TKI Outcomes in AML Similar Across Racial, Ethnic Groups

Tyrosine kinase inhibitors (TKIs) lead to similar outcomes across racial and ethnic groups when used to treat acute myeloid leukemia (AML), according to a new report.

The findings, which were based on an analysis of electronic health record data in real-world settings, suggest that access to TKIs could help promote equitable outcomes among patients with TKIs. The study was published in Blood and Lymphatic Cancer: Targets and Therapy.¹

The authors said TKIs targeting specific AML mutations qualify as a significant advancement in the treatment of patients with AML. Yet, TKIs have not managed to erase disparities in real-world outcomes among patients with AML. A 2011 study, for instance, found that Black patients were more likely to have complex karyotypes than White patients, as well as lower rates of clinical trial participation and hematopoietic stem cell transplantation.² Still, this study also found that overall survival (OS) was not significantly different between Black and White patients.

Targetable mutations in AML appear to be distributed equally across racial and ethnic groups in the US, the authors explained. However, they said there is relatively little research looking at whether TKI therapy works equally well across racial and ethnic demographic groups, as racial and ethnic minorities are often underrepresented in clinical trials, potentially limiting the generalizability of trial outcomes.

To delve into these questions, the investigators turned to the Flatiron Health Research Database, which included data from 280 cancer clinics in the US. Patients were included in the analysis if they were at least 18 years old and were treated with a TKI targeting FLT3, IDH1, or IDH2 as monotherapy or with a hypomethylating agent (HMA). The study’s timeframe was from 2015 to 2023.

A total of 482 patients were identified. They had a median age at diagnosis of 69 years, and a median age of 70 years at TKI initiation. Fifty-two percent of participants were male, 70% were non-Hispanic White ethnicity, 5.6% were Hispanic/Latino, 5.6% were Black and non-Hispanic or an unknown ethnicity, 3.3% were Asian and non-Hispanic or an unknown ethnicity, 8.1% had no race listed and were not Hispanic or had an unknown ethnicity, and 8.1% had no race listed in their health record. Nearly half of patients (48.1%) had Medicare as their primary insurance, and 52% received care in a community cancer center.

In terms of treatment, 18.4% of patients received both a TKI and an HMA, and 10% of patients eventually received a stem cell transplant following TKI therapy, the authors said. In terms of risk profile, most patients had either intermediate (52%) or adverse (42%) risk profiles according to the European Leukemia Net 2017 classifications.

The authors found that outcomes did not differ significantly by race or ethnicity in terms of OS or event-free survival (EFS). For instance, among patients receiving TKIs as a first-line therapy, the real-world EFS was 2.2 months (95% CI, 0.8-5.0) for patients of color and 2.6 months for non-Hispanic White patients (95% CI, 1.1-not reached). For second-line-and-beyond TKI therapy, real-world OS was 9.7 months (95% CI, 8.3-11.5) for patients of color and 9.9 months (95% CI, 7.4-15.1) for non-Hispanic White patients.

The authors found other differences between patients of color and White patients. Patients of color tended to be younger at diagnosis and were more likely to be treated in a community setting, have Medicaid insurance, and be in the lowest socioeconomic quintile.

Overall, though, they said their findings suggest that if access to TKIs is equal among racial and ethnic groups, outcomes might become more equal, too.

“These findings should alert clinicians to consider prioritizing the use of TKIs in diverse populations, while acknowledging that the overall survival outcomes for AML remain poor,” they concluded.

References

1. Espinoza-Gutarra MR, Jarrett BA, Wang X, Afghahi A, Bae S. Health disparities in acute myeloid leukemia patients undergoing treatment with tyrosine kinase inhibitor (TKI) therapy targeting FLT3, IDH1, or IDH2. Blood Lymphat Cancer. 2026;16:559759. doi:10.2147/BLCTT.S559759
2. Bierenbaum J, Davidoff AJ, Ning Y, Tidwell ML, Gojo I, Baer MR. Racial differences in presentation, referral and treatment patterns and survival in adult patients with acute myeloid leukemia: a single-institution experience. Leuk Res. 2012;36(2):140-145. doi:10.1016/j.leukres.2011.10.018