Cystic Fibrosis Healthcare Research Articles

Concern over tobacco and marijuana perceptions and use among adolescents and young adults with cystic fibrosis

BackgroundThis study assesses use and perceptions of short- and long-term harms associated with cigarettes, e-cigarettes, and smoked marijuana among adolescents and young adults (AYAs) with cystic fibrosis (CF). MethodsA total of 205 AYAs with CF completed an online survey querying about use, safety perceptions, and education related to traditional cigarettes, electronic cigarettes (e-cigarettes), and smoked marijuana. In addition, parents of AYAs with CF and CF healthcare providers were asked questions about experiences in avoidance education. ResultsAYA participants with CF reported using tobacco and marijuana at rates lower than that of the general AYA population, with heavy use considerably lower in this population. AYAs with CF perceived lower risk of negative outcomes associated with using e-cigarettes and smoked marijuana compared to combustible cigarettes. Ever-use was correlated with a lower perception of risk across all products. CF providers estimated lower rates of product use in their own patients compared to both the general AYA CF population and the general AYA population, and estimated lower use among the general CF AYA population compared to the general AYA population. Receipt of avoidance education varied greatly when comparing AYAs with CF, parents of individuals with CF, and CF healthcare providers. Reasons for undereducation include but are not limited to lack of familiarity with products, assumption of avoidance, assumption of education, and time constraints. ConclusionsFindings concerning safety perceptions and use of combustible tobacco, e-cigarettes, and marijuana in individuals with cystic fibrosis underscore the importance of providing avoidance education to vulnerable patient populations. Insight derived from this study may also inform pediatric to adult clinic transition education, when chronic disease populations are at greatest risk for engaging in risky behaviors. Implications and contributionWe report data on use, risk perception, and education of cigarettes, electronic cigarettes, and cannabis in individuals with cystic fibrosis, with a focus on adolescents and young adults. Such Findings will inform prevention education, especially during the critical transition period from pediatric to adult care when these behaviors are prevalent.

Economic burden of cystic fibrosis care in British Columbia

RATIONALE: Cystic fibrosis (CF) is a progressive multi-organ disease with significant morbidity placing extensive demands on the health care system. No recent estimate of CF health care costs in a Canadian context exists. OBJECTIVES: Provide up-to-date direct cost estimates of the economic burden of CF from the perspective of a Canadian health care system. METHODS: A longitudinal study of retrospective data was completed in British Columbia by linking the Canadian CF Registry with provincial health care administrative databases for the period between 2007 and 2017. MEASUREMENTS: Health care spending (in constant 2017 Canadian dollars) for CF outpatient services, inpatient hospitalizations, medications, and emergency department visits were included. Generalized estimating equations approach for repeated annual cost data over time was used to estimate health care costs. MAIN RESULTS: The overall total direct annual health care spending for CF care in BC increased from $10.6M to $17.2M from 2007 to 2017 ($2017, mean costs per CF patient increased from $31.7K to $42.2K; Cost ratio: 1.33, 95%CI: 1.12-1.57). Outpatient medications, inpatient hospitalizations, outpatient services, and emergency department visits were responsible for 56%, 36%, 7.4% and 0.5% of total costs, respectively. CF adults also showed substantial increases in costs over time (Cost ratio from 2017 relative to 2007: 1.61, 95%CI: 1.17-2.22). CONCLUSIONS: The economic burden of CF increased from 2007 to 2017 driven by rising outpatient medication costs primarily in adults with CF. With approximately 75% of the population eligible for highly effectively cystic fibrosis transmembrane conductance regulator (CFTR) modulators, the economic burden of CF is expected to increase at unprecedented rates.

Provider Perspectives on Fertility and Fertility Preservation Discussions Among Women With Cystic Fibrosis.

Cystic Fibrosis (CF) is a genetic disorder that primarily impacts the respiratory and gastrointestinal systems. With advances in treatment and medications, the life expectancy of people with CF is continuing to increase with current life expectancy at 47 years of age. Given the increased life expectancy, people with CF are interested in childbearing but may have CF specific fertility issues that should be addressed by their CF healthcare provider. Currently, these conversations are not occurring or are suboptimal. This study aimed to investigate the practices of cystic fibrosis (CF) healthcare providers regarding fertility and fertility preservation (FP) discussions among women with CF. This was a qualitative, descriptive study. Twenty CF healthcare providers were interviewed including nurses, nurse practitioners, social workers, and dieticians among other disciplines. The semi-structured interviews were audio-recorded, transcribed verbatim, and analyzed using thematic analysis. Four themes emerged from the provider interviews about their fertility and FP discussion practices: (1) Change over Time; (2) CF Team Provides Primary Care including Reproductive Health Care; (3) Patient Support and Advocacy; and (4) Barriers and Facilitators to FP Discussions. The results of this study demonstrate an opportunity for CF healthcare providers to deliver patient-centered care. However, CF providers need to be educated on fertility and FP options. Additionally, there is a need for a more standardized structure of care to address the reproductive health of women with CF. The findings from this study may also be useful to non-CF providers who deliver care to women whose chronic illnesses impact their reproductive health.

Impact of Digital Technologies on Clinical Care for Adults with Cystic Fibrosis.

The coronavirus disease 2019 pandemic accelerated the implementation of digital technologies, which have now become embedded as essential tools for the management of chronic disease, including cystic fibrosis (CF). Despite subsequent easing of restrictions and because of improved clinical stability resulting from the introduction of highly effective modulator therapy, digital technologies including video and telephone consultations and remote monitoring are likely to remain integral to the future delivery of CF health care. In this article, we explore some of the key developments in digital technologies, barriers to their adoption, and how the CF community is likely to embrace lessons learned from the recent pandemic to help modernize and reshape the future of CF care.

Clinician perspectives on assessing for disordered eating and body image disturbance in adolescents and young adults with cystic fibrosis

BackgroundMaintaining a healthy weight is a focus of Cystic Fibrosis (CF) care. With the increased use of highly effective CFTR modulators, many people with CF are gaining weight more easily, which may affect eating habits and body image. This study investigates providers’ understanding and current practices surrounding body image disturbance and disordered eating in people with CF. MethodsWe distributed a one-time web-based survey to United States (U.S.)-based CF healthcare providers via CF Foundation list servs. The survey investigated providers’ understanding and perceived importance of issues surrounding disordered eating and body image disturbance in adolescent and young adults (AYA) with CF as well as current screening practices. We used descriptive statistics to analyze participants’ characteristics and practices. ResultsA total of 232 healthcare providers completed the survey. While most participants felt that screening for both body image disturbance and disordered eating should be standardized in CF care (79% and 82%, respectively), fewer than one third felt comfortable screening, and only one quarter actually screened for various eating disordered behaviors in daily practice. Only 2.7% reported using a formal screening tool. Participants reported provider assessment tools (86%), standardized partnerships with eating disorder specialists (80%), and CFF or national guidelines (79%) would be helpful to improve screening and counseling. ConclusionWhile most CF providers believe that body image disturbance and disordered eating are important topics in AYA with CF, few address these topics with their patients. The development of educational sessions and national guidelines may improve screening and counseling practices.

Living with cystic fibrosis during the COVID-19 pandemic: a social connectedness perspective

ABSTRACT Purpose This study explores the concept of social connectedness for adults with Cystic Fibrosis (CF), generally and during the onset of the COVID-19 pandemic, to help inform contemporary CF healthcare. Social connectedness is an essential component of belonging and refers to an individual’s sense of closeness with the social world. Unique disease factors make exploration of social connectedness pertinent, added to by COVID-19, with the CF population potentially facing increased risk for severe illness. Methods Seventeen adults with CF in Western Australia undertook interviews, with findings categorized as overarching themes. Results In a general sense, participants described social connectedness challenges caused by CF, despite which they reported meaningful connections that benefits their mental and physical health. Within a COVID-19 specific context, participants demonstrated resilience in the face of adversity, highlighted the importance of empathy in relation to the pandemic, and described how social support is both an outcome and enhancer of social connectedness. Conclusions This study contributes to limited social connectedness literature within CF and chronic illness in general, highlighting the importance of social connectedness awareness raising, assessments and interventions in CF healthcare inside and outside the COVID-19 pandemic.

Improved survival albeit with persistent disparities in prognosis for people with cystic fibrosis in European countries

Cystic fibrosis (CF), an autosomal recessive disease related to mutations in the gene encoding for the cystic fibrosis transmembrane conductance regulator (CFTR) protein, is the most prevalent severe genetic disease in Caucasian populations [1]. Although the disease affects multiple organs, respiratory disease is the major manifestation of CF and is often responsible for premature death [2], which often occurred in the first years of life before the implementation of modern CF care [3]. Over the past decades, progress in therapeutic management has resulted in a dramatic improvement in the prognosis of people with CF [4]. In a study published more than 20 years ago, Fogarty et al . [5] compared median age at death in seven countries (USA, and Australasian and European countries) and reported that median age at death improved from 8 years in 1974 to 21 years in 1994, albeit with marked differences between countries. More recent studies have confirmed improvement in median age of survival, which was 40.6 years in US patients and 50.9 years in Canadian patients, in a study that used data from between 2009 and 2013 [6]. In European countries, contemporary studies have reported increases in the number of adults with CF [7, 8], with a better prognosis in countries belonging to the European Union (EU) than in those outside of the EU [7, 8], mostly located in Eastern Europe, in which access to appropriate CF care is less available [9]. Collectively these data suggest that prognosis improvement in patients with CF is heavily dependent on the quality of the CF healthcare system. Survival is improving for people with cystic fibrosis in European countries albeit with persistent disparities in prognosis among countries <https://bit.ly/34GF3SA>

A doggy tale: Risk of zoonotic infection with Bordetella bronchiseptica for cystic fibrosis (CF) patients from live licenced bacterial veterinary vaccines for cats and dogs.

Live-attenuated bacterial veterinary vaccines can constitute an infection risk for individuals with any defect in their phagocytic function, including chronic granulomatous disease, leukocyte adhesion deficiency, myeloperoxidase deficiency, as well as Chediak-Higashi syndrome, from accidental acquisition of licenced attenuated live bacterial vaccine, at vaccination or from their vaccinated pet. Ownership of small companion animals, including cats and dogs, is popular within the cystic fibrosis (CF) community. These animals require vaccines as part of their routine care, which may involve live viral and bacterial vaccines, with potential for infection in the CF owner. This report examines the scope of current canine and feline vaccines, with particular emphasis on veterinary vaccination strategies against the Gram-negative pathogen, Bordetella bronchiseptica and describes new vaccine innovations offering protection to both pet and CF owner. The Gram-negative bacterium, Bordetella bronchoseptica, may cause respiratory disease in small companion animals, as well as in certain human vulnerable groups, including those with CF. Live licenced veterinary bacterial vaccines for Bordetella bronchiseptica (Kennel Cough) are available for cats and dogs, which are an infection concern for humans with CF who may come into contact with vaccinated animals. Live licenced veterinary bacterial vaccines for Bordetella bronchiseptica (Kennel Cough) are available for intranasal administration to cats and dogs. These vaccines require a withdrawal period of vaccinated animal from vulnerable owner, ranging from 35days - 11weeks. Recently, a new dead IM vaccine is now available not requiring exclusion of the vaccinated pet from CF owner. CF pharmacists, hospital pharmacists and community pharmacists are important custodians of vaccine-related advice to people with CF, who are frequently consulted for such advice. Pharmacists should be aware of the recent innovations in veterinary medicines, so that they can give appropriate advice to people with CF when asked. Immunocompromised patients, that is those with CF or those with any defect in their phagocytic function (chronic granulomatous disease, leukocyte adhesion deficiency, myeloperoxidase deficiency, Chediak-Higashi syndrome) should avoid exposure to live veterinary bacterial vaccines and seek animal vaccination utilising non-live vaccines. Most importantly, this manuscript highlights the development of a new veterinary vaccine for dogs, which we want to make the CF healthcare community aware of, which is an acellular dead vaccine, so that those patients with dogs needing annual vaccination can select this vaccine pathway, thereby minimising risk of infection from the vaccine strains and avoiding the social exclusion between CF patient and their pet. CF patients should understand the potential infection implications of live-attenuated viral and bacterial strains as vaccines, whether these are small companion animals, exotic animals or large farm animals. Patients should make their veterinarian aware of their CF status, so that a safe and efficacious vaccine strategy is used, both mitigating the potential infection risks from live vaccine components with the CF patient, but simultaneously offering maximum immunological protection to the animal.

Development and evaluation of an internet-based cognitive behavioral therapy intervention for anxiety and depression in adults with cystic fibrosis (eHealth CF-CBT): An international collaboration

IntroductionIndividuals with cystic fibrosis (CF) are at increased risk for anxiety and depression, with negative consequences for adherence, health, and quality of life. New approaches to prevent and treat anxiety and depression that are tailored to the concerns of this population are needed. A CF-specific internet-based cognitive-behavioral therapy (CBT) intervention was developed to increase access to evidence-based mental health care and decrease cost and burden of care for people with CF. ObjectiveTo evaluate the usability and acceptability of “eHealth CF-CBT,” an internet-based program integrating therapist-guided online self-management modules with in-person or virtual sessions. MethodsDutch adults with CF (N = 16) and CF health care providers (N = 16) systematically tested all sessions of the eHealth CF-CBT program, provided qualitative feedback, and completed measures including eHealth Impact Questionnaire (eHIQ), and System Usability Scale (SUS). ResultsPatient and provider ratings of their overall impression of the eHealth CF-CBT program were high, with scores (mean; SD) of 8.3; 0.6 and 8.2; 0.8 respectively on a 10-point scale. Mean ratings of usability by patients (77.0/100) and providers (73.4/100) surpassed the SUS cut point for good favorability. Ratings (pooled mean; SD) on the assessed eHIQ domains Motivation and Confidence to Act (71.3; 10.0), Information and Presentation (78.9; 9.6), and Identification (62.0; 15.1) were positive, as were assessments of specific elements of session content and format. ConclusionseHealth CF-CBT is the first therapist-guided internet-delivered CBT-based intervention for adults with CF. Initial evaluation with key stakeholders demonstrated high levels of acceptability and usability and provided input that was integrated to strengthen the program. Effectiveness testing in the Netherlands will be the next step, as well as future international adaptation and dissemination.

Real-World Safety of CFTR Modulators in the Treatment of Cystic Fibrosis: A Systematic Review.

Cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies target the underlying cause of cystic fibrosis (CF), and are generally well-tolerated; however, real-world studies indicate the frequency of discontinuation and adverse events (AEs) may be higher than what was observed in clinical trials. The objectives of this systematic review were to summarize real-world AEs reported for market-available CFTR modulators (i.e., ivacaftor (IVA), lumacaftor/ivacaftor (LUM/IVA), tezacaftor/ivacaftor (TEZ/IVA), and elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA)), and to identify ways in which the pharmacist on CF healthcare teams may contribute to mitigating and managing these AEs. The MEDLINE, EMBASE, CINAHL, and Web of Science Core Collection online databases were searched from 2012 to 1 Aug 2020. Full manuscripts or conference abstracts of observational studies, case series, and case reports were eligible for inclusion. The included full manuscripts and conference abstracts comprised of 54 observational studies, 5 case series, and 9 case reports. The types of AEs reported generally aligned with what have been observed in clinical trials. LUM/IVA was associated with a higher frequency of respiratory-related AE and discontinuation in real-world studies. A signal for mental health and neurocognitive AEs was identified with all 4 CFTR modulators. A systematic approach to monitoring for AEs in people with CF on CFTR modulators in the real-world setting is necessary to help better understand potential AEs, as well as patient characteristics that may be associated with higher risk of certain AEs. Pharmacists play a key role in the safe initiation and monitoring of people with CF on CFTR modulator therapies.

Human Papilloma Virus Vaccination Among Female Patients Attending French Pediatric Cystic Fibrosis Centers.

To describe human papilloma virus (HPV) vaccination practice among adolescent girls with cystic fibrosis (CF) and to identify reasons for non-vaccination. Cross-sectional multicentric study. Girls aged 9-17years, attending 7 French pediatric CF centers, and their accompanying adult. Administration of a self-report questionnaire. The proportion of girls having received or receiving HPV vaccination, compliance with the vaccination schedule, factors associated with vaccination, and reasons for vaccination and for non-vaccination. A total of 113 girls and 104 accompanying adults participated. The mean age was 13.6years (standard deviation 2.5; range 9-17). A total of 34 (30.9%) patients reported having received HPV vaccination. Among the 34 girls aged 15years or older, 15 (44.1%) were vaccinated. Most patients (58.8%) started vaccination between 11 and 14years of age (mean age 13.9). Most vaccine prescriptions (67.6%) were made by a CF center health care provider. Factors associated with vaccination were older age (odds ratio [OR]=1.27, 95% confidence interval [CI]=1.01-1.6, P=.037 for each year older), previous vaccination by the accompanying parent of one of their children for hepatitis B (OR=8.01, 95% CI=0.96-67.02), P=.055), and parental influence on decision-making (OR=2.77, 95% CI=0.97-7.95, P=.058). Health care providers' positive advice and fear of HPV-related disease were the main reasons given to justify vaccination decisions. Insufficient knowledge and concerns about potential side effects were the main barriers. HPV vaccination remains insufficient among girls with CF. CF health care providers may play a crucial role in HPV vaccination acceptance, and their sensitization to cervical cancer prevention is mandatory.

Managing Cystic Fibrosis in Polish Healthcare.

The quality and length of life of patients with cystic fibrosis (CF) are determined by a number of factors including the quality of healthcare received by patients, as well as access to drug programs dedicated to this particular disease. The purpose of this paper is to present an overview of changes in the average life expectancy and mortality rate of the CF population in Poland between 2000 and 2018. Furthermore, we would like to evaluate access to healthcare services, including the drug program, guaranteed by public healthcare system, and funded by National Health Fund (NHF). The average life expectancy of patients with CF increased in the period in question from ca. 14.5 ± 7.6–24.5 ± 8.9 years (mean ± SD, p = 0.0001). We have observed a drop in the number of deaths in paediatric age during that period. Despite the increase in life expectancy, the use of health resources in patients with CF, especially the drug program, is dramatically low. Considering the fact that in Poland there was no active countrywide CF registry, now it is possible to estimate the frequency of use of CF healthcare services in various provinces exclusively on the basis of database maintained by the Polish NHF.

THE STATE OF FERTILITY PRESERVATION COUNSELING FOR WOMEN WITH CYSTIC FIBROSIS REPORTED BY CYSTIC FIBROSIS PROVIDERS

Fertility preservation (FP) counseling is part of comprehensive care provided to women of childbearing age undergoing oncologic treatments; however, this is not standard for patient populations with other chronic conditions. The purpose of this study was to examine the current state of FP counseling for women with cystic fibrosis (CF) from the perspective of their healthcare providers, with the goal of identifying the gaps, barriers, and facilitators to implementation of counseling in clinical practice. This knowledge can lead to the development of FP guidelines for women with CF and enhance shared decision making by broadening their reproductive options. This study utilized a quantitative, exploratory, cross-sectional design. Healthcare providers (e.g., physicians, nurse practitioners, clinic coordinators, pharmacists, social workers, nurses) were recruited from CF Foundation accredited clinics throughout the United States and via snowball sampling. Participants completed a web-based, anonymous FP survey with 39 items that took approximately 30 minutes to complete. Participants received $20 for their participation. The descriptive statistics were provided using R software. Fifty healthcare providers completed the survey (Mage= 40.8, SD=11.1). The majority of the healthcare providers were female (n=45). Despite the fact that 92% (n=46) of the providers felt that FP discussions should be standard for all female patients with CF, 28% (n=14) of providers reported that FP topics were not discussed with female patients with CF at any age. Most providers felt that initiation of FP discussions was the responsibility of the healthcare team (n=34), but some felt that patients should initiate the FP discussions (n=11). Lack of knowledge (n=17) and lack of time (n=12) were the two most substantial barriers to FP discussions by healthcare providers. According to the providers, the two best facilitators for FP discussions in clinic would be having national guidelines or standards (n=41) and integrating partnerships with sexual and reproductive health specialists (n=39) as part of comprehensive CF care delivery. Having educational materials to foster FP discussions between providers and patients was another facilitator reported (n=50). Healthcare providers preferred having educational information and resources shared via online format (n=46) and written pamphlets (n=44). Fertility preservation counseling is an important part of comprehensive care for women with CF; however, many women do not receive FP information. This national sample of CF healthcare providers reported that they lacked the knowledge and time to discuss FP with their patients. Removing the barriers and optimizing the facilitators to encourage FP discussions between women with CF and their providers will broaden these women’s reproductive options by facilitating fully informed decision making to improve overall health outcomes.

Projecting the impact of delayed access to elexacaftor/tezacaftor/ivacaftor for people with Cystic Fibrosis

BackgroundTherapies that target the underlying defect in Cystic Fibrosis (CF) will likely impact the future characteristics of the CF population and healthcare utilization. The objectives of this study were to estimate the potential impact of elexacaftor/tezacaftor/ivacaftor on morbidity and mortality, and the impact of delayed access. MethodA microsimulation transition model was applied to Canadian CF Registry data to forecast lung disease severity, pulmonary exacerbations, deaths and transplants to 2030 under three scenarios: 1) no availability of elexacaftor/tezacaftor/ivacaftor, 2) availability in 2021 (‘early’) or 3) availability in 2025 (‘delayed’). Published Phase III data on treatment effects were used to estimate transition rates between disease severity states. ResultsUnder specific assumptions regarding disease state and treatment effect applied to the Canadian CF population it is projected that by 2030, early introduction of elexacaftor/tezacaftor/ivacaftor is expected to reduce the number of individuals with severe lung disease by 60% (95% CI 55.3; 63.9), increase the number of individuals with mild lung disease by 18% (95%CI 18.2; 19.0) and reduce the number of pulmonary exacerbations by 19% (95%CI 18.9; 19.5). Earlier introduction of elexacaftor/tezacaftor/ivacaftor could reduce deaths by 15% (95% 13.2; 18.4) and improve the median age of survival by 9.2 years (7.5; 10.8) over a 10-year period. The expected benefits of therapy are cumulative, therefore delayed access to elexacaftor/tezacaftor/ivacaftor will result in preventable health care utilization and deaths. ConclusionsDelayed access to elexacaftor/tezacaftor/ivacaftor will have a negative impact on lung health and survival in the CF population.

The changing demography of the cystic fibrosis population: forecasting future numbers of adults in the UK

Improvements in management of cystic fibrosis (CF) through specialist centres in the UK have been associated with a step-change in life expectancy. With increasing numbers of adult patients there is a need to review health care provision to ensure it is sufficient to meet future needs. We used UK CF Registry data to project the number of patients aged 16–17 and 18 and older up to 2030, and numbers therefore requiring specialist adult CF care. Survival modelling was used to estimate age-specific mortality rates. New-diagnosis rates were estimated using diagnoses observed in the Registry and national population figures. Uncertainty in projections was captured through 95% prediction intervals (PI). The number of adults (aged 18 and older) is expected to increase by 28% from 6,225 in 2017 to 7,988 in 2030 (95% PI 7,803–8,169), assuming current mortality rates. If mortality rates improve at the rate seen over recent years, the projected number increases to 8,579 (95% PI 8,386–8,764). The age distribution is also expected to change, with 36% of CF adults being over 40 in 2030, versus 21% in 2017. There is an urgent requirement to review adult CF health care provision, due to both increasing numbers and the changing care needs of an older population.

Preface

An unintended consequence of cystic fibrosis (CF) newborn screening (NBS) is the identification of infants with a positive NBS test but inconclusive diagnostic testing. These infants are classified as CF transmembrane conductance regulator-related metabolic syndrome (CRMS) in the US and CF screen positive, inconclusive diagnosis (CFSPID) in other countries. Diagnostic and management decisions of these infants are challenges for CF healthcare professionals and stressful situations for families. As CF NBS has become more widespread across the world, increased information about the epidemiology and outcomes of these infants is becoming available. These data were reviewed at the 2015 CF Foundation Diagnosis Consensus Conference, and a harmonized definition of CRMS and CFSPID was developed.At the consensus conference, participants reviewed published and unpublished studies of CRMS/CFSPID and used a modified Delphi methodology to develop a harmonized approach to the definition of CRMS/CFSPID.Several studies of CRMS/CFSPID from populations around the world have been published in the past year. Although the studies vary in the number of infants studied, study design, and outcome measures, there have been some consistent findings. CRMS/CFSPID occurs relatively frequently, with CF:CRMS that ranges from 3 to 5 cases of CF for every 1 case of CRMS/CFSPID in regions where gene sequencing is not used. The incidence varies by NBS protocol used, and in some regions more cases of CRMS/CFSPID are detected than cases of CF. The majority of individuals with CRMS/CFSPID do not develop CF disease or progress to a diagnosis of CF. However, between 10% and 20% of asymptomatic infants can develop clinical features concerning for CF, such as a respiratory culture positive for Pseudomonas aeruginosa. Most studies have only reported short-term outcomes in the first 1-3 years of life; the long-term outcomes of CRMS/CFSPID remain unknown. The European CF Society definition of CFSPID and the CF Foundation definition of CRMS differ only slightly, and the consensus conference was able to create a unified definition of CRMS/CFSPID.CRMS/CFSPID is a relatively common outcome of CF NBS, and clinicians need to be prepared to counsel families whose NBS test falls into this classification. The vast majority of infants with CRMS/CFSPID will remain free from disease manifestations early in life. However, a small proportion may develop clinical features concerning for CF or demonstrate progression to a clinical phenotype compatible with a CF diagnosis, and their long-term outcomes are not known. A consistent international definition of CRMS/CFSPID will allow for better data collection for study of outcomes and result in improved patient care.

1078. Expectations and Attitudes Toward Antimicrobial Stewardship Among Cystic Fibrosis Care Providers

BackgroundTreatment of cystic fibrosis (CF) exacerbations can be challenging secondary to antimicrobial resistance due to chronic airway infection, multiple treatment courses, and frequent use of suppressive antibiotics. For these reasons, many antimicrobial stewardship (AMS) principles may not be practical for the CF population. The objective of this study was to determine perceptions of AMS among CF healthcare providers internationally.MethodsSix questions regarding AMS were incorporated into an email survey focusing on antimicrobial resistance in CF. Healthcare providers (HCP) were identified through list-servs and CF-related organizations internationally.ResultsThree-hundred and seventy-eight HCP from 30 countries responded to the survey (see Figure 1). Within their institutions, more than half had access to a CF-specific pharmacist, infectious disease consultation, and/or written CF exacerbation guidelines. An AMS program was only available for 39% of respondents. Most HCP stated that choosing and dosing antibiotics correctly and minimizing resistance were the main goals of AMS. Stewardship activities they felt would be helpful during CF exacerbations included the following: choice of antibiotics (83%), duration of antibiotics (78%), antibiotic dosing (68%), therapeutic drug monitoring (63%), reducing drug interactions (53%), and avoiding toxicity (50%). Nine percent of HCP stated that they did not think AMS was advantageous during exacerbations.ConclusionAlthough most CF HCP have access to individual aspects of AMS, fewer had access to a formal AMS program. Help with antibiotics during exacerbations was identified as an important aspect for input from AMS programs. Disclosures All authors: No reported disclosures.

Cystic fibrosis: What's new in South Africa in 2019.

Cystic fibrosis (CF) is one of the most common autosomal recessive disorders worldwide. The incidence of CF depends on the prevalence of CFTR (cystic fibrosis transmembrane conductance regulator) gene mutations in the population, which is determined by genetic diversity and ethnicity. Over 2 000 CFTR mutation variants, divided into six distinct functional classes, are now identified, but not all cause CF disease. Advancements in the molecular diagnosis of CF and recognition of a wider spectrum of CF severity have led to recent revision of CF diagnostic nomenclature and criteria. Identifying which CFTR mutations people with CF carry is important, as novel treatments that target the specific CFTR dysfunction at a molecular level are now available, and many new drugs are in the pipeline. These and other advancements in CF are comprehensively covered in the revised 5th edition of the South African Cystic Fibrosis Consensus Guidelines, published in 2017. In addition, the South African Cystic Fibrosis Registry Initiative (SACFRI) was launched in April 2018. SACFRI is a multicentre public-private collaboration of CF healthcare providers across South Africa (SA), which will prospectively collect data relating to CF diagnosis and outcomes in SA. Local SA registry data are critical to understanding the epidemiology of CF in SA, and SACFRI will be an important tool to identify and prioritise areas of CF care that require intervention.

Cystic fibrosis: What’s new in South Africa in 2019

Cystic fibrosis (CF) is one of the most common autosomal recessive disorders worldwide. The incidence of CF depends on the prevalence of CFTR (cystic fibrosis transmembrane conductance regulator) gene mutations in the population, which is determined by genetic diversity and ethnicity. Over 2 000 CFTR mutation variants, divided into six distinct functional classes, are now identified, but not all cause CF disease. Advancements in the molecular diagnosis of CF and recognition of a wider spectrum of CF severity have led to recent revision of CF diagnostic nomenclature and criteria. Identifying which CFTR mutations people with CF carry is important, as novel treatments that target the specific CFTR dysfunction at a molecular level are now available, and many new drugs are in the pipeline. These and other advancements in CF are comprehensively covered in the revised 5th edition of the South African Cystic Fibrosis Consensus Guidelines, published in 2017. In addition, the South African Cystic Fibrosis Registry Initiative (SACFRI) was launched in April 2018. SACFRI is a multicentre public-private collaboration of CF healthcare providers across South Africa (SA), which will prospectively collect data relating to CF diagnosis and outcomes in SA. Local SA registry data are critical to understanding the epidemiology of CF in SA, and SACFRI willbe an important tool to identify and prioritise areas of CF care that require intervention.

Work environment risks for health care workers with cystic fibrosis.

In Australia and New Zealand, >50% of people with cystic fibrosis (CF) are adults and many of these people are pursuing vocational training and undertaking paid employment. More than 6% of adults with CF are working in health care. There is limited guidance in literature to support health care workers with CF (HCWcf) in training and in employment to support safe practice and to provide protection for themselves and their patients from the acquisition of health care associated infection. A multidisciplinary team of CF and Infectious Disease Clinicians, Infection Prevention and Control Practitioners, HCWcf, academic experts in medical ethics and representatives from universities, appraised the available evidence on the risk posed to and by HCWcf. Specific recommendations were made for HCWcf, CF health care teams, hospitals and universities to support the safe practice and appropriate support for HCWcf.