Primary myelodysplastic syndrome in children: the clinical experience in 33 cases

Abstract

We describe the clinicomorphological features in 33 cases of primary myelodysplastic syndrome classified according to the FAB classification which presented to a single centre over a 12 year period. Presenting features were typically related to pancytopenia although hepatosplenomegaly and granulocytic sarcomas were far more prevalent than in the adult population. Morphological assessment of the peripheral blood and the bone marrow showed seven patients had refractory anaemia (RA), 13 patients had RA with excess of blasts (RAEB), nine patients had RAEB in transformation (RAEB-t) and four patients had chronic myelomonocytic leukaemia (CMML). The overall mean survival was short (9.9 months) in all the subgroups and the leukaemic transformation rate was high. None of the patients scored 0-1 according to the Bournemouth Scoring System; four patients scored 2 whereas 29 patients scored 3 to 4. We conclude that unlike adults, the myelodysplastic syndromes in children run an aggressive clinical course, irrespective of the FAB subtype, and the pathogenesis of these diseases in paediatric practice warrants scientific scrutiny. Intensive chemotherapy such as the one used in de novo-AML lead to complete remission in some children and these early results suggest that this should be the treatment of choice in paediatric MDS.

Myelodysplastic syndrome in childhood: a retrospective study of 189 patients in Japan

“…ation between SAA and MDS: (1) AA and MDS are two manifestations of the same fundamental injury to stem cells; (2) MDS develops in patients with AA either as a secondary effect of treatment or from continued exposure to the same agent which induced the original marrow failure. Clonal disorders frequently occur in association with congenital AA, and clonal abnormalities were found in 4% of patients with acquired AA at diagnosis.…”

“…MDS occurs predominantly in the elderly. Although several series of MDS in childhood have been reported, [1][2][3][4][5][6][7][8] epidemiological data are very limited, 1,4,8 in part because pediatric MDS is relatively rare, and also because of difficulties in diagnosis and classification. The classification of MDS is based on the morphology of peripheral blood and bone marrow proposed by the French-American-British (FAB) group in 1982.…”

Allogeneic bone marrow transplantation in children with myelodysplastic syndrome or juvenile myelomonocytic leukemia: the Seattle experience

“…Others have also observed that the aggressive chemotherapy used to treat monosomy 7 associated AML in children and young adults results in survival at best in the range of 15%. 6,15,16 Allogeneic marrow transplantation has been the only potentially curative treatment for adult and pediatric patients with MDS. There are few data regarding the outcome of transplantation for children with various types of MDS.…”

“…5 The actual incidence of MDS in children is not known, but is estimated to be between 1 and 3% of childhood leukemias. [6][7][8] In contrast to adult MDS patients, the preleukemic phase in children is usually short with relatively rapid progression to overt leukemia. 6,9 The less aggressive subtypes, refractory anemia (RA) and refractory anemia with ring sideroblasts (RARS), are extremely rare in childhood and the majority of children also have associated chromosome abnormalities, the most frequent of which, monosomy 7, is found in 40-50%.…”

“…[6][7][8] In contrast to adult MDS patients, the preleukemic phase in children is usually short with relatively rapid progression to overt leukemia. 6,9 The less aggressive subtypes, refractory anemia (RA) and refractory anemia with ring sideroblasts (RARS), are extremely rare in childhood and the majority of children also have associated chromosome abnormalities, the most frequent of which, monosomy 7, is found in 40-50%. 10,11 In fact, children with MDS are frequently referred to as either having 'adult type,' monosomy 7 or JMML.…”