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Journal Articles

Next Generation Sequencing-Based Characterization of T Cell Receptor Repertoire of Patients with Immune Thrombocytopenia

Panpan Han, Xiaoqing You, Clara Lo, MD, Liwen Xu, Haiyu Zhang, James L Zehnder, Bing Melody Zhang

Blood (2019) 134 (Supplement_1): 2347.

https://doi.org/10.1182/blood-2019-127590

Published: 2019

Journal Articles

Stroke Avoidance for Children in República Dominicana (SACRED):a Prospective Trial to Reduce Stroke in Children with Sickle Cell Anemia

Luisanna Sanchez, MD, Rosa M. Nieves, MD, Teresa S. Latham, MA, Adam Lane, PhD, Neelum Jeste, MD, Gabriela S. Urcuyo, MD, Melissa E. Berges, MD, Carla Florencio, MD, Carla Gonzalez, MD, Paola Del Villar, MD, Janet Adams, Susan Stuber, MA, William Schultz, M.H.S., Rafael Mena, MD, Russell E. Ware, MD PhD

Blood (2019) 134 (Supplement_1): 2285.

https://doi.org/10.1182/blood-2019-130214

Published: 2019

Journal Articles

An Ongoing Global, Longitudinal, Observational Study of Patients with Pyruvate Kinase Deficiency: The PEAK Registry

Rachael F. Grace, MD, Paola Bianchi, BSc, PhD, Bertil Glader, MD PhD, Andreas Glenthøj, MD PhD, Bryan Jones, PhD, Hitoshi Kanno, MD PhD, Kevin H. M. Kuo, MDMSc,FRCPC, D. Mark Layton, MB ChB, Eduard J. van Beers, MD PhD, Joan-Lluis Vives Corrons, MD PhD, Tu Xu, MS, PhD

Blood (2019) 134 (Supplement_1): 2223.

https://doi.org/10.1182/blood-2019-123059

Published: 2019

Journal Articles

Probably Pathogenic KMT2D Variants Presenting with Cytopenias and Primary Immune Deficiency

Sun Young Kim, MD PhD, Alexander Ing, MS, CGC, Kristin Clemenz, MS, CGC, Kai Lee Yap, PhD, Sherif M. Badawy, MD, Robert I Liem, MD MS, Alexis A. Thompson, MD MPH, A. Kyle Mack, MD

Blood (2019) 134 (Supplement_1): 2334.

https://doi.org/10.1182/blood-2019-131468

Published: 2019

Journal Articles

2'-O-Methoxyethyl Splice-Switching Oligos to Reverse Splicing from IVS2-745 β-Thalassemia Patient Cells: A Foundation for Potential Therapies

Blood (2019) 134 (Supplement_1): 2244.

https://doi.org/10.1182/blood-2019-127017

Published: 2019

Journal Articles

An Analysis of Racial and Ethnic Backgrounds within the Casire International Cohort of Sickle Cell Disease Patients: Implications for Disease Phenotype and Clinical Research

Blood (2019) 134 (Supplement_1): 2305.

https://doi.org/10.1182/blood-2019-127613

Published: 2019

Journal Articles

Peri-Procedural Management and Outcomes of Patients with Hemophilia on Emicizumab Prophylaxis

Isabella McCary, BS, Christine Guelcher, PPCNP-BC, Jan Kuhn, BA, BSN, MSN, Regina Butler, RN-BC, Michael F. Guerrera, MD, Gita Massey, Leslie J. Raffini, MD

Blood (2019) 134 (Supplement_1): 2396.

https://doi.org/10.1182/blood-2019-126944

Published: 2019

Journal Articles

The Oxygenscan Provides Clinically Relevant Biomarkers for Treatment Efficacy That Are Associated with Frequency of Vaso-Occlusive Crisis in Sickle Cell Disease

Minke A.E. Rab, MD, Richard van Wijk, PhD, Celeste K. Kanne, BA, Brigitte A. van Oirschot, Jennifer Bos, Camille Boisson, Maite E. Houwing, MD, Jorn J. Gerritsma, MD, Karin Fijnvandraat, MD PhD, Celine Renoux, PhD, Marije Bartels, MD PhD, Erfan Nur, MD PhD, Marjon H. Cnossen, MD PhD, Philippe Joly, PhD, Romain Fort, MD, Philippe Connes, PhD, Eduard J. van Beers, MD PhD, Vivien A. Sheehan, MD PhD

Blood (2019) 134 (Supplement_1): 2275.

https://doi.org/10.1182/blood-2019-128870

Published: 2019

Journal Articles

Risk Factors for Pediatric Cerebral Sinovenous Thrombosis: A Case-Control Study with Pediatric Neuroradiologist Case Validation

Austin R Sellers, MS, Anh Thy Nguyen, MSPH, Marisol Betensky, MDMPH, Katie Mills, DNP, APRN, Neil A Goldenberg, MD PhD, Nir Shimony, MD

Blood (2019) 134 (Supplement_1): 2427.

https://doi.org/10.1182/blood-2019-130906

Published: 2019

Journal Articles

Pilot Study on the Safety of Intravenous Arsenic Trioxide and Oral Realgar-Indigo Naturalis Formula Administrated in Children with Acute Promyelocytic Leukemia: A Single-Center Study

Linya Wang, MD, Yuanyuan Zhang, MD, Ruidong Zhang, MD PhD, Jiaole Yu, PhD MD, Ying Wu, MD, Wei Lin, Peijing Qi, Jia Fan, Jing Li, PhD, Jiayan Lin, Xueling Zheng, Hairu Hu, Xiaoxia Peng, Hui Jiang, PhD, Huyong Zheng, MD PhD

Blood (2019) 134 (Supplement_1): 2611.

https://doi.org/10.1182/blood-2019-128196

Published: 2019

Journal Articles

Modeling Transient Myeloproliferative Disorder Using Induced Pluripotent Stem Cells and CRISPR/Cas9 Technology

Sonali Barwe, PhD MSc, E. Anders Kolb, MD, Anilkumar Gopalakrishnapillai, PhD

Blood (2019) 134 (Supplement_1): 2738.

https://doi.org/10.1182/blood-2019-130934

Published: 2019

Journal Articles

Demonstration of the Safety of Post Induction Infusion of Ex-Vivo Expanded Cord Blood Progenitor Cells in Pediatric Patients with Relapsed/Refractory Acute Myelogenous Leukemia

Ann Dahlberg, MD, Muna Qayed, MDMSc, Katherine Tarlock, MD, Jessica A. Pollard, MD, Filippo Milano, MD PhD, Soheil Meshinchi, MD PhD, Colleen Delaney, MD MSc

Blood (2019) 134 (Supplement_1): 2614.

https://doi.org/10.1182/blood-2019-130979

Published: 2019

Journal Articles

Detailed Multi-Method Analysis of Bone Marrow from Pediatric Pre-B-ALL Patients Prior to CD19-CAR-T Therapy Subsequently Evidencing Overt CAR-T Resistance

Katherine Elizabeth Masih, BS, Rebecca Gardner, MD, Berkley Gryder, PhD, Abdelmaksoud Abdallah, BS, Justin Lack, PhD, Benjamin Stanton, PhD, Ashley Wilson, PhD, Sivasish Sindiri, MS, Young Song, PhD, Zachary Rae, BS, Michael Kelly, PhD, Wang Chaoyu, MS, Xinyu Wen, MS, Adam Cheuk, PhD, Jun Wei, PhD, Michael C. Jensen, MD, Javed Khan, MD, Rimas Orentas, PhD

Blood (2019) 134 (Supplement_1): 2744.

https://doi.org/10.1182/blood-2019-130796

Published: 2019

Journal Articles

Transient Elevations in Markers of Hepatic Function during Pediatric Acute Lymphoblastic Leukemia Treatment Are Common but Do Not Influence Outcomes: A Study of 805 Patients from the Learn Consortium

Joanna S. Yi, MD, Tiffany Chambers, MPH, Kelly D Getz, Tamara P. Miller, MDMSc, Evanette Burrows, MPH, Marla Daves, MD, Philip J. Lupo, PhD MPH, Michael E. Scheurer, PhD MPH, Richard Aplenc, MD PhD, Karen R. Rabin, MD PhD, Maria Monica J. Gramatges, MD PhD

Blood (2019) 134 (Supplement_1): 3814.

https://doi.org/10.1182/blood-2019-123992

Published: 2019

Journal Articles

Trends in Inpatient and Intensive Care Resource Utilization after Chimeric Antigen Receptor T Cell Therapy for Pediatric Acute Lymphoblastic Leukemia from 2012-2019

Regina M. Myers, MD, Julie C Fitzgerald, MD, Amanda M. DiNofia, MD, Lisa Wray, MD, Allison Barz Leahy, MD, Yimei Li, PhD, Laura Smith, RN, MSN, CRNP, Evanette Burrows, MPH, Mark Ramos, BS, Laura S Motley, BSc, BSN, RN, Raabia N Khan, MPH, Kelly D Getz, Richard Aplenc, MD PhD, Stephan Grupp, MD PhD, Shannon L. Maude, MD PhD

Blood (2019) 134 (Supplement_1): 61.

https://doi.org/10.1182/blood-2019-126724

Published: 2019

Journal Articles

PP-14, a Novel Structurally-Enhanced Antisickling Allosteric Hemoglobin Effector, Increases Oxygen Affinity and Disrupts Hemoglobin S Polymer Formation

Martin K. Safo, PhD, Piyusha P Pagare, PhD, Mohini Ghatge, PhD, Carla Casu, PhD, Valentina Ghiaccio, PhD, Nancy Anabaraonye, Xiaomeng Xu, MS, Stefano Rivella, PhD, Patrick C. Hines, MD PhD, Ke Liu, PhD, Yan Zhang, PhD, Jürgen Venitz, MD PhD, Osheiza Abdulmalik, DVM

Blood (2019) 134 (Supplement_1): 73.

https://doi.org/10.1182/blood-2019-128646

Published: 2019

Journal Articles

Genomic Characterization of a Pediatric Cohort with Non-Malignant Lymphoproliferative Disorders

Blood (2019) 134 (Supplement_1): 83.

https://doi.org/10.1182/blood-2019-131884

Published: 2019

Journal Articles

Lentiviral Gene Therapy with Autologous Hematopoietic Stem and Progenitor Cells (HSPCs) for the Treatment of Severe Combined Immune Deficiency Due to Adenosine Deaminase Deficiency (ADA-SCID): Results in an Expanded Cohort

Blood (2019) 134 (Supplement_1): 3345.

https://doi.org/10.1182/blood-2019-123432

Published: 2019

Journal Articles

Efficacy and Safety in 15 Hemophilia B Patients Treated with the AAV Gene Therapy Vector Fidanacogene Elaparvovec and Followed for at Least 1 Year

Lindsey A. George, MD, Spencer K. Sullivan, MD, John E.J. Rasko, MBBS, PhD, Adam Giermasz, MD PhD, Benjamin J. Samelson-Jones, MD PhD, Jonathan M. Ducore, MD MPH, Jerome M. Teitel, MD FRCPC, Catherine E. McGuinn, MD, Alexa R. Runowski, MS, Fraser Wright, PhD, Xavier M. Anguela, PhD, Katherine A. High, MD, Denis Rybin, PhD, John E. Murphy, PhD, Jeremy Rupon, MD

Blood (2019) 134 (Supplement_1): 3347.

https://doi.org/10.1182/blood-2019-124091

Published: 2019

Journal Articles

Immunosenescent Characteristics of T Cells in Young Patients Following Haploidentical Stem Cell Transplantation from Parental Donors

Ga Hye Lee, Kyung Taek Hong, MD, Jung Yoon Choi, MD, Hee Young Shin, MD PhD, Won-Woo Lee, PhD DVM, Hyoung Jin Kang, MD PhD

Blood (2019) 134 (Supplement_1): 3283.

https://doi.org/10.1182/blood-2019-125282

Published: 2019

NARROW

Next Generation Sequencing-Based Characterization of T Cell Receptor Repertoire of Patients with Immune Thrombocytopenia

Stroke Avoidance for Children in República Dominicana (SACRED):a Prospective Trial to Reduce Stroke in Children with Sickle Cell Anemia

An Ongoing Global, Longitudinal, Observational Study of Patients with Pyruvate Kinase Deficiency: The PEAK Registry

Probably Pathogenic KMT2D Variants Presenting with Cytopenias and Primary Immune Deficiency

2'-O-Methoxyethyl Splice-Switching Oligos to Reverse Splicing from IVS2-745 β-Thalassemia Patient Cells: A Foundation for Potential Therapies

An Analysis of Racial and Ethnic Backgrounds within the Casire International Cohort of Sickle Cell Disease Patients: Implications for Disease Phenotype and Clinical Research

Peri-Procedural Management and Outcomes of Patients with Hemophilia on Emicizumab Prophylaxis

The Oxygenscan Provides Clinically Relevant Biomarkers for Treatment Efficacy That Are Associated with Frequency of Vaso-Occlusive Crisis in Sickle Cell Disease

Risk Factors for Pediatric Cerebral Sinovenous Thrombosis: A Case-Control Study with Pediatric Neuroradiologist Case Validation

Pilot Study on the Safety of Intravenous Arsenic Trioxide and Oral Realgar-Indigo Naturalis Formula Administrated in Children with Acute Promyelocytic Leukemia: A Single-Center Study

Modeling Transient Myeloproliferative Disorder Using Induced Pluripotent Stem Cells and CRISPR/Cas9 Technology

Demonstration of the Safety of Post Induction Infusion of Ex-Vivo Expanded Cord Blood Progenitor Cells in Pediatric Patients with Relapsed/Refractory Acute Myelogenous Leukemia

Detailed Multi-Method Analysis of Bone Marrow from Pediatric Pre-B-ALL Patients Prior to CD19-CAR-T Therapy Subsequently Evidencing Overt CAR-T Resistance

Transient Elevations in Markers of Hepatic Function during Pediatric Acute Lymphoblastic Leukemia Treatment Are Common but Do Not Influence Outcomes: A Study of 805 Patients from the Learn Consortium

Trends in Inpatient and Intensive Care Resource Utilization after Chimeric Antigen Receptor T Cell Therapy for Pediatric Acute Lymphoblastic Leukemia from 2012-2019

PP-14, a Novel Structurally-Enhanced Antisickling Allosteric Hemoglobin Effector, Increases Oxygen Affinity and Disrupts Hemoglobin S Polymer Formation

Genomic Characterization of a Pediatric Cohort with Non-Malignant Lymphoproliferative Disorders

Lentiviral Gene Therapy with Autologous Hematopoietic Stem and Progenitor Cells (HSPCs) for the Treatment of Severe Combined Immune Deficiency Due to Adenosine Deaminase Deficiency (ADA-SCID): Results in an Expanded Cohort

Efficacy and Safety in 15 Hemophilia B Patients Treated with the AAV Gene Therapy Vector Fidanacogene Elaparvovec and Followed for at Least 1 Year

Immunosenescent Characteristics of T Cells in Young Patients Following Haploidentical Stem Cell Transplantation from Parental Donors

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American Society of Hematology

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ASH Publications

American Society of Hematology

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