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Journal Articles

T-Cell Transcriptomic Signatures in Adults with Primary Untreated Immune Thrombocytopenia Segregate By Age, Revealing Distinct Druggable Pathways

Lipei Shao, Aaron Boothby, S. Lawrence Bailey, Aastha Chauhan, Martin Boechenek, Terry B. Gernsheimer, John W. Semple, James L. Zehnder, V. Koneti Rao, Jin Ping, David F. Stroncek, Moritz Stolla, Sandya R. Panch

Blood (2024) 144 (Supplement 1): 124.

https://doi.org/10.1182/blood-2024-203670

Published: 2024

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

Blood (2023) 142 (Supplement 1): 936.

https://doi.org/10.1182/blood-2023-185110

Published: 2023

Journal Articles

Primary Immune Regulatory Disorders (PIRDs) That Amplify mTOR Signaling Share a T Cell Exhaustion-like Process

Peyton E Conrey, Jose Campos, Andrea Mauracher, Samir Sayed, Jolan E. Walter, Helen Su, Sara Barmettler, Jennifer W Leiding, Megan Cooper, Suzanne P MacFarland, Melanie A Ruffner, Jocelyn R Farmer, V. Koneti Rao, Sarah E Henrickson

Blood (2023) 142 (Supplement 1): 2555.

https://doi.org/10.1182/blood-2023-188195

Published: 2023

Journal Articles

A randomized, placebo-controlled phase 3 trial of the PI3Kδ inhibitor leniolisib for activated PI3Kδ syndrome

Blood (2023) 141 (9): 971–983.

https://doi.org/10.1182/blood.2022018546

Published: 2023

Includes: Supplemental data

Journal Articles

Interim Analysis of Safety and Hematological Parameters of an Ongoing Long-Term Open-Label Extension Study of Investigational PI3Kδ Inhibitor Leniolisib for Patients with Activated PI3K Delta Syndrome (APDS) through December 2021

V. Koneti Rao, Sharon Webster, Anna Sediva, Alessandro Plebani, Catharina Schuetz, Anna Shcherbina, Virgil A.S.H. Dalm, Antonino Trizzino, Yulia Zharankova, Elaine Kulm, Alanvin Orpia, Lavenda Chirombo Kluczynski, Julia Körholz, Vassilios Lougaris, Yulia Rodina, Klaus Kucher, Kath Radford, Jason Bradt, Gulbu Uzel

Blood (2022) 140 (Supplement 1): 1643–1645.

https://doi.org/10.1182/blood-2022-160334

Published: 2022

Journal Articles

Evans syndrome: pathology and genomic hubris

V. Koneti Rao

Blood (2022) 139 (3): 312–313.

https://doi.org/10.1182/blood.2021013636

Published: 2022

Journal Articles

Immunodeficiency and bone marrow failure with mosaic and germline TLR8 gain of function

Blood (2021) 137 (18): 2450–2462.

https://doi.org/10.1182/blood.2020009620

Published: 2021

Includes: Supplemental data

Journal Articles

Autoimmune Cytopenias in Nonmalignant Lymphoproliferative Disorders: Thinking Beyond ALPS(Autoimmune Lymphoproliferative Syndrome)

Elaine Kulm, CRNP-AC, Sharon Webster, BSN, Kate Howe, PA-C, Amy Rump, FNP-BC, Gulbu Uzel, MD, V. Koneti Rao, MD FRCPA

Blood (2020) 136 (Supplement 1): 48.

https://doi.org/10.1182/blood-2020-137640

Published: 2020

Journal Articles

Safety and Efficacy of Long Term Suppression of PI3Kinase Pathway By Small Molecule PI3K-Delta Inhibitor, Leniolisib in Apds (Activated PI3Kδ Syndrome)

V. Koneti Rao, MD FRCPA, Sharon Webster, BSN, Virgil A.S.H. Dalm, MD, Anna Sediva, MD, P. Martin van Hagen, H. Michael Do, MD, Les Folio, MD, Maciej Cabanski, Marie-Anne Valentin, Stefan de Buck, Christoph Kalis, Anke Hasselberg, Christoph Burkhart, Klaus Kucher, MD, Birgitte Sloth, Gulbu Uzel, MD

Blood (2018) 132 (Supplement 1): 3706.

https://doi.org/10.1182/blood-2018-99-113426

Published: 2018

Journal Articles

Clinical Spectrum of Patients with Pathogenic Variant of STAT3 conferring Gain-of-Function: A Mimic of Autoimmune Lymphoproliferative Syndrome

Gregory Constantine, MD, Helen Su, MD, PhD, Les Folio, MD, Joshua Milner, MD, V. Koneti Rao, MD FRCPA

Blood (2018) 132 (Supplement 1): 3723.

https://doi.org/10.1182/blood-2018-99-111950

Published: 2018

Journal Articles

Spare the Spleen in ALPS: It Is Not an Expendable Vestigial Organ

V. Koneti Rao, MD FRCPA, Elaine Kulm, Susan Price, Sharon Webster, BSN, Les Folio, MD, Gulbu Uzel, MD, David T Teachey, MD

Blood (2018) 132 (Supplement 1): 2435.

https://doi.org/10.1182/blood-2018-99-117394

Published: 2018

Journal Articles

Management of Cytopenias in CTLA4 Haploinsufficiency Using Abatacept and Sirolimus

Gulbu Uzel, MD, Djuro Karanovic, MD, Hua Su, PhD, Amy Rump, Anahita Agharahimi, Steven M. Holland, MD, V. Koneti Rao, MD FRCPA

Blood (2018) 132 (Supplement 1): 2409.

https://doi.org/10.1182/blood-2018-99-120185

Published: 2018

Journal Articles

Germline Mutations in CTLA4 and Spectrum of Bone Marrow Features Overlapping with Aplastic Anemia and Large Granular Lymphocyte Lymphocytosis

Laura M Wake, MD, Changqing Xie, MD, Weixin Wang, PhD, Kristian T. Schafernak, MD MPH, V. Koneti Rao, MD FRCPA, Neal S. Young, MD, Danielle M. Townsley, MDMSc, Gulbu Uzel, MD, Katherine R Calvo, MD PhD

Blood (2017) 130 (Supplement 1): 2448.

https://doi.org/10.1182/blood.V130.Suppl_1.2448.2448

Published: 2017

Journal Articles

Effective “activated PI3Kδ syndrome”–targeted therapy with the PI3Kδ inhibitor leniolisib

Blood (2017) 130 (21): 2307–2316.

https://doi.org/10.1182/blood-2017-08-801191

Published: 2017

Includes: Supplemental data

Journal Articles

Indolent Phenotype of RAS-Associated Autoimmune Leukoproliferative Disorder (RALD) Is Characterized By Single Somatic Mutations in RAS Genes with Absence of Cooperating Mutations

Vollter Anastas, B.S., Weixin Wang, PhD, Susan Price, Zhen Zhao, PhD, V. Koneti Rao, MD, Katherine R. Calvo, MD PhD

Blood (2016) 128 (22): 4268.

https://doi.org/10.1182/blood.V128.22.4268.4268

Published: 2016

Journal Articles

Clinical Spectrum of Autoimmune Lymphoproliferative Syndrome Associated with Caspase10 Mutations (ALPS-CASP10)

V. Koneti Rao, MD, Susan Price, Morgan Similuk, Julie Niemela, Joshua D Milner, MD, Sergio Rosenzweig, MD

Blood (2016) 128 (22): 1335.

https://doi.org/10.1182/blood.V128.22.1335.1335

Published: 2016

Journal Articles

Utility of Fluorodeoxyglucose Positron Emission Tomography(FDG-PET) and Biomarkers, Serum Vitamin B12 and HDL for Assessing Lymphoproliferation in Children and Adults with Autoimmune Lymphoproliferative Syndrome Due to FAS Mutations (ALPS-FAS)

V. Koneti Rao, MD, Susan Price, Lori Dodd, Remaley Alan, Millie Whatley, Jorge Carrasquillo, MD, Clara C. Chen, MD

Blood (2016) 128 (22): 4891.

https://doi.org/10.1182/blood.V128.22.4891.4891

Published: 2016

Journal Articles

Serendipity in splendid isolation: rapamycin

V. Koneti Rao

Blood (2016) 127 (1): 5–6.

https://doi.org/10.1182/blood-2015-11-679829

Published: 2016

Journal Articles

JMML and RALD (Ras-associated autoimmune leukoproliferative disorder): common genetic etiology yet clinically distinct entities

Katherine R. Calvo, Susan Price, Raul C. Braylan, Joao Bosco Oliveira, Michael Lenardo, Thomas A. Fleisher, V. Koneti Rao

Blood (2015) 125 (18): 2753–2758.

https://doi.org/10.1182/blood-2014-11-567917

Published: 2015

Includes: Supplemental data

Journal Articles

Early-onset lymphoproliferation and autoimmunity caused by germline STAT3 gain-of-function mutations

Blood (2015) 125 (4): 591–599.

https://doi.org/10.1182/blood-2014-09-602763

Published: 2015

Includes: Supplemental data

NARROW

T-Cell Transcriptomic Signatures in Adults with Primary Untreated Immune Thrombocytopenia Segregate By Age, Revealing Distinct Druggable Pathways

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

Primary Immune Regulatory Disorders (PIRDs) That Amplify mTOR Signaling Share a T Cell Exhaustion-like Process

A randomized, placebo-controlled phase 3 trial of the PI3Kδ inhibitor leniolisib for activated PI3Kδ syndrome

Interim Analysis of Safety and Hematological Parameters of an Ongoing Long-Term Open-Label Extension Study of Investigational PI3Kδ Inhibitor Leniolisib for Patients with Activated PI3K Delta Syndrome (APDS) through December 2021

Evans syndrome: pathology and genomic hubris

Immunodeficiency and bone marrow failure with mosaic and germline TLR8 gain of function

Autoimmune Cytopenias in Nonmalignant Lymphoproliferative Disorders: Thinking Beyond ALPS(Autoimmune Lymphoproliferative Syndrome)

Safety and Efficacy of Long Term Suppression of PI3Kinase Pathway By Small Molecule PI3K-Delta Inhibitor, Leniolisib in Apds (Activated PI3Kδ Syndrome)

Clinical Spectrum of Patients with Pathogenic Variant of STAT3 conferring Gain-of-Function: A Mimic of Autoimmune Lymphoproliferative Syndrome

Spare the Spleen in ALPS: It Is Not an Expendable Vestigial Organ

Management of Cytopenias in CTLA4 Haploinsufficiency Using Abatacept and Sirolimus

Germline Mutations in CTLA4 and Spectrum of Bone Marrow Features Overlapping with Aplastic Anemia and Large Granular Lymphocyte Lymphocytosis

Effective “activated PI3Kδ syndrome”–targeted therapy with the PI3Kδ inhibitor leniolisib

Indolent Phenotype of RAS-Associated Autoimmune Leukoproliferative Disorder (RALD) Is Characterized By Single Somatic Mutations in RAS Genes with Absence of Cooperating Mutations

Clinical Spectrum of Autoimmune Lymphoproliferative Syndrome Associated with Caspase10 Mutations (ALPS-CASP10)

Utility of Fluorodeoxyglucose Positron Emission Tomography(FDG-PET) and Biomarkers, Serum Vitamin B12 and HDL for Assessing Lymphoproliferation in Children and Adults with Autoimmune Lymphoproliferative Syndrome Due to FAS Mutations (ALPS-FAS)

Serendipity in splendid isolation: rapamycin

JMML and RALD (Ras-associated autoimmune leukoproliferative disorder): common genetic etiology yet clinically distinct entities

Early-onset lymphoproliferation and autoimmunity caused by germline STAT3 gain-of-function mutations

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American Society of Hematology

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ASH Publications

American Society of Hematology

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