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.

再生障礙性貧血表現為各類血細胞減少和骨髓造血功能障礙。骨髓移植為治療兒童再生障礙性貧血的理想選擇，如不能提供骨髓移植治療，免疫抑制療法對該症血細胞計數亦有實質性的幫助。本文對瑪麗醫院1990-1997年間7例嚴重再生障礙性貧血進行免疫抑制療法的經驗作一總結。其中男4例、女3例，平均診斷年齡為5.8歲(1.1-8.9歲)。特發性再生障礙性貧血5例，伴EB病毒性肝炎、范可尼綜合症(先天性再生障礙性貧血)各1例。血紅蛋白平均值為6.8 ng/dl (3.2-10.0 ng/dl)，中性粒細胞絕對值為0.2 x 10⁹/L (0.04-0.69 x 10⁹/L)，血小板為9 x 10⁹/L (3-18 x 10⁹/L)。平均隨訪3年(1-7.8年)。7例患兒皆接受各種不同免疫抑制藥物聯合治療，包括抗淋巴細胞球蛋白，環胞霉素和類固醇。2例尚接受粒細胞克隆刺激因子治療。范可尼綜合症患兒僅獲暫時性緩解。其餘6例，4例達到不輸血狀態(其中2例血細胞計數達到或接近正常，另2例僅血小板數目低下)；其它2例中性粒細胞絕對值大於1 x 10⁹/L，但仍依賴於血小板輸入。血細胞計數恢復時間延長。本症患兒對免疫抑制的療效不及國外類似報導。