Pharmacokinetic behavior and appraisal of intravenous busulfan dosing in infants and older children: The results of a population pharmacokinetic study from a large pediatric cohort undergoing hematopoietic stem-cell transplantation

Therapeutic Drug Monitoring

Volumn 34, Issue 2, 2012, Pages 198-208

  Paci, Angelo ^a   Vassal, Gilles ^a   Moshous, Despina ^b   Dalle, Jean Hugues ^c   Bleyzac, Nathalie ^d   Neven, Bénédicte ^b   Galambrun, Claire ^e   Kemmel, Véronique ^f   Abdi, Zeinab D ^g   Broutin, Sophie ^a   Pétain, Aurélie ^g   Nguyen, Laurent ^g  

^a INSTITUT GUSTAVE ROUSSY   (France)

^b HÔPITAL NECKER ENFANTS MALADES   (France)

^c HÔPITAL ROBERT DEBRÉ   (France)

^d HOSPICES CIVILS DE LYON   (France)

^e TIMONE HOSPITAL   (France)

^f UNIVERSITY HOSPITAL OF STRASBOURG   (France)

^g INSTITUT DE RECHERCHE PIERRE FABRE   (France)

Author keywords

busulfan; children; pediatrics; pharmacokinetics; population pharmacokinetics

Indexed keywords

ALEMTUZUMAB; BUSULFAN; CAMPTOTHECIN; CYCLOPHOSPHAMIDE; FLUDARABINE; GLUTATHIONE TRANSFERASE A1; MELPHALAN; THIOTEPA;

ARTICLE; BODY WEIGHT; CHILD; DRUG CLEARANCE; DRUG DISPOSITION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; IMMUNE DEFICIENCY; INFANT; INFANT DISEASE; IRON OVERLOAD; LIVER DYSFUNCTION; MAJOR CLINICAL STUDY; MALIGNANT NEOPLASTIC DISEASE; METABOLIC DISORDER; NEWBORN; OBSERVATIONAL STUDY; PRIORITY JOURNAL;

ADOLESCENT; AGE FACTORS; AREA UNDER CURVE; BODY WEIGHT; BUSULFAN; CHILD; CHILD, PRESCHOOL; COHORT STUDIES; DOSE-RESPONSE RELATIONSHIP, DRUG; EUROPE; GLUTATHIONE TRANSFERASE; HUMANS; IMMUNOSUPPRESSIVE AGENTS; INFANT; INFANT, NEWBORN; INFUSIONS, INTRAVENOUS; MODELS, BIOLOGICAL;

EID: 84858705938     PISSN: 01634356     EISSN: 15363694     Source Type: Journal    
DOI: 10.1097/FTD.0b013e31824c2f60     Document Type: Article

References (57)

1. McCune JS, Gibbs JP, Slattery JT. Plasma concentration monitoring of busulfan: does it improve clinical outcome? Clin Pharmacokinet. 2000; 39:155-165.
2. 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. (Pubitemid 34993856)
3. Bolinger AM, Zangwill AB, Slattery JT, et al. An evaluation of engraftment, toxicity and busulfan concentration in children receiving bone marrow transplantation for leukemia or genetic disease. Bone Marrow Transplant. 2000;25:925-930. (Pubitemid 30255723)
4. 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.
5. 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. (Pubitemid 27172594)
6. Andersson 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 i.v. BuCy2 in chronic myelogenous leukemia. Biol Blood Marrow Transplant. 2002;8:477-485. (Pubitemid 35174821)
7. 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. (Pubitemid 26057312)
8. 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. (Pubitemid 20000799)
9. Ljungman P, Hassan M, Bekassy AN, et al. High busulfan concentrations are associated with increased transplant related mortality in allogeneic bone marrow transplant patients. Bone Marrow Transplant. 1997;20:909-913. (Pubitemid 27510952)
10. Grochow LB. Busulfan disposition: the role of therapeutic monitoring in bone marrow transplantation induction regimens. Semin Oncol. 1993;20: 18-25. (Pubitemid 23239401)
11. 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. (Pubitemid 35174823)
12. 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;6:548-554.
13. Thall PF, Champlin RE, Andersson BS. Comparison of 100-day mortality rates associated with i.v. busulfan and cyclophosphamide vs other preparative regimens in allogeneic bone marrow transplantation for chronic myelogenous leukemia: Bayesian sensitivity analyses of confounded treatment and center effects. Bone Marrow Transplant. 2004;33: 1191-1199. (Pubitemid 38961476)
14. 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. (Pubitemid 39406769)
15. Oechtering D, Schiltmeyer B, Hempel G, et al. Toxicity and pharmacokinetics of i.v. busulfan in children before stem cell transplantation. Anticancer Drugs. 2005;16:337-344. (Pubitemid 40283950)
16. Zwaveling J, Bredius RGM, Cremers SCLM, 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. (Pubitemid 40090051)
17. Nguyen L. The impact of switching from oral to IV busulfan on haematopoietic stem cell transplantation. EJHPP Practice. 2006;12:58-59.
18. Leger F, Nguyen L, Puozzo C. Exposure equivalence between IV (0.8 mg/kg) and oral (1 mg/kg) busulfan in adult patients. Eur J Clin Pharmacol. 2009;65:903-911.
19. 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. (Pubitemid 34285073)
20. Nguyen L, Leger F, Lennon S, et al. Intravenous busulfan in adults prior to haematopoietic stem cell transplantation: a population pharmacokinetic study. Cancer Chemother Pharmacol. 2006;57:191-198. (Pubitemid 41779838)
21. Gibbs JP, Murray G, Risler L. Age-dependent tetrahydrothiophenium ion formation in young children and adults receiving high-dose busulfan. Cancer Res. 1997;57:5509-5516. (Pubitemid 28006757)
22. Bostrom B, Enockson K, Johnson A, et al. Plasma pharmacokinetics of high-dose oral busulfan in children and adults undergoing bone marrow transplantation. Pediatr Transplant. 2003;7(suppl 3):12-18. (Pubitemid 36314216)
23. Hassan M, Ljungman P, Bolme P, et al. Busulfan bioavailability. Blood. 1994;84:2144-2150. (Pubitemid 24296370)
24. Hassan M, Oberg G, Bekassy AN, et al. Pharmacokinetics of high-dose busulphan in relation to age and chronopharmacology. Cancer Chemother Pharmacol. 1991;28:130-134.
25. Gibbs JP, Liacouras CA, Baldassano RN, et al. Up-regulation of glutathione S-transferase activity in enterocytes of young children. Drug Metab Dispos. 1999;27:1466-1469.
26. Nguyen L, Fuller D, Lennon S, et al. I.V. busulfan in pediatrics: a novel dosing to improve safety/efficacy for hematopoietic progenitor cell transplantation recipients. Bone Marrow Transplant. 2004;33:979-987. (Pubitemid 38744783)
27. Vassal G, Michel G, Espérou H, et al. Prospective validation of a novel IV busulfan fixed dosing for paediatric patients to improve therapeutic AUC targeting without drug monitoring. Cancer Chemother Pharmacol. 2008; 61:113-123. (Pubitemid 47512633)
28. Michel G, Valteau-Couanet D, Gentet JC, et al. Weight-based strategy of dose administration in children using intravenous busulfan: clinical and pharmacokinetic results. Pediatr Blood Cancer. 2012;58:90-97.
29. Slattery JT, Risler LJ. Therapeutic monitoring of busulfan in hematopoietic stem cell transplantation. Ther Drug Monit. 1998;20:543-549. (Pubitemid 28463447)
30. Holford NH. A size standard for pharmacokinetics. Clin Pharmacokinet. 1996;30:329-332. (Pubitemid 26177179)
31. Peters HP. Physiological correlates of size. In: Beck E, Birks HJB, Conner EF, eds. The ecological implication of body size. Cambridge University Press, 1983:48-53.
32. West GB, Brown JH, Enquist BJ. A general model for the origin of allometric scaling laws in biology. Science. 1997;276:122-126. (Pubitemid 27161259)
33. Anderson BJ, Meakin GH. Scaling for size: some implications for paediatric anaesthesia dosing. Paediatr Anaesth. 2002;12:219.
34. Anderson BJ, Holford NH. Mechanism-based concepts of size and maturity in pharmacokinetics. Annu Rev Pharmacol Toxicol. 2008;48:303-332. (Pubitemid 351738156)
35. Tod M, Julien V, Pons G. Facilitation of drug evaluation in children by population methods and modelling. Clin Pharmacokinet. 2008; 47:231-243. (Pubitemid 351367434)
36. Yano Y, Beal SL, Sheiner LB. Evaluating pharmacokinetic/pharmacodynamic models using the posterior predictive check. J Pharmacokinet Pharmacodyn. 2001;28:171-192.
37. Booth BP, Rahman A, Dagher R, et al. Pharmacokinetic based dosing of intravenous busulfan in pediatric patients.J ClinPharmacol. 2007;47:111.
38. 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;30:504-510.
39. Yeager AM, Wagner JE Jr, 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.
40. Vassal G, Deroussent A, Challine D, et al. Is 600 mg/m2 the appropriate dosage of busulfan in children undergoing bone marrow transplantation? Blood. 1992;79:2475-2479.
41. Mahmood I. Prediction of drug clearance in children from adults: a comparison of several allometric methods. Br J Clin Pharmacology. 2006;61:545-557.
42. Knibbe CAJ, Krekels EHJ, van den Anker JN, et al. Morphine glucuronidation in preterm neonates, infants and children younger than 3 years. Clin Pharmacokinet. 2009;48:371-385.
43. Peeters MY, Allegaert K, Blusse van Oud-Alblas HJ, et al. Prediction of propofol clearance in children from an allometric model developed in rats, children and adults versus a 0.75 fixed-exponent allometric model. Clin Pharmacokinet. 2010;49:269-275
44. Gaziev J, Nguyen L, Puozzo C, et al. Novel pharmacokinetic behavior of intravenous busulfan in children with thalassemia undergoing hematopoietic stem cell transplantation: a prospective evaluation of pharmacokinetic and pharmacodynamic profile with therapeutic drug monitoring. Blood. 2010;115:4597-4604.
45. Kim SD, Lee JH, Hur EH, 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.
46. Kusuma 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. (Pubitemid 43776023)
47. Johnson A, Orchand PJ, Scott Baker K, et al. Glutathione s-transferase A1 genetic variants reduce busulfan clearance in children undergoing hemotopoietic cell transplantation. J Clin Pharmacol. 2008;48: 1052-1062.
48. Ansari M, Lauzon Joset JF, Vachon M, et al. Influence of GST gene polymorphisms on busulfan pharmacokinetics in children. Bone Marrow Transplant. 2010;45:261-267.
49. Bredschneider M, Klein K, Murdter TE, et al. Genetic polymorphisms of glutathione s-transferase A1, the major glutathione s-transferase in human liver: consequences for enzyme expression and busulfan conjugation. Clin Pharmacol Ther. 2002;71:479-487. (Pubitemid 34701355)
50. Bertholle-Bonnet V, Bleyzac N, Galambrun C, et al. Influence of underlying disease on Busulfan disposition in pediatric bone marrow transplant recipients: a non-parametric population pharmacokinetic study. Ther Drug Monit. 2007;29:184.
51. Vassal G, Fischer A, Challine D, et al. Busulfan disposition below the age of three: alteration in children with lysosomal storage disease. Blood. 1993;82:1030-1034. (Pubitemid 23221692)
52. Hassan M, Oberg G, Bjorkholm M, et al. Influence of prophylactic anticonvulsant therapy on high-dose busulphan kinetics. Cancer Chemother Pharmacol. 1993;33:181-186. (Pubitemid 23361064)
53. Hassan M, Oberg G, Ehrsson H, et al. Pharmacokinetic and metabolic studies of high-dose busulphan in adults. Eur J Clin Pharmacol. 1989;36: 525-530. (Pubitemid 19135219)
54. Geddes M, Kangarloo SB, 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.
55. Nation RL, Evans AM, Milne RW. Pharmacokinetic drug interactions with phenytoin (part I). Clin Pharmacokinet. 1990;18:37-60. (Pubitemid 20040337)
56. Nation RL, Evans AM, Milne RW. Pharmacokinetic drug interactions with phenytoin (part II). Clin Pharmacokinet. 1990;18:131-150. (Pubitemid 20104032)
57. Carreras E, Cahn JY, Puozzo C, et al. Influence on Busilvex pharmacokinetics of clonazepam compared to previous phenytoin historical data. Anticancer Res. 2010;30:2977-2984.