Age-related neutrophil activation in Hermansky-Pudlak Syndrome Type-1

Abstract Hermansky-Pudlak Syndrome (HPS) type 1 (HPS-1) is an autosomal recessive disorder characterized by oculocutaneous albinism, platelet dysfunction, and pulmonary fibrosis (HPS-PF), the leading cause of mortality in these patients. HPS-PF manifests earlier than idiopathic pulmonary fibrosis, t...

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Main Authors: Lourdes Marinna Caro-Rivera, Sonya Malavez-Cajigas, Mercedes Lacourt-Ventura, Andrea P. Rivera-Torres, Dorca E. Marcano-Jiménez, Pablo López-Colon, José Muñiz-Hernández, Enid Rivera-Jiménez, Mónica Egozcue-Dionisi, Rosa Román-Carlo, Wilfredo De Jesús-Rojas, Marcos J. Ramos-Benítez
Format: Article
Language:English
Published: BMC 2025-05-01
Series:Orphanet Journal of Rare Diseases
Online Access:https://doi.org/10.1186/s13023-025-03758-5
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author Lourdes Marinna Caro-Rivera
Sonya Malavez-Cajigas
Mercedes Lacourt-Ventura
Andrea P. Rivera-Torres
Dorca E. Marcano-Jiménez
Pablo López-Colon
José Muñiz-Hernández
Enid Rivera-Jiménez
Mónica Egozcue-Dionisi
Rosa Román-Carlo
Wilfredo De Jesús-Rojas
Marcos J. Ramos-Benítez
author_facet Lourdes Marinna Caro-Rivera
Sonya Malavez-Cajigas
Mercedes Lacourt-Ventura
Andrea P. Rivera-Torres
Dorca E. Marcano-Jiménez
Pablo López-Colon
José Muñiz-Hernández
Enid Rivera-Jiménez
Mónica Egozcue-Dionisi
Rosa Román-Carlo
Wilfredo De Jesús-Rojas
Marcos J. Ramos-Benítez
author_sort Lourdes Marinna Caro-Rivera
collection DOAJ
description Abstract Hermansky-Pudlak Syndrome (HPS) type 1 (HPS-1) is an autosomal recessive disorder characterized by oculocutaneous albinism, platelet dysfunction, and pulmonary fibrosis (HPS-PF), the leading cause of mortality in these patients. HPS-PF manifests earlier than idiopathic pulmonary fibrosis, typically between 30 and 40 years of age. The etiology and drivers of HPS-PF progression remain poorly understood, and no FDA-approved therapies exist. Neutrophil extracellular traps (NETs) and neutrophil-derived mediators have emerged as key players in fibrosis, promoting lung injury, inflammation, and fibroblast activation. This study evaluates the role of neutrophil activation in age-related changes in patients with HPS-1, focusing on differences in inflammatory markers, neutrophil granules, and NETosis capacity. We observed significantly elevated levels of NETs, neutrophil granule proteins (NE, NGAL, LF), and inflammatory cytokines (IL-8, IL-6) in patients with HPS-1 older than 40 years compared to younger patients and healthy controls. Additionally, fibrosis-related markers (MMP-7 and MMP-8) were significantly higher in older patients. Elevated levels of anandamide (AEA), a circulating marker of HPS-PF, were positively associated with neutrophil granule markers in older patients, suggesting its association with fibrosis. Neutrophils from older patients also demonstrated increased NETosis capacity. These findings suggest that age-related neutrophil activation may contribute to an inflammatory environment that promotes fibrosis progression in HPS-1.
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spelling doaj-art-9be78c6f6dd4414c93df25fb8efeb64b2025-08-20T02:25:11ZengBMCOrphanet Journal of Rare Diseases1750-11722025-05-0120111010.1186/s13023-025-03758-5Age-related neutrophil activation in Hermansky-Pudlak Syndrome Type-1Lourdes Marinna Caro-Rivera0Sonya Malavez-Cajigas1Mercedes Lacourt-Ventura2Andrea P. Rivera-Torres3Dorca E. Marcano-Jiménez4Pablo López-Colon5José Muñiz-Hernández6Enid Rivera-Jiménez7Mónica Egozcue-Dionisi8Rosa Román-Carlo9Wilfredo De Jesús-Rojas10Marcos J. Ramos-Benítez11Department of Basic Sciences, Ponce Health Sciences University and Ponce Research InstituteDepartment of Basic Sciences, Ponce Health Sciences University and Ponce Research InstituteDepartment of Basic Sciences, Ponce Health Sciences University and Ponce Research InstituteDepartment of Basic Sciences, Ponce Health Sciences University and Ponce Research InstituteDepartment of Basic Sciences, Ponce Health Sciences University and Ponce Research InstituteDepartment of Basic Sciences, Ponce Health Sciences University and Ponce Research InstituteDepartment of Medicine, San Juan Bautista School of MedicineDepartment of Basic Sciences, Ponce Health Sciences University and Ponce Research InstituteDepartment of Pediatrics, Medical Sciences Campus, University of Puerto RicoDepartment of Basic Sciences, Ponce Health Sciences University and Ponce Research InstituteDepartment of Basic Sciences, Ponce Health Sciences University and Ponce Research InstituteDepartment of Basic Sciences, Ponce Health Sciences University and Ponce Research InstituteAbstract Hermansky-Pudlak Syndrome (HPS) type 1 (HPS-1) is an autosomal recessive disorder characterized by oculocutaneous albinism, platelet dysfunction, and pulmonary fibrosis (HPS-PF), the leading cause of mortality in these patients. HPS-PF manifests earlier than idiopathic pulmonary fibrosis, typically between 30 and 40 years of age. The etiology and drivers of HPS-PF progression remain poorly understood, and no FDA-approved therapies exist. Neutrophil extracellular traps (NETs) and neutrophil-derived mediators have emerged as key players in fibrosis, promoting lung injury, inflammation, and fibroblast activation. This study evaluates the role of neutrophil activation in age-related changes in patients with HPS-1, focusing on differences in inflammatory markers, neutrophil granules, and NETosis capacity. We observed significantly elevated levels of NETs, neutrophil granule proteins (NE, NGAL, LF), and inflammatory cytokines (IL-8, IL-6) in patients with HPS-1 older than 40 years compared to younger patients and healthy controls. Additionally, fibrosis-related markers (MMP-7 and MMP-8) were significantly higher in older patients. Elevated levels of anandamide (AEA), a circulating marker of HPS-PF, were positively associated with neutrophil granule markers in older patients, suggesting its association with fibrosis. Neutrophils from older patients also demonstrated increased NETosis capacity. These findings suggest that age-related neutrophil activation may contribute to an inflammatory environment that promotes fibrosis progression in HPS-1.https://doi.org/10.1186/s13023-025-03758-5
spellingShingle Lourdes Marinna Caro-Rivera
Sonya Malavez-Cajigas
Mercedes Lacourt-Ventura
Andrea P. Rivera-Torres
Dorca E. Marcano-Jiménez
Pablo López-Colon
José Muñiz-Hernández
Enid Rivera-Jiménez
Mónica Egozcue-Dionisi
Rosa Román-Carlo
Wilfredo De Jesús-Rojas
Marcos J. Ramos-Benítez
Age-related neutrophil activation in Hermansky-Pudlak Syndrome Type-1
Orphanet Journal of Rare Diseases
title Age-related neutrophil activation in Hermansky-Pudlak Syndrome Type-1
title_full Age-related neutrophil activation in Hermansky-Pudlak Syndrome Type-1
title_fullStr Age-related neutrophil activation in Hermansky-Pudlak Syndrome Type-1
title_full_unstemmed Age-related neutrophil activation in Hermansky-Pudlak Syndrome Type-1
title_short Age-related neutrophil activation in Hermansky-Pudlak Syndrome Type-1
title_sort age related neutrophil activation in hermansky pudlak syndrome type 1
url https://doi.org/10.1186/s13023-025-03758-5
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