Gentler treatment eliminates early deaths for children with relapsed leukaemia

ALL is the most common childhood cancer. Although cure rates for newly diagnosed disease are high, relapse remains a major challenge. Traditional reinduction chemotherapy is highly intensive and has historically been associated with significant toxicity, including a risk of death during treatment.

Published in Leukemia, the UKALL Rel2020 study introduces a fundamentally different strategy, using a gentler initial course of treatment before moving patients onto blinatumomab, a targeted immunotherapy that directs the immune system to attack leukaemia cells.

The study analysed the outcomes of 188 children and young people aged 1-24 years, treated across all 25 UK paediatric cancer centres between 2020 and 2024, representing a truly national effort.

Despite using significantly less chemotherapy, the new approach achieved remission rates exceeding 90% and long-term survival outcomes comparable to those seen with more intensive traditional treatment plans.

Crucially, no patients died during the early phase of treatment, marking a major advance in the safety of relapse therapy. These findings suggest that highly intensive chemotherapy may not be required to achieve excellent outcomes in relapsed ALL. This approach has the potential to redefine the standard of care and improve both survival and quality of life for children facing relapse.

Dr David O’Connor, consultant in paediatric haematology at GOSH and honorary associate professor at UCL Great Ormond Street Institute of Child Health, said: “This approach marks a major step forward in developing kinder treatments for children with relapsed cancer.

“The early phase of treatment has traditionally carried significant risks. By using lower-intensity chemotherapy and moving rapidly to blinatumomab, we were able to eliminate treatment-related deaths without compromising effectiveness.”

Romyn was two years old when her parents noticed that she couldn’t walk properly. Her parents, Lisa and John, from Dunbar in East Lothian said: “We were on holiday in Yorkshire when we noticed Romyn’s leg was starting to look weak and she was crawling instead of walking, and then she started to get more unwell and stopped eating.”

In October 2018, tests confirmed Romyn had ALL and was taken to NHS Lothian for treatment.

Her parents continued: “Romyn had chemotherapy which had harsh side effects that made her sick. Unfortunately, within 18 months following treatment, she had relapsed. We were offered a bone marrow transplant or to try blinatumomab with a lower dose of chemotherapy. By this time Romyn was 7 and we weighed up the options and what her future might look like.

“Blinatumomab sounded scary as it was unknown, but we thought it would give us more options if she didn’t respond to the treatment, and hopefully she’d recover more quickly as it sounded less severe than the stronger chemotherapy. Dr Baird and her team supported us really well and kept us informed every step of the way. After a few expected side effects to begin with in hospital, Romyn was coping well and was able to have blinatumomab at home via her pump in a backpack. Within days she was back playing on the trampoline with her brothers which was great to see.

“Romyn is now off treatment and recovering well thanks to the blinatumomab being easier for her to tolerate. Since having her chemo port removed, she’s been able to take part in new childhood hobbies like her friends. We went on holiday to Tenerife and she learnt to swim in a week – we now can’t keep her away from a swimming pool!”

Unlike traditional clinical trials, UKALL Rel2020 was delivered as routine NHS care across all 25 paediatric oncology centres, making the results highly representative of real-world patients, including those often excluded from trials.

The results build on a previous study demonstrating the effectiveness of blinatumomab in children with B-ALL who were unable to tolerate intensive chemotherapy.

GOSH is developing a new Children’s Cancer Centre, putting children at the front and centre, with their needs at the very heart of the design. The new facility will also make it easier for clinical teams to combine the very best care with the latest research and innovation - working together to deliver a step change for children with cancer. Find out how you can support the Children’s Cancer Centre.