Randomized trial of two different conditioning regimens for bone marrow transplantation in thalassemia - The role of busulfan pharmacokinetics in determining outcome

Bone Marrow Transplantation

Volumn 36, Issue 10, 2005, Pages 839-845

  Chandy, M ^a   Balasubramanian, P ^a   Ramachandran, S V ^a   Mathews, V ^a   George, B ^a   Dennison, D ^b   Krishnamoorthy, R ^c   Srivastava, A ^a  

^a CHRISTIAN MEDICAL COLLEGE   (India)

^b SULTAN QABOOS UNIVERSITY HOSPITAL   (Oman)

^c HÔPITAL ROBERT DEBRÉ   (France)

Author keywords

Busulfan; Pharmacokinetics; Thalassemia

Indexed keywords

BUSULFAN; CYCLOPHOSPHAMIDE; THYMOCYTE ANTIBODY;

AREA UNDER THE CURVE; ARTICLE; BONE MARROW TRANSPLANTATION; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; DRUG BLOOD LEVEL; DRUG EXPOSURE; DRUG HALF LIFE; DRUG MONITORING; FEMALE; GASTROINTESTINAL HEMORRHAGE; GASTROINTESTINAL TOXICITY; GRAFT REJECTION; GRAFT VERSUS HOST REACTION; HEMORRHAGIC CYSTITIS; HUMAN; IMMUNOSUPPRESSIVE TREATMENT; LIVER TOXICITY; LIVER VENOOCCLUSIVE DISEASE; LUNG TOXICITY; MAJOR CLINICAL STUDY; MALE; NEUROTOXICITY; OUTCOMES RESEARCH; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; RISK REDUCTION; STATISTICAL ANALYSIS; STOMATITIS; SURVIVAL RATE; THALASSEMIA MAJOR;

ANTILYMPHOCYTE SERUM; BONE MARROW TRANSPLANTATION; BUSULFAN; CHILD; CHILD, PRESCHOOL; CYCLOPHOSPHAMIDE; DRUG MONITORING; FEMALE; GRAFT SURVIVAL; HUMANS; IMMUNOSUPPRESSIVE AGENTS; MALE; MYELOABLATIVE AGONISTS; THALASSEMIA; TRANSPLANTATION CONDITIONING; TREATMENT OUTCOME;

EID: 27844545839     PISSN: 02683369     EISSN: 14765365     Source Type: Journal    
DOI: 10.1038/sj.bmt.1705151     Document Type: Article

References (40)

1. Galimberti M, Angelucci E, Baroncianni D et al. Bone marrow transplantation in thalasemia. The experience of Pesaro. Bone Marrow Transplant 1997; 19 (Suppl. 2): 45-47.
2. Schrier SL, Angelucci E. New strategies in the treatment of the thalassemias. Ann Rev Med 2005; 56: 157-171.
3. Ball LM, Lankester AC, Giordano PC et al. Paediatric allogeneic bone marrow transplantation for homozygous beta-thalassaemia, the Dutch experience. Bone Marrow Transplant 2003; 31: 1081-1087.
4. Shing MM, Yuen PM, Li CK et al. A more immunosuppressive pre-transplant conditioning may be required for Chinese patients with thalassaemia. Bone Marrow Transplant 1996; 17: 907-910.
5. Poonkuzhali B, Srivastava A, Quernin MH et al. Pharmacokinetics of oral busulfan in children with beta thalassemia major undergoing allogeneic bone marrow transplantation. Bone marrow Transplant 1999; 24: 5-11.
6. Hassan M, Fasth A, Gerritsen B et al. Busulfan kinetics and limited sampling model in children with leukemia and inherited disorders. Bone Marrow Transplant 1996; 18: 843-850.
7. 2 the appropriate dosage of busulfan in children undergoing bone marrow transplantation? Blood 1992; 79: 2475-2479.
8. Storb R, Blume KG, O'Donnell MR et al. Cyclophosphamide and antithymocyte globulin to condition patients with aplastic anemia for allogeneic marrow transplantations: The experience in four centers. Blood Marrow Transplant 2001; 7: 39-44.
9. Chandy M, Srivastava A, Dennison D et al. Allogeneic bone marrow transplantation in the developing world: Experience from a center in India. Bone Marrow Transplant 2001; 27: 785-790.
10. Glucksberg H, Storb R, Fefer A et al. Clinical manifestations of graft versus host disease in human recipients of marrow from HLA matched sibling donors. Transplantation 1974; 18: 295-304.
11. Bearman SI, Appelbaum FR, Buckner CD et al. Regimen-related toxicity in patients undergoing bone marrow transplantation. J Clin Oncol 1988; 6: 1562-1568.
12. Jones RJ, Lee KSK, Beschorner WE et al. Venocclusive disease of the liver following bone marrow transplantation. Transplantation 1987; 44: 778-783.
13. Quernin MH, Poonkuzhali B, Medard Y et al. High-performance liquid chromatographic method for quantification of busulfan in plasma after derivatization by tetrafluorothiophenol. J Chromatogr 1999; 721: 147-152.
14. Quernin MH, Poonkuzhali B, Montes C et al. Quantification of busulfan in plasma by Gas chromatography-mass spectrometry. J Chromatogr 1998; 709: 47-56.
15. Heinzel G, Woloszezak R, Thoman P. Pharmacokinetic Pharmacodynamic Data Analysis System for the PC. Gustav Fischer: Sturtgart, Germany, 1993.
16. Thomas ED, Buckner CD, Sanders JE et al. Marrow transplantation for thalassaemia. Lancet 1982; 2: 227.
17. Lucarelli G, Andreani M, Angelucci E. The cure of thalassemia by bone marrow transplantation. Blood Rev 2002; 16: 81-85.
18. Chen YC, Lin KH, Huang WS, Tang JL. Bone marrow transplantation in Taiwan: An overview. Bone Marrow Transplant 1994; 13: 907-910.
19. Lawson SE, Roberts IA, Amrolia P et al. Bone marrow transplantation for beta-thalassaemia major: The UK experience in two paediatric centres. Br J Haematol 2003; 120: 289-295.
20. 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.
21. Sodani P, Gaziev D, Polchi P et al. A new approach for bone marrow transplantation in class 3 thalassemic patients aged less than 17 years. Blood 2004; 104: 1201-1203.
22. Brachet C, Azzi N, Demulder A et al. Hydroxyurea treatment for sickle cell disease: Impact on hematopoietic stem cell transplantation outcome. Bone Marrow Transplant 2004; 33: 799-803.
23. 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.
24. Vassal G, Fischer G, Challine D et al. Busulfan disposition below the age of three: Alteration in children with lysosomal storage disease. Blood 1993; 82: 1030-1034.
25. Hassan M, Ljungman P, Bolme P et al. Busulfan bioavailability. Blood 1994; 84: 2144-2150.
26. Shaw PJ, Scharping CE, Brian RJ, Earl JW. Busulfan pharmacokinetics using a single daily high-dose regimen in children with acute leukemia. Blood 1994; 84: 2357-2362.
27. Yeager AM, Wagner JE, Graham ML et al. Optimization of busulfan dosage in children undergoing bone marrow transplantation: A pharmacokinetic study of dose escalation. Blood 1992; 80: 2425-2428.
28. Slattery JT, Sanders JE, Buckner CD et al. Graft rejection and toxicity following bone marrow transplantation in relation to busulfan pharmacokinetics. Bone Marrow Transplant 1995; 16: 31-42.
29. Slattery JT, Clift RA, Buckner CD et al. Marrow transplantation for chronic myeloid leukemia: The influence of plasma busulfan levels on the outcome of transplantation. Blood 1997; 89: 3055-3060.
30. McCune JS, Gooley T, Gibbs JP et al. Busulfan concentration and graft rejection in pediatric patients undergoing hematopoietic stem cell transplantation. Bone Marrow Transplant 2002; 30: 167-173.
31. Pawlowska AB, Blazar BR, Angelucci E et al. Relationship of plasma pharmacokinetics of high-dose oral busulfan to the outcome of allogeneic bone marrow transplantation in children with thalassemia. Bone Marrow Transplant 1997; 20: 915-920.
32. Grochow LB, Jones RJ, Brundrett RB et al. Pharmacokinetics of busulfan: correlation with veno-occlusive disease in patients undergoing bone marrow transplantation. Cancer Chemother Pharmacol 1989; 25: 55-61.
33. Dix SP, Wingard JR, Mullins RE et al. Association of busulfan area under the curve with veno-occlusive disease following BMT. Bone Marrow Transplant 1996; 17: 225-230.
34. Anderson BS, Thall PF, Madden T et al. Busulfan systemic exposure relative to regimen related toxicity and acute graft versus host disease: Defining a therapeutic window for IV BuCy2 in chronic myelogenous leukemia. Biol Blood Marrow Transplant 2002; 8: 477-485.
35. Ljungman P, Hassan M, Bekassy AN et al. Busulfan concentration in relation to permanent alopecia in recipients of bone marrow transplants. Bone Marrow Transplantation 1995; 15: 869-871.
36. Srivastava A, Poonkuzhali B, Shaji RV et al. Glutathione S-transferase M1 polymorphism - a risk factor for hepatic venoocclusive disease in bone marrow transplantation. Blood 2004; 104: 1574-1577.
37. Bleyazac N, Souillet G, Magron P et al. Reduced transplant related mortality by Bayesian individualization of busulfan dosage in children. Bone Marrow Transplant 2001; 28: 743-751.
38. Demirer T, Buckner CD, Appelbaum FR et al. Busulfan, cyclophosphamide and fractionated total body irradiation for allogeneic marrow transplantation in advanced acute and chronic myelogenous leukemia: phase I dose escalation of busulfan based on targeted plasma levels. Bone Marrow Transplant 1996; 17: 341-346.
39. Dupuis LL, Najdova M, Saunders EF. Retrospective appraisal of busulfan dose adjustment in children. Bone Marrow Transplant 2000; 26: 1143-1147.
40. McCune JS, Gibbs JP, Slattery JT. Plasma concentration monitoring of busulfan: Does it improve clinical outcome? Clin Pharmacokinet 2000; 39: 155-165.