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New Drug Combination Helps Young Leukaemia Patients Avoid Painful Chemotherapy

A clinical trial on children's cancer has revealed that a combination of all-trans retinoic acid, which is a metabolite of vitamin A, and arsenic trioxide is found to be very effective in curing children suffering from standard-and high-risk acute promyelocytic leukaemia, or APL.

Almost all the childhood cancer cases, which participated in the trial survived for two years, without suffering a relapse. None of the children needed conventional chemotherapy, and those in the high-risk category, got just four doses of the chemotherapy drug idarubicin or Idamycin PFS. The trial was done by the Children's Oncology Group and funded by the National Cancer Institute, part of the National Institutes of Health.

Commenting on the trial results, Malcolm A. Smith, M.D., of the Cancer Therapy Evaluation Program at the National Cancer Institute, said, "This is a remarkable achievement and will be the new standard of care. Twenty years ago, these patients would have been treated with intensive chemotherapy, including drugs that lead to heart problems later in life. By comparison, all-trans retinoic acid and arsenic trioxide have fewer acute or long-term side effects."

APL accounts for 5-10 percent of acute myeloid leukaemia in children and adolescents. Symptoms of this blood and bone marrow cancer include excessive bleeding, easy bruising, low red blood cell count, fever, and fatigue. The earlier forms of treatment for children with APL included anthracyclines, which could cause heart damage.

An earlier Children's Oncology Group trial had revealed that children with APL who were treated with arsenic trioxide and all-trans retinoic acid, along with chemotherapy drugs like anthracyclines, had high chance of event-free survival and low risk of relapse. Event-free survival is the amount of time after initial treatment that the cancer patient is free of worsening disease, relapse, or death.

The combination of all-trans retinoic acid and arsenic trioxide is at present the initial treatment given to adults with standard-risk APL as well. Adult patients with high-risk APL also undergo chemotherapy as well as maintenance therapy.

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