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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.

Clinical trials
Back to Publications and Scientific Presentations
Safe and successful surgical outcomes in persons with hemophilia A with and without inhibitors treated with emicizumab: A large, single center, real-world experience

Castaman G, Linari S, Pieri L, et al. J Clin Med 2023;12(6):2317.

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A retrospective analysis of the American Thrombosis and Hemostasis Network dataset describing outcomes of dental extractions in persons with hemophilia

Messenger H, Kulkarni R, Recht M, et al. J Clin Med 2023;12(5):1839.

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Surgeries and procedures in people with haemophilia A on emicizumab prophylaxis: Analysis from the ATHN 7 Haemophilia Natural History Study

Buckner TW, Carpenter SL, Daoud N, et al. EAHAD 2023. PO166.

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Peri-operative hemostatic management of tooth extraction in patients with hemophilia A, with and without inhibitors, receiving emicizumab prophylaxis

Yagyuu T, Furukawa S, Zaizen M, et al. Haemophilia 2022. doi: 10.1111/hae.14667.

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Surgical outcomes in people with hemophilia A taking emicizumab prophylaxis: experience from the HAVEN 1-4 studies

Kruse-Jarres R, Peyvandi F, Oldenburg J, et al. Blood Adv. 2022. doi: 10.1182/bloodadvances.2022007458.

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Trends in prescribing practices for management of hemophilia: 1999-2021

Curtis R, Roberts JC, Crook N, et al. Haemophilia 2023;29(3): 761-769.

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Emicizumab prophylaxis in haemophilia A with inhibitors: Three years follow-up from the UK Haemophilia Centre Doctors' Organisation (UKHCDO)

Wall C, Xiang H, Palmer B, et al. Haemophilia 2023;29(3):743-752.

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Nonreplacement therapy for hemophilia in low-income countries: Experience from a prospective study in Ivory Coast

Lambert C, Meité N, Kouassi GK,  et al. Res Pract Thromb Haemost 2022;7(1):1000033.

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Assessment of the clinical perception, quality of life and satisfaction of patients with severe congenital haemophilia A without inhibitor after 1 year of emicizumab therapy

Oka G, Roussel-Robert V, Levivien C, et al. Haemophilia 2023;29(3):709-713.

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Early changes in bleeding perception and quality of life in children and adolescents receiving emicizumab prophylaxis for severe haemophilia A without inhibitor

Marro M, Petit AF, Falaise C, et al. Haemophilia 2023;29(1):397-400.

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A phase IV, multicentre, open-label study of emicizumab prophylaxis in people with haemophilia A with or without FVIII inhibitors undergoing minor surgical procedures

Escobar M, Dunn A, Quon D, et al. Haemophilia 2022. doi: 10.1111/hae.14574

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Switching to emicizumab: A prospective surveillance study in haemophilia A subjects with inhibitors

Dubé E, Merlen C, Bonnefoy A, et al. Haemophilia 2023 Jan;29(1):348-351.

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Patients on emicizumab prophylaxis with previously tolerized inhibitors: Is there a risk of inhibitors recurrence?

Hassan E, Lancashire J, Motwani J. Haemophilia 2022;28(2):e36-e38.

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Hemophilic pseudotumor of the maxillary sinus in an inhibitor-positive patient with hemophilia A receiving emicizumab: A case report

Kawahara F, Shirayama R, Ito T, et al. Int J Hematol 2022;115(6):906-912.

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Effectiveness of emicizumab under real-world conditions in patients of all ages with hemophilia A with and without FVIII inhibitors: Interim analysis of the non-interventional study EMIIL

Oldenburg J, Escuriola-Ettingshausen C, Fontana P, et al. ISTH 2023. OC 43.4.

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Emicizumab prophylaxis in people with hemophilia A aged ≥50 years with comorbidities: Experience from the ATHN 7 hemophilia natural history study

Buckner TW, Daoud N, Lee L, et al. ISTH 2023. PB0625.

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Emicizumab prophylaxis in severe haemophilia A without inhibitors: Outcome and indications. From the UK Haemophilia Centre Doctors' Organisation

Hay C, Wall C, Xiang H, et al. EAHAD 2023. PO106.

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Bleeding control improves after switching to emicizumab: Real world experience from 251 children in the PedNet Registry

Van Der Zwet K, de Kovel M, Motwani J, et al. EAHAD 2023. PO192.

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A real-world evidence analysis of the impact of switching from factor VIII to emicizumab prophylaxis in patients with hemophilia A without inhibitors

Escobar J, Bullano M, Mokdad AG, et al. Expert Rev Hematol 2023;16(6):467-474.

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Evaluation of the safety of emicizumab prophylaxis in people with haemophilia A: An updated summary of thrombotic events and thrombotic microangiopathies

Koparkar S, Barlera F, Nissen R, et al. EAHAD 2023. PO123.

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Emicizumab in children: Bleeding episodes and outcome before and after transition to emicizumab

Glonnegger H, Andresen F, Kapp F, et al. BMC Pediatr 2022;22:487.

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Switching to emicizumab: A prospective surveillance study in haemophilia A subjects with inhibitors

Dubé E, Merlen C, Bonnefoy A, et al. Haemophilia 2022. doi: 10.1111/hae.14685.

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Hemorrhagic and thrombotic adverse events associated with emicizumab and extended half-life factor VIII replacement drugs: EudraVigilance data of 2021

Abbattista M, Ciavarella A, Noone D, et al. J Thromb Haemost 2023;21(3):546-552.

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Real-world data on emicizumab prophylaxis in the Milan Cohort

Arcudi S, Gualtierotti R, Marino S, et al. Haemophilia 2022;28:e141-e144.

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Real-world experience of emicizumab prophylaxis in young children with hemophilia A: Retrospective data from China

Liu G, Huang K, Li G, et al. Front Pediatr 2022;10:992267.

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Emicizumab prophylaxis in people with hemophilia A aged ≥50 years with comorbidities: Experience from the ATHN 7 hemophilia natural history study

Buckner TW, Daoud N, Lee L, et al. ISTH 2023. PB0625.

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Safety of FEIBA and emicizumab (SAFE): Dose escalation study evaluating the safety of in vivo administration of activated prothrombin complex concentrate in haemophilia A patients on emicizumab

Kizilocak H, Marquez-Casas E, Malvar J, et al. Haemophilia 2023;29(1):100-105.

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A model-based framework to inform the dose selection and study design of emicizumab for pediatric patients with hemophilia A

Yoneyama K, Schmitt C, Chang T, et al. J Clin Pharmacol 2022;62(2):232-244.

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Impact of switching prophylaxis treatment from factor VIII to emicizumab in hemophilia A patients without inhibitors

Escobar M, Agrawal N, Chatterjee S, et al. J Med Econ 2023;26(1):574-580.

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Real-world safety of emicizumab: Interim analysis of the European Haemophilia Safety Surveillance (EUHASS) Database

Nissen F, Jiang Y, Hiew HJ, et al. ASH 2022. Oral presentation 192.

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Emicizumab and females with hemophilia A: Case series from ATHN 7

Recht M, Daoud N, Lee L, et al. ASH 2022. Poster 1192.

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Emicizumab prophylaxis in persons with hemophilia A, aged ≥50 years, with comorbidities – pooled data from four phase III studies (HAVEN 1, 3, and 4, and STASEY)

Jiménez-Yuste V, Oldenburg J, Tzeng E, et al. ASH 2021; Poster presentation 2103.

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Exposure-bleeding count modeling of emicizumab for the prophylaxis of bleeding in persons with hemophilia A with/without inhibitors against factor VIII

Jonsson F, Schmitt C, Petry C, et al. Clin Pharmacokinet 2021;60:931-941.

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Real-world outcomes of emicizumab in hemophilia A with or without FVIII inhibitors from the Canadian Hemophilia Bleeding Disorder Registry

Poon MC, Lee A, Germini F, et al. ASH 2022. Poster 3796.

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Estimating the risk of thrombotic events in people with congenital hemophilia A using US claims data.

Faghmous I, Nissen F, Kuebler P, et al. J Comp Eff Res. 2021;10(18):1323-1336.

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Emicizumab dose capping in obese patients with haemophilia A: Early outcomes at a single centre

Krumb E, Lambert C, Hermans C. Haemophilia 2022;28(6):e245-e247.

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Evaluation of the safety of emicizumab prophylaxis in persons with hemophilia A: An updated summary of thrombotic events and thrombotic microangiopathies

Howard M, McKinley D, Sanabria F, et al. ASH 2021; Poster presentation 3186.

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Patient-reported outcomes among adults with hemophilia A on prophylactic treatment

Chupka J, Palmer MD, Lin CW, et al. ASH 2022. Poster 4894.

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Early real-world experience with emicizumab and concomitant factor VIII replacement products in adult males with hemophilia A without inhibitors

Cafuir L, Estrin A, Chen E, et al. J Med Econ 2022;25:984-992.

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A real-world study comparing pre-post billed annualized bleed rates and total cost of care among non-inhibitor patients with hemophilia A switching from FVIII prophylaxis to emicizumab

Batt K, Schultz BG, Caicedo J, et al. Curr Med Res Opin 2022;38:1685-1693.

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Untreated bleeds in people with hemophilia A in a noninterventional study and intrapatient comparison after initiating emicizumab in HAVEN 1-3

Callaghan MU, Asikanius E, Lehle M, et al. Res Pract Thromb Haemost 2022;6:e12782.

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Cost-effectiveness of emicizumab for prophylaxis in people with severe hemophilia A without inhibitors

Decker-Palmer M, Lin CW, Wilson, et al. Blood 2022;140(Suppl 1):13041-13042.

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Emicizumab prophylaxis in hemophilia A with inhibitors

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

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Emicizumab in obese adults with hemophilia A – pooled data from three Phase III studies (HAVEN 1, 3 and 4)

Recht M, Mahlangu J, Minhas M, et al.  ISTH 2021; Poster presentation PB0495.

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Association between physical activity and bleeding events in people with hemophilia A receiving emicizumab prophylaxis: Interim analysis of the TSUBASA study.

Fujii T, Amano K, Sawada A, et al. ISTH 2023. PB0641.

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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 Jul 29. doi: 10.1111/hae.14642.

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Emicizumab prophylaxis improves acute and chronic pain-related outcomes in people with haemophilia A (PwHA): Post hoc analysis of 470 patients from HAVEN 1, 3, 4 and STASEY

Hermans C, Skinner MW, Gentile B, et al. EAHAD 2023. PO107.

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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.

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

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

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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;59(12):1611-1625.

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Breakthrough bleeding episodes in pediatric severe hemophilia A patients with and without inhibitors receiving emicizumab prophylaxis: A single-center retrospective review

Hassan E, Motwani J. Pediatr Hematol Oncol 2022:1-9.

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Pharmacokinetics and pharmacodynamics of emicizumab in persons with hemophilia A with factor VIII inhibitors: HAVEN 1 study

Schmitt C, Adamkewicz JI, Xu J, et al. Thromb Haemost. 2020;121:351-360.

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

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

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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;27:253-260.

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TSUBASA: A prospective study evaluating the association between physical activity and bleeding events in people with hemophilia A during emicizumab prophylaxis

Fujii T, Amano K, Sawada A, et al. WFH 2022; Poster presentation LR-01.01

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

Kiialainen A, Niggli M, Kempton C, et al. ASH 2019; Oral presentation.

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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.

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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

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

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Management of comorbidities in haemophilia

Kempton CL, Makris M, and Holme PA. Haemophilia 2020;27 Suppl 3:37-45.

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Pharmacokinetics and coagulation biomarkers in persons with hemophilia A (PwHA) and FVIII inhibitors receiving emicizumab in the phase IIIb STASEY study

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

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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;134(24):2127-2138.

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The lived experience of a novel disruptive therapy in a group of men and boys with haemophilia A with inhibitors: Emi & Me

Fletcher S, Jenner K, Holland M, Khair K. Health Expect 2022;25(1):443-454.

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Health-related quality of life and caregiver burden of emicizumab in children with haemophilia A and factor VIII inhibitors - results from the HAVEN 2 study

Mancuso ME, Mahlangu J, Sidionio R, et al. Haemophilia 2020;26:1009-1018.

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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;26(6):e340-e342.

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

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

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

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

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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.

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

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

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

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

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Efficacy/safety in children on 2/4-weekly emicizumab prophylaxis: 52-week outcomes in HAVEN 2

Young G, Sidonio R, Oldenburg J, et al. ASPHO 2022; Poster presentation P010.

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Emicizumab in previously untreated and minimally treated infants

Hassan E, Lancashire J, Motwani J. Haemophilia 2022;28(2):e61-e63.

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Surgical experience from the phase III STASEY trial of emicizumab prophylaxis in persons with hemophilia A with FVIII inhibitors: Final analysis

Castaman G, Wndgya J, Alzahrani H, et al. ASH 2021; Oral presentation 344.

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Port removal in patients receiving emicizumab prophylaxis: A single centre experience and review of the literature

Swan D, Paran S, Nolan B. Haemophilia 2022;28(1);42-45.

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Management of people with haemophilia A undergoing surgery while receiving emicizumab prophylaxis: real-world experience from a large comprehensive treatment centre in the US

Lewandowska M, Randall N, Bakeer N, et al. Haemophilia 2021:27:90-99.

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Emicizumab in pediatric hemophilia: Bleeding and surgical outcomes from a single-center retrospective study

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

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Emicizumab prophylaxis in patients with haemophilia A with and without inhibitors

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

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Efficacy of emicizumab is maintained throughout dosing intervals for bleed prophylaxis

Pipe SW, Trzaskoma B, Minhas M, et al. Res Pract Thromb Haemost 2023;7(2):100077.

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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.

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

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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.

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Mortality in congenital hemophilia A - a systematic literature review

Hay CRM, Nissen F, and Pipe SW. J Thromb Haemost 2020;19 (Suppl 1):6-20.

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Real-world safety of emicizumab: the first interim analysis of the European Haemophilia Safety Surveillance (EUHASS) Database

Shang A, Bienz NS, Gadiraju R, et al.  ASH 2020; Poster presentation 2685.

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

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

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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.

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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.

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Application of a hemophilia mortality framework to the Emicizumab Global Safety Database

Peyvandi F, Mahlangu JN, Pipe SW, et al.  J Thromb Haemost. 2020;19 (Suppl 1):32-41.

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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; 19 (Suppl 1):21-31.

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

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Emicizumab and females with hemophilia A: Case series from ATHN 7

Recht M, Daoud N, Lee L, et al. ASH 2022. Poster 1192.

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

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

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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.

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

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

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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.  NHF 2020; Poster presentation P25.

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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.

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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.

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Real-world persistence with and adherence to emicizumab prophylaxis in persons with hemophilia A: a secondary claims database analysis

Mahajerin A, Khairnar R, Meyer CS, et al.  ASH 2020; Poster presentation 866.

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Annualized bleed rates in severe Hemophilia A after switch to emicizumab based on CHESS II data, including an oversample of emicizumab patients

Mancuso ME, Nissen F, Zhang H, et al. ISTH 2022; Poster presentation 0674.

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First report of emicizumab use in a female patient with severe hemophilia A

Verstraete G, Lambert C, Hermans C. Blood Adv 2020;4:2950-2952.

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

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

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Emicizumab dose up-titration in case of suboptimal bleeding control in people with haemophilia A

Schmitt C, Mancuso ME, Chang T, et al. Haemophilia 2022. doi: 10.1111/hae.14679.

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

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

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

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

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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.

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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

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

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Changes in healthcare resource use and costs after emicizumab initiation for hemophilia A

Mahajerin A, Khairnar R, Meyer CS, et al. AMCP Nexus 2020; Poster presentation.

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Long-term outcomes with emicizumab prophylaxis for hemophilia A with/without FVIII inhibitors from the HAVEN 1-4 studies

Callaghan MU, Négrier C, Paz-Priel I, et al. Blood 2021;137:2231-2242.

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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.

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Emicizumab prophylaxis in people with severe haemophilia A without inhibitors: outcomes from the UK Haemophilia Centre Doctors’ Organisation

Wall C, Xiang H, Palmer B, et al. ISTH 2022. Oral presentation 30.1.

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Real-world study of rurioctocog alfa pegol and emicizumab in US clinical practice among patients with hemophilia A

Sun SX, Frick A, Balasa V, et al. Expert Rev Hematol 2022;15:943-950.

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Channeling effects in the prescription of new therapies: the case of emicizumab for hemophilia A

Mahajerin A, Faghmous I, Kuebler P, et al. J Comp Eff Res 2022;11(10):717-728.

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Efficacy of emicizumab is maintained throughout dosing intervals for bleed prophylaxis

Pipe SW, Trzaskoma B, Minhas M, et al. Res Pract Thromb Haemost 2023;7(2):100077.

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

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

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

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

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

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

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

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

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Modelling future usage and cost of factor and emicizumab to treat haemophilia A for the US Western States Region IX haemophilia treatment centres

Stonebraker JS and Ducore JM. Haemophilia 2020;27:e22-e29.

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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

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

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Hemophilia natural history study (ATHN 7): Safety of current therapies for people with hemophilia A or B

Buckner TW, Carpenter SL, Daoud N, et al. Blood 2021;138:2015.

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Long-term outcomes with emicizumab prophylaxis for severe haemophilia A without FVIII inhibitors: Efficacy and safety analyses from HAVEN 3 & 4

Mahlangu J, Jiménez-Yuste V, Ventriglia G, et al. EAHAD 2023. PO135.

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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.

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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.

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Impact of inhibitors on hemophilia A mortality in the United States

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

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Emicizumab and thrombosis: The story so far

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

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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;26:1109-1120.

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Prophylactic emicizumab for hemophilia A in the Asia-Pacific region: A randomized study (HAVEN 5)

Yang R, Wang S, Wang X, et al. Res Pract Thromb Haemost 2022;6(2):e12670.

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Emicizumab outcomes in hemophilia A using real-world data from the Canadian Hemophilia Bleeding Disorder Registry

Poon M-C, Lee A, Germini F, et al. ASH 2021; Oral presentation 347.

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Emicizumab in people with moderate or mild haemophilia A (HAVEN 6): A multicentre, open-label, single-arm, phase 3 study

Négrier C, Mahlangu J, Lehle M, et al. Lancet Haematol 2023;10(3):e168-e177.

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Emicizumab dose up-titration in case of suboptimal bleeding control in people with haemophilia A

Schmitt C, Mancuso ME, Chang T, et al. Haemophilia 2022. doi: 10.1111/hae.14679.

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Low immunogenicity of emicizumab in persons with haemophilia A

Schmitt C, Emrich T, Chebon S, et al. Haemophilia. 2021;27:984-992.

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Detailed analysis of anti-emicizumab antibody decreasing drug efficacy, using plasma samples from a patient with hemophilia A

Kaneda M, Kawasaki R, Matsumoto N, et al.  J Thromb Haemost. 2021;19(12):2938-2946.

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Emicizumab prophylaxis for the treatment of people with moderate or mild hemophilia A without factor VIII inhibitors: Results from the primary analysis of the HAVEN 6 study

Hermans C, Negrier C, Lehle M, et al. ISTH 2022; Oral presentation 30.5.

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Non-inhibitory antibodies inducing increased emicizumab clearance in a severe haemophilia A inhibitor patient

Harroche A, Sefiane T, Desvages M, et al. Haematologica 2021;106:2287-2290.

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Low immunogenicity of emicizumab in persons with haemophilia A

Schmitt C, Emrich T, Asikanius E, et al. EAHAD 2021; Poster presentation ABS141.

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Immune tolerance induction using low-dose Fc fusion recombinant factor FVIII 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.

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

Harkins Druzgal CH, Kizilocak H, Brown J, et al. J Thromb Haemost 2020;18:2205-2208.

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Emicizumab prophylaxis in persons with mild or moderate hemophilia A: Results from the interim analysis of the HAVEN 6 study

Négrier C, Mahlangu J, Lehle M, et al. ASH 2021; Oral presentation 343.

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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.

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Characterization of the neutralizing anti-emicizumab antibody in a patient with hemophilia A and inhibitor

Valsecchi C, Gobbi M, Beeg M, et al. J Thromb Haemost 2021;19:711-718.

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Emicizumab prophylaxis for the treatment of infants with severe hemophilia A without factor VIII inhibitors: Results from the interim analysis of the HAVEN 7 study

Pipe SW, Collins P, Dhalluin C, et al. ASH 2022; Oral presentation 187.

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

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

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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.

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Safety and efficacy of long-term emicizumab prophylaxis in hemophilia A with factor VIII inhibitors: A phase 3b, multicenter, single-arm study (STASEY)

Jiménez-Yuste V, Peyvandi F, Klamroth R, et al. Res Pract Thromb Haemost 2022;6:e12837.

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

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

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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.

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Final analysis of the STASEY trial: A single-arm, multicenter, open-label, phase III clinical trial evaluating the safety and tolerability of emicizumab prophylaxis in persons with hemophilia A with factor VIII inhibitors

Jiménez-Yuste V, Peyvandi F, Klamroth R, et al. ISTH 2021; Poster presentation PB0521.

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

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

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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.

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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

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

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Emicizumab in pediatric hemophilia: Bleeding and surgical outcomes from a single-center retrospective study

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

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Clot waveform analysis for perioperative hemostatic monitoring of arthroscopic synovectomy in a pediatric patient with hemophilia A and inhibitor receiving emicizumab prophylaxis

Mizumachi K, Tsumura K, Nakajima Y, et al. Int J Hematol 2021;113:930-935.

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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 2021;35(11):3299-3302.

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Consecutive complex percutaneous coronary interventions using emicizumab and recombinant activated factor VII in a patient with severe haemophilia A and high-titre inhibitor

Chen Y, Chen W, Tseng M, et al. Haemophilia 2021;27(3):e385-e388.

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

Shima M, Nagao A, Taki M, et al.  ISTH 2020; Poster presentation PB0950.

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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.

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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:e28942.

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Long-term safety and efficacy of emicizumab for up to 5.8 years and patients' perceptions of symptoms and daily life: a phase 1/2 study in patients with severe haemophilia A

Shima M, Nagao A, Taki M, et al. Haemophilia 2021;27:81–89.

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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;189:e100-e103.

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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;27:253-260.

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Real-world cost estimates of initiating emicizumab in US patients with haemophilia A

Samelson-Jones BJ, Guelcher C, Kuhn J, et al.  Haemophilia. 2021;00:1-8.

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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.

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

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

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

Kizilocak H, Yukhtman CL, Marquez-Casas E, et al. Ther Adv Hematol 2019;10:2040620719860025.

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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.

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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.

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Emicizumab use in major orthopedic surgery

Seaman CD, Ragni MV. Blood Adv 2019;3:1722-1724.

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Hemophilia Natural History Study (ATHN 7): Baseline characteristics, adverse events, and self-reported health status of individuals with hemophilia A and B

Buckner TW, Carpenter SL, Croteau SE, et al. Blood 2020;136 (Supplement 1):2-3.

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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.

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Emicizumab prophylaxis in patients with haemophilia A with and without inhibitors

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

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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.

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Safety of FEIBA and emicizumab (SAFE): Dose escalation study evaluating the safety of in vivo administration of activated prothrombin complex concentrate in haemophilia A patients on emicizumab

Kizilocak H, Marquez-Casas E, Malvar J, et al. Haemophilia 2022. doi: 10.1111/hae.14684.

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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.

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Comorbidities in aging persons with hemophilia A (PwHA)

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

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Emicizumab pediatric experience in clinical studies and beyond

Levy GG. WFH 2020: Oral presentation.

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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;42:e71-e75.

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Effects and interference of emicizumab, a humanised bispecific antibody mimicking activated factor VIII cofactor function, on coagulation assays

Adamkewicz JI, Chen DC, Paz-Priel I. Thromb Haemost 2019;119(7):1084-1093.

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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.

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Real-world persistence with and adherence to emicizumab prophylaxis in people with hemophilia A –Results from two secondary claims database analyses

Khairnar R, Meyer C, Schuldt R, et al. WFH 2022. Oral presentation 1122212.

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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.

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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.

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Comorbidities in aging persons with hemophilia A (PwHA)

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

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

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

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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.

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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.

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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.

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