Bleeding Disorder News # 69: Special INHIBITOR Edition

Special Inhibitor Edition:

A Study of Clinical Profile and Treatment in Adult Hemophilia Patients with Special Reference to the Inhibitor Levels
https://www.cureus.com/articles/219460-a-study-of-clinical-profile-and-treatment-in-adult-hemophilia-patients-with-special-reference-to-the-inhibitor-levels#!/

PRESent-2 Research Study Available:  Hemophilia A/B with Inhibitors
The Efficacy and Safety of SerpinPC in Participants with Severe Hemophilia A or Moderately Severe to Severe Hemophilia B
https://clinicaltrials.gov/study/NCT05789524?cond=nct05789524&rank=1#locations
 
PRESent-3 Research Study Available:  Hemophilia B with Inhibitors
The Efficacy and Safety of SerpinPC in Participants with Hemophilia B with Inhibitors
https://clinicaltrials.gov/search?cond=nct05789537

TiumBio plans Phase 1b TU7710 trial in hemophilia with inhibitors
https://hemophilianewstoday.com/news/phase-1b-trial-tu7710-set-hemophilia-a-b-patients-with-inhibitors/

Investigational Drug Candidate For the Potential Treatment of Severe Hemophilia
https://checkrare.com/serpinpc-for-severe-hemophilia/

Motivate Study: The New Era of Immune Tolerance. - Sidonio, Robert (2021) LifeLines for Health, vol 8, 7-14.
https://ches.education/new-blog/2023/1/16/motivate-study-the-new-era-of-immune-tolerance-induction?rq=motivate

Race and ethnicity and the success of immune tolerance induction among people with severe haemophilia A in the United States. - Fedewa, S. A., & Kempton, C. L. (2024). Haemophilia, hae.14980.
https://doi.org/10.1111/hae.14980

Determinants of bleeding before and during immune tolerance in 222 boys with severe hemophilia A and inhibitors > 5 BU. - Fischer, K., Kenet, G., Kurnik, K., Carcao, M., Oldenburg, J., Stamm-Mikkelsen, T., Cid Haro, A. R., Koskenvuo, M., Blatny, J., & Königs, C. (2024). Blood Advances8(2), 369–377.
https://doi.org/10.1182/bloodadvances.2023011442

Defining the impact of immune tolerance induction on clinically relevant outcomes in a US cohort of severe hemophilia A. - Kempton, C. L., & Fedewa, S. A. (2024). Blood Advances8(5), 1190–1199.
https://doi.org/10.1182/bloodadvances.2023011974

Nonacog beta pegol prophylaxis in children with hemophilia B: Safety, efficacy, and neurodevelopmental outcomes for up to 8 years. - Walsh, K. S., Mrakotsky, C., Carcao, M., Chan, A. K. C., Nielsen, P. H., Holst, H., & Shapiro, K. (2024). Research and Practice in Thrombosis and Haemostasis8(2), 102341.
https://doi.org/10.1016/j.rpth.2024.102341

Effect of emicizumab-neutralizing antibodies on activated partial thromboplastin time-based clotting time test results in patients treated with emicizumab. - Novembrino, C., Boscolo-Anzoletti, M., Galbiati, E., Shinohara, S., & Peyvandi, F. (2023). Research and practice in thrombosis and haemostasis7(8), 102260.
https://doi.org/10.1016/j.rpth.2023.102260

Efficacy and safety evaluation of eptacog beta (coagulation factor VIIa [recombinant]-jncw) for the treatment of hemophilia A and B with inhibitors. - Pipe, S. W., Dunn, A. L., & Young, G. (2023). Expert review of hematology16(10), 715–729.
https://doi.org/10.1080/17474086.2023.2248385

Thrombosis and novel hemophilia therapies: the fine line between clotting and bleeding. - Hoffman M. (2021). Blood advances5(18), 3736.
https://doi.org/10.1182/bloodadvances.2021004802C

Non-factor therapies for bleeding disorders: A primer for the general haematologist. - Swan, D., Mahlangu, J., & Thachil, J. (2022). EJHaem3(3), 584–595.
https://doi.org/10.1002/jha2.442

Fitusiran prophylaxis in people with hemophilia A or B who switched from prior BPA/CFC prophylaxis (ATLAS-PPX). - Kenet, G., Nolan, B., Zülfikar, O. B., Antmen, B., Kampmann, P., Matsushita, T., You, C. W., Vilchevska, K., Bagot, C. N., Sharif, A., Peyvandi, F., Young, G., Negrier, C. G., Chi, J., Kittner, B., Sussebach, C., Shammas, F., Mei, B., Andersson, S. R., & Kavakli, K. (2024).  Blood, blood.2023021864. Advance online publication.
https://doi.org/10.1182/blood.2023021864

Concizumab as a Subcutaneous Prophylactic Treatment Option for Patients with Hemophilia A or B: A Review of the Evidence and Patient's Perspectives. - Pasca S. (2022). Journal of blood medicine13, 191–199.
https://doi.org/10.2147/JBM.S242219

Long-term efficacy and safety of subcutaneous concizumab prophylaxis in hemophilia A and hemophilia A/B with inhibitors. - Shapiro, A. D., Angchaisuksiri, P., Astermark, J., Benson, G., Castaman, G., Eichler, H., Jiménez-Yuste, V., Kavakli, K., Matsushita, T., Poulsen, L. H., Wheeler, A. P., Young, G., Zupančić-Šalek, S., Oldenburg, J., & Chowdary, P. (2022).  Blood advances6(11), 3422–3432.
https://doi.org/10.1182/bloodadvances.2021006403

Long-term safety and efficacy of the anti-tissue factor pathway inhibitor marstacimab in participants with severe haemophilia: Phase II study results. - Mahlangu, J., Luis Lamas, J., Cristobal Morales, J., Malan, D. R., Teeter, J., Charnigo, R. J., Hwang, E., & Arkin, S. (2023).  British journal of haematology200(2), 240–248.
https://doi.org/10.1111/bjh.18495

Hemostatic efficacy of marstacimab alone or in combination with bypassing agents in hemophilia plasmas and a mouse bleeding model. - Pittman, D. D., Rakhe, S., Bowley, S. R., Jasuja, R., Barakat, A., & Murphy, J. E. (2022). Research and practice in thrombosis and haemostasis6(2), e12679.
https://doi.org/10.1002/rth2.12679