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Journal Articles

Revumenib Maintenance Therapy Following Revumenib-Induced Remission and Transplant

Andrius Zucenka, Ghayas C. Issa, Martha Arellano, Sajad Khazal, Nandita Khera, Wendy Stock, Branko Cuglievan, Yu Gu, Huy Van Nguyen, Angela R. Smith, Eytan M. Stein

Blood (2023) 142 (Supplement 1): 4950.

https://doi.org/10.1182/blood-2023-189036

Published: 2023

Journal Articles

Updated Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia

Mark C. Walters, Angela R. Smith, Gary J. Schiller, Erica B. Esrick, David A. Williams, Tatiana Gogoleva, Didier Rouy, Bettina M. Cockroft, Gregory M. Vercellotti

Blood (2021) 138 (Supplement 1): 3974.

https://doi.org/10.1182/blood-2021-147907

Published: 2021

Journal Articles

Low Incidence of Chronic Graft-Versus-Host Disease in Myeloablative Allogeneic Hematopoietic Cell Transplantation with Post-Transplant Cyclophosphamide Using Matched Related or Unrelated Donors: Phase II Study Interim Analysis

Blood (2021) 138 (Supplement 1): 1811.

https://doi.org/10.1182/blood-2021-147569

Published: 2021

Journal Articles

Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report

Lauri M. Burroughs, Aleksandra Petrovic, Ruta Brazauskas, Xuerong Liu, Linda M. Griffith, Hans D. Ochs, Jack J. Bleesing, Stephanie Edwards, Christopher C. Dvorak, Sonali Chaudhury, Susan E. Prockop, Ralph Quinones, Frederick D. Goldman, Troy C. Quigg, Shanmuganathan Chandrakasan, Angela R. Smith, Suhag Parikh, Blachy J. Dávila Saldaña, Monica S. Thakar, Rachel Phelan, Shalini Shenoy, Lisa R. Forbes, Caridad Martinez, Deepak Chellapandian, Evan Shereck, Holly K. Miller, Neena Kapoor, Jessie L. Barnum, Hey Chong, David C. Shyr, Karin Chen, Rolla Abu-Arja, Ami J. Shah, Katja G. Weinacht, Theodore B. Moore, Avni Joshi, Kenneth B. DeSantes, Alfred P. Gillio, Geoffrey D. E. Cuvelier, Michael D. Keller, Jacob Rozmus, Troy Torgerson, Michael A. Pulsipher, Elie Haddad, Kathleen E. Sullivan, Brent R. Logan, Donald B. Kohn, Jennifer M. Puck, Luigi D. Notarangelo, Sung-Yun Pai, David J. Rawlings, Morton J. Cowan

Blood (2020) 135 (23): 2094–2105.

https://doi.org/10.1182/blood.2019002939

Published: 2020

Journal Articles

Romiplostim Improves Platelet Recovery after UCB Transplant

Georgios E. Christakopoulos, MD, Todd E. DeFor, MS, Stefanie M Hage, RN, John E. Wagner, MD, Michael A. Linden, MD PhD, Claudio G Brunstein, MD PhD, Nelli Bejanyan, MD, Michael R Verneris, MD, Angela R. Smith, MD MS

Blood (2019) 134 (Supplement_1): 1979.

https://doi.org/10.1182/blood-2019-125424

Published: 2019

Journal Articles

Preliminary Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia

Angela R. Smith, MD MS, Gary J. Schiller, MD, Gregory M Vercellotti, MD, Janet L. Kwiatkowski, MD MSCE, Lakshmanan Krishnamurti, MD, Erica B. Esrick, MD, David A. Williams, MD, Weston P. Miller, MD, Adrian Woolfson, MD PhD, Mark C. Walters, MD

Blood (2019) 134 (Supplement_1): 3544.

https://doi.org/10.1182/blood-2019-125743

Published: 2019

Journal Articles

Immune Mediated Cytopenias Following Allogeneic Hematopoietic Cell Transplantation for Non-Malignant Disease: Risk Factors and Treatment Response in the Pediatric Population

Robert T. Galvin, MD, Qing Cao, MS, Weston P. Miller, MD, Angela R. Smith, MD MS, Christen L. Ebens, MD MPH

Blood (2018) 132 (Supplement 1): 3377.

https://doi.org/10.1182/blood-2018-99-117718

Published: 2018

Journal Articles

Reduced Toxicity Conditioning Is Better Tolerated but Has Higher Graft Failure Compared to Myeloablative Conditioning in Children with Inherited Metabolic Disorders

Michael Downey, BA, Ashish O. Gupta, MBBS, MPH, Ryan Shanley, M.S, Weston P. Miller, MD, Paul Orchard, MD, Angela R. Smith, MD MS

Blood (2018) 132 (Supplement 1): 3352.

https://doi.org/10.1182/blood-2018-99-117573

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

Final Analysis of Day +100 Survival By Prior Hematopoietic Stem Cell Transplant Type from an Expanded Access Study of Defibrotide for Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome

Paul G Richardson, MD, Angela R. Smith, MD MS, Brandon Triplett, MD, Nancy Kernan, MD, Leslie Lehmann, MD, Robert J. Ryan, Robin L. Hume, MS, William Martin Tappe, MD, Stephan A Grupp, MD PhD

Blood (2017) 130 (Supplement 1): 3224.

https://doi.org/10.1182/blood.V130.Suppl_1.3224.3224

Published: 2017

Journal Articles

Pooled Analysis of Day +100 Survival for Defibrotide-Treated Patients with Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) and Ventilator or Dialysis Dependence Following Hematopoietic Stem Cell Transplantation (HSCT)

Paul G Richardson, MD, Angela R. Smith, MD MS, Nancy A. Kernan, MD, Joseph H. Antin, MD, Leslie Lehmann, MD, Robert J. Ryan, Robin L. Hume, MS, William Martin Tappe, MD, Stephan A Grupp, MD PhD

Blood (2017) 130 (Supplement 1): 1946.

https://doi.org/10.1182/blood.V130.Suppl_1.1946.1946

Published: 2017

Journal Articles

Efficacy and Safety of Defibrotide in Pediatric Patients with Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) after Hematopoietic Stem Cell Transplantation (HSCT): Final Results from the Expanded-Access Program

Nancy A. Kernan, MD, Angela R. Smith, MD MS, Brandon Triplett, MD, Leslie Lehmann, MD, Robert J. Ryan, Wei Liang, Robin L. Hume, MS, William Martin Tappe, MD, Stephan A Grupp, MD PhD

Blood (2017) 130 (Supplement 1): 1948.

https://doi.org/10.1182/blood.V130.Suppl_1.1948.1948

Published: 2017

Journal Articles

Validation of a Modified Comorbidity Index for Allogeneic Hematopoietic Cell Transplant

Todd E. DeFor, MS, Chap Le, PhD, Angela R Smith, MD MS, Erica D. Warlick, MD, Nelli Bejanyan, MD, Daniel J. Weisdorf, MD

Blood (2016) 128 (22): 1167.

https://doi.org/10.1182/blood.V128.22.1167.1167

Published: 2016

Journal Articles

Treatment of Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) Post-Hematopoietic Stem Cell Transplantation (HSCT) in Patients with Acute Leukemias: A Subgroup Analysis from the Defibrotide Expanded-Access Program

Paul G. Richardson, MD, Angela R. Smith, MD, Brandon M. Triplett, MD, Nancy A. Kernan, MD, Stephan A. Grupp, MD PhD, Joseph H. Antin, MD, Leslie Lehmann, MD, Wei Liang, PhD, Robin Hume, MS, William Tappe, MD, Robert J. Soiffer, MD

Blood (2016) 128 (22): 3412.

https://doi.org/10.1182/blood.V128.22.3412.3412

Published: 2016

Journal Articles

Impact on Outcomes of Baseline Bilirubin in Patients with Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome Receiving Defibrotide Treatment: A Post-Hoc Analysis

Blood (2016) 128 (22): 2213.

https://doi.org/10.1182/blood.V128.22.2213.2213

Published: 2016

Journal Articles

Timing of Initiation of Defibrotide Post-Diagnosis of Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome Post-Hematopoietic Stem Cell Transplantation: Exploratory Age-Group Analysis from an Expanded Access Study

Stephan A. Grupp, MD PhD, Angela R. Smith, MD, Brandon M. Triplett, MD, Nancy A. Kernan, MD, Joseph H. Antin, MD, Leslie Lehmann, MD, Wei Liang, PhD, Robin Hume, MS, William Tappe, MD, Paul G. Richardson, MD

Blood (2016) 128 (22): 66.

https://doi.org/10.1182/blood.V128.22.66.66

Published: 2016

Journal Articles

Early Initiation of Defibrotide in Patients with Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome Following Hematopoietic Stem Cell Transplantation Improves Day +100 Survival

Paul G. Richardson, MD, Angela R. Smith, MD MS, Brandon M. Triplett, MD, Nancy A. Kernan, MD, Stephan A. Grupp, MD PhD, Joseph H. Antin, MD, Leslie E. Lehmann, MD, Maja Miloslavsky, PhD, Robin L. Hume, MS, Alison L. Hannah, MD, Bijan Nejadnik, MD, Robert J. Soiffer, MD

Blood (2015) 126 (23): 4311.

https://doi.org/10.1182/blood.V126.23.4311.4311

Published: 2015

Journal Articles

Defibrotide for the Treatment of Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome with Multi-Organ Dysfunction: Final Results from a Pivotal, Historically Controlled, Phase 3 Trial

Blood (2015) 126 (23): 737.

https://doi.org/10.1182/blood.V126.23.737.737

Published: 2015

Journal Articles

The Impact of Graft/Recipient ABO Compatibility on Outcomes after Umbilical Cord Blood Transplant for Non-Malignant Disease

Matthew R Kudek, MD, Ryan Shanley, MS, Nicole D. Zantek, MD, PhD, Angela R. Smith, MD MS, Weston P Miller, IV, MD

Blood (2015) 126 (23): 4369.

https://doi.org/10.1182/blood.V126.23.4369.4369

Published: 2015

NARROW

Revumenib Maintenance Therapy Following Revumenib-Induced Remission and Transplant

Updated Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia

Low Incidence of Chronic Graft-Versus-Host Disease in Myeloablative Allogeneic Hematopoietic Cell Transplantation with Post-Transplant Cyclophosphamide Using Matched Related or Unrelated Donors: Phase II Study Interim Analysis

Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report

Romiplostim Improves Platelet Recovery after UCB Transplant

Preliminary Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia

Immune Mediated Cytopenias Following Allogeneic Hematopoietic Cell Transplantation for Non-Malignant Disease: Risk Factors and Treatment Response in the Pediatric Population

Reduced Toxicity Conditioning Is Better Tolerated but Has Higher Graft Failure Compared to Myeloablative Conditioning in Children with Inherited Metabolic Disorders

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

Final Analysis of Day +100 Survival By Prior Hematopoietic Stem Cell Transplant Type from an Expanded Access Study of Defibrotide for Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome

Pooled Analysis of Day +100 Survival for Defibrotide-Treated Patients with Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) and Ventilator or Dialysis Dependence Following Hematopoietic Stem Cell Transplantation (HSCT)

Efficacy and Safety of Defibrotide in Pediatric Patients with Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) after Hematopoietic Stem Cell Transplantation (HSCT): Final Results from the Expanded-Access Program

Validation of a Modified Comorbidity Index for Allogeneic Hematopoietic Cell Transplant

Treatment of Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) Post-Hematopoietic Stem Cell Transplantation (HSCT) in Patients with Acute Leukemias: A Subgroup Analysis from the Defibrotide Expanded-Access Program

Impact on Outcomes of Baseline Bilirubin in Patients with Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome Receiving Defibrotide Treatment: A Post-Hoc Analysis

Timing of Initiation of Defibrotide Post-Diagnosis of Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome Post-Hematopoietic Stem Cell Transplantation: Exploratory Age-Group Analysis from an Expanded Access Study

Early Initiation of Defibrotide in Patients with Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome Following Hematopoietic Stem Cell Transplantation Improves Day +100 Survival

Defibrotide for the Treatment of Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome with Multi-Organ Dysfunction: Final Results from a Pivotal, Historically Controlled, Phase 3 Trial

The Impact of Graft/Recipient ABO Compatibility on Outcomes after Umbilical Cord Blood Transplant for Non-Malignant Disease

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American Society of Hematology

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American Society of Hematology

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