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Journal Articles

Favorable Outcomes in Pediatric Patients in the Phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) Studies of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia

Alexis A. Thompson, MD, Janet L. Kwiatkowski, MD MSCE, John B. Porter, MA, MD, FRCP, FRCPath, Suradej Hongeng, MD, Evangelia Yannaki, MD, Andreas E. Kulozik, MD PhD, Martin G. Sauer, MD, Adrian J. Thrasher, MBBS, PhD FMedSci, Isabelle Thuret, MD, Ashutosh Lal, MD, Ruiting Guo, MS, Weijian Liu, PhD, Richard A. Colvin, MD PhD, Mark C. Walters, MD, Franco Locatelli, MD PhD

Blood (2020) 136 (Supplement 1): 52–54.

DOI: https://doi.org/10.1182/blood-2020-135857

Published: 2020

Journal Articles

Health-Related Quality of Life Outcomes for Patients with Transfusion-Dependent Beta-Thalassemia Treated with Luspatercept in the Believe Trial

Maria Domenica Cappellini, Ali T. Taher, Antonio Piga, MD, Farrukh Shah, Ersi Voskaridou, Vip Viprakasit, John B. Porter, MA, MD, FRCP, FRCPath, Olivier Hermine, Ellis J. Neufeld, Alexis A. Thompson, MD, Derek Tang, Peiwen Yu, Shien Guo, Jeevan K. Shetty, Dimana Miteva, Tatiana Zinger, Jay T. Backstrom, Esther Natalie Oliva, MD

Blood (2020) 136 (Supplement 1): 8–9.

DOI: https://doi.org/10.1182/blood-2020-136312

Published: 2020

Journal Articles

Sustained Reductions in Red Blood Cell (RBC) Transfusion Burden and Events in β-Thalassemia with Luspatercept: Longitudinal Results of the Believe Trial

Ali T. Taher, Vip Viprakasit, Olivier Hermine, John B. Porter, MA, MD, FRCP, FRCPath, Antonio Piga, MD, Kevin H.M. Kuo, MDMSc,FRCPC, Thomas D. Coates, MD, Ersi Voskaridou, Abderrahim Khelif, Antonis Kattamis, Jeevan K. Shetty, George Zhang, Yu (Olivia) Tian, Dimana Miteva, Tatiana Zinger, Derek Tang, Jay T. Backstrom, Maria Domenica Cappellini

Blood (2020) 136 (Supplement 1): 45–46.

DOI: https://doi.org/10.1182/blood-2020-140310

Published: 2020

Journal Articles

Longitudinal Effect of Luspatercept Treatment on Iron Overload and Iron Chelation Therapy (ICT) in Adult Patients (Pts) with β-Thalassemia in the Believe Trial

Olivier Hermine, Maria Domenica Cappellini, Ali T. Taher, Thomas D. Coates, MD, Vip Viprakasit, Ersi Voskaridou, Ashutosh Lal, MD, Hong Keng Liew, Silverio Perrotta, Abderrahim Khelif, Antonis Kattamis, Jeevan K. Shetty, George Zhang, Yu (Olivia) Tian, Dimana Miteva, Tatiana Zinger, Derek Tang, Jay T. Backstrom, John B. Porter, MA, MD, FRCP, FRCPath

Blood (2020) 136 (Supplement 1): 47–48.

DOI: https://doi.org/10.1182/blood-2020-136517

Published: 2020

Journal Articles

Early-Onset Osteopenia and Osteoporosis in Patients with Pyruvate Kinase Deficiency

Hanny Al-Samkari, MD, Rachael F. Grace, MD, Andreas Glenthoej, MD, Oliver Andres, MD, Wilma Barcellini, Frédéric Galactéros, MDPhD, Kevin H.M. Kuo, MDMSc,FRCPC, D. Mark Layton, MB BS, Marta Morado, PhD, Vip Viprakasit, Yan Dong, PhD, Feng Tai, PhD, Peter Hawkins, PhD, Sarah Gheuens, MD PhD, Chris Bowden, MD, John B. Porter, MA, MD, FRCP, FRCPath, Eduard van Beers, MD PhD

Blood (2020) 136 (Supplement 1): 30–32.

DOI: https://doi.org/10.1182/blood-2020-136598

Published: 2020

Journal Articles

Response of Patients with Transfusion-Dependent β-Thalassemia (TDT) to Betibeglogene Autotemcel (beti-cel; LentiGlobin for β-Thalassemia) Gene Therapy Based on HBB Genotype and Disease Genetic Modifiers

Mark C. Walters, MD, David H.K. Chui, MD FRCPC, John J Farrell, PhD, Ashutosh Lal, MD, Franco Locatelli, MD PhD, Janet L. Kwiatkowski, MD MSCE, John B. Porter, MA, MD, FRCP, FRCPath, Martin G. Sauer, MD, Isabelle Thuret, MD, Suradej Hongeng, MD, Andreas E. Kulozik, MD PhD, Adrian J. Thrasher, MBBS, PhD FMedSci, Evangelia Yannaki, MD, Julia Yang, Dustin Whitney, PhD, Alexandria Petrusich, Richard A. Colvin, MD PhD, Alexis A. Thompson, MD

Blood (2020) 136 (Supplement 1): 1–3.

DOI: https://doi.org/10.1182/blood-2020-137642

Published: 2020

Journal Articles

Dose-Dependent Iron Chelating Effects of Eltrombopag on in Vitro Human Megakaryopoiesis

Zhi-Jian Liu, PhD, Emoke Deschmann, MD, Haley E. Ramsey, PhDMSc, Henry Feldman, Bethan Psaila, MD PhD, Nichola Cooper, MD MRCP, Evangelia Vlachodimtropoulou Koumoutsea, MBBS,PhDBSc, John B. Porter, MA MDFRCP FRCPath, James B. Bussel, MD, Michael Georgieff, MD, Martha Sola-Visner, MD

Blood (2019) 134 (Supplement_1): 2475.

DOI: https://doi.org/10.1182/blood-2019-131895

Published: 2019

Journal Articles

NTBI Is Transiently Generated from Two Compartments during a Single Dose of Intravenous Iron - a Randomized Controlled Clinical Study

Maciej W Garbowski, MD PhD, Sukhvinder S Bansal, PhD, John B. Porter, MA MDFRCP FRCPath, Susanna Burckhardt, PhD, Robert Hider, PhD

Blood (2019) 134 (Supplement_1): 2234.

DOI: https://doi.org/10.1182/blood-2019-125618

Published: 2019

Journal Articles

Northstar-3: Interim Results from a Phase 3 Study Evaluating Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Either a β0 or IVS-I-110 Mutation at Both Alleles of the HBB Gene

Ashutosh Lal, MD, Franco Locatelli, MD PhD, Janet L. Kwiatkowski, MD MSCE, Andreas E. Kulozik, MD PhD, Evangelia Yannaki, MD, John B. Porter, MA MDFRCP FRCPath, Isabelle Thuret, MD, Martin G. Sauer, MD, Heidi Elliot, Ying Chen, PhD, Richard A. Colvin, MD PhD, Alexis A. Thompson, MD MPH

Blood (2019) 134 (Supplement_1): 815.

DOI: https://doi.org/10.1182/blood-2019-128482

Published: 2019

Journal Articles

Evaluating the Volumetric Liver Fat Fraction in Patients Referred for Investigation of Hyperferritinemia

Aoife Dervin, MBBS, BSc, MRCP, Perla Eleftheriou, FRCPath, MRCP, Deepak Suri, MBBS FRCP, Sara Trompeter, MBChB BSc MRCPCH FRCPath MSc, Maciej W Garbowski, MD PhD, Tim St. Pierre, John B. Porter, MA MDFRCP FRCPath, Emma Drasar, MBBS MRCP FRCPath PhD

Blood (2019) 134 (Supplement_1): 3539.

DOI: https://doi.org/10.1182/blood-2019-128122

Published: 2019

Journal Articles

Northstar-2: Updated Safety and Efficacy Analysis of Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Non-β0/β0 Genotypes

Alexis A. Thompson, MD MPH, Mark C. Walters, MD, Janet L. Kwiatkowski, MD MSCE, Suradej Hongeng, MD, John B. Porter, MA MDFRCP FRCPath, Martin G. Sauer, MD, Adrian J. Thrasher, MBBS, PhD FMedSci, Isabelle Thuret, MD, Heidi Elliot, Ge Tao, PhD, Richard A. Colvin, MD PhD, Franco Locatelli, MD PhD

Blood (2019) 134 (Supplement_1): 3543.

DOI: https://doi.org/10.1182/blood-2019-126046

Published: 2019

Journal Articles

Mitapivat (AG-348) in Adults with Pyruvate Kinase Deficiency Who Are Regularly Transfused: A Phase 3, Open-Label, Multicenter, Study (ACTIVATE-T) in Progress

Megan Lynch, MSN, Lei Hua, PhD, Chris Mix, MD, John B. Porter, MA MDFRCP FRCPath

Blood (2019) 134 (Supplement_1): 3526.

DOI: https://doi.org/10.1182/blood-2019-123107

Published: 2019

Journal Articles

Predicting Serum Ferritin Levels in Patients with Iron Overload Treated with the Film-Coated Tablet of Deferasirox during the Eclipse Study

Ali Taher, MDPhDFRCP, Sebastian Weber, Jackie Han, Andreas Bruederle, John B. Porter, BA MB BChir MRCP(Path) FRCP(Path) MD

Blood (2017) 130 (Supplement 1): 3508.

DOI: https://doi.org/10.1182/blood.V130.Suppl_1.3508.3508

Published: 2017

Journal Articles

Exploring the Clinical Utility of Renal Safety Biomarkers during Iron Chelation Therapy in Patients with β-Thalassemia and Other Anemias

Maria Domenica Cappellini, MD, Erhard Quebe-Fehling, PhD, Celine Pallaud, Frank Dieterle, John B. Porter, BA MB BChir MRCP(Path) FRCP(Path) MD

Blood (2017) 130 (Supplement 1): 4762.

DOI: https://doi.org/10.1182/blood.V130.Suppl_1.4762.4762

Published: 2017

Journal Articles

Results from the Hgb-207 (Northstar-2) Trial: A Phase 3 Study to Evaluate Safety and Efficacy of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia (TDT) in Patients with Non-β 0 /β 0 Genotypes

Mark C. Walters, MD, Suradej Hongeng, MD, Janet L. Kwiatkowski, MD, Franco Locatelli, MD PhD, John B. Porter, BA MB BChir MRCP(Path) FRCP(Path) MD, Martin G. Sauer, Adrian J Thrasher, MD PhD FMedSci, Isabelle Thuret, MD, Evangelia Yannaki, MD PhD, Heidi Elliot, Marisa Gayron, Mohammed Asmal, MD PhD, Alexis A Thompson, MD MPH

Blood (2017) 130 (Supplement 1): 526.

DOI: https://doi.org/10.1182/blood.V130.Suppl_1.526.526

Published: 2017

Journal Articles

Myocardial Hemosiderosis Correlates with Plasma NTBI Species That Represent Chelated Iron in Transfusion-Dependent Thalassemia

Maciej W. Garbowski, MD, Amna Abdel-Gadir, MBBS MRCP, James Moon, BA MBBCh MRCP MD, John B. Porter, BA MB BChir MRCP(Path) FRCP(Path) MD

Blood (2016) 128 (22): 203.

DOI: https://doi.org/10.1182/blood.V128.22.203.203

Published: 2016

Journal Articles

Eltrombopag (ELT) Reverses Iron Mediated Suppression of Insulin Secretion in Pancreatic Cells By Chelating Iron and Decreasing ROS

Evangelia Vlachodimitropoulou Koumoutsea, MBBS, Pimpisid Koonyosying, BSc, John B. Porter, BA MB BChir MRCP(Path) FRCP(Path) MD, Nichola Cooper, MA, MD MRCP, FRCPath, Bethan Psaila, Martha Sola-Visner, MD

Blood (2016) 128 (22): 1278.

DOI: https://doi.org/10.1182/blood.V128.22.1278.1278

Published: 2016

Journal Articles

Long-Term Dosing in Sickle Cell Disease Subjects with GBT440, a Novel HbS Polymerization Inhibitor

Josh Lehrer-Graiwer, MDMPhil, FACC, Jo Howard, MB, BChir, Claire Jane Hemmaway, MD, Moji Awogbade, Paul Telfer, FRCPath,MD, Mark Layton, MB, FRCP, John B. Porter, MD, Timothy Mant, MB, FFPM, FRCP, Kobina Dufu, PhD, Athiwat Hutchaleelaha, PhD, Donna Oksenberg, PhD, Mira Patel, PhD, Margaret Eleanor Tonda, PharmD, Kenneth Bridges, MD, Eleanor Ramos, MD

Blood (2016) 128 (22): 2488.

DOI: https://doi.org/10.1182/blood.V128.22.2488.2488

Published: 2016

Journal Articles

New Film-Coated Tablet Formulation of Deferasirox Is Well Tolerated in Patients with Thalassemia or MDS: Results of the Randomized, Phase II E.C.L.I.P.S.E. Study

Ali T. Taher, MD PhD, Raffaella Origa, Silverio Perrotta, Alexandra Kouraklis, Giovan Battista Ruffo, Antonis Kattamis, MD, Ai-Sim Goh, Annelore Cortoos, Vicky Huang, Marine Weill, Raquel Merino Herranz, John B. Porter, BA MB BChir MRCP(Path) FRCP(Path) MD

Blood (2016) 128 (22): 1285.

DOI: https://doi.org/10.1182/blood.V128.22.1285.1285

Published: 2016

Journal Articles

Improved Patient-Reported Outcomes with a Film-Coated Versus Dispersible Tablet Formulation of Deferasirox: Results from the Randomized, Phase II E.C.L.I.P.S.E. Study

Blood (2016) 128 (22): 850.

DOI: https://doi.org/10.1182/blood.V128.22.850.850

Published: 2016

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NARROW

Favorable Outcomes in Pediatric Patients in the Phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) Studies of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia

Health-Related Quality of Life Outcomes for Patients with Transfusion-Dependent Beta-Thalassemia Treated with Luspatercept in the Believe Trial

Sustained Reductions in Red Blood Cell (RBC) Transfusion Burden and Events in β-Thalassemia with Luspatercept: Longitudinal Results of the Believe Trial

Longitudinal Effect of Luspatercept Treatment on Iron Overload and Iron Chelation Therapy (ICT) in Adult Patients (Pts) with β-Thalassemia in the Believe Trial

Early-Onset Osteopenia and Osteoporosis in Patients with Pyruvate Kinase Deficiency

Response of Patients with Transfusion-Dependent β-Thalassemia (TDT) to Betibeglogene Autotemcel (beti-cel; LentiGlobin for β-Thalassemia) Gene Therapy Based on HBB Genotype and Disease Genetic Modifiers

Dose-Dependent Iron Chelating Effects of Eltrombopag on in Vitro Human Megakaryopoiesis

NTBI Is Transiently Generated from Two Compartments during a Single Dose of Intravenous Iron - a Randomized Controlled Clinical Study

Northstar-3: Interim Results from a Phase 3 Study Evaluating Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Either a β0 or IVS-I-110 Mutation at Both Alleles of the HBB Gene

Evaluating the Volumetric Liver Fat Fraction in Patients Referred for Investigation of Hyperferritinemia

Northstar-2: Updated Safety and Efficacy Analysis of Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Non-β0/β0 Genotypes

Mitapivat (AG-348) in Adults with Pyruvate Kinase Deficiency Who Are Regularly Transfused: A Phase 3, Open-Label, Multicenter, Study (ACTIVATE-T) in Progress

Predicting Serum Ferritin Levels in Patients with Iron Overload Treated with the Film-Coated Tablet of Deferasirox during the Eclipse Study

Exploring the Clinical Utility of Renal Safety Biomarkers during Iron Chelation Therapy in Patients with β-Thalassemia and Other Anemias

Results from the Hgb-207 (Northstar-2) Trial: A Phase 3 Study to Evaluate Safety and Efficacy of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia (TDT) in Patients with Non-β 0 /β 0 Genotypes

Myocardial Hemosiderosis Correlates with Plasma NTBI Species That Represent Chelated Iron in Transfusion-Dependent Thalassemia

Eltrombopag (ELT) Reverses Iron Mediated Suppression of Insulin Secretion in Pancreatic Cells By Chelating Iron and Decreasing ROS

Long-Term Dosing in Sickle Cell Disease Subjects with GBT440, a Novel HbS Polymerization Inhibitor

New Film-Coated Tablet Formulation of Deferasirox Is Well Tolerated in Patients with Thalassemia or MDS: Results of the Randomized, Phase II E.C.L.I.P.S.E. Study

Improved Patient-Reported Outcomes with a Film-Coated Versus Dispersible Tablet Formulation of Deferasirox: Results from the Randomized, Phase II E.C.L.I.P.S.E. Study

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American Society of Hematology

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American Society of Hematology

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