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Journal Articles

A Phase 3 Study of Eltrombopag Vs. Standard First-Line Management for Newly Diagnosed Immune Thrombocytopenia in Children: Trial in Progress Update

Amanda B. Grimes, Rachael F. Grace, Melissa J. Rose, Sanjay J. Shah, Shipra Kaicker, Carolyn Bennett, Michael Briones, Taizo A. Nakano, Stephanie A. Fritch Lilla, Jeffrey D. Lebensburger, Alexis A. Thompson, Rukhmi Bhat, Rohith Jesudas, Michele P. Lambert, Shelley E. Crary, Kerry Hege, Cathy A. Lee-Miller, Suvankar Majumdar, James B. Ford, Joshua J. Bies, Vandy Black, John Fort, Manpreet Kochhar, Julia MW Wong, Bogdan Dinu, Pei-Chi Kao, Wendy B. London, Jenny M. Despotovic, Robert J. Klaassen, Ellis J. Neufeld, Cindy Neunert, Kristin A. Shimano
Journal: Blood
Blood (2023) 142 (Supplement 1): 1223.
https://doi.org/10.1182/blood-2023-172819
Published: 2023
Journal Articles

Patient and Parent-Reported Outcomes Post-Treatment with Shmir-Based Lentiviral Gene Therapy for Sickle Cell Disease

Lauren Jimenez-Kurlander, Pei-Chi Kao, Mira Magner, Emily Morris, Amy Federico, Theodore Moore, Satiro DeOliveira, Augustine Fernandes, Donald B. Kohn, Wendy B. London, David A. Williams, Erica B. Esrick
Journal: Blood
Blood (2023) 142 (Supplement 1): 3750.
https://doi.org/10.1182/blood-2023-186915
Published: 2023
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

Transplant Associated Thrombotic Microangiopathy (TA-TMA): Lessons Learned from a Large Pediatric Center

Michelle Schoettler, MD, Leslie E. Lehmann, MD, Pei-Chi Kao, MS, Maia Lee, Clement Ma, PhD, Christine Duncan, MD MSc
Journal: Blood
Blood (2018) 132 (Supplement 1): 2090.
https://doi.org/10.1182/blood-2018-99-110185
Published: 2018