Disparities in Clinical Trial Participation in Children and Adolescent Patients With a Hematologic Malignancy

Low socioeconomic groups and racial/ethnic minorities continue to experience pediatric cancer outcome disparities, and remain underrepresented in clinical trials. It is vital to understand why underrepresentation exists and to address it in order to generalize trial findings to all groups. This study examined institutional disparities in clinical trial offerings and enrollment for children and adolescents with hematologic malignancies.

We conducted a single-institution retrospective analysis of clinical trial participation in patients less than 18 years old with newly diagnosed hematologic malignancies between 2011 and 2017. Patient demographics (e.g., parental primary language, race) were abstracted, and patient address at diagnosis was geocoded to characterize neighborhood socioeconomic status. Endpoints were frontline therapeutic clinical trial offering and enrollment. Multivariable logistic regression was constructed to examine predictors of trial enrollment.

Among 464 trial-eligible patients, 90.1% were offered clinical trial participation, of which 85% enrolled. There was no significant difference in enrollment by age, sex, parental primary language, neighborhood socioeconomic status, or rurality. However, non-Hispanic Black patients [OR: 0.4 (95% CI: 0.20-0.8), p = 0.01] and patients with lymphoma [OR: 0.15 (95% CI: 0.04-0.6), p = 0.01] were less likely to enroll on a clinical trial in our adjusted analysis.

Despite a high institutional clinical trial enrollment rate for eligible patients, we found racial and disease-type disparities. Further work is needed to more granularly determine reasons for not offering trial participation or for not enrolling. By better-defining barriers to clinical trial enrollment, targeted institution-level interventions can be created to improve trial enrollment and reduce outcome disparities.