在轻链 (AL) 氨基粉症中开发和初步验证AL-PROfile患者报告的结果指标
Anita D'Souza1, Aniko Szabo2, Idayat Akinola1
1Division of Hematology/Oncology, Medical College of Wisconsin, Milwaukee, Wisconsin, USA.
European journal of haematology
|February 13, 2024
概括
该AL-PROfile证明了可接受的可靠性和有效性,用于评估系统性轻链氨基粉症患者报告的结果. 这种患者报告的结果衡量标准适用于该患者群体的临床使用.
科学领域:
- 临床评估 临床评估
- 患者报告的结果.
- 心理测量评估是一种心理评估.
背景情况:
- 系统性轻链氨基粉症 (AL) 需要强大的患者报告结果措施 (PROMs) 进行有效的管理.
- 现有的PROM可能无法完全捕捉AL氨基粉症对患者生活的多方面的影响.
- 该AL-PROfile是为了整合关键的PROMIS和PRO-CTCAE组件进行全面评估而开发的.
研究的目的:
- 评估AL-PROfile的心理测量特性,这是一种针对系统性轻链氨基粉症的新型PROM.
- 在患者队列中评估AL-PROfile的内容有效性,可靠性和有效性.
主要方法:
- 研究1:对20名AL氨基粉症患者进行认知报告采访,以评估内容的有效性.
- 研究2:对297名参与者的AL-PROfile进行心理测量评估,对SF-36.6等既有措施的可靠性和有效性进行评估.
- 统计分析包括内部一致性,测试重复测试可靠性,收/区分有效性和已知组有效性.
主要成果:
- 认知总结证实了AL-PROfile的相关性,尽管一些患者发现某些问题与他们的经验无关.
- 在大多数领域中,AL-PROfile表现出可接受的内部一致性和测试重复测试可靠性,除了PROMIS认知功能和PRO-CTCAE恶心的例外.
- 有效性分析显示了类似领域之间的预期相关性,以及不同领域之间的较小相关性,支持了该测量的构造有效性. 疾病阶段与身体功能,疲劳和其他症状相关.
结论:
- 该AL-PROfile表现出可接受的可靠性和有效性,用于全身轻链氨基粉症患者.
- 这种PROM是捕捉AL氨基粉症临床实践和研究中患者报告的结果的一个有希望的工具.
- 进一步的改进可能会提高特定领域的性能,例如认知功能.
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