Utilization of electronic patient-reported outcome measures in cystic fibrosis research: Application to the GALAXY study,Journal of Cystic Fibrosis

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Utilization of electronic patient-reported outcome measures in cystic fibrosis research: Application to the GALAXY study
Journal of Cystic Fibrosis ( IF 5.4 ) Pub Date : 2021-07-22 , DOI: 10.1016/j.jcf.2021.07.002
Meghana Sathe ₁ , Baha Moshiree ₂ , Phuong T Vu ₃ , Umer Khan ₃ , Sonya L Heltshe ₄ , Melita Romasco ₃ , Steven D Freedman ₅ , Sarah Jane Schwarzenberg ₆ , Christopher H Goss ₇ , A Jay Freeman ₈

Affiliation

Division of Pediatric Gastroenterology, Hepatology and Nutrition, University of Texas Southwestern/Children's Health, Dallas, TX.
Division of Gastroenterology, Atrium Health, Charlotte, NC.
Cystic Fibrosis Foundation Therapeutics Development Network Coordinating Center, Seattle Children's Research Institute, Seattle WA.
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.
Division of Gastroenterology, Beth Israel Deaconess Medical Center, Boston, MA.
University of Minnesota Masonic Children's Hospital, Minneapolis, MN.
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.
Division of Gastroenterology, Hepatology and Nutrition, Children's Healthcare of Atlanta, Emory University, Atlanta, GA.

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.

中文翻译：

在囊性纤维化研究中使用电子患者报告的结果测量：在 GALAXY 研究中的应用

背景

美国食品和药物管理局认为患者报告的结果测量 (PROM) 是批准新药和现有药物新适应症的临床研究的重要组成部分。GALAXY 评估了电子 PROM (ePROMS) 对囊性纤维化 (pwCF) 患者的胃肠道 (GI) 症状进行综合评估的可行性。

方法

三个经过验证的 GI ePROM（PAC-SYM、PAGI-SYM 和 PAC-QOL）与粪便特异性问卷相结合，组成了 GALAXY ePROM，并在美国 26 个 CF 中心进行前瞻性管理。ePROM 在注册访问时完成，然后在第 1、2 和 4 周以电子方式完成。至少 2 岁及以上的 PwCF 有资格参加这项研究。提醒仅在学习窗口期间由移动应用程序提供。

结果

GALAXY 共有 402 名参与者。其中，169 人（42%）未满 18 岁，193 人（48%）为女性。至少 1 个 ePROM 完全完成的所有随访周的比例为 80%，≥18 岁的患者 (82.5%) 略高于 18 岁的患者 (76.5%)。在评估所有 4 个 ePROM 的完成率时，百分比为 77.6%，在 18 岁以上的人群中也更高（81.5% 对 <18 岁的 72.2%）。