A preliminary quality of life questionnaire-bronchiectasis: a patient-reported outcome measure for bronchiectasis.

BACKGROUND: The Quality of Life Questionnaire-Bronchiectasis (QOL-B) is the first disease-specific, patient-reported outcome measure for patients with bronchiectasis. Content validity, cognitive testing, responsivity to open-label treatment, and psychometric analyses are presented. METHODS: Reviews of literature, existing measures, and physician input were used to generate the initial QOL-B. Modifications following preliminary cognitive testing (N = 35 patients with bronchiectasis) generated version (V) 1.0. An open-ended patient interview study (N = 28) provided additional information and was content analyzed to derive saturation matrices, which summarized all disease-related topics mentioned by each participant. This resulted in QOL-B V2.0. Psychometric analyses were carried out using results from an open-label phase 2 trial, in which 89 patients were enrolled and treated with aztreonam for inhalation solution. Responsivity to open-label treatment was observed. Additional analyses generated QOL-B V3.0, with 37 items on eight scales: respiratory symptoms; physical, role, emotional, and social functioning; vitality; health perceptions; and treatment burden. For each scale, scores are standardized on a 0-to-100-point scale; higher scores indicate better health-related quality of life. No total score is calculated. A final cognitive testing study (N = 40) resulted in a minor change to one social functioning scale item (QOL-B V3.1). RESULTS: Content validity, cognitive testing, responsivity to open-label treatment, and initial psychometric analyses supported QOL-B items and structure. CONCLUSIONS: This interim QOL-B is a promising tool for evaluating the efficacy of new therapies for patients with bronchiectasis and for measuring symptoms, functioning, and quality of life in these patients on a routine basis. A final psychometric validation study is needed and is forthcoming. TRIAL REGISTRY: ClinicalTrials.gov; No.: NCT00805025; URL: www.clinicaltrials.gov.

Utilization of patient-reported outcomes as a step towards collaborative medicine.

Patient-reported outcomes (PROs) have been successfully developed for a variety of chronic respiratory diseases, such as asthma and cystic fibrosis (CF). They have recently been used to evaluate the efficacy of new medications and assess current patient functioning. Although regulatory bodies have favored PROs that measures symptoms, other domains of functioning, such as treatment burden, should be considered. This review examines current guidelines for the development and application of PROs in clinical trials, describes methods for selecting appropriate measures for paediatric populations, and presents a model incorporating PROs into clinical practice. Guidance on interpretation of these measures and graphic presentation of results are illustrated. PROs can serve as the link between the health care provider and patient to foster collaborative and personalized medicine. This model promotes greater patient responsibility, facilitates communication with providers, encourages shared decision-making, and enhances adherence.

Cell phone intervention to improve adherence: cystic fibrosis care team, patient, and parent perspectives.

BACKGROUND: Treatment regimens for patients with cystic fibrosis (CF) are time-consuming and complex, resulting in consistently low adherence rates. To date, few studies have evaluated innovative technologies to improve adherence in this population. Current infection control guidelines for patients with CF seek to minimize patient-to-patient transmission of potential pathogens. Thus, interventions must avoid face-to-face contact and be delivered individually, limiting opportunities for peer support. This study aimed to develop and assess a web-enabled cell phone, CFFONE, designed to provide CF information and social support to improve adherence in adolescents with CF. METHODS: The acceptability, feasibility, and utility of CFFONE were evaluated with health care professionals (n = 17) adolescents with CF aged 11-18 years old (n = 12), adults with CF aged 21-36 years old (n = 6), parents of adolescents with CF (n = 12), and technology experts (n = 8). Adolescents also tested a prototype of CFFONE (n = 9). Qualitative and quantitative data were collected. RESULTS: Focus group data with health care professionals indicated a need for this intervention, and indicated that CFFONE would be likely to improve knowledge and social support, and somewhat likely to improve adherence. Adolescent, adults, and parents all rated CFFONE as likely to improve adherence. Technology experts rated the prototype design and format as appropriate. CONCLUSIONS: The current study provided some support from key stakeholders for this intervention to improve adherence in adolescents with CF. Next steps include a multi-center trial of the efficacy and safety of CFFONE.

Anxiety and depression in cystic fibrosis.

Symptoms of anxiety and depression have important consequences for patients with chronic disease, including lower rates of adherence and increased morbidity. This review discusses the prevalence of anxiety and depression in patients with cystic fibrosis (CF) across the life span and highlights current understanding of how these symptoms affect adherence, health outcomes, and health-related quality of life (HRQOL). Anxiety and depression appear to be more common in patients with CF than in the general population. Recent studies also indicate that symptoms of anxiety occur more frequently than depression in this population. Both anxiety and depression have been shown to be associated with poorer disease outcomes, including HRQOL. Depression, specifically, leads to lower adherence to recommended therapies. Given the prevalence and impact of anxiety and depression in CF, it seems appropriate to recommend annual screening using a validated approach, with referral for either pharmacotherapy or cognitive behavioral therapy of those with apparent symptoms. A large-scale epidemiological study of anxiety and depression in CF is under way and should provide better prevalence estimates and more information on the effects of these psychological symptoms.

Brief report: the association between peer victimization, prosocial support, and treatment adherence in children and adolescents with Inflammatory Bowel Disease.

OBJECTIVE: To examine the relationship between peer victimization, prosocial support, and treatment adherence in children and adolescents with Inflammatory Bowel Disease (IBD). METHOD: Thirty-eight children diagnosed with IBD, between the ages of 7-19 years, and their parents were recruited from an outpatient Gastroenterology Clinic. Each child completed the Social Experience Questionnaire. The child, parent, and treating physician completed a one-item measure of child medication adherence. RESULTS: Child reported positive social interactions moderated the relationship between child reported peer victimization and self-reported medication adherence (t = -2.09; p = .045). These relationships held when parent report of child adherence was substituted for child reported adherence in this model (t = -2.37; p = .024). CONCLUSIONS: The findings from this pilot study suggest that prosocial support may buffer children with IBD from experiencing the more negative effects of peer victimization on treatment adherence and highlight the importance of social interactions in youth with IBD. Implications for treatment are discussed.

Impact of psychosocial factors on quality of life in overweight youth.

OBJECTIVE: The psychosocial functioning of overweight youth is a growing concern. Research has shown that overweight children report lower quality of life (QOL) than their non-overweight peers. This study sought to extend the literature by examining the association between peer victimization, child depressive symptoms, parent distress, and health-related QOL in overweight youth. Mediator models are used to assess the effect of child depressive symptoms on the relationship between psychosocial variables and QOL. RESEARCH METHODS AND PROCEDURES: The sample consisted of 96 overweight and at-risk-for-overweight children (mean age=12.8 years) and their parents who were recruited from a Pediatric Endocrinology Obesity Clinic. Parents completed a demographic questionnaire, the Pediatric Quality of Life Inventory-parent-proxy version, and the Brief Symptom Inventory. Children completed the Children's Depression Inventory-Short Form, the Schwartz Peer Victimization Scale, and the Pediatric Quality of Life Inventory. RESULTS: Increased parent distress, child depressive symptoms, and peer victimization were associated with lower QOL by both parent-proxy and self-report. Child depressive symptoms mediated the relationship between psychosocial variables (parent distress and peer victimization) for self-reported QOL but not for parent-proxy-reported QOL. DISCUSSION: This study documented the important impact of peer victimization and parental distress on the QOL of overweight children. Expanding our understanding of how overweight children experience and interact with their environment is critical. Further research is needed to examine the mechanisms by which parent distress and peer victimization impact the development of depressive symptoms in overweight children, including coping and support strategies that may buffer these children against the development of depressive symptoms and ultimately lower QOL.

A teacher-consultation approach to social skills training for pre-kindergarten children: treatment model and short-term outcome effects.

This study evaluated the post-treatment outcome effects of a classroom-based social skills program for pre-kindergarten children, using a teacher-consultation model. The pre-K RECAP (Reaching Educators, Children, and Parents) program is a semi-structured, cognitive-behavioral skills training program that provides teachers with in-classroom consultation on program implementation and classroom-wide behavior management. Data on children's social skills and behavior problems were collected from parents and teachers at pre- and post-treatment, for 149 children aged 4-5 years (of whom 56% were girls). Significant treatment effects were found for teacher but not parent reports, with treatment group children improving significantly more than comparison group children in their teacher-rated social skills and internalizing and externalizing problems. These results provide some preliminary support for the efficacy of the program on children's social skills and behavior problems, and for a teacher-consultation model for training teachers to implement school-based mental health programs.