Haberland Claudia, Filonenko Anna, Seitz Christian, Börner Matthias, Gerlinger Christoph, Doll Helen, Wessiepe Dorothea
Market Access, Public Affairs & Sustainability, TA Pulmonology / Innovative WHC, Bayer AG, Building S157, 3.315, 13342, Berlin, Germany.
Market Access, Public Affairs & Sustainability, TA Pulmonology / Innovative WHC, Bayer AG, Building S157, R318, 13342, Berlin, Germany.
J Patient Rep Outcomes. 2020 Nov 13;4(1):97. doi: 10.1186/s41687-020-00263-0.
To evaluate the psychometric and measurement properties of two patient-reported outcome instruments, the menstrual pictogram superabsorbent polymer-containing version 3 (MP SAP-c v3) and Uterine Fibroid Daily Bleeding Diary (UF-DBD). Test-retest reliability, criterion, construct validity, responsiveness, missingness and comparability of the MP SAP-c v3 and UF-DBD versus the alkaline hematin (AH) method and a patient global impression of severity (PGI-S) were analyzed in post hoc trial analyses.
Analyses were based on data from up to 756 patients. The full range of MP SAP-c v3 and UF-DBD response options were used, with score distributions reflecting the cyclic character of the disease. Test-retest reliability of MP SAP-c v3 and UF-DBD scores was supported by acceptable intraclass correlation coefficients when stability was defined by the AH method and Patient Global Impression of Severity (PGI-S) scores (0.80-0.96 and 0.42-0.94, respectively). MP SAP-c v3 and UF-DBD scores demonstrated strong and moderate-to-strong correlations with menstrual blood loss assessed by the AH method. Scores increased in monotonic fashion, with greater disease severities, defined by the AH method and PGI-S scores; differences between groups were mostly statistically significant (P < 0.05). MP SAP-c v3 and UF-DBD were sensitive to changes in disease severity, defined by the AH method and PGI-S. MP SAP-c v3 and UF-DBD showed a lower frequency of missing patient data versus the AH method, and good agreement with the AH method.
This evidence supports the use of the MP SAP-c v3 and UF-DBD to assess clinical efficacy endpoints in UF phase III studies replacing the AH method.
为评估两种患者报告结局工具，即含超吸收性聚合物的月经象形图第3版（MP SAP-c v3）和子宫肌瘤每日出血日记（UF-DBD）的心理测量和测量属性。在事后试验分析中，分析了MP SAP-c v3和UF-DBD相对于碱性正铁血红素（AH）法和患者总体严重程度印象（PGI-S）的重测信度、标准效度、结构效度、反应性、缺失情况和可比性。
分析基于多达756名患者的数据。使用了MP SAP-c v3和UF-DBD的全范围反应选项，分数分布反映了疾病的周期性特征。当以AH法和患者总体严重程度印象（PGI-S）分数定义稳定性时，MP SAP-c v3和UF-DBD分数的重测信度得到了可接受的组内相关系数的支持（分别为0.80 - 0.96和0.42 - 0.94）。MP SAP-c v3和UF-DBD分数与通过AH法评估的月经失血量显示出强和中到强的相关性。分数随疾病严重程度增加呈单调增加，疾病严重程度由AH法和PGI-S分数定义；组间差异大多具有统计学意义（P < 0.05）。MP SAP-c v3和UF-DBD对由AH法和PGI-S定义的疾病严重程度变化敏感。与AH法相比，MP SAP-c v3和UF-DBD显示患者数据缺失频率较低，且与AH法一致性良好。
该证据支持在子宫肌瘤III期研究中使用MP SAP-c v3和UF-DBD评估临床疗效终点，以替代AH法。
