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Journal Articles

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

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

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

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

Blood (2024) 144 (Supplement 1): 511.

https://doi.org/10.1182/blood-2024-199743

Published: 2024

Journal Articles

Pediatric Autoimmune Hemolytic Anemia Is Associated with a High Incidence of an Underlying Immune Disorder and High Mortality Rate

Blood (2024) 144 (Supplement 1): 795.

https://doi.org/10.1182/blood-2024-207419

Published: 2024

Journal Articles

Efficacy, Safety, and Health-Related Quality of Life (HRQOL) in Patients with Sickle Cell Disease (SCD) Who Have Received Lovotibeglogene Autotemcel (Lovo-cel) Gene Therapy: Up to 60 Months of Follow-up

Julie Kanter, Alexis A. Thompson, Janet L. Kwiatkowski, Suhag Parikh, Markus Mapara, Stacey Rifkin-Zenenberg, Banu Aygun, Kimberly A. Kasow, Ashish O. Gupta, Lixin Zhang, Emily Sheldon-Waniga, Meghan Gallagher, Katiana Gruppioni, Anjulika Chawla, Heidi Elliot, Francis J. Pierciey, Mark C. Walters, John F. Tisdale

Blood (2023) 142 (Supplement 1): 1051.

https://doi.org/10.1182/blood-2023-174229

Published: 2023

Journal Articles

Lovo-cel (bb1111) Gene Therapy for Sickle Cell Disease: Updated Clinical Results and Investigations into Two Cases of Anemia from Group C of the Phase 1/2 HGB-206 Study

Mark C. Walters, Alexis A. Thompson, Janet L. Kwiatkowski, Suhag Parikh, Markus Y. Mapara, Stacey Rifkin-Zenenberg, Banu Aygun, Kimberly A. Kasow, Alex Miller, Lixin Zhang, Anjulika Chawla, Elizabeth R. Macari, Francis J. Pierciey, Jr., John F. Tisdale, Julie Kanter

Blood (2022) 140 (Supplement 1): 26–28.

https://doi.org/10.1182/blood-2022-162288

Published: 2022

Journal Articles

Standardizing the Diagnostic and Therapeutic Approach to Newly Diagnosed Children with ITP: Prospective Data from the ITP Consortium of North America (ICON) Quality Improvement Initiative

Hannah Elkus, Rachael F. Grace, Taylah Buissereth, Carolyn Bennett, Stacey Rifkin-Zenenberg, Elizabeth R Gunn, Jeffrey D. Lebensburger, Pablo Angulo, Monica Davini, Alexander W. Rankin, Taizo A. Nakano, Corinna L. Schultz, Ashley Pinchinat, Kirsty Hillier, Sherif M. Badawy

Blood (2022) 140 (Supplement 1): 1208–1210.

https://doi.org/10.1182/blood-2022-170067

Published: 2022

Journal Articles

Polyclonality Strongly Correlates with Biological Outcomes and Is Significantly Increased Following Improvements to the Phase 1/2 HGB-206 Protocol and Manufacturing of LentiGlobin for Sickle Cell Disease (SCD; bb1111) Gene Therapy (GT)

Blood (2021) 138 (Supplement 1): 561.

https://doi.org/10.1182/blood-2021-147760

Published: 2021

Journal Articles

Sustained Improvements in Patient-Reported Quality of Life up to 24 Months Post-Treatment with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

Mark C. Walters, John F. Tisdale, Markus Y. Mapara, Lakshmanan Krishnamurti, Janet L. Kwiatkowski, Banu Aygun, Kimberly A. Kasow, Stacey Rifkin-Zenenberg, Jennifer Jaroscak, Diana Garbinsky, Costel Chirila, Meghan E. Gallagher, Xinyan Zhang, Pei-Ran Ho, Alexis A. Thompson, Julie Kanter

Blood (2021) 138 (Supplement 1): 7.

https://doi.org/10.1182/blood-2021-146905

Published: 2021

Journal Articles

Standardizing the Diagnostic and Therapeutic Approach to Newly Diagnosed Children with ITP: An ITP Consortium of North America (ICON) Quality Improvement Initiative

Alexander W. Rankin, Sherif M. Badawy, Carolyn M. Bennett, Taylah Buissereth, Kristen Campbell, Hannah Elkus, Rachael F. Grace, Allison Remiker, Stacey Rifkin, Taizo A. Nakano

Blood (2021) 138 (Supplement 1): 755.

https://doi.org/10.1182/blood-2021-146936

Published: 2021

Journal Articles

Rate of thrombosis in children and adolescents hospitalized with COVID-19 or MIS-C

Blood (2021) 138 (2): 190–198.

https://doi.org/10.1182/blood.2020010218

Published: 2021

Journal Articles

Resolution of Serious Vaso-Occlusive Pain Crises and Reduction in Patient-Reported Pain Intensity: Results from the Ongoing Phase 1/2 HGB-206 Group C Study of LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

Alexis A. Thompson, MD, Mark C. Walters, MD, Markus Y Mapara, MD PhD, Janet L. Kwiatkowski, MD MSCE, Lakshmanan Krishnamurti, MD, Banu Aygun, MD, Kimberly A. Kasow, DO, Stacey Rifkin-Zenenberg, DO, Manfred Schmidt, PhD, Jay DelCarpini, Francis J. Pierciey, Jr., Alexandra L. Miller, Meghan E. Gallagher, MSc, Ren Chen, Sunita Goyal, MD, Julie Kanter, MD, John F. Tisdale, MD

Blood (2020) 136 (Supplement 1): 16–17.

https://doi.org/10.1182/blood-2020-134940

Published: 2020

NARROW

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

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

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

Pediatric Autoimmune Hemolytic Anemia Is Associated with a High Incidence of an Underlying Immune Disorder and High Mortality Rate

Efficacy, Safety, and Health-Related Quality of Life (HRQOL) in Patients with Sickle Cell Disease (SCD) Who Have Received Lovotibeglogene Autotemcel (Lovo-cel) Gene Therapy: Up to 60 Months of Follow-up

Lovo-cel (bb1111) Gene Therapy for Sickle Cell Disease: Updated Clinical Results and Investigations into Two Cases of Anemia from Group C of the Phase 1/2 HGB-206 Study

Standardizing the Diagnostic and Therapeutic Approach to Newly Diagnosed Children with ITP: Prospective Data from the ITP Consortium of North America (ICON) Quality Improvement Initiative

Polyclonality Strongly Correlates with Biological Outcomes and Is Significantly Increased Following Improvements to the Phase 1/2 HGB-206 Protocol and Manufacturing of LentiGlobin for Sickle Cell Disease (SCD; bb1111) Gene Therapy (GT)

Sustained Improvements in Patient-Reported Quality of Life up to 24 Months Post-Treatment with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

Standardizing the Diagnostic and Therapeutic Approach to Newly Diagnosed Children with ITP: An ITP Consortium of North America (ICON) Quality Improvement Initiative

Rate of thrombosis in children and adolescents hospitalized with COVID-19 or MIS-C

Resolution of Serious Vaso-Occlusive Pain Crises and Reduction in Patient-Reported Pain Intensity: Results from the Ongoing Phase 1/2 HGB-206 Group C Study of LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

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American Society of Hematology

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American Society of Hematology

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