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Journal Articles

OEF Measured with Diffuse Optical Spectroscopies As a Biomarker of Silent Stroke in Pediatric Sickle Cell Anemia

Rowan Brothers, Michael Arrington, Kristin Wubbena, Ayesha Quadri, Mariam Akbar, Katherine Turrentine, Eswar Damaraju, Richard Jones, Jack Knight-Scott, Bryan Philbrook, Clark Brown, Beatrice E Gee, Kirshma Khemani, Seung Yup Lee, Adam Goldman-Yassen, Erin Buckley

Blood (2024) 144 (Supplement 1): 3885.

https://doi.org/10.1182/blood-2024-199747

Published: 2024

Journal Articles

A Novel Direct Hemoglobin S Polymerization Inhibitor for the Treatment of Sickle Cell Disease - In Vivo Efficacy of Ilx-002 in Humanized Mice

Osheiza Abdulmalik, Kandace Gollomp, Veronica Bochenek, Conroy O Field, Mariana Macias, Benita Balogun, Salma Roland, Martin K Safo, Yan Zhang, Akua Donkor, Abdelsattar Omar, Moustafa El-Araby, David R. Light, Clark Brown, Andrew N Fleischman

Blood (2024) 144 (Supplement 1): 173.

https://doi.org/10.1182/blood-2024-202714

Published: 2024

Journal Articles

The influence of voxelotor on cerebral blood flow and oxygen extraction in pediatric sickle cell disease

Rowan O. Brothers, Katherine B. Turrentine, Mariam Akbar, Sydney Triplett, Hongting Zhao, Tara M. Urner, Adam Goldman-Yassen, Richard A. Jones, Jack Knight-Scott, Sarah S. Milla, Shasha Bai, Amy Tang, R. Clark Brown, Erin M. Buckley

Blood (2024) 143 (21): 2145–2151.

https://doi.org/10.1182/blood.2023022011

Published: 2024

Includes: Supplemental data

Journal Articles

Over 4 Years of Safety and Efficacy with Voxelotor Treatment for Patients with Sickle Cell Disease: Updated Results from an Open-Label Extension of the Phase 3 HOPE Trial

Maureen Okam Achebe, Hoda MA Hassab, Salam Al-Kindi, Clark Brown, Paul Telfer, Bart J. Biemond, Thokozeni Lipato, Mark Davis, Sarah Gray, Victor R. Gordeuk

Blood (2023) 142 (Supplement 1): 2527.

https://doi.org/10.1182/blood-2023-188616

Published: 2023

Journal Articles

Transcranial Doppler Screening of Children with Sickle Cell Disease for a Large, Multinational Interventional Study: Experience from the Phase 3 HOPE-Kids 2 Trial Investigating the Effect of Voxelotor Treatment on Transcranial Doppler Flow Velocity

Blood (2023) 142 (Supplement 1): 3871.

https://doi.org/10.1182/blood-2023-188677

Published: 2023

Journal Articles

A randomized clinical trial of the efficacy and safety of rivipansel for sickle cell vaso-occlusive crisis

Carlton D. Dampier, Marilyn Jo Telen, Ted Wun, R. Clark Brown, Payal Desai, Fuad El Rassi, Beng Fuh, Julie Kanter, Yves Pastore, Jennifer Rothman, James G. Taylor, VI, David Readett, Krupa M. Sivamurthy, Brinda Tammara, Li-Jung Tseng, Jay Nelson Lozier, Helen Thackray, John L. Magnani, Kathryn L. Hassell

Blood (2023) 141 (2): 168–179.

https://doi.org/10.1182/blood.2022015797

Published: 2023

Includes: Supplemental data

Journal Articles

Individualized, PK-Guided Dosing of Hydroxyurea Is Not Associated with Increased Hematologic Toxicity Compared to Weight-Based Initial Dosing: Interim Results from the Hops Trial

Abena Appiah-Kubi, Seethal A Jacob, Matthew M. Heeney, Clark Brown, Susan E. Creary, Christine R Hollenkamp, Emily Riehm Meier, Omar Niss, Connie M. Piccone, Maa-Ohui Quarmyne, Charles T. Quinn, Allison Remiker, Kay L Saving, Min Dong, Patrick T. McGann

Blood (2022) 140 (Supplement 1): 5416–5417.

https://doi.org/10.1182/blood-2022-158623

Published: 2022

Journal Articles

Hydroxyurea Dosing in Very Young Children with Sickle Cell Anemia: A Multicenter, Randomized, Controlled Trial (HUGKISS)

Meghna Dua, Winfred C. Wang, R. Clark Brown, Melissa A. McNaull, Zora R. Rogers, Martha Barton, Jane Hankins, Jeffrey Gossett, Julie Richardson, Jerlym S. Porter, Guolian Kang, Jeremie H. Estepp

Blood (2022) 140 (Supplement 1): 445–446.

https://doi.org/10.1182/blood-2022-160485

Published: 2022

Journal Articles

Cerebral Metabolic Stress Improves after Voxelotor Treatment in Pediatric Sickle Cell Disease

Rowan O. Brothers, Katherine B. Turrentine, Mariam Akbar, Sydney Triplett, Tara M. Urner, Amy Y. Tang, Clark Brown, Erin M. Buckley

Blood (2022) 140 (Supplement 1): 8296–8297.

https://doi.org/10.1182/blood-2022-169025

Published: 2022

Journal Articles

Hydroxyurea Dosing in Very Young Children (HUGKISS): Challenges of Study Design

Meghna Dua, Winfred C. Wang, Timothy McCavit, Suvankar Majumdar, R. Clark Brown, Zora R. Rogers, Jeffrey Gossett, Guolian Kang, Jeremie H. Estepp

Blood (2022) 140 (Supplement 1): 11108–11109.

https://doi.org/10.1182/blood-2022-165760

Published: 2022

Journal Articles

Active Study: Impact of Voxelotor on Sleep Quality, Physical Activity, and Patient-Reported Outcomes

Nirmish Shah, Clark Brown, Biree Andemariam, Modupe Idowu, Alexander Glaros, Jeffrey Glassberg, Robert M Cronin, Barbara Moehring, Carolyn Hoppe, Sandy Dixon, Deepa Manwani

Blood (2022) 140 (Supplement 1): 5411–5413.

https://doi.org/10.1182/blood-2022-168317

Published: 2022

Journal Articles

Etavopivat, an Allosteric Activator of Pyruvate Kinase-R, Improves Sickle RBC Functional Health and Survival and Reduces Systemic Markers of Inflammation and Hypercoagulability in Patients with Sickle Cell Disease: An Analysis of Exploratory Studies in a Phase 1 Study

Theodosia A. Kalfa, Marilyn J. Telen, Santosh L. Saraf, R. Clark Clark Brown, Katie Giger Seu, Sandra K. Larkin, Maria D. Ribadeneira, Patricia Schroeder, Eric Wu, Patrick Kelly, Frans A. Kuypers

Blood (2021) 138 (Supplement 1): 8.

https://doi.org/10.1182/blood-2021-147078

Published: 2021

Journal Articles

Activation of Pyruvate Kinase-R with Etavopivat (FT-4202) Is Well Tolerated, Improves Anemia, and Decreases Intravascular Hemolysis in Patients with Sickle Cell Disease Treated for up to 12 Weeks

R. Clark Clark Brown, Santosh L. Saraf, Kimberly Cruz, Modupe Idowu, Theodosia A. Kalfa, James Geib, Sanjeev Forsyth, Patricia Schroeder, Eric Wu, Patrick Kelly, Marilyn J. Telen

Blood (2021) 138 (Supplement 1): 9.

https://doi.org/10.1182/blood-2021-147091

Published: 2021

Journal Articles

Initial Safety and Efficacy Results from the Phase II, Multicenter, Open-Label Solace-Kids Trial of Crizanlizumab in Adolescents with Sickle Cell Disease (SCD)

Matthew M. Heeney, David C. Rees, Mariane De Montalembert, Isaac Odame, R. Clark Clark Brown, Yasser Wali, Thu Thuy Nguyen, Du Lam, Nadege Pfender, Julie Kanter

Blood (2021) 138 (Supplement 1): 12.

https://doi.org/10.1182/blood-2021-144730

Published: 2021

Journal Articles

Trial in Progress: A Phase 2, Open-Label Study Evaluating the Safety and Efficacy of the PKR Activator Etavopivat (FT-4202) in Patients with Thalassemia or Sickle Cell Disease

Ashutosh Lal, R. Clark Clark Brown, Thomas D. Coates, Theodosia A. Kalfa, Janet L. Kwiatkowski, Julie Brevard, Von Potter, Ken W. Wood, Sujit Sheth

Blood (2021) 138 (Supplement 1): 4162.

https://doi.org/10.1182/blood-2021-145752

Published: 2021

Journal Articles

Long-Term Safety and Efficacy of Voxelotor for Patients with Sickle Cell Disease: Results from an Open-Label Extension of the Phase 3 HOPE Trial

Maureen Achebe, Hoda Hassab, Salam Alkindi, R. Clark Clark Brown, Paul Telfer, Bart J. Biemond, Victor R. Gordeuk, Thokozeni Lipato, Margaret Tonda, Sarah Gray, Joanna Howard

Blood (2021) 138 (Supplement 1): 3114.

https://doi.org/10.1182/blood-2021-153582

Published: 2021

Journal Articles

GBT021601, a Next Generation HbS Polymerization Inhibitor: Results of Safety, Tolerability, Pharmacokinetics and Pharmacodynamics in Adults Living with Sickle Cell Disease and Healthy Volunteers

R. Clark Clark Brown, Andrew Redfern, Eleanor Lisbon, Carla Washington, Irene Agodoa, Kim Smith-Whitley

Blood (2021) 138 (Supplement 1): 3099.

https://doi.org/10.1182/blood-2021-152924

Published: 2021

Journal Articles

Study Design and Initial Baseline Characteristics in Solace-Kids: Crizanlizumab in Pediatric Patients with Sickle Cell Disease

Matthew M. Heeney, MD, David C. Rees, MD, Mariane de Montalembert, MD PhD, Isaac Odame, MD, R. Clark Brown, MD PhD, Yasser Wali, MD, Thu Thuy Nguyen, Du Lam, MD, Raquel Merino Herranz, Julie Kanter, MD

Blood (2020) 136 (Supplement 1): 22–24.

https://doi.org/10.1182/blood-2020-137081

Published: 2020

Journal Articles

FT-4202, an Allosteric Activator of Pyruvate Kinase-R, Demonstrates Proof of Mechanism and Proof of Concept after a Single Dose and after Multiple Daily Doses in a Phase 1 Study of Patients with Sickle Cell Disease

R. Clark Brown, MD PhD, Kimberly Cruz, MD MS, Theodosia A. Kalfa, MD PhD, Frans A. Kuypers, PhD, Santosh L. Saraf, MD, Jeremie H. Estepp, MD, Luke R. Smart, MD, Punam Malik, MD, Mark Lerman, MD, Renae Mayer, MD, Maria D. Ribadeneira, PhD, Sanjeev Forsyth, MS, Patricia Schroeder, PhD, Eric Wu, PhD, Patrick Kelly, MD, Marilyn J. Telen, MD

Blood (2020) 136 (Supplement 1): 19–20.

https://doi.org/10.1182/blood-2020-134269

Published: 2020

Journal Articles

Early Initiation of Treatment with Rivipansel for Acute Vaso-Occlusive Crisis in Sickle Cell Disease (SCD) Achieves Earlier Discontinuation of IV Opioids and Shorter Hospital Stay: Reset Clinical Trial Analysis

Carlton D. Dampier, MD, Marilyn J. Telen, MD, Ted Wun, MD, Wally R Smith, MD, R. Clark Brown, MD PhD, Payal Desai, MD, Fuad A El Rassi, MD, Julie Kanter, MD, Beng R. Fuh, MD, Yves D. Pastore, MD, Jennifer A. Rothman, MD, James G. Taylor, MD, David Readett, BM, BS, MRCP, Jay N Lozier, MDPhD, John L. Magnani, PhD, Helen M Thackray, MD, Kathryn L. Hassell, MD

Blood (2020) 136 (Supplement 1): 18–19.

https://doi.org/10.1182/blood-2020-134803

Published: 2020

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OEF Measured with Diffuse Optical Spectroscopies As a Biomarker of Silent Stroke in Pediatric Sickle Cell Anemia

A Novel Direct Hemoglobin S Polymerization Inhibitor for the Treatment of Sickle Cell Disease - In Vivo Efficacy of Ilx-002 in Humanized Mice

The influence of voxelotor on cerebral blood flow and oxygen extraction in pediatric sickle cell disease

Over 4 Years of Safety and Efficacy with Voxelotor Treatment for Patients with Sickle Cell Disease: Updated Results from an Open-Label Extension of the Phase 3 HOPE Trial

Transcranial Doppler Screening of Children with Sickle Cell Disease for a Large, Multinational Interventional Study: Experience from the Phase 3 HOPE-Kids 2 Trial Investigating the Effect of Voxelotor Treatment on Transcranial Doppler Flow Velocity

A randomized clinical trial of the efficacy and safety of rivipansel for sickle cell vaso-occlusive crisis

Individualized, PK-Guided Dosing of Hydroxyurea Is Not Associated with Increased Hematologic Toxicity Compared to Weight-Based Initial Dosing: Interim Results from the Hops Trial

Hydroxyurea Dosing in Very Young Children with Sickle Cell Anemia: A Multicenter, Randomized, Controlled Trial (HUGKISS)

Cerebral Metabolic Stress Improves after Voxelotor Treatment in Pediatric Sickle Cell Disease

Hydroxyurea Dosing in Very Young Children (HUGKISS): Challenges of Study Design

Active Study: Impact of Voxelotor on Sleep Quality, Physical Activity, and Patient-Reported Outcomes

Etavopivat, an Allosteric Activator of Pyruvate Kinase-R, Improves Sickle RBC Functional Health and Survival and Reduces Systemic Markers of Inflammation and Hypercoagulability in Patients with Sickle Cell Disease: An Analysis of Exploratory Studies in a Phase 1 Study

Activation of Pyruvate Kinase-R with Etavopivat (FT-4202) Is Well Tolerated, Improves Anemia, and Decreases Intravascular Hemolysis in Patients with Sickle Cell Disease Treated for up to 12 Weeks

Initial Safety and Efficacy Results from the Phase II, Multicenter, Open-Label Solace-Kids Trial of Crizanlizumab in Adolescents with Sickle Cell Disease (SCD)

Trial in Progress: A Phase 2, Open-Label Study Evaluating the Safety and Efficacy of the PKR Activator Etavopivat (FT-4202) in Patients with Thalassemia or Sickle Cell Disease

Long-Term Safety and Efficacy of Voxelotor for Patients with Sickle Cell Disease: Results from an Open-Label Extension of the Phase 3 HOPE Trial

GBT021601, a Next Generation HbS Polymerization Inhibitor: Results of Safety, Tolerability, Pharmacokinetics and Pharmacodynamics in Adults Living with Sickle Cell Disease and Healthy Volunteers

Study Design and Initial Baseline Characteristics in Solace-Kids: Crizanlizumab in Pediatric Patients with Sickle Cell Disease

FT-4202, an Allosteric Activator of Pyruvate Kinase-R, Demonstrates Proof of Mechanism and Proof of Concept after a Single Dose and after Multiple Daily Doses in a Phase 1 Study of Patients with Sickle Cell Disease

Early Initiation of Treatment with Rivipansel for Acute Vaso-Occlusive Crisis in Sickle Cell Disease (SCD) Achieves Earlier Discontinuation of IV Opioids and Shorter Hospital Stay: Reset Clinical Trial Analysis

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