Zum Inhalt springen


  1. The Haemophilia Society. Haemophilia. https://haemophilia.org.uk/bleeding-disorders/haemophilia-a-and-b/. Accessed November 24, 2021.
  2. National Organization for Rare Disorders (NORD). Rare Disease Database-Hemophilia B. https://rarediseases.org/rare-diseases/hemophilia-b/.Accessed October 27, 2021.
  3. Oldenburg J. Optimal treatment strategies for hemophilia: achievements and limitations of current prophylactic regimens. Blood. 2015;125(13):2038-2044.
  4. National Organization for Rare Disorders (NORD). Rare Disease Database-Hemophilia A. https://rarediseases.org/rare-diseases/hemophilia-a/. Accessed October 27, 2021.
  5. De la Corte-Rodriguez H, Rodriguez-Merchan EC. The ICF (International Classification of Functioning, Disability and Health) developed by the WHO for measuring function in hemophilia. Expert Review of Hemotology. 2016;9:7.
  6. Buckner TW, Batt K, Quon D, et al. Assessments of pain, functional impairment, anxiety, and depression in US adults with hemophilia across patient-reported outcome instruments in the Pain, Functional Impairment, and Quality of Life (P-FiQ) study. Eur J Haemotol. 2018;100(suppl. 1):5-13.
  7. Pinto PR, Paredes AC, Moreira P, et al. Emotional distress in haemophilia: Factors associated with the presence of anxiety and depression symptoms among adults. Haemophilia. 2018:1-10.
  8. Cutter S, Molter D, Dunn S, et al. Impact of mild to severe hemophilia on education and work by US men, women, and caregivers of children with hemophilia B: The Bridging Hemophilia B Experiences, Results and Opportunities into Solutions (B-HERO-S) study. Eur J Haematol. 2017.98:18-24.
  9. Witkop M, Guelcher C, Forsyth A, et al. Treatment outcomes, quality of life, and impacts of hemophilia on young adults (aged 18-30 years) with hemophilia. Am J Hematol. 2015;90(S2):S3-S10.
  10. Khair K, Holland M, Bladen M, et al. Study of physical function in adolescents with haemophilia: the SO-FIT study. Haemophilia. 2017;1-8.
  11. Perrin GQ, Herzog RW, Markusic DM. Update on clinical gene therapy for hemophilia. Blood. 2019;133(5):407-414.
  12. Grady PA, Gough LL. Self-management: a comprehensive approach to management of chronic conditions. Framing Health Matters. 2014;104(8):e25-e31.
  13. Hemophilia Federation of America (HFA). Bleeding Disorders Historical Timeline. https://www.hemophiliafed.org/updated-historical-timeline/. Accessed November 10, 2021.
  14. Ingram GIC. The history of haemophilia. J Clin Path. 1976;29:469-479.
  15. Biggs R, Douglas AS, Macfarlane RG, et al. Christmas disease a condition previously mistaken for haemophilia. Br Med J. 1952;2(4799):1378-82.
  16. Bennett B, Ratnoff OD. Detection of the carrier state for classic hemophilia. New Engl J Med. 1973;288(7):342-345.
  17. European Medicines Agency (EMA). BeneFIX. https://www.ema.europa.eu/en/medicines/human/EPAR/benefix. Accessed November 30, 2021.
  18. European Medicines Agency (EMA). ReFacto AF. https://www.ema.europa.eu/en/medicines/human/EPAR/refacto-af. Accessed November 30, 2021.
  19. Tagliaferri A, Rivolta GF, Iorio A, et al. Mortality and causes of death in Italian persons with haemophilia, 1990-2007. Haemophilia. 2010;16:437-446.
  20. Garber K. rFactor VIII deficit questioned. Nat Biotech. 2000;18:1133.
  21. Srivastava A, Santagostino E, Dougall A, et al. WFH guideline for the management of hemophilia, 3rd edition. Haemophilia. 2020;00:1-158.
  22. Nathwani AC, Reiss UM, Tuddenham EGD, et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med. 2014;371:1994-2004.
  23. Rangarajan S, Walsh L, Lester W, et al. AAV5-Factor VII Gene Transfer in Severe Hemophilia A. N Engl J Med. 2017;377(26):2519-2530.