使用“临界差异”统计标准可提高健康评估问卷残疾指数评分对类风湿关节炎患者的预测效用。
Use of a "critical difference" statistical criterion improves the predictive utility of the Health Assessment Questionnaire-Disability Index score in patients with rheumatoid arthritis.
作者信息
Behrens Frank, Koehm Michaela, Schwaneck Eva C, Schmalzing Marc, Gnann Holger, Greger Gerd, Tony Hans-Peter, Burkhardt Harald
机构信息
1Division of Rheumatology, University Hospital Frankfurt, Goethe University, Frankfurt am Main, Germany.
Fraunhofer Institute for Molecular Biology and Applied Ecology IME, Project Group Translational Medicine & Pharmacology TMP, Frankfurt am Main, Germany.
出版信息
BMC Rheumatol. 2019 Dec 10;3:51. doi: 10.1186/s41927-019-0095-2. eCollection 2019.
BACKGROUND
The Health Assessment Questionnaire-Disability Index (HAQ-DI) is used to assess functional status in rheumatoid arthritis (RA), but the change required for meaningful improvements remains unclear. A minimum clinically important difference (MCID) of 0.22 is frequently used in RA trials. The aim of this study was to determine a statistically defined critical difference for HAQ-DI (HAQ-DI-d) and evaluate its association with therapeutic outcomes.
METHODS
We retrospectively analyzed data from adult German patients with RA enrolled in a multicenter observational trial in which they received adalimumab therapy at the decision of the treating clinician during routine clinical care. The HAQ-DI-d, defined as the minimum change that can be reliably discriminated from random long-term variations in patients on stable therapy, was determined by evaluating intra-individual variation in patient scores. Other outcomes of interest included Disease Activity Score-28 joints and patient-reported pain and fatigue.
RESULTS
The HAQ-DI-d was calculated as an improvement (decrease) from baseline of 0.68 in a discovery cohort ( = 1645) of RA patients on stable therapy and with moderate disease activity (mean DAS28 [standard deviation] of 4.4 [1.6]). In the full patient cohort ( = 2740), 22.1% of patients achieved a HAQ-DI-d improvement at month 6. Compared with patients with a small improvement in HAQ-DI (decrease of ≥0.22 to < 0.68) or no improvement (< 0.22), patients achieving a HAQ-DI-d at month 6 had better therapeutic outcomes at months 12 and 24, including stable functional improvements. Change in pain was the most important predictor of HAQ-DI improvement during the first 6 months of therapy.
CONCLUSIONS
A HAQ-DI-d of 0.68 is a reliable measure of functional improvement. This measure may be useful in routine clinical care and clinical trials.
TRIAL REGISTRATION
ClinicalTrials.gov NCT01076205. Registered on February 26, 2010 (retrospectively registered).
背景
健康评估问卷残疾指数（HAQ-DI）用于评估类风湿关节炎（RA）的功能状态，但有意义改善所需的变化仍不明确。RA试验中经常使用的最小临床重要差异（MCID）为0.22。本研究的目的是确定HAQ-DI的统计学定义临界差异（HAQ-DI-d），并评估其与治疗结果的关联。
方法
我们回顾性分析了参加多中心观察性试验的成年德国RA患者的数据，这些患者在常规临床护理中由治疗医生决定接受阿达木单抗治疗。HAQ-DI-d定义为与稳定治疗患者的随机长期变化可靠区分的最小变化，通过评估患者评分的个体内差异来确定。其他感兴趣的结果包括28个关节疾病活动评分以及患者报告的疼痛和疲劳。
结果
在稳定治疗且疾病活动度中等（平均DAS28[标准差]为4.4[1.6]）的RA患者发现队列（n = 1645）中，HAQ-DI-d计算为自基线改善（降低）0.68。在整个患者队列（n = 2740）中，22.1%的患者在第6个月实现了HAQ-DI-d改善。与HAQ-DI改善较小（降低≥0.22至<0.68）或无改善（<0.22）的患者相比，在第6个月实现HAQ-DI-d的患者在第12个月和第24个月有更好的治疗结果，包括稳定的功能改善。疼痛变化是治疗前6个月HAQ-DI改善的最重要预测因素。
结论
HAQ-DI-d为0.68是功能改善的可靠指标。该指标可能在常规临床护理和临床试验中有用。
试验注册
ClinicalTrials.gov NCT01076205。于2010年2月26日注册（追溯注册）。
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