From the Unit of Lymphoid Malignancies (A.J.M.F.), UTMO & Hematology Unit, Department of Onco-Hematology (F.C.), and Medical Oncology Unit, Department of Oncology (M.R.), San Raffaele Scientific Institute, Milan; Department of Medical Oncology (A.A.B.), Bellaria-Maggiore Hospital, Azienda Unità Sanitaria Locale, Bologna; Division of Hematology (M.M.), Ospedale di Ancona; Department of Medical Oncology and Hematology (M.B.), Istituto Clinico Humanitas, Rozzano; Division of Medical Oncology A (M.S.), National Cancer Institute, Aviano; Division of Hematology (F.I.), Ospedale di Reggio Emilia; Division of Hematology (F.Z.), Ospedale di Udine; Division of Hematology (C.S.), Ospedale di Reggio Calabria; Division of Hematology (F.B.), Ospedale Maggiore di Novara; Division of Hematology (G.C.), Istituto Nazionale dei Tumori Pascale di Napoli; and the Division of Hematology (L.B.), Ospedale Maggiore di Milano, Italy.
Neurology. 2014 Apr 15;82(15):1370-3. doi: 10.1212/WNL.0000000000000314. Epub 2014 Mar 14.
We report updated results at a median follow-up of 12 years of a phase II trial assessing first-line MATILDE chemotherapy and response-tailored radiotherapy in patients with primary CNS lymphomas (PCNSL).
Forty-one HIV-negative patients (18-70 years; Eastern Cooperative Oncology Group performance status ≤3) with histologically confirmed PCNSL received 3 courses of MATILDE chemotherapy followed by whole-brain radiotherapy (WBRT). Chemotherapy activity was the primary endpoint.
Overall response rate was 76% (95% confidence interval [CI] 63%-89%) after chemotherapy and 83% (95% CI 71%-95%) after chemotherapy ± radiotherapy. At a median follow-up of 144 months (range 47-153), 31 patients experienced an event: relapse in 24, progressive disease in 3, and toxic death in 4, with a 5-year progression-free survival of 24% ± 8%. Two patients experienced a late relapse (100 and 101 months). Nine patients are alive and disease-free, 8 of whom are alive at >10 years, with a 5-year overall survival of 30% ± 7%. At 10 years from diagnosis, no patient showed chronic hematologic and nonhematologic toxicities, with a Mini-Mental State Examination score of ≥29 in all cases but one.
At a median follow-up of 12 years, MATILDE regimen followed by WBRT confirmed the previously reported survival plateau, which further proves its long-lasting efficacy with acceptable neurologic deficits.
This study provides Class IV evidence that in patients with PCNSL, MATILDE chemotherapy followed by response-tailored radiotherapy increases the probability of disease remission at 12 years.
我们报告了一项 II 期临床试验的更新结果，该试验评估了一线 MATILDE 化疗和针对反应的放疗在原发性中枢神经系统淋巴瘤（PCNSL）患者中的应用，中位随访时间为 12 年。
41 例 HIV 阴性患者（18-70 岁；东部肿瘤协作组体能状态≤3）经组织学证实患有 PCNSL，接受 3 个疗程的 MATILDE 化疗，随后进行全脑放疗（WBRT）。化疗活性是主要终点。
化疗后总缓解率为 76%（95%置信区间[CI] 63%-89%），化疗后加放疗为 83%（95% CI 71%-95%）。在中位随访 144 个月（范围 47-153）时，31 例患者发生事件：24 例复发，3 例进展性疾病，4 例因毒性死亡，5 年无进展生存率为 24%±8%。2 例患者发生迟发性复发（100 和 101 个月）。9 例患者存活且无疾病，其中 8 例存活时间超过 10 年，5 年总生存率为 30%±7%。从诊断开始 10 年后，没有患者出现慢性血液和非血液毒性，除 1 例外所有患者的简易精神状态检查评分均≥29。
在中位随访 12 年时，MATILDE 方案联合 WBRT 证实了先前报道的生存平台，进一步证明了其具有可接受的神经功能缺损的长期疗效。
这项研究提供了 IV 级证据，表明在 PCNSL 患者中，MATILDE 化疗后加针对反应的放疗可提高 12 年时疾病缓解的概率。
