Phase II Randomized, Double-Masked, Vehicle-Controlled Trial of Recombinant Human Nerve Growth Factor for Neurotrophic Keratitis
Phase II Randomized, Double-Masked, Vehicle-Controlled Trial of Recombinant Human Nerve Growth Factor for Neurotrophic Keratitis
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DOI：
10.1016/j.ophtha.2018.02.022
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发表时间：
2018-09-01
期刊：
影响因子：
13.7
通讯作者：
Mantelli, Flavio
中科院分区：
文献类型：
--
作者：
Bonini, Stefano;Lambiase, Alessandro;Mantelli, Flavio
目的：评估局部重组人神经生长因子 (rhNGF) 治疗中重度神经营养性角膜炎 (NK) 的安全性和有效性，这是一种由角膜神经支配受损引起的罕见退行性角膜疾病。设计：II 期多中心、随机、双盲、载体对照试验。参与者：1 只眼睛患有 2 期（中度）或 3 期（重度）NK 的患者。方法：REPARO II 期试验研究评估了 156 名患者的安全性和有效性，这些患者以 1:1:1 的比例随机接受 rhNGF 10 μg/ml、20 μg/ml 或载体。治疗每天 6 滴，持续 8 周。然后患者进入 48 或 56 周的随访期。对接受研究治疗的所有患者进行安全性评估，而疗效则根据治疗意向进行评估。 主要结果指标：角膜愈合（定义为
Purpose: To evaluate the safety and efficacy of topical recombinant human nerve growth factor (rhNGF) for treating moderate-to-severe neurotrophic keratitis (NK), a rare degenerative corneal disease resulting from impaired corneal innervation.Design: Phase II multicenter, randomized, double-masked, vehicle-controlled trial.Participants: Patients with stage 2 (moderate) or stage 3 (severe) NK in 1 eye.Methods: The REPARO phase II study assessed safety and efficacy in 156 patients randomized 1:1:1 to rhNGF 10 mu g/ml, 20 mu g/ml, or vehicle. Treatment was administered 6 drops per day for 8 weeks. Patients then entered a 48- or 56-week follow-up period. Safety was assessed in all patients who received study treatment, whereas efficacy was by intention to treat.Main Outcome Measures: Corneal healing (defined as