Publications by authors named "C M Khayat"
Background: Prophylaxis with a von Willebrand factor (VWF) concentrate is recommended in von Willebrand disease (VWD) patients with a history of frequent and severe bleeds. Despite nosebleeds being a frequent manifestation of VWD, few studies have investigated the efficacy of factor prophylaxis in preventing nosebleeds in patients with severe VWD.
Methods: This post-hoc analysis of a prospective, 12-month, phase 3 study assessed the efficacy of wilate in the prevention of nosebleeds in 33 patients aged ≥6 years with severe type 1, type 2 or type 3 VWD.
Article Synopsis
- GRID1 and GRID2 are genes that encode proteins essential for the organization and development of synapses in the central nervous system, with variations linked to neurodevelopmental issues.
- The study analyzed human variants from various sources, finding many variants in intolerant domains that could disrupt normal protein function, particularly in the amino terminal and M3 transmembrane domains.
- Experiments showed that certain variants lead to dysfunction in receptor interaction and activity, with pentamidine effectively inhibiting hyperactive GluD2 variants, highlighting the importance of these receptors in both healthy function and disease contexts.
Purpose: Limited research exists on the time course of long-term retinal and cerebral deficits in diabetic rodents. Previously, we examined short term (4-8 weeks) deficits in the Goto-Kakizaki (GK) rat model of Type II diabetes. Here, we investigated the long-term (1-8 months) temporal appearance of functional deficits (retinal, cognitive, and motor), retinal vascular pathology, and retinal dopamine levels in the GK rat.
View Article and Find Full Text PDFIntroduction: Congenital afibrinogenemia treatment with plasma-derived fibrinogen concentrates in pediatric patients is limited. This study investigated the pharmacokinetics, surrogate efficacy, and safety of a plasma-derived fibrinogen concentrate (FIB Grifols) in pediatric patients with congenital afibrinogenemia.
Methods: Patients aged <18 years old diagnosed with congenital afibrinogenemia were included in this prospective, multinational, phase 1-2, single-arm study.
Background: Congenital fibrinogen deficiency (CFD) is a rare coagulation disorder placing patients at increased bleeding risk. Human fibrinogen concentrate (HFC) represents current standard of care for fibrinogen replacement in CFD, however, limited data are available on HFC for prophylactic administration before/during surgery. Here, we report results and dosing considerations for HFC treatment in perioperative bleeding management in adult, adolescent, and pediatric patients with CFD.
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