Research progress in drug therapy for primary central nervous system lymphoma

doi:10.19803/j.1672-8629.20260016

Abstract

Abstract: Objective To summarize the current drug therapies for and research progress in treatment strategies for primary central nervous system lymphoma (PCNSL) so as to provide a reference for the safe and rational clinical use of drugs. Methods The mechanism of action, clinical efficacy, and safety of PCNSL therapeutic drugs were analyzed. By focusing on the challenges posed by the blood-brain barrier (BBB) and tumor heterogeneity, this study elaborated the current clinical applications based on the latest literature. Results Chemotherapy regimens that revolved around on high-dose methotrexate (HD-MTX) were the basic approaches to PCNSL. This drug could improve the prognosis of patients by crossing the blood-brain barrier. However, single-drug use had both limitations in efficacy and toxic side effects. Therefore, combined therapy (such as combination with other chemotherapeutic drugs, radiotherapy or targeted therapy) became the mainstream one, which could enhance the tumor-killing effect and prolong the progression-free survival (PFS) of patients. Targeted therapy (e.g., Bruton’s tyrosine kinase inhibitor, BTKi) and immunotherapy opened up new avenues for PCNSL treatment, which were particularly effective for relapsed or refractory PCNSL cases. They could not only increase clinical treatment options, but also mitigate the resistance to traditional chemotherapy. As a breakthrough precision immunotherapy technology, chimeric antigen receptor T-cell (CAR-T) therapy was capable of precise killing of tumor cells by modifying the patient’s autologous immune cells, and showed significant efficacy in some patients with refractory PCNSL. Conclusion Drug therapy for PCNSL is under rapid development. The multimodal treatment strategy combining traditional chemotherapy with emerging targeted therapy and immunotherapy provides a new line of thought for improving the prognosis of PCNSL patients.

Key words: Primary Central Nervous System Lymphoma (PCNSL), Methotrexate (MTX), Chemotherapy, Drug Therapy, Chimeric Antigen Receptor T-Cell (CAR-T) Therapy, Targeted Therapy, Immunotherapy

CLC Number:

Gao Lei, Zhang Min, Ren Xiaohui, Liu Qian. Research progress in drug therapy for primary central nervous system lymphoma[J]. Chinese Journal of Pharmacovigilance, 2026, 23(10): 1081-1085.

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https://zgywjj.magtechjournal.com/EN/Y2026/V23/I10/1081

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