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Matthew M Heeney
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Journal Articles

Individualized, PK-Guided Dosing of Hydroxyurea for Young Children with Sickle Cell Anemia: Final Results from the Hydroxyurea Optimization through Precision Study (HOPS)

Abena Appiah-Kubi, Seethal A Jacob, Matthew M. Heeney, Connie M. Piccone, Omar Niss, Charles T. Quinn, Stephanie A. Fritch Lilla, Maa-Ohui Quarmyne, Angeli Rampersad, Allison Remiker, Amy Tang, Min Dong, Kathryn McElhinney, Joshua R. Tanzer, Patrick T. McGann
Journal: Blood
Blood (2024) 144 (Supplement 1): 175.
https://doi.org/10.1182/blood-2024-200426
Published: 2024
Journal Articles

Initial Results from the BEACON Clinical Study: A Phase 1/2 Study Evaluating the Safety and Efficacy of a Single Dose of Autologous CD34+ Base Edited Hematopoietic Stem Cells (BEAM-101) in Patients with Sickle Cell Disease with Severe Vaso-Occlusive Crises

Ashish O. Gupta, Akshay Sharma, Haydar Frangoul, Jignesh Dalal, Julie Kanter, Asif Alavi, John DiPersio, Mary Eapen, Jennifer J. Jaroscak, Ernesto Ayala, Edward D. Ziga, Stacey Rifkin-Zenenberg, Alex C. Minella, Guo Chen, Yinzhong Chen, Priya S. Chockalingam, Ling Lin, Marcelyne Joseney-Antoine, Leanne Ianniello, Beth Gardner, Adam J. Hartigan, Giuseppe Ciaramella, Sunita Goyal, Amy Simon, Alexis Thompson, Matthew M. Heeney
Journal: Blood
Blood (2024) 144 (Supplement 1): 513.
https://doi.org/10.1182/blood-2024-204888
Published: 2024
Journal Articles

Non-Canonical Genetics of Hereditary Spherocytosis: A Clinical Case Report

Alexander S Banks, Sarah N Flier, Matthew M. Heeney
Journal: Blood
Blood (2024) 144 (Supplement 1): 5240.
https://doi.org/10.1182/blood-2024-211650
Published: 2024
Journal Articles

Pharmacokinetics/Pharmacodynamics, Safety, and Efficacy of Crizanlizumab in Patients with Sickle Cell Disease Aged 6 to <12 Years: 2-Year Data from the Phase 2 Solace-Kids Study

Matthew M. Heeney, David C Rees, Slimane Allali, Isaac Odame, Rodolfo D Cançado, Adlette Inati, Elena Cela, Deborah Keefe, Evgeniya Reshetnyak, Velusamy Shanmuganathan Muthusamy, Lidiya Bebrevska, Michele L Nassin, Julie Kanter
Journal: Blood
Blood (2024) 144 (Supplement 1): 180.
https://doi.org/10.1182/blood-2024-207207
Published: 2024
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

Modupe Idowu, Michael R. DeBaun, Arthur Burnett, Deepika S. Darbari, Soheir Adam, Alan Randall Anderson, Julie Kanter, Henny H. Billett, Darla K. Liles, Robert S. Nickel, Laura M. Decastro, Biree Andemariam, Gershwin Theophilus Blyden, Matthew M. Heeney, Poornima Ramadas, Morgan L. McLemore, Yogita Kolekar, Dramane Laine, Jincy Paulose, Rajendra Sarkar, Mary Beth Vignogna, Fuad El Rassi
Journal: Blood
Blood (2023) 142 (Supplement 1): 146.
https://doi.org/10.1182/blood-2023-179042
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
Journal: Blood
Blood (2023) 142 (Supplement 1): 560.
https://doi.org/10.1182/blood-2023-178659
Published: 2023
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
Journal: Blood
Blood (2023) 142 (Supplement 1): 1139.
https://doi.org/10.1182/blood-2023-188639
Published: 2023
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
Journal: Blood
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
Journal: Blood
Blood (2022) 140 (Supplement 1): 2151–2152.
https://doi.org/10.1182/blood-2022-158215
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
Journal: Blood
Blood (2022) 140 (Supplement 1): 7902–7903.
https://doi.org/10.1182/blood-2022-170858
Published: 2022
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
Journal: Blood
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
Journal: Blood
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
Journal: Blood
Blood (2022) 140 (Supplement 1): 5715–5716.
https://doi.org/10.1182/blood-2022-170450
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

Erica B. Esrick, Amy Federico, Daniela Abriss, Myriam Armant, Kari Boardman, Christian Brendel, Marioara-Felicia Ciuculescu, Heather Daley, Colleen Dansereau, Augustine Fernandes, Matthew M. Heeney, David G. Justus, Pei-Chi Kao, Annette S. Kim, Donald B. Kohn, Leslie E. Lehmann, Donghui Liu, Wendy B. London, John P Manis, Theodore B. Moore, Emily Morris, Danilo Pellin, Ellen Proeung, Shanna Richard, Gavin D. Roach, Kit L. Shaw, Dayna Terrazas, Shanna L White, David A. Williams
Journal: Blood
Blood (2022) 140 (Supplement 1): 10665–10667.
https://doi.org/10.1182/blood-2022-169089
Published: 2022
Journal Articles

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

Clinical Trials & Observations
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
Journal: Blood
Blood (2022) 140 (13): 1470–1481.
https://doi.org/10.1182/blood.2021014095
Published: 2022
Includes: Supplemental data
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
Journal: Blood
Blood (2021) 138 (Supplement 1): 12.
https://doi.org/10.1182/blood-2021-144730
Published: 2021
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
Journal: Blood
Blood (2021) 138 (Supplement 1): 2037.
https://doi.org/10.1182/blood-2021-144723
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
Journal: Blood
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
Journal: Blood
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
Journal: Blood
Blood (2018) 132 (Supplement 1): 2359.
https://doi.org/10.1182/blood-2018-99-114136
Published: 2018