Effects of Nocturnal Non-invasive Ventilation in Patients With Amyotrophic Lateral Sclerosis
Aim 1. To determine the incidence of hypoventilation in Amyotrophic Lateral Sclerosis (ALS) patients.
Aim 2. To identify the clinical characteristics and risk factors associated .
Aim 3. To determine the effect of early intervention with nocturnal NIV on the prognosis of ALS patients.
|Study Design:||Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Effects of Early Intervention With Nocturnal Non-invasive Ventilation in Patients With Amyotrophic Lateral Sclerosis: A Randomized, Controlled Trial|
- Survival [ Time Frame: 36 months ] [ Designated as safety issue: No ]
- Changes of PtcCO2 and PaCO2, admission or unexpected clinical visiting, and QoL [ Time Frame: 36 months ] [ Designated as safety issue: No ]
|Study Start Date:||January 2009|
|Study Completion Date:||April 2013|
|Primary Completion Date:||April 2013 (Final data collection date for primary outcome measure)|
ALS with non-invasive ventilation
Device: non-invasive ventilation
No Intervention: 1
ALS without non-invasive ventilation
Objectives: Amyotrophic lateral sclerosis is the commonest motor neuron disease with incidence of 0.8 person-years in Chinese. Respiratory muscle function has been proposed to be a strong predictor of quality of life (QoL) and survival in ALS. Some studies suggest that most patients with ALS developed hypoventilation when their vital capacity (FVC) is less than 50% of predicted value. However, the incidence of hypoventilation and factors associated with hypoventilation in ALS patients is not clear. Also, there is still no consensus as to which physiologic marker should be used as a trigger for the initiation of non-invasive ventilation (NIV) in ALS patients. The conflicts of studies come from variable subgroup of ALS, pulmonary function at enrollment, techniques used to diagnose ALS, time to apply NIV, and target endpoint. Therefore, this project aimed to study ALS patients who had relatively preserved respiratory muscle function and no respiratory failure at clinical to achieve three goals: (1) To determine the incidence of hypoventilation in ALS patients (2) To identify the clinical characteristics and risk factors associated with hypoventilation in ALS patients (3) To determine the effect of early intervention with NIV on the prognosis of ALS patients Study design: Randomized, controlled trial Participants: ALS patients whose FVC 40%-80% of predict, Pimax <60mmHg, and daytime PaCO2<50mmHg Protocol: Eligible patients with whole-night polysomnography (PSG) and transcutaneous CO2 (PtcCO2). Enrolled patients were randomized to standard treatment or NIV. The primary endpoint of prognosis was survival. The secondary endpoint was changes of PtcCO2 and PaCO2, unexpected admission or clinic visiting, daytime function and QoL.
Statistic: The baseline demographics of patients with or without hypoventilation were compared to determine the factors associated with hypoventilation in ALS patients. The impact of NIV in ALS patients was determined by comparing the primary and secondary goals between standard treatment and NIV group. A two-sided p value of < 0.05 was considered statistically significant.
Clinical implication: Hypoventilation at ALS patients who had relatively preserved respiratory muscle function and no respiratory failure at clinical sleep was common, early identification through PSG screening and PtcCO2 will allow for the early diagnosis and intervention. Understanding the time of applying NIV and the effect on prognosis in ALS will allow for the early intervention and prediction of outcomes.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00958048
|Taipei, Taiwan, 100|
|Principal Investigator:||Peilin Lee, M.D.||National Taiwan University Hospital|
|Principal Investigator:||Whey Dong Wu, M.D.||Naitonal Taiwan University Hospital|