Publications by authors named "C McGuinn"
Article Synopsis
- Severe aplastic anemia (SAA) is a rare and serious blood disorder that can be life-threatening.
- Treatment outcomes are generally positive, but there are differences in how SAA is managed in adults versus children, and ongoing clinical trials are exploring various treatment options.
- Due to its rarity, some pediatric hematologists may lack extensive experience in treating SAA, highlighting the importance of current evidence-based recommendations for managing children with relapsed or refractory cases.
Article Synopsis
- Severe aplastic anemia (SAA) is a rare but serious blood disorder that can be life-threatening.
- Treatment for SAA works well, but how it's handled can be different for kids and adults.
- Some doctors who treat children may not have a lot of experience with SAA since it's so rare, so there are new recommendations to help them.
Congenital fibrinogen deficiency (CFD) is a rare bleeding disorder caused by mutations in FGA, FGB, and FGG. We sought to comprehensively characterize patients with CFD using PRO-RBDD (Prospective Rare Bleeding Disorders Database). Clinical phenotypes, laboratory, and genetic features were investigated using retrospective data from the PRO-RBDD.
View Article and Find Full Text PDFIntroduction: Hereditary factor X (FX) deficiency (FXD) is a rare autosomal recessive bleeding disorder. Plasma-derived FX (pdFX) is a high-purity FX concentrate approved in the United States and Europe for the treatment and prophylaxis of bleeding episodes and for peri-operative management in patients with hereditary FXD (HFXD).
Aim: To review pharmacokinetic dosing, efficacy, and safety data for pdFX as routine prophylaxis for HFXD.
Introduction: The B-Natural study is a multicentre, multinational, observational study of haemophilia B (HB) designed to increase understanding of clinical manifestations, treatment and quality of life (QoL).
Aim: To characterise and compare QoL in HB across disease severity groups and individuals with inhibitors to identify gaps in treatment.
Methods: A total of 224 individuals from 107 families were enrolled from a total of 24 centres in North America (n = 16), Europe (n = 7) and Asia (n = 1).