METHODS: This was a phase IV, multicenter, prospective, open-label, uncontrolled trial. Adults with stage 1 NK (Mackie criteria) and decreased corneal sensitivity (≤ 4 cm) received 1 drop of cenegermin 20 mcg/ml in the affected eye(s) 6 times/day for 8 weeks with a 24-week follow-up.
RESULTS: Of 37 patients, corneal epithelial healing was observed in 84.8% (95% confidence interval [CI] 68.1-94.9%; P < 0.001) at week 8; 95.2% (95% CI 76.2-99.9%; P < 0.001) of those patients remained healed at the end of the 24-week follow-up (week 32). At week 8, 91.2% (95% CI 76.3-98.1%; P < 0.001) of patients experienced improved corneal sensitivity; this improvement was observed in 82.1% (95% CI 63.1-93.9%; P < 0.001) of patients at week 32. Mean best-corrected distance visual acuity change from baseline at week 8 was - 0.10 logMAR (standard deviation [SD], 0.15; 95% CI - 0.16 to - 0.05; P < 0.001) and at week 32 was - 0.05 logMAR (SD, 0.16; 95% CI - 0.11 to 0.01; P = 0.122). At weeks 8 and 32, 15.2% (95% CI 5.1-31.9%; P < 0.001) and 10.7% (95% CI 2.3-28.2%; P < 0.001) of patients, respectively, had a 15-letter gain from baseline. At least one adverse event (AE) was reported by 73.0% and 45.7% of patients during the treatment and follow-up periods, respectively. The most common treatment-related, treatment-emergent AEs were eye pain (37.8%), blurred vision (10.8%), and eyelid pain (8.1%); these were mostly mild or moderate and were only reported during the treatment period.
CONCLUSIONS: These results support the potential use of cenegermin for treating patients with stage 1 NK, and future confirmatory studies would be beneficial to elaborate on these findings.
BACKGROUND: DEFENDO; NCT04485546.
方法:这是第四阶段,多中心,prospective,开放标签,不受控制的审判。患有1期NK(Mackie标准)和角膜敏感性降低(≤4cm)的成年人在受影响的眼睛中接受1滴cenegermin20mcg/ml,每天6次,共8周,并进行24周的随访。
结果:在37例患者中,在第8周,观察到84.8%(95%置信区间[CI]68.1~94.9%;P<0.001)的角膜上皮愈合;在24周随访结束时(第32周),这些患者中有95.2%(95%CI76.2~99.9%;P<0.001)仍愈合.在第8周,91.2%(95%CI76.3-98.1%;P<0.001)的患者角膜敏感性改善;在第32周,82.1%(95%CI63.1-93.9%;P<0.001)的患者观察到这种改善。在第8周时,与基线相比的平均最佳校正距离视力变化为-0.10logMAR(标准偏差[SD],0.15;95%CI-0.16至-0.05;P<0.001),第32周为-0.05logMAR(SD,0.16;95%CI-0.11至0.01;P=0.122)。在第8周和第32周,15.2%(95%CI5.1-31.9%;P<0.001)和10.7%(95%CI2.3-28.2%;P<0.001)的患者,分别,从基线增加了15个字母。在治疗和随访期间,73.0%和45.7%的患者至少报告了一次不良事件(AE)。分别。最常见的治疗相关,治疗中出现的不良事件是眼痛(37.8%),视力模糊(10.8%),和眼睑疼痛(8.1%);这些大多是轻度或中度,仅在治疗期间报告。
结论:这些结果支持cenegermin用于治疗1期NK患者的潜在用途。未来的验证性研究将有助于详细阐述这些发现。
背景:DEFENDO;NCT04485546。