Results 91 to 100 of about 12,413,959 (307)

Efficacy and Safety of Prophylaxis With a Plasma‐Derived von Willebrand Factor/Factor VIII Concentrate (Wilate) in Patients With Type 3 von Willebrand Disease—A WIL‐31 Study Sub‐Analysis

European Journal of Haematology, EarlyView.
ABSTRACT Objectives The WIL‐31 study demonstrated efficacy and safety of prophylaxis with the plasma‐derived von Willebrand factor/factor VIII concentrate wilate in von Willebrand disease (VWD) of all types and was the only prospective study with an on‐demand run‐in study as an intra‐individual comparator.
Claudia Djambas Khayat, Leonid Dubey, Adlette Inati, Toshko Lissitchkov, Dzmitry Novik, Elina Peteva, Robert F. Sidonio Jr, Ali T. Taher, Kateryna V. Vilchevska, Vladimir Vdovin, Ana Boban   +10 more
wiley   +1 more source

Exploring patient and clinician perspectives on the benefits and risks of emerging therapies for the treatment of haemophilia: a qualitative study

The Journal of Haemophilia Practice
Enhanced horizon scanning for emerging treatments has identified that both haemophilia A and haemophilia B pathways will be enriched with a range of new medicines with varying benefit-risk profiles.
Spoors John, Payne Katherine, Wright Stuart, Horsley Will, Bell Sadie, Cairns John   +5 more
doaj   +1 more source

The relationship between target joints and direct resource use in severe haemophilia

Health Economics Review, 2018
Objectives Target joints are a common complication of severe haemophilia. While factor replacement therapy constitutes the majority of costs in haemophilia, the relationship between target joints and non drug-related direct costs (NDDCs) has not been ...
Jamie O’Hara, Shaun Walsh, Charlotte Camp, Giuseppe Mazza, Liz Carroll, Christina Hoxer, Lars Wilkinson   +6 more
doaj   +1 more source

Intracranial haemorrhage in haemophilia A and B [PDF]

British Journal of Haematology, 2007
SummaryIn countries with a good standard of health care, intracranial haemorrhage (ICH) during the neonatal period affects 3·5–4·0% of all haemophilia boys, which is considerably (40–80 times) higher than expected in the normal population. ICHs are also frequent after the neonatal period, affecting 3–10% of the haemophilia population who are mainly ...
openaire   +2 more sources

Contrasting Approaches in the Implementation of GRADE Methodology in Guidelines for Haemophilia and Von Willebrand Disease

Haemophilia, EarlyView.
ABSTRACT Introduction The 2024 ISTH clinical practice guideline (CPG) for treatment of congenital haemophilia, the NBDF‐McMaster Guideline on Care Models for Haemophilia Management, and ASH ISTH NBDF WFH guidelines on the diagnosis and management of VWD all utilised GRADE methodology.
Mark W. Skinner, Manuela Albisetti, Jesús Ardila, Jan Astermark, Jan Blatny, Manuel Carcao, Pratima Chowdary, Nathan T. Connell, Miguel Crato, Yesim Dargaud, Roseline d'Oiron, Amy L. Dunn, Miguel A. Escobar, Carmen Escuriola‐Ettingshausen, Emna Gouider, Annie Harroche, Cedric Hermans, Victor Jimenez‐Yuste, Radoslaw Kaczmarek, Gili Kenet, Liane Khoo, Robert Klamroth, Florian Langer, David Lillicrap, Johnny Mahlangu, Christoph Male, Tadashi Matsushita, Sandrine Meunier, Wolfgang Miesbach, Beatrice Nolan, Johannes Oldenburg, Brian O'Mahony, Margareth Ozelo, Glenn F. Pierce, Gloria Ramos, Michael Recht, Olivia Romero‐Lux, Dawn Rotellini, Rita C. Santoro, Tammuella C. Singleton, Alok Srivastava, Sophie Susen, Kate Talks, Huyen Tran, Leonard A. Valentino, Jerzy Windyga, Renchi Yang, Maria Elisa Mancuso, also on behalf of AICE, AHAD‐AP, AHCDC, ATHN, CLAHT, EHC, French CRH & MHEMO, HTCCNC, NBDF, PEDNET, UKHCDO, WFH   +59 more
wiley   +1 more source

Liver gene therapy by lentiviral vectors reverses anti‐factor IX pre‐existing immunity in haemophilic mice

EMBO Molecular Medicine, 2013
A major complication of factor replacement therapy for haemophilia is the development of anti‐factor neutralizing antibodies (inhibitors). Here we show that liver gene therapy by lentiviral vectors (LVs) expressing factor IX (FIX) strongly reduces pre ...
Andrea Annoni, Alessio Cantore, Patrizia Della Valle, Kevin Goudy, Mahzad Akbarpour, Fabio Russo, Sara Bartolaccini, Armando D'Angelo, Maria Grazia Roncarolo, Luigi Naldini   +9 more
doaj   +1 more source

Health‐Related Quality of Life in Adult Patients With von Willebrand Disease From Germany: Results of the WIL‐QoL Study

Haemophilia, EarlyView.
ABSTRACT Introduction Assessment of health‐related quality of life (HRQoL) is relatively new in von Willebrand disease (VWD). So far, generic questionnaires have mainly been used for HRQoL assessment in VWD. Aims To assess generic and disease‐specific HRQoL in adult VWD patients and compare HRQoL with the general German population.
Sylvia von Mackensen, Carolin Moorthi, Ronald Fischer, Susan Halimeh, Christine Heller, Wolfgang Miesbach, Freimut H. Schilling, Cornelia Wermes, Guenter Auerswald, the WIL‐QoL study group, Karin Beutel, Georg Goldmann, Bettina‐Kemkes Matthes, Robert Klamroth, Ralf Knöfler, Christoph Male, Johannes Oldenburg, Ute Scholz   +17 more
wiley   +1 more source

Surgery in a Patient with Haemophilia A and Lymphoma

Case Reports in Hematology, 2020
An increased incidence of haematologic malignancies and other cancer types among patients with haemophilia compared with matched controls has been reported in several longitudinal studies.
M. S. Cruz, J. Santillan, J. Lesser, J. Ortiz, L. Forzani, P. Plaza   +5 more
doaj   +1 more source

Safety of Biological Medicinal Products for Hereditary Coagulopathies: An Analysis of the Russian Pharmacovigilance Database

Безопасность и риск фармакотерапии
INTRODUCTION. With the expanding product range and the introduction of novel biological medicinal products for haemophilia A, haemophilia B, and von Willebrand disease in the Russian pharmaceutical market, post-marketing safety surveillance becomes ...
D. S. Fokina, O. V. Zhukova, A. L. Khokhlov   +2 more
doaj   +1 more source

Safety and Effectiveness of Emicizumab in People With Haemophilia A Enrolled in the ATHN 7 Haemophilia Natural History Study

Haemophilia, EarlyView.
ABSTRACT Background The Natural History Study of the Safety, Effectiveness, and Practice of Treatment for People with Haemophilia (ATHN 7) monitors use of contemporary haemophilia therapies, including emicizumab, a bispecific antibody substituting for activated factor (F)VIII in people with haemophilia A (HA).
Tyler W. Buckner, Shannon L. Carpenter, Nabil Daoud, Christine L. Kempton, Lucy Lee, Lynn Malec, Thomas W. McLean, Paul Morton, Carrie O'Neill, Janice M. Staber, Michael Wang, Stacy E. Croteau, Michael Recht   +12 more
wiley   +1 more source