Childhood Cancer Survivors

Researchers Conduct Groundbreaking Clinical Trial to Prevent Heart Failure Among Childhood Cancer Survivors


Researchers at City of Hope, in collaboration with the Children’s Oncology Group (COG), have recently completed the largest clinical trial to date aimed at reducing the risk of childhood cancer survivors developing heart failure. The findings, published in The Lancet Oncology, reveal that the medication carvedilol, which relaxes blood vessels, is safe for childhood cancer survivors and may improve indicators of heart injury caused by chemotherapy exposure.

One of the long-term side effects of anthracycline chemotherapy, commonly used to treat childhood cancers, is an increased risk of heart failure. This occurs when the heart is unable to pump enough blood to meet the body’s needs. This gradual process is marked by changes in the heart muscle, such as thinning and enlargement of its chambers. Unfortunately, once heart function decline sets in, it is irreversible. This highlights the urgent need for early prevention strategies.

As the number of childhood cancer survivors continues to grow, it is crucial to develop interventions that can improve their long-term health. Merely ensuring their survival is not enough. We must also focus on optimizing their health, so they do not have to face life-threatening side effects years after being cancer-free, explained Saro H. Armenian, D.O., M.P.H., Barron Hilton Chair in Pediatrics at City of Hope Children’s Cancer Center and corresponding author of The Lancet Oncology study.

This groundbreaking clinical trial, led by physicians from City of Hope, was a randomized, double-blind Phase IIb study conducted at 30 COG-member hospitals in the United States and Canada (COG Study ID: ALTE1621). A total of 182 participants were enrolled, and they were given relatively low doses of either carvedilol or a placebo equivalent for two years. The study found no significant differences in side effects between the two groups, suggesting that carvedilol is well-tolerated by childhood cancer survivors.

Although the trial did not achieve its original objective of reducing heart muscle thinning and chamber enlargement, it did show significant improvements in heart left ventricular end-systolic wall stress, an earlier biomarker of deteriorating heart health.

The study demonstrated the greatest benefits in participants who were long-term survivors and those who strictly adhered to the study medication regimen. Additionally, out of the eight patients who experienced clinically significant heart function decline while on the study, six were in the placebo group and two were receiving carvedilol, noted Armenian.

The results of this research lay the foundation for a Phase III clinical trial, which may provide substantial benefits for high-risk patients who are at risk of irreversible heart function decline after completing cancer therapy.

Douglas S. Hawkins, M.D., COG group chair and a professor of hematology-oncology at Seattle Children’s Hospital, remarked, “Conducting this study across 30 institutions and among long-term survivors of childhood cancer illustrates the strengths of the COG network. An intervention study on this scale would not be feasible outside of COG.” He emphasized that future research should focus on determining the optimal timing, duration, and target population for carvedilol intervention.

This study represents a crucial first step towards developing future studies that aim to optimize the long-term well-being and health of childhood cancer survivors, who are expected to live for decades after their initial cancer diagnosis.

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