A gentle treatment could boost survival and quality of life for children whose leukemia has returned, a study led by a UCL academic has found.
The new regimen significantly reduces the need for intensive chemotherapy and eliminates treatment-related deaths in children with relapsed leukemia.
The study, led by experts including Dr David O’Connor (UCL Great Ormond Street Institute of Child Health), found that children and young people with relapsed acute lymphoblastic leukemia (ALL) can be safely treated with significantly less intensive chemotherapy.
The UKALL Rel2020 study, published in the journal Leukemia, involved young people taking less intensive chemotherapy before switching to blinatumomab, a targeted immunotherapy that tells the immune system to attack leukemia cells.
The study involved around 188 children and young people aged one to 24, drawn from 25 UK pediatric cancer centers between 2020 and 2024.
The new approach achieved a remission rate of 92%, while long-term survival (82% after three years) was comparable to children on more intensive conventional treatment plans.
No patients died during the initial phase of treatment, which experts said is a huge leap in care. ALL is the most common childhood cancer, with around 400 new cases each year in the UK.
Although cure rates for newly diagnosed disease are high, cancer coming back remains an issue.
Dr O’Connor, who is also a consultant in pediatric hematology at Great Ormond Street Hospital, said: “This approach is a major step forward in developing compassionate treatments for children suffering from relapsed cancer.
“The early stages of treatment traditionally carry significant risks. By using less intense chemotherapy and rapidly moving to blinatumomab, we were able to eliminate treatment-related deaths without compromising efficacy.”
patient’s story
Romine Winters was two years old when her parents noticed that she was unable to walk properly.
Her parents, Lisa and John, from Dunbar, East Lothian in Scotland, said: “We were on holiday in Yorkshire when we noticed Romain’s leg was starting to look weak and she was crawling rather than walking, and then she started to become more unwell and stopped eating.”
In October 2018, tests confirmed Romine had ALL and she was taken to NHS Lothian for treatment.
Her parents said: “Romin was given chemotherapy which had serious side effects which left her ill. Unfortunately, within 18 months after treatment, she fell ill again.
“We were offered a bone marrow transplant or trying blinatumomab with a low dose of chemotherapy. By this time Romine was seven years old and we considered the options and what her future might look like.
“Blinatumomab seemed scary because it was unknown, but we thought it would give us more options if she didn’t respond to treatment, and hopefully she would recover more quickly because it seemed less severe than stronger chemotherapy.
“Her medical team really supported us well and kept us informed every step of the way. After some side effects initially in hospital, Romine was recovering well and was able to take blinatumomab at home via her pump in her backpack.
“Within a few days, she was back playing on the trampoline with her brothers, which was great to see. Romine is now undergoing treatment and is recovering well, the blinatumomab has made it easier for her to tolerate.
“Since having her chemo port removed, she has been able to take part in new childhood hobbies. We went on holiday to Tenerife and she learned to swim in a week – now we can’t keep her away from the swimming pool.”
The clinical trial was different from others because it was delivered as routine NHS care, making the results more representative of real-world patients, Dr O’Connor said.
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