New Biological Insights in von Willebrand Disease: Angiogenesis and Angiodysplasia
In the Tuesday afternoon session entitled von Willebrand Disease: Clinical Conundrums, Anna Randi describes the mechanisms that regulate angiogenesis proposing a possible role of VWF in this regulation and discussing therapeutic implications of the latest findings in this field.
von Willebrand Disease: Current Approaches to Diagnosis and Treatment
In the Tuesday afternoon session entitled von Willebrand Disease: Clinical Conundrums, Frank Leebek presents the current challenges in VWD diagnosis and treatment, namely bleeding score variability, overlap of type 1 VWD with low VWF, and the hampering effect of biological variation on test results, as well as the effect of DDAVP on VWF levels.
Investigational SPK-9001: AAV-Mediated Gene Transfer for Hemophilia B – Persistent, Stable Factor IX Activity at One Year Independent of Downstream Purification Method
In the Tuesday morning session entitled Free Papers – Gene Therapy, Spencer Sullivan presents clinical results of FIX gene transfer using in vivo recombinant AAV delivery with the novel bioengineered vector SPA-9001, discussing vector design, FIX activity attained, and safety.
New Approaches to Tolerance: Oral Delivery of Blood Clotting Factors Bioencapsulated in Plant Cells
In the Monday morning session entitled Inhibitors: Clinical Aspects, Henry Daniell presents current advances in the development of new, affordable systems for oral delivery of clotting factors.
What is… Health Economics and Health Economic Evaluation
In the Wednesday morning session entitled Can’t or Won’t Pay: Health Economics and Bleeding Disorders, Jamie O’Hara describes the many variables and types of analyses that go into health economics assessments.
How to Set Up Chromogenic FVIII and FIX Assays
In the Tuesday afternoon session entitled Role of Chromogenic Assays in Hemophilia Diagnosis, Annette Bowyer explains how to set up chromogenic assays for FVIII and FIX, covering technical aspects such as calibration, accuracy, and quality control in the context of these assays, as well as the cost implications of using them appropriately.
Role of Chromogenic FVIII Assays in Hemophilia A Diagnosis
In the Tuesday afternoon session entitled Role of Chromogenic Assays in Hemophilia Diagnosis, Elizabeth Duncan presented a comparison of clotting assays versus the chromogenic assay in quantifying FVIII, highlighting genotype and phenotype discrepancies and reverse discrepancies.
Women and Girls with Bleeding Disorders
In the Tuesday morning session entitled Women With Bleeding Disorders: Clinical and Psychological Issues, Claire McLintock discussed the effect misinformation and taboos around menstruation can have on the life of women with bleeding disorders.
Low-Dose Prophylaxis – India Experience
In the Tuesday afternoon session entitled Prophylaxis: Global Models of Low-Dose Prophylaxis, Shashikant Apte presents the results of the implementation of tertiary prophylaxis programs in India, focusing on reduction of annual bleed rate (ABR) in hemophilia A and B.
FXI Deficiency: Genotype, Phenotype and Management
In the Monday afternoon session entitled Rare Bleeding Disorders, Gillian Gidley reviews the genotypes and phenotypes associated with FXI deficiency, shows data on different diagnostic assays, and covers recommendations for management of patients with FXI deficiency.
The Role of the Laboratory in Diagnosis and Management of Inhibitory Antibodies in Hemophilia
In the Monday morning session entitled Inhibitors: Clinical Aspects, Jan Astermark described the different diagnostic assays laboratories can carry out to measure inhibitors.
Rare Bleeding Disorders: Overview of Clinical Assessment and Diagnosis
In the Monday afternoon session entitled Rare Bleeding Disorders, Flora Peyvandi gives an overview of RBDs in Europe and discusses current technical limitations in laboratory testing, presents current treatments, and also discusses novel treatment strategies.