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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.

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.

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.

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.

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.

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.

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

Published: 2020

Journal Articles

Changes In Health Status and Quality of Life In Adults with Thalassemia: Year 1 Report of the Thalassemia Longitudinal Cohort Study.

Amy Sobota, MD, Robert Yamashita, Ph.D, Yan Xu, MS, Felicia Trachtenberg, PhD, Pamela Kohlbry, PhD, RN, Dorothy A Kleinert, NP, MPH, Patricia Giardina, MD, Janet L Kwiatkowski, MD, MSCE, Dru Haines, RN, CPNP, MSN, Vivekanandan Thayalasuthan, MD, John B. Porter, MA, MD, FRCP, FRCPath, Alexis A. Thompson, MD, MPH, Leann Schilling, MPH, Charles T. Quinn, MD, MS, Ellis J. Neufeld, MD, PhD

Blood (2010) 116 (21): 1533.

https://doi.org/10.1182/blood.V116.21.1533.1533

Published: 2010

Journal Articles

DMT1-Independent Uptake of Non-Transferrin Bound Iron into Human Hepatoma Cells Is Selectively Modulated by T-Type Voltage-Dependent Calcium Channel Inhibitors.

Roozina Rafique, BSc, PhD, Monica S.D. Mascarenhas, BSc, MSc, Bala S. Ramesh, BSc, PhD, Kaila S. Srai, BSc, PhD, John B. Porter, MA,MD,FRCP,FRCPath

Blood (2006) 108 (11): 1542.

https://doi.org/10.1182/blood.V108.11.1542.1542

Published: 2006

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

Changes In Health Status and Quality of Life In Adults with Thalassemia: Year 1 Report of the Thalassemia Longitudinal Cohort Study.

DMT1-Independent Uptake of Non-Transferrin Bound Iron into Human Hepatoma Cells Is Selectively Modulated by T-Type Voltage-Dependent Calcium Channel Inhibitors.

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American Society of Hematology

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ASH Publications

American Society of Hematology

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