Department of Biomedical and Clinical Science Luigi Sacco, Luigi Sacco Hospital, University of Milan, Milan, Italy.
Department of Ophthalmology & Visual Sciences, The Chinese University of Hong Kong, Hong Kong Eye Hospital, Hong Kong, Republic of China.
Ophthalmology. 2018 Jun;125(6):850-862. doi: 10.1016/j.ophtha.2017.12.002. Epub 2018 Jan 20.
To evaluate the efficacy and safety of ranibizumab 0.5 mg in adult patients with macular edema (ME) resulting from any cause other than diabetes, retinal vein occlusion, or neovascular age-related macular degeneration.
A phase 3, 12-month, double-masked, randomized, sham-controlled, multicenter study.
One hundred seventy-eight eligible patients aged ≥18 years.
Patients were randomized 2:1 to receive either ranibizumab 0.5 mg (n = 118) or sham (n = 60) at baseline and month 1. From month 2, patients in both arms received open-label individualized ranibizumab treatment based on disease activity. A preplanned subgroup analysis was conducted on the primary end point on 5 predefined baseline ME etiologies (inflammatory/post-uveitis, pseudophakic or aphakic, central serous chorioretinopathy, idiopathic, and miscellaneous).
Changes in best-corrected visual acuity (BCVA; Early Treatment Diabetic Retinopathy Study letters) from baseline to month 2 (primary end point) and month 12 and safety over 12 months.
Overall, 156 patients (87.6%) completed the study. The baseline characteristics were well balanced between the treatment arms. Overall, ranibizumab showed superior efficacy versus sham from baseline to month 2 (least squares mean BCVA, +5.7 letters vs. +2.9 letters; 1-sided P = 0.0111), that is, a treatment effect (TE) of +2.8 letters. The mean BCVA gain from baseline to month 12 was 9.6 letters with ranibizumab. The TE at month 2 was variable in the 5 predefined etiology subgroups, ranging from >5-letter gain to 0.5-letter loss. The safety findings were consistent with the well-established safety profile of ranibizumab.
The primary end point was met and ranibizumab showed superiority in BCVA gain over sham in treating ME due to uncommon causes, with a TE of +2.8 letters versus sham at month 2. At month 12, the mean BCVA gain was high (9.6 letters) in the ranibizumab arm; however, the TE was observed to be variable across the different etiology subgroups, reaching a >1-line TE in BCVA in patients with ME resulting from inflammatory conditions/post-uveitis or after cataract surgery. Overall, ranibizumab was well tolerated with no new safety findings up to month 12.
评估雷珠单抗 0.5mg 治疗非糖尿病性、视网膜静脉阻塞或新生血管性年龄相关性黄斑变性引起的黄斑水肿（ME）的疗效和安全性。
一项为期 12 个月的、3 期、双盲、随机、假对照、多中心研究。
178 名年龄≥18 岁的合格患者。
患者按 2：1 的比例随机分组，分别于基线和第 1 个月接受雷珠单抗 0.5mg（n=118）或假治疗（n=60）。从第 2 个月开始，两组患者均根据疾病活动度接受开放标签个体化雷珠单抗治疗。对 5 种预先设定的 ME 病因（炎症/葡萄膜炎后、白内障或无晶状体、中心性浆液性脉络膜视网膜病变、特发性和其他）的主要终点进行了预先计划的亚组分析。
从基线到第 2 个月（主要终点）和第 12 个月的最佳矫正视力（BCVA；糖尿病视网膜病变早期治疗研究字母）变化以及 12 个月期间的安全性。
共有 156 名患者（87.6%）完成了研究。治疗组之间的基线特征均衡。总体而言，与假治疗相比，雷珠单抗从基线到第 2 个月（最小二乘均值 BCVA，+5.7 个字母 vs. +2.9 个字母；单侧 P=0.0111）显示出更好的疗效，即治疗效果（TE）为+2.8 个字母。雷珠单抗组从基线到第 12 个月的平均 BCVA 增益为 9.6 个字母。第 2 个月时的 TE 在 5 个预先设定的病因亚组中各不相同，从增益超过 5 个字母到损失 0.5 个字母。安全性发现与雷珠单抗良好的安全性特征一致。
主要终点达到，与假治疗相比，雷珠单抗在治疗非常见原因引起的 ME 时在 BCVA 增益方面具有优势，第 2 个月时的 TE 为+2.8 个字母。在第 12 个月时，雷珠单抗组的平均 BCVA 增益较高（9.6 个字母）；然而，在不同病因亚组中观察到 TE 存在差异，在炎症/葡萄膜炎后或白内障手术后发生 ME 的患者中，BCVA 的 TE 达到 1 行以上。总体而言，雷珠单抗耐受性良好，截至第 12 个月时无新的安全性发现。
