Identifying who will benefit from non-invasive ventilation in amyotrophic lateral sclerosis/motor neurone disease in a clinical cohort

Abstract

Between 1991 and 2011, 1198 patients diagnosed with ALS/MND were registered. 929 patients (77.5%) fulfilled the selection criteria and their data were analysed. Median tracheostomy free survival from symptom onset was 28 months in NIV-treated patients compared to 15 months in untreated (Univariate Cox regression HR=0.61 (0.51 to 0.73), p<0.001). The positive survival effect of NIV persisted when the model was adjusted for age, gender, riluzole and percutaneous endoscopic gastrostomy use (HR=0.72 (0.60 to 0.88, p=0.001). In contrast with the only randomised controlled trial, NIV statistically significantly increased survival by 19 months in those with ALS-bulbar onset (Univariate HR=0.50 (0.36 to 0.70), multivariate HR=0.59 (0.41 to 0.83)). These data confirm that NIV improves survival in MND/ALS. The overall magnitude of benefit is 13 months and was largest in those with ALS-bulbar disease. Future research should explore the optimal timing of NIV initiation within phenotypes in order to optimise respiratory function, quality of life and survival.

Multidisciplinary Care Improves Survival of Patients with Amyotrophic Lateral Sclerosis in the Unique Health System (SUS) in Brazil

“…Similar to other studies that showed that only [23] 6% to 30% of ALS patients use NIV [30,31], the frequency of NIV use was low in the present study (18%), which is related to economic factors and to the long delay in diagnostic time. However, strong evidence exists that shows that NIV prolongs survival in ALS patients [4,30,32].…”

“…However, strong evidence exists that shows that NIV prolongs survival in ALS patients [4,30,32]. In a recent study [30], the median tracheostomy-free survival from symptom onset was 28 months in NIV-treated patients, compared to 15 months for untreated patients, and the benefit persisted after adjusting for age, gender, riluzole use and gastrostomy use. However, the only randomized controlled trial of NIV did not support this finding [33].…”

“…Reductions in pulmonary function decline and respiratory complications have been observed in patients who undergo [30,32], which suggests that future studies are needed to explore the optimal timing for NIV to optimize respiratory function, quality of life and survival.…”

Targeted assessment of lower motor neuron burden is associated with survival in amyotrophic lateral sclerosis

“…Some factors, such as site of disease onset (bulbar versus limb), have a more complex effect on survival that is influenced by age and gender (13,14). Survival can be prolonged through initiation of non-invasive ventilation (NIV) (15), which is sometimes considered as a secondary endpoint for disease progression.…”

Clinical and laboratory features of ALS

“…Edavarone has also been found to be beneficial [10] . Noninvasive ventilation (NIV) is available when the disease involves their respiratory system and can prolong the survival of the patients [11] . In the last decades, some drugs shown encouraging results on the rodent ALS models, but have proved to be ineffective in clinical trials of ALS patients [12] .…”