中华器官移植杂志
中華器官移植雜誌
중화기관이식잡지
CHINESE JOURNAL OF ORGAN TRANSPLANTATION
2011年
3期
144-147
,共4页
刘容容%李桥川%章忠明%罗建明%陈炜%施玲玲%赖颖晖%姚奕斌%周贻振%赖永榕
劉容容%李橋川%章忠明%囉建明%陳煒%施玲玲%賴穎暉%姚奕斌%週貽振%賴永榕
류용용%리교천%장충명%라건명%진위%시령령%뢰영휘%요혁빈%주이진%뢰영용
造血干细胞移植%异基因%珠蛋白生成障碍性贫血%临床疗效
造血榦細胞移植%異基因%珠蛋白生成障礙性貧血%臨床療效
조혈간세포이식%이기인%주단백생성장애성빈혈%림상료효
Hematopoietic stem cell transplantation%Allogene%Thalassemia%Treatment outcome
目的 探讨异基因造血干细胞移植(allo-HSCT)治疗重型β珠蛋白生成障碍性贫血的临床疗效.方法 PesaroⅡ-Ⅲ度重型β珠蛋白生成障碍性贫血患者24例接受allo-HSCT治疗,其中男性18例,女性6例,患者年龄中位数为4岁(2～15岁).24例中,同胞供者23例,母亲供者1例;HLA 6个抗原全相合23例,5个抗原相合1例;骨髓混合外周血干细胞移植15例,脐带血移植9例.采用白消安+环磷酰胺+氟达拉滨的预处理方案.环孢素A(CsA)+甲氨蝶呤(MTX)+抗胸腺细胞球蛋白(ATG)+吗替麦考酚酯(MMF)联用预防移植物抗宿主病(GVHD).中位随访时间13个月(3～69个月).结果 移植后22例患者造血功能顺利恢复.至随访结束,无病存活21例;移植相关死亡1例(4.2%);移植物排斥反应2例(8.3%).21例的3年无病存活率为87.5%,3年总体存活率为91.7%.Ⅱ-Ⅳ度急性GVHD的累积发生率为16.7%,慢性GVHD的累积发生率为20.3%,广泛性慢性GVHD的发生率为5.0%.结论 异基因骨髓混合外周血干细胞移植治疗珠蛋白生成障碍性贫血可获得确切疗效,同时脐带血是珠蛋白生成障碍性贫血移植的重要干细胞来源.CsA+MTX+ATG+小剂量、短疗程MMF的方案可以有效地减少严重急性GVHD的发生,提高移植疗效.
目的 探討異基因造血榦細胞移植(allo-HSCT)治療重型β珠蛋白生成障礙性貧血的臨床療效.方法 PesaroⅡ-Ⅲ度重型β珠蛋白生成障礙性貧血患者24例接受allo-HSCT治療,其中男性18例,女性6例,患者年齡中位數為4歲(2～15歲).24例中,同胞供者23例,母親供者1例;HLA 6箇抗原全相閤23例,5箇抗原相閤1例;骨髓混閤外週血榦細胞移植15例,臍帶血移植9例.採用白消安+環燐酰胺+氟達拉濱的預處理方案.環孢素A(CsA)+甲氨蝶呤(MTX)+抗胸腺細胞毬蛋白(ATG)+嗎替麥攷酚酯(MMF)聯用預防移植物抗宿主病(GVHD).中位隨訪時間13箇月(3～69箇月).結果 移植後22例患者造血功能順利恢複.至隨訪結束,無病存活21例;移植相關死亡1例(4.2%);移植物排斥反應2例(8.3%).21例的3年無病存活率為87.5%,3年總體存活率為91.7%.Ⅱ-Ⅳ度急性GVHD的纍積髮生率為16.7%,慢性GVHD的纍積髮生率為20.3%,廣汎性慢性GVHD的髮生率為5.0%.結論 異基因骨髓混閤外週血榦細胞移植治療珠蛋白生成障礙性貧血可穫得確切療效,同時臍帶血是珠蛋白生成障礙性貧血移植的重要榦細胞來源.CsA+MTX+ATG+小劑量、短療程MMF的方案可以有效地減少嚴重急性GVHD的髮生,提高移植療效.
목적 탐토이기인조혈간세포이식(allo-HSCT)치료중형β주단백생성장애성빈혈적림상료효.방법 PesaroⅡ-Ⅲ도중형β주단백생성장애성빈혈환자24례접수allo-HSCT치료,기중남성18례,녀성6례,환자년령중위수위4세(2～15세).24례중,동포공자23례,모친공자1례;HLA 6개항원전상합23례,5개항원상합1례;골수혼합외주혈간세포이식15례,제대혈이식9례.채용백소안+배린선알+불체랍빈적예처리방안.배포소A(CsA)+갑안접령(MTX)+항흉선세포구단백(ATG)+마체맥고분지(MMF)련용예방이식물항숙주병(GVHD).중위수방시간13개월(3～69개월).결과 이식후22례환자조혈공능순리회복.지수방결속,무병존활21례;이식상관사망1례(4.2%);이식물배척반응2례(8.3%).21례적3년무병존활솔위87.5%,3년총체존활솔위91.7%.Ⅱ-Ⅳ도급성GVHD적루적발생솔위16.7%,만성GVHD적루적발생솔위20.3%,엄범성만성GVHD적발생솔위5.0%.결론 이기인골수혼합외주혈간세포이식치료주단백생성장애성빈혈가획득학절료효,동시제대혈시주단백생성장애성빈혈이식적중요간세포래원.CsA+MTX+ATG+소제량、단료정MMF적방안가이유효지감소엄중급성GVHD적발생,제고이식료효.
Objective To investigate the effect of allgeneic hematopoietic stem cell transplantation (allo-HSCT) for β-thalassemia major. Methods Twenty-four β-thalassemia major patients with median age of 4 years (range: 2～15 years), 18 boys and 6 girls, received allo-HSCT.They were classified into class Ⅱ-Ⅲ according to Pesaro thalassemia classification. Twenty-three transplantations were from sibling donor and 1 was from mother, either HLA-identical (n = 23) or HLA-mismatched (5/6) (n = 1). Fifteen patients received bone marrow transplantation (BMT) plus peripheral blood stem cell transplantation (PBSCT), and 9 were subjected to umbilical cord blood transplantation (UCBT). The conditioning regimen consisted of busalphan, cyclophosphamide,fludarabine, plus hydroxyurea before transplantation. Graft-versus-host disease (GVHD) prophylaxis included CsA, methotrexate, antilymphpcute globulin, and mycophenolate mofetil. The median follow-up period was 13 months (range: 3～69). Results Of 24 patients, there were 21 cases (87. 5 %) of disease-free survival, 1 (4. 2 %) transplantation-related death, and 2 cases (8. 3 %) of rejection. Three-year overall survival and disease-free survival rate was 91.7 % and 87. 5 %respectively. The cumulative incidence of grade Ⅱ -Ⅳ acute GVHD and chronic GVHD was 16. 7 %and 20. 3 %, particularly cumulative extensive chronic GVHD was 5. 0 %. Conclusion The sibling donor BMT plus PBSCT is an effective and safe way to treat β-thalassemia major. Cord blood is an important source of hematopoietic stem cells for HSCT. The protocol GVHD prophylaxis of CsA,MTX, ATG with a low-dose and short course of MMF can effectively reduce the incidence of severe acute GVHD, improve the outcome of thalassemia transplantation.
