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

Abstract

Summary. 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 1AE35 per million (age standardized rate) which is below that reported outside the UK. The overall survival was 45% [standard error (SE) ¼ 4%] at 5 years: 40% (SE ¼ 6%) for JMML and 50% (SE ¼ 6%) for other MDS. Significant adverse prognostic factors for JMML were a platelet count < 40 · 10 9 /l, raised fetal haemoglobin, FPC score and age above 2 years at diagnosis, for other MDS only monosomy 7 was significant. To conclude, the incidence of MDS/JMML in children in the UK appears to be lower than that reported outside the UK. This may be either a real difference in incidence or variation in reporting. Monosomy 7 is associated with poor outcome in MDS other than JMML. The prognosis of JMML depends on age, platelet count and fetal haemoglobin.

Donor leukocyte infusion after hematopoietic stem cell transplantation in patients with juvenile myelomonocytic leukemia

“…Patients with monosomy 7 may represent a subset of patients with different biology, although studies showed the cytogenetic abnormalities including monosomy 7 are not predictive of outcome after first HSCT. 7,31 Third, consistent with previous reports in CML, 32,33 occurrence of GVHD correlated with response to DLI, suggesting a substantial overlap between the GVL effect and GVHD occurrence. It is also noteworthy that in this series of JMML, none of the patients given DLI from a matched sibling donor responded.…”

Juvenile myelomonocytic leukemia and chronic myelomonocytic leukemia

“…Poor prognostic factors include (1) age less than 2 years, (2) low platelet count and (3) elevated fetal hemoglobin levels. 16,84 However, the scientific reasons as to why these factors portend a poor prognosis remain a mystery. Left untreated (which rarely occurs anymore), the natural course of JMML is rapidly fatal with 80% of patients surviving o3 years.…”

The International Prognostic Scoring System (IPSS) for childhood myelodysplastic syndrome (MDS) and juvenile myelomonocytic leukemia (JMML)

“…The score has not been applied to other large series of children with MDS mainly because HbF is not routinely evaluated in MDS patients. 4,7 Poor prognosis in JMML has been associated with low platelet count, elevated HbF, and age at diagnosis of 2 years or more, 1,[10][11][12] confirming two of the factors included in the FPC score. 1 In particular, in the EWOG-MDS experience, a platelet count below 33 Â 10 9 /l at presentation is the strongest negative prognostic factor.…”

“…[1][2][3][4][5][6][7] Some patients show a prolonged and stable clinical course without treatment, but most cases will eventually progress. Hematopoietic stem cell transplantation (HSCT) is the treatment of choice, but the outcome following HSCT is jeopardized if disease progression has occurred.…”