YL Lau, SY Ha, GCF Chan, DCK Chiu, ACW Lee

Abstract

Aplastic anaemia is characterised by pancytopenia and bone marrow failure. The treatment of choice for children is matched related bone marrow transplantation. If that is not available, immunosuppressive therapy has resulted in substantial improvement in blood counts. We review our experience in using immunosuppressive therapy for aplastic anaemia in children seen in Queen Mary Hospital from 1990 to 1997. Seven children (4 boys and 3 girls) with median age at diagnosis of 5.8 years (range 1.1 to 8.9 years) received immunosuppressive therapy for their severe aplastic anaemia during this 8-years period. Five were classified as idiopathic, one had evidence of Epstein-Barr virus-associated hepatitis and one Fanconi anaemia. The median level (range) for haemoglobin was 6.8 gm/dl (3.2 to 10.0), absolute neutrophil count (ANC) 0.2 x 10⁹/L (0.04 to 0.69) and platelet count 9 x 10⁹/L (3 to 18). The median follow-up was 3 years (range 1 to 7.8 years). They all received different combinations of immunosuppressive agents including anti-lymphocyte globulin, cyclosporine and steroid. Two patients also received granulocyte colony-stimulating factor. The patient with Fanconi anaemia only had transient response. Of the remaining 6 patients, 4 achieved transfusion-independent status, with 2 of normal or near normal blood counts and 2 of low platelet counts; the other 2 patients with ANC over 1 x 10⁹/L were still platelet dependent. The time to recovery of blood counts was prolonged. The response to immunosuppressive therapy of our patients may not be as prompt and as good as that reported overseas.