Hemophilia

Srivastava A, Santagostino E, Dougall A, et al. WFH guidelines for the management of hemophilia, 3rd edition. Haemophilia. 2020 Aug;26 Suppl 6:1-158.Full text  Abstract

Collins PW, Chalmers E, Hart DP, et al. Diagnosis and treatment of factor VIII and IX inhibitors in congenital haemophilia: 4th edition. Br J Haematol. 2013 Jan;160(2):153-70.Full text  Abstract

Tiede A, Collins P, Knoebl P, et al. International recommendations on the diagnosis and treatment of acquired hemophilia A. Haematologica. 2020 Jul;105(7):1791-801.Full text  Abstract

National Hemophilia Foundation Medical and Scientific Advisory Council (MASAC). MASAC Document 280 - MASAC recommendations concerning products licensed for the treatment of hemophilia and selected disorders of the coagulation system. Aug 2023 [internet publication].Full text

National Hemophilia Foundation Medical and Scientific Advisory Council (MASAC). Guidelines for emergency department management of individuals with hemophilia and other bleeding disorders. Dec 2019 [internet publication].Full text

1. Blanchette VS, Key NS, Ljung LR, et al. Definitions in hemophilia: communication from the SSC of the ISTH. J Thromb Haemost. 2014 Nov;12(11):1935-9.Full text  Abstract

2. White GC 2nd, Rosendaal F, Aledort LM, et al. Definitions in hemophilia: recommendation of the scientific subcommittee on factor VIII and factor IX of the scientific and standardization committee of the International Society on Thrombosis and Haemostasis. Thromb Haemost. 2001 Mar;85(3):560. Abstract

3. Knoebl P, Marco P, Baudo F, et al. Demographic and clinical data in acquired hemophilia A: results from the European Acquired Haemophilia Registry (EACH2). J Thromb Haemost. 2012 Apr;10(4):622-31.Full text  Abstract

4. Soucie JM, Miller CH, Dupervil B, et al. Occurrence rates of haemophilia among males in the United States based on surveillance conducted in specialized haemophilia treatment centres. Haemophilia. 2020 May;26(3):487-93.Full text  Abstract

5. Centers for Disease Control and Prevention. Data & statistics on hemophilia. Sep 2020 [internet publication].Full text

6. World Federation of Hemophilia. Report on the annual global survey, 2017. Oct 2018 [internet publication].Full text

7. Pruthi RK. Hemophilia: a practical approach to genetic testing. Mayo Clin Proc. 2005 Nov;80(11):1485-99. Abstract

8. Peyvandi F, Jayandharan G, Chandy M, et al. Genetic diagnosis of haemophilia and other inherited bleeding disorders. Haemophilia. 2006 Jul;12 Suppl 3:82-9. Abstract

9. Leuer M, Oldenburg J, Lavergne JM, et al. Somatic mosaicism in hemophilia A: a fairly common event. Am J Hum Genet. 2001 Jul;69(1):75-87.Full text  Abstract

10. Graw J, Brackmann HH, Oldenburg J, et al. Haemophilia A: from mutation analysis to new therapies. Nat Rev Genet. 2005 Jun;6(6):488-501. Abstract

11. Antonarakis SE, Kazazian HH, Tuddenham EG. Molecular etiology of factor VIII deficiency in hemophilia A. Hum Mutat. 1995;5(1):1-22. Abstract

12. Goodeve AC, Peake IR. The molecular basis of hemophilia A: genotype-phenotype relationships and inhibitor development. Semin Thromb Hemost. 2003 Feb;29(1):23-30. Abstract

13. Centers for Disease Control and Prevention. CDC hemophilia mutation project (CHAMP & CHBMP). Jul 2020 [internet publication].Full text

14. Tengborn L, Baudo F, Huth-Kühne A, et al. Pregnancy-associated acquired haemophilia A: results from the European Acquired Haemophilia (EACH2) registry. BJOG. 2012 Nov;119(12):1529-37. Abstract

15. Pinchover LB, Alsharif R, Bernal T. Acquired haemophilia a secondary to multiple myeloma: management of a patient with a mechanical mitral valve. BMJ Case Rep. 2020 Sep 6;13(9):e230798. Abstract

16. Dewarrat N, Gavillet M, Angelillo-Scherrer A, et al. Acquired haemophilia A in the postpartum and risk of relapse in subsequent pregnancies: a systematic literature review. Haemophilia. 2021 Mar;27(2):199-210. Abstract

17. Monroe DM, Hoffman M. What does it take to make the perfect clot? Arterioscler Thromb Vasc Biol. 2006 Jan;26(1):41-8.Full text  Abstract

18. Oldenburg J, Pavlova A. Genetic risk factors for inhibitors to factors VIII and IX. Haemophilia. 2006 Dec;12 Suppl 6:15-22. Abstract

19. Oldenburg J, Schroder J, Brackmann HH, et al. Environmental and genetic factors influencing inhibitor development. Semin Hematol. 2004 Jan;41(1 Suppl 1):82-8. Abstract

20. Schwaab R, Brackmann HH, Meyer C, et al. Haemophilia A: mutation type determinates risk of inhibitor formation. Thromb Haemost. 1995 Dec;74(6):1402-6. Abstract

21. Astermark J, Berntorp E, White GC, et al; MIBS Study Group. The Malmo International Brother Study (MIBS): further support for genetic predisposition to inhibitor development in hemophilia patients. Haemophilia. 2001 May;7(3):267-72. Abstract

22. Astermark J, Oldenburg J, Escobar M, et al; Malmo International Brother Study Group. The Malmo International Brother Study (MIBS): genetic defects and inhibitor development in siblings with severe hemophilia A. Haematologica. 2005 Jul;90(7):924-31.Full text  Abstract

23. Gill JC. The role of genetics in inhibitor formation. Thromb Haemost. 1999 Aug;82(2):500-4. Abstract

24. National Hemophilia Foundation Medical and Scientific Advisory Council (MASAC). MASAC document 264 - MASAC recommendations regarding diagnosis and management of inherited bleeding disorders in girls and women with personal and family history of bleeding. Mar 2021 [internet publication].Full text

25. Shittu OB, Shokunbi WA. Circumcision in haemophiliacs: the Nigerian experience. Haemophilia. 2001 Sep;7(5):534-6. Abstract

26. Kulkarni R, Lusher JM. Intracranial and extracranial hemorrhages in newborns with hemophilia: a review of the literature. J Pediatr Hematol Oncol. 1999 Jul-Aug;21(4):289-95. Abstract

27. Tarantino MD, Gupta SL, Brusky RM. The incidence and outcome of intracranial haemorrhage in newborns with haemophilia: analysis of the Nationwide Inpatient Sample database. Haemophilia. 2007 Jul;13(4):380-2. Abstract

28. Ljung RC. Intracranial haemorrhage in haemophilia A and B. Br J Haematol. 2008 Feb;140(4):378-84. Abstract

29. Nagel K, Pai MK, Paes BA, et al. Diagnosis and treatment of intracranial hemorrhage in children with hemophilia. Blood Coagul Fibrinolysis. 2013 Jan;24(1):23-7. Abstract

30. Konkle BA, Huston H, Fletcher SN, et al. Hemophilia A. In: Adam MP, Mirzaa GM, Pagon RA, et al, eds. GeneReviews [Internet]. Seattle (WA): University of Washington, Seattle; 2000.Full text  Abstract

31. Konkle BA, Huston H, Fletcher SN, et al. Hemophilia B. In: Adam MP, Mirzaa GM, Pagon RA, et al, eds. GeneReviews [Internet]. Seattle (WA): University of Washington, Seattle; 2000.Full text  Abstract

32. Soucie JM, Monahan PE, Kulkarni R, et al. The frequency of joint hemorrhages and procedures in nonsevere hemophilia A vs B. Blood Adv. 2018 Aug 28;2(16):2136-44.Full text  Abstract

33. Arnold WD, Hilgartner MW. Hemophilic arthropathy: current concepts of pathogenesis and management. J Bone Joint Surg Am. 1977 Apr;59(3):287-305. Abstract

34. National Hemophilia Foundation Medical and Scientific Advisory Council (MASAC). MASAC Document 273 - Recommendations on genotyping for persons with hemophilia. Jul 2022 [internet publication].Full text

35. American College of Medical Genetics and Genomics. Five things physicians and patients should question. Choosing Wisely, an initiative of the ABIM Foundation. 2021 [internet publication].Full text

36. Warrier I. Antibodies to factor IX. Haematologica. 2000 Oct;85(10 Suppl):31-3. Abstract

37. Warrier I, Ewenstein BM, Koerper MA, et al. Factor IX inhibitors and anaphylaxis in hemophilia B. J Pediatr Hematol Oncol. 1997 Jan-Feb;19(1):23-7. Abstract

38. Srivastava A, Santagostino E, Dougall A, et al. WFH guidelines for the management of hemophilia, 3rd edition. Haemophilia. 2020 Aug;26 Suppl 6:1-158.Full text  Abstract

39. Astermark J. Basic aspects of inhibitors to factors VIII and IX and the influence of non-genetic risk factors. Haemophilia. 2006 Dec;12 Suppl 6:8-13. Abstract

40. Lee CA, Lillicrap D, Astermark J. Inhibitor development in hemophiliacs: the roles of genetic versus environmental factors. Semin Thromb Hemost. 2006 Jun;32(2 suppl):10-4. Abstract

41. Scharrer I, Bray GL, Neutzling O. Incidence of inhibitors in haemophilia A patients: a review of recent studies of recombinant and plasma-derived factor VIII concentrates. Haemophilia. 1999 May;5(3):145-54. Abstract

42. Hay CR, Ludlam CA, Colvin BT, et al. Factor VIII inhibitors in mild and moderate-severity haemophilia A. Thromb Haemost. 1998 Apr;79(4):762-6. Abstract

43. National Heart, Lung, and Blood Institute. The diagnosis, evaluation, and management of von Willebrand Disease. Dec 2007 [internet publication].Full text

44. Favaloro EJ. The utility of PFA-100 in the identification of von Willebrand disease: a concise review. Semin Thromb Hemost. 2006 Jul;32(5):537-45. Abstract

45. Cariappa R, Wilhite TR, Parvin CA, et al. Comparison of PFA-100 and bleeding time testing in pediatric patients with suspected hemorrhagic problems. J Pediatr Hematol Oncol. 2003 Jun;25(6):474-9. Abstract

46. Nurden AT. Qualitative disorders of platelets and megakaryocytes. J Thromb Haemost. 2005 Aug;3(8):1773-82.Full text  Abstract

47. Astermark J, Donfield SM, Gomperts ED, et al. The polygenic nature of inhibitors in hemophilia A: results from the Hemophilia Inhibitor Genetics Study (HIGS) combined cohort. Blood. 2013 Feb 21;121(8):1446-54.Full text  Abstract

48. Lochan A, Macaulay S, Chen WC, et al. Genetic factors influencing inhibitor development in a cohort of South African haemophilia A patients. Haemophilia. 2014 Sep;20(5):687-92. Abstract

49. Platokouki H, Fischer K, Gouw SC, et al. Vaccinations are not associated with inhibitor development in boys with severe haemophilia A. Haemophilia. 2018 Mar;24(2):283-90. Abstract

50. National Hemophilia Foundation Medical and Scientific Advisory Council (MASAC). MASAC Document 278 - MASAC recommendations on administration of vaccines to individuals with bleeding disorders. May 2023 [internet publication].Full text

51. 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 Jun;8(6):1256-65.Full text  Abstract

52. Gouw SC, van der Bom JG, Ljung R, et al; PedNet and RODIN Study Group. Factor VIII products and inhibitor development in severe hemophilia A. N Engl J Med. 2013 Jan 17;368(3):231-9.Full text  Abstract

53. Peyvandi F, Mannucci PM, Garagiola I, et al. A randomized trial of factor VIII and neutralizing antibodies in hemophilia A. N Engl J Med. 2016 May 26;374(21):2054-64.Full text  Abstract

54. National Hemophilia Foundation Medical and Scientific Advisory Council (MASAC). MASAC recommendation on SIPPET (Survey of Inhibitors in Plasma-Product-Exposed Toddlers): results and recommendations for treatment products for previously untreated patients with hemophilia A. Jun 2016 [internet publication].Full text

55. Verbruggen B. Diagnosis and quantification of factor VIII inhibitors. Haemophilia. 2010 May;16(102):20-4.Full text  Abstract

56. Miller CH, Boylan B, Shapiro AD, et al. Limit of detection and threshold for positivity of the Centers for Disease Control and Prevention assay for factor VIII inhibitors. J Thromb Haemost. 2017 Oct;15(10):1971-6.Full text  Abstract

57. Miller CH, Rice AS, Boylan B, et al. Comparison of clot-based, chromogenic and fluorescence assays for measurement of factor VIII inhibitors in the US Hemophilia Inhibitor Research Study. J Thromb Haemost. 2013 Oct;11(7):1300-9.Full text  Abstract

58. National Hemophilia Foundation Medical and Scientific Advisory Council (MASAC). MASAC document 265 - MASAC guidelines for pregnancy and perinatal management of women with inherited bleeding disorders and carriers of hemophilia A or B. Mar 2021 [internet publication].Full text

59. United Kingdom Haemophilia Centres Doctor's Organisation. Clinical genetics services for haemophilia. 2018 [internet publication].Full text

60. Park JS, Ryu KN. Hemophilic pseudotumor involving the musculoskeletal system: spectrum of radiologic findings. AJR Am J Roentgenol. 2004 Jul;183(1):55-61.Full text  Abstract

61. National Hemophilia Foundation Medical and Scientific Advisory Council (MASAC). MASAC document 268 - recommendation on the use and management of emicizumab-kxwh (Hemlibra®) for hemophilia A with and without inhibitors. Apr 2022 [internet publication].Full text

62. James PD, Connell NT, Ameer B, et al. ASH ISTH NHF WFH 2021 guidelines on the diagnosis of von Willebrand disease. Blood Adv. 2021 Jan 12;5(1):280-300.Full text  Abstract

63. Collins PW, Chalmers E, Hart DP, et al. Diagnosis and treatment of factor VIII and IX inhibitors in congenital haemophilia: 4th edition. Br J Haematol. 2013 Jan;160(2):153-70.Full text  Abstract

64. Brown DL. Congenital bleeding disorders. Curr Probl Pediatr Adolesc Health Care. 2005 Feb;35(2):38-62. Abstract

65. Tiede A, Collins P, Knoebl P, et al. International recommendations on the diagnosis and treatment of acquired hemophilia A. Haematologica. 2020 Jul;105(7):1791-801.Full text  Abstract

66. Warrier I. Management of haemophilia B patients with inhibitors and anaphylaxis. Haemophilia. 1998 Jul;4(4):574-6.Full text  Abstract

67. Hay CR, Baglin TP, Collins PW, et al. The diagnosis and management of factor VIII and IX inhibitors: a guideline from the UK Haemophilia Centre Doctors' Organization (UKHCDO). Br J Haematol. 2000 Oct;111(1):78-90.Full text  Abstract

68. National Hemophilia Foundation Medical and Scientific Advisory Council (MASAC). MASAC Document 274 - Recommendation on administration of inhibitor bypassing agents in the home for patients with hemophilia and inhibitors. Sep 2022 [internet publication].Full text

69. National Hemophilia Foundation Medical and Scientific Advisory Council (MASAC). MASAC document 267 - MASAC recommendation concerning prophylaxis for hemophilia A and B with and without inhibitors. Apr 2022 [internet publication].Full text

70. National Hemophilia Foundation Medical and Scientific Advisory Council (MASAC). MASAC Document 280 - MASAC recommendations concerning products licensed for the treatment of hemophilia and selected disorders of the coagulation system. Aug 2023 [internet publication].Full text

71. Negrier C, Goudemand J, Sultan Y, et al. Multicenter retrospective study on the utilization of FEIBA in France in patients with factor VIII and factor IX inhibitors. Thromb Haemost. 1997 Jun;77(6):1113-9. Abstract

72. Key NS, Aledort LM, Beardsley D, et al. Home treatment of mild to moderate bleeding episodes using recombinant factor VIIa (Novoseven) in haemophiliacs with inhibitors. Thromb Haemost. 1998 Dec;80(6):912-8. Abstract

73. Astermark J, Donfield SM, DiMichele DM, et al. A randomized comparison of bypassing agents in hemophilia complicated by an inhibitor: the FEIBA NovoSeven Comparative (FENOC) Study. Blood. 2007 Jan 15;109(2):546-51.Full text  Abstract

74. Ehrlich HJ, Henzl MJ, Gomperts ED. Safety of factor VIII inhibitor bypass activity (FEIBA): 10-year compilation of thrombotic adverse events. Haemophilia. 2002 Mar;8(2):83-90. Abstract

75. National Hemophilia Foundation Medical and Scientific Advisory Council (MASAC). Guidelines for emergency department management of individuals with hemophilia and other bleeding disorders. Dec 2019 [internet publication].Full text

76. Tena-Sanabria ME, Rojas-Sato YF, Castañeda-Resendiz JC, et al. Treatment with radiosynoviorthesis in hemophilic patients with and without inhibitor. BMC Pediatr. 2020 Apr 20;20(1):173.Full text  Abstract

77. van Vulpen LFD, Thomas S, Keny SA, et al. Synovitis and synovectomy in haemophilia. Haemophilia. 2021 Feb;27 Suppl 3(suppl 3):96-102.Full text  Abstract

78. National Hemophilia Foundation Medical and Scientific Advisory Council (MASAC). MASAC Document 275 - MASAC Recommendations regarding physical therapy management for the care of persons with bleeding disorders. May 2023 [internet publication].Full text

79. Matino D, Makris M, Dwan K, et al. Recombinant factor VIIa concentrate versus plasma-derived concentrates for treating acute bleeding episodes in people with haemophilia and inhibitors. Cochrane Database Syst Rev. 2015 Dec 16;(12):CD004449.Full text  Abstract

80. Kruse-Jarres R, St-Louis J, Greist A, et al. Efficacy and safety of OBI-1, an antihaemophilic factor VIII (recombinant), porcine sequence, in subjects with acquired haemophilia A. Haemophilia. 2015 Mar;21(2):162-70. Abstract

81. D'Arena G, Grandone E, Di Minno MN, et al. Acquired hemophilia a successfully treated with rituximab. Mediterr J Hematol Infect Dis. 2015 Mar 1;7(1):e2015024.Full text  Abstract

82. Collins P, Baudo F, Knoebl P, et al. Immunosuppression for acquired hemophilia A: results from the European Acquired Haemophilia Registry (EACH2). Blood. 2012 Jul 5;120(1):47-55.Full text  Abstract

83. Rayment R, Chalmers E, Forsyth K, et al. Guidelines on the use of prophylactic factor replacement for children and adults with haemophilia A and B. Br J Haematol. 2020 Sep;190(5):684-95.Full text  Abstract

84. Berntorp E, Astermark J, Bjorkman S, et al. Consensus perspectives on prophylactic therapy for haemophilia: summary statement. Haemophilia. 2003 May;9 Suppl 1:1-4. Abstract

85. Gringeri A, Lundin B, von Mackensen S, et al. A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study). J Thromb Haemost. 2011 Apr;9(4):700-10.Full text  Abstract

86. Nilsson IM, Berntorp E, Löfqvist T, et al. Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B. J Intern Med. 1992 Jul;232(1):25-32. Abstract

87. Manco-Johnson MJ, Abshire TC, Shapiro AD, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med. 2007 Aug 9;357(6):535-44.Full text  Abstract

88. Poon M-C, Lee A. Individualized prophylaxis for optimizing hemophilia care: can we apply this to both developed and developing nations? Thrombosis J. 2016;14(suppl 1):32.Full text

89. Oldenburg J. Optimal treatment strategies for hemophilia: achievements and limitations of current prophylactic regimens. Blood. 2015 Mar 26;125(13):2038-44.Full text  Abstract

90. Jiménez-Yuste V, Auerswald G, Benson G, et al. Achieving and maintaining an optimal trough level for prophylaxis in haemophilia: the past, the present and the future. Blood Transfus. 2014 Jul;12(3):314-9.Full text  Abstract

91. Mancuso ME, Santagostino E. Outcome of clinical trials with new extended half-life FVIII/IX concentrates. J Clin Med. 2017 Mar 28;6(4).Full text  Abstract

92. Mahlangu J, Oldenburg J, Paz-Priel I, et al. Emicizumab prophylaxis in patients who have hemophilia A without inhibitors. N Engl J Med. 2018 Aug 30;379(9):811-22.Full text  Abstract

93. Oldenburg J, Mahlangu JN, Kim B, et al. Emicizumab prophylaxis in hemophilia A with inhibitors. N Engl J Med. 2017 Aug 31;377(9):809-18.Full text  Abstract

94. Oldenburg J, Mahlangu JN, Bujan W, et al. The effect of emicizumab prophylaxis on health-related outcomes in persons with haemophilia A with inhibitors: HAVEN 1 Study. Haemophilia. 2019 Jan;25(1):33-44. Abstract

95. Nogami K, Shima M. New therapies using nonfactor products for patients with hemophilia and inhibitors. Blood. 2019 Jan 31;133(5):399-406. Abstract

96. DiMichele DM, Hoots WK, Pipe SW, et al. International workshop on immune tolerance induction: consensus recommendations. Haemophilia. 2007 Jul;13 Suppl 1:1-22.Full text  Abstract

97. Hay CR, DiMichele DM; International Immune Tolerance Study. The principal results of the International Immune Tolerance Study: a randomized dose comparison. Blood. 2012 Feb 9;119(6):1335-44.Full text  Abstract

98. Athale AH, Marcucci M, Iorio A. Immune tolerance induction for treating inhibitors in people with congenital haemophilia A or B. Cochrane Database Syst Rev. 2014 Apr 24;(4):CD010561.Full text  Abstract

99. Lim MY, Nielsen B, Lee K, et al. Rituximab as first-line treatment for the management of adult patients with non-severe hemophilia A and inhibitors. J Thromb Haemost. 2014 Jun;12(6):897-901.Full text  Abstract

100. Chai-Adisaksopha C, Nevitt SJ, Simpson ML, et al. Bypassing agent prophylaxis in people with hemophilia A or B with inhibitors. Cochrane Database Syst Rev. 2017 Sep 25;(9):CD011441.Full text  Abstract

101. US Food and Drug Administration. FDA drug safety communication: FDA restricts use of prescription codeine pain and cough medicines and tramadol pain medicines in children; recommends against use in breastfeeding women. Apr 2017 [internet publication].Full text

102. Medicines and Healthcare products Regulatory Agency. Codeine: restricted use as analgesic in children and adolescents after European safety review. Dec 2014 [internet publication].Full text

103. European Medicines Agency. Restrictions on use of codeine for pain relief in children - CMDh endorses PRAC recommendation. Jun 2013 [internet publication].Full text

104. ClinicalTrials.gov. HOPE-B: trial of AMT-061 in severe or moderately severe hemophilia B patients. Mar 2022 [internet publication].Full text

105. Pipe SW, Leebeek FWG, Recht M, et al. Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B. N Engl J Med. 2023 Feb 23;388(8):706-18.Full text  Abstract

106. Nathwani AC, Reiss UM, Tuddenham EG, et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med. 2014 Nov 20;371(21):1994-2004.Full text  Abstract

107. George LA, Sullivan SK, Giermasz A, et al. Hemophilia B gene therapy with a high-specific-activity factor IX variant. N Engl J Med. 2017 Dec 7;377(23):2215-27.Full text  Abstract

108. Samelson-Jones BJ, Sullivan SK, et al. Follow-up of more than 5 years in a cohort of patients with hemophilia B treated with fidanacogene elaparvovec adeno-associated virus gene therapy. Blood. 2021 Nov 23;138:3975.Full text

109. ClinicalTrials.gov. A study to evaluate the efficacy and safety of factor IX gene therapy with PF-06838435 in adult males with moderately severe to severe hemophilia B (BENEGENE-2). ClinicalTrials.gov Identifier: NCT03861273. Aug 2022 [internet publication].Full text

110. Ward NJ, Buckley SM, Waddington SN, et al. Codon optimization of human factor VIII cDNAs leads to high-level expression. Blood. 2011 Jan 20;117(3):798-807.Full text  Abstract

111. Bunting S, Zhang L, Xie L, et al. Gene therapy with BMN 270 results in therapeutic levels of FVIII in mice and primates and normalization of bleeding in hemophilic mice. Mol Ther. 2018 Feb 7;26(2):496-509.Full text  Abstract

112. Rangarajan S, Walsh L, Lester W, et al. AAV5-factor VIII gene transfer in severe hemophilia A. N Engl J Med. 2017 Dec 28;377(26):2519-30.Full text  Abstract

113. Pasi KJ, Rangarajan S, Mitchell N, et al. Multiyear follow-up of AAV5-hFVIII-SQ gene therapy for hemophilia A. N Engl J Med. 2020 Jan 2;382(1):29-40.Full text  Abstract

114. Pasi K, Rangarajan S, Robinson TM, et al. Hemostatic response is maintained for up to 5 years following treatment with valoctocogene roxaparvovec, an AAV5-hFVIII-SQ gene therapy for severe hemophilia A [abstract]. Res Pract Thromb Haemost. 2021; 5 (2 suppl):OC 67.1.Full text

115. Arruda VR. Why is AAV FVIII gene therapy not approved by the US Food and Drug Administration yet? Blood Adv. 2021 Oct 26;5(20):4313.Full text  Abstract

116. Ozelo MC, Mahlangu J, Pasi KJ, et al. Valoctocogene roxaparvovec gene therapy for hemophilia A. N Engl J Med. 2022 Mar 17;386(11):1013-25. Abstract

117. ClinicalTrials.gov. Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301) (BMN 270-301). ClinicalTrials.gov Identifier: NCT03370913. Dec 2022 [internet publication].Full text

118. George LA, Monahan PE, Eyster ME, et al. Multiyear factor VIII expression after AAV gene transfer for hemophilia A. N Engl J Med. 2021 Nov 18;385(21):1961-73.Full text  Abstract

119. ClinicalTrials.gov. Study to evaluate the efficacy and safety of PF-07055480/giroctocogene fitelparvovec gene therapy in moderately severe to severe hemophilia A adults (AFFINE). Apr 2020 [internet publication].Full text

120. Négrier C. Entering new areas in known fields: recombinant fusion protein linking recombinant factor VIIa with recombinant albumin (rVIIa-FP): advancing the journey. Thromb Res. 2016 May;141 Suppl 3:S9-12.Full text  Abstract

121. Bar-Ilan A, Livnat T, Hoffmann M, et al. In vitro characterization of MOD-5014, a novel long-acting carboxy-terminal peptide (CTP)-modified activated FVII. Haemophilia. 2018 May;24(3):477-86.Full text  Abstract

122. Konkle BA, Shapiro AD, Quon DV, et al. BIVV001 fusion protein as factor VIII replacement therapy for hemophilia A. N Engl J Med. 2020 Sep 10;383(11):1018-27.Full text  Abstract

123. Nolan B, Mahlangu J, Pabinger I, et al. Recombinant factor VIII Fc fusion protein for the treatment of severe haemophilia A: final results from the ASPIRE extension study. Haemophilia. 2020 May;26(3):494-502.Full text  Abstract

124. Nolan B, Klukowska A, Shapiro A, et al. Final results of the PUPs B-LONG study: evaluating safety and efficacy of rFIXFc in previously untreated patients with hemophilia B. Blood Adv. 2021 Jul 13;5(13):2732-9.Full text  Abstract

125. Young G, Mahlangu J, Kulkarni R, et al. Recombinant factor VIII Fc fusion protein for the prevention and treatment of bleeding in children with severe hemophilia A. J Thromb Haemost. 2015 Jun;13(6):967-77.Full text  Abstract

126. Collins PW, Young G, Knobe K, et al. Recombinant long-acting glycoPEGylated factor IX in hemophilia B: a multinational randomized phase 3 trial. Blood. 2014 Dec 18;124(26):3880-6.Full text  Abstract

127. Sehgal A, Barros S, Ivanciu L, et al. An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia. Nat Med. 2015 May;21(5):492-7. Abstract

128. Srivastava A, Rangarajan S, Kavakli K, et al. Fitusiran, an investigational siRNA therapeutic targeting antithrombin for the treatment of hemophilia: first results from a phase 3 study to evaluate efficacy and safety in people with hemophilia a or B without inhibitors (ATLAS-A/B). Blood. 2021 Dec 4; 138 (suppl 2): LBA-3.Full text

129. Chowdary P, Lethagen S, Friedrich U, et al. Safety and pharmacokinetics of anti-TFPI antibody (concizumab) in healthy volunteers and patients with hemophilia: a randomized first human dose trial. J Thromb Haemost. 2015 May;13(5):743-54.Full text  Abstract

130. ClinicalTrials.gov. A trial evaluating the efficacy and safety of prophylactic administration of concizumab in haemophilia A and B patients with inhibitors (explorer™4). Oct 2021 [internet publication].Full text

131. ClinicalTrials.gov. A trial evaluating efficacy and safety of prophylactic administration of concizumab in patients with severe haemophilia A without inhibitors (explorer™5). Nov 2021 [internet publication].Full text

132. ClinicalTrials.gov. Research study to look at how well the drug concizumab works in your body if you have haemophilia without inhibitors (explorer8). Jun 2022 [internet publication].Full text

133. ClinicalTrials.gov. Research study to look at how well the drug concizumab works in your body if you have haemophilia with inhibitors (explorer7). Mar 2022 [internet publication].Full text

134. Shapiro AD, Angchaisuksiri P, Astermark J, et al. Subcutaneous concizumab prophylaxis in hemophilia A and hemophilia A/B with inhibitors: phase 2 trial results. Blood. 2019 Nov 28;134(22):1973-82.Full text  Abstract

135. Shapiro AD. Concizumab: a novel anti-TFPI therapeutic for hemophilia. Blood Adv. 2021 Jan 12;5(1):279.Full text  Abstract

136. Cardinal M, Kantaridis C, Zhu T, et al. A first-in-human study of the safety, tolerability, pharmacokinetics and pharmacodynamics of PF-06741086, an anti-tissue factor pathway inhibitor mAb, in healthy volunteers. J Thromb Haemost. 2018 Sep;16(9):1722-31.Full text  Abstract

137. ClinicalTrials.gov. Study of the efficacy and safety PF-06741086 in adult and teenage participants with severe hemophilia A or moderately severe to severe hemophilia B. Jun 2022 [internet publication].Full text

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