Clustered randomized controlled trial of a clinic-based problem-solving intervention to improve adherence in adolescents with cystic fibrosis.

BACKGROUND: In Cystic Fibrosis (CF), adherence to pulmonary medications is about 50% and decreases during adolescence. Effective interventions have not been integrated into CF care. This effectiveness study tested a brief, clinic-based behavioral intervention to improve adherence. METHODS: iCARE (I Change Adherence and Raise Expectations) was a pragmatic, clustered, 2-arm randomized controlled trial at 18 CF Centers. 607 adolescents with CF, ages 11-20 years, participated. Centers were randomized to IMPACT (n = 9; 300 adolescents), a brief problem-solving + education intervention, or standard care (SC; n = 9; 307 adolescents). IMPACT was delivered during a regularly scheduled clinic visit by a member of the clinical care team. The primary outcome was composite pulmonary medication possession ratio (cMPR); secondary endpoints were lung function, Body Mass Index percentile, courses of IV antibiotics, and health-related quality of life at 12 months. RESULTS: Effectiveness of the intervention was tested using mixed models, generalized estimating equations comparing IMPACT to SC. Fifty-eight percent of problem-solving sessions targeted barriers to airway clearance, exercise or nutrition, while 18% addressed pulmonary medications. Average intervention fidelity score was 67% (SD = 14%; Range = 25-100%). No significant intervention effects were found for cMPR or any of the secondary outcomes compared to SC. CONCLUSIONS: The IMPACT intervention did not improve medication adherence or health outcomes over 12 months. Challenges to implementing the intervention as intended during busy clinic visits were identified. TRIAL REGISTRY: ClinicalTrials.gov; No.: NCT01232478; URL: www.clinicaltrials.gov.

Development of Health-Related Quality of Life Instruments for Young Children With Disorders of Sex Development (DSD) and Their Parents.

Objective: Research in disorders of sex development (DSD) is hindered by a lack of standardized measures sensitive to the experiences of affected children and families. We developed and evaluated parent proxy (children 2-6 years) and parent self-report (children ≤6 years) health-related quality of life (HRQoL) instruments for DSD. Methods: Items were derived from focus groups and open-ended interviews. Clarity and comprehensiveness were assessed with cognitive interviews. Psychometric properties were examined in a field survey of 94 families. Results: Measures demonstrated adequate to good psychometrics, including internal consistency, test-retest reliability, convergent validity, and ability to detect known-group differences. Parents reported greatest stress on Early Experiences , Surgery , and Future Concerns scales. Conclusions: These instruments identify patients' and families' needs, monitor health and quality of life status, and can evaluate clinical interventions. Findings highlight the need for improved psychosocial support during the diagnostic period, better parent-provider communication, and shared decision-making. HRQoL measures are needed for older youth.

Initial evaluation of the Parent Cystic Fibrosis Questionnaire--Revised (CFQ-R) in infants and young children.

BACKGROUND: There is an urgent need to evaluate treatments for young children with cystic fibrosis (CF); however, efforts have been hampered by a lack of reliable, practical endpoints. To examine whether a patient-reported outcome could be reliable in children 4 to 60 months of age, we assessed the psychometric properties of the modified Parent Cystic Fibrosis Questionnaire--Revised (CFQ-R) using data from the Infant Study of Inhaled Saline (ISIS). We also characterized patterns of symptom presentation and daily functioning in children in this age range to inform future measure development. METHODS: Parents (N=314) completed the CFQ-R and Treatment Adherence Questionnaire (TAQ) at five quarterly visits, as well as a weekly Parent Symptom Diary. RESULTS: The Parent CFQ-R demonstrated good construct validity and adequate internal consistency (α's .58-.75). Associations with age, TAQ, and Parent Symptom Diary were observed. The Treatment Burden scale demonstrated responsiveness to change. CONCLUSIONS: Parents were reliable observers of young children's symptoms and daily functioning, and PROs show promise for this age group. Research is needed to identify key symptoms in infants and young children with CF, and to develop a parent proxy PRO according to FDA/EMA guidelines.

Challenges of disorders of sex development: diverse perceptions across stakeholders.

BACKGROUND/AIMS: Disorders of sex development (DSD) are congenital conditions in which chromosomal, gonadal, or anatomic sex development is atypical. Optimal management is patient- and family-centered and delivered by interdisciplinary teams. The present pilot study elicits concerns held by important stakeholders on issues affecting young patients with DSD and their families. METHODS: Content from focus groups with expert clinicians (pediatric urologists (n = 7), pediatric endocrinologists (n = 10), mental health professionals (n = 4), DSD patient advocates (n = 4), and interviews with parents of DSD-affected children (newborn to 6 years; n = 11) was coded and content-analyzed to identify health-related quality of life issues. RESULTS: Key stressors varied across stakeholder groups. In general, family-centered issues were noted more than child-centered. In the child-centered domain, providers worried more about physical functioning; family and advocates emphasized gender concerns and body image. In the family-centered domain, parental concerns about medication management outweighed those of providers. Advocates reported more stressors regarding communication/information than other stakeholders. CONCLUSION: Variability exists across stakeholder groups in the key concerns affecting young children/families with DSD. Interdisciplinary DSD healthcare team development should account for varying perspectives when counseling families and planning treatment.