雷珠单抗联合光动力疗法治疗老年退行性晚期渗出型黄斑变性:自身对照临床试验方案和预试验结果

Ranibizumab combined with photodynamic therapy for the treatment of advanced-stage exudative age-related macular degeneration: study protocol for a self-controlled trial and preliminary results

背景:老年退行性黄斑变性视觉中心视野缺失会对患者日常生活活动造成严重影响,其可根据病理表现的不同分为萎缩型和渗出型2种,其中渗出型老年退行性黄斑变性进入晚期后,黄斑区结构出现老年退行性变化。临床上可采用雷珠单抗联合光动力疗法治疗渗出型老年退行性黄斑变性,但是既往研究很少关注晚期渗出型老年退行性黄斑变性患者。目的:对雷珠单抗联合光动力疗法治疗的晚期渗出型老年退行性黄斑变性患者的视力恢复情况、视网膜厚度及脉络膜新生血管病灶渗漏情况的变化进行分析,探索这种疗法在治疗晚期渗出型老年退行性黄斑变性的有效性。方法:此次前瞻性自身对照临床试验在中国青海大学附属医院眼科完成。试验纳入113例符合标准的晚期渗出型老年退行性黄斑变性患者,采用光动力疗法联合玻璃体腔注射雷珠单抗治疗。结果与结论:试验的主要观察指标为治疗后12个月患者视力改善率。次要观察指标为治疗后1-12个月,每个月的对数视力表评分、视网膜厚度和脉络膜新生血管病灶渗漏治疗以及随访期间的不良事件。预试验结果显示,治疗后12个月时,43只患眼视力比治疗前提高≥2行,患者视网膜厚度平均下降111.21μm,多数患眼脉络膜新生血管病灶渗漏停止或缩小。试验旨在提供关于雷珠单抗联合光动力疗法治疗晚期渗出型老年退行性黄斑变性效果的可靠数据,从而为该方法治疗晚期渗出型老年退行性黄斑变性的合理使用提供依据。试验在中国临床试验注册中心注册(注册号:Chi CTR1800015410),注册方案版本号1.0。

BACKGROUND： The lack of visual field in the visual center of older adults with aged-related macular degeneration（AMD） will have a serious impact on the activities of daily living. There are atrophic and exudative AMD according to the pathological manifestations. Patients with advanced stage of exudative AMD will develop aged-related degeneration in the macular area. Ranibizumab combined with photodynamic therapy is mainly used for current treatment of exudative AMD, but more attention is paid to patients with early exudative AMD. OBJECTIVE： To analyze vision recovery, retinal thickness, and leakage of choroidal neovascular lesions in patients with advanced stage of exudative AMD after treatment with ranibizumab plus photodynamic therapy, and further explore the therapeutic efficacy of the combined therapy. METHODS： The prospective self-controlled trial will be completed in the Department of Ophthalmology, Affiliated Hospital of Qinghai University in China. The study population will comprise 113 patients with advanced exudative AMD who are eligible for inclusion and exclusion criteria. Photodynamic therapy combined with intravitreal injection of ranibizumab will be given as the interventional therapy. RESULTS AND CONCLUSION： The primary outcome measure of the trial is to investigate the visual improvement in patients 12 months after treatment. Secondary outcome measures include logarithmic visual acuity chart scores, retinal thickness, and leakage of choroidal neovascular lesions assessed monthly at 1-12 months after treatment, and adverse events during follow-up. Preliminary results showed that at 12 months after treatment, visual acuity in 43 affected eyes was increased by ≥ 2 lines, and the average retinal thickness decreased by 111.21 μm as compared with before treatment. Leakage of choroidal neovascular lesions in most affected eyes stopped or the lesions were contracted. Findings from the trial are expected to provide reliable data on the efficacy of ranibizumab combined with photodynamic therapy in the treatment of advanced exudative AMD, thereby providing a basis for the rational use of this combined therapy in the treatment of advanced exudative AMD. This trial was registered with the Chinese Clinical Trial Registry（registration number： Chi CTR1800015410）. Protocol version（1.0）.