This clinical trial in the United Kingdom involved 78 children with relapsed or refractory Wilms tumour. The team compared a new risk-stratified protocol against historical observations for similar high-risk groups. The new approach used different chemotherapy combinations depending on the patient's risk group, with Group C receiving high-dose melphalan and autologous stem cell rescue.
After a follow-up period averaging 65.4 months for living patients, the study showed promising results. Four-year event-free survival for the whole group was 63%, while four-year overall survival was 68%. When combining Group A and Group B, survival rates were higher, reaching 77% for event-free survival and 81% for overall survival.
Safety was monitored closely throughout the trial. No transplant-related mortality occurred. However, 25 children died during the study, and all deaths were caused by the tumour itself rather than the treatment. Readers should understand that this evidence comes from a relatively small group of patients and compares a new protocol to past data rather than a current control group.