Hwang Steven R, Godby Richard C, Negaard Brianna J, Mwangi Raphael, Nedved Adrienne N, Barreto Jason N, Micallef Ivana N, Ansell Stephen M, Porrata Luis, Durani Urshila, Thanarajasingam Gita, Habermann Thomas M, Maurer Matthew J, Johnston Patrick B, Khurana Arushi
Division of Hematology, Mayo Clinic, Rochester, MN.
Department of Health Sciences Research, Mayo Clinic, Rochester, MN.
Blood Adv. 2025 Feb 25;9(4):924-932. doi: 10.1182/bloodadvances.2024014073.
Primary central nervous system lymphoma (PCNSL) is a rare form of non-Hodgkin lymphoma involving the brain, cerebrospinal fluid, or retina/vitreous without systemic involvement. Induction with high-dose methotrexate (HD-MTX) followed by consolidation with autologous stem cell transplant (auto-SCT) has become the standard treatment paradigm for most patients. However, limited data are available regarding the efficacy of a maintenance approach with HD-MTX. Herein, we retrospectively reviewed the characteristics and outcomes of 148 patients diagnosed with PCNSL between October 2010 and June 2022, who underwent HD-MTX-based induction therapy followed by either auto-SCT consolidation (n = 70) or HD-MTX maintenance therapy (n = 37). At a median follow-up time of 4.5 years, the progression-free survival (PFS) was 8.3 years and the overall survival (OS) was not reached. Compared to patients who underwent auto-SCT, patients who received maintenance HD-MTX had a higher median age at diagnosis of 72 vs 62 years and a trend toward higher proportion of patients being Eastern Cooperative Oncology Group 2 or higher (41% vs 29%). At 5-years postinduction treatment initiation, the PFS rates in the auto-SCT cohort and HD-MTX maintenance cohort were 74.6% and 72.6%, respectively, and the OS rates were 76.0% and 82.4%, respectively. Overall, there was no significant difference in PFS or OS based on postinduction management strategy. Our data suggest that maintenance HD-MTX may be a reasonable, time-limited treatment strategy for patients with PCNSL responding to initial induction therapy.
原发性中枢神经系统淋巴瘤（PCNSL）是一种罕见的非霍奇金淋巴瘤，累及脑、脑脊液或视网膜/玻璃体，无全身受累。大剂量甲氨蝶呤（HD-MTX）诱导治疗后行自体干细胞移植（auto-SCT）巩固治疗已成为大多数患者的标准治疗模式。然而，关于HD-MTX维持治疗方法的疗效，可用数据有限。在此，我们回顾性分析了2010年10月至2022年6月期间确诊为PCNSL的148例患者的特征和预后，这些患者接受了基于HD-MTX的诱导治疗，随后接受了auto-SCT巩固治疗（n = 70）或HD-MTX维持治疗（n = 37）。中位随访时间为4.5年，无进展生存期（PFS）为8.3年，总生存期（OS）未达到。与接受auto-SCT的患者相比，接受HD-MTX维持治疗的患者诊断时的中位年龄更高，分别为72岁和62岁，东部肿瘤协作组2级或更高分级的患者比例有升高趋势（41%对29%）。诱导治疗开始后5年，auto-SCT队列和HD-MTX维持队列的PFS率分别为74.6%和72.6%，OS率分别为76.0%和82.4%。总体而言，基于诱导后管理策略的PFS或OS无显著差异。我们的数据表明，HD-MTX维持治疗可能是对初始诱导治疗有反应的PCNSL患者的一种合理的、有时间限制的治疗策略。
