ALS
Conditions
Keywords
Telemedicine, Pulmonary Function Testing, Telehealth
Brief summary
Comparison of respiratory outcomes in patients receiving telemedicine-guided remote pulmonary function testing (rPFT) with or without the additional support of nurse coaching. This is a randomized controlled study which assesses the effects rPFT and coaching on respiratory outcomes and quality of life.
Detailed description
This is a two-arm, randomized study to determine 1) whether weekly monitoring of respiratory function can lead to timelier initiation of NIV and 2) whether structured nurse coaching leads to improved self-efficacy for managing disease and better maintenance of respiratory health. For enrollees in both arms, standard FVC and MIP measurements obtained approximately every three months by the respiratory therapist during ALS Clinic are supplemented with self-administered rPFTs performed weekly. Enrollees in the rPFT+NRHC (nurse respiratory health coaching) arm will additionally receive monthly coaching with the study nurse via telehealth. Outcomes include measures of quality of life and self-efficacy for managing disease.
Interventions
Telemedicine delivery of pulmonary function testing in ALS, including Forced Vital Capacity (FVC) and Maximal Inspiratory Pressure (MIP)
Standard clinical delivery of pulmonary function testing in ALS, including Forced Vital Capacity (FVC) and Maximal Inspiratory Pressure (MIP)
BEHAVIORALNurse Respiratory Health Coaching (NRHC)
Nurse Respiratory Health Coaching - The NRHC intervention follows the teamlet model described by Bennett et al. \[Bennett2010\], made up of the research coordinator who performs respiratory testing, and the nurse practitioner who coaches using the GROW (goals, realities, options, wrap-up) model.
Sponsors
ALS Association
Milton S. Hershey Medical Center
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
HEALTH_SERVICES_RESEARCH
Masking
NONE
Intervention model description
A longitudinal parallel group study of repeated measures of remote PFTs, nurse coaching, and their impact on respiratory outcomes.
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Patients: 1. Possess a diagnosis of definite, probable, probable laboratory-supported, or possible ALS by revised El Escorial research criteria \[Brooks2000\]. 2. Be 18 years of age or older. 3. Have a caregiver available to participate in the study 4. Symptom onset within the last three years. 5. Have a computer and home internet service sufficient for engaging in telemedicine sessions. 6. Have a second device capable of downloading the spirometer application from an app store (Android- or iOS-based smartphone or tablet). Caregivers: 1. Be 18 years of age or older, of either gender. 2. Be able and willing to provide informed consent.
Exclusion criteria
Patients: 1. Use of NIV or diaphragm pacer at time of obtaining informed consent. 2. FVC ≤50% predicted or MIP \> -60 cm of water. 3. ALS Functional Rating Scale (ALSFRS-R) \[Cedarbaum1999\] score on day of screening of ≥2 on items for speech, swallowing, and salivation. These items are indicators of bulbar dysfunction, which limits the reliability of PFT administration. 4. Cognitive impairment, as judged by the ALS clinic neurologist, that prevents participation in the study. Caregivers: None
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Self-efficacy for Managing Symptoms | 1 year | The self-efficacy assessment contains questions from the PROMIS item bank on Self-Efficacy for Managing Symptoms (9 items). Cumulative T-scores from each test bank were reported at months 0, 3, 6, 9, and 12, with a mean score of 50±10, higher numbers indicating greater self-efficacy. The reported value is the slope of regression of self-efficacy on time, in T-score points per month. |
| Date of Identification of Noninvasive Ventilation (NIV) Need From rPFT Monitoring | 1 year | NIV is typically prescribed when an individual demonstrates signs of respiratory failure. In this study, forced vital capacity (FVC) of 50% or less of the predicted value serves as a surrogate for respiratory failure and constitutes identification of NIV need. First date of remote measurement of FVC 50% or less of the predicted value is reported here. |
| Date of Identification of NIV Need From Standard PFT Monitoring | From date of enrollment until first identification of non-invasive ventilation need, assessed up to 1.5 years | NIV is typically prescribed when an individual demonstrates signs of respiratory failure. In this study, forced vital capacity (FVC) of 50% or less of the predicted value serves as a surrogate for respiratory failure and constitutes identification of NIV need. First date of standard, in-clinic measurement of FVC 50% or less of the predicted value is reported here. |
| Self-efficacy for Managing Medications and Treatments | 1 year | The self-efficacy assessment contains questions from the Patient-Reported Outcomes Measurement Information System (PROMIS) item banks on Self-Efficacy for Managing Medications and Treatments (4 items). Cumulative T-scores from each test bank were reported at months 0, 3, 6, 9, and 12, with a mean score of 50±10, higher numbers indicating greater self-efficacy. The reported value is the slope of regression of self-efficacy on time, in T-score points per month. |
| Self-efficacy for Managing Social Interactions | 1 year | The self-efficacy assessment contains questions from the PROMIS item bank on Self-Efficacy for Managing Social Interactions (5 items). Cumulative T-scores from each test bank were reported at months 0, 3, 6, 9, and 12, with a mean score of 50±10, higher numbers indicating greater self-efficacy. The reported value is the slope of regression of self-efficacy on time, in T-score points per month. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Dyspnea Characteristics | 1 year | Patient-Reported Outcomes Measurement Information System (PROMIS) Pool v1.0 - Dyspnea Characteristics (5 items), which uses Likert-type scaling that are summed to a total score. Participants complete this questionnaire each month (12 total administrations). Total score range 0-44, with higher numbers indicating more severe dyspnea. The reported value is the slope of regression of total score on time, in point change per month. |
| Respiratory-related Symptoms | 1 year | Subjects were asked to report whether they experienced the following symptoms in the past month during each month on the study (12 administrations): excessive secretions, drooling, choking on secretions, morning headache. Reported value is the sum of experienced symptoms (0-4) summed over 12 months. Participants could experience none of these symptoms (0) up to experiencing all symptoms each month (48). |
| rPFT Adherence | 1 year | Participant adherence to rPFT protocol, defined as the percentage of eligible weeks with a submitted rPFT report. |
| Dyspnea Functional Limitations | 1 year | Patient-Reported Outcomes Measurement Information System (PROMIS) Item Bank v1.0 - Dyspnea Functional Limitations - Short Form 10a (10 items), which uses Likert-type scaling that are summed to a total score. Participants complete this questionnaire each month (12 total administrations). Total score range 0-30, with higher numbers indicating more functional limitations due to dyspnea. The reported value is the slope of regression of total score on time, in point change per month. |
| Sleep Related Impairment | 1 year | Patient-Reported Outcomes Measurement Information System (PROMIS) Item Bank v1.0 - Sleep Related Impairment - Short Form 8a (8 items), which uses Likert-type scaling that are summed to a total score. Participants complete this questionnaire each month (12 total administrations). Total score range 8-40, with higher numbers indicating more sleep-related impairment. The reported value is the slope of regression of total score on time, in point change per month. |
Countries
United States
Participant flow
Pre-assignment details
5 participants exited the study prior to randomization.
Participants by arm
| Arm | Count |
|---|---|
| Remote PFT (rPFT) Longitudinal Subjects will undergo standard pulmonary function testing as part of standard clinical procedure. They will also undergo weekly remote pulmonary function testing using the telemedicine interface and study equipment.
remote pulmonary function testing: Telemedicine delivery of pulmonary function testing in ALS, including Forced Vital Capacity (FVC) and Maximal Inspiratory Pressure (MIP)
standard pulmonary function testing: Standard clinical delivery of pulmonary function testing in ALS, including Forced Vital Capacity (FVC) and Maximal Inspiratory Pressure (MIP) | 12 |
| Remote PFT (rPFT) + Nurse Coaching Longitudinal Subjects will undergo standard pulmonary function testing as part of standard clinical procedure. They will also undergo weekly remote pulmonary function testing using the telemedicine interface and study equipment, and receive monthly coaching from an ALS nurse.
remote pulmonary function testing: Telemedicine delivery of pulmonary function testing in ALS, including Forced Vital Capacity (FVC) and Maximal Inspiratory Pressure (MIP)
standard pulmonary function testing: Standard clinical delivery of pulmonary function testing in ALS, including Forced Vital Capacity (FVC) and Maximal Inspiratory Pressure (MIP)
Nurse Respiratory Health Coaching (NRHC): Nurse Respiratory Health Coaching - The NRHC intervention follows the teamlet model described by Bennett et al. \[Bennett2010\], made up of the research coordinator who performs respiratory testing, and the nurse practitioner who coaches using the GROW (goals, realities, options, wrap-up) model. | 17 |
| Total | 29 |
Baseline characteristics
| Characteristic | Remote PFT (rPFT) + Nurse Coaching Longitudinal | Total | Remote PFT (rPFT) Longitudinal |
|---|---|---|---|
| Age, Continuous | 65 years | 64 years | 61 years |
| Race and Ethnicity Not Collected | — | 0 Participants | — |
| Region of Enrollment United States | 17 participants | 29 participants | 12 participants |
| Sex: Female, Male Female | 10 Participants | 14 Participants | 4 Participants |
| Sex: Female, Male Male | 7 Participants | 15 Participants | 8 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 1 / 12 | 2 / 17 |
| other Total, other adverse events | 0 / 12 | 0 / 17 |
| serious Total, serious adverse events | 1 / 12 | 6 / 17 |
Outcome results
Primary
Date of Identification of NIV Need From Standard PFT Monitoring
NIV is typically prescribed when an individual demonstrates signs of respiratory failure. In this study, forced vital capacity (FVC) of 50% or less of the predicted value serves as a surrogate for respiratory failure and constitutes identification of NIV need. First date of standard, in-clinic measurement of FVC 50% or less of the predicted value is reported here.
Time frame: From date of enrollment until first identification of non-invasive ventilation need, assessed up to 1.5 years
Population: Subset of patients include those who have a date of identification of NIV need from both remote PFT monitoring and standard monitoring.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Total Group | Date of Identification of NIV Need From Standard PFT Monitoring | 259 days after enrollment |
Primary
Date of Identification of Noninvasive Ventilation (NIV) Need From rPFT Monitoring
NIV is typically prescribed when an individual demonstrates signs of respiratory failure. In this study, forced vital capacity (FVC) of 50% or less of the predicted value serves as a surrogate for respiratory failure and constitutes identification of NIV need. First date of remote measurement of FVC 50% or less of the predicted value is reported here.
Time frame: 1 year
Population: Subset of patients include those who have a date of identification of NIV need from both remote PFT monitoring and standard PFT monitoring. Groups are combined because inferences across groups with respect to this outcome were not planned and are not reported.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Total Group | Date of Identification of Noninvasive Ventilation (NIV) Need From rPFT Monitoring | 176 days after enrollment |
Primary
Self-efficacy for Managing Medications and Treatments
The self-efficacy assessment contains questions from the Patient-Reported Outcomes Measurement Information System (PROMIS) item banks on Self-Efficacy for Managing Medications and Treatments (4 items). Cumulative T-scores from each test bank were reported at months 0, 3, 6, 9, and 12, with a mean score of 50±10, higher numbers indicating greater self-efficacy. The reported value is the slope of regression of self-efficacy on time, in T-score points per month.
Time frame: 1 year
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Total Group | Self-efficacy for Managing Medications and Treatments | -.238 points per month | Standard Deviation 0.393 |
| Remote PFT (rPFT) + Nurse Coaching Longitudinal | Self-efficacy for Managing Medications and Treatments | .474 points per month | Standard Deviation 1.583 |
Primary
Self-efficacy for Managing Social Interactions
The self-efficacy assessment contains questions from the PROMIS item bank on Self-Efficacy for Managing Social Interactions (5 items). Cumulative T-scores from each test bank were reported at months 0, 3, 6, 9, and 12, with a mean score of 50±10, higher numbers indicating greater self-efficacy. The reported value is the slope of regression of self-efficacy on time, in T-score points per month.
Time frame: 1 year
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Total Group | Self-efficacy for Managing Social Interactions | -.041 points per month | Standard Deviation 0.551 |
| Remote PFT (rPFT) + Nurse Coaching Longitudinal | Self-efficacy for Managing Social Interactions | -.181 points per month | Standard Deviation 0.688 |
Primary
Self-efficacy for Managing Symptoms
The self-efficacy assessment contains questions from the PROMIS item bank on Self-Efficacy for Managing Symptoms (9 items). Cumulative T-scores from each test bank were reported at months 0, 3, 6, 9, and 12, with a mean score of 50±10, higher numbers indicating greater self-efficacy. The reported value is the slope of regression of self-efficacy on time, in T-score points per month.
Time frame: 1 year
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Total Group | Self-efficacy for Managing Symptoms | -.279 points per month | Standard Deviation 0.636 |
| Remote PFT (rPFT) + Nurse Coaching Longitudinal | Self-efficacy for Managing Symptoms | -.255 points per month | Standard Deviation 0.813 |
Secondary
Dyspnea Characteristics
Patient-Reported Outcomes Measurement Information System (PROMIS) Pool v1.0 - Dyspnea Characteristics (5 items), which uses Likert-type scaling that are summed to a total score. Participants complete this questionnaire each month (12 total administrations). Total score range 0-44, with higher numbers indicating more severe dyspnea. The reported value is the slope of regression of total score on time, in point change per month.
Time frame: 1 year
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Total Group | Dyspnea Characteristics | .029 points per month | Standard Deviation 0.408 |
| Remote PFT (rPFT) + Nurse Coaching Longitudinal | Dyspnea Characteristics | .272 points per month | Standard Deviation 0.964 |
Secondary
Dyspnea Functional Limitations
Patient-Reported Outcomes Measurement Information System (PROMIS) Item Bank v1.0 - Dyspnea Functional Limitations - Short Form 10a (10 items), which uses Likert-type scaling that are summed to a total score. Participants complete this questionnaire each month (12 total administrations). Total score range 0-30, with higher numbers indicating more functional limitations due to dyspnea. The reported value is the slope of regression of total score on time, in point change per month.
Time frame: 1 year
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Total Group | Dyspnea Functional Limitations | -.022 points per month | Standard Deviation 0.35 |
| Remote PFT (rPFT) + Nurse Coaching Longitudinal | Dyspnea Functional Limitations | -.255 points per month | Standard Deviation 0.838 |
Secondary
Respiratory-related Symptoms
Subjects were asked to report whether they experienced the following symptoms in the past month during each month on the study (12 administrations): excessive secretions, drooling, choking on secretions, morning headache. Reported value is the sum of experienced symptoms (0-4) summed over 12 months. Participants could experience none of these symptoms (0) up to experiencing all symptoms each month (48).
Time frame: 1 year
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Total Group | Respiratory-related Symptoms | 11.4 number of symptoms reported | Standard Deviation 6.4 |
| Remote PFT (rPFT) + Nurse Coaching Longitudinal | Respiratory-related Symptoms | 7.6 number of symptoms reported | Standard Deviation 6 |
Secondary
rPFT Adherence
Participant adherence to rPFT protocol, defined as the percentage of eligible weeks with a submitted rPFT report.
Time frame: 1 year
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Total Group | rPFT Adherence | 82.2 Percentage |
Secondary
Sleep Related Impairment
Patient-Reported Outcomes Measurement Information System (PROMIS) Item Bank v1.0 - Sleep Related Impairment - Short Form 8a (8 items), which uses Likert-type scaling that are summed to a total score. Participants complete this questionnaire each month (12 total administrations). Total score range 8-40, with higher numbers indicating more sleep-related impairment. The reported value is the slope of regression of total score on time, in point change per month.
Time frame: 1 year
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Total Group | Sleep Related Impairment | .095 points per month | Standard Deviation 0.32 |
| Remote PFT (rPFT) + Nurse Coaching Longitudinal | Sleep Related Impairment | .140 points per month | Standard Deviation 0.523 |