Association between Busulfan Exposure and Outcome in Children Receiving Intravenous Busulfan before Hematologic Stem Cell Transplantation

Biology of Blood and Marrow Transplantation

Volumn 15, Issue 2, 2009, Pages 231-241

  Bartelink, Imke H ^a   Bredius, Robert G M ^b   Belitser, Svetlana V ^c   Suttorp, Marit M ^b   Bierings, Marc ^a   Knibbe, Catherijne A J ^d   Egeler, Maarten ^b   Lankester, Arjan C ^b   Egberts, Atoine C G ^a   Zwaveling, Juliette ^b   Boelens, Jaap Jan ^a  

^a UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

^b LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

^c UTRECHT UNIVERSITY   (Netherlands)

^d LEIDEN UNIVERSITY   (Netherlands)

Author keywords

Allogeneic stem cell transplantation; Busulfan; Pediatrics; Therapeutic drug monitoring

Indexed keywords

ALEMTUZUMAB; BUSULFAN; CLONAZEPAM; CYCLOPHOSPHAMIDE; CYCLOSPORIN A; DEFIBROTIDE; DIURETIC AGENT; ETOPOSIDE; FLUDARABINE; MELPHALAN; METHOTREXATE; ONDANSETRON; PREDNISONE; RECOMBINANT GRANULOCYTE COLONY STIMULATING FACTOR; THYMOCYTE ANTIBODY;

ACUTE GRAFT VERSUS HOST DISEASE; ADOLESCENT; ADULT; AREA UNDER THE CURVE; ARTICLE; BRONCHIOLITIS OBLITERANS; CHILD; CONTROLLED STUDY; CORRELATIONAL STUDY; DISEASE FREE SURVIVAL; DISEASE SEVERITY; DOSE RESPONSE; DRUG BLOOD LEVEL; DRUG DOSE REDUCTION; DRUG EFFICACY; DRUG EXPOSURE; DRUG SAFETY; DRUG USE; FEMALE; FLUID RESUSCITATION; FOLLOW UP; HEMATOLOGIC DISEASE; HEMATOLOGIC MALIGNANCY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; HUMAN CELL; IDIOPATHIC DISEASE; INFANT; LUNG TOXICITY; MAJOR CLINICAL STUDY; MALE; MOUTH INFLAMMATION; MUCOSA INFLAMMATION; OUTCOME ASSESSMENT; OVERALL SURVIVAL; PNEUMONIA; RETROSPECTIVE STUDY; RISK ASSESSMENT; RISK FACTOR; SURVIVAL TIME; TREATMENT INDICATION; VEIN OCCLUSION;

ADOLESCENT; AREA UNDER CURVE; BUSULFAN; CHILD; CHILD, PRESCHOOL; DRUG INTERACTIONS; GRAFT VS HOST DISEASE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; INCIDENCE; INFANT; MELPHALAN; MUCOSITIS; RETROSPECTIVE STUDIES; SURVIVAL ANALYSIS; TREATMENT OUTCOME; YOUNG ADULT;

EID: 58249143575     PISSN: 10838791     EISSN: 15236536     Source Type: Journal    
DOI: 10.1016/j.bbmt.2008.11.022     Document Type: Article

References (53)

1. Bolinger A.M., Zangwill A.B., Slattery J.T., et al. An evaluation of engraftment, toxicity and busulfan concentration in children receiving bone marrow transplantation for leukemia or genetic disease. Bone Marrow Transplant 25 (2000) 925-930
2. Slattery J.T., Sanders J.E., Buckner C.D., et al. Graft-rejection and toxicity following bone marrow transplantation in relation to busulfan pharmacokinetics. Bone Marrow Transplant 16 (1995) 31-42
3. Tran H.T., Madden T., Petropoulos D., et al. Individualizing high-dose oral busulfan: prospective dose adjustment in a pediatric population undergoing allogeneic stem cell transplantation for advanced hematologic malignancies. Bone Marrow Transplant 26 (2000) 463-470
4. 2 is safe and effective in children of all ages. Bone Marrow Transplant 34 (2004) 197-205
5. 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 12 (1994) 1217-1222
6. Hassan M., Ljungman P., Bolme P., et al. Busulfan bioavailability. Blood 84 (1994) 2144-2150
7. 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 28 (2001) 743-751
8. Andersson B.S., Thall P.F., 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 8 (2002) 477-485
9. Andersson B.S., 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 8 (2002) 145-154
10. Cremers S., Schoemaker R., Bredius R., et al. Pharmacokinetics of intravenous busulfan in children prior to stem cell transplantation. Br J Clin Pharmacol 53 (2002) 386-389
11. Nath C.E., Earl J.W., 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 66 (2008) 50-59
12. Nguyen L., Fuller D., Lennon S., et al. IV busulfan in pediatrics: a novel dosing to improve safety/efficacy for hematopoietic progenitor cell transplantation recipients. Bone Marrow Transplant 33 (2004) 979-987
13. Schechter T., Finkelstein Y., Doyle J., et al. Pharmacokinetic disposition and clinical outcomes in infants and children receiving intravenous busulfan for allogeneic hematopoietic stem cell transplantation. Biol Blood Marrow Transplant 13 (2007) 307-314
14. Takama H., Tanaka H., Nakashima D., et al. Population pharmacokinetics of intravenous busulfan in patients undergoing hematopoietic stem cell transplantation. Bone Marrow Transplant 37 (2006) 345-351
15. Tran H.T., Petropoulos D., Worth L.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 10 (2004) 805-812
16. 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 61 (2008) 113-123
17. Fernandez H.F., Tran H.T., Albrecht F., et al. 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 8 (2002) 486-492
18. 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 8 (2002) 468-476
19. 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 13 (2007) 1095-1105
20. 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 17 (1996) 225-230
21. Grochow L.B., Jones R.J., Brundrett R.B., et al. Pharmacokinetics of busulfan: correlation with veno-occlusive disease in patients undergoing bone marrow transplantation. Cancer Chemother Pharmacol 25 (1989) 55-61
22. Ljungman P., Hassan M., Bekassy A.N., et al. High busulfan concentrations are associated with increased transplant-related mortality in allogeneic bone marrow transplant patients. Bone Marrow Transplant 20 (1997) 909-913
23. McCune J.S., Gibbs J.P., and Slattery J.T. Plasma concentration monitoring of busulfan: does it improve clinical outcome?. Clin Pharmacokinet 39 (2000) 155-165
24. 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 89 (1997) 3055-3060
25. Bolinger A.M., Zangwill A.B., Slattery J.T., et al. Target dose adjustment of busulfan in pediatric patients undergoing bone marrow transplantation. Bone Marrow Transplant 28 (2001) 1013-1018
26. Bouligand J., Boland I., Valteau-Couanet D., et al. In children and adolescents, the pharmacodynamics of high-dose busulfan is dependent on the second alkylating agent used in the combined regimen (melphalan or thiotepa). Bone Marrow Transplant 32 (2003) 979-986
27. Booth B.P., Rahman A., Dagher R., et al. Population pharmacokinetic-based dosing of intravenous busulfan in pediatric patients. J Clin Pharmacol 47 (2007) 101-111
28. Zwaveling J., Bredius R.G., Cremers S.C., 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 35 (2005) 17-23
29. Zwaveling J., den Hartigh J., Lankester A.C., et al. Once-daily intravenous busulfan in children prior to stem cell transplantation: study of pharmacokinetics and early clinical outcomes. Anticancer Drugs 17 (2006) 1099-1105
30. Bartelink I.H., Bredius R.G., Ververs T.T., 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 14 (2008) 88-98
31. Proost J.H., and Meijer D.K. MW/Pharm, an integrated software package for drug dosage regimen calculation and therapeutic drug monitoring. Comput Biol Med 22 (1992) 155-163
32. Bearman S.I. The syndrome of hepatic veno-occlusive disease after marrow transplantation. Blood 85 (1995) 3005-3020
33. Corbacioglu S., and Lacobelli M. VOD-DF study, a prospective study of the incidence and outcome of venoocclusive disease (VOD) with the prophylactic use of defibrotide in pediatric stem cell transplantation. Version 4.0 (2004)
34. Glucksberg H., Storb R., Fefer A., et al. Clinical manifestations of graft-versus-host disease in human recipients of marrow from HL-A-matched sibling donors. Transplantation 18 (1974) 295-304
35. Hensley M.L., Hagerty K.L., Kewalramani T., et al. American Society of Clinical Oncology 2008 Clinical Practice Guideline Update: Use of Chemotherapy and Radiation Therapy Protectants. J Clin Oncol (2008)
36. 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 14 (2008) 220-228
37. Carreras E., Rosinol L., Terol M.J., et al. Veno-occlusive disease of the liver after high-dose cytoreductive therapy with busulfan and melphalan for autologous blood stem cell transplantation in multiple myeloma patients. Biol Blood Marrow Transplant 13 (2007) 1448-1454
38. DeLeve L.D., and Wang X. Role of oxidative stress and glutathione in busulfan toxicity in cultured murine hepatocytes. Pharmacology 60 (2000) 143-154
39. Jones R.J., Lee K.S., and Beschorner W.E. Venocclusive disease of the liver following bone marrow transplantation. Transplantation 44 (1975) 778-783
40. McDonald G.B., Hinds M.S., Fisher L.D., et al. Veno-occlusive disease of the liver and multiorgan failure after bone marrow transplantation: a cohort study of 355 patients. Ann Intern Med 118 (1993) 255-267
41. Boelens J.J., Wynn R.F., O'Meara A., et al. Outcomes of hematopoietic stem cell transplantation for Hurler's syndrome in Europe: a risk factor analysis for graft failure. Bone Marrow Transplant 40 3 (2007) 225-233
42. Cesaro S., Pillon M., Talenti E., et al. A prospective survey on incidence, risk factors and therapy of hepatic veno-occlusive disease in children after hematopoietic stem cell transplantation. Haematologica 90 (2005) 1396-1404
43. Tiedemann K., Waters K.D., Tauro G.P., et al. Results of intensive therapy in childhood acute myeloid leukemia, incorporating high-dose melphalan and autologous bone marrow transplantation in first complete remission. Blood 82 (1993) 3730-3738
44. Chae Y.S., Sohn S.K., Kim J.G., et al. New myeloablative conditioning regimen with fludarabine and busulfan for allogeneic stem cell transplantation: comparison with BuCy2. Bone Marrow Transplant 40 (2007) 541-547
45. Gandhi V., and Plunkett W. Cellular and clinical pharmacology of fludarabine. Clin Pharmacokinet 41 (2002) 93-103
46. Iravani M., Evazi M.R., Mousavi S.A., et al. Fludarabine and busulfan as a myeloablative conditioning regimen for allogeneic stem cell transplantation in high- and standard-risk leukemic patients. Bone Marrow Transplant 40 (2007) 105-110
47. El K.N., Legouvello S., Joseph C.M., et al. High-resolution HLA class I and II typing and CTLp frequency in unrelated donor transplantation: a single-institution retrospective study of 69 BMTs. Bone Marrow Transplant 27 (2001) 35-43
48. Yu L.C., Wall D.A., Sandler E., et al. Unrelated cord blood transplant experience by the pediatric blood and marrow transplant consortium. Pediatr Hematol Oncol 18 (2001) 235-245
49. Flomenberg N., Baxter-Lowe L.A., Confer D., et al. Impact of HLA class I and class II high-resolution matching on outcomes of unrelated donor bone marrow transplantation: HLA-C mismatching is associated with a strong adverse effect on transplantation outcome. Blood 104 (2004) 1923-1930
50. Beatty P.G., Anasetti C., Hansen J.A., et al. Marrow transplantation from unrelated donors for treatment of hematologic malignancies: effect of mismatching for one HLA locus. Blood 81 (1993) 249-253
51. Davies S.M., Shu X.O., Blazar B.R., et al. Unrelated donor bone marrow transplantation: influence of HLA-A and -B incompatibility on outcome. Blood 86 (1995) 1636-1642
52. Spencer A., Brookes P.A., Kaminski E., et al. Cytotoxic T lymphocyte precursor frequency analyses in bone marrow transplantation with volunteer unrelated donors: value in donor selection. Transplantation 59 (1995) 1302-1308
53. Dupuis L.L., Schechter T., Ansari M., et al. Evaluation of 2 i.v. busulfan pediatric dosing guidelines. Biol Blood Marrow Transplant 14 (2008) 167