PFS24 as a prognostic milestone in patients with newly diagnosed primary CNS lymphoma

Abstract High-dose chemotherapy followed by autologous hematopoietic stem cell transplantation has significantly improved overall survival (OS) in primary central nervous system lymphoma (PCNSL). However, early identification of long-term survivors remains a challenge. Progression-free survival at 2...

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Main Authors: Vanja Zeremski, Tobias R. Haage, Hanno M. Witte, Louisa Adolph, Sina A. Beer, Gerhard Behre, Benedikt Jacobs, Christoph Kahl, Chrysavgi Lalayanni, Jens Panse, Sotirios Papageorgiou, Marina P. Siakantaris, Jessica Schneider, Ulf Schnetzke, Alexander Schulz, Theodoros P. Vassilakopoulos, Jeanette Walter, Dimitrios Mougiakakos
Format: Article
Language:English
Published: BMC 2025-04-01
Series:Journal of Hematology & Oncology
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Online Access:https://doi.org/10.1186/s13045-025-01700-7
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Summary:Abstract High-dose chemotherapy followed by autologous hematopoietic stem cell transplantation has significantly improved overall survival (OS) in primary central nervous system lymphoma (PCNSL). However, early identification of long-term survivors remains a challenge. Progression-free survival at 24 months (PFS24) has emerged as a key prognostic marker in diffuse large B-cell lymphoma, but its relevance in PCNSL is still unclear. In this retrospective multicenter study, we analyzed data from 146 newly diagnosed, transplant-eligible PCNSL patients treated with MATRix-like regimens across 14 hospitals. With a median follow-up of 48 months, the 2-year PFS and OS rates were 50.4% and 65.6%, respectively. Of the 139 patients evaluable for PFS24-analysis, 51.1% reached PFS24, with a subsequent 5-year OS of 96.7%. Of note, the annual hazard rate for progression and death decreased to under 5% after 24 months, remaining stable thereafter. The patients who failed to reach PFS24 had a median OS of only 6.0 months. Key predictors of PFS failure included impaired Karnofsky performance status and treatment dose-reduction. In conclusion, PFS24 was identified as an important prognostic marker in PCNSL. Patients who achieve PFS24 have a favorable prognosis, whereas those who do not face poor outcomes and require innovative treatment approaches. This insight could aid in risk stratification and support the use of PFS24 as a surrogate endpoint in clinical trials.
ISSN:1756-8722