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EMICIZUMAB DATA

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Publications

Select publications and scientific data presentations with the strongest and most relevant data are provided below.

Bone and Joint Health

Effect of Emicizumab Prophylaxis on Bone and Joint Health Markers in People with Haemophilia A Without Factor VIII Inhibitors in the HAVEN 3 Study

Kiialainen A, Niggli M, Kempton C, et al. Haemophilia. 2022. doi: 10.1111/hae.14642.

Bone and Joint Health Markers in Persons With Hemophilia A (PwHA) Treated With Emicizumab in the HAVEN 3 Clinical Trial

Kiialainen A, Castaman G, Chang T, et al. Presented at the Virtual NHF Scientific Meeting; August 1-8, 2020. 

Healthcare Costs and Value

Valoctocogene Roxaparvovec and Emicizumab for Hemophilia A Without Inhibitors: Effectiveness and Value (Report-at-a-Glance)

Institute for Clinical and Economic Review, 2020

Hemophilia A and Mortality

Mortality in Congenital Hemophilia A - A Systematic Literature Review

Hay CRM, Nissen F, and Pipe SW. J Thromb Haemost. 2021; Suppl 1(Suppl 1):6–20. 

Establishment of a Framework for Assessing Mortality in Persons With Congenital Hemophilia A and Its Application to an Adverse Event Reporting Database

Pipe SW, Kruse-Jarres R, Mahlangu JN, et al. J Thromb Haemost. 2021; Suppl 1(Suppl 1):21–31.

Application of a Hemophilia Mortality Framework to the Emicizumab Global Safety Database

Peyvandi F, Mahlangu JN, Pipe SW, et al. J Thromb Haemost. 2021; Suppl 1(Suppl 1):32–41.

A Systematic Review of Mortality Statistics and Causes of Death in People With Congenital Hemophilia A (PwcHA)

Hay C, Nissen F, and Pipe S. Presented at the Virtual NHF Scientific Meeting; August 1-8, 2020.

A Contemporary Framework For Understanding Mortality in People With Congenital Hemophilia A (PwcHA)

Pipe S, Kruse-Jarres R, Mahlangu J, et al. Presented at the Virtual NHF Scientific Meeting; August 1-8, 2020. 

An Analysis of Fatalities in Persons With Congenital Hemophilia A Reported in the FDA Adverse Event Reporting System (FAERS) Database

De Ford C, Kuebler P, Shang A, et al. Presented at the Virtual NHF Scientific Meeting: August 1-8, 2020.

Impact of Inhibitors on Hemophilia A Mortality in the United States

Walsh CE, Soucie JM, Miller CH, et al. Am J Hematol. 2015;90:400–405.

Real-World Experience

Safety and Efficacy of Emicizumab in People With Hemophilia A Enrolled in the Hemophilia Natural History Study (ATHN 7)

Buckner TW, Carpenter SL, Kempton CL, et al. ISTH 2022; Oral presentation 40.4.

Real-World Experience on the Tolerability and Safety of Emicizumab Prophylaxis in Paediatric Patients With Severe Haemophilia A With and Without FVIII Inhibitors

Hassan E, Jonathan L, Jayashree M. Haemophilia 2021;27:e698-e703.

Emicizumab Initiation and Bleeding Outcomes in People With Hemophilia A With and Without Inhibitors: A Single-Center Report

Warren BB, Chan A, Manco-Johnson M, et al.  Res Pract Thromb Haemost 2021;5:e12571.

Real-World Data on Bleeding Patterns of Hemophilia A Patients Treated With Emicizumab

Levy-Mendelovich S, Brutman-Barazani T, Budnik I, et al. J Clin Med 2021;10(19):4303.

Emicizumab in Pediatric Hemophilia: Bleeding and Surgical Outcomes from a Single-Center Retrospective Study

Cohen CT, Diaz R. Pediatr Blood Cancer 2021;68:e29325.

Real-World Case Series and Summary of Current Literature of Infants and Toddlers With Severe Hemophilia A With Inhibitor on Prophylaxis With Emicizumab

Garcia J and Zia A. Pediatr Blood Cancer. 2021;68(5):e28942.

Emicizumab Prophylaxis in Haemophilia Patients Older than 50 Years With Cardiovascular Risk Factors: Real-World Data

Misgav M, Brutman-Barazani T, Budnik I, et al. Haemophilia. 2021;27(2):253-260.

 Emicizumab Treatment and Monitoring in a Paediatric Cohort: Real-World Data

Barg AA, Livnat T, Budnik I,  et al. Br J Haematol. 2020;191(2):282-290.

Real-World Use of Emicizumab in Patients With Haemophilia A: Bleeding Outcomes and Surgical Procedures

McCary I, Guelcher C, Kuhn J, et al. Haemophilia. 2020;26:631636.

Emicizumab Prophylaxis in Patients With Haemophilia A With and Without Inhibitors

Ebbert PT, Xavier F, Seaman D, et al. Haemophilia. 2020;26:41–46.

Emicizumab Prophylaxis Among Infants and Toddlers With Severe Hemophilia A and Inhibitors-A Single-Center Cohort

Barg AA, Avishai E, Budnik I, et al. Pediatr Blood Cancer. 2019;66:e27886.