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ORIGINAL RESEARCH

Effectiveness of Prophylactic Coagulation Factor Replacement Therapy in Patients with Severe Hemophilia A in Taiwan – A Population-Based Study

, , , & ORCID Icon
Pages 1501-1510 | Received 29 Sep 2022, Accepted 02 Dec 2022, Published online: 13 Dec 2022

References

  • Srivastava A, Santagostino E, Dougall A, et al. WFH guidelines for the management of hemophilia. Haemophilia. 2020;26(S6):1–158. doi:10.1111/hae.14046
  • Manco-Johnson M. Comparing prophylaxis with episodic treatment in haemophilia A: implications for clinical practice. Haemophilia. 2007;13(Suppl 2):4–9. doi:10.1111/j.1365-2516.2007.01499.x
  • O’Hara J, Sima CS, Frimpter J, Paliargues F, Chu P, Presch I. Long-term outcomes from prophylactic or episodic treatment of haemophilia A: a systematic review. Haemophilia. 2018;24(5):e301–e311. doi:10.1111/hae.13546
  • Manco-Johnson MJ, Kempton CL, Reding MT, et al. Randomized, controlled, parallel-group trial of routine prophylaxis vs. on-demand treatment with sucrose-formulated recombinant factor VIII in adults with severe hemophilia A (SPINART). J Thromb Haemost. 2013;11(6):1119–1127. doi:10.1111/jth.12202
  • Manco-Johnson MJ, Abshire TC, Shapiro AD, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med. 2007;357(6):535–544. doi:10.1056/NEJMoa067659
  • Zhao Y, Xiao J, Yang R, et al. Efficacy of standard prophylaxis versus on-demand treatment with Bayer’s sucrose-formulated recombinant FVIII (rFVIII-FS) in Chinese children with severe hemophilia A. Pediatr Hematol Oncol. 2017;34(3):138–148. doi:10.1080/08880018.2017.1313921
  • Andersson NG, Auerswald G, Barnes C, et al. Intracranial haemorrhage in children and adolescents with severe haemophilia A or B - the impact of prophylactic treatment. Br J Haematol. 2017;179(2):298–307. doi:10.1111/bjh.14844
  • Manco-Johnson MJ, Soucie JM, Gill JC. Joint outcomes committee of the universal data collection USHTCN. prophylaxis usage, bleeding rates, and joint outcomes of hemophilia, 1999 to 2010: a surveillance project. Blood. 2017;129(17):2368–2374. doi:10.1182/blood-2016-02-683169
  • Dunn AL, Abshire TC. Current issues in prophylactic therapy for persons with hemophilia. Acta Haematol. 2006;115(3–4):162–171. doi:10.1159/000090930
  • O’Mahony B, Noone D, Prihodova L. Survey of coagulation factor concentrates tender and procurement procedures in 38 European Countries. Haemophilia. 2015;21(4):436–443. doi:10.1111/hae.12720
  • Saxena K. Barriers and perceived limitations to early treatment of hemophilia. J Blood Med. 2013;4:49–56. doi:10.2147/jbm.S43734
  • National Health Insurance Administration Ministry of Health and Welfare. National health insurance reimbursement guideline for hematological drugs in Taiwan; 2014. Available from: https://www.nhi.gov.tw/Content_List.aspx?n=E70D4F1BD029DC37&topn=3FC7D09599D25979. Accessed September 19, 2021.
  • Hsieh MH-C, Chiou -S-S, Liao T-C, Kao Yang Y-H, Lai E-C-C. Trends in coagulation factor replacement therapy and medical costs in patients with haemophilia in Taiwan: a population-based, 15-year analysis. Haemophilia. 2021;27(2):e187–e193. doi:10.1111/hae.14250
  • Ingerslev J, Lethagen S, Hvitfeldt Poulsen L, et al. Long-standing prophylactic therapy vs. episodic treatment in young people with severe haemophilia: a comparison of age-matched Danish and Russian patients. Haemophilia. 2014;20(1):58–64. doi:10.1111/hae.12242
  • Oldenburg J, Zimmermann R, Katsarou O, et al. Controlled, cross-sectional MRI evaluation of joint status in severe haemophilia A patients treated with prophylaxis vs. on demand. Haemophilia. 2015;21(2):171–179. doi:10.1111/hae.12539
  • Hilliard P, Zourikian N, Blanchette V, et al. Musculoskeletal health of subjects with hemophilia A treated with tailored prophylaxis: Canadian Hemophilia Primary Prophylaxis (CHPS) Study. J Thromb Haemost. 2013;11(3):460–466. doi:10.1111/jth.12113
  • Meng L, Zhuang J, Sun X, et al. FVIII剂量对青少年重型血友病A的关节结构与功能的影响 43例前瞻性队列研究中期随访报告 [Effect of FVIII doses on joint structure and function in adolescents with severe hemophilia A: mid-term results of a prospective cohort study]. Nan Fang Yi Ke Da Xue Xue Bao. 2021;41(4):536–542. Chinese. doi:10.12122/j.issn.1673-4254.2021.04.08
  • Nagae C, Yamashita A, Ashikaga T, et al. A cohort study of the usefulness of primary prophylaxis in patients with severe haemophilia A. Int J Hematol. 2016;104(2):208–215. doi:10.1007/s12185-016-2005-3
  • Hsieh CY, Su CC, Shao SC, et al. Taiwan’s national health insurance research database: past and future. Clin Epidemiol. 2019;11:349–358. doi:10.2147/clep.S196293
  • Jadhav U, Mukherjee K. Assessment of healthcare measures, healthcare resource use, and cost of care among severe hemophilia A patients in Mumbai region of India. J Postgrad Med. 2018;64(3):138–144. doi:10.4103/jpgm.JPGM_701_16
  • Croteau SE, Cook K, Sheikh L, et al. Health care resource utilization and costs among adult patients with hemophilia A on factor VIII prophylaxis: an administrative claims analysis. J Manag Care Spec Pharm. 2021;27(3):316–326. doi:10.18553/jmcp.2021.27.3.316
  • Oladapo AO, Lu M, Walsh S, O’Hara J, Kauf TL. Inhibitor clinical burden of disease: a comparative analysis of the CHESS data. Orphanet J Rare Dis. 2018;13(1):198. doi:10.1186/s13023-018-0929-9
  • Chen Y-C, Chang C-Y, Cheng S-N, et al. Evolution of congenital haemophilia care in Taiwan. J Formos Med Assoc. 2021;2021. doi:10.1016/j.jfma.2021.07.017
  • Franklin JM, Shrank WH, Pakes J, et al. Group-based trajectory models: a new approach to classifying and predicting long-term medication adherence. Med Care. 2013;51(9):789–796. doi:10.1097/MLR.0b013e3182984c1f
  • Ds Nagin. Analyzing developmental trajectories: a semiparametric, group-based approach. Psychol Methods. 1999;42(2):139–157.
  • Nagin DS, Nagin D, Nagin TH. Group-Based Modeling of Development. Harvard University Press; 2005.
  • Nagin DS, Odgers CL. Group-based trajectory modeling in clinical research. Annu Rev Clin Psychol. 2010;6:109–138. doi:10.1146/annurev.clinpsy.121208.131413
  • Gupta S, Siddiqi A-EA, Soucie JM, et al. The effect of secondary prophylaxis versus episodic treatment on the range of motion of target joints in patients with haemophilia. Br J Haematol. 2013;161(3):424–433. doi:10.1111/bjh.12267
  • Austin PC. An introduction to propensity score methods for reducing the effects of confounding in observational studies. Multivariate Behav Res. 2011;46(3):399–424. doi:10.1080/00273171.2011.568786
  • Jackson JW, Schmid I, Stuart EA. Propensity scores in pharmacoepidemiology: beyond the Horizon. Curr Epidemiol Rep. 2017;4(4):271–280. doi:10.1007/s40471-017-0131-y
  • Austin PC, Stuart EA. Moving towards best practice when using inverse probability of treatment weighting (IPTW) using the propensity score to estimate causal treatment effects in observational studies. Stat Med. 2015;34(28):3661–3679. doi:10.1002/sim.6607
  • Gringeri A, Lundin B, von Mackensen S, Mantovani L, Mannucci PM. A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study). J Thromb Haemost. 2011;9(4):700–710. doi:10.1111/j.1538-7836.2011.04214.x
  • Kavakli K, Yang R, Rusen L, Beckmann H, Tseneklidou-Stoeter D, Maas Enriquez M. Prophylaxis vs. on-demand treatment with BAY 81-8973, a full-length plasma protein-free recombinant factor VIII product: results from a randomized trial (LEOPOLD II). J Thromb Haemost. 2015;13(3):360–369. doi:10.1111/jth.12828
  • Tagliaferri A, Feola G, Molinari AC, et al. Benefits of prophylaxis versus on-demand treatment in adolescents and adults with severe haemophilia A: the POTTER study. Thromb Haemost. 2015;114(1):35–45. doi:10.1160/th14-05-0407
  • Ay C, Perschy L, Rejtö J, Kaider A, Pabinger I. Treatment patterns and bleeding outcomes in persons with severe hemophilia A and B in a real-world setting. Ann Hematol. 2020;99(12):2763–2771. doi:10.1007/s00277-020-04250-9
  • National Health Insurance Administration Ministry of Health and Welfare. Medication Reimbursement Guideline of Taiwan National Health Insurance Program; 2022. Available from: https://mohwlaw.mohw.gov.tw/FINT/FINTQRY04.aspx?starDate=00000000&endDate=99991231&no=&n1=&n2=&kt=&kw=%e5%87%9d%e8%a1%80%e5%9b%a0%e5%ad%90&kw2=&kw3=&kw4=&valid=3&type=etype_&RowNo=20. Accessed Aug 28, 2022.
  • Dunkley S, Lam JCM, John MJ, et al. Principles of haemophilia care: the Asia-Pacific perspective. Haemophilia. 2018;24(3):366–375. doi:10.1111/hae.13425
  • Fischer K, Steen Carlsson K, Petrini P, et al. Intermediate-dose versus high-dose prophylaxis for severe hemophilia: comparing outcome and costs since the 1970s. Blood. 2013;122(7):1129–1136. doi:10.1182/blood-2012-12-470898
  • Feldman BM, Rivard GE, Babyn P, et al. Tailored frequency-escalated primary prophylaxis for severe haemophilia A: results of the 16-year Canadian Hemophilia Prophylaxis Study longitudinal cohort. Lancet Haematol. 2018;5(6):e252–e260. doi:10.1016/s2352-3026(18)30048-6
  • Jun SNL, Testa D, Cox-Buckley B, Jain N. Characterization of women and girls with hemophilia treated in the US from A claims database; 2017.