Identification of a homozygous recessive variant in PTGS1 resulting in a congenital aspirin-like defect in platelet function
Author(s)
Type
Journal Article
Abstract
We have identified a rare missense variant on chromosome 9, position 125145990 (GRCh37), in exon 8 in PTGS1 (the gene encoding cyclo-oxygenase 1, COX-1, the target of anti-thrombotic aspirin therapy). We report that in the homozygous state within a large consanguineous family this variant is associated with a bleeding phenotype and alterations in platelet reactivity and eicosanoid production. Western blotting and confocal imaging demonstrated that COX-1 was absent in the platelets of three family members homozygous for the PTGS1 variant but present in their leukocytes. Platelet reactivity, as assessed by aggregometry, lumi-aggregometry and flow cytometry, was impaired in homozygous family members, as were platelet adhesion and spreading. The productions of COX-derived eicosanoids by stimulated platelets were greatly reduced but there were no changes in the levels of urinary metabolites of COX-derived eicosanoids. The proband exhibited additional defects in platelet aggregation and spreading which may explain why her bleeding phenotype was slightly more severe than those of other homozygous affected relatives. This is the first demonstration in humans of the specific loss of platelet COX-1 activity and provides insight into its consequences for platelet function and eicosanoid metabolism. Notably despite the absence of thromboxane A2 (TXA2) formation by platelets, urinary TXA2 metabolites were in the normal range indicating these cannot be assumed as markers of in vivo platelet function. Results from this study are important benchmarks for the effects of aspirin upon platelet COX-1, platelet function and eicosanoid production as they define selective platelet COX-1 ablation within humans.
Date Issued
2021-05-01
Date Acceptance
2020-04-09
Citation
Haematologica: the hematology journal, 2021, 106 (5), pp.1423-1432
ISSN
0390-6078
Publisher
Ferrata Storti Foundation
Start Page
1423
End Page
1432
Journal / Book Title
Haematologica: the hematology journal
Volume
106
Issue
5
Copyright Statement
© 2020, Ferrata Storti Foundation.
Sponsor
Medical Research Council (MRC)
Identifier
https://www.ncbi.nlm.nih.gov/pubmed/32299908
PII: haematol.2019.235895
Grant Number
MR/J011711/1
Subjects
Disorders of Platelet Function
Platelets
cyclo-oxygenase
genetics
platelet dysfunction
Publication Status
Published
Coverage Spatial
Italy
Date Publish Online
2020-04-16