Children with acute myeloid leukemia (AML) are often treated with a class of drugs called anthracyclines, which work by disrupting cancer cell DNA to halt growth. Patients treated with anthracyclines need careful monitoring due to potential cardiac side effects. A study reported in JAMA Oncology found that dexrazoxane, a cardioprotective agent, reduces cardiovascular complications in pediatric AML patients.
The study utilized data from the Pediatric Health Information System (PHIS) encompassing more than 40 children’s hospitals in the United States. Researchers examined more than 2000 children with AML to determine the impact of dexrazoxane on heart-related healthcare services, including whether patients required intensive care unit (ICU) resources for cardiovascular complications.
The study found that the use of dexrazoxane significantly increased from 0.5% in 2010 to 98.5% in 2021. This rise coincided with a reduction in the need for ICU level cardiovascular care, which dropped from 24.3% in 2010 to 11.3% in 2021. Patients who started on dexrazoxane had a 36% lower chance of needing ICU-level care. Additionally, the six-month mortality rate was 7.0% for patients using dexrazoxane, which was slightly lower than the 8.9% mortality rate for those who did not use it.
“Our findings highlight a significant shift in pediatric AML treatment practices, with near-universal adoption of dexrazoxane over the last decade leading to reduced cardiovascular complications,” said Daniel J. Zheng, MD, MHS, MSHP, the study’s lead author and an attending physician in the Division of Oncology at Children’s Hospital of Philadelphia. “This cardioprotective strategy demonstrates improved healthcare outcomes and suggests potential benefits for a wider range of cancer patients who receive anthracyclines.”
The authors noted that further research is needed to comprehensively understand dexrazoxane's impact on more direct measurements of cardiac health beyond resource utilization.
Dr. Zheng was supported by the American Society of Clinical Oncology Conquer Cancer (Novartis Oncology Endowed) Young Investigator Award in Health Disparities, a St. Baldrick’s Scholar Award and National Cancer Institute (K12CA07693). The research was additionally supported by grants from National Heart, Lung, and Blood Institute (NHLBI) (R01HL164925 PI: Getz and R01HL163657 PI: Aplenc/Getz).
Zheng et al. “National trends in dexrazoxane and cardiovascular healthcare utilization for children with AML.” JAMA Oncol. Online October 23, 2025. DOI: 10.1001/jamaoncol.2025.4365.
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Children with acute myeloid leukemia (AML) are often treated with a class of drugs called anthracyclines, which work by disrupting cancer cell DNA to halt growth. Patients treated with anthracyclines need careful monitoring due to potential cardiac side effects. A study reported in JAMA Oncology found that dexrazoxane, a cardioprotective agent, reduces cardiovascular complications in pediatric AML patients.
The study utilized data from the Pediatric Health Information System (PHIS) encompassing more than 40 children’s hospitals in the United States. Researchers examined more than 2000 children with AML to determine the impact of dexrazoxane on heart-related healthcare services, including whether patients required intensive care unit (ICU) resources for cardiovascular complications.
The study found that the use of dexrazoxane significantly increased from 0.5% in 2010 to 98.5% in 2021. This rise coincided with a reduction in the need for ICU level cardiovascular care, which dropped from 24.3% in 2010 to 11.3% in 2021. Patients who started on dexrazoxane had a 36% lower chance of needing ICU-level care. Additionally, the six-month mortality rate was 7.0% for patients using dexrazoxane, which was slightly lower than the 8.9% mortality rate for those who did not use it.
“Our findings highlight a significant shift in pediatric AML treatment practices, with near-universal adoption of dexrazoxane over the last decade leading to reduced cardiovascular complications,” said Daniel J. Zheng, MD, MHS, MSHP, the study’s lead author and an attending physician in the Division of Oncology at Children’s Hospital of Philadelphia. “This cardioprotective strategy demonstrates improved healthcare outcomes and suggests potential benefits for a wider range of cancer patients who receive anthracyclines.”
The authors noted that further research is needed to comprehensively understand dexrazoxane's impact on more direct measurements of cardiac health beyond resource utilization.
Dr. Zheng was supported by the American Society of Clinical Oncology Conquer Cancer (Novartis Oncology Endowed) Young Investigator Award in Health Disparities, a St. Baldrick’s Scholar Award and National Cancer Institute (K12CA07693). The research was additionally supported by grants from National Heart, Lung, and Blood Institute (NHLBI) (R01HL164925 PI: Getz and R01HL163657 PI: Aplenc/Getz).
Zheng et al. “National trends in dexrazoxane and cardiovascular healthcare utilization for children with AML.” JAMA Oncol. Online October 23, 2025. DOI: 10.1001/jamaoncol.2025.4365.
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