Cystic Fibrosis, Cystic Fibrosis Pulmonary Exacerbation, Lung Infection
Conditions
Keywords
Cystic Fibrosis, Pulmonary Exacerbation, Lung Infection, Cystic Fibrosis Foundation, Cystic Fibrosis Foundation National Patient Registry
Brief summary
The goal of this research study is to better understand current treatment practices for pulmonary exacerbations (lung infections) and whether the Cystic Fibrosis National Patient Registry (CFFNPR)can be used for this type of study.
Detailed description
Cystic fibrosis (CF), a life-shortening genetic disease, is marked by acute episodes during which symptoms of lung infection increase and lung function decreases. These pulmonary exacerbations (PEs) are treated with varying antibiotics for varying time periods based on needs determined by individual patients, their families, and the health care providers. Cystic fibrosis pulmonary guidelines for the treatment of PE published by the Cystic Fibrosis Foundation (CFF) in 2009 provided recommendations for treatment and also identified key questions for which additional studies were needed. Standard treatment for PE involves many facets including selection of antibiotics, duration of use, and outcomes that define treatment success. Understanding current treatment practices and measures of treatment success are needed before a study can be designed to define optimal treatment strategies. This is a multi-center, prospective, observational study designed to prospectively follow patients with CF that are initially admitted to the hospital for treatment of a pulmonary exacerbation.
Interventions
None listed
Sponsors
Medical University of South Carolina
Cystic Fibrosis Foundation
University of Washington
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Male or female ≥12 years of age at Visit 1 * Enrolled in the CFFNPR (Patients may enroll in the Registry at Visit 1 if not previously enrolled.) * Current hospitalization for treatment of a pulmonary exacerbation * Planned hospital admission of at least 5 days with intravenous (IV) antibiotics at Visit 1 * Able to perform spirometry at admission and willing to perform spirometry on subsequent treatment and visit days * Willing and able to complete symptom score daily * Willing to return for a follow up visit at end of treatment (if necessary) and 28 days after start of IV antibiotic therapy * Written informed consent (and assent when applicable) obtained from the participant or participant's legal representative
Exclusion criteria
* Previous enrollment in this study * Treatment with IV antibiotics in the 6 weeks prior to Visit 1 * Admission to the intensive care unit for current pulmonary exacerbation * Pneumothorax on admission * Current hospitalization for scheduled pulmonary clean out * Current hospitalization for sinusitis as the primary diagnosis * Massive hemoptysis defined as \> 250 cc in a 24 hour period, or 100 cc/day over 4 consecutive days occurring within one week of Visit 1 * Current pulmonary exacerbation thought to be due to allergic bronchopulmonary aspergillosis (ABPA) * Ongoing treatment with prednisone equivalent \>10 mg/day for greater than 2 weeks initiated prior to Visit 1 * History of solid organ transplantation Currently receiving antimicrobial therapy to treat non-tuberculous mycobacterium (e.g., M. abscessus, M. avium complex)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Feasibility of using the CFF National Patient Registry | 28 days from time of start of IV antibiotic therapy. | Assess feasibility using the CFF National Patient Registry as measured by the accuracy of data entry |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Physician and patient level clinical outcomes for their use in comparative studies of CF pulmonary exacerbations to determine the optimal treatment endpoints | During hospitalization and during a period following discharge of 28 days from time of start of IV antibiotic therapy. | Evaluate physician assessment of treatment response as measured by the physician treatment assessment questionnaire. Evaluate change in lung function and patient reported respiratory symptoms and quality of life in response to treatment of an acute pulmonary exacerbation. |
Other
| Measure | Time frame | Description |
|---|---|---|
| Variability of practicing clinicians' treatment objectives, approaches, and assessment of outcomes related to CF pulmonary exacerbation | during hospitalization and during a period following discharge of 28 days from time of start of IV antibiotic therapy. | Describe the variability of practicing clinicians' treatment objectives as measured by the physician assessment questionnaire, treatment approaches as measured by the choices of medications (specifically antibiotics), and assessment of patient level outcomes related to CF pulmonary exacerbation (change in lung function as measured by spirometry, respiratory symptoms as measure by the CFRSD-CRISS questionnaire) and health related quality of life (as measured by the EQ-5D questionnaire). |
| Inform the design of future pragmatic research of CF pulmonary exacerbation | During hospitalization and during a period following discharge of 28 days from time of start of IV antibiotic therapy. | Identify the appropriate target patient population for future clinical trials by clarifying required stratification factors, and estimating target treatment effects and variability of treatment response. |
Countries
United States
Outcome results
None listed