摘要
目的探讨HLA不全相合非血缘脐血移植(CBT)治疗X连锁无丙种球蛋白血症(Bruton病)的疗效;观察其造血及免疫重建过程。方法为1例男性14岁Bruton病患儿行HLA1个位点不相合的非血缘CBT。预处理方案为Bu/Cy(白消安/环磷酰胺)加抗CD3单抗。移植物抗宿主病(GVHD)预防采用环孢菌素A+霉酚酸酯+甲氨蝶呤方案。移植有核细胞数0.42×108/kg,其中CD34+细胞0.35×106/kg。结果移植后30天(+30天)中性粒细胞数0.5×109/L,BPC20×109/L,显示造血重建。+45天性染色体检测46,XX∶46,XY为4∶1。+100天性染色体均为46,XX;受者血型由AB型转为O型(供者型);IgG从1.1g/L升高到3.5g/L;成熟B细胞在外周血中从0上升到占0.05,显示免疫重建。随访360d,患者血常规、骨髓象正常,IgG13.5g/L及成熟B细胞在外周血中占0.10,均为正常水平,未发生急、慢性GVHD,显示造血功能、免疫系统稳定重建。结论本例为首例非血缘CBT治疗Bruton病获得成功;HLA不全相合非血缘CBT治疗非恶性血液病安全、有效。
Objective To evaluate cord blood stem cell transplantation (CBT) in the treatment of X-linked agammaglobulinemia, and observe the courses of the hematopoietic and immune reconstitution. Methods A 14-year-old male patient with agammaglobulinemia received CBT from a 1/6 HLA-mismatched unrelated cord blood. The conditioning regimen was Bu/Cy/anti-CD3 antibody. CsA was given together with MMF and MTX for prophylaxis of GVHD. The patient received 0.42× 10 8 nucleated cells/kg, containing 0.35× 106 CD34 + cells/kg. Results The recipient showed hematopoietic reconstitution on day 30 post-transplantation when ANC was 0.5×10 9/L and BPC 20×109/L. Sex chromosome analysis showed engraftment (donor 46,XX / recipient 46,XY=4∶1) on day 45. The recipient’s blood group changed from AB to O, IgG from 1.1 g/L to 3.5 g/L, sex chromosome from 46,XY to full 46,XX, and mature B cells in peripheral blood from 0 to 5% on day 100, indicating immune reconstitution. At the last follow-up of 360 days, the patient without acute or chronic GVHD showed normal hemogram and myelogram, IgG 13.5 g/L and 10% mature B cells in peripheral blood, indicating the hematopoiesis and immune persistent reconstitution. No acute or chronic GVHD was developed.Conclusion This is the first case report of successful treatment of X-linked agammaglobulinemia by HLA-mismatched unrelated CBT.
出处
《中华血液学杂志》
CAS
CSCD
北大核心
2005年第7期401-403,共3页
Chinese Journal of Hematology
