Research Coordination Objectives
The key objectives of Neutro-NARPS are:
a) To identify genetic causes of unclassified congenital CNP and clarify the role of inflammation in disease progression. This includes detecting undiagnosed patients, integrating NGS data to find relevant variants, and validating their function in granulopoiesis and MDS/AL development through collaborative efforts.
b) To elucidate the mechanisms of newly recognized CNP entities by identifying patients with likely-acquired, idiopathic, or lifestyle-related (LEA-associated) CNP, and analyzing GWAS and WES/WGS data to uncover genetic and molecular factors linked to immune dysregulation and neutrophil production.
c) To investigate CNP associated with novel therapies by identifying affected patients and analyzing biological samples to understand how treatments such as immunotherapies and targeted agents impact neutrophil production and survival.
d) To develop machine learning models using clinical and multi-omics data from international registries, aiming to improve diagnosis, classification, and prognosis, predict disease progression (e.g., MDS/AL), and identify new biomarkers and disease subtypes.
e) To establish a regulatory framework that supports clinical trials for innovative treatments, including gene therapy and repurposed drugs, facilitating the translation of research into clinical practice through collaboration with clinicians, researchers, and industry partners.
f) To develop and implement electronic tools for patient-reported outcomes (PROs) to improve quality of life (QoL), enabling personalized care and broader application in other bone marrow failure syndromes.
Capacity-building Objectives
The Action builds on a strong multidisciplinary and interdisciplinary network aligned with COST missions, aiming to improve diagnosis and harmonize the management of CNP patients across Europe and beyond. The Neutro-NARPS network brings together clinicians, biomedical and computational researchers, and physician-scientists, alongside patient registries, organizations, pharmaceutical companies, SMEs, and clinical research infrastructures such as the European Clinical Research Infrastructure Network. It is further supported by leading scientific bodies like the European Hematology Association and the Severe Chronic Neutropenia International Registry, as well as national associations and regulatory authorities, ensuring expertise in scientific, ethical, and regulatory aspects and facilitating dissemination and clinical translation.
The Action emphasizes the active involvement of Young Researchers and Innovators (YRIs), particularly from inclusiveness target countries, aiming to build the next generation of CNP experts while promoting gender balance and integrating emerging research groups into European networks. It fosters collaboration among researchers, clinicians, patient organizations, and industry to advance innovation and translate research into clinical applications.
Its capacity-building objectives are to:
a) strengthen collaboration and knowledge exchange on rare and novel CNP entities and therapies;
b) bridge clinical, biomedical, and computational disciplines to develop machine learning models and patient-reported outcome (PRO) tools;
c) promote collaboration for the development and exploitation of innovative diagnostic and prognostic tools;
d) act as a comprehensive stakeholder platform covering all aspects of CNP research, treatment, and patient support;
e) train and empower a new generation of researchers, particularly from underrepresented regions, enhancing European research capacity and inclusiveness.
Action Of European Cooperation in Science and Technology
This website is based upon work from COST Action Neutro-NARPS, CA24124, supported by COST (European Cooperation in Science and Technology).
COST (European Cooperation in Science and Technology) is a funding agency for research and innovation networks. Our Actions help connect research initiatives across Europe and enable scientists to grow their ideas by sharing them with their peers. This boosts their research, career and innovation.
www.cost.eu
