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Journal Articles

Emicizumab prophylaxis in infants with hemophilia A (HAVEN 7): primary analysis of a phase 3b open-label trial

Steven W. Pipe, Peter Collins, Christophe Dhalluin, Gili Kenet, Christophe Schmitt, Muriel Buri, Víctor Jiménez-Yuste, Flora Peyvandi, Guy Young, Johannes Oldenburg, Maria Elisa Mancuso, Kaan Kavakli, Anna Kiialainen, Sonia Deb, Markus Niggli, Tiffany Chang, Michaela Lehle, Karin Fijnvandraat

Blood (2024) 143 (14): 1355–1364.

https://doi.org/10.1182/blood.2023021832

Published: 2024

Includes: Supplemental data

Journal Articles

Low Rebleed Incidence with Eptacog Beta Treatment through 24 and 48 Hours in Children (<12 years) with Hemophilia a or B with Inhibitors

Blood (2023) 142 (Supplement 1): 5480.

https://doi.org/10.1182/blood-2023-181299

Published: 2023

Journal Articles

Emicizumab Prophylaxis in Infants with Severe Hemophilia A without Factor VIII Inhibitors: Results from the Primary Analysis of the HAVEN 7 Study

Steven Pipe, Peter Collins, Christophe Dhalluin, Gili Kenet, Christophe Schmitt, Muriel Buri, Víctor Jiménez-Yuste, Flora Peyvandi, Guy Young, Johannes Oldenburg, Maria Elisa Mancuso, Kaan Kavakli, Anna Kiialainen, Tiffany Chang, Michaela Lehle, Markus Niggli, Karin Fijnvandraat

Blood (2023) 142 (Supplement 1): 505.

https://doi.org/10.1182/blood-2023-177963

Published: 2023

Journal Articles

Physical Activity, Bleedings and Quality of Life in Patients with Haemophilia a without Inhibitors - a Multicenter, Observational Italian Study with a Wearable Device

Maria Elisa Mancuso, Chiara Biasoli, Renato Marino, Andrea Buzzi, Daniele Preti, Sara Bendinelli, Rosaria Tempre, Elena Pompeo, Luigi Sannino, Giacomo Siri, Giancarlo Castaman

Blood (2023) 142 (Supplement 1): 663.

https://doi.org/10.1182/blood-2023-179089

Published: 2023

Journal Articles

Pharmacodynamic Biomarkers in Infants with Hemophilia A Receiving Emicizumab in HAVEN 7

Anna Kiialainen, Steven Pipe, Karin Fijnvandraat, Gili Kenet, Christophe Schmitt, Muriel Buri, Víctor Jiménez-Yuste, Johannes Oldenburg, Maria Elisa Mancuso, Christophe Dhalluin, Michaela Lehle, Peter Collins

Blood (2023) 142 (Supplement 1): 1238.

https://doi.org/10.1182/blood-2023-173015

Published: 2023

Journal Articles

Eptacog Beta Efficacy in Treating Mild or Moderate Bleeds in Target Joints of Individuals with Hemophilia A or B and Inhibitors in PERSEPT 1

Blood (2022) 140 (Supplement 1): 5597–5599.

https://doi.org/10.1182/blood-2022-156110

Published: 2022

Journal Articles

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

Steven W. Pipe, Peter Collins, Christophe Dhalluin, Gili Kenet, Christophe Schmitt, Muriel Buri, Víctor Jiménez-Yuste, Flora Peyvandi, Guy Young, Johannes Oldenburg, Maria Elisa Mancuso, Anna Kiialainen, Tiffany Chang, Michaela Lehle, Karin Fijnvandraat

Blood (2022) 140 (Supplement 1): 457–459.

https://doi.org/10.1182/blood-2022-157264

Published: 2022

Journal Articles

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

Blood (2021) 137 (16): 2231–2242.

https://doi.org/10.1182/blood.2020009217

Published: 2021

Includes: Supplemental data

Journal Articles

BAY 94-9027 Provides Safe and Effective Long-Term Prophylaxis in Pediatric Patients: Results from the PROTECT VIII Kids Extension Study

Sanjay Ahuja, MD, Kathelijn Fischer, Tina Biss, Monika Maas Enriquez, Maria-Elisa Mancuso, MacGregor Steele, MD FRCPC, Maria Wang, Gili Kenet

Blood (2020) 136 (Supplement 1): 40–41.

https://doi.org/10.1182/blood-2020-140303

Published: 2020

Journal Articles

Safety and Efficacy of Emicizumab in Persons with Hemophilia a with or without FVIII Inhibitors: Pooled Data from Four Phase III Studies (HAVEN 1-4)

Blood (2020) 136 (Supplement 1): 3–5.

https://doi.org/10.1182/blood-2020-137438

Published: 2020

Journal Articles

Potency-Adjusted Analyses of a Head-to-Head Pharmacokinetic Study of Damoctocog Alfa Pegol (BAY 94-9027) and Efmoroctocog Alfa (rFVIIIFc) in Patients with Severe Hemophilia A

Alexander Solms, Anita Shah, Sara Wiegmann, Maurice Ahsman, Erik Berntorp, Andreas Tiede, MD, Alfonso Iorio, MDPhD, Maria Elisa Mancuso, Tihomir Zhivkov, Toshko Lissitchkov

Blood (2020) 136 (Supplement 1): 24–25.

https://doi.org/10.1182/blood-2020-136586

Published: 2020

Journal Articles

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

Guy Young, Ri Liesner, Tiffany Chang, Robert Sidonio, Jr, Johannes Oldenburg, Victor Jiménez-Yuste, Johnny Mahlangu, Rebecca Kruse-Jarres, Michael Wang, Marianne Uguen, Michelle Y. Doral, Lilyan Y. Wright, Christophe Schmitt, Gallia G. Levy, Midori Shima, Maria Elisa Mancuso

Blood (2019) 134 (24): 2127–2138.

https://doi.org/10.1182/blood.2019001869

Published: 2019

Includes: Supplemental data

Journal Articles

Prediction of Anti-FVIII Inhibitor Persistence By Anti-FVIII IgG Subclasses in Patients with Severe Hemophilia — A in the Sippet Cohort Study

Flora Peyvandi, MD PhD, Paolo Bucciarelli, MD, Carla Valsecchi, BSc, Marco Boscarino, Roberta Palla, PhD, Elena Santagostino, Amal El-Beshlawy, Mohsen Saleh Elalfy, MD, Vijay M. Ramanan, MBBS,MDDM, Peyman Eshghi, MD, Suresh Hanagavadi, MD, Mehran Karimi, MD, Cecil Ross, MD, Mamta V Manglani, MD, Guy Young, Tulika Seth, MDMBBS,FAAP, Shashikant Apte, MD FRCPA, Dinesh M Nayak, MD, Maria Elisa Mancuso, Johnny Mahlangu, Bonanad Santiago, MD, Monica H Cerqueira, MD PhD, Nadia P Ewing, MD, Christoph Male, MD, Tarek Owaidah, MDFRCPA, Veronica Soto Arellano, MD, Suvankar Majumdar, MBCHB, Mindy Simpson, MD, Mathew Thomas, MD, Ezio Zanon, Marilyn J Manco-Johnson, MD, Monica Martinez, MD, Maria Gabriella Mazzucconi, MD, Daniela Neme, MD, Rogelio Alejandro Paredes-Aguilera, MD, Alessandra N L Prezotti, MD MSc, Klaus Schmitt, MD, Bulent Zulfikar, Prof, Pier Mannuccio Mannucci, MD, Frits Richard Rosendaal, MD

Blood (2018) 132 (Supplement 1): 384.

https://doi.org/10.1182/blood-2018-99-114599

Published: 2018

Journal Articles

Emicizumab Prophylaxis Provides Flexible and Effective Bleed Control in Children with Hemophilia Α with Inhibitors: Results from the HAVEN 2 Study

Guy Young, Ri Liesner, Robert F. Sidonio, Jr., Johannes Oldenburg, Victor Jimenez-Yuste, Johnny Mahlangu, Rebecca Kruse-Jarres, Michael Wang, Tiffany Chang, Marianne Uguen, Michelle Doral, Christophe Schmitt, Gallia G. Levy, Midori Shima, MD PhD, Maria Elisa Mancuso

Blood (2018) 132 (Supplement 1): 632.

https://doi.org/10.1182/blood-2018-99-118153

Published: 2018

Journal Articles

Prospective Study of the Immunological Mechanisms of Immune Tolerance Induction in Severe Haemophilia a Patients with Inhibitors: Preliminary Analysis of a Multi-Center Longitudinal Study

Davide Matino, MD MSc, Giancarlo Castaman, Giovanni Di Minno, Paola Giordano, MD, Maria Elisa Mancuso, Emanuela Marchesini, Flora Peyvandi, MD PhD, Elena Santagostino, Cristina Santoro, MD PhD, Michele Schiavulli, Antonella De Luca, PhD, Marco Gargaro, Paolo Puccetti, MD, Francesca Fallarino, PhD, Alfonso Iorio, MD PhD

Blood (2018) 132 (Supplement 1): 3781.

https://doi.org/10.1182/blood-2018-99-119568

Published: 2018

Journal Articles

Emicizumab Prophylaxis in Adolescent/Adult Patients with Hemophilia A Previously Receiving Episodic or Prophylactic Bypassing Agent Treatment: Updated Analyses from the HAVEN 1 Study

Maria Elisa Mancuso, Michael U. Callaghan, Rebecca Kruse-Jarres, Christine L Kempton, Jin Xu, Olivier Catalani, Elina Asikanius, Gallia G. Levy, Midori Shima, MDPhD, Guy Young, Johannes Oldenburg

Blood (2017) 130 (Supplement 1): 1071.

https://doi.org/10.1182/blood.V130.Suppl_1.1071.1071

Published: 2017

Journal Articles

Management of Joint Replacement in Hemophilia a with Inhibitors during Emicizumab Prophylaxis

Elena Santagostino, Maria Elisa Mancuso, MD PhD, Cristina Novembrino, Massimo Anzoletti Boscolo, Marigrazia Clerici, Gianluigi Pasta, MD, Luigi Piero Solimeno, MD, Flora Peyvandi

Blood (2017) 130 (Supplement 1): 2360.

https://doi.org/10.1182/blood.V130.Suppl_1.2360.2360

Published: 2017

Journal Articles

Risk Factors for the Development of High-Titer Inhibitors in 260 Children with Severe Hemophilia a Born Between 1990 and 2009: The Remain Study

Blood (2016) 128 (22): 3774.

https://doi.org/10.1182/blood.V128.22.3774.3774

Published: 2016

Journal Articles

Characterization of Bleeding in Hemophilia Carriers and Comparison to Women with Type 1 Von Willebrand Disease, Type 3 Von Willebrand Disease Obligate Carriers and Controls

Natasha Amrita Satkunam, MBBS, MSc, Johnny Mahlangu, MD, Christoph Bidlingmaier, MD PD, Dr. MD, Maria Eva Mingot-Castellano, PhD, Meera B. Chitlur, MD, Patrick F. Fogarty, MD, Adam Cuker, MD MS, Maria Elisa Mancuso, MD PhD, Pal André Holme, PhD MD, Julie Grabell, BA, CCRP, Wilma Hopman, MA, Prasad Mathew, MD, Paula D. James, MD

Blood (2016) 128 (22): 875.

https://doi.org/10.1182/blood.V128.22.875.875

Published: 2016

Journal Articles

Whole-exome sequencing to identify genetic risk variants underlying inhibitor development in severe hemophilia A patients

Marcin M. Gorski, Kevin Blighe, Luca A. Lotta, Emanuela Pappalardo, Isabella Garagiola, Ilaria Mancini, Maria Elisa Mancuso, Maria Rosaria Fasulo, Elena Santagostino, Flora Peyvandi

Blood (2016) 127 (23): 2924–2933.

https://doi.org/10.1182/blood-2015-12-685735

Published: 2016

NARROW

Emicizumab prophylaxis in infants with hemophilia A (HAVEN 7): primary analysis of a phase 3b open-label trial

Low Rebleed Incidence with Eptacog Beta Treatment through 24 and 48 Hours in Children (<12 years) with Hemophilia a or B with Inhibitors

Emicizumab Prophylaxis in Infants with Severe Hemophilia A without Factor VIII Inhibitors: Results from the Primary Analysis of the HAVEN 7 Study

Physical Activity, Bleedings and Quality of Life in Patients with Haemophilia a without Inhibitors - a Multicenter, Observational Italian Study with a Wearable Device

Pharmacodynamic Biomarkers in Infants with Hemophilia A Receiving Emicizumab in HAVEN 7

Eptacog Beta Efficacy in Treating Mild or Moderate Bleeds in Target Joints of Individuals with Hemophilia A or B and Inhibitors in PERSEPT 1

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

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

BAY 94-9027 Provides Safe and Effective Long-Term Prophylaxis in Pediatric Patients: Results from the PROTECT VIII Kids Extension Study

Safety and Efficacy of Emicizumab in Persons with Hemophilia a with or without FVIII Inhibitors: Pooled Data from Four Phase III Studies (HAVEN 1-4)

Potency-Adjusted Analyses of a Head-to-Head Pharmacokinetic Study of Damoctocog Alfa Pegol (BAY 94-9027) and Efmoroctocog Alfa (rFVIIIFc) in Patients with Severe Hemophilia A

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

Prediction of Anti-FVIII Inhibitor Persistence By Anti-FVIII IgG Subclasses in Patients with Severe Hemophilia — A in the Sippet Cohort Study

Emicizumab Prophylaxis Provides Flexible and Effective Bleed Control in Children with Hemophilia Α with Inhibitors: Results from the HAVEN 2 Study

Prospective Study of the Immunological Mechanisms of Immune Tolerance Induction in Severe Haemophilia a Patients with Inhibitors: Preliminary Analysis of a Multi-Center Longitudinal Study

Emicizumab Prophylaxis in Adolescent/Adult Patients with Hemophilia A Previously Receiving Episodic or Prophylactic Bypassing Agent Treatment: Updated Analyses from the HAVEN 1 Study

Management of Joint Replacement in Hemophilia a with Inhibitors during Emicizumab Prophylaxis

Risk Factors for the Development of High-Titer Inhibitors in 260 Children with Severe Hemophilia a Born Between 1990 and 2009: The Remain Study

Characterization of Bleeding in Hemophilia Carriers and Comparison to Women with Type 1 Von Willebrand Disease, Type 3 Von Willebrand Disease Obligate Carriers and Controls

Whole-exome sequencing to identify genetic risk variants underlying inhibitor development in severe hemophilia A patients

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American Society of Hematology

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American Society of Hematology

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