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Journal Articles

Higher Hemoglobin Levels Achieved with Voxelotor Are Associated with Lower Vaso-occlusive Crisis Incidence: 72-Week Analysis from the HOPE Study

Elliott Vichinsky, MD, Victor R. Gordeuk, MD, Paul Telfer, DM, FRCP, Adlette Inati, MD FAAP, Margaret Tonda, PharmD, Sarah Gray, PhD, Irene Agodoa, MD, Kenneth I. Ataga, MD

Blood (2020) 136 (Supplement 1): 31–32.

https://doi.org/10.1182/blood-2020-140863

Published: 2020

Journal Articles

In Utero Stem Cell Transplantation in Patients with Alpha Thalassemia Major: Interim Results of a Phase 1 Clinical Trial

Tippi C. MacKenzie, MD, Michela Frascoli, Renan Sper, Billie Rachael Lianoglou, MS, Juan Gonzalez Velez, Christopher C. Dvorak, MD, Sandhya Kharbanda, MD, Elliott Vichinsky, MD

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

https://doi.org/10.1182/blood-2020-142698

Published: 2020

Journal Articles

Efficacy and Safety of Voxelotor in Adolescents and Adults with Sickle Cell Disease: HOPE Trial 72-Week Analysis

Jo Howard, MBBChir, MRCP, FRCPath, Kenneth I. Ataga, MD, R. Clark Brown, MD PhD, Maureen Achebe, MD MPH, Videlis Nduba, MD, Amal El-Beshlawy, MD, Hoda Hassab, MD, Irene Agodoa, MD, Margaret Tonda, PharmD, Sarah Gray, PhD, Joshua Lehrer-Graiwer, MD MPhil, FACC, Elliott Vichinsky, MD

Blood (2020) 136 (Supplement 1): 19.

https://doi.org/10.1182/blood-2020-140553

Published: 2020

Journal Articles

Correlation of Voxelotor Exposure with Hemoglobin Response and Measures of Hemolysis in Patients from the HOPE Study

Jo Howard, MB, BChir, Elliott Vichinsky, MD UCSF, Jennifer Knight-Madden, MBBS, PhD, Margaret Tonda, PharmD, Carla Washington, Barbara Tong, PhD, Joshua Lehrer-Graiwer, MD MPhil, FACC, Victor R Gordeuk, MD

Blood (2019) 134 (Supplement_1): 1020.

https://doi.org/10.1182/blood-2019-130802

Published: 2019

Journal Articles

Trends in Iron Overload over Past Two Decades: Results from the Natural History of Iron Burden Study with the SQUID Biosusceptometer

Ashutosh Lal, MD, Roland Fischer, PhD, Elliott Vichinsky, MD UCSF, Marcela Weyhmiller, PhD

Blood (2019) 134 (Supplement_1): 961.

https://doi.org/10.1182/blood-2019-129701

Published: 2019

Journal Articles

Results from Part A of the Hemoglobin Oxygen Affinity Modulation to Inhibit HbS Polymerization (HOPE) Trial (GBT440-031), a Placebo-Controlled Randomized Study Evaluating Voxelotor (GBT440) in Adults and Adolescents with Sickle Cell Disease

Blood (2018) 132 (Supplement 1): 505.

https://doi.org/10.1182/blood-2018-99-118508

Published: 2018

Journal Articles

Significantly Improved Long Term Health Related Quality of Life (HRQL) and Neurocognition Following Familial Haploidentical Stem Cell Transplantation (HISCT) Utilizing CD34 Enrichment and Mononuclear (CD3) Addback in High Risk Patients with Sickle Cell Disease (SCD)

Blood (2018) 132 (Supplement 1): 162.

https://doi.org/10.1182/blood-2018-162

Published: 2018

Journal Articles

Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia Following Completion of the Northstar HGB-204 Study

John E.J. Rasko, MBBS, PhD, Alexis A. Thompson, MD MPH, Janet L. Kwiatkowski, MD, Suradej Hongeng, MD, Gary J. Schiller, MD, Usanarat Anurathapan, MD, Marina Cavazzana, MD PhD, P. Joy Ho, MBBS DPhil FRACP FRCPA, Manfred Schmidt, PhD, Morris Kletzel, MD MBA, Philippe Leboulch, MD, Elliott Vichinsky, MD, Briana Deary, Ying Chen, PhD, Mohammed Asmal, MD PhD, Mark C. Walters, MD

Blood (2018) 132 (Supplement 1): 167.

https://doi.org/10.1182/blood-2018-167

Published: 2018

Journal Articles

Systemic Biomarkers Show Elevated Oxidative Stress and Chronic Inflammation in Two Disorders of Neurodegeneration with Brain Iron Accumulation (NBIA)

Michael Minkley, BSc, Nancy Sweeters, PNP, Shiva Kasravi, BS, Jennifer Ferguson, BS MS, Annie Higa, BS, Angela Jackson, BSc MSc, Derek Smith, BS MS, Christoph Borchers, PhD, Raad Nashmi, PhD, Patrick Macleod, MD, Lynne D. Neumayr, MD, Elliott Vichinsky, MD, Patrick B Walter, MSc, PhD

Blood (2017) 130 (Supplement 1): 943.

https://doi.org/10.1182/blood.V130.Suppl_1.943.943

Published: 2017

Journal Articles

Clinical Outcomes up to 3 Years Following Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar Hgb-204 Study

Janet L. Kwiatkowski, MD, Alexis A Thompson, MD MPH, John Rasko, MBBS, PhD, Suradej Hongeng, MD, Gary J. Schiller, MD, Usanarat Anurathapan, Marina Cavazzana, MD PhD, Phoebe Joy Ho, Christof von Kalle, MD, Morris Kletzel, MD MBA, Philippe Leboulch, MD, Elliott Vichinsky, MD, Briana Deary, Mohammed Asmal, MD PhD, Mark C. Walters, MD

Blood (2017) 130 (Supplement 1): 360.

https://doi.org/10.1182/blood.V130.Suppl_1.360.360

Published: 2017

Journal Articles

Promising Results at 1 Year Follow-up Following Familial Haploidentical (FHI) T-Cell Depleted (TCD) with CD34 Enrichment and T-Cell (CD3) Addback Allogeneic Stem Cell Transplantation in Patients with High-Risk Sickle Cell Disease (SCD) (IND 14359)

Julie-An Talano, MD, Theodore B Moore, MD, Carolyn A. Keever-Taylor, DPhil, Shalini Shenoy, MD, Mark C. Walters, MD, Susan K Parsons, MD MRP, Allen J Dozor, MD, Deborah Friedman, MD, Qiuhu Shi, PhD, Suzanne Braniecki, PhD, Brenda J. Grossman, MD, Rona Singer Weinberg, PhD, Elliott Vichinsky, MD, Yaya Chu, PhD, Erin Morris, RN, BSN, Sandra Fabricatore, RN, PNP, MSN, Janet Ayello, MS, MT(ASCP), Lee Ann Baxter-Lowe, PhD, Mitchell S. Cairo, MD

Blood (2017) 130 (Supplement 1): 4602.

https://doi.org/10.1182/blood.V130.Suppl_1.4602.4602

Published: 2017

Journal Articles

Community Survey of Knowledge of Thalassemia

Jin Mei, BA, Robert Yamashita, PhD, Ashutosh Lal, MD, Nancy F. Olivieri, MD, Elliott Vichinsky, MD

Blood (2017) 130 (Supplement 1): 5580.

https://doi.org/10.1182/blood.V130.Suppl_1.5580.5580

Published: 2017

Journal Articles

County Level Surveillance of Sickle Cell Disease Population, Healthcare Utilization, and Fragmentation to Support Improvements in Access to Care

Susan T Paulukonis, MA, MPH, Elliott Vichinsky, MD, Marsha Treadwell, PhD, Mary Hulihan, PhD MPH

Blood (2017) 130 (Supplement 1): 3369.

https://doi.org/10.1182/blood.V130.Suppl_1.3369.3369

Published: 2017

Journal Articles

Pituitary Iron: Effect on Pituitary Volume and Function in Transfusion Dependent Thalassemia

Sylvia Titi Titi Singer, MD, Roland Fischer, PhD, Qing Yuan, PhD, Ashutosh Lal, MD, Isabel E Allen, PhD, Elliott Vichinsky, MD, Zhiyue J Wang, PhD

Blood (2017) 130 (Supplement 1): 3502.

https://doi.org/10.1182/blood.V130.Suppl_1.3502.3502

Published: 2017

Journal Articles

Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia: Update from the Northstar Hgb-204 Phase 1/2 Clinical Study

Alexis A. Thompson, MD MPH, Janet Kwiatkowski, MD, John Rasko, MBBS, PhD, Suradej Hongeng, MD, Gary J. Schiller, MD, Usanarat Anurathapan, MD, Marina Cavazzana, MD PhD, P. Joy Ho, Christof von Kalle, MD, Morris Kletzel, MD FAAP, MBA, Philippe Leboulch, MD, Elliott Vichinsky, MD, Alexandria Petrusich, Mohammed Asmal, MD PhD, Mark C. Walters, MD

Blood (2016) 128 (22): 1175.

https://doi.org/10.1182/blood.V128.22.1175.1175

Published: 2016

Journal Articles

Episodic Patterns of High Emergency Department Utilization Among Sickle Cell Disease Patients

Susan Paulukonis, MA, MPH, Lisa Feuchtbaum, DrPH, MPH, Elliott Vichinsky, MD, Mary Hulihan, MPH, PhD

Blood (2016) 128 (22): 316.

https://doi.org/10.1182/blood.V128.22.316.316

Published: 2016

Journal Articles

Threshold Ferritin Values to Predict Control of Liver Iron Burden in Thalassemia

Ashutosh Lal, MD, Marcela G Weyhmiller, PhD, Elliott Vichinsky, MD

Blood (2016) 128 (22): 4824.

https://doi.org/10.1182/blood.V128.22.4824.4824

Published: 2016

Journal Articles

Iron Level and Monocyte Morphology Predict TLR4 Expression and Reactive Oxygen Species Production Which Influences Chronic Inflammation in β-Thalassemia

Patrick B. Walter, PhD, Nick Slater, BS, Paul Harmatz, MD, Annie Higa, BS, Vivian Ng, BS, Marcels Weyhmiller, Patricia Evans, PhD, John B Porter, Nancy Sweeters, Jackson Price, BS, David Killilea, PhD, Lynne Neumayr, MD, Ashutosh Lal, MD, Elliott Vichinsky, MD

Blood (2015) 126 (23): 950.

https://doi.org/10.1182/blood.V126.23.950.950

Published: 2015

Journal Articles

Update of Results from the Northstar Study (HGB-204): A Phase 1/2 Study of Gene Therapy for Beta-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex-Vivo with a Lentiviral Beta AT87Q-Globin Vector (LentiGlobin BB305 Drug Product)

Mark C. Walters, MD, John Rasko, MBBS, PhD, Suradej Hongeng, MD, Janet Kwiatkowski, MD, Gary J Schiller, MD, Morris Kletzel, MD, P. Joy Ho, MBBS, DPhil, Elliott Vichinsky, MD, Christof von Kalle, MD, Marina Cavazzana, MD PhD, Philippe Leboulch, MD, Alexandria Petrusich, Sandeep Soni, MD, Alexis A. Thompson, MD

Blood (2015) 126 (23): 201.

https://doi.org/10.1182/blood.V126.23.201.201

Published: 2015

Journal Articles

The Effects of Glutamine Supplementation on Markers of Autophagy and Apoptosis in Peripheral Blood Mononuclear Cells from Patients with Sickle Cell Disease

Patrick B Walter, PhD, Leah Hohman, BS, Andrew Rokeby, BS, Julian Lum, PhD, Robert Hagar, MD, Melinee Stewart, BS, Lisa Lavrisha, BS, Elliott Vichinsky, MD, Claudia R. Morris, MD

Blood (2015) 126 (23): 3412.

https://doi.org/10.1182/blood.V126.23.3412.3412

Published: 2015

NARROW

Higher Hemoglobin Levels Achieved with Voxelotor Are Associated with Lower Vaso-occlusive Crisis Incidence: 72-Week Analysis from the HOPE Study

In Utero Stem Cell Transplantation in Patients with Alpha Thalassemia Major: Interim Results of a Phase 1 Clinical Trial

Efficacy and Safety of Voxelotor in Adolescents and Adults with Sickle Cell Disease: HOPE Trial 72-Week Analysis

Correlation of Voxelotor Exposure with Hemoglobin Response and Measures of Hemolysis in Patients from the HOPE Study

Trends in Iron Overload over Past Two Decades: Results from the Natural History of Iron Burden Study with the SQUID Biosusceptometer

Results from Part A of the Hemoglobin Oxygen Affinity Modulation to Inhibit HbS Polymerization (HOPE) Trial (GBT440-031), a Placebo-Controlled Randomized Study Evaluating Voxelotor (GBT440) in Adults and Adolescents with Sickle Cell Disease

Significantly Improved Long Term Health Related Quality of Life (HRQL) and Neurocognition Following Familial Haploidentical Stem Cell Transplantation (HISCT) Utilizing CD34 Enrichment and Mononuclear (CD3) Addback in High Risk Patients with Sickle Cell Disease (SCD)

Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia Following Completion of the Northstar HGB-204 Study

Systemic Biomarkers Show Elevated Oxidative Stress and Chronic Inflammation in Two Disorders of Neurodegeneration with Brain Iron Accumulation (NBIA)

Clinical Outcomes up to 3 Years Following Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar Hgb-204 Study

Promising Results at 1 Year Follow-up Following Familial Haploidentical (FHI) T-Cell Depleted (TCD) with CD34 Enrichment and T-Cell (CD3) Addback Allogeneic Stem Cell Transplantation in Patients with High-Risk Sickle Cell Disease (SCD) (IND 14359)

Community Survey of Knowledge of Thalassemia

County Level Surveillance of Sickle Cell Disease Population, Healthcare Utilization, and Fragmentation to Support Improvements in Access to Care

Pituitary Iron: Effect on Pituitary Volume and Function in Transfusion Dependent Thalassemia

Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia: Update from the Northstar Hgb-204 Phase 1/2 Clinical Study

Episodic Patterns of High Emergency Department Utilization Among Sickle Cell Disease Patients

Threshold Ferritin Values to Predict Control of Liver Iron Burden in Thalassemia

Iron Level and Monocyte Morphology Predict TLR4 Expression and Reactive Oxygen Species Production Which Influences Chronic Inflammation in β-Thalassemia

Update of Results from the Northstar Study (HGB-204): A Phase 1/2 Study of Gene Therapy for Beta-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex-Vivo with a Lentiviral Beta AT87Q-Globin Vector (LentiGlobin BB305 Drug Product)

The Effects of Glutamine Supplementation on Markers of Autophagy and Apoptosis in Peripheral Blood Mononuclear Cells from Patients with Sickle Cell Disease

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American Society of Hematology

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American Society of Hematology

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