RT Journal Article A1 Nguyen, Diem Trang A1 Cassoux, Nathalie A1 Choquet, Sylvain A1 Soussain, Carole A1 Le Cossec, Chloé A1 Omuro, Antonio A1 Lehoang, Phuc A1 Bodaghi, Bahram A1 Hoang Xuan, Khe A1 Touitou, Valerie A1 Reseau LOC (INCa) T1 Primary Oculo-Cerebral lymphoma (POCL): Efficacy of intravenous high-dose methotrexate (MTX) based polychemotherapy without radiotherapy on intraocular disease control JF Investigative Ophthalmology & Visual Science JO Invest. Ophthalmol. Vis. Sci. YR 2014 VO 55 IS 13 SP 6023 OP 6023 SN 1552-5783 AB Chemotherapy with radiotherapy (RT) is the current standard treatment of primary oculo-cerebral lymphoma (POCL) but exposes to a high risk of radiation-induced neurotoxicity, especially in the elderly. We recently reported encouraging results of 2 intravenous (IV) high-dose (HD) MTX based polychemotherapy regimens without RT in a randomized multicenter phase II trial (“CNS trial”) for primary central nervous system lymphoma patients over 60 years old (MPVA: HD-MTX, procarbazine, vincristine, and cytarabine versus HD-MTX-Tmz: MTX, temozolomide).The objective was to evaluate the efficacy of IV MTX-based chemotherapy on intraocular lymphoma (IOL) in POCL without RT. Patients with pathologically proven POCL randomized in the CNS trial, with baseline and follow-up ocular evaluations available were retrospectively analysed. The primary study end-points were the ocular response and event-free survival. Secondary outcome measures were: cerebral event-free survival and overall survival (OS) of both treatments. Thirteen patients out of 98 (9 males / 4 females, median age 63 years old) met the inclusion criteria. Six patients were randomized into the MPVA group and 7 into the MTX-Tmz group. Overall ocular complete response rate was 58% (MPVA: 67%, MTX-Tmz: 50% - p=0.99). Disease progressed in 17% of patients (MPVA: 17%, MTX-Tmz: 17%) and remained stable in 25% (MPVA: 17%, MTX-Tmz: 33%). With a median follow-up of 32 months, ocular relapse after an initial complete response was observed in 42% of patients (MPVA: 75%, MTX-Tmz: 67% - p=0.71). The median ocular event-free survival was 15 months (0-43) in the MPVA group, and 16.5 months (5-69) in the MTX-Tmz group (p=0.997). The median OS for the entire population was 32 months (6-69) without significant difference between the two groups (p=0.481), neither in the cerebral event-free survival (p=0.572). Systemic toxicities occurred in both groups (p>0.05) but were managable. Neurocognitve functions were well preserved. IV HD MTX based polychemotherapy as first line treatment is active in IOL in the setting of POCL. MPVA and MTX-Tmz demonstrate similar efficacy. Despite a good response rate, relapses remain frequent and improvement of intra-ocular disease control is needed. Future chemotherapy regimens and treatment strategies have to focus on this issue. RD 3/6/2021