High-risk paediatric leukaemia: immunotherapy halves the rate of relapse
According to the study’s findings, the new strategy has ‘almost halved the number of relapses’ and significantly reduced the serious side effects associated with intensive chemotherapy
The challenge in high-risk paediatric acute lymphoblastic leukaemia (ALL) is to strike a balance between treatment efficacy and toxicity. An international study recently published in the New England Journal of Medicine shows that this balance can be shifted: by replacing two cycles of intensive chemotherapy with two cycles of immunotherapy, relapse rates were almost halved and serious side effects were reduced.
The trial – which involved over 100 centres across eight countries between 2018 and 2023 – focused specifically on that group of paediatric patients who, whilst benefiting from the significant therapeutic advances made in ALL, are at a higher-than-average risk of relapse. Today, in industrialised countries, around 90 per cent of children with acute lymphoblastic leukaemia can achieve long-term remission. However, around one in five patients falls into a category considered high-risk and therefore requires more targeted treatment strategies.
It was in this group that the researchers trialled replacing two cycles of high-intensity chemotherapy with two cycles of blinatumomab-based immunotherapy. According to the study’s findings, the new strategy ‘almost halved the number of relapses’ and significantly reduced the severe side effects associated with intensive chemotherapy. The rationale behind the treatment is to harness the patient’s own immune defences directly. Blinatumomab is designed to bring T-lymphocytes – key cells of the immune system – into contact with leukaemia cells. In this way, it facilitates the recognition and destruction of cancer cells by the immune system itself.
The result is particularly significant because, in high-risk paediatric ALL, intensifying treatment has so far been one of the strategies used to prevent relapses. However, more intensive treatment can lead to significant toxicity, with side effects that can be serious and, in some cases, fatal. The aim of the strategy tested in the study is therefore twofold: to maintain control of the disease and to reduce the biological cost of treatment.
“The aim of this important study was to try to further improve the chances of recovery, whilst minimising as far as possible the toxicity of the treatment for children and its impact on their families,” explains Andrea Biondi, scientific director of the Tettamanti Foundation and the study’s principal investigator for Italia.
