22 November 2021

A treatment for children with an aggressive type of acute myeloid leukaemia could become “the new standard of care” following a successful trial in the USA.

The trial, conducted by the Children’s Oncology Group, found that combining all-trans retinoic acid and arsenic trioxide is highly effective in children with standard- and high-risk acute promyelocytic leukaemia (APL) – eliminating or reducing the need for chemotherapy.

Dr Malcolm Smith, of the Cancer Therapy Evaluation Program at the US National Cancer Institute, which funded the multi-institutional, non-randomised phase 3 co-operative group trial, described the results as “remarkable”.

The combination of all-trans retinoic acid and arsenic trioxide resulted in almost all patients in the trial surviving for two years without experiencing a relapse.

None of the children with standard-risk APL required conventional chemotherapy, while those with high-risk APL received just four doses of the chemotherapy drug idarubicin.

“This is a remarkable achievement and will be the new standard of care,” said Dr Smith.

“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 between 5% and 10% of acute myeloid leukaemia diagnoses in children and adolescents.

In this study, 154 children and young people, aged between one year and 22 years, who were newly diagnosed with standard- or high-risk APL were given oral all-trans retinoic acid, with intravenous arsenic trioxide, every day for at least 28 days.

Children with high-risk APL also received four doses of the anthracycline idarubicin during the initial phase of treatment.

The trial found that children with standard- and high-risk APL had two-year overall survival rates of 99% and 100%, respectively, and the two-year event-free survival rates were 98% and 96%, respectively.

One child with standard risk APL died early in treatment, and three children – one with standard risk APL and two with high-risk APL – experienced a relapse.

Lead investigator Dr Matthew Kutny, of Children’s of Alabama and the University of Alabama, said because there was no need for maintenance therapy, the length of time the children were undergoing treatment was reduced from more than two years to approximately nine months.

However, more follow-up will be needed to determine the long-term effects of treating children with all-trans retinoic acid and arsenic trioxide.


Kutny MA, Alonzo TA, Abla O, Rajpurkar M, Gerbing RB, Wang YC, Hirsch BA, Raimondi S, Kahwash S, Hardy KK, Hardy S, Meshinchi S, Gamis AS, Kolb EA, Feusner JH, Gregory J Jr. (2021) “Assessment of Arsenic Trioxide and All-trans Retinoic Acid for the Treatment of Pediatric Acute Promyelocytic Leukemia A Report From the Children’s Oncology Group AAML1331 Trial.” JAMA Oncology, doi: 10.1001/jamaoncol.2021.5206.

 Link: https://jamanetwork.com/journals/jamaoncology/fullarticle/2786069   

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