관련근거
1.Hematology: Basic Principles and Practice, 8e, 2022, Chapter 134. Hemophilia A and B
2.Conn's Current Therapy 2025, Chapter_Hemophilia and Related Conditions
3.Current Medical Diagnosis & Treatment 2026, 16-08_ Congenital Disorders of Coagulation
4.Williams Hematology, 10e, 2021_Chapter 122_ Hemophilia A and Hemophilia B
5.WFH Guidelines for the Management of Hemophilia 3rd Edition, 2020
6.MASAC Document #268 - RECOMMENDATION ON THE USE AND MANAGEMENT OF EMICIZUMAB-KXWH(HEMLIBRA®) FOR HEMOPHILIA A WITH AND WITHOUT INHIBITORS (2022)
7.Specialist services for haemophilia and related bleeding disorders (adults and children), 2025
8.DRAFT Recommendations for public comments: The annual bleeding rate (ABR) in hemophilia ？ Harmonizing definition, recording and reporting: Recommendations of ISTH ？ SSC FVIII / IX Sub-committee Working Group(2024)
9.Rayment R, et al. Guidelines on the use of prophylactic factor replacement for children and adults with Haemophilia A and B. Br J Haematol. 2020 Sep;190(5):684-695.
10.Holstein K, et al. Practical Guidance of the GTH Haemophilia Board on the Use of Emicizumab in Patients with Haemophilia A. Hamostaseologie 2020 Dec;40(5):561？571.
11.Nordic Hemophilia Council Hemophilia Guidelines, 2025
12.Coppola A, et al. Management of patients with severe haemophilia a without inhibitors on prophylaxis with emicizumab: AICE recommendations with focus on emergency in collaboration with SIBioC, SIMEU, SIMEUP, SIPMeL and SISET. Haemophilia. 2020 Nov;26(6): 937-945.
13.Oldenburg J, et al. Emicizumab Prophylaxis in Hemophilia A with Inhibitors. N Engl J Med. 2017 Aug 31;377(9):809-818.
14.Young G, et al. A multicenter, open-label phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors. Blood. 2019 Dec 12;134(24):2127-2138.
15.Mahlangu J, et al. Emicizumab Prophylaxis in Patients Who Have Hemophilia A without Inhibitors. N Engl J Med. 2018 Aug 30;379(9):811-822.
16.Pipe SW, et al. Efficacy, safety, and pharmacokinetics of emicizumab prophylaxis given every 4 weeks in people with haemophilia A (HAVEN 4): a multicentre, open-label, non-randomised phase 3 study. Lancet Haematol. 2019 Jun;6(6):e295-e305.
17.Shima M, et al. A multicentre, open-label study of emicizumab given every 2 or 4 weeks in children with severe haemophilia A without inhibitors. Haemophilia. 2019 Nov;25(6):979-987.
18.Jimenez-Yuste V, et al. Safety and efficacy of long-term emicizumab prophylaxis in hemophilia A with factor VIII inhibitors: A phase 3b, multicenter, single-arm study (STASEY). Res Pract Thromb Haemost. 2022 Nov 14;6(8):e12837.
19.Mahlangu J, et al. Long-term outcomes with emicizumab in hemophilia A without inhibitors: results from the HAVEN 3 and 4 Studies. Res Pract Thromb Haemost. 2024 Mar 1;8(2):102364
20.Kiialainen A, et al. Effect of emicizumab prophylaxis on bone and joint health markers in people with haemophilia A without factor VIII inhibitors in the HAVEN 3 study. Haemophilia. 2022 Nov;28(6):1033-1043.
21.Kruse-Jarres R, et al. Evolution of joint health and physical activity in people with hemophilia A without factor VIII inhibitors switching to emicizumab prophylaxis: A second interim analysis of the BEYOND ABR study. Blood 146(2025) 1285-1286.
22.Shima M, et al. AOZORA study: 3-year interim analysis of safety and joint health in pediatric people with hemophilia A receiving emicizumab prophylaxis. Res Pract Thromb Haemost. 2025 Oct 21;9(8):103228.
23.Poenaru DV, et al. Orthopaedic and surgical features in the management of patients with haemophilia. Eur J Orthop Surg Traumatol. 2014 Jul;24(5):685-92.
24.Gualtierotti R, et al. Hemophilic arthropathy: Current knowledge and future perspectives. J Thromb Haemost. 2021 Sep;19(9):2112-2121.
25.Kenet G, et al. Real-World Rates of Bleeding, Factor VIII Use, and Quality of Life in Individuals with Severe Haemophilia A Receiving Prophylaxis in a Prospective, Noninterventional Study. J Clin Med. 2021 Dec 18;10(24):5959.
26.Kempton C, et al. Development and testing of the Satisfaction Questionnaire with Intravenous or Subcutaneous Hemophilia Injection and resultsfrom the Phase 3 HAVEN 3 study of emicizumab prophylaxis in persons with haemophilia A without FVIII inhibitors. Haemophilia. 2021 Mar;27(2):221-228.
27.Schmitt C, et al. Emicizumab dose up-titration in case of suboptimal bleeding control in people with haemophilia A. Haemophilia. 2023 Jan;29(1):90-99.
28.NHS England Reference: 170134/P(August 2019) Clinical Commissioning Policy: Emicizumab as prophylaxis in people with congenital haemophilia A without factor VIII inhibitors (all ages)
29.Aetna: Specialty Pharmacy Clinical Policy Bulletins Aetna Non-Medicare Prescription Drug Plan Subject: Hemlibra SGM 2417-A, 3536-A P2025(May 20, 2025)