Designing the GALAXY study: Partnering with the cystic fibrosis community to optimize assessment of gastrointestinal symptoms

A Jay Freeman; Meghana Sathe; Enid Aliaj; Drucy Borowitz; Barbra Fogarty; Christopher H Goss; Steven Freedman; Sonya L Heltshe; Umer Khan; Dara Riva; Christina Roman; Melita Romasco; Sarah Jane Schwarzenberg; Carmen A Ufret-Vincenty; Baha Moshiree

doi:10.1016/j.jcf.2020.12.021

Designing the GALAXY study: Partnering with the cystic fibrosis community to optimize assessment of gastrointestinal symptoms

J Cyst Fibros. 2021 Jul;20(4):598-604. doi: 10.1016/j.jcf.2020.12.021. Epub 2021 Jan 13.

Authors

Affiliations

¹ Division of Gastroenterology, Hepatology and Nutrition, Children's Healthcare of Atlanta, Emory University, Atlanta, GA, USA. Electronic address: afreem6@emory.edu.
² Division of Gastroenterology, Hepatology and Nutrition, Children's Health University of Texas Southwestern, Dallas, TX, USA.
³ Cystic Fibrosis Foundation, Bethesda, MD, USA.
⁴ Cystic Fibrosis Foundation, Bethesda, MD, USA; Jacobs School of Medicine and Biomedical Sciences, Buffalo, NY, USA.
⁵ Seattle Children's Hospital Research Institute, Seattle, WA, USA.
⁶ Seattle Children's Hospital Research Institute, Seattle, WA, USA; Department of Medicine, University of Washington, Seattle, WA, USA; Department of Pediatrics, University of Washington, Seattle, WA, USA.
⁷ Division of Gastroenterology, Beth Israel Deaconess Medical Center, Boston, MA, USA.
⁸ Seattle Children's Hospital Research Institute, Seattle, WA, USA; Department of Pediatrics, University of Washington, Seattle, WA, USA.
⁹ University of Minnesota Masonic Children's Hospital, Minneapolis, MN, USA.
¹⁰ University of North Carolina, Atrium Health, Charlotte, NC, USA.

Abstract

Background: Gastrointestinal (GI) involvement among persons with cystic fibrosis (CF) is highly prevalent, representing a significant source of morbidity. Persons with CF have identified GI concerns as a top research priority, yet universal clinical outcome measures capturing many of the GI symptoms experienced in CF are lacking. The GALAXY study was envisioned to address this unmet need.

Methods: The GALAXY study team partnered with Community Voice, a community of patients with CF and their caregivers, to identify the patient reported outcome measures that most accurately reflected their experience with GI symptoms in CF. We also surveyed CF care teams to identify the comfort level of various team members (providers, nurses and dieticians) in managing a variety of GI conditions.

Results: Members of Community Voice identified the combination of PAC-SYM, PAGI-SYM, PAC-QOL and the Bristol Stool scale with three additional symptom-specific questions as patient-reported outcome measures that comprehensively captured the CF experience with GI disease. CF care team providers reported a high level of comfort in treating GI conditions including constipation (92%), GERD (93%), and gassiness (77%), however comfort level was limited to only first-line interventions.

Conclusion: By partnering with persons with CF as well as their caregivers and medical providers, the GALAXY study is designed to uniquely capture the prevalence and severity of GI involvement among persons with CF in a manner that reflects the CF patient experience. The results of GALAXY will inform the development of future interventional trials and serve as a reproducible and objective study endpoint.

Keywords: Community Involvement; Gastrointestinal; Patient reported outcome measures (proms).

Publication types

Multicenter Study
Observational Study
Research Support, Non-U.S. Gov't

MeSH terms

Cystic Fibrosis / complications
Cystic Fibrosis / diagnosis*
Gastrointestinal Diseases / diagnosis*
Gastrointestinal Diseases / etiology
Humans
Longitudinal Studies
Patient Reported Outcome Measures
Prospective Studies
Symptom Assessment

Grants and funding

P30 DK089507/DK/NIDDK NIH HHS/United States