Update on leukaemia

In the 1980s and 1990s, successive large, national leukaemia trials helped to determine basic treatment strategies that are effective in most children with leukaemia. In acute lymphoblastic leukaemia, the UKALL studies addressed CNS prophylaxis, duration of therapy, and intensive treatment blocks in improving event-free survival. Sufficient patients were recruited to permit direct comparison of individual drugs and deliver the answers within a timescale relevant to clinical practice. In acute myeloid leukaemia, collaboration with UK adult trials led to results in children that were not bettered anywhere in the world. The results of these trials were improved by advances in supportive care that were highly effective in reducing treatment-related mortality. The emphasis for paediatric leukaemia studies has changed over the last decade; more attention is being paid to patient subgroups that are performing badly, such as infants, Philadelphia chromosome-positive leukaemias, and relapsed and refractory acute myeloid leukaemia. Studies of these rare patients have been made possible by increased international collaboration that has allowed patients from many different countries to enter the same clinical trials. Interest in these difficult therapeutic areas has also been stimulated by the development of new agents and treatment strategies that have come directly from improved understanding of leukaemia biology.