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References

1 Guidelines for the Management of Hemophilia (General Care and Management of Hemophilia: 1.1 What is haemophilia?). World Federation of Hemophilia. http://www1.wfh.org/publications/files/pdf-1494.pdf. Last updated April 2013. Accessed 22nd August 2018

2 National Organization for Rare Disorders. Hemophilia A. https://rarediseases.org/rare-diseases/hemophilia-a/. Accessed 22nd August 2018

3 National Organization for Rare Disorders. Hemophilia B. https://rarediseases.org/rare-diseases/hemophilia-b/. Accessed 22nd August 2018

4 UK National Haemophilia Database. UK Bleeding Disorder Statistics for April 2016 to March 2017. http://www.ukhcdo.org/wp-content/uploads/2018/02/Bleeding-Disorder-Statistics-for-April-2016-to-March-2017-for-UKHCDO.pdf. Accessed 5th November 2018

5 World Federation of Hemophilia. How do you get haemophilia? https://www.wfh.org/en/page.aspx?pid=644. Accessed 22nd August 2018

6 World Federation of Hemophilia. Carriers and women with haemophilia. https://www.wfh.org/en/abd/carriers/carriers-and-women-with-hemophilia-en. Accessed 22nd August 2018

7 World Federation of Hemophilia. Inheritance of haemophilia. https://www.wfh.org/en/abd/carriers/carriers-inheritance-of-hemophilia-en. Accessed 5th November 2018

8 Haemophilia. BMJ Best Practice. https://bestpractice.bmj.com/topics/en-gb/468/aetiology. Last updated April 2017. Accessed 22nd August 2018

9 Haemophilia. BMJ Best Practice. https://bestpractice.bmj.com/topics/en-gb/468/diagnosis-approach. Last updated April 2017. Accessed 22nd August 2018

10 World Federation of Hemophilia. Carriers and women with haemophilia. https://www.wfh.org/en/abd/carriers/carriers-and-women-with-hemophilia-en. Accessed 22nd August 2018

11 NHS. Causes Haemophlia. https://www.nhs.uk/conditions/haemophilia/causes/. Accessed 22nd August 2018

12 NHS. Symptoms Haemophilia. https://www.nhs.uk/conditions/haemophilia/symptoms/. Accessed 22nd August 2018

13 Guidelines for the Management of Hemophilia (General Care and Management of Hemophilia: 1.4 Fitness and physical activity). World Federation of Hemophilia. http://www1.wfh.org/publications/files/pdf-1494.pdf. Last updated April 2013. Accessed 22nd August 2018

14 The Haemophilia Society. What to expect from your treatment centre. https://haemophilia.org.uk/bleeding-disorders/treatment-centres/expectations-treatment-centre/. Accessed 22nd August 2018

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17 NHS. Treatment. https://www.nhs.uk/conditions/haemophilia/treatment/ Last updated April 2017. Accessed 22nd August 2018

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19 Valentino LA1, Ewenstein B, Navickis RJ, Wilkes MM. Haemophilia. Central venous access devices in haemophilia. 2004 Mar;10(2):134-46. https://www.ncbi.nlm.nih.gov/pubmed/14962202 . Accessed November 2018

20 Haemophilia. BMJ Best Practice. https://bestpractice.bmj.com/topics/en-gb/468/history-exam. Last updated April 2017. Accessed 22nd August 2018

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22 Hemophilia A (Overview: management). Medscape. https://emedicine.medscape.com/article/779322-overview. Last updated December 2017 Accessed 22nd August 2018

23 Desmopressin (Mild to moderate haemophilia and von Willebrand’s disease). BNF. https://bnf.nice.org.uk/drug/desmopressin.html. Accessed 22nd August 2018

24 Hemophilia B (Overview: management). Medscape. https://emedicine.medscape.com/article/779434-overview. Last updated June 2017. Accessed 22nd August 2018

25 Guidelines for the Management of Hemophilia (General Care and Management of Hemophilia: 1.6 Prophylactic factor replacement therapy). World Federation of Hemophilia. http://www1.wfh.org/publications/files/pdf-1494.pdf. Last updated April 2013. Accessed 22nd August 2018

26 Haemophilia (Management: Approach). BMJ Best Practice. https://bestpractice.bmj.com/topics/en-gb/468/management-approach. Last updated April 2017. Accessed 22nd August 2018

27 Mahlangu J, Powell JS, Ragni MV et al. Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A. Blood, 2014, vol 123, issue 3, pp317-25. http://www.bloodjournal.org/content/bloodjournal/early/2013/11/13/blood-2013-10-529974.full.pdf

28 Santagostino E, Martinowitx U, Lissitchkov T et al. Long-acting recombinant coagulation factor IX albumin fusion protein (rIX-FP) in hemophilia B: results of a phase 3 trial. BLOOD, April 2016, 127 (14), pp1761-9. In.

http://www.bloodjournal.org/content/bloodjournal/early/2016/01/11/blood-2015-09-669234.full.pdf. Accessed 22nd August 2018

29 Clinical Commissioning Policy: Emicizumab as prophylaxis in people with congenital haemophilia A with factor VIII inhibitors (all ages) NHS England. https://www.england.nhs.uk/wp-content/uploads/2018/07/1717-emicizumab.pdf. Published July 2018. Accessed 22nd August 2018

30 Powell JS, Pasi KJ, Ragni MV et al. Phase 3 Study of Recombinant Factor IX Fc Fusion Protein in Hemophilia B

NEJM, 2013, 369 (24), pp2313-23. In. https://www.nejm.org/doi/full/10.1056/nejmoa1305074. Accessed 22nd August 2018

31 Shima M, Hanabusa H, Taki M, et al. Long-term safety and efficacy of emicizumab in a phase 1/2 study in patients with hemophilia A with or without inhibitors (Discussion). Blood Advances. 2017 1:1891-1899; http://www.bloodadvances.org/content/1/22/1891?sso-checked=true#sec-12. Accessed 22nd August 2018

32 Guidelines for the Management of Hemophilia (General Care and Management of Hemophilia: 1.7 Home therapy). World Federation of Hemophilia. http://www1.wfh.org/publications/files/pdf-1494.pdf. Last updated April 2013. Accessed 22nd August 2018

33 Oxford Handbook of Clinical Pharmacy (3 ed.) (Therapy-related issues: cardiovascular system: Understanding anticoagulation: Coagulation factors). Oxford Medicine Online. http://oxfordmedicine.com/view/10.1093/med/9780198735823.001.0001/med-9780198735823-chapter-16?rskey=6N7NwM&result=5. Published online April 2017. Accessed 22nd August 2018

34 Current Issues in Prophylaxis. World Federation of Hemophilia. https://www.wfh.org/en/abd/prophylaxis/current-issues-in-prophylaxis. Last updated March 2016. Accessed 22nd August 2018

34 Updates to the Guidelines for the Management of Hemophilia. World Hemophilia Federation. http://www1.wfh.org/publications/files/pdf-1512.pdf. Published April 2013. Accessed 22nd August 2018

35 Guidelines for the Management of Hemophilia (General Care and Management of Hemophilia: 1.9 Pain management). World Federation of Hemophilia. http://www1.wfh.org/publications/files/pdf-1494.pdf. Last updated April 2013. Accessed 22nd August 2018

36 Haemophilia (Complications). BMJ Best Practice. https://bestpractice.bmj.com/topics/en-gb/468/complications. Last updated April 2017. Accessed 22nd August 2018

37 What are inhibitors? World Hemophilia Federation. https://www.wfh.org/en/page.aspx?pid=651. Accessed 22nd August 2018

38 How does immune tolerance induction work? World Hemophilia Federation. https://www.wfh.org/en/page.aspx?pid=647. Last updated December 2014. Accessed 22nd August 2018

39 Guidelines for the Management of Hemophilia (General Care and Management of Hemophilia: Table 1-3: Approximate frequency of bleeding at different sites). World Federation of Hemophilia. http://www1.wfh.org/publications/files/pdf-1494.pdf. Last updated April 2013. Accessed 22nd August 2018

40 Hemophilia A (Overview: Background). Medscape. https://emedicine.medscape.com/article/779322-overview#a2. Last updated December 2017. Accessed 22nd August 2018

41 Hemophilia A Treatment and Management (Approach considerations). Medscape. https://emedicine.medscape.com/article/779322-treatment?src=refgatesrc1. Last updated December 2017. In

42 NHS England, 2013/14 NHS STANDARD CONTRACT FOR HAEMOPHILIA (ALL AGES) https://www.england.nhs.uk/wp-content/uploads/2013/06/b05-haemophilia.pdf. Last accessed December 2018

43 Guidelines for the Management of Hemophilia (1.11 Dental care and management). World Federation of Hemophilia. http://www1.wfh.org/publications/files/pdf-1472.pdf. Last updated April 2013. Accessed 22nd August 2018

44 Drugs that can cause bleeding. World Hemophilia Federatio. https://www.wfh.org/en/page.aspx?pid=639. Last updated February 2010. Accessed 22nd August 2018

45 Guidelines for the Management of Hemophilia (2.4 Vaccinations). World Federation of Hemophilia. http://www1.wfh.org/publications/files/pdf-1472.pdf. Last updated April 2013. Accessed 22nd August 2018

46 Understanding Haemophilia (Medical and dental treatment: Medicines). The Haemophilia Society. https://haemophilia.org.uk/wp-content/uploads/2017/04/Understanding_haemophilia_WEB.pdf. Published February 2017. Accessed 22nd August 2018

47 Careers with a bleeding disorder and the world of work. The Haemophilia Society. https://haemophilia.org.uk/wp-content/uploads/2017/02/careers_info.pdf.pdf. Last updated November 2014. Accessed 22nd August 2018

48 Specialised Services Circular (SSC) 1652 Sept 2016, on advice of NHS England

49 Elocta SmPC. Electronic Medicines Compendium. https://www.medicines.org.uk/emc/product/5100/smpc. Last updated December 2017. Accessed 22nd August 2018

50 Idelvion SmPC. Electronic Medicines Compendium. https://www.medicines.org.uk/emc/product/2146/smpc. Last updated January 2018. Accessed 22nd August 2018

51 Alprolix SmPC. Electronic Medicines Compendium. https://www.medicines.org.uk/emc/product/7277/smpc. Last updated July 2018. Accessed 22nd August 2018

52 Nathwani AC, Davidoff AM, Tuddenham EGD/ Gene Therapy for Hemophilia. Hematology/Oncology Clinics of North America, October 2017, vol 31, issue 5, pp853-68. https://www.sciencedirect.com/science/article/pii/S0889858817301041?via%3Dihub. Accessed 22nd August 2018

53 HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients. Clinical Trials.Gov

https://clinicaltrials.gov/ct2/show/NCT03569891?term=hemophilia+B&rank=15. Last updated July 2018. Accessed 22nd August 2018

54 Long-term Safety and Efficacy Study of SPK-9001 in Individuals with Hemophilia B. Clinical Trials.Gov. https://clinicaltrials.gov/ct2/show/NCT03307980?term=SPK9001&rank=1. Last updated July 2018. Accessed 22nd August 2018

55 Single-Arm Study to Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia Patients (BMN270-301). Clinical Trials.Gov. https://clinicaltrials.gov/ct2/show/NCT03370913. Last updated July 2018. Accessed 22nd August 2018

56 Pasi KJ, Georgiev P, Mant T et al. A subcutaneously administered investigational RNAi therapeutic (Fitusiran, ALN-AT3) targeting antithrombin for treatment of hemophilia: interim weekly and monthly dosing results in patients with hemophilia A or B. Presentation to American Hematology Society annual meeting, 2015. http://www.alnylam.com/web/assets/ASH-2015_fitusiran_010715.pdf. Accessed 22nd August 2018

57 A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors. Clinical Trials.gov

https://clinicaltrials.gov/ct2/show/NCT03417245. Last updated June 2018. Accessed 22nd August 2018

59 Korte W. The Potential Close Future of Hemophilia Treatment – Gene Therapy, TFPI Inhibition, Antithrombin Silencing, and Mimicking Factor VIII with an Engineered Antibody. Transfusion Medicine and Hemotherapy, 2018, 45, 2. https://www.karger.com/Article/FullText/488152. Accessed 22nd August 2018

60 A Trial Evaluating the Efficacy and Safety of Prophylactic Administration of Concizumab in Haemophilia A and B Patients with Inhibitors (explorer™4). https://clinicaltrials.gov/ct2/show/NCT03196284?term=concizumab&cond=Hemophilia&rank=3 Last updated October 2018. Accessed 22nd August 2018

61 A Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients with Severe Haemophilia A Without Inhibitors (explorer™5). https://clinicaltrials.gov/ct2/show/NCT03196297?term=concizumab&cond=Hemophilia&rank=1 Last updated September 2018. Accessed 22nd August 2018

62 Multiple Escalating Dose Study of BAY1093884 in Adults With Haemophilia A or B With or Without Inhibitors https://clinicaltrials.gov/ct2/show/NCT03597022?term=anti-TFPI&cond=Hemophilia&rank=1 Last updated October 2018. Accessed 5th November 2018