Adult Cystic Fibrosis Program Research Articles

"I eat chocolate milk for dinner because we just have nothing in our fridge": The invisible burden and dire consequences of food insecurity for people with cystic fibrosis in the United States.

One-third of people with cystic fibrosis (pwCF) are food insecure, with profound negative implications for their health. This qualitative study explored lived experiences with food insecurity among pwCF or their caregivers and summarized their perspectives on food insecurity screening in the cystic fibrosis (CF) programs where they receive care. Semi-structured qualitative interviews were conducted with two groups: (1) adults with CF and (2) parents or caregivers of children with CF. PwCF or their caregivers with previously documented food insecurity were referred for participation by pediatric and adult CF programs across the United States. Interviews were recorded and transcribed, and data were coded and analyzed by two independent coders using a content-analysis approach with a constant comparative method to generate themes. A total of 26 participants from 22 CF programs were interviewed. The sample included 17 adults with CF and nine parents of children with CF. Participants were predominantly White (88%) and female (92%). Five overarching themes emerged: (1) food insecurity among CF patients and their families is onerous, (2) financial constraints imposed by the CF disease contribute to food insecurity, (3) federal and state programs provide limited food assistance, and other support is minimal, (4) shame and stigma engulf conversations around food insecurity with CF care teams, and (5) food insecurity screening in clinical settings is critical. Food insecurity among pwCF is invisible, but its consequences are dire. Assistance is limited, screening is inconsistent, and stigma is widespread. There is an urgent need to normalize food insecurity screening, standardize the screening process, and expand food assistance programs for pwCF.

Genomic epidemiology of Mycobacterium abscessus at an adult cystic fibrosis programme reveals low potential for healthcare-associated transmission.

Nontuberculous mycobacteria (NTM) has been reported to be transmitted between people with cystic fibrosis (CF) attending CF centres. A suspected Mycobacterium abscessus outbreak was investigated at the University of Texas Southwestern (UTSW) Adult CF Program using a combination of pathogen genomic sequencing and epidemiologic methods. The objectives of the present study were to apply the Healthcare-Associated Links in Transmission of NTM (HALT NTM) study to investigate the occurrence of potential healthcare-associated transmission and/or acquisition of NTM among people with CF infected with genetically similar NTM isolates. Whole-genome sequencing of respiratory M. abscessus isolates from 50 people with CF receiving care at UTSW was performed to identify genetically similar isolates. Epidemiologic investigation, comparison of respiratory and environmental isolates, and home residence watershed mapping were studied. Whole-genome sequencing analysis demonstrated seven clusters of genetically similar M. abscessus (four ssp. abscessus and three ssp. massiliense). Epidemiologic investigation revealed potential opportunities for healthcare-associated transmission within three of these clusters. Healthcare environmental sampling did not recover M. abscessus, but did recover four human disease-causing species of NTM. No subjects having clustered infections lived in the same home residence watershed. Some subjects were infected with more than one M. abscessus genotype, both within and outside of the dominant circulating clones. Healthcare-associated person-to-person transmission of M. abscessus appears to be rare at this centre. However, polyclonal infections of M. abscessus species and subspecies, not originating from the endemic hospital environment, suggest multiple shared modes of acquisition outside the healthcare setting.

Investigating Nontuberculous Mycobacteria Transmission at the Colorado Adult Cystic Fibrosis Program.

Rationale: Healthcare-associated transmission of nontuberculous mycobacteria (NTM) among people with cystic fibrosis (pwCF) has been investigated at CF centers worldwide, with conflicting conclusions. We investigated transmission at the Colorado Adult CF Program. Objectives: To systematically investigate healthcare-associated transmission and/or acquisition of NTM to determine similarity among respiratory and environmental isolates, and to compare home residence watershed mapping among pwCF having genetically similar NTM isolates. Methods: Whole-genome sequencing of NTM isolates from 80 pwCF was conducted to identify genetically similar isolate clusters (⩽30 SNP differences). Epidemiology, comparison of respiratory and environmental isolates, and home residence watershed mapping were analyzed. Measurements and Main Results: Whole-genome sequencing analysis revealed 11 clusters of NTM [6 Mycobacterium abscessus subspecies (ssp.) abscessus, 1 M. abscessus ssp. massiliense, 2 Mycobacterium avium, and 2 Mycobacterium intracellulare] among pwCF. Epidemiologic investigation demonstrated opportunities for healthcare-associated transmission in two M. abscessus and two M. avium clusters. Respiratory and healthcare environmental isolate comparisons revealed no genetic similarity. Individuals comprising one M. abscessus cluster, with no plausible healthcare-associated transmission, resided in the same watershed. Conclusions: This study suggests healthcare-associated transmission of M. abscessus is rare and includes a report of potential healthcare-associated transmission of M. avium among pwCF. One M. abscessus cluster possibly had common acquisition arising from residing in the same watershed. The presence of genetically similar isolates is insufficient to demonstrate healthcare-associated NTM transmission. Standardizing epidemiologic investigation, combined with environmental sampling and watershed analysis, will improve understanding of the frequency and nature of healthcare-associated NTM transmission among pwCF.

Bone mineral density screening by DXA for people with cystic fibrosis: A registry analysis of patient and program factors influencing rates of screening

Background: Current guidelines recommend screening for Cystic Fibrosis (CF) related bone disease (CFBD) using dual-energy x-ray absorptiometry (DXA) for all people ≥ 18 years of age and select people ≥ 8 years of age. However, adherence to these guidelines is variable. This study aims to evaluate screening practices among adult programs in the US and identify patient and program-based characteristics which may influence screening.Methods: The CF Foundation Patient Registry (CFFPR) was used to identify all people over the age of 18 who were seen at adult CF programs and received screening for CFBD using DXA at least once between 2014 and 2018. Associations with patient and program level characteristics were assessed using the Chi Square test. Patient level variables were also examined using standardized difference to assess for meaningful clinical differences in rates of screening.Results: From 2014 to 2018, a total of 15,134 people over the age of 18 were identified in the CFFPR. Of these people, 9,023 (60%) received a DXA during the time period. The median rate of screening by program was 66% and programs in the highest quartile of screening obtained DXAs on >76% of their population. At the program level, larger size and increased adherence to other guideline practices such as OGTT screening and 4 visits, 4 cultures in a year correlated with higher rates of screening for CFBD. At the patient-level, people with lower lung function (FEV1 <90%) and those with CF related diabetes were more likely to be screened. People without health insurance were less likely to receive recommended screening.Conclusion: Screening practices for CFBD vary widely across adult programs in the US despite recommendations to screen all people over the age of 18. Factors identified in this analysis may be used to identify those people at highest risk of missing appropriate screening. CFBD has significant implications for our patients and therefore routine screening should be emphasized as part of standard care moving forward.

Exploring provider attitudes and perspectives related to men's health in cystic fibrosis

BackgroundNew modulator therapies have markedly improved the health of people with cystic fibrosis (CF), allowing an increased focus on quality-of-life improvements for men with CF, including those related to sexual and reproductive health (SRH). This study explored CF providers’ attitudes and experiences with addressing men's health in CF. MethodsWe interviewed geographically diverse adult and pediatric United States (U.S.) CF program directors via semi-structured telephone interviews exploring their perspectives and practices related to men's SRH in CF. Two coders analyzed transcribed interviews and created a codebook to identify key themes. ResultsWe interviewed 20 providers and identified the following themes: 1) Men's SRH is important to address within CF care, but there is no standardization around this aspect of care; 2) There is no consensus about the recommendation or utilization of semen analysis to assess men's infertility; 3) There are many barriers to men's SRH care provision in CF centers, including the low priority of SRH concerns and provider discomfort and lack of expertise in SRH; 4) Providers desire clear evidence-based guidelines and patient resources related to men's SRH in CF; and 5) Providers believe future research should focus on testosterone and the impact of modulators on men's SRH. ConclusionsCF center directors acknowledge the importance of addressing SRH with men with CF, but there is a lack of standardization and research in this aspect of care. Existing barriers to optimal SRH care and identified facilitators in this study can serve as targets for interventions in the CF care model.

INCREASED CYTOKINE LEVELS DEFINE THE AIRWAY OF STABLE CYSTIC FIBROSIS PATIENTS COMPARED TO HEALTHY CONTROLS

TOPIC: Genetic and Developmental Disorders TYPE: Original Investigations PURPOSE: Cystic Fibrosis (CF) is a multisystem autosomal recessive disease caused by mutations in the CFTR (CF transmembrane conductance regulator) gene. Airway inflammation and infection in CF leads to tissue injury, worsening lung function, and disease progression. Increased cytokine levels reflect the exaggerated inflammation characteristic of the CF airway environment and contribute to pathogenesis even in the absence of respiratory symptoms. We sought to define individual CF-specific cytokine variations as a means to characterize the difference between healthy and CF airways, and to identify patients at risk of clinical deterioration. METHODS: Sputum samples were obtained from Forty-four adult patients with confirmed CF diagnosis from the Yale Adult CF Program. Patients were classified as CF stable at each visit if they did not meet predefined criteria for an acute exacerbation. Ten healthy controls (HC) were recruited to undergo sputum induction. Sputum samples were assayed for twelve cytokines using a multiplexed indirect ELISA. Differences in cytokine levels between CF stable and healthy controls were analyzed using the Mann-Whitney Test with Bonferroni correction. RESULTS: Twelve inflammatory cytokine levels were measured. Six cytokines were significantly increased in the sputum of stable CF patients compared to healthy controls (median values, pg/mL). These cytokines included: IL-1beta (CF 1720; HC 39.7), IL-8 (CF 28173; HC 3468), TNF-alpha (CF 98.65; HC 10.70), and IFN-alpha (CF 38.90; HC 5.60) [p < 0.0001] as well as IFN-gamma (CF 68.45; HC 17.10) and IL-13 (20.10; 0.00) [p < 0.01]. Cytokines tested without significant difference between groups included: CXCL10, G-CSF, IFNI, IL-6, MCP1, and MIP-alpha. CONCLUSIONS: Inflammatory cytokine levels of IL-1beta, IL-8, TNF-alpha, IFN-a, IFN-gamma and IL-13 are significantly increased in the sputum of stable CF patients compared to the sputum of healthy controls. CLINICAL IMPLICATIONS: Monitoring basal sputum cytokine levels may help identify patients with worsening inflammation before symptoms develop and thus prevent irreversible lung damage and disease progression. Additionally, further understanding of inflammatory cytokine dysregulation in stable CF airways may serve as the basis for a sputum inflammation marker panel that guides future therapeutic interventions. DISCLOSURES: No relevant relationships by Clemente Britto, source=Web Response Consultant relationship with AstraZeneca Please note: >$100000 by Geoffrey Chupp, source=Web Response, value=Consulting fee Consultant relationship with Genentech Please note: $20001 - $100000 by Geoffrey Chupp, source=Web Response, value=Consulting fee Consultant relationship with Genzyme Corporation Please note: $5001 - $20000 by Geoffrey Chupp, source=Web Response, value=Consulting fee Consultant relationship with Novartis Please note: $5001 - $20000 by Geoffrey Chupp, source=Web Response, value=Consulting fee Consultant relationship with Circassia Please note: $1001 - $5000 by Geoffrey Chupp, source=Web Response, value=Consulting fee Consultant relationship with Boehringer Ingelheim Please note: $1001 - $5000 by Geoffrey Chupp, source=Web Response, value=Consulting fee Consultant relationship with GlaxoSmithKline Please note: $1-$1000 by Geoffrey Chupp, source=Web Response, value=Consulting fee Consultant relationship with Regeneron Please note: 5/2019-present Added 04/26/2021 by Lauren Cohn, source=Web Response, value=Consulting fee Speaker/Speaker's Bureau relationship with Genentech Please note: 5/2019-11/2019 Added 04/26/2021 by Lauren Cohn, source=Web Response, value=Honoraria Advisory Committee Member relationship with novartis Please note: 10/2019 Added 04/26/2021 by Lauren Cohn, source=Web Response, value=Consulting fee Advisory Committee Member relationship with glaxosmithkline Please note: 12/2019 Added 04/26/2021 by Lauren Cohn, source=Web Response, value=Consulting fee Advisory Committee Member relationship with sanofi Please note: 5/2020 Added 04/26/2021 by Lauren Cohn, source=Web Response, value=Consulting fee Advisory Committee Member relationship with Pieris Please note: 5/2019 Added 04/26/2021 by Lauren Cohn, source=Web Response, value=Consulting fee Advisory Committee Member relationship with AstraZeneca Please note: 5/2019-7/2020 Added 04/26/2021 by Lauren Cohn, source=Web Response, value=Consulting fee Consultant relationship with Biohaven Please note: 6/23/2020 Added 04/26/2021 by Lauren Cohn, source=Web Response, value=Consulting fee No relevant relationships by Sara Khanal, source=Web Response No relevant relationships by qing liu, source=Web Response No relevant relationships by Jana Zielonka, source=Web Response

Health Disparities among adults cared for at an urban cystic fibrosis program

BackgroundEvidence is conflicting regarding differential health outcomes in racial and ethnic minorities with cystic fibrosis (CF), a rare genetic disease affecting approximately 28,000 Americans. We performed a cross-sectional analysis of health outcomes in Black/Latinx patients compared with non-Hispanic Caucasian patients cared for in a CF center in New York City. Adult patients enrolled in the CF Foundation Patient Registry at the Columbia University Adult CF Program and seen at least once during 2019 were included. Health metrics were compared between Black/Latinx and non-Hispanic Caucasian patients.Results262 patients were eligible. 39 patients (15%) identified as Black/Latinx or non-Hispanic Caucasian. Descriptive statistics are reported with mean (standard deviation). Current age was 35.9 (13.3) years for non-Hispanic Caucasian and 32.0 (9.3) years for Black/Latinx patients (p = 0.087). Age of diagnosis did not differ between groups; 9.56 (15.96) years versus 11.59 (15.8) years for non-Hispanic Caucasian versus Black/Latinx respectively (p = 0.464). Pulmonary function, measured as mean forced expiratory volume in one second (FEV1) was 70.6 (22.5) percent predicted in non-Hispanic Caucasian versus 59.50 (27.9) percent predicted in Black/Latinx patients (p = 0.010). Number of visits to the CF clinic were similar between groups. When controlled for age, gender, co-morbidities, median income, and insurance status, there was a continued association between minority status and lower FEV1.ConclusionsMinorities with CF have significantly lower pulmonary function, the major marker of survival, than non-Hispanic Caucasians, even when controlled for a variety of demographic and socioeconomic factors that are known to affect health status in CF. Significant health disparities based on race and ethnicity exist at a single CF center in New York City, despite apparent similarities in access to guideline based care at an accredited CF Center. This data confirms the importance of design of culturally appropriate preventative and management strategies to better understand how to direct interventions to this vulnerable population with a rare disease.

Preserving Multidisciplinary Care Model and Patient Safety During Reopening of Ambulatory Cystic Fibrosis Clinic for Nonurgent Care: A Hybrid Telehealth Model.

Introduction: The University of Virginia's (UVA's) adult cystic fibrosis (CF) program implemented a rapid and successful transition to telemedicine care mid-March of 2020 in response to the coronavirus disease 2019 (COVID-19) pandemic. In May 2020, the adult UVA CF program redesigned the care model to adjust to the reopening of ambulatory operations and introduced hybrid clinics. The goal remained to minimize person-to-person contacts for patients and care team members (CTMs) while ensuring patient access to quarterly, coproduced, synchronous, multidisciplinary CF care, similar to pre-COVID-19 era regular CF care. Methods: Using quality improvement tools, the UVA adult CF program created a standardized hybrid model of care for in-clinic visits, which included combined components of in-person and synchronous virtual interactions with members of the multidisciplinary team. Results: A total of 16 hybrid visits occurred between May 14 and June 11, 2020. All hybrid visits were multidisciplinary and fulfilled patient requests to see CTMs. All patients seen by hybrid encounter participated in coproduced agenda setting, underwent spirometry, and obtained blood work; 75% provided sputum for surveillance culture. Each hybrid visit type was attended by an average of four CTMs and amounted to 63 separate interactions. Of these interactions, 28 were completed virtually, reducing in-person contacts and personal protection equipment utilization by 44% compared with a fully in-person model of care. Conclusions: Combining in-person and telehealth components in a multidisciplinary CF care model reduces patient and staff interactions and personal protective equipment utilization. The hybrid model of in-person/remote combined care enables reliable access to biological data to support medical decision making while mitigating the risks of person-to-person contact for patients and staff.

Sleep screening for cystic fibrosis patients: A survey of cystic fibrosis programs.

The prevalence of sleep disorders in patients with cystic fibrosis (CF) is unknown, and no standardized screening or treatment guidelines exist to address sleep disorders in CF. The objective of this study is to characterize current sleep screening practices in adult, pediatric, combined, and affiliate CF programs. A survey was developed in Research Electronic Data Capture and distributed to program directors of accredited CF programs in the United States. Eighty-eight program directors responded (36% adult, 43% pediatric, 16% combined, 3% affiliate, and 1% unidentified). Of the respondents, 68% were part of an academic institution, 24% were associated with an academic institution, and 8% were part of a community or private program. Program sizes ranged from less than 50 to more than 500 patients. Routine or informal sleep screening was not performed in 44% of adults, 29% of pediatricians, and 35% of combined and affiliate programs. Most programs (>80%) have access to otolaryngology and sleep medicine although not all of these programs refer patients for evaluation of sleep disorders. Most program directors (77%) perceive sleep disorders as a problem in CF and would recommend routine sleep screening. Possible barriers to sleep screening included clinic flow, screening fatigue, and lack of recommendations for sleep screening. Formal sleep screening is inconsistent among CF care centers although most survey respondents would recommend the inclusion of screening in routine CF care. Future work is needed to further evaluate the impact of sleep disorders in CF and determine best practices for standardization of sleep screening and treatment.

Editorial introductions

Editorial introductions

Contraceptive use among women with cystic fibrosis: A pilot study linking reproductive health questions to the Cystic Fibrosis Foundation National Patient Registry

ObjectivesTo examine contraceptive use, pregnancy intention and the association of hormonal contraceptive type with adverse health outcomes among women with cystic fibrosis (CF). Study designWe recruited 150 women with CF, ages 18–49 from three adult CF programs to complete an online survey regarding their pregnancy and contraceptive use history. Survey findings were merged with retrospective clinical information from the CF Foundation Patient Registry (CFFPR). We used descriptive analyses to report contraceptive method and pregnancy frequencies, and logistic regression to examine the association between contraceptive method type and adverse health outcomes. ResultsCombined hormonal contraceptives were the most commonly used methods (42%), followed by condoms (34%), and long-acting reversible contraceptives methods (27%). Thirty-three percent (n = 50) reported ever being pregnant, half of whom reported having at least one unplanned pregnancy. We found no significant association for mucoid Pseudomonas aeruginosa infection among progestin-only (aOR 1.53, 95% CI 0.07–32.2) and estrogen-containing hormonal contraceptive users (aOR 3.9, 95 % CI 0.20–76.5). Risk of osteoporosis was elevated among women with CF who used depot-medroxyprogesterone acetate compared to non-users (OR 5.36, 95% CI 1.00–29.12). ConclusionsBoth contraceptive use and unplanned pregnancy among women with CF are common. Associations between hormonal contraceptive use and adverse pulmonary or bone outcomes among women with CF are inconclusive due to the study s small sample size. Larger studies are warranted. ImplicationsWomen with CF should be informed about the risks and benefits of contraceptives in the context of their disease. CFFPR data capturing contraceptive method use may be the most efficient way to elucidate the association of hormonal contraceptives on disease in women with CF.

PREVALENCE OF RESTLESS LEGS SYNDROME IN CYSTIC FIBROSIS IN AN ACADEMIC ADULT CYSTIC FIBROSIS PROGRAM

SESSION TITLE: Wednesday Abstract Posters SESSION TYPE: Original Investigation Posters PRESENTED ON: 10/23/2019 09:45 AM - 10:45 AM PURPOSE: Restless Legs Syndrome (RLS) is a clinical syndrome that affects approximately 10% of the general population. It is more prevalent in patients with advanced age, renal failure, pregnancy, diabetes mellitus, medications, myopathy, neuropathy, rheumatoid arthritis, and iron deficiency anemia. Increased awareness of RLS in patients with these disorders has led to more timely treatment. However, the prevalence of RLS in patients with Cystic Fibrosis (CF) has not been fully investigated. The objective of the study was to determine the prevalence of RLS symptoms in patients with CF. We also evaluate the impact of this disorder on quality of life via RLS Quality of Life (QoL) survey and severity of symptoms via an International RLS Study Group (IRLS) Rating Scale. METHODS: This research project was done via prospective survey and chart review. Previously validated RLS survey tools were used to measure the presence of RLS. A one-page survey featuring the core symptoms of RLS was administered to 77 subjects with CF in an academic adult CF program. RLS herald symptoms as agreed upon by the IRLS were incorporated as 4 key questions addressing RLS. This survey was considered positive for RLS if 4 of 4 core symptoms were present and those patients were subsequently administered the IRLS Rating Scale and RLS QoL questionnaire. RESULTS: Results of our study show RLS symptoms prevalence of 27% in subjects with CF. Average calculated score on IRLS Rating Scale was 16, suggesting moderate severity. RLS QoL questionnaire, reveals that RLS affects concentration , social activity, sexual health and work productivity in this population. CONCLUSIONS: The association of RLS symptoms and CF has not been well defined. Our study suggests that RLS symptoms are prevalent in CF and affects their quality of life. The pathophysiology of this relationship has not been fully described, however it may involve iron deficiency anemia from chronic inflammation, hemoptysis, gastrointestinal blood loss or iron malabsorption. Additionally, colonization with Pseudomonas Aeruginosa can result in iron deficiency as it competes with its host for iron in creating its own biofilm. Furthermore, medication toxicity, renal failure and diabetes mellitus in this patient population can also be contributing factors. Further investigations are being done to better understand this association. CLINICAL IMPLICATIONS: CF patients, over time, may suffer from significant fatigue mainly because of their advancing lung disease. However, heightened awareness of other contributing factors such as RLS can aid in timely treatment and improving their overall quality of life. DISCLOSURES: No relevant relationships by Rose Franco, source=Web Response No relevant relationships by Jeanette Graf, source=Web Response No relevant relationships by Erin Hubertz, source=Web Response No relevant relationships by Dmitriy Kogan, source=Web Response No relevant relationships by Jatan Shah, source=Web Response

Fertility considerations and attitudes about family planning among women with cystic fibrosis

ObjectiveOur objective was to explore knowledge, attitudes and beliefs about pregnancy and contraceptive decision-making among reproductive-aged women with cystic fibrosis. Study designIn this qualitative study, we used purposive sampling of reproductive age women enrolled in the Adult Cystic Fibrosis program at Penn Medicine. We conducted semistructured one-on-one interviews lasting 30–60 min. Interviews were audio-recorded and transcribed verbatim. Data were analyzed using thematic analysis. ResultsWe interviewed 24 women. Participants described shortened life span, personal health status and impact of cystic fibrosis on a potential child as three factors that were central to family planning. Future pregnancy intentions, perception of fertility including misconceptions and provider counseling influenced related contraception utilization. ConclusionsWomen with cystic fibrosis described thoughtful considerations about desired pregnancies to be planned during times of optimal health. Gaps in knowledge may limit a woman’s ability to make the most informed family planning decisions. ImplicationsHaving cystic fibrosis directly affects women’s decisions around pregnancy and family planning. Understanding what motivates women with cystic fibrosis to use or not to use contraception can improve family planning care delivery to this population.

Corticosteroid use and increased CXCR2 levels on leukocytes are associated with lumacaftor/ivacaftor discontinuation in cystic fibrosis patients homozygous for the F508del CFTR mutation.

Cystic fibrosis (CF) is the most common life-shortening genetic disease and is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Several current therapies aim at improving availability and/or function of the mutant CFTR proteins. The combination therapeutic lumacaftor/ivacaftor (Orkambi, luma/iva) partially corrects folding and potentiates CFTR function impaired by the F508del mutation. Despite the potential for clinical benefit, a substantial number of patients discontinue treatment due to intolerable adverse effects. The aim of the present study is to identify differences between individuals who continued treatment and those who discontinued due to adverse respiratory effects to potentially inform treatment decisions. Clinical data from the year prior to treatment initiation were analyzed from 82 patients homozygous for the F508del mutation treated at the Colorado Adult CF Program. Blood samples were collected from 30 of these subjects before initiation of treatment to examine expression of circulating leukocyte surface antigens and cytokines. Clinical and demographic characteristics were analyzed along with inflammatory markers to determine biomarkers of drug discontinuation. The use of oral prednisone and/or nasal budesonide in the year prior to luma/iva initiation was more prevalent in CF subjects who did not tolerate luma/iva (82% vs. 43%). Increased age, but not gender or initial lung function, was associated with higher probability of discontinuing treatment due to side effects overall. Worse lung function (lower ppFEV1, ppFEF25-75 ≤ 60%) was associated with higher incidence of discontinuing treatment due to pulmonary adverse effects. In a nested cohort of patients, increased surface levels of CXCR2 on CD14+CD16- monocytes were associated with discontinuation. Overall, the patients who tolerated luma/iva were distinguishable from those who did not tolerate the drug based on clinical and cellular markers obtained prior to treatment initiation.

Clinical Model of Exercise-Related Dyspnea in Adult Patients With Cystic Fibrosis

Dyspnea is a highly distressing symptom of pulmonary disease that can make performing physical activities challenging. However, little is known regarding the strongest predictors of exercise-related dyspnea in adult cystic fibrosis (CF). Therefore, the purpose of the present study was to determine the best clinical model of exercise-related dyspnea in this patient group. A retrospective analysis of pulmonary function and cardiopulmonary exercise testing data from patients with CF being followed up at the Adult CF Program at St Michael's Hospital, Toronto, Canada, from 2002 to 2008 were used for the analysis. Patients (n = 88) were male 66%; aged 30.4 ± 9.4 years; body mass index (BMI) 23.1 ± 3.3 kg/m; forced expiratory volume in 1 second (FEV1) 70% ± 19% predicted; and peak oxygen uptake 74% ± 20% predicted. A multivariate linear regression model assessing the effects of age, sex, BMI, airway obstruction (FEV1), perceived muscular leg fatigue, and dynamic hyperinflation explained 54% of the variance in dyspnea severity at peak exercise (P < .01). Relative importance analysis showed that the presence of dynamic hyperinflation and perceived muscular leg fatigue were the largest contributors. Pulmonary rehabilitation programs may consider strategies to reduce dynamic hyperinflation and promote muscular function to best improve exercise-related dyspnea in this patient group.

Risk Factors for Gaps in Care during Transfer from Pediatric to Adult Cystic Fibrosis Programs in the United States.

With improved survival into adulthood, the number of dedicated adult cystic fibrosis (CF) care programs has expanded in the United States over the past decade. Transfer from pediatric to adult CF programs represents a potential time for lapses in recommended health care. To describe variability in transfer between pediatric and adult CF care programs and to identify factors associated with prolonged gaps in care. Using the U.S. CF Foundation Patient Registry, we identified individuals with CF who transferred care from a pediatric to an adult CF care program during 2007 to 2013. A gap in care was defined as the time in days between the last recorded pediatric encounter and the first recorded adult encounter. A hierarchical multivariable regression model was applied to investigate the effect of program- and patient-level factors on gaps in care. There were 1,946 individuals at 155 pediatric CF programs who transferred to an adult CF program during the analytic period. The mean age at transfer was 21.1 years, with 68% transferring care between ages 18 and 21 years. The mean gap in care during transfer was 183 days (median, 106 d; range, 2-1,843 d); 47% had a less than 100-day gap, and 13% had a greater than or equal to 365-day gap (prolonged gap). Prolonged gaps in care were more likely to occur among those younger than age 18 years (odds ratio, 3.33; 95% confidence interval, 2.06-5.37) at the time of transfer and those who transferred to an adult program that was in a different city from their pediatric or affiliate program (odds ratio, 2.16; 95% confidence interval, 1.48-3.17). Having any health insurance coverage was associated with decreased likelihood of prolonged gaps (private insurance vs. no insurance [odds ratio, 0.15; 95% confidence interval, 0.09-0.23] or any government insurance versus no insurance [odds ratio, 0.11; 95% confidence interval, 0.07-0.18]). Lung function, nutritional status, and receipt of intravenous antibiotics in the final year of pediatric care were not associated with the length of gap in care during transfer. In the United States, the majority of individuals transferring from pediatric to adult CF care do so between ages 18 and 21 years. A minority had a gap greater than or equal to 365 days during transfer to adult care, suggesting that most did not have a disruption in recommended quarterly care visits during the transfer period. Risk factors for prolonged gaps in care include younger age at transfer, lack of health insurance, and relocation. Care coordination during transition in CF may minimize lapses in care by identifying and more closely addressing the needs of individuals at highest risk.

Contraceptive choices and preferences in a cohort of women with cystic fibrosis

ObjectivesAs life expectancy for women with cystic fibrosis (CF) improves, reproductive decision-making is becoming increasingly relevant. We sought to characterize fertility intentions, contraceptive use, and preferences among adult females with CF. Study designWe conducted a cross-sectional survey of contraceptive use and preferences in reproductive-aged women with CF. Subjects were recruited via an electronic mailing list from the Penn Adult Cystic Fibrosis Program. ResultsFifty-three women with CF responded and met eligibility criteria. Most (83%) were sexually active, however only 49% used contraception. Condoms and the oral contraceptive pill were the most commonly used methods. Compared to the national contraceptive use among Caucasian women (65%), use in our cohort was significantly lower (p = 0.01). Participants prioritized effectiveness and ease of use in their contraceptive method; they wanted to avoid side effects and interference with sexual enjoyment. Although these priorities are similar to Caucasian women in the literature, additional contraceptive priorities differed significantly between the groups. ConclusionsThis CF cohort is sexually active but contraceptive utilization was low, and women with CF identify specific contraceptive preferences. There is a significant unmet need for contraception among women with CF.

Provider and Patient Attitudes Regarding Sexual Health in Young Women With Cystic Fibrosis.

To explore the attitudes, preferences, and experiences of patients with cystic fibrosis (CF) and CF providers toward sexual and reproductive health (SRH) care for young women with CF. Young women with CF aged 18 to 30 years from a US CF care center and pediatric and adult CF program directors from a national sample participated in qualitative interviews investigating their experiences regarding SRH care and their attitudes and preferences toward SRH care provision in the CF setting. Interviews were audio-recorded, transcribed, and coded by using a thematic analysis approach. Twenty-two patient participants and 16 CF program directors were interviewed. Themes shared by both groups included the importance of SRH discussion in the CF care setting, patient and provider discomfort as a barrier to SRH care, and the need for SRH educational resources and provider training to improve SRH care. Providers highlighted the lack of standardization around SRH care in the current CF care model. Patients desired SRH educational resources coupled with early SRH discussions initiated by their CF provider. Both CF providers and patients agree that the CF provider has a fundamental role in providing CF-specific SRH care. Educational resources coupled with individualized SRH discussions may facilitate improved SRH care for young women with CF. Investigation into the implementation of SRH education and services into pediatric-onset chronic disease care models is needed.

Clinical Outcomes and Prognostic Factors in a Cohort of Adults With Cystic Fibrosis: A 7-Year Follow-Up Study.

Due to the heterogeneity of cystic fibrosis (CF), the longer survival observed in cohorts of adult subjects, and inter-population variations, there is a clear need to seek further information about clinical outcomes and prognostic factors in different cohorts of subjects with CF. Our objectives were to evaluate clinical outcomes and prognostic factors in a cohort of adult subjects with CF after a 7-y follow-up period and investigate longitudinal changes in clinical scores, spirometry, 6-min walk test performance, and pulmonary artery systolic pressure as assessed by Doppler echocardiography. A cohort of clinically stable subjects (≥16 y old) who were enrolled in an adult CF program in 2004-2005 underwent clinical evaluation. Outcome was classified as good (survival) or poor (survival with lung transplantation or death). In 2011-2012, survivors were re-examined. Of 40 subjects with CF evaluated in 2004-2005, 32 (80%) survived, 2 (5%) survived with lung transplantation, and 6 (15%) died. Logistic regression analysis showed that a low percent-of-predicted FEV1 was associated with poor outcome. An FEV1 cut-off value of ≤30% and pulmonary artery systolic pressure of ≥42 mm Hg predicted poor outcome with high sensitivity, specificity, and positive and negative predictive values. Deterioration was observed in clinical scores (P = .03), FVC (P = .02), FEV1 (P < .001), distance walked in the 6-min walk test (P = .002), baseline SpO2 (P < .001), and final SpO2 (P < .001). After 7 y of follow-up, 20% of subjects with CF had a poor outcome. Pulmonary artery systolic pressure of ≥42 mm Hg and FEV1 of ≤30% were the most significant prognostic predictors of poor outcome. Clinical and functional deterioration was observed in survivors.

Contraceptive Needs and Preferences in a Cohort of Women With Cystic Fibrosis [209

INTRODUCTION: As the median age of survival of women with cystic fibrosis rises, reproductive health has become increasingly important in this population. We sought to characterize contraceptive preferences and use among adult females with cystic fibrosis (CF). METHODS: We used questions from previously validated surveys to build an instrument that assessed reproductive health and contraceptive variables. Female participants ages 17–45 years were recruited through the electronic mailing list of the Penn Adult CF Program. RESULTS: Fifty-three of 100 adult female patients with CF have responded to date. Eighty-three percent are sexually active and 31% have been pregnant. Twenty-two percent of reported pregnancies resulted in abortion, secondary to either unplanned pregnancy or suboptimal health status. Forty-nine percent of participants use contraception. Condoms (35%) and oral contraceptive pills (27%) are most commonly used. Respondents prioritize effectiveness and ease of use in their contraceptive method; they want to avoid side effects, medication interactions, and diminished sexual enjoyment. CONCLUSION: Our CF population is sexually active, but contraceptive use is low and unplanned and mistimed pregnancies are common. Uptake of contraceptive methods that fit these women's preferences is low. There is significant unmet need for contraception in this population.