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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

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

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

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

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

Lentiviral Gene Therapy with Low Dose Busulfan for Infants with X-SCID Results in the Development of a Functional Normal Immune System: Interim Results of an Ongoing Phase I/II Clinical Study

Blood (2019) 134 (Supplement_1): 2058.

https://doi.org/10.1182/blood-2019-126746

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

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

B-cell differentiation and IL-21 response in IL2RG/JAK3 SCID patients after hematopoietic stem cell transplantation

Blood (2018) 131 (26): 2967–2977.

https://doi.org/10.1182/blood-2017-10-809822

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

Reticular dysgenesis: international survey on clinical presentation, transplantation, and outcome

Blood (2017) 129 (21): 2928–2938.

https://doi.org/10.1182/blood-2016-11-745638

Published: 2017

Includes: Supplemental data

Journal Articles

SCID patients with ARTEMIS vs RAG deficiencies following HCT: increased risk of late toxicity in ARTEMIS-deficient SCID

Blood (2014) 123 (2): 281–289.

https://doi.org/10.1182/blood-2013-01-476432

Published: 2014

Includes: Supplemental data

Journal Articles

IL-21 is the primary common γ chain-binding cytokine required for human B-cell differentiation in vivo

Blood (2011) 118 (26): 6824–6835.

https://doi.org/10.1182/blood-2011-06-362533

Published: 2011

Includes: Supplemental data

Journal Articles

Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study

Blood (2011) 118 (6): 1675–1684.

https://doi.org/10.1182/blood-2010-11-319376

Published: 2011

Includes: Supplemental data

Journal Articles

HLA-matched sibling bone marrow transplantation for β-thalassemia major

Mitchell Sabloff, Mammen Chandy, Zhiwei Wang, Brent R. Logan, Ardeshir Ghavamzadeh, Chi-Kong Li, Syed Mohammad Irfan, Christopher N. Bredeson, Morton J. Cowan, Robert Peter Gale, Gregory A. Hale, John Horan, Suradej Hongeng, Mary Eapen, Mark C. Walters

Blood (2011) 117 (5): 1745–1750.

https://doi.org/10.1182/blood-2010-09-306829

Published: 2011

Journal Articles

The graft-versus-leukemia effect using matched unrelated donors is not superior to HLA-identical siblings for hematopoietic stem cell transplantation

Blood (2009) 113 (13): 3110–3118.

https://doi.org/10.1182/blood-2008-07-163212

Published: 2009

Includes: Supplemental data

Journal Articles

Megadose CD34-Selected Haplocompatible Donor Stem Cell Transplantation in Children.

Barbara S. Sleight, MD, Morton J. Cowan, MD, Biljana Horn, MD, Jennifer Jaroscak, MD, Joseph McGuirk, MD, Andrew Gilman, MD

Blood (2006) 108 (11): 5417.

https://doi.org/10.1182/blood.V108.11.5417.5417

Published: 2006

Journal Articles

Effects of B7.2 −/− Mature Dendritic Cells on Tolerance Induction to Alloantigens in Fetal Mice Following In Utero Transplantation with Lineage Depleted Bone Marrow.

Swati Bhattacharyya, PhD, Morton J. Cowan, MD.

Blood (2004) 104 (11): 2134.

https://doi.org/10.1182/blood.V104.11.2134.2134

Published: 2004

Journal Articles

Impact of donor type on outcome of bone marrow transplantation for Wiskott-Aldrich syndrome: collaborative study of the International Bone Marrow Transplant Registry and the National Marrow Donor Program

Blood (2001) 97 (6): 1598–1603.

https://doi.org/10.1182/blood.V97.6.1598

Published: 2001

NARROW

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

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)

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

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

Reduced Toxicity Conditioning for Non-Malignant Hematopoietic Cell Transplants

Lentiviral Gene Therapy with Low Dose Busulfan for Infants with X-SCID Results in the Development of a Functional Normal Immune System: Interim Results of an Ongoing Phase I/II Clinical Study

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

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

B-cell differentiation and IL-21 response in IL2RG/JAK3 SCID patients after hematopoietic stem cell transplantation

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

Reticular dysgenesis: international survey on clinical presentation, transplantation, and outcome

SCID patients with ARTEMIS vs RAG deficiencies following HCT: increased risk of late toxicity in ARTEMIS-deficient SCID

IL-21 is the primary common γ chain-binding cytokine required for human B-cell differentiation in vivo

Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study

HLA-matched sibling bone marrow transplantation for β-thalassemia major

The graft-versus-leukemia effect using matched unrelated donors is not superior to HLA-identical siblings for hematopoietic stem cell transplantation

Megadose CD34-Selected Haplocompatible Donor Stem Cell Transplantation in Children.

Effects of B7.2 −/− Mature Dendritic Cells on Tolerance Induction to Alloantigens in Fetal Mice Following In Utero Transplantation with Lineage Depleted Bone Marrow.

Impact of donor type on outcome of bone marrow transplantation for Wiskott-Aldrich syndrome: collaborative study of the International Bone Marrow Transplant Registry and the National Marrow Donor Program

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American Society of Hematology

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American Society of Hematology

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