Disease and outcome disparities in multiple myeloma: exploring the role of race/ethnicity in the Cooperative Group clinical trials

Disease and outcome disparities in multiple myeloma: exploring the role of race/ethnicity in the Cooperative Group clinical trials
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DOI:
10.1038/s41408-018-0102-7
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发表时间:
2018-07-06
影响因子:
12.8
通讯作者:
Rajkumar, S. Vincent
Rajkumar, S. Vincent
中科院分区:
医学1区
文献类型:
--
作者:
Ailawadhi, Sikander;Jacobus, Susanna;Rajkumar, S. Vincent

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多发性骨髓瘤(MM)是一种无法治愈的血液系统恶性肿瘤,其预后在不同种族之间存在差异,可能是由于获得有效治疗方式的差异所致。临床试验可以提供获得循证医学的途径,但在治疗性临床试验中,少数族裔的代表性一直令人沮丧。我们评估了来自9个大型国家合作小组临床试验的患者种族对新诊断MM的影响。在20多年来登记的2896名患者中,只有18%是非白人,少数族裔的登记人数在最近几年(2002-2011年)实际上有所下降。非洲裔美国人更年轻,有更频繁的不良风险标志,包括贫血和乳酸脱氢酶升高。在使用新型治疗药物的试验中,拉美裔患者的比例最小。虽然不利的人口统计学因素(年龄增加)和临床因素(表现状态、分期、贫血、肾功能不全)与较差的存活率有关,但患者种族对客观应答率、无进展或总体存活率没有影响。尽管不同种族-民族的多发性骨髓瘤患者的发病率和预后存在显著差异,但这种差异似乎可以通过获得适当的治疗方案来缓解,例如,临床试验提供的治疗方案。改善治疗临床试验中的少数人收益需要成为优先事项。
Multiple myeloma (MM) is an incurable hematologic malignancy with disparities in outcomes noted among racialethnic subgroups, likely due to disparities in access to effective treatment modalities. Clinical trials can provide access to evidence-based medicine but representation of minorities on therapeutic clinical trials has been dismal. We evaluated the impact of patient race-ethnicity in pooled data from nine large national cooperative group clinical trials in newly diagnosed MM. Among 2896 patients enrolled over more than two decades, only 18% were non-White and enrollment of minorities actually decreased in most recent years (2002-2011). African-Americans were younger and had more frequent poor-risk markers, including anemia and increased lactate dehydrogenase. Hispanics had the smallest proportion of patients on trials utilizing novel therapeutic agents. While adverse demographic (increased age) and clinical (performance status, stage, anemia, kidney dysfunction) factors were associated with inferior survival, patient race-ethnicity did not have an effect on objective response rates, progression-free, or overall survival. While there are significant disparities in MM incidence and outcomes among patients of different racial-ethnic groups, this disparity seems to be mitigated by access to appropriate therapeutic options, for example, as offered by clinical trials. Improved minority accrual in therapeutic clinical trials needs to be a priority.