This clinical report represents the first English-language recommendations for the management of pediatric AP. Future aims should include prospective multicenter pediatric studies to further validate these recommendations and optimize care for children with AP.
The vast majority of costs allocated to out-patient IBD medications in the USA is attributed to increasing use of biologic therapies despite the relative minority of biologic-taking patients.
We performed a comprehensive epidemiologic analysis of privately insured, non-elderly adults and children with AP and CP in the United States. Changes in gallstone formation, smoking, and alcohol consumption, along with advances in pancreatitis management, may be responsible for the stabilization and even decrease in the incidences of AP and CP.
Highlights d Cas9 RNP and AAV can be used to efficiently gene edit human airway basal stem cells d This method yields >30% allelic correction without selection markers or antibiotics d Correction of >30% DF508 alleles restores CFTR function to near non-CF levels d Corrected stem cells can differentiate after embedding in the scaffold for engraftment
Objectives Cysteamine prevents organ damage in children with cystinosis, but may cause gastrointestinal (GI) symptoms.In this study we evaluated the nature of GI disease, and the value of omeprazole in controlling GI symptoms in these children.Study design Upper GI disease was evaluated with endoscopy, gastrin levels, and acid secretion studies after oral administration of cysteamine, before and after 16 weeks of therapy with omeprazole. A symptom score was devised.Results Eleven children (mean age, 5.7 years) were studied. After cysteamine ingestion, before and after omeprazole therapy, the mean maximum acid output was significantly higher than the mean basal acid output. The maximum acid output was measured within 60 minutes of cysteamine ingestion and was reduced by omeprazole therapy (P < .01). The mean peak gastrin level was 30 minutes postcysteamine and was higher than baseline (P < .01). The initial mean symptom score (maximum score, 14) was 6.9 and fell to 0.7 (P < .0001) after 16 weeks of omeprazole therapy. At endoscopy, two children had diffuse gastric nodularity, and nearly all had cystine crystal deposits.Conclusions GI symptoms in children with cystinosis receiving cysteamine are often acid-mediated and improve with omeprazole. Cystine crystals were detected in the GI tract and may signify inadequate treatment with cysteamine. (J Pediatr 2003;143:224-30)
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