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Tolvaptan Used to The Treatment of Autosomal Dominant Polycystic Kidney Disease


自體顯性多囊性腎臟疾病(autosomal dominant polycystic kidney disease, ADPKD)是一種遺傳性腎臟疾病,病人的腎臟會出現囊泡,並隨時間不斷增多並變大,總腎體積(total kidney volume, TKV)會增大及造成慢性腎臟病,且可能有高血壓、腰痛、尿結石等相關併發症。當腎功能惡化至末期腎病變時則需洗腎或換腎。以往並無有效治療方式,主要治療方式係針對血壓控制或採取低蛋白飲食,以期能延緩病人腎功能惡化。近年,全球第一個核准於延緩腎功能惡化的ADPKD藥物tolvaptan上市並納入健保給付。Tolvaptan是血管增壓素V2受體拮抗劑(vasopressin V2 receptor antagonist),能幫助ADPKD患者降低TKV增長及減緩腎臟功能惡化,儘管在Tolvaptan Efficacy and Safety in Management of Autosomal Dominant Polycystic Kidney Disease and Its Outcomes(TEMPO)4:4擴展試驗中,tolvaptan長遠來看並未顯示出對TKV的持續緩解作用,但在減緩腎功能下降的作用仍可再維持超過2年,能減少腎臟疼痛,增加生活品質,並延緩病人至末期腎病變的時間。

Parallel abstracts

Autosomal dominant polycystic kidney disease (ADPKD) is hereditary in nature. Vesicles appear in kidneys of patients with ADPKD that increase in number and become larger over time, resulting in larger kidneys. This increase in total kidney volume (TKV) leads to chronic kidney diseases and complications, such as high blood pressure, lower back pain, and urinary stones. When renal functions deteriorate to the level of end-stage renal diseases, kidney dialysis or transplantation is required. There were no effective treatments in the past. The primary treatment involved controlling blood pressure or adopting a low-protein diet to delay the deterioration of renal functions. In recent years, tolvaptan, the first ADPKD drug to delay the deterioration of renal functions, was approved and adopted by the Taiwan Health Insurance. Tolvaptan is a vasopressin V2 receptor antagonist, which helps reduce the TKV increase and slows down renal function deterioration in patients with ADPKD. Although tolvaptan did not have a sustained relieving effect on TKV in the long-term Tolvaptan Efficacy and Safety in Management of Autosomal Dominant Polycystic Kidney Disease and Its Outcomes (TEMPO) 4:4 extended trial, it maintained the effect of slowing down renal function deterioration for more than 2 years. In addition, tolvaptan reduced kidney pain, increased the quality of life, and postponed the onset of end-stage renal diseases.