Favorable preliminary results using TLI/ATG-based immunomodulatory conditioning for matched unrelated donor allogeneic hematopoietic stem cell transplantation in pediatric severe aplastic anemia.
Favorable preliminary results using TLI/ATG-based immunomodulatory conditioning for matched unrelated donor allogeneic hematopoietic stem cell transplantation in pediatric severe aplastic anemia.
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DOI：
10.1111/j.1399-3046.2011.01542.x
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发表时间：
2011-09
影响因子：
1.3
通讯作者：
Leung W
中科院分区：
文献类型：
--
作者：
Pillai A;Hartford C;Wang C;Pei D;Yang J;Srinivasan A;Triplett B;Dallas M;Leung W
为了评估耐受诱导方案是否可以应用于重型再生障碍性贫血(SAA)的无关(MUD)骨髓移植，我们回顾了我们的HCT经验，使用的是来自匹配同胞供者(MSD)和MUD供者的未经处理的10/10相合的骨髓移植。MSD组(n=9)用环磷酰胺200 mg/kg(CTX)+兔抗胸腺细胞球蛋白(ATG)10 mg/kg；MHD组(n=5)用全淋巴照射(TLI)+CTX/ATG。免疫预防采用环孢素A(CsA)和短程甲氨蝶呤。患者的中位年龄为14.7岁，至HCT的中位时间为1.5年，中位随访时间为3.0年。结果测量包括MSD和MUD队列的无事件生存(EFS)、植入时间和GVHD(GVHD的CIN)的累积发生率。EFS和稳定植入率均为100%。急性移植物抗宿主病的CIN：MSD I-II级1例(11%)，III-IV级0%，MUD I-II级1例(20%)，III-IV级1例(20%)。慢性移植物抗宿主病的CIN为：MSD，局限性：1(11%)，广泛性：0%；MUD，局限性：0%，广泛性：0%。所有免疫抑制依从性的患者都成功地摆脱了免疫抑制。虽然在有限的患者中，我们的结果表明，在主干CTX/ATG条件反射的基础上加用免疫调节性TLI是治疗SAA患者MUD HCT的一种有前途的选择，我们将在前瞻性临床试验中对其进行检验。
To assess whether a tolerance-induction regimen could be applied for unrelated (MUD) HCT in severe aplastic anemia (SAA), we retrospectively reviewed our HCT experience using unmanipulated 10/10 HLA-matched bone marrow grafts from matched sibling donors (MSD) versus MUD donors. Conditioning was cyclophosphamide 200 mg/kg (CTX) + rabbit anti-thymocyte globulin 10 mg/kg (ATG) for MSD (n = 9) and total lymphoid irradiation (TLI, 800 cGy) + CTX/ATG for MUD HCT (n = 5). Immunoprophylaxis was cyclosporine A (CSA) and short-course methotrexate. Median patient age was 14.7 years, median time to HCT 1.5 years, and median follow-up 3.0 years. Outcome measures included event-free survival (EFS), time to engraftment, and cumulative incidence of GVHD (CIN of GVHD) for MSD and MUD cohorts. EFS and stable engraftment rate were 100%. CIN of acute GVHD was: MSD, Grade I-II: 1 (11%), Grade III-IV: 0%; MUD, Grade I-II: 1 (20%), Grade III-IV: 1 (20%). CIN of chronic GVHD was: MSD, limited: 1 (11%), extensive: 0%; MUD, limited: 0%, extensive: 0%. All immunosuppressive-compliant patients successfully weaned immunosuppression. Though in limited patients, our results suggest that immunomodulatory TLI added to backbone CTX/ATG conditioning is a promising option for MUD HCT in SAA patients which we will examine in a prospective clinical trial.
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DOI：
10.3324/haematol.10075
发表时间：
2007-01-01
期刊：
HAEMATOLOGICA-THE HEMATOLOGY JOURNAL
影响因子：
--
作者：
Locasciulli, Anna;Oneto, Rosi;Fuhrer, Monika
通讯作者：
Fuhrer, Monika
影响因子：
158.5
作者：
Lowsky, R;Takahashi, T;Strober, S
通讯作者：
Strober, S
影响因子：
158.5
作者：
KERNAN, NA;BARTSCH, G;BLUME, KG
通讯作者：
BLUME, KG
供体型CD4（+）CD25（+）调节性T细胞在同种异体骨髓移植后抑制致命的急性移植物抗宿主疾病。
影响因子：
15.3
作者：
Hoffmann, Petra;Ermann, Joerg;Edinger, Matthias;Fathman, C Garrison;Strober, Samuel
通讯作者：
Strober, Samuel
影响因子：
8.9
作者：
Chin, C;Hunt, S;Bernstein, D
通讯作者：
Bernstein, D