Lin Huamao Mark, Davis Keith L, Kaye James A, Luptakova Katarina, Nagar Saurabh P, Mohty Mohamad
Millennium Pharmaceuticals, Inc., A Wholly Owned Subsidiary of Takeda Pharmaceutical Company Limited, 40 Landsdowne Street, Cambridge, MA 02139, USA.
RTI Health Solutions, 200 Park Offices Drive, Research Triangle Park, NC 27709, USA.
Adv Hematol. 2019 Jan 29;2019:4625787. doi: 10.1155/2019/4625787. eCollection 2019.
Limited data are available from real-world practices in Europe describing prevailing treatment patterns and outcomes in relapsed/refractory multiple myeloma (RRMM), particularly by cytogenetic risk.
A retrospective medical record review was conducted in 200 RRMM patients in France. From first relapse, patients were assessed on second-/third-line treatments, progression-free survival (PFS), overall survival (OS), and healthcare utilization.
Fifty-five high risk and 113 standard risk patients were identified. Overall, 192 patients (96%) received second-line therapy after relapse. Lenalidomide-based regimens were most common (>50%) in second line. Hospitalization incidence in high risk patients was approximately twice that of standard risk patients. From Kaplan-Meier estimation, median (95% CI) second-line PFS was 21.4 (17.5, 25.0) months (by high versus standard risk: 10.6 [6.4, 17.0] versus 28.7 [22.1, 37.3] months). Among second-line recipients, 47.4% were deceased at data collection. Median second-line OS was 59.4 (38.8, NE) months (by high versus standard risk: 36.5 [17.4, 50.6] versus 73.6 [66.5, NE] months).
The prognostic importance of cytogenetic risk in RRMM was apparent, whereby high (versus standard) risk patients had decidedly shorter PFS and OS. Frequent hospitalizations indicated potentially high costs associated with RRMM, particularly for high risk patients. These findings may inform economic evaluations of RRMM therapies.
欧洲实际临床实践中关于复发/难治性多发性骨髓瘤（RRMM）的主流治疗模式和结局的数据有限，尤其是按细胞遗传学风险分类的数据。
对法国200例RRMM患者进行回顾性病历审查。从首次复发起，评估患者的二线/三线治疗、无进展生存期（PFS）、总生存期（OS）和医疗资源利用情况。
确定了55例高危患者和113例标危患者。总体而言，192例患者（96%）在复发后接受了二线治疗。二线治疗中，基于来那度胺的方案最为常见（>50%）。高危患者的住院发生率约为标危患者的两倍。根据Kaplan-Meier估计，二线治疗的中位（95%CI）PFS为21.4（17.5，25.0）个月（高危组与标危组分别为：10.6[6.4，17.0]个月与28.7[22.1，37.3]个月）。在接受二线治疗的患者中，47.4%在数据收集时已死亡。二线治疗的中位OS为59.4（38.8，NE）个月（高危组与标危组分别为：36.5[17.4，50.6]个月与73.6[66.5，NE]个月）。
细胞遗传学风险在RRMM中的预后重要性明显，高危（与标危）患者的PFS和OS明显更短。频繁住院表明RRMM可能带来高昂成本，尤其是高危患者。这些发现可能为RRMM治疗的经济学评估提供参考。
