Blood Res.  2021 Sep;56(3):141-149. 10.5045/br.2021.2020289.

Clinical analysis of hospitalized patients with hemophilia A: single-hemophilia treatment center experience in Korea over 10 years

• Kim JY
• Park SJ
• You CW

• KMID: 2520584
• DOI: http://doi.org/10.5045/br.2021.2020289

Abstract

Background
There is lack of data on admitted hemophilia patients in Korea. For this reason, this study was intended to analyze the hospitalization data of hemophilia patients in a regional Hemophilia Treatment Center (HTC) for the first time in Korea.
Methods
In this retrospective study, we surveyed hospitalized patients with Hemophilia A (HA) in a HTC for 14 years. Medical records of these hemophiliacs were reviewed and data regarding demographic characteristics, cause of admissions and their outcomes in each patient were obtained. In addition, the data of admitted days, type and amount of Coagulation factor concentrate (CFC) used, treatments other than CFC infusion during the admission days were also obtained from the medical record of each patient.
Results
A total 107 patients with hemophilia A were admitted during 14 years. Annual rate of admission of patients with HA was 8%. Mean age on admission was 29.63±19.51 years old and mean admission days were 11.28±5.46 days. Most admissions were occurred in severe and moderate hemophilia patients. The most common cause of admission was bleed control followed by surgery and other reasons. With modified WFH CFC supplementation guideline, all the bleeds were successfully controlled and all surgeries were also successfully conducted with less total CFC consumption compared to the consumed dose of other reports.
Conclusion
These results suggest that it is necessary to develop more specified regimens different from WFH.

Keyword

Hemophilia A; Admitted patient; Analysis; Factor VIII concentrate consumption; Korea

Reference

1. Mannucci PM, Tuddenham EG. 2001; The hemophilias--from royal genes to gene therapy. N Engl J Med. 344:1773–9. DOI: 10.1056/NEJM200106073442307. PMID: 11396445.

2. White GC 2nd, Rosendaal F, Aledort LM, et al. 2001; Definitions in hemophilia. Recommendation of the scientific subcommittee on factor VIII and factor IX of the scientific and standardization committee of the International Society on Thrombosis and Haemostasis. Thromb Haemost. 85:560. DOI: 10.1055/s-0037-1615621. PMID: 11307831.

3. Shih MY, Wang JD, Yin JD, Tsan YT, Chan WC. 2019; Differences in major bleeding events between patients with severe hemophilia A and hemophilia B: a nationwide, population-based cohort study. Clin Appl Thromb Hemost. 25:1076029619888023. DOI: 10.1177/1076029619888023. PMID: 31928075. PMCID: PMC7098207.

4. Manco-Johnson MJ, Soucie JM, Gill JC. Joint Outcomes Committee of the Universal Data Collection, US Hemophilia Treatment Center Network. 2017; Prophylaxis usage, bleeding rates, and joint outcomes of hemophilia, 1999 to 2010: a surveillance project. Blood. 129:2368–74. DOI: 10.1182/blood-2016-02-683169. PMID: 28183693. PMCID: PMC5409445.

5. Srivastava A, Santagostino E, Dougall A, et al. 2020; WFH guidelines for the management of hemophilia, 3rd edition. Haemophilia. 26(Suppl 6):1–158. DOI: 10.1111/hae.14046. PMID: 32744769.

6. Boadas A, Fernández-Palazzi F, De Bosch NB, Cedeño M, Ruiz-Sáez A. 2011; Elective surgery in patients with congenital coagulopathies and inhibitors: experience of the National Haemophilia Centre of Venezuela. Haemophilia. 17:422–7. DOI: 10.1111/j.1365-2516.2010.02427.x. PMID: 21118333.

7. Klukowska A, Windyga J, Batorova A. 2011; Clinical efficacy of a novel VWF-containing FVIII concentrate, Wilate(Ⓡ), in the prophylaxis and treatment of bleeding episodes in previously treated haemophilia A patients. Thromb Res. 127:247–53. DOI: 10.1016/j.thromres.2010.11.030. PMID: 21220152.

8. Santagostino E, Lentz SR, Misgav M, et al. 2015; Safety and efficacy of turoctocog alfa (NovoEightⓇ) during surgery in patients with haemophilia A: results from the multinational guardian^TM clinical trials. Haemophilia. 21:34–40. DOI: 10.1111/hae.12518. PMID: 25273984. PMCID: PMC4309503.

9. Dingli D, Gastineau DA, Gilchrist GS, Nichols WL, Wilke JL. 2002; Continuous factor VIII infusion therapy in patients with haemophilia A undergoing surgical procedures with plasma- derived or recombinant factor VIII concentrates. Haemophilia. 8:629–34. DOI: 10.1046/j.1365-2516.2002.00650.x. PMID: 12199670.

10. Lusher JM, Lee CA, Kessler CM, Bedrosian CL. ReFacto Phase 3 Study Group. 2003; The safety and efficacy of B-domain deleted recombinant factor VIII concentrate in patients with severe haemophilia A. Haemophilia. 9:38–49. DOI: 10.1046/j.1365-2516.2003.00708.x. PMID: 12558777.

11. Berntorp E, Björkman S. 2003; The pharmacokinetics of clotting factor therapy. Haemophilia. 9:353–9. DOI: 10.1046/j.1365-2516.2003.00762.x. PMID: 12828668.

12. Hermans C, Altisent C, Batorova A, et al. 2009; Replacement therapy for invasive procedures in patients with haemophilia: literature review, European survey and recommendations. Haemophilia. 15:639–58. DOI: 10.1111/j.1365-2516.2008.01950.x. PMID: 19444969.

13. Ludlam CA, Lee RJ, Prescott RJ, et al. 2000; Haemophilia care in central Scotland 1980-94. I. Demographic characteristics, hospital admissions and causes of death. Haemophilia. 6:494–503. DOI: 10.1046/j.1365-2516.2000.00405.x. PMID: 11012692.

14. Triemstra AH, Smit C, VAN DER Ploeg HM, Briët E, Rosendaal FR. 1995; Two decades of haemophilia treatment in the Netherlands, 1972-92. Haemophilia. 1:165–71. DOI: 10.1111/j.1365-2516.1995.tb00061.x. PMID: 27214533.

15. Björkman S, Berntorp E. 2001; Pharmacokinetics of coagulation factors: clinical relevance for patients with haemophilia. Clin Pharma-cokinet. 40:815–32. DOI: 10.2165/00003088-200140110-00003. PMID: 11735604.

16. Björkman S, Carlsson M. 1997; The pharmacokinetics of factor VIII and factor IX: methodology, pitfalls and applications. Haemophilia. 3:1–8. DOI: 10.1046/j.1365-2516.1997.00074.x. PMID: 27214611.

17. Allain JP. 1984; Principles of in vivo recovery and survival studies of VIII:C. Scand J Haematol Suppl. 41:123–30. DOI: 10.1111/j.1600-0609.1984.tb02773.x. PMID: 6440278.

18. Björkman S, Carlsson M, Berntorp E, Stenberg P. 1992; Pharmacokinetics of factor VIII in humans. Obtaining clinically relevant data from comparative studies. Clin Pharmacokinet. 22:385–95. DOI: 10.2165/00003088-199222050-00005. PMID: 1505144.

19. Batorova A, Martinowitz U. 2000; Intermittent injections vs. continuous infusion of factor VIII in haemophilia patients undergoing major surgery. Br J Haematol. 110:715–20. DOI: 10.1046/j.1365-2141.2000.02226.x. PMID: 10997985.

20. Pipe S. 2009; Antihemophilic factor (recombinant) plasma/albumin-free method for the management and prevention of bleeding episodes in patients with hemophilia A. Biologics. 3:117–25. DOI: 10.2147/BTT.S3191. PMID: 19707401. PMCID: PMC2726050.

21. Blanchette VS, Shapiro AD, Liesner RJ, et al. 2008; Plasma and albumin-free recombinant factor VIII: pharmacokinetics, efficacy and safety in previously treated pediatric patients. J Thromb Haemost. 6:1319–26. DOI: 10.1111/j.1538-7836.2008.03032.x. PMID: 18503631.

22. Négrier C, Shapiro A, Berntorp E, et al. 2008; Surgical evaluation of a recombinant factor VIII prepared using a plasma/albumin-free method: efficacy and safety of Advate in previously treated patients. Thromb Haemost. 100:217–23. DOI: 10.1160/TH08-02-0117. PMID: 18690340.

23. Carlsson M, Berntorp E, Björkman S, Lethagen S, Ljung R. 1997; Improved cost-effectiveness by pharmacokinetic dosing of factor VIII in prophylactic treatment of haemophilia A. Haemophilia. 3:96–101. DOI: 10.1046/j.1365-2516.1997.00091.x. PMID: 27214717.

24. Björkman S, Folkesson A, Jönsson S. 2009; 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. 65:989–98. DOI: 10.1007/s00228-009-0676-x. PMID: 19557401.