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Journal Articles

Long Term Outcomes of 63 Patients with Transfusion-Dependent β-Thalassemia (TDT) Followed up to 7 Years Post-Treatment with betibeglogene autotemcel (beti-cel) Gene Therapy and Exploratory Analysis of Predictors of Successful Treatment Outcomes in Phase 3 Trials

Blood (2022) 140 (Supplement 1): 5355–5357.

https://doi.org/10.1182/blood-2022-162400

Published: 2022

Journal Articles

Hypersensitivity Reactions to Native E. coli L-Asparaginase in Children with Acute Lymphoblastic Leukemia May Vary By Treatment Schedule and Type of Glucocorticoid in Induction: Results of Trial ALL-BFM 2000

Blood (2022) 140 (Supplement 1): 11676–11678.

https://doi.org/10.1182/blood-2022-163142

Published: 2022

Journal Articles

Long-Term Patient-Reported Outcomes Following Treatment with betibeglogene autotemcel in Patients with Transfusion-Dependent β-Thalassemia

Franco Locatelli, Mark C. Walters, Janet L. Kwiatkowski, Marina Cavazzana, Suradej Hongeng, John B. Porter, Adrian J. Thrasher, Andreas E. Kulozik, Isabelle Thuret, John E.J. Rasko, Evangelia Yannaki, Martin G. Sauer, Shamshad Ali, Himal Thakar, Katiana Gruppioni, Alexis A. Thompson

Blood (2022) 140 (Supplement 1): 8200–8202.

https://doi.org/10.1182/blood-2022-163178

Published: 2022

Journal Articles

Combining daratumumab with CD47 blockade prolongs survival in preclinical models of pediatric T-ALL

Blood (2022) 140 (1): 45–57.

https://doi.org/10.1182/blood.2021014485

Published: 2022

Includes: Supplemental data

Journal Articles

Constitutional PIGA mutations cause a novel subtype of hemochromatosis in patients with neurologic dysfunction

Lena Muckenthaler, Oriana Marques, Silvia Colucci, Joachim Kunz, Piotr Fabrowski, Thomas Bast, Sandro Altamura, Britta Höchsmann, Hubert Schrezenmeier, Monika Langlotz, Paulina Richter-Pechanska, Tobias Rausch, Nicole Hofmeister-Mielke, Nikolas Gunkel, Matthias W. Hentze, Andreas E. Kulozik, Martina U. Muckenthaler

Blood (2022) 139 (9): 1418–1422.

https://doi.org/10.1182/blood.2021013519

Published: 2022

Includes: Supplemental data

Journal Articles

TP53 and KRAS Variants at Initial Diagnosis Identify an Ultra-High Risk Group of Pediatric T-Lymphoblastic Leukemia (T-ALL)

Tamara Kempter, Joachim B. Kunz, Paulina Richter-Pechanska, Katarzyna Tomska, Tobias Rausch, Busra Erarslan-Uysal, Cornelia Eckert, Martin Zimmermann, Martin Stanulla, Martin Schrappe, Gunnar Cario, Gabriele Escherich, Renate Kirschner-Schwabe, Martina U. Muckenthaler, Jan O. Korbel, Andreas E. Kulozik

Blood (2021) 138 (Supplement 1): 1315.

https://doi.org/10.1182/blood-2021-148420

Published: 2021

Journal Articles

Benefits of a Disease Management Program for SCD in Germany 2011 - 2019: The Increased Use of Hydroxyurea Correlates with a Reduced Frequency of Acute Chest Syndrome

Joachim B. Kunz, Andreas Schlotmann, Andrea Daubenbüchel, Stephan Lobitz, Andrea Jarisch, Regine Grosse, Holger Cario, Lena Oevermann, Dani Hakimeh, Laura Tagliaferri, Andreas E. Kulozik

Blood (2021) 138 (Supplement 1): 973.

https://doi.org/10.1182/blood-2021-148550

Published: 2021

Journal Articles

The First Real-World Experience with Betibeglogene Autotemcel (beti-cel) Gene Therapy Treatment for Transfusion-Dependent β-Thalassemia (TDT)

Joachim B. Kunz, Eva Roth, Adil Mirza, Johann Greil, Petra Pavel, Anita Schmitt, Michael Schmitt, Alexander Kunz, Andreas E. Kulozik

Blood (2021) 138 (Supplement 1): 4917.

https://doi.org/10.1182/blood-2021-148460

Published: 2021

Journal Articles

Improvement in Health-Related Quality of Life Following Treatment with Betibeglogene Autotemcel in Patients with Transfusion-Dependent β-Thalassemia Enrolled in Phase 3 Studies

Janet L. Kwiatkowski, Franco Locatelli, Mark C. Walters, John B. Porter, Suradej Hongeng, Evangelia Yannaki, Andreas E. Kulozik, Martin G. Sauer, Adrian J. Thrasher, Isabelle Thuret, Ashutosh Lal, Ruiting Guo, Richard A. Colvin, Katiana Gruppioni, Alexis A. Thompson

Blood (2021) 138 (Supplement 1): 3085.

https://doi.org/10.1182/blood-2021-150392

Published: 2021

Journal Articles

Restoring Iron Homeostasis in Pts Who Achieved Transfusion Independence after Treatment with Betibeglogene Autotemcel Gene Therapy: Results from up to 7 Years of Follow-up

Alexis A. Thompson, Franco Locatelli, Evangelia Yannaki, Mark C. Walters, John B. Porter, Suradej Hongeng, Andreas E. Kulozik, Martin G. Sauer, Adrian J. Thrasher, Isabelle Thuret, Ashutosh Lal, Marina Cavazzana, John E.J. Rasko, Lin Du, Richard A. Colvin, Janet L. Kwiatkowski

Blood (2021) 138 (Supplement 1): 573.

https://doi.org/10.1182/blood-2021-148177

Published: 2021

Journal Articles

Co-Targeting of CD38 and CD47 in T Cell Acute Lymphoblastic Leukemia

Fotini Vogiatzi, PhD, Kristina Müller, Dorothee Winterberg, Thies Rösner, PhD, Lennart Lenk, PhD, Gunnar Cario, MD PhD, Martin Schrappe, MDPhD, Andreas E. Kulozik, MD PhD, Beat Bornhauser, Jean-Pierre Bourquin, MD PhD, Thomas Valerius, MD, Matthias Peipp, PhD, Christian Kellner, PhD, Denis Martin Schewe, MD

Blood (2020) 136 (Supplement 1): 39–40.

https://doi.org/10.1182/blood-2020-140270

Published: 2020

Journal Articles

Response of Patients with Transfusion-Dependent β-Thalassemia (TDT) to Betibeglogene Autotemcel (beti-cel; LentiGlobin for β-Thalassemia) Gene Therapy Based on HBB Genotype and Disease Genetic Modifiers

Mark C. Walters, MD, David H.K. Chui, MD FRCPC, John J Farrell, PhD, Ashutosh Lal, MD, Franco Locatelli, MD PhD, Janet L. Kwiatkowski, MD MSCE, John B. Porter, MA, MD, FRCP, FRCPath, Martin G. Sauer, MD, Isabelle Thuret, MD, Suradej Hongeng, MD, Andreas E. Kulozik, MD PhD, Adrian J. Thrasher, MBBS, PhD FMedSci, Evangelia Yannaki, MD, Julia Yang, Dustin Whitney, PhD, Alexandria Petrusich, Richard A. Colvin, MD PhD, Alexis A. Thompson, MD

Blood (2020) 136 (Supplement 1): 1–3.

https://doi.org/10.1182/blood-2020-137642

Published: 2020

Journal Articles

Favorable Outcomes in Pediatric Patients in the Phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) Studies of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia

Alexis A. Thompson, MD, Janet L. Kwiatkowski, MD MSCE, John B. Porter, MA, MD, FRCP, FRCPath, Suradej Hongeng, MD, Evangelia Yannaki, MD, Andreas E. Kulozik, MD PhD, Martin G. Sauer, MD, Adrian J. Thrasher, MBBS, PhD FMedSci, Isabelle Thuret, MD, Ashutosh Lal, MD, Ruiting Guo, MS, Weijian Liu, PhD, Richard A. Colvin, MD PhD, Mark C. Walters, MD, Franco Locatelli, MD PhD

Blood (2020) 136 (Supplement 1): 52–54.

https://doi.org/10.1182/blood-2020-135857

Published: 2020

Journal Articles

In Vitro Drug Response Profiling in BCP- and T-ALL Primary Samples Adds a Robust Functional Layer Enabling Optimized Guidance of Individualized Therapy in Relapsed and Refractory Pediatric Acute Leukemia Patients

Blood (2020) 136 (Supplement 1): 15–16.

https://doi.org/10.1182/blood-2020-142314

Published: 2020

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

Daratumumab eradicates minimal residual disease in a preclinical model of pediatric T-cell acute lymphoblastic leukemia

Fotini Vogiatzi, Dorothee Winterberg, Lennart Lenk, Swantje Buchmann, Gunnar Cario, Martin Schrappe, Matthias Peipp, Paulina Richter-Pechanska, Andreas E. Kulozik, Jana Lentes, Anke K. Bergmann, Thomas Valerius, Fabian-Simon Frielitz, Christian Kellner, Denis M. Schewe

Blood (2019) 134 (8): 713–716.

https://doi.org/10.1182/blood.2019000904

Published: 2019

Includes: Supplemental data

Journal Articles

Longitudinal Multilevel Omic Analysis of Pediatric T-ALL Reveals Distinct Mechanisms for Disease Progression in Type 1 and in Type 2 Relapses

Blood (2018) 132 (Supplement 1): 2826.

https://doi.org/10.1182/blood-2018-99-117978

Published: 2018

Journal Articles

Lentiglobin Gene Therapy for Patients with Transfusion-Dependent β-Thalassemia (TDT): Results from the Phase 3 Northstar-2 and Northstar-3 Studies

Franco Locatelli, Mark C. Walters, MD, Janet L. Kwiatkowski, MD, John Porter, MA MDFRCP FRCPath, Martin G. Sauer, MD, Isabelle Thuret, MD, Suradej Hongeng, MD, Andreas E. Kulozik, MD PhD, Ashutosh Lal, MD, Adrian J. Thrasher, MD PhD FMedSci, Evangelia Yannaki, MD, Heidi Elliot, Ge Tao, PhD, Mohammed Asmal, MD PhD, Alexis A. Thompson, MD MPH

Blood (2018) 132 (Supplement 1): 1025.

https://doi.org/10.1182/blood-2018-99-112667

Published: 2018

Journal Articles

Pediatric T-ALLs Developing into a Type 2 Relapse Originate from Cells That Carry the Potential of Variable Maturation into Subclones with Distinct Chromatin Landscapes

Blood (2018) 132 (Supplement 1): 1545.

https://doi.org/10.1182/blood-2018-99-118255

Published: 2018

Journal Articles

Response to Front-Line Imatinib Treatment in Children and Adolescents with CML - Data from a Large Pediatric Cohort

Blood (2017) 130 (Supplement 1): 898.

https://doi.org/10.1182/blood.V130.Suppl_1.898.898

Published: 2017

NARROW

Long Term Outcomes of 63 Patients with Transfusion-Dependent β-Thalassemia (TDT) Followed up to 7 Years Post-Treatment with betibeglogene autotemcel (beti-cel) Gene Therapy and Exploratory Analysis of Predictors of Successful Treatment Outcomes in Phase 3 Trials

Hypersensitivity Reactions to Native E. coli L-Asparaginase in Children with Acute Lymphoblastic Leukemia May Vary By Treatment Schedule and Type of Glucocorticoid in Induction: Results of Trial ALL-BFM 2000

Long-Term Patient-Reported Outcomes Following Treatment with betibeglogene autotemcel in Patients with Transfusion-Dependent β-Thalassemia

Combining daratumumab with CD47 blockade prolongs survival in preclinical models of pediatric T-ALL

Constitutional PIGA mutations cause a novel subtype of hemochromatosis in patients with neurologic dysfunction

TP53 and KRAS Variants at Initial Diagnosis Identify an Ultra-High Risk Group of Pediatric T-Lymphoblastic Leukemia (T-ALL)

Benefits of a Disease Management Program for SCD in Germany 2011 - 2019: The Increased Use of Hydroxyurea Correlates with a Reduced Frequency of Acute Chest Syndrome

The First Real-World Experience with Betibeglogene Autotemcel (beti-cel) Gene Therapy Treatment for Transfusion-Dependent β-Thalassemia (TDT)

Improvement in Health-Related Quality of Life Following Treatment with Betibeglogene Autotemcel in Patients with Transfusion-Dependent β-Thalassemia Enrolled in Phase 3 Studies

Restoring Iron Homeostasis in Pts Who Achieved Transfusion Independence after Treatment with Betibeglogene Autotemcel Gene Therapy: Results from up to 7 Years of Follow-up

Co-Targeting of CD38 and CD47 in T Cell Acute Lymphoblastic Leukemia

Response of Patients with Transfusion-Dependent β-Thalassemia (TDT) to Betibeglogene Autotemcel (beti-cel; LentiGlobin for β-Thalassemia) Gene Therapy Based on HBB Genotype and Disease Genetic Modifiers

Favorable Outcomes in Pediatric Patients in the Phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) Studies of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia

In Vitro Drug Response Profiling in BCP- and T-ALL Primary Samples Adds a Robust Functional Layer Enabling Optimized Guidance of Individualized Therapy in Relapsed and Refractory Pediatric Acute Leukemia Patients

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

Daratumumab eradicates minimal residual disease in a preclinical model of pediatric T-cell acute lymphoblastic leukemia

Longitudinal Multilevel Omic Analysis of Pediatric T-ALL Reveals Distinct Mechanisms for Disease Progression in Type 1 and in Type 2 Relapses

Lentiglobin Gene Therapy for Patients with Transfusion-Dependent β-Thalassemia (TDT): Results from the Phase 3 Northstar-2 and Northstar-3 Studies

Pediatric T-ALLs Developing into a Type 2 Relapse Originate from Cells That Carry the Potential of Variable Maturation into Subclones with Distinct Chromatin Landscapes

Response to Front-Line Imatinib Treatment in Children and Adolescents with CML - Data from a Large Pediatric Cohort

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American Society of Hematology

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American Society of Hematology

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