Body weight-dependent pharmacokinetics of busulfan in paediatric haematopoietic stem cell transplantation patients: Towards individualized dosing

Clinical Pharmacokinetics

Volumn 51, Issue 5, 2012, Pages 331-345

  Bartelink, Imke H ^a,b   Boelens, Jaap J ^a   Bredius, Robbert G M ^c   Egberts, Antoine C G ^a,d   Wang, Chenguang ^b   Bierings, Marc B ^a   Shaw, Peter J ^e,f   Nath, Christa E ^e   Hempel, George ^g   Zwaveling, Juliette ^c   Danhof, Meindert ^b   Knibbe, Catherijne A J ^b,h  

^a UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

^b LEIDEN UNIVERSITY   (Netherlands)

^c LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

^d UTRECHT UNIVERSITY   (Netherlands)

^e CHILDREN S HOSPITAL AT WESTMEAD   (Australia)

^f UNIVERSITY OF SYDNEY   (Australia)

^g UNIVERSITY OF MÜNSTER   (Germany)

^h ST ANTONIUS HOSPITAL   (Netherlands)

Author keywords

Busulfan; Children; Modelling; Pharmacokinetics; Pharmacology; Population pharmacokinetics; Stem cell transplant

Indexed keywords

BUSULFAN;

ALLOMETRY; BODY WEIGHT; DRUG CLEARANCE; DRUG DISTRIBUTION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; META ANALYSIS; NOMOGRAM; PRIORITY JOURNAL; REVIEW;

ADOLESCENT; ADULT; AGE FACTORS; BODY WEIGHT; BUSULFAN; CHILD; CHILD, PRESCHOOL; DOSE-RESPONSE RELATIONSHIP, DRUG; FEMALE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; INDIVIDUALIZED MEDICINE; INFANT; INFANT, NEWBORN; MALE; MODELS, BIOLOGICAL; MYELOABLATIVE AGONISTS; NOMOGRAMS; NONLINEAR DYNAMICS; PROSPECTIVE STUDIES; SOFTWARE; TISSUE DISTRIBUTION; YOUNG ADULT;

EID: 84859558030     PISSN: 03125963     EISSN: 11791926     Source Type: Journal    
DOI: 10.2165/11598180-000000000-00000     Document Type: Review

References (62)

1. Ciurea SO, Andersson BS. Busulfan in hematopoietic stem cell transplantation 1 Biol Blood Marrow Transplant 2009 May; 15 (5): 523-36
2. Thomas ED, Buckner CD, Banaji M, et al. One hundred patients with acute leukemia treated by chemotherapy, total body irradiation, and allogeneic marrow transplantation. Blood 1977 Apr; 49 (4): 511-33 (Pubitemid 8080182)
3. Lu C, Braine HG, Kaizer H, et al. Preliminary results of high-dose busulfan and cyclophosphamide with syngeneic or autologous bone marrow rescue. Cancer Treat Rep 1984 May; 68 (5): 711-7 (Pubitemid 14115195)
4. 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 Dec 1; 309 (22): 1347-53 (Pubitemid 14246556)
5. Shaw PJ, Nath C, Berry A, et al. Busulphan given as four single daily doses of 150 mg/m2 is safe and effective in children of all ages. Bone Marrow Transplant 2004 Aug; 34 (3): 197-205 (Pubitemid 39136825)
6. Michel G, Gluckman E, Esperou-Bourdeau H, et al. Allogeneic bone marrow transplantation for children with acute myeloblastic leukemia in first complete remission: impact of conditioning regimen without total-body irradiation: a report from the Societe Francaise de Greffe de Moelle. J Clin Oncol 1994 Jun; 12 (6): 1217-22
7. Hassan M, Ljungman P, Bolme P, et al. Busulfan bioavailability. Blood 1994 Oct 1; 84 (7): 2144-50 (Pubitemid 24296370)
8. 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 Oct; 28 (8): 743-51 (Pubitemid 33089217)
9. McCune JS, Gibbs JP, Slattery JT. Plasma concentration monitoring of busulfan: does it improve clinical outcome? Clin Pharmacokinet 2000 Aug; 39 (2): 155-65
10. 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 Apr 15; 89 (8): 3055-60 (Pubitemid 27172594)
11. Bhagwatwar HP, Phadungpojna S, Chow DS, et al. Formulation and stability of busulfan for intravenous administration in high-dose chemotherapy. Cancer Chemother Pharmacol 1996; 37 (5): 401-8 (Pubitemid 26054548)
12. 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 Apr; 12 (4): 472-9 (Pubitemid 43380964)
13. Poonkuzhali B, Chandy M, Srivastava A, et al. Glutathione S-transferase activity influences busulfan pharmacokinetics in patients with beta thalassemia major undergoing bone marrow transplantation. Drug Metab Dispos 2001 Mar; 29 (3): 264-7 (Pubitemid 32179525)
14. Russell JA, Kangarloo SB. Therapeutic drug monitoring of busulfan in transplantation. Curr Pharm Des 2008; 14 (20): 1936-49
15. Tse WT, Duerst R, Schneiderman J, et al. Age-dependent pharmacokinetic profile of single daily dose i.v. busulfan in children undergoing reduced-intensity conditioning stem cell transplant. Bone Marrow Transplant 2009 Aug; 44 (3): 145-56
16. Wall DA, Chan KW, Nieder ML, et al. Safety, efficacy, and pharmacokinetics of intravenous busulfan in children undergoing allogeneic hematopoietic stem cell transplantation. Pediatr Blood Cancer 2010 Feb; 54 (2): 291-8
17. 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 Aug; 30 (4): 504-10
18. 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 Jan; 14 (1): 88-98 (Pubitemid 350286741)
19. Trame MN, Bergstrand M, Karlsson MO, et al. Population pharmacokinetics of busulfan in children: increased evidence for body surface area and allometric body weight dosing of busulfan in children. Clin Cancer Res 2011; 17: 6867-77
20. 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 May; 33 (10): 979-87 (Pubitemid 38744783)
21. Vassal G, Fischer A, Challine D, et al. Busulfan disposition below the age of three: alteration in children with lysosomal storage disease. Blood 1993 Aug 1; 82 (3): 1030-4 (Pubitemid 23221692)
22. Nath CE, Earl JW, Pati N, et al. 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 Jul; 66 (1): 50-9 (Pubitemid 351822161)
23. Bertholle-Bonnet V, Bleyzac N, Galambrun C, et al. Influence of underlying disease on busulfan disposition in pediatric bone marrow transplant recipients: a nonparametric population pharmacokinetic study. Ther Drug Monit 2007 Apr; 29 (2): 177-84 (Pubitemid 46716183)
24. 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 Feb; 15 (2): 231-41
25. Oechtering D, Schiltmeyer B, Hempel G, et al. Toxicity and pharmacokinetics of i.v. busulfan in children before stem cell transplantation. Anticancer Drugs 2005 Mar; 16 (3): 337-44 (Pubitemid 40283950)
26. Bergstrand M, Karlsson MO. Handling data below the limit of quantification in mixed effect models. AAPS J 2009 Jun; 11 (2): 371-80
27. Karlsson MO, Sheiner LB. The importance of modeling interoccasion variability in population pharmacokinetic analyses. J Pharmacokinet Biopharm 1993 Dec; 21 (6): 735-50 (Pubitemid 24052760)
28. Wilkins JJ. NONMEMory: a run management tool for NONMEM. Comput Methods Programs Biomed 2005 Jun; 78 (3): 259-67 (Pubitemid 40719673)
29. Karlsson MO, Savic RM. Diagnosing model diagnostics. Clin Pharmacol Ther 2007 Jul; 82 (1): 17-20 (Pubitemid 46944205)
30. Product information: Busulfex, busulfan. Otsuka America Pharmaceuticals, 2009 [online]. Available from URL: http://busulfex.com/06US11L-0497A- Busulfex%20USPI.pdf [Accessed 2012 Mar 7]
31. Hassan M, Oberg G, Bjorkholm M, et al. Influence of prophylactic anticonvulsant therapy on high-dose busulphan kinetics. Cancer Chemother Pharmacol 1993; 33 (3): 181-6 (Pubitemid 23361064)
32. Nilsson C, Aschan J, Hentschke P, et al. The effect of metronidazole on busulfan pharmacokinetics in patients undergoing hematopoietic stem cell transplantation. Bone Marrow Transplant 2003 Mar; 31 (6): 429-35 (Pubitemid 36511807)
33. Browning B, Thormann K, Donaldson A, et al. Busulfan dosing in children with BMIs ‡85% undergoing HSCT: a new optimal strategy. Biol Blood Marrow Transplant 2011; 17 (9): 1383-8
34. van den Broek MPH, Huitema AD, van Hasselt JG, et al. Lidocaine (lignocaine) dosing regimen based upon a population pharmacokinetic model for preterm and term neonates with seizures. Clin Pharmacokinet 2011; 50 (7): 461-9
35. Wang C, Peeters MY, Allegaert K, et al. A bodyweight-dependent allometric exponent for scaling clearance across the human life-span. Pharm Res. Epub 2012 Jan 28
36. Knibbe CA, Krekels EH, van den Anker JN, et al. Morphine glucuronidation in preterm neonates, infants and children younger than 3 years. Clin Pharmacokinet 2009; 48 (6): 371-85
37. Wahlby U, Jonsson EN, Karlsson MO. Comparison of stepwise covariate model building strategies in population pharmacokinetic-pharmacodynamic analysis. AAPS PharmSci 2002; 4 (4): E27
38. Krekels EH, van Hasselt JG, Tibboel D, et al. Systematic evaluation of the descriptive and predictive performance of paediatric morphine population models. Pharm Res 2010 Dec 14; 28 (4): 797-811
39. Lindbom L, Pihlgren P, Jonsson EN. PsN-Toolkit-a collection of computer intensive statistical methods for non-linear mixed effect modeling using NONMEM. Comput Methods Programs Biomed 2005 Sep; 79 (3): 241-57 (Pubitemid 41196689)
40. Pulsipher MA, Boucher KM, Wall D, et al. Reduced-intensity allogeneic transplantation in pediatric patients ineligible for myeloablative therapy: results of the Pediatric Blood and Marrow Transplant Consortium Study ONC0313. Blood 2009 Aug 13; 114 (7): 1429-36
41. 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 Jun 3; 115 (22): 4597-604
42. Busilvex, busulfan [product information]. Idron, France: Pierre Fabre Médicament Production, site Aquitaine Pharm International, 2007
43. Vassal G, Michel G, Esperou 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 (1): 113-23 (Pubitemid 47512633)
44. Gordon N, Mullen CA, Tran H, et al. Fludarabine and once-daily intravenous busulfan for allogeneic bone marrow transplantation for Chediak-Higashi syndrome. J Pediatr Hematol Oncol 2003 Oct; 25 (10): 824-6 (Pubitemid 37238778)
45. 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 May 1; 79 (9): 2475-9
46. de Lima M, Couriel D, Thall PF, et al. Once-daily intravenous busulfan and fludarabine: clinical and pharmacokinetic results of a myeloablative, reducedtoxicity conditioning regimen for allogeneic stem cell transplantation inAML and MDS. Blood 2004 Aug 1; 104 (3): 857-64 (Pubitemid 38970585)
47. Gibbs JP, Murray G, Risler L, et al. Age-dependent tetrahydrothiophenium ion formation in young children and adults receiving high-dose busulfan. Cancer Res 1997 Dec 15; 57 (24): 5509-16 (Pubitemid 28006757)
48. Gibbs JP, Liacouras CA, Baldassano RN, et al. Up-regulation of glutathione S-transferase activity in enterocytes of young children. Drug Metab Dispos 1999 Dec; 27 (12): 1466-9
49. Kanamori M, Takahashi H, Echizen H. Developmental changes in the liver weight-and body weight-normalized clearance of theophylline, phenytoin and cyclosporine in children. Int J Clin Pharmacol Ther 2002 Nov; 40 (11): 485-92 (Pubitemid 35331932)
50. Ginsberg G, Hattis D, Sonawane B, et al. Evaluation of child/adult pharmacokinetic differences from a database derived from the therapeutic drug literature. Toxicol Sci 2002 Apr; 66 (2): 185-200 (Pubitemid 34285113)
51. Abdi Z.D, Paci A, Moshous D, et al. Screening for potential covariates influencing the pharmacokinetics of intravenous busulfan: results from a large pediatric cohort undergoing hematopoietic stem-cell transplantation [online]. Available from URL: http://ash.confex.com/ash/2010/webprogram/Paper 28407.html [Accessed 2011 Sep 15]
52. Anderson BJ, Holford NH. Mechanism-based concepts of size and maturity in pharmacokinetics. Annu Rev Pharmacol Toxicol 2008; 48: 303-32 (Pubitemid 351738156)
53. Orrenius S, Moldeus P. The multiple roles of glutathione in drug metabolism. Trends Pharmacol Sci 1984 Oct; 5: 432-5
54. Yeh RF, Pawlikowski MA, Blough DK, et al. Accurate targeting of daily intravenous busulfan with 8-hour blood sampling in hospitalized adult hematopoietic cell transplant recipients. Biol Blood Marrow Transplant 2012; 18 (2): 265-72
55. Andersson BS, de LM, Thall PF, 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 inAML/MDS. Biol Blood Marrow Transplant 2008 Jun; 14 (6): 672-84 (Pubitemid 351664062)
56. Russell JA, Tran HT, 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 (9): 468-76 (Pubitemid 35174820)
57. Lee JW, Kang HJ, Lee SH, et al. Highly variable pharmacokinetics of oncedaily intravenous busulfan when combined with fludarabine in pediatric patients: phase I clinical study for determination of optimal once-daily busulfan dose using pharmacokinetic modeling. Biol Blood Marrow Transplant. Epub 2011 Dec 7
58. Boelens JJ, Bartelink IH, Flinsenberg TWH, et al. Busulfan + fludarabine: an effective and low toxic conditioning regimen prior to HSCT in children with either malignant or non-malignant diseases [abstract]. Biol Blood Marrow Transplant 2011; 18 (2): s229
59. 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 Feb; 14 (2): 220-8
60. Gü ngö r T, Schanz U, Seger R, et al. Successful half-dose busulfan/full-dose fludarabine based reduced intensity conditioning in high-risk pediatric and adult chronic granulomatous disease (CGD) patients. Biol Blood Marrow Transplant 2010 Jan 1; 16 (1): S181-2
61. Worth LL, Andersson BS, KazerooniR, et al. Thiotepa (TT), busulfan (BU) and clofarabine (CLO) as conditioning therapy for allogeneic hematopoetic stemcell transplant for patients with high risk malignancies: early response and engraftment data. Biol Blood Marrow Transplant 2010 Jan 1; 16 (1): S271-2
62. Andersson BS, de Lima M, Valdez BC, et al. Clofarabine +-fludarabine with IV busulfan and allogeneic stem cell transplantation for relapsed, refractory myeloid leukemia (ML) and MDS. Biol Blood Marrow Transplant 2010 Jan 1; 16 (1): S271-2