Study Exposes Demographic Imbalance in Clinical Trials for Leukemias, Myeloma

By Leah Sherwood - Last Updated: November 16, 2022

There are significant demographic and geographic imbalances in pivotal clinical trials leading to drug approvals for leukemias and multiple myeloma (MM) compared with the population affected, according to a report published in the Journal of Clinical Oncology.

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The study, which was intended to evaluate the extent to which clinical trials match the demographic and geographic diversity of populations affected by leukemias and MM, was described in a paper by Mycal Casey, DO, MPH, and his colleagues at Augusta University in Georgia.

The researchers collected demographic and geographic data from ClinicalTrials.gov and primary manuscripts and then analyzed the data statistically by race, ethnicity, sex, and malignancy subtypes.

The results showed that pivotal randomized clinical trials for new agents in myeloma and leukemias are not representative of the afflicted population, underrepresenting Black, Native American, and Hispanic patients.

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. Proportions for Blacks, Native Americans, and Hispanics were significantly lower, reflecting underrepresentation in trials compared with the proportion in the general population.

Asian-Pacific Islanders and Blacks had the highest representation in chronic myeloid leukemia (n=147 [12.7%] and n=61 [5.3%], respectively) and lowest in chronic lymphocytic leukemia (n=55 [3%] and n=20 [1.1%], respectively).

Females were also underrepresented in acute myeloid leukemia, and males in MM and chronic myeloid leukemia.

The geographic distribution of trials showed inadequate regional and state participation compared with mortality for all malignancies except MM.

“There are significant disparities in care and outcomes for patients with leukemias and MM,” the authors wrote. “These disparities need to be addressed to make results applicable to all relevant populations.”

Casey M, Odhiambo L, Aggarwal N, et al. Are pivotal clinical trials for drugs approved for leukemias and multiple myeloma representative of the population at risk? J Clin Oncol. 2022. doi:10.1200/JCO.22.00504

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