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Rationale: Although inadequate health literacy has been associated with lower asthma medication knowledge and worse metered-dose inhaler (MDI) technique, the relationship between health literacy and the capacity to learn asthma self-management skills is unknown.Objectives: In this prospective cohort study of adults hospitalized for severe asthma exacerbations at two inner-city hospitals, we examined the relationship between inadequate health literacy and difficulties learning and retaining instructions about discharge medications and appropriate MDI technique.Methods: At hospital discharge, participants received one-on-one, 30-min, guideline-based, written and oral instruction about their asthma discharge regimen as well as appropriate MDI technique.Measurements and Main Results: Seventy-three patients were enrolled. Inadequate health literacy was identified in 16 (22%) participants. Before instruction, inadequate health literacy was associated with lower asthma medication knowledge (5.2/10 vs. 7.2/10, p < 0.001) and worse MDI technique (3.2/6 vs. 3.9/6, p = 0.03). However, inadequate health literacy was not associated with difficulty learning (p = 0.33) or retaining (p = 0.35) instructions about the discharge regimen. Similarly, inadequate health literacy was not associated with difficulty learning (p = 0.26) or retaining (p = 0.97) appropriate MDI technique. Results were similar in multivariable models adjusted for demographic characteristics and asthma severity indicators.Conclusions: These findings suggest that inadequate health literacy is a surmountable barrier to learning and remembering key asthma self-management skills. [ABSTRACT FROM AUTHOR]

Despite the efficacy of corticosteroid therapy, patients hospitalized for asthma exacerbations are at high risk for re-exacerbation and death after discharge. The objective of this prospective cohort study was to evaluate adherence to inhaled corticosteroids (ICS) and oral corticosteroids (OCS) after discharge in adults hospitalized for asthma exacerbations. ICS and OCS were equipped with electronic medication monitors and were provided at discharge. Adherence (use/prescribed use x 100%) was measured by self-report and canister weight (ICS), pill count (OCS), and electronic medication monitors (both ICS and OCS) 2 weeks after discharge. Poor adherence was defined as adherence of less than 50%. The Asthma Control Questionnaire was used to assess symptom control. Sixty patients were enrolled (age 42.2 years, 98.3% African American, 65.0% female, 46.7% with history of near-fatal asthma). Electronically measured adherence to both corticosteroids dropped to approximately 50% within 7 days of discharge. Poor adherence to both corticosteroids predicted significantly worse symptom control (p = 0.04). Self-report, canister weight, and pill count all had low sensitivity (29.2%, 65.0%, and 7.7%, respectively) for detecting poor adherence. We conclude that adherence to ICS and OCS deteriorates within days of hospital discharge but may not be recognized in a substantial proportion of patients. [ABSTRACT FROM AUTHOR]

Despite the growing numbers of HIV-infected school-aged children, we know very little about the mental health status of this group. This longitudinal study examined the frequency of psychological distress symptoms in HIV-infected children between the ages of 6 and 11 years at three time points over a period of two and one-half years. Children were assessed using the Dominic, a pictorial instrument that assesses for 7 psychological distress symptoms of childhood. In addition, family and demographic variables were collected at Time 1. Children were found to be relatively well-adjusted, with low to moderate incidence of psychological distress. While there were no significant changes in frequency of psychological distress symptoms from Time 1 to Time 3, the prevalence of overanxious and depressive symptomatology increased over time. Implications for clinical practice and future research will be discussed. [ABSTRACT FROM AUTHOR]

OBJECTIVE: To assess the extent to which caregivers and their child's physician agree about the prescribed asthma medication regimen and evaluate factors associated with medication concordance. METHODS: A cross-sectional, descriptive survey was administered to 318 caregivers of inner-city children with asthma, aged 5 to 12 years, and their caregiver-identified primary care physician at elementary schools and participants' homes. Concordance between caregiver- and physician-reported controller medication prescription was measured. RESULTS: Only 42% of physicians and 32% of caregivers reported a controller medication prescription (78% agreement, kappa = 0.54; 95% confidence interval: 0.45-0.63) despite that 73% of the children were rated by their caregiver as currently experiencing persistent asthma symptoms. When the physician reported a controller prescription, 38% of the caregivers denied use of a controller. Having a course of oral steroids in the past year (chi(2) = 9.85) and positive caregiver beliefs toward asthma care (chi(2) = 18.40) were associated with caregiver-physician concordance. Multivariate analysis found that when caregivers had high Asthma Beliefs Scale summary scores versus low scores, they were almost 10 times as likely to be concordant with the physician (odds ratio: 9.76; 95% confidence interval: 2.85-33.46). CONCLUSIONS: Our data support previous reports of physician underprescribing of controller medication among inner-city children. However, even when prescribed by a physician, more than one third of caregivers did not report a controller prescription, and this discordance was related to caregivers' beliefs about treatment. Efforts to improve physician adherence to asthma guidelines will not result in proper treatment unless caregiver-physician communication about asthma therapy is improved. [ABSTRACT FROM AUTHOR]

Focuses on the use, feasibility and impact of a psychological behavioral screening service in a primary care setting. Identification of children possibly experiencing behavioral problems; Prescription of psychotropic medications; Mental health services.

Investigates the implications of family nonparticipation in studies of treatment adherence among adolescents with insulin-dependent diabetes. Lower treatment adherence scores for families that agreed to participate but failed to return the questionnaires; Frequency of testing blood sugar.

Caregivers of children with asthma can become overwhelmed by the burden of care provision. Guided by the socioecological framework, we examined individual and system-level factors associated with caregiver health-related quality of life (HRQoL) among preschool children (aged two to six years) enrolled in a multilevel home- and school-based asthma educational intervention in Baltimore, Maryland. Primary outcome was caregiver HRQoL measured at baseline and six months. Independent variables were baseline individual-level factors (caregiver's health literacy, self-efficacy to manage asthma, and depressive symptoms) and system-level factors (barriers to asthma care measured in five subdomains-pragmatics, health knowledge and beliefs, expectations, skills, and marginalization-and neighborhood social cohesion). Multiple regression models were used to assess association between baseline individual and system-level factors and caregiver HRQoL at baseline and six-month follow-up adjusting for child's asthma control, caregiver's age, household income, and study arm assignment/intervention. 398 caregivers completed the baseline survey and 328 (82%) completed the six-month follow-up survey. In the adjusted multiple regression model, lower caregiver's health literacy and higher barriers to asthma care related to pragmatics were significantly associated with lower caregiver HRQoL at baseline (b= 0.38, 95% CI, 0.15 - 0.61, p  = 0.002; and b = 0.01, 95% CI, 0.004 - 0.02, p  < 0.001, respectively). The results were similar at six-month follow-up. Caregiver health literacy and barriers to asthma care related to pragmatics and skills were found to be important determinants of asthma caregiver HRQoL. Addressing these factors may improve quality of life among caregivers of preschool children with asthma.

Background: Cystic Fibrosis Foundation guidelines recommend human milk (HM) as the ideal source of nutrition for children with CF (cwCF). Despite known pulmonary and nutritional benefits, fewer cwCF ever receive HM compared to the general population. Early nutrition choices are preference-sensitive, yet little is known about the factors that impede or sustain HM feeding among parents of cwCF., Objectives: Explore perceptions and experiences of mothers of cwCF who initiated HM feeding., Methods: Mothers of cwCF aged ≤ 10 years completed audio-taped, semi-structured interviews describing their experiences with HM feeding. Interviews were transcribed and two researchers independently coded the transcripts and conducted content and thematic analysis using an inductive approach., Results: Participants included 28 mothers who initiated HM feeding. Major themes included: (1) the impact of a CF diagnosis on HM feeding plans; (2) CF-specific challenges to HM feeding; (3) mixed perceptions of the CF care team's support for HM feeding and of the role of formula in CF nutritional care; and (4) the benefit of lactation consultants as part of the CF care team., Conclusion: Many parents prioritize HM for their cwCF given the well-established health benefits. However, CF-specific barriers to HM feeding are common and nutritional challenges necessitating fortification add additional barriers to sustained HM feeding efforts. While HM may improve long-term pulmonary outcomes, our findings demonstrate the need for personalized support for mothers desiring to HM feed to facilitate shared decision-making around options to optimize early nutritional status among cwCF., (© 2025 Wiley Periodicals LLC.)

Background: Elexacaftor/tezacaftor/ivacaftor (ETI) has profoundly affected the health and lives of many people with CF (pwCF). The rapid change in health for pwCF taking ETI provided them an opportunity to reflect on what "being healthy" means. The goal of this secondary analysis was to document changes in the health and well-being of pwCF after starting ETI, beyond the expected physical benefits., Methods: The Qualitative Understanding of Experiences with the SIMPLIFY Trial (QUEST) study evaluated pwCF's experiences on ETI, including participation in a withdrawal study, treatment burden, and experiences of health. Two sixty-minute interviews were conducted approximately four months apart, audio-recorded and transcribed. A phenomenological approach was used to identify text of interest and create a formal codebook., Results: Ninety-one pwCF (mean age=27.8 years; range 14-67; 51 % male) and 23 caregivers of the teenagers completed at least one interview. Beyond the expected benefits of ETI, four themes were identified: (1) Night and Day Change in Health, (2) Reduced Cognitive Burden, (3) Shifting to Managing Overall Wellness and Co-Morbidities, and (4) Social/Self Identity Changes., Conclusion: ETI has raised pwCF's expectations for their health and well-being. Current symptom and QOL measures may no longer capture the less perceptible ways pwCF on ETI experience changes in symptoms or health. CF care may need to adapt to focus more on general health, managing other comorbidities, and planning for the future., Competing Interests: Declaration of competing interest Sawicki, Riekert, Everhart, Butcher, Gifford, Nichols, Prickett, Goodman, Bacon, Yablon, Sconiers, McWilliams, Gifford, and Maggs received support from the CFF. Sawicki received fees from Vertex for participating in an advisory board meeting that focused on his clinical expertise; Riekert received speaker fees from Vertex., (Copyright © 2024 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.)

Background: As people with cystic fibrosis (PWCF) live longer due to the breakthrough drug elexacaftor-tezacaftor-ivacaftor (ETI), they have questioned whether other CF therapies could be safely discontinued. SIMPLIFY was the first prospective, randomized trial to evaluate non-inferiority of discontinuing versus continuing two therapies. The QUEST (Qualitative Understanding of Experiences in the SIMPLIFY Trial) study was conducted to understand experiences of PWCF enrolled in SIMPLIFY, including why they joined, perceptions of randomization, decision-making around study withdrawal, and considerations for future discontinuation studies., Methods: QUEST enrolled SIMPLIFY participants 14 years or older stable on ETI and caregivers of the 14-17 year-olds. Interviews were audio-recorded, transcribed, and coded. A phenomenological approach was used to inductively develop codes with no a priori hypotheses; identified themes were then organized around current research and recruitment literature., Results: 114 interviews were completed (68 adults, 23 teenagers, and 23 caregivers). Among PWCF, median age was 27.8 years, 49 % were female and 80 % had participated in research before SIMPLIFY. Five themes were identified: (1) Experience with SIMPLIFY randomization, [2] Trust, [3] Altruism, [4] Perceived personal benefits, and [5]) Perceived risks and protocol burden., Conclusion: QUEST findings highlight how a long-standing culture of research and thoughtful protocol design contributed to SIMPLIFY's successful recruitment and retention. This included understanding the importance of remaining in the trial despite not being randomized to their preferred treatment assignment. Using patient-centered approaches to select research questions, design a protocol to minimize participant barriers, and frame recruitment materials messaging contribute to successful research participation., Competing Interests: Declaration of competing interest The Authors declare that there is no conflict of interest with respect to the research, authorship, and/or publication of this article., (Copyright © 2024. Published by Elsevier Inc.)

Background: This report summarizes the 2023 inaugural annual meeting of the Cystic Fibrosis Foundation's Prioritizing Research in Mental Health (PRIME) working group. This workshop focused on mental health and elexacaftor/tezacaftor/ivacaftor (ETI)., Methods: We reviewed existing literature and identified key gaps and study design considerations in preclinical work, pharmacokinetics/pharmacodynamics, mood/anxiety, quality of life/self-perception, neuropsychological symptoms, sleep, and symptom management., Results: Limited studies have identified behavioral changes with modulator exposure in rodent models of depression, anxiety, and cognition. Longitudinal human studies reporting mean changes generally show no change or improvement. However, case reports and single-center studies identify subgroups reporting new or worsening symptoms., Conclusions: Future studies should focus on understanding the role of CFTR in the nervous system, defining ETI impacts in preclinical models, and mechanistic investigations. Innovative methods with larger samples and comprehensive assessments are needed to determine the incidence of new/worsening symptoms throughout the lifespan and effective management strategies., Competing Interests: Declaration of competing interest Bathgate (CJB) reports grants, travel support, and honorarium fees from the Cystic Fibrosis Foundation; and personal fees from Vertex Pharmaceuticals. Everhart (RSE) reports grants, travel support, and honorarium fees from the Cystic Fibrosis Foundation; grant funding from the National Heart, Lung, and Blood Institute. Fedele (DAF) reports grants, travel support, and honorarium fees from the Cystic Fibrosis Foundation; grant funding from the National Heart, Lung and Blood Institute. Georgiopoulos (AMG) reports personal fees from the Belgian Cystic Fibrosis Foundation/King Baudouin Foundation; grants, personal fees and travel reimbursement from the Cystic Fibrosis Foundation, grants from the Dutch Cystic Fibrosis Foundation; travel reimbursement from the European Cystic Fibrosis Society; travel reimbursement from the French Cystic Fibrosis Society; personal fees from the Italian Cystic Fibrosis Research Foundation; grant funding from the National Heart, Lung and Blood Institute, grants and personal fees from Vertex Pharmaceuticals; and personal fees from Virginia Commonwealth University. He (JH) reports grants and honorarium fees from the Cystic Fibrosis Foundation; grant funding from the National Institute of Health (NIH). Kirby (KK) reports honoraria from the Cystic Fibrosis Foundation. Liu (FFL) reports grant funding, travel support, and honorarium fees from the Cystic Fibrosis Foundation; grant funding from the National Institute of Mental Health (NIMH). Powers (SWP) reports grants to his institution from the National Institutes of Health (NINDS, NCCIH, NIDDK, NINR, NCATS), the Patient Centered Outcomes Research Institute, and the Cystic Fibrosis Foundation; travel reimbursement and honoraria from the Cystic Fibrosis Foundation. Reznikov (LRR) reports grants, travel support, and honorarium fees from the Cystic Fibrosis Foundation; grant funding from the National Heart, Lung, and Blood Institute; honoraria and travel support for committee leadership role from NIH, committee leadership role in American Physiological Society. Raffensperger (KR) reports honoraria from the Cystic Fibrosis Foundation. Riekert (KAR) reports grants, travel support, and honorarium fees from the Cystic Fibrosis Foundation and grant funding from the National Institutes of Health (NHLBI, NIDDK). Tillman (EMT) reports grant support and travel reimbursement from the Cystic Fibrosis Foundation. Traver (KLT) reports travel reimbursement and honoraria from the Cystic Fibrosis Foundation, Cystic Fibrosis Learning Network (CFLN) and Cincinnati Children's Medical Center (CCHMC)., (Copyright © 2024. Published by Elsevier B.V.)

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.)

Rationale: Evaluating approaches to reduce treatment burden is a research priority among people with cystic fibrosis on highly effective modulators, including elexacaftor-tezacaftor-ivacaftor (ETI). Objectives: We sought to evaluate the impact of discontinuing both hypertonic saline (HS) and dornase alfa (DA) versus continuing both therapies among a subgroup of participants in the SIMPLIFY study who sequentially participated in trials evaluating the independent clinical effects of discontinuing HS and DA. Methods: SIMPLIFY participants ≥12 years old on ETI and constituting a subgroup using both HS and DA at study entry were randomized to the HS or DA trial and then randomized 1:1 to continue or discontinue the applicable therapy for 6 weeks. After completion of the first trial, eligible participants could enroll in the second trial beginning with a 2-week run-in. Study outcomes were compared across the duration of SIMPLIFY participation between a cohort remaining on both therapies during SIMPLIFY and a cohort that sequentially discontinued both as a result of trial randomizations. Multivariable regression models were used to estimate treatment differences, adjusted for time between trials, trial order, baseline age, sex at birth, and percent predicted forced expiratory volume in 1 second (ppFEV 1 ) at study entry. Results: Forty-three participants discontinued both therapies by the end of SIMPLIFY, and 63 remained on both, with overall average ppFEV 1 of 96.7% at study entry and 3.9 months as the average duration of follow-up from beginning of the first trial to completion of the second trial, including time between trials. No clinically meaningful difference in the change in ppFEV 1 from baseline to completion of the second trial was observed between those who discontinued and those who remained on both therapies (difference: 0.22% off-on; 95% confidence interval = -1.60, 2.03). Changes in lung clearance index at 2.5% starting concentration, patient-reported outcomes, and safety outcomes were also comparable. Patient-reported treatment burden, as measured by a Cystic Fibrosis Questionnaire-Revised subscale, significantly decreased in those who discontinued both therapies. Conclusions: SIMPLIFY participants who sequentially discontinued both HS and DA experienced no meaningful changes in clinical outcomes and reported decreased treatment burden as compared with those who remained on both therapies. These data continue to inform a new era of postmodulator care of people with cystic fibrosis.

Background: A personalized approach to assessing medication knowledge may identify opportunities for education to support self-management of cystic fibrosis (CF). This project describes the development, scoring, and preliminary validity of the Personalized CF Medication Questionnaire (PCF-MQ), designed to assess knowledge of prescribed CF medication purpose, administration, and dose and frequency., Methods: Participants completed the PCF-MQ, the Knowledge of Disease Management (KDM-CF), and the Cystic Fibrosis-Medication Beliefs Questionnaire (CF-MBQ). Prescribed regimens were abstracted from medical records. Eligibility criteria were age 12 years and older, diagnosed with CF, and prescribed a CF medication. Statistical analyses were conducted using R software. Spearman rho was used to test correlations between measures., Results: Sixty people with CF (pwCF) were enrolled; three people reported a regimen that substantially deviated from the medical record and were excluded from the analyses. The mean (SD) age was 20.2 (7.3) years, 54 % were female, and 74 % had a FEV1pp ≥70 %. The mean (SD) PCF-MQ total score was 77.8 (12.3) and knowledge scores ranged from a low of 58.3 for levalbuterol to 100 for ivacaftor. The PCF-MQ total score correlated with the KDM total score and subscales (Spearman Rho= 0.32-0.59, p < 0.05) and was not correlated with the CF-MBQ subscales (p > 0.05))., Conclusions: The PCF-MQ was correlated with another measure of general CF knowledge, but not health beliefs; because of the small sample size, this should be considered preliminary evidence of its validity. Advantages over existing CF knowledge measures include its practicality for use to help assess pwCF's knowledge about their prescribed regimen., Competing Interests: Declaration of competing interest Presented as a poster presentation at the 2020 North American Cystic Fibrosis Conference with abstract published in Pediatric Pulmonology (Phan H, Daines CL, Woo T, Goodman, A, Sawicki GS, Riekert KA. Design And Validation of The CF Medication Questionnaire. Pediatr Pulmonol. 2020; 55(S2): 264.), (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.)

Central nervous system (CNS) injury is common in sickle cell disease (SCD) and occurs early in life. Hydroxyurea is safe and efficacious for treatment of SCD, but high-quality evidence from randomized trials to estimate its neuroprotective effect is scant. HU Prevent was a randomized (1:1), double-blind, phase II feasibility/pilot trial of dose-escalated hydroxyurea vs. placebo for the primary prevention of CNS injury in children with HbSS or HbS-β 0 -thalassemia subtypes of SCD age 12-48 months with normal neurological examination, MRI of the brain, and cerebral blood flow velocity. We hypothesized that hydroxyurea would reduce by 50% the incidence of CNS injury. Two outcomes were compared: primary-a composite of silent cerebral infarction, elevated cerebral blood flow velocity, transient ischemic attack, or stroke; secondary-a weighted score estimating the risk of suffering the consequences of stroke (the Stroke Consequences Risk Score-SCRS), based on the same outcome events. Six participants were randomized to each group. One participant in the hydroxyurea group had a primary outcome vs. four in the placebo group (incidence rate ratio [90% CI] 0.216 [0.009, 1.66], p = .2914) (~80% reduction in the hydroxyurea group). The mean SCRS score was 0.078 (SD 0.174) in the hydroxyurea group, 0.312 (SD 0.174) in the placebo group, p = .072, below the p-value of .10 often used to justify subsequent phase III investigations. Serious adverse events related to study procedures occurred in 3/41 MRIs performed, all related to sedation. These results suggest that hydroxyurea may have profound neuroprotective effect in children with SCD and support a definitive phase III study to encourage the early use of hydroxyurea in all infants with SCD., (© 2024 Wiley Periodicals LLC.)

Background: There are numerous mobile health (mHealth) interventions for treatment adherence and self-management; yet, little is known about user engagement or interaction with these technologies., Objective: This systematic review aimed to answer the following questions: (1) How is user engagement defined and measured in studies of mHealth interventions to promote adherence to prescribed medical or health regimens or self-management among people living with a health condition? (2) To what degree are patients engaging with these mHealth interventions? (3) What is the association between user engagement with mHealth interventions and adherence or self-management outcomes? (4) How often is user engagement a research end point?, Methods: Scientific database (Ovid MEDLINE, Embase, Web of Science, PsycINFO, and CINAHL) search results (2016-2021) were screened for inclusion and exclusion criteria. Data were extracted in a standardized electronic form. No risk-of-bias assessment was conducted because this review aimed to characterize user engagement measurement rather than certainty in primary study results. The results were synthesized descriptively and thematically., Results: A total of 292 studies were included for data extraction. The median number of participants per study was 77 (IQR 34-164). Most of the mHealth interventions were evaluated in nonrandomized studies (157/292, 53.8%), involved people with diabetes (51/292, 17.5%), targeted medication adherence (98/292, 33.6%), and comprised apps (220/292, 75.3%). The principal findings were as follows: (1) >60 unique terms were used to define user engagement; "use" (102/292, 34.9%) and "engagement" (94/292, 32.2%) were the most common; (2) a total of 11 distinct user engagement measurement approaches were identified; the use of objective user log-in data from an app or web portal (160/292, 54.8%) was the most common; (3) although engagement was inconsistently evaluated, most of the studies (99/195, 50.8%) reported >1 level of engagement due to the use of multiple measurement methods or analyses, decreased engagement across time (76/99, 77%), and results and conclusions suggesting that higher engagement was associated with positive adherence or self-management (60/103, 58.3%); and (4) user engagement was a research end point in only 19.2% (56/292) of the studies., Conclusions: The results revealed major limitations in the literature reviewed, including significant variability in how user engagement is defined, a tendency to rely on user log-in data over other measurements, and critical gaps in how user engagement is evaluated (infrequently evaluated over time or in relation to adherence or self-management outcomes and rarely considered a research end point). Recommendations are outlined in response to our findings with the goal of improving research rigor in this area., Trial Registration: PROSPERO International Prospective Register of Systematic Reviews CRD42022289693; https://www.crd.york.ac.uk/prospero/display&#95;record.php?ID=CRD42022289693., (©Cyd Eaton, Natalie Vallejo, Xiomara McDonald, Jasmine Wu, Rosa Rodríguez, Nishanth Muthusamy, Nestoras Mathioudakis, Kristin A Riekert. Originally published in the Journal of Medical Internet Research (https://www.jmir.org), 24.09.2024.)

Background: Nutritional challenges are common in early CF care and stressful for caregivers of children with CF (cwCF) to navigate. Gastrostomy tube (G-tube) placement can improve weight gain, however the decision to proceed with placement is personalized and preference-sensitive. Little is known about the experiences of caregivers of cwCF and the G-tube decision-making process., Objectives: The present study used a qualitative approach to explore the perceptions and experiences of caregivers of cwCF with G-tube introductions and recommendations, as well as factors influencing G-tube decision-making., Methods: Caregivers of cwCF aged ≤ 10 years completed audio-taped, semi-structured interviews describing their experiences with G-tube placement discussions. Interviews were transcribed and two independent researchers coded the transcripts and conducted content and thematic analysis using an inductive approach., Results: Participants included 43 caregivers, 84 % were mothers (36/43). CwCF had a mean age of 4 years (SD=2.6), 84 % were White (36/43), and 60 % reported weights below <50th percentile (26/43). All caregivers knew about G-tubes, 44 % (19/43) were recommended a G-tube and 35 % (15/43) had a G-tube placed. Major findings included descriptions of the stages of G-tube decision-making from a heads up, to the game plan, to making a first difficult decision and finally living with the decision to pursue G-tube placement., Conclusion: G-tube decision-making is an emotional and personalized journey for caregivers of cwCF. Efforts to explore the values and priorities of caregivers is imperative to supporting families making difficult decisions in CF care., Competing Interests: Declaration of competing interest All authors disclose that they have no financial interests in the subject of this manuscript. The funding sources have no role in the analysis or drafting of the manuscript., (Copyright © 2024 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.)

The COVID-19 pandemic necessitated a rapid shift in clinical research to perform virtual visits and remote endpoint assessments, providing a key opportunity to optimize the use of remote endpoints for clinical trials in cystic fibrosis. The use of remote endpoints could allow more diverse participation in clinical trials while minimizing participant burden but must be robustly evaluated to ensure adequate performance and feasibility. In response, the Cystic Fibrosis Foundation convened the Remote Endpoint Task Force (Supplemental Table 1), a multidisciplinary group of CF researchers with remote endpoint expertise and community members tasked to better understand the current and future use of remote endpoints for clinical research. Here, we describe the current use of remote endpoints in CF clinical research, address key unanswered questions regarding their use and feasibility, and discuss the next steps to determine clinical trial readiness., Competing Interests: Declaration of competing interest Related to this work, MEJ reports employment by the Cystic Fibrosis Foundation (CFF). Unrelated to this work the authors report the following: DB reports consulting fees and stocks from Anagram Therapeutics Scientific Advisory Group and is a member of the Board of Directors for Anagram Therapeutics Scientific Advisory Group. CE reports honoraria for teaching presentations from Vertex and Chiesi. CHG reports grant funding from NIH NIDDK and NCRR, CFF, FDA Orphan Prod. Div. He received consulting fees from Enterprise Therapeutics and honoraria from Gilead and Vertex. He has received travel support from Vertex Pharmaceuticals and Enterprise Therapeutics. He participates on the DSMB for Novartis. He is a deputy editor for Annals of the ATS. He has stock in Air Therapeutics. GH reports grant funding from NIH NHLBI and CFF and support from CFCanada for travel. JEH reports grant funding from CFF and NIH, travel support from CF Canada, consulting fees from Vertex Pharmaceuticals and participation on an advisory board for Vertex Pharmaceuticals. DP reports grant funding from the CFF and consulting fees from Renexion Pharma. MSP reports grant funding from Dexcom Inc, CFF and Vertex Pharmaceuticals. She also reports consulting fees from Anagram Therapeutics and honoraria from Vertex Pharmaceuticals and TD Cowan. She is a member of the CFF DSMB and an associate editor for Endocrine Practice: Journal of the American Association of Clinical Endocrinologists. KAR reports grant funding from the CFF. MS reports grant funding from the CFF and Anagram Therapeutics. She has received consulting fees from Nestle International and Alcresta Therapeutics. She has received travel support from the CFF and serves on the CFF DSMB and reports a leadership role in the CFF. JS reports being a member of the CFF DSMB and advisory boards. ST reports honoraria from Vertex Pharmaceuticals. ETZ reports grant funding from CFF, NIH NHLBI, Vertex Pharmaceuticals. She has received consulting fees from CFF and Vertex Phamaceuticals and honoraria from the CFF and Asosciation for Diagnostic and Laboratory Medicine. She has received travel support from Vertex Pharmaceuticals, CFF and European Cystic Fibrosis Society. She has participated on advisory boards for CFF, CFF Therapeutics Development Network and Vertex Pharmaceuticals. MB, NL, KP and MR have no conflicts to report., (Copyright © 2024. Published by Elsevier B.V.)

Rationale & Objective: Vaccination for influenza is strongly recommended for people with chronic kidney disease (CKD) due to their immunocompromised state. Identifying risk factors for not receiving an influenza vaccine (non-vaccination) could inform strategies for improving vaccine uptake in this high-risk population., Study Design: Longitudinal observational study., Setting & Participants: 3,692 Chronic Renal Insufficiency Cohort Study (CRIC) participants., Exposure: Demographic factors, social determinants of health, clinical conditions, and health behaviors., Outcome: Influenza non-vaccination, which was assessed based on a receipt of influenza vaccine ascertained during annual clinic visits in a subset of participants who were under nephrology care., Analytical Approach: Mixed-effects Poisson models to estimate adjusted prevalence ratios (APRs)., Results: Between 2009 and 2020, the pooled mean vaccine uptake was 72% (mean age, 66 years; 44% female; 44% Black race). In multivariable models, factors significantly associated with influenza non-vaccination were younger age (APR, 2.16 [95% CI, 1.85-2.52] for<50 vs≥75 years), Black race (APR, 1.58 [95% CI, 1.43-1.75] vs White race), lower education (APR, 1.20 [95% CI, 1.04-1.39 for less than high school vs college graduate]), lower annual household income (APR, 1.26 [95% CI, 1.06-1.49] for <$20,000 vs >$100,000), formerly married status (APR, 1.22 [95% CI, 1.09-1.35] vs currently married), and nonemployed status (APR, 1.13 [95% CI, 1.02-1.24] vs employed). In contrast, participants with diabetes (APR, 0.80 [95% CI, 0.73-0.87] vs no diabetes), chronic obstructive pulmonary disease (COPD) (APR, 0.80 [95% CI, 0.70-0.92] vs no COPD), end-stage kidney disease (APR, 0.64 [0.56 to 0.76] vs estimated glomerular filtration rate≥60mL/min/1.73m 2 ), frailty (APR, 0.86 [95% CI, 0.74-0.99] vs no frailty), and ideal physical activity (APR, 0.90 [95% CI, 0.82-0.99] vs. physically inactive) were less likely to have non-vaccination status., Limitations: Possible residual confounding., Conclusions: Among adults with CKD receiving nephrology care, younger adults, Black individuals, and those with adverse social determinants of health were more likely to have the influenza non-vaccination status. Strategies are needed to address these disparities and reduce barriers to vaccination., Plain-Language Summary: Identifying risk factors for not receiving an influenza vaccine ("non-vaccination") in people living with kidney disease, who are at risk of influenza and its complications, could inform strategies for improving vaccine uptake. In this study, we examined whether demographic factors, social determinants of health, and clinical conditions were linked to the status of not receiving an influenza vaccine among people living with kidney disease and receiving nephrology care. We found that younger adults, Black individuals, and those with adverse social determinants of health were more likely to not receive the influenza vaccine. These findings suggest the need for strategies to address these disparities and reduce barriers to vaccination in people living with kidney disease., (Copyright © 2023 National Kidney Foundation, Inc. Published by Elsevier Inc. All rights reserved.)

Background: Poor adherence habits are key contributors to nonadherence but there are few clinically feasible methods for evaluating adherence habits, particularly for youths with chronic kidney disease (CKD). This study investigated how participants' qualitative responses to three interview questions about adherence habits mapped to primary principles of habit formation and objectively measured medication adherence in youths with CKD., Methods: Participants (ages 11-21 years) were recruited from a pediatric nephrology clinic as part of a larger study. Participants' daily objective antihypertensive medication adherence was measured with an electronic pill bottle over a 4-week baseline period. Qualitative interviews about adherence habits and routines were conducted with a subset of participants (N = 18)., Results: Clear qualitative differences emerged in how participants with high-medium adherence (80-100%) discussed adherence habits compared to participants with low adherence (0-79%). Participants with high-medium adherence discussed situational cues for taking medicine, including locations that cue adherence, step-by-step events leading up to taking medicine, and people who cue adherence. Participants with high-medium adherence regularly described taking medicine as "automatic," "second nature," and a "habit." Participants with low adherence rarely discussed these habit features nor did they explicitly acknowledge currently missing doses. Participants with low adherence tended to discuss challenges with organization and routines for taking medicine., Conclusions: Evaluating patient responses to questions about adherence habits may uncover challenges with adherence habit formation, provide direction for habit-strengthening intervention focused on developing automatic cues for taking medication, and support adherence successes for youths with CKD., Clinical Trial Registration Number: NCT03651596. A higher resolution version of the Graphical abstract is available as Supplementary information., (© 2023. The Author(s), under exclusive licence to International Pediatric Nephrology Association.)

Introduction: Medication adherence is suboptimal in childhood asthma. Children rely on caregivers to manage medication administration. It is important to detect families who are at risk for poor adherence or to identify potential areas that can assist families with better adherence to asthma medications in order to improve asthma outcomes. We investigated the association between asthma routines, family asthma management knowledge and skills, and caregiver depressive symptoms with daily controller medication adherence among Head Start preschool children in Baltimore City., Methods: Our study included 256 low-income urban preschool children who were prescribed a daily controller medication. Asthma routinization (by the Asthma Routines Questionnaire), family asthma management [by the Family Asthma Management System Scale (FAMSS)], and caregiver depressive symptoms (by the Center for Epidemiological Studies - Depression) were assessed at baseline. The medication possession ratio (MPR) to measure adherence to daily controller medications was calculated at baseline and 12 months from pharmacy fill records. Multiple regression models evaluated the relationship between asthma routinization, the FAMSS, the CES-D, and MPR., Results: Results indicated that only 7% of families had an MPR above 80% at baseline, and 24% of caregivers had clinically significant depressive symptoms. Higher asthma medication routines were associated with higher MPR at baseline ( b  = 0.05, p  = 0.03). Higher family asthma management was associated with higher MPR at both baseline ( b  = 0.04, p  < 0.01) and 12 months ( b  = 0.05, p  < 0.01)., Discussion: Our findings highlight the importance of family asthma management and maintaining medication routines over time to improve asthma controller medication adherence., Competing Interests: The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest., (© 2023 Lu, Ruvalcaba, McQuaid, Rand, Riekert and Eakin.)

Background: The daily treatment regimen for an individual with cystic fibrosis (CF) can take more than 2 h to complete, and chronic treatment adherence rates are low. Developing partnerships between CF clinical researchers and the CF community is essential in developing acceptable, feasible, and effective strategies to improve self-management and adherence., Methods: The Success with Therapies Research Consortium (STRC) was formed as a multi-center US collaborative to conduct rigorous research studies of adherence to CF treatments. A multidisciplinary team of researchers from 15 sites, collaborating with members of the CF community, is charged with developing, implementing, and disseminating real-world, patient-centered interventions for people living with CF., Results: Since 2014, the STRC has conducted 8 studies. The CF community, people with CF (pwCF), and caregivers have come to serve in multiple valuable capacities on the STRC, including as members of the Steering Committee and Co-Principal Investigators. Additionally, while people with CF are irreplaceable participants in STRC studies, their influence, and that of their families and healthcare professionals, extends beyond the traditional research participant role., Conclusions: Engaging broadly with the CF community is the optimal model for developing interventions to support those living with CF in sustaining daily care. Input and direct involvement from people with CF, their families, and their caregivers has enabled the STRC to advance its mission through innovative clinical research approaches., Competing Interests: Declaration of Competing Interest The authors declare that they have no conflict of interest., (Copyright © 2023. Published by Elsevier B.V.)

Background: Higher growth percentiles are associated with more favorable lung function in cystic fibrosis (CF), prompting the creation of CF Foundation (CFF) nutritional guidelines., Objectives: To describe early childhood growth trajectories within CF, to determine if growth trajectories are associated with differences in lung function at age six, and to identify factors that differ between trajectory groups., Methods: Retrospective cohort study of children diagnosed with CF and born 2000-2011 using the US CFF Patient Registry. Annualized growth parameters prior to age six were included in group-based trajectory modeling to identify unique early life growth trajectories. FEV1 percent predicted (FEV1pp) at age six was compared between trajectory groups using linear regression. Factors associated with group membership were identified using multinomial logistic regression., Results: 6,809 children met inclusion criteria. Six discrete growth trajectories were identified, including three groups that began with growth parameters >50th percentile, termed: "always high", "gradual decliner", "rapid decliner", and three which began with growth parameters <50th percentile, termed: "rapid riser", "gradual riser", "always low". FEV1pp at age six was highest for the Always High trajectory. The Always Low trajectory was nearly 10% lower than the Always High trajectory. Sex, ethnicity, newborn screening and pancreatic function were associated with trajectory class membership., Conclusions: Distinct early life growth trajectories were identified within CF. Trajectories that met CFF nutritional guideline recommendations were associated with higher FEV1pp at age six. CF care teams should continue to partner with families to encourage interventions to support optimal growth to improve lung function in CF., Competing Interests: Declaration of Competing Interest All authors disclose that they have no financial interests in the subject of this manuscript. The funding sources have no role in the analysis or drafting of the manuscript., (Copyright © 2023. Published by Elsevier B.V.)

Background: Reducing treatment burden is a priority for people with cystic fibrosis, whose health has benefited from using new modulators that substantially increase CFTR protein function. The SIMPLIFY study aimed to assess the effects of discontinuing nebulised hypertonic saline or dornase alfa in individuals using the CFTR modulator elexacaftor plus tezacaftor plus ivacaftor (ETI)., Methods: The SIMPLIFY study included two parallel, multicentre, open-label, randomised, controlled, non-inferiority trials at 80 participating clinics across the USA in the Cystic Fibrosis Therapeutics Development Network. We included individuals with cystic fibrosis aged 12-17 years with percent predicted FEV 1 (ppFEV 1 ) of 70% or more, or those aged 18 years or older with ppFEV 1 of 60% or more, if they had been taking ETI and either (or both) mucoactive therapies (≥3% hypertonic saline or dornase alfa) for at least 90 days before screening. Participants on both hypertonic saline and dornase alfa were randomly assigned to one of the two trials, and those on a single therapy were assigned to the applicable trial. All participants were then randomly assigned 1:1 to continue or discontinue therapy for 6 weeks using permuted blocks of varying size, stratified by baseline ppFEV 1 (week 0; ≥90% or <90%), single or concurrent use of hypertonic saline and dornase alfa, previous SIMPLIFY study participation (yes or no), and age (≥18 or <18 years). For participants randomly assigned to continue their therapy during a given trial, this therapy was instructed to be taken at least once daily according to each participant's pre-existing, clinically prescribed regimen. Hypertonic saline concentration was required to be at least 3%. The primary objective for each trial was to determine whether discontinuing was non-inferior to continuing, measured by the 6-week change in ppFEV 1 in the per-protocol population. We established a non-inferiority margin of -3% for the difference between groups in the 6-week change in ppFEV 1 . Safety outcomes were analysed in the intention-to-treat population. This study is registered with ClinicalTrials.gov, NCT04378153., Findings: From Aug 25, 2020, to May 25, 2022, a total of 672 unique participants were screened for eligibility for one or both trials, resulting in 847 total random assignments across both trials with 594 unique participants. 370 participants were randomly assigned in the hypertonic saline trial and 477 in the dornase alfa trial. Participants across both trials had an average ppFEV 1 of 96·9%. Discontinuing treatment was non-inferior to continuing treatment with respect to the absolute 6-week change in ppFEV 1 in both the hypertonic saline trial (-0·19% [95% CI -0·85 to 0·48] in the discontinuation group [n=133] vs 0·14% [-0·51 to 0·78] in the continuation group [n=140]; between-group difference -0·32% [-1·25 to 0·60]) and dornase alfa trial (0·18% [-0·38 to 0·74] in the discontinuation group [n=199] vs -0·16% [-0·73 to 0·41] in the continuation group [n=193]; between-group difference 0·35% [-0·45 to 1·14]), with consistent results in the intention-to-treat populations. In the hypertonic saline trial, 64 (35%) of 184 in the discontinuation group versus 44 (24%) of 186 participants in the continuation group and, in the dornase alfa trial, 89 (37%) of 240 in the discontinuation group versus 55 (23%) of 237 in the continuation group had at least one adverse event., Interpretation: In individuals with cystic fibrosis on ETI with relatively well preserved pulmonary function, discontinuing daily hypertonic saline or dornase alfa for 6 weeks did not result in clinically meaningful differences in pulmonary function when compared with continuing treatment., Competing Interests: Declaration of interests GR-B reports grants and contracts from Vertex Pharmaceuticals and the Cystic Fibrosis Foundation (CFF). SHD reports contracts from AstraZeneca, Calithera, CFF, US National Institutes of Health (NIH), Vertex Pharmaceuticals; consulting fees from Polarean, 501 Ventures, and Chiesi USA; fees for advisory boards for Enterprise Therapeutics and Gilead Sciences; and participation on a data safety monitoring board (DSMB) for Abbvie and Boehringer Ingleheim. JLT-C reports grants and contracts from CFF, Vertex Pharmaceutics, Eloxx, and 4DMT; consulting fees from Vertex Phamaceuticals, Insmed, and 4DMT; participation on a DSMB for Abbvie; and serving on an advisory board for CFF, American Thoracic Society (ATS), Journal of Cystic Fibrosis, and Emily's Entourage. KAR reports grants from CFF; royalties from Springer Publishing; honoraria from Vertex Pharmaceuticals; and serving on an advisory board for ATS. AB reports grants from the CFF for Therapeutics Development Network (TDN) studies. FR reports grants from Vertex Phamaceuticals and consulting fees from Vertex Phamaceuticals, Proteostasis, Translate Bio, Boehringer Ingelheim, and Calithera. JKY reports grants from CFF. SM reports grants from CFF to support TDN studies. DPN reports grants from CFF and NIH; consulting fees from BiomX, Clarametyx, Genentech, GlaxoSmithKline, Nabriva, Respirion, and Vertex Pharmaceuticals; and advisory board membership for CFF and Kither Biotechnology. DR reports grants and contracts from CFF and Vertex Pharmaceuticals. AHG reports grants and contracts from CFF, Insmed, AbbVie, and 4D Molecular Therapeutics. GSS reports advisory board participation for Vertex Pharmaceuticals and Gilead Sciences. CHG reports grants and contracts from CFF, NIH, and the US Food and Drug Administration; consulting fees from Enterprise Therapeutics; and honoraria from Gilead Sciences, Novartis, Boehringer Ingelheim, Vertex Phamaceuticals, and stock options in Aer Therapeutics. JPC reports employment at the CFF. NM-H reports grants from CFF, NIH, and US Food and Drug Administration; consulting fees from Enterprise Therapeutics; and DSMB membership for the NIH. BPO, KO-D, AHG, GO, CA, RR, and KM declare no competing interests., (Copyright © 2023 Elsevier Ltd. All rights reserved.)

Background: People with cystic fibrosis (CF) are living longer and healthier lives as a result of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies, and are pursuing pregnancy. As the number of pregnancies in CF continue to increase, clinician attitudes and practices regarding care of pregnant people with CF remain largely unknown., Objective: To evaluate the current attitudes and practices of CF clinicians regarding pregnancy planning and care in CF., Methods: We conducted a national survey investigating practice patterns related to pregnancy care in CF. We used descriptive statistics to summarize responses and paired t-tests to compare population means., Results: A total of 93 clinicians completed the survey. Eighty-six percent of respondents believed family planning and pregnancy discussions should start before the age of 21 years, of which 67% believed these discussions should occur prior to age 18 years. Our results demonstrate variability in CF clinician comfort and management of various aspects of pregnancy care in CF including 1) potential complications of pregnancy 2) continuation of chronic CF therapies 3) continuation of CFTR modulators during pregnancy and lactation, and 4) approach to treatment of pulmonary exacerbation during pregnancy., Conclusions: As more people with CF pursue pregnancy in the era of CFTR modulators, CF providers should be initiating discussions surrounding pregnancy early and often. Establishing best practices in the management of pregnancy in CF, expanding peri‑pregnancy expertise within the CF community, and future studies investigating the maternal-fetal effects of CF therapies are needed., Competing Interests: Declaration of Competing Interest All authors have no financial conflict of interest to disclose related to this study., (Copyright © 2022 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.)

Introduction: Using self-determination theory, we explored relationships between autonomous motivation (AM) and perceived competence (PC) with previously validated measures of motivation and adolescent-reported asthma medication adherence., Method: Data were from adolescents (n = 260) enrolled in the School-Based Asthma Care for Teens study and taking preventive medication at baseline. Eligible adolescents (aged 12-16 years) had physician-diagnosed persistent asthma or poor control., Results: Adolescents taking daily preventive medicine reported higher AM and PC for adherence, whereas adolescents likely to miss ≥1 dose in the next 2 weeks had lower AM and PC. Adolescents taking medicines as prescribed, with plans to continue, and those feeling able to follow provider care plans, had higher AM and PC. Findings remained significant in regressions with control variables., Discussion: Many factors interfere with adolescent medication-taking. Clinicians' efforts to build AM and PC with patients and caregivers may be key to promoting adherence in this group., (Copyright © 2022 National Association of Pediatric Nurse Practitioners. Published by Elsevier Inc. All rights reserved.)

Health insurance coverage is associated with outcomes in cystic fibrosis (CF). A fraction of individuals in the United States are covered through Tricare, a federally funded program for military members and their dependents. The role of Tricare on CF health outcomes is unknown. Using a retrospective CF Foundation Patient Registry cohort born 2000-2011, insurance status was defined as any Tricare (n = 328) with reference groups of always private (n = 3,455) and exclusively public (n = 2,669) during the first 6 years of life. Subjects with Tricare coverage attended more CF care centers and lived in more zip codes by age 6 than their counterparts. BMI did not differ between groups. Subjects with Tricare had a higher FEV 1 at age 6 compared to those with always public insurance. Overall, outcomes for those with Tricare insurance appeared more similar to those with always private insurance. Future research should consider treating Tricare coverage similar to private insurance., Competing Interests: Declaration of Competing Interest All authors disclose that they have no financial interests in the subject of this manuscript. The funding sources have no role in the analysis or drafting of the manuscript., (Copyright © 2022 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.)

Background: This study aimed to compare attended home blood pressure (BP) measurements (HBPM) with ambulatory BP monitor (ABPM) readings and examine if level of agreement between measurement modalities differs overall and by subgroup., Methods: This was a secondary analysis of data from a 2-year, multicenter observational study of children 11-19 years (mean 15, SD = 2.7) with chronic kidney disease. Participants had 3 standardized resting oscillometric home BPs taken by staff followed by 24-h ABPM within 2 weeks of home BP. BP indices (measured BP/95%ile BP) were calculated for mean triplicate attended HBPM and mean ABPM measurements. Paired HBPM and ABPM measurements taken during any of 5 study visits were compared using linear regression with robust standard errors. Generalized estimating equation-based logistic regression determined sensitivity, specificity, negative, and positive predictive values with ABPM as the gold standard. Analyses were conducted for the group overall and by subgroup., Results: A total of 103 participants contributed 251 paired measurements. Indexed systolic BP did not differ between HBPM and daytime APBM (mean difference - 0.002; 95% CI: - 0.006, 0.003); the difference in indexed diastolic BP was minimal (mean difference - 0.033; 95% CI: - 0.040, - 0.025). Overall agreement between HBPM and 24-h ABPM in identifying abnormal BP was high (81.8%). HBPM had higher sensitivity (87.5%) than specificity (77.4%) and greater negative (89.8%) than positive (73.3%) predictive value, and findings were consistent in subgroups., Conclusions: Attended HBPM may be reasonable for monitoring BP when ABPM is unavailable. The greater accessibility and feasibility of attended HBPM may potentially help improve BP control among at-risk youth. A higher resolution version of the Graphical abstract is available as Supplementary information., (© 2022. The Author(s), under exclusive licence to International Pediatric Nephrology Association.)

Urban adolescents with asthma often have inadequate preventive care. We tested the effectiveness of the School-Based Asthma Care for Teens (SB-ACT) program on asthma morbidity and preventive medication adherence. Methods: Subjects/Setting - 12-16yr olds with persistent asthma in Rochester, NY schools. Design - 3-group randomized trial (2014-2019). SB-ACT Intervention - Two core components: 1) Directly observed therapy (DOT) of preventive asthma medications, provided in school for at least 6-8 weeks for the teen to learn proper technique and experience the benefits of daily preventive therapy; 2) 4-6 weeks later, 3 sessions of motivational interviewing (MI) to discuss potential benefits from DOT and enhance motivation to take medication independently. We included 2 comparison groups: 1) DOT-only for 6-8wks, and 2) asthma education (AE) attention control. Masked follow-up assessments were conducted at 3, 5, and 7mos. Outcomes - Mean number of symptom-free days (SFDs)/2 weeks and medication adherence. Analyses - Modified intention-to-treat repeated measures analysis. Results: We enrolled 430 teens (56% Black, 32% Hispanic, 85% Medicaid). There were no group differences at baseline. We found no difference in SFDs at any follow-up timepoint. More teens in the SB-ACT and DOT-only groups reported having a preventive asthma medication at each follow-up ( p <.001), and almost daily adherence at 3 and 5-months ( p <.001, p =.003) compared to AE. By 7 months there were no significant differences between groups in adherence ( p =.49). Conclusion: SB-ACT improved preventive medication availability and short-term adherence but did not impact asthma symptoms. Further work is needed to create developmentally appropriate and effective interventions for this group.

Background: Urban minority preschool children are disproportionately affected by asthma with increased asthma morbidity and mortality. It is important to understand how families manage asthma in preschool children to improve asthma control., Objective: To evaluate family asthma management and asthma outcomes among a low-income urban minority population of Head Start preschool children., Methods: The family asthma management system scale (FAMSS) evaluates how families manage a child's asthma. A total of 388 caregivers completed the FAMSS at baseline. Asthma outcomes were evaluated at baseline and prospectively at 6 months, including asthma control (based on the Test for Respiratory and Asthma Control in Kids), courses of oral corticosteroids (OCSs) required, and caregiver health-related quality of life (Pediatric Asthma Caregiver's Quality of Life Questionnaire [PACQLQ]). Multiple regression models evaluated the relationship between the FAMSS total score, FAMSS subscales, and asthma outcomes., Results: Higher FAMSS total scores were associated with fewer courses of OCSs required (b = -0.23, P < .01) and higher PACQLQ scores (b = 0.07, P < .05). At baseline, higher integration subscale scores (b = -0.19, P < .05) were associated with fewer courses of OCSs required, and higher family response scores were associates with higher PACQLQ scores (b = 0.06, P < .05). Nevertheless, higher collaboration scores were associated with lower PACQLQ at baseline (b = -0.06, P < .05) and 6 months (b = -0.07, P < .05)., Conclusion: Among this population of low-income minority preschool children, understanding how a family manages their child's asthma may help identify gaps for education to possibly improve caregiver asthma-related quality of life and reduce courses of OCSs., Trial Registration: ClinicalTrials.gov Identifier: NCT01519453 (https://clinicaltrials.gov/ct2/show/NCT01519453); protocol available from meakin1@jhmi.edu., (Copyright © 2021 American College of Allergy, Asthma & Immunology. Published by Elsevier Inc. All rights reserved.)

Background: Lower socioeconomic status (SES) has consistently been associated with poorer outcomes in individuals with cystic fibrosis (CF). Previous studies have compared outcomes for children with and without private insurance coverage, however the potential role of changes in insurance status on early health outcomes in children with CF remains unknown., Objectives: To describe the variability in insurance status in early childhood and to evaluate whether insurance variability was associated with poorer outcomes at age 6., Methods: Retrospective observational study using the Cystic Fibrosis Foundation Patient Registry. Insurance status was defined as: always private (including Tricare), exclusively public, or intermittent private insurance (private insurance and exclusively public insurance in separate years) during the first 6 years of life. Outcomes at age 6 included body mass index (BMI) and FEV 1  percent predicted (maxFEV 1 pp)., Results: From a 2000-2011 birth cohort (n = 8,109), 42.3% always had private insurance, 30.0% had exclusively public insurance, and 27.6% had intermittent private insurance. BMI percentiles did not differ between groups; however, children with intermittent private insurance and exclusively public insurance had a 3.3% and 6.6% lower maxFEV 1 pp at age 6, respectively, compared to those with always private insurance., Conclusions: A substantial proportion of young children in a modern CF cohort have public or intermittent private insurance coverage. While public insurance has been associated with poorer health outcomes in CF, variability in health insurance coverage may also be associated with an intermediate risk of disparities in lung function as early as age 6., Competing Interests: Declaration of Competing Interest All authors disclose that they have no financial interests in the subject of this manuscript. The funding sources have no role in the analysis or drafting of the manuscript., (Copyright © 2021 Elsevier Ltd. All rights reserved.)

Background: Females with cystic fibrosis (CF) have been shown to have worse pulmonary exacerbation (PEx) related outcomes compared to males. However, it is unknown if sex differences in treatment patterns are contributing to these outcomes. Thus, we sought to explore sex differences in treatment patterns in the Standardized Treatment of Pulmonary Exacerbations (STOP) cohort., Methods: Data for 220 participants from the STOP cohort were analyzed. Multivariable regression models were used to assess if female sex was associated with duration of treatment with IV antibiotics and inpatient length of stay. Secondary outcomes included antibiotic selection, adjunctive therapies, mean FEV1pp and CFRSD-CRISS respiratory symptom scores at the four study assessments., Results: In our adjusted model, the average number of IV antibiotic treatment days was 13% higher in females compared to males (IRR 1.13, 95% CI=1.02,1.25; p=0.02). We found no sex differences in inpatient length of stay, number of IV antibiotics, antibiotic selection or initiation of adjunctive therapies. Overall, females had higher CFRSD-CRISS scores at the end of IV therapy indicating worse symptom severity (23.6 for females vs. 18.5 for males, p=0.03)., Conclusions: Despite females having a longer treatment duration, our findings demonstrate that males and females are receiving similar treatments which suggest that the outcome disparities in females with CF may not be due to failure to provide the same level of care. Further research dedicated to sex differences in CF is necessary to understand why clinical outcomes differ between males and females., Competing Interests: Declaration of Competing Interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper., (Copyright © 2021 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.)

Substance use and mental health (SU/MH) disorders are insufficiently recognized in HIV care. We examined whether conveying SU/MH screening results to patients and providers increased SU/MH discussions and action plans. Intervention participants completed a computerized patient-reported questionnaire before their HIV visit; screened positive on ≥ 1 measure: depression, anxiety, PTSD symptoms, at-risk alcohol use, or drug use; and reviewed screening results to decide which to prioritize with their provider. Screening results and clinical recommendations were conveyed to providers via medical record. A historic control included patients with positive screens but no conveyance to patient or provider. The patient-provider encounter was audio-recorded, transcribed, and coded. For the overall sample (n = 70; 38 control, 32 intervention), mean age (SD) was 51.8 (10.3), 61.4% were male, and 82.9% were Black. Overall, 93.8% raised SU/MH in the intervention compared to 50.0% in the control (p < 0.001). Action plans were made for 40.0% of intervention and 10.5% of control encounters (p = 0.049). Conveying screening results with clinical recommendations increased SU/MH action plans, warranting further research on this intervention to address SU/MH needs., (© 2021. The Author(s), under exclusive licence to Springer Science+Business Media, LLC part of Springer Nature.)

Background: Well-designed clinical research needs to obtain information that is applicable to the general population. However, most current studies fail to include substantial cohorts of racial/ethnic minority populations. Such underrepresentation may lead to delayed diagnosis or misdiagnosis of disease, wide application of approved interventions without appropriate knowledge of their usefulness in certain populations, and development of recommendations that are not broadly applicable. Goals: To develop best practices for recruitment and retention of racial/ethnic minorities for clinical research in pulmonary, critical care, and sleep medicine. Methods: The American Thoracic Society convened a workshop in May of 2019. This included an international interprofessional group from academia, industry, the NIH, and the U.S. Food and Drug Administration, with expertise ranging from clinical and biomedical research to community-based participatory research methods and patient advocacy. Workshop participants addressed historical and current mistrust of scientific research, systemic bias, and social and structural barriers to minority participation in clinical research. A literature search of PubMed and Google Scholar was performed to support conclusions. The search was not a systematic review of the literature. Results: Barriers at the individual, interpersonal, institutional, and federal/policy levels were identified as limiting to minority participation in clinical research. Through the use of a multilevel framework, workshop participants proposed evidence-based solutions to the identified barriers. Conclusions: To date, minority participation in clinical research is not representative of the U.S. and global populations. This American Thoracic Society research statement identifies potential evidence-based solutions by applying a multilevel framework that is anchored in community engagement methods and patient advocacy.

The care for individuals with cystic fibrosis (CF) with at least one F508del mutation will greatly change as a result of the unparalleled clinical benefits observed with the new triple-combination CFTR (CF transmembrane regulator)-modulator therapy elexacaftor/tezacaftor/ivacaftor (ETI). Incorporating ETI into the standard of care creates new motivation and opportunity to consider reductions in overall treatment burden and evaluate whether other chronic medications can now be safely discontinued without loss of clinical benefit. SIMPLIFY is a master protocol poised to test the impact of discontinuing versus continuing two commonly used chronic therapies in people with CF who are at least 12 years of age or older and stable on ETI therapy. The protocol is composed of two concurrent randomized controlled trials designed to evaluate the independent short-term effects of discontinuing hypertonic saline or dornase alfa, enabling individuals on both therapies to participate in one or both trials. The primary objective for each trial is to determine whether discontinuing treatment is noninferior to continuing treatment after establishment of ETI, as measured by the 6-week absolute change in the percent-predicted forced expiratory volume in 1 second. Developing this study required a balance between ideal study-design principles and feasibility. SIMPLIFY will be the largest multicenter, randomized, controlled medication-withdrawal study in CF. This study is uniquely positioned to provide timely evidence on whether the daily treatment burden can be reduced among individuals on CFTR-modulator therapy. Clinical trial registered with www.clinicaltrials.gov (NCT04378153).

Objective: To evaluate whether engagement and affective communication among adolescents and young adults (AYAs) with chronic kidney disease (CKD), caregivers, and pediatric nephrology providers during outpatient clinic visits predicts antihypertensive medication adherence., Methods: AYAs (n = 60, M age = 15.4 years, SD = 2.7, 40% female, 43% African American/Black) and caregivers (n = 60, 73% female) attended audio-recorded clinic visits with pediatric nephrologists (n = 12, 75% female). Recordings were analyzed using global affect ratings of the Roter Interactional Analysis System. Antihypertensive medication adherence was monitored electronically before and after clinic visits. A linear regression model evaluated associations between affect ratings and post-visit adherence., Results: AYAs took 84% of doses (SD = 20%) pre-visit and 82% of doses (SD = 24%) post-visit. Higher AYA engagement (β = 0.03, p = .01) and the absence of provider negative affect (β=-0.15, p = .04) were associated with higher post-visit adherence, controlling for pre-visit adherence, AYA sex, age, and race, and clustered by provider., Conclusions: Post-visit adherence was higher when AYAs were rated as more engaged and providers as less negative., Practice Implications: AYAs with lower engagement may benefit from further adherence assessment. Communication strategies designed to more actively engage AYAs in their care and diminish provider conveyance of negative affect during clinic visits may positively influence adherence among AYAs with CKD., Competing Interests: Declaration of Competing Interest The authors report no declarations of interest., (Copyright © 2020 Elsevier B.V. All rights reserved.)

Background: The prevalence of burnout and depressive symptoms is high among physician trainees., Research Question: What is the burden of burnout and depressive symptoms among fellows training in pulmonary and critical care medicine (PCCM) and what are associated individual fellow, program, and institutional characteristics?, Study Design and Methods: We conducted a cross-sectional electronic survey of fellows enrolled in pulmonary, PCCM, and critical care medicine training programs in the United States to assess burnout and depressive symptoms. Burnout symptoms were measured using the Maslach Burnout Index two-item measure. The two-item Primary Care Evaluation of Mental Disorders Procedure was used to screen for depressive symptoms. For each of the two outcomes (burnout and depressive symptoms), we constructed three multivariate logistic regression models to assess individual fellow characteristics, program structure, and institutional polices associated with either burnout or depressive symptoms., Results: Five hundred two of the 976 fellows who received the survey completed it-including both outcome measures-giving a response rate of 51%. Fifty percent of fellows showed positive results for either burnout or depressive symptoms, with 41% showing positive results for depressive symptoms, 32% showing positive results for burnout, and 23% showing positive results for both. Reporting a coverage system in the case of personal illness or emergency (adjusted OR [aOR], 0.44; 95% CI, 0.26-0.73) and access to mental health services (aOR, 0.14; 95% CI, 0.04-0.47) were associated with lower odds of burnout. Financial concern was associated with higher odds of depressive symptoms (aOR, 1.13; 95% CI, 1.05-1.22). Working more than 70 hours in an average clinical week and the burdens of electronic health record (EHR) documentation were associated with a higher odds of both burnout and depressive symptoms., Interpretation: Given the high prevalence of burnout and depressive symptoms among fellows training in PCCM, an urgent need exists to identify solutions that address this public health crisis. Strategies such as providing an easily accessible coverage system, access to mental health resources, reducing EHR burden, addressing work hours, and addressing financial concerns among trainees may help to reduce burnout or depressive symptoms and should be studied further by the graduate medical education community., (Copyright © 2020 American College of Chest Physicians. Published by Elsevier Inc. All rights reserved.)

Background: Burnout is common among physicians who care for critically ill patients and is known to contribute to worse patient outcomes. Fellows training in pulmonary and critical care medicine (PCCM) have risk factors that make them susceptible to burnout; for example, clinical environments that require increased intellectual and emotional demands with long hours. The Accreditation Council for Graduate Medical Education has recognized the increasing importance of trainee burnout and encourages training programs to address burnout. Objective: To assess factors related to training and practice that posed a threat to the well-being among fellows training in PCCM and to obtain suggestions regarding how programs can improve fellow well-being. Methods: We conducted a qualitative content analysis of data collected from a prior cross-sectional electronic survey with free-response questions of fellows enrolled in pulmonary, PCCM, and critical care medicine training programs in the United States. Fellows were asked what factors posed a threat to their well-being and what changes their training program could implement. Responses were qualitatively coded and categorized into themes using thematic analysis. Results: A total of 427 fellows (44% of survey respondents) completed at least one free-response question. The majority of respondents (60%) identified as male and white/non-Hispanic (59%). The threats to well-being and burnout were grouped into five themes: clinical burden, individual factors, team culture, limited autonomy, and program resources. Clinical burden was the most common threat discussed by fellows. Fellows highlighted factors contributing to burnout that specifically pertained to trainees including challenging interpersonal relationships with attending physicians and limited protected educational time. Fellows proposed solutions addressing clinical care, changes at the program or institution level, and organizational culture changes to improve well-being. Conclusion: This study provides insight into factors fellows report as contributors to burnout and decreased well-being in addition to investigating fellow-driven solutions toward improving well-being. These solutions may help pulmonary, PCCM, and critical care medicine program directors better address fellow well-being in the future., (Copyright © 2021 by the American Thoracic Society.)

Rationale: Decisions in medicine are made on the basis of knowledge and reasoning, often in shared conversations with patients and families in consideration of clinical practice guideline recommendations, individual preferences, and individual goals. Observational studies can provide valuable knowledge to inform guidelines, decisions, and policy. Objectives: The American Thoracic Society (ATS) created a multidisciplinary ad hoc committee to develop a research statement to clarify the role of observational studies-alongside randomized controlled trials (RCTs)-in informing clinical decisions in pulmonary, critical care, and sleep medicine. Methods: The committee examined the strengths of observational studies assessing causal effects, how they complement RCTs, factors that impact observational study quality, perceptions of observational research, and, finally, the practicalities of incorporating observational research into ATS clinical practice guidelines. Measurements and Main Results: There are strengths and weakness of observational studies as well as RCTs. Observational studies can provide evidence in representative and diverse patient populations. Quality observational studies should be sought in the development of ATS clinical practice guidelines, and medical decision-making in general, when 1 ) no RCTs are identified or RCTs are appraised as being of low- or very low-quality ( replacement ); 2 ) RCTs are of moderate quality because of indirectness, imprecision, or inconsistency, and observational studies mitigate the reason that RCT evidence was downgraded ( complementary ); or 3 ) RCTs do not provide evidence for outcomes that a guideline committee considers essential for decision-making (e.g., rare or long-term outcomes; " sequential" ). Conclusions: Observational studies should be considered in developing clinical practice guidelines and in making clinical decisions.

Importance: Asthma is the most common chronic childhood disease, with Black children experiencing worse morbidity and mortality. It is important to evaluate the effectiveness of efficacious interventions in community settings that have the greatest likelihood of serving at-risk families., Objective: To evaluate the effectiveness of a multilevel home- and school (Head Start)-based asthma educational program compared with a Head Start-based asthma educational program alone in improving asthma outcomes in children., Design, Setting, and Participant: This randomized clinical trial included 398 children with asthma enrolled in Head Start preschool programs in Baltimore, Maryland, and their primary caregivers. Participants were recruited from April 1, 2011, to November 31, 2016, with final data collection ending December 31, 2017. Data were analyzed from March 18 to August 30, 2018., Interventions: Asthma Basic Care (ABC) family education combined with Head Start asthma education compared with Head Start asthma education alone., Main Outcomes and Measures: Asthma control as measured by the Test for Respiratory and Asthma Control in Kids (TRACK) score., Results: Among the 398 children included in the analysis (247 boys [62.1%]; mean [SD] age, 4.2 [0.7] years), the ABC plus Head Start program improved asthma control (β = 6.26; 95% CI, 1.77 to 10.75; P < .001), reduced courses of oral corticosteroids (β = -0.61; 95% CI, -1.13 to -0.09; P = .02), and reduced hospitalizations (odds ratio, 0.36; 95% CI, 0.21-0.61; P < .001) during a 12-month period., Conclusion and Relevance: In this randomized clinical trial, combined family and preschool asthma educational interventions improved asthma control and reduced courses of oral corticosteroids and hospitalizations. Multilevel interventions implemented in community settings that serve low-income minority families may be key to reducing disparities in asthma outcomes., Trial Registration: ClinicalTrials.gov Identifier: NCT01519453.

Objective: To identify differences in perspectives of people with cystic fibrosis (PwCF) and caregivers versus healthcare providers on adherence barriers. Mismatched perspectives may lead to miscommunication and missed opportunities to reduce barriers and improve CF outcomes., Methods: PwCF, caregivers, and CF providers completed audio-taped, semi-structured interviews about adherence barriers. Interviews were transcribed and coded for themes. Themes were reviewed to identify when PwCF-caregiver perspectives differed from providers'., Results: Participants included 14 adolescents with CF (mean age = 15.89 years, 64 % female, 71 % Caucasian), 14 adults with CF (mean age = 30.03 years, 64 % female, 57 % Caucasian), 29 caregivers (76 % female; 72 % Caucasian), and 42 providers. Four barriers were identified that could generate miscommunication between PwCF-caregivers and providers: Tired = Fatigued/Sleepy versus Tired = Burnout, Vacation and Travel, Knowledge and Skills About CF Regimen, and Daily Habits or Routines. PwCF and caregivers used similar words as providers, but conceptualized barriers differently. PwCF and caregivers discussed barriers pragmatically, however, providers viewed certain barriers more abstractly or unidimensionally, or did not discuss them., Conclusions: PwCF-caregivers and providers may not align in how they discuss barriers, which may contribute to miscommunication about adherence challenges., Practice Implications: Patient-centered communication strategies may enhance providers' understandings of PwCF-caregiver perspectives on barriers and facilitate adherence interventions., Competing Interests: Declaration of Competing Interest The authors declare that they do not have any conflicts of interest to disclose., (Copyright © 2020 Elsevier B.V. All rights reserved.)

Objective: Among adolescents and young adults (AYAs) with chronic illness, effective provider communication is essential for patient-centered care during a sensitive developmental period. However, communication in chronic illness care for AYAs is not well studied. Our objectives were to describe the provider communication skills in pediatric chronic kidney disease (CKD) care visits; and determine if communication skills differ by AYA characteristics., Methods: We adapted a global consultation rating system for pediatric subspecialty care using audiotaped clinic encounters of 18 pediatric nephrologists with 99 AYAs (age M(SD) = 14.9(2.6)) with CKD stages 1-5 and 96 caregivers. We hypothesized that provider communication skills would differ by AYA characteristics (age, gender, and race)., Results: The strongest provider skills included initiating the session and developing rapport; lowest rated skills were asking patient's perspective and checking understanding. Communication scores did not consistently differ by AYA age or race, but were rated higher with female AYAs in several domains (ps<0.05)., Conclusions: Pediatric providers generally had adequate or good communication scores with AYAs, but improvement in certain skills, particularly with male AYAs, may further support patient-centered care., Practice Implications: To achieve consistent, patient-centered communication with AYAs, an observation-based global assessment may identify areas for provider improvement., Competing Interests: Declaration of Competing Interest None of the authors have any conflicts of interest, including financial interests or gains., (Copyright © 2020 Elsevier B.V. All rights reserved.)