Chemotherapy-free regimen benefits children with acute promyelocytic leukemia
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Children with acute promyelocytic leukemia achieved survival benefit with an all-trans retinoic acid and arsenic trioxide chemotherapy-free regimen, according to study results published in JAMA Oncology.
Researchers additionally reported efficacy of the regimen plus four doses of idarubicin during induction therapy only and no maintenance therapy among children with high-risk acute promyelocytic leukemia (APL).
“We demonstrated very high cure rates for children with APL treated with an all-trans retinoic acid and arsenic trioxide regimen, and children experienced fewer acute and chronic side effects compared with traditional chemotherapy regimens,” Matthew A. Kutny, MD, associate professor and director of The University of Alabama at Birmingham’s leukemia, lymphoma and histiocytosis program, told Healio.
Rationale
All-trans retinoic acid, a metabolite of vitamin A, and arsenic trioxide are established targeted therapies for patients with APL.
“The treatment regimen for children with acute promyelocytic leukemia additionally includes intensive cycles of conventional chemotherapy that may cause adverse events in children,” Kutny said.
Methodology
Researchers assessed the safety of all-trans retinoic acid and arsenic trioxide treatment regimen and whether it allowed for elimination or significant reduction of chemotherapy use among 154 patients aged 1 to 22 years (median age, 14.4 years; 52.6% male) with either standard-risk (n = 98) or high-risk (n = 56) APL. Patients received treatment daily for at least 28 days. During induction therapy only, children with high-risk APL additionally received four doses of idarubicin.
“We tested treatment regimens with little or no conventional chemotherapy — depending on risk group — to establish better and less toxic treatments for children with APL,” Kutny said. “This cooperative group clinical trial had a primary goal of achieving excellent cure rates for children with APL with fewer side effects from treatment.”
Key findings
Researchers reported median follow-up of 24.7 months (range, 0-49.5) for the standard-risk group and 22.8 months (range, 0-47.7) for the high-risk group.
Patients at standard-risk for APL experienced a 2-year EFS rate of 98% and an OS rate of 99%. In this group, one patient died during induction therapy and one patient experienced relapse.
Patients with high-risk APL experienced a 2-year EFS rate of 96.4% and an OS rate of 100%. Two high-risk patients experienced relapse, and no patient deaths occurred.
Implications
“The treatment used on this clinical trial was highly effective and well tolerated, but the arsenic trioxide is an intravenous infusion that requires numerous visits to a hospital or clinic,” Kutny said. “Based on these findings, future research plans of the Children’s Oncology Group, supported by the National Cancer Institute and St Baldrick’s Foundation, include a new oral formulation of arsenic trioxide that would significantly decrease the burden of care for patients and their families.”
For more information:
Matthew A. Kutny, MD, can be reached at The University of Alabama at Birmingham, 1600 Seventh Ave. S, Lowder 512, Birmingham, AL 35233; email: mkutny@peds.uab.edu.
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