Westborg Inger, Al-Najjar Ayat, Norberg Helena
Department of Clinical Sciences/Ophthalmology, Umea University Faculty of Medicine, Umea, Sweden.
Department of Medical and Translational Biology, Umea University Faculty of Medicine, Umea, Sweden.
BMJ Open. 2025 Feb 5;15(2):e089801. doi: 10.1136/bmjopen-2024-089801.
To investigate the eligibility for faricimab in a real-world diabetic macular oedema (DMO) population to the YOSEMITE and RHINE trials, and to compare the eligible DMO populations to the trial populations.
DESIGN, SETTINGS AND PARTICIPANTS: This retrospective cross-sectional analysis used data from the Swedish Macula Registry (SMR) between 1 January 2019 and 31 August 2023. Eligibility criteria mirrored the main criteria of the YOSEMITE and RHINE trials: (1) DMO diagnosis, (2) treatment-naïve, (3) 18 years or older, (4) central retinal thickness (CRT) 325 µm or higher and (5) best-corrected visual acuity (BCVA) ranging from 25 to 73 letters. Individuals with registered proliferative diabetic retinopathy (DR) at the start of treatment were excluded. A secondary selection of eligible individuals was conducted using the same criteria, except for BCVA, which ranged from 25 to 77 letters according to national guidelines (treatment practice).
Characteristics at the initial visit of the two eligible SMR populations were compared with baseline data from the clinical trials, respectively.
In total, 3777 individuals with DMO were selected from SMR. Of these, 2357 (62.4%) individuals were treatment-naïve, all were 18 years or older, 1928 (51.0%) exhibited CRT≥325 µm, 1175 (31.1%) had 25-73 letters based on phase III studies, while 1528 (40.5%) had 25-77 letters according to treatment practice. After excluding individuals with registered proliferative DR 1171 (31.0%) individuals in the SMR met all criteria based on phase III studies, while 1522 (40.3%) individuals fulfilled the criteria according to treatment practice. The SMR and treatment practice populations were older (YOSEMITE 67.5±11.6 vs 62.8±10.0 years, p<0.001 and RHINE 67.5±11.6 vs 61.6±10.1 years, p<0.001) than those in the phase III studies and had lower CRT (YOSEMITE 446±96 vs 486±131 µm, p<0.001 and RHINE 446±96 vs 471±127 µm, p=0.001).
Approximately 30% of DMO patients in the SMR met the main trial criteria from YOSEMITE and RHINE, while around 40% met the criteria based on treatment practice. The SMR and treatment practice cohorts were older and had less severe DMO than the trial cohorts. Further research into the safety profile of faricimab in clinical settings is necessary, along with the consideration of additional eligibility criteria when implementing faricimab in ophthalmology practice.
研究在真实世界糖尿病性黄斑水肿（DMO）人群中，法西单抗符合优山美地（YOSEMITE）和莱茵河（RHINE）试验入选标准的情况，并将符合标准的DMO人群与试验人群进行比较。
设计、背景与参与者：这项回顾性横断面分析使用了瑞典黄斑登记处（SMR）2019年1月1日至2023年8月31日的数据。入选标准与优山美地和莱茵河试验的主要标准一致：（1）DMO诊断；（2）未接受过治疗；（3）年龄18岁及以上；（4）中心视网膜厚度（CRT）≥325µm；（5）最佳矫正视力（BCVA）为25至73个字母。治疗开始时登记有增殖性糖尿病视网膜病变（DR）的个体被排除。使用相同标准对符合条件的个体进行二次筛选，但根据国家指南（治疗实践），BCVA范围为25至77个字母。
将两个符合条件的SMR人群初次就诊时的特征分别与临床试验的基线数据进行比较。
共从SMR中选取了3777例DMO患者。其中，2357例（62.4%）未接受过治疗，均为18岁及以上，1928例（51.0%）CRT≥325µm，根据III期研究，1175例（31.1%）BCVA为25至73个字母，而根据治疗实践，1528例（40.5%）BCVA为25至77个字母。排除登记有增殖性DR的个体后，根据III期研究，SMR中有1171例（31.0%）个体符合所有标准，而根据治疗实践，1522例（40.3%）个体符合标准。SMR人群和治疗实践人群比III期研究中的人群年龄更大（优山美地试验：67.5±11.6岁对62.8±10.0岁，p<0.001；莱茵河试验：67.5±11.6岁对61.6±10.1岁，p<0.001），CRT更低（优山美地试验：446±96µm对486±131µm，p<0.001；莱茵河试验：446±96µm对471±127µm，p=0.001）。
SMR中约30%的DMO患者符合优山美地和莱茵河试验的主要标准，约40%的患者符合基于治疗实践的标准。SMR人群和治疗实践队列比试验队列年龄更大，DMO病情较轻。有必要进一步研究法西单抗在临床环境中的安全性，同时在眼科实践中应用法西单抗时考虑额外的入选标准。
