Cystic Fibrosis
Conditions
Keywords
Cystic Fibrosis Medication Adherence
Brief summary
Inhaled tobramycin is a Cystic Fibrosis Foundation recommended effective treatment for individuals with cystic fibrosis for the management of Pseudomonas aeruginosa airway colonization and improves the FEV1 and reduces the number of acute pulmonary exacerbations of CF. Patients typically use the inhaled tobramycin for a period of 28 days. Unfortunately, the standard nebulizer method for delivering tobramycin inhaled solution (TIS) is time-consuming and may result in missed therapy doses and suboptimal care. A new inhaled formulation and delivery device, the TOBI Podhaler (TPI), an alternative method of administering inhaled Tobramycin will be used and assessed. This new pocket-sized disposable inhaler is maintenance-free, requires no refrigeration or power source, and should greatly increase patient mobility and improve time management.
Detailed description
Adult cystic fibrosis patients from the Rush University Medical Center Adult Cystic Fibrosis Program will be recruited for a prospective before and after cohort study to evaluate patient preference for either the TOBI Podhaler or inhaled tobramycin solution and compare the efficacy of the TOBI Podhaler to tobramycin inhaled solution. This project will evaluate the potential differences in efficacy, medication adherence, treatment time, side effects/adverse effects, quality of life, and identify patient preferences between the use of TIP and TIS. Measurements after a 28-day cycle of tobramycin inhaled solution, first 28-day cycle of TOBI Podhaler and third 28-day cycle of TOBI Podhaler will include FEV1, number of acute pulmonary exacerbations, adherence, cough frequency, sputum characteristics, and side effects or adverse events. The quality of life as assessed by the Cystic Fibrosis Questionnaire-Revised (CFQ-R) will be measured after a 28-day cycle of tobramycin inhaled solution and the third 28-day cycle of TOBI Podhaler. The FEV1 values, number of missed treatments, and total treatment time will be analyzed through a repeated measures analysis of variance. Comparisons of the CFQ-R data before and after the switch to the TPI will be made with the Student's t-test.
Interventions
DRUGTOBIpodhaler
Sponsors
Rush University Medical Center
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Adult (≥ 18 years) diagnosed with cystic fibrosis 2. Airway colonization with Pseudomonas aeruginosa that is sensitive to tobramycin in vitro and currently managed with Tobramycin inhaled solution who are interested in switching to the TOBI Podhaler.
Exclusion criteria
1. Persons unable to communicate in English, 2. Pregnant patients, 3. Patients \< 18 years of age
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Increased Adherence to the Medical Therapeutic Regimen | 6 months | Number of Participants with Adherence to Tobramycin Inhaled Solution Treatment compared with TOBI Podhaler Treatment based on improved efficacy and time required for administration and translate into improved respiratory status for adult patients with cystic fibrosis. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Preference for TOBIpodhaler Treatment | 6 months | The subject will indicate a preference for TOBIpodhaler vs prior treatment. |
| Spirometry Data | 6 months | spirometry was performed at baseline and at the end of the study. |
Countries
United States
Participant flow
Recruitment details
Recruitment was very slow related to cost of the medication. We also experienced difficulty getting subjects to return the questionnaire related to time involved and satisfaction with the treatment.
Pre-assignment details
Subjects were asked to compare the time and satisfaction of the TOBIpodhaler treatment to their prior inhaled antibiotic administration time and satisfaction and to compare their Quality of Life (using CFR-Q) measured before and after the TOBIpodhaler.
Participants by arm
| Arm | Count |
|---|---|
| Subject Response to TOBIpodhaler Were Compared to Baseline Individuals who were on inhaled antibiotic therapy as part of their CF management of Pseudomonas in the airway were selected if they were able to change their inhaled antibiotic to the TOBIpodhaler. These subjects would then compare their time involved in airway treatment, satisfaction with the treatment, and FEV1 to their prior-to-change value. | 5 |
| Total | 5 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Lost to Follow-up | 1 |
| Overall Study | poor adherence of subjects to protocol and withdrawal from study. | 4 |
Baseline characteristics
| Characteristic | Subject Response to TOBIpodhaler Were Compared to Baseline |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 5 Participants |
| Age, Continuous | 27.4 years STANDARD_DEVIATION 5.95 |
| FEV1 | 0 Participants |
| Region of Enrollment United States | 5 Participants |
| Sex: Female, Male Female | 3 Participants |
| Sex: Female, Male Male | 2 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 5 | 0 / 5 |
| other Total, other adverse events | 0 / 5 | 0 / 5 |
| serious Total, serious adverse events | 0 / 5 | 0 / 5 |
Outcome results
Primary
Increased Adherence to the Medical Therapeutic Regimen
Number of Participants with Adherence to Tobramycin Inhaled Solution Treatment compared with TOBI Podhaler Treatment based on improved efficacy and time required for administration and translate into improved respiratory status for adult patients with cystic fibrosis.
Time frame: 6 months
Population: 4 of 5 patients preferred TOBIpodhaler
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Patient Served as Own Control | Increased Adherence to the Medical Therapeutic Regimen | 4 participants |
Secondary
Preference for TOBIpodhaler Treatment
The subject will indicate a preference for TOBIpodhaler vs prior treatment.
Time frame: 6 months
Population: Data were not collected since patients did not return for follow-up.
Secondary
Spirometry Data
spirometry was performed at baseline and at the end of the study.
Time frame: 6 months
Population: Data were not collected since patients did not return for follow-up.