基于药动学的A型血友病的凝血因子Ⅷ个体治疗的研究进展

殷怡维, 刘晓芹, 焦正

中国药学杂志 ›› 2021, Vol. 56 ›› Issue (24) : 1968-1973.

PDF(1366 KB)
中国药学杂志
PDF(1366 KB)
中国药学杂志 ›› 2021, Vol. 56 ›› Issue (24) : 1968-1973. DOI: 10.11669/cpj.2021.24.003
综述

基于药动学的A型血友病的凝血因子Ⅷ个体治疗的研究进展

  • 殷怡维1, 刘晓芹2, 焦正1*
作者信息 +

Pharmacokinetics-Informed Individualized Dosing of Factor Ⅷ in Patients with Haemophilia A

  • YIN Yi-wei1, LIU Xiao-qin2, JIAO Zheng1*
Author information +
文章历史 +

摘要

A型血友病是由凝血因子Ⅷ(FⅧ)缺乏而引起的出血性疾病,患者需长期补充FⅧ进行治疗。然而不同患者的治疗目标和药动学特征存在较大差异,依据个体药动学参数和需求制定个体的给药方案,能够有效地预防出血,减轻患者经济负担,具有显著的疗效优越性。近年来,基于药动学进行个体化给药得到快速发展,国内外已有基于群体药动学和最大后验贝叶斯法的给药方案设计工具,协助医务工作者实现个体化给药。笔者将阐述 FⅧ产品的药动学特征、影响因素以及基于药动学指导的个体化治疗方法和临床应用,以期为A型血友病患者的个体化给药研究及实践提供参考。

Abstract

Haemophilia A is a clotting disorder caused by factor Ⅷ deficiency. Patients who have haemophilia A need long-term factor Ⅷ replacement. However, different patients have different goals of treatment and pharmacokinetics characteristics. Developing individualized dosing based on individual patients' pharmacokinetics and goals has shown its advantage in effectively preventing bleeding and reducing the economic burden. In recent years, there is a growing use of pharmacokinetics-informed individualized dosing in clinical practice. To further assist the calculation of individualized dosing, researchers have developed dosing tools based on population pharmacokinetics and maximum a posteriori Bayesian (MAPB) estimation in China and other countries. In this review, we summarized the pharmacokinetics characteristics of factor Ⅷ and its influential factors. Moreover, the individualized dosing methods of factor Ⅷ based on pharmacokinetics, especially with MAPB approach are described to optimize the clinical management of patients with Haemophilia A.

关键词

血友病 / 凝血因子Ⅷ / 药动学 / 最大后验贝叶斯法 / 个体化给药

Key words

haemophilia / factor Ⅷ / pharmacokinetics / maximum a posteriori bayesian / individualized dosing

引用本文

EndNote

Ris (Procite)

Bibtex

导出引用
殷怡维, 刘晓芹, 焦正. 基于药动学的A型血友病的凝血因子Ⅷ个体治疗的研究进展[J]. 中国药学杂志, 2021, 56(24): 1968-1973 https://doi.org/10.11669/cpj.2021.24.003
YIN Yi-wei, LIU Xiao-qin, JIAO Zheng. Pharmacokinetics-Informed Individualized Dosing of Factor Ⅷ in Patients with Haemophilia A[J]. Chinese Pharmaceutical Journal, 2021, 56(24): 1968-1973 https://doi.org/10.11669/cpj.2021.24.003
中图分类号： R969.3   

参考文献

[1] IORIO A, STONEBRAKER J S, CHAMBOST HE, et al. Stablishing the prevalence and prevalence at birth of hemophilia in males:a meta-analytic approach using national registries. Ann Intern Med, 2019, 171(8):540-546.
[2] SONG X W, ZHONG J, XUE F, et al. An overview of patients with haemophilia A in China:Epidemiology, disease severity and treatment strategies. Haemophilia, 2021, 27(1):e51-e59.
[3] SRIVASTAVA A, SANTAGOSTINO E, DOUGALL A, et al. WFH guidelines for the management of hemophilia, 3rd edition. Haemophilia, 2020, 26(Suppl 6):S151-S158.
[4] STONEBRAKER J S, BOLTON-MAGGS P H B, BROOKER M. The world federation of hemophilia annual global survey 1999-2018. Haemophilia, 2020, 26(4):591-600.
[5] THROMBOSIS AND HEMOSTASIS GROUP, CHINESE SOCIETY OF HEMATOLOGY, CHINESE MEDICAL ASSOCIATION/HEMOPHILIA TREATMENT CENTER COLLABORATIVE NETWORK OF CHINA. Chinese guidelines on the treatment of hemophilia (version 2020). Chin J Hematol(中华血液学杂志), 2020, 41(4):265-271.
[6] RAYMENT R, CHALMERS E, FORSYTH K, et al. Guidelines on the use of prophylactic factor replacement for children and adults with Haemophilia A and B. Br J Haematol, 2020, 190(5):684-695.
[7] SCHIAVONI M, NAPOLITANO M, GIUFFRIDA G, et al. Status of recombinant factor viii concentrate treatment for hemophilia a in italy:characteristics and clinical benefits. Front Med (Lausanne), 2019, 6:261.
[8] KEATING G. BAY 81-8973 (Octocog Alfa; Kovaltry): A Review in Haemophilia A. Bio Drugs, 2016, 30(5):453-459.
[9] MAHLANGU J, AHUJA S, WINDYGA, et al. Bay 81-8973, a full-length recombinant factor Ⅷ for the treatment of hemophilia a: product review. Ther Adv Hematol, 2018,9(7):191-205.
[10] KEATING G, DHILLON S. 2012 Octocog alfa (Advate): a guide to its use in hemophilia A. Bio Drugs, 2012, 26(4):269-273.
[11] PAPR. New drug information. Product: Xyntha. JAAPA, 2008, 21(12):15.
[12] COLLINS P W, FISCHER K, MORFINI M, et al. Implications of coagulation factor Ⅷ and IX pharmacokinetics in the prophylactic treatment of haemophilia. Haemophilia, 2011, 17(1):2-10.
[13] BJORKMAN S, OH M, SPOTTS G, et al. Population pharmacokinetics of recombinant factor Ⅷ:the relationships of pharmacokinetics to age and body weight. Blood, 2012, 119(2):612-618.
[14] JI S M, JI X W, YU A P, et al. Research status of hemophilia treatment. Chin J Clin Pharmacol(中国临床药理学杂志), 2018,34(19):2359-2365.
[15] MAHLANGU J, YOUNG G, HERMANS C, et al. Defining extended half-life rFⅧ-A critical review of the evidence. Haemophilia, 2018, 24(3):348-358.
[16] OLDENBURG J. Optimal treatment strategies for hemophilia:achievements and limitations of current prophylactic regimens. Blood, 2015, 125(13):2038-2044.
[17] IORIO A, EDGINTON A N, BLANCHETTE V, et al. Performing and interpreting individual pharmacokinetic profiles in patients with Hemophilia A or B:Rationale and general considerations. Res Pract Thromb Haemost, 2018, 2(3):535-548.
[18] VALENTINO L A, PIPE S W, COLLINS P W, et al. Association of peak factor Ⅷ levels and area under the curve with bleeding in patients with haemophilia A on every third day pharmacokinetic-guided prophylaxis. Haemophilia, 2016, 22(4):514-520.
[19] BRODERICK C R, HERBERT R D, LATIMER J, et al. Association between physical activity and risk of bleeding in children with hemophilia. JAMA, 2012, 308(14):1452-1459.
[20] DELAVENNE X,DARGAUD Y. Pharmacokinetics for haemophilia treaters:Meaning of PK parameters, interpretation pitfalls, and use in the clinic. Thromb Res, 2020, 192:52-60.
[21] CHOW S C. Bioavailability and bioequivalence in drug development. Wiley Interdiscip Rev Comput Stat, 2014, 6(4):304-312.
[22] COLLINS P W, BJORKMAN S, FISCHER K, et al. Factor Ⅷ requirement to maintain a target plasma level in the prophylactic treatment of severe hemophilia A:influences of variance in pharmacokinetics and treatment regimens. J Thromb Haemost, 2010, 8(2):269-275.
[23] HAZENDONK H, VAN MOORT I, MATHOT R A A, et al. Setting the stage for individualized therapy in hemophilia:What role can pharmacokinetics play?. Blood Rev, 2018, 32(4):265-271.
[24] MCLEAY S C, MORRISH G A, KIRKPATRICK C M, et al. The relationship between drug clearance and body size:systematic review and meta-analysis of the literature published from 2000 to 2007. Clin Pharmacokinet, 2012, 51(5):319-330.
[25] BJORKMAN S. Comparative pharmacokinetics of factor Ⅷ and recombinant factor IX:for which coagulation factors should half-life change with age?. Haemophilia, 2013, 19(6):882-886.
[26] AI D, CHEN Z P, WU R H, et al. Individualized prophylaxis for hemophilia children based on pharmacokinetics application. J China Pediatr Blood Cancer(中国小儿血液与肿瘤), 2020, 25(4):68-71.
[27] AR M C, BALKAN C, KAVAKLI K. Extended half-life coagulation factors:a new era in the management of hemophilia patients. Turk J Haematol, 2019, 36(3):141-154.
[28] ANZENGRUBER J, FEICHTINGER M, BARNTHALER P, et al. How full-length fviii benefits from its heterogeneity-insights into the role of the b-domain. Pharm Res, 2019, 36(5):77-79.
[29] EPSTEIN J, XIONG Y, WOO P, et al. Retrospective analysis of differences in annual factor Ⅷ utilization among haemophilia A patients. Haemophilia, 2012, 18(2):187-192.
[30] CHEN Z P, LI P J, LI G, et al. Pharmacokinetic studies of factor viii in chinese boys with severe hemophilia a:a single-center study. Chin Med J (Engl), 2018, 131(15):1780-1785.
[31] ZHANG Y, ROBERTS J, TORTORICI M, et al. Population pharmacokinetics of recombinant coagulation factor Ⅷ-SingleChain in patients with severe hemophilia A. J Thromb Haemost, 2017, 15(6):1106-1114.
[32] MIESBACH W, ALESCI S, KREKELER S, et al. Comorbidities and bleeding pattern in elderly haemophilia A patients. Haemophilia, 2009, 15(4):894-899.
[33] KEPA S, HORVATH B, REITTER-PFOERTNER S, et al. Parameters influencing FⅧ pharmacokinetics in patients with severe and moderate haemophilia A. Haemophilia, 2015, 21(3):343-350.
[34] VAN MOORT I, PREIJERS T, HAZENDONK H. Dosing of factor Ⅷ concentrate by ideal body weight is more accurate in overweight and obese haemophilia A patients. Br J Clin Pharmacol, 2021, 87(6):2602-2613.
[35] GRAHAM A, JAWORSKI K. Pharmacokinetic analysis of anti-hemophilic factor in the obese patient. Haemophilia, 2014, 20(2):226-229.
[36] BJRKMAN S, FOLKESSON A, JONSSON S. Pharmacokinetics and dose requirements of factor Ⅷ over the age range 3-74 years:a population analysis based on 50 patients with long-term prophylactic treatment for haemophilia A. Eur J Clin Pharmacol, 2009, 65(10):989-998.
[37] PIPE S W, MONTGOMERY R R, PRATT K P. Life in the shadow of a dominant partner:the FⅧ-VWF association and its clinical implications for hemophilia A. Blood, 2016, 128(16):2007-2016.
[38] SHAPIRO A D, RAGNI M V, KULKARNI R. Recombinant factor Ⅷ Fc fusion protein:extended-interval dosing maintains low bleeding rates and correlates with von Willebrand factor levels. J Thromb Haemost, 2014, 12(11):1788-1800.
[39] MOHLKE K L,GINSBURG D. Von Willebrand disease and quantitative variation in von Willebrand factor. J Lab Clin Med, 1997, 130(3):252-261.
[40] VLOT A J, KOPPELMAN S J, VAN DEN BERG M H, et al. The affinity and stoichiometry of binding of human factor Ⅷ to von Willebrand factor. Blood, 1995, 85(11):3150-3157.
[41] FIJNVANDRAAT K, PETERS M, TEN CATE J W. Inter-individual variation in half-life of infused recombinant factor Ⅷ is related to pre-infusion von Willebrand factor antigen levels. Br J Haematol, 1995, 91(2):474-476.
[42] MORFINI M. The History of Clotting Factor Concentrates Pharmacokinetics. J Clin Med, 2017, 6(3):35.
[43] IORIO A, BLANCHETTE V, BLATNY J. Estimating and interpreting the pharmacokinetic profiles of individual patients with hemophilia A or B using a population pharmacokinetic approach:communication from the SSC of the ISTH. J Thromb Haemost, 2017, 15(12):2461-2465.
[44] BERNTORP E, SPOTTS G, PATRONE L, et al. Advancing personalized care in hemophilia A:ten years′ experience with an advanced category antihemophilic factor prepared using a plasma/albumin-free method. Biologics, 2014, 8:115-127.
[45] LIU X Q, JIAO Z, GAO Y C. Progress in Development and Application of Decision-making Systems for Individualized Dosing. Chin Pharm J(中国药学杂志), 2019, 54(1):1-8.
[46] SHAO J, JIAO Z, WEI J X. The progress of population pharmacokinetics in adult liver transplantation oral tacrolimus recipients. Chin Pharm J(中国药学杂志), 2018, 53(17):1433-1437.
[47] ZHANG J H, PENG W X, DONG J. Individualized medication of warfarin by quantitative pharmacology. Chin Pharm J(中国药学杂志), 2018, 53(19):1703-1708.
[48] YING Y Q, LIN W W, JIAO Z. Development of software for individualized dosage regimen of valproic acid and carbamazepine. Chin Pharm J(中国药学杂志), 2014, 49(2):163-166.
[49] DE ROSE D U, CAIROLI S, DIONISI M, et al. Therapeutic drug monitoring is a feasible tool to personalize drug administration in neonates using new techniques:an overview on the pharmacokinetics and pharmacodynamics in neonatal age. Int J Mol Sci, 2020, 21(16):5898-5923.
[50] SRIDHARAN K,SIVARAMAKRISHNAN G. Which antimicrobial agent is likely to be the best for treating clostridium difficile infections? a bayesian network meta-analysis of randomized clinical trials. Drug Res (Stuttg), 2019, 69(4):194-200.
[51] WOILLARD J B, SAINT-MARCOUX F, DEBORD J. Pharmacokinetic models to assist the prescriber in choosing the best tacrolimus dose. Pharmacol Res, 2018, 130:316-321.
[52] ZHOU E J, PAN Y, JIAO Z. Advance of research on individualized dosing based on maximum a posteriori Bayesian method. Prog Pharm Sci(药学进展), 2020, 44(6):411-417.
[53] BJRKMAN S. Limited blood sampling for pharmacokinetic dose tailoring of FⅧ in the prophylactic treatment of haemophilia A. Haemophilia, 2010, 16(4):597-605.
[54] PASCA S, MILAN M, SAROLO L. PK-driven prophylaxis versus standard prophylaxis:When a tailored treatment may be a real and achievable cost-saving approach in children with severe hemophilia A. Thromb Res, 2017, 157:58-63.
[55] MEGAS-VERICAT J E, BONANAD S, HAYA S. Bayesian pharmacokinetic-guided prophylaxis with recombinant factor Ⅷ in severe or moderate haemophilia A. Thromb Res, 2019, 174:151-162.
[56] PASCA S, ZANON E. Savings without changing:How to use the MyPKfit(R) device to improve treatment strategies in a cohort of patients with haemophilia A. Thromb Res, 2019, 183:1-3.
PDF(1366 KB)

Accesses

Citation

Detail
段落导航
相关文章

/

〈 〉