Giuliani Anna, Marturano Davide, Corradi Valentina, Caprara Carlotta, Rigato Matteo, Marcello Matteo, Gastaldon Fiorella, Ronco Claudio, Zanella Monica
Dipartimento di Nefrologia, Dialisi e Trapianto, Ospedale San Bortolo, Vicenza, Italy; International Renal Research Institute of Vicenza (IRRIV), Vicenza, Italy.
International Renal Research Institute of Vicenza (IRRIV), Vicenza, Italy; DIMED, Università di Padova, Padova, Italy.
G Ital Nefrol. 2021 Oct 26;38(5):2021-vol5.
Autosomal dominant polycystic kidney disease (ADPKD) is the most common hereditary kidney disease and accounts for∼10% of patients on renal replacement therapy. In the last decade, no specific treatment was available and only preventive measures could be put in place to delay the onset of ESRD. Following the results of the TEMPO 3:4 study, tolvaptan was approved in many countries, for the purpose of slowing the progression of renal insufficiency. In Italy tolvaptan is available since 2016 for patients with chronic kidney disease (CKD) stage 1-3, and since 2020 for patients with CKD stage 4, who fulfil the criteria of "rapid disease progression", according to the European recommendations. After this approval, Italian nephrology units have had to change their organization to be able to identify the patients eligible for the drug and to guarantee frequent patient monitoring. In this paper, we present our three-year experiences with tolvaptan, focusing on its safety profile and tolerability, but also on the high burden of care that such therapy represents not only for doctors, but also for patients. Strategies to implement remote monitoring may be useful to reduce the burden of assistance on one side, and the medicalization of ADPKD patients in the early stage of the disease, on the other.
常染色体显性多囊肾病(ADPKD)是最常见的遗传性肾病,约占接受肾脏替代治疗患者的10%。在过去十年中,尚无特效治疗方法,只能采取预防措施来延缓终末期肾病(ESRD)的发病。基于TEMPO 3:4研究结果,托伐普坦在许多国家获批,用于减缓肾功能不全的进展。在意大利,自2016年起,慢性肾脏病(CKD)1-3期患者可使用托伐普坦;自2020年起,符合“疾病进展迅速”标准的CKD 4期患者也可使用,这是根据欧洲的建议。获批后,意大利的肾脏病科室不得不调整其组织架构,以便能够识别 eligible for the drug 可使用该药物的患者,并确保对患者进行频繁监测。在本文中,我们介绍了使用托伐普坦三年的经验,重点关注其安全性和耐受性,同时也关注这种治疗不仅给医生,也给患者带来的高护理负担。实施远程监测的策略可能有助于一方面减轻护理负担,另一方面减少疾病早期ADPKD患者的医疗化程度。 (注:原文中“eligible for the drug”翻译为“可使用该药物的”,这里英文表述不太完整,完整准确的应该是“符合使用该药物条件的” )