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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

Validation of BCL11A As Therapeutic Target in Sickle Cell Disease: Results from the Adult Cohort of a Pilot/Feasibility Gene Therapy Trial Inducing Sustained Expression of Fetal Hemoglobin Using Post-Transcriptional Gene Silencing

Erica B. Esrick, MD, Maureen Achebe, MD MPH, Myriam Armant, PhD, Pablo Bartolucci, MD PhD, Marioara Felicia Ciuculescu, MD, Heather Daley, Colleen Dansereau, MSN, RN, CPN, Giuseppe Di Caprio, PhD, Bronner Goncalves, MD PhD, Nicolas Hebert, Matthew M Heeney, MD, John M. Higgins, MD PhD, Leslie E. Lehmann, MD, John P Manis, MD, Olivier Negre, PhD, Sarah Nikiforow, MD PhD, Ethan Schonbrun, PhD, David K. Wood, PhD, David A. Williams, MD

Blood (2019) 134 (Supplement_2): LBA-5.

https://doi.org/10.1182/blood-2019-132745

Published: 2019

Journal Articles

Optimized Beta-Globin Expression and Enucleation from Induced Red Blood Cells for In Vitro Modeling of Sickle Cell Disease

Tolulope O Rosanwo, BA, Melissa Kinney, PhD, Martha A Clark, PhD, Linda T Vo, PhD, R. Grant Rowe, MD PhD, Natasha M. Archer, MD, Matthew M Heeney, MD, Thorsten Schlaeger, PhD, Daniel E. Bauer, MD PhD, Mark D. Fleming, MD DPhil, Manoj T Duraisingh, PhD, Stuart H. Orkin, MD, Carlo Brugnara, MD, Trista North, PhD, George Q. Daley, MD PhD

Blood (2018) 132 (Supplement 1): 2359.

https://doi.org/10.1182/blood-2018-99-114136

Published: 2018

Journal Articles

Flipping the Switch: Initial Results of Genetic Targeting of the Fetal to Adult Globin Switch in Sickle Cell Patients

Blood (2018) 132 (Supplement 1): 1023.

https://doi.org/10.1182/blood-2018-99-116733

Published: 2018

Journal Articles

Hscb , a Mitochondrial Iron-Sulfur Cluster Assembly Co-Chaperone, Is a Novel Candidate Gene for Congenital Sideroblastic Anemia

Andrew Crispin, BS, Paul Schmidt, PhD, Dean Campagna, BS, Chang Cao, PhD, Daniel Lichtenstein, BS, Anoop Sendamarai, PhD, Chaoshe Guo, PhD, Caiyong Chen, PhD, Gordon J Hildick-Smith, MD, Nicolas C Huston, BA, Jeanne Boudreaux, MD, Sylvia S. Bottomley, MD, Matthew M Heeney, MD, Barry H. Paw, MD PhD, Mark D. Fleming, MD DPhil, Sarah Ducamp, PhD

Blood (2017) 130 (Supplement 1): 79.

https://doi.org/10.1182/blood.V130.Suppl_1.79.79

Published: 2017

Journal Articles

Variation in Serial TCD Velocity Measurements in the TCD with Transfusions Changing to Hydroxyurea (TWiTCH) Trial

Russell E. Ware, MD PhD, Adam Lane, PhD, William H Schultz, MHS PA, Hamayun Imran, MD, Margaret T. Lee, MD, Matthew M Heeney, MD, Clark Brown, MD PhD, Lori Luchtman-Jones, MD, Jennifer Webb, MD, Sherron Jackson, MD, Banu Aygun, MD, Scott T. Miller, MD, Jennifer A Rothman, MD, Alexis A. Thompson, MD MPH, Judy Luden, CCRP, Barry R Davis, MD PhD, Robert J. Adams, MD

Blood (2016) 128 (22): 1019.

https://doi.org/10.1182/blood.V128.22.1019.1019

Published: 2016

Journal Articles

Geographic Differences in Phenotype and Treatment of Children with Sickle Cell Anemia from the Multinational DOVE Study

Baba Inusa, Raffaella Colombatti, MD PhD, David C Rees, MA, FRCP, Matthew M Heeney, MD, Carolyn C Hoppe, MD, Bernhards Ogutu, MD PhD, Hoda M Hassab, MD, Chunmei Zhou, MS, Suqin Yao, PhD, Patricia B Brown, BSN, RN, Lori E Heath, MS, Joseph A. Jakubowski, PhD, Miguel R Abboud, MD

Blood (2016) 128 (22): 3653.

https://doi.org/10.1182/blood.V128.22.3653.3653

Published: 2016

Journal Articles

Changes in Extrahepatic Iron Load in Response to Iron Chelation Versus Phlebotomy: Observations from the Twitch Trial

John C Wood, MD PhD, Alvarez Ofelia, MD, Matthew M Heeney, MD, Alex George, MD PhD, Cynthia Gauger, MD, Beng Fuh, MD, Stephen C Nelson, MD, Carla W Roberts, MD, Melissa Rhodes, MD, Jamie Coleman, MD, Russell E. Ware, MD PhD

Blood (2016) 128 (22): 202.

https://doi.org/10.1182/blood.V128.22.202.202

Published: 2016

Journal Articles

TCD with Transfusions Changing to Hydroxyurea (TWiTCH): Hydroxyurea Therapy As an Alternative to Transfusions for Primary Stroke Prevention in Children with Sickle Cell Anemia

Russell E. Ware, MD PhD, Barry R Davis, MD PhD, William H Schultz, MHS PA, Clark Brown, MD PhD, Banu Aygun, MD, Sharada A. Sarnaik, MD, Isaac Odame, MD, Beng Fuh, MD, Alex George, MD, PhD, William Owen, MD, Lori Luchtman-Jones, MD, Zora R. Rogers, MD, Lee Hilliard, MD, Cynthia Gauger, MD, Connie M. Piccone, MD, Margaret T. Lee, MD, Janet Kwiatkowski, MD, Sherron Jackson, MD, Scott T. Miller, MD, Carla W Roberts, MD, Matthew M. Heeney, MD FRCP(C), Theodosia A. Kalfa, MD PhD, Stephen C Nelson, MD, Hamayun Imran, MD, Kerri A Nottage, MD MPH, Ofelia A. Alvarez, MD, Melissa Rhodes, MD, Alexis A. Thompson, MD, Jennifer Rothman, MD, Kathleen J. Helton, MD, Donna Roberts, MD, Jamie Coleman, MD, Melanie J Bonner, PhD, Abdullah Kutlar, MD, Niren Patel, MD, John C Wood, MD PhD, Linda Piller, MD, Peng Wei, PhD, Judy Luden, CCRP, Nicole A. Mortier, MHS, PA-C, Susan Stuber, MA, Naomi L.C. Luban, MD, Alan R. Cohen, MD, Sara L Pressel, MS, Robert J. Adams, MD MS

Blood (2015) 126 (23): 3.

https://doi.org/10.1182/blood.V126.23.3.3

Published: 2015

Journal Articles

Comparison of Clinical Outcomes Between Adult and Pediatric Patients (pts) with Sickle Cell Disease (SCD): 3-Year (y) Follow-up in a Prospective, Longitudinal, Noninterventional Registry Trial

Matthew M. Heeney, MD FRCP(C), Brad Baltz, MD, Patricia Adams-Graves, MD, Elizabeth Yang, MD PhD, Carole Paley, MD, Jason Esposito, MSHS, Katie McNamara, MSHS, CCRA, Elliott Vichinsky, MD, Alex George, MD, PhD

Blood (2014) 124 (21): 4890.

https://doi.org/10.1182/blood.V124.21.4890.4890

Published: 2014

Journal Articles

Clinical Outcomes For Patients With Sickle Cell Disease: 24-Month Follow-Up In An Ongoing 3-Year, Prospective, Non-Interventional Registry Trial

Matthew M Heeney, MD, Brigitta U. Mueller, MD, Brad Baltz, MD, Patricia Adams-Graves, MD, Elizabeth Yang, MD, PhD, Carole Paley, MD, Jason Esposito MSHS, McNamara Katie MSHS CCRA, Elliott Vichinsky, MD

Blood (2013) 122 (21): 988.

https://doi.org/10.1182/blood.V122.21.988.988

Published: 2013

Journal Articles

12-Month Follow-up for Patients with Sickle Cell Disease in an Ongoing 3-Year, Prospective, Non-Interventional Registry Trial

Matthew M. Heeney, MD, Brigitta U. Mueller, MD, Brad Baltz, MD, Patricia Adams-Graves, MD, Elizabeth Yang, MD, PhD, Carole Paley, MD, Jason Esposito, MSHS, Elliott P. Vichinsky, MD

Blood (2012) 120 (21): 1010.

https://doi.org/10.1182/blood.V120.21.1010.1010

Published: 2012

Journal Articles

Prasugrel in Children with Sickle Cell Disease: Pharmacokinetic and Pharmacodynamic Characteristics from an Open-Label, Adaptive-Design, Dose-Ranging Study

Lori Styles, MD, Joseph A. Jakubowski, PhD, Darell E. Heiselman, MD, Lori E. Heath, MS, Chunmei Zhou, MS, Charles T. Quinn, MD, MS, Matthew M. Heeney, MD, Rupa Redding-Lallinger, MD, David S. Small, PhD, Brian A. Moser, MS, Kenneth J. Winters, MD

Blood (2012) 120 (21): 3213.

https://doi.org/10.1182/blood.V120.21.3213.3213

Published: 2012

Journal Articles

Platelet Activation and Inhibition in Sickle Cell Disease (PAINS) Study

Andrew L. Frelinger, III, PhD, Joseph A. Jakubowski, PhD, Julie K. Brooks, MS, Sabrina L. Zayas, BS, Michelle A. Berny-Lang, PhD, Marc R. Barnard, MS, Matthew M. Heeney, MD, Alan D. Michelson, MD

Blood (2012) 120 (21): 5147.

https://doi.org/10.1182/blood.V120.21.5147.5147

Published: 2012

Journal Articles

In Vivo Platelet Activation and Its In Vitro Inhibition by Prasugrel's Active Metabolite In Adolescents with Sickle Cell Disease

Andrew L. Frelinger, III, PhD, Joseph A. Jakubowski, PhD, Julie K. Brooks, MS, Anu Nigam, MS, Michelle A. Berny-Lang, Ph.D., Marc R. Barnard, MS, Matthew M. Heeney, MD, Alan D. Michelson, MD

Blood (2011) 118 (21): 2141.

https://doi.org/10.1182/blood.V118.21.2141.2141

Published: 2011

Journal Articles

Baseline Characteristics of Patients with Sickle Cell Disease in An Ongoing 5-Year, Prospective, Noninterventional Registry Trial

Matthew M. Heeney, MD, Brigitta U. Mueller, MD, Brad Baltz, Carole Paley, Jason Esposito, Surabhi Sharma, Elliott Vichinsky, MD

Blood (2011) 118 (21): 1060.

https://doi.org/10.1182/blood.V118.21.1060.1060

Published: 2011

Journal Articles

Long-Term Safety and Efficacy of Deferasirox (Exjade ® ) In Transfused Patients with Sickle Cell Disease Treated for up to 5 Years

Elliott Vichinsky, MD, Francoise Bernaudin, MD, Gian Luca Forni, MD, Renee Gardner, MD, Kathryn Hassell, MD, Matthew M. Heeney, MD, Baba Inusa, MD, Abdullah Kutlar, MD, Peter A. Lane, MD, Liesl Mathias, MD, John B. Porter, MD, Cameron K Tebbi, MD, Felicia L. Wilson, MD, Louis Griffel, MD, Wei Deng, PhD, Vanessa Giannone, BSN, Thomas D. Coates, MD

Blood (2010) 116 (21): 845.

https://doi.org/10.1182/blood.V116.21.845.845

Published: 2010

Journal Articles

The Clinical and Genetic Spectrum of TMPRSS6 Mutations Leading to Inappropriate Hepcidin Expression and Iron Refractory Iron Deficiency Anemia (IRIDA).

Matthew M Heeney, MD, Dean R. Campagna, Mark Westerman, PhD, Mark D. Fleming, MD, DPhil

Blood (2009) 114 (22): 629.

https://doi.org/10.1182/blood.V114.22.629.629

Published: 2009

Journal Articles

Collaborative Data Project [C-DATA] of the Comprehensive Sickle Cell Centers Program.

Zora R. Rogers, MD, Susan Lieff, PhD, Marsha McMurray, Carlton Dampier, MD, Winfred C. Wang, MD, Melanie Chelednik, George R. Buchanan, MD, Kenneth I. Ataga, MD, Laura M. De Castro, MD, Matthew M. Heeney, MD, Karen Kalinyak, MD, Kim Smith-Whitley, MD, Elliott Vichinsky, MD, Ronald W. Helms, PhD

Blood (2006) 108 (11): 1200.

https://doi.org/10.1182/blood.V108.11.1200.1200

Published: 2006

NARROW

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

Validation of BCL11A As Therapeutic Target in Sickle Cell Disease: Results from the Adult Cohort of a Pilot/Feasibility Gene Therapy Trial Inducing Sustained Expression of Fetal Hemoglobin Using Post-Transcriptional Gene Silencing

Optimized Beta-Globin Expression and Enucleation from Induced Red Blood Cells for In Vitro Modeling of Sickle Cell Disease

Flipping the Switch: Initial Results of Genetic Targeting of the Fetal to Adult Globin Switch in Sickle Cell Patients

Hscb , a Mitochondrial Iron-Sulfur Cluster Assembly Co-Chaperone, Is a Novel Candidate Gene for Congenital Sideroblastic Anemia

Variation in Serial TCD Velocity Measurements in the TCD with Transfusions Changing to Hydroxyurea (TWiTCH) Trial

Geographic Differences in Phenotype and Treatment of Children with Sickle Cell Anemia from the Multinational DOVE Study

Changes in Extrahepatic Iron Load in Response to Iron Chelation Versus Phlebotomy: Observations from the Twitch Trial

TCD with Transfusions Changing to Hydroxyurea (TWiTCH): Hydroxyurea Therapy As an Alternative to Transfusions for Primary Stroke Prevention in Children with Sickle Cell Anemia

Comparison of Clinical Outcomes Between Adult and Pediatric Patients (pts) with Sickle Cell Disease (SCD): 3-Year (y) Follow-up in a Prospective, Longitudinal, Noninterventional Registry Trial

Clinical Outcomes For Patients With Sickle Cell Disease: 24-Month Follow-Up In An Ongoing 3-Year, Prospective, Non-Interventional Registry Trial

12-Month Follow-up for Patients with Sickle Cell Disease in an Ongoing 3-Year, Prospective, Non-Interventional Registry Trial

Prasugrel in Children with Sickle Cell Disease: Pharmacokinetic and Pharmacodynamic Characteristics from an Open-Label, Adaptive-Design, Dose-Ranging Study

Platelet Activation and Inhibition in Sickle Cell Disease (PAINS) Study

In Vivo Platelet Activation and Its In Vitro Inhibition by Prasugrel's Active Metabolite In Adolescents with Sickle Cell Disease

Baseline Characteristics of Patients with Sickle Cell Disease in An Ongoing 5-Year, Prospective, Noninterventional Registry Trial

Long-Term Safety and Efficacy of Deferasirox (Exjade ® ) In Transfused Patients with Sickle Cell Disease Treated for up to 5 Years

The Clinical and Genetic Spectrum of TMPRSS6 Mutations Leading to Inappropriate Hepcidin Expression and Iron Refractory Iron Deficiency Anemia (IRIDA).

Collaborative Data Project [C-DATA] of the Comprehensive Sickle Cell Centers Program.

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American Society of Hematology

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