Comparison Of Long-Term Outcomes Between Children With Aplastic Anemia And Refractory Cytopenia Of Childhood Who Received Immunosuppressive Therapy With Antithymocyte Globulin And Cyclosporine

Comparison Of Long-Term Outcomes Between Children With Aplastic Anemia And Refractory Cytopenia Of Childhood Who Received Immunosuppressive Therapy With Antithymocyte Globulin And Cyclosporine

Hama A (2015) Haematologica  (Link to Abstract)            


The 2008 World Health Organization classification proposed a new entity in childhood myelodysplastic syndrome, refractory cytopenia of childhood (RCC). However, it is unclear whether this morphological classification reflects clinical outcomes. This study is a review on the outcome among 186 children who had received immunosuppressive therapy (IST, horse ATG / cyclosporine) for acquired aplastic anemia. Patients were divided into 3 groups after re-classification by bone marrow pathology: aplastic anemia (AA), refractory cytopenia of childhood (RCC), refractory cytopenia with multilineage dysplasia (RCMD). The AA group had significantly lower leukocyte/neutrophil/reticulocyte/platelet counts compared to the RCC and RCMD groups. No difference in response rates to IST among the 3 groups was seen. Cumulative incidence of total clonal evolution showed no significant difference among the groups. However incidence of development of monosomy 7 was higher in the AA group. Longer duration of G-CSF administration was associated with development of monosomy 7 in AA patients. 10-year failure-free survival did not differ significantly among the 3 groups. 10-year overall survival was significantly lower in the AA group (85%) vs. the RCC group (97%) and the RCMD group (100%).


* The 3 groups did not appear to be distinctly different in terms of biology[NW1]  and outcome in this study. Clonal evolution is still seen in the AA group. This hints that the current morphological classification may not be reliable enough to distinguish aplastic anemia from hypocellular MDS.