Allogeneic hematopoietic stem cell transplantation is superior to immunosuppressive therapy in Indian children with aplastic anemiaa single-center analysis of 100 patients

Pediatric Hematology and Oncology

Volumn 27, Issue 2, 2010, Pages 122-131

  George, Biju ^a   Mathews, Vikram ^a   Viswabandya, Auro ^a   Lakshmi, Kavitha M ^a   Srivastava, Alok ^a   Chandy, Mammen ^a  

^a CHRISTIAN MEDICAL COLLEGE   (India)

Author keywords

Aplastic anemia; Children; HSCT; Immunosuppressive therapy

Indexed keywords

ANTIVIRUS AGENT; BUSULFAN; CORTICOSTEROID; COTRIMOXAZOLE; CYCLOPHOSPHAMIDE; CYCLOSPORIN; FLUDARABINE; LYMPHOCYTE ANTIBODY; METHOTREXATE; PREDNISOLONE; THYMOCYTE ANTIBODY;

ACUTE GRAFT VERSUS HOST DISEASE; ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; ALLOGENEIC PERIPHERAL BLOOD STEM CELL TRANSPLANTATION; APLASTIC ANEMIA; ARTICLE; CHILD; CHRONIC GRAFT VERSUS HOST DISEASE; CONTROLLED STUDY; DISEASE FREE SURVIVAL; ENGRAFTMENT; FEMALE; FOLLOW UP; GRAFT REJECTION; GRAFT VERSUS HOST REACTION; HERPES SIMPLEX; HUMAN; IMMUNOSUPPRESSIVE TREATMENT; INFECTION; INTERMETHOD COMPARISON; MAJOR CLINICAL STUDY; MALE; MORTALITY; OVERALL SURVIVAL; PNEUMOCYSTOSIS; SCHOOL CHILD; SURVIVAL RATE; SURVIVAL TIME; TREATMENT RESPONSE;

ANEMIA, APLASTIC; CHILD; DISEASE-FREE SURVIVAL; FEMALE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; IMMUNOSUPPRESSIVE AGENTS; INDIA; MALE; SURVIVAL RATE; TRANSPLANTATION, HOMOLOGOUS;

EID: 77749271196     PISSN: 08880018     EISSN: 15210669     Source Type: Journal    
DOI: 10.3109/08880010903540542     Document Type: Article

References (29)

1. Davies JK, Guinan EC. An update on the management of severe idiopathic aplastic anaemia in children. Br J Haematol. 2007;136:549-564.
2. Kennedy-Nasser AA, Leung KS, Mahajan A, et al. Comparable outcomes of matched-related and alternative donor stem cell transplantation for pediatric severe aplastic anemia. Biol Blood Marrow Transplant. 2006; 12: 1277-1284.
3. Munoz Villa A, Ortega Aramburu JJ, Bureo Dacal E, et al. Allogeneic bone marrow transplantation in children with severe acquired aplastic anemia. The long-term results. The Spanish Group for Bone Marrow Transplantation in Children (GETMON). An Esp Pediatr. 1999; 50:29-32.
4. Locasciulli A, Oneto R, Bacigalupo A, et al; Severe Aplastic Anemia Working Party of the European Blood and Marrow Transplant Group. 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). Haematologica. 2007;92:11-18.
5. Azuma E, Kojima S, Kato K, et al. Conditioning with cyclophosphamide/ antithymocyte globulin for allogeneic bone marrow transplantation from HLA-matched siblings in children with severe aplastic anemia. Bone Marrow Transplant. 1997; 19: 1085-1087.
6. Kahl C, Leisenring W, Deeg HJ, 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.
7. Kobayashi R, Yabe H, Hara J, et al. Preceding immunosuppressive therapy with antithymocyte globulin and cyclosporin increases the incidence of graft rejection in children with aplastic anaemia who underwent allogeneic bone marrow transplantation from HLA-identical siblings. Br J Haematol. 2006;135:693-696.
8. Fuhrer M, Rampf U, Baumann I, et al. Immunosuppressive therapy for aplastic anemia in children: a more severe disease predicts better survival. Blood. 2005;106:2102-2104.
9. Kojima S, Horibe K, Inaba J, et al. Long-term outcome of acquired aplastic anaemia in children: comparison between immunosuppressive therapy and bone marrow transplantation. Br J Haematol. 2000;111:321-328.
10. Feng YH, Yen CJ, Huang WT, et al. Clinical response of antilymphocyte globulin-based treatment in patients in Taiwan with aplastic anemia: positive hepatitis C antibody may represent a response predictor. Int J Hematol. 2004;79:133-137.
11. Kojima S, Hibi S, Kosaka Y, et al. Immunosuppressive therapy using antithymocyte globulin, cyclosporine, and danazol with or without human granulocyte colony-stimulating factor in children with acquired aplastic anemia. Blood. 2000;96:2049-2054.
12. de-Medeiros CR, Ribeiro RC, Bittencourt MA, et al. Long-term outcome of 25 children and adolescents with severe aplastic anemia treated with antithymocyte globulin. Braz J Med Biol Res. 2000; 33:553-558.
13. 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;19:110-114.
14. Rosenfeld SJ, Kimball J, Vining D, Young NS. Intensive immunosuppression with antithymocyte globulin and cyclosporine as treatment for severe acquired aplastic anemia. Blood. 1995;85:3058-3065.
15. Camitta BM. What is the definition of cure for aplastic anemia? Acta Haematol. 2000;103:16-18.
16. George B, Mathews V, Viswabandya A, et al. Fludarabine based reduced intensity conditioning regimens in children undergoing allogeneic stem cell transplantation for severe aplastic anemia. Pediatr Transplant. 2008;12:14-19.
17. Slavin S, Nagler A, Naparstek E, et al. Nonmyeloablative stem cell transplantation and cell therapy as an alternative to conventional bone marrow transplantation with lethal cytoreduction for the treatment of malignant and nonmalignant hematologic diseases. Blood. 1998;91:756-763.
18. Schrezenmeier H, Passweg JR, Marsh JC, et al. Worse outcome and more chronic GVHD with peripheral blood progenitor cells than bone marrow in HLA-matched sibling donor transplants for young patients with severe acquired aplastic anemia. Blood. 2007;110:1397-1400.
19. Lo Ten Foe JR, Kwee ML, Rooimans MA, et al. Somatic mosaicism in Fanconi anemia: molecular basis and significance. Eur J Hum Genet. 1997;5:137-148.
20. Federman N, Sakamoto KM. The genetic basis of bone marrow failure syndromes in children. Mol Genet Metab. 2005;86:100-109.
21. Calado RT, Graf SA, Wilkerson KL, et al. Mutations in the SBDS gene in acquired aplastic anemia. Blood. 2007;110:1141-1146.
22. 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;352:1413-1424.
23. Ortmann CA, Niemeyer CM, Wawer A, et al. TERC mutations in children with refractory cytopenia. Haematologica. 2006;91:707-708.
24. Solomou EE, Gibellini F, Stewart B, et al. Perforin gene mutations in patients with acquired aplastic anemia. Blood. 2007;109:5234-5237.
25. 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;3:18-24.
26. Bacigalupo A, Locatelli F, Lanino E, et al; Severe Aplastic Anemia Working Party of the European Group for Blood and Marrow Transplantation. Fludarabine, cyclophosphamide and anti-thymocyte globulin for alternative donor transplants in acquired severe aplastic anemia: a report from the EBMT-SAA Working Party. Bone Marrow Transplant. 2005;36:947-950.
27. Passweg JR, Perez WS, Eapen M, et al. Bone marrow transplants from mismatched related and unrelated donors for severe aplastic anemia. Bone Marrow Transplant. 2006;37:641-649.
28. Gluckman E, Rocha V, Chastang C. European results of unrelated cord blood transplants. Eurocord group. Bone Marrow Transplant. 1998;21(Suppl 3):S87-S91.
29. Tzeng CH, Chen PM, Fan S, et al. CY/TBI-800 as a pretransplant regimen for allogeneic bone marrow transplantation for severe aplastic anemia using HLA-haploidentical family donors. Bone Marrow Transplant. 1996;18:273-277.