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Journal Articles

Clinical and functional spectrum of RAC2-related immunodeficiency

Blood (2024) 143 (15): 1476–1487.

https://doi.org/10.1182/blood.2023022098

Published: 2024

Includes: Supplemental data

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

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

Treatment of Individuals with Severe Sickle Cell Disease with OTQ923, an Autologous, Ex Vivo , CRISPR/Cas9-Edited, CD34+ Hematopoietic Stem and Progenitor Cell Product, Leads to Durable Engraftment and Fetal Hemoglobin Induction

Akshay Sharma, Jaap Jan Boelens, Maria I. Cancio, Jane Hankins, Prafulla A Bhad, Andrew Lewandowski, Xiaojun Zhao, Shripad Chitnis, Radhika Peddinti, Yan Zheng, Neena Kapoor, Fabio Ciceri, Jianping Yuan, Vionnie W. Yu, Susan C. Stevenson, Serena De Vita, James LaBelle

Blood (2022) 140 (Supplement 1): 1906–1908.

https://doi.org/10.1182/blood-2022-166254

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

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

Administration of BPX-501 Cells Following Aβ T and B-Cell-Depleted HLA Haploidentical HSCT (haplo-HSCT) in Children with Acute Leukemias

Franco Locatelli, Annalisa Ruggeri, MD PhD, Pietro Merli, MD, Swati Naik, MBBS, Rajni Agarwal, MD, Victor Aquino, MD, David A. Jacobsohn, MD, Waseem Qasim, MD PhD, Eneida R. Nemecek, MD, Lakshmanan Krishnamurti, MD, Deepa Manwani, MD, Melissa Kuhn, PharmD, Neena Kapoor

Blood (2018) 132 (Supplement 1): 307.

https://doi.org/10.1182/blood-2018-99-119481

Published: 2018

Journal Articles

Administration of Rimiducid Following Haploidentical BPX-501 Cells in Children with Malignant or Non-Malignant Disorders Who Develop Graft-Versus-Host-Disease (GvHD)

Reem Elkeky, MBBS, David A. Jacobsohn, MD, Rajni Agarwal, MD, Swati Naik, MBBS, Neena Kapoor, MD, Lakshmanan Krishnamurti, MD, Mary Slatter, MD, Federica Galaverna, MD, Pietro Merli, MD, Melissa Aldinger, PharmD, Franco Locatelli

Blood (2018) 132 (Supplement 1): 2207.

https://doi.org/10.1182/blood-2018-99-119792

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

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

Allogeneic Hematopoietic Stem Cell Transplantation in Older Patients Aged 50 Years or Older with Severe Aplastic Anaemia: Results from the European Society for Blood and Marrow Transplant and the Center for International Blood and Marrow Transplant Research

Blood (2017) 130 (Supplement 1): 2446.

https://doi.org/10.1182/blood.V130.Suppl_1.2446.2446

Published: 2017

Journal Articles

Outcome of Children with Primary Immune-Deficiencies (PIDs) Enrolled in a Phase I-II Trial Based on the Infusion of BPX-501 Donor T Cells Genetically Modified with a Novel Suicide Gene (inducible Caspase 9, iC9) after T-Cell Depleted HLA-Haploidentical Allogeneic Stem Cell Transplantation (haplo-HSCT)

Neena Kapoor, MD, Alice Bertaina, MD PhD, Pietro Merli, MD, Swati Naik, MD, Waseem Qasim, MBBS PhD, Mary Slatter, MD, Reem Elfeky, MD, Barbarella Lucarelli, MD, Monika Smogorzewska, PhD, Giuseppina Li Pira, PhD, Valentina Bertaina, PhD, Annemarie Moseley, MD PhD, Franco Locatelli, MD

Blood (2016) 128 (22): 72.

https://doi.org/10.1182/blood.V128.22.72.72

Published: 2016

Journal Articles

Improved outcomes associated with hematopoietic stem cell transplantation for patients with juvenile myelomonocytic leukemia

Quan Zhao, Kris Michael Mahadeo, Neena Kapoor, Hisham Abdel-Azim

Blood (2015) 126 (4): 561–562.

https://doi.org/10.1182/blood-2015-05-644161

Published: 2015

Journal Articles

Hematopoietic stem cell transplantation for infantile osteopetrosis

Paul J. Orchard, Anders L. Fasth, Jennifer Le Rademacher, Wensheng He, Jaap Jan Boelens, Edwin M. Horwitz, Amal Al-Seraihy, Mouhab Ayas, Carmem M. Bonfim, Farid Boulad, Troy Lund, David K. Buchbinder, Neena Kapoor, Tracey A. O’Brien, Miguel A. Diaz Perez, Paul A. Veys, Mary Eapen

Blood (2015) 126 (2): 270–276.

https://doi.org/10.1182/blood-2015-01-625541

Published: 2015

Journal Articles

Long Term Humoral Immune Reconstitution Kinetics After Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) In Children

Amro Elshoury, MD, Neena Kapoor, MD, Ami J Shah, MD, Bhakti Mehta, MD, Kris M. Mahadeo, MD, Hisham Abdel-Azim, MD

Blood (2013) 122 (21): 4627.

https://doi.org/10.1182/blood.V122.21.4627.4627

Published: 2013

Journal Articles

Improved Outcomes After Reduced Intensity Conditioning Matched Unrelated Donor Hematopoietic Stem Cell Transplantation In Children With Chronic Granulomatous Disease

Bhakti Mehta, MD, Kris M. Mahadeo, MD, Ami J Shah, MD, Neena Kapoor, MD, Hisham Abdel-Azim, MD

Blood (2013) 122 (21): 2272.

https://doi.org/10.1182/blood.V122.21.2272.2272

Published: 2013

Journal Articles

Multicenter study of banked third-party virus-specific T cells to treat severe viral infections after hematopoietic stem cell transplantation

Ann M. Leen, Catherine M. Bollard, Adam M. Mendizabal, Elizabeth J. Shpall, Paul Szabolcs, Joseph H. Antin, Neena Kapoor, Sung-Yun Pai, Scott D. Rowley, Partow Kebriaei, Bimalangshu R. Dey, Bambi J. Grilley, Adrian P. Gee, Malcolm K. Brenner, Cliona M. Rooney, Helen E. Heslop

Blood (2013) 121 (26): 5113–5123.

https://doi.org/10.1182/blood-2013-02-486324

Published: 2013

Includes: Supplemental data

Journal Articles

Impact of Thymic Function On Chronic Graft-Versus-Host Disease (cGVHD).

Kris M. Mahadeo, M.D., M.P.H., Ami J Shah, MD, Bernadette Masinsin, BS, Neena Kapoor, MD, Hisham Abdel-Azim, MD, Robertson Parkman, M.D.

Blood (2012) 120 (21): 3052.

https://doi.org/10.1182/blood.V120.21.3052.3052

Published: 2012

Journal Articles

Multicenter Study of “off-the-Shelf” Third Party Virus-Specific T Cells (VSTs) to Treat Adenovirus (Adv), Cytomegalovirus (CMV) or Epstein Barr Virus (EBV) Infection After Hemopoietic Stem Cell Transplantation (HSCT)

Ann M. Leen, PhD, Catherine M. Bollard, MD, Adam M. Mendizabal, MS, Elizabeth J. Shpall, MD, Paul Szabolcs, MD, Joseph H. Antin, MD, Neena Kapoor, MD, Sung-Yun Pai, MD, Scott D. Rowley, MD, Partow Kebriaei, M.D., Bambi J. Grilley, Adrian P. Gee, PhD, Malcolm K. Brenner, MD, PhD, Cliona M. Rooney, PhD, Helen E. Heslop, MD

Blood (2012) 120 (21): 457.

https://doi.org/10.1182/blood.V120.21.457.457

Published: 2012

NARROW

Clinical and functional spectrum of RAC2-related immunodeficiency

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

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

Treatment of Individuals with Severe Sickle Cell Disease with OTQ923, an Autologous, Ex Vivo , CRISPR/Cas9-Edited, CD34+ Hematopoietic Stem and Progenitor Cell Product, Leads to Durable Engraftment and Fetal Hemoglobin Induction

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

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

Administration of BPX-501 Cells Following Aβ T and B-Cell-Depleted HLA Haploidentical HSCT (haplo-HSCT) in Children with Acute Leukemias

Administration of Rimiducid Following Haploidentical BPX-501 Cells in Children with Malignant or Non-Malignant Disorders Who Develop Graft-Versus-Host-Disease (GvHD)

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

Allogeneic Hematopoietic Stem Cell Transplantation in Older Patients Aged 50 Years or Older with Severe Aplastic Anaemia: Results from the European Society for Blood and Marrow Transplant and the Center for International Blood and Marrow Transplant Research

Improved outcomes associated with hematopoietic stem cell transplantation for patients with juvenile myelomonocytic leukemia

Hematopoietic stem cell transplantation for infantile osteopetrosis

Long Term Humoral Immune Reconstitution Kinetics After Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) In Children

Improved Outcomes After Reduced Intensity Conditioning Matched Unrelated Donor Hematopoietic Stem Cell Transplantation In Children With Chronic Granulomatous Disease

Multicenter study of banked third-party virus-specific T cells to treat severe viral infections after hematopoietic stem cell transplantation

Impact of Thymic Function On Chronic Graft-Versus-Host Disease (cGVHD).

Multicenter Study of “off-the-Shelf” Third Party Virus-Specific T Cells (VSTs) to Treat Adenovirus (Adv), Cytomegalovirus (CMV) or Epstein Barr Virus (EBV) Infection After Hemopoietic Stem Cell Transplantation (HSCT)

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American Society of Hematology

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