在囊性纤维化患者的腹部症状评分
Harold Tabori1,2,3, Anton Barucha1,3,4, Carlos Zagoya1
1Cystic Fibrosis Centre, Brandenburg Medical School (MHB) University, Klinikum Westbrandenburg, 14770 Brandenburg an der Havel, Germany.
高效的CFTR调节疗法 (HEMT) 已将重点转移到囊性纤维化 (CF) 中的消化系统参与. 新的CF特异性患者报告结果测量 (PROM) 现在有效地跟踪CF患者的腹部症状 (AS).
科学领域:
- 胃肠病学 胃肠病学
- 肺部病理学 肺部病理学
- 临床治疗学 临床治疗学
背景情况:
- 高效的CFTR调节疗法 (HEMT) 的出现改变了许多患有囊性纤维化 (pwCF) 的人的疾病轨迹.
- 这种转变使得临床和科学关注范围从肺部扩大到消化系统参与CF.
- 因此,对敏感工具的需求越来越大,特别是患者报告的结果测量 (PROM),以评估腹部症状 (AS).
研究的目的:
- 审查和分析用于评估囊性纤维化 (pwCF) 患者腹部症状 (AS) 的方法.
- 评估pwCF中AS评估工具的演变,特别是在HEMT的背景下.
- 为了确定CF特异性胃肠PROMs的开发和验证.
主要方法:
- 进行了系统的文献审查.
- 截至2022年11月,已获取了5224个出版物.
- 最终包括了39项研究在分析中.
主要成果:
- 从历史上看,pwCF中的AS使用单个问题或未经验证的工具进行评估.
- 使用了生活质量 (QOL) PROMs与有限的胃肠道 (GI) 问题以及其他胃肠道病理的PROMs.
- 最近开发和验证的CF特异性GI-PROM显示出对变化的高度敏感性.
结论:
- 新的CF特异性GI-PROM已根据FDA指导方针开发和验证.
- 这些新型PROM对治疗变化敏感,并有效捕捉症状负担.
- 实施HEMT导致了pwCF中AS负担的显著降低,正如这些新措施所证明的那样.
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