Background: The simultaneous presentation of systemic diffuse large B cell lymphoma (DLBCL) with central nervous system (CNS) disease is not well controlled by either R-CHOP or systemic methotrexate (MTX) alone. Methods: We conducted a pilot trial with 6 patients who were initially diagnosed with systemic DLBCL with CNS involvement. Patients were treated with a systemic MTX plus R-CHOP regimen. Results: The overall response rate was 4/6 (66.7%). The CNS response rate and systemic response rate were 4/6 (66.7%) and 5/6 (83.3%), respectively. The median response duration of the 4 patients with complete remission at completion was 25.5 months, and the median survival of all patients was 25.1 months. CNS lesions progressed in all relapsed and refractory patients, while systemic disease progression was observed in 1 patient. No fatal hematologic adverse effects, hepatotoxicity or nephrotoxicity were observed. Conclusions: The dose of systemic MTX (1∼1.5 g/m2) or dose intensity (4-week interval in 4 patients) used in this trial was considered insufficient. Therefore, the dose of MTX or interval of each chemotherapy cycle should be modified in future trials to control CNS disease involved with DLBCL. © 2014 S. Karger AG, Basel

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