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Clinical outcomes associated with conditioning regimens in patients with primary central nervous system lymphoma undergoing consolidation autologous stem cell transplantation

Yu-Na Lee, Hyungwoo Cho, J. Hyung, In Hye Song, H. Go, Ji Eun Park, Ho Sung Kim, Minyoung Oh et al.

Blood, 2025

71 participants · 1 citation

Abstract

Background: Primary central nervous system lymphoma (PCNSL) is a rare, aggressive extra-nodal non-Hodgkin lymphoma confined to the central nervous system or eyes. While high-dose methotrexate (HD-MTX)-based induction therapy achieves high response rates, frequent relapses require consolidation treatment. Recent pivotal randomized controlled trials have established high-dose chemotherapy with autologous stem cell transplantation (HDC-ASCT) as the preferred consolidation strategy, demonstrating improved survival and reduced neurotoxicity compared to whole brain radiotherapy. Among conditioning regimens, thiotepa-based approaches have shown superior efficacy, but optimal regimen selection among those thiotepa-containing ones remains unclear. This study compared clinical outcomes of consolidation ASCT according to conditioning regimens: thiotepa/busulfan/cyclophosphamide (TBC), busulfan/thiotepa (BuTT), and non-thiotepa regimens.

Methods: This retrospective study included 160 newly diagnosed PCNSL patients treated at Asan Medical Center from 2004 to 2023 who responded to HD-MTX-based induction chemotherapy and proceeded to consolidation ASCT. Induction regimens included HD-MTX, HD-MTX/procarbazine/vincristine (MPV), or rituximab-MPV (R-MPV). Patients were categorized by conditioning regimen: non-thiotepa (busulfan/cytarabine/etoposide[BuCyE], busulfan/melphalan/etoposide[BuMelE], or BCNU/etoposide/cytarabine/melphalan[BEAM]; n=28), TBC (n=71), and BuTT (n=61). We evaluated…

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Evidence Hub reads this as a randomized trial, done in people. Its evidence level is 2 of 5 (High · randomized trial). Randomized trials of a reasonable size are the strongest single-study design. Reported size: 71 participants or records.

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