"Time out for an hour, every day, my whole life": understanding treatment burden in cystic fibrosis in the era of Elexacaftor/Tezacaftor/Ivacaftor (ETI).

Background: Reducing treatment burden is a priority within the CF community. Improved outcomes for many people with CF (PWCF) following treatment with Elexacaftor/Tezacaftor/Ivacaftor (ETI) have led to considering whether daily CF regimens can be reduced. It is important to understand perceptions of PWCF when making decisions about care changes.

Associations Between Socioeconomic Status and Adherence to Medications in People With Cystic Fibrosis.

Background: Medication adherence is essential in managing cystic fibrosis (CF). The role of socioeconomic factors for medication adherence in people with CF is poorly understood, and their differential impact across the life course is underexplored. This study investigates associations between measures of socioeconomic status (SES)-educational attainment, household income, and health insurance type-and adherence to CF medications across age groups.

Developing a framework for clinical conversations using a qualitative analysis of the patient experience of SIMPLIFY.

Objectives: We explored the impact of taking part in a medication discontinuation study for people with cystic fibrosis (CF) on subsequent clinical conversations and what interviewees valued as characteristics of these conversations.

Methods: This analysis is part of the Qualitative Understanding of the Experience of SIMPLIFY Trial (QUEST), a qualitative companion study to a discontinuation trial of two commonly prescribed medications for people with CF. We interviewed 109 total individuals (87 people with CF and 22 caregivers).

Nephrology Provider Perceptions about Recommending Influenza and COVID-19 Vaccines: A Nationwide Survey.

Introduction: Vaccination rates for influenza and COVID-19 remain low among people with chronic kidney disease (CKD). Nephrology care offers an opportunity to boost vaccination rates. Understanding provider perceptions can be key to developing effective intervention programs.

Tacrolimus IPV Group Membership Does Not Consistently Track With Electronically Monitored or Self-reported Adherence in Adolescent and Young Adult Kidney Transplant Recipients.

Background: High intrapatient variability (IPV) of tacrolimus trough concentrations (SD ≥ 2, coefficient of variation (CV%) ≥ 30) is associated with rejection and graft loss and may indicate nonadherence. However, the association between tacrolimus IPV and electronically monitored or self-reported adherence is unknown.

Methods: This secondary analysis of the TAKE-IT (Teen Adherence in Kidney Transplant Effectiveness of Intervention Trial) compared electronically monitored and self-reported adherence between participants with high and low tacrolimus IPV among 103 adolescent and young adult kidney transplant recipients (mean age = 15.

Development of a Diabetes Navigator Toolkit to Support Diabetes Technology Uptake: The IMPACT Toolkit.

Advanced diabetes technologies (ADTs), including continuous glucose monitoring devices, insulin pumps, and automated insulin delivery systems, improve glycemic control in type 1 diabetes but are underutilized, particularly in minority and underserved populations. This article reports on a qualitative study to develop a diabetes toolkit. Participants identified barriers to the use of ADTs and proposed solutions to these barriers, which were then incorporated into a diabetes toolkit designed for use by a diabetes navigator or other diabetes care team members seeking to improve the uptake and sustained use of ADTs.

Provider Practices in Pulmonary Exacerbations of Cystic Fibrosis in the Era of Highly Effective Modulator Therapy.

Since the approval of elexacaftor/tezacaftor/ivacaftor (ETI), data suggest that there have been changes in the management of pulmonary exacerbations (PEx) of cystic fibrosis. Given the subjective nature of PEx diagnosis and management, we sought to characterize provider PEx management practices in people with cystic fibrosis (pwCF) prescribed highly effective modulator therapy (HEMT) and to identify practice changes that may impact clinical outcomes. We conducted semistructured qualitative interviews among clinicians in the United States from late 2021 to 2022 to investigate changes in the management of PEx in pwCF prescribed ETI.

Mental health and adherence in CF: Self-efficacy and perceived barriers as mediators.

Background: Symptoms of depression and anxiety can contribute to lower medical treatment adherence. Given that people with cystic fibrosis (PWCF) have higher rates of depressive and anxiety symptoms than those without cystic fibrosis (CF), this study examined factors that mediated the association between mental health and adherence.

Methods: Participants were 294 adults (M age=25 years) with CF who were enrolled in the Daily Care Check-in Validation Study.

Individual and system-level determinants of caregivers' quality of life among inner-city preschool children with asthma.

Objective: 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.

Methods: The primary outcome was caregiver HRQoL measured at baseline and six months.

"It's Like You're Feeding Your Child Twice": Barriers and Facilitators to Human Milk Feeding Children With Cystic Fibrosis.

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.

Coping and learning to Manage Stress with cystic fibrosis (CALM): A multisite telehealth randomized controlled trial to reduce depression and anxiety symptoms in adults with cystic fibrosis.

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).

Qualitative understanding of experiences of people with cystic fibrosis in a treatment discontinuation trial: The QUEST study.

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.

Supporting adherence to the cystic fibrosis regimen: Development and validation of The Daily Care Check-In (DCC).

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.

User Engagement With mHealth Interventions to Promote Treatment Adherence and Self-Management in People With Chronic Health Conditions: Systematic Review.

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.

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

Evaluating approaches to reduce treatment burden is a research priority among people with cystic fibrosis on highly effective modulators, including elexacaftor-tezacaftor-ivacaftor (ETI). 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. 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.

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.

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).

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.

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.

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.

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.

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.