Long-term safety and efficacy of turoctocog alfa in prophylaxis and treatment of bleeding episodes in severe haemophilia A: Final results from the guardian 2 extension trial

被引：17

作者：

Lentz, Steven R. ^{[1
]}

Janic, Dragana ^{[2
]}

Kavakli, Kaan ^{[3
]}

Miljic, Predrag ^{[4
]}

Oldenburg, Johannes ^{[5
]}

Ozelo, Margareth C. ^{[6
]}

Santagostino, Elena ^{[7
]}

Suzuki, Takashi ^{[8
]}

Salek, Silva Zupancic ^{[9
,10
,11
]}

Korsholm, Lars ^{[12
]}

Matytsina, Irina ^{[12
]}

Tiede, Andreas ^{[13
]}

机构：

[1] Univ Iowa, Carver Coll Med, Div Hematol Oncol & Blood & Marrow Transplantat, Internal Med, Iowa City, IA 52242 USA

[2] Univ Belgrade, Univ Childrens Hosp, Sch Med, Belgrade, Serbia

[3] Ege Univ, Childrens Hosp, Dept Pediat Hematol, Fac Med, Izmir, Turkey

[4] Univ Belgrade, Clin Ctr Serbia, Sch Med, Clin Hematol, Belgrade, Serbia

[5] Univ Clin Bonn, Inst Expt Haematol & Transfus Med, Bonn, Germany

[6] Univ Estadual Campinas, IHTC Claudio LP Correa, INCT Sangue Hemoctr UNICAMP, Sao Paulo, Brazil

[7] Fdn IRCCS Ca Granda, Maggiore Hosp Policlin, Angelo Bianchi Bonomi Hemophilia & Thrombosis Ctr, Milan, Italy

[8] Tokyo Med Univ, Tokyo, Japan

[9] Univ Hosp Ctr Zagreb, Zagreb, Croatia

[10] Med Sch Zagreb, Zagreb, Croatia

[11] Med Sch Osijek, Osijek, Croatia

[12] Novo Nordisk AS, Soborg, Denmark

[13] Hannover Med Sch, Hannover, Germany

来源：

HAEMOPHILIA | 2018年 / 24卷 / 06期

关键词：

annualized bleeding rate; haemophilia A; prophylaxis; recombinant factor VIII; turoctocog alfa; NOVOEIGHT(R);

D O I：

10.1111/hae.13617

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Introduction Turoctocog alfa is a recombinant factor VIII (FVIII) molecule, approved for treatment and prophylaxis of bleeding in patients with haemophilia A. In the guardian 1 (adolescents/adults) and guardian 3 (children) phase 3 trials, turoctocog alfa demonstrated a favourable efficacy and safety profile. Guardian 1 or 3 completers could enrol in the guardian 2 extension. Final guardian 2 results are reported here. Aim Methods Investigate long-term safety and efficacy of turoctocog alfa administered for prophylaxis and treatment of bleeds. In this phase 3b open-label trial, previously treated males of all ages with severe haemophilia A received prophylaxis regimens of turoctocog alfa or on-demand treatment of bleeds. The primary safety endpoint was frequency of FVIII inhibitor development. Efficacy endpoints included annualized bleeding rate (ABR) during prophylaxis, haemostatic response in treatment of bleeds and number of injections required to treat bleeds. Results Conclusion Overall, 213 patients were dosed with turoctocog alfa; 207 patients received prophylaxis; 19 received on-demand treatment. No FVIII inhibitors (>= 0.6 BU) were reported. For all patients on prophylaxis, overall median ABR was 1.37 bleeds/y; success rate for treatment of bleeds was 90.2%; and 88.2% of bleeds were controlled with 1-2 injections of turoctocog alfa. For the on-demand regimen, overall median ABR was 30.44 bleeds/y; success rate for treatment of bleeds was 96.7%; and 94.9% of bleeds were controlled with 1-2 injections of turoctocog alfa. Extended use of turoctocog alfa is safe and effective for prevention and treatment of bleeding episodes in previously treated patients with haemophilia A across all ages.

引用

页码：E391 / E394

页数：4

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