Chest. 2021 Jun 17:S0012-3692(21)01127-2. doi: 10.1016/j.chest.2021.06.017. Online ahead of print.

ABSTRACT

Cystic Fibrosis (CF) is an autosomal recessive genetic condition with multi-systemic disease manifestations, the most prominent of which occur in the respiratory system. Despite significant developments in disease understanding and therapeutics, each contributing to improved lung function and survival in persons with CF, several pulmonary complications including pneumothorax, massive hemoptysis, and respiratory failure continue to occur. In this review, we briefly describe each of these complications and their management, as well as discuss how they impact the care and disease trajectory of individuals in whom they occur. Finally, we discuss the evolving role that palliative care and cystic fibrosis transmembrane conductance regular modulator therapies play in the natural disease course and care of persons with CF.

PMID:34147501 | DOI:10.1016/j.chest.2021.06.017

J Cyst Fibros. 2021 Jun 16:S1569-1993(21)00173-9. doi: 10.1016/j.jcf.2021.05.016. Online ahead of print.

ABSTRACT

Gastrointestinal manifestations in patients with cystic fibrosis (CF) are extremely common and have recently become a research focus. Gastrointestinal (GI) dysfunction is poorly understood in the CF population, despite many speculations including the role of luminal pH, bacterial overgrowth, and abnormal microbiome. Nevertheless, dysmotility is emerging as a possible key player in CF intestinal symptoms. Our review article aims to explore the sequelae of defective cystic fibrosis transmembrane conductance regulator (CFTR) genes on the GI tract as studied in both animals and humans, describe various presentations of intestinal dysmotility in CF, review newer diagnostic motility techniques including intraluminal manometry, and review the current literature regarding the potential role of dysmotility in CF-related intestinal pathologies.

PMID:34147362 | DOI:10.1016/j.jcf.2021.05.016

Indian J Med Res. 2020 Dec;152(6):575-583. doi: 10.4103/ijmr.IJMR_906_18.

ABSTRACT

BACKGROUND & OBJECTIVES: Due to limited information available on the frequency and spectrum of cystic fibrosis (CF) transmembrane conductance regulator (CFTR) gene mutations in congenital bilateral absence of vas deferens (CBAVD) in Indian population, it is difficult to provide accurate genetic counselling to couples. The present study was undertaken to investigate the spectrum and frequency of CFTR gene mutations in Indian men with CBAVD and to determine the female CF carrier status.

METHODS: Direct DNA sequencing of the CFTR gene was carried out in eighty CBAVD men, their female partners and fifty controls from the general population. Pathological significance of the identified novel CFTR gene variants was carried out using in silico tools. Appropriate genetic counselling was provided to the couples prior to intracytoplasmic sperm injection (ICSI).

RESULTS: A significant association was observed for CFTR gene variants in Indian CBAVD men versus controls (odds ratio: 12.1; 95% confidence interval: 4.8-30.4; P<0.0001). A total of 20 CFTR gene variants were identified in 53 CBAVD men. Eight novel missense CFTR gene variants (L214V, A238P, E379V, L578I, F587L, L926W, R1325K and R1453Q); two novel splice-site gene variants (c.1-30C>G and IVS1+2T>G) and ten previously reported mutations (R75Q, c.1210-12[5], F508del, A309G, R334W, I444T, R668C, R709X, A1285V and Q1352H) were detected in CBAVD men. The novel and reported CFTR gene mutations were L926W (2.5%, P=0.26), R1453Q (2.5%, P=0.26), F508del (8.75%, P=0.03) and c.1210-12[5] (42.5%, P=0.002). A total of 13 (16.2%) female partners were found to be a CF carrier. Nine couples had a risk of transmitting mutant CFTR allele to the offspring.

INTERPRETATION & CONCLUSIONS: The heterogeneous spectrum of CFTR gene in Indian population suggests the necessity of screening CBAVD men and female partners for accurate genetic counselling prior to undergoing ICSI.

PMID:34145097 | DOI:10.4103/ijmr.IJMR_906_18

J Cyst Fibros. 2021 Jun 15:S1569-1993(21)00164-8. doi: 10.1016/j.jcf.2021.05.013. Online ahead of print.

ABSTRACT

BACKGROUND: . In CLEAR-108-a phase 3, randomised, open-label study-once-daily amikacin liposome inhalation suspension (ALIS) was noninferior to twice-daily tobramycin inhalation solution (TIS) in improving lung function in patients with cystic fibrosis (CF) and chronic Pseudomonas aeruginosa infection after 3 treatment cycles (28 days on/28 days off). The CLEAR-110 extension study (ClinicalTrials.gov: NCT01316276; EudraCT: 2011-000443-24) assessed long-term safety, tolerability, and efficacy of ALIS in eligible patients who completed CLEAR-108.

METHODS: . Patients received once-daily ALIS 590 mg for 12 treatment cycles (96 weeks). Patients were grouped by prior treatment: the "prior-ALIS" cohort received ALIS in CLEAR-108, and the "ALIS-naive" cohort received TIS in CLEAR-108.

RESULTS: . Overall, 206 patients (prior-ALIS, n=92; ALIS-naive, n=114) entered CLEAR-110 and received ≥1 dose of ALIS. Most patients (88.8%) experienced ≥1 treatment-emergent adverse event (TEAE) through day 672 (end of year 2). Most TEAEs (72.3%) were mild or moderate in severity. Severe TEAEs were reported in 31 patients (15.0%). Two life-threatening TEAEs (haemoptysis; intestinal obstruction) and 1 death (cardiac failure) were reported. Twenty-one patients (10.2%) discontinued treatment due to a TEAE (mostly infective pulmonary exacerbation of CF). Mean change from baseline in forced expiratory volume in 1 second percent predicted at day 672 was -3.1% (prior-ALIS, -4.0%; ALIS-naive, -2.3%). Mean change from baseline in sputum density of P. aeruginosa at day 672 was 0.02 (prior-ALIS, -0.16; ALIS-naive, 0.19) log CFU/g.

CONCLUSIONS: . Long-term treatment with ALIS was well tolerated with a favourable adverse event profile and demonstrated continued antibacterial activity in CF patients with chronic P. aeruginosa infection.

PMID:34144923 | DOI:10.1016/j.jcf.2021.05.013

Radiographics. 2021 Jun 18:200164. doi: 10.1148/rg.2021200164. Online ahead of print.

ABSTRACT

A wide spectrum of hereditary syndromes predispose patients to distinct pancreatic abnormalities, including cystic lesions, recurrent pancreatitis, ductal adenocarcinoma, nonductal neoplasms, and parenchymal iron deposition. While pancreatic exocrine insufficiency and recurrent pancreatitis are common manifestations in cystic fibrosis and hereditary pancreatitis, pancreatic cysts are seen in von Hippel-Lindau disease, cystic fibrosis, autosomal dominant polycystic kidney disease, and McCune-Albright syndrome. Ductal adenocarcinoma can be seen in many syndromes, including Peutz-Jeghers syndrome, familial atypical multiple mole melanoma syndrome, Lynch syndrome, hereditary breast and ovarian cancer syndrome, Li-Fraumeni syndrome, and familial pancreatic cancer syndrome. Neuroendocrine tumors are commonly seen in multiple endocrine neoplasia type 1 syndrome and von Hippel-Lindau disease. Pancreatoblastoma is an essential component of Beckwith-Wiedemann syndrome. Primary hemochromatosis is characterized by pancreatic iron deposition. Pancreatic pathologic conditions associated with genetic syndromes exhibit characteristic imaging findings. Imaging plays a pivotal role in early detection of these conditions and can positively affect the clinical outcomes of those at risk for pancreatic malignancies. Awareness of the characteristic imaging features, imaging-based screening protocols, and surveillance guidelines is crucial for radiologists to guide appropriate patient management. ©RSNA, 2021.

PMID:34143711 | DOI:10.1148/rg.2021200164

Pediatr Pulmonol. 2021 Jun 18. doi: 10.1002/ppul.25538. Online ahead of print.

ABSTRACT

BACKGROUND: The goal of this study was to identify clinical features associated with abnormal infant pulmonary function tests (iPFTs), specifically functional residual capacity (FRC), in infants with cystic fibrosis (CF) diagnosed via newborn screen (NBS). We hypothesized that poor nutritional status in the first 6-12 months would be associated with increased FRC at 12-24 months.

METHODS: This study utilized a combination of retrospectively and prospectively collected data from ongoing research studies and iPFTs performed for clinical indications. Demographic and clinical features were obtained from the electronic medical record. Forced expiratory flows and volumes were obtained using the raised volume rapid thoracoabdominal technique (RVRTC) and FRC was measured via plethysmography.

RESULTS: A total of 45 CF NBS infants had iPFTs performed between 12-24 months. Mean forced vital capacity, forced expiratory volume in 0.5 second, and forced expiratory flows were all within normal limits. In contrast, the mean FRC z-score was 2.18 (95%CI=1.48, 2.88) and the mean respiratory rate (RR) z-score was 1.42 (95%CI=0.95, 1.89). There was no significant association between poor nutritional status and abnormal lung function. However, there was a significant association between higher RR and increased FRC, and a RR cutoff of 36 breaths/min resulted in 92% sensitivity to detect hyperinflation with 32% specificity.

CONCLUSIONS: These results suggest that FRC is a more sensitive measure of early CF lung disease than RVRTC measurements and that RR may be a simple, non-invasive clinical marker to identify CF NBS infants with hyperinflation. This article is protected by copyright. All rights reserved.

PMID:34143539 | DOI:10.1002/ppul.25538

Stat Methods Med Res. 2021 Jun 18:9622802211023975. doi: 10.1177/09622802211023975. Online ahead of print.

ABSTRACT

Proportional rates models are frequently used for the analysis of recurrent event data with multiple event categories. When some of the event categories are missing, a conventional approach is to either exclude the missing data for a complete-case analysis or employ a parametric model for the missing event type. It is well known that the complete-case analysis is inconsistent when the missingness depends on covariates, and the parametric approach may incur bias when the model is misspecified. In this paper, we aim to provide a more robust approach using a rate proportion method for the imputation of missing event types. We show that the log-odds of the event type can be written as a semiparametric generalized linear model, facilitating a theoretically justified estimation framework. Comprehensive simulation studies were conducted demonstrating the improved performance of the semiparametric method over parametric procedures. Multiple types of Pseudomonas aeruginosa infections of young cystic fibrosis patients were analyzed to demonstrate the feasibility of our proposed approach.

PMID:34142905 | DOI:10.1177/09622802211023975

Chron Respir Dis. 2021 Jan-Dec;18:14799731211017895. doi: 10.1177/14799731211017895.

ABSTRACT

What is the impact of including an allied health assistant (AHA) role on physiotherapy service delivery in an acute respiratory service? A pragmatic pre-post design study examined physiotherapy services across two 3-month periods: current service delivery [P1] and current service delivery plus AHA [P2]. Clinical and non-clinical activity quantified as number, type and duration (per day) of all staff activity categorised for skill level (AHA, junior, senior). Physiotherapy service delivery increased in P2 compared to P1 (n = 4730 vs n = 3048). Physiotherapists undertook fewer respiratory (p < 0.001) and exercise treatments (p < 0.001) but increased reviews for inpatients (p < 0.001) and at multidisciplinary clinics in P2 (56% vs 76%, p < 0.01). The AHA accounted for 20% of all service provision. AHA activity comprised mainly non-direct clinical care including oversight of respiratory equipment use (e.g. supply, set-up, cleaning, loan audits) and other patient-related administrative tasks associated with delegation handovers, supervision and clinical documentation (72%), delegated supervision of established respiratory (5%) and exercise treatments (10%) and delegated exercise tests (3%). The AHA completed most of the exercise tests (n = 25). AHA non-direct clinical tasks included departmental management activities (11%). No adverse events were reported. AHA inclusion in an acute respiratory care service changed physiotherapy service provision. The AHA completed delegated routine clinical and non-clinical tasks. Physiotherapists increased clinic activity and annual reviews. Including an AHA role offers sustainable options for enhancing physiotherapy service provision in acute respiratory care.

PMID:34142582 | DOI:10.1177/14799731211017895

iScience. 2021 May 15;24(6):102542. doi: 10.1016/j.isci.2021.102542. eCollection 2021 Jun 25.

ABSTRACT

Ivacaftor (VX-770) was the first cystic fibrosis transmembrane conductance regulator (CFTR) modulatory drug approved for the treatment of patients with cystic fibrosis. Electron cryomicroscopy (cryo-EM) studies of detergent-solubilized CFTR indicated that VX-770 bound to a site at the interface between solvent and a hinge region in the CFTR protein conferred by transmembrane (tm) helices: tm4, tm5, and tm8. We re-evaluated VX-770 binding to CFTR in biological membranes using photoactivatable VX-770 probes. One such probe covalently labeled CFTR at two sites as determined following trypsin digestion and analysis by tandem-mass spectrometry. One labeled peptide resides in the cytosolic loop 4 of CFTR and the other is located in tm8, proximal to the site identified by cryo-EM. Complementary data from functional and molecular dynamic simulation studies support a model, where VX-770 mediates potentiation via multiple sites in the CFTR protein.

PMID:34142049 | PMC:PMC8184517 | DOI:10.1016/j.isci.2021.102542

Ann Med Surg (Lond). 2021 May 27;66:102449. doi: 10.1016/j.amsu.2021.102449. eCollection 2021 Jun.

ABSTRACT

INTRODUCTION AND IMPORTANCE: Congenital unilateral absence of the vas deferens and ipsilateral renal agenesis is a rare condition in which the vas deferens is absent unilateral due to a congenital anomaly. This report illustrates the clinical relevance of absence of the vasa deferentia. This occurs either unilateral, which is associated with renal anomalies, or bilateral which leads to azoospermia and often has a genetic link to cystic fibrosis.

CASE PRESENTATION: We report the first incidental finding of congenital unilateral absence of the vas deferens and ipsilateral renal agenesis during bilateral laparoscopic totally extraperitoneal inguinal hernia repair in a 70 year old patient.

CONCLUSION: Surgeons who encounter unilateral absence of the vas deferens should assess bilateral absence of the vasa deferentia by palpation in adult patients or by laparoscopic exploration of the contralateral inguinal canal in pediatric patients. Renal ultrasonography should be performed in all patients to assess renal anomalies. Genetic counselling is advised when absence of the vasa deferentia is present bilaterally of unilaterally with associated infertility.

PMID:34141420 | PMC:PMC8184504 | DOI:10.1016/j.amsu.2021.102449

ACS Med Chem Lett. 2021 May 18;12(6):949-950. doi: 10.1021/acsmedchemlett.1c00260. eCollection 2021 Jun 10.

NO ABSTRACT

PMID:34141077 | PMC:PMC8201489 | DOI:10.1021/acsmedchemlett.1c00260

BMJ Support Palliat Care. 2021 Jun 17:bmjspcare-2021-003019. doi: 10.1136/bmjspcare-2021-003019. Online ahead of print.

ABSTRACT

BACKGROUND: Children with a life-limiting condition often require extensive and complex care, much of which is provided by their parents at home. There is a growing body of research that aims to understand the experiences of these parents, but the majority of this research is from mothers' perspectives, meaning that fathers' experiences are not well understood.

OBJECTIVES: To identify and synthesise findings from existing qualitative studies that have explored the experiences of fathers of children with a life-limiting condition.

METHODS: A systematic review of qualitative research was conducted using thematic synthesis. Searches were conducted in MEDLINE, CINAHL, EMBASE, PsycINFO and Social Science Citation Index.

RESULTS: Findings from 30 studies were included, representing the experiences of 576 fathers of children with a range of diagnoses including cancer, cystic fibrosis, genetic and neurological conditions. Themes detailed fathers' experiences of uncertainty and shock around the time of their child's diagnosis, their accounts of a 'new normal', difficulties in discussing their emotions, forming relationships with and seeking support from professionals and working fathers' role conflicts. They discussed the life-changing nature of their child's diagnosis, an event that affected all aspects of their lives from everyday activities, to their relationships, spirituality, values and ambitions.

CONCLUSIONS: Fathers experience many difficulties in response to their child's diagnosis and ongoing treatment. Findings highlight the need for healthcare professionals to recognise individual family dynamics and the evolving role of the father. Fathers' responses are not widely understood, and research that directly addresses their own well-being is warranted.

PMID:34140322 | DOI:10.1136/bmjspcare-2021-003019

Eur Respir J. 2021 Jun 17:2100014. doi: 10.1183/13993003.00014-2021. Online ahead of print.

ABSTRACT

INTRODUCTION: France implemented a high emergency lung transplantation (HELT) program nationally in 2007. A similar program does not exist in Canada. The objectives of our study were to compare health outcomes within France as well as between Canada and France before and after the HELT program in a population with Cystic Fibrosis (CF).

METHODS: This population-based cohort study utilised data from the French and Canadian CF registries. A cumulative incidence curve assessed time to transplant with death without transplant as competing risks. The Kaplan-Meier method was used to estimate post-transplant survival.

RESULTS: Between 2002 and 2016, there were 1075 (13.0%) people with CF in France and 555 (10.2%) people with CF in Canada who underwent lung transplantation. The proportion of lung transplant increased in France after the HELT program was initiated (4.5% versus 10.1%) whereas deaths pre-transplant decreased from 85.3% in the pre-HELT to 57.1% in the post-HELT period. Between 2008-2016, people in France were significantly more likely to receive a transplant (Hazard Ratio (HR) 1.56, 95% CI 1.37-1.77, p<0.001) than die (HR 0.55, 95% CI 0.46-0.66, p<0.001) compared to Canada. Post-transplant survival was similar between the countries and there was no difference in survival when comparing pre- and post-HELT period in France.

CONCLUSION: Following the implementation of the HELT program, people living with CF in France were more likely to receive a transplant than die. Post-transplant survival in the post-HELT period in France did not change compared to the pre-HELT period, despite potentially sicker patients being transplanted, and is comparable to Canada.

PMID:34140297 | DOI:10.1183/13993003.00014-2021

An Pediatr (Engl Ed). 2021 Jun 14:S2341-2879(21)00101-0. doi: 10.1016/j.anpede.2020.04.022. Online ahead of print.

ABSTRACT

INTRODUCTION: Neonatal cystic fibrosis (CF) screening has enabled the disease to be diagnosed early, and is a determining factor in the increase in survival of these patients. Its main disadvantage is its low specificity and elevated number of false positives. The aim of this study is to analyse the differences in immunoreactive trypsin (IRT) between the different groups of newborns (NB) with a positive neonatal screen depending on whether they were healthy, healthy carriers, affected by CF, or CFSPID (Cystic Fibrosis Screen Positive, Inconclusive Diagnosis).

MATERIAL: Retrospective analytical study of the concentrations of IRT in NB with a positive neonatal screen for CF born in a tertiary hospital during an 8-year period.

RESULTS: A total of 790 NB with a positive neonatal screen for CF were analysed. Of these 86.3% were term, 53% girls, and 11.8% were admitted. The mean IRT value was 79.16 ng/mL (range 60-367). Significantly higher concentrations of IRT were found in those affected by CF compared to the other groups (P < .001). There were higher levels in large prematures (P = .007) and admitted patients (P = .002). There were no differences as regards gender or season. There was a direct correlation of 64% (P = .001) between IRT and sweat test in those affected by CF and CFSPID. A cut-off value of IRT for the diagnosis of CF was calculated from the ROC curve (76.2 ng/mL (S = 95.7%, Sp = 64.5%).

CONCLUSIONS: NB with CF have significantly higher levels of IRT than healthy ones, or carriers and CFSPID. Prematurity and hospital admission may also have an influence. A higher IRT value is associated with a higher level in the sweat test.

PMID:34140271 | DOI:10.1016/j.anpede.2020.04.022

J Cyst Fibros. 2021 Jun 14:S1569-1993(21)00162-4. doi: 10.1016/j.jcf.2021.05.011. Online ahead of print.

ABSTRACT

BACKGROUND: Non-invasive ventilation (NIV) is an established treatment option for cystic fibrosis (CF) patients with type 2 respiratory failure but the benefits of this therapy remain unclear. This study examined the long-term outcomes and response to NIV in a large adult CF cohort.

METHODS: All patients attending a UK adult CF Centre receiving NIV as treatment for hypercapnic respiratory failure over a nine-year period were studied prospectively. Detailed clinical data was recorded and longitudinal data measurements were examined for the three years pre and post NIV initiation to assess effect of this intervention.

RESULTS: 94 patients, mean age 29.9 (SD 9.7) years, percent predicted FEV1 21.5 (7.3), received NIV. All patients commenced NIV in a hospital setting. 21 remain alive, 24 received double lung transplant, 49 died without lung transplantation. NIV use was associated with a stabilisation and improvement in both FEV1 and FVC from NIV set up to three years post follow-up, in addition to an increase in body mass index and attenuation of PCO2 (all p<0.001). No single parameter was found to predict long-term NIV response but baseline PCO2 (p=0.005), CRP (p=0.004) and age (p=0.009) were identified as independent predictors of mortality.

CONCLUSIONS: NIV use in CF adults is associated with improvements in lung function and attenuation of hypercapnia which is maintained for up to three years post NIV initiation. Outcomes for CF patients with severe pulmonary disease commenced on NIV have significantly improved with fifty percent of patients expected to survive for approximately five years.

PMID:34140250 | DOI:10.1016/j.jcf.2021.05.011

J Cyst Fibros. 2021 Jun 14:S1569-1993(21)00126-0. doi: 10.1016/j.jcf.2021.04.016. Online ahead of print.

ABSTRACT

Cystic fibrosis is a genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, leading to dysfunction of the CFTR protein. CFTR dysfunction leads to disease in the respiratory and gastrointestinal systems. Disorders of the cardiovascular system in individuals with CF are usually attributed to secondary effects from progressive lung disease. However, CFTR has been localized to vascular endothelium and smooth muscle, suggesting that CFTR dysfunction may directly impact cardiovascular function. As treatments for CF improve and life-expectancy increases, the risk of vascular disease may increase in prevalence related to primary and secondary CFTR dysfunction, chronic systemic inflammation, nutritional health and hyperglycemia in individuals with CF related diabetes. Here we review the available literature on CF and the cardiovascular system, examining the secondary effects and evidence for direct CFTR dysfunction in the heart, aorta, pulmonary vessels, and vasculature, as well as future directions and treatment options.

PMID:34140249 | DOI:10.1016/j.jcf.2021.04.016

J Clin Endocrinol Metab. 2021 Jun 17:dgab442. doi: 10.1210/clinem/dgab442. Online ahead of print.

NO ABSTRACT

PMID:34139767 | DOI:10.1210/clinem/dgab442

Respir Med. 2021 Jun 9;185:106505. doi: 10.1016/j.rmed.2021.106505. Online ahead of print.

ABSTRACT

BACKGROUND: Serum biomarkers associated with the severity of non-cystic fibrosis (CF) bronchiectasis are insufficient. This study determined the association of serum hepatocyte growth factor (HGF), osteopontin, and pentraxin-3 levels with disease severity and exacerbation in patients with non-CF bronchiectasis.

METHODS: Serum levels of HGF, osteopontin, and pentraxin-3 were measured in patients with clinically stable non-CF bronchiectasis (n = 61). The correlation between the biomarkers and bronchiectasis severity index (BSI) and FACED score was assessed using univariate and multivariate linear regression analyses. Predictive variables associated with exacerbation were analyzed using a Cox proportional hazards model and the time to first exacerbation in high and low HGF groups during the observation period was compared using Kaplan-Meier survival curves.

RESULTS: The BSI showed significant correlation with HGF (r = 0.423; p = 0.001) and pentraxin-3 (r = 0.316; p = 0.013). The FACED score was significantly correlated with HGF (r = 0.406; p = 0.001). Univariate and multivariate linear regression analysis revealed that serum level of HGF was independently associated with both scoring systems. The high HGF group showed a significantly shorter time to first exacerbation (Log-rank test, p = 0.014). Multivariate Cox proportional hazards regression analysis revealed that high serum HGF level and colonization with non-pseudomonas organisms were independent predictors of future exacerbations (HR 2.364; p = 0.024 and HR 2.438; p = 0.020, respectively).

CONCLUSION: Serum level of HGF is a potential biomarker that is closely associated with disease severity and future risk of exacerbations in patients with non-CF bronchiectasis.

PMID:34139579 | DOI:10.1016/j.rmed.2021.106505

Pediatr Pulmonol. 2021 Jun 17. doi: 10.1002/ppul.25537. Online ahead of print.

ABSTRACT

BACKGROUND: Little is known about the impact of COVID-19 and the United Kingdom's (UK) national shielding advice on people with cystic fibrosis (CF) and their families. This study explored the experiences and support needs of children and young adults (CYAs) with CF, and parents who have a child with CF during the COVID-19 pandemic.

METHODS: CYAs with CF and parents of CYAs with CF completed a UK wide online survey with open and closed questions exploring experiences, information and support needs and decision-making processes. Qualitative thematic content analysis and descriptive quantitative analyses were undertaken.

RESULTS: CYAs aged 10-30 years (n=99) and parents of CYAs aged 0-34 years (n=145) responded. Parents (72.7%) and CYAs (50.0%) worried about the virus, and both were vigilant for virus symptoms (82.7% and 79.7%). Over three-quarters of CYAs were worried about their own health if they caught the virus. CYAs worried about feeling more isolated during the virus (64.9%). Qualitative findings reported the following themes: 1) Disruption- caused by isolation 2) Impact on psychological wellbeing 3) Safety of Shielding and 4) Healthcare and Treatment provision- changes to care, access and support.

CONCLUSIONS: The impact of COVID-19 and UK shielding advice to have no contact with anyone outside the household caused disruption to the lives and routines of individuals in relation to work, education, social lives, relationships, CF management routines and support. Parents and CYAs highlighted the need for clear, up-to-date and tailored advice on individualised risks and shielding. This article is protected by copyright. All rights reserved.

PMID:34138526 | DOI:10.1002/ppul.25537

Pediatr Pulmonol. 2021 Jun 17. doi: 10.1002/ppul.25535. Online ahead of print.

ABSTRACT

BACKGROUND: Adverse health impacts of cystic fibrosis (CF) can be present in children before respiratory complications are observed. Children with CF show progressive health decline, with increasing lung function decline in adolescence. This study aims to quantify the healthcare resource utilization (HCRU) and costs attributable to CF by comparing children with CF with the general pediatric population.

METHODS: This retrospective, cross-sectional, observational study compared HCRU and costs among children with CF in the US with demographically similar children without CF (comparison group) over a 12-month period using administrative claims data spanning 2010-2017. Analyses were conducted by insurance type (commercially insured [COM] and Medicaid insured [MED]) and stratified by age (<2 years, 2 to <6 years, 6 to <12 years, 12-17 years).

RESULTS: Children with CF (2,831 COM and 1,896 MED) were matched to children in the comparison group (8,493 COM and 5,688 MED). Higher prevalence of comorbidities was seen in children with CF vs the comparison group across all ages. Across all ages, HCRU attributable to CF was substantial (higher hospitalization rates, more outpatient and emergency room visits, and greater use of prescription medications), and there were higher associated costs (all P values <0.05), in COM and MED populations. HCRU and costs attributable to CF were highest for children aged 12-17 years.

CONCLUSIONS: Substantial HCRU and costs are evident among children with CF across all ages, starting as young as infancy, with highest HCRU and costs among adolescents. Effective treatments from an early age are needed for children with CF. This article is protected by copyright. All rights reserved.

PMID:34138523 | DOI:10.1002/ppul.25535