Hemofilia

1.Schramm W. The history of haemophilia: a short review. Thromb Res. 2014;134(suppl 1):S4–S9

2.Sachdeva A, Gunasekaran V, Ramya HN, et al; ‘Consensus in Diagnosis and Management of Hemophilia’ Committee*, Indian Academy of Pediatrics. Consensus Statement of the Indian Academy of Pediatrics in Diagnosis and Management of Hemophilia. Indian Pediatr. 2018;55(7):582–590

3.Thornburg CD. How I approach: previously untreated patients with severe congenital hemophilia A. Pediatr Blood Cancer. 2018;65(12):e27466

4.Iorio A, Stonebraker JS, Chambost H, et al; Data and Demographics Committee of the World Federation of Hemophilia. Establishing the prevalence and prevalence at birth of hemophilia in males: a meta-analytic approach using national registries. Ann Intern Med. 2019;171(8):540–546

5.World Federation of Hemophilia website. Available at: https://www.wfh.org/en/home. Accessed December 15, 2020

6.Benson G, Auerswald G, Dolan G, et al. Diagnosis and care of patients with mild haemophilia: practical recommendations for clinical management. Blood Transfus. 2018;16(6):535–544

7.Konkle BA, Johnsen JM, Wheeler M, Watson C, Skinner M, Pierce GF; My Life Our Future programme. Genotypes, phenotypes and whole genome sequence: approaches from the My Life Our Future haemophilia project. Haemophilia. 2018;24(suppl 6):87–94

8.Soucie JM, Nuss R, Evatt B, et al; The Hemophilia Surveillance System Project Investigators. Mortality among males with hemophilia: relations with source of medical care. Blood. 2000;96(2):437–442

9.Soucie JMMC, Miller CH, Dupervil B, Le B, Buckner TW. Occurrence rates of haemophilia among males in the United States based on surveillance conducted in specialized haemophilia treatment centres. Haemophilia. 2020;26(3):487–493

10.Gale AJ. Continuing education course #2: current understanding of hemostasis. Toxicol Pathol. 2011;39(1):273–280

11.Peyvandi F, Garagiola I, Young G. The past and future of haemophilia: diagnosis, treatments, and its complications. Lancet. 2016;388(10040):187–197

12.Arruda VR, Doshi BS, Samelson-Jones BJ. Novel approaches to hemophilia therapy: successes and challenges. Blood. 2017;130(21):2251–2256

13.Johnsen JM, Fletcher SN, Huston H, et al. Novel approach to genetic analysis and results in 3000 hemophilia patients enrolled in the My Life, Our Future initiative. Blood Adv. 2017;1(13):824–834

14.Bertamino M, Riccardi F, Banov L, Svahn J, Molinari AC. Hemophilia care in the pediatric age. J Clin Med. 2017;6(5):E54

15.Zimmerman B, Valentino LA. Hemophilia: in review. Pediatr Rev. 2013;34(7):289–294, quiz 295

16.Hetzel D, Strauss W, Bernard K, Li Z, Urboniene A, Allen LF. A phase III, randomized, open-label trial of ferumoxytol compared with iron sucrose for the treatment of iron deficiency anemia in patients with a history of unsatisfactory oral iron therapy. Am J Hematol. 2014;89(6):646–650

17.Anderst JD, Carpenter SL, Presley R, et al. Relevance of abusive head trauma to intracranial hemorrhages and bleeding disorders. Pediatrics. 2018;141(5):e20173485

18.Querol F, Rodriguez-Merchan EC. The role of ultrasonography in the diagnosis of the musculo-skeletal problems of haemophilia. Haemophilia. 2012;18(3):e215–e226

19.Bakeer N, Shapiro AD. Merging into the mainstream: the evolution of the role of point-of-care musculoskeletal ultrasound in hemophilia. F1000Res. 2019;8:F1000

20.McCoy HE III, Kitchens CS. Small bowel hematoma in a hemophiliac as a cause of pseudoappendicitis: diagnosis by CT imaging. Am J Hematol. 1991;38(2):138–139

21.Prentice CR, Lindsay RM, Barr RD, et al. Renal complications in haemophilia and Christmas disease. Q J Med. 1971;40(157):47–61

22.Plug I, Mauser-Bunschoten EP, Bröcker-Vriends AHJT, et al. Bleeding in carriers of hemophilia. Blood. 2006;108(1):52–56

23.Kasper CK, Lin JC. Prevalence of sporadic and familial haemophilia. Haemophilia. 2007;13(1):90–92

24.Carcao MD. The diagnosis and management of congenital hemophilia. Semin Thromb Hemost. 2012;38(7):727–734

25.Srivastava A, Brewer AK, Mauser-Bunschoten EP, et al; Treatment Guidelines Working Group on Behalf of the World Federation of Hemophilia. Guidelines for the management of hemophilia. Haemophilia. 2013;19(1):e1–e47

26.Srivastava A, Santagostino E, Dougall A, et al. WFH Guidelines for the Management of Hemophilia, 3rd edition. Haemophilia. 2020;26(suppl 6):1–158

27.Kasper CK. Judith Graham Pool and the discovery of cryoprecipitate. Haemophilia. 2012;18(6):833–835

28.Schimpf K, Mannucci PM, Kreutz W, et al. Absence of hepatitis after treatment with a pasteurized factor VIII concentrate in patients with hemophilia and no previous transfusions. N Engl J Med. 1987;316(15):918–922

29.Horowitz MS, Rooks C, Horowitz B, Hilgartner MW. Virus safety of solvent/detergent-treated antihaemophilic factor concentrate. Lancet. 1988;2(8604):186–189

 30.Addiego JE Jr, Gomperts E, Liu SL, et al. Treatment of hemophilia A with a highly purified factor VIII concentrate prepared by anti-FVIIIc immunoaffinity chromatography. Thromb Haemost. 1992;67(1):19–27

31.National Hemophilia Foundation. MASAC recommendations concerning products licensed for the treatment of hemophilia and other bleeding disorders. Available at:

https://www.hemophilia.org/sites/default/files/document/files/263_treatment.pdf. Accessed xxxx

32.Bray GL, Gomperts ED, Courter S, et al; Recombinate Study Group. A multicenter study of recombinant factor VIII (recombinate): safety, efficacy, and inhibitor risk in previously untreated patients with hemophilia A. Blood. 1994;83(9):2428–2435

33.Lusher JM, Arkin S, Abildgaard CF, Schwartz RS; Kogenate Previously Untreated Patient Study Group. Recombinant factor VIII for the treatment of previously untreated patients with hemophilia A: safety, efficacy, and development of inhibitors. N Engl J Med. 1993; 328(7): 453–459

34.Harrison S, Adamson S, Bonam D, et al. The manufacturing process for recombinant factor IX. Semin Hematol. 1998;35(2)(suppl 2):4–10

35.Bush L, Webb C, Bartlett L, Burnett B. The formulation of recombinant factor IX: stability, robustness, and convenience. Semin Hematol. 1998;35(2)(suppl 2):18–21

36.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;357(6):535–544

37.Fischer K, Steen Carlsson K, Petrini P, et al. Intermediate-dose versus high-dose prophylaxis for severe hemophilia: comparing outcome and costs since the 1970s. Blood. 2013;122(7):1129–1136

38.Nilsson IM, Berntorp E, Löfqvist T, Pettersson H. Twenty-five years’ experience of prophylactic treatment in severe haemophilia A and B. J Intern Med. 1992; 232(1): 25–32

39.Teitel JM, Barnard D, Israels S, Lillicrap D, Poon MC, Sek J. Home management of haemophilia. Haemophilia. 2004;10(2):118–133

40.Armstrong EP, Malone DC, Krishnan S, Wessler MJ. Adherence to clotting factors among persons with hemophilia A or B. Hematology. 2015;20(3):148–153

41.Duncan N, Shapiro A, Ye X, Epstein J, Luo MP. Treatment patterns, health-related quality of life and adherence to prophylaxis among haemophilia A patients in the United States. Haemophilia. 2012;18(5):760–765

42.Schrijvers LH, Uitslager N, Schuurmans MJ, Fischer K. Barriers and motivators of adherence to prophylactic treatment in haemophilia: a systematic review. Haemophilia. 2013;19(3):355–361

43.Roberts JC, Lattimore S, Recht M, et al. Goal Attainment Scaling for haemophilia (GAS-Hēm): testing the feasibility of a new patient-centric outcome measure in people with haemophilia. Haemophilia. 2018;24(4):e199–e206

44.Lissitchkov T, Hampton K, von Depka M, et al. Novel, human cell line-derived recombinant factor VIII (human-cl rhFVIII; Nuwiq (®)) in adults with severe haemophilia A: efficacy and safety. Haemophilia. 2016;22(2):225–231