VMAT2抑制剂治疗亨廷顿舞蹈病的研究进展OA

Huntington's disease(HD)is an autosomal dominant neurodegenerative disease,characterized clinically by choreiform involuntary movements,cognitive impairment and psychiatric symptoms,with insidious onset,slow progression,poor prognosis,high rate of disability,and a heavy disease burden.At present,no mature disease-modifying therapy is available,and pharmacological management remains largely symptomatic.Vesicular monoamine transporter 2(VMAT2)inhibitors reduce excessive dopaminergic overactivity in the striatum by inhibiting presynaptic vesicular monoamine uptake and depleting dopamine stores,and have become one of the main drug options for the treatment of chorea in patients with HD,including tetrabenazine,deutetrabenazine and valbenazine.This review summarizes the pharmacological properties,clinical evidence and safety profiles of these VMAT2 inhibitors.Current studies indicate that all three VMAT2 inhibitors can significantly improve choreic symptoms in patients with HD.However,adverse effects such as depression,increased suicide risk and extrapyramidal symptoms still warrant close attention.Future head-to-head trials and real-world studies are needed to further optimize patient selection and dosing strategies for different agents and to explore their integration with potential disease-modifying therapies.