Utilization of electronic patient-reported outcome measures in cystic fibrosis research: Application to the GALAXY study |
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| Affiliation: | 1. Division of Pediatric Gastroenterology, Hepatology and Nutrition, University of Texas Southwestern/Children''s Health, Dallas, TX;2. Division of Gastroenterology, Atrium Health, Charlotte, NC;3. Cystic Fibrosis Foundation Therapeutics Development Network Coordinating Center, Seattle Children''s Research Institute, Seattle WA;4. Department of Pediatrics, University of Washington School of Medicine, Seattle, WA;5. Division of Gastroenterology, Beth Israel Deaconess Medical Center, Boston, MA;6. University of Minnesota Masonic Children''s Hospital, Minneapolis, MN;7. Department of Medicine, University of Washington School of Medicine, Seattle, WA;8. Division of Gastroenterology, Hepatology and Nutrition, Children''s Healthcare of Atlanta, Emory University, Atlanta, GA;1. Center for Public Health Innovation at CI International, Littleton, CO, United States;2. University of Colorado Anschutz Medical Center, Aurora, Colorado United States;3. Departments of Pediatrics and Population Health Sciences. UW School of Medicine and Public Health, Madison, WI, United States;4. Children''s Hospital Colorado, Aurora, Colorado, United States;5. Indiana University School of Medicine, United States;6. Riley Hospital for Children, United States;7. Ann & Robert H. Lurie Children''s Hospital, United States;8. Northwestern University Feinberg School of Medicine, United States;1. The University of Alabama at Birmingham, Birmingham, AL, United States;2. Johns Hopkins Medical Institutions, Baltimore, MD, United States;1. Department of Pharmacy Services, Queens University, Belfast, UK;2. Department of Pediatrics, University of Washington, Seattle Children''s Hospital, Seattle, WA, USA;3. Marisco Lung Institute, University of North Carolina, Chapel Hill, NC, USA;4. Department of Pediatrics, University of North Carolina, Chapel Hill, NC, USA;1. Respiratory Division, Vrije Universiteit Brussel (VUB), Universitair Ziekenhuis Brussel (UZ Brussel), Laarbeeklaan 101, 1090 Brussels, Belgium;2. Gastro-enterology Division, Vrije Universiteit Brussel (VUB), Universitair Ziekenhuis Brussel (UZ Brussel), Laarbeeklaan 101, 1090 Brussels, Belgium;1. Mycology Reference Centre Manchester, ECMM Centre of Excellence for Medical Mycology and the Department of Infectious Diseases, Manchester University NHS Foundation Trust, Wythenshawe Hospital, Manchester, UK;2. Paediatric Respiratory Department, Royal Manchester Children''s Hospital, Manchester University Hospital NHS Foundation Trust, Manchester, UK;3. Department of General Paediatrics, Birmingham Children''s Hospital, Birmingham Women''s and Children''s NHS Foundation Trust, Birmingham, UK;4. Division of Infection, Immunity & Respiratory Medicine, Faculty of Biology, Medicine and Health, University of Manchester, Manchester, UK;5. Manchester Adult Cystic Fibrosis Centre, Manchester University Hospitals NHS Foundation Trust, Manchester, UK |
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| Abstract: | BACKGROUNDThe Food and Drug Administration considers patient-reported outcome measures (PROMs) an essential part of clinical research studies for approval of new drugs and new indications for existing drugs. GALAXY evaluated the feasibility of electronic PROMs (ePROMS) to conduct a comprehensive evaluation of gastrointestinal (GI) symptoms in persons with cystic fibrosis (pwCF).METHODSThree validated GI ePROMs (PAC-SYM, PAGI-SYM and PAC-QOL) were combined with a Stool-Specific questionnaire to make up the GALAXY ePROMs and administered prospectively across 26 CF centers in the United States. The ePROMs were completed at enrollment visit and then electronically at weeks 1, 2 and 4. PwCF at least 2 years and older were eligible for the study. Reminders were only provided by the mobile application during the study window.RESULTSThere were 402 participants enrolled in GALAXY. Of those, 169 (42%) were under 18 years old and 193 (48%) were female. The proportion of all follow-up weeks with at least 1 ePROM fully completed was 80%, slightly higher in those ≥18 years of age (82.5%) compared to those <18 years of age (76.5%). When assessing the completion for all 4 ePROMs, the percentage was 77.6%, also higher among those ≥18 year of age (81.5% versus 72.2% for < 18 years of age).CONCLUSIONUsing ePROMs, our study demonstrated that GI symptoms can be feasibly collected with good reproducibility and with minimal involvement of research coordinator time. This mechanism of symptom collection may provide an efficient tool for future CF trials. |
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