Menter Alan, Kosinski Mark, Bresnahan Brian W, Papp Kim A, Ware John E
Baylor Medical Center, Dallas, Texas, USA.
J Drugs Dermatol. 2004 Jan-Feb;3(1):27-38.
The objective of this study was to document the disease burden associated with moderate to severe plaque psoriasis, and assess the impact of efalizumab psoriasis treatment in improving patient-reported outcomes. This included analysis of patient-reported dermatology-related quality of life (DRQL) and psoriasis symptom scores among patients with moderate to severe psoriasis participating in three phase III, randomized, double-blinded, parallel-group, placebo-controlled, multi-center clinical trials conducted to evaluate the efficacy and safety of efalizumab. A total of 1,242 patients with moderate to severe psoriasis treated either with efalizumab 1.0 mg/kg/wk or placebo were followed for 12 weeks. DRQL and psoriasis symptom severity were assessed at baseline (pre-treatment) and at the end of the first treatment phase (12 weeks). DRQL was measured using the Dermatology Life Quality Index (DLQI). Symptoms were measured using the Psoriasis Symptom Assessment (PSA) and an Itch scale. Disease burden was assessed at baseline by examining responses to individual questions of the DLQI, PSA, and Itch patient-reported outcome measures. The impact of treatment on disease burden was assessed over a 12-week double-blind study period by comparing changes in DLQI, PSA, and Itch scale scores between the active treatment and placebo groups. Patient-reported outcomes were also assessed during a 12-week extended treatment phase. Prior to treatment, the responses to DLQI and PSA items revealed significant disease burden. Greater than 90% of patients reported being embarrassed or self conscious because of their skin, 53% reported that their skin prevented them from working or studying. and 98% reported that scaling and itching was bothersome. Compared to placebo-treated patients, efalizumab-treated patients showed significant improvement in patient-reported outcomes, reducing the limitations and burden associated with moderate to severe psoriasis within each of the three studies, as measured by DLQI (p<0.001), PSA-Severity (p<0.001), PSA-Frequency (p<0.001), and Itch (p<0.001) scores. Across all measures, the proportion of patients that improved on both statistical and clinical criteria for meaningful improvement was at least twofold greater among efalizumab-treated patients than in placebo-treated patients. The benefit of efalizumab was maintained over the course of an additional 12 weeks during an extended treatment phase. In conclusion, patients with moderate to severe plaque psoriasis reported significant DRQL burden and symptom severity at baseline, but efalizumab significantly improved patient-reported DRQL and reduced the frequency and severity of psoriasis symptoms during 12-week double-blind and 12-week extended treatment periods.
本研究的目的是记录与中度至重度斑块状银屑病相关的疾病负担，并评估依法利珠单抗治疗银屑病对改善患者报告结局的影响。这包括对参与三项III期、随机、双盲、平行组、安慰剂对照、多中心临床试验以评估依法利珠单抗疗效和安全性的中度至重度银屑病患者的患者报告的皮肤病相关生活质量（DRQL）和银屑病症状评分进行分析。共有1242例中度至重度银屑病患者接受了1.0mg/kg/周的依法利珠单抗或安慰剂治疗，随访12周。在基线（治疗前）和第一个治疗阶段结束时（12周）评估DRQL和银屑病症状严重程度。使用皮肤病生活质量指数（DLQI）测量DRQL。使用银屑病症状评估（PSA）和瘙痒量表测量症状。通过检查对DLQI、PSA和瘙痒患者报告结局测量的各个问题的回答，在基线时评估疾病负担。通过比较活性治疗组和安慰剂组之间DLQI、PSA和瘙痒量表评分的变化，在为期12周的双盲研究期间评估治疗对疾病负担的影响。在为期12周的延长治疗阶段也评估了患者报告的结局。治疗前，对DLQI和PSA项目的回答显示出显著的疾病负担。超过90%的患者报告因皮肤问题感到尴尬或自觉，53%的患者报告皮肤问题妨碍了他们工作或学习，98%的患者报告鳞屑和瘙痒令人烦恼。与接受安慰剂治疗的患者相比，接受依法利珠单抗治疗的患者在患者报告结局方面有显著改善，在三项研究中的每一项中，均降低了与中度至重度银屑病相关的限制和负担，这通过DLQI（p<0.001）、PSA-严重程度（p<0.001）、PSA-频率（p<0.001）和瘙痒（p<0.001）评分来衡量。在所有测量指标中，依法利珠单抗治疗的患者中在统计学和临床意义上有显著改善的患者比例至少是接受安慰剂治疗患者的两倍。在延长治疗阶段的另外12周期间，依法利珠单抗的益处得以维持。总之，中度至重度斑块状银屑病患者在基线时报告有显著的DRQL负担和症状严重程度，但依法利珠单抗在12周双盲和12周延长治疗期间显著改善了患者报告的DRQL，并降低了银屑病症状的频率和严重程度。
