Unrelated donor marrow transplantation in children with severe aplastic anaemia using cyclophosphamide, anti‐thymocyte globulin and total body irradiation
Unrelated donor marrow transplantation in children with severe aplastic anaemia using cyclophosphamide, anti‐thymocyte globulin and total body irradiation
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使用环磷酰胺、抗胸腺细胞球蛋白和全身照射对患有严重再生障碍性贫血的儿童进行无关供体骨髓移植
DOI：
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发表时间：
2001
影响因子：
6.5
通讯作者：
T. Matsuyama
中科院分区：
文献类型：
--
作者：
S. Kojima;J. Inaba;A. Yoshimi;Y. Takahashi;N. Watanabe;K. Kudo;K. Horibe;N. Maeda;Koji Kato;T. Matsuyama
我们报告了15例严重再生障碍性贫血(SAA)患者，他们年龄20岁，接受了来自人类白细胞抗原( )匹配的非血缘供者的骨髓移植，结果良好。所有患者对强化免疫抑制治疗(IST)均无反应，并多次输血。预适应方案包括环磷酰胺(60 mg/kg/d， −4和−3)、抗胸腺细胞球蛋白(2.5 mg/kg/d，d −5~−2)和全身照射(2.5 × 2/d，d −2和−1)。除后4名患者接受他克莫司替代环孢素A外，其余患者接受环孢素A和甲氨蝶呤预防移植物抗宿主病(GVHD)。经血清学分型，供受者对的HLAI、II类抗原完全相同，但高分辨分型发现有4对在HLAA、-B或-DRB1位点存在不匹配。所有患者均获得快速植入，移植后存活2~86 月(中位随访期51 月)。15例患者中有5例发生中到重度急性移植物抗宿主病(33%)；只有1例患者发展为广泛的慢性移植物抗宿主病。考虑到我们令人鼓舞的结果，无血缘关系的供者移植治疗SAA被推荐作为对IST无反应者的抢救治疗。
We report a favourable outcome in 15 patients with severe aplastic anaemia (SAA) who were < 20 years of age and who underwent bone marrow transplantation (BMT) from a human leucocyte antigen (HLA)‐matched unrelated donor. All patients were non‐responders to intensive immunosuppressive therapy (IST) and were multiply transfused. The conditioning regimen consisted of cyclophosphamide (60 mg/kg/d, on d −4 and −3), anti‐thymocyte globulin (2·5 mg/kg/d, on d −5 to −2) and total body irradiation (2·5 Gy × 2/d, on d −2 and −1). Patients received cyclosporine and methotrexate for prophylaxis of graft‐versus‐host disease (GVHD), except for the last four who received tacrolimus instead of cyclosporine. Donor/recipient pairs were identical for HLA class I and II antigens by serological typing, but four pairs were found to have a mismatch at the HLA‐A, ‐B or ‐DRB1 locus by high‐resolution typing. All patients achieved rapid engraftment and are alive at 2–86 months after transplantation (median follow‐up, 51 months). Moderate to severe acute GVHD occurred in 5 out of 15 patients (33%); only one patient developed extensive chronic GVHD. Considering our encouraging results, unrelated donor transplantation for SAA is recommended as a salvage therapy in non‐responders to IST.
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影响因子：
20.3
作者：
R. Curtis;L. Travis;P. Rowlings;G. Socié;D. Kingma;P. Banks;E. Jaffe;G. Sale;M. Horowitz
通讯作者：
R. Curtis;L. Travis;P. Rowlings;G. Socié;D. Kingma;P. Banks;E. Jaffe;G. Sale;M. Horowitz
影响因子：
20.3
作者：
Deeg, HJ;Socie, G;Storb, R
通讯作者：
Storb, R
影响因子：
20.3
作者：
R. Storb;R. Etzioni;C. Anasetti;F. Appelbaum;Buckner Cd;W. Bensinger;E. Bryant;R. Clift;H. Deeg-H
通讯作者：
R. Storb;R. Etzioni;C. Anasetti;F. Appelbaum;Buckner Cd;W. Bensinger;E. Bryant;R. Clift;H. Deeg-H
影响因子：
20.3
作者：
Richard;-E.;Champlin;Mary;-M.;Horowitz;Dirk;-W.;Van;Bekkum;Bruce;Camitta;Gerald;Elfenbein;Robert;Peter;Gale;Eliane;Gluckman;-A.;Good;Alfred;Rimm;Ciril;Rozman;Bruno;Speck;Mortimer;Bortin
通讯作者：
Richard;-E.;Champlin;Mary;-M.;Horowitz;Dirk;-W.;Van;Bekkum;Bruce;Camitta;Gerald;Elfenbein;Robert;Peter;Gale;Eliane;Gluckman;-A.;Good;Alfred;Rimm;Ciril;Rozman;Bruno;Speck;Mortimer;Bortin
影响因子：
20.3
作者：
H. Deeg;C. Anasetti;E. Petersdorf;R. Storb;K. Doney;J. Hansen;J. Sanders;K. Sullivan;Appelbaum Fr
通讯作者：
H. Deeg;C. Anasetti;E. Petersdorf;R. Storb;K. Doney;J. Hansen;J. Sanders;K. Sullivan;Appelbaum Fr