Effects of Long Term Ventilation Support on the Quality of Life of ALS Patients and Their Families

Study Overview
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Brief Summary
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Amyotrophic lateral sclerosis (ALS) is a serious rapidly progressive disease of the nervous system. The average survival from the time of diagnosis is two to three years. The patient physical and psychological sufferings in ALS are immense, and apart from Riluzole, there is no effective treatment. Care of advanced ALS have an estimated cost of 4-8 million NOK per year. Perhaps the most challenging topic of ALS care is the decision to extend ventilation support into the stages of disease that require treatment both during day and night. In these cases, treatment is clearly life-sustaining and although quality of life may be maintained, the burden of caregiving imposed upon family or health care workers is huge, regardless of tracheostomy (TIV) or non-invasive (NIV) modality. The present study is a longitudinal questionnaire study in Norway measuring overall quality of life, health-related quality of life, and disease-specific quality of life in ALS patients, partners and children before and after the introduction of life sustaining ventilation support. The investigators aim to increase the knowledge on how life-sustaining ventilation support with NIV or TIV affects the quality of life in ALS patients, life partners and children. The results from the study may provide crucial information for clinicians and patients on one of the most difficult ethical issues of ALS treatment. The investigators anticipate that this information will facilitate a shared decision making processes, weighing benefits and disadvantages in a wider perspective.

Official Title
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Effects of Long Term Ventilation Support on the Quality of Life of ALS Patients and Their Families

Conditions
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Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Nervous System Diseases, Spinal Cord Diseases, Neurodegenerative Diseases, TDP-43 Proteinopathies

Intervention / Treatment
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* Device: Long term mechanical ventilation support
* Device: No long term mechanical ventilation support


Participation Criteria
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Eligibility Criteria
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Inclusion criteria for patients: A clinical diagnosis of probable ALS according to the revised El Escorial criteria Progression of the illness leading the consulting physician to offer treatment with LTMV Can communicate in Norwegian Inclusion criteria for partners of ALS patients: Partner of a patient with ALS with progression of the illness leading the consulting physician to offer treatment with LTMV Can communicate in Norwegian Inclusion criteria for children: Children from 8 years and older having a parent who suffers from ALS with progression of the illness leading the consulting physician to offer treatment with LTMV Can communicate in Norwegian Exclusion criteria for patients, partners and children of ALS patients: 1. Potential participants with cognitive impairment or dementia.

Ages Eligible for Study
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Minimum Age: 8 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: ALS patients that choose life prolonging treatment with LTMV and their families<br> | Device: Long term mechanical ventilation support<br>* Patients that choose life prolonging treatment with long term mechanical ventilation support<br>|
| Active Comparator: ALS patients that decline life prolonging treatment with LTMV and their families<br> | Device: No long term mechanical ventilation support<br>* Patients that decline life prolonging treatment with long term mechanical ventilation support<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall quality of life assessed by the Quality of Life Scale | Quality of Life Scale total score (range 16-112). Higher score indicates better quality of life. | At inclusion | 
| Overall quality of life assessed by the Quality of Life Scale | Quality of Life Scale total score (range 16-112). Higher score indicates better quality of life. | 3 months after inclusion | 
| Overall quality of life assessed by the Quality of Life Scale | Quality of Life Scale total score (range 16-112). Higher score indicates better quality of life. | 9 months after inclusion | 
| Overall quality of life assessed by the Quality of Life Scale | Quality of Life Scale total score (range 16-112). Higher score indicates better quality of life. | 15 months after inclusion | 
| Overall quality of life assessed by the Quality of Life Scale | Quality of Life Scale total score (range 16-112). Higher score indicates better quality of life. | 21 months after inclusion | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Health-related quality of life assessed by Severe Respiratory Insufficiency Questionnaire | Severe Respiratory Insufficiency Questionnaire score (range 0-100), with 100 reflecting the best health state. | 3 months after inclusion | 
| Health-related quality of life assessed by Severe Respiratory Insufficiency Questionnaire | Severe Respiratory Insufficiency Questionnaire score (range 0-100), with 100 reflecting the best health state. | 9 months after inclusion | 
| Health-related quality of life assessed by Severe Respiratory Insufficiency Questionnaire | Severe Respiratory Insufficiency Questionnaire score (range 0-100), with 100 reflecting the best health state. | 15 months after inclusion | 
| Health-related quality of life assessed by Severe Respiratory Insufficiency Questionnaire | Severe Respiratory Insufficiency Questionnaire score (range 0-100), with 100 reflecting the best health state. | 21 months after inclusion | 
| Health-related quality of life assessed by Kidsscreen-27 | Kidsscreen-27 score where mean (±SD) scores of 50 ± 10 define normality for children and adolescents aged 8-18 years across Europe. Higher scores indicate a better health-related quality of life. | At inclusion | 
| Health-related quality of life assessed by Kidsscreen-27 | Kidsscreen-27 score where mean (±SD) scores of 50 ± 10 define normality for children and adolescents aged 8-18 years across Europe. Higher scores indicate a better health-related quality of life. | 3 months after inclusion | 
| Health-related quality of life assessed by Kidsscreen-27 | Kidsscreen-27 score where mean (±SD) scores of 50 ± 10 define normality for children and adolescents aged 8-18 years across Europe. Higher scores indicate a better health-related quality of life. | 9 months after inclusion | 
| Health-related quality of life assessed by Kidsscreen-27 | Kidsscreen-27 score where mean (±SD) scores of 50 ± 10 define normality for children and adolescents aged 8-18 years across Europe. Higher scores indicate a better health-related quality of life. | 15 months after inclusion | 
| Health-related quality of life assessed by Kidsscreen-27 | Kidsscreen-27 score where mean (±SD) scores of 50 ± 10 define normality for children and adolescents aged 8-18 years across Europe. Higher scores indicate a better health-related quality of life. | 21 months after inclusion | 
| Caregiver burden assessed by Zarit Burden Interview | Zarit Burden Interview total score (range 0-88 where a higher score indicates greater burden). | At inclusion | 
| Caregiver burden assessed by Zarit Burden Interview | Zarit Burden Interview total score (range 0-88 where a higher score indicates greater burden). | 3 months after inclusion | 
| Caregiver burden assessed by Zarit Burden Interview | Zarit Burden Interview total score (range 0-88 where a higher score indicates greater burden). | 9 months after inclusion | 
| Caregiver burden assessed by Zarit Burden Interview | Zarit Burden Interview total score (range 0-88 where a higher score indicates greater burden). | 15 months after inclusion | 
| Caregiver burden assessed by Zarit Burden Interview | Zarit Burden Interview total score (range 0-88 where a higher score indicates greater burden). | 21 months after inclusion | 
| Health-related quality of life assessed by the EQ-5D-5L | EQ-5D-5L index score and EQ-VAS score (range 0-100) where higher scores indicates better health-related quality of life. | At inclusion | 
| Health-related quality of life assessed by the EQ-5D-5L | EQ-5D-5L index score and EQ-VAS score (range 0-100) where higher scores indicates better health-related quality of life. | 3 months after inclusion | 
| Health-related quality of life assessed by the EQ-5D-5L | EQ-5D-5L index score and EQ-VAS score (range 0-100) where higher scores indicates better health-related quality of life. | 9 months after inclusion | 
| Health-related quality of life assessed by the EQ-5D-5L | EQ-5D-5L index score and EQ-VAS score (range 0-100) where higher scores indicates better health-related quality of life. | 15 months after inclusion | 
| Health-related quality of life assessed by the EQ-5D-5L | EQ-5D-5L index score and EQ-VAS score (range 0-100) where higher scores indicates better health-related quality of life. | 21 months after inclusion | 
| Disease-specific quality of life assessed by Amyotrophic lateral sclerosis assessment scale - 5 items (ALSAQ-5) | ALSAQ-5 score (range 0-100), with 0 reflecting the best health state | At inclusion | 
| Disease-specific quality of life assessed by Amyotrophic lateral sclerosis assessment scale - 5 items (ALSAQ-5) | ALSAQ-5 score (range 0-100), with 0 reflecting the best health state | 3 months after inclusion | 
| Disease-specific quality of life assessed by Amyotrophic lateral sclerosis assessment scale - 5 items (ALSAQ-5) | ALSAQ-5 score (range 0-100), with 0 reflecting the best health state | 9 months after inclusion | 
| Disease-specific quality of life assessed by Amyotrophic lateral sclerosis assessment scale - 5 items (ALSAQ-5) | ALSAQ-5 score (range 0-100), with 0 reflecting the best health state | 15 months after inclusion | 
| Disease-specific quality of life assessed by Amyotrophic lateral sclerosis assessment scale - 5 items (ALSAQ-5) | ALSAQ-5 score (range 0-100), with 0 reflecting the best health state | 21 months after inclusion | 
| Health-related quality of life assessed by Severe Respiratory Insufficiency Questionnaire | Severe Respiratory Insufficiency Questionnaire score (range 0-100), with 100 reflecting the best health state. | At inclusion | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Quality of life, Overall quality of life, Health related quality of life, Disease specific quality of life, Ventilation support, Non-invasive ventilation support, Invasive ventilation support