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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

Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia Following Completion of the Northstar HGB-204 Study

John E.J. Rasko, MBBS, PhD, Alexis A. Thompson, MD MPH, Janet L. Kwiatkowski, MD, 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, Philippe Leboulch, MD, Elliott Vichinsky, MD, Briana Deary, Ying Chen, PhD, Mohammed Asmal, MD PhD, Mark C. Walters, MD

Blood (2018) 132 (Supplement 1): 167.

https://doi.org/10.1182/blood-2018-167

Published: 2018

Journal Articles

SCID genotype and 6-month posttransplant CD4 count predict survival and immune recovery

Elie Haddad, Brent R. Logan, Linda M. Griffith, Rebecca H. Buckley, Roberta E. Parrott, Susan E. Prockop, Trudy N. Small, Jessica Chaisson, Christopher C. Dvorak, Megan Murnane, Neena Kapoor, Hisham Abdel-Azim, Imelda C. Hanson, Caridad Martinez, Jack J. H. Bleesing, Sharat Chandra, Angela R. Smith, Matthew E. Cavanaugh, Soma Jyonouchi, Kathleen E. Sullivan, Lauri Burroughs, Suzanne Skoda-Smith, Ann E. Haight, Audrey G. Tumlin, Troy C. Quigg, Candace Taylor, Blachy J. Dávila Saldaña, Michael D. Keller, Christine M. Seroogy, Kenneth B. Desantes, Aleksandra Petrovic, Jennifer W. Leiding, David C. Shyr, Hélène Decaluwe, Pierre Teira, Alfred P. Gillio, Alan P. Knutsen, Theodore B. Moore, Morris Kletzel, John A. Craddock, Victor Aquino, Jeffrey H. Davis, Lolie C. Yu, Geoffrey D. E. Cuvelier, Jeffrey J. Bednarski, Frederick D. Goldman, Elizabeth M. Kang, Evan Shereck, Matthew H. Porteus, James A. Connelly, Thomas A. Fleisher, Harry L. Malech, William T. Shearer, Paul Szabolcs, Monica S. Thakar, Mark T. Vander Lugt, Jennifer Heimall, Ziyan Yin, Michael A. Pulsipher, Sung-Yun Pai, Donald B. Kohn, Jennifer M. Puck, Morton J. Cowan, Richard J. O'Reilly, Luigi D. Notarangelo

Blood (2018) 132 (17): 1737–1749.

https://doi.org/10.1182/blood-2018-03-840702

Published: 2018

Includes: Supplemental data

Journal Articles

Immune reconstitution and survival of 100 SCID patients post–hematopoietic cell transplant: a PIDTC natural history study

Blood (2017) 130 (25): 2718–2727.

https://doi.org/10.1182/blood-2017-05-781849

Published: 2017

Includes: Supplemental data

Journal Articles

Clinical Outcomes up to 3 Years Following Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar Hgb-204 Study

Janet L. Kwiatkowski, MD, Alexis A Thompson, MD MPH, John Rasko, MBBS, PhD, Suradej Hongeng, MD, Gary J. Schiller, MD, Usanarat Anurathapan, Marina Cavazzana, MD PhD, Phoebe Joy Ho, Christof von Kalle, MD, Morris Kletzel, MD MBA, Philippe Leboulch, MD, Elliott Vichinsky, MD, Briana Deary, Mohammed Asmal, MD PhD, Mark C. Walters, MD

Blood (2017) 130 (Supplement 1): 360.

https://doi.org/10.1182/blood.V130.Suppl_1.360.360

Published: 2017

Journal Articles

Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia: Update from the Northstar Hgb-204 Phase 1/2 Clinical Study

Alexis A. Thompson, MD MPH, Janet Kwiatkowski, MD, John Rasko, MBBS, PhD, Suradej Hongeng, MD, Gary J. Schiller, MD, Usanarat Anurathapan, MD, Marina Cavazzana, MD PhD, P. Joy Ho, Christof von Kalle, MD, Morris Kletzel, MD FAAP, MBA, Philippe Leboulch, MD, Elliott Vichinsky, MD, Alexandria Petrusich, Mohammed Asmal, MD PhD, Mark C. Walters, MD

Blood (2016) 128 (22): 1175.

https://doi.org/10.1182/blood.V128.22.1175.1175

Published: 2016

Journal Articles

Update of Results from the Northstar Study (HGB-204): A Phase 1/2 Study of Gene Therapy for Beta-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex-Vivo with a Lentiviral Beta AT87Q-Globin Vector (LentiGlobin BB305 Drug Product)

Mark C. Walters, MD, John Rasko, MBBS, PhD, Suradej Hongeng, MD, Janet Kwiatkowski, MD, Gary J Schiller, MD, Morris Kletzel, MD, P. Joy Ho, MBBS, DPhil, Elliott Vichinsky, MD, Christof von Kalle, MD, Marina Cavazzana, MD PhD, Philippe Leboulch, MD, Alexandria Petrusich, Sandeep Soni, MD, Alexis A. Thompson, MD

Blood (2015) 126 (23): 201.

https://doi.org/10.1182/blood.V126.23.201.201

Published: 2015

Journal Articles

Robust Immune Reconstitution in Children with Severe Primary Immunodeficiency after Reduced-Intensity Conditioning Hematopoietic Stem Cell Transplantation

William T. Tse, MD PhD, Jessica Ward, MSN, cPNP, Jennifer Schneiderman, MD MS, Sonali Chaudhury, MD, Ramsay Fuleihan, MD, Reggie Duerst, MD, Morris Kletzel, MD

Blood (2014) 124 (21): 3923.

https://doi.org/10.1182/blood.V124.21.3923.3923

Published: 2014

Journal Articles

Transplantation for children with acute myeloid leukemia: a comparison of outcomes with reduced intensity and myeloablative regimens

Blood (2014) 123 (10): 1615–1620.

https://doi.org/10.1182/blood-2013-10-535716

Published: 2014

Journal Articles

Does Methotrexate Affect Transplant Outcomes? Transplant Related Mortality (TRM), Engraftment, Graft Vs. Host Disease (GvHD), Overall Survival (OS) and Event Free Survival (EFS) in 142 Consecutive Pediatric Cord Blood Transplants (CBT) At Children's Memorial Hospital (CMH)

Morris Kletzel, MD, Mebhoob Merchant, Nazneen Merchant, CRA, Alfred Rademaker, STATISTICIAN PHD, Reggie Duerst, MD, Sonali Chaudhury, M.D., Jennifer Schneiderman, MD, MS, William T. Tse, MD, PhD

Blood (2011) 118 (21): 3028.

https://doi.org/10.1182/blood.V118.21.3028.3028

Published: 2011

Journal Articles

A Low-Cost, Open-Source Informatics Framework for Clinical Trials and Outcomes Research

William T. Tse, MD, PhD, Kevin K. Duh, Morris Kletzel, MD

Blood (2011) 118 (21): 4763.

https://doi.org/10.1182/blood.V118.21.4763.4763

Published: 2011

Journal Articles

Juvenile Myelo-Monocytic Leukemia (JMML): the Effect of Granulocyte Monocyte-Colony Stimulating Factor (GM-CSF) on Wilms Tumor Gene ( WT1 ) by Quantitative Real Time-Polymerase Chain Reaction (q-RT-PCR) and Flow Cytometry

Morris Kletzel, MD, Sana Khan, Wei Huang, Marie Olszewski

Blood (2010) 116 (21): 5063.

https://doi.org/10.1182/blood.V116.21.5063.5063

Published: 2010

Journal Articles

Pediatric Myelodysplastic Syndromes and Allogeneic Hematopoietic Progenitor Cell Transplantation (HPCT): A Single Center Perspective.

Jeffrey R. Andolina, MD, Morris Kletzel, MD, David Jacobsohn, MD, ScM, William T. Tse, MD, PhD, Jennifer Schneiderman, MD, MS, Reggie Duerst, MD, Irene Helenowski, Sonali Chaudhury, M.D.

Blood (2009) 114 (22): 1780.

https://doi.org/10.1182/blood.V114.22.1780.1780

Published: 2009

Journal Articles

Reduced-intensity allogeneic transplantation in pediatric patients ineligible for myeloablative therapy: results of the Pediatric Blood and Marrow Transplant Consortium Study ONC0313

Michael A. Pulsipher, Kenneth M. Boucher, Donna Wall, Haydar Frangoul, Michel Duval, Rakesh K. Goyal, Peter J. Shaw, Ann E. Haight, Michael Grimley, Stephan A. Grupp, Morris Kletzel, Richard Kadota

Blood (2009) 114 (7): 1429–1436.

https://doi.org/10.1182/blood-2009-01-196303

Published: 2009

Includes: Supplemental data

Journal Articles

Age-Dependent Pharmacokinetic Profile of Single Daily Dose Intravenous Busulfan in Children Undergoing Reduced-Intensity Conditioning Stem Cell Transplantation

William T. Tse, MD, PhD, Reggie Duerst, MD, Jennifer Schneiderman, MD, MS, Sonali Chaudhury, M.D., David Jacobsohn, MD, Morris Kletzel, MD

Blood (2008) 112 (11): 796.

https://doi.org/10.1182/blood.V112.11.796.796

Published: 2008

Journal Articles

Potential Increase in Graft Failure Risk after Reduced Intensity Conditioning Stem Cell Transplant (RIT) Using Umbilical Cord Blood for Children with Primary Immunodeficiency.

Jessica Guarino, MSN, MPH, Morris Kletzel, M.D., Reggie Duerst, M.D., David Jacobsohn, M.D., M.S., Jennifer Schneiderman, M.D., M.S., William T. Tse, M.D., Ph.D.

Blood (2007) 110 (11): 2006.

https://doi.org/10.1182/blood.V110.11.2006.2006

Published: 2007

Journal Articles

Successful Engraftment Using Varied Stem Cell Sources, Low Toxicity, and Long-Term Survival Using a Bu/Flu/ATG Reduced Intensity Allogeneic Transplantation in High Risk Pediatric Patients Ineligible for Myelablative Therapy: Results of the Pediatric Blood and Marrow Transplant Consortium (PBMTC) Study ONC0313.

Michael A. Pulsipher, Haydar Frangoul, Michel Duval, Rakesh Goyal, Peter J. Shaw, Ann Haight, Richard Kadota, Michael Grimley, Stephan A. Grupp, Morris Kletzel

Blood (2007) 110 (11): 623.

https://doi.org/10.1182/blood.V110.11.623.623

Published: 2007

Journal Articles

Reduced Intensity Conditioning (RIC) and Hematopoietic Stem Cell Transpalntation (HSCT) Utilizing Extracorporeal Photopheresis (ECP), Fludarabine, and Targeted Dose Busulfan in Children.

Jennifer Schneiderman, MD, MS, Reggie Duerst, MD, Kim Campbell, PhD, Amy Krutsick, MS, William Tse, MD, PhD, Kimberly Thormann, CPNP, Morris Kletzel, MD, David A. Jacobsohn, MD

Blood (2006) 108 (11): 5307.

https://doi.org/10.1182/blood.V108.11.5307.5307

Published: 2006

Journal Articles

Reduced Intensity Conditioning (RIC) with FLU-BU-ATG and Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) for Pediatric Malignancies.

Reggie E. Duerst, MD, David A. Jacobsohn, MD, William Tse, MD, PhD, Morris Kletzel, MD

Blood (2006) 108 (11): 5377.

https://doi.org/10.1182/blood.V108.11.5377.5377

Published: 2006

Journal Articles

Why target the WT1 gene?

Morris Kletzel

Blood (2006) 107 (12): 4578–4579.

https://doi.org/10.1182/blood-2006-03-012187

Published: 2006

NARROW

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

Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia Following Completion of the Northstar HGB-204 Study

SCID genotype and 6-month posttransplant CD4 count predict survival and immune recovery

Immune reconstitution and survival of 100 SCID patients post–hematopoietic cell transplant: a PIDTC natural history study

Clinical Outcomes up to 3 Years Following Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar Hgb-204 Study

Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia: Update from the Northstar Hgb-204 Phase 1/2 Clinical Study

Update of Results from the Northstar Study (HGB-204): A Phase 1/2 Study of Gene Therapy for Beta-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex-Vivo with a Lentiviral Beta AT87Q-Globin Vector (LentiGlobin BB305 Drug Product)

Robust Immune Reconstitution in Children with Severe Primary Immunodeficiency after Reduced-Intensity Conditioning Hematopoietic Stem Cell Transplantation

Transplantation for children with acute myeloid leukemia: a comparison of outcomes with reduced intensity and myeloablative regimens

Does Methotrexate Affect Transplant Outcomes? Transplant Related Mortality (TRM), Engraftment, Graft Vs. Host Disease (GvHD), Overall Survival (OS) and Event Free Survival (EFS) in 142 Consecutive Pediatric Cord Blood Transplants (CBT) At Children's Memorial Hospital (CMH)

A Low-Cost, Open-Source Informatics Framework for Clinical Trials and Outcomes Research

Juvenile Myelo-Monocytic Leukemia (JMML): the Effect of Granulocyte Monocyte-Colony Stimulating Factor (GM-CSF) on Wilms Tumor Gene ( WT1 ) by Quantitative Real Time-Polymerase Chain Reaction (q-RT-PCR) and Flow Cytometry

Pediatric Myelodysplastic Syndromes and Allogeneic Hematopoietic Progenitor Cell Transplantation (HPCT): A Single Center Perspective.

Reduced-intensity allogeneic transplantation in pediatric patients ineligible for myeloablative therapy: results of the Pediatric Blood and Marrow Transplant Consortium Study ONC0313

Age-Dependent Pharmacokinetic Profile of Single Daily Dose Intravenous Busulfan in Children Undergoing Reduced-Intensity Conditioning Stem Cell Transplantation

Potential Increase in Graft Failure Risk after Reduced Intensity Conditioning Stem Cell Transplant (RIT) Using Umbilical Cord Blood for Children with Primary Immunodeficiency.

Reduced Intensity Conditioning (RIC) and Hematopoietic Stem Cell Transpalntation (HSCT) Utilizing Extracorporeal Photopheresis (ECP), Fludarabine, and Targeted Dose Busulfan in Children.

Reduced Intensity Conditioning (RIC) with FLU-BU-ATG and Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) for Pediatric Malignancies.

Why target the WT1 gene?

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American Society of Hematology

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ASH Publications

American Society of Hematology

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