Armbrust Karen R, Fox Austin R, Jeffrey Brett G, Sherry Patti, Sen H Nida
Laboratory of Immunology, National Eye Institute, National Institutes of Health, Bethesda, MD, USA.
Department of Ophthalmology, Veterans Affairs Health Care System, Minneapolis, MN, USA.
Taiwan J Ophthalmol. 2020 Jul 27;11(1):64-70. doi: 10.4103/tjo.tjo_32_20. eCollection 2021 Jan-Mar.
This prospective study evaluates whether rituximab is a safe and potentially effective treatment for nonparaneoplastic autoimmune retinopathy (npAIR).
Five npAIR patients were enrolled in a Phase I/II, prospective, nonrandomized, open-label, single-center study. All patients received a cycle of 1000 mg intravenous rituximab at weeks 0 and 2, with a second cycle of rituximab 6 to 9 months later. Clinical evaluation was performed at baseline, 6 and 12 weeks after each rituximab cycle, and then every 3 months for a total duration of 18 months. The primary outcome for this study was treatment success based on visual field and full-field electroretinography at 6 months. The secondary outcomes included treatment success at months 12 and 18, drug-related adverse events, changes in visual symptoms, and changes in quality of life.
Two patients met criteria for treatment success: one based solely on electroretinography and the other based solely on visual field area, but treatment success was not sustained. Clinical response over the course of the 18-month study showed disease stabilization in three patients and treatment failure in two patients. There were no severe drug-related adverse events.
This is the first clinical trial prospectively evaluating the effect of rituximab in npAIR and, although rituximab was well tolerated, there was no clear-cut clinical improvement conferred by B cell depletion with rituximab.
本前瞻性研究评估利妥昔单抗治疗非副肿瘤性自身免疫性视网膜病变（npAIR）是否安全且可能有效。
五名npAIR患者参加了一项I/II期前瞻性、非随机、开放标签、单中心研究。所有患者在第0周和第2周接受一个周期的1000mg静脉注射利妥昔单抗，6至9个月后接受第二个利妥昔单抗周期治疗。在基线、每个利妥昔单抗周期后的第6周和第12周进行临床评估，然后每3个月评估一次，共持续18个月。本研究的主要结局是基于6个月时的视野和全视野视网膜电图评估治疗是否成功。次要结局包括12个月和18个月时的治疗成功情况、药物相关不良事件、视觉症状变化和生活质量变化。
两名患者达到治疗成功标准：一名仅基于视网膜电图，另一名仅基于视野面积，但治疗成功未持续。在18个月的研究过程中，临床反应显示三名患者病情稳定，两名患者治疗失败。未出现严重的药物相关不良事件。
这是第一项前瞻性评估利妥昔单抗对npAIR疗效的临床试验，尽管利妥昔单抗耐受性良好，但利妥昔单抗导致的B细胞耗竭并未带来明显的临床改善。
