Introduction: Limited data are available on optimal prophylaxis regimens of factor IX (FIX) replacements for patients with haemophilia B. Aim: This multicentre, open-label study evaluated the efficacy and safety of once-weekly prophylaxis with nonacog alfa compared with on-demand treatment in adolescent and adult patients. Methods: Males aged 12-65 years with moderately severe to severe haemophilia B (FIX:C <= 2%) were eligible for enrolment. Patients received on-demand treatment for 26 weeks, followed by once-weekly prophylaxis of 100 IU kg(-1) for 52 weeks. The primary efficacy end point was the annualized bleeding rate (ABR). Secondary end points included response to on-demand treatment, the number of infusions used to treat bleeding events, and the incidence of less-than-expected therapeutic effect (LETE). FIX: C was measured on day 1 and at weeks 26 and 78. Results: Mean (+/- SD) ABR was lower during prophylaxis vs. on-demand treatment [3.6 (+/- 4.6) vs. 32.9 (+/- 17.4) events, respectively; P < 0.0001]. The majority (88.4%) of bleeding events had excellent or good responses upon the first infusion; 82.1% of events responded to the first infusion. No incident of LETE occurred. No thrombotic events or FIX inhibitors were reported. Eight of 17 FIX: C approximately 1 week after dosing were >2 IU dL(-1) (min-max of 2.13-10.39 IU dL(-1)). Conclusions: Once-weekly prophylaxis of 100 IU kg(-1) was associated with lower ABR compared with on-demand treatment in adolescents and adults with moderately severe to severe haemophilia B. Once-weekly prophylaxis was well tolerated, with a similar safety profile as that reported during the on-demand treatment period. Residual FIX: C may be supportive of effectiveness.
机构:
Karolinska Univ Hosp, Karolinska Inst, Dept Hematol, Coagulat Unit, Stockholm, Sweden
McMaster Univ, Dept Med, Hamilton, ON, CanadaKarolinska Univ Hosp, Karolinska Inst, Dept Hematol, Coagulat Unit, Stockholm, Sweden
Schulman, S.
;
Eelde, A.
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Karolinska Univ Hosp, Karolinska Inst, Dept Hematol, Coagulat Unit, Stockholm, SwedenKarolinska Univ Hosp, Karolinska Inst, Dept Hematol, Coagulat Unit, Stockholm, Sweden
Eelde, A.
;
Holmstroem, M.
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Karolinska Univ Hosp, Karolinska Inst, Dept Hematol, Coagulat Unit, Stockholm, SwedenKarolinska Univ Hosp, Karolinska Inst, Dept Hematol, Coagulat Unit, Stockholm, Sweden
Holmstroem, M.
;
Stahlberg, G.
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机构:Karolinska Univ Hosp, Karolinska Inst, Dept Hematol, Coagulat Unit, Stockholm, Sweden
Stahlberg, G.
;
Odeberg, J.
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Karolinska Univ Hosp, Dept Med, Karolinska Inst, Atherosclerosis Res Unit, Stockholm, SwedenKarolinska Univ Hosp, Karolinska Inst, Dept Hematol, Coagulat Unit, Stockholm, Sweden
Odeberg, J.
;
Blombaeck, M.
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Karolinska Univ Hosp, Dept Mol Med & Surg, Karolinska Inst, Stockholm, SwedenKarolinska Univ Hosp, Karolinska Inst, Dept Hematol, Coagulat Unit, Stockholm, Sweden
机构:
Christian Med Coll & Hosp, Dept Hematol, Vellore 632004, Tamil Nadu, IndiaChristian Med Coll & Hosp, Dept Hematol, Vellore 632004, Tamil Nadu, India
Srivastava, A.
;
Brewer, A. K.
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Royal Infirm, Dept Oral Surg, Glasgow G31 2ER, Lanark, ScotlandChristian Med Coll & Hosp, Dept Hematol, Vellore 632004, Tamil Nadu, India
Brewer, A. K.
;
Mauser-Bunschoten, E. P.
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Univ Med Ctr Utrecht, Van Creveldklin, Utrecht, Netherlands
Univ Med Ctr Utrecht, Dept Hematol, Utrecht, NetherlandsChristian Med Coll & Hosp, Dept Hematol, Vellore 632004, Tamil Nadu, India
Mauser-Bunschoten, E. P.
;
Key, N. S.
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机构:
Univ N Carolina, Dept Med, Chapel Hill, NC USAChristian Med Coll & Hosp, Dept Hematol, Vellore 632004, Tamil Nadu, India
Key, N. S.
;
Kitchen, S.
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Royal Hallamshire Hosp, Sheffield Haemophilia & Thrombosis Ctr, Sheffield S10 2JF, S Yorkshire, EnglandChristian Med Coll & Hosp, Dept Hematol, Vellore 632004, Tamil Nadu, India
Kitchen, S.
;
Llinas, A.
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Fdn Santa Fe Univ Hosp Fdn Cosme & Damian, Dept Orthopaed & Traumatol, Bogota, Colombia
Univ Los Andes, Bogota, Colombia
Univ Rosario, Bogota, ColombiaChristian Med Coll & Hosp, Dept Hematol, Vellore 632004, Tamil Nadu, India
Llinas, A.
;
Ludlam, C. A.
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Royal Infirm, Comprehens Care Haemophilia & Thrombosis Ctr, Edinburgh, Midlothian, ScotlandChristian Med Coll & Hosp, Dept Hematol, Vellore 632004, Tamil Nadu, India
Ludlam, C. A.
;
Mahlangu, J. N.
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Johannesburg Hosp, Haemophilia Comprehens Care Ctr, Johannesburg, South Africa
Natl Hlth Lab Serv, Fac Hlth Sci, Dept Mol Med & Haematol, Johannesburg, South Africa
Univ Witwatersrand, Johannesburg, South AfricaChristian Med Coll & Hosp, Dept Hematol, Vellore 632004, Tamil Nadu, India
Mahlangu, J. N.
;
Mulder, K.
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机构:
Hlth Sci Ctr, Bleeding Disorders Clin, Winnipeg, MB, CanadaChristian Med Coll & Hosp, Dept Hematol, Vellore 632004, Tamil Nadu, India
机构:
Karolinska Univ Hosp, Karolinska Inst, Dept Hematol, Coagulat Unit, Stockholm, Sweden
McMaster Univ, Dept Med, Hamilton, ON, CanadaKarolinska Univ Hosp, Karolinska Inst, Dept Hematol, Coagulat Unit, Stockholm, Sweden
Schulman, S.
;
Eelde, A.
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h-index: 0
机构:
Karolinska Univ Hosp, Karolinska Inst, Dept Hematol, Coagulat Unit, Stockholm, SwedenKarolinska Univ Hosp, Karolinska Inst, Dept Hematol, Coagulat Unit, Stockholm, Sweden
Eelde, A.
;
Holmstroem, M.
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h-index: 0
机构:
Karolinska Univ Hosp, Karolinska Inst, Dept Hematol, Coagulat Unit, Stockholm, SwedenKarolinska Univ Hosp, Karolinska Inst, Dept Hematol, Coagulat Unit, Stockholm, Sweden
Holmstroem, M.
;
Stahlberg, G.
论文数: 0引用数: 0
h-index: 0
机构:Karolinska Univ Hosp, Karolinska Inst, Dept Hematol, Coagulat Unit, Stockholm, Sweden
Stahlberg, G.
;
Odeberg, J.
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h-index: 0
机构:
Karolinska Univ Hosp, Dept Med, Karolinska Inst, Atherosclerosis Res Unit, Stockholm, SwedenKarolinska Univ Hosp, Karolinska Inst, Dept Hematol, Coagulat Unit, Stockholm, Sweden
Odeberg, J.
;
Blombaeck, M.
论文数: 0引用数: 0
h-index: 0
机构:
Karolinska Univ Hosp, Dept Mol Med & Surg, Karolinska Inst, Stockholm, SwedenKarolinska Univ Hosp, Karolinska Inst, Dept Hematol, Coagulat Unit, Stockholm, Sweden
机构:
Christian Med Coll & Hosp, Dept Hematol, Vellore 632004, Tamil Nadu, IndiaChristian Med Coll & Hosp, Dept Hematol, Vellore 632004, Tamil Nadu, India
Srivastava, A.
;
Brewer, A. K.
论文数: 0引用数: 0
h-index: 0
机构:
Royal Infirm, Dept Oral Surg, Glasgow G31 2ER, Lanark, ScotlandChristian Med Coll & Hosp, Dept Hematol, Vellore 632004, Tamil Nadu, India
Brewer, A. K.
;
Mauser-Bunschoten, E. P.
论文数: 0引用数: 0
h-index: 0
机构:
Univ Med Ctr Utrecht, Van Creveldklin, Utrecht, Netherlands
Univ Med Ctr Utrecht, Dept Hematol, Utrecht, NetherlandsChristian Med Coll & Hosp, Dept Hematol, Vellore 632004, Tamil Nadu, India
Mauser-Bunschoten, E. P.
;
Key, N. S.
论文数: 0引用数: 0
h-index: 0
机构:
Univ N Carolina, Dept Med, Chapel Hill, NC USAChristian Med Coll & Hosp, Dept Hematol, Vellore 632004, Tamil Nadu, India
Key, N. S.
;
Kitchen, S.
论文数: 0引用数: 0
h-index: 0
机构:
Royal Hallamshire Hosp, Sheffield Haemophilia & Thrombosis Ctr, Sheffield S10 2JF, S Yorkshire, EnglandChristian Med Coll & Hosp, Dept Hematol, Vellore 632004, Tamil Nadu, India
Kitchen, S.
;
Llinas, A.
论文数: 0引用数: 0
h-index: 0
机构:
Fdn Santa Fe Univ Hosp Fdn Cosme & Damian, Dept Orthopaed & Traumatol, Bogota, Colombia
Univ Los Andes, Bogota, Colombia
Univ Rosario, Bogota, ColombiaChristian Med Coll & Hosp, Dept Hematol, Vellore 632004, Tamil Nadu, India
Llinas, A.
;
Ludlam, C. A.
论文数: 0引用数: 0
h-index: 0
机构:
Royal Infirm, Comprehens Care Haemophilia & Thrombosis Ctr, Edinburgh, Midlothian, ScotlandChristian Med Coll & Hosp, Dept Hematol, Vellore 632004, Tamil Nadu, India
Ludlam, C. A.
;
Mahlangu, J. N.
论文数: 0引用数: 0
h-index: 0
机构:
Johannesburg Hosp, Haemophilia Comprehens Care Ctr, Johannesburg, South Africa
Natl Hlth Lab Serv, Fac Hlth Sci, Dept Mol Med & Haematol, Johannesburg, South Africa
Univ Witwatersrand, Johannesburg, South AfricaChristian Med Coll & Hosp, Dept Hematol, Vellore 632004, Tamil Nadu, India
Mahlangu, J. N.
;
Mulder, K.
论文数: 0引用数: 0
h-index: 0
机构:
Hlth Sci Ctr, Bleeding Disorders Clin, Winnipeg, MB, CanadaChristian Med Coll & Hosp, Dept Hematol, Vellore 632004, Tamil Nadu, India