Simoctocog alfa (Nuwiq®) in previously untreated patients with severe haemophilia A-Final efficacy and safety results from the NuProtect study

被引：1

作者：

Mathias, Mary ^{[1
]}

Abraham, Aby ^{[2
]}

Belletrutti, Mark J. ^{[3
,4
]}

Carcao, Manuel ^{[5
]}

Carvalho, Manuela ^{[6
]}

Chambost, Herve ^{[7
]}

Chan, Anthony K. C. ^{[8
]}

Dubey, Leonid ^{[9
]}

Ducore, Jonathan ^{[10
]}

Gattens, Michael ^{[11
]}

Gresele, Paolo ^{[12
]}

Gruel, Yves ^{[13
]}

Guillet, Benoit ^{[14
]}

Jimenez-Yuste, Victor ^{[15
]}

Kitanovski, Lidija ^{[16
]}

Klukowska, Anna ^{[17
]}

Lohade, Sunil ^{[18
]}

Mancuso, Maria Elisa ^{[19
]}

Oldenburg, Johannes ^{[20
]}

Pollio, Berardino ^{[21
]}

Sigaud, Marianne ^{[22
]}

Vilchevska, Kateryna ^{[23
]}

Wu, John K. M. ^{[3
,4
]}

Jansen, Martina ^{[24
]}

Belyanskaya, Larisa ^{[25
]}

Walter, Olaf ^{[25
]}

Knaub, Sigurd ^{[25
]}

Neufeld, Ellis J. ^{[26
]}

机构：

[1] Great Ormond St Hosp Sick Children, Haemophilia Comprehens Care Ctr, NIHR GOSH BRC, Haemophilia Ctr, London, England

[2] Christian Med Coll & Hosp, Dept Hematol, Vellore, India

[3] Univ British Columbia, Dept Pediat, Div Hematol Oncol BMT, Vancouver, BC, Canada

[4] British Columbia Childrens Hosp, Vancouver, BC, Canada

[5] Res Inst Hosp Sick Children, Dept Paediat, Div Haematol Oncol & Child Hlth Evaluat Sci, Toronto, ON, Canada

[6] Sao Joao Univ Hosp Ctr, Congenital Coagulopathies Reference Ctr, Porto, Portugal

[7] Aix Marseille Univ, Children Hosp La Timone, AP HM, Dept Pediat Hematol Oncol,INSERM,INRA,C2VN, Marseille, France

[8] McMaster Univ, McMaster Ctr Transfus Res, Dept Pediat, Hamilton, ON, Canada

[9] Western Ukrainian Specialized Childrens Med Ctr, Dept Paediat, Lvov, Ukraine

[10] Univ Calif Davis, Dept Pediat, Davis Med Ctr, Sacramento, CA USA

[11] Cambridge Univ Hosp NHS Fdn Trust, Addenbrookes Hosp, Dept Paediat Haematol & Oncol, Cambridge, England

[12] Univ Perugia, Dept Med & Surg, Perugia, Italy

[13] Hop Trousseau, Ctr Reg Traitement Hemophilie, Tours, France

[14] Univ Rennes, Irset Inst Rech Sante Environm & Travail, Haemophilia Treatment Ctr, CHU Rennes,UMR S 1085,INSERM,EHESP, Rennes, France

[15] Univ Autonoma Madrid, Hosp Univ La Paz, Serv Hematol, Madrid, Spain

[16] Univ Med Ctr Ljubljana, Dept Haematooncol, Div Paediat, Ljubljana, Slovenia

[17] Polish Soc Haematol & Transfusiol, Haemostasis Grp, Warsaw, Poland

[18] Sahyadri Special Hosp, Dept Hematol, Pune, India

[19] IRCCS Humanitas Res Hosp, Ctr Thrombosis & Hemorrhag Dis, Milan, Italy

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

[21] Regina Margherita Children Hosp, Reg Reference Ctr Inherited Bleeding & Thrombot D, Turin, Italy

[22] Univ Hosp Nantes, Ctr Reg Traitement Hemophilie, Nantes, France

[23] OHMATDYT Natl Specialized Childrens Hosp, Dept Hematol, Kiev, Ukraine

[24] Octapharma Pharmazeut Prod GesmbH, Vienna, Austria

[25] Octapharma AG, Lachen, Switzerland

[26] St Jude Childrens Res Hosp, Dept Hematol, Memphis, TN USA

来源：

EUROPEAN JOURNAL OF HAEMATOLOGY | 2023年 / 111卷 / 04期

关键词：

FVIII inhibitors; haemophilia A; NuProtect; Nuwiq; simoctocog alfa; HUMAN-CL RHFVIII; RECOMBINANT FACTOR-VIII; HUMAN CELL-LINE; INHIBITOR DEVELOPMENT; CHILDREN; ADULTS; IMMUNOGENICITY; PROPHYLAXIS; PRODUCTS; 1ST;

D O I：

10.1111/ejh.14040

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Introduction Simoctocog alfa (Nuwiq (R)) is a 4th generation recombinant FVIII with proven efficacy for the prevention and treatment of bleeding episodes (BEs) in previously treated patients with severe haemophilia A. The NuProtect study assessed the immunogenicity, efficacy and safety of simoctocog alfa in 108 previously untreated patients (PUPs). The incidence of high-titre inhibitors was 16.2% and no patients with non-null F8 mutations developed inhibitors.Aim To report the efficacy and safety results from the NuProtect study.Methods PUPs received simoctocog alfa for prophylaxis, treatment of BEs, or as surgical prophylaxis. The efficacy of prophylaxis (during inhibitor-free periods) was assessed using annualised bleeding rates (ABRs). The efficacy in treating BEs and in surgical prophylaxis was assessed using a 4-point scale. Adverse events were recorded throughout the study.Results Of 108 PUPs treated with simoctocog alfa, 103 received at least one prophylactic dose and 50 received continuous prophylaxis for at least 24 weeks. In patients on continuous prophylaxis, the median ABR was 0 (mean 0.5) for spontaneous BEs and 2.5 (mean 3.6) for all BEs. In 85 patients who had BEs, efficacy of BE treatment was excellent or good for 92.9% (747/804) of rated BEs; 92.3% of BEs were treated with 1 or 2 infusions. The efficacy of surgical prophylaxis was excellent or good for 94.7% (18/19) of rated procedures. There were no safety concerns and no thromboembolic events.Conclusion Simoctocog alfa was efficacious and well tolerated as prophylaxis, surgical prophylaxis and for the treatment of BEs in PUPs with severe haemophilia A.

引用

页码：544 / 552

页数：9

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