CNS-derived microparticles: a hypothesis for biomarker-based assessment of neurological complications in β-thalassemia

Abstract

β-thalassemia, a prevalent hereditary hematological condition, is associated with multi-organ impairment, including well-documented neurological complications. These neurological sequelae are primarily attributed to chronic anemia and iron overload-induced neurotoxicity. While existing diagnostic methods assess neurological deficits, a more practical and sensitive strategy is critically needed for early detection and effective treatment monitoring in β-thalassemia patients. A comprehensive review of the literature provides compelling evidence that 1) diverse cell types within the central nervous system (CNS) release specific microparticles (MPs); 2) the concentrations of these MPs in biological fluids are directly influenced by pathological damage to their originating cells; and 3) the unique pathological state of β-thalassemia e.g., oxidative stress, hypoxia, inflammation actively contributes to this MP release. Therefore, we hypothesize that elevated concentrations of CNS-derived MPs in β-thalassemia patients will directly correlate with the severity of neurological abnormalities and CNS injury. If rigorously validated through further in vivo and clinical studies, our proposed MP-based assessment could represent a novel and practical approach for determining the early stages of neurological involvement and effectively monitoring CNS recovery following therapy in β-thalassemia.