Treatment of severe von Willebrand disease with a high-purity von Willebrand factor concentrate (Wilfactin (R)): a prospective study of 50 patients

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Treatment of severe von Willebrand disease with a high-purity von Willebrand factor concentrate (Wilfactin (R)): a prospective study of 50 patients. / Borel-Derlon, A.; Federici, A. B.; Roussel-Robert, V.; Goudemand, J.; Lee, C. A.; Scharrer, I.; Rothschild, C.; Berntorp, Erik; Henriet, C.; Tellier, Z.; Bridey, F.; Mannucci, P. M.

In: Journal of Thrombosis and Haemostasis, Vol. 5, No. 6, 2007, p. 1115-1124.

Research output: Contribution to journalArticle

Harvard

Borel-Derlon, A, Federici, AB, Roussel-Robert, V, Goudemand, J, Lee, CA, Scharrer, I, Rothschild, C, Berntorp, E, Henriet, C, Tellier, Z, Bridey, F & Mannucci, PM 2007, 'Treatment of severe von Willebrand disease with a high-purity von Willebrand factor concentrate (Wilfactin (R)): a prospective study of 50 patients', Journal of Thrombosis and Haemostasis, vol. 5, no. 6, pp. 1115-1124. https://doi.org/10.1111/j.1538-7836.2007.02562.x

APA

CBE

Borel-Derlon A, Federici AB, Roussel-Robert V, Goudemand J, Lee CA, Scharrer I, Rothschild C, Berntorp E, Henriet C, Tellier Z, Bridey F, Mannucci PM. 2007. Treatment of severe von Willebrand disease with a high-purity von Willebrand factor concentrate (Wilfactin (R)): a prospective study of 50 patients. Journal of Thrombosis and Haemostasis. 5(6):1115-1124. https://doi.org/10.1111/j.1538-7836.2007.02562.x

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Author

Borel-Derlon, A. ; Federici, A. B. ; Roussel-Robert, V. ; Goudemand, J. ; Lee, C. A. ; Scharrer, I. ; Rothschild, C. ; Berntorp, Erik ; Henriet, C. ; Tellier, Z. ; Bridey, F. ; Mannucci, P. M. / Treatment of severe von Willebrand disease with a high-purity von Willebrand factor concentrate (Wilfactin (R)): a prospective study of 50 patients. In: Journal of Thrombosis and Haemostasis. 2007 ; Vol. 5, No. 6. pp. 1115-1124.

RIS

TY - JOUR

T1 - Treatment of severe von Willebrand disease with a high-purity von Willebrand factor concentrate (Wilfactin (R)): a prospective study of 50 patients

AU - Borel-Derlon, A.

AU - Federici, A. B.

AU - Roussel-Robert, V.

AU - Goudemand, J.

AU - Lee, C. A.

AU - Scharrer, I.

AU - Rothschild, C.

AU - Berntorp, Erik

AU - Henriet, C.

AU - Tellier, Z.

AU - Bridey, F.

AU - Mannucci, P. M.

PY - 2007

Y1 - 2007

N2 - Background and objectives: A plasma-derived von Willebrand factor (VWF) concentrate with low factor VIII (FVIII) content was specifically developed to treat von Willebrand disease (VWD). Efficacy and safety were investigated by merging the results of two comparable protocols conducted prospectively in 5 European and 12 French centers. Methods and results: Fifty patients with clinically severe VWD (72% had VWF ristocetin cofactor activity less than 10 IU dL(-1) and 46% had FVIII < 20 IU dL(-1)) were treated with the concentrate as the only therapy, except for clinical situations requiring a priming dose of FVIII to rapidly correct an intrinsic coagulation defect. A total of 139 spontaneous bleeding episodes were treated; only 53 (38%) needed a concomitant FVIII dose. Outcome was excellent or good in 89% of the episodes. Forty-four patients underwent 108 surgical or invasive procedures. Outcome was excellent or good in 95 scheduled procedures (only VWF was infused) and 13 emergency procedures (a priming FVIII dose was co-administered with the first VWF infusion). There were no thrombotic complications and none of the 18 patients with type 3 VWD developed anti-VWF or anti-FVIII antibodies. Conclusions. This concentrate safely and effectively provides hemostasis in patients with clinically severe VWD.

AB - Background and objectives: A plasma-derived von Willebrand factor (VWF) concentrate with low factor VIII (FVIII) content was specifically developed to treat von Willebrand disease (VWD). Efficacy and safety were investigated by merging the results of two comparable protocols conducted prospectively in 5 European and 12 French centers. Methods and results: Fifty patients with clinically severe VWD (72% had VWF ristocetin cofactor activity less than 10 IU dL(-1) and 46% had FVIII < 20 IU dL(-1)) were treated with the concentrate as the only therapy, except for clinical situations requiring a priming dose of FVIII to rapidly correct an intrinsic coagulation defect. A total of 139 spontaneous bleeding episodes were treated; only 53 (38%) needed a concomitant FVIII dose. Outcome was excellent or good in 89% of the episodes. Forty-four patients underwent 108 surgical or invasive procedures. Outcome was excellent or good in 95 scheduled procedures (only VWF was infused) and 13 emergency procedures (a priming FVIII dose was co-administered with the first VWF infusion). There were no thrombotic complications and none of the 18 patients with type 3 VWD developed anti-VWF or anti-FVIII antibodies. Conclusions. This concentrate safely and effectively provides hemostasis in patients with clinically severe VWD.

KW - von Willebrand disease

KW - von Willebrand factor

KW - concentrate

U2 - 10.1111/j.1538-7836.2007.02562.x

DO - 10.1111/j.1538-7836.2007.02562.x

M3 - Article

VL - 5

SP - 1115

EP - 1124

JO - Journal of Thrombosis and Haemostasis

T2 - Journal of Thrombosis and Haemostasis

JF - Journal of Thrombosis and Haemostasis

SN - 1538-7933

IS - 6

ER -