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Journal Articles

Genotype, oxidase status, and preceding infection or autoinflammation do not affect allogeneic HCT outcomes for CGD

Jennifer W. Leiding, Danielle E. Arnold, Suhag Parikh, Brent Logan, Rebecca A. Marsh, Linda M. Griffith, Ruizhe Wu, Sharon Kidd, Kanwaldeep Mallhi, Deepak Chellapandian, Stephanie J. Si Lim, Eyal Grunebaum, E. Liana Falcone, Luis Murguia-Favela, Debbi Grossman, Vinod K. Prasad, Jennifer R. Heimall, Fabien Touzot, Lauri M. Burroughs, Jack Bleesing, Neena Kapoor, Jasmeen Dara, Olatundun Williams, Malika Kapadia, Benjamin R. Oshrine, Jeffrey J. Bednarski, Ahmad Rayes, Hey Chong, Geoffrey D. E. Cuvelier, Lisa R. Forbes Satter, Caridad Martinez, Mark T. Vander Lugt, Lolie C. Yu, Shanmuganathan Chandrakasan, Avni Joshi, Susan E. Prockop, Blachy J. Dávila Saldaña, Victor Aquino, Larisa A. Broglie, Christen L. Ebens, Lisa M. Madden, Kenneth DeSantes, Jordan Milner, Hemalatha G. Rangarajan, Ami J. Shah, Alfred P. Gillio, Alan P. Knutsen, Holly K. Miller, Theodore B. Moore, Pamela Graham, Andrea Bauchat, Nancy J. Bunin, Pierre Teira, Aleksandra Petrovic, Sharat Chandra, Hisham Abdel-Azim, Morna J. Dorsey, Olga Birbrayer, Morton J. Cowan, Christopher C. Dvorak, Elie Haddad, Donald B. Kohn, Luigi D. Notarangelo, Sung-Yun Pai, Jennifer M. Puck, Michael A. Pulsipher, Troy R. Torgerson, Harry L. Malech, Elizabeth M. Kang

Blood (2023) 142 (24): 2105–2118.

https://doi.org/10.1182/blood.2022019586

Published: 2023

Includes: Supplemental data

Journal Articles

Real-World Analysis of the Underdiagnosis, Clinical Outcomes and Associated Burden of Hematopoietic Stem Cell Transplantation-Associated Thrombotic Microangiopathy (HSCT-TMA) in the United States of America

Yan Wang, Andrew Rava, Marlene Smuzynski, Bonny Shah, Anusorn Thanataveerat, Imad Al Dakkak, Moh-Lim Ong, Christopher C Dvorak, Vincent Ho

Blood (2023) 142 (Supplement 1): 491.

https://doi.org/10.1182/blood-2023-177671

Published: 2023

Journal Articles

High Exposure of Rabbit Anti-Thymocyte Globulin (ATG) Is Strongly Associated with Inferior DFS in Pediatric Patients with Malignancy Undergoing Alpha-Beta T-Cell Depleted Haploidentical HCT

Christopher C Dvorak, Lucia Holbrook-Brown, Janel R. Long-Boyle, Hisham Abdel-Azim, Anant Vatsayan, Julie-An Talano, Nancy Bunin, Eric Jon Anderson, Allyson Flower, Nahal Lalefar, Christine S Higham, Neena Kapoor, Maryanne Odinakachukwu, David A Jacobsohn, Michael A. Pulsipher

Blood (2023) 142 (Supplement 1): 3533.

https://doi.org/10.1182/blood-2023-178925

Published: 2023

Journal Articles

Risk Stratified Treatment for Patients with Newly Diagnosed Juvenile Myelomonocytic Leukemia: A Phase 1/2 Non-Randomized Study of Trametinib and Azacitidine with or without Chemotherapy

Elliot Stieglitz, Yueh-Yun Chi, Bill H. Chang, Sarah K. Tasian, Marielle Yohe, Christopher C Dvorak, Erica Southworth, Janel R. Long-Boyle, Jessica Van Ziffle, Zied Abdullaev, Roy Leong, Alan S. Wayne, Deepa Bhojwani, Mignon L. Loh

Blood (2023) 142 (Supplement 1): 3210.

https://doi.org/10.1182/blood-2023-177838

Published: 2023

Journal Articles

Treatment of Refractory Adenovirus (ADV) Infections Using Adv-Specific Cytotoxic T-Lymphocytes (CTLs) in Children, Adolescents and Young Adults (CAYA) Post Allogeneic Hematopoietic Stem Cell Transplantation (AlloHSCT), Solid Organ Transplantation (SOT), or with Primary Immunodeficiency (PID)

Blood (2023) 142 (Supplement 1): 4836.

https://doi.org/10.1182/blood-2023-188013

Published: 2023

Journal Articles

KIR-favorable TCR-αβ/CD19-depleted haploidentical HCT in children with ALL/AML/MDS: primary analysis of the PTCTC ONC1401 trial

Michael A. Pulsipher, Kwang W. Ahn, Nancy J. Bunin, Nahal Lalefar, Eric Anderson, Allyson Flower, Mitchell S. Cairo, Julie-An Talano, Sonali Chaudhury, Carrie L. Kitko, Jamie L. Duke, Dimitrios Monos, Wing Leung, Christopher C. Dvorak, Hisham Abdel-Azim

Blood (2022) 140 (24): 2556–2572.

https://doi.org/10.1182/blood.2022015959

Published: 2022

Includes: Supplemental data

Journal Articles

MAC Plus Study: A Retrospective Study Correlating DNA Variants, Clinical Course and Outcomes from 187 Patients Who Underwent Testing Using an Atypical Hemolytic Uremic Syndrome and Thrombotic Microangiopathy Genetic Sequencing Panel

Jihyun Moon, Jamey Kain, Fernando C. Fervenza, Stephen B Erickson, Prince Singh, Shahrzad Tehranian, Craig B. Langman, Ramy Hanna, Paul Brakeman, Christopher C. Dvorak, Shaun David Lawicki, Amita Sharma, Jooeun Lim, Sujatha Venkataramani, Mike Ero, Bradley Lewis

Blood (2022) 140 (Supplement 1): 2776–2777.

https://doi.org/10.1182/blood-2022-162838

Published: 2022

Journal Articles

Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC

Geoffrey D. E. Cuvelier, Brent R. Logan, Susan E. Prockop, Rebecca H. Buckley, Caroline Y. Kuo, Linda M. Griffith, Xuerong Liu, Alison Yip, Michael S. Hershfield, Paul G. Ayoub, Theodore B. Moore, Morna J. Dorsey, Richard J. O’Reilly, Neena Kapoor, Sung-Yun Pai, Malika Kapadia, Christen L. Ebens, Lisa R. Forbes Satter, Lauri M. Burroughs, Aleksandra Petrovic, Deepak Chellapandian, Jennifer Heimall, David C. Shyr, Ahmad Rayes, Jeffrey J. Bednarski, Sharat Chandra, Shanmuganathan Chandrakasan, Alfred P. Gillio, Lisa Madden, Troy C. Quigg, Emi H. Caywood, Blachy J. Dávila Saldaña, Kenneth DeSantes, Hesham Eissa, Frederick D. Goldman, Jacob Rozmus, Ami J. Shah, Mark T. Vander Lugt, Monica S. Thakar, Roberta E. Parrott, Caridad Martinez, Jennifer W. Leiding, Troy R. Torgerson, Michael A. Pulsipher, Luigi D. Notarangelo, Morton J. Cowan, Christopher C. Dvorak, Elie Haddad, Jennifer M. Puck, Donald B. Kohn

Blood (2022) 140 (7): 685–705.

https://doi.org/10.1182/blood.2022016196

Published: 2022

Includes: Supplemental data

Journal Articles

JSP191 As a Single-Agent Conditioning Regimen Results in Successful Engraftment, Donor Myeloid Chimerism, and Production of Donor Derived Naïve Lymphocytes in Patients with Severe Combined Immunodeficiency (SCID)

Blood (2021) 138 (Supplement 1): 554.

https://doi.org/10.1182/blood-2021-153074

Published: 2021

Journal Articles

The pulmonary metatranscriptome prior to pediatric HCT identifies post-HCT lung injury

Matt S. Zinter, Caroline A. Lindemans, Birgitta A. Versluys, Madeline Y. Mayday, Sara Sunshine, Gustavo Reyes, Marina Sirota, Anil Sapru, Michael A. Matthay, Sandhya Kharbanda, Christopher C. Dvorak, Jaap J. Boelens, Joseph L. DeRisi

Blood (2021) 137 (12): 1679–1689.

https://doi.org/10.1182/blood.2020009246

Published: 2021

Includes: Supplemental data

Journal Articles

Conditioning Regimens and Outcomes after Allogeneic Hematopoietic Cell Transplant for Hyperinflammatory Inborn Errors of Immunity

Rebecca A. Marsh, Soyoung Kim, Kyle Hebert, Christopher C. Dvorak, MD, Victor Aquino, MD, K. Scott Scott Baker, MD MS, Deepak Chellapandian, MD, Blachy Davila, MD, Christine Duncan, MD, Michael J. Eckrich, MD MPH, George E. Georges, MD, Timothy S. Olson, MD PhD, Michael A Pulsipher, MD, Shalini Shenoy, MD, Elizabeth Stenger, MD, Mark Vander Lugt, MD, Lolie C. Yu, MD, Andrew Gennery, MD, Mary Eapen, MBBS, MS

Blood (2020) 136 (Supplement 1): 36–37.

https://doi.org/10.1182/blood-2020-139930

Published: 2020

Journal Articles

In Utero Stem Cell Transplantation in Patients with Alpha Thalassemia Major: Interim Results of a Phase 1 Clinical Trial

Tippi C. MacKenzie, MD, Michela Frascoli, Renan Sper, Billie Rachael Lianoglou, MS, Juan Gonzalez Velez, Christopher C. Dvorak, MD, Sandhya Kharbanda, MD, Elliott Vichinsky, MD

Blood (2020) 136 (Supplement 1): 1.

https://doi.org/10.1182/blood-2020-142698

Published: 2020

Journal Articles

First Report of Non-Genotoxic Conditioning with JSP191 (anti-CD117) and Hematopoietic Stem Cell Transplantation in a Newly Diagnosed Patient with Severe Combined Immune Deficiency

Blood (2020) 136 (Supplement 1): 10.

https://doi.org/10.1182/blood-2020-137762

Published: 2020

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

Incidence and Cost of Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome with and without Multi-Organ Dysfunction: Analysis of the Premier Healthcare Database

Christopher C. Dvorak, MD, Zhun Cao, PhD, Kwanza Price, MPH, JeanPierre Coaquira, Craig Lipkin, Scott Robinson, Partow Kebriaei, MD

Blood (2019) 134 (Supplement_1): 4706.

https://doi.org/10.1182/blood-2019-123499

Published: 2019

Journal Articles

Next Generation Sequence Minimal Residual Disease (NGS-MRD) Predicts Outstanding Event Free Survival (EFS) Regardless of Hematopoietic Cell Transplantation (HCT) Preparative Approach or Graft Alpha/Beta Depletion in Children with Acute Lymphoblastic Leukemia (ALL)

Hisham Abdel-Azim, Christopher C. Dvorak, MD, Nancy Bunin, MD, Mark C. Walters, MD, Julie-An Talano, MD, Eric Jon Anderson, MD, Jemily Malvar, MS, May Al Jassani, Sonali Chaudhury, MBBS,MD, Mitchell S Cairo, MD, Carrie Kitko, MD, Allison P. Jacob, Dimitri S. Monos, PhD, Leung Wing, MD PhD, Michael A. Pulsipher, MD

Blood (2019) 134 (Supplement_1): 4624.

https://doi.org/10.1182/blood-2019-131706

Published: 2019

Journal Articles

Non-Genotoxic Anti-CD117 Antibody Conditioning Results in Successful Hematopoietic Stem Cell Engraftment in Patients with Severe Combined Immunodeficiency

Rajni Agarwal, MD, Christopher C. Dvorak, MD, Hye-Sook Kwon, PhD, Janel R Long-Boyle, PharmD, PhD, Susan S Prohaska, PhD MS, Janice W Brown, MD, Anne Le, Alyssa Guttman-Klein, Irving L Weissman, MD, Morton J Cowan, MD, Aaron C. Logan, MD PhD, Kenneth I. Weinberg, MD, Robertson Parkman, MD, Maria-Grazia Roncarolo, MD, Judith A Shizuru, MD PhD

Blood (2019) 134 (Supplement_1): 800.

https://doi.org/10.1182/blood-2019-126239

Published: 2019

Journal Articles

Reduced Toxicity Conditioning for Non-Malignant Hematopoietic Cell Transplants

Cristina F Contreras, BS, Janel R Long-Boyle, PharmD, PhD, Kristin A. Shimano, MD, Jasmeen Dara, MD, James N Huang, MD, Sandhya Kharbanda, MD, Alexis Melton, MD PhD, Christine Higham, MD, Morton J Cowan, MD, Christopher C. Dvorak, MD

Blood (2019) 134 (Supplement_1): 4481.

https://doi.org/10.1182/blood-2019-127218

Published: 2019

Journal Articles

Role of Disease Mechanism in Hematopoietic Cell Transplantation Outcomes for Hemophagocytic Lymphohistiocytosis

Aleksandra S. Dain, MD, Michelle L. Hermiston, MD PhD, Kristin A. Shimano, MD, Sandhya Kharbanda, MD, Alexis Melton, MD PhD, Jasmeen Dara, MD, James N Huang, MD, Christopher C. Dvorak, MD

Blood (2019) 134 (Supplement_1): 3343.

https://doi.org/10.1182/blood-2019-127397

Published: 2019

Journal Articles

In ALD, I feel the need for speed

Sandhya Kharbanda, Christopher C. Dvorak

Blood (2019) 133 (12): 1275–1277.

https://doi.org/10.1182/blood-2019-02-897629

Published: 2019

NARROW

Genotype, oxidase status, and preceding infection or autoinflammation do not affect allogeneic HCT outcomes for CGD

Real-World Analysis of the Underdiagnosis, Clinical Outcomes and Associated Burden of Hematopoietic Stem Cell Transplantation-Associated Thrombotic Microangiopathy (HSCT-TMA) in the United States of America

High Exposure of Rabbit Anti-Thymocyte Globulin (ATG) Is Strongly Associated with Inferior DFS in Pediatric Patients with Malignancy Undergoing Alpha-Beta T-Cell Depleted Haploidentical HCT

Risk Stratified Treatment for Patients with Newly Diagnosed Juvenile Myelomonocytic Leukemia: A Phase 1/2 Non-Randomized Study of Trametinib and Azacitidine with or without Chemotherapy

Treatment of Refractory Adenovirus (ADV) Infections Using Adv-Specific Cytotoxic T-Lymphocytes (CTLs) in Children, Adolescents and Young Adults (CAYA) Post Allogeneic Hematopoietic Stem Cell Transplantation (AlloHSCT), Solid Organ Transplantation (SOT), or with Primary Immunodeficiency (PID)

KIR-favorable TCR-αβ/CD19-depleted haploidentical HCT in children with ALL/AML/MDS: primary analysis of the PTCTC ONC1401 trial

MAC Plus Study: A Retrospective Study Correlating DNA Variants, Clinical Course and Outcomes from 187 Patients Who Underwent Testing Using an Atypical Hemolytic Uremic Syndrome and Thrombotic Microangiopathy Genetic Sequencing Panel

Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC

JSP191 As a Single-Agent Conditioning Regimen Results in Successful Engraftment, Donor Myeloid Chimerism, and Production of Donor Derived Naïve Lymphocytes in Patients with Severe Combined Immunodeficiency (SCID)

The pulmonary metatranscriptome prior to pediatric HCT identifies post-HCT lung injury

Conditioning Regimens and Outcomes after Allogeneic Hematopoietic Cell Transplant for Hyperinflammatory Inborn Errors of Immunity

In Utero Stem Cell Transplantation in Patients with Alpha Thalassemia Major: Interim Results of a Phase 1 Clinical Trial

First Report of Non-Genotoxic Conditioning with JSP191 (anti-CD117) and Hematopoietic Stem Cell Transplantation in a Newly Diagnosed Patient with Severe Combined Immune Deficiency

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

Incidence and Cost of Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome with and without Multi-Organ Dysfunction: Analysis of the Premier Healthcare Database

Next Generation Sequence Minimal Residual Disease (NGS-MRD) Predicts Outstanding Event Free Survival (EFS) Regardless of Hematopoietic Cell Transplantation (HCT) Preparative Approach or Graft Alpha/Beta Depletion in Children with Acute Lymphoblastic Leukemia (ALL)

Non-Genotoxic Anti-CD117 Antibody Conditioning Results in Successful Hematopoietic Stem Cell Engraftment in Patients with Severe Combined Immunodeficiency

Reduced Toxicity Conditioning for Non-Malignant Hematopoietic Cell Transplants

Role of Disease Mechanism in Hematopoietic Cell Transplantation Outcomes for Hemophagocytic Lymphohistiocytosis

In ALD, I feel the need for speed

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American Society of Hematology

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American Society of Hematology

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