Naranjo Javier, Borrego Francisco, Rocha José Luis, Salgueira Mercedes, Martín-Gomez Maria Adoración, Orellana Cristhian, Morales Ana, Vallejo Fernando, Hidalgo Pilar, Rodríguez Francisca, Garófano Remedios, González Isabel, Esteban Rafael, Espinosa Mario
Department of Nephrology, Hospital Universitario Puerta del Mar, Cádiz, Spain.
Department of Nephrology, Complejo Hospitalario de Jaén, Jaén, Spain.
Front Med (Lausanne). 2022 Sep 29;9:987092. doi: 10.3389/fmed.2022.987092. eCollection 2022.
Tolvaptan (TV) is the first vasopressin-receptor antagonist approved for the treatment of autosomal dominant polycystic kidney disease (ADPKD). No publications report TV experience in real clinical practice during the first year of treatment.
A prospective study of an initial cohort of 220 rapidly progressing patients treated with TV for 12 months. The tolerability of TV, the evolution of the estimated glomerular filtration rate (eGFR), analytical parameters, and blood pressure were analyzed.
A total of 163 patients (78.2%) received TV for 1 year. The main causes of treatment withdrawal were the aquaretic effects (11%), eGFR deterioration (5%), and hepatic toxicity (2.3%). eGFR decreased significantly after 1 month of treatment without further changes. The decrease in eGFR in the first month was higher in patients with an initially higher eGFR. The eGFR drop during the first year of treatment with TV was lower than that reported by patients in the 2 years prior to TV treatment (-1.7 ± 7.6 vs. -4.4 ± 4.8 mL/min, = 0.003). Serum sodium and uric acid concentrations increased, and morning urinary osmolality decreased in the first month, with no further changes. Blood pressure decreased significantly without changes in antihypertensive medication.
TV treatment is well tolerated by most patients. Liver toxicity is very rare and self-limited. TV reduces eGFR in the first month without showing further changes during the first year of treatment. Patients with a higher starting eGFR will suffer a greater initial drop, with a longer recovery. We suggest using the eGFR observed after a month of treatment as the reference for future comparisons and calculating the rate of eGFR decline in patients undergoing TV treatment.
托伐普坦（TV）是首个被批准用于治疗常染色体显性多囊肾病（ADPKD）的血管加压素受体拮抗剂。尚无关于TV在治疗第一年真实临床实践中的经验报道。
对220例接受TV治疗12个月的快速进展患者进行了一项前瞻性初始队列研究。分析了TV的耐受性、估计肾小球滤过率（eGFR）的变化、分析参数和血压。
共有163例患者（78.2%）接受TV治疗1年。治疗中断的主要原因是利水作用（11%）、eGFR恶化（5%）和肝毒性（2.3%）。治疗1个月后eGFR显著下降，之后无进一步变化。初始eGFR较高的患者在第一个月eGFR下降幅度更大。TV治疗第一年的eGFR下降幅度低于TV治疗前两年患者报告的下降幅度（-1.7±7.6 vs. -4.4±4.8 mL/min，P = 0.003）。第一个月血清钠和尿酸浓度升高，晨尿渗透压降低，之后无进一步变化。血压显著下降，且降压药物未改变。
大多数患者对TV治疗耐受性良好。肝毒性非常罕见且为自限性。TV在第一个月会降低eGFR，在治疗的第一年未显示出进一步变化。起始eGFR较高的患者初始下降幅度更大，恢复时间更长。我们建议将治疗1个月后观察到的eGFR作为未来比较的参考，并计算接受TV治疗患者的eGFR下降率。
