Guan Wenxue, Wang Liang, Peng Xiaoyan
Department of Ophthalmology, Beijing Tongren Hospital, Capital Medical University, Beijing, 100005, China.
Department of Hematology, Beijing Tongren Hospital, Capital Medical University, Beijing, 100005, China.
Exp Hematol Oncol. 2022 Nov 8;11(1):95. doi: 10.1186/s40164-022-00354-2.
Vitreoretinal lymphoma (VRL) is strongly linked to central nervous system (CNS) progression with no standard treatment approaches. Commonly used strategies include repeated intraocular injections of low-dose methotrexate or local radiotherapy, with great inconvenience, long-term side effects, and high risk of CNS relapse. In this study, we evaluated the efficacy and safety of bruton's tyrosine kinase inhibitors (BTKi) in the treatment of VRL. This prospective single-center study enrolled patients with relapsed or newly diagnosed VRL between October 2020 and April 2022. Patients received BTKi monotherapy until disease progression or unacceptable toxicity. The primary endpoint was the disease control (DC) rate after one month of treatment; secondary endpoints include toxicity, overall survival (OS), and progression-free survival (PFS). Ten consecutive patients with VRL were enrolled into this study. After 1-month treatment, 9 patients (90%) achieved a DC, with 7 patients (70%) achieving a complete response (CR). With a median follow-up of 8.3 (2.5-21.4) months, 4 patients were confirmed to have disease progression, with a PFS of 1.2, 7.5, 9.1, and 11.6 months, respectively. The remaining 6 patients have durable control of disease and were still on treatment at time of the analysis. BTKi were well-tolerated and no patients discontinued the drug because of adverse events. In conclusion, targeting BTK in VRL is viable, and our findings could pave the way for a paradigm change in VRL therapy choices. Further large-scale studies, however, are required to give stronger evidence about the efficacy and safety.
玻璃体视网膜淋巴瘤（VRL）与中枢神经系统（CNS）进展密切相关，且尚无标准治疗方法。常用策略包括反复眼内注射低剂量甲氨蝶呤或局部放疗，但存在极大不便、长期副作用以及CNS复发的高风险。在本研究中，我们评估了布鲁顿酪氨酸激酶抑制剂（BTKi）治疗VRL的疗效和安全性。这项前瞻性单中心研究纳入了2020年10月至2022年4月期间复发或新诊断的VRL患者。患者接受BTKi单药治疗，直至疾病进展或出现不可接受的毒性。主要终点是治疗1个月后的疾病控制（DC）率；次要终点包括毒性、总生存期（OS）和无进展生存期（PFS）。连续10例VRL患者纳入本研究。治疗1个月后，9例患者（90%）达到DC，7例患者（70%）达到完全缓解（CR）。中位随访8.3（2.5 - 21.4）个月时，4例患者被证实疾病进展，PFS分别为1.2、7.5、9.1和11.6个月。其余6例患者疾病得到持久控制，在分析时仍在接受治疗。BTKi耐受性良好，没有患者因不良事件停药。总之，在VRL中靶向BTK是可行的，我们的发现可能为VRL治疗选择的范式转变铺平道路。然而，需要进一步的大规模研究来提供关于疗效和安全性的更有力证据。
