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Journal Articles

Disease stigma and salivary stress biomarkers among adolescents and young adults with sickle cell disease

Free
Kemar Prussien, Margaret Mary Burke, Sana Bakir, Lori Crosby, Rosalind Wright, Alexis Thompson, Lisa Schwartz
Journal: Blood
Blood (2025) 146 (Supplement 1): 2670.
https://doi.org/10.1182/blood-2025-2670
Published: 2025
Journal Articles

Robust HbF induction and improvement of anemia and hemolysis with base editing in sickle cell disease: Safety and efficacy findings from the ongoing BEACON study

Free
Ashish Gupta, Akshay Sharma, Haydar Frangoul, Julie Kanter, Markus Mapara, Jignesh Dalal, Asif Alavi, Jennifer Jaroscak, Ernesto Ayala, John DiPersio, Edward D. Ziga, Mary Eapen, Stacey Rifkin-Zenenberg, Srikanth Ambati, Yinzhong Chen, Sarah Chesler, Marcelyne Joseney-Antoine, Leanne Ianniello, Bahru Habtemariam, Priya Chockalingam, Ling Lin, Sunita Goyal, Amy Simon, Alexis Thompson, Matthew Heeney
Journal: Blood
Blood (2025) 146 (Supplement 1): 2532.
https://doi.org/10.1182/blood-2025-2532
Published: 2025
Journal Articles

End-of-study results from the ICON3 pines trial, a phase 3, randomized trial of eltrombopag vs. standard first-line treatment for newly diagnosed immune thrombocytopenia in children

Free
Kristin Shimano, Amanda Grimes, Melissa Rose, Shipra Kaicker, Sanjay Shah, Michael Briones, Elizabeth Gunn, Taizo Nakano, Jeffrey Lebensburger, Michele Lambert, Stephanie Fritch Lilla, Rohith Jesudas, Cathy Lee-Miller, Alexis Thompson, Rukhmi Bhat, Stacey Rifkin-Zenenberg, Suvankar Majumdar, Manpreet Kochhar, Shelley Crary, Kerry Hege, Jennifer Rothman, James Ford, Joshua Bies, Tung Wynn, Loan Hsieh, Maritza Ruiz, Bogdan Dinu, Julia Wong, Pei-Chi Kao, Staci Arnold, Carolyn Bennett, Jenny Despotovic, Robert Klaassen, Ellis Neufeld, Cindy Neunert, Wendy London, Rachael Grace
Journal: Blood
Blood (2025) 146 (Supplement 1): 738.
https://doi.org/10.1182/blood-2025-738
Published: 2025
Journal Articles

The ASH consa consortium on newborn screening in Africa for sickle cell disease: Interim updates of progress in screening, follow-up and supporting initiatives

Free
Obiageli Nnodu, Bernard Awuonda, Lulu Chirande, Charles Kiyaga, Catherine Segbefia, Catherine Chunda-Liyoka, Patience Franklin, Andrew Zapfel, Marie Benoit, Alexis Thompson, Venee N. Tubman, Isaac Odame, Maureen Okam Achebe, Nancy Berliner, Theresa Coetzer, Russell Ware, Enrico Novelli, Nancy S. Green
Journal: Blood
Blood (2025) 146 (Supplement 1): 944.
https://doi.org/10.1182/blood-2025-944
Published: 2025
Journal Articles

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

Free
Amy Drendel, Daniel Tsze, Samina Ali, Fahd Ahmad, Gladstone Airewele, Bolanle Akinsola, Nitya Bakshi, David Brousseau, Kathleen Brown, Andrew Campbell, T. Charles Casper, Todd Chang, Corrie Chumpitazi, Daniel Cohen, Keli Coleman, Andrea Cruz, Christopher Denton, Angela Ellison, Melanie Fields, Monica Harding, Dunia Hatabah, Hailey Jensen, Rawan Korman, Sara Leibovich, Derek Meyer, Chris Rees, Allison Remiker, Nidhi Singh, Alexis Thompson, Elliott Vichinsky, Anthony Villella, Bridget Wynn, Carlton Dampier, Claudia Morris
Journal: Blood
Blood (2025) 146 (Supplement 1): 4722.
https://doi.org/10.1182/blood-2025-4722
Published: 2025
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

Free
Dunia Hatabah, Fahd Ahmad, Gladstone Airewele, Bolanle Akinsola, Nitya Bakshi, David Brousseau, Kathleen Brown, Andrew Campbell, T. Charles Casper, Todd Chang, Corrie Chumpitazi, Daniel Cohen, Keli Coleman, Andrea Cruz, Christopher Denton, Angela Ellison, Melanie Fields, Hailey Jensen, Elizabeth Klings, Rawan Korman, Sara Leibovich, Derek Meyer, Seth Otto, Jonathan Race, Chris Rees, Allison Remiker, Nidhi Singh, Alexis Thompson, Elliott Vichinsky, Anthony Villella, Bridget Wynn, Carlton Dampier, Claudia Morris
Journal: Blood
Blood (2025) 146 (Supplement 1): 2946.
https://doi.org/10.1182/blood-2025-2946
Published: 2025
Journal Articles

Multicenter assessment of absolute neutrophil counts (ANC) and white blood cell counts (WBC) in healthy participants with the erythrocyte duffy null phenotype or genotype

Free
Lauren Merz, Gilles Attipoe, Christina Barriteau, Michael Blechner, Eric Burks, Cecelia Calhoun, Sally Campbell Lee, Vivian Chang, Stella Chou, Adam Cuker, Emmanuel Fadeyi, Warren Fingrut, Victoria Forbes, Ivo Francischetti, Vijayakrishna Gadi, Richard Godby, Lucy Godley, Abraham Haimed, Hyojeong Han, Richard Haspel, Chibuzo Ilonze, Cyril Jacquot, Peng Ji, Justin Juskewitch, Matthew Karafin, Kimberly Klein, Nicole Kucine, Priya Kumar, Margarita Kushnir, David Levitz, Michael Lewis, Geling Li, Zhen Mei, Sabrina Racine Brzostek, Gary Rose, Corinna Schultz, Alexa Siddon, Christine Sillings, John Mark Sloan, Amy Soni, Don Stevens, Charlotte Story, James Szymanski, Clifford Takemoto, Alexis Thompson, Rahma Warsame, Angela Weyand, Samuel Wilson, Nibal Zaghloul, Yadashe Belay, Aliza Fink, Patricia Frustace, Jamie Tyson, Nathalie Van Havre, Maureen Okam Achebe
Journal: Blood
Blood (2025) 146 (Supplement 1): 1074.
https://doi.org/10.1182/blood-2025-1074
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

Free
Nitya Bakshi, Derek Meyer, Jonathan Race, Fahd Ahmad, Gladstone Airewele, Bolanle Akinsola, Kathleen Brown, David Brousseau, Andrew Campbell, Todd Chang, Corrie Chumpitazi, Daniel Cohen, Keli Coleman, Andrea Cruz, Carlton Dampier, Christopher Denton, Angela Ellison, Melanie Fields, Dunia Hatabah, Hailey Jensen, Rawan Korman, Sara Leibovich, Chris Rees, Allison Remiker, Nidhi Singh, Alexis Thompson, Elliott Vichinsky, Anthony Villella, Bridget Wynn, T. Charles Casper, Claudia Morris
Journal: Blood
Blood (2025) 146 (Supplement 1): 6524.
https://doi.org/10.1182/blood-2025-6524
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)

Free
Rawan Korman, Fahd Ahmad, Gladstone Airewele, Bolanle Akinsola, Noor Alzraikat, Nitya Bakshi, David Brousseau, Kathleen Brown, Andrew Campbell, T. Charles Casper, Todd Chang, Corrie Chumpitazi, Daniel Cohen, Keli Coleman, Andrea Cruz, Carlton Dampier, Christopher Denton, Angela Ellison, Melanie Fields, Dunia Hatabah, Hailey Jensen, Sara Leibovich, Derek Meyer, Jonathan Race, Chris Rees, Allison Remiker, Nidhi Singh, Alexis Thompson, Elliott Vichinsky, Anthony Villella, Bridget Wynn, Claudia Morris
Journal: Blood
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

Free
Claudia Morris, Fahd Ahmad, Gladstone Airewele, Bolanle Akinsola, Nitya Bakshi, David Brousseau, Kathleen Brown, Andrew Campbell, T. Charles Casper, Todd Chang, Corrie Chumpitazi, Daniel Cohen, Keli Coleman, Andrea Cruz, Christopher Denton, Angela Ellison, Melanie Fields, Monica Harding, Dunia Hatabah, Hailey Jensen, Rawan Korman, Sara Leibovich, Chris Rees, Allison Remiker, Nidhi Singh, Alexis Thompson, Elliott Vichinsky, Anthony Villella, Bridget Wynn, Carlton Dampier
Journal: Blood
Blood (2025) 146 (Supplement 1): 616.
https://doi.org/10.1182/blood-2025-616
Published: 2025
Journal Articles

Betibeglogene Autotemcel (beti-cel) Gene Addition Therapy Results in Durable Hemoglobin a Production with up to 10 Years of Follow-up with Transfusion-Dependent β-Thalassemia

Free
Alexis Thompson, Janet L. Kwiatkowski, Jennifer Schneiderman, Isabelle Thuret, Andreas E. Kulozik, Evangelia Yannaki, Marina Cavazzana, Suradej Hongeng, Timothy S. Olson, Martin G. Sauer, Adrian J. Thrasher, Ashutosh Lal, John E. J. Rasko, Shamshad Ali, Ge Tao, Himal L. Thakar, Ami Deora, Clark Paramore, Niki Witthuhn, Mark C. Walters, Franco Locatelli
Journal: Blood
Blood (2024) 144 (Supplement 1): 2194.
https://doi.org/10.1182/blood-2024-200152
Published: 2024
Journal Articles

Reni-Cel, an Investigational AsCas12a Gene-Edited Cell Medicine, Led to Successful Engraftment, Increased Hemoglobin, and Reduced Transfusion Dependence in Patients with Transfusion-Dependent Beta-Thalassemia Treated in the Edithal Trial

Free
Haydar Frangoul, Rabi Hanna, Mark C. Walters, Kai-Hsin Chang, Michael Jaskolka, Keunpyo Kim, Baisong Mei, Olubunmi Afonja, Alexis Thompson
Journal: Blood
Blood (2024) 144 (Supplement 1): 7456.
https://doi.org/10.1182/blood-2024-208623
Published: 2024
Journal Articles

Acceptability and Feasibility of Salivary Stress Research with Adolescents and Young Adults with Sickle Cell Disease

Free
Kemar V. Prussien, Lori E. Crosby, Margaret Mary Burke, Rosalind J. Wright, Alexis Thompson, Lisa A. Schwartz
Journal: Blood
Blood (2024) 144 (Supplement 1): 1067.
https://doi.org/10.1182/blood-2024-205076
Published: 2024
Journal Articles

Innovative Model for Creation and Use of Co-Developed Pain Management Plans for Patients with Sickle Cell Disease

Free
Alexis Thompson, Jasmine Umana, Julia B Sabrick, Therese McKnight, Leslie D Williams, Krupal Rana, William Baar, Lauren Coogle, Osvaldo Mecado, Char M Witmer, Angela M. Ellison, Cynthia F Norris
Journal: Blood
Blood (2024) 144 (Supplement 1): 603.
https://doi.org/10.1182/blood-2024-194151
Published: 2024
Journal Articles

Participants with a History of Stroke in Lovotibeglogene Autotemcel (Lovo-cel) Clinical Trials

Free
Jennifer J. Jaroscak, Julie Kanter, Robert I. Liem, Anjulika Chawla, Melissa A. Kinney, Ankit Lodaya, Lin Pan, Emily Sheldon-Waniga, Francis J. Pierciey, Jr., Alexis Thompson
Journal: Blood
Blood (2024) 144 (Supplement 1): 3576.
https://doi.org/10.1182/blood-2024-199642
Published: 2024
Journal Articles

Clinical Management Bundle for Patients with Sickle Cell Disease: Improving Completion of Age-Specific Screening Tests and Disease-Modifying Therapy Use

Free
Cynthia F Norris, Leigh Foppert, Elizabeth Brooks, Alicia Brandemarte, Rebecca Brugger, Julia B Sabrick, Kyisha Knox, Alexandra Kaspin, Sharena Robinson, Therese McKnight, Elizabeth Pumiglia, Drew H Scoles, Brian Maloney, Alexis Thompson
Journal: Blood
Blood (2024) 144 (Supplement 1): 519.
https://doi.org/10.1182/blood-2024-201040
Published: 2024
Journal Articles

Efficacy Findings in a Phase 3, Randomized Trial of Eltrombopag Vs. Standard First-Line Treatment for Newly Diagnosed Immune Thrombocytopenia in Children

Free
Kristin A. Shimano, Amanda B. Grimes, Melissa J Rose, Shipra Kaicker, Sanjay J. Shah, Michael Briones, Elizabeth Gunn, Taizo A. Nakano, Jeffrey D Lebensburger, Michele P. Lambert, Stephanie A. Fritch Lilla, Rohith Jesudas, Cathy A. Lee-Miller, Alexis Thompson, Rukhmi Bhat, Stacey Rifkin-Zenenberg, Suvankar Majumdar, Manpreet Kochhar, Shelley E Crary, Kerry Hege, Jennifer A. Rothman, James B. Ford, Joshua J Bies, John Fort, Loan Hsieh, Maritza E. Ruiz, Kathryn M. Carrier, Bogdan Dinu, Julia MW Wong, Pei-Chi Kao, Wendy B. London, Staci D. Arnold, Carolyn M. Bennett, Jenny Despotovic, Robert J. Klaassen, Ellis J. Neufeld, Cindy E Neunert, Rachael F. Grace
Journal: Blood
Blood (2024) 144 (Supplement 1): 709.
https://doi.org/10.1182/blood-2024-193644
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

Free
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

An Update on Lovotibeglogene Autotemcel (Lovo-cel) Clinical Trials for Sickle Cell Disease (SCD) and Analysis of Early Predictors of Response to Lovo-Cel

Free
Stacey Rifkin-Zenenberg, Julie Kanter, Melissa A. Kinney, Janet L. Kwiatkowski, Robert S. Nickel, Mark C. Walters, Suhag Parikh, Alexis Thompson, Anil P. George, Markus Mapara, Paul L. Martin, Anjulika Chawla, Ankit Lodaya, Lin Pan, Emily Sheldon-Waniga, Francis J. Pierciey, Jr., John Tisdale, Ashish O. Gupta
Journal: Blood
Blood (2024) 144 (Supplement 1): 511.
https://doi.org/10.1182/blood-2024-199743
Published: 2024
Journal Articles

Outcomes of Immunosuppressive Therapy in Children Less Than Three Years of Age with Idiopathic Severe Aplastic Anemia: A North American Collaboration

Free
Talya Wittmann Dayagi, Laura Willis, Catherine Corriveau-Bourque, Michaela Cada, Zora R. Rogers, Taizo A. Nakano, Timothy S. Olson, Winfred C. Wang, Alfred Gillio, Thomas D. Coates, Anjulika Chawla, Paul Castillo, Peter Kurre, Christopher Gamper, Staci D. Arnold, Sarita Joshi, Amy E. Geddis, Jessica Boklan, Jennifer A. Rothman, James N. Huang, Gary M. Kupfer, Bertil Glader, Kelly J. Walkovich, Alexis Thompson, Rabi Hanna, David A. Williams, Akiko Shimamura, Yigal Dror
Journal: Blood
Blood (2024) 144 (Supplement 1): 2691.
https://doi.org/10.1182/blood-2024-198302
Published: 2024