Department of Neurology, University of California, Irvine, CA, United States.
Department of Neurology, Brigham and Women's Hospital, Harvard Medical School, Boston, MA, United States.
Neuromuscul Disord. 2022 Oct;32(10):800-805. doi: 10.1016/j.nmd.2022.08.005. Epub 2022 Aug 24.
This study aimed to evaluate the correlation between various outcome measures used to assess disease severity and progression in inclusion body myositis (IBM) clinical trials. A cross-sectional study, involving 51 IBM patients meeting the European Neuromuscular Center (ENMC) 2011 criteria for clinically defined or probable IBM, was completed at the University of California, Irvine. Clinical details, demographic data, and functional data including timed get up (TGU), manual muscle testing, hand grip, pinch dynamometry, as well as IBM functional rating scale (IBMFRS), modified Rankin score, forced vital capacity (FVC), and modified ocular bulbar facial respiratory scale (mOBFRS) were collected on all patients. Descriptive statistics and Pearson's r correlation were performed to analyze the data. Age of the patient did not correlate with any of the outcome measures. Greater severity of IBMFRS scores correlated with longer disease duration as well as greater severity for FVC, strength outcomes, TGU, modified Rankin, and mOBFRS. Additionally, TGU strongly correlated with muscle strength measurements, modified Rankin, and mOBFRS. mOBFRS moderately correlated with IBMFRS, muscle strength, FVC, TGU and modified Rankin score. We demonstrate moderate to strong correlations among the disease severity outcome measures in this study.
本研究旨在评估用于评估包涵体肌炎 (IBM) 临床试验中疾病严重程度和进展的各种结果测量之间的相关性。在加利福尼亚大学欧文分校完成了一项涉及 51 名符合欧洲神经肌肉中心 (ENMC) 2011 年临床定义或可能 IBM 标准的 IBM 患者的横断面研究。收集了所有患者的临床详细信息、人口统计学数据以及功能数据,包括定时起身 (TGU)、手动肌肉测试、手握力、捏力计测量,以及 IBM 功能评定量表 (IBMFRS)、改良 Rankin 评分、用力肺活量 (FVC) 和改良眼-球-面-呼吸量表 (mOBFRS)。进行了描述性统计和 Pearson r 相关性分析。患者的年龄与任何结果测量均无相关性。更严重的 IBMFRS 评分与疾病持续时间较长以及 FVC、肌肉力量、TGU、改良 Rankin 和 mOBFRS 的严重程度增加相关。此外,TGU 与肌肉力量测量、改良 Rankin 和 mOBFRS 具有很强的相关性。mOBFRS 与 IBMFRS、肌肉力量、FVC、TGU 和改良 Rankin 评分中度相关。我们在本研究中证明了疾病严重程度结果测量之间存在中度到高度相关性。