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Journal Articles

ENERGIZE-T: A Global, Phase 3, Double-Blind, Randomized, Placebo-Controlled Study of Mitapivat in Adults with Transfusion-Dependent Alpha- or Beta-Thalassemia

Blood (2024) 144 (Supplement 1): 409.

https://doi.org/10.1182/blood-2024-200867

Published: 2024

Journal Articles

Efficacy and Safety of Luspatercept in Patients Enrolled in the BELIEVE Trial: Data from the Phase 3b Long-Term Rollover Study

Maria Domenica Cappellini, Ali T Taher, Vip Viprakasit, Pencho Georgiev, Kevin H.M. Kuo, Natalia Holot, Valérie Vilmont, Loyse Felber Medlin, Wen-Ling Kuo, Yinzhi Lai, Luciana Moro Bueno, Sujit Sheth

Blood (2023) 142 (Supplement 1): 3849.

https://doi.org/10.1182/blood-2023-174089

Published: 2023

Journal Articles

Individualized Dosing of Ropeginterferon Alfa-2b Ensures Optimal Response in Patients with Low-Risk Polycythemia Vera (PV)

Heinz Gisslinger, Christoph Klade, Pencho Georgiev, Dorota Krochmalczyk, Liana Gercheva-Kyuchukova, Miklos Egyed, Petr Dulicek, Arpad Illes, Halyna Pylypenko, Liliya Sivcheva, Jiri Mayer, Vera Yablokova, Kurt Krejcy, Victoria Empson, Hans Hasselbalch, Robert Kralovics, Jean-Jacques Kiladjian

Blood (2023) 142 (Supplement 1): 4563.

https://doi.org/10.1182/blood-2023-173499

Published: 2023

Journal Articles

Efficacy and Safety of Long-Term Ropeginterferon Alfa-2b Treatment in Patients with Low-Risk and High-Risk Polycythemia Vera (PV)

Jean-Jacques Kiladjian, Christoph Klade, Pencho Georgiev, Dorota Krochmalczyk, Liana Gercheva-Kyuchukova, Miklos Egyed, Petr Dulicek, Arpad Illes, Halyna Pylypenko, Lylia Sivcheva, Jiří Mayer, Vera Yablokova, Kurt Krejcy, Victoria Empson, Hans Carl Hasselbalch, Robert Kralovics, Heinz Gisslinger

Blood (2022) 140 (Supplement 1): 9663–9664.

https://doi.org/10.1182/blood-2022-156889

Published: 2022

Journal Articles

Polycythemia Vera Patients Respond Better to Ropeginterferon Alfa-2b Than HU/BAT Irrespective of Pretreatment or Mutational Status; Results from 5 Years' Treatment in a Randomized, Controlled Setting in the PROUD-PV/Continuation-PV Trials

Heinz Gisslinger, Christoph Klade, Pencho Georgiev, Dorota Krochmalczyk, Liana Gercheva-Kyuchukova, Miklos Egyed, Petr Dulicek, Arpad Illes, Halyna Pylypenko, Lylia Sivcheva, Jiri Mayer, Vera Yablokova, Kurt Krejcy, Victoria Empson, Hans C. Hasselbalch, Robert Kralovics, Jean-Jacques Kiladjian

Blood (2021) 138 (Supplement 1): 3660.

https://doi.org/10.1182/blood-2021-152328

Published: 2021

Journal Articles

Long-Term Use of Ropeginterferon Alpha-2b in Polycythemia Vera: 5-Year Results from a Randomized Controlled Study and Its Extension

Heinz Gisslinger, MD, Christoph Klade, PhD, Pencho Georgiev, MD, Dorota Krochmalczyk, MD, Liana Gercheva-Kyuchukova, MD, Miklos Egyed, MD, Petr Dulicek, MD, Árpád Illés, MD PhD, Halyna Pylypenko, MD, Lylia Sivcheva, MD, Jiří Mayer, Vera Yablokova, MD, Kurt Krejcy, MD, Victoria Empson, MSc, Hans C. Hasselbalch, MD, Robert Kralovics, PhD, Jean Jacques Kiladjian, MD PhD

Blood (2020) 136 (Supplement 1): 33.

https://doi.org/10.1182/blood-2020-136973

Published: 2020

Journal Articles

Thromboembolic Risk Reduction and High Rate of Complete Molecular Response with Long-Term Use of Ropeginterferon Alpha-2b in Polycythemia Vera: Results from a Randomized Controlled Study

Jean-Jacques Kiladjian, MD PhD, Christoph Klade, PhD, Pencho Georgiev, MD, Dorota Krochmalczyk, MD, Liana Gercheva-Kyuchukova, MD, Miklos Egyed, Viktor Rossiev, MD, Petr Dulicek, MD, Árpád Illés, MD D.Sc, Halyna Pylypenko, MD, Lylia Sivcheva, MD, Jiri Mayer, MD, Vera Yablokova, MD, Kurt Krejcy, MD, Hans Hasselbalch, MD, Robert Kralovics, PhD, Heinz Gisslinger, MD

Blood (2019) 134 (Supplement_1): 553.

https://doi.org/10.1182/blood-2019-122233

Published: 2019

Journal Articles

The Believe Trial: Results of a Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Luspatercept in Adult Beta-Thalassemia Patients Who Require Regular Red Blood Cell (RBC) Transfusions

Blood (2018) 132 (Supplement 1): 163.

https://doi.org/10.1182/blood-2018-163

Published: 2018

Journal Articles

The Role of Antithrombin Lowering in Patients with Hemophilia: Hemostatic Control Pre- and Post-Fitusiran Dosing Interruption

Margaret V. Ragni, MD MPH, Pencho Georgiev, MD, Michael Desmond Creagh, Toshko Jelev Lissitchkov, MD, Steve K. Austin, BMedSci, MBBS, Charles Richard Morris Hay, MDFRCP, FRCPath, Inga Hegemann, MD, Syed Rashid Saeed Kazmi, MBBS, Pratima Chowdary, MD, Savita Rangarajan, MBBS, MD, Meredith C. Foster, ScD, Qifeng Yu, PhD, Craig Benson, MD MS, Kate Madigan, MD, Huy Van Nguyen, PharmD, Shariq Ali, PhD, Ketaki Kadam, MS, Pallavi Jain, PharmD, K. John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH

Blood (2018) 132 (Supplement 1): 2477.

https://doi.org/10.1182/blood-2018-99-112998

Published: 2018

Journal Articles

Evidence for Superior Efficacy and Disease Modification after Three Years of Prospective Randomized Controlled Treatment of Polycythemia Vera Patients with Ropeginterferon Alfa-2b Vs. HU/BAT

Heinz Gisslinger, Christoph Klade, PhD, Pencho Georgiev, MD, Dorota Krochmalczyk, Liana Gercheva-Kyuchukova, Miklos Egyed, MD PhD, Viktor Rossiev, Petr Dulicek, Arpad Illes, MD, Halyna Pylypenko, MD, Lylia Sivcheva, MD, Jiri Mayer, MD, Vera Yablokova, Kurt Krejcy, PhD, Barbara Grohmann-Izay, MD MSc, Hans Carl Hasselbalch, Professor, dr.MD, Robert Kralovics, PhD, Jean-Jacques Kiladjian, MD PhD

Blood (2018) 132 (Supplement 1): 579.

https://doi.org/10.1182/blood-2018-99-118715

Published: 2018

Journal Articles

Ropeginterferon Alfa-2b Induces High Rates of Clinical, Hematological and Molecular Responses in Polycythemia Vera: Two-Year Results from the First Prospective Randomized Controlled Trial

Heinz Gisslinger, Christoph Klade, PhD, Pencho Georgiev, MD, Dorota Krochmalczyk, Liana Gercheva, MD PhD, Miklos Egyed, MD PhD, Viktor Rossiev, Petr Dulicek, Árpád Illés, MD PhD, Halyna Pylypenko, MD, Lylia Sicheva, Jiri Mayer, Prof, MD SCs, Vera Yablokova, Kurt Krejcy, PhD, Barbara Grohmann-Izay, MD MSc, Hans Carl Hasselbalch, MD Professor, DMSc, Robert Kralovics, PhD, Jean-Jacques Kiladjian, MD PhD

Blood (2017) 130 (Supplement 1): 320.

https://doi.org/10.1182/blood.V130.Suppl_1.320.320

Published: 2017

Journal Articles

Perioperative Management in Patients with Hemophilia Receiving Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia

Claude Negrier, MD, Margaret V. Ragni, MD MPH, Pencho Georgiev, MD, Toshko Jelev Lissitchkov, MD PhD, Huy Van Nguyen, PharmD, Kate Madigan, K John Pasi

Blood (2017) 130 (Supplement 1): 2366.

https://doi.org/10.1182/blood.V130.Suppl_1.2366.2366

Published: 2017

Journal Articles

Final Results from PROUD-PV a Randomized Controlled Phase 3 Trial Comparing Ropeginterferon Alfa-2b to Hydroxyurea in Polycythemia Vera Patients

Heinz Gisslinger, MD, Christoph Klade, Pencho Georgiev, Aleksander Skotnicki, MD PhD, Liana Gercheva-Kyuchukova, MD, Miklos Egyed, Viktor Rossiev, MD, Petr Dulicek, Arpad Illes, MD PhD, Halyna Pylypenko, MD, Liliya Sivcheva, Jiri Mayer, MD, Barbara Grohmann-Izay, MD, Hans Hasselbalch, MD, Robert Kralovics, Jean-Jacques Kiladjian, MD PhD

Blood (2016) 128 (22): 475.

https://doi.org/10.1182/blood.V128.22.475.475

Published: 2016

Journal Articles

Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: Updated Results from a Phase 1 and Phase 1/2 Extension Study in Patients without Inhibitors

Margaret V. Ragni, MD, Pencho Georgiev, Tim Mant, Michael Desmond Creagh, Toshko Lissitchkov, David Bevan, Steve Austin, Charles R Hay, Inga Hegemann, Rashid Kazmi, Pratima Chowdary, Savita Rangarajan, Chang-Heok Soh, Akin Akinc, Angela M. Partisano, Benny Sorenson, K John Pasi

Blood (2016) 128 (22): 2572.

https://doi.org/10.1182/blood.V128.22.2572.2572

Published: 2016

Journal Articles

Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: Updated Results from a Phase 1 and Phase 1/2 Extension Study in Patients with Inhibitors

K John Pasi, Pencho Georgiev, Tim Mant, Toshko Lissitchkov, Michael Desmond Creagh, David Bevan, Steve Austin, Charles R Hay, Inga Hegemann, Rashid Kazmi, Pratima Chowdary, Margaret V. Ragni, MD, Chang-Heok Soh, Akin Akinc, Angela M. Partisano, Benny Sorenson, Savita Rangarajan

Blood (2016) 128 (22): 1397.

https://doi.org/10.1182/blood.V128.22.1397.1397

Published: 2016

Journal Articles

A Subcutaneously Administered Investigational RNAi Therapeutic (ALN-AT3) Targeting Antithrombin for Treatment of Hemophilia: Interim Weekly and Monthly Dosing Results in Patients with Hemophilia A or B

K John Pasi, Pencho Georgiev, Tim Mant, Michael Desmond Creagh, Toshko Lissitchkov, MD, David Bevan, Steve Austin, Charles R Hay, Brigit Brand, Rashid Kazmi, Pratima Chowdary, Margaret V Ragni, Jihong Chen, Akin Akinc, Benny Sorensen, Savita Rangarajan

Blood (2015) 126 (23): 551.

https://doi.org/10.1182/blood.V126.23.551.551

Published: 2015

NARROW

ENERGIZE-T: A Global, Phase 3, Double-Blind, Randomized, Placebo-Controlled Study of Mitapivat in Adults with Transfusion-Dependent Alpha- or Beta-Thalassemia

Efficacy and Safety of Luspatercept in Patients Enrolled in the BELIEVE Trial: Data from the Phase 3b Long-Term Rollover Study

Individualized Dosing of Ropeginterferon Alfa-2b Ensures Optimal Response in Patients with Low-Risk Polycythemia Vera (PV)

Efficacy and Safety of Long-Term Ropeginterferon Alfa-2b Treatment in Patients with Low-Risk and High-Risk Polycythemia Vera (PV)

Polycythemia Vera Patients Respond Better to Ropeginterferon Alfa-2b Than HU/BAT Irrespective of Pretreatment or Mutational Status; Results from 5 Years' Treatment in a Randomized, Controlled Setting in the PROUD-PV/Continuation-PV Trials

Long-Term Use of Ropeginterferon Alpha-2b in Polycythemia Vera: 5-Year Results from a Randomized Controlled Study and Its Extension

Thromboembolic Risk Reduction and High Rate of Complete Molecular Response with Long-Term Use of Ropeginterferon Alpha-2b in Polycythemia Vera: Results from a Randomized Controlled Study

The Believe Trial: Results of a Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Luspatercept in Adult Beta-Thalassemia Patients Who Require Regular Red Blood Cell (RBC) Transfusions

The Role of Antithrombin Lowering in Patients with Hemophilia: Hemostatic Control Pre- and Post-Fitusiran Dosing Interruption

Evidence for Superior Efficacy and Disease Modification after Three Years of Prospective Randomized Controlled Treatment of Polycythemia Vera Patients with Ropeginterferon Alfa-2b Vs. HU/BAT

Ropeginterferon Alfa-2b Induces High Rates of Clinical, Hematological and Molecular Responses in Polycythemia Vera: Two-Year Results from the First Prospective Randomized Controlled Trial

Perioperative Management in Patients with Hemophilia Receiving Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia

Final Results from PROUD-PV a Randomized Controlled Phase 3 Trial Comparing Ropeginterferon Alfa-2b to Hydroxyurea in Polycythemia Vera Patients

Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: Updated Results from a Phase 1 and Phase 1/2 Extension Study in Patients without Inhibitors

Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: Updated Results from a Phase 1 and Phase 1/2 Extension Study in Patients with Inhibitors

A Subcutaneously Administered Investigational RNAi Therapeutic (ALN-AT3) Targeting Antithrombin for Treatment of Hemophilia: Interim Weekly and Monthly Dosing Results in Patients with Hemophilia A or B

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American Society of Hematology

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American Society of Hematology

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