Long-term observation of treatment strategies for primary vitreoretinal lymphoma in a multicenter Chinese cohort

To compare outcomes of different treatment regimens for primary vitreoretinal lymphoma (PVRL) without central nervous system (CNS) involvement, specifically assessing their association with subsequent onset of central nervous system lymphoma (CNSL). This retrospective study collected data from 64 PVRL patients (118 eyes) from January 2015 to June 2023 at 4 ophthalmologic centers in China. Clinical, laboratory, and imaging data of these patients has been reviewed, focusing on the incidence of CNS manifestations during follow-up. Among the 64 patients with PVRL, 26 patients received only intravitreal injection of methotrexate (group A), and 38 patients received combined intraocular and systemic treatment (group B). The median follow-up time was 36.1 months, the median CNS progression-free survival (PFS) was 23.5 months, and the median overall survival (OS) was not-reached. CNSL developed in 44 of the 64 patients (68.8%) during the follow-up, including 19 of the 26 patients (73.1%) in group A, and 25 of the 38 (65.8%) in group B. Increased cerebrospinal fluid IL-10 levels (range: 2.2–472 pg/ml, mean 63.715 pg/ml) were significantly correlated with the progression of CNSL (P < 0.001). In this observational cohort, combined therapeutic approach of systemic and intraocular therapy was associated with higher OS (P = 0.002). No significant differences in CNS-PFS was observed between the two treatment groups (P = 0.095). In this cohort of patients with PVRL, combined intraocular and systemic therapy may be associated with longer OS, while no significant difference in CNS-PFS was observed. Primary vitreoretinal lymphoma is an aggressive intraocular malignancy with a high risk of subsequent central nervous system (CNS) involvement, but the optimal treatment strategy for patients without CNS disease at diagnosis remains controversial. In this multicenter Chinese cohort (64 patients without baseline CNS involvement), combined intraocular and systemic therapy appeared to be associated with improved overall survival compared with intravitreal methotrexate alone. The risk of CNS lymphoma progression remained high regardless of treatment strategy, and no significant difference in CNS progression-free survival was observed between treatment groups. Elevated cerebrospinal fluid IL-10 levels were significantly associated with subsequent CNS involvement, supporting its potential role as a prognostic biomarker in clinical decision-making.