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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

Outcome of Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome

Blood (2019) 134 (Supplement_1): 4629.

https://doi.org/10.1182/blood-2019-126161

Published: 2019

Journal Articles

Prevention and Management of Daratumumab Mediated Infusion Related Hypersensitivity Reactions Pre-and Post-Implementation of Rapid Infusion Protocol

My L Nguyen, PharmD, Catherine Burdalski, PharmD, BCOP, Syed Abbas Ali, MD, Lindsay Kohler, BS, PharmD Candidate, Steven Gilmore, PharmD, BCOP

Blood (2019) 134 (Supplement_1): 5811.

https://doi.org/10.1182/blood-2019-125391

Published: 2019

Journal Articles

Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy for Wiskott-Aldrich Syndrome (WAS): Up to 8 Years of Follow up in 17 Subjects Treated Since 2010

Blood (2019) 134 (Supplement_1): 3346.

https://doi.org/10.1182/blood-2019-124665

Published: 2019

Journal Articles

Plasma Thymus and Activation-Regulated Chemokine (TARC) As Diagnostic Marker in Pediatric Hodgkin Lymphoma

Eline Zijtregtop, MD PhDcandidate, Waichu Wong, BSc, Friederike Meyer- Wentrup, MD PhD, Martha Lopez-Yurda, PhD, Raoull Hoogendijk, MSc, Patty Kok, BSc, Michel Zwaan, Prof., MD PhD, Auke Beishuizen, MD PhD

Blood (2018) 132 (Supplement 1): 5312.

https://doi.org/10.1182/blood-2018-99-111861

Published: 2018

Journal Articles

All Itching Is Not Benign; A Case of Refractory Hypereosinophilic Syndrome Treated with Off Label CD52 Inhibitor-Alemtuzumab

Rehan Ansari, MD, Elva Nora Osuna Salazar, MD, Nabeel Sarhill, MD, Laura E Garcia, MD, James Hanley, MD

Blood (2018) 132 (Supplement 1): 5485.

https://doi.org/10.1182/blood-2018-99-118271

Published: 2018

Journal Articles

Safety and Activity of the Highly Specific BTK Inhibitor BGB-3111 in Combination with the PD-1 Inhibitor BGB-A317 in Patients with B-Cell Lymphoid Malignancies

Gavin Cull, MB, BS, FRACP, FRCPA, Stephen Opat, FRACP, FRCPA, Judith Trotman, MBChB, FRACP, FRCPA, James Hilger, Xiaoping Zhang, Shibao Feng, PhD, Sunhee Ro, PhD, Jane Huang, MD, Constantine S. Tam, MBBS, MD FRACP, FRCPA

Blood (2017) 130 (Supplement 1): 4057.

https://doi.org/10.1182/blood.V130.Suppl_1.4057.4057

Published: 2017

Journal Articles

First in Human, Phase I/II Trial of the Bruton's Tyrosine Kinase Inhibitor (BTKi) M7583 in Patients with B Cell Malignancies: Study Design and Initial Outcomes

Wojciech Jurczak, Simon Rule, MD, William Townsend, David Tucker, Martin Dyroff, Barbara Sarholz, Jürgen Scheele, John G. Gribben, MDDSc FMedSci, Pier Luigi Zinzani, MD

Blood (2017) 130 (Supplement 1): 2778.

https://doi.org/10.1182/blood.V130.Suppl_1.2778.2778

Published: 2017

Journal Articles

Rare Case of Adult Onset DOCK8 Mutated Hyper Ige Syndrome

Damien Hansra, MD, Maureen Cioffi-Lavina, MD, Hugo Granada, MD, Brendan McIntyre, Cory Patrick, Sean Keniff

Blood (2017) 130 (Supplement 1): 4825.

https://doi.org/10.1182/blood.V130.Suppl_1.4825.4825

Published: 2017

Journal Articles

The WASP P460S Mutation Causes a New Phenotype of WASP Mutations Related Disorder: X-Linked Pancytopenia

Yunjing Zheng, MD, Shuiyan Wu, Xiaoliang Yu, Meng Wu, Qin Lu, Sudan He, Shaoyan Hu, MD PhD

Blood (2017) 130 (Supplement 1): 1044.

https://doi.org/10.1182/blood.V130.Suppl_1.1044.1044

Published: 2017

Journal Articles

Expanded Phenotypic and Genetic Heterogeneity in the Clinical Spectrum of FPD-AML: Lymphoid Malignancies and Skin Disorders Are Common Features in Carriers of Germline RUNX1 Mutations

Anna L Brown, PhD, Christopher N Hahn, PhD, Catherine Carmichael, Ella Wilkins, Milena Babic, BMPharBio (Hons), Chan-Eng Chong, PhD, Xiao-Chun Li, Joelle Michaud, Ping Cannon, Nicola Poplawski, MBChB, DipPaed, FRACP, MD, Meryl Altree, Kerry Phillips, Louise Jaensch, Miriam Fine, Andreas W Schreiber, PhD, Jinghua Feng, PhD, Lesley Rawlings, PhD, Cassandra Vakulin, Carolyn Butcher, Richard D'Andrea, PhD, Ian D Lewis, MBBS, PhD FRACP, FRCPA, Nigel Patton, Cecily Forsyth, MBBS, FRACP, FRCPA, Sally Mapp, PhD MD, Helen Mar Fan, Rachel Susman, Sue Morgan, FRACP, FRCPA, MBBS, Julian Cooney, MD, Mark S Currie, MD, Uday R. Popat, MD, Kenneth Bradstock, MBBS, PhD, April D. Sorrell, MD, Carolyn J. Owen, MD MDes(UK), FRCPC, Marshall S Horwitz, MD PhD, Devendra Hiwase, MBBS, FRACP, FRCPA, MD PhD, Alwin Krämer, MD, Stefan Fröhling, MD, Lucy A Godley, MD PhD, Jane E Churpek, MD, Hamish S Scott, BSc, PhD FFSc (RCPA), FAHMS

Blood (2016) 128 (22): 1212.

https://doi.org/10.1182/blood.V128.22.1212.1212

Published: 2016

Journal Articles

Generation of Functional Regulatory T Cells By FOXP3 Gene Transfer into CD4 T Cells from Scurfy Mice and IPEX Patients

Marianne Delville, MD, Emmanuelle Six, PhD, Florence Bellier, Nelly Sigrist, David Zemmour, PhD, Marie-André Bessard, MD, Fabienne Mazerolles, PhD, David Gross, PhD, Axel Schambach, MD PhD, Frederic Rieux-Laucat, PhD, Christoph Benoist, MD PhD, Isabelle Andre-Schmutz, Marina Cavazzana, MD PhD

Blood (2016) 128 (22): 2526.

https://doi.org/10.1182/blood.V128.22.2526.2526

Published: 2016

Journal Articles

N-WASP is required for B-cell–mediated autoimmunity in Wiskott-Aldrich syndrome

Stefano Volpi, Elettra Santori, Katrina Abernethy, Masayuki Mizui, Carin I. M. Dahlberg, Mike Recher, Kelly Capuder, Eva Csizmadia, Douglas Ryan, Divij Mathew, George C. Tsokos, Scott Snapper, Lisa S. Westerberg, Adrian J. Thrasher, Fabio Candotti, Luigi D. Notarangelo

Blood (2016) 127 (2): 216–220.

https://doi.org/10.1182/blood-2015-05-643817

Published: 2016

Includes: Supplemental data

Journal Articles

Safety and Clinical Benefit of Lentiviral Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome

Blood (2015) 126 (23): 259.

https://doi.org/10.1182/blood.V126.23.259.259

Published: 2015

Journal Articles

Gene Therapy Using a Self-Inactivating Lentiviral Vector Improves Clinical and Laboratory Manifestations of Wiskott-Aldrich Syndrome

Blood (2015) 126 (23): 260.

https://doi.org/10.1182/blood.V126.23.260.260

Published: 2015

Journal Articles

Efficacy and Safety of Deferasirox in Chinese Patients with Non-Transfusion-Dependent Thalassemia: 1-Year Results from the Thetis Study

Yong-Rong Lai, Rong Rong Liu, M Domenica Cappellini, Yesim Aydinok, John B Porter, Zewen Zhu, Candace Wang, Xue Qi, Ali T Taher

Blood (2015) 126 (23): 4570.

https://doi.org/10.1182/blood.V126.23.4570.4570

Published: 2015

Journal Articles

Autoimmune Lymphoproliferative Syndrome (ALPS) and ALPS-Related Disorders. Different Bio-Clinical Profile and Similar Response to Treatment: A Single Centre Experience

Maurizio Miano, MD, Andrea Todiere, MD, Ilaria Caviglia, Elena Palmisani, MD, Johanna Svahn, MD, Concetta Micalizzi, MD, Paola Terranova, Tiziana Lanza, Carlo Dufour, MD, Francesca Fioredda

Blood (2015) 126 (23): 4618.

https://doi.org/10.1182/blood.V126.23.4618.4618

Published: 2015

Journal Articles

B Cell Reconstitution after Gene Therapy in Patients with Wiskott Aldrich Syndrome and Comparison with Mismatched Allogeneic Hematopoietic Stem Cell Transplantation

Alessandra Magnani, MD PhD, Cécile Roudaut, MSc, Aurelie Gabrion, Laure Caccavelli, PhD, Fabien Touzot, MDPhD, Benedicte Neven, MD PhD, Despina Moshous, MD, Nizar Mahlaoui, Emmanuel Clave, Antoine Toubert, Alain Fischer, MD PhD, Marina Cavazzana, MD PhD

Blood (2015) 126 (23): 3235.

https://doi.org/10.1182/blood.V126.23.3235.3235

Published: 2015

Journal Articles

The North American Shwachman-Diamond Syndrome Registry: Genetically Undefined Shwachman-Diamond Syndrome

Kasiani C. Myers, MD, Audrey Anna Bolyard, RN, BS, Jamie Leung, Joan Moore, Sara Loveless, Leann Mount, Richard E. Harris, MD, Stella M. Davies, MBBS, PhD, Sioban Keel, MD, David C. Dale, MD, Akiko Shimamura, MD PhD

Blood (2015) 126 (23): 3614.

https://doi.org/10.1182/blood.V126.23.3614.3614

Published: 2015

Journal Articles

Haploidentical Transplantation for DOCK8 Deficiency

Nirali N Shah, MD, Alexandra F Freeman, MD, Mark Parta, MD, Helen Su, MD PhD, Gulbu Uzel, MD, Juan C. Gea-Banacloche, MD, Steven M. Holland, MD, Dennis D. Hickstein, MD

Blood (2015) 126 (23): 2229.

https://doi.org/10.1182/blood.V126.23.2229.2229

Published: 2015

NARROW

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

Outcome of Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome

Prevention and Management of Daratumumab Mediated Infusion Related Hypersensitivity Reactions Pre-and Post-Implementation of Rapid Infusion Protocol

Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy for Wiskott-Aldrich Syndrome (WAS): Up to 8 Years of Follow up in 17 Subjects Treated Since 2010

Plasma Thymus and Activation-Regulated Chemokine (TARC) As Diagnostic Marker in Pediatric Hodgkin Lymphoma

All Itching Is Not Benign; A Case of Refractory Hypereosinophilic Syndrome Treated with Off Label CD52 Inhibitor-Alemtuzumab

Safety and Activity of the Highly Specific BTK Inhibitor BGB-3111 in Combination with the PD-1 Inhibitor BGB-A317 in Patients with B-Cell Lymphoid Malignancies

First in Human, Phase I/II Trial of the Bruton's Tyrosine Kinase Inhibitor (BTKi) M7583 in Patients with B Cell Malignancies: Study Design and Initial Outcomes

Rare Case of Adult Onset DOCK8 Mutated Hyper Ige Syndrome

The WASP P460S Mutation Causes a New Phenotype of WASP Mutations Related Disorder: X-Linked Pancytopenia

Expanded Phenotypic and Genetic Heterogeneity in the Clinical Spectrum of FPD-AML: Lymphoid Malignancies and Skin Disorders Are Common Features in Carriers of Germline RUNX1 Mutations

Generation of Functional Regulatory T Cells By FOXP3 Gene Transfer into CD4 T Cells from Scurfy Mice and IPEX Patients

N-WASP is required for B-cell–mediated autoimmunity in Wiskott-Aldrich syndrome

Safety and Clinical Benefit of Lentiviral Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome

Gene Therapy Using a Self-Inactivating Lentiviral Vector Improves Clinical and Laboratory Manifestations of Wiskott-Aldrich Syndrome

Efficacy and Safety of Deferasirox in Chinese Patients with Non-Transfusion-Dependent Thalassemia: 1-Year Results from the Thetis Study

Autoimmune Lymphoproliferative Syndrome (ALPS) and ALPS-Related Disorders. Different Bio-Clinical Profile and Similar Response to Treatment: A Single Centre Experience

B Cell Reconstitution after Gene Therapy in Patients with Wiskott Aldrich Syndrome and Comparison with Mismatched Allogeneic Hematopoietic Stem Cell Transplantation

The North American Shwachman-Diamond Syndrome Registry: Genetically Undefined Shwachman-Diamond Syndrome

Haploidentical Transplantation for DOCK8 Deficiency

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American Society of Hematology

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