User menu

Accès à distance ? S'identifier sur le proxy UCLouvain

Managing haemophilia for life: 4th haemophilia global summit

Primary tabs

Astermark, Jan Dolan, Gerry Hilberg, Thomas Jiménez-Yuste, Víctor Laffan, Michael Lobet, Sébastien [UCL] Perno, Carlo Federico

The 4th Haemophilia Global Summit was held in Potsdam, Germany, in September 2013 and brought together an international faculty of haemophilia experts and delegates from multidisciplinary backgrounds. The programme was designed by an independent Scientific Steering Committee of haemophilia experts and explored global perspectives in haemophilia care, discussing practical approaches to the optimal management of haemophilia now and in the future. The topics outlined in this supplement were selected by the Scientific Steering Committee for their relevance and potential to influence haemophilia care globally. In this supplement from the meeting, Jan Astermark reviews current understanding of risk factors for the development of inhibitory antibodies and discusses whether this risk can be modulated and minimized. Factors key to the improvement of joint health in people with haemophilia are explored, with Carlo Martinoli and Víctor Jiménez-Yuste discussing the utility of ultrasound for the early detection of haemophilic arthropathy. Other aspects of care necessary for the prevention and management of joint disease in people with haemophilia are outlined by Thomas Hilberg and Sébastian Lobet, who highlight the therapeutic benefits of physiotherapy and sports therapy. Riitta Lassila and Carlo-Federico Perno describe current knowledge surrounding the risk of transmission of infectious agents via clotting factor concentrates. Finally, different types of extended half-life technology are evaluated by Mike Laffan, with a focus on the practicalities and challenges associated with these products. © 2014 John Wiley & Sons Ltd.

Document type Article de périodique (Journal article) – Synthèse de littérature
Access type Accès restreint
Publication date 2014
Language Anglais
Journal information "Haemophilia" - Vol. 20, no. Suppl. 5, p. 1-20 (2014)
Peer reviewed yes
Publisher Wiley-Blackwell Publishing Ltd. ((United Kingdom) Osney Mead, Oxford)
issn 1351-8216
e-issn 1365-2516
Publication status Publié
Affiliations UCL - (SLuc) Service de médecine physique et de réadaptation motrice
UCL - (SLuc) Service d'hématologie
UCL - SSS/IONS/COSY - Systems & cognitive Neuroscience
Links
  1. Berntorp Erik, Shapiro Amy D, Modern haemophilia care, 10.1016/s0140-6736(11)61139-2
  2. Steen-Carlsson K Personal communication
  3. WATERS B., LILLICRAP D., The molecular mechanisms of immunomodulation and tolerance induction to factor VIII, 10.1111/j.1538-7836.2009.03538.x
  4. Saint-Remy J-M. R., Lacroix-Desmazes S., Oldenburg J., Inhibitors in haemophilia: pathophysiology, 10.1111/j.1365-2516.2004.01009.x
  5. ASTERMARK J., Why do inhibitors develop? Principles of and factors influencing the risk for inhibitor development in haemophilia, 10.1111/j.1365-2516.2006.01261.x
  6. CAO O., LODUCA P. A., HERZOG R. W., Role of regulatory T cells in tolerance to coagulation factors, 10.1111/j.1538-7836.2009.03417.x
  7. Pordes A. G., Baumgartner C. K., Allacher P., Ahmad R. U., Weiller M., Schiviz A. N., Schwarz H. P., Reipert B. M., T cell-independent restimulation of FVIII-specific murine memory B cells is facilitated by dendritic cells together with toll-like receptor 7 agonist, 10.1182/blood-2011-02-336198
  8. Oldenburg J., El-Maarri O., Schwaab R., Inhibitor development in correlation to factor VIII genotypes, 10.1046/j.1351-8216.2001.00134.x
  9. GOUW S. C., VAN DER BOM J. G., VAN DEN BERG H. M., ZEWALD R. A., PLOOS VAN AMSTEL J. K., MAUSER-BUNSCHOTEN E. P., Influence of the type of F8 gene mutation on inhibitor development in a single centre cohort of severe haemophilia A patients : F8 GENOTYPE AND INHIBITOR DEVELOPMENT, 10.1111/j.1365-2516.2010.02420.x
  10. Astermark J., Berntorp E., White G. C., Kroner B. L., , The Malmo International Brother Study (MIBS): further support for genetic predisposition to inhibitor development, 10.1046/j.1365-2516.2001.00510.x
  11. Astermark, Haematologica, 90, 924 (2005)
  12. Gouw S. C., van den Berg H. M., Oldenburg J., Astermark J., de Groot P. G., Margaglione M., Thompson A. R., van Heerde W., Boekhorst J., Miller C. H., le Cessie S., van der Bom J. G., F8 gene mutation type and inhibitor development in patients with severe hemophilia A: systematic review and meta-analysis, 10.1182/blood-2011-09-379453
  13. Schwarz J., Astermark J., Menius E. D., Carrington M., Donfield S. M., Gomperts E. D., Nelson G. W., Oldenburg J., Pavlova A., Shapiro A. D., Winkler C. A., Berntorp E., , F8haplotype and inhibitor risk: results from the Hemophilia Inhibitor Genetics Study (HIGS) Combined Cohort, 10.1111/hae.12004
  14. Pandey Gouri Shankar, Yanover Chen, Miller-Jenkins Lisa M, Garfield Susan, Cole Shelley A, Curran Joanne E, Moses Eric K, Rydz Natalia, Simhadri Vijaya, Kimchi-Sarfaty Chava, Lillicrap David, Viel Kevin R, Przytycka Teresa M, Pierce Glenn F, Howard Tom E, Sauna Zuben E, Lusher Jeanne, Chitlur Meera, Ameri Afshin, Natarajan Kavita, Iyer Rathi V, Thompson Alexis A, Watts Raymond G, Kempton Christine L, Kessler Craig, Barrett John C, Martin Erica J, Key Nigel, Kruse-Jarres Rebecca, Lessinger Cindy, Pratt Kathleen P, Josephson Neil, McRedmond Kevin, Withycombe Janice, Walsh Christopher, Matthews Dana, Mahlangu Johnny, Krause Amanda, Schwyzer Rosemary, Thejpal Rajendra, Rapiti Nadine, Goga Yasmin, Coetzee Marius, Stones David, Mann Kenneth, Butenas Saulius, Almasy Laura, Blangero John, Carless Mel, Raja Rajalingam, Reed Elaine, Endogenous factor VIII synthesis from the intron 22–inverted F8 locus may modulate the immunogenicity of replacement therapy for hemophilia A, 10.1038/nm.3270
  15. WHITE G. C., KEMPTON C. L., GRIMSLEY A., NIELSEN B., ROBERTS H. R., Cellular immune responses in hemophilia: why do inhibitors develop in some, but not all hemophiliacs?, 10.1111/j.1538-7836.2005.01375.x
  16. Astermark J., Polymorphisms in the IL10 but not in the IL1beta and IL4 genes are associated with inhibitor development in patients with hemophilia A, 10.1182/blood-2005-09-3918
  17. Astermark J., Oldenburg J., Carlson J., Pavlova A., Kavakli K., Berntorp E., Lefvert A.-K., , Polymorphisms in the TNFA gene and the risk of inhibitor development in patients with hemophilia A, 10.1182/blood-2006-05-024711
  18. ASTERMARK J., WANG X., OLDENBURG J., BERNTORP E., LEFVERT A.-K., , Polymorphisms in the CTLA-4 gene and inhibitor development in patients with severe hemophilia A, 10.1111/j.1538-7836.2007.02290.x
  19. Saba R., Booth S.A., The Genetics of Susceptibility to Variant Creutzfeldt-Jakob Disease, 10.1159/000345203
  20. Mehra Narinder, Sharma Gaurav, Kaur Gurvinder, Genetic correlates influencing immunopathogenesis of HIV infection, 10.4103/0971-5916.92623
  21. PAVLOVA A., DELEV D., LACROIX-DESMAZES S., SCHWAAB R., MENDE M., FIMMERS R., ASTERMARK J., OLDENBURG J., Impact of polymorphisms of the major histocompatibility complex class II, interleukin-10, tumor necrosis factor-α and cytotoxic T-lymphocyte antigen-4 genes on inhibitor development in severe hemophilia A, 10.1111/j.1538-7836.2009.03636.x
  22. Lu Y., Ding Q., Dai J., Wang H., Wang X., Impact of polymorphisms in genes involved in autoimmune disease on inhibitor development in Chinese patients with haemophilia A : , 10.1160/th11-06-0425
  23. Pergantou H., Varela I., Moraloglou O., Economou M., Spanou K., Kapsimali Z., Constantinidou N., Platokouki H., Impact of HLA alleles and cytokine polymorphisms on inhibitors development in children with severe haemophilia A, 10.1111/hae.12168
  24. LOZIER J. N., ROSENBERG P. S., GOEDERT J. J., MENASHE I., A case-control study reveals immunoregulatory gene haplotypes that influence inhibitor risk in severe haemophilia A : IMMUNOGENETICS OF FACTOR VIII INHIBITORS, 10.1111/j.1365-2516.2010.02473.x
  25. Chaves D., Belisário A., Castro G., Santoro M., Rodrigues C., Analysis of cytokine genes polymorphism as markers for inhibitor development in haemophilia A, 10.1111/j.1744-313x.2009.00893.x
  26. Gaitonde Puneet, Peng Aaron, Straubinger Robert M., Bankert Richard B., Balu-iyer Sathy V., Downregulation of CD40 Signal and Induction of TGF-β by Phosphatidylinositol Mediates Reduction in Immunogenicity Against Recombinant Human Factor VIII, 10.1002/jps.22746
  27. Astermark J., Donfield S. M., Gomperts E. D., Schwarz J., Menius E. D., Pavlova A., Oldenburg J., Kessing B., DiMichele D. M., Shapiro A. D., Winkler C. A., Berntorp E., , The polygenic nature of inhibitors in hemophilia A: results from the Hemophilia Inhibitor Genetics Study (HIGS) Combined Cohort, 10.1182/blood-2012-06-434803
  28. Pradeu Thomas, Cooper Edwin L., The danger theory: 20 years later, 10.3389/fimmu.2012.00287
  29. IORIO A., HALIMEH S., HOLZHAUER S., GOLDENBERG N., MARCHESINI E., MARCUCCI M., YOUNG G., BIDLINGMAIER C., BRANDAO L. R., ETTINGSHAUSEN C. E., GRINGERI A., KENET G., KNÖFLER R., KREUZ W., KURNIK K., MANNER D., SANTAGOSTINO E., MANNUCCI P. M., NOWAK-GÖTTL U., Rate of inhibitor development in previously untreated hemophilia A patients treated with plasma-derived or recombinant factor VIII concentrates: a systematic review : Inhibitor rate after plasma-derived or recombinant FVIII concentrates, 10.1111/j.1538-7836.2010.03823.x
  30. Gouw Samantha C., van der Bom Johanna G., Ljung Rolf, Escuriola Carmen, Cid Ana R., Claeyssens-Donadel Ségolène, van Geet Christel, Kenet Gili, Mäkipernaa Anne, Molinari Angelo Claudio, Muntean Wolfgang, Kobelt Rainer, Rivard George, Santagostino Elena, Thomas Angela, van den Berg H. Marijke, Factor VIII Products and Inhibitor Development in Severe Hemophilia A, 10.1056/nejmoa1208024
  31. Lusher Jeanne M., Arkin Steven, Abildgaard Charles F., Schwartz Richard S., Recombinant Factor VIII for the Treatment of Previously Untreated Patients with Hemophilia A -- Safety, Efficacy, and Development of Inhibitors, 10.1056/nejm199302183280701
  32. Yoshioka A., Fukutake K., Takamatsu J., Shirahata A., Clinical Evaluation of Recombinant Factor VIII Preparation (Kogenate) in Previously Treated Patients with Hemophilia A: Descriptive Meta-Analysis of Post-Marketing Study Data, 10.1532/ijh97.06019
  33. Kreuz, Thromb Haemost, 93, 457 (2005)
  34. Coppola Antonio, Rocino Angiola, Santagostino Elena, Tagliaferri Annarita, Zanon Ezio, Morfini Massimo, Franchini Massimo, , Systematic Review of the Role of FVIII Concentrates in Inhibitor Development in Previously Untreated Patients with Severe Hemophilia A: A 2013 Update, 10.1055/s-0033-1356715
  35. ASTERMARK J., ALTISENT C., BATOROVA A., DINIZ M. J., GRINGERI A., HOLME P. A., KARAFOULIDOU A., LOPEZ-FERNÁNDEZ M. F., REIPERT B. M., ROCINO A., SCHIAVONI M., Von DEPKA M., WINDYGA J., FIJNVANDRAAT K., , Non-genetic risk factors and the development of inhibitors in haemophilia: a comprehensive review and consensus report : NON-GENETIC RISK FACTORS FOR INHIBITOR FORMATION, 10.1111/j.1365-2516.2010.02231.x
  36. Gouw S. C., van der Bom J. G., Marijke van den Berg H., , Treatment-related risk factors of inhibitor development in previously untreated patients with hemophilia A: the CANAL cohort study, 10.1182/blood-2006-11-056291
  37. Rivard G. E., Lillicrap D., Poon M. C., Demers C., Lepine M., St-Louis J., Warner M., Can activated recombinant factor VII be used to postpone the exposure of infants to factor VIII until after 2 years of age?, 10.1111/j.1365-2516.2005.01088.x
  38. KURNIK K., BIDLINGMAIER C., ENGL W., CHEHADEH H., REIPERT B., AUERSWALD G., New early prophylaxis regimen that avoids immunological danger signals can reduce FVIII inhibitor development : NEW REGIMEN CAN REDUCE INHIBITOR DEVELOPMENT, 10.1111/j.1365-2516.2009.02122.x
  39. AUERSWALD G., BIDLINGMAIER C., KURNIK K., Early prophylaxis/FVIII tolerization regimen that avoids immunological danger signals is still effective in minimizing FVIII inhibitor developments in previously untreated patients - long-term follow-up and continuing experience : LETTER TO THE EDITOR, 10.1111/j.1365-2516.2011.02659.x
  40. Auerswald G Kurnik K Blatny J et al The EPIC Study: A Clinical Trial To Assess Whether Early Low Dose Prophylaxis In The Absence Of Immunological Danger Signals Reduces Inhibitor Incidence In Previously Untreated Patients (PUPs) With Hemophilia A
  41. Gouw S. C., van den Berg H. M., Fischer K., Auerswald G., Carcao M., Chalmers E., Chambost H., Kurnik K., Liesner R., Petrini P., Platokouki H., Altisent C., Oldenburg J., Nolan B., Garrido R. P., Mancuso M. E., Rafowicz A., Williams M., Clausen N., Middelburg R. A., Ljung R., van der Bom J. G., , Intensity of factor VIII treatment and inhibitor development in children with severe hemophilia A: the RODIN study, 10.1182/blood-2012-09-457036
  42. ZUKOTYNSKI K., JARRIN J., BABYN P. S., CARCAO M., PAZMINO-CANIZARES J., STAIN A. M., DORIA A. S., Sonography for assessment of haemophilic arthropathy in children: a systematic protocol, 10.1111/j.1365-2516.2006.01414.x
  43. ACHARYA S. S., SCHLOSS R., DYKE J. P., MINTZ D. N., CHRISTOS P., DIMICHELE D. M., ADLER R. S., Power Doppler sonography in the diagnosis of hemophilic synovitis - a promising tool, 10.1111/j.1538-7836.2008.03160.x
  44. MELCHIORRE D., LINARI S., INNOCENTI M., BISCOGLIO I., TOIGO M., CERINIC M. M., MORFINI M., Ultrasound detects joint damage and bleeding in haemophilic arthropathy: a proposal of a score : SONOGRAPHIC SCORE IN HAEMOPHILIC ARTHROPATHY, 10.1111/j.1365-2516.2010.02380.x
  45. QUEROL F., RODRIGUEZ-MERCHAN E. C., The role of ultrasonography in the diagnosis of the musculo-skeletal problems of haemophilia : THE ROLE OF US IN THE DIAGNOSIS OF MUSCULO-SKELETAL PROBLEMS OF HAEMOPHILIA, 10.1111/j.1365-2516.2011.02680.x
  46. MUÇA-PERJA M., RIVA S., GROCHOWSKA B., MANGIAFICO L., MAGO D., GRINGERI A., Ultrasonography of haemophilic arthropathy : ULTRASONOGRAPHY OF HAEMOPHILIC ARTHROPATHY, 10.1111/j.1365-2516.2011.02672.x
  47. Martinoli C., Della Casa Alberighi O., di Minno G., Graziano E., Molinari A. C., Pasta G., Russo G., Santagostino E., Tagliaferri A., Tagliafico A., Morfini M., Development and definition of a simplified scanning procedure and scoring method for Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) : , 10.1160/th12-11-0874
  48. Jansen Nathalie W. D., Roosendaal Goris, Lafeber Floris P. J. G., Understanding haemophilic arthropathy: an exploration of current open issues, 10.1111/j.1365-2141.2008.07386.x
  49. VALENTINO L. A., HAKOBYAN N., RODRIGUEZ N., HOOTS W. K., Pathogenesis of haemophilic synovitis: experimental studies on blood-induced joint damage, 10.1111/j.1365-2516.2007.01534.x
  50. Zourikian, Current and future issues in hemophilia care (2011)
  51. FORSYTH A. L., RIVARD G.-É., VALENTINO L. A., ZOURIKIAN N., HOFFMAN M., MONAHAN P. E., VAN MEEGEREN M. E. R., FORRIOL F., Consequences of intra-articular bleeding in haemophilia: science to clinical practice and beyond : CONSEQUENCES OF INTRA-ARTICULAR BLEEDING IN HAEMOPHILIA, 10.1111/j.1365-2516.2012.02835.x
  52. Hakobyan N., Kazarian T., Valentino L. A., Synovitis in a murine model of human factor VIII deficiency, 10.1111/j.1365-2516.2005.01080.x
  53. Charalambides Charalambos, Beer Marilyn, Melhuish John, Williams Robert J, Cobb Andrew G, Bandaging technique after knee replacement, 10.1080/00016470510030382
  54. Gurcay Eda, Eksioglu Emel, Ezer Ustun, Cakir Banu, Cakci Aytul, A prospective series of musculoskeletal system rehabilitation of arthropathic joints in young male hemophilic patients, 10.1007/s00296-007-0474-7
  55. LOBET S., DETREMBLEUR C., LANTIN A.-C., HAENECOUR L., HERMANS C., Functional impact of custom-made foot orthoses in patients with haemophilic ankle arthropathy : FOOT ORTHOSES IN PATIENTS WITH HAEMOPHILIA, 10.1111/j.1365-2516.2011.02711.x
  56. LOBET S., PENDEVILLE E., DALZELL R., DEFALQUE A., LAMBERT C., POTHEN D., HERMANS C., The role of physiotherapy after total knee arthroplasty in patients with haemophilia, 10.1111/j.1365-2516.2008.01748.x
  57. DE KLEIJN P., BLAMEY G., ZOURIKIAN N., DALZELL R., LOBET S., Physiotherapy following elective orthopaedic procedures, 10.1111/j.1365-2516.2006.01266.x
  58. Hilberg T., Herbsleb M., Gabriel H. H. W., Jeschke D., Schramm W., Proprioception and isometric muscular strength in haemophilic subjects, 10.1046/j.1365-2516.2001.00563.x
  59. Brunner A., Stäuber F., Göhler S., Czepa D., Krüger S., Wendel M., Seuser A., Hilberg T., Quadriceps strength, inter-extremity difference (IED) and joint status in adult persons with severe haemophilia in different age stages, 10.1111/hae.12034
  60. Hilberg T., Herbsleb M., Puta C., Gabriel H. H. W., Schramm W., Physical training increases isometric muscular strength and proprioceptive performance in haemophilic subjects, 10.1046/j.1365-2516.2003.00679.x
  61. Czepa D., von Mackensen S., Hilberg T., Haemophilia & Exercise Project (HEP): The impact of 1-year sports therapy programme on physical performance in adult haemophilia patients, 10.1111/hae.12031
  62. Massion Jean, Postural control system, 10.1016/0959-4388(94)90137-6
  63. KURZ E., HERBSLEB M., ANDERS C., PUTA C., VOLLANDT R., CZEPA D., ZIEZIO R., SCHOLLE H.-C., HILBERG T., SEMG activation patterns of thigh muscles during upright standing in haemophilic patients : SEMG PATTERNS OF KNEE MUSCLES IN HAEMOPHILIACS, 10.1111/j.1365-2516.2010.02466.x
  64. Haemophilia and Exercise Project www.haemophilia-exercise.de
  65. Hilberg Thomas, Czepa Doerte, Freialdenhoven David, Boettger Michael Karl, Joint pain in people with hemophilia depends on joint status : , 10.1016/j.pain.2011.04.030
  66. Key Nigel S, Negrier Claude, Coagulation factor concentrates: past, present, and future, 10.1016/s0140-6736(07)61199-4
  67. Franchini Massimo, Mannucci Pier, Past, present and future of hemophilia: a narrative review, 10.1186/1750-1172-7-24
  68. Makris, in Textbook of Haemophilia (2010)
  69. Merry Concepta, Mcmahon Corrina, Ryan Mairin, O'shea, Mulcahy Fiona, Smith Owen P., Successful use of protease inhibitors in HIV-infected haemophilia patients, 10.1046/j.1365-2141.1998.00734.x
  70. Kernoff P. B. A., Lee C. A., Karayiannis P., Thomas H. C., High risk of non-A non-B hepatitis after a first exposure to volunteer or commercial clotting factor concentrates: effects of prophylactic immune serum globulin, 10.1111/j.1365-2141.1985.tb07444.x
  71. Brettler, Blood, 76, 254 (1990)
  72. The impact of HIV on mortality rates in the complete UK haemophilia population : , 10.1097/00002030-200402200-00020
  73. Darby Sarah C, Ewart David W, Giangrande Paul LF, Spooner Rosemary JD, Rizza Charles R, Dusheiko Geoffrey M, Lee Christine A, Ludlam Christopher A, Preston F Eric, Mortality from liver cancer and liver disease in haemophilic men and boys in UK given blood products contaminated with hepatitis C, 10.1016/s0140-6736(97)05413-5
  74. National Hemophilia Foundation HIV/AIDS www.hemophilia.org/NHFWeb/MainPgs/MainNHF.aspx?menuid=43&contentid=39
  75. Klamroth Robert, Gröner Albrecht, Simon Toby L., Pathogen inactivation and removal methods for plasma-derived clotting factor concentrates : Plasma Concentrate Pathogen Safety, 10.1111/trf.12423
  76. Di Minno G., Canaro M., Ironside J. W., Navarro D., Perno C. F., Tiede A., Gurtler L., Pathogen safety of long-term treatments for bleeding disorders: still relevant to current practice, 10.3324/haematol.2013.084145
  77. Canaro Mariana, Ironside James, Navarro David, Perno Carlo, Tiede Andreas, Guertler Lutz, Di Minno Giovanni, Pathogen Safety of Long-Term Treatments for Bleeding Disorders: (Un)Predictable Risks and Evolving Threats, 10.1055/s-0033-1354420
  78. Woolhouse M. E. J., Dye C., Preface, 10.1098/rstb.2001.0899
  79. Zappa, Blood Transfus, 7, 167 (2009)
  80. Soucie J. Michael, De Staercke Christine, Monahan Paul E., Recht Michael, Chitlur Meera B., Gruppo Ralph, Hooper W. Craig, Kessler Craig, Kulkarni Roshni, Manco-Johnson Marilyn J., Powell Jerry, Pyle Meredith, Riske Brenda, Sabio Hernan, Trimble Sean, , Evidence for the transmission of parvovirus B19 in patients with bleeding disorders treated with plasma-derived factor concentrates in the era of nucleic acid test screening : TRANSMISSION OF B19V, 10.1111/j.1537-2995.2012.03907.x
  81. PEDEN A., McCARDLE L., HEAD M. W., LOVE S., WARD H. J. T., COUSENS S. N., KEELING D. M., MILLAR C. M., HILL F. G. H., IRONSIDE J. W., Variant CJD infection in the spleen of a neurologically asymptomatic UK adult patient with haemophilia : vCJD INFECTION IN A UK HAEMOPHILIC PATIENT, 10.1111/j.1365-2516.2009.02181.x
  82. Archer P Jones N Willets J www.archercbbp.com/files/report/76_Lord%20Archer%20Report.DOC
  83. Kleinman Steven H., Glynn Simone A., Lee Tzong-Hae, Tobler Leslie, Montalvo Leilani, Todd Deborah, Kiss Joseph E., Shyamala Venkatakrishna, Busch Michael P., , Prevalence and quantitation of parvovirus B19 DNA levels in blood donors with a sensitive polymerase chain reaction screening assay, 10.1111/j.1537-2995.2007.01341.x
  84. Burnouf T., Radosevich M., Nanofiltration of plasma-derived biopharmaceutical products, 10.1046/j.1365-2516.2003.00701.x
  85. Sharp Colin P., Lail Alice, Donfield Sharyne, Gomperts Edward D., Simmonds Peter, Virologic and clinical features of primary infection with human parvovirus 4 in subjects with hemophilia: frequent transmission by virally inactivated clotting factor concentrates : TRANSMISSION OF PARV4, 10.1111/j.1537-2995.2011.03420.x
  86. Norja Päivi, Lassila Riitta, Makris Mike, Parvovirus transmission by blood products- a cause for concern?, 10.1111/bjh.12060
  87. Mannucci PM WFH 2004 www1.wfh.org/publication/files/pdf-1267.pdf
  88. Cohen B., Fortnightly Review: Parvovirus B19: an expanding spectrum of disease, 10.1136/bmj.311.7019.1549
  89. Ludlam Christopher A, Powderly William G, Bozzette Samuel, Diamond Michael, Koerper Marion A, Kulkarni Roshni, Ritchie Bruce, Siegel Jamie, Simmonds Peter, Stanley Samuel, Tapper Michael L, von Depka Mario, Clinical perspectives of emerging pathogens in bleeding disorders, 10.1016/s0140-6736(06)68036-7
  90. EMA 2011 position statement www.ema.europa.eu/docs/en_GB/document_library/Position_statement/2011/06/WC500108071.pdf
  91. Knight Richard, Stanley Samuel, Wong Michael, Dolan Gerard, Hemophilia Therapy and Blood-Borne Pathogen Risk, 10.1055/s-2006-946908
  92. Lusher Jeanne M., Scharrer Inge, Evolution of recombinant factor VIII safety: KOGENATE® and Kogenate® FS/Bayer, 10.1007/s12185-009-0435-x
  93. Franchini, Blood Transfus, 8, 292 (2010)
  94. KEELING D., TAIT C., MAKRIS M., Guideline on the selection and use of therapeutic products to treat haemophilia and other hereditary bleeding disorders, 10.1111/j.1365-2516.2008.01695.x
  95. Harrison, Semin Hematol, 35, 4 (1998)
  96. KELLEY B., JANKOWSKI M., BOOTH J., An improved manufacturing process for Xyntha/ReFacto AF : XYNTHA/REFACTO AF IMPROVED MANUFACTURING PROCESS, 10.1111/j.1365-2516.2009.02160.x
  97. Gringeri, Blood Transfus, 9, 366 (2011)
  98. World Federation of Hemophilia Annual Global Survey 2011 www1.wfh.org/publications/files/pdf-1488.pdf
  99. Zeiler, Infusionsther Transfusionsmed, 21, 362 (1994)
  100. Makris M., Calizzani G., Fischer K., Gilman E.A., Hay C.R.M., Lassila R., Lambert T., Ludlam C.A., Mannucci P.M., EUHASS: The European Haemophilia Safety Surveillance system, 10.1016/s0049-3848(10)70150-x
  101. Vlot, Blood, 87, 1809 (1996)
  102. LENTING P. J., VAN SCHOOTEN C. J. M., DENIS C. V., Clearance mechanisms of von Willebrand factor and factor VIII, 10.1111/j.1538-7836.2007.02572.x
  103. Bovenschen N., Elevated plasma factor VIII in a mouse model of low-density lipoprotein receptor-related protein deficiency, 10.1182/blood-2002-07-2081
  104. Bjorkman S., Oh M., Spotts G., Schroth P., Fritsch S., Ewenstein B. M., Casey K., Fischer K., Blanchette V. S., Collins P. W., Population pharmacokinetics of recombinant factor VIII: the relationships of pharmacokinetics to age and body weight, 10.1182/blood-2011-07-360594
  105. Vlot, Thromb Haemost, 83, 65 (2000)
  106. Mei B., Pan C., Jiang H., Tjandra H., Strauss J., Chen Y., Liu T., Zhang X., Severs J., Newgren J., Chen J., Gu J.-M., Subramanyam B., Fournel M. A., Pierce G. F., Murphy J. E., Rational design of a fully active, long-acting PEGylated factor VIII for hemophilia A treatment, 10.1182/blood-2009-11-254755
  107. Radke S Walsch R Kerschgens B et al Clinical Development Program for BAY 94-9027, a Long-Acting PEGylated B-Domain−Deleted Recombinant Factor VIII for Patients With Hemophilia A 2013
  108. Baxter Bioscience BAX 855 a PEGylated rFVIII product with prolonged half-life. Presented at: Optimal use of clotting factors and immunoglobulins 2013
  109. Stennicke H. R., Kjalke M., Karpf D. M., Balling K. W., Johansen P. B., Elm T., Ovlisen K., Moller F., Holmberg H. L., Gudme C. N., Persson E., Hilden I., Pelzer H., Rahbek-Nielsen H., Jespersgaard C., Bogsnes A., Pedersen A. A., Kristensen A. K., Peschke B., Kappers W., Rode F., Thim L., Tranholm M., Ezban M., Olsen E. H. N., Bjorn S. E., A novel B-domain O-glycoPEGylated FVIII (N8-GP) demonstrates full efficacy and prolonged effect in hemophilic mice models, 10.1182/blood-2012-01-407494
  110. Tiede A., Brand B., Fischer R., Kavakli K., Lentz S. R., Matsushita T., Rea C., Knobe K., Viuff D., Enhancing the pharmacokinetic properties of recombinant factor VIII: first-in-human trial of glycoPEGylated recombinant factor VIII in patients with hemophilia A, 10.1111/jth.12161
  111. Powell J. S., Josephson N. C., Quon D., Ragni M. V., Cheng G., Li E., Jiang H., Li L., Dumont J. A., Goyal J., Zhang X., Sommer J., McCue J., Barbetti M., Luk A., Pierce G. F., Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patients, 10.1182/blood-2011-09-382846
  112. Shapiro A. D., Ragni M. V., Valentino L. A., Key N. S., Josephson N. C., Powell J. S., Cheng G., Thompson A. R., Goyal J., Tubridy K. L., Peters R. T., Dumont J. A., Euwart D., Li L., Hallen B., Gozzi P., Bitonti A. J., Jiang H., Luk A., Pierce G. F., Recombinant factor IX-Fc fusion protein (rFIXFc) demonstrates safety and prolonged activity in a phase 1/2a study in hemophilia B patients, 10.1182/blood-2011-07-367003
  113. Ivens I. A., Baumann A., McDonald T. A., Humphries T. J., Michaels L. A., Mathew P., PEGylated therapeutic proteins for haemophilia treatment: a review for haemophilia caregivers, 10.1111/j.1365-2516.2012.02931.x
  114. Petrini P., What factors should influence the dosage and interval of prophylactic treatment in patients with severe haemophilia A and B?, 10.1046/j.1365-2516.2001.00471.x
Bibliographic reference Astermark, Jan ; Dolan, Gerry ; Hilberg, Thomas ; Jiménez-Yuste, Víctor ; Laffan, Michael ; et. al. Managing haemophilia for life: 4th haemophilia global summit. In: Haemophilia, Vol. 20, no. Suppl. 5, p. 1-20 (2014)
Permanent URL http://hdl.handle.net/2078.1/164128