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An ultrapure plasma-derived monoclonal antibody-purified factor IX concentrate (Nonafact®), results of phase III and IV clinical studies

2011; Wiley; Volume: 17; Issue: 3 Linguagem: Inglês

10.1111/j.1365-2516.2010.02453.x

1365-2516

Eveline P. Mauser‐Bunschoten, Ilona Kleine Budde, S Łopaciuk, Mmw Koopman, F.J.M. van der Meer, I.R.O. Nováková, Paula F. Ypma, P.W.G. van der Linden, Jerzy Windyga, P. F. W. Strengers,

Blood Coagulation and Thrombosis Mechanisms

HaemophiliaVolume 17, Issue 3 p. 439-445 ORIGINAL ARTICLE Treatment An ultrapure plasma-derived monoclonal antibody-purified factor IX concentrate (Nonafact®), results of phase III and IV clinical studies E. P. MAUSER-BUNSCHOTEN, E. P. MAUSER-BUNSCHOTEN Van Creveldkliniek and Hematology, University Medical Centre Utrecht, Utrecht, The NetherlandsSearch for more papers by this authorI. KLEINE BUDDE, I. KLEINE BUDDE Sanquin Blood Supply Foundation, Amsterdam, The NetherlandsSearch for more papers by this authorS. LOPACIUK, S. LOPACIUK Institute of Hematology and Blood Transfusion, Warsaw, PolandSearch for more papers by this authorM. M. W. KOOPMAN, M. M. W. KOOPMAN Academisch Medisch Centrum, Amsterdam, The Netherlands Current address: Sanquin Blood Supply Foundation, Amsterdam, The Netherlands.Search for more papers by this authorF. J. M. Van Der MEER, F. J. M. Van Der MEER Leiden University Medical Centre, Leiden, The NetherlandsSearch for more papers by this authorI. R. O. NOVÀKOVÀ, I. R. O. NOVÀKOVÀ University Medical Centre St. Radboud, Nijmegen, The NetherlandsSearch for more papers by this authorP. YPMA, P. YPMA HAGA Hospital, Leyenburg, Den Haag, The NetherlandsSearch for more papers by this authorP. W. G. Van Der LINDEN, P. W. G. Van Der LINDEN Kennemer Gasthuis, Haarlem, The NetherlandsSearch for more papers by this authorJ. WINDYGA, J. WINDYGA Institute of Hematology and Blood Transfusion, Warsaw, PolandSearch for more papers by this authorP. F. W. STRENGERS, P. F. W. STRENGERS Sanquin Blood Supply Foundation, Amsterdam, The NetherlandsSearch for more papers by this author E. P. MAUSER-BUNSCHOTEN, E. P. MAUSER-BUNSCHOTEN Van Creveldkliniek and Hematology, University Medical Centre Utrecht, Utrecht, The NetherlandsSearch for more papers by this authorI. KLEINE BUDDE, I. KLEINE BUDDE Sanquin Blood Supply Foundation, Amsterdam, The NetherlandsSearch for more papers by this authorS. LOPACIUK, S. LOPACIUK Institute of Hematology and Blood Transfusion, Warsaw, PolandSearch for more papers by this authorM. M. W. KOOPMAN, M. M. W. KOOPMAN Academisch Medisch Centrum, Amsterdam, The Netherlands Current address: Sanquin Blood Supply Foundation, Amsterdam, The Netherlands.Search for more papers by this authorF. J. M. Van Der MEER, F. J. M. Van Der MEER Leiden University Medical Centre, Leiden, The NetherlandsSearch for more papers by this authorI. R. O. NOVÀKOVÀ, I. R. O. NOVÀKOVÀ University Medical Centre St. Radboud, Nijmegen, The NetherlandsSearch for more papers by this authorP. YPMA, P. YPMA HAGA Hospital, Leyenburg, Den Haag, The NetherlandsSearch for more papers by this author … See all authors First published: 01 March 2011 https://doi.org/10.1111/j.1365-2516.2010.02453.xCitations: 10 Dr Ilona K. Budde, Medical Department, Sanquin Blood Supply Foundation, Plesmanlaan 125, 1066 CX Amsterdam, The Netherlands.Tel.: +31 20 512 3239; fax: +31 20 512 3914;e-mail: i.kleinebudde@sanquin.nl Read the full textAboutRelatedInformationPDFPDF ToolsRequest permissionExport citationAdd to favoritesTrack citation ShareShare Give accessShare full text accessClose modalShare full-text accessPlease review our Terms and Conditions of Use and check box below to share full-text version of article.I have read and accept the Wiley Online Library Terms and Conditions of UseShareable LinkUse the link below to share a full-text version of this article with your friends and colleagues. Learn more.Copy URL Abstract Summary. Nonafact®, an ultrapure, monoclonal antibody-purified factor IX concentrate (FIX) was developed to minimize risk of thrombotic complications and viral transmission. To investigate the pharmacokinetics, efficacy and safety, phase III/IV studies were performed in the Netherlands and Poland from 1996 to 2007. The mean half-life, in vivo response and recovery of Nonafact® were 18.7 (SD 2.0) h, 1.1 (SD 0.2) IU dL−1 per IU kg−1 b.w. of FIX infused and 49% (SD 10%), respectively. Eleven surgical procedures were performed in eight patients. During two surgeries, both high-risk, blood loss was observed. No postoperative bleeding occurred. The in vivo recovery of FIX was higher than expected. In the phase III follow-up study, 26 previously treated patients (PTP) were included with a median follow-up of 1130 days. From the 1617 minor bleedings, 80.5% was stopped after a single infusion. In the phase IV study thirteen patients were treated for a median study period of 737 days. In the two follow-up studies the investigators rated the effect of Nonafact® as excellent/good in 95% of major bleedings. Surgeries for which Nonafact® was given prophylactically were without bleeding problems. In total more than 10 million units of Nonafact® were used during almost 120 person-years. Only one minor adverse event was reported. No inhibitors, viral transmissions and thrombogenic events occurred. In conclusion, Nonafact® is safe and provides excellent haemostasis in haemophilia B patients treated for spontaneous bleeding or undergoing surgical procedures. Due to the excellent in vivo recovery characteristic, treatment with Nonafact® is cost saving compared to other FIX products. Citing Literature Volume17, Issue3May 2011Pages 439-445 RelatedInformation RecommendedAn open clinical study assessing the efficacy and safety of Factor IX Grifols®, a high-purity Factor IX concentrate, in patients with severe haemophilia BT. LISSITCHKOV, M. MATYSIAK, K. ZAWILSKA, L. GERCHEVA, A. ANTONOV, M. MONTAÑES, A. PAEZ, HaemophiliaEfficacy and safety of monoclonal antibody purified factor IX concentrate in haemophilia B patients undergoing surgical proceduresA. D. Shapiro, G. C. White II, H. C. Kim, J. M. Lusher, G. E. Bergman, HaemophiliaPharmacokinetic study of a high-purity factor IX concentrate (Factor IX Grifols®) with a 6-month follow up in previously treated patients with severe haemophilia BJ. A. AZNAR, N. CABRERA, M. MATYSIAK, K. ZAWILSKA, L. GERCHEVA, A. ANTONOV, M. MONTAÑÉS, A. M. PÁEZ, T. LISSITCHKOV, HaemophiliaClinical efficacy, safety and pharmacokinetic properties of the plasma-derived factor IX concentrate Haemonine® in previously treated patients with severe haemophilia BM. SERBAN, A. B. SKOTNICKI, M. COLOVIC, C. JINCA, A. KLUKOWSKA, P. LAGUNA, D. M. WOLF, HaemophiliaClinical use of recombinant factor VIII Fc and recombinant factor IX Fc in patients with haemophilia A and BC. Wang, G. Young, Haemophilia