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

References

• van Besien K, Artz A, Smith S, et al Fludarabine, melphalan, and alemtuzumab conditioning in adults with standard-risk advanced acute myeloid leukemia and myelodysplastic syndrome. J Clin Oncol 2005;23:5728–5738.
   PubMed Web of Science ®Google Scholar
• Armitage JO. Bone marrow transplantation. N Engl J Med 1994;330:827–838.
   PubMed Web of Science ®Google Scholar
• Galton DA. Myleran in chronic myeloid leukaemia; results of treatment. Lancet 1953;264:208–213.
   PubMed Web of Science ®Google Scholar
• Canellos GP, Griffin JD. Chronic granulocytic leukemia: the heterogeneity of stem cell differentiation within a single disease entity. Semin Oncol 1985;12:281–288.
   PubMed Web of Science ®Google Scholar
• 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.
   PubMed Web of Science ®Google Scholar
• 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.
   PubMed Web of Science ®Google Scholar
• Coppell JA, Brown SA, Perry DJ. Veno-occlusive disease: cytokines, genetics, and haemostasis. Blood Rev 2003;17:63–70.
   PubMed Web of Science ®Google Scholar
• 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.
   PubMed Web of Science ®Google Scholar
• 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.
   PubMed Web of Science ®Google Scholar
• Essell JH, Thompson JM, Harman GS, et al Marked increase in veno-occlusive disease of the liver associated with methotrexate use for graft-versus-host disease prophylaxis in patients receiving busulfan/cyclophosphamide. Blood 1992;79:2784–2788.
   PubMed Web of Science ®Google Scholar
• McDonald GB, Slattery JT, Bouvier ME, et al Cyclophosphamide metabolism, liver toxicity, and mortality following hematopoietic stem cell transplantation. Blood 2003;101:2043–2048.
   PubMed Web of Science ®Google Scholar
• Dulley FL, Kanfer EJ, Appelbaum FR, et al Venocclusive disease of the liver after chemoradiotherapy and autologous bone marrow transplantation. Transplantation 1987;43:870–873.
   PubMed Web of Science ®Google Scholar
• Moscardo F, Urbano-Ispizua A, Sanz GF, et al Positive selection for CD34+ reduces the incidence and severity of veno-occlusive disease of the liver after HLA-identical sibling allogeneic peripheral blood stem cell transplantation. Exp Hematol 2003;31:545–550.
   PubMed Web of Science ®Google Scholar
• McDonald GB, Hinds MS, Fisher LD, et al Veno-occlusive disease of the liver and multiorgan failure after bone marrow transplantation: a cohort study of 355 patients. Ann Intern Med 1993;118:255–267.
   PubMed Web of Science ®Google Scholar
• 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;8:468–476.
   PubMed Web of Science ®Google Scholar
• Grochow LB. Parenteral busulfan: is therapeutic monitoring still warranted? Biol Blood Marrow Transplant 2002;8:465–467.
   PubMed Web of Science ®Google Scholar
• Slattery JT, Risler LJ. Therapeutic monitoring of busulfan in hematopoietic stem cell transplantation. Ther Drug Monit 1998;20:543–549.
   PubMed Web of Science ®Google Scholar
• Eberly AL, Anderson GD, Bubalo JS, McCune JS. Optimal prevention of seizures induced by high-dose busulfan. Pharmacotherapy 2008;28:1502–1510.
   PubMed Web of Science ®Google Scholar
• Kline J, Pollyea DA, Stock W, et al Pre-transplant ganciclovir and post transplant high-dose valacyclovir reduce CMV infections after alemtuzumab-based conditioning. Bone Marrow Transplant 2006;37:307–310.
   PubMed Web of Science ®Google Scholar
• Vaughan WP, Carey D, Perry S, Westfall AO, Salzman DE. A limited sampling strategy for pharmacokinetic directed therapy with intravenous busulfan. Biol Blood Marrow Transplant 2002;8:619–624.
   PubMed Web of Science ®Google Scholar
• Bostrom B, Enockson K, Johnson A, Bruns A, Blazar B. Plasma pharmacokinetics of high-dose oral busulfan in children and adults undergoing bone marrow transplantation. Pediatr Transplant 2003;7(Suppl. 3):12–18.
   PubMed Web of Science ®Google Scholar
• Faries D. Practical modifications of the continual reassessment method for phase I cancer clinical trials. J Biopharm Stat 1994;4:147–164.
   PubMedGoogle Scholar
• Goodman SN, Zahurak ML, Piantadosi S. Some practical improvements in the continual reassessment method for phase I studies. Stat Med 1995;14:1149–1161.
   PubMed Web of Science ®Google Scholar
• Vassal G, Re M, Gouyette A. Gas chromatographic-mass spectrometric assay for busulfan in biological fluids using a deuterated internal standard. J Chromatogr A 1988;428:357–361.
   PubMed Web of Science ®Google Scholar
• Bland JM, Altman DG. Statistical methods for assessing agreement between two methods of clinical measurement. Lancet 1986;1:307–310.
   PubMed Web of Science ®Google Scholar
• Dewe W. Review of statistical methodologies used to compare (bio)assays. J Chromatogr B Analyt Technol Biomed Life Sci 2009;877:2208–2213.
   PubMed Web of Science ®Google Scholar
• de Lima M, Couriel D, Thall PF, 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.
   PubMed Web of Science ®Google Scholar
• 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;309:1347–1353.
   PubMed Web of Science ®Google Scholar
• 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.
   PubMed Web of Science ®Google Scholar
• Beri R, Chunduri S, Sweiss K, et al Reliability of a pretransplant i.v. BU test dose performed 2 weeks before myeloablative FluBu conditioning regimen. Bone Marrow Transplant 2010;45:249–253.
   PubMed Web of Science ®Google Scholar
• Russell JA, Kangarloo SB. Therapeutic drug monitoring of busulfan in transplantation. Curr Pharm Des 2008;14:1936–1949.
   PubMed Web of Science ®Google Scholar
• 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 first dose strategy. Anticancer Drugs 2004;15:453–459.
   PubMed Web of Science ®Google Scholar
• Walko CM, McLeod H. Pharmacogenomic progress in individualized dosing of key drugs for cancer patients. Nat Clin Pract Oncol 2009;6:153–162.
   PubMedGoogle Scholar
• Toh HC, McAfee SL, Sackstein R, Cox BF, Colby C, Spitzer TR. Late onset veno-occlusive disease following high-dose chemotherapy and stem cell transplantation. Bone Marrow Transplant 1999;24:891–895.
   PubMed Web of Science ®Google Scholar
• van Besien K, Kunavakkam R, Rondon G, et al Fludarabine-melphalan conditioning for AML and MDS: alemtuzumab reduces acute and chronic GVHD without affecting long-term outcomes. Biol Blood Marrow Transplant 2009;15:610–617.
   PubMed Web of Science ®Google Scholar
• 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.
   PubMed Web of Science ®Google Scholar
• Platzbecker U, von Bonin M, Goekkurt E, et al Graft-versus-host disease prophylaxis with everolimus and tacrolimus is associated with a high incidence of sinusoidal obstruction syndrome and microangiopathy: results of the EVTAC trial. Biol Blood Marrow Transplant 2009;15:101–108.
   PubMed Web of Science ®Google Scholar
• Wood WA, Walko CM, Rao KV, et al Identification of an AUC of 7603 umol-min/hr per day x 4 days as the maximum tolerated dose (MTD) of IV continuous infusion (CI) busulfan (Bu) with fixed dose fludarabine (Flu) in a pharmacokinetically-based dose phase I study in patients (pts) with hematologic malignancies undergoing allogeneic stem cell transplantation. Biol Blood Marrow Transplant 2009;15(Suppl.): Abstract 270.
   Google Scholar