Highly Variable Pharmacokinetics of Once-Daily Intravenous Busulfan When Combined with Fludarabine in Pediatric Patients: Phase I Clinical Study for Determination of Optimal Once-Daily Busulfan Dose Using Pharmacokinetic Modeling

Biology of Blood and Marrow Transplantation

Volumn 18, Issue 6, 2012, Pages 944-950

  Lee, Ji Won ^a   Kang, Hyoung Jin ^a   Lee, Seung Hwan ^a   Yu, Kyung Sang ^a   Kim, Nam Hee ^a   Yuk, Yen Ju ^a   Jang, Mi Kyoung ^a   Han, Eun Jong ^a   Kim, Hyery ^a   Song, Sang Hoon ^a   Park, Kyung Duk ^a   Shin, Hee Young ^a   Jang, In Jin ^a   Ahn, Hyo Seop ^a  

^a Seoul National University College of Medicine   (South Korea)

Author keywords

Busulfan; Fludarabine; Pharmacokinetics; Stem cell transplantation

Indexed keywords

BUSULFAN; CYCLOSPORIN; ETOPOSIDE; FLUDARABINE; GLUTATHIONE TRANSFERASE; METHOTREXATE; MYCOPHENOLIC ACID; PREDNISOLONE; TACROLIMUS;

ADOLESCENT; ADULT; AREA UNDER THE CURVE; ARTICLE; CHILD; CLINICAL ARTICLE; CONTROLLED STUDY; DRUG CLEARANCE; DRUG MONITORING; FEMALE; GRAFT VERSUS HOST REACTION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; INFANT; MALE; PHASE 1 CLINICAL TRIAL; PRESCHOOL CHILD; PROSPECTIVE STUDY; PROTEIN POLYMERIZATION; SCHOOL CHILD; UNSPECIFIED SIDE EFFECT; VEIN OCCLUSION;

ADOLESCENT; ANTINEOPLASTIC COMBINED CHEMOTHERAPY PROTOCOLS; AREA UNDER CURVE; BONE MARROW TRANSPLANTATION; BUSULFAN; CHILD; CHILD, PRESCHOOL; DRUG ADMINISTRATION SCHEDULE; FEMALE; GRAFT VS HOST DISEASE; HUMANS; INFANT; LEUKEMIA, MYELOID, ACUTE; MALE; MODELS, STATISTICAL; PERIPHERAL BLOOD STEM CELL TRANSPLANTATION; PRECURSOR CELL LYMPHOBLASTIC LEUKEMIA-LYMPHOMA; TRANSPLANTATION, HOMOLOGOUS; TREATMENT OUTCOME; VIDARABINE;

EID: 84862807889     PISSN: 10838791     EISSN: 15236536     Source Type: Journal    
DOI: 10.1016/j.bbmt.2011.11.025     Document Type: Article

References (43)

1. Copelan E.A., Bechtel T.P., Avalos B.R., et al. Busulfan levels are influenced by prior treatment and are associated with hepatic veno-occlusive disease and early mortality but not with delayed complications following marrow transplantation. Bone Marrow Transplant 2001, 27:1121-1124.
2. Dix S.P., Wingard J.R., Mullins R.E., et al. Association of busulfan area under the curve with veno-occlusive disease following BMT. Bone Marrow Transplant 1996, 17:225-230.
3. Geddes M., Kangarloo S.B., Naveed F., et al. High busulfan exposure is associated with worse outcomes in a daily i.v. busulfan and fludarabine allogeneic transplant regimen. Biol Blood Marrow Transplant 2008, 14:220-228.
4. Ljungman P., Hassan M., Bekassy A.N., Ringden O., Oberg G. High busulfan concentrations are associated with increased transplant-related mortality in allogeneic bone marrow transplant patients. Bone Marrow Transplant 1997, 20:909-913.
5. McCune J.S., Gibbs J.P., Slattery J.T. Plasma concentration monitoring of busulfan: does it improve clinical outcome?. Clin Pharmacokinet 2000, 39:155-165.
6. Slattery J.T., Clift R.A., Buckner C.D., et al. Marrow transplantation for chronic myeloid leukemia: the influence of plasma busulfan levels on the outcome of transplantation. Blood 1997, 89:3055-3060.
7. Madden T., de Lima M., Thapar N., et al. Pharmacokinetics of once-daily IV busulfan as part of pretransplantation preparative regimens: a comparison with an every 6-hour dosing schedule. Biol Blood Marrow Transplant 2007, 13:56-64.
8. Russell J.A., Tran H.T., Quinlan D., et al. Once-daily intravenous busulfan given with fludarabine as conditioning for allogeneic stem cell transplantation: study of pharmacokinetics and early clinical outcomes. Biol Blood Marrow Transplant 2002, 8:468-476.
9. Chiesa R., Cappelli B., Crocchiolo R., et al. Unpredictability of intravenous busulfan pharmacokinetics in children undergoing hematopoietic stem cell transplantation for advanced beta thalassemia: limited toxicity with a dose-adjustment policy. Biol Blood Marrow Transplant 2010, 16:622-628.
10. Nath C.E., Earl J.W., Pati N., Stephen K., Shaw P.J. Variability in the pharmacokinetics of intravenous busulphan given as a single daily dose to paediatric blood or marrow transplant recipients. Br J Clin Pharmacol 2008, 66:50-59.
11. Tran H., Petropoulos D., Worth L., et al. Pharmacokinetics and individualized dose adjustment of intravenous busulfan in children with advanced hematologic malignancies undergoing allogeneic stem cell transplantation. Biol Blood Marrow Transplant 2004, 10:805-812.
12. Russell J.A., Kangarloo S.B. Therapeutic drug monitoring of busulfan in transplantation. Curr Pharm Des 2008, 14:1936-1949.
13. Bartelink I.H., Bredius R.G., Ververs T.T., et al. Once-daily intravenous busulfan with therapeutic drug monitoring compared with conventional oral busulfan improves survival and engraftment in children undergoing allogeneic stem cell transplantation. Biol Blood Marrow Transplant 2008, 14:88-98.
14. Bleyzac N., Souillet G., Magron P., et al. Improved clinical outcome of paediatric bone marrow recipients using a test dose and Bayesian pharmacokinetic individualization of busulfan dosage regimens. Bone Marrow Transplant 2001, 28:743-751.
15. Kletzel M., Jacobsohn D., Duerst R. Pharmacokinetics of a test dose of intravenous busulfan guide dose modifications to achieve an optimal area under the curve of a single daily dose of intravenous busulfan in children undergoing a reduced-intensity conditioning regimen with hematopoietic stem cell transplantation. Biol Blood Marrow Transplant 2006, 12:472-479.
16. Lemaistre J.A., Bachier C., Smith B., Lemaistre C., Shaughnessy P.J. Once daily busulfan cyclophosphamide is well tolerated and effective as a preparative regimen for allogeneic hematopoietic stem cell transplant. J Oncol Pharm Pract 2011 Jan 12, [Epub ahead of print].
17. Russell J.A., Duan Q., Chaudhry M.A., et al. Transplantation from matched siblings using once-daily intravenous busulfan/fludarabine with thymoglobulin: a myeloablative regimen with low nonrelapse mortality in all but older patients with high-risk disease. Biol Blood Marrow Transplant 2008, 14:888-895.
18. Ryu S.G., Lee J.H., Choi S.J., et al. Randomized comparison of four-times-daily versus once-daily intravenous busulfan in conditioning therapy for hematopoietic cell transplantation. Biol Blood Marrow Transplant 2007, 13:1095-1105.
19. Zwaveling J., den Hartigh J., Lankester A.C., Guchelaar H.J., Egeler R.M., Bredius R.G. Once-daily intravenous busulfan in children before stem cell transplantation: study of pharmacokinetics and early clinical outcomes. Anticancer Drugs 2006, 17:1099-1105.
20. Andersson B.S., de Lima M., Thall P.F., et al. Once daily i.v. busulfan and fludarabine (i.v. Bu-Flu) compares favorably with i.v. busulfan and cyclophosphamide (i.v. BuCy2) as pretransplant conditioning therapy in AML/MDS. Biol Blood Marrow Transplant 2008, 14:672-684.
21. Bornhauser M., Storer B., Slattery J.T., et al. Conditioning with fludarabine and targeted busulfan for transplantation of allogeneic hematopoietic stem cells. Blood 2003, 102:820-826.
22. de Lima M., Couriel D., Thall P.F., 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. Lee J.H., Choi J., Kwon K.A., et al. Fludarabine-based myeloablative regimen as pretransplant conditioning therapy in adult acute leukemia/myelodysplastic syndrome: comparison with oral or intravenous busulfan with cyclophosphamide. Korean J Hematol 2010, 45:102-108.
24. 2 is safe and effective in children of all ages. Bone Marrow Transplant 2004, 34:197-205.
25. Kang H.J., Shin H.Y., Choi H.S., Ahn H.S. Fludarabine, cyclophosphamide plus thymoglobulin conditioning regimen for unrelated bone marrow transplantation in severe aplastic anemia. Bone Marrow Transplant 2004, 34:939-943.
26. Kim M.S., Kang H.J., Park H.J., et al. Development of multiplex PCR method for the analysis of glutathione S-transferase polymorphisms. Mol Diagn Ther 2011.
27. Andersson B.S., Madden T., Tran H.T., et al. Acute safety and pharmacokinetics of intravenous busulfan when used with oral busulfan and cyclophosphamide as pretransplantation conditioning therapy: a phase I study. Biol Blood Marrow Transplant 2000, 6:548-554.
28. Cremers S., Schoemaker R., Bredius R., et al. Pharmacokinetics of intravenous busulfan in children prior to stem cell transplantation. Br J Clin Pharmacol 2002, 53:386-389.
29. Fernandez H.F., Tran H.T., Albrecht F., Lennon S., Caldera H., Goodman M.S. Evaluation of safety and pharmacokinetics of administering intravenous busulfan in a twice-daily or daily schedule to patients with advanced hematologic malignant disease undergoing stem cell transplantation. Biol Blood Marrow Transplant 2002, 8:486-492.
30. McCune J.S., Holmberg L.A. Busulfan in hematopoietic stem cell transplant setting. Expert Opin Drug Metab Toxicol 2009, 5:957-969.
31. Johnson L., Orchard P.J., Baker K.S., et al. Glutathione S-transferase A1 genetic variants reduce busulfan clearance in children undergoing hematopoietic cell transplantation. J Clin Pharmacol 2008, 48:1052-1062.
32. Kusama M., Kubota T., Matsukura Y., et al. Influence of glutathione S-transferase A1 polymorphism on the pharmacokinetics of busulfan. Clin Chim Acta 2006, 368:93-98.
33. Ansari M., Lauzon-Joset J.F., Vachon M.F., et al. Influence of GST gene polymorphisms on busulfan pharmacokinetics in children. Bone Marrow Transplant 2010, 45:261-267.
34. Elhasid R., Krivoy N., Rowe J.M., et al. Influence of glutathione S-transferase A1, P1, M1, T1 polymorphisms on oral busulfan pharmacokinetics in children with congenital hemoglobinopathies undergoing hematopoietic stem cell transplantation. Pediatr Blood Cancer 2010, 55:1172-1179.
35. Kim S.D., Lee J.H., Hur E.H., et al. Influence of GST gene polymorphisms on the clearance of intravenous busulfan in adult patients undergoing hematopoietic cell transplantation. Biol Blood Marrow Transplant 2011, 17:1222-1230.
36. Zwaveling J., Press R.R., Bredius R.G., et al. Glutathione S-transferase polymorphisms are not associated with population pharmacokinetic parameters of busulfan in pediatric patients. Ther Drug Monit 2008, 30:504-510.
37. Adkins J.C., Peters D.H., Markham A. Fludarabine. An update of its pharmacology and use in the treatment of haematological malignancies. Drugs 1997, 53:1005-1037.
38. Bonin M., Pursche S., Bergeman T., et al. F-ara-A pharmacokinetics during reduced-intensity conditioning therapy with fludarabine and busulfan. Bone Marrow Transplant 2007, 39:201-206.
39. Takamatsu Y., Sasaki N., Eto T., et al. Individual dose adjustment of oral busulfan using a test dose in hematopoietic stem cell transplantation. Int J Hematol 2007, 86:261-268.
40. Bartelink I.H., Bredius R.G., Belitser S.V., et al. Association between busulfan exposure and outcome in children receiving intravenous busulfan before hematologic stem cell transplantation. Biol Blood Marrow Transplant 2009, 15:231-241.
41. O'Donnell P.H., Artz A.S., Undevia S.D., et al. Phase I study of dose-escalated busulfan with fludarabine and alemtuzumab as conditioning for allogeneic hematopoietic stem cell transplant: reduced clearance at high doses and occurrence of late sinusoidal obstruction syndrome/veno-occlusive disease. Leuk Lymphoma 2010, 51:2240-2249.
42. Shaw P.J., Scharping C.E., Brian R.J., Earl J.W. Busulfan pharmacokinetics using a single daily high-dose regimen in children with acute leukemia. Blood 1994, 84:2357-2362.
43. Nakamura H., Sato T., Okada K., et al. Population pharmacokinetics of oral busulfan in young Japanese children before hematopoietic stem cell transplantation. Ther Drug Monit 2008, 30:75-83.