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The Emicizumab Medical Website now redirects users here. This Medical and Scientific Information website provides the latest information related to Genentech products and the diseases they treat on a single platform. The content from the previous website has been updated based on relevant topics of interest to the hemophilia A community, and has been redesigned and reformatted to align with this new platform.
Key Topics of Interest
The information in this section may include content beyond what is in the FDA-approved label. Because the FDA has not approved such content, no conclusions regarding safety or efficacy may be made. Providing this information should not be construed as recommendation for use of a Genentech product for unapproved uses. For FDA-approved products please consult the product’s full prescribing information for a complete discussion of risks and benefits of the product(s) for its approved indication(s). The list of publications and congress materials is not an exhaustive list of published materials on the product. The list of references by data topics is selected per evidence-based medicine criteria.
Registrational Trials
Emicizumab Prophylaxis in Hemophilia A with Inhibitors
Oldenburg J, Mahlangu JM, Kim B, et al. N Engl J Med. 2017;377:809-818.
A Multicenter, Open-Label, Phase 3 Study of Emicizumab Prophylaxis in Children with Hemophilia A with Inhibitors
Young G, Liesner R, Chang T, et al. Blood. 2019;134:2127-2138.
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.
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 N, Lehle M, et al. Lancet Haematol. 2019;6:e295-e305.
Long-Term Outcomes
Long-Term Outcomes With Emicizumab in Hemophilia A Without Inhibitors: Results from the HAVEN 3 and 4 Studies
Mahlangu J, Jiménez-Yuste V, Ventriglia G, et al. Res Pract Thromb Haemost. 2024;8:102364.
Long-Term Outcomes with Emicizumab Prophylaxis for Hemophilia A with or Without FVIII Inhibitors from the HAVEN 1-4 Studies
Callaghan MU, Negrier C, Paz-Priel I, et al. Blood. 2021;137:2231-2242.
Long-Term Outcomes with Emicizumab Prophylaxis for Severe Haemophilia A Without FVIII Inhibitors: Safety and Efficacy Analyses from HAVEN 3 & 4
Mahlangu J, Jiménez-Yuste V, Ventriglia G, et al. Presented at: 16th Congress of the European Association for Haemophilia and Allied Disorders; February 7-10, 2023; Manchester, England. Poster PO135.
Thrombotic Microangiopathy/Thrombotic Events
Post‑Marketing Surveillance of the Safety and Effectiveness of Emicizumab in Japanese Patients with Congenital Hemophilia A with Inhibitors
Shima M, Fukutake K, Matsumoto M, et al. Presented at: 66th Congress of the American Society of Hematology; December 7-10, 2024; San Diego, California. Poster #2583.
Emicizumab Prophylaxis in People with Haemophilia A: Summary of 10 Years of Safety Data on Thromboembolic Events and Thrombotic Microangiopathy
Sarouei K, Barlera S, Polito L, et al. Presented at: 17th Congress of the European Association for Haemophilia and Allied Disorders; February 6-9, 2024; Frankfurt, Germany. Poster #PO126.
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. Presented at: 16th Congress of the European Association for Haemophilia and Allied Disorders; February 7-10, 2023; Manchester, England. Poster #PO123.
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;10:1323-1336.
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:e340-e342.
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.
Immunogenicity
Characterization of Anti-Emicizumab Antibodies Using Repository Samples Obtained in Clinical Studies of Emicizumab Conducted in Japan
Matsumoto N, Abe H, Kawasaki R, et al. TH Open. 2023;7:e241-e243.
Emicizumab Dose Up-Titration in Case of Suboptimal Bleeding Control in People with Haemophilia A
Schmitt C, Mancuso ME, Chang T, et al. Haemophilia. 2023;29:90-99.
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;19:2938-2946.
Low Immunogenicity of Emicizumab in Persons with Haemophilia A
Schmitt C, Emrich T, Chebon S, et al. Haemophilia. 2021;27:984-992.
Infants
Emicizumab Prophylaxis in Infants with Hemophilia A (HAVEN 7): Primary Analysis of a Phase 3b, Open-Label Trial
Pipe SW, Collins P, Dhalluin C, et al. Blood. 2024;143:1355-1364.
FVIII Exposure Analysis from the HAVEN 7 Study of Emicizumab in Infants with Severe Hemophilia A
Young G, Eakle K, Lim E, et al. Presented at: 66th Congress of the American Society of Hematology; December 7-10, 2024; San Diego, California; Poster #2589.
Bleed Patterns in Infants, from Birth to 12 Months of Age, with Hemophilia A Treated with Emicizumab: Exploratory Analysis of the HAVEN 7 Study
Carpenter SL, Rosenfelt D, Tzeng E, et al. Presented at: 66th Congress of the American Society of Hematology; December 7-10, 2024; San Diego, California; Poster #1214.
Emicizumab Prophylaxis in Infants with Severe Hemophilia A Without Factor VIII Inhibitors: Results from the Primary Analysis of the HAVEN 7 Study
Pipe S, Collins P, Dhalluin C, et al. Presented at: 65th Congress of the American Society of Hematology; December 9-12, 2023; San Diego, California. Oral #505.
Pharmacodynamic Biomarkers in Infants with Hemophilia A Receiving Emicizumab in HAVEN 7
Kiialainen A, Pipe SW, Fijnvandraat K, et al. Presented at: 65th Congress of the American Society of Hematology; December 9-12, 2023; San Diego, California. Oral #P1238.
Emicizumab Prophylaxis for the Treatment of Infants with Severe Haemophilia A Without Factor VIII Inhibitors: Results from the Interim Analysis of the HAVEN 7 Study on Behalf of the HAVEN 7 Study Investigators
Pipe S, Collins P, Dhalluin C, et al. Presented at: 16th Congress of the European Association for Haemophilia and Allied Disorders; February 7-10, 2023; Manchester, England.
Bone Health and Physical Activity
Association of Physical Activity with Bleeding Events and Safety in Patients with Haemophilia A Starting Emicizumab Prophylaxis: An Interim Analysis of the TSUBASA Study
Nogami K, Fujii T, Sawada A, et al. Int J Hematol. 2024;119:14-23.
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 CL, et al. Haemophilia. 2022;28:1033-1043.
TSUBASA Study: A Prospective Study Evaluating the Association Between Physical Activity and Bleeding Events, Quality of Daily Life, and Safety in People with Hemophilia A Without FVIII Inhibitors
Nogami K, Fujii T, Sawada A, et al. Presented at: 66th Congress of the American Society of Hematology; December 7-10, 2024; San Diego, California. Poster #1211.
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. Presented at: 31st Congress of the International Society of Thrombosis and Haemostasis; June 24-28, 2023; Montreal, Canada. Poster #PB0641.
Surgeries and Procedures
Surgical Experience from the STASEY Study of Emicizumab Prophylaxis in People with Hemophilia A with Factor VIII Inhibitors
Castaman G, Peyvandi F, Kremer Hovinga JA, et al. TH Open. 2024;8:e42-e54.
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;6:6140-6150.
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;28:e105-e108.
Mild/Moderate Hemophilia A
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:e168-e177.
Factor VIII Use in the Treatment of Breakthrough Bleeds in People with Moderate or Mild Hemophilia A Without Factor VIII Inhibitors Receiving Emicizumab Prophylaxis: The Phase III HAVEN 6 Experience
Jiménez-Yuste V, Lim E, Mistry K, et al. Presented at: 65th Congress of the American Society of Hematology; December 9-12, 2023; San Diego, California. Poster #P3999.
Comorbidities
Emicizumab Is Efficacious in People with Hemophilia A with Comorbidities Aged ≥50 Years: Analysis of 4 Phase III Trials
Jiménez-Yuste V, Oldenburg J, Tzeng E, et al. Res Pract Thromb Haemost. 2024;8:102405.
Emicizumab in People with Moderate or Mild Haemophilia Aged ≥40 Years, with and Without Comorbidities
Jiménez-Yuste V, Tzeng E, Lim E, et al. Presented at: 17th Congress of the European Association for Haemophilia and Allied Disorders; February 6-9, 2024; Frankfurt, Germany. Poster PO122.
Female Patients
Emicizumab Use in Females with Moderate or Mild Hemophilia A Without Factor VIII Inhibitors Who Warrant Prophylaxis
Hermans C, Ventriglia G, Obaji S, et al. Res Pract Thromb Haemost. 2023;7:102239.
Mortality
Mortality in Congenital Hemophilia A - A Systematic Literature Review
Hay CRM, Nissen F, Pipe SW. J Thromb Haemost. 2021;19 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;19 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;19 Suppl 1:32-41.
Real-World Evidence
Journal articles describing efficacy, safety, or surgical outcomes related to emicizumab in ≥50 patients in the real-world setting.
Evaluation of Safety and Efficacy of Emicizumab Prophylaxis in Egyptian Pediatric Patients with Hemophilia A: Single Center Cross Sectional Study
Hassan T, Zakaria M, Fathy M, et al. Turk J Haematol. 2024 Aug 22. doi: 10.4274/tjh.galenos.2024.2024.0220.
Bleeding Control Improves After Switching to Emicizumab: Real-World Experience of 177 Children in the PedNet Registry
van der Zwet K, de Kovel M, Motwani J, et al. Haemophilia. 2024;30:685-692.
Intraindividual Bleeding Outcomes in Patients with Hemophilia A on Emicizumab Prophylaxis in Australia
Ramanan R, Parikh S, Aung LL, et al. Blood Vessels Thromb Hemost. 2024;1:100005.
Comparison of Thrombotic Adverse Events in Patients Treated with Factor VIII Products and Emicizumab Using the 2018-2022 US Food and Drug Administration Adverse Event Reporting System Data
Cho H, Yoo KY, Shin JY, et al. J Thromb Haemost. 2024;22:1640-1648.
Factor VIII Inhibitors in Hemophilia A Treated with Emicizumab: Longitudinal Follow-Up of Outcomes
Levy-Mendelovich S, Atia N, Budnik I, et al. Res Pract Thromb Haemost. 2023;7:100278.
Emicizumab Prophylaxis in Haemophilia A with Inhibitors: Three Years Follow-Up from the UK Haemophilia Centre Doctors' Organisation (UKHCDO)
Wall C, Ziang H, Palmer B, et al. Haemophilia. 2023;29:743-752.
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;21:546-552.
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;39:418-426.
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.
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;10:4303.
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.
Emicizumab Prophylaxis: Prospective Longitudinal Real-World Follow-Up and Monitoring
Barg AA, Budnik I, Avishai E, et al. Haemophilia. 2021;27:383-391.
Real-World Use of Emicizumab in Patients with Haemophilia A: Bleeding Outcomes and Surgical Procedures
McCary I, Guelcher C, Kohn J, et al. Haemophilia. 2020;26:631-636.
Congress presentations describing efficacy, safety, or surgical outcomes related to emicizumab in ≥50 patients in the real-world setting.
Real-World Experience with Emicizumab for Hemophilia A from the Physician Perspective Based on Survey Data
Recht M, Salehi H, Blazos C, et al. Presented at: 66th Congress of the American Society of Hematology; December 7-10, 2024; San Diego, California. Poster #5078.
Impact of Emicizumab on Mental Liberation in Adult Patients with Severe and Moderate Factor VIIII Deficiency
Hermans C, Lambert C, Lobet S, et al. Presented at: 66th Congress of the American Society of Hematology; December 7-10, 2024; San Diego, California. Poster #1205.
Unmet Needs, Factor Consumption, and Healthcare Resource Use Among People with Hemophilia A: Real-World Analysis of the Adelphi Hemophilia Disease Specific Programme™
Malec L, Matino D, Wynne-Cattanach K, et al. Presented at: 66th Congress of the American Society of Hematology; December 7-10, 2024; San Diego, California. Poster #3973.
Real-World Bleeding Rates on Emicizumab Using Digital Treatment Diary data
Brands MR, Taal L, Driessens MHE, et al. Presented at: The 32nd Congress of the International Society on Thrombosis and Haemostasis; June 22-26, 2024; Bangkok, Thailand. Poster PB0525.
Bleeds During the First Year of Emicizumab Prophylaxis: Preliminary Results of the Brazilian Emicizumab Registry (EMCase Project)
Camelo RM, Henriques LCM, Ferreira CB, et al. Presented at: The 32nd Congress of the International Society on Thrombosis and Haemostasis; June 22-26, 2024; Bangkok, Thailand. Poster PB1204.
Management and Outcomes of Surgical Procedures in People with Haemophilia Receiving Emicizumab
Dix C, Naeve L, Faruqi U, et al. Presented at: The 32nd Congress of the International Society on Thrombosis and Haemostasis; June 22-26, 2024; Bangkok, Thailand. Oral OC 14.3.
Emicizumab Prophylaxis in Previously Untreated and Minimally Treated Boys with Severe Haemophilia A: Summary of United Kingdom Experience
Mathias MC, Kelly AM, Alamelu J, et al. Presented at: The 32nd Congress of the International Society on Thrombosis and Haemostasis; June 22-26, 2024; Bangkok, Thailand. Poster PB0204.
Effectiveness of Emicizumab Under Real-World Conditions in Patients of All Ages with Hemophilia A with and Without FVIII Inhibitors: Third Interim Analysis of the Non-Interventional Study EMIIL
Oldenburg J, Eichler H, Escuriola-Ettingshausen C, et al. Presented at: The 32nd Congress of the International Society on Thrombosis and Haemostasis; June 22-26, 2024; Bangkok, Thailand. Poster PB0262.
Relationship Between Current and Historic Clinical Characteristics and Bleeding Outcomes in People with Severe Haemophilia A on Emicizumab: A Multivariate Analysis of Data from the CHESS Studies
Polito L, Oellerich M, Chafaie R, et al. Presented at: The 32nd Congress of the International Society on Thrombosis and Haemostasis; June 22-26, 2024; Bangkok, Thailand. Poster PB0259.
Long-Term Effectiveness and Safety of Emicizumab in People with Hemophilia A in the Canadian Hemophilia Bleeding Disorders Registry
Poon M-C, Le A, Germini F, et al. Presented at: The 32nd Congress of the International Society on Thrombosis and Haemostasis; June 22-26, 2024; Bangkok, Thailand. Poster PB0202.
Long-Term Safety and Effectiveness of Emicizumab in People with Hemophilia A in the Hemophilia Natural History Study (ATHN 7)
Recht M, Carpenter Sl, Lee L, et al. Presented at: The 32nd Congress of the International Society on Thrombosis and Haemostasis; June 22-26, 2024; Bangkok, Thailand. Poster PB0205.
Uptake of Emicizumab in PUPs with Severe Haemophilia A and Changes in Inhibitor Incidence
Fischer K, Lassila R, Peyvandi F, et al. Haemophilia. 2024;30(Suppl S1):34-188.
Effectiveness of Starting Emicizumab in Routine Clinical Practice for Patients with Severe Hemophilia A Without FVIII Inhibitor: Results Using PicnicHealth Data in the United States
Pipe SW, et al. Presentated at: World Federation of Hemophillia 2024 World Congress; April 21–24, 2024; Madrid, Spain.
Quality of Life in Emicizumab-Treated People with Hemophilia A in the ATHN 7 Hemophilia Natural History Study – an Assessment of Baseline PROMIS®-29
Buckner TW, Daoud N, Lee L, et al. Presented at: 65th Congress of the American Society of Hematology; December 9-12, 2023; San Diego, California. Poster #P3998.
Risk Factors for Joint Bleeding in Severe Hemophilia A and B: Analysis of the Community Counts Longitudinal Surveillance Cohort
Manco-Johnson M, Acharya S, Ahuja S, et al. Blood. 2023;142:287.
Experience with Emicizumab Among People with Hemophilia A in the Canadian Hemophilia Bleeding Disorders Registry
Poon M-C, Lee A, Germini F, et al. Presented at: 65th Congress of the American Society of Hematology; December 9-12, 2023; San Diego, California. Poster #P1240.
Real-World Unmet Needs in Patients with Hemophilia A Without Inhibitors in the US: Results from the PicnicHealth Database
Thornburg C, Wheeler AP, Bozzi S, et al. Presented at: 65th Congress of the American Society of Hematology; December 9-12, 2023; San Diego, California. Poster #P2380.
Should Immune Tolerance Be Pursued in the Era of Emicizumab? Longitudinal Follow Up of a FVIII Inhibitor Cohort - Real World Data
Levy-Mendelovich S, Atia N, Budnik I, et al. Res Pract Thromb Haemost. 2023;7(Suppl 2):100425.
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. Res Pract Thromb Haemost. 2023;7(Suppl 2):100432.
Comparison of Intraindividual Bleeding Outcomes Pre- and Post-Emicizumab in Australian Adults and Children with Haemophilia A with and Without Inhibitors: A Nationwide Real-World Experience
Ramanan R, Parikh S, McFadyen J, et al. Res Pract Thromb Haemost. 2023;7(Suppl 2):101296.
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. Presented at: 16th Congress of the European Association for Haemophilia and Allied Disorders; February 7-10, 2023; Manchester, England. Poster #PO166.
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. Haemophilia. 2023;29(Suppl 1):24-202.
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. Haemophilia. 2023;29(Suppl 1):24-202.
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Publications and Congresses
The information in this section may include content beyond what is in the FDA-approved label. Because the FDA has not approved such content, no conclusions regarding safety or efficacy may be made. Providing this information should not be construed as recommendation for use of a Genentech product for unapproved uses. For FDA-approved products please consult the product’s full prescribing information for a complete discussion of risks and benefits of the product(s) for its approved indication(s).
Publications: The list of publications is not an exhaustive list of published materials on the product. The list of references by data topics is selected per evidence-based medicine criteria. To browse a full listing of published scientific literature:
Congresses: The list of congresses is a subset of Roche/Genentech posters and oral presentations for the product with data presented at scientific meetings in the recent 24 months. To browse full Roche/Genentech congress presentations and posters:
Showing 66 out of 66 results
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20250205 Joint Health and Physical Activity in People with Haemophilia A without Factor VIII Inhibitors Before Switching to Emicizumab Prophylaxis: BEYOND ABR Study Interim Analysis
Castaman G, Di Minno MND, Kruse-Jarres R, et al. Presented at: 18th Congress of the European Association for Haemophilia and Allied Disorders in Milan, Italy; February 4-7, 2025. EAHAD Poster #PO105.Supportive Studies -
20241218 Pain-Related Quality of Life Outcomes in People With Haemophilia A Receiving Emicizumab: A Post Hoc Analysis of the HAVEN 1, 3 and 4 and STASEY Studies
Hermans C, Skinner MW, Gentile B, et al. Haemophilia. 2025;31(1):87-98.Exploratory Analysis -
20241208 FVIII Exposure Analysis from the HAVEN 7 Study of Emicizumab in Infants with Severe Hemophilia A
Young G, Eakle K, Lim E, et al. Presented at: 66th Congress of the American Society of Hematology; December 7-10, 2024; San Diego, California. ASH Poster #2589.Supportive Studies -
20241208 Real-World Adherence and Persistence to Emicizumab Using a National US Claims Database
Shapiro AD, Schuldt R, Xia Z, et al. Presented at: 66th Congress of the American Society of Hematology; December 7-10, 2024; San Diego, California. ASH Poster #3699.Health Economics and Outcomes Research (HEOR) -
20241208 Emicizumab and Immune Tolerance Induction in People with Hemophilia A and Factor VIII Inhibitors: 96-Week Interim Analysis of the AKATSUKI Study
Nagao A, Nogami K, Suzuki N, et al. Presented at: 66th Congress of the American Society of Hematology; December 7-10, 2024; San Diego, California. ASH Poster #2588.Safety -
20241208 Post-Marketing Surveillance of the Safety and Effectiveness of Emicizumab in Japanese Patients with Congenital Hemophilia A with Inhibitors
Shima M, Fukutake K, Matsumoto M, et al. Presented at: 66th Congress of the American Society of Hematology; December 7-10, 2024; San Diego, California. ASH Poster #2583.Safety -
20241207 Health Equity Impacts of Funding Emicizumab in People with Severe Hemophilia a in the US: An Aggregate Distributional Cost-Effectiveness Analysis
Majda T, Lee JS, Curtis R, et al. Presented at: 66th Congress of the American Society of Hematology; December 7-10, 2024; San Diego, California. ASH Oral #279.Health Economics and Outcomes Research (HEOR) -
20241207 Bleed Patterns in Infants, from Birth to 12 Months of Age, with Hemophilia A Treated with Emicizumab: Exploratory Analysis of the HAVEN 7 Study
Carpenter SL, Rosenfelt D, Tzeng E, et al. Presented at: 66th Congress of the American Society of Hematology; December 7-10, 2024; San Diego, California. ASH Poster #1214.Supportive Studies -
20240623 Hemoglobin Improvement Among Patients with Hemophilia A Treated with Emicizumab Prophylaxis: Post Hoc Analysis of the HAVEN 1, 3, 4 and STASEY Trials
Batsuli G, Gupta G, Tzeng E, et al. Presented at: 32nd Congress of the International Society on Thrombosis and Haemostasis; June 22-26, 2024; Bangkok, Thailand. Poster PB0216.Exploratory Analysis -
20240410 Emicizumab Is Efficacious in People with Hemophilia A with Comorbidities Aged ≥50 Years: Analysis of 4 Phase III Trials
Jiménez-Yuste V, Oldenburg J, Tzeng E, et al. Res Pract Thromb Haemost. 2024;8:102405.Exploratory Analysis -
20240404 Emicizumab Prophylaxis in Infants with Hemophilia A (HAVEN 7): Primary Analysis of a Phase 3b, Open-Label Trial
Pipe SW, Collins P, Dhalluin C, et al. Blood. 2024;143:1355-1364.Supportive Studies -
20240301 Long-Term Outcomes with Emicizumab in Hemophilia A Without Inhibitors: Results from the HAVEN 3 and 4 Studies
Mahlangu J, Jiménez-Yuste V, Ventriglia G, et al. Res Pract Thromb Haemost. 2024;8:102364.Exploratory Analysis -
20240207 Emicizumab Prophylaxis in People with Haemophilia A: Summary of 10 Years of Safety Data on Thromboembolic Events and Thrombotic Microangiopathy
Sarouei K, Barlera S, Polito L, et al. Presented at: 17th Congress of the European Association for Haemophilia and Allied Disorders in Frankfurt, Germany; February 6-9, 2024. EAHAD Poster #PO126.Safety -
20240207 Emicizumab in People with Moderate or Mild Haemophilia Aged ≥40 Years, with and Without Comorbidities
Jiménez-Yuste V, Tzeng E, Lim E, et al. Presented at: 17th Congress of the European Association for Haemophilia and Allied Disorders; February 6-9, 2024; Frankfurt, Germany. Poster PO122.Supportive Studies -
20240112 Surgical Experience from the STASEY Study of Emicizumab Prophylaxis in People with Hemophilia A with Factor VIII Inhibitors
Castaman G, Peyvandi F, Kremer Hovinga JA, et al. TH Open. 2024;8:e42-e54.Supportive Studies -
20240101 Association of Physical Activity with Bleeding Events and Safety in Patients with Haemophilia A Starting Emicizumab Prophylaxis: An Interim Analysis of the TSUBASA Study
Nogami K, Fujii T, Sawada A, et al. Int J Hematol. 2024;119:14-23.Supportive Studies -
20231211 Factor VIII Use in the Treatment of Breakthrough Bleeds in People with Moderate or Mild Hemophilia A Without Factor VIII Inhibitors Receiving Emicizumab Prophylaxis: The Phase III HAVEN 6 Experience
Jiménez-Yuste V, Lim E, Mistry K, et al. Presented at: 65th Congress of the American Society of Hematology in San Diego, CA, USA; December 9-12, 2023. ASH Poster #P3999.Supportive Studies -
20231211 Cost-Effectiveness of Emicizumab vs Efanesoctocog Alfa, Standard Half Life (SHL) and Other Extended Half Life (EHL) FVIII Products for Prophylaxis in People with Severe Hemophilia A Without Inhibitors
Curtis R, Decker-Palmer M, Wilson MR, et al. Presented at: 65th Congress of the American Society of Hematology in San Diego, CA, USA; December 9-12, 2023. ASH Poster #5053.Health Economics and Outcomes Research (HEOR) -
20231210 Emicizumab Prophylaxis in Infants with Severe Hemophilia A Without Factor VIII Inhibitors: Results from the Primary Analysis of the HAVEN 7 Study
Pipe S, Collines P, Dhalluin C, et al. Presented at: 65th Congress of the American Society of Hematology; December 9-12, 2023; San Diego, California. Oral #505.Supportive Studies -
20231209 Pharmacodynamic Biomarkers in Infants with Hemophilia A Receiving Emicizumab in HAVEN 7
Kiialainen A, Pipe S, Fijnvandraat K, et al. Presented: 65th Congress of the American Society of Hematology in San Diego, CA, USA; December 9-12, 2023. ASH Poster #P1238.Supportive Studies -
20231021 Emicizumab Use in Females with Moderate or Mild Hemophilia A Without Factor VIII Inhibitors Who Warrant Prophylaxis
Hermans C, Ventriglia G, Obaji S, et al. Res Pract Thromb Haemost. 2023;7:102239.Supportive Studies -
20230828 Characterization of Anti-Emicizumab Antibodies Using Repository Samples Obtained in Clinical Studies of Emicizumab Conducted in Japan
Matsumoto N, Abe H, Kawasaki R, et al. TH Open. 2023;7:e241-e243.Safety -
20230626 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. Presented at: 31st Congress of the International Society of Thrombosis and Haemostasis in Montreal, Canada; June 24-28, 2023. ISTH Poster #PB0641.Supportive Studies -
20230301 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:e168-e177.Supportive Studies -
20230210 Emicizumab Prophylaxis for the Treatment of Infants with Severe Haemophilia A Without Factor VIII Inhibitors: Results from the Interim Analysis of the HAVEN 7 Study on Behalf of the HAVEN 7 Study Investigators
Pipe S, Collins P, Dhalluin C, et al. Presented at: 16th Congress of the European Association for Haemophilia and Allied Disorders; February 7-10, 2023; Manchester, England.Supportive Studies -
20230208 Efficacy of Emicizumab Is Maintained Throughout Dosing Intervals for Bleed Prophylaxis
Pipe SW, Trzaskoma B, Minhas M, et al. Res Pract Thromb Haemost. 2023;7:100077.Exploratory Analysis -
20230207 Long-Term Outcomes with Emicizumab Prophylaxis for Severe Haemophilia A Without FVIII Inhibitors: Safety and Efficacy Analyses from HAVEN 3 & 4
Mahlangu J, Jiménez-Yuste V, Ventriglia G, et al. Presented at: 16th Congress of the European Association for Haemophilia and Allied Disorders; February 7-10, 2023; Manchester, England. Poster PO135.Exploratory Analysis -
20230207 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. Presented at: 16th Congress of the European Association for Haemophilia and Allied Disorders in Manchester, England; February 7-10, 2023. EAHAD Poster #PO123.Safety -
20230207 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. Presented at: 16th Congress of the European Association for Haemophilia and Allied Disorders in Manchester, England; February 7-10, 2023. EAHAD Poster #PO107.Exploratory Analysis -
20230101 Emicizumab Dose Up-Titration in Case of Suboptimal Bleeding Control in People with Haemophilia A
Schmitt C, Mancuso ME, Chang T, et al. Haemophilia. 2023;29:90-99.Exploratory Analysis -
20221227 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;6:6140-6150.Exploratory Analysis -
20221115 Cost-Effectiveness of Emicizumab for Prophylaxis in People with Severe Hemophilia A Without Inhibitors
Decker-Palmer M, Lin CW, Wilson M, et al. Blood. 2022;140(Suppl 1):13041-13042.Health Economics and Outcomes Research (HEOR) -
20221114 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.Supportive Studies -
20221101 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 CL, et al. Haemophilia. 2022;28:1033-1043.Exploratory Analysis -
20220913 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.Exploratory Analysis -
20220701 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;11:717-728.Health Economics and Outcomes Research (HEOR) -
20220701 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;28:e105-e108.Supportive Studies -
20220307 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:e12670.Supportive Studies -
20220201 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;62:232-244.Pharmacokinetics (PK) and Pharmacodynamics (PD) -
20211201 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;19:2938-2946.Safety -
20211101 Patient Preference for Emicizumab Versus Prior Factor Therapy in People with Haemophilia A: Results from the HAVEN 3 and HAVEN 4 Studies
Parnes A, Mahlangu JN, Pipe SW, et al. Haemophilia. 2021;27:e772-e775.Exploratory Analysis -
20211101 Low Immunogenicity of Emicizumab in Persons with Haemophilia A
Schmitt C, Emrich T, Chebon S, et al. Haemophilia. 2021;27(6):984-992.Safety -
20210901 The Effect of Emicizumab Prophylaxis on Long-Term, Self-Reported Physical Health in Persons with Haemophilia A Without Factor VIII Inhibitors in the HAVEN 3 and HAVEN 4 Studies
Skinner MW, Négrier C, Paz-Priel I, et al. Haemophilia. 2021;27:854-865.Exploratory Analysis -
20210701 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.Pharmacokinetics (PK) and Pharmacodynamics (PD) -
20210422 Long-Term Outcomes with Emicizumab Prophylaxis for Hemophilia A With/Without FVIII Inhibitors from the HAVEN 1-4 Studies
Callaghan MU, Negrier C, Paz-Priel I, et al. Blood. 2021;137:2231-2242.Exploratory Analysis -
20210301 Development and Testing of the Satisfaction Questionnaire with Intravenous or Subcutaneous Hemophilia Injection and Results from the Phase 3 HAVEN 3 Study of Emicizumab Prophylaxis in Persons with Haemophilia A Without FVIII Inhibitors
Kempton C, Trask P, Parnes A, et al. Haemophilia. 2021;27:221-228.Exploratory Analysis -
20210301 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. 2021;121:351-360.Pharmacokinetics (PK) and Pharmacodynamics (PD) -
20210101 Mortality in Congenital Hemophilia A - A Systematic Literature Review
Hay CRM, Nissen F, Pipe SW. J Thromb Haemost. 2021;19 Suppl 1:6-20.Safety -
20210101 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.Safety -
20210101 Application of a Hemophilia Mortality Framework to the Emicizumab Global Safety Database
Peyvandi F, Mahlangu JN, Pipe SW, et al. J Thromb Haemost. 2021;19 Suppl 1:32-41.Safety -
20210101 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.Supportive Studies -
20210101 Evaluation of the Pharmacokinetics, Pharmacodynamics, and Safety of a Single Dose of Emicizumab in Healthy Chinese Subjects
Li H, Zhang W, Petry C, et al. Clin Pharmacol Drug Dev. 2021;10:30-38.Pharmacokinetics (PK) and Pharmacodynamics (PD) -
20201201 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;59:1611-1625.Pharmacokinetics (PK) and Pharmacodynamics (PD) -
20201101 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, Sidonio R Jr, et al. Haemophilia. 2020;26:1009-1018.Exploratory Analysis -
20201101 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:e340-e342.Safety -
20200901 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.Health Economics and Outcomes Research (HEOR) -
20191212 A Multicenter, Open-Label, Phase 3 Study of Emicizumab Prophylaxis in Children with Hemophilia A with Inhibitors
Young G, Liesner R, Chang T, et al. Blood. 2019;134:2127-2138.Pivotal Trial(s) -
20191201 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.Health Economics and Outcomes Research (HEOR) -
20191201 Efficacy of Emicizumab Prophylaxis Versus Factor VIII Prophylaxis for Treatment of Hemophilia A Without Inhibitors: Network Meta-Analysis and Sub-Group Analyses of the Intra-Patient Comparison of the HAVEN 3 Trial
Reyes A, Révil C, Niggli M, et al. Curr Med Res Opin. 2019;35:2079-2087.Exploratory Analysis -
20191101 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.Supportive Studies -
20190901 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.Safety -
20190601 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.Pivotal Trial(s) -
20190101 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.Exploratory Analysis -
20180830 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(9):811-822.Pivotal Trial(s) -
20170831 Emicizumab Prophylaxis in Hemophilia A with Inhibitors
Oldenburg J, Mahlangu JN, Kim B, et al. N Engl J Med. 2017;377(9):809-818.Pivotal Trial(s) -
20160526 Factor VIII-Mimetic Function of Humanized Bispecific Antibody in Hemophilia A
Shima M, Hanabusa H, Taki M, et al. N Engl J Med. 2016;374:2044-2053.Supportive Studies
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Medical Resources
SECTIONS ON THIS PAGE
Emicizumab Patient Website
This website is intended to provide community members with balanced clinical and scientific information on the safety and effectiveness of emicizumab for educational purposes, and to support informed and meaningful treatment discussions with healthcare providers.
Guidelines and Recommendations
The below links to evidence-based clinical guidelines and recommendations provide guidance on the use of emicizumab in patients with hemophilia A.
National Blood Disorders Foundation Medical and Scientific Advisory Council
Document #268
Recommendations on the use and management of emicizumab-kxwh (Hemlibra®) for hemophilia A with and without inhibitors.
Document #267
Recommendations on prophylaxis for hemophilia A and B with and without inhibitors.
World Federation of Hemophilia
Guidelines for the Management of Hemophilia, 3rd edition
Srivastava A, Santagostino E, Dougall A, et al. Haemophilia. 2020;26 Suppl 6:1-158.
International Society on Thrombosis and Haemostasis
Clinical Practice Guideline
Rezende SM, Neumann I, Angchaisuksiri P, et al. J Thromb Haemost. 2024 Sep;22(9):2629-2652.
Patient Organizations
Hemophilia Federation of America
National Bleeding Disorders Foundation
Formerly the National Hemophilia Foundation