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

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

A Jay Freeman  1 , Meghana Sathe  2 , Enid Aliaj  3 , Drucy Borowitz  4 , Barbra Fogarty  5 , Christopher H Goss  6 , Steven Freedman  7 , Sonya L Heltshe  8 , Umer Khan  5 , Dara Riva  3 , Christina Roman  3 , Melita Romasco  5 , Sarah Jane Schwarzenberg  9 , Carmen A Ufret-Vincenty  3 , Baha Moshiree  10
Affiliations
Observational Study

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

A Jay Freeman et al. J Cyst Fibros. 2021 Jul.

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).

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Conflict of interest statement

Declaration of Competing Interest Each of the authors have funding through Cystic Fibrosis Foundation (CFF) with five of the authors employed by the CFF (EA, DB, DR.., CR and CUF). The authors have no other conflicts of interest related to this manuscript.

Comment in

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