Pilot study of paediatric regional lung function assessment via X-ray velocimetry (XV) imaging in children with normal lungs and in children with cystic fibrosis.

INTRODUCTION: Cystic fibrosis (CF) is a life-limiting autosomal recessive genetic condition. It is caused by mutations in the gene that encodes for a chloride and bicarbonate conducting transmembrane channel. X-ray velocimetry (XV) is a novel form of X-ray imaging that can generate lung ventilation data through the breathing cycle. XV technology has been validated in multiple animal models, including the ß-ENaC mouse model of CF lung disease. It has since been assessed in early-phase clinical trials in adult human subjects; however, there is a paucity of data in the paediatric cohort, including in CF. The aim of this pilot study was to investigate the feasibility of performing a single-centre cohort study in paediatric patients with CF and in those with normal lungs to demonstrate the appropriateness of proceeding with further studies of XV in these cohorts. METHODS AND ANALYSIS: This is a cross-sectional, single-centre, pilot study. It will recruit children aged 3-18 years to have XV lung imaging performed, as well as paired pulmonary function testing. The study will aim to recruit 20 children without CF with normal lungs and 20 children with CF. The primary outcome will be the feasibility of recruiting children and performing XV testing. Secondary outcomes will include comparisons between XV and current assessments of pulmonary function and structure. ETHICS AND DISSEMINATION: This project has ethical approval granted by The Women's and Children's Hospital Human Research Ethics Committee (HREC ID 2021/HRE00396). Findings will be disseminated through peer-reviewed publication and conferences. TRIAL REGISTRATION NUMBER: ACTRN12623000109606.

Current cough and sputum assessed by the cough and sputum assessment-questionnaire (CASA-Q) is associated with quality of life impairment in cystic fibrosis.

BACKGROUND: Cough and sputum are major symptoms in cystic fibrosis (CF) that contribute to the impairment of quality of life. METHODS: This prospective single centre cross-sectional pilot study aimed to evaluate the results of a self-administered questionnaire assessing cough and sputum symptoms (2 domains), and their impact (2 domains) on daily activities in the previous week, named the Cough and Sputum Assessment Questionnaire (CASA-Q) in CF adult patients at stable state, and to analyse associations with clinical, functional, microbiological, radiological data, and two quality of life scales: the Cystic Fibrosis Questionnaire Revised (CFQ-R) and the Saint George Respiratory Questionnaire (SGRQ). RESULTS: Forty-eight patients were included in this analysis (69% men; median age of 27.8 ± 8.1 years; median body mass index of 21.8 + 3.3 kg/m²; mean FEV1 of 64 ± 30% of the predicted value). The mean values of the CASA-Q domains were 58 ± 23 for cough symptoms, 77 ± 24 for cough impact, 62 ± 25 for sputum symptoms and 84 ± 21 for sputum impact. Impairment in CASA-Q cough and sputum domains was associated with dyspnea mMRC scale (p < 0.005 for all 4 domains of CASA-Q) and exacerbations in the previous year (p < 0.05 for CASA-Q symptoms domains). We also found correlations between all domains of the CASA-Q and quality of life questionnaires including SGRQ (p < 0.001) and to a lesser extend CFQ-R. We identified a clinical phenotype (female gender, ΔF508 heterozygous mutation, dyspnea mMRC scale) associated with an impairment of CASA-Q score and quality of life using a 2-step cluster analysis. CONCLUSIONS: CASA-Q allows the assessment of cough and sputum in CF adult patients and is associated with quality of life impairment. This simple easy-to-use tool could be used in routine clinical practice and in clinical studies to assess cough and sputum in CF patients. TRIAL REGISTRATION: The study was registered on ClinicalTrials.gov (NCT02924818, first posted on 5th October 2016).

Critical disease burdens of Australian adults with cystic fibrosis: Results from an online survey.

BACKGROUND: The objective of this study was to conduct a web-based questionnaire to investigate self-reported phenotypes and disease burdens of individuals living in Australia and diagnosed with cystic fibrosis (CF) using a case-control study design. METHODS: An online questionnaire was distributed to individuals with CF and healthy control subjects. Overall health rating, medications, family history, education, clinical indicators of disease, and symptoms, including their severity and frequency, were evaluated. RESULTS: There was a total of 119 respondents consisting of 59 people living with CF and 60 controls. The CF cohort had significantly lower tertiary educational levels compared to controls. The analysis specific to the CF cohort depicted a significant correlation between the frequency of hospitalizations and the level of education in the CF cohort. Of the 26 self-reported symptoms of CF that were analyzed, 14 were significantly higher in the people living with CF. The CF cohort reporting symptoms of chronic pain (25%) described an increase in the burden of disease, depicting a 30% longer mean hospitalization, increased consumption of medications and significant relationships with four other symptoms, including muscle aches, digestive issues, pancreatic insufficiency, and abdominal swelling. CONCLUSIONS: The nationwide survey identified a diverse range of clinical manifestations experienced by the Australian CF population. Chronic pain, linked to aging and the changing landscape of disease, was a significant indicator of the burden of disease. A comprehensive understanding of the phenotypic profiles and symptom variability will contribute to future research and provide insights into the impacts of disease and the burden of therapy, particularly in children, at the start of their health journey.

Epidemiology and management of nontuberculous mycobacterial disease in people with cystic fibrosis, the Netherlands.

BACKGROUND: Nontuberculous mycobacteria (NTM) are opportunistic, difficult to treat pathogens. With increasing prevalence of NTM infections in people with cystic fibrosis (pwCF) and the improved life expectancy, the burden is expected to grow. METHODS: We assessed the epidemiology and management of NTM isolation and disease in pwCF in the Netherlands using a survey and retrospective, case-controlled data from the Dutch CF Registry. We determined the isolation prevalence, treatment and outcomes from 2013-2019. RESULTS: NTM isolation prevalence increased from 1.0% to 3.6% (2013-2019). This was a single NTM isolation in 53.7% of the adults and 60.0% of the children. M. abscessus and M. avium complex (MAC) were most frequent (47.1 and 30.9%). Of the treated pwCF, 48.5% attained culture conversion of M. abscessus; 54.5% for MAC. Children with an NTM isolation showed more infections with S. maltophilia and/or A. fumigatus (p < 0.001) compared to controls. In the year prior to NTM isolation, children in the NTM group had a lower mean FEV1% predicted (81.5 ± 16.7 vs. 88.6 ± 15.3, p = 0.024), while adults in the NTM group had more IV antibiotic days compared to controls (60 vs. 17, p = 0.047). In the following years, FEV1% predicted declined faster in pwCF with NTM than the control group (children: -3.8% vs. -1.6%, p = 0.023; adults: -0.7% and 0.4%, ns). CONCLUSIONS: The isolation prevalence of 3.6%, poor treatment outcomes and associated lung function decline emphasize that NTM pulmonary disease (NTM-PD) is a significant health issue among pwCF in the Netherlands. Its prevention and treatment require increased attention.

Prevalence of unmet pain and symptom management needs in adults with cystic fibrosis.

BACKGROUND: Although people living with CF (PLwCF) commonly report pain and other symptoms, little is known regarding their experiences of living with and accessing treatment for burdensome symptoms. METHODS: PLwCF completed online questionnaires assessing symptom prevalence and distress and were also asked about experiences accessing pain and symptom treatment, using both closed-ended and free-text entries. RESULTS: Pain was the most prevalent symptom experienced among the 55 participants (76%) and the symptom that most commonly caused distress (64%). PLwCF not on CFTR modulator therapy were likelier to endorse pain as distressing (p = 0.007). Respondents expressed that their pain was commonly underrecognized and undermanaged, they desired a multi-modal approach to treatment, and noted concerns about disease progression affecting their symptom management options. CONCLUSIONS: Our study suggests that PLwCF often have unmet symptom management needs that may impair quality of life.

Longitudinal assessment of renal function after lung transplantation for cystic fibrosis: transition from post-operative acute kidney injury to acute kidney disease and chronic kidney failure.

INTRODUCTION: The clinical trajectory of post-operative acute kidney injury (AKI) following lung transplantation for cystic fibrosis is unknown. METHODS: Incidence and risk factors for post-operative AKI, acute kidney disease (AKD) and chronic kidney disease (CKD) were retrospectively analyzed in cystic fibrosis patients undergoing lung transplantation. Logistic regressions, Chi-square, Cuzick rank tests, and Cox-proportional hazard models were used. RESULTS: Eighty-three patients were included. Creatinine peaked 3[2-4] days after transplantation, with 15(18%), 15(18%), and 20(24%) patients having post-operative AKI stages 1, 2, and 3, while 15(18%), 19(23%) and 10(12%) developed AKD stage 1, stage 2 and 3, respectively. Higher AKI stage was associated with worsening AKD (p = 0.009) and CKD (p = 0.015) stages. Of the 50 patients with AKI, 32(66%) transitioned to AKD stage > 0, and then 27 (56%) to CKD stage > 1. Female sex, extracorporeal membrane oxygenation support as a bridge to lung transplant and at the end of the surgery, the use of intraoperative blood components, and cold-ischemia time were associated with increased risk of post-operative AKI and AKD. Higher AKI stage prolonged invasive mechanical ventilation (p = 0.0001), ICU stay (p = 0.0001), and hospital stay (p = 0.0001), and increased the incidence of primary graft dysfunction (p = 0.035). Both AKI and AKD stages > 2 worsened long-term survival with risk ratios of 3.71 (1.34-10.2), p = 0.0131 and 2.65(1.02-6.87), p = 0.0443, respectively. DISCUSSION: AKI is frequent in cystic fibrosis patients undergoing lung transplantation, it often evolves to AKD and to chronic kidney disease, thereby worsening short- and long-term outcomes.

Assessment of Liver Fibrosis with the Use of Elastography in Paediatric Patients with Diagnosed Cystic Fibrosis.

Background: Complications of cystic fibrosis-associated liver disease (CFLD) are a leading nonpulmonary cause of death. Noninvasive tests enabling early detection of liver changes, especially in children are sought. The aim of the study was to assess the scale of liver fibrosis with the use of elastography in paediatric patients with diagnosed cystic fibrosis (CF) and its comparison with other tests (APRI and Fibrotest). Methods: We examined 41 children, in the age range 2-21 years, with diagnosed CF. The analysis a included clinical picture, laboratory parameters of liver damage, and cholestasis. Aspartate aminotransferase-to-platelet ratio index (APRI) and Fibrotest were done in all patients. Liver stiffness measurements were acquired using shear-wave elastography (SWE). Results: CFLD was diagnosed in 16/41 patients (39%). Abnormal elastography was observed in 19/41 patients (46.3%), and in 5/41 (12.2%), the changes were advanced (F4). Abnormal elastography was observed in 12/16 (75%) of the patients with CFLD, and in 7/25 (28%), there were no lesions observed in the liver in the course of cystic fibrosis. In all patients with F4, we observed abnormal results of the APRI and Fibrotest. In most patients with small changes in elastography, we found normal results of the APRI and Fibrotest. Conclusion: Elastography seems to be a noninvasive examination useful in everyday clinical work in detecting early liver changes and monitoring of progression in paediatric patients with diagnosed cystic fibrosis, ahead of changes in laboratory tests. The cost-effectiveness of this test, the possibility of its repetition, and its availability are additional benefits.

Health care costs in a randomized trial of antimicrobial duration among cystic fibrosis patients with pulmonary exacerbations.

BACKGROUND: The purpose of these analyses was to determine whether overall costs were reduced in cystic fibrosis (CF) patients experiencing pulmonary exacerbation (PEx) who received shorter versus longer durations of treatment. METHODS: Among people with CF experiencing PEx, we calculated 30-day inpatient, outpatient, emergency room, and medication costs and summed these to derive total costs in 2020 USD. Using the Kaplan-Meier sample average (KMSA) method, we calculated adjusted costs and differences in costs within two pairs of randomized groups: early robust responders (ERR) randomized to receive treatment for 10 days (ERR-10 days) or 14 days (ERR-14 days), and non-early robust responders (NERR) randomized to receive treatment for 14 days (NERR-14 days) or 21 days (NERR-21 days). RESULTS: Patients in the shorter treatment duration groups had shorter lengths of stay per hospitalization (mean ± standard deviation (SD) for ERR-10 days: 7.9 ± 3.0 days per hospitalization compared to 10.1 ± 4.2 days in ERR-14 days; for NERR-14 days: 8.7 ± 4.9 days per hospitalization compared to 9.6 ± 6.5 days in NERR-21 days). We found statistically significantly lower adjusted mean costs (95% confidence interval) among those who were randomized to receive shorter treatment durations (ERR-10 days: $60,800 ($59,150 - $62,430) vs $74,420 ($72,610 - $76,450) in ERR-14 days; NERR-14 days: $66,690 ($65,960-$67,400) versus $74,830 ($73,980-$75,650) in NERR-21 days). CONCLUSIONS: Tied with earlier evidence that shorter treatment duration was not associated with worse clinical outcomes, our analyses indicate that treating with shorter antimicrobial durations can reduce costs without diminishing clinical outcomes.

Review article: epidemiology, pathogenesis and management of liver disease in adults with cystic fibrosis.

BACKGROUND: Cystic fibrosis-related liver disease (CFLD) is the leading cause of death in cystic fibrosis (CF), after pulmonary disease. To improve identification and management of this condition requires an understanding of the underlying disease mechanism. AIMS: This review summarises the current understanding of CFLD epidemiology, pathology, diagnosis and management. METHODS: Relevant reports on cystic fibrosis liver disease were identified using a literature search and summarised. RESULTS: CFLD is a heterogeneous condition with several different co-existent pathologies, including environmental and genetic factors. Incidence of clinically significant CFLD continues at a linear rate into early adulthood and has been described in up to 25% of CF patients. Diagnosis strategies lack precision and patient risk stratification needs to look beyond Childs-Pugh scoring. Efficacious therapies are lacking and, at present, newer modulator therapies lack data in CFLD and carry an increased risk of hepatotoxicity. Outcomes of liver transplant are comparable to non-CF transplant indications. CONCLUSIONS: The incidence of CFLD increases with age and hence is increasingly important to adult patients with CF. Effective therapies are lacking. For progress to be made a better understanding of pathogenesis and disease detection are required.

Probiotic use in adults with cystic fibrosis is common and influenced by gastrointestinal health needs: A cross-sectional survey study.

BACKGROUND: Cystic fibrosis (CF) primarily affects the lung, however, gastrointestinal disorders and symptoms, including dysbiosis, also impact on morbidity and quality of life. There is interest in strategies to modulate the gastrointestinal microbiota, including probiotics, although the evidence remains inadequate to guide practice, and information on use is limited. The present study aimed to characterise probiotic use, beliefs and experiences of adults with CF. METHODS: A cross-sectional questionnaire study was conducted in adults with CF (n = 205) and a general population Control group (n = 158), recruited from Victoria, Australia. Participants were classified as probiotic 'Ever Users' or 'Never Users'. Outcomes included self-reported probiotic use and factors associated with probiotic use, which were analysed using logistic regression analysis. Open-ended questionnaire responses were thematically analysed. RESULTS: In total, 70% of adults with CF had ever used probiotics (supplements and/or foods), comparable to Controls (80%) (p = 0.03). Key reasons for CF probiotic use were gastrointestinal- and antibiotic-related (75%). Most CF Ever Users (73%) did not discuss probiotic use with CF clinicians and 33% were uncertain if probiotics had been helpful. Female gender (odds ratio [OR] = 2.82; 95% confidence interval [CI] = 1.36-5.87; p = 0.005), university-level education (OR = 2.73; 95% CI = 1.24-6.01; p = 0.01) and bloating on antibiotics (OR = 2.14; 95% CI = 1.04-4.40; p = 0.04) were independently associated with probiotic use in CF; as was female gender in Controls (OR = 2.84; 95% CI = 1.20-6.71; p = 0.02). CONCLUSIONS: Probiotics were used by adults with CF for gastrointestinal- and antibiotic-related reasons often without informing clinicians and despite uncertainty about perceived helpfulness. Further research investigating gastrointestinal outcomes of probiotics will inform practice recommendations guiding their use in CF and other chronic diseases.

Cystic fibrosis and noninvasive liver fibrosis assessment methods in children.

BACKGROUND: Noninvasive assessments of liver fibrosis are currently used to evaluate cystic fibrosis (CF)-related liver disease. However, there is scarce data regarding their repeatability and reproducibility, especially in children with CF. The present study aimed to evaluate the repeatability and reproducibility of transient elastography (TE) (FibroScan®) and point shear-wave elastography using virtual touch quantification (pSWE VTQ) in children with CF. METHODS: TE and pSWE VTQ were performed in 56 children with CF by two different operators. Analysis of repeatability and reproducibility was available in 33 patients for TE and 46 patients for pSWE VTQ. Intra- and interobserver agreement were assessed using the intraclass correlation coefficient (ICC) and their 95% confidence interval (CI), and Bland and Altman graphs. RESULTS: For TE, ICC was 0.91 (0.83-0.95) for intraobserver agreement and 0.92 (95% CI: 0.86-0.96) for interobserver agreement. For pSWE VTQ, ICC was 0.83 (0.72-0.90) for intraobserver agreement and 0.67 (0.48-0.80) for interobserver agreement. CONCLUSIONS: Both technics can be proposed in the follow-up of patients, according to their availability in CF centers. IMPACT: This study shows that TE and pSWE VTQ are reliable methods to evaluate liver fibrosis in children with CF. This study shows for the first time that TE and pSWE VTQ are both repeatable and reproducible in children with CF. These data indicate that both TE and pSWE VTQ can be proposed for the follow-up of patients with CF, according to their availability in each CF center.

Arm muscle area for the longitudinal assessment of nutritional status in paediatric patients with cystic fibrosis - A single centre experience.

BACKGROUND: In cystic fibrosis, adequate nutrition contributes to good long-term prognosis. A body mass index (BMI) at or above the 50th percentile for age and sex in all children has been recommended. As researchers have described a depletion of fat-free mass despite normal BMI, longitudinal studies using more sensitive nutritional parameters are warranted. We evaluated anthropometric measurements in an attempt to identify early indicators of deteriorating nutritional status in our paediatric cohort. METHODS: We analysed datasets from children and adolescents between 2 and 17.9 years with at least two entries for triceps skinfold thickness and upper arm circumference in our patient database between January 1995 and December 2018. Arm muscle area (AMA) was calculated, and all values were expressed as z-scores from CDC growth charts. RESULTS: A total of 4,862 encounters from 161 paediatric patients (78 girls) were available, representing a median number of 28 visits during a median follow-up of 8.1 years per patient. Linear mixed effects models revealed relatively stable courses for weight, height, BMI and skinfold thickness up to adulthood. AMA was the only parameter which declined slightly (r = -0.036), particularly in boys. Kaplan-Meier-analyses showed that AMA was the earliest parameter to decrease below -1 z-score between 6 and 18 years. CONCLUSIONS: The present data suggest that compared with weight or BMI, AMA could serve as an earlier indicator of a deteriorating nutritional status. The benefit of assessing skinfold thickness and arm circumference routinely and calculating AMA from these measurements should be evaluated in large, prospective, multi-centre studies.

Assessment of body composition in cystic fibrosis: agreement between skinfold measurement and densitometry.

Introduction: Introduction: few studies have evaluated body composition (BC) through different techniques, and the degree of agreement between them in adults with cystic fibrosis (CF). Objectives: to describe BC using techniques to assess nutritional status and to test their concordance in CF. Methods: a cross-sectional study in CF patients in a clinically stable situation. Nutritional assessment was performed using skinfold measurement (SM) and densitometry (DXA). Fat-free mass index (FFMI) was also determined. The diagnosis of malnutrition was established if body mass index (BMI) < 18.5 kg/m2. Fat-free mass (FFM) malnutrition was diagnosed when FFMI was < 17 kg/m2 in males and < 15 kg/m2 in females (FFMI: fat-free mass in kg/height in m2). Results: forty-one patients were studied (twenty-two females, 53.7 %); median age was 29.8 (interquartile range, 20.9-33.7); BMI was 21.6 (19.8-23.0). Only four (9.8 %) patients had a BMI < 18.5. By DXA, FFM (kg) results were: median, 52.8 (47.8-56.9) with FFMI of 17.9 (16.7-19.3) in males and 36.7 (33.1-38.9) in females, FFMI of 14.7 (14.2-15.8). Twenty (48.6 %) patients presented FFM malnutrition, with 16.7 % of males and 59.1 % of females being affected. By SM, the FFMI was 18.7 (17.2-20.0) in males and 14.9 (14.2-15.8) in females; moreover, sixteen (39.1 %) patients presented malnutrition of FFM, with 20.8 % of males and 61.8 % of females being affected. For FFM (kg), a high concordance was obtained between SM and DXA (intraclass correlation coefficient of 0.950); likewise when they were compared by applying the ESPEN criteria for FFM malnutrition. However, when the techniques were compared to classify malnutrition according to FFMI, the kappa coefficient was only moderate (k = 0.440). The mean difference between FFM by DXA and SM was +1.44 ± 0.62 kg in favor of SM, with greater dispersion as FFM increased. Conclusions: the prevalence of FFM malnutrition is high in adult CF patients, despite a normal BMI, especially in females. Notwithstanding the good statistical agreement between SM and DXA, concordance was moderate. Therefore, DXA remains the technique of choice, and SM may be used when the former is not available.

Introducción: Introducción: pocos estudios han evaluado la composición corporal (BC) mediante diferentes técnicas y el grado de concordancia entre ellas en adultos con fibrosis quística (FQ). Objetivos: describir la BC mediante técnicas de evaluación nutricional y comprobar su concordancia en la FQ. Métodos: estudio transversal de adultos con FQ en situación de estabilidad clínica. La evaluación nutricional se realizó mediante medición de pliegues cutáneos (SM) y densitometría (DXA). También se determinó el índice de masa libre de grasa (FFMI). El diagnóstico de desnutrición se estableció si el índice de masa corporal era < 18,5 kg/m2. Se diagnosticó desnutrición por masa libre de grasa (FFM) cuando el FFMI era < 17 kg/m2 en 4 hombres y < 15 kg/m2 en mujeres (FFMI: masa libre de grasa en kg/estatura en m2). Resultados: se estudiaron 41 pacientes (22 mujeres (53,7 %), con una edad media de 29,8 años (rango intercuartílico, 20,9-33,7) e IMC de 21,6 (19,8-23,0). Solo 4 (9,8 %) pacientes tenían un IMC < 18,5. Mediante DXA, los resultados de FFM (kg) fueron (mediana y RIC): 52,8 (47,8-56,9) con FFMI de 17,9 (16,7-19,3) en los varones y 36,7 (33,1-38,9) en las mujeres con FFMI de 14,7 (14,2-15,8). Veinte (48,6 %) pacientes presentaban desnutrición del FFM, con el 16,7 % de varones y el 59,1 % de mujeres afectados. Mediante el SM, el FFMI fue de 18,7 (17,2-20,0) en los varones y de 14,9 (14,2-15,8) en las mujeres. En el caso de la FFM (kg), se obtuvo una alta concordancia entre el SM y la DXA (coeficiente de correlación intraclase de 0,950); igualmente cuando se compararon las técnicas aplicando los criterios ESPEN para la desnutrición de la FFM. Sin embargo, cuando se compararon las técnicas para clasificar la malnutrición según el FFMI, el coeficiente kappa fue solo moderado (coeficiente kappa = 0,440). La diferencia media entre el FFM por DXA y el SM fue de +1,44 ± 0,62 kg a favor del SM, con mayor dispersión a medida que aumenta el FFM. Conclusiones: la prevalencia de la malnutrición por FFM es elevada en pacientes adultos con FQ, a pesar de presentar un IMC normal, especialmente en el caso de las mujeres. A pesar de existir una buena correlación estadística entre el SM y la DXA, la concordancia fue moderada. Por lo tanto, la DXA sigue siendo la técnica de elección y el SM puede ser una alternativa cuando la DXA no esté disponible.

Sleep assessment in cystic fibrosis patients using a minimal-impact biomotion system.

PURPOSE: In our study we aimed to analyze sleep variability and activity in patients with cystic fibrosis (CF) during their hospital stay. METHODS: Forty-three CF patients were recruited and have been divided into two subgroups: exacerbated (n = 18) and non-exacerbated (n = 25). During the course of their hospital stay we used VitaLog, a minimal-impact biomotion device, in order to determine total sleep time (TST), time in bed (TIB), sleep efficiency (SE) and intra patient standard deviation (IPSD) of TST. RESULTS: TST was 5.1 h ± 1.5 h and ranged from 0.6h to 7.9 h.TIB was 17.7 h ± 3.8 h and ranged from 5.6h to 23.9 h. SE was 70.0% ± 17.0% and ranged from 13.6% to 98.5%. TST was higher in non-exacerbated patients (5.3 h ± 1.4 h vs. 4.8 h ± 1.6 h, p = 0.008) whereas TIB was lower in non-exacerbated patients (17.0 h ± 3.7 h vs. 18.5 h ± 3.8 h, p = 0.002). We also found that SE was better in non-exacerbated patients (73.1% ± 14.6% vs. 66.6% ± 18.8%, p = 0.002). Furthermore, we observed that IPSD of TST was higher in exacerbated patients (1.3 h ± 0.5 h vs. 0.9 h ± 0.4 h, p = 0.004). CONCLUSION: In general, in CF patients TST was short and SE poor during the night. Furthermore, in the course of their hospital stay patients showed low activity. In exacerbated patients sleep quality was lower compared to non-exacerbated patients.

Characteristics and Outcomes of Children With Cystic Fibrosis Hospitalized With Cirrhosis in the United States.

INTRODUCTION: To describe the characteristics and outcomes of children with cystic fibrosis (CF) hospitalized with cirrhosis in the United States. METHODS: We conducted a population-based cohort study of hospitalizations among children with CF using the 2016 Kid's Inpatient Database. RESULTS: In total, 9,615 admissions were analyzed. Diagnosis of cirrhosis was present in 509 (5.3%) and was significantly associated with increased mortality, length of stay, and hospital charges compared with those without cirrhosis. Hepatic encephalopathy was significantly associated with death in children with cirrhosis. DISCUSSION: Future interventions should be designed to support children with CF who have cirrhosis to improve clinical outcomes.

Designing the GALAXY study: Partnering with the cystic fibrosis community to optimize assessment of gastrointestinal symptoms.

BACKGROUND: Gastrointestinal (GI) involvement among persons with cystic fibrosis (CF) is highly prevalent, representing a significant source of morbidity. Persons with CF have identified GI concerns as a top research priority, yet universal clinical outcome measures capturing many of the GI symptoms experienced in CF are lacking. The GALAXY study was envisioned to address this unmet need. METHODS: The GALAXY study team partnered with Community Voice, a community of patients with CF and their caregivers, to identify the patient reported outcome measures that most accurately reflected their experience with GI symptoms in CF. We also surveyed CF care teams to identify the comfort level of various team members (providers, nurses and dieticians) in managing a variety of GI conditions. RESULTS: Members of Community Voice identified the combination of PAC-SYM, PAGI-SYM, PAC-QOL and the Bristol Stool scale with three additional symptom-specific questions as patient-reported outcome measures that comprehensively captured the CF experience with GI disease. CF care team providers reported a high level of comfort in treating GI conditions including constipation (92%), GERD (93%), and gassiness (77%), however comfort level was limited to only first-line interventions. CONCLUSION: By partnering with persons with CF as well as their caregivers and medical providers, the GALAXY study is designed to uniquely capture the prevalence and severity of GI involvement among persons with CF in a manner that reflects the CF patient experience. The results of GALAXY will inform the development of future interventional trials and serve as a reproducible and objective study endpoint.

Longitudinal Assessment of Patients With Cystic Fibrosis Lung Disease With Multivolume Noncontrast MRI and Spirometry.

BACKGROUND: MRI has been suggested as a radiation-free imaging modality to investigate early structural alterations and regional functional impairment in cystic fibrosis (CF) lung disease. PURPOSE/HYPOTHESIS: To compare functional and morphological MRI changes over the course of the disease to changes in spirometry. STUDY TYPE: Longitudinal retrospective study. POPULATION: Twenty patients with CF lung disease (at baseline, age = 16.5 (13.3-20.6) years, forced expiratory volume in 1 second (as % of predicted [%pred]) FEV1 = 71 (59-87) %pred, forced expiratory flow at 25-75% of forced vital capacity FEF25-75 = 39 (25-63) %pred. FIELD STRENGTH/SEQUENCE: 1.5T / T2 -weighted HASTE; T2 -weighted TSE-PROPELLER; T2 -weighted bSSFP; T1 -weighted 3D GRE. ASSESSMENT: Nonenhanced chest MRI and spirometry were retrospectively collected over a 3-year period from the initial recruitment visit. Images acquired at end-inspiration and end-expiration were registered by software using the optical flow method to measure expiratory-inspiratory differences in MR signal-intensity (Δ1 H-MRI). Measures of CF functional impairment were defined from Δ1 H-MRI: Δ1 H-MRI median, Δ1 H-MRI quartile coefficient of variation (QCV), and percent low-signal-variation volume (LVV). MR images were also evaluated by three readers using a CF-specific scoring system. STATISTICAL TESTS: Spearman correlation analysis, Spearman rank correlation analysis, linear mixed-effect model analysis, intraclass correlation coefficient. RESULTS: Functional imaging parameters and total morphological score correlated with all spirometric measures, as did subscores of bronchial wall thickening/bronchiectasis, mucus plugging, and consolidation. Overall, the percent change of Δ1H-MRI median correlated with the percent change of FEV1 (ΔFEV1 , r = 0.41, P < 0.01) and the percent change of FEF25-75 (ΔFEF25-75%, r = 0.38, P < 0.01). The percent change of LVV correlated with ΔFEV1 (r = -0.47, P < 0.001) and ΔFEF25-75 (r = -0.50, P < 0.001). DATA CONCLUSION: These preliminary results suggest that nonenhanced multivolume MRI may provide a feasible tool to regionally map early pulmonary alterations for longitudinal evaluation of CF lung disease, without exposing the patients to ionizing radiation. LEVEL OF EVIDENCE: 3T TECHNICAL EFFICACY STAGE: 5.