Romiplostim dose-response in patients with myelodysplastic syndromes

British Journal of Clinical Pharmacology

Volumn 75, Issue 6, 2013, Pages 1445-1454

  Perez Ruixo, Juan Jose ^a   Doshi, Sameer ^b   Wang, Yow Ming C ^b   Mould, Diane R ^c  

^a Amgen SA   (Spain)

^b AMGEN INC   (United States)

^c Projections Research Inc   (United States)

Author keywords

Kinetics of drug action model; Myelodysplastic syndromes; Platelets; Romiplostim

Indexed keywords

ANTINEOPLASTIC AGENT; ROMIPLOSTIM;

ADULT; AGED; ARTICLE; BLOOD; BODY WEIGHT; BONE MARROW; CANCER CHEMOTHERAPY; CELL COMPARTMENTALIZATION; CELL DIFFERENTIATION; CELL MATURATION; CELL PROLIFERATION; CELL STIMULATION; CLINICAL ARTICLE; CONTROLLED STUDY; DIAGNOSTIC TEST ACCURACY STUDY; DOSE RESPONSE; DRUG DISPOSITION; DRUG DOSE ESCALATION; DRUG EFFICACY; DRUG SAFETY; FEMALE; HUMAN; HUMAN CELL; IDIOPATHIC THROMBOCYTOPENIC PURPURA; MALE; MEGAKARYOCYTE; MULTICENTER STUDY; MYELODYSPLASTIC SYNDROME; PHARMACODYNAMICS; PHASE 1 CLINICAL TRIAL; PHASE 2 CLINICAL TRIAL; PREDICTIVE VALUE; PRIORITY JOURNAL; RACE DIFFERENCE; RISK FACTOR; SEX DIFFERENCE; STEM CELL; THROMBOCYTE; THROMBOCYTE COUNT; THROMBOCYTE LIFESPAN; THROMBOCYTOPENIA; TREATMENT RESPONSE;

ADULT; AGED; AGED, 80 AND OVER; BLOOD PLATELETS; DOSE-RESPONSE RELATIONSHIP, DRUG; FEMALE; HUMANS; MALE; MIDDLE AGED; MODELS, BIOLOGICAL; MYELODYSPLASTIC SYNDROMES; PLATELET COUNT; RECEPTORS, FC; RECEPTORS, THROMBOPOIETIN; RECOMBINANT FUSION PROTEINS; THROMBOPOIETIN;

EID: 84878161398     PISSN: 03065251     EISSN: 13652125     Source Type: Journal    
DOI: 10.1111/bcp.12041     Document Type: Article

References (32)

1. Hofmann WK, Koeffler HP. Myelodysplastic syndrome. Annu Rev Med 2005; 56: 1-16.
2. Cortes J. Treatment of myelodysplastic syndromes with valproic acid alone or in combination with all-trans retinoic acid. Curr Hematol Rep 2005; 4: 173-174.
3. Rollison DE, Howlader N, Smith MT, Strom SS, Merritt WD, Ries LA, Edwards BK, List AF. Epidemiology of myelodysplastic syndromes and chronic myeloproliferative disorders in the United States, 2001-2004, using data from the NAACCR and SEER programs. Blood 2008; 112: 45-52.
4. Greenberg P, Cox C, LeBeau MM, Fenaux P, Morel P, Sanz G, Sanz M, Vallespi T, Hamblin T, Oscier D, Ohyashiki K, Toyama K, Aul C, Mufti G, Bennett J. International scoring system for evaluating prognosis in myelodysplastic syndromes. Blood 1997; 89: 2079-2088.
5. Psaila B, Bussel JB, Frelinger AL, Babula B, Linden MD, Li Y, Barnard MR, Tate C, Feldman EJ, Michelson AD. Differences in platelet function in patients with acute myeloid leukemia and myelodysplasia compared to equally thrombocytopenic patients with immune thrombocytopenia. J Thromb Haemost 2011; 9: 2302-2310.
6. Garcia-Manero G, Shan J, Faderl S, Cortes J, Ravandi F, Borthakur G, Wierda WG, Pierce S, Estey E, Liu J, Huang X, Kantarjian H. A prognostic score for patients with lower risk myelodysplastic syndrome. Leukemia 2008; 22: 538-543.
7. Bryan J, Jabbour E, Prescott H, Garcia-Manero G, Issa JP, Kantarjian H. Current and future management options for myelodysplastic syndromes. Drugs 2010; 70: 1381-1394.
8. Broudy VC, Lin NL. AMG531 stimulates megakaryopoiesis in vitro by binding to Mpl. Cytokine 2004; 25: 52-60.
9. Wang B, Nichol JL, Sullivan JT. Pharmacodynamics and pharmacokinetics of AMG 531, a novel thrombopoietin receptor ligand. Clin Pharmacol Ther 2004; 76: 628-638.
10. Kantarjian H, Fenaux P, Sekeres MA, Becker PS, Boruchov A, Bowen D, Hellstrom-Lindberg E, Larson RA, Lyons RM, Muus P, Shammo J, Siegel R, Hu K, Franklin J, Berger DP. Safety and efficacy of romiplostim in patients with lower-risk myelodysplastic syndrome and thrombocytopenia. J Clin Oncol 2010; 28: 437-444.
11. Kantarjian HM, Giles FJ, Greenberg PL, Paquette RL, Wang ES, Gabrilove JL, Garcia-Manero G, Hu K, Franklin JL, Berger DP. Phase 2 study of romiplostim in patients with low- or intermediate-risk myelodysplastic syndrome receiving azacitidine therapy. Blood 2010; 116: 3163-3170.
12. Sekeres MA, Kantarjian H, Fenaux P, Becker P, Boruchov A, Guerci-Bresler A, Hu K, Franklin J, Wang YM, Berger D. Subcutaneous or intravenous administration of romiplostim in thrombocytopenic patients with lower risk myelodysplastic syndromes. Cancer 2011; 117: 992-1000.
13. Perez-Ruixo JJ, Green B, Doshi S, Wang YM, Mould DR. Romiplostim dose response in patients with immune thrombocytopenia. J Clin Pharmacol 2012; 52: 1540-1551.
14. Wang YM, Krzyzanski W, Doshi S, Xiao JJ, Perez-Ruixo JJ, Chow AT. Pharmacodynamics-mediated drug disposition (PDMDD) and precursor pool lifespan model for single dose of romiplostim in healthy subjects. AAPS J 2010; 12: 729-740.
15. Krzyzanski W, Sutjandra L, Perez-Ruixo JJ, Sloey B, Chow AT, Wang YM. Pharmacokinetic and pharmacodynamic modeling of romiplostim in animals. Pharm Res 2013 [in press].
16. Beal SL, Sheiner LB, eds. NONMEM Users Guides. Hanover: GloboMax, LLC, 1992.
17. Friberg LE, Henningsson A, Maas H, Nguyen L, Karlsson MO. Model of chemotherapy-induced myelosuppression with parameter consistency across drugs. J Clin Oncol 2002; 20: 4713-4721.
18. van Kesteren C, Zandvliet AS, Karlsson MO, Mathot RA, Punt CJ, Armand JP, Raymond E, Huitema AD, Dittrich C, Dumez H, Roche HH, Droz JP, Ravic M, Yule SM, Wanders J, Beijnen JH, Fumoleau P, Schellens JH. Semi-physiological model describing the hematological toxicity of the anti-cancer agent indisulam. Invest New Drugs 2005; 23: 225-234.
19. Jacqmin D, Snoeck E, van Schaick EA, Gieschke R, Pillai P, Steimer JL, Girard P. Modeling response time profiles in the absence of drug concentrations: definition and performance of the K-PD model. J Pharmacokinet Pharmacodyn 2007; 34: 57-85.
20. Pillai G, Gieschke R, Goggin T, Jacqmin P, Schimmer RC, Steimer JL. A semimechanistic and mechanistic population PK-PD model for biomarker response to ibandronate, a new bisphosphonate for the treatment of osteoporosis. Br J Clin Pharmacol 2004; 58: 618-631.
21. Ramon-Lopez A, Nalda MR, Valenzuela B, Perez-Ruixo JJ. Semi-mechanistic model for neutropenia after high dose of chemotherapy in breast cancer patients. Pharm Res 2009; 26: 1952-1962.
22. Mandema JW, Verotta D, Sheiner LB. Building population pharmacokinetic-pharmacodynamic models. I. Models for covariate effects. J Pharmacokinet Biopharm 1992; 20: 511-528.
23. Karlsson MO, Savic RM. Diagnosing model diagnostics. Clin Pharmacol Ther 2007; 82: 17-20.
24. Yano Y, Beal SL, Sheiner LB. Evaluating pharmacokinetic/pharmacodynamic models using the posterior predictive check. J Pharmacokinet Pharmacodyn 2001; 28: 171-192.
25. Hooker AC, Staatz CE, Karlsson MO. Conditional weighted residuals (CWRES): a model diagnostic for the FOCE method. Pharm Res 2007; 24: 2187-2197.
26. Gabrielsson J, Jusko WJ, Alari L. Modeling of dose-response-time data: four examples of estimating the turnover parameters and generating kinetic functions from response profiles. Biopharm Drug Dispos 2000; 21: 41-52.
27. Jin F, Krzyzanski W. Pharmacokinetic model of target-mediated disposition of thrombopoietin. AAPS J 2004; 6: 86-93.
28. Yan X, Lowe PJ, Fink M, Berghout A, Balser S, Krzyzanski W. Population pharmacokinetic and pharmacodynamic model-based comparability assessment of a recombinant human epoetin alfa and the biosimilar HX575. J Clin Pharmacol 2012; 52: 1624-1644.
29. Yang BB, Kido A. Pharmacokinetics and pharmacodynamics of pegfilgrastim. Clin Pharmacokinet 2011; 50: 295-306.
30. Krzyzanski W. Interpretation of transit compartments pharmacodynamic models as lifespan based indirect response models. J Pharmacokinet Pharmacodyn 2011; 38: 179-204.
31. Krzyzanski W, Perez Ruixo JJ. Lifespan based indirect response models. J Pharmacokinet Pharmacodyn 2012; 39: 109-123.
32. Emmons RV, Reid DM, Cohen RL, Meng G, Young NS, Dunbar CE, Shulman NR. Human thrombopoietin levels are high when thrombocytopenia is due to megakaryocyte deficiency and low when due to increased platelet destruction. Blood 1996; 87: 4068-4071.