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Journal Articles

Clinical Outcomes over 3 Years of Once-Weekly Efanesoctocog Alfa Treatment in Adults and Adolescents with Severe Hemophilia A: Second Interim Analysis from the Phase 3 XTEND-ed Long-Term Extension Study

Robert Klamroth, Stephanie P'ng, Sophie Susen, Davide Matino, Pratima Chowdary, Angela C Weyand, Toshko Lissitchkov, Young-Shil Park, María Teresa Alvarez-Román, Liqi Feng, Helena Palmborg, Jennifer Dumont, Elena Santagostino, Lara Mamikonian, Keiji Nogami

Blood (2024) 144 (Supplement 1): 717.

https://doi.org/10.1182/blood-2024-204533

Published: 2024

Journal Articles

Sustained Improvement of Survival in Patients Receiving Emicizumab Prophylaxis Instead of Immunosuppression during Early Management of Acquired Hemophilia a (AHA)

Inga M. Schimansky, Andreas Tiede, Christiane Dobbelstein, Dr. Robert Klamroth, Christina Hart, Ulrich J. Sachs, Richard Greil, Paul N. Knöbl, Johannes Oldenburg, Christian Pfrepper, Karolin Trautmann-Grill, Patrick Moehnle, Katharina Holstein, Wolfgang A. Miesbach

Blood (2024) 144 (Supplement 1): 555.

https://doi.org/10.1182/blood-2024-205490

Published: 2024

Journal Articles

Characterizing a Cohort of Patients with Hemophilia B Treated with Fidanacogene Elaparvovec from the Phase 3 Benegene-2 Study Who Returned to Factor IX Prophylaxis

Laurent Frenzel, Kaan Kavakli, Robert Klamroth, Shyh-Shin Chiou, Amy D. Shapiro, Pengling Sun, Joanne Fuiman, John McKay, Annie F. Fang, Francesca Biondo, Frank Plonski, Jeremy Rupon

Blood (2023) 142 (Supplement 1): 2257.

https://doi.org/10.1182/blood-2023-181223

Published: 2023

Journal Articles

Design of an Observational Study (PROVE) to Assess the Long-Term Effects of Prophylaxis with Simoctocog Alfa or Emicizumab on Joint and Bone Health in Hemophilia a Patients

Robert Klamroth, Robert F Sidonio, Jr., Yesim Dargaud, Víctor Jiménez-Yuste, Rolf Ljung, Guy Young, Pratima Chowdary

Blood (2023) 142 (Supplement 1): 1255.

https://doi.org/10.1182/blood-2023-181725

Published: 2023

Journal Articles

Long-Term Bleeding Protection, Sustained FIX Activity, Reduction of FIX Consumption and Safety of Hemophilia B Gene Therapy: Results from the HOPE-B Trial 3 Years after Administration of a Single Dose of Etranacogene Dezaparvovec in Adult Patients with Severe or Moderately Severe Hemophilia B

Blood (2023) 142 (Supplement 1): 1055.

https://doi.org/10.1182/blood-2023-187624

Published: 2023

Journal Articles

Emicizumab Versus Immunosuppression for Acquired Hemophilia Α (AHA)

Christina Hart, Robert Klamroth, Ulrich J. Sachs, Richard Greil, Paul N. Knoebl, Johannes Oldenburg, Wolfgang Miesbach, Christian Pfrepper, Karolin Trautmann-Grill, Isabell Pekrul, Katharina Holstein, Hermann Eichler, Carmen Weigt, Dorothea Schipp, Sonja Werwitzke, Andreas Tiede

Blood (2023) 142 (Supplement 1): 2612.

https://doi.org/10.1182/blood-2023-174958

Published: 2023

Journal Articles

Fitusiran, an Investigational siRNA Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: First Results from a Phase 3 Study to Evaluate Efficacy and Safety in People with Hemophilia a or B without Inhibitors (ATLAS-A/B)

Alok Srivastava, Savita Rangarajan, Kaan Kavakli, Robert Klamroth, Gili Kenet, Liane Khoo, Chur-Woo You, Weiqun Xu, Niel Malan, Laurent Frenzel, Catherine N. Bagot, Oleksandra Stasyshyn, Chia-Yau Chang, Stacey Poloskey, Shauna Andersson, Zhiying Qiu, Baisong Mei, Steven W. Pipe

Blood (2021) 138 (Supplement 2): LBA-3.

https://doi.org/10.1182/blood-2021-155018

Published: 2021

Journal Articles

Health-Related Quality of Life Following Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A in the Phase 3 Trial GENEr8-1

Blood (2021) 138 (Supplement 1): 4916.

https://doi.org/10.1182/blood-2021-146021

Published: 2021

Journal Articles

Pharmacokinetic-Guided Prophylaxis with Rurioctocog Alfa Pegol Improves Bleeding Rates over Standard Prophylaxis: Real-World Observational Data from the Ahead German Study

Robert Klamroth, Karin Kurnik, Swee Wenning, Carmen Escuriola-Ettingshausen, Stephan Regensburger, Leilei Tang, Joan Gu, Randy Guerra, Johannes Oldenburg

Blood (2021) 138 (Supplement 1): 3187.

https://doi.org/10.1182/blood-2021-146162

Published: 2021

Journal Articles

Prophylactic Treatment with Rurioctocog Alfa Pegol Results in a Dose-Dependent Normalization of Biomarkers of Joint Health in Severe Hemophilia a: An Exploratory Analysis from the Propel Study

Tina Manon-Jensen, Srilatha Tangada, Cecilie Bager, Pratima Chowdary, Robert Klamroth, Annette von Drygalski, Jerzy Windyga, Miguel A. Escobar, Peder Frederiksen, Werner Engl, Bruce Ewenstein, Morten Karsdal

Blood (2021) 138 (Supplement 1): 2102.

https://doi.org/10.1182/blood-2021-150139

Published: 2021

Journal Articles

Rurioctocog alfa pegol PK-guided prophylaxis in hemophilia A: results from the phase 3 PROPEL study

Robert Klamroth, Jerzy Windyga, Vlad Radulescu, Peter W. Collins, Oleksandra Stasyshyn, Hishamshah Mohd Ibrahim, Werner Engl, Srilatha D. Tangada, William Savage, Bruce Ewenstein

Blood (2021) 137 (13): 1818–1827.

https://doi.org/10.1182/blood.2020005673

Published: 2021

Includes: Supplemental data

Journal Articles

First Data from the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults with Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing Antibodies

Steven W. Pipe, MD, Michael Recht, MD PhD, Nigel S. Key, MD, Frank W.G. Leebeek, MD PhD, Giancarlo Castaman, MD, Susan U. Lattimore, R.N., Paul Van Der Valk, Kathelijne Peerlinck, MD PhD, Michiel Coppens, MD, Niamh O'Connell, MD PhD, John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH, Peter Kampmann, MD, Karina Meijer, MD PhD, Annette von Drygalski, MD Pharm D, Guy Young, MD, Cedric Hermans, MDMRCP,PhD, Jan Astermark, MD PhD, Robert Klamroth, MD PhD, Richard S. Lemons, MD, Nathan Visweshwar, MD, Shelley Crary, MD MS, Rashid Kazmi, MBBS, Emily Symington, Miguel A. Escobar, MD, Esteban Gomez, MD, Rebecca Kruse-Jarres, MD, Adam Kotowski, MD, Doris Quon, MD PhD, Michael Wang, MD, Allison P. Wheeler, MD, Eileen K Sawyer, PhD, Stephanie Verweij, BSc, Valerie Colletta, MSc, Naghmana Bajma, MD, Robert Gut, MD PhD, Wolfgang A. Miesbach, MD PhD

Blood (2020) 136 (Supplement_2): LBA-6.

https://doi.org/10.1182/blood-2020-143560

Published: 2020

Journal Articles

AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B Confirm Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 5 Years

Frank W.G. Leebeek, MD PhD, Karina Meijer, MD PhD, Michiel Coppens, MD, Peter Kampmann, MD, Robert Klamroth, Roger Schutgens, MD PhD MSc, Giancarlo Castaman, MD, Erhard Seifried, MD PhD, Joachim Schwaeble, Halvard Bönig, Eileen K Sawyer, PhD, Wolfgang A. Miesbach, MD

Blood (2020) 136 (Supplement 1): 26.

https://doi.org/10.1182/blood-2020-139225

Published: 2020

Journal Articles

Effectiveness and Safety Outcomes in Patients with Hemophilia a Receiving Antihemophilic Factor (Recombinant) for at Least 5 Years in a Real-World Setting: 6-Year Interim Analysis of the Ahead International and German Studies

Dimitrios A. Tsakiris, MD, Johannes Oldenburg, Robert Klamroth, Benoît Guillet, Kate Khair, Angela Huth-Kühne, MD, Karin Kurnik, Stephan Regensburger, Jaco Botha, Alejandro Fernandez, Leilei Tang, Margareth C. Ozelo

Blood (2020) 136 (Supplement 1): 1.

https://doi.org/10.1182/blood-2020-139873

Published: 2020

Journal Articles

Bleeding and response to hemostatic therapy in acquired hemophilia A: results from the GTH-AH 01/2010 study

Katharina Holstein, Xiaofei Liu, Andrea Smith, Paul Knöbl, Robert Klamroth, Ulrich Geisen, Hermann Eichler, Wolfgang Miesbach, Andreas Tiede

Blood (2020) 136 (3): 279–287.

https://doi.org/10.1182/blood.2019003639

Published: 2020

Includes: Supplemental data

Journal Articles

Reduction in Annualized Bleeding and Factor IX Consumption up to 2.5 Years in Adults with Severe or Moderate-Severe Hemophilia B Treated with AMT-060 (AAV5-hFIX) Gene Therapy

Frank Leebeek, K Meijer, MD, Michiel Coppens, MD, Peter Kampmann, MD, Robert Klamroth, Roger Schutgens, MD PhD MSc, Giancarlo Castaman, Erhard Seifried, MD, J Schwaeble, MD, Halvard Bonig, MD, Eileen K Sawyer, PhD, W Miesbach

Blood (2018) 132 (Supplement 1): 3476.

https://doi.org/10.1182/blood-2018-99-109995

Published: 2018

Journal Articles

Practical Utilisation of Octapharma FVIII Concentrates in Previously Untreated and Minimally Treated Haemophilia a Patients Entering Routine Clinical Treatment with Nuwiq ® , Octanate ® or Wilate ® — the Protect-NOW Study

Johannes Oldenburg, MD PhD, Anna Pavlova, MD PhD, Susan Halimeh, MD, Robert Klamroth, Joris Versteden, Martina Jansen, Larisa Belyanskaya, PhD

Blood (2018) 132 (Supplement 1): 5034.

https://doi.org/10.1182/blood-2018-99-116577

Published: 2018

Journal Articles

Efficacy of an Extended Half-Life GlycoPEGylated rFVIII (N8-GP): Pooled Analysis of ABR (Results from Two Clinical Trials)

Pratima Chowdary, MD, Lone Hvitfeldt Poulsen, Miguel A Escobar, MD, Susan Kearney, Meera B. Chitlur, MD, Robert Klamroth, Mudi Misgav, Claude Negrier, Wan Hui Ong Clausen, Frank Driessler, Andrea Landorph, Elena Santagostino

Blood (2018) 132 (Supplement 1): 1177.

https://doi.org/10.1182/blood-2018-99-110308

Published: 2018

Journal Articles

Gene therapy with adeno-associated virus vector 5–human factor IX in adults with hemophilia B

Wolfgang Miesbach, Karina Meijer, Michiel Coppens, Peter Kampmann, Robert Klamroth, Roger Schutgens, Marco Tangelder, Giancarlo Castaman, Joachim Schwäble, Halvard Bonig, Erhard Seifried, Federica Cattaneo, Christian Meyer, Frank W. G. Leebeek

Blood (2018) 131 (9): 1022–1031.

https://doi.org/10.1182/blood-2017-09-804419

Published: 2018

Includes: Supplemental data

Journal Articles

Factor VIII Activity and Risk of Bleeding in Acquired Hemophilia a: Results from the Gth-AH 01/2010 Study

Katharina Holstein, MD, Andrea Smith, Paul Knöbl, MD PhD, Christiane Dobbelstein, MD, Wolfgang A. Miesbach, MD PhD, Michael Spannagl, MD PhD, Jürgen Heinz, MD PhD, Ute Scholz, MD, Hermann Eichler, MD, Angela Huth-Kühne, MD, Robert Klamroth, MD PhD, Armin Koch, MD PhD, Andreas Tiede, MD

Blood (2017) 130 (Supplement 1): 22.

https://doi.org/10.1182/blood.V130.Suppl_1.22.22

Published: 2017

NARROW

Clinical Outcomes over 3 Years of Once-Weekly Efanesoctocog Alfa Treatment in Adults and Adolescents with Severe Hemophilia A: Second Interim Analysis from the Phase 3 XTEND-ed Long-Term Extension Study

Sustained Improvement of Survival in Patients Receiving Emicizumab Prophylaxis Instead of Immunosuppression during Early Management of Acquired Hemophilia a (AHA)

Characterizing a Cohort of Patients with Hemophilia B Treated with Fidanacogene Elaparvovec from the Phase 3 Benegene-2 Study Who Returned to Factor IX Prophylaxis

Design of an Observational Study (PROVE) to Assess the Long-Term Effects of Prophylaxis with Simoctocog Alfa or Emicizumab on Joint and Bone Health in Hemophilia a Patients

Long-Term Bleeding Protection, Sustained FIX Activity, Reduction of FIX Consumption and Safety of Hemophilia B Gene Therapy: Results from the HOPE-B Trial 3 Years after Administration of a Single Dose of Etranacogene Dezaparvovec in Adult Patients with Severe or Moderately Severe Hemophilia B

Emicizumab Versus Immunosuppression for Acquired Hemophilia Α (AHA)

Fitusiran, an Investigational siRNA Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: First Results from a Phase 3 Study to Evaluate Efficacy and Safety in People with Hemophilia a or B without Inhibitors (ATLAS-A/B)

Health-Related Quality of Life Following Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A in the Phase 3 Trial GENEr8-1

Pharmacokinetic-Guided Prophylaxis with Rurioctocog Alfa Pegol Improves Bleeding Rates over Standard Prophylaxis: Real-World Observational Data from the Ahead German Study

Prophylactic Treatment with Rurioctocog Alfa Pegol Results in a Dose-Dependent Normalization of Biomarkers of Joint Health in Severe Hemophilia a: An Exploratory Analysis from the Propel Study

Rurioctocog alfa pegol PK-guided prophylaxis in hemophilia A: results from the phase 3 PROPEL study

First Data from the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults with Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing Antibodies

AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B Confirm Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 5 Years

Effectiveness and Safety Outcomes in Patients with Hemophilia a Receiving Antihemophilic Factor (Recombinant) for at Least 5 Years in a Real-World Setting: 6-Year Interim Analysis of the Ahead International and German Studies

Bleeding and response to hemostatic therapy in acquired hemophilia A: results from the GTH-AH 01/2010 study

Reduction in Annualized Bleeding and Factor IX Consumption up to 2.5 Years in Adults with Severe or Moderate-Severe Hemophilia B Treated with AMT-060 (AAV5-hFIX) Gene Therapy

Practical Utilisation of Octapharma FVIII Concentrates in Previously Untreated and Minimally Treated Haemophilia a Patients Entering Routine Clinical Treatment with Nuwiq ® , Octanate ® or Wilate ® — the Protect-NOW Study

Efficacy of an Extended Half-Life GlycoPEGylated rFVIII (N8-GP): Pooled Analysis of ABR (Results from Two Clinical Trials)

Gene therapy with adeno-associated virus vector 5–human factor IX in adults with hemophilia B

Factor VIII Activity and Risk of Bleeding in Acquired Hemophilia a: Results from the Gth-AH 01/2010 Study

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American Society of Hematology

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American Society of Hematology

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