To determine the relationships between respiratory function, secretion management, RTIs and mortality in order to optimize medical decision-making.
ID
Source
Brief title
Condition
- Muscle disorders
- Neuromuscular disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Rate of respiratory and functional decline, mortality and RTIs.
Secondary outcome
• Examine the relationships between rates of decline in respiratory function
measures and prognosis / life expectancy / NIV initiation.
• Examine the relationship between respiratory tract infection morbidity and
respiratory function.
• Examine the relationship between respiratory function and respiratory
impairment symptoms.
• Correlate rates of decline in respiratory function measures and overall
functional decline using the ALS functional rating scale (ALSFRS-R).
• Examine the respiratory tract infection morbidity rates.
• Examine the predictive value across a range of respiratory function
constructs to predict carbon dioxide pressure (PCO2).
• Explore the potential relationships between other demographic
characteristics, baseline variables, and outcome measures as defined above
Background summary
Respiratory insufficiency is the primary cause of mortality in amyotrophic
lateral sclerosis (ALS). Death occurs, on average, three years after symptom
onset. Therapies that improve respiratory function may have the potential to
improve both quality of life and survival in ALS. A timely start of
non-invasive ventilation (NIV), for example, could improve overall survival
with nine months. The generation of exact evidence in support of such
beneficial outcomes has been, however, challenged by variance in measurement of
respiratory status. Measurement errors also complicate the medical
decision-making surrounding respiratory interventions. Furthermore, due to the
respiratory muscle weakness, ALS patients are more prone to respiratory tract
infections. It is, however, not well document what are the relationships
between respiratory measurements, initiation of secretion management and
reduction of respiratory tract infections (RTIs) or mortality.
Study objective
To determine the relationships between respiratory function, secretion
management, RTIs and mortality in order to optimize medical decision-making.
Study design
Longitudinal cohort study for 18 months with quarterly follow-up visits and
monthly follow-up by phone or email.
Study burden and risks
patient will be asked to come to the UMC Utrecht for 7 visits, each visits will
last maximum 1.5 hours. therefor, the total burden for the patient in the
hospital will be 10.5 hours. Besides the hospital visits the patients will
receive a monthly email with 3 questions, it will take them 5 minutes to answer
those questions. Maximum time for the study: [7 x 1.5hours] + [71 x 5min] = 16
hours 30 minutes
Heidelberglaan 100
Utrecht 3584CX
NL
Heidelberglaan 100
Utrecht 3584CX
NL
Listed location countries
Age
Inclusion criteria
1. Age > 18 years
2. Diagnosis of ALS according to the El Escorial criteria
3. Kings Stage 2,3 and 4A
4. Ability to give informed consent
5. Ability to complete respiratory function measurement as defined by the
ability to generate consistent scores on FVC (2 valid scores within 10%).
6. Ability to correspond remotely by email /text message independently or with
the assistance of a carer.
Exclusion criteria
1. Diagnosis of another respiratory condition requiring current active
management (e.g. Asthma, COPD, bronchiectasis, lung cancer etc).
2. Use of NIV at time of enrolment (King Stage 4B).
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| CCMO | NL64066.041.17 |