Vermeulen Christie, van der Burg Lars L J, van Geloven Nan, Eggink Catharina A, Cheng Yanny Y Y, Nuijts Rudy M M A, Wisse Robert P L, van Luijk Chantal M, Nieuwendaal Carla, Remeijer Lies, van der Meer Pieter F, de Korte Dirk, Klei Thomas R L
Department of Product and Process Development, Sanquin Blood Bank, Plesmanlaan 125, 1066 CX, Amsterdam, The Netherlands.
Department of Biomedical Data Sciences, Leiden University Medical Center, Leiden, The Netherlands.
Ophthalmol Ther. 2023 Dec;12(6):3347-3359. doi: 10.1007/s40123-023-00827-5. Epub 2023 Oct 16.
Allogeneic serum from blood donors is starting to be used to treat patients with dry eye disease (DED). However, the optimal dose is not known. We therefore aimed to evaluate the clinical efficaciousness and user-friendliness of micro-sized versus conventional-sized allogeneic serum eye drops (SEDs).
In a randomized trial, patients with DED first receive micro-sized SEDs (7 µl/unit) for 1 month, followed by a 1-month washout, before receiving conventional-sized SEDs (50 µl/unit) for 1 month; or vice versa. The primary endpoint was the Ocular Surface Disease Index (OSDI) score. Secondary endpoints were tear break-up time (TBT), tear production (TP), and presence of corneal punctate lesions (CP). The user-friendliness of both application systems was also compared. A linear mixed model for cross-over design was applied to compare both treatments.
Forty-nine patients completed the trial. The mean OSDI score significantly improved from 52 ± 3 to 41 ± 3 for micro-sized SEDs, and from 54 ± 3 to 45 ± 3 for conventional-sized SEDs. Non-inferiority (margin = 6) of micro-sized SEDs was established. We demonstrate a significant improvement for TBT in case of conventional-sized SEDs and for CP in both treatment groups. TP trended towards an improvement in both treatment groups. The user-friendliness of the conventional drop system was significantly higher.
For the first time, non-inferiority of micro-sized allogeneic SEDs was established. The beneficial effect of both SED volumes was similar as measured by the OSDI score. Although user-friendliness of the micro drop system was significantly lower, it is an attractive alternative as it saves valuable donor serum.
ClinicalTrials.gov (NCT03539159).
来自献血者的异体血清开始用于治疗干眼症（DED）患者。然而，最佳剂量尚不清楚。因此，我们旨在评估微型与传统尺寸的异体血清滴眼液（SED）的临床疗效和用户友好性。
在一项随机试验中，DED患者首先接受微型SED（7微升/单位）治疗1个月，随后进行1个月的洗脱期，然后接受传统尺寸的SED（50微升/单位）治疗1个月；或者反之。主要终点是眼表疾病指数（OSDI）评分。次要终点是泪膜破裂时间（TBT）、泪液分泌（TP）和角膜点状病变（CP）的存在情况。还比较了两种给药系统的用户友好性。采用交叉设计的线性混合模型来比较两种治疗方法。
49名患者完成了试验。微型SED的平均OSDI评分从52±3显著改善至41±3，传统尺寸SED的平均OSDI评分从54±3显著改善至45±3。确定了微型SED的非劣效性（界值 = 6）。我们证明传统尺寸SED治疗时TBT有显著改善，两个治疗组的CP均有改善。两个治疗组的TP均有改善趋势。传统滴眼液系统的用户友好性显著更高。
首次确定了微型异体SED的非劣效性。通过OSDI评分衡量，两种SED体积的有益效果相似。尽管微型滴眼液系统的用户友好性显著较低，但它是一种有吸引力的选择，因为它节省了宝贵的供体血清。
ClinicalTrials.gov（NCT03539159）。
