The US National Institutes of Health (NIH) has announced results from its two-year Phase III trial of a combination leukemia therapy.
The therapy is comprised of all-trans retinoic acid and arsenic trioxide and has proven to be effective in both standard and high-risk acute promyelocytic leukemia patients.
The trial was conducted by the Children’s Oncology Group and followed a non-randomized decentralized group trial structure. 15f children and young people were involved, aged 1 to 22. The therapy was given orally in addition to intravenous administration of arsenic trioxide for 28 days.
Nearly all trial subjects survived for two years without any disease relapse following treatment. Standard chemotherapy was not needed for any of the children with standard-risk leukemia, while the higher-risk children were given four doses of a chemotherapy drug known as idarubicin.
For the standard-risk group, survival rates were 99%, and for the high-risk group 100%, with event-free survival rates reported as 98% and 96% respectively.
It should be noted that a child with standard-risk APL died early in the trial, and one relapsed later on. Two children with high-risk APL also experienced a relapse.
Severe side effects were uncommon but included high blood sugar, liver irritation, and bleeding.
The trial’s lead investigator, Matthew Kutny described working as a pediatrician and oncologist and the difficult talks with families about the intense treatment their children would have to receive, and said of the new treatment: “being able to offer a therapy that is less intense and has fewer side effects, but at the same time has amazingly high survival rates, is a really good feeling.”
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