30 September 2024

Many children with B-cell acute lymphoblastic leukaemia (ALL) can be given low intensity therapy, according to a major new US study.

A clinical trial led by researchers from at St Jude Children's Research Hospital, Memphis, Tennessee now suggests that, for patients with two genetic subtypes, ETV6::RUNX1 and high-hyperdiploid, low intensity treatment is safe and effective – even if they are designated ‘high risk’ by the National Cancer Institute classification.

The ETV6::RUNX1 subtype affects 25% of patients, while the high-hyperdiploid subtype affects 30% of patients. The findings of the study came as part of a major research project at the centre, evaluating links between genomics, risk assessment and patient outcomes, running since the year 2000.

93% of the patients with the two genetic subtypes – more than 500 in total – received low intensity treatment. Reporting in Blood, the researchers say the outcomes were “positive”, with good event-free survival and overall survival.

The researchers report that patients experienced a reduction in side effects, such as thrombosis and pancreatitis.

Researcher Dr Hiroto Inaba said: “We tailored chemotherapy to better match patient needs and successfully identified who could benefit from low-intensity therapy. This strategy highlights our goal of personalising treatment based on individual patient characteristics for better outcomes."

Fellow researcher Dr Katelyn Purvis said: “We now have tangible evidence that reduced therapy can be beneficial for some patients - which decreases toxicity. Our goal for every child who walks through our doors with leukaemia is to not only cure them but also to extend their lives by decades with minimal side effects. We’re proud that we can achieve this for most of our leukaemia patients, which is truly remarkable.” 

Source:

Purvis K, Zhou Y, Karol SE, Rubnitz JE, Ribeiro RC, Lee SH, Yang JJ, Bowman WP, Wang L, Dixon SB, Roberts KG, Gao Q, Cheng C, Mullighan CG, Jeha S, Pui CH, Inaba H. (2024) “Outcomes in patients with ETV6::RUNX1 or high-hyperdiploid B-ALL treated in the St. Jude Total Therapy XV/XVI studies.” Blood, 24 September 2024, doi: 10.1182/blood.2024024936.

Link: https://ashpublications.org/blood/article-abstract/doi/10.1182/blood.2024024936/517953/Outcomes-in-patients-with-ETV6-RUNX1-or-high

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