The importance of therapeutic drug monitoring (TDM) for parenteral busulfan dosing in conditioning regimen for hematopoietic stem cell transplantation (HSCT) in children

Annals of Transplantation

Volumn 19, Issue 1, 2014, Pages 214-224

  Tesfaye, Hndie ^a   Branova, Romana ^a   Klapkova, Eva ^a   Prusa, Richard ^a   Janeckova, Daniela ^a   Riha, Petr ^a   Sedlacek, Petr ^a   Keslova, Petra ^a   Malis, Josef ^a  

^a CHARLES UNIVERSITY   (Czech Republic)

Author keywords

High dose busulfan target AUC; Limited sampling strategy TDM; Pediatrie HSCT

Indexed keywords

BUSULFAN;

ADOLESCENT; ADULT; AREA UNDER THE CURVE; ARTICLE; BLOOD SAMPLING; BODY WEIGHT; CHILD; CLINICAL ARTICLE; CLINICAL ASSESSMENT; CONTROLLED STUDY; DOSE CALCULATION; DRUG EFFICACY; DRUG MEGADOSE; DRUG MONITORING; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HIGH PERFORMANCE LIQUID CHROMATOGRAPHY; HUMAN; INFANT; MEDICATION COMPLIANCE; QUANTITATIVE ASSAY; TREATMENT OUTCOME; YOUNG ADULT;

ADJUVANTS, IMMUNOLOGIC; ADOLESCENT; BODY WEIGHT; BUSULFAN; CHILD; CHILD, PRESCHOOL; CHROMATOGRAPHY, HIGH PRESSURE LIQUID; DITIOCARB; DOSE-RESPONSE RELATIONSHIP, DRUG; DRUG MONITORING; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; INFANT; INFUSIONS, INTRAVENOUS; MYELOABLATIVE AGONISTS; TRANSPLANTATION CONDITIONING;

EID: 84901242037     PISSN: 14259524     EISSN: None     Source Type: Journal    
DOI: 10.12659/AOT.889933     Document Type: Article

References (64)

1. Iwamoto T, Hiraku Y, Oikawa S et al: DNA intrastrand cross-link at the 5'-GA-3' sequence formed by Busulfan and its role in the cytotoxic efect. Cancer Sci, 2004; 95: 454-58
2. Karstens A, Kramer I: Chemical and physical stability of diluted Busulfan infusion solutions. EJHP Science, 2007; 13: 40-47
3. 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: 1347-54
4. Eberly AL, Anderson, GD, Bubalo JS, McCune JS: Optimal prevention of seizures induced by high-dose Busulfan. Pharmacotherapy, 2008; 28: 1502-10
5. 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: 386-89
6. van den Bongard HJ, Mathot RA, Beijnen JH, Schellens JH: Pharmacokinetically guided administration of chemotherapeutic agents. Clin Pharmacokinet, 2000; 39: 345-67
7. Dupuis LL, Najdova M, Saunders EF: Retrospective appraisal of Busulfan dose adjustment in children. Bone Marrow Transplant, 2000; 26: 1143-47
8. Dupuis LL, Sibbald C, Schechter T et al: IV Busulfan Dose Individualization in Children undergoing Haematopoietic Stem Cell Transplant: Limited Sampling Strategie. Biol Blood Marrow Transplant, 2008; 14: 576-82
9. Chow DSL, Bhagwatwar HP, Phadungpojna S, Andersson BS: Stability-indicating high-performance liquid chromatographic assay of Busulfan in aqueous and plasma samples. J Chromatogr B, 1997; 704: 277-88
10. Rifai N, Sakamoto M, Laf M, Guinan E: Measurement of Plasma Busulfan Concentration by High-Performance Liquid Chromatography with Ultra Violet Detection, Ther Drug Monit, 1997; 19: 169-74
11. 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: 225-30
12. Russell JA, Kangarloo SB: Therapeutic drug monitoring of Busulfan in transplantation. Current Pharmaceutical Design, 2008; 14: 1936-49
13. Nguyen L, Leger F, Lennon S, Puozzo C: Intravenous Busulfan in adults prior to haematopoietic stem cell transplantation: a population pharmacoki-netic study. Cancer Chemother Pharmacol, 2006; 57: 191-98
14. Tran H, Petropoulos D, Worth L et al: Pharmacokinetics and individualized dose adjustment of intravenous Busulfan in children with advanced hae-matologic malignancies undergoing allogeneic stem cell transplantation. Biol Blood Marrow Transplant, 2004; 10: 805-12
15. Vaughan WP, Carey D, Perry S et al: A limited sampling strategy for phar-macokinetic directed therapy with intravenous Busulfan. Biol Blood Marrow Transplant, 2002; 8: 619-24
16. Balasubramanian P, Chandy M, Krishnamoorthy R, Srivastava A: Evaluation of existing limited in children with beta thalassaemia major undergoing bone marrow sampling models for Busulfan kinetics transplantation. Bone Marrow Transplant, 2001; 28: 821-25
17. Slattery JT, Risler LJ: Therapeutic monitoring of Busulfan in haematopoietic stem cell transplantation. Ther Drug Monit, 1998; 20: 543-49
18. Grochow LB, Krivit W, Whitley CB, Blazar B: Busulfan disposition in children. Blood, 1990; 75: 1723-27
19. Hassan M, Öberg G, Bekassy AN: Pharmacokinetics of high-dose busulphan in relation to age and chronopharmacology. Cancer Chemother Pharmacol, 1991; 28: 130-34
20. Vassal G, Fischer A, Challine D et al: Busulfan disposition below the age of three: alteration in children with lysosomal storage disease. Blood, 1993; 82: 1030-34
21. Regazzi MB, Locatelli F, Buggia I et al: Disposition of high-dose Busulfan in pediatric patients undergoing bone marrow transplantation: a pharmaco-kinetic study of dose escalation. Blood, 1992; 80: 2425-28
22. Hassan M, Fasth A, Gerritsen B et al: Busulphan pharmacokinetics and limited sampling model in children with leukemia and inherited disorders. Bone Marrow Transplant, 1996; 18: 843-50
23. Olavarria E, Hassan M, Eades A et al: A phase I/II study of multiple-dose intravenous Busulfan as myeloablation prior to stem cell transplantation. Leukemia, 2000; 14: 1954-59
24. Andersson BS, Gajewski J, Donato M et al: Allogeneic stem cell transplantation (BMT) for AML and MDS following i.v. Busulfan and Cyclophosphamide (i.v. BuCy). Bone Marrow Transplant, 2000; 25(Suppl.2): S35-38
25. Busulfex Product information. Orphan Medical Inc, Minnetonka, Minnesota, USA, 2000
26. Andersson BS, Madden T, Tran HT 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: 548-54
27. Hassan Z, Ljungman P, Ringden O et al: Pharmacokinetics of liposomal bu-sulphan in man. Bone Marrow Transplant, 2001; 27: 479-85
28. Johnson-Davis KL, McMillin GA, Juenke JM et al: Which dose of Busulfan is best? Clin Chem, 2010; 56: 1061-64
29. Chattergoon DS, Saunders EF, Klein J et al: An improved limited sampling method for individualised busulphan dosing in bone marrow transplantation in children. Bone Marrow Transplant, 1997; 20: 347-54
30. Bartelink IH, Bredius RG, Ververs TT et al: Once-daily intravenous Busulfan with therapeutic drug monitoring compared to conventional oral Busulfan improves survival and engraftment in children undergoing allogeneic stem cell transplantation. Biol Blood Marrow Transplant, 2008; 14: 88-98
31. th Congress of the European Association for Clinical Pharmacology and Therapeutics - EACPT (July 12-15, 2009, Edinburgh, Scotland, United Kingdom), 2009; 215-19
32. Lindley C, Shea T, McCune J et al: Intraindividual variability in Busulfan pharmacokinetics in patients undergoing a bone marrow transplant: assessment of a test dose and frst dose strategy. Anticancer Drugs, 2004; 15: 453-59
33. Lee JW, Kang HJ, Lee SH et al: Highly variable pharmacokinetics of once-daily intravenous Busulfan when combined with fudarabine in pediatric patients: phase I clinical study for determination of optimal once-daily Busulfan dose using pharmacokinetic modeling. Biol Blood Marrow Transplant, 2012; 18: 944-50
34. Malr R, Sjöö F, Rentsch K et al: Therapeutic drug monitoring is essential for intravenous Busulfan therapy in pediatric haematopoietic stem cell recipients. Pediatr Transplantation, 2011; 15: 580-88
35. Proost JH, Meijer DK: MW/Pharm. An integrated software package for drug dosage regimen calculation and therapeutic drug monitoring. Comput Biol Med, 1992; 22: 155-63
36. Schechter T, Finkelstein Y, Doyle J et al: Pharmacokinetic disposition and clinical outcomes in infants and children receiving intravenous Busulfan for allogeneic haematopoietic stem cell transplantation. Biol Blood Marrow Transplant, 2007; 13: 307-14
37. McPherson ME, Hutcherson D, Olson E et al: Safety and efcacy of targeted Busulfan therapy in children undergoing myeloablative matched sibling donor BMT for sickle cell disease. Bone Marrow Transplant, 2011; 46: 27-33
38. Bredschneider M, Klein K, Mürdter TE et al: Genetic polymorphisms of glu-tathione S-transferase A1, the major glutathione S-transferase in human liver: consequences for enzyme expression and Busulfan conjugation. Clin Pharmacol Ther, 2002; 71: 479-87
39. Czerwinski M, Gibbs JP, Slattery JT: Busulfan conjugation by glutathione S-transferases alpha, mu, and pi. Drug Metab Dispos, 1996; 24: 1015-19
40. Kim I, Keam B, Lee KH et al: Glutathione S-transferase A1 polymorphisms and acute graft-vs.-host disease in HLA-matched sibling allogeneic haematopoietic stem cell transplantation. Clin Transplant, 2007; 21: 207-13
41. Ansari M, Lauzon-Joset JF, Vachon MF et al: Infuence of GST gene polymorphisms on Busulfan pharmacokinetics in children. Bone Marrow Transplant, 2010; 45: 261-67
42. Ansari M, Rezgui MA, Theoret Y et al: Glutathione S-transferase Gene variations infuence Bu pharmacokinetics and outcomes of hemopoietic SCT in pediatric patients. BMT, 2013; 48: 939-46
43. Elhasid R, Krivoy N, Rowe JM et al: Infuence of glutathione S-transferase A1, P1, M1, T1 polymorphisms on oral Busulfan pharmacokinetics in children with congenital hemoglobinopathies undergoing haematopoietic stem cell transplantation. Pediatr Blood Cancer, 2010; 55: 1172-79
44. Zwaveling J, Press RR, Bredius RG et al: Glutathione S-transferase polymorphisms are not associated with population pharmacokinetic parameters of Busulfan in pediatric patients. Ther Drug Monit, 2008; 30: 504-10
45. ten Brink MH, Wessels JA, den Hartigh J et al: Efect of genetic polymorphisms in genes encoding GST isoenzymes on BU pharmacokinetics in adult patients undergoing haematopoietic SCT. Bone Marrow Transplant, 2012; 47: 190-95
46. Kim SD, Lee JH, Hur EH et al: Infuence of GST gene polymorphisms on the clearance of intravenous Busulfan in adult patients undergoing haematopoietic cell transplantation. Biol Blood Marrow Transplant, 2011; 17: 1222-30
47. Tesfaye H, Vydra J, Havrankova V, Bránova R: Therapeutic drug monitoring (TDM) Uncovers unreliability of the frst dose to predict Busulfan next doses for haemopoietic stem cell transplantation conditioning therapy: A case study. Czech conference of Clinical pharmacology June 28-30, 2012, Zámek Stirin, Czech Republic. abstract pp 19-20, Farmakon press
48. Strober S: Protective conditioning against GVHD and graft rejection after combined organ and haematopoietic cell transplantation. Blood Cells Mol Dis, 2008; 40: 48-54
49. Hofer E, Akria L, Tabak A et al: A simple approximation for Busulfan dose adjustment in adult patients undergoing bone marrow transplantation. Ther Drug Monit, 2004; 26: 331-35
50. McCune JS, Gooley T, Gibbs JP et al: Busulfan concentration and graft rejection in pediatric patients undergoing haematopoietic stem cell transplantation. Bone Marrow Transplant, 2002; 30: 167-73
51. Zwaveling J, Bredius RG, Cremers SC et al: Intravenous Busulfan in children prior to stem cell transplantation: study of pharmacokinetics in association with early clinical outcome and toxicity. Bone Marrow Transplant, 2005; 35: 17-23
52. Andersson BS, Thall PF, Madden T et al: Busulfan systemic exposure relative to regimen-related toxicity and acute graft-versus-host disease: de-fning a therapeutic window for i.v. BuCy2 in chronic myelogenous leukemia. Biol Blood Marrow Transplant, 2002; 8: 477-85
53. Bolinger AM, Zangwill AB, Slattery JT et al: Target dose adjustment of Busulfan in pediatric patients undergoing bone marrow transplantation. Bone Marrow Transplant, 2001; 28: 1013-18
54. Walters MC, Storb R, Patience M et al: Impact of bone marrow transplantation for symptomatic sickle cell disease: an interim report. Multicentre investigation of bone marrow transplantation for sickle cell disease. Blood, 2000; 95: 1918-24
55. Vassal G, Michel G, Gentet J et al: A new IV Busulfan fixed dosing for better exposure targeting in children undergoing allogeneic or autologous haematopoietic stem cell transplantation Journal of Clinical Oncology, 2005 ASCO Annual Meeting Proceedings. Vol 23, No. 16S, Part I of II (June 1 Supplement), 2005; 8535
56. O'Donnell PH, Artz AS, Undevia SD et al: Phase I study of dose-escalated Busulfan with fudarabine and alemtuzumab as conditioning for alloge-neic haematopoietic stem cell transplant: reduced clearance at high doses and occurrence of late sinusoidal obstruction syndrome/veno-occlusive disease. Leuk Lymphoma, 2010; 51: 2240-49
57. Bartelink IH, Bredius RG, Belitser SV et al: Association between Busulfan exposure and outcome in children receiving intravenous Busulfan before hematologic stem cell transplantation. Biol Blood Marrow Transplant, 2009; 15: 231-41
58. Tesfaye H, Branova R, Riha P et al: Current challenges of body weight based intravenous busulfan dosing versus dose adjustment based on therapeutic drug monitoring. Biomed Pap Med Fac Univ Palacky Olomouc Czech Repub, 2013; 157(Suppl.1): 81-85
59. Hassan M: The role of Busulfan in bone marrow transplantation. Med Oncol, 1999; 16: 166-76
60. Pai RK, van Besien K, Hart J et al: Clinicopathologic features of late-onset veno-occlusive disease/sinusoidal obstruction syndrome after high dose intravenous Busulfan and haematopoietic cell transplant. Leuk Lymphoma, 2012; 53: 1552-57
61. Yeh RF, Pawlikowski MA, Blough DK et al: Accurate targeting of daily intravenous Busulfan with 8-hour blood sampling in hospitalized adult haematopoietic cell transplant recipients. Biol Blood Marrow Transplant, 2012; 18: 265-72
62. Clarke W: Commentary on: Which dose of Busulfan is best? Clin Chem, 2010; 56: 1061-64, Clin Chem, 2010; 56: 1064-65
63. Kellogg MD: Comment on Which dose of Busulfan is best? Clin Chem, 2010; 56: 1061-64
64. Vassal G, Michel G, Espérou H et al: Prospective validation of a novel IV bu-sulfan fixed dosing for paediatric patients to improve therapeutic AUC targeting without drug monitoring. Cancer Chemother Pharmacol, 2008; 61: 113-23