Plasma-Derived von Willebrand Factor/Factor VIII Concentrate (Haemate P) in von Willebrand Disease : A Systematic Review and Pharmacovigilance Update
(2025) In Haemophilia 31(2). p.247-262- Abstract
Background: Von Willebrand disease (VWD) is an inherited bleeding disorder caused by deficient or dysfunctional von Willebrand factor (VWF). VWF replacement therapy is indicated in VWD management. Methods: This systematic review was conducted to evaluate all available evidence of the efficacy, safety, dosing and consumption of pasteurized plasma-derived human coagulation FVIII/human VWF (pdVWF/FVIII; Haemate P/Humate-P) concentrate for on-demand (OD) treatment, surgical prophylaxis and long-term prophylaxis of patients with VWD. A systematic search was performed in MEDLINE and Cochrane Library databases to identify studies (7 June 1982–31 May 2023) reporting the use of pdVWF/FVIII in VWD according to predefined selection criteria.... (More)
Background: Von Willebrand disease (VWD) is an inherited bleeding disorder caused by deficient or dysfunctional von Willebrand factor (VWF). VWF replacement therapy is indicated in VWD management. Methods: This systematic review was conducted to evaluate all available evidence of the efficacy, safety, dosing and consumption of pasteurized plasma-derived human coagulation FVIII/human VWF (pdVWF/FVIII; Haemate P/Humate-P) concentrate for on-demand (OD) treatment, surgical prophylaxis and long-term prophylaxis of patients with VWD. A systematic search was performed in MEDLINE and Cochrane Library databases to identify studies (7 June 1982–31 May 2023) reporting the use of pdVWF/FVIII in VWD according to predefined selection criteria. Pharmacovigilance data were also retrieved for the same period. Results: Fifteen studies were identified, 12 being observational and three interventional. Efficacy and safety assessments and treatment protocols varied across the studies which hindered direct comparisons. Haemostatic efficacy of pdVWF/FVIII was rated excellent/good for OD treatment in 95%–98% of bleeds and in 94%–100% of surgeries. In two separate studies, prophylactic efficacy was rated excellent/good in 100% of treatment cycles. Where reported, median annualized bleeding rates decreased from 3–24 prior prophylaxis to 0.5–6 during prophylaxis. Analysis of pharmacovigilance safety reports showed that pdVWF/FVIII was associated with a low rate of adverse events. Conclusions: This systematic literature review and analysis of pharmacovigilance data summarize evidence of over 40 years of clinical use of pdVWF/FVIII, supporting its safety and efficacy in VWD.
(Less)
- author
- Ettingshausen, Carmen Escuriola ; Lassila, Riitta ; Escolar, Gines ; Male, Christoph ; Schirner, Kathrin ; Heyder, Lisa and Berntorp, Erik LU
- organization
- publishing date
- 2025-03
- type
- Contribution to journal
- publication status
- published
- keywords
- factor VIII, safety, systematic review, von Willebrand disease, von Willebrand factor
- in
- Haemophilia
- volume
- 31
- issue
- 2
- pages
- 16 pages
- publisher
- John Wiley & Sons Inc.
- external identifiers
-
- pmid:39924968
- scopus:85217374938
- ISSN
- 1351-8216
- DOI
- 10.1111/hae.15138
- language
- English
- LU publication?
- yes
- id
- 053d147a-d3b0-41f8-9924-bc76a1eea0c3
- date added to LUP
- 2025-04-09 11:16:31
- date last changed
- 2025-06-04 15:19:44
@article{053d147a-d3b0-41f8-9924-bc76a1eea0c3,
abstract = {{<p>Background: Von Willebrand disease (VWD) is an inherited bleeding disorder caused by deficient or dysfunctional von Willebrand factor (VWF). VWF replacement therapy is indicated in VWD management. Methods: This systematic review was conducted to evaluate all available evidence of the efficacy, safety, dosing and consumption of pasteurized plasma-derived human coagulation FVIII/human VWF (pdVWF/FVIII; Haemate P/Humate-P) concentrate for on-demand (OD) treatment, surgical prophylaxis and long-term prophylaxis of patients with VWD. A systematic search was performed in MEDLINE and Cochrane Library databases to identify studies (7 June 1982–31 May 2023) reporting the use of pdVWF/FVIII in VWD according to predefined selection criteria. Pharmacovigilance data were also retrieved for the same period. Results: Fifteen studies were identified, 12 being observational and three interventional. Efficacy and safety assessments and treatment protocols varied across the studies which hindered direct comparisons. Haemostatic efficacy of pdVWF/FVIII was rated excellent/good for OD treatment in 95%–98% of bleeds and in 94%–100% of surgeries. In two separate studies, prophylactic efficacy was rated excellent/good in 100% of treatment cycles. Where reported, median annualized bleeding rates decreased from 3–24 prior prophylaxis to 0.5–6 during prophylaxis. Analysis of pharmacovigilance safety reports showed that pdVWF/FVIII was associated with a low rate of adverse events. Conclusions: This systematic literature review and analysis of pharmacovigilance data summarize evidence of over 40 years of clinical use of pdVWF/FVIII, supporting its safety and efficacy in VWD.</p>}},
author = {{Ettingshausen, Carmen Escuriola and Lassila, Riitta and Escolar, Gines and Male, Christoph and Schirner, Kathrin and Heyder, Lisa and Berntorp, Erik}},
issn = {{1351-8216}},
keywords = {{factor VIII; safety; systematic review; von Willebrand disease; von Willebrand factor}},
language = {{eng}},
number = {{2}},
pages = {{247--262}},
publisher = {{John Wiley & Sons Inc.}},
series = {{Haemophilia}},
title = {{Plasma-Derived von Willebrand Factor/Factor VIII Concentrate (Haemate P) in von Willebrand Disease : A Systematic Review and Pharmacovigilance Update}},
url = {{http://dx.doi.org/10.1111/hae.15138}},
doi = {{10.1111/hae.15138}},
volume = {{31}},
year = {{2025}},
}