Background For the treatment of von Willebrand disease (VWD), von Willebrand factor (VWF) concentrates can be used in on-demand, long-term prophylaxis, and surgical prophylaxis regimens. Methods This systematic literature review was conducted to evaluate the efficacy, consumption, and safety of plasma-derived human coagulation FVIII/human VWF (pdVWF/FVIII; Voncento/Biostate) for the treatment of patients with any inherited VWD type. An electronic search was conducted in MEDLINE and Cochrane Library databases on VWD therapies. All retrieved publications were assessed against predefined inclusion/exclusion criteria following the Cochrane group recommendations. Associated pharmacovigilance data were collected across the same time period. Results Eleven publications from eight study cohorts were identified for data retrieval. All were from multicenter studies and included both pediatric and adult patients. Eight publications included evaluations of the efficacy of pdVWF/FVIII for on-demand treatment, eight included long-term prophylactic treatment, and eight included surgical prophylaxis. Treatment protocols and VWF administration methods differed between studies, as did safety evaluations. The clinical response was rated as excellent/good for on-demand treatment in 66 to 100% of nonsurgical bleeds, 89 to 100% in the treatment of breakthrough bleeds during long-term prophylaxis treatment, and hemostatic efficacy in surgical procedures was 75 to 100%. Pharmacovigilance data confirmed a low incidence of adverse events in treated patients. Conclusion This review provides a comprehensive summary of studies that evaluated the use of pdVWF/FVIII in VWD demonstrating the long-term effectiveness and safety of this pdVWF/FVIII across all ages, types of VWD, and treatment settings.
机构:
Fdn IRCCS, Sci Direct, Ca Granda Osped Maggiore Policlin, Milan, ItalyUniv Milan, Angelo Bianchi Bonomi Haemophilia & Thrombosis Ct, Fdn IRCCS, Ca Granda Osped Maggiore Policlin,Dept Pathophysi, I-20122 Milan, Italy
Mannucci, Pier M.
Valsecchi, Carla
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Univ Milan, Angelo Bianchi Bonomi Haemophilia & Thrombosis Ct, Fdn IRCCS, Ca Granda Osped Maggiore Policlin,Dept Pathophysi, I-20122 Milan, ItalyUniv Milan, Angelo Bianchi Bonomi Haemophilia & Thrombosis Ct, Fdn IRCCS, Ca Granda Osped Maggiore Policlin,Dept Pathophysi, I-20122 Milan, Italy
Valsecchi, Carla
Pontiggia, Silvia
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Univ Milan, Angelo Bianchi Bonomi Haemophilia & Thrombosis Ct, Fdn IRCCS, Ca Granda Osped Maggiore Policlin,Dept Pathophysi, I-20122 Milan, ItalyUniv Milan, Angelo Bianchi Bonomi Haemophilia & Thrombosis Ct, Fdn IRCCS, Ca Granda Osped Maggiore Policlin,Dept Pathophysi, I-20122 Milan, Italy
Pontiggia, Silvia
Farina, Claudio
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Kedr SpA, R&D Dept, Lucca, ItalyUniv Milan, Angelo Bianchi Bonomi Haemophilia & Thrombosis Ct, Fdn IRCCS, Ca Granda Osped Maggiore Policlin,Dept Pathophysi, I-20122 Milan, Italy
Farina, Claudio
Retzios, Anastassios D.
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Bay Clin R&D Serv LLC, San Ramon, CA USAUniv Milan, Angelo Bianchi Bonomi Haemophilia & Thrombosis Ct, Fdn IRCCS, Ca Granda Osped Maggiore Policlin,Dept Pathophysi, I-20122 Milan, Italy
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Rhode Isl Hosp, COBRE Ctr Canc Res Dev, Providence, RI 02903 USA
Rhode Isl Hosp, Dept Med, Div Med Oncol, Providence, RI 02903 USA
Brown Univ, Providence, RI 02912 USARhode Isl Hosp, COBRE Ctr Canc Res Dev, Providence, RI 02903 USA
Clifton, James G.
Huang, Feilei
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Rhode Isl Hosp, COBRE Ctr Canc Res Dev, Providence, RI 02903 USA
Rhode Isl Hosp, Dept Med, Div Med Oncol, Providence, RI 02903 USA
Brown Univ, Providence, RI 02912 USARhode Isl Hosp, COBRE Ctr Canc Res Dev, Providence, RI 02903 USA
Huang, Feilei
Kovac, Spomenka
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JJ Strossmayer Univ, Dept Chem, Osijek, CroatiaRhode Isl Hosp, COBRE Ctr Canc Res Dev, Providence, RI 02903 USA
Kovac, Spomenka
Yang, Xinli
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Rhode Isl Hosp, COBRE Ctr Canc Res Dev, Providence, RI 02903 USA
Rhode Isl Hosp, Dept Med, Div Med Oncol, Providence, RI 02903 USA
Brown Univ, Providence, RI 02912 USARhode Isl Hosp, COBRE Ctr Canc Res Dev, Providence, RI 02903 USA
Yang, Xinli
Hixson, Douglas C.
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Rhode Isl Hosp, COBRE Ctr Canc Res Dev, Providence, RI 02903 USA
Rhode Isl Hosp, Dept Med, Div Med Oncol, Providence, RI 02903 USA
Brown Univ, Providence, RI 02912 USARhode Isl Hosp, COBRE Ctr Canc Res Dev, Providence, RI 02903 USA
Hixson, Douglas C.
Josic, Djuro
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Rhode Isl Hosp, COBRE Ctr Canc Res Dev, Providence, RI 02903 USA
Rhode Isl Hosp, Dept Med, Div Med Oncol, Providence, RI 02903 USA
Brown Univ, Providence, RI 02912 USA
JJ Strossmayer Univ, Dept Chem, Osijek, CroatiaRhode Isl Hosp, COBRE Ctr Canc Res Dev, Providence, RI 02903 USA