Advances in Hemophilia: Experimental Aspects and Therapy

Hematology/Oncology Clinics of North America

Volumn 24, Issue 1, 2010, Pages 181-198

  Rodriguez, Nidra I ^a,b   Hoots, W Keith ^a,b  

^a UNIVERSITY OF TEXAS MEDICAL SCHOOL   (United States)

^b Gulf States Hemophilia and Thrombophilia Center   (United States)

Author keywords

Hemophilia; Inhibitors; Prophylaxis

Indexed keywords

ACTIVATED PROTHROMBIN COMPLEX; AMINOCAPROIC ACID; ANTIFIBRINOLYTIC AGENT; BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 8 CONCENTRATE; BLOOD CLOTTING FACTOR 9; BLOOD CLOTTING FACTOR 9 CONCENTRATE; BLOOD CLOTTING FACTOR CONCENTRATE; DESMOPRESSIN; FIBRIN GLUE; IRON; LIPOSOME; MACROGOL; PARVOVIRUS VECTOR; PHOSPHORUS 32; POLYSIALIC ACID; RECOMBINANT BLOOD CLOTTING FACTOR 7A; RECOMBINANT BLOOD CLOTTING FACTOR 8; RECOMBINANT BLOOD CLOTTING FACTOR 9; RITUXIMAB; THROMBIN; TRANEXAMIC ACID; VON WILLEBRAND FACTOR; YTTRIUM 90;

ACUTE LYMPHOBLASTIC LEUKEMIA; ADJUVANT THERAPY; ANTIGENICITY; ARTHROPATHY; AUTOIMMUNE HEMOLYTIC ANEMIA; B CELL LEUKEMIA; BIOENGINEERING; BLEEDING; CLINICAL EFFECTIVENESS; CLINICAL TRIAL; DISEASE SEVERITY; DOSAGE SCHEDULE COMPARISON; DOSE RESPONSE; DRUG BLOOD LEVEL; DRUG DOSAGE FORM COMPARISON; DRUG DOSE ESCALATION; DRUG DOSE INCREASE; DRUG EFFICACY; DRUG FORMULATION; DRUG HALF LIFE; DRUG MEGADOSE; DRUG POTENTIATION; DRUG SAFETY; EARLY INTERVENTION; GENE MUTATION; GENETIC ANALYSIS; HEALTH CARE COST; HEMARTHROSIS; HEMOPHILIA; HEMOPHILIA A; HEMOPHILIA B; HEMOSTASIS; HUMAN; HUMAN VERSUS ANIMAL COMPARISON; IDIOPATHIC THROMBOCYTOPENIC PURPURA; IMMUNOLOGICAL TOLERANCE; IMMUNOPATHOLOGY; INFLAMMATION; LOW DRUG DOSE; MUCOSAL BLEEDING; NEOVASCULARIZATION (PATHOLOGY); NONHUMAN; PATIENT SAFETY; PRIMARY PREVENTION; PRIORITY JOURNAL; RADIATION INJURY; RADIOACTIVE SYNOVECTOMY; RADIODIAGNOSIS; REVIEW; RISK BENEFIT ANALYSIS; SECONDARY PREVENTION; SYNOVECTOMY; SYNOVIORTHESIS; SYNOVITIS; VIRAL GENE THERAPY; VON WILLEBRAND DISEASE;

FACTOR IX; FACTOR VIII; HEMARTHROSIS; HEMOPHILIA A; HUMANS; SYNOVITIS;

EID: 74749087269     PISSN: 08898588     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.hoc.2009.11.003     Document Type: Review

References (54)

1. Kessler C.M. New perspectives in hemophilia treatment. Hematology Am Soc Hematol Educ Program (2005) 429-435
2. Berntorp E., Astermark J., Bjorkman S., et al. Consensus perspectives on prophylactic therapy for haemophilia: summary statement. Haemophilia 9 Suppl 1 (2003) 1-4
3. Galiè N., Seeger W., Naeije R., et al. Comparative analysis of clinical trials and evidence-based treatment algorithm in pulmonary arterial hypertension. J Am Coll Cardiol 43 (2004) 81-88
4. Manco-Johnson M.J., Abshire T.C., Shapiro A.D., et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med 357 6 (2007) 535-544
5. Astermark J., Petrini P., Tengborn L., et al. Primary prophylaxis in severe haemophilia should be started at an early age but can be individualized. Br J Haematol 105 4 (1999) 1109-1113
6. Funk M., Schmidt H., Escuriola-Ettingshausen C., et al. Radiological and orthopedic score in pediatric hemophilic patients with early and late prophylaxis. Ann Hematol 77 4 (1998) 171-174
7. Kreuz W., Escuriola-Ettingshausen C., Funk M., et al. When should prophylactic treatment in patients with haemophilia A and B start?-The German experience. Haemophilia 4 4 (1998) 413-417
8. Petrini P., Lindvall N., Egberg N., et al. Prophylaxis with factor concentrates in preventing hemophilic arthropathy. Am J Pediatr Hematol Oncol 13 3 (1991) 280-287
9. Fischer K., van der Bom J.G., Mauser-Bunschoten E.P., et al. The effects of postponing prophylactic treatment on long-term outcome in patients with severe hemophilia. Blood 99 7 (2002) 2337-2341
10. Aledort L.M., Haschmeyer R.H., and Pettersson H. A longitudinal study of orthopaedic outcomes for severe factor-VIII-deficient haemophiliacs. The Orthopaedic Outcome Study Group. J Intern Med 236 4 (1994) 391-399
11. Smith P.S., Teutsch S.M., Shaffer P.A., et al. Episodic versus prophylactic infusions for hemophilia A: a cost-effectiveness analysis. J Pediatr 129 3 (1996) 424-431
12. Szucs T.D., Offner A., Kroner B., et al. Resource utilisation in haemophiliacs treated in Europe: results from the European Study on Socioeconomic Aspects of Haemophilia Care. The European Socioeconomic Study Group. Haemophilia 4 4 (1998) 498-501
13. Fischer K., van der Bom J.G., Molho P., et al. Prophylactic versus on-demand treatment strategies for severe haemophilia: a comparison of costs and long-term outcome. Haemophilia 8 6 (2002) 745-752
14. Petrini P. What factors should influence the dosage and interval of prophylactic treatment in patients with severe haemophilia A and B?. Haemophilia 7 1 (2001) 99-102
15. van den Berg H.M., Fischer K., Mauser-Bunschoten E.P., et al. Long-term outcome of individualized prophylactic treatment of children with severe haemophilia. Br J Haematol 112 3 (2001) 561-565
16. Feldman B.M., Pai M., Rivard G.E., et al. Tailored prophylaxis in severe hemophilia A: interim results from the first 5 years of the Canadian Hemophilia Primary Prophylaxis Study. J Thromb Haemost 4 6 (2006) 1228-1236
17. Villar A., Jimenez-Yuste V., Quintana M., et al. The use of haemostatic drugs in haemophilia: desmopressin and antifibrinolytic agents. Haemophilia 8 (2002) 189-193
18. Galves A., Gomez-Ortiz G., Diaz-Ricart M., et al. Desmopressin (DDAVP) enhances platelet adhesion to the extracellular matrix of cultured human endothelial cells through increased expression of tissue factor. Thromb Haemost 77 (1997) 975-980
19. Winterbottom N., Kuo J.M., Nguyen K., et al. Antigenic responses to bovine thrombin exposure during surgery: a prospective study of 309 patients. Journal of Applied Research 2 (2002) 1-11
20. Bruce M.E., Will R.G., Ironside J.W., et al. Transmissions to mice indicate that 'new variant' CJD is caused by the BSE agent. Nature 389 6650 (1997) 498-501
21. Chapman W.C., Singla N., Genyk Y., et al. A phase 3, randomized, double-blind comparative study of the efficacy and safety of topical recombinant human thrombin and bovine thrombin in surgical hemostasis. J Am Coll Surg 205 2 (2007 Aug) 256-265
22. O'Connell N., Mc Mahon C., Smith J., et al. Recombinant factor VIIa in the management of surgery and acute bleeding episodes in children with haemophilia and high responding inhibitors. Br J Haematol 116 3 (2002) 632-635
23. Hedner U. Treatment of patients with factor VIII and IX inhibitors with special focus on the use of recombinant factor VIIa. Thromb Haemost 82 2 (1999) 531-539
24. Rodriguez-Merchan E.C. Effects of hemophilia on articulations of children and adults. Clin Orthop Relat Res 328 (1996) 7-13
25. Dunn A.L., Busch M.T., Wyly J.B., et al. Radionuclide synovectomy for hemophilic arthropathy: a comprehensive review of safety and efficacy and recommendation for a standarized treatment protocol. Thromb Haemost 87 (2002) 383-393
26. Manco-Johnson M.J., Nuss R., Lear J., et al. 32P radiosynoviorthesis in children with hemophilia. J Pediatr Hematol Oncol 24 (2002) 534-539
27. Infante-Rivard C., Rivard G., Winikoff R., et al. Is there an increased risk of cancer associated with radiosynoviorthesis? [abstract 18FP539]. Haemophilia 12 2 (2006)
28. Astermark J. Basic aspects of inhibitors to factors VIII and IX and the influence of non-genetic risk factors. Haemophilia 12 6 (2006) 8-14
29. Scharrer I., Bray G.L., and Neutzling O. Incidence of inhibitors in haemophilia A patients-a review of recent studies of recombinant and plasma-derived factor VIII concentrates. Haemophilia 5 (1999) 145-154
30. Goudemand J., Rothschild C., Demiguel V., et al. Influence of the type of factor VIII concentrate on the incidence of factor VIII inhibitors in previously untreated patients with severe hemophilia A. Blood 107 1 (2006) 46-51
31. Gouw S.C., van der Bom J.G., Auerswald G., et al. Recombinant versus plasma-derived factor VIII products and the development of inhibitors in previously untreated patients with severe hemophilia A: the CANAL cohort study. Blood 109 11 (2007) 4693-4697
32. Scandella D., de Graaf Mahoney S., Mattingly M., et al. Epitope mapping of human FVIII inhibitor antibodies by deletion analysis of FVIII fragments expressed in Escherichia coli. Proc Natl Acad Sci U S A 85 (1988) 6152-6156
33. Auerswald G., Spranger T., and Brackmann H.H. The role of plasma-derived factor VIII/von Willebrand factor concentrates in the treatment of hemophilia A patients. Haematologica 88 9 (2003) 16-20
34. Suzuki T., Arai M., Amano K., et al. Factor VIII inhibitor antibodies with C2 domain specificity are less inhibitory to factor VIII complexed with von Willebrand factor. Thromb Haemost 76 (1996) 749-754
35. Gensana M., Altisent C., Aznar J.A., et al. Influence of von Willebrand factor on the reactivity of human factor VIII inhibitors with factor VIII. Haemophilia 7 (2001) 369-374
36. Behrmann M., Pasi J., Saint-Remy J.M., et al. von Willebrand factor modulates factor VIII immunogenicity: comparative study of different factor VIII concentrates in a haemophilia A mouse model. Thromb Haemost 88 (2002) 221-229
37. Gringeri A., Monzini M., Tagariello G., et al. Occurrence of inhibitors in previously untreated or minimally treated patients with haemophilia A after exposure to a plasma-derived solvent-detergent factor VIII concentrate. Haemophilia 12 (2006) 128-132
38. Gringeri A., Musso R., Mazzucconi G., et al. Immune tolerance induction with a high purity von Willebrand factor/VIII complex concentrate in haemophilia A patients with inhibitors at high risk of a poor response. Haemophilia 13 (2007) 373-379
39. Gopal A.K., and Press O.W. Clinical applications of anti CD 20 antibodies. J Lab Clin Med 134 (1999) 445-450
40. Stasi R., Pagano A., Stipa E., et al. Rituximab chimeric anti-CD 20 monoclonal antibody treatment for adults with chronic idiopathic thrombocytopenic purpura. Blood 98 (2001) 952-957
41. Zecca M., Nobili B., Ramenghi U., et al. Rituximab for the treatment of refractory autoimmune hemolytic anemia in children. Blood 101 (2003) 3857-3861
42. Carcao M., St. Louis J., Poon M.C., et al. Rituximab for congenital haemophiliacs with inhibitors: a Canadian experience. Haemophilia 12 (2006) 7-18
43. Fox R.A., Neufeld E.J., and Bennett C.M. Rituximab for adolescents with haemophilia and high titre inhibitors. Haemophilia 12 (2006) 218-222
44. Lawn R.M. The molecular genetics of hemophilia: blood clotting factors VIII and IX. Cell 42 (1985) 405-406
45. Goodeve A.C., and Peake I.R. The molecular basis of hemophilia A: genotype-phenotype relationships and inhibitor development. Semin Thromb Hemost 29 1 (2003) 23-30
46. Pierce G.F., Lillicrap D., Pipe S.W., et al. Gene therapy, bioengineered clotting factors and novel technologies for hemophilia treatment. J Thromb Haemost 5 (2007) 901-906
47. Ponder K.P. Gene therapy for hemophilia. Curr Opin Hematol 13 5 (2006) 301-307
48. Miao H.Z., Sirachainan N., Palmer L., et al. Bioengineering of coagulation factor VIII for improved secretion. Blood 103 (2004) 3412-3419
49. Pipe S.W. The promise and challenges of bioengineered recombinant clotting factors. J Thromb Haemost 3 (2005) 1692-1701
50. Abuchowski A., van Es T., Palczuk N.C., et al. Alteration of immunological properties of bovine serum albumin by covalent attachment of polyethylene glycol. J Biol Chem 252 (1977) 3578-3581
51. Molineaux G. Pegylation: engineering improved biopharmaceuticals for oncology. Pharmacotherapy 23 (2003) 3S-8S
52. Baru M., Carmel-Goren L., Barenholz Y., et al. Factor VIII efficient and specific non-covalent binding to PEGylated liposomes enables prolongation of its circulation time and haemostatic efficacy. Thromb Haemost 93 (2005) 1061-1068
53. Goyal P., Goyal K., Vijaya Kumar S.G., et al. Liposomal drug delivery systems-clinical applications. Acta Pharm 55 (2005) 1-25
54. Spira J., Ply Ushch O.P., Andreeva T.A., et al. Prolonged bleeding-free period following prophylactic infusion of recombinant factor VIII (Kogenate (R) FS) reconstituted with pegylated liposomes. Blood 108 (2006) 3668-3673