Gelhorn Heather L, Ye Xin, Speck Rebecca M, Tong Sandra, Healey John H, Bukata Susan V, Lackman Richard D, Murray Lindsey, Maclaine Grant, Lenderking William R, Hsu Henry H, Lin Paul S, Tap William D
Evidera, 7101 Wisconsin Avenue, Suite 1400, Bethesda, MD, 20814, USA.
Daiichi Sankyo Pharma Development, 399 Thornall St, Edison, NJ, 08837, USA.
J Patient Rep Outcomes. 2019 Feb 4;3(1):6. doi: 10.1186/s41687-019-0099-0.
Tenosynovial giant cell tumor (TGCT), a rare, locally aggressive neoplasm of the synovium of joints and tendon sheaths, is associated with joint destruction, pain and swelling. Impacts on physical function (PF) vary depending on tumor size and location. The aim of this study was to identify relevant items, and demonstrate the content validity of custom measures of lower extremity PF from the Patient-Reported Outcomes Measurement Information System Physical Function Physical Function (PROMIS-PF) item bank among patients with TGCT.
Patients were recruited for qualitative research interviews to identify predominant TGCT symptoms and impacts. Patients completed a checklist to evaluate the relevance of each PROMIS-PF item. The publicly available PROMIS-PF item response theory (IRT) parameters were used to select items representing the range of the latent PF trait.
Participants (n = 20) were 75% female, mean age 42.5 years. TGCTs were located in the knee (n = 15), hip (n = 3), and ankle (n = 2). Fifty-four PROMIS-PF items were identified as relevant by ≥20% of the participants. PF concepts discussed by participants during the qualitative interviews were also used to select relevant items. Selected items (n = 13) were used to create a physical function subscale specific to lower extremity tumors.
We describe a novel method of combining qualitative research and IRT-based item information to select a relevant and content valid subset of PROMIS-PF items to assess heterogeneous impacts on PF in TGCT, a rare disease population.
腱鞘巨细胞瘤（TGCT）是一种罕见的、局部侵袭性的关节和腱鞘滑膜肿瘤，与关节破坏、疼痛和肿胀相关。对身体功能（PF）的影响因肿瘤大小和位置而异。本研究的目的是识别相关条目，并在TGCT患者中验证来自患者报告结局测量信息系统身体功能（PROMIS-PF）条目库的定制下肢PF测量指标的内容效度。
招募患者进行定性研究访谈，以确定TGCT的主要症状和影响。患者完成一份清单，以评估每个PROMIS-PF条目的相关性。使用公开可用的PROMIS-PF条目反应理论（IRT）参数来选择代表潜在PF特质范围的条目。
参与者（n = 20）中75%为女性，平均年龄42.5岁。TGCT位于膝关节（n = 15）、髋关节（n = 3）和踝关节（n = 2）。54个PROMIS-PF条目被≥20%的参与者确定为相关。定性访谈中参与者讨论的PF概念也用于选择相关条目。选择的条目（n = 13）用于创建特定于下肢肿瘤的身体功能子量表。
我们描述了一种新方法，将定性研究与基于IRT的条目信息相结合，以选择相关且内容有效的PROMIS-PF条目子集，来评估TGCT（一种罕见疾病群体）对PF的异质性影响。
