Health Outcomes of Parents With Cystic Fibrosis-Aim 2

Pulmonary Function Tests (PFTs) - Forced Vital Capacity (FVC)

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

Time frame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5

Pulmonary Function Tests (PFTs) - Forced Expiratory Volume (FEV1)

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

Time frame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5

Pulmonary Function Tests (PFTs) - Forced Expiratory Flow at 25 and 75 percent (FEF25-75)

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.

Time frame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5

History of CF Diagnosis

Date of diagnosis, as reported in the medical record.

Time frame: Year 1

History of CF Genotype Information

Genotype information, as reported in the medical record.

Time frame: Year 1

History of CF Mutation

Mutation selection, as reported in the medical record.

Time frame: Year 1

Number of Hospitalizations

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

Time frame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5

Length of Hospitalizations

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

Time frame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5

Number of Pulmonary Exacerbations

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)

Time frame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5

Severity of Pulmonary Exacerbations

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

Time frame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5

Treatment of Pulmonary Exacerbations

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

Time frame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5

Rate of Hospitalizations Due to Pulmonary Exacerbations

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

Time frame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5

Clinic Visit Attendance

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

Time frame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5

Microbiologic Profile

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

Time frame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5

Medication use

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

Time frame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5

History of CF-Related Diabetes Status

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

Time frame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5

CF-Related Diabetes Control - Hemoglobin A1C

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

Time frame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5

CF-Related Diabetes Control - Glucose Tolerance

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

Time frame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5

CF-Related Diabetes Control - Insulin Prescription Usage

Insulin prescription use and dosage as reported in the medical record

Time frame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5

Liver Disease Status

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

Time frame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5

Transplantation Status

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.

Time frame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5

Transplantation Type

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

Time frame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5

BMI

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

Time frame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5

Sleep Quality

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.

Time frame: Up to 4 times in Year 1, Up to 2 times in Year 2 through Year 5.

Parental Stress

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.

Time frame: Up to 4 times in Year 1, Up to 2 times in Year 2 through Year 5.

Life Stressors

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%.

Time frame: Up to 2 times per year in Year 1 through Year 5.

Cystic Fibrosis Questionnaire to evaluate quality of life domains

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.

Time frame: Up to 2 times per year in Year 1 through Year 5.

Parental Responsibility

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.

Time frame: Up to 4 times in Year 1, Up to 2 times in Year 2 through Year 5.

Daily Care Check-In

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).

Time frame: Up to 2 times per year in Year 1 through Year 5.

Social Support

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.

Time frame: Up to 4 times in Year 1, Up to 2 times in Year 2 through Year 5.

Anxiety

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.

Time frame: Up to 4 times in Year 1, Up to 2 times in Year 2 through Year 5.

Depression

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.

Time frame: Up to 4 times in Year 1, Up to 2 times in Year 2 through Year 5.

Self-Reported Adherence

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.

Time frame: Up to 2 times per year in Year 1 through Year 5.