CA24124 - Network for the Advancement of Neutropenia Research and Patient Support (Neutro-NARPS)

Chronic Neutropenia (CNP) is a rare disorder characterized by persistently low absolute neutrophil counts and may occur as idiopathic entity or as a manifestation of a wide spectrum, mild to life-threatening, congenital or acquired, diseases. The  principal challenge of the Network for the Advancement of Neutropenia Research and Patient Support (Neutro-NARPS) is to establish a multi-disciplinary consortium of top-level European and International experts and young investigators with high interest in CNP from the fields of medicine, molecular biology, bioinformatics and computational biology. These partners will work in close collaboration with patient Registries and Organizations, biotech and pharmaceutical companies, the European Haematology Association, and Competent Authorities to bridge the gap between basic research, translational medicine, and clinical practice. The Action’s network will focus on (a) the understanding of the pathogenesis and natural history of CNP, (b) improvement of diagnosis, (c) development and translation to the clinic of novel, repurposed or targeted treatment approaches including gene therapy, and (d) evaluation of the effect of the disease and treatment on patients’ Quality of Life (QoL). Neutro-NARPS envisions elucidating the pathogenetic mechanisms particularly associated with newly recognized CNP entities, investigating the role of nutrition and inflammation on disease initiation and evolution, identifying biomarkers for targeted therapies, and enhancing diagnostic and prognostic precision by applying machine learning (ML) technologies. The Action also aims to facilitate the translation of novel and potentially curative therapies from bench-to-bedside and to improve QoL by developing novel tools for the evaluation of patient reported outcomes (PROs).