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Journal Articles

Clonal landscape and clinical outcomes of telomere biology disorders: somatic rescue and cancer mutations

Fernanda Gutierrez-Rodrigues, Emma M. Groarke, Natthakan Thongon, Juan Jose Rodriguez-Sevilla, Luiz Fernando B. Catto, Marena R. Niewisch, Ruba Shalhoub, Lisa J. McReynolds, Diego V. Clé, Bhavisha A. Patel, Xiaoyang Ma, Dalton Hironaka, Flávia S. Donaires, Nina Spitofsky, Barbara A. Santana, Tsung-Po Lai, Lemlem Alemu, Sachiko Kajigaya, Ivana Darden, Weiyin Zhou, Paul V. Browne, Subrata Paul, Justin Lack, David J. Young, Courtney D. DiNardo, Abraham Aviv, Feiyang Ma, Michel Michels De Oliveira, Ana Paula de Azambuja, Cynthia E. Dunbar, Malgorzata Olszewska, Emmanuel Olivier, Eirini P. Papapetrou, Neelam Giri, Blanche P. Alter, Carmem Bonfim, Colin O. Wu, Guillermo Garcia-Manero, Sharon A. Savage, Neal S. Young, Simona Colla, Rodrigo T. Calado
Journal: Blood
Blood (2024) 144 (23): 2402–2416.
https://doi.org/10.1182/blood.2024025023
Published: 2024
Includes: Supplemental data
Journal Articles

Single Cell Transcriptome Analysis of CD34+ Cells from Patients with RUNX1-Fpdmm at Different Stages of Disease Progression

Erica Bresciani, Bian J Capaldo, Amra Kajdic, Natalie T. Deuitch, Kathleen Craft, Shawn Chong, Abdel G. Elkahloun, Morgan Park, Alice Young, David J. Young, Daoud Meerzaman, Qingrong Chen, Paul P. Liu
Journal: Blood
Blood (2024) 144 (Supplement 1): 1324.
https://doi.org/10.1182/blood-2024-210378
Published: 2024
Journal Articles

Minority Patients in the NIH RUNX1-FPDMM Natural History Study Present Mostly with Large Deletions Affecting RUNX1

Rialnat A. Lawal, Natalie T. Deuitch, Erica Bresciani, Amra Kajdic, Shawn Chong, Kathleen Craft, David J. Young, Paul P. Liu
Journal: Blood
Blood (2024) 144 (Supplement 1): 798.
https://doi.org/10.1182/blood-2024-205563
Published: 2024
Journal Articles

Natural history study of patients with familial platelet disorder with associated myeloid malignancy

CME Clinical Trials & Observations
Lea Cunningham, Matthew Merguerian, Katherine R. Calvo, Joie Davis, Natalie T. Deuitch, Alina Dulau-Florea, Nisha Patel, Kai Yu, Keith Sacco, Sumona Bhattacharya, Monica Passi, Neval Ozkaya, Seila De Leon, Shawn Chong, Kathleen Craft, Jamie Diemer, Erica Bresciani, Kevin O’Brien, Elizabeth J. Andrews, Nguyen Park, Londa Hathaway, Edward W. Cowen, Theo Heller, Kerry Ryan, Amisha Barochia, Khanh Nghiem, Julie Niemela, Sergio Rosenzweig, David J. Young, Pamela A. Frischmeyer-Guerrerio, Raul Braylan, Paul P. Liu
Journal: Blood
Blood (2023) 142 (25): 2146–2158.
https://doi.org/10.1182/blood.2023019746
Published: 2023
Includes: Supplemental data
Journal Articles

Bone Marrow, Laboratory, and Clinical Features in Pediatric Patients with RUNX1 Familial Platelet Disorder with Associated Myeloid Malignancy (FPDMM)

Seila De Leon, Michelly Sampaio De Melo, Nisha Patel, Alina Dulau Florea, Irina Maric, Raul C. Braylan, Weixin Wang, Matthew Merguerian, Elizabeth J. Andrews, Lea Cunningham, David J. Young, Shawn Chong, Natalie Deuitch, Joie Davis, Kathleen Craft, Theo Heller, Pamela Frischmeyer-Guerrerio, Paul Pu Liu, Katherine R. Calvo
Journal: Blood
Blood (2023) 142 (Supplement 1): 4109.
https://doi.org/10.1182/blood-2023-187600
Published: 2023
Journal Articles

A Phase I/II, Intra-Patient Dose-Escalation Study of Bitopertin, a Selective Inhibitor of GlyT1 and Heme Synthesis, for Steroid-Refractory Diamond-Blackfan Anemia

David J. Young, Tania Rene Machado, Raymond T. Doty, Colin O. Wu, Janis L. Abkowitz, William Savage, Cynthia E. Dunbar
Journal: Blood
Blood (2023) 142 (Supplement 1): 1086.
https://doi.org/10.1182/blood-2023-173821
Published: 2023
Journal Articles

Arginine metabolism regulates human erythroid differentiation through hypusination of eIF5A

Pedro Gonzalez-Menendez, Ira Phadke, Meagan E. Olive, Axel Joly, Julien Papoin, Hongxia Yan, Jérémy Galtier, Jessica Platon, Sun Woo Sophie Kang, Kathy L. McGraw, Marie Daumur, Marie Pouzolles, Taisuke Kondo, Stéphanie Boireau, Franciane Paul, David J. Young, Sylvain Lamure, Raghavendra G. Mirmira, Anupama Narla, Guillaume Cartron, Cynthia E. Dunbar, Myriam Boyer-Clavel, Natalie Porat-Shliom, Valérie Dardalhon, Valérie S. Zimmermann, Marc Sitbon, Thomas E. Dever, Narla Mohandas, Lydie Da Costa, Namrata D. Udeshi, Lionel Blanc, Sandrina Kinet, Naomi Taylor
Journal: Blood
Blood (2023) 141 (20): 2520–2536.
https://doi.org/10.1182/blood.2022017584
Published: 2023
Includes: Supplemental data
Journal Articles

A Retrospective Cohort Study of Switching Patterns Among Hemophilia a and B Patients Using Optum Electronic Health Records (EHR) Database in the United States (US)

Yun Gu, Kui Huang, Xunming Sun, Xiaofeng Zhou, Rongjun Shen, Lisa J. Young, Amit Chhabra, Gregory Di Russo, Jeremy Rupon, Ian Winburn
Journal: Blood
Blood (2022) 140 (Supplement 1): 5603–5605.
https://doi.org/10.1182/blood-2022-163569
Published: 2022
Journal Articles

Granulocyte Transfusions As an Adjunctive Therapy in Severe Aplastic Anemia

Roma V. Rajput, Vaani Shah, Ruba Shalhoub, Kamille West-Mitchell, Cathy Conry-Cantilena, David J. Young, Cynthia E. Dunbar, Bhavisha A. Patel, Richard W. Childs, Neal S. Young, Colin O. Wu, Emma M. Groarke, Shelley S. Kalsi
Journal: Blood
Blood (2022) 140 (Supplement 1): 8556–8557.
https://doi.org/10.1182/blood-2022-162595
Published: 2022
Journal Articles

Treatment of Refractory Diamond-Blackfan Anemia with Eltrombopag

Brynn B Duncan, Jennifer Lotter, Jeanine Superata, Ma Evette Barranta, Ivana Darden, Sanjay Venugopal, Colin O. Wu, Cynthia E. Dunbar, David J. Young
Journal: Blood
Blood (2022) 140 (Supplement 1): 5824–5825.
https://doi.org/10.1182/blood-2022-160297
Published: 2022
Journal Articles

Studies of a mosaic patient with DBA and chimeric mice reveal erythroid cell–extrinsic contributions to erythropoiesis

Raymond T. Doty, Xing Fan, David J. Young, Jialiu Liang, Komudi Singh, Zahra Pakbaz, Ronan Desmond, Sara K. Young-Baird, Settara C. Chandrasekharappa, Frank X. Donovan, Susan R. Phelps, Thomas Winkler, Cynthia E. Dunbar, Janis L. Abkowitz
Journal: Blood
Blood (2022) 139 (23): 3439–3449.
https://doi.org/10.1182/blood.2021013507
Published: 2022
Includes: Supplemental data
Journal Articles

Strong Predictors of Chromosomal Aberrations and Myeloid Neoplasia Following Immunosuppression Therapy for Severe Aplastic Anemia: A Retrospective Cohort Study

Emma M. Groarke, Bhavisha A. Patel, Ruba Shalhoub, Fernanda Gutierrez-Rodrigues, Parth Desai, Harshraj Leuva, Casey Paton, Nina Spitofsky, Jennifer Lotter, Olga Rios, Richard W. Childs, David J. Young, Alina Dulau-Florea, Cynthia E. Dunbar, Katherine R. Calvo, Colin O. Wu, Neal S. Young
Journal: Blood
Blood (2021) 138 (Supplement 1): 601.
https://doi.org/10.1182/blood-2021-146070
Published: 2021
Journal Articles

Long-Term Eltrombopag for Bone Marrow Failure Depletes Total Body Iron

David J Young, MDPhD, Xing Fan, MD, Emma Groarke, MD, Bhavisha A Patel, MD, Ronan Desmond, MD, Thomas Winkler, MD, Andre Larochelle, MD PhD, Katherine R. Calvo, MD PhD, Neal S. Young, MD, Cynthia E. Dunbar, MD
Journal: Blood
Blood (2020) 136 (Supplement 1): 39–40.
https://doi.org/10.1182/blood-2020-141375
Published: 2020
Journal Articles

Treatment optimization and genomic outcomes in refractory severe aplastic anemia treated with eltrombopag

Clinical Trials & Observations
Thomas Winkler, Xing Fan, James Cooper, Ronan Desmond, David J. Young, Danielle M. Townsley, Phillip Scheinberg, Sophia Grasmeder, Andre Larochelle, Marie Desierto, Janet Valdez, Jennifer Lotter, Colin Wu, Ruba N. Shalhoub, Katherine R. Calvo, Neal S. Young, Cynthia E. Dunbar
Journal: Blood
Blood (2019) 133 (24): 2575–2585.
https://doi.org/10.1182/blood.2019000478
Published: 2019
Journal Articles

Analysis of US Claims Real World Data of Hemophilia a & B Patients: Units Dispensed & Expenditures Associated with Utilization of and Switching from Standard to Extended Half-Life Factor Products

Bartholomew J. Tortella, MD MBA, Amit Chhabra, MBBS, MPH, José Alvir, DrPH, Emily R. Rubinstein, MPH, Lisa J. Young, MSc, Patrick F. Fogarty, MD
Journal: Blood
Blood (2018) 132 (Supplement 1): 3508.
https://doi.org/10.1182/blood-2018-99-119133
Published: 2018
Journal Articles

Combinatorial Approaches to Overcome Plasma Protein Inhibition of FLT3 Tyrosine Kinase Inhibitors

David J Young, MD PhD, Jun O Liu, PhD, Donald Small, MD PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 1362.
https://doi.org/10.1182/blood-2018-99-118820
Published: 2018
Journal Articles

A Novel Method for Overcoming Plasma Protein Inhibition of FLT3 Tyrosine Kinase Inhibitors

David J Young, MD PhD, Donald Small, MD PhD
Journal: Blood
Blood (2017) 130 (Supplement 1): 1241.
https://doi.org/10.1182/blood.V130.Suppl_1.1241.1241
Published: 2017
Journal Articles

Blood T-cell receptor diversity decreases during the course of HIV infection, but the potential for a diverse repertoire persists

Paul D. Baum, Jennifer J. Young, Diane Schmidt, Qianjun Zhang, Rebecca Hoh, Michael Busch, Jeffrey Martin, Steven Deeks, Joseph M. McCune
Journal: Blood
Blood (2012) 119 (15): 3469–3477.
https://doi.org/10.1182/blood-2011-11-395384
Published: 2012
Includes: Supplemental data
Journal Articles

Donor-lymphocyte infusion induces transplantation tolerance by activating systemic and graft-infiltrating double-negative regulatory T cells

Kevin J. Young, Liming Yang, M. James Phillips, Li Zhang
Journal: Blood
Blood (2002) 100 (9): 3408–3414.
https://doi.org/10.1182/blood-2002-01-0235
Published: 2002
Journal Articles

Splenectomy selectively affects the distribution and mobility of the recirculating lymphocyte pool

Brief Report
Tim J. Seabrook, Wayne R. Hein, Lisbeth Dudler, Alan J. Young
Journal: Blood
Blood (2000) 96 (3): 1180–1183.
https://doi.org/10.1182/blood.V96.3.1180
Published: 2000