Risk factors for inhibitor development in severe hemophilia A

I Garagiola, R Palla, F Peyvandi - Thrombosis research, 2018 - Elsevier
Although significant advances in hemophilia treatment have improved patient outcomes and
quality of life, one of the greatest complications in severe hemophilia A is the development of …

Laboratory monitoring in emicizumab-treated persons with hemophilia A

J Müller, I Pekrul, B Pötzsch, B Berning… - Thrombosis and …, 2019 - thieme-connect.com
Hemophilia A (HA) is an X-linked hereditary bleeding disorder caused by deficiency of
coagulation factor (F) VIII activity. One of the greatest complications in the treatment of HA is …

Expression and assembly of largest foreign protein in chloroplasts: oral delivery of human FVIII made in lettuce chloroplasts robustly suppresses inhibitor formation in …

KC Kwon, A Sherman, WJ Chang… - Plant biotechnology …, 2018 - Wiley Online Library
Inhibitor formation is a serious complication of factor VIII (FVIII) replacement therapy for the X‐
linked bleeding disorder haemophilia A and occurs in 20%–30% of patients. No prophylactic …

B cell–activating factor modulates the factor VIII immune response in hemophilia A

BS Doshi, J Rana, G Castaman… - The Journal of …, 2021 - Am Soc Clin Investig
Inhibitors of factor VIII (FVIII) remain the most challenging complication of FVIII protein
replacement therapy in hemophilia A (HA). Understanding the mechanisms that guide FVIII …

Application of systems biology to identify pharmacological mechanisms of thrombotic microangiopathy evoked by combined activated prothrombin complex …

R Valls, J Wagg, I Paz-Priel, G Man, L Artigas… - Scientific Reports, 2023 - nature.com
Emicizumab is a bispecific monoclonal antibody that substitutes for the function of missing or
deficient factor VIII (FVIII) in people with hemophilia A (PwHA). Long-term safety and efficacy …

Platelet-targeted gene therapy induces immune tolerance in hemophilia and beyond

S Kumar, JA Schroeder, Q Shi - Journal of Thrombosis and Haemostasis, 2024 - Elsevier
Blood platelets have unique storage and delivery capabilities. Platelets play fundamental
roles in hemostasis, inflammatory reactions, and immune responses. Beyond their functions …

Optimization and evaluation of a two-stage chromogenic assay procedure for measurement of emicizumab plasma levels

NS Hamedani, J Oldenburg, B Pötzsch, J Müller - Plos one, 2022 - journals.plos.org
Emicizumab mimics the hemostatic activity of activated factor VIII (FVIIIa) within the tenase
complex. Despite functional similarities between FVIIIa and emicizumab, conventional …

[HTML][HTML] Hemofilia: abordaje diagnóstico y terapéutico. Revisión bibliográfica

LM Martínez-Sánchez, LF Álvarez-Hernández… - … Facultad Nacional de …, 2018 - scielo.org.co
Antecedentes: La hemofilia es una enfermedad de origen genético, recesiva y ligada al
cromosoma X; su principal manifestación clínica es la hemorragia, cuyo grado depende del …

Two‐center validation of assays for the detection of binding and neutralizing anti‐factor VIII antibodies

J Müller, S Neimanis, J Kahle, T Albert… - …, 2024 - Wiley Online Library
Introduction Patients with hemophilia A treated with coagulation Factor VIII (FVIII) products
are at risk for developing anti‐FVIII antibodies. The ABIRISK Consortium aimed to provide …

Engineering a therapeutic protein to enhance the study of anti-drug immunity

PE Zerra, ET Parker, WH Baldwin, JF Healey, SR Patel… - Biomedicines, 2022 - mdpi.com
The development of anti-drug antibodies represents a significant barrier to the utilization of
protein-based therapies for a wide variety of diseases. While the rate of antibody formation …