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.

Self-reported chronic therapy use after 24-weeks of follow-up by participants who completed the simplify randomized, controlled trial.

Background: Highly effective CFTR modulator therapy (HEMT) has improved the health of many people with cystic fibrosis (pwCF), offering opportunities to discontinue burdensome therapies. SIMPLIFY included randomized, controlled trials that confirmed non-inferiority of discontinuing versus continuing dornase alfa (DA) or hypertonic saline (HS) for 6 weeks in pwCF on HEMT. In this study of post-trial treatment use by SIMPLIFY participants, we hypothesized that randomization to discontinue DA or HS during the trial would be associated with a higher likelihood of non-use of each medication during follow-up.

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).

Effectiveness of artificial intelligence vs. human coaching in diabetes prevention: a study protocol for a randomized controlled trial.

Background: Prediabetes is a highly prevalent condition that heralds an increased risk of progression to type 2 diabetes, along with associated microvascular and macrovascular complications. The Diabetes Prevention Program (DPP) is an established effective intervention for diabetes prevention. However, participation in this 12-month lifestyle change program has historically been low.

Cross-Cutting mHealth Behavior Change Techniques to Support Treatment Adherence and Self-Management of Complex Medical Conditions: Systematic Review.

Background: Mobile health (mHealth) interventions have immense potential to support disease self-management for people with complex medical conditions following treatment regimens that involve taking medicine and other self-management activities. However, there is no consensus on what discrete behavior change techniques (BCTs) should be used in an effective adherence and self-management-promoting mHealth solution for any chronic illness. Reviewing the extant literature to identify effective, cross-cutting BCTs in mHealth interventions for adherence and self-management promotion could help accelerate the development, evaluation, and dissemination of behavior change interventions with potential generalizability across complex medical conditions.

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.

The effect of elexacaftor/tezacaftor/ivacaftor on non-pulmonary symptoms in adults with cystic fibrosis.

Background: Elexacaftor/Tezacaftor/Ivacaftor (ETI) is a CFTR modulator that has led to large benefits in lung function, pulmonary exacerbation rates, and respiratory symptoms. Less is known about the effect of ETI on non-pulmonary symptoms. The objective of this study was to examine the changes in patient reported outcomes after starting ETI in multiple non-pulmonary symptoms.

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.

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.

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.

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.

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.

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.

Understanding barriers to and strategies for medication adherence in COPD: a qualitative study.

Background: Medication adherence in chronic obstructive pulmonary disease (COPD) is low, though not enough is known about the factors that affect adherence in COPD. This study uses qualitative methods to understand the patient perspective on facilitators and barriers to medication adherence in COPD as well as patient-reported strategies for self-management of disease.

Methods: Semi-structured interviews were conducted with 30 individuals (n = 30).

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.