Emicizumab Data by Topic

 

The below peer-reviewed scientific and clinical data provide a balanced cross-section of the current information available on key topics of interest for emicizumab. Please note that the references provided are not an exhaustive list of published materials on emicizumab but include selections of the most relevant and rigorous studies available for each topic of interest. The reference lists include journal publications that present original scientific research, analysis, or data on emicizumab. The posters and presentations provided are Genentech/Roche-sponsored congress publications, and also include congress abstracts from national hemophilia registries. Single case reports are generally excluded, with the exception of journal publications related to safety (e.g., thrombotic microangiopathy, thrombotic events, major surgical procedures).

For further information or inquiries on specific topics of interest for emicizumab, you may contact Genentech Medical Information.

Bone and joint health markers in persons with hemophilia A (PwHA) treated with emicizumab in HAVEN 3

Oral Presentation, ASH 2019: Kiialainen A, Niggli M, Kempton C, et al.

Emicizumab Prophylaxis in Hemophilia A with Inhibitors

Oldenburg J, Mahlangu JN, Kim B, et al. N Engl J Med 2017;377:809–818.

Timing of treated spontaneous bleeding in persons with haemophilia A (PwHA) with inhibitors in the HAVEN 1 study

Poster presentation P191, EAHAD 2020: Kruse-Jarres R, Trzaskoma B, Raimundo K, et al.

Summary of thromboembolic (TE) or thrombotic microangiopathy (TMA) events in persons taking emicizumab

Oral presentation P131, EAHAD 2020: Lee L, Moreno K, Kuebler P, et al.

The effect of emicizumab prophylaxis on health-related outcomes in persons with haemophilia A with inhibitors: HAVEN 1 study

Oldenburg J, Mahlangu J.N, Bujan W, et al. Haemophilia 2019;25:33-44.

Emicizumab, a humanized bispecific antibody to coagulation factors IXa and X with a factor VIIIa-cofactor activity

Kitazawa T, Shima M. Int J Hematol 2020;111(1):20-30.

Population pharmacokinetic analysis and exploratory exposure-bleeding rate relationship of emicizumab in adult and pediatric persons with hemophilia A

Retout S, Schmitt C, Petry C, et al. Clin Pharmacokinet 2020; DOI: 10.1007/s40262-020-00904-z.

Emicizumab treatment and monitoring in a paediatric cohort: real-world data

Barg AA, Livnat T, Budnik I, et al. Br J Haematol 2020; DOI: 10.1111/bjh.16964.

Pharmacokinetics and Coagulation Biomarkers in Persons with Hemophilia A (PwHA) and FVIII Inhibitors Receiving Emicizumab in the Phase IIIb STASEY Study

Poster presentation PB092, ISTH 2019: Kiialainen A, Petry C, Jimenez-Yuste V, et al.

Estimating the risk of myocardial infarction in persons with hemophiliaA using a machine-learning approach with US claims data

Poster presentation P-1133, ASH 2019: Faghmous I, Flores C, Sarouei K, et al.

A multicenter, open-label, phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors

Young G, Liesner R, Chang TY, et al. Blood. 2019; https://doi.org/10.1182/blood.2019001869.

Factor VIII use in the treatment of breakthrough bleeds in hemophilia A patients without inhibitors on emicizumab prophylaxis: the phase 3 HAVEN 3 study experience

Poster presentation P-2395, ASH 2019: Callaghan M, Trzaskoma B, Ko RH, et al.

Second interim analysis results from the STASEY trial: A single-arm, multicenter, open-label, phase III clinical trial to evaluate the safety and tolerability of emicizumab prophylaxis in people with hemophilia A (PwHA) with FVIII inhibitors

Poster presentation PB0958, ISTH 2020: Jiménez-Yuste V, Klamroth R, Castaman G, et al.

Emicizumab prophylaxis in paediatric persons with haemophilia A (PWHA) with inhibitors: Impact on health-related outcomes and caregiver burden in the HAVEN 2 study

Oral presentation OR10, EAHAD 2018: Mancuso ME, Mahlangu J, Sidonio RF, et al.

Emicizumab, A Bispecific Antibody to Factors IX/IXa and X/Xa, Does Not Interfere with Antithrombin or TFPI Activity In Vitro

Noguchi-Sasaki M, Soeda T, Ueyama A, et al. TH Open 2018;2:e96-e103.

Exposure–response modeling of emicizumab for the prophylaxis of bleeding in haemophilia A patients with and without inhibitors against factor VIII

Poster presentation PB0325, ISTH 2019: Jonsson F, Schmitt C, Petry C, et al.

Management of comorbidities in haemophilia

Kempton CL, Makris M, and Holme PA. Haemophilia 2020; DOI: 10.1111/hae.14013.

Emicizumab Prophylaxis in Patients Who Have Hemophilia A without Inhibitors

Mahlangu J, Oldenburg J, Paz-Priel I, et al. N Engl J Med 2018;379:811-822.

Adverse event reporting in haemophilia A patients with and without Inhibitors treated with emicizumab: A report from the Co- morbidity Working Party of the UK Haemophilia Centres Doctors´ Organisation (UKHCDO)

Wall C, Hall G, Collins P, et al. Res Pract Thromb Haemost 2020;4 Supplement 1:484.

Emicizumab prophylaxis improves long-term Physical Health scores in persons with haemophilia A (PwHA) with or without inhibitors: update from the HAVEN 3 and HAVEN 4 studies.

Poster presentation PB0698, ISTH 2019: Skinner M, Negrier C, Paz-Priel I, et al.

Emicizumab-mediated haemostatic function in patients with haemophilia A is down-regulated by activated protein C through inactivation of activated factor V

Yada K, Nogami K, Shinozawa K, et al. Br J Haematol 2018;183:257-266.

Pharmacokinetics and biomarkers in persons with haemophilia A (PwHA) receiving emicizumab every 2 or 4 weeks

Poster presentation P021, EAHAD 2019: Kiialainen A, Schmitt C, Adamkewicz JI, et al.

Safety analysis of rFVIIa with emicizumab dosing in congenital hemophilia A with inhibitors: Experience from the HAVEN clinical program

Levy G.G, Asikanius E, Kuebler P, et al. J Thromb Haemost 2019;17:1470-1477.

Efficacy, safety, and pharmacokinetics of emicizumab prophylaxis given every 4 weeks in people with haemophilia A (HAVEN 4): a multicentre, open-label, non-randomised phase 3 study

Pipe S.W, Shima M, Lehle M, et al. Lancet Haematol 2019;6:e295-e305.

Factor VIIIa-mimetic cofactor activity of a bispecific antibody to factors IX/IXa and X/Xa, emicizumab, depends on its ability to bridge the antigens

Kitazawa T, Esaki K, Tachibana T, et al. Thromb Haemost 2017;117:1348-1357.

Pharmacokinetics and biomarkers in persons with haemophilia A (PwHA) without FVIII inhibitors receiving emicizumab once weekly in the phase 3 HAVEN 3 study

Poster presentation P022, EAHAD 2019: Kiialainen A, Schmitt C, Oldenburg J, et al.

ATHN 7: A natural history cohort study of the safety, effectiveness, and practice of treatment for people with hemophilia - Demographics and preliminary results

Buckner T, Daoud N, Croteau SE, et al. Res Pract Thromb Haemost 2020;4 Supplement 1:513.

A contemporary framework for understanding mortality in people with congenital hemophilia A (PwcHA)

Poster presentation P23, NHF 2020: Pipe SW, Kruse-Jarres R, Mahlangu JN, et al.

Emicizumab treatment is efficacious and well tolerated long term in persons with haemophilia (PwHA) with or without FVIII inhibitors: pooled data from four HAVEN studies

Oral presentation OC 60.2, ISTH 2019: Callaghan M, Negrier C, Paz-Priel I, et al.

Timing of treated spontaneous bleeding in persons with haemophilia A (PwHA) with inhibitors in the HAVEN 3 study

Poster presentation PB0694, ISTH 2019: Mahlangu J, Oldenburg J, O'Connell N, et al.

Case report of a fatal rectal haemorrhage in a person with severe haemophilia A receiving emicizumab and high-dose bypassing agents in the HAVEN 1 study

Khoo L, Matthews S, Kershaw G, et al. Haemophilia 2020;DOI: 10.1111/hae.14110.

Identification and multidimensional optimization of an asymmetric bispecific IgG antibody mimicking the function of factor VIII cofactor activity

Sampei Z, Igawa T, Soeda T, et al. PLoS One 2013;8(2):e57479.

Relative and Absolute Bioavailability Study of Emicizumab to Bridge Drug Products and Subcutaneous Injection Sites in Healthy Volunteers

Kotani N, Yoneyama K, Kawakami N, et al. Clin Pharma Drug Dev 2019;8:702-712.

Real world efficacy of emicizumab in haemophilia A with inhibitors: A report from the UK National Haemophilia Database

Wall C, Xiang H, Palmer B, et al. Res Pract Thromb Haemost 2020;4 Supplement 1:462.

Activated coagulation time and hepcon protamine titration device to manage unfractionated heparin during cardiopulmonary bypass in a hemophilia A patient on emicizumab

Isaacs J, Welsby IJ, Schroder JN, et al. J Cardiothorac Vasc Anesth 2020; DOI: 10.1053/j.jvca.2020.08.058.

An analysis of fatalities in persons with congenital hemophilia A reported in the FDA Adverse Event Reporting System (FAERS) database

Poster presentation P25, NHF 2020: De Ford C, Kuebler P, Shang A, et al.

A Randomized, Multicenter, Open-label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus No Prophylaxis in Persons with Hemophilia A in the Asia-Pacific Region (HAVEN 5)

Poster presentation PB0957, ISTH 2020: Wang S, Zhao X, Wang X, et al.

Use of bypassing agents prior to and post bypassing agent dosing guidance during emicizumab prophylaxis: Analyses from the HAVEN 1 study

Poster presentation P-3688, ASH 2017: Callaghan MU, Negrier C, Young G, et al. 

ST-segment elevation myocardial infarction (STEMI) and pulmonary embolism in a hemophilia A patient receiving emicizumab and recombinant activated factor VII

Gundabolu K, Goldsweig A, Bhatt VR, et al. Haemophilia 2019;26:e5-e8.

Pharmacokinetics (PK), pharmacodynamics (PD) and PK/PD relationships of emicizumab in persons with haemophilia A (PwHA) with inhibitors from adolescent/adult (HAVEN 1) and paediatric (HAVEN 2) phase 3 studies

Oral presentation, EAHAD 2018: Schmitt C, Adamkewicz J, Xu J, et al.

Hemlibra treatment in pediatric hemophilia A patients – real world data of safety and uptake in the PedNet cohorts

Kenet G, Oldenburg J, Santagostino E, et al. Res Pract Thromb Haemost 2020;4 Supplement 1:459.

Immune tolerance induction in paediatric patients with haemophilia A and inhibitors receiving emicizumab prophylaxis

Batsuli G, Zimowski KL, Tickle K, et al. Haemophilia 2019;25:789-796.

A systematic review of mortality statistics and causes of death in people with congenital hemophilia A (PwcHA)

Poster presentation P32, NHF 2020: Hay CR, Nissen F, Pipe SW

A multicentre, open-label study of emicizumab given every 2 or 4 weeks in children with severe haemophilia A without inhibitors

Shima M, Nogami K, Nagami S, et al. Haemophilia 2019;25:979-987.

Central line-associated thrombus formation in an inhibitor negative severe hemophilia A patient on emicizumab

George N.B., Paschke A., Scott K. Blood 2019;134:Supplement 1.

Pharmacodynamic data and coagulation biomarkers in persons with hemophilia A (PWHA) with inhibitors: Results from the HAVEN 1 emicizumab (ACE910) phase 3 study

Oral presentation OC 47.1, ISTH 2017: Adamkewicz JI, Schmitt C, Calatzis A, et al.

Emicizumab pediatric experience in clinical studies and beyond

Oral Presentation, WFH 2020: Levy G

Second Interim Analysis Results from the STASEY Trial: A Single-arm, Multicenter, Open-label, Phase III Clinical Trial to Evaluate the Safety and Tolerability of EmicizumabProphylaxis in People with Hemophilia A (PwHA) with FVIII Inhibitors

Poster Presentation PB0958, ISTH 2020: Jiménez-Yuste V, Klamroth R, Castaman G, et al.

Impact of inhibitors on hemophilia A mortality in the United States

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

Inhibitor status of patients with Hemophilia A who transition to Emicizumab after Immune Tolerance Induction

Batsuli G, Greene A, Meeks S, et al. WFH 2020 Abstract #MED-FP-011.

Emicizumab and thrombosis: The story so far

Makris M, Iorio A, Lenting PJ. J Thromb Haemost 2019;17:1269-1272.

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;e27886.

Factor VIII-Mimetic Function of Humanized Bispecific Antibody in Hemophilia A

Shima M, Hanabusa H, Taki M, et al. N Engl J Med 2016;374:2044-53.

Immunogenicity of emicizumab in people with hemophilia A: results from the HAVEN 1–4 studies

Oral Presentation, ASH 2018; Paz-Priel I, Chang T, Asikanius E, et al.

Immune Tolerance Induction Using Low-dose Fc Fusion Recombinant Factor VIII on Emicizumab in Hemophilia A Patients with Inhibitors, Tokyo Protocol

Nagao A, Katayama H, Nishiyama A, et al. Res Pract Thromb Haemost 2019;3 Supplement 1:263-264.

Long-term safety and efficacy for up to >5 years in a phase 1/2 study in patients with severe hemophilia A

Poster Presentaton PB0950, ISTH 2020: Shima M, Nagao A, Taki M, et al.

Neutralizing antidrug antibody to emicizumab in a patient with severe hemophilia A with inhibitors: New case with detailed laboratory evaluation

Druzgal CH, Kizilocak H, Brown J, et al. J Thromb Haemost 2020; DOI # 10.1111/jth.14957.

Surgical experience from four phase III studies (HAVEN 1–4) of emicizumab in persons with haemophilia A (PwHA) with or without FVIII inhibitors

Oral presentation OC 60.1, ISTH 2019: Santagostino E, Oldenburg J, Chang T, et al.

Model of Short- and Long-Term Outcomes of Emicizumab Prophylaxis Treatment for Persons with Hemophilia A

Zhou ZY, Raimundo K, Patel AM, et al. J Manag Care Spec Pharm 2020;DOI: 10.18553/jmcp.2020.19406.

Characterization of neutralizing anti-emicizumab antibody developed in a hemophilia A patient

Valsecchi C, Gobbi M, Schiavone L, et al. Res Pract Thromb Haemost 2020; 4 Supplement 1:587.

Surgical experience from the phase III STASEY trial of emicizumab prophylaxis in persons with hemophiliaA (PwHA) with FVIII inhibitors: Data from the second interim analysis

Poster presentation PB0939, ISTH 2020: Castaman G, Kremer Hovinga JA, Schutgens R, et al.

Emicizumab prophylaxis in patients with haemophilia A with and without inhibitors

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

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;26:631–636.

Total knee replacement with and without emicizumab: a unique comparison of perioperative management

Evans MS, Davis C, Eyster ME. Blood Adv 2020;4:855-857.

Major surgery management in patients with haemophilia A and inhibitors on emicizumab prophylaxis without global coagulation monitoring

Biron-Andreani C, Diaz-Cau I, Ranc A, et al. Br J Haematol 2020:DOI: 10.1111/bjh.16512.

Management of perioperative hemostasis in a severe hemophilia A patient with inhibitors on emicizumab using global hemostasis assays

Kizilocak H, Yukhtman C.L, Marquez-Casas E, et al. Ther Adv Hematol 2019;DOI: 10.1177/2040620719860025.

Emicizumab use in major orthopedic surgery

Seaman C.D, Ragni M.V. Blood Adv 2019;3:1722-1724.

Rescue FVIII replacement to secure haemostasis in a patient with haemophilia A and inhibitors on emicizumab prophylaxis undergoing hip replacement

Santagostino E, Mancuso M.E, Novembrino C, et al. Haematologica 2019;104:e380-e382.

Economic impact model of delayed inhibitor development in patients with hemophilia A receiving emicizumab for the prevention of bleeding events

Patel AM, Corman SL, Chaplin S, et al. J Med Econ 2019:22:1328-1337.

Effects and Interferences of Emicizumab, a Humanised Bispecific Antibody Mimicking Activated Factor VIII Cofactor Function, on Coagulation Assays

Adamkewicz J.I, Chen D.C, Paz-Priel I. Thromb Haemost 2019;119(7):1084-1093.

Effects and interferences of emicizumab, a humanized bispecific antibody mimicking activated factor VIII cofactor function, on lupus anticoagulant assays

Adamkewicz JI, Kiialainen A, Paz-Priel I. Int J Lab Hematol 2019;DOI:10.1111/ijlh.13114.

Effect of emicizumab on global coagulation assays for plasma supplemented with apixaban or argatroban

Tripodi A, Chantarangul V, Padovan L, et al. J Thromb Thrombolysis 2020;49:413-419.

Comorbidities in aging persons with hemophilia A (PwHA)

Poster presentation P019, EAHAD 2019: Patel AM, Yang E, Raimundo K, Ko RH et al.

Global coagulation function assessed by rotational thromboelastometry predicts coagulation-steady state in individual hemophilia A patients receiving emicizumab prophylaxis

Yada K, Nogami K, Ogiwara K, et al. Int J Hematol 2019;110:419–430.

Is the comprehensive assessment tool of challenges in hemophilia (CATCH) appropriate to measure the impact of hemophilia and its treatment? First quantitative survey

Poster presentation P617382, HTRS 2019: Regnault A, Raimundo K, Patel AM, et al.

Modified clot waveform analysis to measure plasma coagulation potential in the presence of the anti-factor IXa/factor X bispecific antibody emicizumab

Nogami K, Matsumoto T, Tabuchi Y, et al. J Thromb Haemost 2018;16:1078–1088.

Routine measurements of factor VIII activity and inhibitor titer in the presence of emicizumab utilizing anti-idiotype monoclonal antibodies

Nogami K, Soeda T, Matsumoto T, et al. J Thromb Haemost 2018;16:1383-1390.

Comorbidities in aging persons with hemophilia A (PwHA)

Poster presentation P019, EAHAD 2019: Patel AM, Yang E, Raimundo K, Ko RH

Safety analysis of rFVIIa with emicizumab dosing in congenital hemophilia A with inhibitors: Experience from the HAVEN clinical program

Levy G.G, Asikanius E, Kuebler P, et al. J Thromb Haemost. 2019; 17:1470-1477.

Archived data reports on thrombotic microangiopathy, serious thrombotic events, and fatalities were last updated June 30, 2020. Please note that there will be no further updates of these archived materials.