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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

Association between Hemophilia Joint Health Score and Quality of Life Using Results from the Xtend-1 Efanesoctocog Alfa Phase 3 Trial

Christoph Königs, Doris V. Quon, Cedric Hermans, Nana Kragh, Jérôme Msihid, Emilie Gerard, Duygu Bozkaya, Linda Bystrická, Cecile Le Camus, Elena Santagostino, Annemieke Willemze, Annette von Drygalski

Blood (2024) 144 (Supplement 1): 719.

https://doi.org/10.1182/blood-2024-193761

Published: 2024

Journal Articles

Once-Weekly Efanesoctocog Alfa Prophylaxis Provided High Sustained Factor VIII Activity Levels, Independent of Blood Group, in Children <12 Years of Age with Severe Hemophilia A

Flora Peyvandi, Cristina Tarango, Mindy Simpson, Manuela Albisetti, Maria Teresa Álvarez-Roman, Anthony KC Chan, Julie Curtin, Linda Bystrická, Elena Santagostino, Ekaterina Gresko, Sriya Gunawardena, Chandravathi Vage, Nancy Wong

Blood (2023) 142 (Supplement 1): 506.

https://doi.org/10.1182/blood-2023-188649

Published: 2023

Journal Articles

Treatment of Bleeding Episodes with Efanesoctocog Alfa in Children with Severe Hemophilia A in the XTEND-Kids Phase 3 Study

Lynn Malec, Amy Dunn, Manuel Carcao, Bulent Zulfikar, Ruben Berrueco, Simon Brown, Umer Khan, Sriya Gunawardena, Graham Neill, Lydia Abad-Franch, Linda Bystrická, Elena Santagostino, Sophie Susen

Blood (2023) 142 (Supplement 1): 3993.

https://doi.org/10.1182/blood-2023-188739

Published: 2023

Journal Articles

Efficacy of Efanesoctocog Alfa on Pain in Patients with Hemophilia A: Results from the XTEND-1 Phase 3 Clinical Trial in Previously Treated Patients with Hemophilia A

Amanda Wilson, Nana Kragh, Jerome Mshijid, Jennifer Dumont, Annemieke Willemze, Elena Santagostino, Chuanwu Zhang, Roshni Kulkarni, Annette von Drygalski, Pratima Chowdary

Blood (2022) 140 (Supplement 1): 5610–5611.

https://doi.org/10.1182/blood-2022-166849

Published: 2022

Journal Articles

Rationale and Design of an Observational Multicenter Study to Evaluate the Use and Effectiveness of Avatrombopag in Adult Patients with Immune Thrombocytopenia: The Adopt Study

Vickie McDonald, Axel Matzdorff, Åsa Mellbring, Jameel Nazir, Daniel Lindqvist, Elena Santagostino

Blood (2021) 138 (Supplement 1): 4210.

https://doi.org/10.1182/blood-2021-144690

Published: 2021

Journal Articles

Efficacy and Safety of BAY 94-9027 (Damoctocog Alfa Pegol) Prophylaxis in Patients with Severe Hemophilia a and Comorbidities: A Post Hoc Analysis of PROTECT VIII Data

Wolfgang A. Miesbach, Giovanni Di Minno, Elena Santagostino, Dr. Klamroth, Inga Bayh, Amaryllis Soto, Cedric Hermans

Blood (2019) 134 (Supplement_1): 1117.

https://doi.org/10.1182/blood-2019-128486

Published: 2019

Journal Articles

Improvement in Quality of Life Outcomes and Bleeding Rates among Patients Switching from On-demand FVIII to Prophylactic BAY 94-9027 in the PROTECT VIII Study

Jonathan M. Ducore, MD MPH, Shadan Lalezari, PhD, Elena Santagostino, MD PhD, Sanjay P Ahuja, MD, Monika Maas Enriquez, Claudia Tueckmantel, PhD, Mark Reding

Blood (2019) 134 (Supplement_1): 1129.

https://doi.org/10.1182/blood-2019-123947

Published: 2019

Journal Articles

Interim Analysis of the Extension Study with rVIII-SingleChain in Previously Untreated Patients (PUPs) with Severe Hemophilia A (CSL627-3001)

Elena Santagostino, Kathelijn Fischer, Christoph Koenigs, MD PhD, Claudia Djambas Khayat, MD BSc, Samantha Lucas, Blanca Salazar, Andres Brainsky, MD, Thomas Chung, Brahm Goldstein, Johnny Mahlangu, MMed, FCPath

Blood (2019) 134 (Supplement_1): 162.

https://doi.org/10.1182/blood-2019-123981

Published: 2019

Journal Articles

Timing of inhibitor development in more than 1000 previously untreated patients with severe hemophilia A

H. Marijke van den Berg, Kathelijn Fischer, Manuel Carcao, Hervé Chambost, Gili Kenet, Karin Kurnik, Chris Königs, Christoph Male, Elena Santagostino, Rolf Ljung

Blood (2019) 134 (3): 317–320.

https://doi.org/10.1182/blood.2019000658

Published: 2019

Includes: Supplemental data

Journal Articles

Characteristics of Bleed-Free Patients on Every-5-Day Dosing in the Protect VIII (BAY 94-9027) Study

Elena Santagostino, MD PhD, Bryce A. Kerlin, MD, Claude Negrier, Liu Tian, Dr, Jonathan M. Ducore, MD

Blood (2018) 132 (Supplement 1): 2486.

https://doi.org/10.1182/blood-2018-99-116031

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

Untreated Bleeds May Be Historically Under-Reported and More Prevalent in People with Hemophilia A with Inhibitors: An Examination of Bleed Data from a Prospective, Non-Interventional Study

Michael U. Callaghan, MD, Elina Asikanius, Michaela Lehle, Johannes Oldenburg, Johnny Mahlangu, Marianne Uguen, Sammy Chebon, Rebecca Kruse-Jarres, Victor Jimenez-Yuste, Midori Shima, MD PhD, Peter Trask, Christine L. Kempton, Craig M. Kessler, Gallia G. Levy, Elena Santagostino

Blood (2018) 132 (Supplement 1): 383.

https://doi.org/10.1182/blood-2018-99-118160

Published: 2018

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

Data Coming out of the Human Inhibitor PUP Study (HIPS) Reveal 4 Subgroups of Patients with Distinct Antibody Signatures

Blood (2018) 132 (Supplement 1): 3774.

https://doi.org/10.1182/blood-2018-99-115979

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

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

Recombinant Fusion Protein Linking Coagulation Factor IX with Albumin (RIX-FP) in Previously Treated Adolescents/Adults with Hemophilia B: Long-Term Efficacy and Safety from an Ongoing Phase 3b Extension Clinical Trial

Ingrid Pabinger, MD, Elena Santagostino, Andres Brainsky, MD, Jerry S. Powell, MD, Yanyan Li, PhD, Wilfried Seifert, PhD

Blood (2017) 130 (Supplement 1): 3690.

https://doi.org/10.1182/blood.V130.Suppl_1.3690.3690

Published: 2017

Journal Articles

Bleeding Events and Safety Outcomes in Pediatric Persons with Hemophilia A with Inhibitors: The First Non-Interventional Study (NIS) from a Real-World Setting

Johannes Oldenburg, Midori Shima, MDPhD, Rebecca Kruse-Jarres, Elena Santagostino, Johnny Mahlangu, Nives Selak Bienz, Sammy Chebon, Elina Asikanius, Gallia G. Levy

Blood (2017) 130 (Supplement 1): 1089.

https://doi.org/10.1182/blood.V130.Suppl_1.1089.1089

Published: 2017

Journal Articles

Nonacog Beta Pegol for Prophylactic Treatment of Children with Hemophilia B: Up to 3.5-Year Data from the Paradigm™5 Clinical Trial

Susan Kearney, MD, Elena Santagostino, Manuel Carcao, MD, Faraizah Abdul Karim, MD, Masashi Taki, MD PhD, Meng Yao Lu, MD, May-Lill Garly, MD PhD, Marek Zak, MD PhD

Blood (2017) 130 (Supplement 1): 1092.

https://doi.org/10.1182/blood.V130.Suppl_1.1092.1092

Published: 2017

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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

Association between Hemophilia Joint Health Score and Quality of Life Using Results from the Xtend-1 Efanesoctocog Alfa Phase 3 Trial

Once-Weekly Efanesoctocog Alfa Prophylaxis Provided High Sustained Factor VIII Activity Levels, Independent of Blood Group, in Children <12 Years of Age with Severe Hemophilia A

Treatment of Bleeding Episodes with Efanesoctocog Alfa in Children with Severe Hemophilia A in the XTEND-Kids Phase 3 Study

Efficacy of Efanesoctocog Alfa on Pain in Patients with Hemophilia A: Results from the XTEND-1 Phase 3 Clinical Trial in Previously Treated Patients with Hemophilia A

Rationale and Design of an Observational Multicenter Study to Evaluate the Use and Effectiveness of Avatrombopag in Adult Patients with Immune Thrombocytopenia: The Adopt Study

Efficacy and Safety of BAY 94-9027 (Damoctocog Alfa Pegol) Prophylaxis in Patients with Severe Hemophilia a and Comorbidities: A Post Hoc Analysis of PROTECT VIII Data

Improvement in Quality of Life Outcomes and Bleeding Rates among Patients Switching from On-demand FVIII to Prophylactic BAY 94-9027 in the PROTECT VIII Study

Interim Analysis of the Extension Study with rVIII-SingleChain in Previously Untreated Patients (PUPs) with Severe Hemophilia A (CSL627-3001)

Timing of inhibitor development in more than 1000 previously untreated patients with severe hemophilia A

Characteristics of Bleed-Free Patients on Every-5-Day Dosing in the Protect VIII (BAY 94-9027) 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

Untreated Bleeds May Be Historically Under-Reported and More Prevalent in People with Hemophilia A with Inhibitors: An Examination of Bleed Data from a Prospective, Non-Interventional Study

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

Data Coming out of the Human Inhibitor PUP Study (HIPS) Reveal 4 Subgroups of Patients with Distinct Antibody Signatures

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

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

Recombinant Fusion Protein Linking Coagulation Factor IX with Albumin (RIX-FP) in Previously Treated Adolescents/Adults with Hemophilia B: Long-Term Efficacy and Safety from an Ongoing Phase 3b Extension Clinical Trial

Bleeding Events and Safety Outcomes in Pediatric Persons with Hemophilia A with Inhibitors: The First Non-Interventional Study (NIS) from a Real-World Setting

Nonacog Beta Pegol for Prophylactic Treatment of Children with Hemophilia B: Up to 3.5-Year Data from the Paradigm™5 Clinical Trial

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