Post ISTH & ASH Headlines 2019

BLEEDING DISORDERS: CONGENITAL HAEMOPHILIA

Abstracts selected by Professor Mike Makris (Sheffield)

Abstract 636
BIVV001: The First Investigational Factor VIII Therapy to Break Through the VWF Ceiling in Hemophilia A, with Potential for Extended Protection for One Week or Longer
Abstract (PDF)

 

Abstract 632
Emicizumab Prophylaxis Provides Flexible and Effective Bleed Control in Children with Hemophilia Α with Inhibitors: Results from the HAVEN 2 Study
Abstract (PDF)

 

Abstract 634
The Atlanta Protocol: Immune Tolerance Induction in Pediatric Patients with Hemophilia a and Inhibitors on Emicizumab
Abstract (PDF)

 

Abstract 633
Immunogenicity of Emicizumab in People with Hemophilia A (PwHA): Results from the HAVEN 1-4 Studies
Abstract (PDF)

 

Abstract 635
Maintaining Perioperative Hemostasis in Patients with Severe Hemophilia A and Inhibitors Receiving Emicizumab Prophylaxis
Abstract (PDF)

 

Abstract 2476
Emicizumab for the Treatment of Acquired Hemophilia_A: Lessons Learned from 4 Very Different Cases
Abstract (PDF)

 

Abstract 491
Adeno-Associated Mediated Gene Transfer for Hemophilia B:8 Year Follow up and Impact of Removing “Empty Viral Particles” on Safety and Efficacy of Gene Transfer
Abstract (PDF)

 

Abstract 489
GO-8: Preliminary Results of a Phase I/II Dose Escalation Trial of Gene Therapy for Haemophilia a Using a Novel Human Factor VIII Variant
Abstract (PDF)

 

Abstract 631
A Single Intravenous Infusion of FLT180a Results in Factor IX Activity Levels of More Than 40% and Has the Potential to Provide a Functional Cure for Patients with Haemophilia B
Abstract (PDF)