Virginia Eye Consultants (J.S.), Norfolk, VA; Kannarr Eye Care (S.K.), Pittsburg, KS; Florida Vision Institute (J.L.), West Palm Beach, FL; Ophthalmology Associates (R.M.), St. Louis, MO; Sun Pharmaceutical Industries, Inc. (A.J., A.O.), Princeton, NJ; Sun Pharma Advanced Research Company Ltd (C.D.), Princeton, NJ; and North Bay Eye Associates (J.B.), Petaluma, CA.
Eye Contact Lens. 2020 Jan;46 Suppl 1:S14-S19. doi: 10.1097/ICL.0000000000000636.
OTX-101 (CEQUA™) is approved in the United States for treatment of keratoconjunctivitis sicca (KCS). This pooled analysis of 2 studies (phase 2b/3 and phase 3) evaluates the efficacy and safety of OTX-101 0.09% in the intent-to-treat (ITT) population and the subgroup of patients with a baseline Schirmer score less than 10 mm.
In these randomized, multicenter, double-masked, vehicle-controlled studies, patients received 1 drop of either OTX-101 or vehicle in both eyes twice daily. A Schirmer's test was performed at baseline and day 84/early discontinuation. Symptom Assessment iN Dry Eye (SANDE) scores and adverse events were monitored at each visit.
The pooled analysis included 523 and 525 patients randomized to OTX-101 0.09% and vehicle, respectively. In the ITT population, 16.6% of eyes receiving OTX-101 and 9.0% of eyes receiving vehicle showed a day 84 increase in Schirmer score ≥10 mm from baseline (P<0.0001). In the subgroup with Schirmer score less than 10 mm at baseline, 18.7% and 10.2% of eyes receiving OTX-101 and vehicle, respectively, exhibited this outcome (P=0.0001). The mean (SD) percent change from baseline in global SANDE scores on day 84 in the ITT population was -29.0% (39.0%) and -30.4% (39.5%) for OTX-101 and vehicle groups, respectively. In the subgroup, the mean (SD) percent change was -27.3% (39.7%) and -31.4% (38.3%) for OTX-101 and vehicle groups, respectively. Adverse events were mostly mild to moderate.
OTX-101 improved tear production compared with vehicle. Both OTX-101 and vehicle showed improved SANDE scores over baseline. OTX-101 was well tolerated in patients with KCS.
OTX-101（CEQUA™）在美国获批用于治疗干燥性角膜结膜炎（KCS）。这两项 2 期 b/3 期和 3 期研究的汇总分析评估了 OTX-101 0.09%在意向治疗（ITT）人群和基线 Schirmer 评分小于 10mm 的患者亚组中的疗效和安全性。
在这些随机、多中心、双盲、安慰剂对照研究中，患者每天两次在双眼各滴用 1 滴 OTX-101 或安慰剂。在基线和第 84 天/提前停药时进行 Schirmer 测试。在每次就诊时监测症状评估干燥眼（SANDE）评分和不良事件。
汇总分析纳入了 523 例和 525 例分别随机分配至 OTX-101 0.09%和安慰剂的患者。在 ITT 人群中，16.6%接受 OTX-101 治疗的眼和 9.0%接受安慰剂治疗的眼在第 84 天从基线时 Schirmer 评分增加≥10mm（P<0.0001）。在基线时 Schirmer 评分小于 10mm 的亚组中，18.7%和 10.2%接受 OTX-101 和安慰剂治疗的眼分别观察到这一结果（P=0.0001）。在 ITT 人群中，第 84 天从基线的全球 SANDE 评分的平均（SD）百分比变化为 -29.0%（39.0%）和 -30.4%（39.5%），分别为 OTX-101 和安慰剂组。在亚组中，平均（SD）百分比变化分别为 -27.3%（39.7%）和 -31.4%（38.3%），分别为 OTX-101 和安慰剂组。不良事件大多为轻度至中度。
与安慰剂相比，OTX-101 增加了泪液产生。OTX-101 和安慰剂均较基线时 SANDE 评分改善。OTX-101 在 KCS 患者中耐受良好。
