van Hoorn Evelien S, Willems Sterre P E, Al Arashi Wala, de Moor Annick S, van Kwawegen Calvin B, Teela Lorynn, Oude Voshaar Martijn A H, Kremer Hovinga Idske C L, Schutgens Roger E G, Schols Saskia E M, Leebeek Frank W G, Haverman Lotte, Cnossen Marjon H, Gouw Samantha C, Lingsma Hester F
Department of Public Health, Erasmus MC, University Medical Center Rotterdam, Rotterdam, The Netherlands.
Department of Hematology, Radboud university medical center Nijmegen, Hemophilia Treatment Center Nijmegen-Eindhoven-Maastricht, Nijmegen, The Netherlands.
Res Pract Thromb Haemost. 2024 Jun 13;8(4):102474. doi: 10.1016/j.rpth.2024.102474. eCollection 2024 May.
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).
To evaluate the feasibility, measurement properties, and relevance of PROMIS measures in adults with VWD, IPFDs, and RBDs.
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.
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.
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.
患者报告结局测量信息系统（PROMIS）测量工具可用于测量患者报告结局。PROMIS测量工具，包括计算机自适应测试（CATs）和简表，已证明能够充分评估血友病患者的结局。然而，尚不清楚PROMIS测量工具是否适用于血管性血友病（VWD）、遗传性血小板功能障碍（IPFDs）和罕见出血性疾病（RBDs）患者。
评估PROMIS测量工具在VWD、IPFDs和RBDs成年患者中的可行性、测量特性及相关性。
在这项横断面多中心研究中，患有VWD、IPFDs和RBDs的成年患者通过电子方式完成了9项PROMIS测量工具和简明健康状况调查简表第2版（SF-36v2）。可行性通过完成项目的数量和地板效应/天花板效应来确定。测量特性包括基于多特质多方法分析的结构效度以及使用信度系数和最低下限的信度。相关性通过与荷兰普通人群进行比较来评估。
共有111名患者（中位年龄57岁[四分位间距，44 - 67岁]；60%为VWD，16%为IPFD，24%为RBD）参与。VWD患者中，每项PROMIS CAT回答的平均项目数在5.3至8.7之间（范围为4 - 12）。所有CAT的结构效度均得到支持，所有工具均具有良好的信度（≥0.70）。PROMIS测量工具的天花板效应低于SF-36v2。
对于主要为非重度VWD形式的患者，PROMIS测量工具是SF-36v2可行、有效且可靠的替代工具。所选测量工具的相关性有限。有必要进行更多研究以评估IPFDs和RBDs成年患者中的PROMIS测量工具。
