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Journal Articles

Clinical Evidence of Granulocyte-Monocyte Progenitor (GMP) Stem Cell Involvement in Plinabulin's Mechanism of Action (MoA) for the Prevention of Docetaxel (Doc) Chemotherapy (Chemo)-Induced Neutropenia (CIN)

Douglas W. Blayney, MD, Lan Huang, PhD, Ramon Mohanlal, PhD MD

Blood (2019) 134 (Supplement_1): 4861.

https://doi.org/10.1182/blood-2019-127227

Published: 2019

Journal Articles

Hematopoietic Engraftment of Fanconi Anemia Patients through 3 Years after Gene Therapy

Blood (2019) 134 (Supplement_1): 4627.

https://doi.org/10.1182/blood-2019-129686

Published: 2019

Journal Articles

Long-Term Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar (HGB-204) Study

Janet L. Kwiatkowski, MD MSCE, Alexis A. Thompson, MD MPH, John E.J. Rasko, MBBS, PhD, Suradej Hongeng, MD, Gary J. Schiller, MD, Usanarat Anurathapan, MD, Marina Cavazzana, MD PhD, P. Joy Ho, MBBS DPhil FRACP FRCPA, Manfred Schmidt, PhD, Morris Kletzel, MD MBA, Elliott P. Vichinsky, MD, Briana Deary, Ying Chen, PhD, Alexandria Petrusich, Mark C. Walters, MD

Blood (2019) 134 (Supplement_1): 4628.

https://doi.org/10.1182/blood-2019-125807

Published: 2019

Journal Articles

Durable and Robust Fetal Globin Induction without Anemia in Rhesus Monkeys Following Autologous Hematopoietic Stem Cell Transplant with BCL11A Erythroid Enhancer Editing

Blood (2019) 134 (Supplement_1): 4632.

https://doi.org/10.1182/blood-2019-129394

Published: 2019

Journal Articles

Mgta-456, an Aryl Hydrocarbon Receptor (AHR) Antagonist Based Expansion of CD34+ Hematopoietic Stem Cells (HSC), Permits Selection of Better HLA Matched Cord Blood Units (CBUs) and Promotes Faster Neutrophil Recovery and Uniform Engraftment with Potentially Less Acute Graft-Vs-Host Disease (GVHD)

Heather Stefanski, MD PhD, Claudio G Brunstein, MD PhD, David H. McKenna, MD, Darin Sumstad, MLS, Jeffrey S. Miller, MD, Bruce R. Blazar, MD, Todd E. DeFor, MS, Anthony E. Boitano, PhD, Michael P. Cooke, PhD, Glen D. Raffel, MD PhD, John C. Davis, Jr., MD MPH, MS, John E. Wagner, MD

Blood (2019) 134 (Supplement_1): 804.

https://doi.org/10.1182/blood-2019-122329

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

Outcomes after Plerixafor Plus FLAG-IDA (PLERIFLAG) Versus FLAG-IDA +/- Gentuzumab for Adult Patients with First Relapsed/Refractory AML: A Propensity Score Analysis from the Pethema Registry

Blood (2019) 134 (Supplement_1): 1321.

https://doi.org/10.1182/blood-2019-128568

Published: 2019

Journal Articles

Procarbazine-Free Escalated Beacopdac in Frontline Therapy of Advanced Hodgkin Lymphoma Reduces Red Cell Transfusion Requirements and May Shorten Time to Menstrual Period Recovery Compared to Escalated Beacopp and Appears to be As Efficacious

Blood (2019) 134 (Supplement_1): 1564.

https://doi.org/10.1182/blood-2019-125256

Published: 2019

Journal Articles

Phase 1 Study of Decitabine and Vorinostat Followed By Fludarabine, Cytarabine and G-CSF (FLAG) in Children, Adolescents and Young Adults with Relapsed/Refractory AML: Report from the Therapeutic Advances in Childhood Leukemia and Lymphoma (TACL) Consortium

Lauren Pommert, MDMS, Deepa Bhojwani, MD, Richard Sposto, PhD, Ellynore Florendo, Eric Schafer, MD, Nathan Gossai, MD, Van Thu Huynh, MD, Sridhar Rao, MD PhD, Michael J. Burke, MD

Blood (2019) 134 (Supplement_1): 1325.

https://doi.org/10.1182/blood-2019-127298

Published: 2019

Journal Articles

Cladribine, Cytarabine, Granulocyte Colony Stimulating Factor ± Mitoxantrone (CLAG ± M) Is Highly Effective Therapy for Secondary and Relapsed/Refractory Acute Myeloid Leukemia

Amanda Przespolewski, DO, Craig W. Freyer, PharmD, Wenyan Ji, MA, Tara L. Cronin, BA, Swapna Thota, MD, Elizabeth A. Griffiths, MD, James E. Thompson, MD, Amro Elshoury, MBBChir, Jeffrey Baron, PharmD, Mark Walsh, PharmD, Eunice S. Wang, MD

Blood (2019) 134 (Supplement_1): 1361.

https://doi.org/10.1182/blood-2019-130582

Published: 2019

Journal Articles

Enhanced Transduction Lentivector Gene Therapy for Treatment of Older Patients with X-Linked Severe Combined Immunodeficiency

Blood (2019) 134 (Supplement_1): 608.

https://doi.org/10.1182/blood-2019-127439

Published: 2019

Journal Articles

Β-Catenin-Tcf/Lef Signaling Promotes Steady State and Emergency Granulopoiesis through G-CSF Receptor Upregulation

Petr Danek, MSc, Miroslava Kardosova, PhD, Lucie Janeckova, PhD, Vladimir Korinek, PhD, Touati Benoukraf, PhD, Daniel G. Tenen, MD, Meritxell Alberich-Jorda

Blood (2019) 134 (Supplement_1): 1193.

https://doi.org/10.1182/blood-2019-122254

Published: 2019

Journal Articles

Response to High Dose G-CSF Treatment (20µg/kg/d or Higher) of Patients with Congenital Neutropenia: An Analysis By the Scnir in Europe

Cornelia Zeidler, Natali Gerschmann, Sabine Mellor-Heineke, Fabian Frömling, Julia Skokowa, MD PhD, Karl Welte

Blood (2019) 134 (Supplement_1): 1225.

https://doi.org/10.1182/blood-2019-129844

Published: 2019

Journal Articles

15-PGDH Inhibition Mitigates Aplastic Anemia Severity

Julianne N. P. Smith, PhD, Folashade Otegbeye, MBBChir,MPH, Alvin Jogasuria, Sanford Markowitz, MD PhD, Amar Desai, PhD

Blood (2019) 134 (Supplement_1): 1220.

https://doi.org/10.1182/blood-2019-131353

Published: 2019

Journal Articles

Phase I Clinical Trial of CK0801 (cord blood regulatory T cells) in Patients with Bone Marrow Failure Syndrome (BMF) Including Aplastic Anemia, Myelodysplasia and Myelofibrosis

Tapan M. Kadia, MD, Hongbing Ma, MD PhD, Ke Zeng, MD PhD, Mitsutaka Nishimoto, MD PhD, Mi-Ae Lyu, PhD, Meixian Huang, Musa Yilmaz, MD, Courtney D. DiNardo, MDMSc, Ghayas C. Issa, MD, Simrit Parmar, MBBS, Swami P. Iyer, MD, Parameswaran Hari, MBBS, MD, Naval G. Daver, MD, Elias Jabbour, MD, Gautam M. Borthakur, MD, Srdan Verstovsek, MD PhD

Blood (2019) 134 (Supplement_1): 1221.

https://doi.org/10.1182/blood-2019-127702

Published: 2019

Journal Articles

Feasibility of Peripheral Blood Stem Cell Collection from Heterozygoussickle Cell Trait Donors

Juergen Foell, MD, Morad Morez, Anja Troeger, MD, Katharina Kleinschmidt, MD PhD, Beatrix Pfirstinger, Andreas Brosig, MD PhD, Ralph Burkhardt, MD PhD, Selim Corbacioglu, MD, Norbert Ahrens

Blood (2019) 134 (Supplement_1): 1171.

https://doi.org/10.1182/blood-2019-131984

Published: 2019

Journal Articles

Canonical Signaling By TGF Family Members in Mesenchymal Stromal Cells Is Dispensable for Hematopoietic Niche Maintenance Under Basal and Stress Conditions

Joseph R. Krambs, BS, Grazia Abou Ezzi, PhD, Juo-Chin Yao, Justin T. Li, Daniel C. Link, MD

Blood (2019) 134 (Supplement_1): 1209.

https://doi.org/10.1182/blood-2019-128693

Published: 2019

Journal Articles

Efficient Correction of ELANE mutations in Primary HSPCs of Severe Congenital Neutropenia Patients Using CRISPR/Cas9 and rAVV6 HDR Repair Templates

Malte U Ritter, Benjamin Secker, Maksim Klimiankou, Masoud Nasri, MSc, Narges Aghaallaei, Benjamin Dannenmann, Regine Bernhard, Ingeborg Steiert, Karin Haehnel, Baubak Bajoghli, Cornelia Zeidler, Karl Welte, Julia Skokowa, MD PhD

Blood (2019) 134 (Supplement_1): 1036.

https://doi.org/10.1182/blood-2019-131708

Published: 2019

Journal Articles

102 Patients with Primary Autoimmune Neutropenia : An Analysis of the European Severe Chronic Neutropenia International Registry (SCNIR)

Fabian Frömling, Sabine Mellor-Heineke, Natali Gerschmann, Lena Röbbel, Julia Skokowa, MD PhD, Karl Welte, Cornelia Zeidler

Blood (2019) 134 (Supplement_1): 1039.

https://doi.org/10.1182/blood-2019-130031

Published: 2019

Journal Articles

Efficacy and Safety of a FLAG-Sequential Regimen Followed By Hematopoietic Stem Cell Transplantation for Myelodysplasia or Acute Leukemia in Fanconi Anemia: A Franco-Brazilian Report

Pierre-Edouard Debureaux, Flore Sicre de Fontbrune, Carmem M. S. Bonfim, MD, Jean-Hugues Dalle, MD PhD, Nimrod Buchbinder, MD, Yves Bertrand, MD, Cecile Renard, MD, Edouard Forcade, MD, Juliana Fernandes, MD, Alexis Talbot, MD, Ricardo Pasquini, MD, Jean Soulier, MD PhD, Thierry Leblanc, MD, Gerard Socie, MD PhD, Regis Peffault de Latour, MD PhD

Blood (2019) 134 (Supplement_1): 2498.

https://doi.org/10.1182/blood-2019-122007

Published: 2019

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Clinical Evidence of Granulocyte-Monocyte Progenitor (GMP) Stem Cell Involvement in Plinabulin's Mechanism of Action (MoA) for the Prevention of Docetaxel (Doc) Chemotherapy (Chemo)-Induced Neutropenia (CIN)

Hematopoietic Engraftment of Fanconi Anemia Patients through 3 Years after Gene Therapy

Long-Term Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar (HGB-204) Study

Durable and Robust Fetal Globin Induction without Anemia in Rhesus Monkeys Following Autologous Hematopoietic Stem Cell Transplant with BCL11A Erythroid Enhancer Editing

Mgta-456, an Aryl Hydrocarbon Receptor (AHR) Antagonist Based Expansion of CD34+ Hematopoietic Stem Cells (HSC), Permits Selection of Better HLA Matched Cord Blood Units (CBUs) and Promotes Faster Neutrophil Recovery and Uniform Engraftment with Potentially Less Acute Graft-Vs-Host Disease (GVHD)

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

Outcomes after Plerixafor Plus FLAG-IDA (PLERIFLAG) Versus FLAG-IDA +/- Gentuzumab for Adult Patients with First Relapsed/Refractory AML: A Propensity Score Analysis from the Pethema Registry

Procarbazine-Free Escalated Beacopdac in Frontline Therapy of Advanced Hodgkin Lymphoma Reduces Red Cell Transfusion Requirements and May Shorten Time to Menstrual Period Recovery Compared to Escalated Beacopp and Appears to be As Efficacious

Phase 1 Study of Decitabine and Vorinostat Followed By Fludarabine, Cytarabine and G-CSF (FLAG) in Children, Adolescents and Young Adults with Relapsed/Refractory AML: Report from the Therapeutic Advances in Childhood Leukemia and Lymphoma (TACL) Consortium

Cladribine, Cytarabine, Granulocyte Colony Stimulating Factor ± Mitoxantrone (CLAG ± M) Is Highly Effective Therapy for Secondary and Relapsed/Refractory Acute Myeloid Leukemia

Enhanced Transduction Lentivector Gene Therapy for Treatment of Older Patients with X-Linked Severe Combined Immunodeficiency

Β-Catenin-Tcf/Lef Signaling Promotes Steady State and Emergency Granulopoiesis through G-CSF Receptor Upregulation

Response to High Dose G-CSF Treatment (20µg/kg/d or Higher) of Patients with Congenital Neutropenia: An Analysis By the Scnir in Europe

15-PGDH Inhibition Mitigates Aplastic Anemia Severity

Phase I Clinical Trial of CK0801 (cord blood regulatory T cells) in Patients with Bone Marrow Failure Syndrome (BMF) Including Aplastic Anemia, Myelodysplasia and Myelofibrosis

Feasibility of Peripheral Blood Stem Cell Collection from Heterozygoussickle Cell Trait Donors

Canonical Signaling By TGF Family Members in Mesenchymal Stromal Cells Is Dispensable for Hematopoietic Niche Maintenance Under Basal and Stress Conditions

Efficient Correction of ELANE mutations in Primary HSPCs of Severe Congenital Neutropenia Patients Using CRISPR/Cas9 and rAVV6 HDR Repair Templates

102 Patients with Primary Autoimmune Neutropenia : An Analysis of the European Severe Chronic Neutropenia International Registry (SCNIR)

Efficacy and Safety of a FLAG-Sequential Regimen Followed By Hematopoietic Stem Cell Transplantation for Myelodysplasia or Acute Leukemia in Fanconi Anemia: A Franco-Brazilian Report

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