Ommati L V M, Rodrigues C A, Silva A R, Silva L P, Chaufaille M L L F, Oliveira J S R
Disciplina de Hematologia e Hemoterapia, Departamento de Oncologia Clínica e Experimental, Escola Paulista de Medicina, Universidade Federal de São Paulo, São Paulo, SP, Brasil.
Braz J Med Biol Res. 2009 Mar;42(3):244-50. doi: 10.1590/s0100-879x2009000300005.
Allogeneic hematopoietic stem cell transplantation (AHSCT) is the treatment of choice for young patients with severe aplastic anemia (SAA). The association of antithymocyte globulin (ATG) and cyclophosphamide (CY) is the most frequently used conditioning regimen for this disease. We performed this retrospective study in order to compare the outcomes of HLA-matched sibling donor AHSCT in 41 patients with SAA receiving cyclophosphamide plus ATG (ATG-CY, N = 17) or cyclophosphamide plus busulfan (BU-CY, N = 24). The substitution of BU for ATG was motivated by the high cost of ATG. There were no differences in the clinical features between the two groups, including age, gender, cytomegalovirus status, ABO match, interval between diagnosis and transplant, and number of total nucleated cells infused. No differences were observed in the time to neutrophil and platelet engraftment, or in the risk of veno-occlusive disease and hemorrhage. However, there was a higher risk of mucositis in the BU-CY group (71 vs 24%, P = 0.004). There were no differences in the incidence of neutrophil and platelet engraftment, acute and chronic graft-versus-host disease, and transplant-related mortality. There was a higher incidence of late rejection in the ATG-CY group (41 vs 4%, P = 0.009). Although the ATG-CY group had a longer follow-up (101 months) than the BU-CY group (67 months, P = 0.04), overall survival was similar between the groups (69 vs 58%, respectively, P = 0.32). We conclude that the association BU-CY is a feasible option to the conventional ATG-CY regimen in this population.
异基因造血干细胞移植（AHSCT）是年轻重型再生障碍性贫血（SAA）患者的首选治疗方法。抗胸腺细胞球蛋白（ATG）与环磷酰胺（CY）联合是该疾病最常用的预处理方案。我们进行这项回顾性研究，以比较41例接受环磷酰胺加ATG（ATG-CY，N = 17）或环磷酰胺加白消安（BU-CY，N = 24）的SAA患者中，HLA匹配同胞供体AHSCT的结局。用BU替代ATG是因为ATG成本高昂。两组患者的临床特征无差异，包括年龄、性别、巨细胞病毒状态、ABO血型匹配、诊断至移植的间隔时间以及输注的总核细胞数。在中性粒细胞和血小板植入时间、静脉闭塞性疾病和出血风险方面未观察到差异。然而，BU-CY组黏膜炎风险更高（71% 对24%，P = 0.004）。中性粒细胞和血小板植入率、急慢性移植物抗宿主病以及移植相关死亡率无差异。ATG-CY组晚期排斥发生率更高（41% 对4%，P = 0.009）。尽管ATG-CY组随访时间（101个月）比BU-CY组长（67个月，P = 0.04），但两组总体生存率相似（分别为69% 对58%，P = 0.32）。我们得出结论，在该人群中，BU-CY联合方案是传统ATG-CY方案的可行选择。
