Premature infants with severe retinopathy of prematurity (ROP) are often treated using anti–vascular endothelial growth factor drugs such as bevacizumab. Intravitreal bevacizumab reaches the systemic circulation and is associated with reduced serum vascular endothelial growth factor levels in premature infants.¹ Vascular endothelial growth factor is necessary for normal development of organs, so blocking its systemic action could be harmful to premature infants.^2,3 We conducted a dose de-escalation phase 1 study, which found that very low doses of bevacizumab are effective in treating severe ROP.^4,5 To determine which dose to carry forward to a large-scale randomized clinical trial, we applied a bayesian approach to analyze data across 8 dose levels. Herein we report those results (NCT04634604).

患有严重早产儿视网膜病变 (ROP) 的早产儿通常使用贝伐单抗等抗血管内皮生长因子药物进行治疗。玻璃体内贝伐单抗到达体循环并与早产儿血清血管内皮生长因子水平降低有关。¹血管内皮生长因子是器官正常发育所必需的，因此阻断其全身作用可能对早产儿有害。^{2 ,3}我们进行了剂量降级 1 期研究，发现极低剂量的贝伐单抗可有效治疗严重 ROP。^{4 ,5}为了确定将哪种剂量用于大规模随机临床试验，我们应用贝叶斯方法分析 8 个剂量水平的数据。我们在此报告这些结果 (NCT04634604)。