Are You a Healthcare Professional?
This European website, initiated and developed by CSL Behring, has two separate sections with the aim to provide information on haemophilia for an international audience, either to European healthcare professionals or to the general public.*
Yes, I am a healthcare professional*
No, I am not a healthcare professional
- The Haemophilia Society. Haemophilia. https://haemophilia.org.uk/bleeding-disorders/haemophilia-a-and-b/. Accessed November 24, 2021.
- National Organization for Rare Disorders (NORD). Rare Disease Database-Hemophilia B. https://rarediseases.org/rare-diseases/hemophilia-b/. Accessed October 27, 2021.
- Oldenburg J. Optimal treatment strategies for hemophilia: achievements and limitations of current prophylactic regimens. Blood. 2015;125(13):2038-2044.
- National Organization for Rare Disorders (NORD). Rare Disease Database-Hemophilia A. https://rarediseases.org/rare-diseases/hemophilia-a/. Accessed October 27, 2021.
- 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 Rev of Hematol. 2016;9:7.
- 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.
- 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.
- 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.
- 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.
- Khair K, Holland M, Bladen M, et al. Study of physical function in adolescents with haemophilia: the SO-FIT study. Haemophilia. 2017;1-8.
- Perrin GQ, Herzog RW, Markusic DM. Update on clinical gene therapy for hemophilia. Blood. 2019;133(5):407-414.
- Grady PA, Gough LL. Self-management: a comprehensive approach to management of chronic conditions. Framing Health Matters. 2014;104(8):e25-e31.
- Hemophilia Federation of America (HFA). Bleeding Disorders Historical Timeline. https://www.hemophiliafed.org/history-of-bleeding-disorders/. Accessed July 14, 2023.
- Ingram GIC. The history of haemophilia. J Clin Path. 1976;29:469-479.
- Biggs R, Douglas AS, Macfarlane RG, et al. Christmas disease a condition previously mistaken for haemophilia. Br Med J. 1952;2(4799):1378-82.
- Bennett B, Ratnoff OD. Detection of the carrier state for classic hemophilia. New Engl J Med. 1973;288(7):342-345.
- European Medicines Agency (EMA). BeneFIX. https://www.ema.europa.eu/en/medicines/human/EPAR/benefix. Accessed November 30, 2021.
- European Medicines Agency (EMA). ReFacto AF. https://www.ema.europa.eu/en/medicines/human/EPAR/refacto-af. Accessed November 30, 2021.
- 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.
- Garber K. rFactor VIII deficit questioned. Nat Biotech. 2000;18:1133.
- Srivastava A, Santagostino E, Dougall A, et al. WFH guideline for the management of hemophilia, 3rd edition. Haemophilia. 2020;00:1-158.
- 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.
- Rangarajan S, Walsh L, Lester W, et al. AAV5-Factor VIII gene transfer in severe hemophilia A. N Engl J Med. 2017;377(26):2519-2530.
- European Medicines Agency (EMA). Roctavian. https://www.ema.europa.eu/en/medicines/human/EPAR/roctavian-0. Accessed January 28, 2023.
- Food and Drug Administration. Approved Cellular and Gene Therapy Products. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products. Accessed September 5, 2023.
- European Medicines Agency (EMA). Hemgenix. https://www.ema.europa.eu/en/medicines/human/EPAR/hemgenix. Accessed July 14, 2023.