Sil Soumitri, Arnold Lesley M, Lynch-Jordan Anne, Ting Tracy V, Peugh James, Cunningham Natoshia, Powers Scott W, Lovell Daniel J, Hashkes Philip J, Passo Murray, Schikler Kenneth N, Kashikar-Zuck Susmita
Department of Pediatrics, Emory University School of Medicine, Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Atlanta, GA, USA Department of Psychiatry and Behavioral Neuroscience, University of Cincinnati College of Medicine, Cincinnati, OH, USA Division of Behavioral Medicine and Clinical Psychology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA William S. Rowe Division of Rheumatology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA Pediatric Rheumatology Unit, Shaare Zedek Medical Center, Shaare Zedek, Jerusalem, Israel Division of Rheumatology and Immunology, Medical University of South Carolina, Charleston, SC, USA Division of Pediatric Rheumatology, University of Louisville School of Medicine, Louisville, KY, USA.
Pain. 2014 Jul;155(7):1206-1212. doi: 10.1016/j.pain.2014.03.005. Epub 2014 Mar 17.
The primary objective of this study was to estimate a clinically significant and quantifiable change in functional disability to identify treatment responders in a clinical trial of cognitive-behavioral therapy (CBT) for youth with juvenile fibromyalgia (JFM). The second objective was to examine whether baseline functional disability (Functional Disability Inventory), pain intensity, depressive symptoms (Children's Depression Inventory), coping self-efficacy (Pain Coping Questionnaire), and parental pain history predicted treatment response in disability at 6-month follow-up. Participants were 100 adolescents (11-18 years of age) with JFM enrolled in a recently published clinical trial comparing CBT to a fibromyalgia education (FE) intervention. Patients were identified as achieving a clinically significant change in disability (i.e., were considered treatment responders) if they achieved both a reliable magnitude of change (estimated as a > or = 7.8-point reduction on the FDI) using the Reliable Change Index, and a reduction in FDI disability grade based on established clinical reference points. Using this rigorous standard, 40% of patients who received CBT (20 of 50) were identified as treatment responders, compared to 28% who received FE (14 of 50). For CBT, patients with greater initial disability and higher coping efficacy were significantly more likely to achieve a clinically significant improvement in functioning. Pain intensity, depressive symptoms, and parent pain history did not significantly predict treatment response. Estimating clinically significant change for outcome measures in behavioral trials sets a high bar but is a potentially valuable approach to improve the quality of clinical trials, to enhance interpretability of treatment effects, and to challenge researchers to develop more potent and tailored interventions.
本研究的主要目的是评估青少年纤维肌痛(JFM)患者在认知行为疗法(CBT)临床试验中功能残疾方面具有临床意义且可量化的变化,以识别治疗反应者。第二个目的是检验基线功能残疾(功能残疾量表)、疼痛强度、抑郁症状(儿童抑郁量表)、应对自我效能感(疼痛应对问卷)和父母疼痛史是否能预测6个月随访时残疾方面的治疗反应。研究参与者为100名患有JFM的青少年(11 - 18岁),他们参与了一项最近发表的临床试验,该试验将CBT与纤维肌痛教育(FE)干预进行比较。如果患者使用可靠变化指数实现了可靠的变化幅度(估计FDI减少≥7.8分),并且根据既定的临床参考点FDI残疾等级有所降低,则被确定为在残疾方面实现了具有临床意义的变化(即被视为治疗反应者)。采用这一严格标准,接受CBT的患者中有40%(50例中的20例)被确定为治疗反应者,而接受FE的患者这一比例为28%(50例中的14例)。对于CBT,初始残疾程度较高且应对效能较高的患者在功能方面显著更有可能实现具有临床意义的改善。疼痛强度、抑郁症状和父母疼痛史并不能显著预测治疗反应。在行为试验中估计结局指标具有临床意义的变化设定了很高的标准,但这是一种潜在的有价值的方法,可提高临床试验的质量、增强治疗效果的可解释性,并促使研究人员开发更有效且更具针对性的干预措施。