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Absence of mTOR Inhibitor Effect on Hepatic Cyst Growth: A Case Report of a Kidney Transplant Recipient with Autosomal Dominant Polycystic Kidney Disease.

机译:缺乏mTOR抑制剂对肝囊肿生长的影响:肾移植受者常染色体显性多囊肾疾病的一例报道。

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摘要

Some experimental studies have suggested a beneficial effect of the mammalian target of rapamycin (mTOR) inhibitor use on hepatic and renal cyst growth in patients with autosomal dominant polycystic kidney disease (ADPKD). However, the results of clinical studies are conflicting and the role of mTOR inhibitors is still uncertain. We report the case of a patient with ADPKD who underwent deceased kidney transplantation because of an end-stage renal disease. The evolution was uneventful with an excellent graft function under cyclosporine (CsA) monotherapy. Some years later, the patient developed a symptomatic hepatomegaly due to growth of cysts. CsA was replaced by sirolimus, an mTOR inhibitor, in order to reduce or control the increase in the cyst and liver volume. Despite the switch, the hepatic volume increased by 25% in two years. Finally sirolimus was stopped because of the lack of effect on hepatic cyst growth and the presence of sirolimus side effects. The interest of our case resides in the followup by MRI imaging during the mTOR inhibitor treatment and 15 months after the restart of the initial immunosuppressive therapy. This observation indicates that mTOR inhibitors did not have significant effect on cyst-associated hepatic growth in our patient, which is consistent with some results of recent large clinical studies.
机译:一些实验研究表明,使用雷帕霉素(mTOR)抑制剂的哺乳动物靶标对常染色体显性遗传性多囊肾病(ADPKD)患者的肝和肾囊肿生长具有有益作用。但是,临床研究结果相互矛盾,mTOR抑制剂的作用仍不确定。我们报道了由于末期肾脏疾病而进行过肾脏移植手术的ADPKD患者。在环孢素(CsA)单一疗法下,移植物具有良好的移植功能,进展平稳。几年后,由于囊肿的生长,患者出现了症状性肝肿大。为了减少或控制囊肿和肝脏体积的增加,用mTOR抑制剂西罗莫司代替了CsA。尽管发生了变化,但两年内肝脏体积增加了25%。最后,由于缺乏对肝囊肿生长的作用以及存在西罗莫司的副作用,因此停止了西罗莫司的治疗。我们病例的兴趣在于在mTOR抑制剂治疗期间以及重新开始初始免疫抑制治疗后15个月的MRI成像随访。该观察结果表明,mTOR抑制剂对我们患者中与囊肿相关的肝生长没有显着影响,这与最近的大型临床研究的一些结果一致。

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