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Treatment for myeloid leukaemia of Down syndrome: population‐based experience in the UK and results from the Medical Research Council AML 10 and AML 12 trials
Author(s) -
Rao Anupama,
Hills Robert K.,
Stiller Charles,
Gibson Brenda E.,
Graaf Siebold S.N.,
Hann Ian M.,
O'Marcaigh Aengus,
Wheatley Keith,
Webb David K.H.
Publication year - 2006
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/j.1365-2141.2005.05906.x
Subject(s) - medicine , myeloid leukaemia , down syndrome , clinical trial , pediatrics , population , myeloid leukemia , chemotherapy , myeloid , disease , psychiatry , environmental health
Summary Down syndrome (DS) children are at an increased risk of developing myelodysplasia and acute myeloid leukaemia (AML). We retrospectively analysed the population‐based data on 81 children with myeloid leukaemia of Down syndrome (ML‐DS) from the UK National Registry of Childhood Tumours and experience in the Medical Research Council (MRC) AML 10 and AML 12 trials, which enrolled 46 children with ML‐DS from 1988 to 2002. Eight per cent of UK children with AML had DS, but DS children comprised only 5% of children registered in MRC trials. The unique clinical characteristics of ML‐DS were confirmed. Overall survival (OS) of ML‐DS at 5 years increased from 47% in UK children diagnosed from 1988 to 1995 to 75% in children diagnosed from 1996 to 2002. OS for DS children registered in AML 10 and AML 12 was 74% in 5 years and improved from AML 10 to AML 12 (56% vs. 83%) There was no significant difference in OS between DS and non‐DS children (OS: 74% vs. 62%, P = 0·4) in the trials, but this result masked a significant increase in early death amongst DS children, with a significant reduction in mortality later on. Relapse was significantly reduced (3% vs. 39%, P = 0·0003), leading to the improved disease‐free survival (83% vs. 56%, P = 0·02). Given the increased number of earl treatment‐related deaths, future treatment protocols should aim to reduce chemotherapy dosage or intensity whilst maintaining low rates of resistant and recurrent disease.