■患者报告结果测量信息系统(PROMIS)测量可用于测量患者报告结果。PROMIS措施,包括计算机自适应测试(CAT)和简短表格,已证明有能力充分评估血友病患者的结局。是的,然而,不清楚PROMIS措施是否适合血管性血友病(VWD)患者,遗传性血小板功能障碍(IPFDs),和罕见的出血性疾病(RBD)。
■为了评估可行性,测量属性,以及PROMIS措施在VWD成人中的相关性,IPFD,和RBDs。
■在这项横断面多中心研究中,成人VWD,IPFD,和RBD以电子方式完成了9项PROMIS措施和简短表格-36版本2(SF-36v2)。可行性由已完成项目的数量和地板/天花板效应决定。测量属性包括基于多特征多方法分析的结构效度和使用可靠性系数和最大下限的可靠性。根据与荷兰普通人群的比较评估相关性。
■总共,111例患者(中位年龄,57年[IQR,44-67];60%VWD,16%IPFD,24%RBD)参与。回答的平均项目数从5.3到8.7不等(范围,4-12)VWD患者的每个PROMISCAT。所有CAT都支持结构效度,所有仪器都具有良好的可靠性(≥0.70)。与SF-36v2相比,PROMIS措施的上限效应较小。
■PROMIS措施是可行的,有效,在主要为非严重型VWD的患者中,SF-36v2的可靠替代方案。选定措施的相关性有限。需要进一步的研究来评估患有IPFD和RBD的成年人的PROMIS措施。
UNASSIGNED: Patient-reported outcomes measurement information system (PROMIS) measures can be used to measure patient-reported outcomes. PROMIS measures, including computer adaptive tests (CATs) and short forms, have demonstrated the ability to adequately assess outcomes in patients with hemophilia. It is, however, unclear if PROMIS measures are suitable for patients with von Willebrand disease (VWD), inherited platelet function disorders (IPFDs), and rare bleeding disorders (RBDs).
UNASSIGNED: To evaluate the feasibility, measurement properties, and relevance of PROMIS measures in adults with VWD, IPFDs, and RBDs.
UNASSIGNED: In this cross-sectional multicenter study, adults with VWD, IPFDs, and RBDs completed 9 PROMIS measures and the Short Form-36 version 2 (SF-36v2) electronically. Feasibility was determined by the number of completed items and floor/ceiling effects. Measurement properties included construct validity based on a multitrait-multimethod analysis and reliability using the reliability coefficient and greatest lower bound. Relevance was evaluated based on comparison with the Dutch general population.
UNASSIGNED: In total, 111 patients (median age, 57 years [IQR, 44-67]; 60% VWD, 16% IPFD, 24% RBD) participated. Mean number of items answered varied from 5.3 to 8.7 (range, 4-12) per PROMIS CAT in patients with VWD. Construct validity was supported for all CATs and all instruments had a good reliability (≥0.70). The PROMIS measures had less ceiling effects than the SF-36v2.
UNASSIGNED: The PROMIS measures are a feasible, valid, and reliable alternative for the SF-36v2 in patients with primarily nonsevere forms of VWD. The relevance of the selected measures was limited. Additional research is necessary to evaluate the PROMIS measures in adults with IPFDs and RBDs.