Health Outcomes of Parents With Cystic Fibrosis-Aim 2

Rate of change in ppFEV1 as reflected in the medical record from Year 1 to Year 5

Occurrence of PFTs at each clinic visit/hospitalization. Measures include FVC (Forced vital capacity), and the percent predicted.

Occurrence of PFTs at each clinic visit/hospitalization. Measures include FEV1(Forced expiratory volume in one second), and the percent predicted.

Occurrence of PFTs at each clinic visit/hospitalization. Measures include FEF25-75 (forced expiratory flow at 25 percent and 75 percent of the pulmonary volume), and the percent predicted for each.

Date of diagnosis, as reported in the medical record.

Genotype information, as reported in the medical record.

Mutation selection, as reported in the medical record.

The number of hospitalizations, as reported in the medical record, will be used to compute the rate of hospitalizations.

The length of each hospitalization, as reported in the medical record, will be used to compute the rate of hospitalizations.

The number of pulmonary exacerbations as reported in the medical record will be used to compute the rate of pulmonary exacerbations.

, hospitalizations (number and length)

The severity of pulmonary exacerbations (mild, moderate, severe), as reported in the medical record.

The type of treatment used for pulmonary exacerbations (IV antibiotic, oral antibiotic), as reported in the medical record.

The number and length of hospitalizations, combined, due to pulmonary exacerbations will be used to compute the rate of pulmonary exacerbations.

Number of outpatient clinic visits attended, as reported in the Cystic Fibrosis Foundation Patient Registry (CFFPR).

Number of positive screenings of specific types of bacteria, mycobacteria, fungus in cultures as reported in the CFFPR

Number of positive screenings of medication use, as reported in the medical record, including CFTR modulator therapy, chronic antibiotic use, and home IV treatment.

Instances of positive screenings for CF-Related Diabetes as reported in the medical record.

Highest Hemoglobin A1C values, as reported in the medical record.

Oral glucose tolerance test results, as reported in the medical record.

Insulin prescription use and dosage as reported in the medical record

Liver disease status (gall stones, liver disease with cirrhosis, liver disease non-cirrhosis, acute liver failure, hepatic steatosis) as reported by the medical record.

Current transplant status of the patient (not pertinent, accepted on waiting list, evaluated with final decision pending, evaluated and rejected, or had transplantation), as reported in the medical record.

If participant had transplantation in previous years, the transplant type will also be recorded as shown in the medical record

Height and weight combined, as reported in the medical record, will be collected to calculate BMI and BMI percentile

The Pittsburgh Sleep Quality Index (PSQI) is a self-rated questionnaire which assesses sleep quality and disturbances over a 1-month period. Nineteen individual items generate seven "component" scores: subjective sleep quality, sleep latency, sleep duration, sleep efficiency, sleep disturbances, use of sleeping medication, and daytime dysfunction. The seven component scores are derived, each scored 0 (no difficulty) to 3 (severe difficulty). The component scores are summed to produce a global score (range 0 to 21). Higher scores indicate worse sleep quality.

The Parental Stress Scale is a self-reported questionnaire which assesses feelings and perceptions about the experience of being a parent. Eighteen-item measure with 5-point Likert scale questions from 1 (strongly disagree) to 5 (strongly agree). Score is determined by the sum (possible range of 18 to 90) with higher scores indicating a higher measured level of parental stress.

Holmes-Rahe Life Stress Inventory (HRLSI) is a forty-three-item measure which assesses life stressors by accounting for life events over a 1-year period. Each life event corresponds to a point value (range 11-100). Scores are determined by the sum of the values for all checked life events. Scores of 150 or less refer to a relatively low amount of life change and low susceptibility to stress-induced health breakdowns. Scores of 151 to 300 imply about a 50% chance of a major health breakdown in the next two years. Scores greater than 301 raise the odds of a major health breakdown to about 80%.

The Cystic Fibrosis Questionnaire-Revised (CFQ-R) is a disease-specific instrument designed to measure the impact on overall health, daily life, well-being and symptoms. It consists of 50 individual measures assessing 9 quality of life domains: physical, role/school, vitality, emotion, social, body image, eating, treatment burden, and health perceptions. Each question is scored with the following scales: for questions 1 - 6: Very True = 1, Mostly True = 2, Somewhat True = 3, Not at all True = 4. For questions 7 - 17: Always = 1, Often = 2, Sometimes = 3, Never = 4. For questions 18 - 30: Very True = 1, Mostly True = 2, Somewhat True = 3, Not at all True = 4. For questions 31 - 35: Always = 1, Often = 2, Sometimes = 3, Never = 4. Scaled scores are calculated using the following formula: (Sum of responses - Minimal Possible sum (n x 1)) / (Maximum possible sum (n x 4) - Minimum possible sum (n x 1)) x 100.

Adapted Parental Responsibility Scale (APRS) is a forty-item measure which assesses responsibility forms of parental involvement. Each form of responsibility is to be rated on a 5-point Likert scale (I always do, I usually do, co-parent and I equally do, co-parent usually does, and co-parent always does) to designate who had primary responsibility for each task.

The Daily Care Check-In Tool is an 18-item measure that assesses the occurrence and frequency of facing barriers to completing treatments over a 6-month period. The Daily Care Check-In Occurrence Scale score can range from 0 (no barriers) to 18 (experiences all barriers). Each barrier is further scored based on a 5-point Likert scale (always, often, sometimes, rarely, never) to assess the frequency of each interference to treatment. Scores for the Daily Care Check-In Interference Scale are calculated by summing the point values assigned to each score. Scores can range from 0 (no barriers) to 90 (experiences all barriers and they always interfere with completing treatments).

Interpersonal Support Evaluation List-Short Form (ISEL-SF) is a 12-item measure that assesses the perceived availability of social support from family, friends, and others. Each statement is scored using a 4-point scale (definitely false, probably false, probably true, definitely true) to measure subscales of appraisal, belonging, and tangible support. All items are summed to a total score ranging from 0 to 36 with higher scores indicating greater perceived social support.

The Generalized Anxiety Disorder Assessment (GAD-7) is a seven-item measure that is used to assess the severity of generalized anxiety disorder over a 2-week period. The frequency of symptoms is evaluated using a 4-point scale (not at all, several days, more than half the days, nearly every day). The whole scale score can range from 0 to 21. Scores of 0 to 4 indicate minimal anxiety, scores 5-9 indicate mild anxiety, scores 10-15 indicate moderate anxiety and scores greater than 15 indicate severe anxiety.

The Patient Health Questionnaire depression scale (PHQ-8) is an 8-item screening tool used to assess depressive symptoms and severity over a 2-week period. The frequency of symptoms is evaluated using a 4-point scale (not at all, several days, more than half the days, nearly every day). Total score (range of 0 to 24) is determined by adding together the scores of each of the items. Scores of 5, 10, 15, and 20 represent the cutoff points for mild, moderate, moderately severe and severe depression, respectively.

The Self-Reported Adherence to Highly Effective Modulator Therapy scale is a 4-item questionnaire aimed at identifying the class of modulator (ivacaftor, elexacaftor-tezacaftor-ivacaftor, tezacaftor-ivacaftor, lumacaftor-ivacaftor), presence of altered dosing schedules (specific time or days), and missed treatments (yes, no). Adherence is further evaluated by assessing the number of days that treatment occurred as prescribed (0-3 days, 4-6 days, 7-10 days, 11-14 days) over a 2-week period. This measure serves as descriptive data only, and therefore requires no scoring calculations.