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Updated: Sep 2, 2025

Pre-clinical Evaluation of Tyrosine Kinase Inhibitors for Treatment of Acute Leukemia
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Are Pivotal Clinical Trials for Drugs Approved for Leukemias and Multiple Myeloma Representative of the Population at

Mycal Casey1, Lorriane Odhiambo2, Nidhi Aggarwal3

  • 1Internal Medicine, Medical College of Georgia at Augusta University, Augusta, GA.

Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology
|August 9, 2022
PubMed
Abstract

Insights

Clinical trials for leukemias and multiple myeloma (MM) show significant demographic and geographic under-representation. These disparities mean trial results may not apply to all affected populations, highlighting a need for broader inclusion in cancer research.

Area of Science:

  • Hematology
  • Clinical Trials Research
  • Health Disparities

Background:

  • Significant disparities exist in the care and outcomes for patients diagnosed with leukemias and multiple myeloma (MM).
  • The representativeness of clinical trials (CTs) in reflecting the diversity of affected populations is crucial for generalizability.

Purpose of the Study:

  • To evaluate the extent to which clinical trials leading to drug approval for leukemias and MM match the demographic and geographic diversity of the populations impacted by these diseases.
  • To identify imbalances in patient representation within pivotal clinical trials.

Main Methods:

  • Identified CTs leading to drug approval using US Food and Drug Administration databases.
  • Collected demographic and geographic data from ClinicalTrials.gov and primary manuscripts.
  • Summarized data using descriptive statistics, analyzing representation by race, ethnicity, sex, and malignancy subtypes.

Main Results:

  • Of 41 trials reporting race and 20 reporting ethnicity, most patients (81.6%) were White.
  • Minority groups, including Asian-Pacific Islanders and Blacks, showed varied representation across leukemia subtypes, with overall low proportions for Blacks, Native Americans, and Hispanics compared to the general population.
  • Females were under-represented in acute myeloid leukemia trials, while males were under-represented in multiple myeloma and chronic myeloid leukemia trials. Geographic distribution also showed inadequate participation relative to mortality rates.

Conclusions:

  • Pivotal CTs for leukemias and MM demonstrate significant demographic and geographic under-representation and imbalances when compared to the affected populations.
  • Addressing these disparities is essential to ensure that clinical trial outcomes are applicable to all relevant patient groups.

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