A new tool could help children with leukemia on blinatumomab avoid chemotherapy they do not need

Children with the most common childhood cancer may be able to safely skip some chemotherapy, according to a UK-wide study led by Newcastle University and Great Ormond Street Hospital (GOSH), Newcastle University said on 21 October. The study, published in Blood Advances, looked at more than 200 children with B-cell acute lymphoblastic leukemia (B-ALL) treated at 24 centers in the UK and Ireland.

Old warning signs stopped working

The children were treated with blinatumomab, an immunotherapy that replaces parts of standard chemotherapy. The researchers found that the usual predictors of relapse, such as age, white blood cell count, high-risk genetic features and the amount of leukemia left before treatment, were no longer reliable for these patients.

Instead, the strongest predictor was how much leukemia remained after the first cycle of blinatumomab. Two genetic subtypes, IKZF1plus and DUX4-rearranged leukemia, were linked to a poorer response and a higher risk of relapse. The team combined early response and genetics into a risk tool called BlinPredict.

“Less chemotherapy, more days at school, more time at home.” — Dr Sujith Samarasinghe, consultant pediatric hematologist at GOSH and joint lead author

What it means for patients now

This is not a new drug but a better way to decide who needs what. Children who respond strongly early on may do well with reduced chemotherapy, which can mean fewer side effects, while higher-risk children can be identified for a different approach. The findings have already led to revisions of the UK's national treatment guidelines and are guiding how children across the UK are treated, the university said.

Families should discuss treatment decisions with their child's care team; the tool applies to children receiving blinatumomab as part of frontline treatment. “This research will help clinicians identify which patients are likely to do exceptionally well with reduced chemotherapy,” said Prof. Anthony Moorman of Newcastle University.

Adults and resistant leukemia are next

The researchers now plan to study why some leukemias resist blinatumomab and to look for risk factors in other groups, including adults with ALL. The work was funded by Horizon Europe, Marie Skłodowska-Curie Actions and Cancer Research UK. Other progress in blood cancer is collected under cancer treatment.