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Research Article

Pre-formulation investigations for establishing a protocol for treosulfan handling and activation

ORCID Icon, , ORCID Icon, & ORCID Icon
Pages 639-648 | Received 19 May 2018, Accepted 20 Nov 2018, Published online: 10 Jan 2019

References

  • Abedi-Valugerdi M, Wolfsberger J, Pillai PR, Zheng W, Sadeghi B, Zhao Y, Hassan M. 2016. Suppressive effects of low-dose 5-fluorouracil, busulfan or treosulfan on the expansion of circulatory neutrophils and myeloid derived immunosuppressor cells in tumor-bearing mice. Int Immunopharmacol. 40:41–49.
  • Beelen DW, Trenschel R, Casper J, Freund M, Hilger RA, Scheulen ME, Basara N, Fauser AA, Hertenstein B, Mylius HA. 2005. Dose-escalated treosulphan in combination with cyclophosphamide as a new preparative regimen for allogeneic haematopoietic stem cell transplantation in patients with an increased risk for regimen-related complications. Bone Marrow Transplant. 35:233–241.
  • Chekerov R, Kaltenecker G, Reichert D, Gohler T, Klare P, Oskay-Ozcelik G, Sauer U, Wischnik A, Vehling-Kaiser U, Becker M. 2015. Treosulfan in the treatment of advanced ovarian cancer – results of a German Multicenter Non-interventional Study. Anticancer Res. 35:6869–6875.
  • Danielak D, Twardosz J, Kasprzyk A, Wachowiak J, Kałwak K, Główka F. 2018. Population pharmacokinetics of treosulfan and development of a limited sampling strategy in children prior to hematopoietic stem cell transplantation. Eur J Clin Pharmacol. 74:79–89.
  • Duncan ID, Clayton LA. 1985. Combination chemotherapy of ovarian carcinoma with cisplatinum and treosulfan – a phase II study. Br J Obstet Gynaecol. 92:762–767.
  • Feit PW, Rastrup-Andersen N, Matagne R. 1970. Studies on epoxide formation from (2S,3S)-threitol 1,4-bismethanesulfonate. The preparation and biological activity of (2S,3S)-1,2-epoxy-3,4-butanediol 4-methanesulfonate. J Med Chem. 13:1173–1175.
  • Gil L, Styczynski J, Dytfeld D, Debski R, Kazmierczak M, Kolodziej B, Rafinska B, Kubicka M, Nowicki A, Komarnicki M, et al. 2007. Activity of bortezomib in adult de novo and relapsed acute myeloid leukemia. Anticancer Res. 27:4021–4025.
  • Glowka F, Kasprzyk A, Romanski M, Wrobel T, Wachowiak J, Szpecht D, Kalwak K, Wiela-Hojenska A, Dziatkiewicz P, Tezyk A. 2015. Pharmacokinetics of treosulfan and its active monoepoxide in pediatric patients after intravenous infusion of high-dose treosulfan prior to HSCT. Eur J Pharm Sci: Off J Eur Federation Pharm Sci. 68:87–93.
  • Główka FK, Karaźniewicz-Łada M, Grund G, Wróbel T, Wachowiak J. 2008. Pharmacokinetics of high-dose i.v. treosulfan in children undergoing treosulfan-based preparative regimen for allogeneic haematopoietic SCT. Bone Marrow Transplant. 42(Suppl 2):S67–S70.
  • Glowka FK, Lada MK, Grund G, Wachowiak J. 2007. Determination of treosulfan in plasma and urine by HPLC with refractometric detection; pharmacokinetic studies in children undergoing myeloablative treatment prior to haematopoietic stem cell transplantation. J Chromatogr B: Analyt Technol Biomed Life Sci. 850:569–574.
  • Glowka FK, Romanski M, Tezyk A, Zaba C. 2013. Direct high-performance liquid chromatography method with refractometric detection designed for stability studies of treosulfan and its biologically active epoxy-transformers. J Pharm Biomed Anal. 72:145–149.
  • Greystoke B, Bonanomi S, Carr TF, Gharib M, Khalid T, Coussons M, Jagani M, Naik P, Rao K, Goulden N, et al. 2008. Treosulfan-containing regimens achieve high rates of engraftment associated with low transplant morbidity and mortality in children with non-malignant disease and significant co-morbidities. Br J Haematol. 142:257–262.
  • Gropp M, Meier W, Hepp H. 1998. Treosulfan as an effective second-line therapy in ovarian cancer. Gynecol Oncol. 71:94–98.
  • Gyurkocza B, Gutman J, Nemecek ER, Bar M, Milano F, Ramakrishnan A, Scott B, Fang M, Wood B, Pagel JM. 2014. Treosulfan, fludarabine, and 2-Gy total body irradiation followed by allogeneic hematopoietic cell transplantation in patients with myelodysplastic syndrome and acute myeloid leukemia. Biol Blood Marrow Transplant. 20:549–555.
  • Hartley JA, O'Hare CC, Baumgart J. 1999. DNA alkylation and interstrand cross-linking by treosulfan. Br J Cancer. 79:264–266.
  • Hilger RA, Harstrick A, Eberhardt W, Oberhoff C, Skorzec M, Baumgart J, Seeber S, Scheulen ME. 1998. Clinical pharmacokinetics of intravenous treosulfan in patients with advanced solid tumors. Cancer Chemother Pharmacol. 42:99–104.
  • Hilger RA, Jacek G, Oberhoff C, Kredtke S, Baumgart J, Seeber S, Scheulen ME. 2000. Investigation of bioavailability and pharmacokinetics of treosulfan capsules in patients with relapsed ovarian cancer. Cancer Chemother Pharmacol. 45:483–488.
  • Holowiecki J, Giebel S, Wojnar J, Krawczyk-Kulis M, Markiewicz M, Holowiecka-Goral A, Freund M, Casper J. 2008. Treosulfan and fludarabine low-toxicity conditioning for allogeneic haematopoietic stem cell transplantation in chronic myeloid leukaemia. Br J Haematol. 142:284–292.
  • Holtick U, Herling M, Pflug N, Chakupurakal G, Leitzke S, Wolf D, Hallek M, Scheid C, Chemnitz JM. 2017. Similar outcome after allogeneic stem cell transplantation with a modified FLAMSA conditioning protocol substituting 4 Gy TBI with treosulfan in an elderly population with high-risk AML. Ann Hematol. 96:479–487.
  • Housman G, Byler S, Heerboth S, Lapinska K, Longacre M, Snyder N, Sarkar S. 2014. Drug resistance in cancer: an overview. Cancers (Basel). 6:1769–1792.
  • Huang J, Bathena SP, Alnouti Y. 2010. Metabolite profiling of praziquantel and its analogs during the analysis of in vitro metabolic stability using information-dependent acquisition on a hybrid triple quadrupole linear ion trap mass spectrometer. Drug Metab Pharmacokinet. 25:487–499.
  • Koyyalamudi SR, Kuzhiumparambil U, Nath CE, Byrne JA, Fraser CJ, O'Brien TA, Earl JW, Shaw PJ. 2016. Development and validation of a high pressure liquid chromatography-UV method for the determination of treosulfan and its epoxy metabolites in human plasma and its application in pharmacokinetic studies. J Chromatogr Sci. 54:326–333.
  • Lin JH, Lu AY. 1998. Inhibition and induction of cytochrome P450 and the clinical implications. Clin Pharmacokinet. 35:361–390.
  • Mahner S, Oskay-Ozcelik G, Heidrich-Lorsbach E, Fuxius S, Sommer H, Klare P, Belau A, Ruhmland B, Heuser T, Kolbl H. 2012. A prospective multicenter study of treosulfan in elderly patients with recurrent ovarian cancer: results of a planned safety analysis. J Cancer Res Clin Oncol. 138:1413–1419.
  • Martins LM, Mesner PW, Kottke TJ, Basi GS, Sinha S, Tung JS, Svingen PA, Madden BJ, Takahashi A, McCormick DJ, et al. 1997. Comparison of caspase activation and subcellular localization in HL-60 and K562 cells undergoing etoposide-induced apoptosis. Blood. 90:4283–4296.
  • Maschan M, Shelikhova L, Ilushina M, Kurnikova E, Boyakova E, Balashov D, Persiantseva M, Skvortsova Y, Laberko A, Muzalevskii Y, et al. 2016. TCR-alpha/beta and CD19 depletion and treosulfan-based conditioning regimen in unrelated and haploidentical transplantation in children with acute myeloid leukemia. Bone Marrow Transplant. 51:668–674.
  • Mohanan E, Panetta JC, Lakshmi KM, Edison ES, Korula A, Na F, Abraham A, Viswabandya A, George B, Mathews V, et al. 2018. Pharmacokinetics and pharmacodynamics of treosulfan in patients with Thalassemia major undergoing allogeneic hematopoietic stem cell transplantation. Clin Pharmacol Therapeut. 104:575–583.
  • Munkelt D, Koehl U, Kloess S, Zimmermann SY, Kalaaoui RE, Wehner S, Schwabe D, Lehrnbecher T, Schubert R, Kreuter J, et al. 2008. Cytotoxic effects of treosulfan and busulfan against leukemic cells of pediatric patients. Cancer Chemother Pharmacol. 62:821–830.
  • Nagler A, Labopin M, Beelen D, Ciceri F, Volin L, Shimoni A, Foa R, Milpied N, Peccatori J, Polge E, et al. 2017. Long-term outcome after a treosulfan-based conditioning regimen for patients with acute myeloid leukemia: a report from the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation. Cancer. 123:2671–2679.
  • Nemecek ER, Guthrie KA, Sorror ML, Wood BL, Doney KC, Hilger RA, Scott BL, Kovacsovics TJ, Maziarz RT, Woolfrey AE, et al. 2011. Conditioning with treosulfan and fludarabine followed by allogeneic hematopoietic cell transplantation for high-risk hematologic malignancies. Biol Blood Marrow Transplant: J Am Soc Blood Marrow Transplant. 17:341–350.
  • O'Hare T, Eide CA, Deininger MW. 2007. Bcr-Abl kinase domain mutations, drug resistance, and the road to a cure for chronic myeloid leukemia. Blood. 110:2242–2249.
  • Remberger M, Torlen J, Serafi IE, Garming-Legert K, Bjorklund A, Ljungman P, Sundin M, Hassan M, Mattsson J. 2017. Toxicological effects of fludarabine and treosulfan conditioning before allogeneic stem-cell transplantation. Int J Hematol. 106:471–475.
  • Romański M, Teżyk A, Zaba C, Główka FK. 2014. Rapid and sensitive liquid chromatography-tandem mass spectrometry method for determination of protein-free pro-drug treosulfan and its biologically active monoepoxy-transformer in plasma and brain tissue. Talanta. 127:123–132.
  • Romański M, Urbaniak B, Kokot Z, Główka FK. 2015. Activation of prodrug treosulfan at pH 7.4 and 37 °C accompanied by hydrolysis of its active epoxides: kinetic studies with clinical relevance. J Pharm Sci. 104:4433–4442.
  • Sakellari I, Mallouri D, Gavriilaki E, Batsis I, Kaliou M, Constantinou V, Papalexandri A, Lalayanni C, Vadikolia C, Athanasiadou A. 2017. Survival advantage and comparable toxicity in reduced-toxicity treosulfan-based versus reduced-intensity busulfan-based conditioning Regimen in Myelodysplastic Syndrome and acute myeloid leukemia patients after allogeneic hematopoietic cell transplantation. Biol Blood Marrow Transplan. 23:445–451.
  • Sanchez-Garcia I, Martin-Zanca D. 1997. Regulation of Bcl-2 gene expression by BCR-ABL is mediated by Ras. J Mol Biol. 267:225–228.
  • Scheulen ME, Hilger RA, Oberhoff C, Casper J, Freund M, Josten KM, Bornhauser M, Ehninger G, Berdel WE, Baumgart J. 2000. Clinical phase I dose escalation and pharmacokinetic study of high-dose chemotherapy with treosulfan and autologous peripheral blood stem cell transplantation in patients with advanced malignancies. Clin Cancer Res: Off J Am Assoc Cancer Res. 6:4209–4216.
  • Schmidmaier R, Oellerich M, Baumgart J, Emmerich B, Meinhardt G. 2004. Treosulfan-induced apoptosis in acute myeloid leukemia cells is accompanied by translocation of protein kinase C delta and enhanced by bryostatin-1. Exp Hematol. 32:76–86.
  • Sehouli J, Tome O, Dimitrova D, Camara O, Runnebaum IB, Tessen HW, Rautenberg B, Chekerov R, Muallem MZ, Lux MP, et al. 2017. A phase III, open label, randomized multicenter controlled trial of oral versus intravenous treosulfan in heavily pretreated recurrent ovarian cancer: a study of the North-Eastern German Society of Gynecological Oncology (NOGGO). J Cancer Res Clin Oncol. 143:541–550.
  • Shah VP, Midha KK, Dighe S, McGilveray IJ, Skelly JP, Yacobi A, Layloff T, Viswanathan CT, Cook CE, McDowall RD, et al. 1991. Analytical methods validation: bioavailability, bioequivalence and pharmacokinetic studies. Conference report. Eur J Drug Metab Pharmacokinet. 16:249–255.
  • Shah VP, Midha KK, Findlay JW, Hill HM, Hulse JD, McGilveray IJ, McKay G, Miller KJ, Patnaik RN, Powell ML, et al. 2000. Bioanalytical method validation-a revisit with a decade of progress. Pharm Res. 17:1551–1557.
  • Sjoo F, Hassan Z, Abedi-Valugerdi M, Griskevicius L, Nilsson C, Remberger M, Aschan J, Concha H, Gaughan U, Hassan M. 2006. Myeloablative and immunosuppressive properties of treosulfan in mice. Exp Hematol. 34:115–121.
  • Slatter MA, Rao K, Amrolia P, Flood T, Abinun M, Hambleton S, Nademi Z, Goulden N, Davies G, Qasim W, et al. 2011. Treosulfan-based conditioning regimens for hematopoietic stem cell transplantation in children with primary immunodeficiency: United Kingdom experience. Blood. 117:4367–4375.
  • Ten Brink MH, Ackaert O, Zwaveling J, Bredius RG, Smiers FJ, den Hartigh J, Lankester AC, Guchelaar HJ. 2014a. Pharmacokinetics of treosulfan in pediatric patients undergoing hematopoietic stem cell transplantation. Ther Drug Monit. 36:465–472.
  • Ten Brink MH, Zwaveling J, Swen JJ, Bredius RG, Lankester AC, Guchelaar HJ. 2014b. Personalized busulfan and treosulfan conditioning for pediatric stem cell transplantation: the role of pharmacogenetics and pharmacokinetics. Drug Discov Today. 19:1572–1586.
  • Wachowiak J, Sykora KW, Cornish J, Chybicka A, Kowalczyk JR, Gorczynska E, Choma M, Grund G, Peters C, Party EPDW. 2011. Treosulfan-based preparative regimens for allo-HSCT in childhood hematological malignancies: a retrospective study on behalf of the EBMT pediatric diseases working party. Bone Marrow Transplant. 46:1510–1518.
  • van der Stoep M, Bertaina A, Ten Brink MH, Bredius RG, Smiers FJ, Wanders DCM, Moes D, Locatelli F, Guchelaar HJ, Zwaveling J, et al. 2017. High interpatient variability of treosulfan exposure is associated with early toxicity in paediatric HSCT: a prospective multicentre study. Br J Haematol. 179:772–780.