Journal article
Accelerating the Drug Delivery Pipeline for Acute and Chronic Pancreatitis: Summary of the Working Group on Drug Development and Trials in Recurrent Acute Pancreatitis at the National Institute of Diabetes and Digestive and Kidney Diseases Workshop
Pancreas, Vol.47(10), pp.1193-1199
11/2018
DOI: 10.1097/MPA.0000000000001164
PMCID: PMC6195328
PMID: 30325857
Abstract
Recurrent acute pancreatitis (RAP) is a complex clinical syndrome with significant morbidity, unpredictable outcomes, and limited treatment options. The National Institute of Diabetes and Digestive and Kidney Disease sponsored a workshop on July 25, 2018, in Pittsburgh, Pennsylvania, to address research gaps impeding development of effective therapies for pancreatitis. The RAP working group identified challenges to clinical progress using existing definitions, risk assessment, diagnostic and severity criteria, disease trajectories, outcomes, and research methods. Recurrent acute pancreatitis includes all the risk of acute pancreatitis and often progresses to chronic pancreatitis with variable complications of chronic pain, exocrine insufficiency, diabetes, and pancreatic cancer. However, the great variability among individuals with RAP requires better precision in defining the risks, individual episodes, as well as their frequency, pathogenic pathways, and specific outcome measures for each of the systems affected by pancreatic inflammation. Because of disease complexity, few patients are similar enough for traditional studies and methods to conduct clinical trials with small sample sizes are required. The need for genetic testing, biomarker development, and better imaging methods was highlighted. Adaptive and N-of-one study designs, better endpoints, and outcome measures including patient-reported outcomes should considered early in developing future therapeutic trial design and include all stakeholders.
Details
- Title: Subtitle
- Accelerating the Drug Delivery Pipeline for Acute and Chronic Pancreatitis: Summary of the Working Group on Drug Development and Trials in Recurrent Acute Pancreatitis at the National Institute of Diabetes and Digestive and Kidney Diseases Workshop
- Creators
- Mark E LoweMarc T Goodman - Department of Medicine, Cedars-Sinai Medical Center, Los Angeles, CAGregory A Coté - Department of Medicine, Medical University of South Carolina, Charleston, SCMarshall J Glesby - Department of Medicine, Weill Cornell Medical College, New York, NYMark Haupt - ARIEL Precision Medicine, Pittsburgh, PANicholas J Schork - Department of Quantitative Medicine, The Transcriptional Genomics Research Institute, Phoenix, AZVikesh K Singh - Department of Medicine, Johns Hopkins University School of Medicine, Baltimore, MDDana K Andersen - Division of Digestive Diseases and Nutrition, National Institutes of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health, Bethesda, MDStephen J Pandol - Division of Digestive and Liver Diseases, Department of Medicine, Cedars-Sinai Medical Center, Los Angeles, CAAliye Uc - Division of Gastroenterology, Hepatology, Pancreatology and Nutrition, Stead Family Department of Pediatrics, University of Iowa Carver College of Medicine, Iowa City, IADavid C Whitcomb - Department of Medicine, University of Pittsburgh School of Medicine and UPMC, Pittsburgh, PA
- Resource Type
- Journal article
- Publication Details
- Pancreas, Vol.47(10), pp.1193-1199
- DOI
- 10.1097/MPA.0000000000001164
- PMID
- 30325857
- PMCID
- PMC6195328
- NLM abbreviation
- Pancreas
- ISSN
- 0885-3177
- eISSN
- 1536-4828
- Publisher
- United States
- Grant note
- P50 AA011999 / NIAAA NIH HHS P01 DK098108 / NIDDK NIH HHS R01 AA024464 / NIAAA NIH HHS U01 DK108334 / NIDDK NIH HHS P30 DK054759 / NIDDK NIH HHS U01 DK108314 / NIDDK NIH HHS
- Language
- English
- Date published
- 11/2018
- Academic Unit
- Stead Family Department of Pediatrics; Radiation Oncology; Gastroenterology, Hepatology, Pancreatology, and Nutrition
- Record Identifier
- 9984046822202771
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