National and Kapodistrian University of Athens School of Medicine, Athens, Greece.
Dana-Farber Cancer Institute, Boston, MA, USA.
Lancet Haematol. 2022 Jun;9(6):e403-e414. doi: 10.1016/S2352-3026(22)00103-X. Epub 2022 May 9.
BACKGROUND: Elotuzumab plus lenalidomide and dexamethasone has shown improved progression-free and overall survival versus lenalidomide and dexamethasone in patients with relapsed or refractory multiple myeloma. We aimed to assess these regimens in patients with newly diagnosed multiple myeloma who are ineligible for haematopoietic stem-cell transplantation (HSCT). METHODS: ELOQUENT-1 is an open-label, multicentre, randomised, phase 3 trial conducted at 185 hospitals, oncology practices, and research centres in 19 countries. Eligible patients were aged 18 years or older with newly diagnosed, untreated, symptomatic myeloma and not candidates for high-dose therapy plus HSCT, and an Eastern Cooperative Oncology Group (ECOG) performance status of 2 or lower. Patients were randomly assigned (1:1) to receive elotuzumab plus lenalidomide and dexamethasone or lenalidomide and dexamethasone using an interactive voice response system, stratified by the International Staging System (ISS; stage I-II vs III), age (<75 years vs ≥75 years), and ECOG performance status (0 vs 1-2). Patients in the elotuzumab plus lenalidomide and dexamethasone group received elotuzumab administered intravenously at 10 mg/kg on days 1, 8, 15, and 22 during cycles 1 and 2, days 1 and 15 during cycles 3-18, and then at 20 mg/kg on day 1 for subsequent cycles. In both treatment groups, patients received 25 mg lenalidomide orally on days 1-21 of each cycle and 40 mg dexamethasone on days 1, 8, 15, and 22 of each cycle. The primary endpoint was progression-free survival, as per the primary definition from European Society for Blood and Marrow Transplantation criteria in all randomly assigned patients (intention-to-treat population). This study is registered with ClinicalTrials.gov, NCT01335399 (completed). FINDINGS: Between Aug 4, 2011, and June 19, 2014, 748 patients were randomly assigned (374 in each treatment group) and 742 patients received treatment (333 (90%) of 371 in the elotuzumab plus lenalidomide and dexamethasone group vs 339 (91%) of 371 in the lenalidomide and dexamethasone group discontinued treatment). The median age of patients was 73·0 years (IQR 69·0-78·0), 294 (39%) patients were 75 years or older. Most patients were White (711 [95%]) and male (412 [55%]). At a minimum follow-up of 65·3 months, the median progression-free survival was not significantly different between the groups: 31·4 months (95% CI 26·2-36·8) in the elotuzumab plus lenalidomide and dexamethasone group versus 29·5 months (23·5-34·3) in the lenalidomide and dexamethasone group (HR 0·93, 95·71% CI 0·77-1·12; stratified log-rank p=0·44). The median follow-up was 70·6 months (IQR 35·1-79·2). The most common grade 3-4 treatment-related adverse event was neutropenia (64 [17%] of 371 vs 79 [21%] of 371). Study drug toxicity was the reported cause of death in five (1%) versus four (1%) patients. INTERPRETATION: Elotuzumab plus lenalidomide and dexamethasone did not significantly improve progression-free survival versus lenalidomide and dexamethasone in patients with newly diagnosed multiple myeloma who are ineligible for HSCT. Although these data contribute to the treatment landscape, further research is needed to find ways to improve treatments in the front-line setting. FUNDING: Bristol Myers Squibb.
背景:在复发或难治性多发性骨髓瘤患者中,与来那度胺和地塞米松相比,依鲁替尼联合来那度胺和地塞米松可改善无进展生存期和总生存期。我们旨在评估这些方案在不适合造血干细胞移植(HSCT)的新诊断多发性骨髓瘤患者中的疗效。
方法:ELOQUENT-1 是一项在 19 个国家的 185 家医院、肿瘤诊所和研究中心进行的开放标签、多中心、随机、3 期临床试验。入组患者年龄为 18 岁或以上,患有新诊断、未经治疗、有症状的骨髓瘤,不适合高剂量治疗加 HSCT,东部肿瘤协作组(ECOG)体能状态为 2 或更低。患者通过交互式语音应答系统以 1:1 的比例随机分配(1:1)接受依鲁替尼联合来那度胺和地塞米松或来那度胺和地塞米松治疗,按国际分期系统(ISS;I-II 期 vs III 期)、年龄(<75 岁 vs ≥75 岁)和 ECOG 体能状态(0 与 1-2)分层。依鲁替尼联合来那度胺和地塞米松组的患者在第 1 周期和第 2 周期的第 1、8、15 和 22 天,第 3-18 周期的第 1 和第 15 天,以及随后的周期在第 1 天接受 10 mg/kg 的依鲁替尼静脉注射,随后的周期在第 1 天接受 20 mg/kg 的依鲁替尼静脉注射。在两个治疗组中,患者在每个周期的第 1-21 天接受 25 mg 来那度胺口服,在每个周期的第 1、8、15 和 22 天接受 40 mg 地塞米松。主要终点是无进展生存期,根据欧洲血液和骨髓移植协会标准的主要定义,适用于所有随机分配的患者(意向治疗人群)。该研究在 ClinicalTrials.gov 上注册,NCT01335399(已完成)。
结果:在 2011 年 8 月 4 日至 2014 年 6 月 19 日期间,共 748 例患者随机分配(每组 374 例),742 例患者接受了治疗(371 例患者中 333 例[90%]接受了依鲁替尼联合来那度胺和地塞米松治疗,371 例患者中 339 例[91%]接受了来那度胺和地塞米松治疗)。患者的中位年龄为 73.0 岁(IQR 69.0-78.0),294 例(39%)患者年龄为 75 岁或以上。大多数患者为白人(711 [95%])和男性(412 [55%])。在最低随访 65.3 个月时,两组之间的中位无进展生存期无显著差异:依鲁替尼联合来那度胺和地塞米松组为 31.4 个月(95%CI 26.2-36.8),来那度胺和地塞米松组为 29.5 个月(23.5-34.3)(HR 0.93,95%CI 0.77-1.12;分层对数秩检验 p=0.44)。中位随访时间为 70.6 个月(IQR 35.1-79.2)。最常见的 3-4 级治疗相关不良事件是中性粒细胞减少症(64 [17%]例患者接受了 371 例治疗,79 [21%]例患者接受了 371 例治疗)。研究药物毒性是 5 例(1%)患者和 4 例(1%)患者死亡的报告原因。
解释:依鲁替尼联合来那度胺和地塞米松并未显著改善不适合 HSCT 的新诊断多发性骨髓瘤患者的无进展生存期。尽管这些数据为治疗现状提供了参考,但仍需要进一步研究以寻找改善一线治疗的方法。
资金来源:百时美施贵宝。
Mol Biomed. 2024-7-4