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雷珠单抗:早产儿视网膜病变的治疗进展。

Ranibizumab: A Review in Retinopathy of Prematurity.

机构信息

Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand.

出版信息

Paediatr Drugs. 2021 Jan;23(1):111-117. doi: 10.1007/s40272-020-00433-z. Epub 2021 Jan 15.

Abstract

Ranibizumab (Lucentis) is a monoclonal antibody fragment targeted against VEGF-A that is the first approved anti-VEGF agent for the treatment of retinopathy of prematurity (ROP). In the pivotal, randomized, phase III RAINBOW trial in infants with ROP, the majority of intravitreal ranibizumab recipients experienced treatment success at 24 weeks, with a numerically greater treatment success rate in the ranibizumab 0.2 mg (80% of patients) than laser therapy (66%) group without reaching statistical significance for superiority. Long-term effects on vision following ranibizumab treatment are not yet known, but interim analyses from the RAINBOW extension study do not show evidence of degraded vision. Adverse reactions to ranibizumab in pediatric patients were consistent with the known safety profile in adults, with most adverse reactions attributed to the intravitreal injection procedure. Furthermore, systemic VEGF suppression was not observed in clinical trials, which is congruent with the rapid systemic clearance of ranibizumab. Overall, ranibizumab is an effective and generally well tolerated treatment for ROP and is not associated with systemic VEGF suppression. Although results for its long-term effects on vision are not yet available, ranibizumab is a promising alternative option to laser therapy for treating ROP.

摘要

雷珠单抗(Lucentis)是一种针对 VEGF-A 的单克隆抗体片段,是首个被批准用于治疗早产儿视网膜病变(ROP)的抗 VEGF 药物。在 ROP 婴儿的关键性、随机、III 期 RAINBOW 试验中,大多数接受玻璃体内雷珠单抗治疗的患者在 24 周时达到治疗成功,雷珠单抗 0.2mg 组(80%的患者)的治疗成功率高于激光治疗(66%)组,但未达到优势的统计学意义。雷珠单抗治疗后的长期视力影响尚不清楚,但 RAINBOW 扩展研究的中期分析并未显示视力下降的证据。儿科患者使用雷珠单抗的不良反应与成人已知的安全性特征一致,大多数不良反应归因于玻璃体内注射过程。此外,临床试验中未观察到全身性 VEGF 抑制,这与雷珠单抗的快速全身清除一致。总体而言,雷珠单抗是 ROP 的一种有效且通常耐受良好的治疗方法,与全身性 VEGF 抑制无关。尽管其对视力的长期影响结果尚不清楚,但雷珠单抗是治疗 ROP 的激光治疗的一种有前途的替代选择。

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