Results 21 to 30 of about 112,813 (257)

Factor VIII/V C-domain swaps reveal discrete C-domain roles in factor VIII function and intracellular trafficking

open access: yesHaematologica, 2017
Factor VIII C-domains are believed to have specific functions in cofactor activity and in interactions with von Willebrand factor. We have previously shown that factor VIII is co-targeted with von Willebrand factor to the Weibel-Palade bodies in blood ...
Eduard H.T.M. Ebberink   +7 more
doaj   +1 more source

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

open access: yesResearch 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   +24 more
doaj   +1 more source

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

open access: yesHaematologica, 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   +7 more
doaj   +1 more source

Desmopressin in moderate hemophilia A patients: a treatment worth considering

open access: yesHaematologica, 2018
Desmopressin increases endogenous factor VIII levels in hemophilia A. Large inter-individual variation in the response to desmopressin is observed.
Janneke I. Loomans   +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]

open access: yesPLoS 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   +8 more
doaj   +1 more source

Effects of Cancer Treatment on Somatosensory and Nociceptive Processing in Children and Adolescents: A Systematic Review

open access: yesPediatric Blood &Cancer, EarlyView.
ABSTRACT Chemotherapy‐induced peripheral neuropathy remains a major complication in pediatric cancer, with disrupted somatosensory and nociceptive processing being a key aspect. This review synthesizes empirical studies on alterations in somatosensory and nociceptive processing in children and adolescents with cancer.
Julia Schweiger   +4 more
wiley   +1 more source

Results of the Childhood Cancer and Leukaemia Group's United Kingdom Relapsed Wilms Tumour Trial

open access: yesPediatric Blood &Cancer, EarlyView.
ABSTRACT Background The United Kingdom relapsed Wilms tumour (UKW‐R) trial aimed to improve the historically low survival rates after relapse of Wilms tumour (WT) through a prospective national risk‐stratified protocol. The trial also evaluated efficacy and toxicity of high‐dose melphalan.
Sucheta J. Vaidya   +10 more
wiley   +1 more source

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

open access: yesHaematologica, 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   +7 more
doaj   +1 more source

Efficacy and Tolerability of Topotecan/Cyclophosphamide/Dinutuximab in Relapsed and Refractory High‐Risk Neuroblastoma: A Multi‐Institutional Retrospective Study

open access: yesPediatric Blood &Cancer, EarlyView.
ABSTRACT Purpose Chemoimmunotherapy with irinotecan, temozolomide, and dinutuximab (I/T/DIN) has emerged as first‐line therapy for relapsed/refractory (r/r) high‐risk neuroblastoma (HRNB) in North America. Topotecan and cyclophosphamide (T/C) are often used in combination with dinutuximab in the setting of lack of response, progression, or incomplete ...
Benjamin J. Lerman   +17 more
wiley   +1 more source

Clinical Course and Impact of Breaks in Therapy for Children With Relapsed/Refractory Solid Tumors

open access: yesPediatric Blood &Cancer, EarlyView.
ABSTRACT Introduction Pediatric relapsed or refractory (R/R) solid tumors carry a dismal prognosis, and postrelapse patient experiences are not well described. We present postrelapse outcomes, including number of R/R events and subsequent therapy regimens.
Matthew T. McEvoy   +5 more
wiley   +1 more source

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