Patient and Caregiver Perceptions of Airway Clearance Methods Used for Cystic Fibrosis.

Introduction: Cystic Fibrosis Foundation guidelines recommend people with CF perform daily airway clearance. This can be difficult for patients, as some find it time consuming or uncomfortable. Data comparing airway clearance methods are limited. We surveyed patients and their families to understand which methods are preferred and identify obstacles to performing airway clearance. Methods: We designed a REDCap survey and enrolled participants in 2021. Respondents reported information on airway clearance usage, time commitment, and medication use. They rated airway clearance methods for effectiveness, comfort, time commitment, importance, and compatibility with other treatments. The analysis included descriptive statistics and clustering. Results: 60 respondents started and 52 completed the survey. The median patient age was 20 years. Respondents experienced a median of four airway clearance methods in their lifetime, including chest wall oscillation (vest, 92%), manual chest physical therapy (CPT, 88%), forced expiration technique (huff or cough, 77%), and exercise (75%). Past 30-day use was highest for exercise (62%) and vest (57%). The time commitment was generally less than 2 hours daily. Of those eligible for CFTR modulators, 53% reported decreased time commitment to airway clearance after starting treatment. On a scale of 0-100, respondents rated CFTR modulators as their most important treatment (median 99.5), followed by exercise (88). Discussion. Patients and caregivers are familiar with several methods of airway clearance for CF. They report distinct strengths and limitations of each method. Exercise and vest are the most common methods of airway clearance. The use of CFTR modulators may reduce patient-reported time commitment to airway clearance.

Bracing in Adolescent Idiopathic Scoliosis Trial (BrAIST): Development and Validation of a Prognostic Model in Untreated Adolescent Idiopathic Scoliosis Using the Simplified Skeletal Maturity System.

STUDY DESIGN: Prognostic study and validation using prospective clinical trial data. OBJECTIVE: To derive and validate a model predicting curve progression to ≥45° before skeletal maturity in untreated patients with adolescent idiopathic scoliosis (AIS). SUMMARY OF BACKGROUND DATA: Studies have linked the natural history of AIS with characteristics such as sex, skeletal maturity, curve magnitude, and pattern. The Simplified Skeletal Maturity Scoring System may be of particular prognostic utility for the study of curve progression. The reliability of the system has been addressed; however, its value as a prognostic marker for the outcomes of AIS has not. The BrAIST trial followed a sample of untreated AIS patients from enrollment to skeletal maturity, providing a rare source of prospective data for prognostic modeling. METHODS: The development sample included 115 untreated BrAIST participants. Logistic regression was used to predict curve progression to ≥45° (or surgery) before skeletal maturity. Predictors included the Cobb angle, age, sex, curve type, triradiate cartilage, and skeletal maturity stage (SMS). Internal and external validity was evaluated using jackknifed samples of the BrAIST data set and an independent cohort (n = 152). Indices of discrimination and calibration were estimated. A risk classification was created and the accuracy evaluated via the positive (PPV) and negative predictive values (NPV). RESULTS: The final model included the SMS, Cobb angle, and curve type. The model demonstrated strong discrimination (c-statistics 0.89-0.91) and calibration in all data sets. The classification system resulted in PPVs of 0.71-0.72 and NPVs of 0.85-0.93. CONCLUSIONS: This study provides the first rigorously validated model predicting a short-term outcome of untreated AIS. The resultant estimates can serve two important functions: 1) setting benchmarks for comparative effectiveness studies and 2) most importantly, providing clinicians and families with individual risk estimates to guide treatment decisions. LEVEL OF EVIDENCE: Level 1, prognostic.