The modern treatment of haemophilia: A narrative review

Blood Transfusion

Volumn 11, Issue 2, 2013, Pages 178-182

  Franchini, Massimo ^a  

^a CARLO POMA HOSPITAL   (Italy)

Author keywords

Factor concentrates; Haemophilia; Hepatitis; HIV; Therapy

Indexed keywords

BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 8 CONCENTRATE; BLOOD CLOTTING FACTOR 8 PLUS VON WILLEBRAND FACTOR; BLOOD CLOTTING FACTOR 9; BLOOD CLOTTING FACTOR 9 CONCENTRATE; DESMOPRESSIN; FANHDI; HAEMOBIONINE; HAEMOCTIN; IMMUNATE STIM PLUS; IMMUNINE STIM PLUS; RECOMBINANT BLOOD CLOTTING FACTOR 8; RECOMBINANT BLOOD CLOTTING FACTOR 9; UNCLASSIFIED DRUG;

DISEASE SEVERITY; DISEASE SURVEILLANCE; DRUG SAFETY; HEMOPHILIA; HEMOPHILIA A; HEMOPHILIA B; HISTORY OF MEDICINE; HUMAN; HYPERTHERMIC THERAPY; IMMUNOAFFINITY CHROMATOGRAPHY; ION EXCHANGE CHROMATOGRAPHY; LIFESPAN; NANOFILTRATION; PREVALENCE; REVIEW; SUBSTITUTION THERAPY; ULTRAFILTRATION;

FACTOR IX; FACTOR VIII; HEMOPHILIA A; HISTORY, 20TH CENTURY; HISTORY, 21ST CENTURY; HUMANS; RECOMBINANT PROTEINS;

EID: 84876939197     PISSN: 17232007     EISSN: None     Source Type: Journal    
DOI: 10.2450/2012.0166-11     Document Type: Review

References (29)

1. Mannucci PM, Tuddenham EGD. The hemophiliac - from royal genes to gene therapy. N Engl J Med 2001;344:1773-9. (Pubitemid 32497033)
2. Bolton-Maggs PH, Pasi KJ. Hemophilias A and B. Lancet 2003;361:1801-9.
3. Hoyer LH. Hemophilia A. N Engl J Med 1994;330:38-47.
4. Mannucci PM. Back to the future: a recent history of haemophilia treatment. Haemophilia 2008;14(Suppl. 3):10-8. (Pubitemid 351722377)
5. Pool JG, Gershgold EJ, Pappenhagen AR. High-potency antihaemophilic factor concentrate prepared from cryoglobulin precipitate. Nature 1964;203:312.
6. Mannucci PM: Hemophilia: treatment options in the twentyfirst century. J Thromb Haemost 2005;1:1349-55.
7. Nilsson IM: Experience with prophylaxis in Sweden. Semin Hematol 1993;30(3Suppl.2):16-9. (Pubitemid 23212311)
8. Mannucci PM. Desmopressin (DDAVP) in the treatment of bleeding disorders: the first twenty years. Haemophilia 2000;6(Suppl. 1):60-7. (Pubitemid 30468295)
9. White GC, McMillan CW, Kingdon HS, Shoemaker CB. Use of recombinant antihemophilic factor in the treatment of two patients with classic hemophilia. N Engl J Med 1989;320:166-70. (Pubitemid 19033095)
10. Franchini M, Mannucci PM. Co-morbidities and quality of life in elderly persons with haemophilia. Br J Haematol 2010;148:522-33.
11. Franchini M, Tagliaferri A, Mannucci PM. The management of hemophilia in elderly patients. Clin Interv Aging 2007;2:361-8.
12. Franchini M, Lippi G, Montagnana M, et al. Hemophilia and cancer: a new challenge for hemophilia centers. Cancer Treat Rev 2009;35:374-7.
13. Azzi A. Ciappi S, Zakvrewska K, et al. Human parvovirus B 19 infection in hemophiliacs first infused with two high-purity, virally attenuated, factor VIII concentrates. Am J Hematol 1992;39:228-30.
14. Peden A, McCardle L, Head MW, et al. Variat CJD infection in the spleen of a neurologically asymptomatic UK adult patient with haemophilia. Haemophilia 2010:16:296-304.
15. Makris M, Calizzani G, Fischer K, et al. EUHASS: The European Haemophilia Safety Surveillance system. Thromb Res 2011;127(Suppl. 2):S22-5.
16. Franchini M, Lippi G. Recombinant FVIII products. Semin Thromb Hemost 2010;36:537-49.
17. Astermark J. Inhibitor development: patient-determined risk factors. Haemophilia 2010;16:66-70.
18. Astermark J. Overview of inhibitors. Semin Hematol 2006;43(Suppl. 2):S3-S7.
19. Franchini M, Lippi G. VWF-containing FVIII concentrates and inhibitors in hemophilia A: a critical literature review. Thromb Haemost 2010;104:931-40.
20. Mannucci PM. Plasma-derived versus recombinant factor VIII concentrates for the treatment of haemophilia A: plasma-derived is better. Blood Transfus 2010;8:288-91.
21. Franchini M. Plasma-derived versus recombinant FVIII concentrates for the treatment of hemophilia A: recombinant is better. Blood Transfus 2010;8:292-6.
22. Gringeri A. Factor VIII safety: plasma-derived versus recombinant products. Blood Transfus 2011;9:366-70.
23. Wight J, Paisley S. The epidemiology of inhibitors in haemophilia A: a systematic review. Haemophilia 2003;9:418-35. (Pubitemid 36874391)
24. Scharrer I, Ehrlich HJ. Reported inhibitor incidence in FVIII PUP studies: comparing apples with oranges? Haemophilia 2004;10:197-8. (Pubitemid 38312651)
25. Iorio A, Halimeh S, Holzhauer S, et al. Rate of inhibitor development in previously-untreated hemophilia a patients treated with plasma-derived or recombinant factor VIII concentrates. A systematic review. J Thromb Haemost 2010;8:1256-65.
26. Franchini M, Tagliaferri A, Mengoli C, Cruciani M. Cumulative inhibitor incidence in previously untreated patients with severe hemophilia A treated with plasma-derived versus recombinant factor FVIII concentrates: a critical systematic review. Crit Rev Oncol Hematol 2012;81:82-93.
27. Aledort LM, Navickis RJ, Wilkes MM. Can B-domain deletion alter the immunogenicity of recombinant factor VIII? A metaanalysis of prospective clinical studies. J Thromb Haemost 2011;9:2180-92.
28. Mannucci PM, Gringeri A, Peyvandi F, Santagostino E. Factor VIII products and inhibitor development: the SIPPET study (survey of inhibitors in plasma-product exposed toddlers). Haemophilia 2007;13(Suppl. 5):65-8. (Pubitemid 350253407)
29. Mannucci PM. Treatment of hemophilia: building on strength in the third millennium. Haemophilia 2011;17(Suppl. 3):1-24.