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

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Designing the GALAXY study: Partnering with the cystic fibrosis community to optimize assessment of gastrointestinal symptoms
Journal of Cystic Fibrosis ( IF 5.4 ) Pub Date : 2021-01-13 , DOI: 10.1016/j.jcf.2020.12.021
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

Affiliation

Division of Gastroenterology, Hepatology and Nutrition, Children's Healthcare of Atlanta, Emory University, Atlanta, GA, USA.
Division of Gastroenterology, Hepatology and Nutrition, Children's Health University of Texas Southwestern, Dallas, TX, USA.
Cystic Fibrosis Foundation, Bethesda, MD, USA.
Cystic Fibrosis Foundation, Bethesda, MD, USA; Jacobs School of Medicine and Biomedical Sciences, Buffalo, NY, USA.
Seattle Children's Hospital Research Institute, Seattle, WA, USA.
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.
Division of Gastroenterology, Beth Israel Deaconess Medical Center, Boston, MA, USA.
Seattle Children's Hospital Research Institute, Seattle, WA, USA; Department of Pediatrics, University of Washington, Seattle, WA, USA.
University of Minnesota Masonic Children's Hospital, Minneapolis, MN, USA.
University of North Carolina, Atrium Health, Charlotte, NC, USA.

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.

中文翻译：

设计 GALAXY 研究：与囊性纤维化社区合作优化胃肠道症状评估

背景

囊性纤维化 (CF) 患者的胃肠道 (GI) 受累非常普遍，是发病率的重要来源。CF 患者已将 GI 问题确定为首要研究重点，但缺乏捕捉 CF 经历的许多 GI 症状的通用临床结果测量。GALAXY 研究旨在解决这一未满足的需求。

方法

GALAXY 研究团队与社区之声（一个由 CF 患者及其护理人员组成的社区）合作，以确定最准确反映他们在 CF 中的胃肠道症状经历的患者报告的结果测量。我们还对 CF 护理团队进行了调查，以确定不同团队成员（提供者、护士和营养师）在管理各种胃肠道疾病方面的舒适度。

结果

Community Voice 的成员将 PAC-SYM、PAGI-SYM、PAC-QOL 和 Bristol Stool 量表与另外三个针对特定症状的问题相结合，作为患者报告的结果测量，全面捕捉 CF 对 GI 疾病的体验。CF 护理团队提供者报告称，在治疗 GI 疾病（包括便秘 (92%)、GERD (93%) 和胀气 (77%) 时，舒适度很高，但舒适度仅限于一线干预措施。