Bacigalupo A, Hows J, Gluckman E, Nissen C, Marsh J, Van Lint M T, Congiu M, De Planque M M, Ernst P, McCann S
Department of Haematology, Ospedale San Martino, Genova, Italy.
Br J Haematol. 1988 Oct;70(2):177-82. doi: 10.1111/j.1365-2141.1988.tb02460.x.
This is an analysis of 509 patients with severe aplastic anaemia (SAA) treated in Europe between 1981 and 1986; 218 patients were treated by allogeneic bone marrow transplantation (BMT) from HLA identical sibling donors and 291 with immunosuppressive therapy (IS) with antilymphocyte globulin (ALG). The overall actuarial survival was 63% after BMT and 61% after IS therapy at 6 years. All patients fulfilled the criteria of SAA; however, most patients with a neutrophil count of less than 0.2 x 10(9)/l also had infections and haemorrhages. Therefore a further subclassification was defined by pretreatment peripheral blood neutrophil count: very severe aplastic anaemia (vSAA) (less than 0.2 x 10(9)/l neutrophils) and moderately severe aplastic anaemia (mSAA) (0.2-0.5 x 10(9)/l neutrophils). A Cox regression analysis showed that the only significant pre-treatment variables were a low neutrophil count (P = 0.001) and increasing age (P = 0.05). Thus it seemed reasonable to analyse survival data after combined stratification for neutrophils (vSAA versus mSAA) and age (cut off at 20 years). BMT was superior to IS in patients with vSAA under 20 years of age (64% v. 38%; P = 0.01). IS was superior to BMT in patients with mSAA aged 20 or more (82% v. 62%; P = 0.002). The two treatments gave comparable results in young patients with mSAA (BMT = 58%, IS = 62%; P = 0.1), and in older patients with vSAA (BMT = 44%, IS = 43%; P = 0.06). Overall 75/218 and 87/291 patients, given BMT or IS respectively, died. The major cause of failure in BMT patients was graft rejection (n = 22) or problems associated with graft-versus-host disease. For ALG patients the major problem was persistence of the aplasia with haemorrhage (n = 32) or infections (n = 46). This study indicates that over 60% of patients with SAA can be successfully treated with either BMT or IS. Overall survival does not differ in the two groups, though significant differences emerge after stratification for severity of the aplasia and age.
这是一项对1981年至1986年间在欧洲接受治疗的509例重型再生障碍性贫血（SAA）患者的分析；218例患者接受了来自人类白细胞抗原（HLA）相同同胞供者的异基因骨髓移植（BMT），291例患者接受了抗淋巴细胞球蛋白（ALG）免疫抑制治疗（IS）。6年时，BMT后的总体精算生存率为63%，IS治疗后为61%。所有患者均符合SAA标准；然而，大多数中性粒细胞计数低于0.2×10⁹/L的患者也有感染和出血情况。因此，根据预处理外周血中性粒细胞计数进一步进行了亚分类：极重型再生障碍性贫血（vSAA）（中性粒细胞低于0.2×10⁹/L）和中型重型再生障碍性贫血（mSAA）（中性粒细胞0.2 - 0.5×10⁹/L）。一项Cox回归分析表明，唯一显著的预处理变量是低中性粒细胞计数（P = 0.001）和年龄增加（P = 0.05）。因此，对中性粒细胞（vSAA与mSAA）和年龄（以20岁为界）进行联合分层后分析生存数据似乎是合理的。在20岁以下的vSAA患者中，BMT优于IS（64%对38%；P = 0.01）。在20岁及以上的mSAA患者中IS优于BMT（82%对62%；P = 0.002）。在年轻的mSAA患者（BMT = 58%，IS = 62%；P = 0.1）和老年的vSAA患者中（BMT = 44%，IS = 43%；P = 0.06），两种治疗效果相当。总体而言，分别接受BMT或IS治疗的患者中，有75/218和87/291例死亡。BMT患者失败的主要原因是移植物排斥（n = 22）或与移植物抗宿主病相关的问题。对于接受ALG治疗的患者，主要问题是再生障碍持续伴出血（n = 32）或感染（n = 46）。这项研究表明，超过60%的SAA患者可以通过BMT或IS成功治疗。两组的总体生存率没有差异，尽管在根据再生障碍的严重程度和年龄分层后出现了显著差异。
