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Journal Articles

Revisiting acute chest syndrome (ACS) associated with sickle cell disease (SCD) vaso-occlusive pain episodes (VOE): Insight from a prospective, multicenter Phase-3 randomized controlled trial

Blood (2025) 146 (Supplement 1): 2946.

https://doi.org/10.1182/blood-2025-2946

Published: 2025

Journal Articles

Secretory phospholipase A2 (sPLA2) during vaso-occlusive pain episodes (VOE) and acute chest syndrome (ACS) in sickle cell disease (SCD): Results from a prospective multi-center randomized controlled trial (RCT)

Blood (2025) 146 (Supplement 1): 2923.

https://doi.org/10.1182/blood-2025-2923

Published: 2025

Journal Articles

Sickle cell disease treatment with arginine therapy (STArT) – results of a phase-3 randomized controlled trial

Blood (2025) 146 (Supplement 1): 616.

https://doi.org/10.1182/blood-2025-616

Published: 2025

Journal Articles

The psychometric properties of the stoplight pain scale in children with sickle cell disease

Blood (2025) 146 (Supplement 1): 4722.

https://doi.org/10.1182/blood-2025-4722

Published: 2025

Journal Articles

Impact of chronic pain on acute pain trajectories in sickle cell disease: Insights from the sickle cell disease treatment with arginine therapy (STArT) randomized controlled trial

Blood (2025) 146 (Supplement 1): 6524.

https://doi.org/10.1182/blood-2025-6524

Published: 2025

Journal Articles

Uric acid - a potent antioxidant with protective and toxic affects on renal function

Robert Hagar, Shiran Chen, Sam Walters, Nicole Herrera, Ali Odeh, Elliott Vichinsky

Blood (2025) 146 (Supplement 1): 1173.

https://doi.org/10.1182/blood-2025-1173

Published: 2025

Journal Articles

An experiential learning intervention with community advisory boards to enhance clinical trial literacy in sickle cell disease: A baseline analysis

Tilicia Mayo-Gamble, Titilope Fasipe, Justine Nicholas, Elliott Vichinsky, Ellen Fung, Betty Pace, Abdullah Kutlar, Melanie Aviles, Olufunke Martin, Charles Abrams, Kenneth Rivlin

Blood (2025) 146 (Supplement 1): 4374.

https://doi.org/10.1182/blood-2025-4374

Published: 2025

Journal Articles

Long-Term Efficacy and Safety of the Oral Pyruvate Kinase Activator Mitapivat in Adults with Non-Transfusion-Dependent Alpha- or Beta-Thalassemia

Kevin H.M. Kuo, D. Mark Layton, Ashutosh Lal, Hanny Al-Samkari, Joy Bhatia, Penelope A. Kosinski, Bo Tong, Megan Lynch, Katrin Uhlig, Elliott Vichinsky

Blood (2021) 138 (Supplement 1): 576.

https://doi.org/10.1182/blood-2021-150386

Published: 2021

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

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

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

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

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

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

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

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

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Revisiting acute chest syndrome (ACS) associated with sickle cell disease (SCD) vaso-occlusive pain episodes (VOE): Insight from a prospective, multicenter Phase-3 randomized controlled trial

Secretory phospholipase A2 (sPLA2) during vaso-occlusive pain episodes (VOE) and acute chest syndrome (ACS) in sickle cell disease (SCD): Results from a prospective multi-center randomized controlled trial (RCT)

Sickle cell disease treatment with arginine therapy (STArT) – results of a phase-3 randomized controlled trial

The psychometric properties of the stoplight pain scale in children with sickle cell disease

Impact of chronic pain on acute pain trajectories in sickle cell disease: Insights from the sickle cell disease treatment with arginine therapy (STArT) randomized controlled trial

Uric acid - a potent antioxidant with protective and toxic affects on renal function

An experiential learning intervention with community advisory boards to enhance clinical trial literacy in sickle cell disease: A baseline analysis

Long-Term Efficacy and Safety of the Oral Pyruvate Kinase Activator Mitapivat in Adults with Non-Transfusion-Dependent Alpha- or Beta-Thalassemia

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

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

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

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

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

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)

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)

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

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