57th ASH Annual Meeting and Exposition (December 5-8, 2015): 322. Disorders of Coagulation or Fibrinolysis: Novel Treatment Strategies in Hemophilia

Disorders of Coagulation or Fibrinolysis Oral and Poster Abstracts
Oral

W311ABCD, Level 3 (Orange County Convention Center)

Moderators:

Rodney M. Camire, PhD, Children's Hospital of Philadelphia and Yesim G. Dargaud, MD, PhD, Hopital Edouard Herriot Pav. E

Disclosures:

Camire: Pfizer: Consultancy , Patents & Royalties , Research Funding ; Novo Nordisk: Research Funding ; Spark Therapeutics: Membership on an entity’s Board of Directors or advisory committees .

10:30 AM

547

Personalized Prophylaxis with Human-Cl Recombinant FVIII in HA Patients

Robert Klamroth¹^*, Toshko Lissitchkov, MD²^*, Luminita Rusen³^*, Olaf Walter, MD, MBA⁴^*, Johann Bichler, PhD⁴^*, K John Pasi⁵, Andreas Tiede, MD, PhD⁶ and Sigurd Knaub, PhD⁴

¹Vivantes Clinic Friedrichshain, Berlin, Germany
²Department of Coagulation Disorders and Anemia, SHAT Joan Pavel, Sofia, Bulgaria
³Sanador SR, Bucharest, Romania
⁴Octapharma AG, Lachen SZ, Switzerland
⁵Royal London Haemophilia Centre, Barts and the London School of Medicine and Dentistry, London, United Kingdom
⁶Hematology, Hemostasis, Oncology and Stem Cell Transplantation, Hannover Medical School, Hannover, Germany

10:45 AM

548

Efficacy and Safety Results of Prolong-9FP Clinical Program of Recombinant Fusion Protein Linking Coagulation Factor IX with Albumin (rIX-FP) in Previously Treated Patients with Hemophilia B

Elena Santagostino, MD, PhD¹, Christine Voigt, PhD²^*, Denise Wolko²^*, Grace Cole²^*, Yanyan Li, PhD²^* and Iris C. Jacobs, MD²

¹Centro Emofilia e Trombosi, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy
²CRD, CSL Behring, King of Prussia, PA

11:00 AM

549

The Effect of Prophylactic Recombinant Factor IX and Glycopegylated Factor IX to Normalize Articular Wound Healing Following Hemarthrosis Examined in Hemophilia B Mice

Junjiang Sun, MD¹^*, Eric Livingston²^*, Tom Knudsen, DVM, PhD³^*, Dougald M. Monroe III, PhD⁴, Mirella Ezban, Ph.D.⁵^*, Ted Bateman, PhD⁶^* and Paul E. Monahan, MD⁷

¹Gene Therapy Center, University of North Carolina, Chapel Hill, NC
²Department of Biomedical Engineering, University of North Carolina at Chapel Hill, Chapel Hill, NC, NC
³Novo Nordisk A/S, Maaloev, Denmark
⁴Division of Hematology/Oncology, Department of Medicine, University of North Carolina, Chapel Hill, NC
⁵Novo Nordisk, Maaloev, Denmark
⁶Department of Biomedical Engineering, University of North Carolina at Chapel Hill, Chapel Hill, NC
⁷Department of Pediatrics and Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC

11:15 AM

550

Towards Restoring Coagulation in Hemophilia By Antibody-Mediated Inhibition of Antithrombin

Peter Boross, PhD¹^*, Cafer Yildiz²^*, Kamran Bakhtiari, PhD³^*, Joost C.M. Meijers, Prof, PhD³ and C. Erik Hack, MD, PhD^1,2^*

¹Prothix B.V., Leiden, Netherlands
²Laboratorium for Translational Immunology, UMC Utrecht, Utrecht, Netherlands
³Department of Plasma Proteins, Sanquin Research, Amsterdam, Netherlands

11:30 AM

551

A Subcutaneously Administered Investigational RNAi Therapeutic (ALN-AT3) Targeting Antithrombin for Treatment of Hemophilia: Interim Weekly and Monthly Dosing Results in Patients with Hemophilia A or B

K John Pasi¹, Pencho Georgiev²^*, Tim Mant³^*, Michael Desmond Creagh⁴^*, Toshko Lissitchkov, MD⁵^*, David Bevan⁶^*, Steve Austin⁷^*, Charles R Hay⁸^*, Brigit Brand⁹, Rashid Kazmi¹⁰^*, Pratima Chowdary¹¹^*, Margaret V Ragni¹², Jihong Chen¹³^*, Akin Akinc¹³^*, Benny Sorensen¹³ and Savita Rangarajan¹⁴

¹Royal London Haemophilia Centre, Barts and the London School of Medicine and Dentistry, London, United Kingdom
²University Multiprofile Hospital for Active Treatment “Sveti Georgi’, Plovdiv, Bulgaria
³Quintiles Drug Research Unit, London, United Kingdom
⁴Royal Cornwall Hospitals NHS Trust, Truro, United Kingdom
⁵Department of Coagulation Disorders and Anemia, SHAT Joan Pavel, Sofia, Bulgaria
⁶Guy's and St Thomas' Hospital NHS Trust, London, United Kingdom
⁷St. George’s Healthcare NHS Trust Haemophilia Centre, London, United Kingdom
⁸Manchester Royal Infirmary, Manchester, United Kingdom
⁹Universitaetsspital Zuerich, Zeurich, Switzerland
¹⁰University Hospital Southampton NHS Foundation Trust, Southampton, United Kingdom
¹¹Royal Free Hospital, London, United Kingdom
¹²University of Pittsburgh and Hemophilia Center of Western Pennsylvania, Pittsburgh, PA
¹³Alnylam Pharmaceuticals, Cambridge, MA
¹⁴Southern Haemophilia Network, Hampshire Hospitals NHF Foundation Trust, London, United Kingdom

11:45 AM

552

Antithrombin Reduction Corrected Thrombin Generation in Samples from Hemophilia A and B Patients with Inhibitors

Gili Kenet^1,2, Tami Livnat³^*, Emma Fosbury⁴^*, Pratima Chowdary⁴^*, Alfica Sehgal⁵^*, Akin Akinc⁵^* and Benny Sorensen⁵

¹Sackler Faculty of Medicine, Tel-Aviv University, Tel-Aviv, Israel
²National hemophilia center and the Institue of Thrombosis and Hemostasis, The Chaim Sheba Medical Centre, Ramat Gan, Israel
³Tel Hashomer Hospital, Tel Aviv, Israel
⁴Royal Free London NHS Foundation Trust, London, United Kingdom
⁵Alnylam Pharmaceuticals, Cambridge, MA

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322.O2.6 322. Disorders of Coagulation or Fibrinolysis: Novel Treatment Strategies in Hemophilia