Impact of Discontinuing Both Hypertonic Saline and Dornase Alfa After Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis.

Rationale Evaluating approaches to reduce treatment burden is a research priority among people with CF (pwCF) on highly effective modulators including elexacaftor/tezacaftor/ivacaftor (ETI). Objective 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 comprising 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 versus 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 one second (ppFEV1) at study entry. Results There were 43 participants who discontinued both therapies by the end of SIMPLIFY and 63 who remained on both, with overall average ppFEV1 at study entry 96.7% and average duration of follow up from beginning of the first trial to completion of the second trial 3.9 months, including time between trials. No clinically meaningful difference in the change in ppFEV1 from baseline to completion of the second trial was observed between those who discontinued versus remained on both therapies (difference: 0.22% Off-On, 95% CI: -1.60,2.03). Changes in LCI2.5, patient reported, and safety outcomes were also comparable. Patient reported treatment burden, as measured by a CFQ-R subscale, significantly decreased in those discontinuing 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 to those who remained on both therapies. These data continue to inform a new era of post-modulator care of pwCF.

Hydroxyurea to prevent brain injury in children with sickle cell disease (HU Prevent)-A randomized, placebo-controlled phase II feasibility/pilot study.

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-ß0-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.

Development and preliminary validation of the personalized cystic fibrosis medication questionnaire (PCF-MQ).

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.

Remote endpoints for clinical trials in cystic fibrosis: Report from the U.S. CF foundation remote endpoints task force.

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.

An emotional journey: caregiver experiences with gastrostomy tube decision-making for children with cystic fibrosis.

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.

Factors Associated With Non-vaccination for Influenza Among Patients With CKD: Findings From the Chronic Renal Insufficiency Cohort (CRIC) Study.

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/1.73m2), 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.

Asthma routinization, family asthma management, caregiver depressive symptoms, and medication adherence in Head Start preschool children.

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.

Medication adherence in youths with CKD: habits for success.

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.

Partners in research: The success with therapies research consortium and the CF community unite to improve self-management.

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.

Early life growth trajectories in cystic fibrosis are associated with lung function at age six.

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.

Clinician attitudes and practices on pregnancy planning and care in cystic fibrosis.

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 peri­pregnancy expertise within the CF community, and future studies investigating the maternal-fetal effects of CF therapies are needed.

Discontinuation versus continuation of hypertonic saline or dornase alfa in modulator treated people with cystic fibrosis (SIMPLIFY): results from two parallel, multicentre, open-label, randomised, controlled, non-inferiority trials.

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 FEV1 (ppFEV1) of 70% or more, or those aged 18 years or older with ppFEV1 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 ppFEV1 (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 ppFEV1 in the per-protocol population. We established a non-inferiority margin of -3% for the difference between groups in the 6-week change in ppFEV1. 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 ppFEV1 of 96·9%. Discontinuing treatment was non-inferior to continuing treatment with respect to the absolute 6-week change in ppFEV1 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.

Self-Determination Theory and Preventive Medication Adherence: Motivational Considerations to Support Historically Marginalized Adolescents With Asthma.

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.

Clinical outcomes in cystic fibrosis at 6 years of age with tricare insurance coverage.

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 FEV1 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.

Agreement between attended home and ambulatory blood pressure measurements in adolescents with chronic kidney disease.

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.

Association between insurance variability and early lung function in children with cystic fibrosis.

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 FEV1 percent predicted (maxFEV1pp). 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 maxFEV1pp 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.

Effect of the School-Based Asthma Care for Teens (SB-ACT) program on asthma morbidity: a 3-arm randomized controlled trial.

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.

Family management of asthma in Head Start preschool children.

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.

Enhancing Recruitment and Retention of Minority Populations for Clinical Research in Pulmonary, Critical Care, and Sleep Medicine: An Official American Thoracic Society Research Statement.

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.