Results 31 to 40 of about 13,061,190 (344)

Inhibitor development upon switching from plasma-derived to recombinant factor VIII in previously untreated patients with severe hemophilia A: the PUP-SWITCH study

Research and Practice in Thrombosis and Haemostasis
Background: The SIPPET randomized clinical trial showed that in previously untreated patients (PUPs) with severe hemophilia A, treatment with plasma-derived factor (F)VIII (pdFVIII) within the first 50 exposure days (EDs) was associated with a lower ...
Syna Miri, Frits R. Rosendaal, Kaan Kavakli, Peyman Eshghi, Soha Mohammadi Moghaddam, Sara Scardo, Behnaz Habibpanah, Mohsen Elalfy, Susan Halimeh, Gabriella Nicolò, Dilek Gökçebay, Namık Özbek, Tiraje Celkan, Ahmad Mohammadi, Mehran Karimi, Amin Shahsavani, Bariş Yılmaz, Canan Albayrak, Burcak Gunes, Zühre Kaya, Yilmaz Ay, Sinan Akbayram, Nazan Sarper, Pier Mannuccio Mannucci, Flora Peyvandi   +24 more
doaj   +1 more source

The factor VIII protein and its function.

Acta Biochimica Polonica, 2016
Factor VIII (FVIII), an essential blood coagulation protein, is a key component of the fluid phase blood coagulation system. Human factor VIII is a single chain of about 300 kDa consisting of domains described as A1-A2-B-A3-C1-C2.
A. Mazurkiewicz-Pisarek, G. Płucienniczak, T. Ciach, A. Plucienniczak   +3 more
semanticscholar   +1 more source

N-linked glycosylation modulates the immunogenicity of recombinant human factor VIII in hemophilia A mice

Haematologica, 2018
Immune responses to factor VIII remain the greatest complication in the treatment of severe hemophilia A. Recent epidemiological evidence has highlighted that recombinant factor VIII produced in baby hamster kidney cells is more immunogenic than factor ...
Jesse D. Lai, Laura L. Swystun, Dominique Cartier, Kate Nesbitt, Cunjie Zhang, Christine Hough, James W. Dennis, David Lillicrap   +7 more
doaj   +1 more source

Desmopressin in moderate hemophilia A patients: a treatment worth considering

Haematologica, 2018
Desmopressin increases endogenous factor VIII levels in hemophilia A. Large inter-individual variation in the response to desmopressin is observed.
Janneke I. Loomans, Marieke J.H.A. Kruip, Manuel Carcao, Shannon Jackson, Alice S. van Velzen, Marjolein Peters, Elena Santagostino, Helen Platokouki, Erik Beckers, Jan Voorberg, Johanna G. van der Bom, Karin Fijnvandraat, for the RISE consortium   +12 more
doaj   +1 more source

The 1.7 Å X-ray crystal structure of the porcine factor VIII C2 domain and binding analysis to anti-human C2 domain antibodies and phospholipid surfaces. [PDF]

PLoS ONE, 2015
The factor VIII C2 domain is essential for binding to activated platelet surfaces as well as the cofactor activity of factor VIII in blood coagulation. Inhibitory antibodies against the C2 domain commonly develop following factor VIII replacement therapy
Caileen M Brison, Steven M Mullen, Michelle E Wuerth, Kira Podolsky, Matthew Cook, Jacob A Herman, Justin D Walter, Shannon L Meeks, P Clint Spiegel   +8 more
doaj   +1 more source

Pazopanib Combined With Vincristine and Irinotecan in Relapsed Wilms Tumor: Encouraging Outcomes in a Heavily Pretreated Pediatric Cohort

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background While Wilms tumor (WT) typically has a favorable prognosis, relapsed cases—especially those with high‐risk histology—remain therapeutically challenging after intensive frontline therapy. The combination of vincristine and irinotecan has demonstrated activity in pediatric solid tumors, and pazopanib, a multi‐targeted tyrosine kinase ...
Maria Debora De Pasquale, Alessandro Crocoli, Giuseppe Maria Milano, Pierluigi Di Paolo, Francesca Diomedi Camassei, Ida Russo, Annalisa Serra   +6 more
wiley   +1 more source

Factor VIII activity and bleeding risk during prophylaxis for severe hemophilia A: a population pharmacokinetic model

Haematologica, 2020
During factor VIII prophylaxis for severe hemophilia A, bleeding risk increases with time when factor VIII activity is below 1%. Maintaining trough activity above 1% does not protect all patients from bleeding.
Andreas Tiede, Faraizah Abdul Karim, Victor Jiménez-Yuste, Robert Klamroth, Sandra Lejniece, Takashi Suzuki, Andreas Groth, Elena Santagostino   +7 more
doaj   +1 more source

Psychosocial Outcomes in Patients With Endocrine Tumor Syndromes: A Systematic Review

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Introduction The combination of disease manifestations, the familial burden, and varying penetrance of endocrine tumor syndromes (ETSs) is unique. This review aimed to portray and summarize available data on psychosocial outcomes in patients with ETSs and explore gaps and opportunities for future research and care.
Daniël Zwerus, Floortje Strobbe, Annemarie A. Verrijn Stuart, Hanneke M. van Santen, Gerlof D. Valk, Sasja A. Schepers, Rachel S. van Leeuwaarde   +6 more
wiley   +1 more source

Survival for Children Diagnosed With Wilms Tumour (2012–2022) Registered in the UK and Ireland Improving Population Outcomes for Renal Tumours of Childhood (IMPORT) Study

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background The Improving Population Outcomes for Renal Tumours of childhood (IMPORT) is a prospective clinical observational study capturing detailed demographic and outcome data on children and young people diagnosed with renal tumours in the United Kingdom and the Republic of Ireland.
Naomi Ssenyonga, Angela Lopez‐Cortes, Reem Al‐Saadi, Suzanne Tugnait, Kristina Dzhuma, Catriona Duncan, Gordan Vujanic, Tanzina Chowdhury, Claudia Allemani, Michel P. Coleman, Kathy Pritchard‐Jones, the Children's Cancer and Leukaemia Group (CCLG) Renal Tumour Special Interest Group, Hugh Bishop, Anthony McCarthy, Pamela Kearns, Helen Rees, Amos Burke, Meriel Jenney, Jane Pears, Hamish Wallace, Milind Ronghe, Tanzina Chowdhury, Susan Picton, Emma Ross, Lisa Howell, Bernadette Brennan, Gail Halliday, Richard Grundy, Shaun Wilson, Sucheta Vaidya, Daniel Yeomanson, Jessica Bate, Vesna Pavasovic, Jessica Bate, Sabine Irtan, Jesper Brok, Minou Oostveen, Kristina Dzhuma, Mark Weeks, Richard Williams, Mark Powis, Max Pachl, Bruce Okoye, Naima Smeulders, Gordan Vujanic, Oystein Olsen, Tom Watson, Susan Shelmerdine, Dan Saunders, Angela Lopez-Cortes, Taryn Treger, Tom Jackson, Suzanne Tugnait, Lisa Howell, Tanzina Chowdhury, Sam Behjati, Angela Polanco   +56 more
wiley   +1 more source

The MedSupport Multilevel Intervention to Enhance Support for Pediatric Medication Adherence: Development and Feasibility Testing

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Introduction We developed MedSupport, a multilevel medication adherence intervention designed to address root barriers to medication adherence. This study sought to explore the feasibility and acceptability of the MedSupport intervention strategies to support a future full‐scale randomized controlled trial.
Elizabeth G. Bouchard, Hital Patel, Paula C. Vincent, Justine Kahn, Kira Bona, Thai Hoa Tran, Elisa M. Rodriguez, Kristopher Attwood, Kara M. Kelly   +8 more
wiley   +1 more source