Cystic Fibrosis Guidelines Research Articles

Assessing effectiveness of cleaning and disinfection of equipment and environmental surfaces in cystic fibrosis clinics using an ATP assay.

Infection control guidelines for cystic fibrosis (CF) stress cleaning of environmental surfaces and patientcare equipment in CF clinics. This multicenter study measured cleanliness of frequently touched surfaces in CF clinics using an ATP bioluminescence assay to assess the effectiveness of cleaning/disinfection and the impact of feedback. Eight surfaces were tested across 19 clinics (10 pediatric, 9 adult) over 5 rounds of testing. Rounds 1 and 2 served as uncleaned baseline, and Round 3 occurring after routine cleaning. Rounds 4 and 5 were performed after feedback provided to staff and measured after cleaning. Pass rates defined as <250 relative light units were the primary outcome. Of the 750 tests performed, 72% of surfaces passed at baseline, and 79%, 83%, and 85% of surfaces passed in Rounds 3, 4, and 5, respectively. The overall pass-rate was significantly higher in adult compared to pediatric clinics (86% vs 71%; P < 0.001). In pediatric clinics, blood pressure equipment and computer keyboards in the pulmonary function lab consistently passed, but the exam room patient/visitor chairs consistently failed in all rounds. In adult clinics blood pressure equipment, keyboards in exam rooms and exam tables passed in all rounds and no surface consistently failed. We demonstrate the feasibility of an ATP bioluminescence assay to measure cleanliness of patient care equipment and surfaces in CF clinics. Pass rates improved after cleaning and feedback for certain surfaces. We found that surfaces are more challenging to keep clean in clinics taking care of younger patients.

Bronchiectasis: literature review for preparation of 2024 clinical guidelines

Bronchiectasis, ICD-10 – J47, (BE) is a chronic respiratory disease characterized clinically by cough, sputum production and bronchial infection, and radiographically by abnormal and persistent dilation of the bronchi. Common causes include cystic fibrosis, primary ciliary dyskinesia, immune disorders, systemic inflammatory diseases and infections, and other factors. However, some cases are idiopathic, when the cause cannot be identified. In practice, patients with bronchiectasis are divided into two groups: associated and not associated with cystic fibrosis. The prevalence of the disease varies significantly worldwide; it is not reliably known in the Russian Federation.The aim of the review is to analyze the literature data on modern approaches to the diagnosis of BE and to familiarize readers with diagnostic methods and basic approaches to the treatment.Methods. Data from 77 articles and the expert opinion of specialists providing care to patients with BE were used.Results. The main causes, frequency of occurrence, clinical phenotypes and treatment approaches for BE are described. There are many clinical, laboratory, instrumental and radiological features that provide insight into the etiology of BE. The European consensus is that the goal of treating BE is to restore or maintain normal lung function. There are no randomized trials on the treatment of BE, so all treatment guidelines are based on very low-level evidence or extrapolated from cystic fibrosis guidelines. Recommendations for mucolytic, antibacterial and anti-inflammatory therapy for BE are described, taking into account international and national experience.Conclusion. The development of a new version of clinical guidelines with modern relevant information will improve the diagnosis and treatment of BE in the Russian Federation.

Effect of Disinfection Method and Testing Methodology on the Performance of a Breath-Enhanced Jet Nebulizer.

National guidelines for cystic fibrosis recommend cleaning and disinfecting nebulizers after each use. We tested two groups of five reusable breath-enhanced nebulizers after 0, 5, 10, 15, 20, 30, 60, 90, 120, 150, and 180 sterilization (baby bottle sterilizer) or cleaning cycles. The nebulizers were operated for 7 min (6 L/min) after loading albuterol (2.5 mg/3 mL), and they were evaluated with and without breathing simulation after cleaning/sterilization (0-180 and 0-60 cycles, respectively). Over the course of 180 cleaning/sterilization cycles, the mean (SD) solution output was 1.33 mL (0.12 mL)/1.29 mL (0.08 mL); the nebulizer mass remaining in the nebulizer was 61.5% (5.2%)/63% (4%); sputtering time was 4.7 min (0.8 min)/4.8 s (0.6 min); inspiratory filter was 19% (3%)/18.5% (2.4%); expiratory filter was 6.7% (1.1%)/6.7% (0.8%); and difference in drug output calculated using the solution output and nebulizer mass was 6.8% (4%)/5.2% (2.9%). Thermal disinfection with a baby-bottle sterilizer did not alter the performance of a reusable breath-enhanced nebulizer. The nebulizer test performed without breathing simulation underestimated its performance. The calculation of the drug output based on the solution output resulted in its overestimation.

Association of Pseudomonas aeruginosa incident infections with adherence to cystic fibrosis foundation care guidelines

BackgroundCare guidelines for cystic fibrosis (CF) have been developed to enhance consistent care and to improve health outcomes. We determined if adherence to CF care guidelines predicted P. aeruginosa incidence rates (Pa-IR) at U.S. CF centers in 2018. MethodsThis cross-sectional CF Foundation Patient Registry study included 82 adult and 132 pediatric centers. Adherence to 12 guidelines was defined categorically (guideline met) or as a continuous measure (proportion of patients being treated/evaluated per guideline). Association of adherence to individual guidelines with Pa-IR, accounted for center and patient characteristics relevant to Pa-IR and were modeled using random forests and weighted-least-squares (WLS) analyses. ResultsThe mean Pa-IR was 0.2 cases/patient-years at risk (SE 0.0074) for all centers combined. Guideline adherence was lowest for ≥4 bacterial cultures/year (54% of centers) and annual oral glucose tolerance test (OGTT) (48% of centers), and highest for annual non-tuberculous mycobacteria (NTM) sputum culture (98%). The mean number of guidelines met was 6.7 and higher for pediatric (7.3) than adult (5.6) centers, (p<0.001). The number of guidelines met correlated negatively with Pa-IR (β=-0.007, p = 0.043). Macrolide prescription and annual OGTT per guideline were associated with lower and higher Pa-IR, respectively. Centers with lower center-wide lung function, higher proportion of pwCF with low body-mass index, and location in the Southwest had higher Pa-IR. ConclusionOverall adherence to guidelines was high except for performing ≥4 bacterial cultures/year and OGTT. Higher Pa-IR was associated with center characteristics and lower guideline adherence. The lower Pa-IR with greater adherence to guidelines suggests that focusing on quality care can positively impact Pa-IR.

P339 Diet quality assessment of adults with cystic fibrosis - comparison to population dietary guidelines: a cross-sectional study

CFTR modulator therapy improves respiratory function with consequential reductions in resting energy expenditure in people with Cystic Fibrosis (CF). Traditionally, CF dietetic management focuses on meeting high energy needs with typically high fat, sugar, and salt foods aetiologically linked to diet-related chronic diseases. This study aims to analyse habitual dietary intakes of Irish adults with CF, comparing to CF dietary guidelines and assess diet quality with population specific healthy eating guidelines. Participant information is gathered using demographic questionnaires and dietary data via three-day food diaries. Dietary data is statistically analysed in SPSS® and compared to CF and population dietary guidelines.

Comparison of mental health in individuals with primary ciliary dyskinesia, cystic fibrosis, and parent caregivers

IntroductionIndividuals with chronic respiratory diseases and caregivers are at higher risk for depression and anxiety. Primary ciliary dyskinesia (PCD) and cystic fibrosis (CF) are both rare genetic diseases, characterized by recurrent respiratory infections. This study compared depression and anxiety in people with PCD (pwPCD) and CF (pwCF), and caregivers, using the screening tools recommended in the CF guidelines. MethodsPatient Health Questionnaire (PHQ-9) and Generalized Anxiety Disorder (GAD-7) were administered to a PCD and CF sample. Given that PCD is extremely rare, they were matched on age and sex to pwCF at a 1:2 ratio. Similar procedures were performed with parents. ResultsA total of 63 patients and 129 caregivers participated: 21 pwPCD and 42 pwCF (ages 12–34 years) plus 43 caregivers of pwPCD and 86 caregivers of pwCF. A high percentage of patients scored above the cut-off for depression (PCD: 33%; CF: 43%) and anxiety (PCD and CF both: 43%), mostly mild. Similarly, a high percentage of caregivers scored above the cut-off for depression (PCD: 42–54%; CF: 45–46%) and anxiety (PCD: 47–54%; CF: 39–56%). Suicidal ideation was endorsed by 9.5% of pwPCD, 20% of mothers and 10% of fathers and 5% of pwCF, 3% of mothers, but no fathers. ConclusionA large percentage of patients and caregivers reported elevated psychological distress and suicidal ideation. Addressing psychological symptoms is critical given they are associated with poor adherence, missed clinic visits, increased inflammation and worse quality of life. Mental health screening and treatment should be integrated into PCD care.

The inequity of targeted cystic fibrosis reproductive carrier screening tests in Australia.

European and Australian guidelines for cystic fibrosis (CF) reproductive carrier screening recommend testing a small number of high frequency CF causing variants, rather than comprehensive CFTR sequencing. The study objective was to determine variant detection rates of commercially available targeted reproductive carrier screening tests in Australia. Next-generation DNA sequencing of the CFTR gene was performed on 2552 individuals from a whole population sample to identify CF causing variants. The variant detection rates of two commercially available Australian reproductive carrier screening tests, which target 50 or 175 CF causing variants, in this population were calculated. The ethnicity of individuals was determined using principal component analysis. Variant detection rates of the tests for 50 and 175 CF causing variants were 88.2% and 90.8%, respectively. No CF causing variants in individuals of East Asian ethnicity (n=3) were detected by either test, while >86.6% (n=69) of CF causing variants in Europeans would be identified by either test. Reproductive carrier screening tests for a targeted set of high frequency CF variants are unable to detect approximately 10% of CF variants in a multiethnic Australian population, and individuals of East Asian ethnicity are disproportionally affected by this test limitation.

Take on transplant: human-centered design of a patient education tool to facilitate informed discussions about lung transplant among people with cystic fibrosis.

Lung transplant (LTx) saves lives in cystic fibrosis (CF). However, many potential candidates express uncertainty about LTx and die before receiving this treatment. CF guidelines recommend LTx education and clinical discussions well before the need for LTx arises, but limited patient resources exist. We engaged people with CF and CF physicians in human-centered design of "Take On Transplant" (TOT), a web-based education tool to prepare patients for LTx discussions. Across 3 phases, needs assessment, design groups, and iterative user testing of TOT, we refined TOT from wireframe prototypes, to an interactive website, to a fully functional intervention ready for clinical trials. Fifty-five people with CF and 105 physicians identified information needs to prepare for LTx discussions. Design groups (n = 14 participants) then established core requirements: didactic education ("Resource Library"), patient narratives ("CF Stories"), frequently asked questions ("FAQ"), and self-assessment to tailor content ("My CF Stage"). Iterative usability testing (n = 39) optimized the design of CF Stories and prototype layout. We then developed the TOT website and demonstrated feasibility and preliminary efficacy of use through 2-week field testing (n = 9). Our human-centered design process provided guidance for educational tools to serve the evolving needs of potential LTx candidates. Our findings support the process of patient deliberation as a foundation for shared decision-making in CF, and inform educational tools that could potentially translate beyond LTx. TOT fills a critical gap in preparing people with CF for shared decision-making about LTx and may serve as a model for educational tools for other preference-sensitive decisions.

Primary ciliary dyskinesia: review of the draft clinical guidelines, 2022

Primary ciliary dyskinesia (PCD) is a rare hereditary disease from the group of ciliopathies, which is based on a defect in the cilia ultrastructure of the respiratory epithelium and similar structures (sperm flagella, villi of the fallopian tubes, ventricular ependyma, etc.), leading to motor function impairment. The prevalence of the disease varies significantly around the world and is not known reliably in the Russian Federation.The aim of the review was to analyze literature data on modern approaches to the diagnosis and treatment of PCD.Methodology. The data of 90 articles and the opinions of experts providing care to patients with PCD were used.Results. The classic manifestations of PCD depend on age. The leading manifestations of the disease in patients with PCD are recurrent inflammatory diseases of the upper and lower respiratory tract (bronchitis, pneumonia), with the formation of bronchiectasis, damage to the ENT organs (chronic rhinitis, rhinosinusitis, nasal polyposis, repeated otitis media, progressive hearing loss). Currently, there is no single method which could serve as a “gold” standard for diagnosing PCD. The diagnosis of PCD is based on the characteristic clinical picture in combination with the results of special tests (nitric oxide in exhaled air, DNA diagnostics, high-speed video microscopy, transmission electron microscopy). The genetic diagnostics has not been developed sufficiently in the global practice yet and is unavailable in our country. The approach to the treatment of a patient with PCD should be multidisciplinary due to multiple organ lesions. According to the European consensus, the goal of PCD therapy is to restore or maintain normal lung function. There have been no randomized trials of treatment for PCD, and therefore all treatment recommendations are based on very low-level evidence or extrapolated from cystic fibrosis guidelines. Recommendations on mucolytic, antibacterial and anti-inflammatory therapy of PCD are given with consideration for the international and domestic experience.Conclusion. The development of a new version of clinical guidelines containing up-to-date relevant information will improve the diagnosis and treatment of PCD in the Russian Federation.

Findings from a feasibility study of estradiol for hypogonadal women with cystic fibrosis-related bone disease

BackgroundAdvancements in therapies for patients with cystic fibrosis (CF) have decreased mortality, leading to increased prevalence of chronic complications including bone disease. CF-related bone disease (CFBD) is characterized by low bone mineral density (BMD) and fragility fractures. Estrogen deficiency increases bone resorption, resulting in decreased BMD that can be restored with estrogen replacement. Current CF guidelines recommend treating female hypogonadal patients with CFBD with estrogen replacement, but no prospective study has investigated the effects of estrogen supplementation on CFBD. Estrogen is known to modulate inflammatory markers and autoimmune diseases.We proposed to test the hypothesis that estrogen status plays a critical role in optimizing bone health, modulating inflammation, preserving lung function, and maximizing quality of life in premenopausal women with CF.MethodsWe planned a randomized, placebo-controlled, investigator- and patient-blinded, pilot trial with two parallel arms. Eligible subjects were women with CF 18–50 years old with hypogonadism and low BMD who were not taking systemic glucocorticoids, had not had a prior transplant, and did not have contraindications to oral estradiol. Subjects would be block randomized to receive oral estradiol or placebo for 6 months. The primary outcome was feasibility metrics. Secondary outcomes included relative changes in estradiol, bone turnover markers, lung function, inflammatory markers, and quality of life metrics. The study was funded through departmental funds.ResultsOf 233 subjects screened, 86 subjects were women with CF 18–50 years old and none were eligible for participation. Most subjects were excluded due to absent DXA report (24%), normal BMD (22%), or use of systemic estrogen (16%). Due to difficulty recruiting the planned 52 subjects, the trial was closed for recruitment and no subjects were randomized.ConclusionThis study was designed to investigate the feasibility of a safety and efficacy trial of estrogen therapy for women with CF. Unfortunately, due to eligibility criteria, the study was unable to recruit subjects. This feasibility study highlights the need for improved BMD screening in young women with CF. Future study designs may require the incorporation of a screening DXA as part of subject recruitment.Trial registrationThe study was registered on ClinicalTrials.gov (NCT03724955).

Cystic fibrosis mutation classes in pediatric otitis media — Fickle or faulty?

PurposeRecent data have challenged the historical paradigm that cystic fibrosis (CF) protects against otitis media (OM). These findings raised questions about the pathogenesis of this ostensible change. In this study our aim is to characterize acute OM (AOM) risk based on CF genotype. MethodsA retrospective chart review was completed at a tertiary care pediatric hospital. Charts of 159 CF patients seen at our facility from 2010 to 2019 were reviewed. Data collected included demographics, AOM infections, cystic fibrosis transmembrane conductance regulator (CFTR) allele mutations, pulmonary exacerbations (PE), and pancreatic insufficiency (PI) status. Mutation alleles were divided into five classes based on CF guidelines, which were further classified as severe (classes I–III) or mild (classes IV–V). Results54% of patients had at least one episode of AOM with a mean of 1.5 episodes of AOM (standard deviation = 2.3). 86% of patients had severe/severe (S/S) alleles and 14% had severe/mild (S/M). S/S patients had significantly more PE (p = .004) and increased rates of PI (p < .001). Of the 131 patients with S/S mutations, 57% had an episode of AOM while only 46% the 22 S/M patients had an AOM episode (p = .357). ConclusionsTo our knowledge this is the first report showing a clinical trend towards increased middle ear disease in patients with severe CFTR mutations. Future prospective studies will be powered to demonstrate whether this trend is statistically significant. Patients with S/S mutations not only have more severe clinical phenotypes but may have additional unexpected complications such as middle ear disease.

Evaluation of Home Medication Reconciliation by Clinical Pharmacists for Adult and Pediatric Cystic Fibrosis Patients.

Medication reconciliation is an important aspect of a patient’s care process that is ideally performed by clinical pharmacists. Despite literature supporting this process in other patient populations, cystic fibrosis (CF) lacks research in this area. To address this, we designed a retrospective, multi-centered, non-controlled, cross-sectional study at four CF Foundation-accredited centers in the United States to evaluate the medication reconciliation process for adult and pediatric CF patients by documenting the number of home medications reconciled by clinical pharmacists and the number of patients with home medications that did not align with the current CF guidelines published in 2013. There were 105 adult patients and 72 pediatric patients included in the study analysis with a mean number of medications reconciled by clinical pharmacists of 17.4 (standard deviation (SD) 6.7) for adults and 13 (SD 4.6) for pediatric patients. The mean number of discrepancies from guidelines per patient was 1.61 (SD 1.2) for adult patients and 0.63 (SD 0.9) for pediatric patients. Pharmacists play an essential role in identifying and managing medication interactions and further research is necessary to investigate pharmacist impact on medication reconciliation.

Energy and macronutrient intakes in children with cystic fibrosis

Rationale: Adequate nutrition is essential for growth in children with cystic fibrosis (CF). The new guidelines for CF bring attention to monitoring macronutrient intake as well as total energy. In general the goal for children with CF is to achieve optimal growth by consuming a daily energy (kcal) intake of 110–200% of the recommended daily allowance (RDA). The aim of this study was to evaluate daily energy and macronutrient intakes and compare according to the recommendations of the nutritional guidelines for CF.

Evaluating the impact of 2006 Australasian Clinical Practice Guidelines for nutrition in children with cystic fibrosis in Australia.

To determine the association between the implementation of the 2006 Australasian Clinical Practice Guidelines for Nutrition in Cystic Fibrosis (CF) and the nutritional status of children participating in the Australian Cystic Fibrosis Data Registry (ACFDR). This research consisted of a quantitative study using ACFDR data and a survey of clinicians and dietitians treating children with CF. Two independent cohorts of children (2-5 years and 6-11 years) were selected from ACFDR between 1998 and 2014 (N = 2304). Generalised estimating equation model was used to assess weight, height and body mass index (BMI) z-scores for each patient before and after the implementation of the nutrition guidelines. A nationwide online survey was sent to 48 clinicians to explore the enablers and barriers to implementation of the guidelines. Data analysis showed significant increase (p < 0.05) in mean weight, height and BMI z-scores ranging from 0.06 to 0.18 after implementation of the guidelines in both cohorts of children. Nineteen (39%) clinicians participated in the survey. The majority of the respondents adopted the recommendations into their practice and used the guidelines as part of their professional development. Structural barriers included a lack of adequate staff resources and clinic space for consultations, inappropriate staff classification, high staff turnover and lack of mentoring support. In children participating in the ACFDR, nutritional status improved after the implementation of the 2006 guidelines. Survey results revealed enablers and barriers to guideline implementation and will inform implementation strategies for the revised Australasian nutrition guidelines for CF, released in 2017.

326 Nutrition guidelines for cystic fibrosis in Australia and New Zealand: development of evidence based and consensus recommendations

326 Nutrition guidelines for cystic fibrosis in Australia and New Zealand: development of evidence based and consensus recommendations

428 Are cystic fibrosis guidelines credible? Evaluating methodological issues

428 Are cystic fibrosis guidelines credible? Evaluating methodological issues

188 Nutrition guidelines for cystic fibrosis in Australia and New Zealand: development of evidence based and consensus recommendations

188 Nutrition guidelines for cystic fibrosis in Australia and New Zealand: development of evidence based and consensus recommendations

Maintaining Nursing Care Quality—Clinical Care Guidelines for Cystic Fibrosis: Outpatient and Inpatient

The Care Committee of the Mukoviszidose e.V., headed by Cornelia Meyer, has drawn up a completely revised version of the German Guidelines to Care in Cystic Fibrosis. It addresses all nursing staff concerning children, adolescents or adults with cystic fibrosis (CF) either in an inpatient and outpatient setting or in rehab clinics. The guidelines evolve along with the increased life expectancy of cystic fibrosis patients and improved quality of life which is described by CF patients and can be observed by the therapeutic CF team. For example, the treatment of secondary diseases like CFDR (CF related diabetes) which comes along with an increased life expectancy has been added over the years. It is essential that within such a complex disease pattern, professional experience of care staff who worked in specialized CF care centers (50+ patients per year) for many years has to be transferred to other nursing stuff or beginners. This is especially important in the context of the heterogeneous characteristics of this disease. Often it can be seen that the quality of the care for CF patients depends on the know-how and professional experience of the interdisciplinary CF therapists team. Moreover, it depends on the knowledge which the patient has gathered by himself by experience or learning. And, of course, it depends also on the knowledge of the relatives especially parents or partner respectively.

Infection control strategies that successfully controlled an outbreak of Mycobacterium abscessus at a cystic fibrosis center

Mycobacterium abscessus infection in patients with cystic fibrosis (CF) can result in accelerated clinical decline and the potential for direct or indirect transmission between patients has been recently demonstrated. Data on the outcomes of M abscessus outbreaks and the efficacy of specific infection control procedures in patients with CF remain limited. This study provides follow-up from an outbreak of pulmonary M abscessus in our center, highlighting outcomes and strategies that appear to have prevented further spread of the organism. Data from our adult CF center (1989-2015) were analyzed, including chart reviews of all patients with positive mycobacterial sputum cultures, cultures from environment surfaces, and epidemiologic evaluation of infected patients. Following an M abscessus outbreak in 2009, infection control policies were intensified based on CF guidelines and surveillance data were collected and reviewed. Five cases of M abscessus were involved in the outbreak; 3 patients died during follow-up. An environment search failed to reveal an intermediary source of transmission between patients. After implementation of infection control measures composed of staff/patient education, environment sterilization, and patient isolation, no new cases were detected. Direct or indirect patient-to-patient transmission of M abscessus is a threat in the CF population. A multifaceted infection control strategy based on CF guidelines was effective in halting transmission in our center.

236 Vitamin status in infants with cystic fibrosis during the first year of life

Objectives Nutritional guidelines for cystic fibrosis recommend vitamin supplements [1]. Newborn screening has resulted in better nutrition at diagnosis. We assessed vitamin A, D, E status in the first year of life and vitamin supplements. Methods We identified 55 newborn infants with CF at our regional CF centre, over a 3 year period (2011–13). Vitamin A, D, E levels were measured at 3 & 12 months, according to local guidelines. We examined the association between vitamin levels and pancreatic status, vitamin supplements, meconium ileus, milk formulation, nutrition status. TableSerum vitamin levelsSerum levelNumber (%) of subjectsVit A (0.7–1.5 mmol/L)Vit D (50/75–150 nmol/L)Vit E (12–24 mmol/L)3 mo (n = 35)12 mo (n = 35)3 mo (n = 35)12 mo (n = 35)3 mo (n = 34)12 mo (n = 35)Low1 (2.8%)1 (2.8%)3 (8.6%)9 (25.7%)2 (5.9%)3 (8.6%)Normal31 (88.6%)22 (62.9%)32 (91.4%)26 (74.3%)7 (20.6%)16 (45.7%)High3 (8.6%)12 (34.3%)0025 (73.5%)16 (45.7%) Results See the table. Vitamins were commenced by age 1 month in pancreatic insufficient infants. There was no relationship between vitamin status and bowel resection, milk formulation and nutrition status. Vitamin A supplementation was appropriate at 3 months, but by 12 months, a third of infants had high levels. Vitamin D status was normal at 3 months, but by 12 months a quarter of infants were deficient. Vitamin E levels were high in most infants at 3 months, and by 12 months, high vitamin E levels correlated with the amount of supplements. Conclusion Our audit of vitamin assays during the first year of life in CF infants indicates the need to review our guidelines, particularly to achieve adequate vitamin D status and avoid excess vitamin E levels.