There are significant disparities in care and outcomes for patients with leukemias and multiple myeloma (MM). To evaluate the extent to which clinical trials (CTs) match the demographic and geographic diversity of populations affected by leukemias and MM.

CTs leading to drug approval were identified from the US Food and Drug Administration databases. Demographic and geographic data were collected from ClinicalTrials.gov and primary manuscripts. Standard descriptive statistics were used to summarize the data in frequencies and proportions, including 95% CIs, by race, ethnicity, sex, and malignancy subtypes.

A total of 41 (67.2%) trials leading to drug approval reported data on race and 20 (48.8%) on ethnicity. These trials included 13,731 patients, of whom 11,209 (81.6%) were White. Among minorities, Asian-Pacific Islanders and Blacks had the highest representation in chronic myeloid leukemia, 147 (12.7%) and 61 (5.3%), and lowest in chronic lymphocytic leukemia, 55 (3%) and 20 (1.1%), respectively. Proportions for Blacks, Native Americans, and Hispanics were significantly low, reflecting under-representation in trials compared with the proportion in the general population. Females were also under-represented in acute myeloid leukemia (44.7% v 60.5%, P < .0001), and males in MM (55.3% v 60.2%, P < .0001) and chronic myeloid leukemia (55.2% v 62.9%, P < .0001). The geographic distribution of trials showed inadequate regional and state participation compared with mortality for all malignancies except MM.

There are significant demographic and geographic under-representation and imbalances in pivotal CTs leading to drug approvals for leukemias and MM compared with the population affected. These disparities need to be addressed to make results applicable to all relevant populations.

白血病和多发性骨髓瘤 (MM) 患者的护理和结果存在显着差异。评估临床试验 (CT) 与受白血病和 MM 影响的人群的人口统计学和地理多样性相匹配的程度。

导致药物批准的 CT 是从美国食品和药物管理局数据库中识别出来的。人口统计和地理数据是从 ClinicalTrials.gov 和主要手稿中收集的。标准描述性统计用于按种族、种族、性别和恶性肿瘤亚型按频率和比例（包括 95% CI）总结数据。

导致药物批准的共有 41 项 (67.2%) 试验报告了种族数据，20 项 (48.8%) 报告了种族数据。这些试验包括 13,731 名患者，其中 11,209 名 (81.6%) 是白人。在少数民族中，亚太岛民和黑人在慢性粒细胞白血病中的比例最高，分别为 147 例（12.7%）和 61 例（5.3%），在慢性淋巴细胞白血病中的比例最低，分别为 55 例（3%）和 20 例（1.1%） . 黑人、美洲原住民和西班牙裔的比例非常低，反映出与普通人群相比，试验中的代表性不足。女性在急性髓性白血病中的代表性也不足（44.7%对60.5%，P < .0001），而在 MM 中男性（55.3%对60.2%，P< .0001) 和慢性粒细胞白血病 (55.2% v 62.9%, P < .0001)。试验的地理分布表明，与除 MM 以外的所有恶性肿瘤的死亡率相比，区域和州参与不足。

与受影响人群相比，导致白血病和 MM 药物批准的关键 CT 存在显着的人口和地理代表性不足和不平衡。需要解决这些差异，以使结果适用于所有相关人群。