Contraceptive use and pregnancy in cystic fibrosis: Survey findings from 10 cystic fibrosis centers.

Background: Reproductive life planning is key, now that people with cystic fibrosis (pwCF) may live into their 60s. This study explores contraceptive use, pregnancy trends, and whether concomitant cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy reduces contraceptive effectiveness.

Methods: Females with CF aged 18-45 years from 10 U.

Cystic Fibrosis Experience of Care Survey: Patient-Caregiver-Clinician Collaborative Design and Implementation.

People with cystic fibrosis (PwCF), families, and clinicians, partner to co-produce care, navigate access barriers, address mental health and social factors, follow specific infection prevention and control practices, and share decision-making regarding treatments and daily care. Standard patient satisfaction and experience of care surveys are not tailored to return relevant, actionable data for specific populations. To improve the care experience, the U.

Health care transition quadruple aim outcomes for IDD: Scoping review.

Purpose: Structured HCT models addressing planning, transfer, and integration into adult care for adolescents and young adults with childhood-acquired chronic conditions are becoming more prevalent. However, consensus on outcome measures to assess health care transition (HCT) interventions particularly for intellectual and developmental disabilities (IDD) population is lacking. This scoping review identified potential HCT outcome measures for young adults (aged 18-26) with IDD using the Quadruple Aim Framework.

Healthcare transition readiness of families of youth with cystic fibrosis during COVID-19: A correlational multicenter analysis.

Background: Enhancing family readiness for the healthcare transition (HCT) to adult-focused care can help adolescents and young adults (AYA) thrive in adulthood. We aimed to explore modifiable and non-modifiable individual, family, and healthcare factors associated with HCT readiness of AYA among families of AYA with cystic fibrosis (CF) during COVID-19.

Methods: A multi-site, cross-sectional design was used and an online survey was deployed among families and their AYA from three US pediatric CF centers.

Impact of Cystic Fibrosis Transmembrane Conductance Regulator Modulators on Maternal Outcomes During and After Pregnancy.

Background: Cystic fibrosis (CF) transmembrane conductance regulator (CFTR) modulators are available to the majority of people with CF in the United States; little is known about pregnancy outcomes with modulator use. The aim of this retrospective study was to determine the impact of CFTR modulators on maternal outcomes.

Research Question: Does pregnancy differentially affect outcomes in female individuals with CF with and without CFTR modulator exposure?

Study Design And Methods: Data on pregnancies from 2010 to 2021 were collected from 11 US adult CF centers.

Continuous glucose monitoring and advanced glycation endproducts for prediction of clinical outcomes and development of cystic fibrosis-related diabetes in adults with CF.

Introductions: Cystic fibrosis-related diabetes (CFRD) is associated with pulmonary decline, compromised nutritional status, and earlier mortality. Onset is often insidious, so screening for early detection of glycemic abnormalities is important. Continuous glucose monitoring (CGM) has been validated in people with CF and has been shown to detect early glycemic variability otherwise missed on 2-hour oral glucose tolerance testing (OGTT).

Randomized Trial of the Insulin-Only iLet Bionic Pancreas for the Treatment of Cystic Fibrosis- Related Diabetes.

Objective: Cystic fibrosis-related diabetes (CFRD) affects up to 50% of adults with cystic fibrosis and adds significant morbidity and treatment burden. We evaluated the safety and efficacy of automated insulin delivery with the iLet bionic pancreas (BP) in adults with CFRD in a single-center, open-label, random-order, crossover trial.

Research Design And Methods: Twenty participants with CFRD were assigned in random order to 14 days each on the BP or their usual care (UC).

Cystic fibrosis-related mortality in the United States from 1999 to 2020: an observational analysis of time trends and disparities.

Cystic fibrosis transmembrane conductance regulator modulators have revolutionized cystic fibrosis (CF) care in the past decade. This study explores the CF-related mortality trends in the US from 1999 to 2020. We extracted CF-related mortality data from the CDC WONDER database.

Association between unplanned pregnancies and maternal exacerbations in cystic fibrosis.

Introduction: Following availability of the highly effective cystic fibrosis (CF) transmembrane conductance regulator modulator, elexacaftor/tezacaftor/ivacaftor, there was a near doubling of pregnancies reported in the United States (US) in people with CF. We sought to determine health impacts of planned (PP) versus unplanned pregnancies (UP).

Methods: We collected retrospective pregnancy data from January 2010-December 2020 from 11 US CF centers.

Development and Validation of a Respiratory-Responsive Vocal Biomarker-Based Tool for Generalizable Detection of Respiratory Impairment: Independent Case-Control Studies in Multiple Respiratory Conditions Including Asthma, Chronic Obstructive Pulmonary Disease, and COVID-19.

Background: Vocal biomarker-based machine learning approaches have shown promising results in the detection of various health conditions, including respiratory diseases, such as asthma.

Objective: This study aimed to determine whether a respiratory-responsive vocal biomarker (RRVB) model platform initially trained on an asthma and healthy volunteer (HV) data set can differentiate patients with active COVID-19 infection from asymptomatic HVs by assessing its sensitivity, specificity, and odds ratio (OR).

Methods: A logistic regression model using a weighted sum of voice acoustic features was previously trained and validated on a data set of approximately 1700 patients with a confirmed asthma diagnosis and a similar number of healthy controls.

Safety and efficacy of vanzacaftor-tezacaftor-deutivacaftor in adults with cystic fibrosis: randomised, double-blind, controlled, phase 2 trials.

Background: Elexacaftor-tezacaftor-ivacaftor has been shown to be safe and efficacious in people with cystic fibrosis and at least one F508del allele. Our aim was to identify a novel cystic fibrosis transmembrane conductance regulator (CFTR) modulator combination capable of further increasing CFTR-mediated chloride transport, with the potential for once-daily dosing.

Methods: We conducted two phase 2 clinical trials to assess the safety and efficacy of a once-daily combination of vanzacaftor-tezacaftor-deutivacaftor in participants with cystic fibrosis who were aged 18 years or older.

The Relationship between Body Composition, Dietary Intake, Physical Activity, and Pulmonary Status in Adolescents and Adults with Cystic Fibrosis.

Measures of body fat and lean mass may better predict important clinical outcomes in patients with cystic fibrosis (CF) than body mass index (BMI). Little is known about how diet quality and exercise may impact body composition in these patients. Dual X-ray absorptiometry (DXA) body composition, 24-h dietary recall, and physical activity were assessed in a cross-sectional analysis of 38 adolescents and adults with CF and 19 age-, race-, and gender-matched healthy volunteers.

Continuous Glucose Monitoring and HbA1c in Cystic Fibrosis: Clinical Correlations and Implications for CFRD Diagnosis.

Context: The clinical utility and implications of continuous glucose monitoring (CGM) in cystic fibrosis (CF) are unclear.

Objective: We examined the correlation between CGM measures and clinical outcomes in adults with CF, investigated the relationship between hemoglobin A1c (HbA1c) and CGM-derived average glucose (AG), and explored CGM measures that distinguish cystic fibrosis-related diabetes (CFRD) from normal and abnormal glucose tolerance.

Methods: This prospective observational study included 77 adults with CF who had CGM and HbA1c measured at 2 to 3 time points 3 months apart.

Issues affecting young people with asthma through the transition period to adult care.

Asthma is among the most common medical conditions affecting children and young people, with adolescence a recognised period of increased risk, overrepresented in analyses examining recent increasing asthma mortality rates. Asthma may change significantly during this period and management also occurs in the context of patients seeking increased autonomy and self-governance whilst navigating increasing academic and social demands. A number of disease factors can destabilise asthma during adolescence including: increased rates of anaphylaxis, anxiety, depression, obesity, and, in females, an emerging resistance to corticosteroids and the pro-inflammatory effects of oestrogen.

Empire-CF study: A phase 2 clinical trial of leukotriene A4 hydrolase inhibitor acebilustat in adult subjects with cystic fibrosis.

Background: Cystic fibrosis (CF) is characterized by neutrophilic inflammation in the airways. Leukotriene B4 (LTB) is a neutrophil chemoattractant and has been implicated in CF pathogenesis. Acebilustat, a novel, synthetic, small-molecule leukotriene A4 hydrolase inhibitor, reduces LTB production.

The effect of elexacaftor/tezacaftor/ivacaftor (ETI) on glycemia in adults with cystic fibrosis.

Background: Cystic fibrosis related diabetes (CFRD) is associated with pulmonary decline and compromised nutritional status. Emerging data suggest that CFTR dysfunction may play a direct role in the pathogenesis of CFRD; however, studies investigating the effect of CFTR modulators on glycemic outcomes in patients with cystic fibrosis (CF) have shown mixed results. The impact of elexacaftor-tezacaftor-ivacaftor (ETI) on glycemic control is currently unknown.

Family-building and parenting considerations for people with cystic fibrosis.

As people with cystic fibrosis (CF) live longer and healthier lives, increasing numbers are considering the full range of reproductive options for their futures, including parenthood, pregnancy, or pregnancy prevention. As the face of CF changes, the CF care model must adapt to meet the reproductive health needs of both parents and nonparents with CF. This article summarizes the reproductive goals and family-building concerns faced by people with CF, including fertility, pregnancy, and alternative paths to parenthood, the impact of parenthood on mental and physical health, and important future research.

The Effects of Ivacaftor on Bone Density and Microarchitecture in Children and Adults with Cystic Fibrosis.

Context: Cystic fibrosis (CF) transmembrane conductance (CFTR) dysfunction may play a role in CF-related bone disease (CFBD). Ivacaftor is a CFTR potentiator effective in improving pulmonary and nutritional outcomes in patients with the G551D-CFTR mutation. The effects of ivacaftor on bone health are unknown.

Supporting the Medically Fragile: Individualized Approach to Empowering Young Adults With Chronic Disease During the COVID-19 Pandemic.

Young adults with chronic and complex diseases face systemic barriers, care fragmentation, and increased vulnerabilities. Novel coronavirus pandemic has proven to further complicate care coordination for young adult patients with medical and psychosocial complexities. The BRIDGES Young Adult Program at Boston Children's Hospital has 6 years of experience advocating for and empowering young adults with chronic medical conditions, and their families, through outpatient consults aimed to assist with subspecialty guidance and defragmentation of care during the time of transition from pediatric to adult care.

Integration of mental health screening and treatment into cystic fibrosis clinics: Evaluation of initial implementation in 84 programs across the United States.

Background: A large-scale epidemiological study of 6088 individuals with cystic fibrosis (CF) and 4102 caregivers in nine countries documented elevated symptoms of depression and anxiety, leading to international guidelines for annual screening and follow-up. To facilitate national implementation, 84 CF programs funded a mental health coordinators (MHC). Implementation was evaluated after 1 year using the consolidated framework for implementation research (CFIR) to identify facilitators and barriers.

Prevalence and clinical associations of Staphylococcus aureus small-colony variant respiratory infection in children with cystic fibrosis (SCVSA): a multicentre, observational study.

Background: Staphylococcus aureus is the bacterium cultured most often from respiratory secretions of people with cystic fibrosis. Both meticillin-susceptible S aureus and meticillin-resistant S aureus (MRSA) can adapt to form slow-growing, antibiotic-resistant isolates known as small-colony variants that are not routinely identified by clinical laboratories. We aimed to determine the prevalence and clinical significance of S aureus small-colony variants and their subtypes among children with cystic fibrosis.