575
Views
6
CrossRef citations to date
0
Altmetric
Hematology

Long-term efficacy and safety of prophylaxis with recombinant factor VIII in Chinese pediatric patients with hemophilia A: a multi-center, retrospective, non-interventional, phase IV (ReCARE) study

, , , , , , , , , , , , & show all
Pages 1223-1230 | Received 15 Dec 2016, Accepted 23 Feb 2017, Published online: 25 Apr 2017

References

  • Klamroth R. Treatment of haemophilia A with recombinant antihaemophilic factor VIII products. Eur Haematol Rev 2007;1:11-15
  • Fast Facts About Bleeding Disorders. National Hemophilia Foundation. Available at: https://www.hemophilia.org/About-Us/Fast-Facts [Last accessed 1 January 2016]
  • Hemophilia A. National Organization for Rare Disorders. Available at: https://rarediseases.org/rare-diseases/hemophilia-a/ [Last accessed 1 January 2016]
  • Oldenburg J. Optimal treatment strategies for hemophilia: achievements and limitations of current prophylactic regimens. Blood 2015;125:2038-44
  • MASAC Recommendation Concerning Prophylaxis. National Hemophilia Foundation. Available at: https://www.hemophilia.org/Researchers-Healthcare-Providers/Medical-and-Scientific-Advisory-Council-MASAC/MASAC-Recommendations/MASAC-Recommendation-Concerning-Prophylaxis [Last accessed 1 January 2016]
  • The World Federation of Hemophilia’s Sixth Global Forum on the Safety and Supply of Treatment Products for Bleeding Disorders. Available at: http://www1.wfh.org/publications/files/pdf-1274.pdf [Last accessed 21 December 2015]
  • Franchini M. Plasma-derived versus recombinant Factor VIII concentrates for the treatment of haemophilia A: recombinant is better. Blood Transfus 2010;8:292-6
  • Qu Y, Nie X, Yang Z, et al. The prevalence of hemophilia in mainland China: a systematic review and meta-analysis. Southeast Asian J Trop Med Public Health 2014;45:455-66
  • Aygoren-Pursun E, Scharrer I. A multicenter pharmacosurveillance study for the evaluation of the efficacy and safety of recombinant factor VIII in the treatment of patients with hemophilia A. German Kogenate Study Group. Thromb Haemost 1997;78:1352-6
  • Yoshioka A, Shima M, Fukutake K, et al. Safety and efficacy of a new recombinant FVIII formulated with sucrose (rFVIII-FS) in patients with haemophilia A: a long-term, multicentre clinical study in Japan. Haemophilia 2001;7:242-9
  • Giangrande PL; Kogenate Bayer Study Group. Safety and efficacy of Kogenate Bayer in previously untreated patients (PUPs) and minimally treated patients (MTPs). Haemophilia 2002;8(Suppl 2):19-22
  • Scharrer I; Kogenate Bayer Study Group. Experience with Kogenate Bayer in surgical procedures. Haemophilia 2002;8(Suppl 2):15-18
  • Kreuz W, Gill JC, Rothschild C, et al. Full-length sucrose-formulated recombinant factor VIII for treatment of previously untreated or minimally treated young children with severe haemophilia A: results of an international clinical investigation. Thromb Haemost 2005;93:457-67
  • Yoshioka A, Fukutake K, Takamatsu J. Clinical evaluation of recombinant factor VIII preparation (Kogenate) in previously treated patients with hemophilia A: descriptive meta-analysis of post-marketing study data. Int J Hematol 2006;84:158-65
  • Tarantino MD, Collins PW, Hay CR, et al. Clinical evaluation of an advanced category antihaemophilic factor prepared using a plasma/albumin-free method: pharmacokinetics, efficacy, and safety in previously treated patients with haemophilia A. Haemophilia 2004;10:428-37
  • Brand B, Gruppo R, Wynn TT, et al. Efficacy and safety of pegylated full-length recombinant factor VIII with extended half-life for perioperative haemostasis in haemophilia A patients. Haemophilia 2016;22:e251-8
  • Stidl R, Fuchs S, Bossard M, et al. Safety of PEGylated recombinant human full-length coagulation factor VIII (BAX 855) in the overall context of PEG and PEG conjugates. Haemophilia 2016;22:54-64
  • Shi J, Zhao Y, Wu J, et al. Safety and efficacy of a sucrose-formulated recombinant factor VIII product for the treatment of previously treated patients with haemophilia A in China. Haemophilia 2007;13:351-6
  • Xin L, Jing WU, Ping S. Recombinant factor VIII for treatment of patients with hemophilia A in China. Chin J Thromb Hemost 2007;13:57-9
  • Lei Z, Liping J, Mengsu T. Efficacy of recombinant factor VIII in 16 hemophilia patients [in Chinese]. Chin J Pract Intern Med 2008;28:943-5
  • Qing LW, Jing S, Hua SD. Efficacy and inhibitor generation of recombinant factor VIII in Chinese patients with hemophilia A [in Chinese]. Chin J Thromb Hemost 2008;14:257-60
  • Dan L, Ming X, Xian Z. A preliminary study on treatment of joint bleeding in children with severe and moderate hemophilia A: low-dose short-course recombinant human factor concentration as secondary prophylaxis. West China Med J 2011;26:1136-8
  • Wu R, Luke KH. Low dose secondary prophylaxis reduces joint bleeding in severe and moderate haemophilic children: a pilot study in China. Haemophilia 2011;17:70-4
  • Blanchette VS, Key NS, Ljung LR, et al. Definitions in hemophilia: communication from the SSC of the ISTH. J Thromb Haemost 2014;12:1935-9
  • Introductory Guide MedDRA Version 18.0. MedDra Web site. Available at: http://www.meddra.org/sites/default/files/guidance/file/intguide_18_0_english.pdf [Last accessed 1 January 2016]
  • Common Terminology Criteria for Adverse Events v4.0 (CTCAE). National Institute of Health. Available at: https://www.acrin.org/Portals/0/Administration/Regulatory/CTCAE_4.02_2009-09-15_QuickReference_5x7.pdf [Last accessed 1 January 2016]
  • Berntorp E, Boulyjenkov V, Brettler D, et al. Modern treatment of haemophilia. Bull World Health Org 1995;73:691-701
  • Berntorp E. Guidelines on treatment of haemophilia in Sweden. Haemophilia 1998;4:425-6
  • Collins P, Faradji A, Morfini M, et al. Efficacy and safety of secondary prophylactic vs. on-demand sucrose-formulated recombinant factor VIII treatment in adults with severe hemophilia A: results from a 13-month crossover study. J Thromb Haemost 2010;8:83-9
  • Lusher JM, Arkin S, Abildgaard CF, et al. Recombinant factor VIII for the treatment of previously untreated patients with hemophilia A. Safety, efficacy, and development of inhibitors. Kogenate Previously Untreated Patient Study Group. N Engl J Med 1993;328:453-9
  • Bray GL, Gomperts ED, Courter S, et al. A multicenter study of recombinant factor VIII (recombinate): safety, efficacy, and inhibitor risk in previously untreated patients with hemophilia A. The Recombinate Study Group. Blood 1994;83(9):2428-35
  • Oldenburg J, Zimmermann R, Katsarou O, et al. Controlled, cross-sectional MRI evaluation of joint status in severe haemophilia A patients treated with prophylaxis vs. on demand. Haemophilia 2015;21:171-9
  • Kavakli K, Yang R, Rusen L. Prophylaxis vs. on-demand treatment with BAY 81-8973, a full-length plasma protein-free recombinant factor VIII product: results from a randomized trial (LEOPOLD II). J Thromb Haemost 2015;13:360-9
  • Tang L, Wu R, Sun J, et al. Short-term low-dose secondary prophylaxis for severe/moderate haemophilia A children is beneficial to reduce bleed and improve daily activity, but there are obstacle in its execution: a multi-centre pilot study in China. Haemophilia 2013;19:27-34
  • Konkle BA, Stasyshyn O, Chowdary P, et al. Pegylated, fulllength, recombinant factor VIII for prophylactic and on-demand treatment of severe hemophilia A. Blood 2015;126:1078-85
  • Mahlangu J, Powell JS, Ragni MV, et al. Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A. Blood 2014;123:317-25
  • Manco-Johnson MJ, Ma AD, Klamroth R, et al. Joint bleeding patterns in patients treated prophylactically with an extended half-life, pegylated, full-length recombinant factor VIII (BAX 855). Blood 2015;126:2300
  • White GC, McMillan CW, Blatt PM, et al. Factor VIII inhibitors: a clinical overview. Am J Hematol 1982;13:335-42
  • Valentino LA, Mamonov V, Hellmann A, et al. A randomized comparison of two prophylaxis regimens and a paired comparison of on-demand and prophylaxis treatments in hemophilia A management. J Thromb Haemost 2012;10:359-67
  • Wang XF, Zhao YQ, Yang RC, et al. The prevalence of factor VIII inhibitors and genetic aspects of inhibitor development in Chinese patients with haemophilia A. Haemophilia 2010;16:632-9
  • Ewenstein BM, Gomperts ED, Pearson S, O’Banion ME. Inhibitor development in patients receiving recombinant factor VIII (Recombinate rAHF/Bioclate): a prospective pharmacovigilance study. Haemophilia 2004;10:491-8
  • Gomez K, Klamroth R, Mahlangu J, et al. Key issues in inhibitor management in patients with haemophilia. Blood Transfus 2014;12(Suppl 1):S319-S29
  • Xi M, Makris M, Marcucci M, et al. Inhibitor development in previously treated hemophilia A patients: a systematic review, meta-analysis, and meta-regression. J Thromb Haemost 2013;11:1655-62
  • Kempton CL. Inhibitors in previously treated patients: a review of the literature. Haemophilia 2010;16:61-5

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.