Community Perspective on Pancreatic Enzyme Replacement Therapy in Cystic Fibrosis.
Journal
Journal of pediatric gastroenterology and nutrition
ISSN: 1536-4801
Titre abrégé: J Pediatr Gastroenterol Nutr
Pays: United States
ID NLM: 8211545
Informations de publication
Date de publication:
01 11 2022
01 11 2022
Historique:
pubmed:
8
9
2022
medline:
2
11
2022
entrez:
7
9
2022
Statut:
ppublish
Résumé
People with cystic fibrosis (CF) and exocrine pancreatic insufficiency must take pancreatic enzyme replacement therapy (PERT) to prevent malnutrition and gastrointestinal (GI) symptoms. Finding better ways to manage GI complaints is a high priority for the CF community. We fielded a survey to assess the perspective of people affected by CF regarding symptoms attributed to and challenges associated with current PERT, to identify factors that affect participation in PERT studies, and to understand attitudes toward an outcome measure that could be an alternative to the coefficient of fat absorption test. Persistent GI symptoms are commonly ascribed to PERT. Minimizing time commitment and maximizing patient safety were factors affecting participation in research. We demonstrate 4 generalizable ways to incorporate patient experience early in the research process to aid in development of new medications and help improve study enrollment.
Identifiants
pubmed: 36070542
doi: 10.1097/MPG.0000000000003606
pii: 00005176-202211000-00019
doi:
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
e94-e97Informations de copyright
Copyright © 2022 by European Society for European Society for Pediatric Gastroenterology, Hepatology, and Nutrition and North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition.
Références
Singh VK, Schwarzenberg SJ. Pancreatic insufficiency in cystic fibrosis. J Cyst Fibros. 2017;16:S70–8.
Goetz DM, Savant AP. Review of CFTR modulators 2020. Pediatr Pulmonol. 2021;56:3595–606.
Nichols AL, Davies JC, Jones D, et al. Restoration of exocrine pancreatic function in older children with cystic fibrosis on ivacaftor. Paediatr Respir Rev. 2020;35:99–102.
Cystic Fibrosis Foundation Patient Registry 2020 Annual Data Report, Bethesda, MD, ©2021 Cystic Fibrosis Foundation.
United States Food and Drug Administration. Available at: https://www.fda.gov/news-events/press-announcements/fda-approves-new-breakthrough-therapy-cystic-fibrosis . Accessed September 9, 2022.
Hendeles L, Borowitz D. Pancreatic enzyme products. Pediatric Allergy Immunol Pulmonol. 2010;23:291–4.
Freedman S, Orenstein D, Black P, et al. Increased fat absorption from enteral formula through an in-line digestive cartridge in patients with cystic fibrosis. J Pediatr Gastroenterol Nutr. 2017;65:97–101.
Hollin IL, Donaldson SH, Roman C, et al. Beyond the expected: identifying broad research priorities of researchers and the cystic fibrosis community. J Cyst Fibros. 2019;18:375–7.
Community Voice: Cystic Fibrosis Foundation. Available at: https://www.cff.org/get-involved/community-voice . Accessed September 9, 2022.
Roman CN, Lazzara J, Aliaj E, et al. Lessons learned about publication of results of community surveys when regulatory oversight has not occurred. SSM - Qualitative Res Health. 2022, 2:100091.
Borowitz DA, Cummings LC, Maqbool A, et al. Coefficient of fat absorption to measure the efficacy of pancreatic enzyme replacement therapy in people with cystic fibrosis: gold standard or coal standard? Pancreas. 2022;51:310–318.
Borowitz D, Stevens C, Brettman LR, et al. International phase III trial of liprotamase efficacy and safety in pancreatic-insufficient cystic fibrosis patients. J Cyst Fibros. 2011;10:443–52.
Eton DT, Ramalho de Oliveira D, Egginton JS, et al. Building a measurement framework of burden of treatment in complex patients with chronic conditions: a qualitative study. Patient Relat Outcome Meas. 2012;3:39–49.
Farrell B, French Merkley V, Ingar N. Reducing pill burden and helping with medication awareness to improve adherence. Can Pharm J (Ott). 2013;146:262–9.
Nicolais CJ, Bernstein R, Saez-Flores E, et al. Identifying factors that facilitate treatment adherence in cystic fibrosis: qualitative analyses of interviews with parents and adolescents. J Clin Psychol Med Settings. 2019;26:530–40.
Murad MH, Shah ND, Van Houten HK, et al. Individuals with diabetes preferred that future trials use patient-important outcomes and provide pragmatic inferences. J Clin Epidemiol. 2011;64:743–8.
Freeman AJ, Sathe M, Aliaj E, et al. Designing the GALAXY study: Partnering with the cystic fibrosis community to optimize assessment of gastrointestinal symptoms. J Cyst Fibros. 2021;20:598–604.
Sathe M, Moshiree B, Vu PT, et al. Utilization of electronic patient-reported outcome measures in cystic fibrosis research: application to the GALAXY study. J Cyst Fibros. 2021;20:605–11.
Constitution of The World Health Organization. The World Health Organization. 1995. Available at: https://www.who.int/about/governancy/constitution . Accessed September 9, 2022.
Linton MJ, Dieppe P, Medina-Lara A. Review of 99 self-report measures for assessing well-being in adults: exploring dimensions of well-being and developments over time. BMJ Open. 2016;6:e010641.
Hunter J, Marshall J, Corcoran K, et al. A positive concept of health - interviews with patients and practitioners in an integrative medicine clinic. Complement Ther Clin Pract. 2013;19:197–203.
Heltshe SL, West NE, VanDevanter DR, et al. Study design considerations for the Standardized Treatment of Pulmonary Exacerbations 2 (STOP2): a trial to compare intravenous antibiotic treatment durations in CF. Contemp Clin Trials. 2018;64:35–40.
Sacristan JA, Aguaron A, Avendano-Sola C, et al. Patient involvement in clinical research: why, when, and how. Patient Prefer Adherence. 2016;10:631–40.