Fred Hutchinson Cancer Research Center, Seattle, WA 98109-1024, USA.
Br J Haematol. 2012 Jul;158(1):120-8. doi: 10.1111/j.1365-2141.2012.09130.x. Epub 2012 Apr 26.
Allogeneic marrow transplantation offers curative therapy for children with severe aplastic anaemia (SAA). We report the outcomes of 148 children with SAA who received human leucocyte antigen (HLA)-matched related marrow grafts between 1971 and 2010. Patients were divided into three groups, reflecting changes in conditioning and graft-versus-host disease (GVHD) prophylaxis regimens that occurred over time. Patients in Group 1 were conditioned with cyclophosphamide (CY; 200 mg/kg) followed by 'long' (102 d) methotrexate (MTX). Patients in Groups 2 and 3 received CY alone (Group 2) or combined with anti-thymocyte globulin (Group 3) followed by 'short' (days 1, 3, 6, and 11) MTX and ciclosporin (until day 180). With a median follow-up of 25 years, the 5-year survivals were 66%, 95%, and 100% for Groups 1, 2, and 3, respectively (overall P < 0·0001). The 3-year estimates of graft rejection were 22%, 32%, and 7%, respectively. The probabilities of grades III-IV acute and 2-year chronic GVHD were 15%, 0%, and 3%, and 21%, 21%, and 10%, respectively. Advances in preparative and GVHD prophylaxis regimens, and supportive care during the past 40 years have led to improved outcomes for children with SAA. These results confirm the use of allogeneic marrow transplantation for children with SAA who have HLA-matched related donors.
同种异体骨髓移植为严重再生障碍性贫血（SAA）患儿提供了治愈疗法。我们报告了 1971 年至 2010 年间 148 例 SAA 患儿接受人类白细胞抗原（HLA）匹配相关骨髓移植的结果。患者分为三组，反映了随时间推移而发生的调理和移植物抗宿主病（GVHD）预防方案的变化。第 1 组患者用环磷酰胺（CY；200mg/kg）预处理，随后用“长”（102 天）甲氨蝶呤（MTX）；第 2 组和第 3 组患者仅用 CY（第 2 组）或用抗胸腺细胞球蛋白（第 3 组）联合 CY 预处理，随后用“短”（第 1、3、6 和 11 天）MTX 和环孢素（直至第 180 天）。中位随访 25 年后，第 1、2 和 3 组的 5 年生存率分别为 66%、95%和 100%（总体 P<0·0001）。第 3 年估计的移植物排斥率分别为 22%、32%和 7%。3 级和 4 级急性和 2 年慢性 GVHD 的概率分别为 15%、0%和 3%，21%、21%和 10%。过去 40 年来，在调理和 GVHD 预防方案以及支持性治疗方面的进展，使 SAA 患儿的预后得到改善。这些结果证实了同种异体骨髓移植可用于有 HLA 匹配相关供体的 SAA 患儿。
