We sought to evaluate how PROMIS patient-reported outcome (PRO) measures correlated with disease characteristics in systemic light chain (AL) amyloidosis patients at diagnosis. Newly diagnosed AL patients were recruited at two centres (N = 61). Patients completed the PROMIS Global Health v1.2, PROMIS-29 Profile v2.0 and Fatigue 8a v1.0. We assigned disease severity based on stage, presence of cardiac AL, and number of organs involved. We evaluated a) known groups validity by comparing PROMIS T-scores by disease severity, b) internal consistency using Cronbach’s alpha and c) convergent/discriminant validity based on correlations across the domains and summary scores. Using receiver operating characteristic (ROC) curve analysis, NT-proBNP cut-off level corresponding to normal/mild vs moderate/severe PRO scores was determined. The median age was 68 (48–83) years with 58% males. Sixty-six percent had cardiac involvement and 25% had 3 or more organs involved with AL amyloidosis; 14% had stage 1, 28% stage 2, 36% stage 3 and 16% stage 4 disease. PROMIS measures had acceptable to excellent internal consistency and expected patterns of correlations. PROMIS Global Physical Health score was worse than the Global Mental Health Score at diagnosis; Physical function, fatigue and anxiety were the most impaired domains. PROMIS Global Health summary scores discriminated across AL amyloidosis stage and number of organs involved. Physical Function showed the strongest effects across known groups by stage, cardiac involvement and number of organs involved followed by Ability to Participate in Social Roles and Activities. A diagnostic NT-proBNP cut-off of 4200 pg/ml identified patients with moderate/severe PRO scores for these domains. Our results provide evidence for reliability and validity of select PROMIS short form measures in AL amyloidosis at diagnosis.

我们试图评估PROMIS患者报告的结局（PRO）量度与诊断时系统性轻链（AL）淀粉样变性患者的疾病特征之间的关系。在两个中心招募了新诊断的AL患者（N = 61）。患者完成了PROMIS Global Health v1.2，PROMIS-29 Profile v2.0和Fatigue 8a v1.0。我们根据分期，心脏AL的存在以及涉及的器官数量来确定疾病的严重程度。我们通过比较疾病严重程度的PROMIS T评分来评估a）已知组的有效性，b）使用Cronbach's alpha的内部一致性，以及c）基于域之间的相关性和汇总分数的收敛/区分有效性。使用接收器工作特征（ROC）曲线分析，确定对应于正常/轻度与中度/重度PRO评分的NT-proBNP截止水平。中位年龄为68（48-83）岁，男性为58％。66％有心脏受累，25％有3个或更多与AL淀粉样变性有关的器官；14％的患者患有1期，28％2期，36％3期和16％4期疾病。PROMIS措施具有极好的内部一致性和预期的相关模式，可以接受。诊断时，PROMIS全球身体健康评分低于全球心理健康评分；身体功能，疲劳和焦虑是受损最严重的领域。PROMIS全球健康摘要评分区分了AL淀粉样变性病阶段和涉及的器官数量。身体功能在各个阶段的已知人群中表现出最强的作用，其受累程度，心脏受累程度和受累器官的数量依次为参与社会角色和活动的能力。诊断性NT-proBNP临界值为4200 pg / ml，可确定这些区域的PRO评分为中度/重度的患者。我们的结果提供了在AL淀粉样变性诊断中选择PROMIS短形式量度的可靠性和有效性的证据。