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REFERENCES Young NS, Bacigalupo A, Marsh JC. Aplastic anemia: pathophysiology and treatment. Biol Blood Marrow Transplant 2010; 16:S119. Guinan EC. Acquired aplastic anemia in childhood. Hematol Oncol Clin North Am 2009; 23:171. Young NS, Maciejewski J. The pathophysiology of acquired aplastic anemia. N Engl J Med 1997; 336:1365. Davies JK, Guinan EC. An update on the management of severe idiopathic aplastic anaemia in children. Br J Haematol 2007; 136:549. Korthof ET, Békássy AN, Hussein AA. Management of acquired aplastic anemia in children. Bone Marrow Transplant 2013; 48:191. Teo JT, Klaassen R, Fernandez CV, et al. Clinical and genetic analysis of unclassifiable inherited bone marrow failure syndromes. Pediatrics 2008; 122:e139. Goss JA, Schiller GJ, Martin P, et al. Aplastic anemia complicating orthotopic liver transplantation. Hepatology 1997; 26:865. Walport MJ, Hubbard WN, Hughes GR. Reversal of aplastic anaemia secondary to systemic lupus erythematosus by high-dose intravenous cyclophosphamide. Br Med J (Clin Res Ed) 1982; 285:769. Nissen C. The pathophysiology of aplastic anemia. Semin Hematol 1991; 28:313. Führer M, Rampf U, Baumann I, et al. Immunosuppressive therapy for aplastic anemia in children: a more severe disease predicts better survival. Blood 2005; 106:2102. Shimamura A, Guinan EA. Acquired aplastic anemia. In: Hematology of Infancy and Childhood, Nathan DG, Orkin SH (Eds), WB Saunders, Philadelphia 2003. p.256. Saracco P, Quarello P, Iori AP, et al. Cyclosporin A response and dependence in children with acquired aplastic anaemia: a multicentre retrospective study with long-term observation follow-up. Br J Haematol 2008; 140:197. Pongtanakul B, Das PK, Charpentier K, Dror Y. Outcome of children with aplastic anemia treated with immunosuppressive therapy. Pediatr Blood Cancer 2008; 50:52. Young NS, Kaufman DW. The epidemiology of acquired aplastic anemia. Haematologica 2008; 93:489. Young NS. Aplastic anaemia. Lancet 1995; 346:228. LANGE RD, WRIGHT SW, TOMONAGA M, et al. Refractory anemia occurring in survivors of the atomic bombing in Nagasaki, Japan. Blood 1955; 10:312. Muir KR, Chilvers CE, Harriss C, et al. The role of occupational and environmental exposures in the aetiology of acquired severe aplastic anaemia: a case control investigation. Br J Haematol 2003; 123:906.
LANGE RD, WRIGHT SW, TOMONAGA M, et al. Refractory anemia occurring in survivors of the atomic bombing in Nagasaki, Japan. Blood 1955; 10:312. Muir KR, Chilvers CE, Harriss C, et al. The role of occupational and environmental exposures in the aetiology of acquired severe aplastic anaemia: a case control investigation. Br J Haematol 2003; 123:906. Guiguet M, Baumelou E, Mary JY. A case-control study of aplastic anaemia: occupational exposures. The French Cooperative Group for Epidemiological Study of Aplastic Anaemia. Int J Epidemiol 1995; 24:993. Wallerstein RO, Condit PK, Kasper CK, et al. Statewide study of chloramphenicol therapy and fatal aplastic anemia. JAMA 1969; 208:2045. Kay AG. Myelotoxicity of gold. Br Med J 1976; 1:1266. Brodie MJ, Pellock JM. Taming the brain storms: felbamate updated. Lancet 1995; 346:918. Laporte JR, Ibáñez L, Ballarín E, et al. Fatal aplastic anaemia associated with nifedipine. Lancet 1998; 352:619. Handoko KB, Souverein PC, van Staa TP, et al. Risk of aplastic anemia in patients using antiepileptic drugs. Epilepsia 2006; 47:1232. SCOTT JL, CARTWRIGHT GE, WINTROBE MM. Acquired aplastic anemia: an analysis of thirty-nine cases and review of the pertinent literature. Medicine (Baltimore) 1959; 38:119. Powars D. Aplastic anemia secondary to glue sniffing. N Engl J Med 1965; 273:700. Young NS. Drugs and Chemicals. In: Aplastic Anemia: Acquired and Inherited, Young NS, Alter BP (Eds), WB Saunders, Philadelphia 1994. p.100. LOGE JP. INSECT-STING ALLERGY: QUESTIONNAIRE STUDY OF 2,606 CASES. JAMA 1965; 193:115. Lee KA, Kim SH, Woo HY, et al. Increased frequencies of glutathione S-transferase (GSTM1 and GSTT1) gene deletions in Korean patients with acquired aplastic anemia. Blood 2001; 98:3483. Sutton JF, Stacey M, Kearns WG, et al. Increased risk for aplastic anemia and myelodysplastic syndrome in individuals lacking glutathione S-transferase genes. Pediatr Blood Cancer 2004; 42:122. Kurtzman G, Young N. Viruses and bone marrow failure. Baillieres Clin Haematol 1989; 2:51. Brown KE, Tisdale J, Barrett AJ, et al. Hepatitis-associated aplastic anemia. N Engl J Med 1997; 336:1059. Hadzi�� N, Height S, Ball S, et al. Evolution in the management of acute liver failure-associated aplastic anaemia in children: a single centre experience. J Hepatol 2008; 48:68.
Kurtzman G, Young N. Viruses and bone marrow failure. Baillieres Clin Haematol 1989; 2:51. Brown KE, Tisdale J, Barrett AJ, et al. Hepatitis-associated aplastic anemia. N Engl J Med 1997; 336:1059. Hadzi�� N, Height S, Ball S, et al. Evolution in the management of acute liver failure-associated aplastic anaemia in children: a single centre experience. J Hepatol 2008; 48:68. Osugi Y, Yagasaki H, Sako M, et al. Antithymocyte globulin and cyclosporine for treatment of 44 children with hepatitis associated aplastic anemia. Haematologica 2007; 92:1687. Mary JY, Baumelou E, Guiguet M. Epidemiology of aplastic anemia in France: a prospective multicentric study. The French Cooperative Group for Epidemiological Study of Aplastic Anemia. Blood 1990; 75:1646. Ohara A, Kojima S, Okamura J, et al. Evolution of myelodysplastic syndrome and acute myelogenous leukaemia in children with hepatitis-associated aplastic anaemia. Br J Haematol 2002; 116:151. Oosterkamp HM, Brand A, Kluin-Nelemans JC, Vandenbroucke JP. Pregnancy and severe aplastic anaemia: causal relation or coincidence? . Br J Haematol 1998; 103:315. Young NS. Acquired aplastic anemia. Ann Intern Med 2002; 136:534. Nisticò A, Young NS. gamma-Interferon gene expression in the bone marrow of patients with aplastic anemia. Ann Intern Med 1994; 120:463. Dybedal I, Bryder D, Fossum A, et al. Tumor necrosis factor (TNF)-mediated activation of the p55 TNF receptor negatively regulates maintenance of cycling reconstituting human hematopoietic stem cells. Blood 2001; 98:1782. Maciejewski JP, Selleri C, Sato T, et al. Increased expression of Fas antigen on bone marrow CD34+ cells of patients with aplastic anaemia. Br J Haematol 1995; 91:245. Ball SE, Gibson FM, Rizzo S, et al. Progressive telomere shortening in aplastic anemia. Blood 1998; 91:3582. Yamaguchi H, Baerlocher GM, Lansdorp PM, et al. Mutations of the human telomerase RNA gene (TERC) in aplastic anemia and myelodysplastic syndrome. Blood 2003; 102:916. Yamaguchi H, Calado RT, Ly H, et al. Mutations in TERT, the gene for telomerase reverse transcriptase, in aplastic anemia. N Engl J Med 2005; 352:1413. Zeng W, Chen G, Kajigaya S, et al. Gene expression profiling in CD34 cells to identify differences between aplastic anemia patients and healthy volunteers. Blood 2004; 103:325. Young, NS, Alter, BP. Aplastic Anemia: Acquired and Inherited. WB Saunders, Philadelphia, 1994, p. 13.
Yamaguchi H, Calado RT, Ly H, et al. Mutations in TERT, the gene for telomerase reverse transcriptase, in aplastic anemia. N Engl J Med 2005; 352:1413. Zeng W, Chen G, Kajigaya S, et al. Gene expression profiling in CD34 cells to identify differences between aplastic anemia patients and healthy volunteers. Blood 2004; 103:325. Young, NS, Alter, BP. Aplastic Anemia: Acquired and Inherited. WB Saunders, Philadelphia, 1994, p. 13. Howard SC, Naidu PE, Hu XJ, et al. Natural history of moderate aplastic anemia in children. Pediatr Blood Cancer 2004; 43:545. Rozman C, Marín P, Nomdedeu B, Montserrat E. Criteria for severe aplastic anaemia. Lancet 1987; 2:955. Bacigalupo A, Hows J, Gluckman E, et al. Bone marrow transplantation (BMT) versus immunosuppression for the treatment of severe aplastic anaemia (SAA): a report of the EBMT SAA working party. Br J Haematol 1988; 70:177. Rosenfeld SJ, Kimball J, Vining D, Young NS. Intensive immunosuppression with antithymocyte globulin and cyclosporine as treatment for severe acquired aplastic anemia. Blood 1995; 85:3058. Höchsmann B, Moicean A, Risitano A, et al. Supportive care in severe and very severe aplastic anemia. Bone Marrow Transplant 2013; 48:168. Young NS, Calado RT, Scheinberg P. Current concepts in the pathophysiology and treatment of aplastic anemia. Blood 2006; 108:2509. Passweg JR, Socié G, Hinterberger W, et al. Bone marrow transplantation for severe aplastic anemia: has outcome improved? Blood 1997; 90:858. Gillio AP, Boulad F, Small TN, et al. Comparison of long-term outcome of children with severe aplastic anemia treated with immunosuppression versus bone marrow transplantation. Biol Blood Marrow Transplant 1997; 3:18. Bacigalupo A. Treatment strategies for patients with severe aplastic anemia. Bone Marrow Transplant 2008; 42 Suppl 1:S42. Socié G, Henry-Amar M, Bacigalupo A, et al. Malignant tumors occurring after treatment of aplastic anemia. European Bone Marrow Transplantation-Severe Aplastic Anaemia Working Party. N Engl J Med 1993; 329:1152. Ades L, Mary JY, Robin M, et al. Long-term outcome after bone marrow transplantation for severe aplastic anemia. Blood 2004; 103:2490. Socié G, Stone JV, Wingard JR, et al. Long-term survival and late deaths after allogeneic bone marrow transplantation. Late Effects Working Committee of the International Bone Marrow Transplant Registry. N Engl J Med 1999; 341:14.
Ades L, Mary JY, Robin M, et al. Long-term outcome after bone marrow transplantation for severe aplastic anemia. Blood 2004; 103:2490. Socié G, Stone JV, Wingard JR, et al. Long-term survival and late deaths after allogeneic bone marrow transplantation. Late Effects Working Committee of the International Bone Marrow Transplant Registry. N Engl J Med 1999; 341:14. Paquette RL, Tebyani N, Frane M, et al. Long-term outcome of aplastic anemia in adults treated with antithymocyte globulin: comparison with bone marrow transplantation. Blood 1995; 85:283. Maschan A, Bogatcheva N, Kryjanovskii O, et al. Results at a single centre of immunosuppression with cyclosporine A in 66 children with aplastic anaemia. Br J Haematol 1999; 106:967. Young NS, Barrett AJ. The treatment of severe acquired aplastic anemia. Blood 1995; 85:3367. Rosenfeld S, Follmann D, Nunez O, Young NS. Antithymocyte globulin and cyclosporine for severe aplastic anemia: association between hematologic response and long-term outcome. JAMA 2003; 289:1130. Bacigalupo A, Bruno B, Saracco P, et al. Antilymphocyte globulin, cyclosporine, prednisolone, and granulocyte colony-stimulating factor for severe aplastic anemia: an update of the GITMO/EBMT study on 100 patients. European Group for Blood and Marrow Transplantation (EBMT) Working Party on Severe Aplastic Anemia and the Gruppo Italiano Trapianti di Midolio Osseo (GITMO). Blood 2000; 95:1931. Lawlor ER, Anderson RA, Davis JH, et al. Immunosuppressive therapy: a potential alternative to bone marrow transplantation as initial therapy for acquired severe aplastic anemia in childhood? J Pediatr Hematol Oncol 1997; 19:115. Matloub YH, Smith C, Bostrom B, et al. One course versus two courses of antithymocyte globulin for the treatment of severe aplastic anemia in children. J Pediatr Hematol Oncol 1997; 19:110. Goldenberg NA, Graham DK, Liang X, Hays T. Successful treatment of severe aplastic anemia in children using standardized immunosuppressive therapy with antithymocyte globulin and cyclosporine A. Pediatr Blood Cancer 2004; 43:718. Fuhrer M, Rampf U, Burdach S, et al. Immunosuppressive therapy and bone marrow transplantation for aplastic anemia in children: Results of the study SAA 94 (abstract). Blood 1998; 92 Suppl 1:156a.
Goldenberg NA, Graham DK, Liang X, Hays T. Successful treatment of severe aplastic anemia in children using standardized immunosuppressive therapy with antithymocyte globulin and cyclosporine A. Pediatr Blood Cancer 2004; 43:718. Fuhrer M, Rampf U, Burdach S, et al. Immunosuppressive therapy and bone marrow transplantation for aplastic anemia in children: Results of the study SAA 94 (abstract). Blood 1998; 92 Suppl 1:156a. Scheinberg P, Wu CO, Nunez O, Young NS. Long-term outcome of pediatric patients with severe aplastic anemia treated with antithymocyte globulin and cyclosporine. J Pediatr 2008; 153:814. Tichelli A, Gratwohl A, Nissen C, et al. Morphology in patients with severe aplastic anemia treated with antilymphocyte globulin. Blood 1992; 80:337. Sloand E, Kim S, Maciejewski JP, et al. Intracellular interferon-gamma in circulating and marrow T cells detected by flow cytometry and the response to immunosuppressive therapy in patients with aplastic anemia. Blood 2002; 100:1185. Locasciulli A, van't Veer L, Bacigalupo A, et al. Treatment with marrow transplantation or immunosuppression of childhood acquired severe aplastic anemia: a report from the EBMT SAA Working Party. Bone Marrow Transplant 1990; 6:211. Dearden C, Foukaneli T, Lee P, et al. The incidence and significance of fevers during treatment with antithymocyte globulin for aplastic anaemia. Br J Haematol 1998; 103:846. Schrezenmeier H, Marin P, Raghavachar A, et al. Relapse of aplastic anaemia after immunosuppressive treatment: a report from the European Bone Marrow Transplantation Group SAA Working Party. Br J Haematol 1993; 85:371. Scheinberg P, Cooper JN, Sloand EM, et al. Association of telomere length of peripheral blood leukocytes with hematopoietic relapse, malignant transformation, and survival in severe aplastic anemia. JAMA 2010; 304:1358. Kojima S, Ohara A, Tsuchida M, et al. Risk factors for evolution of acquired aplastic anemia into myelodysplastic syndrome and acute myeloid leukemia after immunosuppressive therapy in children. Blood 2002; 100:786. Griscelli-Bennaceur A, Gluckman E, Scrobohaci ML, et al. Aplastic anemia and paroxysmal nocturnal hemoglobinuria: search for a pathogenetic link. Blood 1995; 85:1354.
Kojima S, Ohara A, Tsuchida M, et al. Risk factors for evolution of acquired aplastic anemia into myelodysplastic syndrome and acute myeloid leukemia after immunosuppressive therapy in children. Blood 2002; 100:786. Griscelli-Bennaceur A, Gluckman E, Scrobohaci ML, et al. Aplastic anemia and paroxysmal nocturnal hemoglobinuria: search for a pathogenetic link. Blood 1995; 85:1354. Schubert J, Vogt HG, Zielinska-Skowronek M, et al. Development of the glycosylphosphatitylinositol-anchoring defect characteristic for paroxysmal nocturnal hemoglobinuria in patients with aplastic anemia. Blood 1994; 83:2323. Di Bona E, Rodeghiero F, Bruno B, et al. Rabbit antithymocyte globulin (r-ATG) plus cyclosporine and granulocyte colony stimulating factor is an effective treatment for aplastic anaemia patients unresponsive to a first course of intensive immunosuppressive therapy. Gruppo Italiano Trapianto di Midollo Osseo (GITMO). Br J Haematol 1999; 107:330. Scheinberg P, Nunez O, Young NS. Retreatment with rabbit anti-thymocyte globulin and ciclosporin for patients with relapsed or refractory severe aplastic anaemia. Br J Haematol 2006; 133:622. Macartney C, Freilich M, Odame I, et al. Complete response to tacrolimus in a child with severe aplastic anemia resistant to cyclosporin A. Pediatr Blood Cancer 2009; 52:525. Audino AN, Blatt J, Carcamo B, et al. High-dose cyclophosphamide treatment for refractory severe aplastic anemia in children. Pediatr Blood Cancer 2010; 54:269. Topic 5923 Version 12.0 © 2013 UpToDate, Inc. All rights reserved. | Subscription and License Agreement | Release: 21.3 - C21.34 Licensed to: Morehouse School of Medicine | Support Tag: [1004-85.214.214.87-4FABCF92AE-S473950.14]