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Journal Articles

Safety Profile of Crizanlizumab 5.0 Mg/Kg in Sickle Cell Disease: Pooled Data from Clinical Trials

Abdullah Kutlar, Vikas Joshi, Andreas Brueckner, Julie Kanter, Darla K. Liles, Jeremie Lincy, Rahul Manjare, Yvonne Dei Adomakoh, Matthew M. Heeney, Mariane de Montalembert, Deborah Keefe, Kwaku Marfo, Kenneth I. Ataga

Blood (2023) 142 (Supplement 1): 1139.

https://doi.org/10.1182/blood-2023-188639

Published: 2023

Journal Articles

Cerebral Hemodynamics in Infants with Sickle Cell Disease Indicate Presence of Early Hemodynamic Stress

Pei-Yi Lin, Rutvi Vyas, Michael Woglom, Jeffrey N. Stout, Erica B. Esrick, Matthew M. Heeney, Natasha Archer, P. Ellen Grant, Jason Sutin

Blood (2023) 142 (Supplement 1): 560.

https://doi.org/10.1182/blood-2023-178659

Published: 2023

Journal Articles

Primary Analysis of Spartan: A Phase 2 Trial to Assess the Efficacy and Safety of Crizanlizumab in Patients with Sickle Cell Disease Related Priapism

Blood (2023) 142 (Supplement 1): 146.

https://doi.org/10.1182/blood-2023-179042

Published: 2023

Journal Articles

Natural History of Pediatric Patients with Sickle Cell Disease and Concurrent Cerebral Vasculopathies: Report of 62 Cases from a Single Institution

Joanna E. Papadakis, Paulina Piwowarczyk, Daniel S. Weber, Anna L. Slingerland, Dylan S. Keusch, Matthew M. Heeney, Edward R. Smith, Alfred P. See, Natasha M. Archer

Blood (2022) 140 (Supplement 1): 5446–5447.

https://doi.org/10.1182/blood-2022-170784

Published: 2022

Journal Articles

Ketamine Use for Management of Vaso-Occlusive Pain in Children with Sickle Cell Disease

Emily M. Harris, Emily Vilk, Carolina Donado, Alexis Williams, Matthew M. Heeney, Jean Solodiuk, Christine Greco, Natasha M. Archer

Blood (2022) 140 (Supplement 1): 5435–5436.

https://doi.org/10.1182/blood-2022-170695

Published: 2022

Journal Articles

Altered Cellular Sedimentation during Apheresis Impacts Outcomes of Peripheral Blood Stem Cell Collection Efficiency in Patients with Sickle Cell Disease

David G. Justus, Erica B. Esrick, Matthew M. Heeney, Ellen Proeung, Carly Howard, Carlo Brugnara, David A. Williams, John P Manis

Blood (2022) 140 (Supplement 1): 5715–5716.

https://doi.org/10.1182/blood-2022-170450

Published: 2022

Journal Articles

Improving Rates of Annual Transcranial Doppler Screening in Children with Sickle Cell Disease: A Quality Improvement Project

Jeffrey G Edwards, Adam P Yan, Ramy Yim, Eileen Hansbury, Matthew M. Heeney, Dave Johnson, Heather McMullan, Chris Wong Quiles, Maya Ilowite, Natasha M. Archer

Blood (2022) 140 (Supplement 1): 7902–7903.

https://doi.org/10.1182/blood-2022-170858

Published: 2022

Journal Articles

Induction of Fetal Hemoglobin and Reduction of Clinical Manifestations in Patients with Severe Sickle Cell Disease Treated with Shmir-Based Lentiviral Gene Therapy for Post-Transcriptional Gene Editing of BCL11A: Updated Results from Pilot and Feasibility Trial

Blood (2022) 140 (Supplement 1): 10665–10667.

https://doi.org/10.1182/blood-2022-169089

Published: 2022

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

Increasing COVID-19 Vaccination Rates Amongst Children with Sickle Cell Disease: A Quality Improvement Project

Adam P Yan, Natasha M. Archer, Dianne Arnold, Eileen Hansbury, Matthew M. Heeney, Dave Johnson, Erika Lichtman, Heather McMullan, Lisa Morrissey, Maya Ilowite

Blood (2022) 140 (Supplement 1): 2151–2152.

https://doi.org/10.1182/blood-2022-158215

Published: 2022

Journal Articles

Ticagrelor vs placebo for the reduction of vaso-occlusive crises in pediatric sickle cell disease: the HESTIA3 study

Matthew M. Heeney, Miguel R. Abboud, Jessie Githanga, Baba P. D. Inusa, Julie Kanter, Alan D. Michelson, Videlis Nduba, Victor Musiime, Mohini Apte, Adlette Inati, Amar M. Taksande, Marielle Andersson, Magnus Åstrand, Noha Maklad, Mohammad Niazi, Anders Himmelmann, Anders R. Berggren

Blood (2022) 140 (13): 1470–1481.

https://doi.org/10.1182/blood.2021014095

Published: 2022

Includes: Supplemental data

Journal Articles

Association of Hospitalization Due to Vaso-Occlusive Crisis with Subsequent Sickle Cell Disease-Related Organ Damage Hospitalization: Retrospective Analysis of 3-Year Observational Study Data

Matthew M. Heeney, Thirupathi Pattipaka, Jilles M. Fermont

Blood (2021) 138 (Supplement 1): 2037.

https://doi.org/10.1182/blood-2021-144723

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

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

Normalizing hepcidin predicts TMPRSS6 mutation status in patients with chronic iron deficiency

Matthew M. Heeney, Dongjing Guo, Luigia De Falco, Dean R. Campagna, Gordana Olbina, Paige P.-C. Kao, Klaus Schmitz-Abe, Fedik Rahimov, Patrick Gutschow, Keith Westerman, Vaughn Ostland, Tracy Jackson, Robert J. Klaassen, Kyriacos Markianos, Karin E. Finberg, Achille Iolascon, Mark Westerman, Wendy B. London, Mark D. Fleming

Blood (2018) 132 (4): 448–452.

https://doi.org/10.1182/blood-2017-03-773028

Published: 2018

Includes: Supplemental data

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

NARROW

Safety Profile of Crizanlizumab 5.0 Mg/Kg in Sickle Cell Disease: Pooled Data from Clinical Trials

Cerebral Hemodynamics in Infants with Sickle Cell Disease Indicate Presence of Early Hemodynamic Stress

Primary Analysis of Spartan: A Phase 2 Trial to Assess the Efficacy and Safety of Crizanlizumab in Patients with Sickle Cell Disease Related Priapism

Natural History of Pediatric Patients with Sickle Cell Disease and Concurrent Cerebral Vasculopathies: Report of 62 Cases from a Single Institution

Ketamine Use for Management of Vaso-Occlusive Pain in Children with Sickle Cell Disease

Altered Cellular Sedimentation during Apheresis Impacts Outcomes of Peripheral Blood Stem Cell Collection Efficiency in Patients with Sickle Cell Disease

Improving Rates of Annual Transcranial Doppler Screening in Children with Sickle Cell Disease: A Quality Improvement Project

Induction of Fetal Hemoglobin and Reduction of Clinical Manifestations in Patients with Severe Sickle Cell Disease Treated with Shmir-Based Lentiviral Gene Therapy for Post-Transcriptional Gene Editing of BCL11A: Updated Results from Pilot and Feasibility Trial

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

Increasing COVID-19 Vaccination Rates Amongst Children with Sickle Cell Disease: A Quality Improvement Project

Ticagrelor vs placebo for the reduction of vaso-occlusive crises in pediatric sickle cell disease: the HESTIA3 study

Association of Hospitalization Due to Vaso-Occlusive Crisis with Subsequent Sickle Cell Disease-Related Organ Damage Hospitalization: Retrospective Analysis of 3-Year Observational Study Data

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

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

Normalizing hepcidin predicts TMPRSS6 mutation status in patients with chronic iron deficiency

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

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American Society of Hematology

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American Society of Hematology

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