异基因造血干细胞移植治疗儿童再生障碍性贫血研究进展
|
摘要
<正>再生障碍性贫血(AA)是一种严重威胁儿童生命健康的获得性血液系统疾病,异基因造血干细胞移植(allo-HSCT)有望彻底治愈AA的治疗手段,越来越多的儿童AA患者能够从allo-HSCT中获益~([1-2])。1儿童再生障碍性贫血AA以骨髓增生减低、外周血全血细胞减少为主要临床表现。在欧美等西方国家儿童AA的发病率约为2～3/百万,东方国家AA的发病率比西方国家高
引文
[1]Dufour C,Veys P,Carraro E,et al.Similar outcome of upfront-unrelated and matched sibling stem cell transplantation in idiopathic paediatric aplastic anaemia.A study on behalf of the UK Paediatric BMT Working Party,Paediatric Diseases Working Party and Severe Aplastic Anaemia Working Party of EBMT[J].Br J Haematol,2015,171(4):585-594.
[2]周健,符粤文,梁利杰,等.无关供者与同胞相合供者异基因造血干细胞移植治疗儿童和青少年重型再生障碍性贫血疗效比较[J].中华内科杂志,2016,55(12):927-931.
[3]Barone A,Lucarelli A,Onofrillo D,et al.Diagnosis and management of acquired aplastic anemia in childhood.Guidelines from the Marrow Failure Study Group of the Pediatric Haemato-Oncology Italian Association(AIEOP)[J].Blood Cell Mol Dis,2015,55(1):40-47.
[4]Dufour C,Ferretti E,Bagnasco F,et al.Changes in cytokine profile pre-and post-immunosuppression in acquired aplastic anemia[J].Haematologica,2009,94(12):1743-1747.
[5]Yoshida N,Kojima S.Updated guidelines for the treatment of acquired aplastic anemia in children[J].Curr Oncol Rep,2018,20(9):67.
[6]Maury S,Balère-Appert ML,Chir Z,et al.Unrelated stem cell transplantation for severe acquired aplastic anemia:improved outcome in the era of high-resolution HLA matching between donor and recipient[J].Haematologica,2007,92(5):589-596.
[7]Locasciulli A,Oneto R,Bacigalupo A,et al.Outcome of patients with acquired aplastic anemia given first line bone marrow transplantation or immunosuppressive treatment in the last decade:a report from the European Group for Blood and Marrow Transplantation(EBMT)[J].Haematologica,2007,92(1):11.
[8]Scheinberg P,Wu CO,Nunez O,et al.Long-term outcome of pediatric patients with severe aplastic anemia treated with antithymocyte globulin and cyclosporine[J].J Pediatr,2008,153(6):814-819.
[9]Jeong DC,Chung NG,Cho B,et al.Long-term outcome after immunosuppressive therapy with horse or rabbit antithymocyte globulin and cyclosporine for severe aplastic anemia in children[J].Haematologica,2014,99(4):664.
[10]Ohara A,Kojima S,Hamajima N,et al.Myelodysplastic syndrome and acute myelogenous leukemia as a late clonal complication in children with acquired aplastic anemia[J].Blood,1997,90(3):1009-1013.
[11]SociéG,Henryamar M,Bacigalupo A,et al.Malignant tumors occurring after treatment of aplastic anemia.European Bone Marrow Transplantation-Severe Aplastic Anaemia Working Party[J].N Engl J Med,1993,329(16):1152-1157.
[12]Kojima S,Horibe K,Inaba J,et al.Long-term outcome of acquired aplastic anaemia in children:comparison between immunosuppressive therapy and bone marrow transplantation[J].Br J Haematol,2010,111(1):321-328.
[13]Dufour C,Pillon M,SocièG,et al.Outcome of aplastic anaemia in children.A study by the severe aplastic anaemia and paediatric disease working parties of the European group blood and bone marrow transplant[J].Br J Haematol,2015,169(4):565-573.
[14]Yoshida N,Kobayashi R,Yabe H,et al.First-line treatment for severe aplastic anemia in children:bone marrow transplantation from a matched family donor vs.immunosuppressive therapy[J].Haematologica,2014,99(12):1784-1791.
[15]Yagasaki H,Takahashi Y,Hama A,et al.Comparison of matchedsibling donor BMT and unrelated donor BMT in children and adolescent with acquired severe aplastic anemia[J].Bone Marrow Transplant,2010,45(10):1508-1513.
[16]罗成娟,陈静,薛惠良,等.125例儿童再生障碍性贫血患者疗效分析[J].中华血液学杂志,2010,31(8):531-535.
[17]Zhu H,Luo RM,Luan Z,et al.Unmanipulated haploidentical haematopoietic stem cell transplantation for children with severe aplastic anaemia[J].Br J Haematol,2016,174(5):799-805.
[18]罗荣牡,张晓妹,杜振兰,等.单倍体造血干细胞移植治疗儿童获得性再生障碍性贫血单中心回顾性分析[J].中华儿科杂志,2018,56(7):529-33.
[19]Yoshimi A,Kojima S,Taniguchi S,et al.Unrelated cord blood transplantation for severe aplastic anemia[J].Biol Blood Marrow Transplant,2008,14(9):1057-1063.
[20]Latour R,Purtill D,Ruggeri A,et al.Influence of nucleated cell dose on overall survival of unrelated cord blood transplantation for patients with severe acquired aplastic anemia:a study by eurocord and the aplastic anemia working party of the european group for blood and marrow transplantation[J].Biol Blood Marrow Transplant,2011,17(1):78-85.
[21]Kudo K,Muramatsu H,Narita A,et al.Unrelated cord blood transplantation in aplastic anemia:is anti-thymocyte globulin indispensable for conditioning?[J].Bone Marrow Transplant,2017,52(12):1659-1661.
[2]周健,符粤文,梁利杰,等.无关供者与同胞相合供者异基因造血干细胞移植治疗儿童和青少年重型再生障碍性贫血疗效比较[J].中华内科杂志,2016,55(12):927-931.
[3]Barone A,Lucarelli A,Onofrillo D,et al.Diagnosis and management of acquired aplastic anemia in childhood.Guidelines from the Marrow Failure Study Group of the Pediatric Haemato-Oncology Italian Association(AIEOP)[J].Blood Cell Mol Dis,2015,55(1):40-47.
[4]Dufour C,Ferretti E,Bagnasco F,et al.Changes in cytokine profile pre-and post-immunosuppression in acquired aplastic anemia[J].Haematologica,2009,94(12):1743-1747.
[5]Yoshida N,Kojima S.Updated guidelines for the treatment of acquired aplastic anemia in children[J].Curr Oncol Rep,2018,20(9):67.
[6]Maury S,Balère-Appert ML,Chir Z,et al.Unrelated stem cell transplantation for severe acquired aplastic anemia:improved outcome in the era of high-resolution HLA matching between donor and recipient[J].Haematologica,2007,92(5):589-596.
[7]Locasciulli A,Oneto R,Bacigalupo A,et al.Outcome of patients with acquired aplastic anemia given first line bone marrow transplantation or immunosuppressive treatment in the last decade:a report from the European Group for Blood and Marrow Transplantation(EBMT)[J].Haematologica,2007,92(1):11.
[8]Scheinberg P,Wu CO,Nunez O,et al.Long-term outcome of pediatric patients with severe aplastic anemia treated with antithymocyte globulin and cyclosporine[J].J Pediatr,2008,153(6):814-819.
[9]Jeong DC,Chung NG,Cho B,et al.Long-term outcome after immunosuppressive therapy with horse or rabbit antithymocyte globulin and cyclosporine for severe aplastic anemia in children[J].Haematologica,2014,99(4):664.
[10]Ohara A,Kojima S,Hamajima N,et al.Myelodysplastic syndrome and acute myelogenous leukemia as a late clonal complication in children with acquired aplastic anemia[J].Blood,1997,90(3):1009-1013.
[11]SociéG,Henryamar M,Bacigalupo A,et al.Malignant tumors occurring after treatment of aplastic anemia.European Bone Marrow Transplantation-Severe Aplastic Anaemia Working Party[J].N Engl J Med,1993,329(16):1152-1157.
[12]Kojima S,Horibe K,Inaba J,et al.Long-term outcome of acquired aplastic anaemia in children:comparison between immunosuppressive therapy and bone marrow transplantation[J].Br J Haematol,2010,111(1):321-328.
[13]Dufour C,Pillon M,SocièG,et al.Outcome of aplastic anaemia in children.A study by the severe aplastic anaemia and paediatric disease working parties of the European group blood and bone marrow transplant[J].Br J Haematol,2015,169(4):565-573.
[14]Yoshida N,Kobayashi R,Yabe H,et al.First-line treatment for severe aplastic anemia in children:bone marrow transplantation from a matched family donor vs.immunosuppressive therapy[J].Haematologica,2014,99(12):1784-1791.
[15]Yagasaki H,Takahashi Y,Hama A,et al.Comparison of matchedsibling donor BMT and unrelated donor BMT in children and adolescent with acquired severe aplastic anemia[J].Bone Marrow Transplant,2010,45(10):1508-1513.
[16]罗成娟,陈静,薛惠良,等.125例儿童再生障碍性贫血患者疗效分析[J].中华血液学杂志,2010,31(8):531-535.
[17]Zhu H,Luo RM,Luan Z,et al.Unmanipulated haploidentical haematopoietic stem cell transplantation for children with severe aplastic anaemia[J].Br J Haematol,2016,174(5):799-805.
[18]罗荣牡,张晓妹,杜振兰,等.单倍体造血干细胞移植治疗儿童获得性再生障碍性贫血单中心回顾性分析[J].中华儿科杂志,2018,56(7):529-33.
[19]Yoshimi A,Kojima S,Taniguchi S,et al.Unrelated cord blood transplantation for severe aplastic anemia[J].Biol Blood Marrow Transplant,2008,14(9):1057-1063.
[20]Latour R,Purtill D,Ruggeri A,et al.Influence of nucleated cell dose on overall survival of unrelated cord blood transplantation for patients with severe acquired aplastic anemia:a study by eurocord and the aplastic anemia working party of the european group for blood and marrow transplantation[J].Biol Blood Marrow Transplant,2011,17(1):78-85.
[21]Kudo K,Muramatsu H,Narita A,et al.Unrelated cord blood transplantation in aplastic anemia:is anti-thymocyte globulin indispensable for conditioning?[J].Bone Marrow Transplant,2017,52(12):1659-1661.