Results 121 to 130 of about 113,823 (359)

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

On a break with the X: the role of repair of double-stranded DNA breaks in X-linked disease [PDF]

, 2012
The problem of managing free reactive DNA ends in eukaryotic cells has resulted in the development of a number of mechanisms in order to ensure that free ends are rendered non-reactive, or that the double-strand DNA breaks generating the free ends are ...
Cecceroni, Lucia, De Caris, Laura, Tummala, Hemanth   +2 more
core   +4 more sources

Mortality, life expectancy, and causes of death of persons with hemophilia in the Netherlands 2001–2018

Journal of Thrombosis and Haemostasis, 2020
Treatment of patients with hemophilia has advanced over the past decades, but it is unknown whether this has resulted in a normal life expectancy in the Netherlands.
S. Hassan, R. Monahan, E. Mauser-Bunschoten, L. V. van Vulpen, J. Eikenboom, E. Beckers, L. Hooimeijer, P. Ypma, L. Nieuwenhuizen, M. Coppens, S. Schols, F. Leebeek, C. Smit, M. Driessens, S. le Cessie, E. C. van Balen, F. Rosendaal, J. G. van der Bom, S. Gouw   +18 more
semanticscholar   +1 more source

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

Hemophilia is associated with accelerated biological aging

Haematologica
Hemophilia is a rare X-linked bleeding disorder caused by mutations in the F8 or F9 gene (hemophilia A or B), leading to deficient factor VIII or IX proteins, respectively.
Marina Trappl, Rafaela Vostatek, Manuel Salzmann, Daniel Kraemmer, Johanna Gebhart, Philipp Hohensinner, Ingrid Pabinger, Cihan Ay   +7 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

Safety and Efficacy of Damoctocog Alfa Pegol Prophylaxis in Patients With Severe Haemophilia A: Results of an Interventional, Post‐Marketing Study

Haemophilia, EarlyView.
ABSTRACT This is a plain language summary of the results of a trial of damoctocog alfa pegol (BAY 94–9027, Jivi).
Pål André Holme, Lone Hvitfeldt Poulsen, Claudia Tueckmantel, Monika Maas Enriquez, María Teresa Alvarez Román, Raimondo De Cristofaro   +5 more
wiley   +1 more source

GENETIC DIAGNOSTICS OF HEMOPHILIA A

Zdravniški Vestnik, 2004
Background. Hemophilia A is a chromosome X-linked bleeding disorder due to mutations in the FVIII gene. There are 163 hemophilia A patients in Slovene registry for Hemophilia.
Maruša Debeljak, Majda Benedik Dolničar, Lana Strmecki   +2 more
doaj  

Novel variant in HPS3 gene in a patient with Hermansky Pudlak syndrome (HPS) type 3

Platelets, 2020
Hermansky-Pudlak syndrome (HPS) is a rare autosomal recessive disorder caused by defects in 10 human HPS genes, characterized by oculocutaneous albinism (OCA) and bleeding diathesis associated to platelet δ-storage pool defect (SPD).
Anna Lecchi, Silvia La Marca, Eti A Femia, Antonia Lenz, Doris Boeckelmann, Andrea Artoni, Flora Peyvandi, Barbara Zieger   +7 more
doaj   +1 more source

Association of inferior vena cava filter placement for venous thromboembolic disease and a contraindication to anticoagulation with 30-day mortality [PDF]

, 2018
Importance: Despite the absence of data from randomized clinical trials, professional societies recommend inferior vena cava (IVC) filters for patients with venous thromboembolic disease (VTE) and a contraindication to anticoagulation therapy.
Brown, David L, Novak, Eric, Saeed, Mohammed J, Turner, Tyson E   +3 more
core   +1 more source