Hemophilia and Rare Bleeding Disorders » Novel Biotherapeutics in Hemophilia

Abstract Number: OC 32.1

Ex vivo Assessment of the Importance of Activated Factor IX (FIXa) Levels in the Procoagulant Effect of Recombinant FIX Concentrates in Emicizumab-treated Patients

E.G Arias-Salgado¹, E. Monzón Manzano¹, P. Acuña¹, I. Fernández-Bello¹, S. García Barcenilla¹, M.T. Álvarez Román¹, M. Martín¹, M.I. Rivas Pollmar¹, T. Cebanu¹, E. González Zorrilla¹, N. Butta¹, V. Jiménez-Yuste^1,2

Abstract Number: OC 48.1

A Blood Coagulation Factor IX Variant that Functions Independently of Factor VIII Mitigates the Hemophilia A Phenotype in Patient Plasma

V.J. Strijbis¹, L.G. Romano², K.L. Cheung¹, Y.P. Liu³, A.C. McCreary³, F.W. Leebeek², M.H. Bos¹

Abstract Number: PB0682

A Systematic Review on the Pharmacokinetics and Associated Pharmacodynamics of Emicizumab in Humans

A. Donners¹, C. Rademaker¹, L. Bevers¹, A. Huitema^1,2,3, R. Schutgens¹, A. Egberts^1,4, K. Fischer¹

Abstract Number: PB0675

Administration of fVIII Protein through Human Placental Cells Reduces the Risk of an Antibody Response in Juvenile Sheep

B. Trevisan¹, M. Rodriguez¹, S. George¹, J. Shields², S. Lankford¹, R. Combs³, M. Gautreaux⁴, J. Owen³, A. Atala¹, C.B. Doering², H.T. Spencer², C.D. Porada¹, MG. Almeida-Porada¹

Abstract Number: LPB0116

An Accurate and Simple Liquid Chromatography Tandem Mass-spectrometric Method for the Quantification of Emicizumab in People with Hemophilia A

A. Donners¹, L. Gerencsér¹, K. van der Elst¹, K. Fischer¹, R. Urbanus¹, M. El Amrani¹

Abstract Number: PB0678

Cost-effectiveness Analysis of the Brazilian Immune Tolerance Induction Protocol with Recombinant Factor VIII and Prophylaxis with Bypassing Agents or Emicizumab

R.M. Camelo¹, S.M. Rezende¹, J. Álvares-Teodoro²

Abstract Number: PB0686

Do New Therapies alter the Treatment of Hemophilia Patients?

H. Duda¹, B. Haschberger¹, J. Hesse¹, H. Windecker¹, A. Hilger¹, C. Keipert¹

Abstract Number: PB0684

Emi and Me: The Real-world Impact of Emicizumab on the Lives of People with Haemophilia A with Inhibitors and their Families

S. Fletcher¹, M. Holland², K. Jenner², K. Khair²

Abstract Number: PB0681

Emicizumab Assays: One Stage vs. Chromogenic Stage Assay

M. Stuart¹, D. Chen¹, R. Pruthi¹

Abstract Number: OC 49.2

Emicizumab: Will it Suffice for Bone Metabolism?

M. Manco-Johnson¹, N. Briones¹, A. Tran¹, D. Thornhill¹, C. Baird¹, L. Jacobson¹, B. Warren¹, S. Kuldanek¹, D. Ambruso¹, G. Karsenty²

Abstract Number: PB0679

Measuring the Chromogenic FVIII Mimetic Activity of the New Bispecific Antibody, Mim8, in Artificially Spiked Severe Haemophilia A Plasma

A. Bowyer¹, M. Ezban², S. Kitchen¹

Abstract Number: PB0680

Measuring the FVIII Mimetic Activity of the New Bispecific Antibody, Mim8, in Severe Haemophilia A Plasma Using APTT and One-stage FVIII Assays

A. Bowyer¹, M. Ezban², S. Kitchen¹

Abstract Number: OC 48.4

Orally Delivered Factor VIII Restores Hemostasis in a Hemophilia A Dog

M.A. Hashim¹, A. Yamaguchi¹, A.T. Vo¹, A.K. Dhalla¹, B. Syed¹, L.C. Fung¹, V. Salgotra¹, A. Dasari¹, G. Schrenk², O. Zoechling², D. Sexton³, G. Allen³, C.T. Long⁴, E.P. Merricks⁴, M. Imran¹, T.C. Nichols⁴

Abstract Number: PB0677

Real-world Experience of Emicizumab Treatment Using Entire Vials Only

K. Fischer¹, A.A. Donners¹, R.T Urbanus¹, I.C. Kremer Hovinga¹, L.F. van Vulpen¹, P.R van der Valk¹, K.P. van Galen¹, N. Uitslager¹, R.E. Schutgens¹

Abstract Number: OC 08.4

Real-world Use of Damoctocog Alfa Pegol in Patients with Hemophilia A: Analysis of PK Profiles and Treatment Regimen Data from the WAPPS-Hemo Database

M.E. Mancuso¹, P. Chelle², E. Iserman³, T. Matthew³, A. Keepanasseril³, A. Edginton², A. Iorio^3,4, WAPPS-Hemo Investigators

Abstract Number: PB0687

Successful Experience of Emicizumab Prophylaxis in a Child with Severe Hemophilia A and Inhibitor without Previous Immune Tolerance Induction

R.M. Camelo¹, M. Palis², M.A.F. de Cerqueira², N. Dantas-Silva³, J. Álvares-Teodoro⁴

Abstract Number: PB0676

Tailoring the Effect of Antithrombin-targeting Therapy in Hemophilia A Using in Silico Thrombin Generation

R. de Laat-Kremers¹, M. Ninivaggi¹, I. van Moort², M. de Maat², B. de Laat¹

Abstract Number: LPB0117

The Association of Emicizumab and rFVIIa for Major Orthopaedic Surgery

C. Carulli¹, S. Linari², L. Pieri², G. Castaman²

Abstract Number: PB0674

The Effect of rFVIIa and Mim8 Combination in Hemophilia A-like Blood

M. Ezban¹, K. Jensen¹, J. Lund¹

Abstract Number: OC 48.3

Therapeutic Knockdown of Plasminogen with siRNA in Hemophilia A and von Willebrand disease Mouse Models

P. Batty¹, A. Strilchuk², K. Nesbitt¹, A. Yong², D. Lillicrap¹, C. Kastrup²