Hemophilia and Rare Bleeding Disorders » Novel Biotherapeutics in Hemophilia

Abstract Number: PB0218

Adaptative methods using one-stage and chromogenic substrate assays for the quantitative measurement of Mim8 in human plasma

N. Martineau¹, L. Fauconnier¹, N. Roland¹, A. Carlo², T. Hervé³, F. Depasse², M. Ezban⁴, L. Nicoud¹

Abstract Number: PB0225

An engineered factor X variant as a novel by-passing agent for hemophilia

E. Tonetto¹, M. Testa¹, R. Tarantino², L. Peretto¹, M. Pinotti¹, A. Branchini¹

Abstract Number: PB0222

Assay of modified factor VIII is modulated by von Willebrand factor. What should we measure ?

C. Kluft, M. Verhoek

Abstract Number: PB0226

Assay of the factor VIIIa mimetic, Mim8, using one-stage clotting assay requires a product-specific reference material

H. Wilmot¹, M. Ezban², E. Gray³

Abstract Number: PB0215

Efficacy, effectiveness, and safety of emicizumab for the treatment of people with hemophilia A: a systematic review

R. Muniz¹, R. Camelo², J. Álvares-Teodoro¹, M. Barbosa¹, M. Araujo³

Abstract Number: PB0216

Efficacy, effectiveness, and safety of extended half-life factor VIII products for the treatment of hemophilia A: protocol of a systematic review

M. Araujo¹, R. Camelo², M. Barbosa³, R. Muniz³, J. Álvares-Teodoro³

Abstract Number: VPB0230

Emicizumab treatment of 4 patients with severe hemophilia A and inhibitors in pediatric age – Resource management to minimize economic costs while maintaining the quality of clinical follow-up

M. Urbano¹, F. Machado², B. Marques¹, C. Guedes³, T. Brites³, O. Lavrukhina³, S. Silva², J. Feio², C. Geraldes¹, R. Salvado³, J. Tomaz³

Abstract Number: PB0229

Emicizumab: In-house method validation

R. Sueldo¹, M. Hepner², J. Frontroth³, E. Annetta⁴, C. Cervio³, M. Bianco⁵, G. Sciuccati⁵

Abstract Number: PB0220

Evaluation of four different reagents on the coagulation analyzers STAR MAX® and ATELLICA® COAG 360 for Emicizumab concentration measurement in severe hemophilia A patients

A. Launois¹, E. de Raucourt², I. Martin-Toutain¹, F. SAMDJEE³, S. LE DORE³, E. FERRE³, C. Flaujac⁴

Abstract Number: PB0224

Evaluation of thrombin generation and emicizumab concentration in a cohort of severe Hemophilia A patients with and without inhibitor during emicizumab prophylaxis

M. Mormile¹, F. Barone², E. Baldacci¹, A. Ferretti², M. Chavez Orellana², G. Maestrini², A. Serrao³, F. Viola², A. Chistolini², C. Santoro¹

Abstract Number: OC 40.1

FRONTIER1: A Phase 1/2 Dose Escalation Study of a Novel Factor VIIIa Mimetic Bispecific Antibody, Mim8, for Evaluation of Safety, Pharmacokinetics, and Efficacy

P. Chowdary¹, F. Jaime², J. Mahlangu³, C. Eskelund⁴, R. Meldgaard⁴, P. Persson⁴, J. Windyga⁵

Abstract Number: PB0217

Impacts of Mim8 on routine coagulation assays, on intrinsic pathway coagulation factor assays and on thrombophilia coagulation tests

L. Fauconnier¹, N. Roland¹, A. Carlo², T. Hervé³, F. Depasse², M. Ezban⁴, L. Nicoud¹, N. Martineau¹

Abstract Number: OC 50.3

Improved Procoagulant Activity of Hemophilia B Causing Dysfunctional Factor IX Variants with Factor VIII Mimetics

J. Chau¹, A. Sternberg¹, A. Pishko², L. George¹, B. Samelson-Jones¹

Abstract Number: VPB0228

Investigation of properties of macropore silica sorbents and active dyes as ligands suitable for purification of factor VIII coagulation

N. Shurko, T. Danysh, I. Yurchyshak, S. Miliashkevych, V. Novak

Abstract Number: VPB0227

Laboratory monitoring in haemophilia A patients with factor VIII inhibitor treated with emicizumab

C. Novembrino¹, M. Boscolo-Anzoletti², E. Galbiati², F. Peyvandi³

Abstract Number: PB0223

Mim8 does not affect FVIII function in hemophilia A

J. Lund, K. Jensen, M. Ezban

Abstract Number: OC 50.5

Mim8 is associated with improved thrombin generation vs. emicizumab in patients with haemophilia A, with and without inhibitors

J. Windyga¹, P. Chowdary², F. Jaime³, C. Eskelund⁴, P. Jørgensen⁴, M. Kreilgaard⁴, J. Mahlangu⁵

Abstract Number: PB0221

Neutralizing antidrug antibody to emicizumab: Detailed laboratory evaluation and review of the literature

H. KIZILOCAK¹, J. Brown², G. Young³

Abstract Number: PB0214

Snake venom-based hemocoagulase restores in vivo hemostasis and limits bleeding in murine model of hemophilia A

N. Rhoads¹, L. Stanger¹, M. Wong², B. Li², M. Holinstat¹, R. Adili³

Abstract Number: PB0219

Thrombin generation measurement in hemophilia A patients with inhibitors receiving emicizumab prophylaxis

Y. Dargaud¹, C. Escuriola Ettingshausen², S. Desage³, S. Meunier⁴, A. Lienhart⁴