The benefit of low dose prophylaxis in the treatment of hemophilia: a focus on China

References

• Srivastava A, Brewer AK, Mauser Bunschoten EP, et al. Guidelines for the management of hemophilia. Haemophilia. 2013;19(1):e1–47.
   PubMed Web of Science ®Google Scholar
• Nilsson IM, Berntorp E, Löfqvist T, et al. Twenty five years: experience of prophylactic treatment in severe hemophilia A and B. J Intern Med. 1992;232:25–32.
   PubMed Web of Science ®Google Scholar
• Ljung R. Prophylactic therapy in hemophilia. Blood Reviews. 2009;23:267–274.
   PubMed Web of Science ®Google Scholar
• Fischer K, Astermark J, Van Der Bom JG, et al. Prophylactic treatment for severe hemophilia: comparison of an intermediate-dose to a high-dose regimen. Haemophilia. 2002;8:753–760.
   PubMed Web of Science ®Google Scholar
• Fischer K, Steen Carlsson K, Petrini P, et al. Intermediate-dose versus high-dose prophylaxis for severe hemophilia: comparing outcome and costs since the 1970. Blood. 2013;122:1129–1136.
   PubMed Web of Science ®Google Scholar
• Feldman BM, Pai M, Rivard G, et al. Tailored prophylaxis in severe hemophilia A: interim results from the first five years of the Canadian Hemophilia Primary Prophylaxis Study. Thromb Hemost. 2006;4:1228–1236.
   Web of Science ®Google Scholar
• Hilliard P, Zourikian N, Blanchette V, et al. Musculoskeletal health of subjects with hemophilia A treated with tailored prophylaxis: Canadian Hemophilia Primary Prophylaxis (CHPS) study. Thromb Hemost. 2013;11:460–466.
   Web of Science ®Google Scholar
• Collins PW, Bjorkman S, Fischer K, et al. Factor VIII requirement to maintain a target plasma level in the prophylactic treatment of severe hemophilia A: influences of variance in pharmacokinetics and treatment regimens. Thromb Hemost. 2010;8:269–275.
   Web of Science ®Google Scholar
• Bjorkman S, Oh M, Spotts G, et al. Population pharmacokinetics of recombinant factor VIII: the relationship of pharmacokinetics to age and body weight. Blood. 2012;119:612–618.
   PubMed Web of Science ®Google Scholar
• 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. Thromb Hemost. 2011;10:359–367.
   Web of Science ®Google Scholar
• Aledort L, Ljung R, Mann K, et al. Factor VIII therapy for hemophilia A: current and future issues. Expert Rev Hematol. 2014;7:373–385.
   PubMed Web of Science ®Google Scholar
• Aronstam A, Arblaster PG, Rainsford SG, et al. Prophylaxis in haemophilia: a double-blind controlled trial. Br J Haematol. 1976;33:81–90.
   PubMed Web of Science ®Google Scholar
• Schimpf K, Fischer B, Rothmann P. [A controlled study of treating haemophilia A on an out-patient basis (author’s transl)] (article in German). Dtsch Med Wochenschr. 1976 Jan;30(101):141–148.
   Google Scholar
• Chuansumrit A, Isarangkura P, Hathirat P. Prophylactic treatment for hemophilia A patients: a pilot study. Southeast Asian J Trop Med Public Health. 1995 Jun;26:243–246.
   PubMedGoogle Scholar
• Verma S, Dutta T, Subramanian N, et al. A randomized study of very low dose FVIII prophylaxis in severe hemophilia A success story from a resource limited country. Haemophilia. 2016;22:342–348.
   PubMed Web of Science ®Google Scholar
• Fischer K. Low dose prophylaxis for severe hemophilia: a little goes a long way (commentary). Haemophilia. 2016;22:331–333.
   PubMed Web of Science ®Google Scholar
• Wu RH, Luke KH, Poon MC, et al. Low dose secondary prophylaxis reduces joint bleeding in severe and moderate hemophilia children: a pilot study in China. Haemophilia. 2011;17:70–74.
   PubMed Web of Science ®Google Scholar
• Tang L, Wu RH, Sun J, et al. Short-term low dose secondary prophylaxis for severe/moderate hemophilia A children is beneficial to reduce bleed and improve daily activity, but there are obstacles in its execution: a multi-centre pilot study in China. Haemophilia. 2013;19:27–34.
   PubMed Web of Science ®Google Scholar
• Li G, Wu RH, Wu XY, et al. Efficacy and safety of recombinant coagulation factor IX in moderate and severe hemophilia B children in China: a signal centre observation study. Chin J Appl Clin Pediatr. 2013;28:1128–1130.
   Google Scholar
• Wu RH, Wu XY, Zhang NN, et al. Long-term low dose secondary prophylaxis for severe and moderated hemophilia children with arthropathy in China: a single-centre observation. Chin J Hematol. 2013;3:632–634.
   Google Scholar
• Wu RH, Xinyi W, Zhang NN, et al. Modified long-term low dose secondary prophylaxis for severe and moderate hemophilia a children with arthropathy: a single-centre prospective study in China. Hemophilia. 2012 WFH 2012 World Congress, abstract. Haemophilia, 2012;18:164.
   Web of Science ®Google Scholar
• Li PJ, Wu RH. In 4 children with severe hemophilia A individualized upgrade ladder prevention treatment process and curative effect of review. Chinese J Thromb Hemost. 2016;22(4):395–398.
   PubMed Web of Science ®Google Scholar
• Liu GQ, Tang L, Wu X, et al. Analysis of individualized primary prophylaxis treatment of 19 cases of children with severe hemophilia A. Chin J Pediatr. 2016;54:923–926.
   Google Scholar
• Yao WR, Xiao J, Cheng X, et al. The efficacy of recombinant FVIII low dose prophylaxis in Chinese pediatric patients with severe hemophilia A: a retrospective analysis from the ReCARE study. Clin Appl Thromb Hemost. 2017 Oct;23(7):851–858. DOI:10.1177/1076029616679507
   PubMed Web of Science ®Google Scholar
• Fu LL, Tang L, Chen ZP, et al. Intracranial hemorrhage occurred in children with severe hemophilia under different treatment. J China Pediatr Blood Cancer. 2017;22:78–81,86.
   Google Scholar
• Lian XY, Li KX, Hua BL, et al. Low dose tertiary prophylactic therapy for severe hemophilia A adults in a single medical centre in Beijing area. Acta Acad Med Sin. 2015;37:398–401.
   Google Scholar
• Hua B, Lee A, Fan L, et al. Low dose factor VIII infusion in Chinese adult hemophilia A patients: pharmacokinetic evidence that daily infusion results in higher trough level that with every-other-day infusion with similar factor VIII consumption. Haemophilia. 2017;23:417–421.
   PubMed Web of Science ®Google Scholar
• Poon M-C, Luke K-H. Hemophilia care in China: achievements of a decade of World Federation of Hemophilia (WFH) treatment center twinning activities. Haemophilia. 2008;14:879–888.
   PubMed Web of Science ®Google Scholar
• Wu R, Wu X, Zhang N, et al. Joint disease status of severe and moderate hemophilia patients at the Beijing Children’s Hospital: early onset and rapid increasing severity of arthropathy in 90% of patients by 6 years of age. Haemophilia. 2014;20:e227–230.
   PubMed Web of Science ®Google Scholar
• He W, Wu X, Chen Z, et al. Analysis of 2-year follow-up findings in 119 cases of clinical bleeding from moderate to severe preschool hemophiliacs in China. Chin J Appl Clin Pediatr. 2014;29:1154–1157.
   Google Scholar
• Tang L, Zhang J, Wu R. Assessment of the quality of life and the family burden of diseases in moderate or severe hemophilia children with joint complications. Chinese J Thromb Hemost. 2014;20:8–12.
   Google Scholar
• Wu R, Sun J, Xiao J, et al. A prospective study of health-related quality of life of boys with severe hemophilia A in China: comparing on-demand to prophylaxis treatment. Haemophilia. 2017;23:430–43610.
   PubMed Web of Science ®Google Scholar
• Wu R, Zhang J, Sun J, et al. Validation of the Chinese version of the Canadian Hemophilia Outcomes-Kids’ Life Assessment Tool (the CHO-KLAT). Haemophilia. 2014;20:794–799.
   PubMed Web of Science ®Google Scholar
• Chen L, Sun J, Hilliard PE, et al. “Train the trainer”: an effective and successful model to accelerate training and improve physiotherapy services for persons with hemophilia in China. Haemophilia. 2014;20:441–445.
   PubMed Web of Science ®Google Scholar
• Sun J, Hilliard PE, Zourikian N, et al. Chinese Hemophilia Joint Health Score 2.1 reliability study. Haemophilia. 2014. 20(15):435–440.
   Google Scholar
• Luke KH, Poon MC, Sun J, et al. A successful 9 year journey by a China-Canada PROFESSIONAL group to transfer current hemophilia assessment tools from Canada to advance pediatric hemophilia care in China. Haemophilia. 2016;22:3–138.
   Web of Science ®Google Scholar