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Journal Articles

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

Clinical Trials & Observations
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
Journal: Blood
Blood (2023) 142 (24): 2105–2118.
https://doi.org/10.1182/blood.2022019586
Published: 2023
Includes: Supplemental data
Journal Articles

Clinical Characteristics, Management, and Allogeneic Hematopoietic Cell Transplantation of Patients with Toll-like Receptor 8 Gain-of-Function Mutations

Danielle E Arnold, Saara Kaviany, Jahnavi Aluri, Luis Murguia-Favela, Nicola A. Wright, Thomas P. Loughran, Jr., Jonathan Powell, Suk See De Ravin, V. Koneti Rao, Steven Holland, Jack Bleesing, Jeffrey J. Bednarski, James A. Connelly, Megan Cooper
Journal: Blood
Blood (2023) 142 (Supplement 1): 936.
https://doi.org/10.1182/blood-2023-185110
Published: 2023
Journal Articles

Long-Term Outcome of Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1) Using an Enhancer-Deleted Self-Inactivating Gammaretroviral Vector

Sung-Yun Pai, Nisha Nagarsheth, Susan Prockop, Myriam Armant, Donald B. Kohn, Rebecca A. Marsh, Claire Booth, John K. Everett, Jack Bleesing, Deepak Chellapandian, Colleen Dansereau, Satiro de Oliveira, Wendy B. London, M. Angélica Marinovic, Theodore B. Moore, Matias Oleastro, Grainne O'Toole, Mark Vander Lugt, Luciano Urdinez, Kelly J. Walkovich, Jolan E. Walter, Axel Schambach, Adrian J. Thrasher, Frederic D. Bushman, David A Williams
Journal: Blood
Blood (2022) 140 (Supplement 1): 10643–10645.
https://doi.org/10.1182/blood-2022-159768
Published: 2022
Journal Articles

Clinical, Immunological Features, Treatments, and Outcomes of Autoimmune Hemolytic Anemia in Patients with RAG Deficiency

Chen Wang, Bijun Sun, Kevin Wu, Jocelyn Farmer, Boglarka Ujhazi, Christoph B. Geier, Sumai Gordon, Emma Westermann-Clark, Sinisa Savic, Sargur Ravishankar, Karin Chen, Cullen M Dutmer, Maria G Kanariou, Mehdi Adeli, Paolo Palma, Carmem Bonfim, Evangelia Lycopoulou, Beata Wolska-Kusnierz, Dbaibo Ghassan, Jack Bleesing, Despina Moshous, Benedicte Neven, Catharina Schuetz, Geha Raif, Luigi D. Notarangelo, Maurizio Miano, David K. Buchbinder, Krisztian Csomos, Wenjie Wang, Ji-Yang Wang, Xiaochuan Wang, Jolan E. Walter
Journal: Blood
Blood (2022) 140 (Supplement 1): 2448–2450.
https://doi.org/10.1182/blood-2022-162845
Published: 2022
Journal Articles

T-follicular helper cell expansion and chronic T-cell activation are characteristic immune anomalies in Evans syndrome

Deepak Kumar, Chengyu Prince, Carolyn M. Bennett, Michael Briones, Laura Lucas, Athena Russell, Kiran Patel, Satheesh Chonat, Sara Graciaa, Holly Edington, Michael H. White, Lisa Kobrynski, Manar Abdalgani, Suhag Parikh, Sharat Chandra, Jack Bleesing, Rebecca Marsh, Sunita Park, Edmund K. Waller, Sampath Prahalad, Shanmuganathan Chandrakasan
Journal: Blood
Blood (2022) 139 (3): 369–383.
https://doi.org/10.1182/blood.2021012924
Published: 2022
Includes: Supplemental data
Journal Articles

Complement blockade for TA-TMA: lessons learned from a large pediatric cohort treated with eculizumab

Clinical Trials & Observations
Sonata Jodele, Christopher E. Dandoy, Adam Lane, Benjamin L. Laskin, Ashley Teusink-Cross, Kasiani C. Myers, Gregory Wallace, Adam Nelson, Jack Bleesing, Ranjit S. Chima, Russel Hirsch, Thomas D. Ryan, Stefanie Benoit, Kana Mizuno, Mikako Warren, Stella M. Davies
Journal: Blood
Blood (2020) 135 (13): 1049–1057.
https://doi.org/10.1182/blood.2019004218
Published: 2020
Includes: Supplemental data
Journal Articles

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

Suk See De Ravin, MD, PhD, Sandra Anaya O'Brien, Nana Kwatemaa, RN, MSN, CCRP, Narda Theobald, BS, Siyuan Liu, Janet Lee, BS, Lela Kardava, Taylor Liu, Frederick Goldman, MDMS, Susan Moir, Jack Bleesing, MD PhD, Benedicte Neven, MD PhD, Jennifer Puck, MD, Morton J Cowan, MD, Ewelina Mamcarz, MD, Stephen Gottschalk, MD, Michael M Meagher, PhD, Luigi Notarangelo, MD, Elizabeth Kang, MD, Xiaolin Wu, PhD, Harry L. Malech, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 608.
https://doi.org/10.1182/blood-2019-127439
Published: 2019
Journal Articles

Characterizing Autoimmune Hemolytic Anemia in RAG Deficiency

Kevin Wu, BSc, Jocelyn Farmer, Boglarka Ujhazi, BS, Emma Westermann-Clark, MD, Savic Sinisa, PhD, Sargur Ravishankar, Karin Chen, MD, Dutmer Cullen, MD, Maria G Kanariou, MD, Mehdi Adeli, Paolo Palma, MD PhD, Carmem M. S. Bonfim, MD PhD, Evangelia Lycopoulou, MD, Beata Wolska-Kusnierz, PhD, Dbaibo Ghassan, MD, Jack Bleesing, MD PhD, Despina Moshous, MD PhD, Benedicte Neven, MD PhD, Scheutz Catharina, Geha Raif, MD, Luigi Notarangelo, MD, Maurizio Miano, David K. Buchbinder, MD MSHS, Krisztian Csomos, PhD, Jolan Walter, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3508.
https://doi.org/10.1182/blood-2019-132274
Published: 2019
Journal Articles

Hematopoietic stem cell transplantation in 29 patients hemizygous for hypomorphic IKBKG /NEMO mutations

Charline Miot, Kohsuke Imai, Chihaya Imai, Anthony J. Mancini, Zeynep Yesim Kucuk, Tokomki Kawai, Ryuta Nishikomori, Etsuro Ito, Isabelle Pellier, Sophie Dupuis Girod, Jeremie Rosain, Shinya Sasaki, Shanmuganathan Chandrakasan, Jana Pachlopnik Schmid, Tsubasa Okano, Estelle Colin, Alberto Olaya-Vargas, Marco Yamazaki-Nakashimada, Waseem Qasim, Sara Espinosa Padilla, Andrea Jones, Alfons Krol, Nyree Cole, Stephen Jolles, Jack Bleesing, Thomas Vraetz, Andrew R. Gennery, Mario Abinun, Tayfun Güngör, Beatriz Costa-Carvalho, Antonio Condino-Neto, Paul Veys, Steven M. Holland, Gulbu Uzel, Despina Moshous, Benedicte Neven, Stéphane Blanche, Stephan Ehl, Rainer Döffinger, Smita Y. Patel, Anne Puel, Jacinta Bustamante, Erwin W. Gelfand, Jean-Laurent Casanova, Jordan S. Orange, Capucine Picard
Journal: Blood
Blood (2017) 130 (12): 1456–1467.
https://doi.org/10.1182/blood-2017-03-771600
Published: 2017
Includes: Supplemental data
Journal Articles

Diagnostic and risk criteria for HSCT-associated thrombotic microangiopathy: a study in children and young adults

Clinical Trials & Observations
Sonata Jodele, Stella M. Davies, Adam Lane, Jane Khoury, Christopher Dandoy, Jens Goebel, Kasiani Myers, Michael Grimley, Jack Bleesing, Javier El-Bietar, Gregory Wallace, Ranjit S. Chima, Zachary Paff, Benjamin L. Laskin
Journal: Blood
Blood (2014) 124 (4): 645–653.
https://doi.org/10.1182/blood-2014-03-564997
Published: 2014
Includes: Supplemental data
Journal Articles

Adolescents and Young Adults With Hemophagocytic Lymphohistiocytosis Who Undergo Allogeneic Hematopoietic Cell Transplantation Are At Increased Risk Of Mortality Compared To Younger Patients

Shanmuganathan Chandrakasan, MD, Rebecca Marsh, MD, Michael Grimley, MD, Jack Bleesing, MD, PhD, Michael Jordan, MD, Alexandra H. Filipovich, MD
Journal: Blood
Blood (2013) 122 (21): 2087.
https://doi.org/10.1182/blood.V122.21.2087.2087
Published: 2013
Journal Articles

Complete Responses in Patients with Autoimmune Lymphoproliferative Syndrome (ALPS) Using the mTOR Inhibitor Sirolimus (rapamycin)

Davi d T. Teachey, MD, Robert J Greiner, MD, Dirk Schwabe, MD, Jack Bleesing, MD, PhD, Catherine Scott Manno, MD, Kathleen Sullivan, MD, PhD, Daniel S. Wechsler, MD, PhD, Stephan A. Grupp, MD, PhD
Journal: Blood
Blood (2008) 112 (11): 2569.
https://doi.org/10.1182/blood.V112.11.2569.2569
Published: 2008