Cystic Fibrosis
Conditions
Brief summary
The investigators know that adherence to medications in children with cystic fibrosis (CF) is poor. Forgetfulness has often been reported as a barrier to adherence by both CF patients and their parents. Many of the investigators patients also report being motivated by the results of their lung function studies (PFTs) to stay adherent to their medications. In this study, the investigators would like to see if providing medication reminders and allowing patients to measure their lung function at home will lead to better adherence. This will be a pilot study to determine the feasibility of providing such as a device to children with CF.
Detailed description
This is a randomized controlled trial to assess the impact of home lung function monitoring and medication reminders on adherence, clinical outcomes and quality of life in children with cystic fibrosis (CF) who are between 10 and 21 years of age. Participants will be randomly assigned to either an intervention group or a control group. Those in the intervention group will receive a personal spirometer device that provides medication reminders and allows for lung function monitoring at home. The control group will receive the current standard of care. All participants will be aware that their adherence is being monitored over the course of the study using prescription refill data. Changes in lung function, body mass index and rate of hospitalization will be used as measures of clinical outcome. Responses to age-appropriate well-validated patient questionnaires will be used to assess the impact of the investigators interventions on quality of life and perceptions of treatment burden. Since this device has never been previously studied in this patient population, the investigators would like to do a pilot study with 5 patients (3 in the intervention group and 2 in the control group) over a 3-month period to determine feasibility. This will also help us with the investigator power calculations and ultimately deciding the number of participants that will be needed for the larger study.
Interventions
DEVICESpiro PD personal spirometer
Sponsors
University of Michigan
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
HEALTH_SERVICES_RESEARCH
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
10 Years to 21 Years
Healthy volunteers
No
Inclusion criteria
1. Age 10-21 years 2. Confirmed diagnosis of cystic fibrosis (CF) either by a sweat chloride ≥ 60mEq/L or the presence of two disease-causing mutations 3. Patients must be clinically stable with at least 1 month from their last hospitalization or use of oral antibiotics for a pulmonary exacerbation 4. Signed informed consent from the patient and/or from the parent/legal guardian, if younger than 18 years.
Exclusion criteria
1. Age less than 10 years or greater than 21 years 2. Clinically unstable
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Medication Adherence | 3 months | Overall adherence to inhaled hypertonic saline, dornase alfa and CF multivitamins based on prescription refill data. The actual number of prescriptions of each of the three medications filled in the 3-month period was divided by the number that should have been filled based on the prescribed amount of each medication and that value was multiplied by a 100 to generate a percentage. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Cystic Fibrosis Questionnaire-Revised (CFQ-R)Treatment Burden Domain Score (Child) | 3 months | Response of the participants to the treatment burden domain of the CFQ-R at the end of 3 months |
| Cystic Fibrosis Questionnaire-Revised (CFQ-R) Treatment Burden Domain Score (Parent) | 3 months | Response of the parents/caregivers to the treatment burden domain of the CFQ-R at the end of 3-months |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Intervention Group Subjects in this group were asked to use the Spiro PD personal spirometer to check their lung function once a week for 3 months. They were also asked to use the medication reminder feature of their device daily. Participants were trained on the appropriate use of their device at the time of enrollment. They also received weekly telephone calls from a respiratory therapist to review that week's lung function results. They filled out a quality of life questionnaire at enrollment and at 3 months. Information regarding their height, weight, lung function and frequency of hospitalizations over the previous 3 months were obtained at enrollment and 3-months from review of their medical records. Pharmacies were contacted for refill data during the 3-month study duration for inhaled hypertonic saline, dornase alfa and CF multivitamins. | 3 |
| Control Subjects in this group had their adherence to inhaled hypertonic saline, dornase alfa and CF multivitamins during the 3-month study duration monitored through the use of prescription refill histories. They filled out a quality of life questionnaire at enrollment and 3 months. Their height, weight, lung function and frequency of hospitalizations over the previous 3 months was obtained at enrollment and 3-months from review of their medical records. | 2 |
| Total | 5 |
Baseline characteristics
| Characteristic | Control | Total | Intervention Group |
|---|---|---|---|
| Age, Continuous | 11.93 years STANDARD_DEVIATION 1.29 | 11.67 years STANDARD_DEVIATION 0.82 | 11.51 years STANDARD_DEVIATION 0.64 |
| Body Mass Index (BMI) Percentile | 41.5 Percentile STANDARD_DEVIATION 16.26 | 55.2 Percentile STANDARD_DEVIATION 20.14 | 64.33 Percentile STANDARD_DEVIATION 19.14 |
| Cystic Fibrosis Questionnaire-Revised (CFQ-R) Treatment Burden Domain (Parent) | 61 units on a scale STANDARD_DEVIATION 24.04 | 60 units on a scale STANDARD_DEVIATION 17.14 | 59.33 units on a scale STANDARD_DEVIATION 17.24 |
| Cystic Fibrosis Questionnaire-Revised (CFQ-R) Treatment Burden Domain Score (Child) | 78 units on a scale STANDARD_DEVIATION 31.11 | 75.8 units on a scale STANDARD_DEVIATION 16.31 | 74.33 units on a scale STANDARD_DEVIATION 6.35 |
| Forced expiratory volume in 1 second (FEV1) | 93 %predicted STANDARD_DEVIATION 1.41 | 81.8 %predicted STANDARD_DEVIATION 15.61 | 74.33 %predicted STANDARD_DEVIATION 16.65 |
| Sex: Female, Male Female | 1 Participants | 4 Participants | 3 Participants |
| Sex: Female, Male Male | 1 Participants | 1 Participants | 0 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 0 / 3 | 0 / 2 |
| serious Total, serious adverse events | 0 / 3 | 0 / 2 |
Outcome results
Primary
Medication Adherence
Overall adherence to inhaled hypertonic saline, dornase alfa and CF multivitamins based on prescription refill data. The actual number of prescriptions of each of the three medications filled in the 3-month period was divided by the number that should have been filled based on the prescribed amount of each medication and that value was multiplied by a 100 to generate a percentage.
Time frame: 3 months
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Intervention Group | Medication Adherence | 91.6 Percent Adherence | Standard Deviation 14.43 |
| Control | Medication Adherence | 100 Percent Adherence | Standard Deviation 0 |
Secondary
Cystic Fibrosis Questionnaire-Revised (CFQ-R)Treatment Burden Domain Score (Child)
Response of the participants to the treatment burden domain of the CFQ-R at the end of 3 months
Time frame: 3 months
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Intervention Group | Cystic Fibrosis Questionnaire-Revised (CFQ-R)Treatment Burden Domain Score (Child) | 81.66 units on a scale | Standard Deviation 16.8 |
| Control | Cystic Fibrosis Questionnaire-Revised (CFQ-R)Treatment Burden Domain Score (Child) | 72.5 units on a scale | Standard Deviation 23.33 |
Secondary
Cystic Fibrosis Questionnaire-Revised (CFQ-R) Treatment Burden Domain Score (Parent)
Response of the parents/caregivers to the treatment burden domain of the CFQ-R at the end of 3-months
Time frame: 3 months
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Intervention Group | Cystic Fibrosis Questionnaire-Revised (CFQ-R) Treatment Burden Domain Score (Parent) | 67 units on a scale | Standard Deviation 19.05 |
| Control | Cystic Fibrosis Questionnaire-Revised (CFQ-R) Treatment Burden Domain Score (Parent) | 66.5 units on a scale | Standard Deviation 31.82 |