|
|
|
| Stem cell transplantation for children with acquired severe aplastic anaemia |
| WANG Bin1 WU Runhui1 PANG Yilin2 ZHU Guanghua1 ZHOU Xuan1 YANG Jun1 LI Sidan1 QIN Maoquan1▲ |
1.Hematology Oncology Center, Beijing Children′s Hospital Affiliated to Capital Medical University Beijing Key Laboratory of Hematological Diseases and Tumor Molecular Classification National Key Discipline of Pediatrics, Beijing 100045, China;
2.Department of Emergency, Beijing Children's Hospital Affiliated to Capital Medical University, Beijing 100045, China |
|
|
|
Abstract Objective To explore the effect of hematopoietic stem cell transplantation (HSCT) in the treatment of severe aplastic anemia, and evaluate its safety and prognosis. Methods Clinical data of 51 children with severe aplastic anaemia received HSCT in Hematology Department of Beijing Children′s Hospital Affiliated to Capital Medical University from January 2006 to December 2015 were retrospectively analyzed. The engraftment, complications and curative effect were evaluated. Results 98 percent of patients had rapid engraftment. The median time of neutrophil exceeded 0.5×109/L and platelets recovery exceeded 20×109/L were 11 and 15 days for all transplantation patients. In this research, 10 patients were diagnosed with CMV viremia. 2 patients developed hemorrhagic cystitis. 7 cases had grade Ⅰ-Ⅱ aGVHD and 2 cases had grade Ⅲ aGVHD. 46 patients were still alive. The estimated 3-year OS of the Allo-HSCT, Haplo-HSCT and MUD-HSCT group was 100%, 89% and 75% respectively. All children less than 5 years of age (without infection) were transplanted successfully. Conclusion HSCT is effective in the treatment of acute severe aplastic anemia in children, with fewer complications and high survival rates
|
|
|
|
|
|
[1] Brodsky RA，Jones RJ. Aplastic anaemia [J]. Lancet，2005， 365（9471）：1647-1656.
[2] Bacigalupo A. Treatment strategies for patients with severe aplastic anemia [J]. Bone Marrow Transplant，2008，42（Suppl 1）：S42-S44.
[3] Bacigalupo A. How I treat acquired aplastic anemia [J]. Blood，2017，129（11）：1428-1436.
[4] Passweg JR，Perez W，Eapen M，et al. Bone marrow transplants from mismatched related and unrelated donors for severe aplastic anemia [J]. Bone Marrow Transplant，2006， 37（7）：641-649.
[5] Passweg JR，Baldomero H，Bader P，et al. Hematopoietic stem cell transplantation in Europe 2014：more than 40?000 transplants annually [J]. Bone Marrow Transplant，2016， 51（6）：786-792.
[6] 中华医学会血液学分会红细胞疾病（贫血）学组.再生障碍性贫血诊断治疗专家共识[J].中华血液学杂志，2010， 31（11）：790-792.
[7] Ogawa S. Clonal hematopoiesis in acquired aplastic anemia [J]. Blood，2016，128（3）：337-347.
[8] Passweg JR，Pérez WS，Eapen M，et al. Bone marrow transplants from mismatched related and unrelated donors for severe aplastic anemia [J]. Bone Marrow Transplant，2006， 37（7）：641-649.
[9] Clé DV，Atta EH，Dias DS，et al. Repeat course of rabbit antithymocyte globulin as salvage following initial therapy with rabbit antithymocyte globulin in acquired aplastic anemia [J]. Haematologica，2015，100（9）：e345-e347.
[10] Passweg JR，Tichelli A. Immunosuppressive treatment for aplastic anemia：are we hitting the ceiling? [J]. Haematologica，2009，94（3）：310-312.
[11] Nishikawa E，Yagasaki H，Hama A，et al. Long-term outcomes of 95 children with moderate aplastic anemia treated with horse antithymocyte globulin and cyclosporine [J].Pediatr Blood Cancer，2017，64（5）：doi：10.1002/pbc.26305.
[12] Wang H，Wu Y，Fu R，et al. Granulocyte transfusion combined with granulocyte colony stimulating factor in severe infection patients with severe aplastic anemia：a single center experience from China [J]. PLoS One，2014，9（2）：e88148.
[13] Inamoto Y，Suzuki R，Kuwatsuka Y，et al. Long-term outcome after bone marrow transplantation for aplastic anemia using cyclophosphamide and total lymphoid irradiation as conditioning regimen [J]. Biol Blood Marrow Transplant，2008，14（1）：43-49.
[14] Bacigalupo A，Giammarco S，Sica S. Bone marrow transplantation versus immunosuppressive therapy in patients with acquired severe aplastic anemia [J]. Int J Hematol，2016，104（2）：168-174.
[15] Zhu H，Luo RM，Luan Z，et al. Unmanipulated haploidentical haematopoietic stem cell transplantation for children with severe aplastic anaemia [J]. British J Haematol，2016， 174（5）：799-805.
[16] 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）：11-18.
[17] Maury S，Aljurf M. Adult patients older than 40 years refractory to at least one immunosuppressive course：HLA-identical sibling HSCT using fludarabine-based conditioning [J]. Bone Marrow Transplant，2013，48（2）：196-197.
[18] 陈静.非血缘造血干细胞移植——儿童重型再生障碍性贫血的一线治疗选择[J].实用儿科临床杂志，2012，27（15）：1144-1146.
[19] Xu LP，Zhang XH，Wang FR，et al. Haploidentical transplantation for pediatric patients with acquired severe aplastic anemia [J]. Bone Marrow Transplant，2017，52（3）：381-387.
[20] Xu LP，Wang SQ，Wu DP，et al. Haploidentical transplantation for acquired severe aplastic anaemia in a multicentre prospective study [J]. British J Haematol，2016， 175（2）：265-274.
|
|
|
|
