BACKGROUND: The 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).
METHODS: Three 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.
RESULTS: There 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).
CONCLUSION: Using 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.
Bibliographical noteFunding Information:
This work was supported by the Cystic Fibrosis Foundation PT-18-1172, P30 DK089507/DK/NIDDK NIH HHS/United States.
We would like to thank all of the participants (both those pwCF and caregivers) for their instrumental role in developing the GALAXY study and participating in it. In addition, we would like to thanks especially our TDNCC study managers, Carmet Ufret-Vincenty (TDNCC, Seattle Children's Research Institute, Seattle WA) and Anna Mead (TDNCC, Seattle Children's Research Institute, Seattle WA). We would also like to thank the Priniciple Investigators and Research Coordinators for their dedication and commitment to this study, allowing it to enroll completely within 4 months. A. Jay Freeman and J. Dangerfield (Children's Healthcare of Atlanta and Emory University, Atlanta, GA); A. Shaikhkhalil and P. Olson (Nationwide Children's Hospital, Columbus, OH); A. Maqbool and E. Donnely (Children's Hospital of Philadelphia, Philadelphia, PA); S. Palle and K. Farley (Oklahoma City Fibrosis Center, Oklahoma City, OK); A. Garcia and P. Nusbaum (Oregon Health Sciences University, Portland, OR); H. Chaney and C. Purdy (Children's National Medical Center, Washington, DC); M. Sathe and D. Kamal (University of Texas Southwestern/Children's Health, Dallas, TX); T. Kremer and J. Longtine (University of Massachuetts Memorial Health Care, Worcester, MA); C. Lapin and M. McIntosh (Central Conecticut Cystic Fibrosis Center, Hartford, CT); K Pancham and C. Payne (University of Kentucky, Lexington, KY); J. Robson and J. Vroom (Primary Children's Cystic Fibrosis Center, Salt Lake City, UT); J. Zea-Hernandez and C. Gile (Helen DeVos Children's Hospital, Grand Rapids, MI); H. Hanes and K. Kennedy (Wake Forest University Baptist Medical Center, Winston-Salem, NC); A. Cairns and C. Millard (Maine Medical Center, Portland, ME); D. Green and D. Hodge (All Children's Hospital, St. Petesburg, FL); C. Milla and A. Remulla (Standford University Medical Center, Palo Alto, CA); D. Patel and F. Branch (SSM Health Cardinal Glennon Childre's Hospital, St. Louis, MO); O. Elidermir and H. Turner (Nemours Children's Clinic, Pensacola, FL); M. Bozic and C. Sawyers (Riley Hospital for Children, Indianapolis, IN); S. Freedman and K. Regan (Boston Children's Hospital, Brigham & Women's Hospital, Boston, MA); A. Stein and R. Nelso (Northwestern University, Chicago, IL); J. Abraham and M. Bailey (The Minnesota Cystic Fibrosis Center, Minneapolis, MN); J. Sullivan and J. Sweet (University of Vermonth Medical Center, Burlington, VT): S. Chittivelu and A. Scott (OSF Saint Francis Medical Center, Peoria, IL); S. Jagpal and C. Varghese (Rutgers-Robert Wood Johnson Medical School, New Brunswick, NJ); B. Moshiree and Rachel Mcafee (Atrium Health Charlotte, NC).
© 2021 Elsevier Ltd
- Electronic PROMs
- Patient reported outcomes measures
- cystic fibrosis
- gastrointestinal manifestations of cystic fibrosis