Meghana Sathe1, Baha Moshiree2, Phuong T Vu3, Umer Khan3, Sonya L Heltshe4, Melita Romasco3, Steven D Freedman5, Sarah Jane Schwarzenberg6, Christopher H Goss7, A Jay Freeman8. 1. Division of Pediatric Gastroenterology, Hepatology and Nutrition, University of Texas Southwestern/Children's Health, Dallas, TX. Electronic address: meghana.sathe@UTSouthwestern.edu. 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. Cystic Fibrosis Foundation Therapeutics Development Network Coordinating Center, Seattle Children's Research Institute, Seattle WA; 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. Cystic Fibrosis Foundation Therapeutics Development Network Coordinating Center, Seattle Children's Research Institute, Seattle WA; Department of Pediatrics, University of Washington School of Medicine, Seattle, WA; 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.
Abstract
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.
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.
Authors: M Boon; J Calvo-Lerma; I Claes; T Havermans; I Asseiceira; A Bulfamante; M Garriga; E Masip; B A M van Schijndel; V Fornes; C Barreto; C Colombo; P Crespo; S Vicente; H Janssens; J Hulst; P Witters; R Nobili; L Pereira; M Ruperto; E Van der Wiel; J G Mainz; K De Boeck; C Ribes-Koninckx Journal: J Cyst Fibros Date: 2020-04-22 Impact factor: 5.482
Authors: Emily A Knapp; Aliza K Fink; Christopher H Goss; Ase Sewall; Josh Ostrenga; Christopher Dowd; Alexander Elbert; Kristofer M Petren; Bruce C Marshall Journal: Ann Am Thorac Soc Date: 2016-07
Authors: Mieke Boon; Ine Claes; Trudy Havermans; Victoria Fornés-Ferrer; Joaquim Calvo-Lerma; Inês Asseiceira; Anna Bulfamante; María Garriga; Etna Masip; Sandra Woodcock; Sylvia Walet; Celeste Barreto; Carla Colombo; Paula Crespo; Els Van der Wiel; Jessie Hulst; Sandra Martinez-Barona; Rita Nobili; Luisa Pereira; Mar Ruperto; Saioa Vicente; Kris De Boeck; Carmen Ribes-Koninckx Journal: PLoS One Date: 2019-12-20 Impact factor: 3.240
Authors: Y Yiannakou; J Tack; H Piessevaux; D Dubois; E M M Quigley; M Y Ke; S Da Silva; A Joseph; R Kerstens Journal: Aliment Pharmacol Ther Date: 2017-10-06 Impact factor: 8.171