AML is a rare disease in children and teenagers (70 cases per annum in children less than 16 years in the UK), but is a significant cause of childhood cancer mortality. This trial is an International collaboration and will recruit patients with newly diagnosed AML, high risk myelodysplastic syndrome (MDS) defined as greater than 10% blasts in the bone marrow, and isolated myeloid sarcoma (MS) up to their 18th birthday. Children with myeloid leukaemia of Down syndrome (ML DS) and acute promyelocytic leukaemia (APL) are excluded. The expected number of patients recruited per year is 120-150 or up to 700 cases in 5-6 years and is sufficient to address the four randomised questions proposed.
All major national groups report similar outcomes for childhood AML: overall survival (OS) 65-70%, event-free survival (EFS) 50-60% and cumulative incidence of relapse (CIR) 35-40% (consensus of the International Berlin Frankfurt Munster (I-BFM) AML group, 2011). Whilst advances in supportive care and better salvage therapy after relapse have led to a moderate improvement in OS, the CIR remains unacceptably high with relapsed disease the commonest cause of death. This study plans to build on experience gained from previous UK, French and international trials and to test a number of strategies with the potential to improve outcome: 1) intensive anthracycline or anthracenedione therapy combined with cytarabine in induction, 2) induction intensification with a higher dose of gemtuzumab ozogamicin, 3) assessment of fludarabine, a purine analogue, in consolidation and 4) evaluation of reduced intensity conditioning (RIC) in allogeneic haemopoietic stem cell transplantation (HSCT) in 1st complete remission (CR1). In addition, risk group stratification will direct therapy and will include cytogenetic/molecular genetic characteristics, morphological response to induction therapy and minimal residual disease (MRD) assessment of treatment response. Different MRD methodologies will be studied for their predictive value. The treatment choices and risk stratification are now discussed with relevance to previous UK and French studies and the literature.
Please note that the trials team cannot give individuals clinical advice. Patients and their families should contact their treating physician to discuss trials for which they may be eligible
||Professor Brenda Gibson
||University of Birmingham
||CRUK (Cancer Research UK)
||Clinical Trial of an Investigational Medical Product
||Open to recruitment
|UKCRN Study ID:
|Open to new sites?
|Recruitment start date:
|Anticipated Recruitment end date
|CRCTU Trial Management Team:
||Children's Cancer Trials Team
|Trial E-mail Address: