A phase II randomized double-masked trial of pegaptanib, an anti-vascular endothelial growth factor aptamer, for diabetic macular edema
A phase II randomized double-masked trial of pegaptanib, an anti-vascular endothelial growth factor aptamer, for diabetic macular edema
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DOI：
10.1016/j.ophtha.2005.06.007
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发表时间：
2005-10-01
期刊：
影响因子：
13.7
通讯作者：
Schwartz, SD
中科院分区：
文献类型：
--
作者：
Cunningham, ET Jr;Adamis, AP;Schwartz, SD
目的：评价培加他尼钠注射液（pegaptanib）治疗糖尿病黄斑水肿（DME）的安全性和有效性。设计：随机、双盲、多中心、剂量范围、对照试验。受试者：研究眼最佳矫正视力（VA）在20/50至20/320之间且研究者判定DME累及黄斑中心的个体可以安全地停止光凝治疗 16 周。 干预：在研究开始时、第 6 周和第 12 周进行玻璃体内培加他尼（0.3 mg、1 mg、3 mg）或假注射，并根据需要进行额外注射和/或局部光凝治疗，持续 18 周。最终评估在第 36 周进行。主要结果指标：最佳矫正视力、通过光学相干断层扫描测量评估的中央子视野中心点的中央视网膜厚度，以及第 12 周至第 36 周之间的光凝附加治疗。结果：172 名患者的基线人口统计学和眼部特征表现出平衡。与假手术 (20/63) 相比，第 36 周时 0.3 mg (20/50) 的中位 VA 更好 (P = 0.04)。接受 0.3 mg 治疗的患者中，较大比例的 VA ≥ 10 个字母（约 2 行）（34% vs. 10%，P = 0.003）和 >= 15 个字母（18% vs. 7%，P = 0.12）。 0.3 mg 组的平均中央视网膜厚度减少 68 Am，而假手术组平均中央视网膜厚度增加 4 Am（P = 0.02）。接受 0.3 mg 治疗的患者中，较大比例的绝对下降量≥100μm（42% vs. 16%，P = 0.02）和≥75μm（49% vs. 19%，P = 0.008）。在每个培加他尼组中，认为需要光凝治疗的受试者较少（0.3 mg 与假手术组，25% 与 48%；P = 0.04）。所有哌加他尼剂量均耐受良好。 652 次注射中有 1 次发生眼内炎（0.15%/次注射；即 1/130 [0.8%] 培加他尼受试者），并且与严重视力丧失无关。结论：在这项 II 期试验中，分配至培加他尼的受试者具有更好的 VA 结果，更有可能显示中央视网膜厚度减少，并且被认为不太可能在随访时需要额外的光凝治疗。
Objective: To evaluate the safety and efficacy of pegaptanib sodium injection (pegaptanib) in the treatment of diabetic macular edema (DME).Design: Randomized, double-masked, multicenter, dose-ranging, controlled trial.Participants: Individuals with a best-corrected visual acuity (VA) between 20/50 and 20/320 in the study eye and DME involving the center of the macula for whom the investigator judged photocoagulation could be safely withheld for 16 weeks.Intervention: Intravitreous pegaptanib (0.3 mg, 1 mg, 3 mg) or sham injections at study entry, week 6, and week 12 with additional injections and/or focal photocoagulation as needed for another 18 weeks. Final assessments were conducted at week 36.Main Outcome Measures: Best-corrected VA, central retinal thickness at the center point of the central subfield as assessed by optical coherence tomography measurement, and additional therapy with photocoagulation between weeks 12 and 36.Results: One hundred seventy-two patients appeared balanced for baseline demographic and ocular characteristics. Median VA was better at week 36 with 0.3 mg (20/50), as compared with sham (20/63) (P = 0.04). A larger proportion of those receiving 0.3 mg gained VAs of >= 10 letters (approximately 2 lines) (34% vs. 10%, P = 0.003) and >= 15 letters (18% vs. 7%, P = 0.12). Mean central retinal thickness decreased by 68 Am with 0.3 mg, versus an increase of 4 Am with sham (P = 0.02). Larger proportions of those receiving 0.3 mg had an absolute decrease of both >= 100 mu m (42% vs. 16%, P = 0.02) and >= 75 mu m (49% vs. 19%, P = 0.008). Photocoagulation was deemed necessary in fewer subjects in each pegaptanib arm (0.3 mg vs. sham, 25% vs. 48%; P = 0.04). All pegaptanib doses were well tolerated. Enclophthalmitis occurred in 1 of 652 injections (0.15%/injection; i.e., 1/130 [0.8%] pegaptanib subjects) and was not associated with severe visual loss.Conclusions: In this phase II trial, subjects assigned to pegaptanib had better VA outcomes, were more likely to show reduction in central retinal thickness, and were deemed less likely to need additional therapy with photocoagulation at follow-up.