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Journal Articles

Introduction to a review series on gene therapy and gene editing for sickle cell disease and hemophilia

Catherine M. Bollard

Blood (2021) 138 (11): 913.

https://doi.org/10.1182/blood.2021012891

Published: 2021

Journal Articles

Pediatric hemophagocytic lymphohistiocytosis

Scott W. Canna, Rebecca A. Marsh

Blood (2020) 135 (16): 1332–1343.

https://doi.org/10.1182/blood.2019000936

Published: 2020

Journal Articles

An Autosomal Dominant SCID Form Due to a Gain of Function Mutation in the RAC2 Gene

Chantal Lagresle-Peyrou, PhD, Aurélien Olichon, Hanem Sadek, Philippe Roche, Claudine Tardy, Cindy Da Silva, Alexandrine Garrigue, Alain Fischer, MD PhD, Despina Moshous, MD PhD, Yves Collette, Capucine Picard, MD PhD, Jean-Laurent Casanova, PhD MD, Isabelle André, PhD, Marina Cavazzana, MD PhD

Blood (2019) 134 (Supplement_1): 3742.

https://doi.org/10.1182/blood-2019-126855

Published: 2019

Journal Articles

Characterizing Dyskeratosis Congenita Caused By Parn Mutations in the Zebrafish

Anna Cordeiro, PhD, Adam P Deveau, MSc, Santhosh Dhanraj, PhD, Yigal Dror, MDFRCPC, Jason Berman, MD

Blood (2019) 134 (Supplement_1): 3744.

https://doi.org/10.1182/blood-2019-130147

Published: 2019

Journal Articles

Phase II Study of Eltrombopag in Subjects with Fanconi Anemia

Daisuke Araki, MD, Ma Evette Barranta, Fariba Chinian, MS, Julie Erb-Alvarez, MPH, Thomas Winkler, MD, Neelam Giri, MD, Andre Larochelle, MD PhD

Blood (2019) 134 (Supplement_1): 3753.

https://doi.org/10.1182/blood-2019-123151

Published: 2019

Journal Articles

Application of High-Throughput Sequencing in the Diagnosis of Inherited Immune-Thrombocytopenia from Children Chronic/Refractory ITP

Ma Jingyao, MD, Zhenping Chen, PhDMD, Huiqing LIU, Jialu Zhang, Hao GU, Runhui Wu, MD PhD

Blood (2019) 134 (Supplement_1): 86.

https://doi.org/10.1182/blood-2019-126771

Published: 2019

Journal Articles

CD244 Marks Non-Functional Hematopoietic Stem Cells with a Mast Cell Signature after Induction of Endoplasmic Reticulum Stress

Valgardur Sigurdsson, PhD, Shuhei Koide, PhD, Visnja Radulovic, PhD, Els Mansell, PhD, Mark Van Der Garde, MSc, PhD, Tiit Örd, PhD, Tonis Örd, PhD, Kenichi Miharada, PhD

Blood (2019) 134 (Supplement_1): 2474.

https://doi.org/10.1182/blood-2019-128239

Published: 2019

Journal Articles

Predictors of Disease Progression and Survival in Patients with Myelodysplastic Syndrome Secondary to Inherited Bone Marrow Failure Syndromes

Blood (2019) 134 (Supplement_1): 2507.

https://doi.org/10.1182/blood-2019-130129

Published: 2019

Journal Articles

Changing the Natural History of Fanconi Anemia Complementation Group-A with Gene Therapy: Early Results of U.S. Phase I Study of Lentiviral-Mediated Ex-Vivo FANCA Gene Insertion in Human Stem and Progenitor Cells

Agnieszka Czechowicz, MD PhD, Maria Grazia Roncarolo, MD, Brian C Beard, PhD, Ken Law, PhD, Eileen Nicoletti, MD, Paula Río, PhD, Juan A. Bueren, PhD, Jonathan D Schwartz, MD, Sandeep Soni, MBBS

Blood (2019) 134 (Supplement_1): 3350.

https://doi.org/10.1182/blood-2019-127352

Published: 2019

Journal Articles

Economic Burden of End Organ Damage Among Patients with Sickle Cell Disease in the US

Xue Song, Andrew D. Campbell, MD, Ze Cong, PhD, Irene Agodoa, MD, Diane Martinez, DrPH, MPH, Carolyn R. Lew, PhD, Danae Black, Helen Varker, Chris Chan, Sophie M. Lanzkron, MD

Blood (2019) 134 (Supplement_1): 3388.

https://doi.org/10.1182/blood-2019-127173

Published: 2019

Journal Articles

Evolution of Moderate Aplastic Anemia into Subclinical PNH/AA and Successful Treatment with Reduced Intensity Haplo-HSCT: A Pediatric Case Report

Meghana Srinivas, Jennifer Krajewski, MD, Nancy Durning, APN, Melanie Hankewycz, APN, Alfred Gillio, MD

Blood (2019) 134 (Supplement_1): 5027.

https://doi.org/10.1182/blood-2019-130407

Published: 2019

Journal Articles

Mitapivat (AG-348) in Adults with Pyruvate Kinase Deficiency Who Are Not Regularly Transfused: A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled Study (ACTIVATE) in Progress

Eduard J. van Beers, MD PhD, Malia P. Judge, BS, RN, Lei Hua, PhD, Chris Mix, MD

Blood (2019) 134 (Supplement_1): 4791.

https://doi.org/10.1182/blood-2019-123100

Published: 2019

Journal Articles

Social Vulnerability Is Associated with Emergency Department Dependency in Pediatric Sickle Cell Disease Patients

Kristina Lai, MPH, Peter A. Lane, MD

Blood (2019) 134 (Supplement_1): 4680.

https://doi.org/10.1182/blood-2019-125351

Published: 2019

Journal Articles

Knockdown of HNF4A Gene Increases Fetal Hemoglobin Synthesis in Hudep-2

Ingrid Grazielle Sousa, BSc, MSc, Priscila Keiko Matsumoto Martin, PhD, Dulcinéia Martins de Albuquerque, BSc, PhD, Carolina Lanaro, BSc, PhD, Ryo Kurita, PhD, Yukio Nakamura, MD PhD, Fernando Ferreira Costa, MD PhD

Blood (2019) 134 (Supplement_1): 968.

https://doi.org/10.1182/blood-2019-130920

Published: 2019

Journal Articles

Hemolysis in the Spleen Drives Erythrocyte Turnover

Thomas Klei, Robin Van Bruggen, PhD, Jill Dalimot, Martijn Veldthuis, Erik Mul, Timo Rademakers, Mark Hoogenboezem, Sietse Quirijn Nagelkerke, Wilfred Van Ijcken, Soren K. Moestrup, MD PhD, Floris van Alphen, Sander Meijer, PhD, Taco Kuijpers, Rob van Zwieten, Pia Svendsen

Blood (2019) 134 (Supplement_1): 946.

https://doi.org/10.1182/blood-2019-124342

Published: 2019

Journal Articles

High Levels of Peripheral Vasoconstriction Detected By Polysomnography Predict More Acute Severe Pain Episodes in Children with Sickle Cell Anemia

Patjanaporn Chalacheva, PhD, Yunhua Ji, MS, Carol L. Rosen, MD, Michael R. DeBaun, MDMPH, Michael C.K. Khoo, PhD, Thomas Coates, MD

Blood (2019) 134 (Supplement_1): 894.

https://doi.org/10.1182/blood-2019-121944

Published: 2019

Journal Articles

Northstar-3: Interim Results from a Phase 3 Study Evaluating Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Either a β0 or IVS-I-110 Mutation at Both Alleles of the HBB Gene

Ashutosh Lal, MD, Franco Locatelli, MD PhD, Janet L. Kwiatkowski, MD MSCE, Andreas E. Kulozik, MD PhD, Evangelia Yannaki, MD, John B. Porter, MA MDFRCP FRCPath, Isabelle Thuret, MD, Martin G. Sauer, MD, Heidi Elliot, Ying Chen, PhD, Richard A. Colvin, MD PhD, Alexis A. Thompson, MD MPH

Blood (2019) 134 (Supplement_1): 815.

https://doi.org/10.1182/blood-2019-128482

Published: 2019

Journal Articles

Home-Based Assessment of Patient Reported Outcome Measures Using a Smartphone App Platform: A Feasibility Study

Sherif M. Badawy, MD, Leonardo Barrera, MPH, Graham Zolkowski, BS, Zeinab Alward, MBChB, MPH

Blood (2019) 134 (Supplement_1): 420.

https://doi.org/10.1182/blood-2019-131334

Published: 2019

Journal Articles

Decline in Forced Expiratory Volume in 1 Second in Older Adults with Sickle Cell Disease Is Similar to Adults with Cystic Fibrosis

Brock Hodges, Jodi-Anne T Stewart, BS, Mark Rodeghier, PhD, Karina L Wilkerson, NP, MSN, Adetola A. Kassim, MDMS, Michael R. DeBaun, MDMPH

Blood (2019) 134 (Supplement_1): 1012.

https://doi.org/10.1182/blood-2019-123428

Published: 2019

Journal Articles

Management Strategies and Satisfaction Levels in Patients with Sickle Cell Disease: Interim Results from the International Sickle Cell World Assessment Survey (SWAY)

John James, MSc, Biree Andemariam, MD, Baba PD Inusa, MD, Fuad El-Rassi, Beverly Francis-Gibson, Alecia C. Nero, MD, Caterina P. Minniti, MD, Cassandra Trimnell, BA, Miguel R. Abboud, MD, Jean-Benoît Arlet, Raffaella Colombatti, Mariane de Montalembert, MD PhD, Suman Jain, Wasil Jastaniah, Erfan Nur, MD PhD, Laurie DeBonnett, MD, Ifeyinwa (ify) Osunkwo, MD MPH

Blood (2019) 134 (Supplement_1): 1017.

https://doi.org/10.1182/blood-2019-123643

Published: 2019

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Introduction to a review series on gene therapy and gene editing for sickle cell disease and hemophilia

Pediatric hemophagocytic lymphohistiocytosis

An Autosomal Dominant SCID Form Due to a Gain of Function Mutation in the RAC2 Gene

Characterizing Dyskeratosis Congenita Caused By Parn Mutations in the Zebrafish

Phase II Study of Eltrombopag in Subjects with Fanconi Anemia

Application of High-Throughput Sequencing in the Diagnosis of Inherited Immune-Thrombocytopenia from Children Chronic/Refractory ITP

CD244 Marks Non-Functional Hematopoietic Stem Cells with a Mast Cell Signature after Induction of Endoplasmic Reticulum Stress

Predictors of Disease Progression and Survival in Patients with Myelodysplastic Syndrome Secondary to Inherited Bone Marrow Failure Syndromes

Changing the Natural History of Fanconi Anemia Complementation Group-A with Gene Therapy: Early Results of U.S. Phase I Study of Lentiviral-Mediated Ex-Vivo FANCA Gene Insertion in Human Stem and Progenitor Cells

Economic Burden of End Organ Damage Among Patients with Sickle Cell Disease in the US

Evolution of Moderate Aplastic Anemia into Subclinical PNH/AA and Successful Treatment with Reduced Intensity Haplo-HSCT: A Pediatric Case Report

Mitapivat (AG-348) in Adults with Pyruvate Kinase Deficiency Who Are Not Regularly Transfused: A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled Study (ACTIVATE) in Progress

Social Vulnerability Is Associated with Emergency Department Dependency in Pediatric Sickle Cell Disease Patients

Knockdown of HNF4A Gene Increases Fetal Hemoglobin Synthesis in Hudep-2

Hemolysis in the Spleen Drives Erythrocyte Turnover

High Levels of Peripheral Vasoconstriction Detected By Polysomnography Predict More Acute Severe Pain Episodes in Children with Sickle Cell Anemia

Northstar-3: Interim Results from a Phase 3 Study Evaluating Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Either a β0 or IVS-I-110 Mutation at Both Alleles of the HBB Gene

Home-Based Assessment of Patient Reported Outcome Measures Using a Smartphone App Platform: A Feasibility Study

Decline in Forced Expiratory Volume in 1 Second in Older Adults with Sickle Cell Disease Is Similar to Adults with Cystic Fibrosis

Management Strategies and Satisfaction Levels in Patients with Sickle Cell Disease: Interim Results from the International Sickle Cell World Assessment Survey (SWAY)

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