Webb D K, Hann I M, Chessells J M
Department of Haematology and Oncology, Hospital for Sick Children, London.
Arch Dis Child. 1991 Jul;66(7):858-61. doi: 10.1136/adc.66.7.858.
Over 15 years, 42 children aged 2-14 years were diagnosed as having acquired aplastic anaemia. Adequate clinical details were available for 38 children who were categorised as very severe (n = 13), severe (n = 16), or nonsevere (n = 9) by the modified Camitta criteria. Treatment varied over the study period. Seven children received a bone marrow allograft from a full match family donor and three a matched unrelated donor transplant after failed treatment with antilymphocyte globulin. The remainder were treated with antilymphocyte globulin (n = 11), antilymphocyte globulin and oxymetholone (n = 4), oxymetholone with or without prednisolone (n = 12), or supportive treatment alone (n = 1). With a minimum follow up of one year since treatment, the five year survival was 70% for bone marrow transplantation with a family donor, 30% for antilymphocyte globulin, and 25% for oxymetholone. All three children with a matched unrelated donor transplant died. The prognosis of acquired aplastic anaemia remains poor for most children and new approaches to treatment are urgently required.
在15年期间，42名2至14岁的儿童被诊断患有获得性再生障碍性贫血。38名儿童有足够的临床细节，根据改良的卡米塔标准，他们被分为极重型（n = 13）、重型（n = 16）或非重型（n = 9）。在研究期间治疗方法各不相同。7名儿童接受了来自全相合家庭供者的骨髓移植，3名儿童在抗淋巴细胞球蛋白治疗失败后接受了匹配的无关供者移植。其余儿童接受抗淋巴细胞球蛋白治疗（n = 11）、抗淋巴细胞球蛋白和羟甲烯龙治疗（n = 4）、羟甲烯龙联合或不联合泼尼松龙治疗（n = 12）或仅接受支持治疗（n = 1）。自治疗后至少随访一年，家庭供者骨髓移植的五年生存率为70%，抗淋巴细胞球蛋白治疗为30%，羟甲烯龙治疗为25%。3名接受匹配无关供者移植的儿童均死亡。大多数儿童获得性再生障碍性贫血的预后仍然很差，迫切需要新的治疗方法。
