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本文引用的文献

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Daratumumab monotherapy in patients with treatment-refractory multiple myeloma (SIRIUS): an open-label, randomised, phase 2 trial.达雷妥尤单抗单药治疗治疗难治性多发性骨髓瘤患者(SIRIUS):一项开放标签、随机、2 期试验。
Lancet. 2016 Apr 9;387(10027):1551-1560. doi: 10.1016/S0140-6736(15)01120-4. Epub 2016 Jan 7.
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Multiple Myeloma: Diagnosis and Treatment.多发性骨髓瘤:诊断与治疗
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Cytogenetics and long-term survival of patients with refractory or relapsed and refractory multiple myeloma treated with pomalidomide and low-dose dexamethasone.泊马度胺联合小剂量地塞米松治疗难治性或复发难治性多发性骨髓瘤患者的细胞遗传学与长期生存情况
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4
Pomalidomide alone or in combination with low-dose dexamethasone in relapsed and refractory multiple myeloma: a randomized phase 2 study.来那度胺单药或联合低剂量地塞米松治疗复发/难治性多发性骨髓瘤的随机 2 期研究。
Blood. 2014 Mar 20;123(12):1826-32. doi: 10.1182/blood-2013-11-538835. Epub 2014 Jan 13.
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Pomalidomide plus low-dose dexamethasone versus high-dose dexamethasone alone for patients with relapsed and refractory multiple myeloma (MM-003): a randomised, open-label, phase 3 trial.泊马度胺联合低剂量地塞米松与高剂量地塞米松单独用于治疗复发和难治性多发性骨髓瘤(MM-003)患者:一项随机、开放标签、3 期试验。
Lancet Oncol. 2013 Oct;14(11):1055-1066. doi: 10.1016/S1470-2045(13)70380-2. Epub 2013 Sep 3.
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Management of double-refractory multiple myeloma.双难治性多发性骨髓瘤的治疗管理。
Curr Hematol Malig Rep. 2013 Dec;8(4):253-60. doi: 10.1007/s11899-013-0173-2.
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New developments in the management and treatment of newly diagnosed and relapsed/refractory multiple myeloma patients.新诊断和复发/难治性多发性骨髓瘤患者管理和治疗的新进展。
Expert Opin Pharmacother. 2013 Aug;14(12):1569-73. doi: 10.1517/14656566.2013.805746. Epub 2013 May 31.
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Novel approaches to treatment of double-refractory multiple myeloma.双难治性多发性骨髓瘤的新型治疗方法。
Am Soc Clin Oncol Educ Book. 2013;2013:302-6. doi: 10.1200/EdBook_AM.2013.33.e302.
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Pomalidomide: first global approval.泊马度胺:全球首次获批。
Drugs. 2013 May;73(6):595-604. doi: 10.1007/s40265-013-0047-x.
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Carfilzomib: in relapsed, or relapsed and refractory, multiple myeloma.卡非佐米:用于治疗复发或复发且难治的多发性骨髓瘤。
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对接受过≥3线既往治疗(包括蛋白酶体抑制剂(PI)和免疫调节药物(IMiD),或对PI和IMiD双重难治)的多发性骨髓瘤患者总生存的真实世界数据进行分析。

Analysis of Real-World Data on Overall Survival in Multiple Myeloma Patients With ≥3 Prior Lines of Therapy Including a Proteasome Inhibitor (PI) and an Immunomodulatory Drug (IMiD), or Double Refractory to a PI and an IMiD.

作者信息

Usmani Saad, Ahmadi Tahamtan, Ng Yvette, Lam Annette, Desai Avinash, Potluri Ravi, Mehra Maneesha

机构信息

Levine Cancer Institute/Carolinas Health Care System, Charlotte, North Carolina, USA.

Janssen Research and Development, LLC, Spring House, Pennsylvania, USA.

出版信息

Oncologist. 2016 Nov;21(11):1355-1361. doi: 10.1634/theoncologist.2016-0104. Epub 2016 Aug 2.

DOI:10.1634/theoncologist.2016-0104
PMID:27486203
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC5189616/
Abstract

BACKGROUND

This retrospective study evaluated the treatment patterns in and overall survival (OS) of multiple myeloma (MM) patients who were refractory to a proteasome inhibitor (PI) and an immunomodulatory drug (IMiD) or who had received three or more prior lines of therapy (LOTs) including a PI and an IMiD.

METHODS

Electronic health records in the IMS LifeLink and OPTUM databases were screened for indexing periods of 2000-2014 and 2007-2014, respectively. Patients who were refractory to both a PI and an IMiD (criterion 1) or who received three or more prior LOTs (including a PI and an IMiD) and showed disease progression within 60 days of their most recent regimen (criterion 2) comprised the eligible population. Median OS from time of last LOT was assessed for the full cohort, cohorts meeting criteria 1 and 2, and clinically important subgroups.

RESULTS

Of 3,929 and 3,837 patients with MM diagnoses evaluated in the IMS LifeLink and OPTUM databases, 500 and 162 met the eligibility criteria, respectively. Similar median OS was observed for eligible patients in the IMS LifeLink and OPTUM databases (7.9 vs. 7.9 months; = .5358). In subgroup analyses of the IMS LifeLink data set, median OS was longer in patients <65 years of age than it was for those ≥65 years at eligibility (9.5 vs 6.7 months; < .01) and in patients with good or unreported versus poor performance status at last claim (7.8 or 8.8 vs. 2.9 months; < .0001).

CONCLUSION

The findings of this survival analysis suggest that outcomes for these patients remain poor despite the availability of newer agents.

IMPLICATIONS FOR PRACTICE

This real-world retrospective study of electronic health records examines the survival outcomes of patients with multiple myeloma who are heavily pretreated or highly refractory to currently approved treatments, including recently approved proteasome inhibitors and immunomodulatory drugs. This survival analysis showed that outcomes for these patients remain poor despite the availability of newer agents, with median overall survival of approximately 8 months. These findings highlight a critical need to develop novel therapies for these patients and also serve as a reference point against which emerging agents for heavily pretreated or highly refractory disease may be evaluated.

摘要

背景

本回顾性研究评估了对蛋白酶体抑制剂(PI)和免疫调节药物(IMiD)难治或接受过包括PI和IMiD在内的三线或更多线先前治疗(LOT)的多发性骨髓瘤(MM)患者的治疗模式和总生存期(OS)。

方法

分别在IMS LifeLink和OPTUM数据库中筛选2000 - 2014年和2007 - 2014年索引期的电子健康记录。对PI和IMiD均难治的患者(标准1)或接受过三线或更多线先前LOT(包括PI和IMiD)且在其最近治疗方案的60天内出现疾病进展的患者(标准2)构成符合条件的人群。评估了整个队列、符合标准1和2的队列以及具有临床重要意义的亚组从最后一次LOT时间起的中位OS。

结果

在IMS LifeLink和OPTUM数据库中评估的3929例和3837例MM诊断患者中,分别有500例和162例符合纳入标准。在IMS LifeLink和OPTUM数据库中,符合条件的患者观察到相似的中位OS(7.9个月对7.9个月;P = 0.5358)。在IMS LifeLink数据集的亚组分析中,年龄<65岁的患者中位OS长于符合纳入标准时年龄≥65岁的患者(9.5个月对6.7个月;P < 0.01),且最后一次就诊时表现状态良好或未报告的患者中位OS长于表现状态差的患者(7.8或8.8个月对2.9个月;P < 0.0001)。

结论

该生存分析的结果表明,尽管有更新的药物可用,但这些患者的预后仍然很差。

对实践的启示

这项对电子健康记录的真实世界回顾性研究检查了对目前批准的治疗(包括最近批准的蛋白酶体抑制剂和免疫调节药物)进行了大量预处理或高度难治的多发性骨髓瘤患者的生存结果。该生存分析表明,尽管有更新的药物可用,但这些患者的预后仍然很差,中位总生存期约为8个月。这些发现突出了为这些患者开发新疗法的迫切需求,也为评估针对大量预处理或高度难治性疾病的新兴药物提供了一个参考点。