Long-acting recombinant factor IX Fc fusion protein (rFIXFc) for perioperative management of subjects with haemophilia B in the phase 3 B-LONG study

British Journal of Haematology

Volumn 168, Issue 1, 2016, Pages 124-134

  Powell, Jerry S ^a   Apte, Shashikant ^b   Chambost, Hervé ^c   Hermans, Cedric ^d   Jackson, Shannon ^e   Josephson, Neil C ^f   Mahlangu, Johnny N ^g   Ozelo, Margareth C ^h   Peerlinck, Kathelijne ⁱ   Pasi, John ^j   Perry, David ^k   Ragni, Margaret V ^l   Wang, Xuefeng ^m   Jiang, Haiyan ⁿ   Li, Shuanglian ⁿ   Cristiano, Lynda M ⁿ   Innes, Alison ⁿ   Nugent, Karen ⁿ   Brennan, Aoife ⁿ   Luk, Alvin ⁿ   Allen, Geoffrey ⁿ   Pierce, Glenn F ⁿ   Robinson, Brian ⁿ   more..

^a UNIVERSITY OF CALIFORNIA   (United States)

^b Sahyadri Hospital   (India)

^c AIX MARSEILLE UNIVERSITY   (France)

^d CLINIQUES UNIVERSITAIRES SAINT LUC   (Belgium)

^e ST PAUL S HOSPITAL   (Canada)

^f PUGET SOUND BLOOD CENTER   (United States)

^g UNIVERSITY OF THE WITWATERSRAND   (South Africa)

^h UNIVERSITY OF CAMPINAS   (Brazil)

ⁱ UNIVERSITY HOSPITALS LEUVEN   (Belgium)

^j QUEEN MARY UNIVERSITY OF LONDON   (United Kingdom)

^k ADDENBROOKE S HOSPITAL   (United Kingdom)

^l UNIVERSITY OF PITTSBURGH   (United States)

^m SHANGHAI SECOND MEDICAL UNIVERSITY   (China)

ⁿ BIOGEN   (United States)

more..

Author keywords

Clinical trial, phase 3; Factor ix; Haemophilia b; Pharmacokinetics; Surgery

Indexed keywords

BLOOD CLOTTING FACTOR; DALTEPARIN; ENOXAPARIN; HEPARIN; RECOMBINANT BLOOD CLOTTING FACTOR 9 FC FUSION PROTEIN; RECOMBINANT PROTEIN; TRANEXAMIC ACID; UNCLASSIFIED DRUG; BLOOD CLOTTING FACTOR 9; HYBRID PROTEIN;

ADOLESCENT; ADULT; ANAPHYLAXIS; ARTICLE; BLOOD TRANSFUSION; CHEMOPROPHYLAXIS; CLINICAL ARTICLE; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; DRUG DOSE REGIMEN; DRUG EFFICACY; DRUG SAFETY; HEMOPHILIA B; HEMOSTASIS; HUMAN; LOADING DRUG DOSE; MALE; MIDDLE AGED; OPEN STUDY; OPERATIVE BLOOD LOSS; ORTHOPEDIC SURGERY; PERIOPERATIVE PERIOD; PHASE 3 CLINICAL TRIAL; PREDICTION; PRIORITY JOURNAL; THROMBOSIS; UNSPECIFIED SIDE EFFECT; BLOOD CLOTTING TEST; CLINICAL TRIAL; COMPLICATION; DISEASE MANAGEMENT; HEMORRHAGE; TREATMENT OUTCOME; YOUNG ADULT;

ADOLESCENT; ADULT; BLOOD COAGULATION TESTS; CHEMOPREVENTION; DISEASE MANAGEMENT; FACTOR IX; HEMOPHILIA B; HEMORRHAGE; HEMOSTASIS, SURGICAL; HUMANS; MALE; MIDDLE AGED; PERIOPERATIVE CARE; RECOMBINANT FUSION PROTEINS; TREATMENT OUTCOME; YOUNG ADULT;

EID: 84916232822     PISSN: 00071048     EISSN: 13652141     Source Type: Journal    
DOI: 10.1111/bjh.13112     Document Type: Article

References (17)

1. Collins, P.W., Moss, J., Knobe, K., Groth, A., Colberg, T. & Watson, E. (2012) Population pharmacokinetic modeling for dose setting of nonacog beta pegol (N9-GP), a glycoPEGylated recombinant factor IX. Journal of Thrombosis and Haemostasis, 10, 2305-2312.
2. Diao, L., Li, S., Ludden, T., Gobburu, J., Nestorov, I. & Jiang, H. (2014) Population Pharmacokinetic Modelling of Recombinant Factor IX Fc Fusion Protein (rFIXFc) in Patients with Haemophilia B. Clinical Pharmacokinetics, 53, 467-477.
3. Hermans, C., Altisent, C., Batorova, A., Chambost, H., De Moerloose P., Karafoulidou, A., Klamroth, R., Richards, M., White, B. & Dolan, G. (2009) Replacement therapy for invasive procedures in patients with haemophilia: literature review, European survey and recommendations. Haemophilia, 15, 639-658.
4. Klitgaard, T. & Nielsen, T.G. (2008) Overview of the human pharmacokinetics of recombinant activated factor VII. British Journal of Clinical Pharmacology, 65, 3-11.
5. Longo, G., Messori, A., Morfini, M., Baudo, F., Ciavarella, N., Cinotti, S., Filimberti, E., Giustarini, G., Molinari, A.C. & Ferrini, P.R. (1989) Evaluation of factor VIII pharmacokinetics in hemophilia-A subjects undergoing surgery and description of a nomogram for dosing calculations. American Journal of Hematology, 30, 140-149.
6. National Hemophilia Foundation. (2007) MASAC Recommendation Concerning Prophylaxis (Regular Administration of Clotting Factor Concentrate to Prevent Bleeding). MASAC Document #179. https://www.hemophilia.org/sites/default/files/document/files/masac179.pdf. Accessed 8-19-2013.
7. Powell, J.S., Pasi, J., Ragni, M.V., Ozelo, M.C., Valentino, L.A., Mahlangu, J.N., Josephson, N.C., Perry, D., Manco-Johnson, M., Apte, S., Baker, R.I., Chan, G.C., Novitzky, N., Wong, R.S., Krassova, S., Allen, G., Jiang, H., Innes, A., Li, S., Cristiano, L.M., Goyal, J., Sommer, J.M., Dumont, J.A., Nugent, K., Vigliani, G., Brennan, A., Luk, A. & Pierce, G.F. (2013) Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia B. New England Journal of Medicine, 369, 2313-2323.
8. Powell, J., Shapiro, A., Ragni, M., Negrier, C., Windyga, J., Ozelo, M., Pasi, J., Baker, R., Potts, J., Li, S., Mei, B., Pierce, G.F. & Robinson, B. (2014) Switching to recombinant factor IX Fc fusion protein prophylaxis results in fewer infusions, decreased factor IX consumption and lower bleeding rates. British Journal of Haematology, doi:10.1111/bjh.13109.
9. Prasad, N., Padmanabhan, V. & Mullaji, A. (2007) Blood loss in total knee arthroplasty: an analysis of risk factors. International Orthopaedics, 31, 39-44.
10. Quon, D.V. & Logan, L. (2011) Safety and efficacy of plasma-derived coagulation factor IX concentrate (AlphaNine(R) SD) in patients with haemophilia B undergoing surgical intervention: a single institution retrospective analysis. Haemophilia, 17, e196-e201.
11. Ragni, M.V., Pasi, K.J., White, G.C., Giangrande, P.L., Courter, S.G. & Tubridy, K.L. (2002) Use of recombinant factor IX in subjects with haemophilia B undergoing surgery. Haemophilia, 8, 91-97.
12. Rudowski, W.J., Scharf, R. & Ziemski, J.M. (1987) Is major surgery in hemophiliac patients safe? World Journal of Surgery, 11, 378-386.
13. Sommer, J., Buyue, Y., Bardan, S., Peters, R.T., Jiang, H., Kamphaus, G.D., Gray, E. & Pierce, G.F. (2014) Comparative field study: impact of laboratory assay variability on the assessment of recombinant factor IX Fc fusion protein (rFIXFc) activity. Thrombosis and Haemostasis, doi:10.1160/TH13-11-0971 [Epub ahead of print].
14. Srivastava, A., Brewer, A.K., Mauser-Bunschoten, E.P., Key, N.S., Kitchen, S., Llinas, A., Ludlam, C.A., Mahlangu, J.N., Mulder, K., Poon, M.C. & Street, A. (2013) Guidelines for the management of hemophilia. Haemophilia, 19, e1-e47.
15. White, G.C., Beebe, A. & Nielsen, B. (1997) Recombinant factor IX. Thrombosis and Haemostasis, 78, 261-265.
16. Windyga, J., Rusen, L., Gruppo, R., O'Brien, A.C., Kelly, P., Roth, D.A. & Arkin, S. (2010) BDDrFVIII (Moroctocog alfa [AF-CC]) for surgical haemostasis in patients with haemophilia A: results of a pivotal study. Haemophilia, 16, 731-739.
17. Windyga, J., Lissitchkov, T., Stasyshyn, O., Mamonov, V., Ghandehari, H., Chapman, M., Fritsch, S., Wong, W.Y., Pavlova, B.G. & Abbuehl, B.E. (2014) Efficacy and safety of a recombinant factor IX (Bax326) in previously treated patients with severe or moderately severe haemophilia B undergoing surgical or other invasive procedures: a prospective, open-label, uncontrolled, multicentre, phase III study. Haemophilia, doi:10.1111/hae.12419 [Epub ahead of print].