LRRC8 complexes are ATP release channels that regulate platelet activation and arterial thrombosis

J John D. Tranter (1Cardiovascular Division, Department of Internal Medicine, Washington University School of Medicine, St. Louis, MO) R Ryan T. Mikami (1Cardiovascular Division, Department of Internal Medicine, Washington University School of Medicine, St. Louis, MO) A Ashutosh Kumar G Gavriel Brown (2Division of Hematology, Department of Medicine, Washington University School of Medicine, St. Louis, MO) T Tarek Mohamed Abd El-Aziz (1Cardiovascular Division, Department of Internal Medicine, Washington University School of Medicine, St. Louis, MO) Y Yonghui Zhao (Center for Low-Carbon Conversion Science and Engineering; State Key Laboratory of Low Carbon Catalysis and Carbon Dioxide Utilization, Shanghai Advanced Research Institute) P Prakash Arullampalam K Katrina Ashworth (1Washington University School of Medicine, Saint Louis, United States) V Vishwanath Jha (2Division of Hematology, Department of Medicine, Washington University School of Medicine, St. Louis, MO) N Nihil Abraham (1Cardiovascular Division, Department of Internal Medicine, Washington University School of Medicine, St. Louis, MO) C Chloe Meyer (1Cardiovascular Division, Department of Internal Medicine, Washington University School of Medicine, St. Louis, MO) A Abigail Ajanel (3Department of Emergency Medicine, Washington University School of Medicine, St. Louis, MO) L Litao Xie Y Yongmei Feng J Juan Hong H Haixia Zhang T Tripti Kumari (2Division of Hematology, Department of Medicine, Washington University School of Medicine, St. Louis, MO) A Adam Balutowski (1Cardiovascular Division, Department of Internal Medicine, Washington University School of Medicine, St. Louis, MO) A Alice Liu (Division of Hematology, Department of Pediatrics, Washington University) D David Bark (Division of Hematology, Department of Pediatrics, Washington University) V Vinayak K. Nair (1Cardiovascular Division, Department of Internal Medicine, Washington University School of Medicine, St. Louis, MO) N Nina M. Lasky (4Division of Pediatric Hematology and Oncology, Washington University in St. Louis, St. Louis, MO) N Nathan O. Stitziel (From the Cardiovascular Division, Department of Medicine, and the Department of Genetics, Washington University School of Medicine, St. Louis.) D Daniel J. Lerner (8Senseion Therapeutics Inc, St. Louis, MO) R Robert A. Campbell (Department of Emergency Medicine, School of Medicine, Washington University) J Jorge Di Paola (Division of Hematology, Department of Pediatrics, Washington University) J Jaehyung Cho R Rajan Sah (1Cardiovascular Division, Department of Internal Medicine, Washington University School of Medicine, St. Louis, MO)

Abstract

Abstract Platelet shape and volume changes are early mechanical events contributing to platelet activation and thrombosis. Here, we identify single-nucleotide polymorphisms in leucine-rich repeat–containing 8 (LRRC8) protein subunits that form the volume-regulated anion channel (VRAC), which are independently associated with altered mean platelet volume. LRRC8A is required for functional VRAC in megakaryocytes (MKs) and regulates platelet volume; adhesion; and agonist-stimulated activation, aggregation, adenosine triphosphate (ATP) secretion, and calcium mobilization. MK-specific LRRC8A conditional knockout mice have reduced laser injury–induced cremaster arteriolar thrombus formation and prolonged FeCl3 induced carotid arterial thrombosis without prolonged bleeding times. Mechanistically, platelet LRRC8A mediates swell-induced cytosolic ATP release to amplify agonist-stimulated calcium–phosphoinositide 3-kinase–protein kinase B signaling. Small-molecule LRRC8 channel inhibitors recapitulate defects observed in LRRC8A-null platelets in vitro and in vivo. These studies identify the mechanoresponsive LRRC8 channel complex as an ATP release channel in platelets, which positively regulates platelet function and thrombosis, providing a proof of concept for a novel antithrombotic drug target.

Article Details

Journal Blood
Volume / Issue Vol. 146, Issue 9
Published August 28, 2025
Pages 1110-1126
ISSN 0006-4971
Publisher Elsevier BV

Journal Info

Blood

Elsevier BV

ISSN: 0006-4971 Health Sciences

Authors (28)

J

John D. Tranter

1Cardiovascular Division, Department of Internal Medicine, Washington University School of Medicine, St. Louis, MO

R

Ryan T. Mikami

1Cardiovascular Division, Department of Internal Medicine, Washington University School of Medicine, St. Louis, MO

A

Ashutosh Kumar

G

Gavriel Brown

2Division of Hematology, Department of Medicine, Washington University School of Medicine, St. Louis, MO

T

Tarek Mohamed Abd El-Aziz

1Cardiovascular Division, Department of Internal Medicine, Washington University School of Medicine, St. Louis, MO

Y

Yonghui Zhao

Center for Low-Carbon Conversion Science and Engineering; State Key Laboratory of Low Carbon Catalysis and Carbon Dioxide Utilization, Shanghai Advanced Research Institute

P

Prakash Arullampalam

K

Katrina Ashworth

1Washington University School of Medicine, Saint Louis, United States

V

Vishwanath Jha

2Division of Hematology, Department of Medicine, Washington University School of Medicine, St. Louis, MO

N

Nihil Abraham

1Cardiovascular Division, Department of Internal Medicine, Washington University School of Medicine, St. Louis, MO

C

Chloe Meyer

1Cardiovascular Division, Department of Internal Medicine, Washington University School of Medicine, St. Louis, MO

A

Abigail Ajanel

3Department of Emergency Medicine, Washington University School of Medicine, St. Louis, MO

L

Litao Xie

Y

Yongmei Feng

J

Juan Hong

H

Haixia Zhang

T

Tripti Kumari

2Division of Hematology, Department of Medicine, Washington University School of Medicine, St. Louis, MO

A

Adam Balutowski

1Cardiovascular Division, Department of Internal Medicine, Washington University School of Medicine, St. Louis, MO

A

Alice Liu

Division of Hematology, Department of Pediatrics, Washington University

D

David Bark

Division of Hematology, Department of Pediatrics, Washington University

V

Vinayak K. Nair

1Cardiovascular Division, Department of Internal Medicine, Washington University School of Medicine, St. Louis, MO

N

Nina M. Lasky

4Division of Pediatric Hematology and Oncology, Washington University in St. Louis, St. Louis, MO

N

Nathan O. Stitziel

From the Cardiovascular Division, Department of Medicine, and the Department of Genetics, Washington University School of Medicine, St. Louis.

D

Daniel J. Lerner

8Senseion Therapeutics Inc, St. Louis, MO

R

Robert A. Campbell

Department of Emergency Medicine, School of Medicine, Washington University

J

Jorge Di Paola

Division of Hematology, Department of Pediatrics, Washington University

J

Jaehyung Cho

R

Rajan Sah

1Cardiovascular Division, Department of Internal Medicine, Washington University School of Medicine, St. Louis, MO