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referencesuptodate· REFERENCES· item f3_41_3738

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.

referencesuptodate· REFERENCES· item f3_41_3738

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.

referencesuptodate· REFERENCES· item f3_41_3738

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.

referencesuptodate· REFERENCES· item f3_41_3738

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.

referencesuptodate· REFERENCES· item f3_41_3738

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.

referencesuptodate· REFERENCES· item f3_41_3738

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.

referencesuptodate· REFERENCES· item f3_41_3738

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]