Haemophilia: Provision of factors and novel therapies: World Federation of Hemophilia goals and achievements

British Journal of Haematology

Volumn 154, Issue 6, 2011, Pages 704-714

  Skinner, Mark W ^a  

^a WORLD FEDERATION OF HEMOPHILIA   (Canada)

Author keywords

Bleeding disorders; Haemophilia; Treatment for All; WFH Global Survey; World Federation of Hemophilia

Indexed keywords

BLOOD CLOTTING FACTOR 8; FRESH FROZEN PLASMA; RECOMBINANT BLOOD CLOTTING FACTOR 8;

AGE DISTRIBUTION; BLEEDING TIME; BLOOD CLOTTING FACTOR 11 DEFICIENCY; DECISION MAKING; DISEASE SEVERITY; DRUG COST; DRUG EFFICACY; DRUG SAFETY; HEALTH CARE COST; HEALTH CARE DELIVERY; HEALTH CARE ORGANIZATION; HEALTH CARE PERSONNEL; HEALTH CARE PLANNING; HEMOPHILIA; HUMAN; IMMUNE RESPONSE; IMMUNOGENICITY; PARTIAL THROMBOPLASTIN TIME; PATIENT CARE; PATIENT COMPLIANCE; PATIENT EDUCATION; POSTPARTUM HEMORRHAGE; PRIORITY JOURNAL; PROTHROMBIN TIME; QUALITY OF LIFE; REVIEW; SEX DIFFERENCE; SUBSTITUTION THERAPY; VON WILLEBRAND DISEASE;

BLEEDING DISORDERS; HAEMOPHILIA; TREATMENT FOR ALL; WFH GLOBAL SURVEY; WORLD FEDERATION OF HEMOPHILIA;

BLOOD COAGULATION DISORDERS, INHERITED; DELIVERY OF HEALTH CARE; FACTOR VIII; FEMALE; HEALTH SERVICES ACCESSIBILITY; HEMOPHILIA A; HUMANS; INTERNATIONAL COOPERATION; MALE; ORGANIZATIONAL OBJECTIVES; WORLD HEALTH;

EID: 80052262310     PISSN: 00071048     EISSN: 13652141     Source Type: Journal    
DOI: 10.1111/j.1365-2141.2011.08765.x     Document Type: Review

References (83)

1. Barr, R.D., Sek, J., Horsman, J., Furlong, W., Saleh, M., Pai, M. & Walker, I. (2003) Health status and health-related quality of life associated with von Willebrand disease. American Journal of Hematology, 73, 108-114.
2. Batorova, A., High, K.A. & Gringeri, A. (2010) Special lectures in haemophilia management. Haemophilia, 16, 22-28.
3. Bolton-Maggs, P.H.B. (2006) Optimal haemophilia care versus the reality. British Journal of Haematology, 132, 671-682.
4. Burnouf, T., Caron, C., Radosevich, M., Goubran, H.A., Goudemand, J. & El-Ekiaby, M. (2008) Properties of a concentrated minipool solvent-detergent treated cryoprecipitate processed in single-use bag systems. Haemophilia, 14, 956-962.
5. Centers for Disease Control Prevention (CDC) (2003) Universal Data Collection Program (UDC) report 2003/Vol.5/No.1. Centers for Disease Control and Prevention, Atlanta, GA.
6. Centers for Disease Control and Prevention (CDC) (2011) Universal Data Collection Program (UDC) Data Report. Last accessed 12 May 2011.
7. Clinical and Laboratory Standards Institute (2008) One stage Prothrombin Time (PT)test and Activated Partial Thromboplsatuin Time (APTT)Test: Approved guideline, 2nd edn. CLSI H47-A2, Wayne, PA.
8. Colvin, B.T., Astermark, J., Fischer, K., Gringeri, A., Lassila, R., Schramm, W., Thomas, A., Ingerslev, J. for The Inter Disciplinary Working Group (2008) European principles of haemophilia care. Haemophilia, 14, 361-374.
9. El-Ekiaby, M., Sayed, M.A., Caron, C., Burnouf, S., El-Sharkawy, N., Goubran, H., Radosevich, M., Goudemand, J., Blum, D., De Melo, L., Soulié, V., Adam, J. & Burnouf, T. (2010) Solvent-detergent filtered (S/D-F) fresh frozen plasma and cryoprecipitate minipools prepared in a newly designed integral disposable processing bag system. Transfusion Medicine, 20, 48-61.
10. European Directorate of the Quality of Medicines & HealthCare (EDQM) (2009) Proceedings of the International Symposium on the Optimal Clinical Use of Blood Components; 24-25 April 2009; Page 332.
11. European Haemophilia Consortium (2007) Summary Report EHC Policy Roundtable on the Role of Patient Registries for People with Haemophilia. European Haemophilia Consortium, Brussels, Belgium.
12. European Medicines Agency (2011) London, UK. Last accessed 12 April 2011.
13. Evatt, B. (2002) Observations from Global Survey 2001: an emerging database for progress. Haemophilia, 8, 153-156.
14. Evatt, B. (2005) Guide to Developing a National Patient Registry. World Federation of Hemophilia, Montreal, QC, Canada.
15. Evatt, B.L. (2006) The natural evolution of haemophilia care: developing and sustaining comprehensive care globally. Haemophilia, 12, 13-21.
16. Evatt, B.L., Austin, H., Leon, G., Ruiz-Sáez, A. & De Bosch, N. (1999) Haemophilia therapy: assessing the cumulative risk of HIV exposure by cryoprecipitate. Haemophilia, 5, 295-300.
17. Farrugia, A. (2008) Guide for the Assessment of Clotting Factor Concentrates for the Treatment of Hemophilia, 2nd edn. World Federation of Hemophilia, Montreal, QC, Canada.
18. Greer, I.A., Lowe, G.D., Walker, J.J. & Forbes, C.D. (1991) Haemorrhagic problems in obstetrics and gynaecology in patients with congenital coagulopathies. British Journal of Obstetrics and Gynaecology, 98, 909-918.
19. Gringeri, A., Lundin, B., Mackensen, S.V., Mantovani, L. & Mannucci, P.M. (2011) A Randomized Clinical Trial of Prophylaxis in Children with Hemophilia A (the ESPRIT Study). Journal of Thrombosis and Haemostasis, 9, 700-710.
20. Guh, S., Grosse, S.D., McAlister, S., Kessler, C.M. & Soucie, J.M. (2011) Costs of care for privately insured males with hemophilia in the United States, 2008. HTRS 2011 Annual Scientific Symposium Abstracts. Haemophilia, 17, 566.
21. Hoveyda, F. & MacKenzie, I.Z. (2001) Secondary postpartum haemorrhage: incidence, morbidity and current management. British Journal of Obstetrics and Gynaecology, 108, 927-930.
22. Institute of Medicine (1995) HIV and the Blood Supply: An Analysis of Crisis Decisionmaking. National Academy Press, Washington, DC, USA.
23. Jennings, I., Kitchen, D.P., Woods, T.A.L., Kitchen, S., Walker, I.D. & Preston, F.E. (2009) Laboratory performance in the World Federation of Hemophilia EQA programme, 2003-2008. Haemophilia, 15, 571-577.
24. Kadir, R.A. & James, A.H. (2009) Reproductive Health in Women with Bleeding Disorders. WFH Treatment of Hemophilia. Monograph; 48: 11. World Federation of Hemophilia, Montreal, QC, Canada.
25. Kadir, R.A., Economides, D.L., Braithwaite, J., Goldman, E. & Lee, C.A. (1997) The obstetric experience of carriers of haemophilia. British Journal of Obstetrics and Gynaecology, 104, 803-810.
26. Kadir, R.A., Lee, C.A., Sabin, C.A., Pollard, D. & Economides, D.L. (1998) Pregnancy in women with von Willebrands disease or factor XI deficiency. British Journal of Obstetrics and Gynaecology, 105, 314-321.
27. Kasper, C.K. & Lin, J.C. (2010) How many carriers are there? Haemophilia, 16, 842.
28. Kitchen, D., McCraw, A. & Enchenagucia, M. (2010) Diagnosis of Haemophilia and Other Bleeding Disorders: A Laboratory Manual, 2nd edn. World Federation of Hemophilia, Montreal, QC, Canada.
29. Lee, C.Y., Madrazo, B. & Drukker, B.H. (1981) Ultrasonic evaluation of the postpartum uterus in the management of postpartum bleeding. Obstetrics and Gynecology, 1981, 58.
30. Lillicrap, D. (2010) Genetic Characterization of Hemophilia and Implications for Novel Therapies. ASH Annual Meeting Abstracts 116: SCI-9.
31. Manco-Johnson, M.J., Abshire, T.C., Shapiro, A.D., Riske, B., Hacker, M.R., Kilcoyne, R., Ingram, J.D., Manco-Johnson, M.L., Funk, S., Jacobson, L., Valentino, L.A., Hoots, W.K., Buchanan, G.R., DiMichele, D., Recht, M., Brown, D., Leissinger, C., Bleak, S., Cohen, A., Mathew, P., Matsunaga, A., Medeiros, D., Nugent, D., Thomas, G.A., Thompson, A.A., McRedmond, K., Soucie, J.M., Austin, H. & Evatt, B.L. (2007) Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. The New England Journal of Medicine, 357, 535-544.
32. Mannucci, P.M. (2004) World Federation of Hemophilia, Occasional Paper No 5. The Safety of Plasma-Derived Versus Recombinant Concentrates. World Federation of Hemophilia, Montreal, QC, Canada.
33. Mauser Bunschoten, E.P., van Houwelingen, J.C., Sjamsoedin Visser, E.J., van Dijken, P.J., Kok, A.J. & Sixma, J.J. (1988) Bleeding symptoms in carriers of hemophilia A and B. Thrombosis and Haemostasis, 59, 349-352.
34. Murphy, S.L. & High, K.A. (2008) Gene therapy for haemophilia. British Journal of Haematology, 140, 479-487.
35. Nathwani, A. (2010a) Haemophilia B gene therapy study in the UK. Late Breaking Abstracts from the WFH Haemophilia 2010 World Congress. Haemophilia, 2010, 16.
36. Nathwani, A., Tuddenham, E., Rosales, C., McIntosh, J., Riddell, A., Rustagi, P., Glader, B., Kay, M., Allay, J., Coleman, J., Sleep, S., High, K.A., Mingozzi, F., Gray, J.T., Reiss, U.M., Nienhuis, A.W. & Davidoff, A. (2010b) Early Clinical Trial Results Following Administration of a Low Dose of a Novel Self Complementary Adeno-Associated Viral Vector Encoding Human Factor IX In Two Subjects with Severe Hemophilia B. Blood (ASH Annual Meeting Abstracts) 116, 248.
37. Nichols, W.L., Hultin, M.B., James, A.H., Manco-Johnson, M.J., Montgomery, R.R., Ortel, T.L., Rick, M.E., Sadler, J.E., Weinstein, M. & Yawn, B.P. (2008) von Willebrand disease (VWD): evidence-based diagnosis and management guidelines, the National Heart, Lung, and Blood Institute (NHLBI) Expert Panel report (USA). Haemophilia, 14, 171-232.
38. O'Mahony, B. (2002) Global Haemophilia Care Challenge and Opportunities. Plenary Presentation World Federation of Hemophilia XXV Congress. World Federation of Hemophilia, Montreal, QC, Canada.
39. O'Mahony, B. (2006) Guide to the National Tenders for the Purchase of Clotting Factor Concentrates. World Federation of Hemophilia, Montreal, QC, Canada.
40. Plug, I., Mauser-Bunschoten, E.P., Brocker-Vriends, A.H.J.T., van Amstel, H.K.P., van der Bom, J.G., van Diemen-Homan, J.E.M., Willemse, J. & Rosendaal, F.R. (2006) Bleeding in carriers of hemophilia. Blood, 108, 52-56.
41. Podda, G.M., Bucciarelli, P., Lussana, F., Lecchi, A. & Cattaneo, M. (2007) Usefulness of PFA-100 testing in the diagnostic screening of patients with suspected abnormalities of hemostasis: comparison with the bleeding time. Journal of Thrombosis & Haemostasis, 5, 2393-2398.
42. Poon, M.-C. & Luke, K.-H. (2008) Haemophilia care in China: achievements of a decade of World Federation of Hemophilia treatment centre twinning activities. Haemophilia, 14, 879-888.
43. Prevention of Postpartum Hemorrhage Initiative, Office of Health, Infectious Diseases and Nutrition, Bureau for Global Health (2007) Website 2007. Available at. Accessed 10 April 2011.
44. Ramsahoye, B., Davies, S., Dasani, H. & Pearson, J. (1995) Obstetric management in von Willebrand's disease: a report of 24 pregnancies and a review of the literature. Haemophilia, 1, 140-144.
45. Rickard, K. (2000) The International Haemophilia Training Centres of the World Federation of Hemophilia: a 30-year review. Haemophilia, 6, 471-473.
46. Rizza, C.R., Rhymes, I.L., Austen, D.E.G., Kernoff, P.B.A. & Aroni, S.A. (1975) Detection of carriers of haemophilia: a 'blind' study. British Journal of Haematology, 30, 447-456.
47. Sekhon, B.S. & Saluja, V. (2011) Biosimilars: an overview. Biosimilars, 1, 1-11.
48. Shankar, M., Chi, C. & Kadir, R.A. (2008) Review of quality of life: menorrhagia in women with or without inherited bleeding disorders. Haemophilia, 2008, 14.
49. Skinner, M.W. (2006) Treatment for all: a vision for the future. Haemophilia, 12, 169-173.
50. Skinner, M.W. (2008) WFH - the cornerstone of global development: 45years of progress. Haemophilia, 14, 1-9.
51. Skinner, M.W. (2010) Building our global family - achieving treatment for all. Haemophilia, 16, 1-10.
52. Soucie, J.M., Nuss, R., Evatt, B., Abdelhak, A., Cowan, L., Hill, H., Kolakoski, M., Wilber, N.; and the Hemophilia Surveillance System Project Investigators (2000) Mortality among males with hemophilia: relations with source of medical care. Blood, 96, 437-442.
53. Srivastava, A. (2004) Dose and response in haemophilia - optimization of factor replacement therapy. British Journal of Haematology, 127, 12-25.
54. Stonebraker, J.S., Bolton-Maggs, P.H.B., Michael Soucie, J., Walker, I. & Brooker, M. (2010a) A study of variations in the reported haemophilia A prevalence around the world. Haemophilia, 16, 20-32.
55. Stonebraker, J.S., Brooker, M., Amand, R.E., Farrugia, A. & Srivastava, A. (2010b) A study of reported factor VIII use around the world. Haemophilia, 16, 33-46.
56. Stonebraker, J.S., Bolton-Maggs, P.H.B., Brooker, M., Farrugia, A. & Srivastava, A. (2011) A study of reported factor IX use around the world. Haemophilia, 17, 446-455.
57. Street, A.M., Ljung, R. & Lavery, S.A. (2008) Management of carriers and babies with haemophilia. Haemophilia, 14(Suppl. 3), 181-187.
58. United Nations (2010). United Nations Millennium Development Goals - Goal 4 Child Health. Available at.
59. Wight, J. & Paisley, S. (2003) The epidemiology of inhibitors in haemophilia A; a systematic review. Haemophilia, 9, 418-435.
60. World Federation of Hemophilia (1963) Minutes for the first meeting of the World Federation of Hemophilia. Copenhagen, Denmark, June 25-28. World Federation of Hemophilia, Montreal, QC, Canada.
61. World Federation of Hemophilia (1999) Global Survey on Hemophilia. World Federation of Hemophilia, Montreal, QC, Canada.
62. World Federation of Hemophilia (2000) Report on the Annual Global Survey 2000. World Federation of Hemophilia, Montreal, QC, Canada.
63. World Federation of Hemophilia (2001) Report on the Annual Global Survey 2001. World Federation of Hemophilia, Montreal, QC, Canada.
64. World Federation of Hemophilia (2002a) Report on the Annual Global Survey 2002. World Federation of Hemophilia, Montreal, QC, Canada.
65. World Federation of Hemophilia (2002b) Improving Care Beyond Our Borders - A Twinning Guide for Hemophilia Treatment Centres. World Federation of Hemophilia, Montreal, QC, Canada.
66. World Federation of Hemophilia (2004) Report on the Annual Global Survey 2003. World Federation of Hemophilia, Montreal, QC, Canada.
67. World Federation of Hemophilia (2005a) Report on the Annual Global Survey 2004. World Federation of Hemophilia, Montreal, QC, Canada.
68. World Federation of Hemophilia (2005b) Guidelines for the Management of Hemophilia. World Federation of Hemophilia, Montreal, QC, Canada.
69. World Federation of Hemophilia (2006a) Treatment for All: A Vision for Improvement. World Federation of Hemophilia, Montreal, QC, Canada.
70. World Federation of Hemophilia (2006b) Report on the Annual Global Survey 2005. World Federation of Hemophilia, Montreal, QC, Canada.
71. World Federation of Hemophilia (2007) Report on the Annual Global Survey 2006. World Federation of Hemophilia, Montreal, QC, Canada.
72. World Federation of Hemophilia (2008a) Report on the Annual Global Survey 2007. World Federation of Hemophilia, Montreal, QC, Canada.
73. World Federation of Hemophilia (2008b) Patient Outreach Guide for Hemophilia and Other Bleeding Disorders. World Federation of Hemophilia, Montreal, QC, Canada.
74. World Federation of Hemophilia (2009a) Report on the Annual Global Survey 2008. World Federation of Hemophilia, Montreal, QC, Canada.
75. World Federation of Hemophilia (2009b) Sixth Global Forum on the Safety and Supply of Treatment Products for Bleeding Disorders Proceedings, 29-36, World Federation of Hemophilia, Montreal, QC, Canada.
76. World Federation of Hemophilia (2010) Accredited National Member Organizations of the WFH. World Federation of Hemophilia, Montreal, QC, Canada.
77. World Federation of Hemophilia (2011a) WFH Annual Report 2010. World Federation of Hemophilia, Montreal, QC, Canada.
78. World Federation of Hemophilia (2011b) Report on the Annual Global Survey 2009. World Federation of Hemophilia, Montreal, QC, Canada.
79. World Health Organization (1998) Developing a National Policy and Guidelines on the Clinical Use of Blood. World Health Organization, Geneva, Switzerland.
80. World Health Organization (2002) Delivery of Treatment for Haemophilia, Report of a Joint WHO/WFH/ISTH Meeting. London, UK. World Health Organization, Geneva, Switzerland.
81. World Health Organization (2010a) WHO Model List of Essential Medicines; 16th list (updated) March 2010. World Health Organization, Geneva, Switzerland.
82. World Health Organization (2010b) WHO Model Formulary for Children 2010 based on the Second Model List of Essential medicines for Children 2009. World Health Organization, Geneva, Switzerland.
83. World Health Organization (2010c) WHO Essential Medicines Fact Sheet 325 (revised June 2010). World Health Organization, Geneva, Switzerland.