Search

The reader will come to appreciate that: • Severe cystic fibrosis liver disease (CLFD) commonly presents in early childhood years, and affects up to 10 % of people with cystic fibrosis by age 30 years. • United States CF Foundation (USCFF) guidelines have recently further defined CF hepatobiliary involvement (CFHBI) and advanced CF liver disease (aCFLD). • Familiarity with Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulators monitoring guidelines is essential in clinical practice, particularly while their role in aCFLD is not established. Cystic fibrosis liver disease (CFLD) is characterised by a wide heterogenity of manifestations and severity. It represents a major cause of morbidity in people with cystic fibrosis (PwCF), which will be of increasing relevance as survival increases in the new era of cystic fibrosis care. No medical therapy currently available has evidence to treat or prevent progression of liver disease. Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulators may be transformative on pulmonary, nutritional and quality of life, but direct effect on long term liver disease outcomes is not yet established. Drug-associated hepatic adverse effects may be common, and clinician familiarity with drug-monitoring recommendations is essential. Longitudinal studies are required to understand the effect of CFTR modulators on the incidence and natural history of CFLD, including with early treatment initiation, in established advanced liver disease, and post liver transplantation. [ABSTRACT FROM AUTHOR]

To support investigation of the etiology and psychophysiology of medical traumatic stress, we developed a standardized set of emotionally-salient medical images, called the 'MEDical Image Collection' (MEDIC), for use in neuroimaging or psychological research. This study aimed to establish internal consistency, test re-test reliability, and congruent validity of the image set. A representative sample of 300 adults in the United States were recruited via research recruitment platform, Prolific. Participants rated 124 images depicting medical stimuli on one of two dimensions: emotional arousal (i.e., how strongly an evoked emotion is felt) or affective valence (i.e., how positive or negative the evoked emotion is). Sociodemographic and health-related characteristics, including experiences during the COVID-19 pandemic, were also assessed. To assess test re-test reliability, a subset (n = 200) rated the images on the same dimension a second time, 3 months later. The MEDIC image set was found to: (a) elicit a range of emotional arousal and valence ratings, (b) have excellent inter-rater reliability, (c) moderate test-retest reliability, and (d) good face validity. Results indicate the new MEDIC 124-image set is a reliable and valid instrument, enabling researchers to provide context-specific and emotionally-salient stimuli to individuals when studying affective responses in relation to health and medicine. • MEDIC is a newly developed image set (124 images) of medical stimuli for research into medical traumatic stress and anxiety. • MEDIC elicited a wide range of emotional arousal and valence ratings in a sample of 300 adults. • The MEDIC image set was found to have excellent inter-rater reliability and moderate test-retest reliability. • We demonstrated the sensitivity of the MEDIC image set to medical experiences. • MEDIC can be accessed for use by contacting the study team. [ABSTRACT FROM AUTHOR]

Objective: The aim of the study was to determine the rate of progression from acute recurrent pancreatitis (ARP) to chronic pancreatitis (CP) in children and assess risk factors., Study Design: Data were collected from the INternational Study group of Pediatric Pancreatitis: In search for a cuRE (INSPPIRE) cohort. Kaplan-Meier curves were constructed to calculate duration of progression from initial attack of acute pancreatitis (AP) to CP. Log-rank test was used to compare survival (nonprogression) probability distribution between groups. Cox proportional hazard regression models were fitted to obtain hazard ratio (with 95% confidence interval [CI]) of progression for each risk variable., Results: Of 442 children, 251 had ARP and 191 had CP. The median time of progression from initial attack of AP to CP was 3.79 years. The progression was faster in those ages 6 years or older at the first episode of AP compared to those younger than 6 years (median time to CP: 2.91 vs 4.92 years; P = 0.01). Children with pathogenic PRSS1 variants progressed more rapidly to CP compared to children without PRSS1 variants (median time to CP: 2.52 vs 4.48 years; P = 0.003). Within 6 years after the initial AP attack, cumulative proportion with exocrine pancreatic insufficiency was 18.0% (95% CI: 12.4%, 25.6%); diabetes mellitus was 7.7% (95% CI: 4.2%, 14.1%)., Conclusions: Children with ARP rapidly progress to CP, exocrine pancreatic insufficiency, and diabetes. The progression to CP is faster in children who were 6 years or older at the first episode of AP or with pathogenic PRSS1 variants. The factors that affect the aggressive disease course in childhood warrant further investigation.

Objective: To determine the clinical presentation, diagnostic variables, risk factors, and disease burden in children with chronic pancreatitis., Study Design: We performed a cross-sectional study of data from the International Study Group of Pediatric Pancreatitis: In Search for a Cure, a registry of children with acute recurrent pancreatitis and chronic pancreatitis. Between-group differences were compared using Wilcoxon rank-sum test., Results: Among 170 subjects in the registry, 76 (45%) had chronic pancreatitis; 57% were female, 80% were white; median age at diagnosis was 9.9 years. Pancreatitis-predisposing genetic mutations were identified in 51 (67%) and obstructive risk factors in 25 (33%). Toxic/metabolic and autoimmune factors were uncommon. Imaging demonstrated ductal abnormalities and pancreatic atrophy more commonly than calcifications. Fifty-nine (77%) reported abdominal pain within the past year; pain was reported as constant and receiving narcotics in 28%. Children with chronic pancreatitis reported a median of 3 emergency department visits and 2 hospitalizations in the last year. Forty-seven subjects (70%) missed 1 day of school in the past month as the result of chronic pancreatitis; 26 (34%) missed 3 or more days. Children reporting constant pain were more likely to miss school (P = .002), visit the emergency department (P = .01), and experience hospitalizations (P = .03) compared with children with episodic pain. Thirty-three children (43%) underwent therapeutic endoscopic retrograde pancreatography; one or more pancreatic surgeries were performed in 30 (39%)., Conclusions: Chronic pancreatitis occurs at a young age with distinct clinical features. Genetic and obstructive risk factors are common, and disease burden is substantial., (Copyright © 2015 Elsevier Inc. All rights reserved.)

Background: The American and European cystic fibrosis (CF) guidelines recommend different diagnostic criteria. This study assessed diagnostic concordance between these recommendations., Methods: Subjects with single organ manifestations suggestive of CF (chronic sinopulmonary disease (RESP), chronic/recurrent pancreatitis (PANC) or obstructive azoospermia (AZOOSP)) were prospectively evaluated by sweat test, nasal potential difference and genotyping. Concordance in diagnostic outcomes between the two algorithms was measured using observed agreement and κ statistics., Results: A total of 208 subjects were evaluated. Observed agreement was 84.8% and level of agreement was excellent (κ=0.87) between the American and European recommendations. The RESP phenotype was associated with the highest degree of concordance (observed agreement ≥90%, κ=0.92) compared with the PANC (observed agreement 86%, κ=0.65) and AZOOSP (observed agreement 80%, κ=0.87) phenotypes. Incorporation of nasal potential difference into the American algorithm failed to improve the overall degree of concordance (good agreement level; κ=0.75); the level of agreement was unchanged in RESP and PANC subjects, but reduced in AZOOSP subjects (from excellent to good). Extensive genotyping had limited clinical utility in the diagnosis of CF in both algorithms., Conclusions: Despite inconsistencies between the American and European diagnostic recommendations, concordance in diagnostic outcomes among subjects presenting with single organ manifestations of CF was good to excellent. These diagnostic guidelines provide guidance and promote rigorous evaluation for the diagnosis of CF but neither guideline should be regarded as dogma.