Paediatric myelodysplastic syndromes and juvenile myelomonocytic leukaemia in the UK: a population‐based study of incidence and survival

• Published in: British journal of haematology 2003-06, Vol.121 (5), p.758-767
• Main Authors: Passmore, S. Jane, Chessells, Judith M., Kempski, Helena, Hann, Ian M., Brownbill, Pat A., Stiller, Charles A.
• Format: Article
• Language: English
• Subjects: Adolescent; Biological and medical sciences; Child; Child, Preschool; Female; Hematologic and hematopoietic diseases; Hematology; Humans; Incidence; Infant; Infant, Newborn; juvenile myelomonocytic leukaemia; Leukemia, Myeloid - mortality; Leukemia, Myeloid - therapy; Leukemias. Malignant lymphomas. Malignant reticulosis. Myelofibrosis; Male; Medical sciences; myelodysplastic syndrome; Myelodysplastic syndromes; Myelodysplastic Syndromes - mortality; Myelodysplastic Syndromes - therapy; Prognosis; prognostic factors; Survival Analysis; Survival Rate; United Kingdom - epidemiology
• ISSN: 0007-1048; 1365-2141
• DOI: 10.1046/j.1365-2141.2003.04361.x

We aimed to identify and classify cases of paediatric myelodysplastic syndromes (MDS) occurring in Britain to estimate the incidence of this rare group of diseases, investigate the results of therapy and identify prognostic risk factors. Patients aged below 15 years at diagnosis were collected from England, Scotland and Wales, inclusively between 1990 and 1999. One hundred and thirty‐five patients were accepted as de novo MDS or juvenile myelomonocytic leukaemia (JMML). The incidence for this period was 1·35 per million (age standardized rate) which is below that reported outside the UK. The overall survival was 45%[standard error (SE) = 4%] at 5 years: 40% (SE = 6%) for JMML and 50% (SE = 6%) for other MDS. Significant adverse prognostic factors for JMML were a platelet count