Full-text resources of PSJD and other databases are now available in the new Library of Science.
Visit https://bibliotekanauki.pl


Preferences help
enabled [disable] Abstract
Number of results


2009 | 4 | 3 | 363-368

Article title

Aplastic anaemia in childhood. Description of two cases and review of the literature


Title variants

Languages of publication



Childhood aplastic anaemia (AA) is an uncommon but potentially fatal haematological disorder. Patients with AA receive supportive care based on transfusions and timely treatment of opportunistic infections, along with specific therapies, which may be bone marrow transplantation and immunosuppressive therapy. Early diagnosis and supportive therapy are required to prevent fatal complications like overwhelming sepsis or life threatening haemorrhages. We report two cases of aplastic anaemia having a different aetiology. The diagnostic work-up and the therapeutic management for each case are described below.










Physical description


1 - 9 - 2009
3 - 7 - 2009


  • Division of Paediatric Oncology, Catholic University of Rome, 00168, Rome, Italy
  • Division of Paediatric Oncology, Catholic University of Rome, 00168, Rome, Italy
  • Division of Paediatric Oncology, Catholic University of Rome, 00168, Rome, Italy
  • Division of Paediatric Oncology, Catholic University of Rome, 00168, Rome, Italy
  • Division of Paediatric Oncology, Catholic University of Rome, 00168, Rome, Italy
  • Division of Paediatric Oncology, Catholic University of Rome, 00168, Rome, Italy
  • Division of Paediatric Oncology, Catholic University of Rome, 00168, Rome, Italy
  • Division of Paediatric Oncology, Catholic University of Rome, 00168, Rome, Italy


  • [1] Kojima S, Matsuyama T, Kato, S, Kigasawa H, Kobayashi R, Kikuta A., Sakamaki H, Ikuta K, Tsuchida, M., Hoshi Y, Morishima Y, Kodera Y. Outcome of 154 patients with severe aplastic anemia who received transplants from unrelated donors: the Japan Marrow Donor Program. Blood 2002;100:799–803 http://dx.doi.org/10.1182/blood.V100.3.799[Crossref]
  • [2] Locasciulli A. Acquired aplastic anemia in children: incidence, prognosis and treatment options. Paediatric Drugs 2002;4:761–766 [Crossref]
  • [3] Young NS. Aplastic anaemia. Lancet 1995; 346:228–832 http://dx.doi.org/10.1016/S0140-6736(95)91273-8[Crossref]
  • [4] The International Agranulocytosis and Aplastic Anemia Study. Incidence of aplastic anemia: the relevance of diagnostic criteria. Blood 1987;70:1718–1721
  • [5] Hirano N, Butler MO, Von Bergwelt-Baildon MS, Maecker B, Schultze, JL, O’Connor KC, Schur PH, Kojima S, Guinan EC, Nadler LM. Autoantibodies frequently detected in patients with aplastic anemia. Blood 2003;102:4567–4575 http://dx.doi.org/10.1182/blood-2002-11-3409[Crossref]
  • [6] Zeng W, Kajigaya S, Chen G, Risitano AM, Nunez O, Young NS. Transcript profile of CD4+ and CD8+ T cells from the bone marrow of acquired aplastic anemia patients. Exp Hematol, 2004;32:806–814 http://dx.doi.org/10.1016/j.exphem.2004.06.004
  • [7] Sloand E. Genetic polymorphisms and the risk of acquired idiopathic aplastic anemia. Haematologica 2005;90:1009B
  • [8] Dufour C, Capasso M, Svahn J, et al. Homozygosis for (12) CA repeats in the first intron of the human IFN-gamma gene is significantly associated with the risk of aplastic anaemia in Caucasian population. Br J Haematol 2004;126:682–685 http://dx.doi.org/10.1111/j.1365-2141.2004.05102.x[Crossref]
  • [9] Gidvani V, Ramkissoon S, Sloand EM, Young NS. Cytokine gene polymorphisms in acquired bone marrow failure. Am J Hematol 2007;82:721–724 http://dx.doi.org/10.1002/ajh.20881[Crossref][WoS]
  • [10] 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;123:906–914 http://dx.doi.org/10.1046/j.1365-2141.2003.04718.x[Crossref]
  • [11] Yanez Rubal JC, Estevez Rodriguez J, Crespo-Lopez MC, Martin- Herranz I. Carbamazepine-induced aplastic anaemia: a case report. Rev Neurol 2002;35:647–649
  • [12] Brown KE, Tisdale J, Barrett AJ, Dunbar CE, Young NS. Hepatitis associated aplastic anemia. N Engl J Med. 1997;336:1059–1064 http://dx.doi.org/10.1056/NEJM199704103361504[Crossref]
  • [13] Park CY, Kim HJ, Kim YJ, et al. Very severe aplastic anemia appearing after thymectomy. Korean J Intern Med 2003;18(1):61–63
  • [14] Deka D, Malhotra N, Sinha A, Banerjee N, Kashyap R, Roy KK. Pregnancy associated aplastic anemia: maternal and fetal outcome. J Obstet Gynaecol Res 2003;29(2):67–72 [Crossref]
  • [15] Schrezenmeier H, Hertenstein B, Wagner B, Raghavachar A, Heimpel H. A pathogenetic link between aplastic anemia and paroxysmal nocturnal hemoglobinuria is suggested by a high frequency of aplastic anemia patients with a deficiency of phosphatidylinositol glycan anchored proteins. Exp Hematol 1995; 23(1):81–87
  • [16] Kim I, Yoon SS, Park S, Kim BK, Kim NK. The treatment of severe aplastic anemia: outcomes of bone marrow transplantation and immunosuppressive therapy in a single institution of Korea. J Korean Med Sci 2003;18:365–371 http://dx.doi.org/10.3346/jkms.2003.18.4.483[Crossref]
  • [17] Torres HA, Bodey GP, Rolston KV, Kantarjian HM, Raad II, Kontoyiannis DP. Infections in patients with aplastic anemia: experience at a tertiary care cancer center. Cancer 2003;98(1):86–93 http://dx.doi.org/10.1002/cncr.11478[Crossref]
  • [18] Bhatnagar, S.K., Chandra, J., Narayan, S., Sharma, S., Singh, V. & Dutta, A.K. Pancytopenia in children: etiological profile. J Trop Pediatr 2005;51: 236–239 http://dx.doi.org/10.1093/tropej/fmi010[Crossref]
  • [19] Safadi R, Or R, Ilan Y, Naparstek E, Nagler A, Klein A, et al. Lack of known hepatitis virus in hepatitis-associated aplastic anemia and outcome after bone marrow transplantation. Bone Marrow Transplant 2001;27:183–190 http://dx.doi.org/10.1038/sj.bmt.1702749[Crossref]
  • [20] Sieff CA, Nisbet-Brown E, Nathan DG. Congenital bone marrow failure syndromes. Br J Haematol. 2000;111:30–42 http://dx.doi.org/10.1046/j.1365-2141.2000.02263.x[Crossref]
  • [21] Locasciulli, A, Oneto R, Bacigalupo A, Socie G, Korthof E, Bekassy A, et al. Outcome of patients with acquired aplastic anaemia given first line bone marrow transplantation or immunosuppression treatment in the last decade: a report from the European Group for Blood and Marrow Transplantation (EBMT). Haematologica 2007; 92:10–17 http://dx.doi.org/10.3324/haematol.10075[Crossref]
  • [22] Ades L, Mary JY, Robin M, Ferry C, Porcher R, Esperou H, et al. Long-term outcome after bone marrow transplantation for severe aplastic anaemia. Blood 2004;103:2490–2497 http://dx.doi.org/10.1182/blood-2003-07-2546[Crossref]
  • [23] Kahl C, Leisenring W, Deeg HJ, Chauncey TR, Flowers ME, Martin PJ, et al. Cyclophosphamide and antithymocyte globulin as a conditioning regimen for allogeneic marrow transplantation in patients with aplastic anaemia: a long term follow up. Br J Haematol 2005;130:747–751 http://dx.doi.org/10.1111/j.1365-2141.2005.05667.x[Crossref]
  • [24] Champlin RE, Perez WS, Passweg J, Klein JP, Camitta BM, Gluckman E, et al. Addition of antithymocyte globulin (ATG) to cyclophosphamide (Cy) for HLA-identical sibling allogeneic bone marrow transplantation (BMT) for severe aplastic anaemia (SAA): results of a randomized controlled trial. Blood 2003;269a
  • [25] Fouladi M, Herman R, Rolland-Grinton M, et al. Improved survival in severe acquired aplastic anemia in childhood. Bone Marrow Transplant 2000;26:1149–1156 http://dx.doi.org/10.1038/sj.bmt.1702699[Crossref]
  • [26] Kojima S, Horibe K, Inaba J, et al. Long-term outcome of acquired aplastic anemia in children: comparison between immunosuppressive therapy and bone marrow transplantation. Br J Haematol 2000;111:321–328 http://dx.doi.org/10.1046/j.1365-2141.2000.02289.x[Crossref]
  • [27] Schrezenmeier H, Marin P, Raghavachar A, McCann S, Hows J, Gluckman E, 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;85:371–377 http://dx.doi.org/10.1111/j.1365-2141.1993.tb03181.x[Crossref]
  • [28] Frickhofen N, Heimpel H, Kaltwasser JP, Schrezenmeier H. 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;101:1236–1242 http://dx.doi.org/10.1182/blood-2002-04-1134[Crossref]
  • [29] Socie G, Rosenfeld S, Frickhofen N, Gluckman E, Tichelli A. Late clonal diseases of treated aplastic anemia. Semin Hematol 2000;37:91–101 http://dx.doi.org/10.1016/S0037-1963(00)90033-7[Crossref]
  • [30] Rosenfeld S, Follman D, Nunez O, Young NS. Antithymocyte globulin and cyclosporine for severe aplastic anemia: association between haematologic response and long-term outcome. JAMA 2003;289:1130–1135 http://dx.doi.org/10.1001/jama.289.9.1130[Crossref]
  • [31] Scheinberg P, Nunez O, Young R. Re-treatment with rabbit antithymocyte globulin and cyclosporin for patients with relapsed or refractory severe aplastic anaemia. Br J Haematol 2006; 133:622–627 http://dx.doi.org/10.1111/j.1365-2141.2006.06098.x[Crossref]
  • [32] Marsh JC, Socie G, Schrezenmeier H, Tichelli A, Gluckman E, Ljungman P, et al. Haemopoietic growth factors in aplastic anaemia: a cautionary note. European Bone Marrow Transplant Working Party for Severe Aplastic Anaemia. Lancet 1994;344:172–173 http://dx.doi.org/10.1016/S0140-6736(94)92763-4[Crossref]
  • [33] Scheinberg P, Wu CO, Nunez O, Young NS. Predicting response and survival or severe aplastic anemia patients treated with immunosuppression. ASH Annual Meeting Abstracts 2007;110:504a
  • [34] Fang JP, Xu HG, Huang SL, Chen C, Huang K. Immunosuppressive treatment of aplastic anemia in Chinese children with antithymocyte globulin and cyclosporine. Pediatr Hematol Oncol 2006;23:45–50 http://dx.doi.org/10.1080/08880010500313389[Crossref]

Document Type

Publication order reference


YADDA identifier

JavaScript is turned off in your web browser. Turn it on to take full advantage of this site, then refresh the page.