Department of Hematology, Huashan Hospital, Shanghai, China.
Institutes of Biomedical Sciences, Fudan University, Shanghai, China.
J Neurooncol. 2023 May;163(1):39-46. doi: 10.1007/s11060-022-04062-z. Epub 2022 Jun 22.
High-dose methotrexate (HD-MTX)-based chemotherapy regimen is the first-line option for primary central nervous system lymphoma (PCNSL). This prospective cohort study aimed to evaluate the efficacy and adverse effects of HD-MTX plus idarubicin (IDA) in patients with newly diagnosed immunocompetent PCNSL.
We recruited newly diagnosed PCNSL patients from January 2017 to August 2020. Patients were assigned into two groups: HD-MTX monotherapy and HD-MTX plus IDA (HD-MTX/IDA). In the HD-MTX monotherapy group, patients were treated with MTX 8 g/m alone on day 1, while the HD-MTX/IDA group received MTX 8 g/m on day 1 and IDA 10 mg/m on day 2. Treatments were repeated every 3 weeks for 8 cycles except for progression and/or unacceptable toxicity.
We recruited 61 PCNSL patients, including 36 in the HD-MTX and 25 in the HD-MTX/IDA group. The CR rate was 68% in the HD-MTX/IDA group and 72.22% of patients in the HD-MTX monotherapy group (p = 0.7221), while the overall response rate was 72% vs. 77.78% (p = 0.6063). Median PFS in HD-MTX/IDA group and HD-MTX monotherapy group were 15.6 months and 18.5 months, respectively (p = 0.6374). Median OS was not reached in both groups. There were no significant differences in adverse effects between the two groups.
The combination of IDA with HD-MTX showed no obvious therapeutic advantage over HD-MTX monotherapy in newly diagnosed patients with PCNSL. HD-MTX dose of 8 g/m monotherapy can still provide better therapeutic benefits in patients with acceptable adverse effects. Future studies could explore HD-MTX in combination with other chemotherapeutic agents in the first-line treatment of PCNSL.
大剂量甲氨蝶呤（HD-MTX）为基础的化疗方案是原发性中枢神经系统淋巴瘤（PCNSL）的一线选择。本前瞻性队列研究旨在评估新诊断的免疫功能正常的 PCNSL 患者中 HD-MTX 联合伊达比星（IDA）的疗效和不良反应。
我们于 2017 年 1 月至 2020 年 8 月期间招募了新诊断的 PCNSL 患者。患者被分为两组：HD-MTX 单药治疗组和 HD-MTX 联合 IDA（HD-MTX/IDA）组。在 HD-MTX 单药治疗组中，患者在第 1 天单独接受 MTX 8 g/m，而在 HD-MTX/IDA 组中，患者在第 1 天接受 MTX 8 g/m，第 2 天接受 IDA 10 mg/m。除进展和/或不可接受的毒性外，每 3 周重复治疗 8 个周期。
我们共招募了 61 例 PCNSL 患者，其中 36 例患者接受 HD-MTX 治疗，25 例患者接受 HD-MTX/IDA 治疗。HD-MTX/IDA 组的完全缓解率为 68%，HD-MTX 单药治疗组的完全缓解率为 72.22%（p=0.7221），总缓解率为 72%与 77.78%（p=0.6063）。HD-MTX/IDA 组和 HD-MTX 单药治疗组的中位 PFS 分别为 15.6 个月和 18.5 个月（p=0.6374）。两组中位 OS 均未达到。两组不良反应无明显差异。
IDA 联合 HD-MTX 在新诊断的 PCNSL 患者中并未显示出明显优于 HD-MTX 单药治疗的疗效。HD-MTX 剂量为 8 g/m 的单药治疗仍可为患者提供更好的治疗效果，同时不良反应可接受。未来的研究可以探索 HD-MTX 联合其他化疗药物在 PCNSL 的一线治疗中的应用。
