV (25.7% vs. 8.3% of all ventilated patients), demonstrating that primary TIV was the prevailing pathway for invasive ventilation. The median (range) survival was significantly longer in the NIV cohort (40.8 [37.2–44.3] months) and the TIV cohort (82.1 [68.7–95.6] months) as compared to the non‐NIV cohort (33.6 [31.6–35.7] months). Conclusions Although NIV represents the standard of care, its utilization rate was low. TIV was mainly started without preceding NIV, suggesting that TIV may not be confined to NIV treatment escalation. However, TIV was pursued in a minority of patients who had previously undergone NIV. The survival benefit observed in the patients with NIV was equal to that reported in a controlled pivotal trial, but the prognosis with TIV is highly variable. The determinants of utilization of NIV/TIV and of survival (bulbar syndrome, availability of ventilation‐related home nursing, cultural factors) warrant further investigation.

A Nation-Wide, Multi-Center Study on the Quality of Life of ALS Patients in Germany

“…The use of non-invasive ventilation was also found to significantly improve QoL. This finding is in line with previous studies, which have found non-invasive ventilation to be efficient at treating respiratory failure, prolonging life and also improving QoL [15,30,[69][70][71][72][73]. Corresponding to our findings regarding tracheostomy and non-invasive ventilation, another previous study found that ventilated patients had a better QoL than non-ventilated patients, potentially due to the beneficial effects of ventilation on lessening daytime sleepiness, which supports our results [74].…”

Factors associated with assisted ventilation use in amyotrophic lateral sclerosis: a nationwide population-based study in Korea

“…The utilization of assisted ventilation varies across countries. NIV use was 23% in Australia 35 , 21% in Germany 36 , 34% in United States 12 , 27% in Japan 37 , and 17% in Taiwan 38 . TIV also showed various utilization rate: 9.5% in Germany 36 , approximately 11% in Italy 39 , above 20% in Japan 37 , and 21% in Taiwan 38 .…”

“…NIV use was 23% in Australia 35 , 21% in Germany 36 , 34% in United States 12 , 27% in Japan 37 , and 17% in Taiwan 38 . TIV also showed various utilization rate: 9.5% in Germany 36 , approximately 11% in Italy 39 , above 20% in Japan 37 , and 21% in Taiwan 38 . These differences in the utilization rates of assisted ventilation could be attributed to various factors, including genetics, healthcare policies covering ventilator use, cultural factors (e.g., religion), and study design such as follow up duration or type of ALS involved in the study.…”

Amyotrophe Lateralsklerose (ALS) – Diagnose, Verlauf und neue Behandlungsoptionen

“…Durch eine fortschreitende Mangelernährung (bedingt durch die Schluckstörung) oder durch eine respiratorische Insuffi-zienz (neuromuskuläre Hypoventilation) führt die ALS zu einer Lebensverkürzung. Beobachtungsstudien zeigten ein medianes Überleben von 38 Monaten nach Symptombeginn [7]. Die Überlebensprognose wird jedoch von zahlreichen Faktoren bestimmtdie Überlebenszeit ist daher individuell und entzieht sich einer Verallgemeinerung.…”