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Aplastic anemia  

Kim, Hack Ki (Department of Pediatrics, College of Medicine, The Catholic University of Korea)
Publication Information
Clinical and Experimental Pediatrics / v.50, no.6, 2007 , pp. 519-523 More about this Journal
Abstract
Aplastic anemia is a rare disease, which is characterized by pancytopenia and hypocellular bone marrow without infiltration of abnormal cells or fibrosis. The incidence in Asia is higher than in the West and new cases are diagnosed at a rate of 5.1 per million pediatric populations per year in Korea. The pathophysiology is understood roughly by defective hematopoiesis, impaired bone marrow micro-environment and immune mechanism. Treatments are performed on basis of pathogenesis and selected depending on the severity. Immunosuppressive therapy with antilymphocyte or antithymocyte globulin and cyclosporine is effective in the majority of patients but has some problems including relapse or clonal evolution. Recently, there have been clinical trials of immunosuppression with hematopoietic growth factors or other drugs. Allogeneic hematopoietic stem cell transplantation (HSCT) is curative in children with severe aplastic anemia. The overall survival in HSCT from HLA-identical sibling is higher than alternative donor, including HLA matched unrelated donor or cord blood. We have to consider quality of life after HSCT because of high survival rate. However, chronic graft versus host disease and graft failure are important factors that affect the quality of life and overall survival. We need further investigation to make new regimens aimed at overcoming these risk factors and perform clinical trials.
Keywords
Aplastic anemia; Immunosuppressive therapy; Allogeneic hematopoietic stem cell transplantation;
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1 Young NS, Calado RT, Scheinberg P. Current concepts in the pathophysiology and treatment of aplastic anemia. Blood 2006;108:2509-19   DOI   ScienceOn
2 Breatnach F, Chessells JM, Creaves MF. The aplastic presentation of childhood leukemia: a feature of common- ALL. Br J Haemtol 1981;49:387-93   DOI   ScienceOn
3 Camitta BM. What is the definition of cure for aplastic anemia? Acta Haematol 2000;103:16-8   DOI   ScienceOn
4 Jeng MR, Naidu PE, Reiman MD, Rodriguez-Galindo C, Nottage KA, Thornton DT, et al. Granulocyte-macrophage colony stimulating factor and immunossupression in the reatment of pediatric acquired aplastic anemia. Pediatr Blood Cancer 2005;45:170-5   DOI   ScienceOn
5 Rosenfeld S, Follmann D, Nunez O, Young NS. Antithymocyte globulin and cyclosporine for severe aplastic anemia. JAMA 2003;289:1130-5   DOI   ScienceOn
6 Hwang WY, Samuel M, Tan D, Koh LP, Lim W, Linn YC. A meta-analysis of unrelated donor umbilical cord blood transplantation versus unrelated donor bone marrow transplantation in adult and pediatric patients. Biol Blood Marrow Transplant. 2007;13:444-53   DOI   ScienceOn
7 Horowitz MM. Current status of allogeneic bone marrow transplantation in acquired aplastic anemia. Semin Hematol 2007;37:30-42
8 Jaime-Perez JC, Ruiz-Arguelles GJ, Gomez-Almaguer D. Haematopoietic stem cell transplantation to treat aplastic anaemia. Expert Opin Biol Ther. 2005;5:617-26   DOI   ScienceOn
9 Takatoku M, Uchiyama T, Okamoto S, Kanakura Y, Sawada K, Tomonaga M, et al. Retrospective nationwide survey of Japanese patients with transfusion-dependent MDS and aplastic anemia highlights the negative impact of iron overload on morbidity/mortality. Eur J Haematol 2007;doi: 10.1111/j.1600-0609.2007.00842
10 Bacigalupo A, Chaple M, Hows J, Van Lint MT, McCann S, Milligan D, et al. Treatment of aplastic anaemia (AA) with antilymphocyte globulin (ALG) and methylprednisolone (MPred) with or without androgens: a randomized trial from the EBMT SAA working party. Br J Haematol 1993;83:145-51   DOI   ScienceOn
11 Kahl C, Leisenring W, Deeg HJ, Chauncey TR, Flowers MED, Martin PJ, et al. Cyclophosphamide and antithymocyte globulin as a conditioning regimen for allogeneic marrow transplantation in patients with aplastic anemia: a long-term follow-up. Br J Haematol 2005;130:747-51   DOI   ScienceOn
12 Speck B, Gluckman E, Haak HL, Van Rood JJ. Treatment of aplastic anemia by antilymphocyte globulin with and without allogeneic bone-marrow infusions. Lancet 1977;2: 1145-8
13 Korbling M, Anderlini P. Peripheral blood stem cell versus bone marrow allotransplantation: does the source of hematopoietic stem cells matter? Blood 2001;98:2900-8.   DOI   ScienceOn
14 Heimpel H. Aplastica anemia before bone marrow transplantation and antilymphocyte globulin. Acta Haematol 2000; 103:11-5   DOI   ScienceOn
15 Pitcher LA, Hann IM, Evans JPM, Veys P, Chessels JM, Webb DKH. Improved prognosis for acquired aplastic anemia. Arch Dis Child 1999;80:158-62   DOI
16 Frickhofen N, Heimpel H, Kaltwasser JP, Schrezenmeier, the 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;101:1236-42   DOI   ScienceOn
17 Kennedy-Nasser AA, Leung KS, Mahajan A, Weiss HL, Arce JA, Gottschalk S, et al. Comparable outcomes of matched-related and alternative donor stem cell transplantation for pediatric severe aplastic anemia. Biol Blood Marrow Transplant 2006;12:1277-84   DOI   ScienceOn
18 Deeg HJ, O'Donnell M, Tolar J, Agarwal R, Harris RE, Feig SA, et al. Optimization of conditioning for marrow transplantation from unrelated donor for patients with aplastic anemia after failure of immunosuppressive therapy. Blood 2006;108:1485-91   DOI   ScienceOn
19 Deeg HJ, Amylon MD, Harris RE, Collins R, Beatty PG, Feig S, et al. Marrow transplants from unrelated donors for patients with aplastic anemia: minimum effective dose of total body irradiation. Biol Blood Marrow Transplant 2001;7:208-15   DOI   ScienceOn
20 Bacigalupo A, Hows J, Gluckman E, Nissen C, Marsh J, Van Lint MT, et al. Bone marrow transplantation (BMT) versus immunosuppression for the treatment of severe aplastic anemia(SAA): a report of the EBMT SAA working party. Br J Haematol 1988;70:177-82   DOI   ScienceOn
21 Geary CG, Harrison CJ, Philpott NJ, Hows JM, Gordon-Smith EC, Marsh JC. Abnormal cytogenetic clones in patients with aplastic anemia: response to immunosuppressive therapy. Br J Haematol 1999;104:271-4   DOI   ScienceOn
22 Fernandes J, Rocha V, Robin M, De Latour RP, Traineau R, Devergie A, et al. Second transplant with two unrelated cord blood units for early graft failure after haematopoietic stem cell transplantation. Br J Haematol 2007;137:248-51   DOI   ScienceOn
23 Jeong DC, Kim HK, Korean Society of Pediatric Hemato-Oncology (KSPHO). Epidemiology and clinical outcome of childhood aplastic anemia in Korea (1991-2005). Clin Pediatr Hematol Oncol, the 26th Spring meeting of the Korean Society of Pediatric Hemato-Oncology; 2006 Apr 14; Seoul.Seoul: The Korean Society of Pediatric Hemato-Oncology, 2006:105-6.
24 Brodsky RA, Jones RJ. Aplastic anemia. Lancet 2005;365: 1647-56   DOI   ScienceOn
25 Kojima S. Aplastic anemia in the Orient. Int J Hematol 2002;76 suppl 2:173-4   DOI
26 Führer M, Rampf U, Baumann I, Faldum A, Niemeyer C, Schaub-Janka G, et al. Immunosuppressive therapy for aplastic anemia in children: a more severe disease predicts better survival. Blood 2005;106:2102-4   DOI   ScienceOn
27 Gupta V, Gordon-Smith EC, Cook G, Parker A, Duguid JKM, Wilson KMO, et al. A third course of anti-thymocyte globulin in aplastic anemia is only beneficial in previous responders. Br J Haematol 2005;129:110-7   DOI   ScienceOn
28 Maciejewski JP, Rivera C, Kook H, Dunn D, Young NS.Relationship between bone marrow failure syndromes and the presence of glycophosphatidyl inositol-anchored proteindeficient clones. Br J Haematol 2001;115:1015-22   DOI   ScienceOn
29 International Agranulocytosis and Aplastic Anemia Study. Incidence of aplastic anemia: relevance of diagnosis criteria. Blood 1987;70:1718-21
30 Davis JK, Guinan EC. An update on the management of severe idopathic aplastic anemia in children. Br J Haematol 2007;136:549-64   DOI   ScienceOn