血液学反应:有中等质量证据表明,抗胸腺细胞球蛋白优于支持性治疗或雄激素。在抗胸腺细胞球蛋白方案中增加环孢素会增强疗效。[20]Marsh J, Schrezenmeier H, Marin P, et al. Prospective randomized multicenter study comparing cyclosporin alone versus the combination of antithymocyte globulin and cyclosporin for treatment of patients with nonsevere aplastic anemia: a report from the European Blood and Marrow Transplant (EBMT) Severe Aplastic Anaemia Working Party. Blood. 1999;93:2191-2195.http://bloodjournal.org/content/93/7/2191.fullhttp://www.ncbi.nlm.nih.gov/pubmed/10090926?tool=bestpractice.com[22]Camitta BM. A controlled prospective trial of antithoracic duct lymphocyte globulin (ATDLG) for treatment of severe aplastic anemia. Prog Clin Biol Res. 1984;148:239-247.http://www.ncbi.nlm.nih.gov/pubmed/6379662?tool=bestpractice.com[23]Champlin R, Ho W, Gale RP. Antithymocyte globulin treatment in patients with aplastic anemia: a prospective randomized trial. N Engl J Med. 1983;308:113-118.http://www.ncbi.nlm.nih.gov/pubmed/6336819?tool=bestpractice.com[24]Frickhofen N, Kaltwasser JP, Schrezenmeier H, et al. Treatment of aplastic anemia with antilymphocyte globulin and methylprednisolone with or without cyclosporine. The German Aplastic Anemia Study Group. N Engl J Med. 1991;324:1297-1304.http://www.ncbi.nlm.nih.gov/pubmed/2017225?tool=bestpractice.com[25]Rosenfeld S, Follmann D, Nuñez O, et al. Antithymocyte globulin and cyclosporine for severe aplastic anemia: association between hematologic response and long-term outcome. JAMA. 2003;289:1130-1135.http://jama.jamanetwork.com/article.aspx?articleid=196101http://www.ncbi.nlm.nih.gov/pubmed/12622583?tool=bestpractice.com在重度或极重度的 AA 患者中,4 个月的应答率为 65%;中位随访>11 年,该方案与 58% 的精算生存率有关。[26]Frickhofen N, Heimpel H, Kaltwasser JP, et al; German Aplastic Anemia Study Group. Antithymocyte globulin with or without cyclosporin A: 11-year follow-up of a randomized trial comparing treatments of aplastic anemia. Blood. 2003;101:1236-1242.http://bloodjournal.org/content/101/4/1236.fullhttp://www.ncbi.nlm.nih.gov/pubmed/12393680?tool=bestpractice.com
系统评价或者受试者>200名的随机对照临床试验(RCT)。
血液学反应:有中等质量证据表明,抗胸腺细胞球蛋白优于支持性治疗或雄激素。在抗胸腺细胞球蛋白方案中增加环孢素会增强疗效。[20]Marsh J, Schrezenmeier H, Marin P, et al. Prospective randomized multicenter study comparing cyclosporin alone versus the combination of antithymocyte globulin and cyclosporin for treatment of patients with nonsevere aplastic anemia: a report from the European Blood and Marrow Transplant (EBMT) Severe Aplastic Anaemia Working Party. Blood. 1999;93:2191-2195.http://bloodjournal.org/content/93/7/2191.fullhttp://www.ncbi.nlm.nih.gov/pubmed/10090926?tool=bestpractice.com[22]Camitta BM. A controlled prospective trial of antithoracic duct lymphocyte globulin (ATDLG) for treatment of severe aplastic anemia. Prog Clin Biol Res. 1984;148:239-247.http://www.ncbi.nlm.nih.gov/pubmed/6379662?tool=bestpractice.com[23]Champlin R, Ho W, Gale RP. Antithymocyte globulin treatment in patients with aplastic anemia: a prospective randomized trial. N Engl J Med. 1983;308:113-118.http://www.ncbi.nlm.nih.gov/pubmed/6336819?tool=bestpractice.com[24]Frickhofen N, Kaltwasser JP, Schrezenmeier H, et al. Treatment of aplastic anemia with antilymphocyte globulin and methylprednisolone with or without cyclosporine. The German Aplastic Anemia Study Group. N Engl J Med. 1991;324:1297-1304.http://www.ncbi.nlm.nih.gov/pubmed/2017225?tool=bestpractice.com[25]Rosenfeld S, Follmann D, Nuñez O, et al. Antithymocyte globulin and cyclosporine for severe aplastic anemia: association between hematologic response and long-term outcome. JAMA. 2003;289:1130-1135.http://jama.jamanetwork.com/article.aspx?articleid=196101http://www.ncbi.nlm.nih.gov/pubmed/12622583?tool=bestpractice.com在重度或极重度的 AA 患者中,4 个月的应答率为 65%;中位随访>11 年,该方案与 58% 的精算生存率有关。[26]Frickhofen N, Heimpel H, Kaltwasser JP, et al; German Aplastic Anemia Study Group. Antithymocyte globulin with or without cyclosporin A: 11-year follow-up of a randomized trial comparing treatments of aplastic anemia. Blood. 2003;101:1236-1242.http://bloodjournal.org/content/101/4/1236.fullhttp://www.ncbi.nlm.nih.gov/pubmed/12393680?tool=bestpractice.com
血液学反应:有中等质量的证据表明,在 AA 患者的血液学反应方面,马 ATG 优于兔 ATG。一项国立卫生研究院的比较兔 ATG 加环孢素和马 ATG 的大型前瞻性随机研究显示,兔 ATG 组的应答率 (35%) 和生存率 (55%) 明显低于马 ATG 组(应答率 62% 和生存率 85%)。[33]Scheinberg P, Nunez O, Weinstein B, et al. Horse versus rabbit antithymocyte globulin in acquired aplastic anemia. N Engl J Med. 2011;365:430-438.http://www.ncbi.nlm.nih.gov/pubmed/21812672?tool=bestpractice.com随后两项较小型的 II 期研究也显示了相似的结果。[34]Marsh J, Socie G, Tichelli A, et al, on behalf of the EBMT Severe Aplastic Anaemia (SAA) Working Party. Prospective phase II pilot study of rabbit antithymocyte globulin with ciclosporin for patients with acquired aplastic anaemia and matched pair analysis with patients treated with horse ATG and ciclosporin. EBMT 2011 physicians' abstracts: no.208. Bone Marrow Transplant. 2011;46(Suppl 1):S30.http://www.nature.com/bmt/journal/v46/n1s/pdf/bmt201147a.pdf[35]Afable MG 2nd, Shaik M, Sugimoto Y, et al. Efficacy of rabbit antithymocyte globulin in severe aplastic anemia. Haematologica. 2011;96:1269-1275.http://www.haematologica.org/content/96/9/1269.fullhttp://www.ncbi.nlm.nih.gov/pubmed/21606164?tool=bestpractice.com
系统评价或者受试者>200名的随机对照临床试验(RCT)。
血液学反应:有中等质量的证据表明,在 AA 患者的血液学反应方面,马 ATG 优于兔 ATG。一项国立卫生研究院的比较兔 ATG 加环孢素和马 ATG 的大型前瞻性随机研究显示,兔 ATG 组的应答率 (35%) 和生存率 (55%) 明显低于马 ATG 组(应答率 62% 和生存率 85%)。[33]Scheinberg P, Nunez O, Weinstein B, et al. Horse versus rabbit antithymocyte globulin in acquired aplastic anemia. N Engl J Med. 2011;365:430-438.http://www.ncbi.nlm.nih.gov/pubmed/21812672?tool=bestpractice.com随后两项较小型的 II 期研究也显示了相似的结果。[34]Marsh J, Socie G, Tichelli A, et al, on behalf of the EBMT Severe Aplastic Anaemia (SAA) Working Party. Prospective phase II pilot study of rabbit antithymocyte globulin with ciclosporin for patients with acquired aplastic anaemia and matched pair analysis with patients treated with horse ATG and ciclosporin. EBMT 2011 physicians' abstracts: no.208. Bone Marrow Transplant. 2011;46(Suppl 1):S30.http://www.nature.com/bmt/journal/v46/n1s/pdf/bmt201147a.pdf[35]Afable MG 2nd, Shaik M, Sugimoto Y, et al. Efficacy of rabbit antithymocyte globulin in severe aplastic anemia. Haematologica. 2011;96:1269-1275.http://www.haematologica.org/content/96/9/1269.fullhttp://www.ncbi.nlm.nih.gov/pubmed/21606164?tool=bestpractice.com
生存率:对于使用匹配的非亲缘供体干细胞移植有质量较差的证据。预期结果表明生存率范围为 61% 至 84%。[28]Bacigalupo A, Locatelli F, Lanino E, et al. Fludarabine, cyclophosphamide and anti-thymocyte globulin for alternative donor transplants in acquired severe aplastic anemia: a report from the EBMT-SAA Working Party. Bone Marrow Transplant. 2005;36:947-950.http://www.ncbi.nlm.nih.gov/pubmed/16205733?tool=bestpractice.com[29]Deeg HJ, O'Donnell M, Tolar J, et al. Optimization of conditioning for marrow transplantation from unrelated donors for patients with aplastic anemia after failure of immunosuppressive therapy. Blood. 2006;108:1485-1491.http://bloodjournal.org/content/108/5/1485.fullhttp://www.ncbi.nlm.nih.gov/pubmed/16684959?tool=bestpractice.com分子水平的 HLA 分型至少可以去掉部分替代供体干细胞移植的副作用。[30]Armand P, Antin JH. Allogeneic stem cell transplantation for aplastic anemia. Biol Blood Marrow Transplant. 2007;13:505-516.http://www.ncbi.nlm.nih.gov/pubmed/17448909?tool=bestpractice.com
系统评价或者受试者>200名的随机对照临床试验(RCT)。
生存率:对于使用匹配的非亲缘供体干细胞移植有质量较差的证据。预期结果表明生存率范围为 61% 至 84%。[28]Bacigalupo A, Locatelli F, Lanino E, et al. Fludarabine, cyclophosphamide and anti-thymocyte globulin for alternative donor transplants in acquired severe aplastic anemia: a report from the EBMT-SAA Working Party. Bone Marrow Transplant. 2005;36:947-950.http://www.ncbi.nlm.nih.gov/pubmed/16205733?tool=bestpractice.com[29]Deeg HJ, O'Donnell M, Tolar J, et al. Optimization of conditioning for marrow transplantation from unrelated donors for patients with aplastic anemia after failure of immunosuppressive therapy. Blood. 2006;108:1485-1491.http://bloodjournal.org/content/108/5/1485.fullhttp://www.ncbi.nlm.nih.gov/pubmed/16684959?tool=bestpractice.com分子水平的 HLA 分型至少可以去掉部分替代供体干细胞移植的副作用。[30]Armand P, Antin JH. Allogeneic stem cell transplantation for aplastic anemia. Biol Blood Marrow Transplant. 2007;13:505-516.http://www.ncbi.nlm.nih.gov/pubmed/17448909?tool=bestpractice.com