评估现实世界患者报告的结果在患者启动生物药物治疗自身免疫性疾病的患者:在四个患者驱动的研究网络的观察研究
Timothy Beukelman1, Millie D Long2, Rennie L Rhee3
1Department of Pediatrics, Division of Rheumatology, University of Alabama at Birmingham, Birmingham, AL, USA.
Patient related outcome measures
|June 19, 2023
概括
患者报告的结局 (PROs) 在开始生物治疗的自身免疫疾病患者中显示出各种基线异常. 那些有基线PRO异常的人更有可能改善,为未来的治疗评估提供信息.
科学领域:
- 类风湿病学 类风湿病学
- 免疫学 免疫学 免疫学
- 临床药理学 临床药理学
背景情况:
- 通过患者报告的结果 (PROs) 来评估生物药物对自身免疫疾病的实际有效性仍然具有挑战性.
- 关于将PRO纳入临床有效性评估中最可靠的方法存在不确定性.
- 这项研究解决了需要更好地了解PROs在自身免疫性疾病的生物治疗中的需求.
研究的目的:
- 评估和比较开始治疗自身免疫性疾病的生物治疗的患者的基线PRO异常.
- 为了确定基线PRO异常对后续治疗反应的影响.
- 在现实世界的有效性研究中提供PRO的有意义的纳入信息.
主要方法:
- 用患者报告结果测量信息系统仪器收集患者报告的结果.
- 美国的PRO分数 (T-分数) 标准化为美国一般人口.
- 分析了基线和随访 (3-8个月) PRO,以确定异常 (≥5个单位比正常情况差) 和显著改善 (≥5个单位).
主要成果:
- 通过自身免疫性疾病观察到基线PRO得分的显著变化.
- 患有异常基线疼痛干扰评分的患者比例在52%至93%之间.
- 患有基线PRO异常的患者表现出经历显著改善的可能性大幅增加.
结论:
- 许多患者在生物药物上有所改善,但并非所有患者都有基线PRO异常.
- 没有基线PRO异常的患者似乎不太可能显著改善.
- 需要进行进一步的研究,以优化患者选择和PRO评估在生物有效性的现实研究中.
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