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Karin Bæk Knudsen,1 Tacjana Pressler,2 Laust Hvas Mortensen,3 Mary Jarden,3,4 Kirsten Arntz Boisen,5 Marianne Skov,2 Alexandra L Quittner,6 Terese Lea Katzenstein1,7 1Department of Infectious Diseases, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark; 2Cystic Fibrosis Center Copenhagen, Department of Pediatric and Adolescent Medicine, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark; 3Department of Public Health, University of Copenhagen, Copenhagen, Denmark; 4University Hospital Center for Health Research (UCSF), Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark; 5Center of Adolescent Medicine, Department of Pediatric and Adolescent Medicine, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark; 6Miami Children’s Research Institute, Miami, FL, USA; 7Department of Clinical Medicine, Faculty of Health and Social Sciences, University of Copenhagen, Copenhagen, Denmark Background: Over the last two decades, lifespan has increased significantly for people living with cystic fibrosis (CF). However, several studies have demonstrated that many young adults with CF report mental health problems and poor adherence to their prescribed treatments, challenging their long-term physical health. Treatment guidelines recommend interventions to improve adherence and self-management. The aim of this study was to test the feasibility of a life coaching intervention for young adults with CF. Methods: A randomized, controlled feasibility study was conducted at the CF Center at Copenhagen University Hospital, Rigshospitalet. Participants were young adults with CF, aged 18–30 years without severe intellectual impairments. Participants were randomized to either life coaching or standard care. The intervention consisted of up to 10 individual, face-to-face or telephone coaching sessions over a period of 1 year. Primary outcomes were recruitment success, acceptability, adherence to the intervention, and retention rates. Secondary outcome measures included health-related quality of life, adherence to treatment, self-efficacy, pulmonary function, body mass index, and blood glucose values. Results: Among the 85 eligible patients approached, 40 (47%) were enrolled and randomized to the intervention or control group; two patients subsequently withdrew consent. Retention rates after 5 and 10 coaching sessions were 67% and 50%, respectively. Reasons for stopping the intervention included lack of time, poor health, perceiving coaching as not helpful, lack of motivation, and no need for further coaching. Coaching was primarily face-to-face (68%). No significant differences were found between the groups on any of the secondary outcomes. Conclusion: Both telephone and face-to-face coaching were convenient for participants, with 50% receiving the maximum offered coaching sessions. However, the dropout rate early in the intervention was a concern. In future studies, eligible participants should be screened for their interest and perceived need for support and life coaching before enrollment. Keywords: life coaching, adherence, depression, quality of life, chronic disease

Ivette Cejas,1 Michael F Hoffman,2 Alexandra L Quittner21Department of Otolaryngology, University of Miami Miller School of Medicine, 2Department of Psychology, University of Miami, Miami, FL, USAAbstract: The number of children with hearing loss with additional disabilities receiving cochlear implantation has increased dramatically over the past decade. However, little is known about their auditory and speech and language development following implantation. The purpose of this review is to evaluate the effects of cochlear implantation on the most common genetic and developmental disorders in children with hearing loss. Benefits of cochlear implantation for children with autism spectrum disorder, developmental delay, CHARGE syndrome, cerebral palsy, learning disorders, Usher syndrome, Waardenburg syndrome, and attention deficit/hyperactivity disorder are reviewed. Our review indicates that children with hearing loss and additional disabilities benefit from cochlear implantation, especially when implanted early. Thus, early interventions seem as important for these children as for deaf children without additional disabilities. Comparisons of outcomes across these disabilities indicate that children with little to no cognitive impairment (eg, Waardenburg sydrome, attention deficit hyperactivity disorder) have better outcomes than those with greater deficits in intellectual functioning (eg, autism, CHARGE syndrome). In addition, parents of children with hearing loss and additional disabilities report higher levels of parenting stress and greater child behavior problems than those without comorbid diagnoses. However, these parents are as sensitive when interacting with their children as parents with typically developing children using cochlear implantation. Given these results, it is critical to evaluate these children's developmental milestones to provide early implantation and intervention, appropriately counsel families regarding realistic expectations for the implant, and facilitate family adaptation.Keywords: cochlear implantation, outcomes, comorbid disorders, speech and language development, auditory skills, childhood deafness

Rationale: The effectiveness and safety of aztreonam lysine for inhalation (AZLI) in patients with cystic fibrosis (CF) on maintenance treatment for Pseudomonas aeruginosa (PA) airway infection was evaluated in this randomized, double-blind, placebo-controlled study.Objectives: To evaluate the safety and efficacy of inhaled aztreonam lysine in controlling PA infection in patients with CF.Methods: After randomization and a 28-day course of tobramycin inhalation solution (TIS), patients (n = 211; > or =6 yr; > or =3 TIS courses within previous year; FEV(1) > or = 25% and < or =75% predicted values) were treated with 75 mg AZLI or placebo, twice or three times daily for 28 days, then monitored for 56 days. The primary efficacy endpoint was time to need for additional inhaled or intravenous antipseudomonal antibiotics. Secondary endpoints included changes in respiratory symptoms (CF Questionnaire-Revised [CFQ-R] Respiratory Scale), pulmonary function (FEV(1)), and sputum PA density. Adverse events and minimum inhibitory concentrations of aztreonam for PA were monitored.Measurements and Main Results: AZLI treatment increased median time to need for additional antipseudomonal antibiotics for symptoms of pulmonary exacerbation by 21 days, compared with placebo (AZLI, 92 d; placebo, 71 d; P = 0.007). AZLI improved mean CFQ-R respiratory scores (5.01 points, P = 0.02), FEV(1) (6.3%, P = 0.001), and sputum PA density (-0.66 log(10) cfu/g, P = 0.006) compared with placebo; no AZLI dose-response was observed. Adverse events reported for AZLI and placebo were comparable and consistent with CF lung disease. Susceptibility of PA to aztreonam at baseline and end of therapy were similar.Conclusions: AZLI was effective in patients with CF using frequent TIS therapy. AZLI delayed time to need for inhaled or intravenous antipseudomonal antibiotics, improved respiratory symptoms and pulmonary function, and was well tolerated. Clinical trial registered with www.clinicaltrials.gov (NCT 00104520). [ABSTRACT FROM AUTHOR]

The purpose of this study was to illustrate how the daily phone diary (DPD) can be used to measure adherence behaviors in 2 pediatric pulmonary populations, cystic fibrosis (CF; n =31) and asthma (n =30). Computerized phone interview data was used to conduct activity pattern analyses, which revealed that parents of children with CF spent significantly more time in medical care and less time in recreation. Reasonable agreement was found for adherence rates between the DPD and electronic monitors. The DPD was also able to identify barriers to adherence, which included oppositional behaviors, forgetting, and competing activities. Overall, these data suggested that the DPD holds promise for measuring adherence behaviors within the family context. [ABSTRACT FROM AUTHOR]

This study examined the effectiveness of a 7-session (9 week) behavioral intervention aimed at increasing caloric intake on dietary calcium (Ca) intake in children with cystic fibrosis (CF). Diet diaries collected 2 weeks before and 2 weeks after treatment were reanalyzed for Ca intake in 44 children with CF ages 3 to 12 years of age. Paired t test analysis revealed a significant increase in mean daily dietary Ca pre- (1,006 mg/Ca/day) to posttreatment (1,467 mg/Ca/day), t(43) = 6.56, p < .001. Follow-up data on 15 participants demonstrated maintenance of these gains 12 months after treatment. Behavioral interventions appear promising for increasing dietary Ca intake in children with CF. [ABSTRACT FROM AUTHOR]

Abstract Background Adherence with tobramycin inhalation solution (TIS) during routine cystic fibrosis (CF) care may differ from recommended guidelines and affect health care utilization. Methods We analyzed 2001-2006 healthcare claims data from 45 large employers. Study subjects had diagnoses of CF and at least 1 prescription for TIS. We measured adherence as the number of TIS therapy cycles completed during the year and categorized overall adherence as: low ≤ 2 cycles, medium >2 to Results Among 804 individuals identified with CF and a prescription for TIS, only 7% (n = 54) received ≥ 4 cycles of TIS per year. High adherence with TIS was associated with a decreased risk of hospitalization when compared to individuals receiving ≤ 2 cycles (adjusted odds ratio 0.40; 95% confidence interval 0.19-0.84). High adherence with TIS was also associated with lower outpatient service costs (IQR: $2,159-$8444 vs. $2,410-$14,423) and higher outpatient prescription drug costs (IQR: $35,125-$60,969 vs. $10,353-$46,768). Conclusions Use of TIS did not reflect recommended guidelines and may impact other health care utilization.

Abstract Background As more patients with cystic fibrosis (CF) reach adulthood and participate in age-appropriate activities (e.g. employment, dating), disclosure of medical status becomes more important. This study assessed rates of disclosure and its perceived impact on relationships using the Adult Data for Understanding Lifestyle and Transitions (ADULT) online survey. Methods Adults with CF participated in the survey via the United States national network of CF Centers. Descriptive and inferential statistics were utilized. Results Participants (n = 865) were more likely to disclose to relatives (94%) and close friends (81%) than to dating partners (73%), bosses/supervisors/teachers (51%) or co-workers (39%). Respondents generally reported a neutral/positive effect on relationships following disclosure. Negative effects of disclosure were infrequent, but more likely with dating partners or bosses/supervisors/teachers. Results also indicated that disclosure may be influenced by severity of lung disease and gender, with those having normal/mild lung disease less likely to disclose their diagnosis to both co-workers (p < 0.01) and bosses/supervisors/teachers (p < 0.01), and women being more likely to disclose to close friends (p < 0.0001) and dating partners (p < 0.05) than men. Conclusions Most adults with CF disclosed their disease to relatives and close friends. Individuals with severe CF lung disease were more likely to disclose their diagnosis to coworkers and supervisors/teachers. It may be helpful to provide support for disclosure of disease in situations such as employment and dating.

Objectives: To examine the effects of observed maternal sensitivity (MS), cognitive stimulation (CS), and linguistic stimulation on the 4-year growth of oral language in young, deaf children receiving a cochlear implant. Previous studies of cochlear implants have not considered the effects of parental behaviors on language outcomes.Study Design: In this prospective, multisite study, we evaluated parent-child interactions during structured and unstructured play tasks and their effects on oral language development in 188 deaf children receiving a cochlear implant and 97 normal-hearing children as controls. Parent-child interactions were rated on a 7-point scale using the National Institute of Child Health and Human Development's Early Childcare Study codes, which have well-established psychometric properties. Language was assessed using the MacArthur Bates Communicative Development Inventories, the Reynell Developmental Language Scales, and the Comprehensive Assessment of Spoken Language.Results: We used mixed longitudinal modeling to test our hypotheses. After accounting for early hearing experience and child and family demographics, MS and CS predicted significant increases in the growth of oral language. Linguistic stimulation was related to language growth only in the context of high MS.Conclusion: The magnitude of effects of MS and CS on the growth of language was similar to that found for age at cochlear implantation, suggesting that addressing parenting behaviors is a critical target for early language learning after implantation. [ABSTRACT FROM AUTHOR]

Background: Adults with cystic fibrosis (AWCF) have higher rates of depression and anxiety than comparable community members. This multisite randomized waitlist-controlled trial tested the efficacy of "Coping and Learning to Manage Stress with CF" (CALM), a 6-session+booster telehealth intervention to improve depression and anxiety symptoms (primary outcomes) and perceived stress, coping self-efficacy, and key health-related quality of life domains (secondary outcomes)., Methods: AWCF reporting mild to severe symptoms of depression and/or anxiety were randomized to receive CALM immediately (immediate, n = 66) or after a 13-week delay (waitlist, n = 66). Group differences post-intervention and at 1-month were examined via linear mixed models. Maintenance of treatment gains from baseline to 3-month follow-up was examined using combined data from both groups. Effect size calculations using Cohen's d assessed treatment effect magnitude., Results: Compared to the waitlist group, those that received CALM immediately reported lower depression and anxiety symptoms post-intervention and at 1-month follow-up (ps<0.001). For depression there was a large effect size post-intervention (d = 0.85) and a medium effect size at 1-month follow-up (d = 0.70); anxiety had a medium effect size post-intervention (d = 0.65) and at 1-month follow-up (d = 0.66). The immediate group also reported significantly higher coping self-efficacy, less stress, and increased vitality post-CALM and at 1-month follow-up (ps<0.01). Treatment gains were maintained at 3-month follow-up for all outcomes., Conclusions: CALM was efficacious for AWCF in reducing symptoms of depression, anxiety, and perceived stress while improving coping self-efficacy and vitality with evidence of treatment sustainability. Next steps are dissemination and implementation to CF psychosocial clinicians., Competing Interests: Declaration of competing interest No conflicts to disclose (Bathgate, Smith, Murphy, Quittner, Riekert, Goralski, Holm)., (Copyright © 2024 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.)

Background: The cystic fibrosis (CF) regimen is time-consuming and burdensome leading to barriers to self-management. This mixed-methods study developed the Daily Care Check-in (DCC) that is specific to the barriers faced by people with CF (PWCF) and evaluated its validity., Methods: Qualitative methods were used to identify barriers to self-management and develop items, with "think aloud" cognitive interviews conducted to refine the items. A multisite, cross-sectional study was conducted to test the internal consistency, test-retest reliability, and validity of the DCC scores, comparing them to objective medication adherence (composite medication possession ratio (cMPR)) and psychosocial measures (self-efficacy, medication beliefs, executive functioning, depressive and anxiety symptoms, treatment burden, and treatment complexity)., Results: The DCC (18 items) includes two scales: Occurrence (score range 0-18) and Interference (score range 0-90). 405 participants completed the DCC, 344 (85 %) completed the survey, and 365 (90 %) had a cMPR calculated. On average, 6.8 barriers were reported (SD = 4.2 Occurrence Scale), and the Interference Scale had a mean score of 18.4 (SD = 14.0). Reliability was acceptable to good. cMPR was negatively correlated with the DCC (rho=-0.26, Occurrence and rho = -0.31, Interference, p-values<0.0001). A priori hypotheses between the DCC and the other measures were supported and demonstrated construct validity., Conclusions: This study provides evidence supporting the validity of the DCC for assessing the presence and impact of barriers to CF self-management, including medication adherence. Formal screening of self-management barriers (e.g., using the DCC) should be considered to facilitate conversations with the care team and identify tailored interventions to support CF self-management., Competing Interests: Declaration of competing interest KAR, CF, AG, TE, AG, and ALQ, received/received grant/salary support from the Cystic Fibrosis Foundation. ALQ has received royalties from iQVIA, consulting fees from Insmed Inc. and Vertex Pharmaceuticals Inc., and honoraria from Vertex Pharmaceuticals Inc., (Copyright © 2024 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.)

Objective: To estimate costs of severe to profound hearing loss, including costs and cost-savings associated with cochlear implantation., Methods: Data was obtained from the National Health Interview Survey, the National Health and Nutrition Examination Survey and national Medicare rates. We used continuous time state transition models with individual patient simulations to estimate the costs of severe to profound hearing loss (SPHL) across the lifespan. The model included four states, normal hearing, severe to profound hearing loss, cochlear implantation, and death., Results: The estimated lifetime cost of an individual born with SPHL is $489,274 [377,518; 616,519]. Costs are lower for those who received a cochlear implant before 18 months of age $390,931 [311,976; 471,475], compared to those who are not implanted $608,167 [442,544; 791,719]. For individuals with a later onset of hearing loss (60 years old) lifetime costs were $154,536 [7,093; 302,936]. The annual societal costs for the US population were estimated to be $37 [8; 187] billion., Conclusions: SPHL is a costly condition, with the primary driver being lost productivity. Medical costs were higher for cochlear implantation, however, the higher income earnings offset the higher medical costs. Overall, early implantation substantially reduced lifetime costs. Access to hearing health care and technology is critical given the documented benefits for language, education, and quality of life. Government and insurance policies should be modified to allow for equal access and coverage for hearing technology, which will ultimately reduce lifetime and societal costs., Levels of Evidence: N/A The current study used existing nationally representative datasets. Thus, these levels of evidence do not apply. Laryngoscope, 134:4358-4365, 2024., (© 2024 The Authors. The Laryngoscope published by Wiley Periodicals LLC on behalf of The American Laryngological, Rhinological and Otological Society, Inc.)

Objective: This study aimed to compare the psychometric performance of the Cystic Fibrosis Questionnaire-Revised-8 Dimensions (CFQ-R-8D), a new, condition-specific, preference-based measure, with that of generic preference-based measures EQ-5D-3L and Short Form 6 dimensions (SF-6D)., Methods: Data from three trials of participants with CF aged ≥ 14 years who completed the CFQ-R and EQ-5D-3L or SF-6D were used. Analyses were undertaken to evaluate convergent validity based on correlations with CFQ-R domain scores. Known-group validity was assessed based on percent predicted forced expiratory volume in one second and pulmonary exacerbations. Responsiveness was based on correlation of change and sensitivity to change based on change in symptom severity. Effect sizes and standardized response means were estimated., Results: CFQ-R-8D utilities and dimensions were strongly correlated with most of the overlapping CFQ-R domain scores (ρ > 0.5); EQ-5D-3L and SF-6D utilities and dimensions had moderate (ρ > 0.3) to strong correlations in dimensions capturing similar concepts. All measures showed evidence of known-group validity (P < 0.05). Change correlations were strong for CFQ-R-8D utilities and dimensions and CFQ-R, but they were moderate for SF-6D and mostly weak ((ρ > 0.1) for EQ-5D-3L. The SF-6D had the largest mean change over time and effect sizes, followed by CFQ-R-8D and then EQ-5D-3L. Neither CFQ-R-8D or SF-6D utility scores had ceiling effects (< 9% responses in full health) compared with those of EQ-5D-3L (61-62%). In participants classified as being in full health by EQ-5D-3L, CFQ-R-8D captured CF-specific health problems, particularly cough, abdominal pain, and breathing difficulty., Conclusions: The CFQ-R-8D reflected known-group differences and changes over time with stronger evidence of good psychometric performance than EQ-5D-3L and similar evidence as SF-6D. Additionally, the CFQ-R-8D captured more condition-specific symptoms than EQ-5D-3L or SF-6D, which are important determinants of health-related quality of life for people with CF., (© 2024. The Author(s).)

Airway inflammation underlies cystic fibrosis (CF) pulmonary exacerbations. In a prospective multicenter study of randomly selected, clinically stable adolescents and adults, we assessed relationships between 24 inflammation-associated molecules and the future occurrence of CF pulmonary exacerbation using proportional hazards models. We explored relationships for potential confounding or mediation by clinical factors and assessed sensitivities to treatments including CF transmembrane regulator (CFTR) protein synthesis modulators. Results from 114 participants, including seven on ivacaftor or lumacaftor-ivacaftor, representative of the US CF population during the study period, identified 10 biomarkers associated with future exacerbations mediated by percent predicted forced expiratory volume in 1 s. The findings were not sensitive to anti-inflammatory, antibiotic, and CFTR modulator treatments. The analyses suggest that combination treatments addressing RAGE-axis inflammation, protease-mediated injury, and oxidative stress might prevent pulmonary exacerbations. Our work may apply to other airway inflammatory diseases such as bronchiectasis and the acute respiratory distress syndrome., Competing Interests: T.G.L., J.A.F., J.L.J., Y.L., K.A.P., and J.B.V. received other support from the CFF (CC132-16AD, LIOU14Y0, LIOU14P0) and the National Heart Lung and Blood Institute (NHLBI) of the National Institutes of Health (NIH) (R01 HL125520) and received support during the current study for performing clinical trials from Abbvie, Calithera Biosciences, Corbus Pharmaceuticals, Gilead Sciences, Laurent Pharmaceuticals, Nivalis Therapeutics, Novartis, Proteostasis, Savara Pharmaceuticals, Translate Bio, and Vertex Pharmaceuticals. F.R.A. received additional other support from the NHLBI/NIH (R01 HL125520), the National Science Foundation (EMSW21-RTG), and the Margolis Family Foundation of Utah. P.S.B. received other support from the CFF (Center and TDC grants) and the NHLBI/NIH (U01 HL114623) and received support for a clinical trial from Alcresta Therapeutics. B.A.C. received other support from the CFF (C112–12, C112-TDC09Y, 10063SUB, 41339154.s132P010379SUB) and received support for clinical trials from Genentech, Novartis, and Vertex Pharmaceuticals. C.L.D. received other support from the CFF (C004–11, C004-TDC09Y, DAINES11Y3) and from the Health Resources and Services Administration (T72MC00012). J.A.F. transitioned to become an employee of ICON plc, a clinical research organization involved in various trials pertinent to CF during the study and is now an employee of Vertex Pharmaceuticals; JAF, ICON, and Vertex had no direct involvement in performance of the study following the initial change in affiliation. T.H. received other support from the CFF (PACE, Center Grant) and received support for clinical trials from Celtaxsys and Vertex Pharmaceuticals. J.R.H. received other support from the NHLBI/NIH (HHSN268200900018C) and the Veterans Administration Healthcare System (I01 BX001533). J.L. received other support from the CFF (C017-11AF). C.N. received other support from the CFF (C138-12). P.R. received other support from the CFF (C003–12, C003-TDC09Y). S.D.S. received other support from the CFF (AQUADEK12K1, SAGEL11CS0, GOAL13K2, NICK13A0, SAGEL14K1, NICK15R0) and the NHLBI/NIH (U54 HL096458) and the NCATS/NIH (Colorado CTSA Grant Number UL1 TR002535). J.L.T.-C. received other support from the CFF (TDC) and the NHLBI/NIH (HL103801) and received support for clinical trials from Vertex Pharmaceuticals. K.N.O. was funded by the intramural research program of the NHLBI, NIH, during the study. Neither the project sponsors nor any sources of other support had direct roles in development and conduct of the study. None of the sponsors of clinical trials mentioned earlier participated in any way with this trial., (© 2024 The Author(s).)

Objective: Treatments for cystic fibrosis (CF) are complex, labour-intensive, and perceived as highly burdensome by caregivers of children with CF. An instrument assessing burden of care is needed., Design: A stepwise, qualitative design was used to create the CLCF with caregiver focus groups, participant researchers, a multidisciplinary professional panel, and cognitive interviews., Main Outcome Measures: Preliminary psychometric analyses evaluated the reliability and convergent validity of the CLCF scores. Cronbach's alpha assessed internal consistency and t-tests examined test-retest reliability. Correlations measured convergence between the Treatment Burden scale of the Cystic Fibrosis Questionnaire-Revised (CFQ-R) and the CLCF. Discriminant validity was assessed by comparing CLCF scores in one vs two-parent families, across ages, and in children with vs without Pseudomonas aeruginosa ( PA )., Results: Six Challenge subscales emerged from the qualitative data and the professional panel constructed a scoresheet estimating the Time and Effort required for treatments. Internal consistency and test-retest reliability were adequate. Good convergence was found between the Total Challenge score and Treatment Burden on the CFQ-R ( r =-0.49, p  = 0.02, n  = 31). A recent PA infection signalled higher Total Challenge for caregivers ( F (23)11.72, p  = 0.002)., Conclusions: The CLCF, developed in partnership with parents/caregivers and CF professionals, is a timely, disease-specific burden measure for clinical research.

Importance: Cochlear implants (CIs) have been shown to be effective in improving auditory skills and speech and language development. However, less is known about the long-term outcomes of CIs on educational functioning or quality of life., Objective: To evaluate long-term educational outcomes and quality of life in adolescents over 13 years postimplantation., Design, Setting, and Participants: This longitudinal cohort study included 188 children with bilateral severe to profound hearing loss with CIs from the Childhood Development After Cochlear Implantation (CDaCI) study from hospital-based CI programs; a cohort of 340 children with severe to profound hearing loss without CIs from a nationally representative survey (National Longitudinal Transition Study-2; NLTS-2), and results from the literature of comparable children without CIs., Exposure(s): Cochlear implantation (early and late)., Main Outcomes and Measures: Adolescent performance on measures of academic achievement (Woodcock Johnson), language (Comprehensive Assessment of Spoken Language), and quality of life (Pediatric Quality of Life Inventory, Youth Quality of Life Instrument-Deaf and Hard of Hearing)., Results: The CDaCI cohort included 188 children, 136 of whom completed the wave 3 postimplantation follow-up visits (77 [55%] female) with CIs; mean [SD] age was 11.47 [1.27] years. The NLTS-2 cohort included 340 children (50% female) with severe to profound hearing loss without CIs. Children with CIs had better academic performance compared with children without CIs with similar levels of hearing loss. The largest benefits were seen for children who received implants early (prior to age 18 months), who performed at or above age and gender norms for language and academic achievement. Similarly, adolescents with CIs reported better quality of life on the Pediatric Quality of Life Inventory compared with children without CIs. On a condition-specific measure (Youth Quality of Life Instrument-Deaf and Hard of Hearing), children who received implants early scored higher across all 3 domains than comparisons without CIs., Conclusions and Relevance: To our knowledge, this is the first study to evaluate long-term educational outcomes and quality of life in adolescents using CIs. This longitudinal cohort study showed better outcomes of CIs in terms of language, academic performance, and quality of life. While the greatest benefits were observed for children who received implants before age 18 months, benefits were also noted for children who received implants later, providing evidence that children with severe to profound hearing loss with CIs can achieve at or above expected levels compared with hearing peers.

Background: Nontuberculous mycobacteria (NTM), predominately Mycobacterium avium complex (MAC), cause chronic pulmonary disease. Improvements in symptoms and health-related quality of life (HRQoL) are important treatment outcomes, but no validated patient-reported outcome (PRO) measure exists., Research Question: What are the validity and responsiveness of the Quality of Life-Bronchiectasis (QOL-B) questionnaire respiratory symptoms scale and key HRQoL measures during the first 6 months of MAC pulmonary disease (MAC-PD) treatment?, Study Design and Methods: Comparison of Two- vs Three-antibiotic Therapy for Pulmonary Mycobacterium Avium Complex Disease (MAC2v3) is an ongoing randomized, multisite pragmatic clinical trial. Patients with MAC-PD were randomized to azithromycin-based two-drug or three-drug therapy; treatment groups were combined for this analysis. PROs were measured at baseline, 3 months, and 6 months. The QOL-B respiratory symptoms, vitality, physical functioning, health perceptions, and NTM symptom domain scores (on a scale of 0-100, with 100 being best) were analyzed separately. We performed psychometric and descriptive analyses in the population enrolled as of the time of analysis and calculated the minimal important difference (MID) using distribution-based methods. Finally, we evaluated responsiveness using paired t tests and latent growth curve analysis in the subset with longitudinal surveys completed by the time of analysis., Results: The baseline population included 228 patients, of whom 144 had completed longitudinal surveys. Patients predominately were female (82%) and had bronchiectasis (88%); 50% were 70 years of age or older. The respiratory symptoms domain showed good psychometric properties (no floor or ceiling effects; Cronbach's α, 0.85) and an MID of 6.4 to 6.9. Vitality and health perceptions domain scores performed similarly. Respiratory symptoms domain scores improved by 7.8 points (P < .0001) and 7.5 points (P < .0001), and the physical functioning domain score improved by 4.6 points (P < .003) and 4.2 points (P = .01) at 3 and 6 months, respectively. Latent growth curve analysis confirmed a nonlinear, statistically significant improvement in respiratory symptoms and physical functioning domain scores by 3 months., Interpretation: The QOL-B respiratory symptoms and physical functioning scales exhibited good psychometric properties in patients with MAC-PD. Respiratory symptoms scores improved beyond the MID by 3 months after treatment initiation., Trial Registry: ClinicalTrials.gov; No.: NCT03672630; URL: www., Clinicaltrials: gov., (Copyright © 2023 American College of Chest Physicians. Published by Elsevier Inc. All rights reserved.)

Objectives: The CF Foundation sponsored competitive awards for Mental Health Coordinators (MHCs) from 2016 to 2018 to implement the international guidelines for mental health screening and treatment in US CF centers. Longitudinal surveys evaluated success in implementing these guidelines using the Consolidated Framework for Implementation Research (CFIR)., Methods: MHCs completed annual surveys assessing implementation from preparation/basic implementation (e.g., using recommended screeners) to full implementation/sustainability (e.g., providing evidence-based treatments). Points were assigned to questions through consensus, with higher scores assigned to more complex tasks. Linear regression and mixed effects models were used to: (1) examine differences in centers and MHC characteristics, (2) identify predictors of success, (3) model the longitudinal trajectory of implementation scores., Results: A total of 122 MHCs (88.4% responded): Cohort 1, N = 80; Cohort 2, N = 30; and Cohort 3, N = 12. No differences in center characteristics were found. Significant improvements in implementation were observed across centers over time. Years of experience on a CF team was the only significant predictor of success; those with 1-5 years or longer reported the highest implementation scores. Change over time was predicted by >5 years of experience., Conclusions: Implementation of the mental health guidelines was highly successful over time. Funding for MHCs with dedicated time was critical. Longitudinal modeling indicated that CF centers with diverse characteristics could implement them, supported by evidence from the CF Patient Registry showing nearly universal uptake of mental health screening in the United States. Years of experience predicted better implementation, suggesting that education and training of MHCs and retention of experienced providers are critical to success., (© 2023 Wiley Periodicals LLC.)

Objectives: Cystic fibrosis (CF) limits survival and negatively affects health-related quality of life (HRQOL). Cost-effectiveness analysis (CEA) may be used to make reimbursement decisions for new CF treatments; nevertheless, generic utility measures used in CEA, such as EQ-5D, are insensitive to meaningful changes in lung function and HRQOL in CF. Here we develop a new, CF disease-specific, preference-based utility measure based on the adolescent/adult version of the Cystic Fibrosis Questionnaire-Revised (CFQ-R), a widely used, CF-specific, patient-reported measure of HRQOL., Methods: Blinded CFQ-R data from 4 clinical trials (NCT02347657, NCT02392234, NCT01807923, and NCT01807949) were used to identify discriminating items for a classification system using psychometric (eg, factor and Rasch) analyses. Thirty-two health states were selected for a time trade-off (TTO) exercise with a representative sample of the UK general population. TTO utilities were used to estimate a preference-based scoring algorithm by regression analysis (tobit models with robust standard errors clustered on participants with censoring at -1)., Results: A classification system with 8 dimensions (CFQ-R-8 dimensions; physical functioning, vitality, emotion, role functioning, breathing difficulty, cough, abdominal pain, and body image) was generated. TTO was completed by 400 participants (mean age, 47.3 years; 49.8% female). Among the regression models evaluated, the tobit heteroscedastic-ordered model was preferred, with a predicted utility range from 0.236 to 1, no logical inconsistencies, and a mean absolute error of 0.032., Conclusion: The CFQ-R-8 dimensions is the first disease-specific, preference-based scoring algorithm for CF, enabling estimation of disease-specific utilities for CEA based on the well-validated and widely used CFQ-R., (Copyright © 2023. Published by Elsevier Inc.)

Introduction: Individuals with chronic respiratory diseases and caregivers are at higher risk for depression and anxiety. Primary ciliary dyskinesia (PCD) and cystic fibrosis (CF) are both rare genetic diseases, characterized by recurrent respiratory infections. This study compared depression and anxiety in people with PCD (pwPCD) and CF (pwCF), and caregivers, using the screening tools recommended in the CF guidelines., Methods: Patient Health Questionnaire (PHQ-9) and Generalized Anxiety Disorder (GAD-7) were administered to a PCD and CF sample. Given that PCD is extremely rare, they were matched on age and sex to pwCF at a 1:2 ratio. Similar procedures were performed with parents., Results: A total of 63 patients and 129 caregivers participated: 21 pwPCD and 42 pwCF (ages 12-34 years) plus 43 caregivers of pwPCD and 86 caregivers of pwCF. A high percentage of patients scored above the cut-off for depression (PCD: 33%; CF: 43%) and anxiety (PCD and CF both: 43%), mostly mild. Similarly, a high percentage of caregivers scored above the cut-off for depression (PCD: 42-54%; CF: 45-46%) and anxiety (PCD: 47-54%; CF: 39-56%). Suicidal ideation was endorsed by 9.5% of pwPCD, 20% of mothers and 10% of fathers and 5% of pwCF, 3% of mothers, but no fathers., Conclusion: A large percentage of patients and caregivers reported elevated psychological distress and suicidal ideation. Addressing psychological symptoms is critical given they are associated with poor adherence, missed clinic visits, increased inflammation and worse quality of life. Mental health screening and treatment should be integrated into PCD care., Competing Interests: Declaration of competing interest There are no conflicts of interest., (Copyright © 2022 Elsevier Ltd. All rights reserved.)

Background: Airway clearance therapies (ACTs) are recommended as an integral part of the management of non-cystic fibrosis bronchiectasis (BE) to prevent inflammation, mucus accumulation, and infection that occur because of ineffective secretion clearance. Adherence to ACTs is low, in part because of perceived burden and a lack of standardization of education and training programs for patients. Poor adherence is associated with more frequent exacerbations, worse health outcomes, and worse quality of life. Structured educational programs increase adherence to ACT among people with cystic fibrosis and may show similar results for people with BE., Objective: This pilot study evaluated the feasibility, clinical utility, sustainability, and expert opinions of this educational program addressing gaps in ACT knowledge and skills in people with BE., Methods: The Individual Management of Patient Airway Clearance Therapy- Bronchiectasis (IMPACT BE) was implemented in nine BE centers with 100 patients. Qualitative and quantitative data were collected from patients and providers., Results: The IMPACT BE program demonstrated good uptake in a clinic setting by multidisciplinary team members, with improvements in medical teams' evaluation of their ability to provide education to patients. All healthcare teams indicated that this program could become a sustainable part of their clinic. Qualitative responses from patients indicated the program was comprehensive and easy to use., Conclusion: In this pilot study, IMPACT BE was found to be useful in teaching airway clearance to people with BE. The open-access toolkit was well received by both patients and a diverse array of providers in a clinic setting., (Copyright © 2023 by the American Thoracic Society.)

Background: A cystic fibrosis (CF)-specific cognitive-behavioral therapy intervention (CF-CBT) was developed in partnership with the CF community to advance preventive mental health care. Multidisciplinary providers across three centers were trained to deliver CF-CBT for this pilot assessing feasibility/acceptability and preliminary effectiveness of an integrated model of care., Methods: The 8-session CF-CBT was delivered to 14 adults with mild depression and/or anxiety symptoms in-person and via audio telehealth. Assessment of attrition, engagement, homework completion, treatment satisfaction, and treatment fidelity informed feasibility/acceptability assessment. Mental health outcomes included depression, anxiety, quality of life (Cystic Fibrosis Questionnaire-Revised [CFQ-R), perceived stress and coping. Preliminary effectiveness was evaluated with Cohen's d metric of effect sizes (ES) of pre-post mean change scores., Results: A total of 108 sessions were conducted; 13 adults completed the intervention; 1 discontinued early. Engagement, homework completion, and treatment acceptability were highly rated (mean = 30; SD = 2, range: 27-32 on a 32-point scale). Fidelity scores ranged from 85.7% to 93.6%. Large ES changes reflected improvements in depressive symptoms (-0.83), CFQ-R (Vitality scale: 1.11), and Relaxation Skills (0.93); moderate ES for CFQ-R Role Functioning (0.63), Awareness of Tension (0.62), Coping Confidence (0.70) and CF-specific Coping (0.55); and small ES for anxiety symptoms (-0.22), perceived stress (-0.25), Behavioral Activation (0.29), and several CFQ-R domains, including Emotional Functioning (0.29). Two CFQ-R subscales decreased (Body Image, Eating Concerns)., Conclusions: Results indicated feasibility and acceptability of CF-CBT and its integration into team-based CF care with promising effectiveness, especially for depression. A multicenter randomized controlled trial of CF-CBT will further examine effectiveness of a CF-specific integrated care model., (© 2022 Wiley Periodicals LLC.)

Background: The spread of COVID-19 was associated with increased stress and new mental health concerns for people with cystic fibrosis (pwCF), already at increased risk for depression and anxiety. This study assessed stress and mental health in adolescents and young adults with CF 1 year from when the pandemic began., Methods: Sixty-six pwCF (mean age = 24; range 14-36) completed a new measure of the impact of COVID-19 (COVID-19 Exposure and Family Impact Scale-Adolescence and Young Adult; CEFIS-AYA); the Patient Health Questionnaire-9, and the Generalized Anxiety Disorder-7. The Italian translation of the CEFIS-AYA was performed., Results: On the CEFIS-AYA, the mean Exposure score was 5.2 (SD = 2.6) out of 28. The mean Impact score was 1.8 (SD = 0.7; negative valence &gt; 2.5). Individuals were more sedentary and undertaking less exercise. Average stress rating was 5.9 (SD = 2), indicating moderate stress. No significant differences were found between those who did (N = 12) and who did not have a COVID infection (N = 54). A high percentage of participants scored above the clinical cut-off for depression (45%) and anxiety (41%), with a low proportion reporting moderate-severe symptomatology., Conclusion: After 1 year, the pandemic was having a less significant impact on patients' daily lives. Sedentary activity and reduced exercise were common. Despite expectations that this group was particularly vulnerable, depression and anxiety scores were similar to the rates described for this population before the pandemic. Overall, these results suggested that pwCF are highly resilient and nearly 1 year after the onset of COVID-19, have returned to similar daily activities and emotional health., (© 2022 Wiley Periodicals LLC.)

Individuals with cystic fibrosis (CF) are at high risk for depression and anxiety, with negative consequences for health and quality of life. Cystic Fibrosis Foundation/European Cystic Fibrosis Society guidelines recommend routine screening, treatment, and preventative efforts. Cognitive-behavioral therapy (CBT) has a large evidence-base for depression/anxiety prevention and treatment. However, traditional CBT protocols require adaptation to address the emotional challenges of coping with CF, stressors related to disease management, and barriers to access to care. The goal of this study was to partner with the CF community to develop an innovative CBT-based intervention for the prevention and treatment of depression and anxiety tailored to CF-specific needs. In-depth feedback was collected via audio-recorded telephone interviews with 16 adults with CF from 3 U.S. CF centers, with purposive sampling across gender, age, ethnicity, and disease severity. A semi-structured interview guide elicited discussion of patient experiences of coping with CF, and perspectives on the acceptability of the content, structure, and delivery model of the proposed intervention. Qualitative analysis utilized a content analytic approach. Participants ranged from 21 to 53 years ( M  = 35); eight were female; three were Hispanic. Patient-reported most recent FEV1, a measure of lung function based on forced expiratory volume in in one second, ranged from 25 to 113% predicted ( M  = 72). One participant was post-double lung transplant. Qualitative interviews were analyzed thematically revealing core themes related to the experience of coping with CF. The most frequently cited CF-related stressors were Treatment Burden, Illness Uncertainty, and Financial/Insurance Stress. Participants talked about the interaction of physical symptoms and emotional distress in their daily lives, a topic not typically discussed in routine CF care. Resilience was also a major theme with participants describing strategies they use to cope with CF and hospitalizations. Description of patients' experiences was incorporated into the program's intervention manual and patient workbook. Participants also provided direct feedback on the proposed program. Feedback was largely positive regarding program content and structure, suggesting the acceptability of a CF-specific CBT-based intervention for adults with CF. Features to increase accessibility of care including telehealth, inpatient delivery, and team-based care were perceived as advantageous, and participants emphasized the value of a CF -specific mental health intervention. Qualitative findings directly informed the development of CF-CBT, a cognitive-behavioral skills-based program to promote emotional well-being for adults with CF., Competing Interests: DF reports grant support from the Cystic Fibrosis Foundation (CFF), Dutch Cystic Fibrosis Foundation, and Vertex Pharmaceuticals, and travel reimbursement from the CFF. BS reports grant support, personal fees and travel reimbursement from CFF. AQ reports consulting fees from Vertex Pharmaceuticals and Insmed, and grant support from the CFF, American Cochlear Implant Alliance, PCORI, and the NIH. AG reports personal fees and travel reimbursement from Cystic Fibrosis Australia; grant support, personal fees, and travel reimbursement from CFF; grant support from the Dutch Cystic Fibrosis Foundation; travel reimbursement from the European Cystic Fibrosis Society; personal fees from Johns Hopkins University/DKBmed; personal fees from Saudi Pediatric Pulmonology Association; and grant support and personal fees from Vertex Pharmaceuticals. MK reports no commercial or financial relationships that could be construed as a potential conflict of interest., (Copyright © 2022 Friedman, Kaskas, Quittner, Smith and Georgiopoulos.)

Competing Interests: Declaration of competing interest Dr Edwina Landau reports speaker reimbursement from the European Cystic Fibrosis Society. Dr Marieke Verkleij reports grants from the Dutch Cystic Fibrosis Foundation, speaker and travel reimbursement from the European Cystic Fibrosis Society and grants from Vertex Pharmaceuticals. Dr Anna Georgiopoulos reports personal fees and travel reimbursement from Cystic Fibrosis Australia; grants, personal fees, and travel reimbursement from Cystic Fibrosis Foundation; grants from the Dutch Cystic Fibrosis Foundation; travel reimbursement from the European Cystic Fibrosis Society; personal fees from Johns Hopkins University/DKBmed; personal fees from Saudi Pediatric Pulmonology Association; grants and personal fees from Vertex Pharmaceuticals. Dr Alexandra Quittner reports grants from the NIH, CF Foundation, and the American Cochlear Implant Alliance; grant and travel reimbursement from Cystic Fibrosis Australia; consulting fees from Vertex Pharmaceuticals and Insmed, Inc. Dr Michael Schechter reports research grants from the NIH, CFF, Abbvie, GlaxoSmithKline, Vertex, consulting fees from Astra-Zeneca and CFF, speaking fees from Sanofi Pasteur.

Objectives: The purpose of this study was to develop and validate a cochlear implant (CI)-specific parenting stress measure using the FDA Guidance on Patient-Reported Outcomes (2009)., Methods: The development and psychometric validation of the Parenting Stress-CI module for both the Early Childhood (EC; 0-5 years) and School-Age (SA; 6-12 years) versions are reported in this article. Instrument development consisted of qualitative interviews with parents of children with CIs (EC: N = 19; SA: N = 21), content analysis, item development, and cognitive testing of the instrument. Last, we conducted the psychometric validation (EC: N = 72; SA: N = 64), including analyses of internal consistency, test-retest reliability (∼2 weeks between administrations; N = 24), and convergent validity with the Parenting Stress Index-4 (PSI-4)., Results: The final EC version includes 15 questions, and the SA version includes 8 questions. Both the EC and SA versions had strong reliability (EC α = .88; SA α = .85), with all items significantly correlated with the overall module (r = .43-.80). Both versions also had strong test-retest reliability (r = .99, p < .001). Last, analyses of convergent validity demonstrated significant correlations with the PSI-4 Total Stress scale for both Parenting Stress-CI versions (EC r = .66, p < .00; SA r = .45, p < .001)., Conclusions: The Parenting Stress-CI modules are reliable and valid condition-specific parenting stress instruments for parents of children with CIs ages 0-12 years, filling a significant gap in the literature. These fully validated instruments can be used to assess parental needs for support and guide the development of targeted, family centered interventions., (© The Author(s) 2022. Published by Oxford University Press on behalf of the Society of Pediatric Psychology. All rights reserved. For permissions, please e-mail: journals.permissions@oup.com.)

Objective: This study evaluated associations among parenting stress, self-efficacy, and involvement in relation to spoken language outcomes in young children 3 years following cochlear implantation., Study Design: Cross-sectional., Setting: Six university tertiary medical centers., Patients: One hundred sixty-four young children with bilateral, severe-to-profound sensorineural hearing loss who had 3 years of experience with a CI; children with substantial cognitive impairments were excluded from the study., Main Outcome Measuress: Family Stress Scale (FSS), Scale of Parental Involvement and Self-Efficacy (SPISE), Oral and Written Language Scales (OWLS)., Results: Correlations were of moderate strength between FSS scores and SPISE scores (Parental Self-Efficacy, r = -0.45, p < 0.01, Parental Involvement r = -0.32, p < 0.01). As hypothesized, parents reporting higher levels of stress reported lower perceptions of self-efficacy and involvement. In addition, results showed that family stress had a direct, negative effect on spoken language (-4.43 [95% confidence interval: -6.97; -1.89]). After controlling for maternal education and activation age, parental self-efficacy mediated the negative effect between family stress and spoken language (indirect effect = -1.91 [3.45; -0.69]; proportion mediated = 0.43). No mediating effects were found for parental involvement., Conclusions: These findings highlight the need for parenting interventions that focus on reducing stressors and increasing parents' perceptions of self-efficacy in families of children using cochlear implants. Integration of mental health screening and tailored parenting interventions in CI clinics may increase parental self-efficacy and involvement, with measurable benefits in the child's use of spoken language., Competing Interests: The authors disclose no conflicts of interest., (Copyright © 2021, Otology & Neurotology, Inc.)

Background: Depression and anxiety are two to four times more prevalent in people with CF (pwCF) than the general population. COVID-19 may exacerbate mental health challenges, increasing demand for psychological services, while decreasing their availability. We assessed the impact of the pandemic on depression and anxiety in pwCF, including how COVID-19 affected the frequency of mental health screening and the types of services provided., Methods: A 38-item internet survey, completed in June 2020, assessed how COVID-19 affected: 1) the mental health clinician's role and screening processes; 2) barriers to screening and resource needs; 3) impact of COVID-19 on depression and anxiety, and 4) positive outcomes and confidence in sustaining mental health screening and treatment, including telehealth services, after the pandemic., Results: Responses were obtained from 131 of the 289 US CF programs. Overall, 60% of programs (n=79) continued mental health screening and treatment, although less frequently; 50% provided individual tele-mental health interventions, and 9% provided telehealth group therapy. Clinically elevated depression symptoms (PHQ-9≥10; moderate to severe), were found in 12% of 785 pwCF, with 3.1% endorsing suicidal ideation. Similarly, elevated anxiety (moderate to severe; GAD-7≥10) was found in 13% of pwCF (n=779)., Conclusions: The COVID-19 pandemic created an opportunity to implement innovative solutions to disruptions in mental health screening and treatment in CF programs. We found that pwCF had increased access to psychological interventions during the pandemic via telehealth, supporting the continued integration of tele-mental health screening and treatment into CF care., (Copyright © 2021. Published by Elsevier B.V.)

Background: . In CLEAR-108-a phase 3, randomised, open-label study-once-daily amikacin liposome inhalation suspension (ALIS) was noninferior to twice-daily tobramycin inhalation solution (TIS) in improving lung function in patients with cystic fibrosis (CF) and chronic Pseudomonas aeruginosa infection after 3 treatment cycles (28 days on/28 days off). The CLEAR-110 extension study (ClinicalTrials.gov: NCT01316276; EudraCT: 2011-000443-24) assessed long-term safety, tolerability, and efficacy of ALIS in eligible patients who completed CLEAR-108., Methods: . Patients received once-daily ALIS 590 mg for 12 treatment cycles (96 weeks). Patients were grouped by prior treatment: the "prior-ALIS" cohort received ALIS in CLEAR-108, and the "ALIS-naive" cohort received TIS in CLEAR-108., Results: . Overall, 206 patients (prior-ALIS, n=92; ALIS-naive, n=114) entered CLEAR-110 and received ≥1 dose of ALIS. Most patients (88.8%) experienced ≥1 treatment-emergent adverse event (TEAE) through day 672 (end of year 2). Most TEAEs (72.3%) were mild or moderate in severity. Severe TEAEs were reported in 31 patients (15.0%). Two life-threatening TEAEs (haemoptysis; intestinal obstruction) and 1 death (cardiac failure) were reported. Twenty-one patients (10.2%) discontinued treatment due to a TEAE (mostly infective pulmonary exacerbation of CF). Mean change from baseline in forced expiratory volume in 1 second percent predicted at day 672 was -3.1% (prior-ALIS, -4.0%; ALIS-naive, -2.3%). Mean change from baseline in sputum density of P. aeruginosa at day 672 was 0.02 (prior-ALIS, -0.16; ALIS-naive, 0.19) log CFU/g., Conclusions: . Long-term treatment with ALIS was well tolerated with a favourable adverse event profile and demonstrated continued antibacterial activity in CF patients with chronic P. aeruginosa infection., Competing Interests: Declaration of Competing Interest D. Bilton: National Institute for Health Research funding support through the Imperial College, Royal Brompton Hospital, Specialist Respiratory Bio-Medical Research Unit; has received research contract support from Insmed Incorporated. I. Fajac: Received research contract support from Insmed Incorporated, has received a research grant from Actelion, and has served on advisory boards for Gilead Sciences and Vertex. T. Pressler: Received research contract support from Insmed Incorporated. J. P. Clancy: Serves on the Scientific Advisory Board and has received clinical trial support for phase II studies of ALIS in CF for Insmed Incorporated. D. Sands: Received research contract support from Insmed Incorporated. P. Minic: Received research contract support from Insmed Incorporated. M. Cipolli: Received research contract support from Insmed Incorporated, a grant from Vertex Pharmaceuticals, and served on advisory boards for Chiesi and Vertex. I. Galeva: Received research contract support from Insmed Incorporated. A. Solé: Has served on the advisory board for Gilead Sciences and Vertex. A. L. Quittner: Consulting for AbbVie, Aradigm, Insmed Incorporated, and Vertex; investigator-initiated studies for the Cystic Fibrosis Foundation. Z. Jumadilova: Employee of Insmed Incorporated. M. Ciesielska: Employee of Insmed Incorporated. M. Konstan: Has served on the Arikayce Clinical Program Steering Committee; has received grants and personal fees from Insmed Incorporated, Laurent Pharmaceuticals, Anthera, the Cystic Fibrosis Foundation, and AzurRX; has received personal fees from Chiesi, Celtazsys, Ionis Pharmaceuticals, Merck, Paranta Biosciences, Santhera, Ph Pharma, and Kala Pharmaceuticals; and has received nonfinancial support from Insmed Incorportated., (Copyright © 2021. Published by Elsevier B.V.)

Background: Primary ciliary dyskinesia (PCD) might be a risk factor for the development of anxiety and depression. This study investigated the associations between anxiety, depression and health-related quality of life (HRQoL) in individuals with PCD and their caregivers., Methods: Children, adolescents and adults with PCD and their caregivers were invited to participate in a mental health screening programme. During regular yearly outpatient visits, measures of anxiety (GAD-7), depression (PHQ-9), HRQoL (QOL-PCD), lung function (forced expiratory volume in 1 s (FEV 1 )) and body mass index (BMI)) were collected and associations of anxiety, depression and HRQoL were estimated., Results: A total of 103 individuals participated in the mental health screening programme. Elevated levels of anxiety (scores ≥10 on GAD-7) were found in 6% of adults (n=33), 14% of children (n=7), 6% of adolescents (n=17) and 20% of caregivers (n=46, 52% of mothers). Elevated depression levels (scores ≥10 on PHQ-9) were found in 18% of adults, 14% of children, 6% of adolescents and 11% of caregivers. Anxiety and depression were associated with scales on the QOL-PCD. Mothers reported higher anxiety scores than fathers (30% versus 9%, p=0.03). A strong negative relationship was found between depression in caregivers and physical functioning (QOL-PCD) of the child. Anxiety and depression were not significantly associated with anxiety/depression in their child., Conclusion: This is the first study investigating anxiety and depression in individuals with PCD and their caregivers. Our results revealed elevated levels of anxiety and depression, which were associated with worse HRQoL. These results suggest the need for psychological support in PCD., Competing Interests: Conflict of interest: M. Verkleij has nothing to disclose. Conflict of interest: I. Appelman has nothing to disclose. Conflict of interest: J. Altenburg has nothing to disclose. Conflict of interest: J. Twisk has nothing to disclose. Conflict of interest: A.L. Quittner has nothing to disclose. Conflict of interest: E. Haarman has nothing to disclose., (Copyright ©The authors 2021.)

Objectives: Severe to profound hearing loss is associated with communication, social, and behavioral difficulties that have been linked to worse health-related quality of life (HRQoL) compared to individuals with normal hearing. HRQoL has been identified as an important health outcome that measures functional ability, particularly for chronic conditions and disabilities. The current study developed the QoL-cochlear implant (CI) for early childhood and adolescents using the recommended Food and Drug Administration and European Medicines Agency guidelines on patient-reported outcomes., Design: Three phases of instrument development were conducted for both the early childhood (0 to 5 years old; parent proxy) and adolescent/young adult (13 to 22 years old) versions of the QoL-CI. Phase 1 included the development of our conceptual framework, which informed the discussion guides for stakeholder focus groups (e.g., audiologists, physicians, and therapists) at CI clinics in Miami and Philadelphia (n = 39). Open-ended interviews with parents (N = 18 for early childhood; N = 6 for adolescent/young adult version) and adolescents/young adults using CIs (n = 17) were then completed at both sites during phase 2. All interviews were transcribed and coded to identify common themes, which were then used to draft items for the QoL-CI. Both versions of the QoL-CI were developed using Qualtrics to allow for quick, easy electronic administration of the instruments on a tablet device. Last, phase 3 included cognitive testing in a new sample (N = 19 early childhood, N = 19 adolescent) to ensure that the draft instruments were clear, comprehensive, and easy to use., Results: Participant responses obtained via the open-ended interviews yielded an early childhood and adolescent version of the QoL-CI that was reportedly easy to complete and comprehensive. The final QoL-CI Early Childhood instrument yielded 35 questions across eight functional domains (environmental sounds, communication, social functioning, behavior, CI device management and routines, school, CI benefits, and early intervention). Similarly, the final QoL-CI adolescent/young adult version consisted of 46 items across eight domains (noisy environments, communication, CI usage and management, advocacy, social functioning, emotional functioning, acceptance, and independence)., Conclusions: The QoL-CI is a condition-specific QoL instrument that can be used for children ages birth through 22 years. These instruments capture the "whole" child by not only focusing on communication and auditory skills but also academic, social and emotional functioning. Once validated, these CI-specific measures will enable providers to track long-term outcomes and evaluate the efficacy of new interventions to improve overall CI use and QoL for pediatric and young adult users., Competing Interests: Dr. Cejas is on the AG Bell Board of Directors and is a consultant for Advanced Bionics. Dr. Sanchez is a consultant for Advanced Bionics and is on the MED-EL bilingual task force. The other authors have no conflicts of interest to disclose., (Copyright © 2021 Wolters Kluwer Health, Inc. All rights reserved.)

Background: The International Depression Epidemiological Study (TIDES) found elevated rates of screen positivity for depression and anxiety among individuals with cystic fibrosis (CF). Depression is associated with worse adherence and health-related quality of life in CF. We investigated the relationship with mortality., Methods: Subjects were untransplanted participants in TIDES 12+ years of age receiving care at one of 45 collaborating US CF care centers who completed the Hospital Anxiety and Depression Scale and/or Center for Epidemiologic Studies Depression Scale during a stable visit between 2006 and 2010. Clinical characteristics and mortality data were obtained from the CF Foundation Patient Registry. The association of a positive screen with 5-year survival was evaluated using Cox Proportional Hazards modeling., Results: Of 1005 eligible patients, 25% screened positive for depression and 34% screened positive for anxiety. Patients who screened positive for depression were more likely to be older, have a residual function mutation, public insurance, and more pulmonary exacerbations in the screening year. There were 96 deaths. The unadjusted 5-year Hazard Ratio (HR) for death among those with depression was 2.0; 95% CI (1.3, 3.0)]. When adjusted for predetermined potential confounders the HR for the entire population was 1.4; 95% CI (0.9, 2.2). The adjusted HR was higher in adults [1.6; 95% CI (1.0, 2.4)] and those screening in the severe range [2.0; 95% CI (1.2, 3.4)]. Anxiety was not associated with mortality., Conclusions: A positive depression screen is associated with increased mortality among adults with CF. Research into the etiology of this relationship is needed., Competing Interests: Declaration of Competing Interest The authors report no conflicts of interest., (Copyright © 2020. Published by Elsevier B.V.)

Background: A large-scale epidemiological study of 6088 individuals with cystic fibrosis (CF) and 4102 caregivers in nine countries documented elevated symptoms of depression and anxiety, leading to international guidelines for annual screening and follow-up. To facilitate national implementation, 84 CF programs funded a mental health coordinators (MHC). Implementation was evaluated after 1 year using the consolidated framework for implementation research (CFIR) to identify facilitators and barriers., Methods: A 45-item internet survey was developed to assess relevant CFIR implementation steps. Surveys were completed in 2016. It assessed five domains tailored to study aims: (a) Intervention characteristics, (b) outer setting, (c) inner setting, (d) characteristics of individuals, and (e) process of implementation., Results: Response rate was 88%, with pediatric and adult programs equally represented. A majority of MHCs were social workers (54.1%) and psychologists (41.9%); 41% had joined the team in the past year. Facilitators across the five domains included universal uptake of screening tools, greater awareness and detection of psychological symptoms, reduced stigma, and positive feedback from patients and families. Barriers included limited staff time, space, and logistics., Discussion: This is the largest systematic effort to integrate mental health screening and treatment into the care of individuals with a serious, chronic illness and their caregivers. MHCs implementing screening, interpretation and follow-up reported positive results, and significant barriers. This national implementation effort demonstrated that depression and anxiety can be efficiently evaluated and treated in a complex, chronic disease. Future efforts include recommending the addition of screening scores to national CF Registries and examining their effects on health outcomes., (© 2020 Wiley Periodicals LLC.)

To maximize health, individuals with cystic fibrosis (CF) follow a complex, burdensome daily care regimen. Managing CF is associated with a range of significant biopsychosocial challenges and places individuals with CF, and their caregivers, at greater risk for developing anxiety and depression. To promote wellness and address the potential barriers that affect management of this complex chronic illness, many individuals would benefit from treatment from a behavioral health provider. Social workers within multidisciplinary CF care teams are well positioned to respond to this need, and an expanding number of social workers serving as behavioral health providers in the community will be sought as a resource to provide treatment to this population. This article serves as a primer for social workers to maximize knowledge of the psychosocial and potential behavioral health needs of individuals with CF across the life span. To best support individuals with CF, authors describe the disease-specific manifestations and outline the numerous potential clinical targets for social work to promote wellness. The article concludes by highlighting the importance of communication with the medical team and considerations for effective collaborative care., (© 2020 National Association of Social Workers.)

Advances in genomic medicine have evolved to include rapid whole genome sequencing (rWGS) in pediatric intensive care settings. Traditionally, genetic testing was conducted in outpatient clinics, with stepwise genetic testing occurring over several years. This delayed the time to diagnosis, making it more difficult to include underrepresented groups, such as those who identify as Black and Latinx. National genetic sequencing programs have also struggled to engage these participants in their studies, leading to a significant disparity in access to new genetic technologies. The purpose of our study was to compare the demographic characteristics of families enrolled in both an Odyssey Program (N = 46), defined as outpatients in the Genetics Clinic who have had prior genetic testing, and a newly implemented rWGS (N = 52) sequencing program. Despite living in a large, ethnically diverse city, our results indicated that parents in the Odyssey program differed significantly from parents in the rWGS program in level of education, family income, and insurance status. For example, 71.5% of parents in the diagnostic Odyssey program had a college or advanced degree, whereas 42% of parents in the rWGS program had this level of education. Family income and insurance also differed, with 48.6% of families in the Odyssey program earning $100,000 or more versus 28.2% in rWGS; 56% of parents in the Odyssey program had private insurance with 26% on Medicaid whereas only 23% of parents in rWGS had private insurance, with the vast majority of children on Medicaid (69%). Thus, our Odyssey program illustrates some of the common pitfalls in implementing genomic testing in an ethnically diverse community, including lack of referrals, travel to outpatient visits, and a cultural mismatch with providers. The successful enrollment of underrepresented groups in the rWGS program demonstrates that given the opportunity to participate in genetic testing, families are interested and aware of the potential benefits of this testing for their child. As genomic sequencing transitions from outpatient to inpatient settings, an opportunity arises to close the health disparity gap. Recommendations for implementing rWGS in pediatric, intensive care settings that address the common barriers faced by underrepresented families are discussed., (© 2020 National Society of Genetic Counselors.)