The Treatment And Prognosis Of Moderate Aplastic Anemia In Children

Objective In contrast to severe aplastic anemia(SAA),the appropriate management of non-severe aplastic anemia(non-SAA) is unclear because of its individual heterogeneity.Recently,it was reported that more than half of some non-SAA in children with hematopoietic function impaired to some extent progressed to SAA,and these patients were proposed to be classified as moderate aplastic anemia(MAA).The study aimed to review the clinical history,therapeutic effects and outcome of children with MAA,and to explore the risk factors for progression.Patients and Methods 120 children with MAA seen in our institution from February 2003 to June 2004 were retrospectively analyzed.Patients were randomized to receive androgen(groupâ… ) or the combination of androgen and cyclosporine A(groupâ…¡),and both groups were given proprietary Chinese medicines as well.Results 120 cases were included with a median age of 12 years(3～18 years),and the male to female ratio was 1.4:1(male 70 cases,female 50 cases).â‘ The response rate at 3 months,6 months,2 years were 41.5%and 29.4%,55.6%and 53.8%,56.0%and 62.5%respectively in groupâ… and groupâ…¡,and there was no significant difference between the two groups.â‘¡The 3-year overall progression-free survival was 71.8%±6.2%,and there was no significant difference between the two groups.â‘¢Of 86 patients followed up,18 cases(20.9%) progressed to SAA with a media follow-up of 33 months (range,3～68 months),and the progressions occurred at a median of 18 months (range,2-63 months) after diagnosis.â‘£Of the initial haematological factors,hemoglobin level≤50g/L,red blood cell count≤2.5×10¹²/L,platelets count≤20×10⁹ / L,the percentage of erythroid and myeloid cells in bone marrow≤45%,severely hypocellular marrows,absence of megakaryocyte,non-hematopoietic cells dominated marrow fragments,no treatment response at 3 months correlated with progression. Among these factors,the initial platelets count≤20×10⁹/L and severely hypocellular marrows were identified as independent risk factors for progression.Conclusion when children with MAA are treated with androgen and/or cyclosporine A,the therapeutic effects are unsatisfactory compared with the general NSAA ones,and at least 1/5 of them may progress to SAA.The initial platelets count≤20×10⁹/L and severely hypocellular marrows are independent risk factors for progression.Probably those with high risk factors for progression should receive more intensively treatments.