Complete substitution of cyclophosphamide by fludarabine and ATG in a busulfan-based preparative regimen for children and adolescents with β-thalassemia

Bone Marrow Transplantation

Volumn 36, Issue 5, 2005, Pages 383-387

  Sauer, Martin ^a   Bettoni, C ^a   Lauten, M ^a   Ghosh, A ^a   Rehe, K ^a   Grigull, L ^a   Beilken, A ^a   Welte, K ^a   Sykora, K W ^a  

^a HANNOVER MEDICAL SCHOOL   (Germany)

Author keywords

ATG; Bone marrow transplantation; Busulfan; Cyclophosphamide; Fludarabine; Preparative regimen; Thalassemia

Indexed keywords

BUSULFAN; CYCLOPHOSPHAMIDE; CYCLOSPORIN A; DEFIBROTIDE; FLUDARABINE; METHOTREXATE; THYMOCYTE ANTIBODY;

ADOLESCENT; ARTICLE; BONE MARROW TRANSPLANTATION; CELL COUNT; CHILD; CHIMERA; CLINICAL ARTICLE; CLINICAL PROTOCOL; DISEASE SEVERITY; DRUG DOSE REGIMEN; DRUG SUBSTITUTION; FEMALE; GRAFT VERSUS HOST REACTION; HLA MATCHING; HUMAN; IMMUNOSUPPRESSIVE TREATMENT; LIVING DONOR; MALE; MUCOSA INFLAMMATION; PILOT STUDY; PRIORITY JOURNAL; SIBLING; THALASSEMIA MAJOR; VEIN OCCLUSION;

ADOLESCENT; BETA-THALASSEMIA; BONE MARROW TRANSPLANTATION; CASE-CONTROL STUDIES; CHILD; CHILD, PRESCHOOL; FEMALE; GRAFT VS HOST DISEASE; HISTOCOMPATIBILITY TESTING; HUMANS; IMMUNOSUPPRESSIVE AGENTS; MALE; TRANSPLANTATION CHIMERA; TRANSPLANTATION CONDITIONING; TRANSPLANTATION, HOMOLOGOUS;

EID: 30944463804     PISSN: 02683369     EISSN: 14765365     Source Type: Journal    
DOI: 10.1038/sj.bmt.1705082     Document Type: Article

References (29)

1. Weatherall DJ, Clegg JB. Thalassemia - a global public health problem. Nat Med 1996; 2: 847-849.
2. Hershko C, Weatherall DJ. Iron-chelating therapy. Crit Rev Clin Lab Sci 1988; 26: 303-345.
3. Lucarelli G, Galimberti M, Polchi P et al. Marrow transplantation in patients with thalassemia responsive to iron chelation therapy. N Engl J Med 1993; 329: 840-844.
4. Lucarelli G, Clift RA, Galimberti M et al. Marrow transplantation for patients with thalassemia: Results in class 3 patients. Blood 1996; 87: 2082-2088.
5. Lucarelli G, Galimberti M, Polchi P et al. Bone marrow transplantation in patients with thalassemia. N Engl J Med 1990; 322: 417-421.
6. Sodani P, Gaziev D, Polchi P et al. New approach for bone marrow transplantation in patients with class 3 thalassemia aged younger than 17 years. Blood 2004; 104: 1201-1203.
7. McDonald GB, Slattery JT, Bouvier ME et al. Cyclophosphamide metabolism, liver toxicity, and mortality following hematopoietic stem cell transplantation. Blood 2003; 101: 2043-2048.
8. Browne RK, Scheltinga DM, Pomering M, Mahony M. Testicular myxosporidiasis in anurans, with a description of Myxobolus fallax n. sp. Syst Parasitol 2002; 52: 97-110.
9. Voorter CE, Rozemuller EH, de Bruyn-Geraets D et al. Comparison of DRB sequence-based typing using different strategies. Tissue Antigens 1997; 49: 471-476.
10. Storb R, Deeg HJ, Whitehead J et al. Methotrexate and cyclosporine compared with cyclosporine alone for prophylaxis of acute graft versus host disease after marrow transplantation for leukemia. N Engl J Med 1986; 314: 729-735.
11. Przepiorka D, Weisdorf D, Martin P et al. 1994 Consensus Conference on Acute GVHD Grading. Bone Marrow Transplant 1995; 15: 825-828.
12. Lucarelli G, Andreani M, Angelucci E. The cure of thalassemia by bone marrow transplantation. Blood Rev 2002; 16: 81-85.
13. Sensenbrenner LL, Owens Jr AH, Heiby JR, Jeejeebhoy HF. Comparative effects of cytotoxic agents on transplanted hematopoietic and antibody-producing cells. J Natl Cancer Inst 1973; 50: 1027-1033.
14. Fried W, Kedo A, Barone J. Effects of cyclophosphamide and of busulfan on spleen colory-forming units and on hematopoietic stroma. Cancer Res 1977; 37: 1205-1209.
15. Thomas ED, Buckner CD, Rudolph RH et al. Allogeneic marrow grafting for hematologic malignancy using HL-A matched donor-recipient sibling pairs. Blood 1971; 38: 267-287.
16. Santos GW, Tutschka PJ, Brookmeyer R et al. Marrow transplantation for acute nonlymphocytic leukemia after treatment with busulfan and cyclophosphamide. N Engl J Med 1983; 309: 1347-1353.
17. Bearman SI. Veno-occlusive disease of the liver. Curr Opin Oncol 2000; 12: 103-109.
18. Keating MJ, O'Brien S, Lerner S et al. Long-term follow-up of patients with chronic lymphocytic leukemia (CLL) receiving fludarabine regimens as initial therapy. Blood 1998; 92: 1165-1171.
19. Chun HG, Leyland-Jones B, Cheson BD. Fludarabine phosphate: A synthetic purine antimetabolite with significant activity against lymphoid malignancies. J Clin Oncol 1991; 9: 175-188.
20. Kashyap A, Wingard J, Cagnoni P et al. Intravenous versus oral busulfan as part of a busulfan/cyclophosphamide preparative regimen for allogeneic hematopoietic stem cell transplantation: Decreased incidence of hepatic venoocclusive disease (HVOD), HVOD-related mortality, and overall 100-day mortality. Biol Blood Marrow Transplant 2002; 8: 493-500.
21. Gandhi V, Plunkett W. Cellular and clinical pharmacology of fludarabine. Clin Pharmacokinet 2002; 41: 93-103.
22. de Lima M, Couriel D, Thall PF et al. Once-daily intravenous busulfan and fludarabine: Clinical and pharmacokinetic results of a myeloablative, reduced-toxicity conditioning regimen for allogeneic stem cell transplantation in AML and MDS. Blood 2004; 104: 857-864.
23. Andersson BS, Kashyap A, Gian V et al. Conditioning therapy with intravenous busulfan and cyclophosphamide (IV BuCy2) for hematologic malignancies prior to allogeneic stem cell transplantation: A phase II study. Biol Blood Marrow Transplant 2002; 8: 145-154.
24. Iannone R, Casella JF, Fuchs EJ et al. Results of minimally toxic nonmyeloablative transplantation in patients with sickle cell anemia and beta-thalassemia. Biol Blood Marrow Transplant 2003; 9: 519-528.
25. Horan JT, Liesveld JL, Fenton P et al. Hematopoietic stem cell transplantation for multiply transfused patients with sickle cell disease and thalassemia after low-dose total body irradiation, fludarabine, and rabbit anti-thymocyte globulin. Bone Marrow Transplant 2005; 35: 171-177.
26. Hongeng S, Chuansumrit A, Hathirat P et al. Full chimerism in nonmyeloablative stem cell transplantation in a beta-thalassemia major patient (class 3 Lucarelli). Bone Marrow Transplant 2002; 30: 409-410.
27. Storb R, Floersheim GL, Weiden PL et al. Effect of prior blood transfusions on marrow grafts: Abrogation of sensitization by procarbazine and antithymocyte serum. J Immunol 1974; 112: 1508-1516.
28. Storb R, Etzioni R, Anasetti C et al. Cyclophosphamide combined with antithymocyte globulin in preparation for allogeneic marrow transplants in patients with aplastic anemia. Blood 1994; 84: 941-949.
29. 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.