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Kasenda, B.; Ihorst, G.; Schrörs, R.; Korfel, A.; Schmidt-Wolf, I.; Egerer, G.; Baumgarten, L. von; Röth, A.; Blöhdorn, J.; Möhle, R.; Binder, M.; Keller, U.; Lamprecht, M.; Pfreundschuh, M.; Valk, E.; Fricker, H.; Schorb, E.; Fritsch, K.; Finke, J.; Illerhaus, G. (2017): High-dose chemotherapy with autologous hämatopoietic stem cell support for relapsed or refractory primary CNS lymphoma: a prospective multicentre trial by the German Cooperative PCNSL study group. In: Leukemia, Vol. 31, No. 12: pp. 2623-2629
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Abstract

To investigate safety and efficacy of high-dose chemotherapy followed by autologous stem cell transplantation (HCT-ASCT) in relapsed/refractory (r/r) primary central nervous system lymphoma (PCNSL), we conducted a single-arm multicentre study for immunocompetent patients ( < 66 years) with PCNSL failing high-dose methotrexate)-based chemotherapy. Induction consisted of two courses of rituximab (375 mg/m(2)), high-dose cytarabine (2 x 3 g/m(2)) and thiotepa (40 mg/m(2)) with collection of stem cells in between. Conditioning for HCT-ASCT consisted of rituximab 375 mg/m(2), carmustine 400 mg/m(2) and thiotepa (4 x 5 mg/kg). Patients commenced HCT-ASCT irrespective of response after induction. Patients not achieving complete remission (CR) after HCT-ASCT received whole-brain radiotherapy. Primary end point was CR after HCT-ASCT. We enrolled 39 patients;median age and Karnofsky performance score are 57 years and 90%, respectively. About 28 patients had relapsed and 8 refractory disease. About 22 patients responded to induction and 32 patients commenced HCT-ASCT. About 22 patients (56.4%) achieved CR after HCT-ASCT. Respective 2-year progression-free survival (PFS) and overall survival (OS) rates were 46.0% (median PFS 12.4 months) and 56.4%;median OS not reached. We recorded four treatment-related deaths. Thiotepa-based HCT-ASCT is an effective treatment option in eligible patients with r/r PCNSL. Comparative studies are needed to further scrutinise the role of HCT-ASCT in the salvage setting.