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新诊断多发性骨髓瘤患者接受来那度胺单药维持治疗、任何维持治疗或无维持治疗的治疗结局和医疗资源利用:来自 Connect MM 登记处的结果。

Treatment Outcomes and Health Care Resource Utilization in Patients With Newly Diagnosed Multiple Myeloma Receiving Lenalidomide-only Maintenance, Any Maintenance, or No Maintenance: Results from the Connect MM Registry.

机构信息

US Oncology Research, Rocky Mountain Cancer Centers, Denver, Colorado.

Mount Sinai Hospital, New York, New York.

出版信息

Clin Ther. 2018 Jul;40(7):1193-1202.e1. doi: 10.1016/j.clinthera.2018.05.017. Epub 2018 Jul 23.

DOI:10.1016/j.clinthera.2018.05.017
PMID:30007443
Abstract

PURPOSE

Maintenance therapy after autologous stem cell transplantation (ASCT) improves clinical outcomes in multiple myeloma (MM), but the effect of continued treatment with lenalidomide-only maintenance, or any maintenance, on health care resource utilization (HCRU) is largely unknown.

METHODS

Here we present an analysis of HCRU and clinical outcomes in a cohort of patients from the Connect MM registry, the largest, ongoing, observational, prospective US registry of patients with symptomatic newly diagnosed MM. In this study, patients with newly diagnosed MM who completed induction and single ASCT without subsequent consolidation received lenalidomide-only maintenance (n = 180), any maintenance (n = 256), or no maintenance (n = 165). HCRU (hospitalization, surgery/procedures, and concurrent medications [growth factors, bisphosphonates, or neuropathic pain medication]) was assessed starting from 100 days post-ASCT for up to 2 years.

FINDINGS

Although the rates of hospitalization per 100 person-years were similar across groups at the end of years 1 and 2, the median duration of hospitalization was numerically longer with no maintenance. The rates of use of growth factors, bisphosphonates, and neuropathic pain medication were generally similar in all 3 groups. The receipt of any maintenance was associated with significantly reduced use of neuropathic pain medications during year 1. Of note, lenalidomide-only maintenance was associated with significantly longer progression-free survival (54.5 vs 30.4 months; hazard ratio [HR] = 0.58; 95% CI, 0.43-0.79; P = 0.0005) and overall survival (OS) (median OS not reached in either group; HR = 0.45; 95% CI, 0.28-0.73; P = 0.001) compared with no maintenance. Likewise, the group treated with any maintenance had significantly longer median progression-free survival (44.7 vs 30.4 months; HR = 0.62; 95% CI, 0.47-0.82; P = 0.0008) and OS (median OS not reached in either group; HR = 0.50; 95% CI, 0.33-0.76; P = 0.001) than did the group that did not receive maintenance.

IMPLICATIONS

These findings suggest that in this largely community-based study population, post-ASCT maintenance therapy, including lenalidomide-only maintenance, improves clinical outcomes without negatively affecting HCRU. ClinicalTrials.gov identifier: NCT01081028.

摘要

目的

自体干细胞移植(ASCT)后的维持治疗可改善多发性骨髓瘤(MM)的临床结局,但继续接受来那度胺单药维持治疗或任何维持治疗对医疗资源利用(HCRU)的影响在很大程度上尚不清楚。

方法

在此,我们展示了 Connect MM 登记处中患者队列的 HCRU 和临床结局分析,这是美国最大、正在进行的、观察性的、前瞻性的新诊断为 MM 的症状性患者登记处。在这项研究中,接受过诱导和单 ASCT 治疗且无后续巩固治疗的新诊断 MM 患者接受来那度胺单药维持治疗(n=180)、任何维持治疗(n=256)或无维持治疗(n=165)。从 ASCT 后 100 天开始,评估 HCRU(住院、手术/操作和同时使用的药物[生长因子、双膦酸盐或神经病理性疼痛药物]),最长可达 2 年。

结果

尽管在第 1 年和第 2 年结束时,各组的 100 人年住院率相似,但无维持治疗组的中位住院时间在数值上更长。在所有 3 组中,生长因子、双膦酸盐和神经病理性疼痛药物的使用率通常相似。接受任何维持治疗与第 1 年神经病理性疼痛药物使用率显著降低相关。值得注意的是,与无维持治疗相比,来那度胺单药维持治疗与显著延长的无进展生存期(54.5 个月 vs 30.4 个月;风险比[HR] = 0.58;95%CI,0.43-0.79;P=0.0005)和总生存期(OS)(两组均未达到中位 OS;HR=0.45;95%CI,0.28-0.73;P=0.001)相关。同样,接受任何维持治疗的组与无维持治疗的组相比,中位无进展生存期(44.7 个月 vs 30.4 个月;HR=0.62;95%CI,0.47-0.82;P=0.0008)和 OS(两组均未达到中位 OS;HR=0.50;95%CI,0.33-0.76;P=0.001)显著延长。

结论

这些发现表明,在这项主要基于社区的研究人群中,ASCT 后维持治疗,包括来那度胺单药维持治疗,可改善临床结局,而不会对 HCRU 产生负面影响。临床试验注册号:NCT01081028。

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