重型β-珠蛋白生成障碍性贫血异基因造血干细胞移植后移植物抗宿主病

Graft-Versus-Host-Disease after Allogeneic Stem Cell Transplantation in Severe β-Thalassemia

目的探讨异基因造血干细胞移植治疗重型β-珠蛋白生成障碍性贫血(地贫)并急(慢)性移植物抗宿主病(GVHD)的规律及预防方案。方法分析本院1997-2004年行58次异基因造血干细胞移植52例重型地贫患儿资料。预处理方案以白消安加环磷酰胺加抗人胸腺免疫球蛋白为主,依据人类白细胞抗原(HLA)配型情况等分别加用氟达拉滨或全身放疗。GVHD预防方案:环孢素(CsA)或他克莫司(FK-506)加甲氨蝶呤(MTX)或霉酚酸脂(MMF)。观察比较不同移植类型、骨髓来源等GVHD的发生情况及预后。结果随访1～104个月,GVHD总发生率为72.7%(24例),其中21例为急性GVHD(占87.5%),3例为慢性GVHD(12.5%);GVHD临床分度为Ⅰ、Ⅱ、Ⅲ～Ⅳ度,发生率分别为27.3%(9例)、27.3%(9例)和18.1%(6例)。非亲缘或单倍体供者移植组GVHD发生率为66.7%,外周血造血干细胞移植(PBSCT)者GVHD发生率为100%。结论地贫移植后GVHD发生以急性为主,临床分度以Ⅰ、Ⅱ度为主,非亲缘供者、单倍体移植组GVHD发生率高,尤以PBSCT发生率高;CsA加MMF是比较有效的预防方法。

Objective To study the characteristic of graft - versus - host - disease （GVHD） after allogeneic stem cell transplantation in severe β - thalassemia and evaluate the result of GVHD prophylaxis protocal Methods From 1997 to 2004,52 cases of β - thalassemia were carried out 58 times hematopoiesis stem cells transplantation. Cyclosporin（CsA） was maily used for GVHD prophylaxis, based on it, methotrexate （MTX） or mycophemolate mofeil （MMF） was added to different cases, some cases were used tacrolimus （ FK - 506 ） instead of CsA. Conditioning regimen consisted of Busul phan ＋ cyclophosphamide ＋ Antihuman thymocyte globulin （ATG）, some patients were aided total body irradiation （TBI） or Fludarabine （Flu）. The incidence rate of GVHD and prognosis of different prophylaxis protocols, source of stem cell were compared. Results In the process of monitoring during 1 to 104 months after transplantion,24 cases developed GVHD（21 cases with acute GVHD ,3 cases with chronic GVHD）. The total occurrenle rate was 72.7 percent. Grade Ⅰ and grade Ⅱ GVHD occured in 9 patients（29.0 percent）respectively,grade Ⅲ -Ⅳ GVHD was 19.4 percent,the occured rate of GVHD of group haploid and non -related donor transplant was 66.7 percent, peripheral blood hematopoietic stem cell transplant （PBSCT） group was 100 percent. Conclusions Grade Ⅰ and grade Ⅱ GVHD were usually happened after transplantation. The incidence of GVHD among the unrelated donor transplantation is higher than other tvoe of transplantation.esoeciallv the PBSCT. So far.CsA ＋ MMF is the ideal nronhvlaxis for GVHD at present.