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

Abstract

We report a retrospective analysis of children with myelodysplastic syndrome (MDS) diagnosed between 1990 and 1997 in Japan. In total, 189 patients were enrolled: 122 cases of primary MDS (26 RA, 18 RAEB, 25 RAEBt, 53 CMML/JMML), 24 cases with constitutional predisposition to MDS, and 43 cases of therapy-related MDS (t-MDS). The frequency of pediatric MDS was estimated to be 7.7% of all leukemias. Cytogenetic abnormalities were observed in 41% of primary MDS and 90% of t-MDS cases. The 4-year survival rate, estimated by Kaplan-Meier analysis, for primary RA was 78.9%, while other types of MDS and JMML had rates lower than 40%, and t-MDS showed an even more unfavorable prognosis. In primary MDS, the survival rate of patients with cytogenetic abnormalities was significantly lower. Among prognostic variables by IPSS, only the cytogenetic pattern was useful for predicting outcome in childhood MDS. There was no apparent advantage to chemotherapy for RA, and the survival rate in patients with primary RA, JMML, or t-MDS receiving stem cell transplantation was significantly higher. More precise designs of our diagnostic and classification systems, as well as therapeutic trials in large-scale prospective studies, are necessary for further improvements in MDS outcome. Leukemia (2001) 15, 1713-1720.

Hematopoietic stem cell transplantation for advanced myelodysplastic syndrome in children: results of the EWOG-MDS 98 study

“…Children with MDS treated on AML protocols have been reported to experience a high rate of induction failure and relapse, resulting in a probability of OS of approximately 30%. 6,7,[27][28][29] Likewise, patients receiving allogeneic HSCT as primary treatment have a considerable risk of relapse, as well as transplantation-related toxicity. 6,9,11 Therefore, the role of intensive chemotherapy before HSCT for patients with advanced MDS has remained a matter of debate.…”

“…Conventional AML-type chemotherapy without HSCT resulted in survival rates below 30%. 6,7 In contrast, it has been shown that a large proportion of children with advanced MDS can be successfully treated with HSCT. [7][8][9][10][11] However, all previously published studies include patients with a wide spectrum of diagnoses, ranging from refractory cytopenia to more advanced MDS and juvenile myelomonocytic leukemia, transplanted following variable conditioning regimens.…”

A pediatric approach to the WHO classification of myelodysplastic and myeloproliferative diseases

“…The WHO classifications recognizes JMML as a separate entity. There are many differences between MDS in children and adults, eg RARS is exceedingly rare in children, [1][2][3][4][5][6] deletion 5q has occasionally been observed in children [12][13][14] but the 5q− syndrome characterized by 5q− as the sole abnormality, BM blasts Ͻ5%, normal or elevated platelet count, an indolent course and long survival has not been described in children. Constitutional abnormalities are often observed in children with MDS and JMML.…”

“…Constitutional abnormalities are often observed in children with MDS and JMML. [1][2][3][4][5][6] The unique features of Down syndrome is not appropriately addressed in the WHO classification. There are no data to indicate whether the blast threshold of 20% is better than the traditional 30% to distinguish MDS from AML in children.…”

“…Myelodysplastic syndrome (MDS) in children and adolescents has hitherto received sparse attention in the pediatric literature and, despite recent attempts, [1][2][3][4][5][6] there has been no generally agreed system of classification or diagnosis. Recommendations for the classification of childhood cancer do not even mention MDS.…”