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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

Inhibitor Development with Simoctocog Alfa in Previously Untreated Patients with Severe Haemophilia a: Final Results of the Nuprotect Study

Ri Liesner, Ellis J. Neufeld, MD

Blood (2019) 134 (Supplement_1): 903.

https://doi.org/10.1182/blood-2019-125520

Published: 2019

Journal Articles

Characterization and treatment of congenital thrombotic thrombocytopenic purpura

Blood (2019) 133 (15): 1644–1651.

https://doi.org/10.1182/blood-2018-11-884700

Published: 2019

Journal Articles

Immune Tolerance Induction with Simoctocog Alfa (Nuwiq ® ) in Six Patients with Severe Haemophilia a and FVIII Inhibitors

Neha Bhatnagar, ND, Kate Khair, Ri Liesner, Alice Wilkinson, Larisa Belyanskaya, PhD

Blood (2018) 132 (Supplement 1): 5037.

https://doi.org/10.1182/blood-2018-99-118041

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

A New Flow-Based Assay to Assess Treatment Response in Congenital Thrombotic Thrombocytopenic Purpura

Ferras Alwan, Chiara Vendramin, Alice Taylor, Mari Thomas, PhD, Ri Liesner, Marie Scully, MD MRCP, FRCPath

Blood (2018) 132 (Supplement 1): 2441.

https://doi.org/10.1182/blood-2018-99-115965

Published: 2018

Journal Articles

99.3% of Inhibitors in Severe Hemophilia a Develop before Exposure Day 75. Time to Change Definition of Previously Treated Patients; Data from 1038 Patients with Severe Hemophilia a of the Pednet Registry

Blood (2018) 132 (Supplement 1): 2472.

https://doi.org/10.1182/blood-2018-99-114518

Published: 2018

Journal Articles

Immunogenicity of Two Plasma-Derived FVIII Products and Simoctocog Alfa in Previously Untreated Patients According to F8 Mutation Type

Ri Liesner, Joris Versteden, Shannely Lowndes, Larisa Belyanskaya, PhD, Johannes Oldenburg, MD PhD, Anna Pavlova, MD PhD

Blood (2018) 132 (Supplement 1): 1204.

https://doi.org/10.1182/blood-2018-99-110608

Published: 2018

Journal Articles

A Multicenter, Retrospective Data Collection Study on the Compassionate Use of a Plasma-Derived Factor X Concentrate to Treat Patients with Hereditary Factor X Deficiency

James N. Huang, MD, Ri Liesner, Chioma Akenezi, BSc, PgDip, Steven K. Austin, BMedSci, MBBS, Kaan Kavakli, MD

Blood (2017) 130 (Supplement 1): 3736.

https://doi.org/10.1182/blood.V130.Suppl_1.3736.3736

Published: 2017

Journal Articles

Characterizing the United Kingdom Congenital Thrombotic Thrombocytopenic Purpura Population

Ferras Alwan, MBBS, Chiara Vendramin, Ri Liesner, Tina Dutt, Amanda Clark, Jayashree Motwani, Will Lester, Vickie McDonald, Rachel Rayment, Nichola Cooper, MA, MD MRCP, FRCPath, Richard Gooding, Hamish Lyall, Tina Biss, MD, Joost van Veen, Henry G Watson, John-Paul Westwood, Mari Thomas, Marie Scully

Blood (2017) 130 (Supplement 1): 3616.

https://doi.org/10.1182/blood.V130.Suppl_1.3616.3616

Published: 2017

Journal Articles

Surgical Experience in Two Multicenter, Open-Label Phase 3 Studies of Emicizumab in Persons with Hemophilia A with Inhibitors (HAVEN 1 and HAVEN 2)

Rebecca Kruse-Jarres, Michael U. Callaghan, Stacy E. Croteau, MD, Victor Jimenez-Yuste, Liane Khoo, Ri Liesner, Tadashi Matsushita, Michael Recht, Guy Young, Tiffany Chang, Christophe Dhalluin, Yunming Mu, Jin Xu, Jenny Devenport, Richard H. Ko, Paul Solari, Johannes Oldenburg

Blood (2017) 130 (Supplement 1): 89.

https://doi.org/10.1182/blood.V130.Suppl_1.89.89

Published: 2017

Journal Articles

HAVEN 2 Updated Analysis: Multicenter, Open-Label, Phase 3 Study to Evaluate Efficacy, Safety and Pharmacokinetics of Subcutaneous Administration of Emicizumab Prophylaxis in Pediatric Patients with Hemophilia A with Inhibitors

Guy Young, Robert F. Sidonio, Ri Liesner, Johannes Oldenburg, Tiffany Chang, Marianne Uguen, Christophe Dhalluin, Christophe Schmitt, Gallia G. Levy, Midori Shima, MDPhD, Johnny Mahlangu

Blood (2017) 130 (Supplement 1): 85.

https://doi.org/10.1182/blood.V130.Suppl_1.85.85

Published: 2017

Journal Articles

Ahead Study: A 3 Year Follow-up of 522 Severe and Moderate Hemophilia a Patients

Johannes Oldenburg, Prof, Dimitrios Tsakiris, Prof, Cedric R. Hermans, MD PhD MRCP, RI Liesner, MD FRCP, FRCPath, MA, MBBChir, Kate Khair, Maria Gabriella Mazzucconi, Prof, Katharina N Steinitz-Trost, PhD, Gerald Spotts, PhD, Armin J Reininger, Prof, Alessandro Gringeri, MD MSc

Blood (2016) 128 (22): 3786.

https://doi.org/10.1182/blood.V128.22.3786.3786

Published: 2016

Journal Articles

A high-throughput sequencing test for diagnosing inherited bleeding, thrombotic, and platelet disorders

Ilenia Simeoni, Jonathan C. Stephens, Fengyuan Hu, Sri V. V. Deevi, Karyn Megy, Tadbir K. Bariana, Claire Lentaigne, Sol Schulman, Suthesh Sivapalaratnam, Minka J. A. Vries, Sarah K. Westbury, Daniel Greene, Sofia Papadia, Marie-Christine Alessi, Antony P. Attwood, Matthias Ballmaier, Gareth Baynam, Emilse Bermejo, Marta Bertoli, Paul F. Bray, Loredana Bury, Marco Cattaneo, Peter Collins, Louise C. Daugherty, Rémi Favier, Deborah L. French, Bruce Furie, Michael Gattens, Manuela Germeshausen, Cedric Ghevaert, Anne C. Goodeve, Jose A. Guerrero, Daniel J. Hampshire, Daniel P. Hart, Johan W. M. Heemskerk, Yvonne M. C. Henskens, Marian Hill, Nancy Hogg, Jennifer D. Jolley, Walter H. Kahr, Anne M. Kelly, Ron Kerr, Myrto Kostadima, Shinji Kunishima, Michele P. Lambert, Ri Liesner, José A. López, Rutendo P. Mapeta, Mary Mathias, Carolyn M. Millar, Amit Nathwani, Marguerite Neerman-Arbez, Alan T. Nurden, Paquita Nurden, Maha Othman, Kathelijne Peerlinck, David J. Perry, Pawan Poudel, Pieter Reitsma, Matthew T. Rondina, Peter A. Smethurst, William Stevenson, Artur Szkotak, Salih Tuna, Christel van Geet, Deborah Whitehorn, David A. Wilcox, Bin Zhang, Shoshana Revel-Vilk, Paolo Gresele, Daniel B. Bellissimo, Christopher J. Penkett, Michael A. Laffan, Andrew D. Mumford, Augusto Rendon, Keith Gomez, Kathleen Freson, Willem H. Ouwehand, Ernest Turro

Blood (2016) 127 (23): 2791–2803.

https://doi.org/10.1182/blood-2015-12-688267

Published: 2016

Includes: Supplemental data

Journal Articles

Factor VIII brand and the incidence of factor VIII inhibitors in previously untreated UK children with severe hemophilia A, 2000-2011

Peter W. Collins, Benedict P. Palmer, Elizabeth A. Chalmers, Daniel P. Hart, Ri Liesner, Savita Rangarajan, Katherine Talks, Michael Williams, Charles R. M. Hay

Blood (2014) 124 (23): 3389–3397.

https://doi.org/10.1182/blood-2014-07-580498

Published: 2014

Journal Articles

A Two Centre Experience Of Use Of a Dual Virally Inactivated FVIII/VWF Product (Wilate®) In Patients With Von Willebrand Disease

Paul A Batty, MBBS, Kate Khair, PhD, Renu Riat, MBBS, Yun-Han Chen, MBBS, Louise Bowles, Daniel P Hart, MBChB, MRCP, FRCPath, PhD, Ri Liesner, MA MBBChir MD FRCP FRCPath MRCPCH, Sean Platton, Clive Burgess, John Pasi, MBChB, PhD, FRCP, FRCPath

Blood (2013) 122 (21): 1112.

https://doi.org/10.1182/blood.V122.21.1112.1112

Published: 2013

Journal Articles

Factor VIII gene ( F8 ) mutation and risk of inhibitor development in nonsevere hemophilia A

Corien L. Eckhardt, Alice S. van Velzen, Marjolein Peters, Jan Astermark, Paul P. Brons, Giancarlo Castaman, Marjon H. Cnossen, Natasja Dors, Carmen Escuriola-Ettingshausen, Karly Hamulyak, Daniel P. Hart, Charles R. M. Hay, Saturnino Haya, Waander L. van Heerde, Cedric Hermans, Margareta Holmström, Victor Jimenez-Yuste, Russell D. Keenan, Robert Klamroth, Britta A. P. Laros-van Gorkom, Frank W. G. Leebeek, Ri Liesner, Anne Mäkipernaa, Christoph Male, Evelien Mauser-Bunschoten, Maria G. Mazzucconi, Simon McRae, Karina Meijer, Michael Mitchell, Massimo Morfini, Marten Nijziel, Johannes Oldenburg, Kathelijne Peerlinck, Pia Petrini, Helena Platokouki, Sylvia E. Reitter-Pfoertner, Elena Santagostino, Piercarla Schinco, Frans J. Smiers, Berthold Siegmund, Annarita Tagliaferri, Thynn T. Yee, Pieter Willem Kamphuisen, Johanna G. van der Bom, Karin Fijnvandraat

Blood (2013) 122 (11): 1954–1962.

https://doi.org/10.1182/blood-2013-02-483263

Published: 2013

Includes: Supplemental data

Journal Articles

Intensity of factor VIII treatment and inhibitor development in children with severe hemophilia A: the RODIN study

Blood (2013) 121 (20): 4046–4055.

https://doi.org/10.1182/blood-2012-09-457036

Published: 2013

Includes: Supplemental data

Journal Articles

IB1001, an Investigational Recombinant Factor IX, Pharmacokinetics in Pediatric Patients with Hemophilia B.

Edward D. Gomperts, MD, Shashikant Apte, MD, Utpal Chaudhuri, MD, Joseph M John, MD, Vijay Ramanan, MD, Ri Liesner, MA MBBChir MD FRCP FRCPath MRCPCH, Amy D. Shapiro, MD, Bonnie J. Mills, PhD, Martin Lee, PhD

Blood (2012) 120 (21): 2225.

https://doi.org/10.1182/blood.V120.21.2225.2225

Published: 2012

Journal Articles

Residual plasmatic activity of ADAMTS13 is correlated with phenotype severity in congenital thrombotic thrombocytopenic purpura

Luca A. Lotta, Haifeng M. Wu, Ian J. Mackie, Marina Noris, Agnes Veyradier, Marie A. Scully, Giuseppe Remuzzi, Paul Coppo, Ri Liesner, Roberta Donadelli, Chantal Loirat, Richard A. Gibbs, April Horne, Shangbin Yang, Isabella Garagiola, Khaled M. Musallam, Flora Peyvandi

Blood (2012) 120 (2): 440–448.

https://doi.org/10.1182/blood-2012-01-403113

Published: 2012

Includes: Supplemental data

NARROW

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

Inhibitor Development with Simoctocog Alfa in Previously Untreated Patients with Severe Haemophilia a: Final Results of the Nuprotect Study

Characterization and treatment of congenital thrombotic thrombocytopenic purpura

Immune Tolerance Induction with Simoctocog Alfa (Nuwiq ® ) in Six Patients with Severe Haemophilia a and FVIII Inhibitors

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

A New Flow-Based Assay to Assess Treatment Response in Congenital Thrombotic Thrombocytopenic Purpura

99.3% of Inhibitors in Severe Hemophilia a Develop before Exposure Day 75. Time to Change Definition of Previously Treated Patients; Data from 1038 Patients with Severe Hemophilia a of the Pednet Registry

Immunogenicity of Two Plasma-Derived FVIII Products and Simoctocog Alfa in Previously Untreated Patients According to F8 Mutation Type

A Multicenter, Retrospective Data Collection Study on the Compassionate Use of a Plasma-Derived Factor X Concentrate to Treat Patients with Hereditary Factor X Deficiency

Characterizing the United Kingdom Congenital Thrombotic Thrombocytopenic Purpura Population

Surgical Experience in Two Multicenter, Open-Label Phase 3 Studies of Emicizumab in Persons with Hemophilia A with Inhibitors (HAVEN 1 and HAVEN 2)

HAVEN 2 Updated Analysis: Multicenter, Open-Label, Phase 3 Study to Evaluate Efficacy, Safety and Pharmacokinetics of Subcutaneous Administration of Emicizumab Prophylaxis in Pediatric Patients with Hemophilia A with Inhibitors

Ahead Study: A 3 Year Follow-up of 522 Severe and Moderate Hemophilia a Patients

A high-throughput sequencing test for diagnosing inherited bleeding, thrombotic, and platelet disorders

Factor VIII brand and the incidence of factor VIII inhibitors in previously untreated UK children with severe hemophilia A, 2000-2011

A Two Centre Experience Of Use Of a Dual Virally Inactivated FVIII/VWF Product (Wilate®) In Patients With Von Willebrand Disease

Factor VIII gene ( F8 ) mutation and risk of inhibitor development in nonsevere hemophilia A

Intensity of factor VIII treatment and inhibitor development in children with severe hemophilia A: the RODIN study

IB1001, an Investigational Recombinant Factor IX, Pharmacokinetics in Pediatric Patients with Hemophilia B.

Residual plasmatic activity of ADAMTS13 is correlated with phenotype severity in congenital thrombotic thrombocytopenic purpura

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