Extracellular vesicle-based drug delivery in pediatric immune thrombocytopenia

Pediatric immune thrombocytopenia (ITP) is an acquired immune-mediated disorder triggered by autoantibody production directed against glycoproteins on the platelet surface, leading to platelet destruction in the spleen and impaired megakaryopoiesis in the bone marrow. The immune response is further compromised by a dysregulated inflammatory environment involving T-cell proliferation, pro-inflammatory macrophage polarization and defective immunomodulatory function of mesenchymal stromal cells (MSCs). Although first-line therapies such as intravenous immunoglobulin G and corticosteroids remain beneficial, they are also associated with relevant toxicities and do not fully address the systemic nature of the disease in pediatric patients. Extracellular vesicles (EVs) as cellular mediators secreted by all kinds of cells are currently under investigation as promising therapeutic nanocarriers. MSC-derived extracellular vesicles (MSC-EVs) in particular, have emerged as therapeutic candidates due to their low immunogenicity, high biocompatibility, and intrinsic ability to transport cargo capable of modulating the immune response. MSC-EVs may simultaneously regulate T-cell balance, influence macrophage polarization, support megakaryopoiesis, and deliver regulatory molecules to specific targets. Bioengineering strategies involving cargo loading and surface functionalization may further improve their therapeutic potential and homing capabilities. Although key barriers to clinical translation remain, MSC-EVs represent a promising platform for a precision and cell-free approach in pediatric ITP. Although direct pediatric-specific evidence remains limited, in this review we provide a comprehensive overview of the biological properties, immunomodulatory mechanisms, and translational challenges of MSC-EVs, comparing their advantages against conventional pharmacological approaches and other available synthetic nanocarriers.

Abstract from PubMed Central (PMID 42996533, PMC13740497). This entry was added automatically by our daily literature monitor because it matches the topics we follow; read the full paper at the original source.