{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,14]],"date-time":"2026-07-14T02:19:30Z","timestamp":1783995570437,"version":"3.55.0"},"reference-count":34,"publisher":"MDPI AG","issue":"1","license":[{"start":{"date-parts":[[2026,1,16]],"date-time":"2026-01-16T00:00:00Z","timestamp":1768521600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Medicina"],"abstract":"<jats:p>Background and Objectives: Autosomal dominant polycystic kidney disease (ADPKD) is the most common monogenic kidney disease, and the only approved pharmacological therapy shown to slow disease progression is tolvaptan. This study presents a long-term observation of ADPKD patients treated at our center, focusing on changes in eGFR approximately one year before and at least 1 year after the initiation of tolvaptan therapy. Materials and Methods: A retrospective analysis of a cohort of ADPKD patients who have received tolvaptan treatment in our center. Results: In total, 20 patients were enrolled in the analysis. Their median time of observation since tolvaptan introduction was 23.5 months. No statistically significant difference was noted in the median monthly decrease in eGFR between the time prior to tolvaptan introduction and during tolvaptan therapy. Analysis of trajectories of eGFR in particular patients enabled the division of the cohort into three subgroups: beneficiaries (n = 7, 35%), stable (n = 8, 40%), and progressors (n = 5, n = 25%). Conclusions: Despite the low number of patients, together with a relatively short observation period, which are the main limitations of our study, our results suggest that, in real-world settings, the efficacy of tolvaptan may be lower than previously reported. There is an urgent need to identify factors responsible for the suboptimal effect of the medicine. Our findings underscore the need to re-evaluate the current inclusion criteria for tolvaptan, particularly in real-world settings where patient variability is broader than in controlled clinical trials. Tailoring treatment qualification to include more practical and region-specific factors may enhance therapeutic outcomes.<\/jats:p>","DOI":"10.3390\/medicina62010194","type":"journal-article","created":{"date-parts":[[2026,1,16]],"date-time":"2026-01-16T14:46:33Z","timestamp":1768574793000},"page":"194","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Trajectories of Kidney Function in Autosomal Dominant Polycystic Kidney Disease Patients Treated with Tolvaptan"],"prefix":"10.3390","volume":"62","author":[{"ORCID":"https:\/\/orcid.org\/0009-0001-9448-7512","authenticated-orcid":false,"given":"Zofia","family":"Jankowska","sequence":"first","affiliation":[{"name":"Department of Transplantology, Immunology, Nephrology, and Internal Diseases, Medical University of Warsaw, 02-006 Warszawa, Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8592-8814","authenticated-orcid":false,"given":"Mariusz","family":"Niemczyk","sequence":"additional","affiliation":[{"name":"Department of Transplantology, Immunology, Nephrology, and Internal Diseases, Medical University of Warsaw, 02-006 Warszawa, Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2026,1,16]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"705","DOI":"10.1111\/nep.12658","article-title":"KHA-CARI guideline recommendations for the diagnosis and management of autosomal dominant polycystic kidney disease","volume":"21","author":"Rangan","year":"2016","journal-title":"Nephrology"},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"103","DOI":"10.1016\/S0140-6736(98)03495-3","article-title":"Comparison of phenotypes of polycystic kidney disease types 1 and 2. European PKD1-PKD2 Study Group","volume":"353","author":"Hateboer","year":"1999","journal-title":"Lancet"},{"key":"ref_3","doi-asserted-by":"crossref","first-page":"234","DOI":"10.1016\/j.kint.2024.07.010","article-title":"KDIGO 2025 clinical practice guideline for the evaluation, management, and treatment of autosomal dominant polycystic kidney disease (ADPKD): Executive summary","volume":"107","author":"Torres","year":"2025","journal-title":"Kidney Int."},{"key":"ref_4","doi-asserted-by":"crossref","first-page":"825","DOI":"10.1093\/ndt\/gfab312","article-title":"An update on the use of tolvaptan for autosomal dominant polycystic kidney disease: Consensus statement on behalf of the ERA Working Group on Inherited Kidney Disorders, the European Rare Kidney Disease Reference Network and Polycystic Kidney Disease International","volume":"37","author":"Muller","year":"2022","journal-title":"Nephrol. Dial. Transpl."},{"key":"ref_5","doi-asserted-by":"crossref","first-page":"303","DOI":"10.1007\/s40265-019-1056-1","article-title":"Tolvaptan: A review in autosomal dominant polycystic kidney disease","volume":"79","author":"Blair","year":"2019","journal-title":"Drugs"},{"key":"ref_6","doi-asserted-by":"crossref","first-page":"1930","DOI":"10.1056\/NEJMoa1710030","article-title":"Tolvaptan in Later-Stage Autosomal Dominant Polycystic Kidney Disease","volume":"377","author":"Torres","year":"2017","journal-title":"N. Engl. J. Med."},{"key":"ref_7","doi-asserted-by":"crossref","first-page":"130","DOI":"10.5603\/mrj.99177","article-title":"Tolvaptan in autosomal dominant polycystic kidney disease\u2014A real-life experience","volume":"9","author":"Borowiecka","year":"2024","journal-title":"Med. Res. J."},{"key":"ref_8","doi-asserted-by":"crossref","first-page":"147","DOI":"10.2147\/DHPS.S338050","article-title":"Clinical Utility and Tolerability of Tolvaptan in the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)","volume":"14","author":"Raina","year":"2022","journal-title":"Drug Healthc. Patient Saf."},{"key":"ref_9","doi-asserted-by":"crossref","first-page":"1764","DOI":"10.1093\/ndt\/gfaf048","article-title":"The long-term effect of tolvaptan treatment on kidney function and volume in patients with ADPKD","volume":"40","author":"Geertsema","year":"2025","journal-title":"Nephrol. Dial. Transpl."},{"key":"ref_10","doi-asserted-by":"crossref","first-page":"1007","DOI":"10.2215\/CJN.13861119","article-title":"Urinary Lithogenic Risk Profile in ADPKD Patients Treated with Tolvaptan","volume":"15","author":"Bargagli","year":"2020","journal-title":"Clin. J. Am. Soc. Nephrol."},{"key":"ref_11","doi-asserted-by":"crossref","first-page":"1645","DOI":"10.1093\/ndt\/gfac298","article-title":"Tolvaptan treatment is associated with altered mineral metabolism parameters and increased bone mineral density in ADPKD patients","volume":"38","author":"Bargagli","year":"2023","journal-title":"Nephrol. Dial. Transpl."},{"key":"ref_12","first-page":"731","article-title":"The safety and efficacy of tolvaptan in the treatment of patients with autosomal dominant polycystic kidney disease: A systematic review and meta-analysis","volume":"43","author":"Li","year":"2023","journal-title":"Nefrol. Engl. Ed."},{"key":"ref_13","doi-asserted-by":"crossref","unstructured":"Sahin, A.Z., and Ozdemir, O. (2025). The effect of tolvaptan on renal progression and systemic inflammation in ADPKD. Sci. Rep., 15.","DOI":"10.1038\/s41598-025-15681-7"},{"key":"ref_14","doi-asserted-by":"crossref","unstructured":"Rigato, M., Carraro, G., Cirella, I., Dian, S., Di Vico, V., Stefanelli, L.F., Ravarotto, V., Bertoldi, G., Nalesso, F., and Cal\u00f2, L.A. (2022). Effects Effects of Tolvaptan on Oxidative Stress in ADPKD: A Molecular Biological Approach. J. Clin. Med., 11.","DOI":"10.3390\/jcm11020402"},{"key":"ref_15","doi-asserted-by":"crossref","first-page":"2458","DOI":"10.1681\/ASN.2018060590","article-title":"A Practical Guide for Treatment of Rapidly Progressive ADPKD with Tolvaptan","volume":"29","author":"Chebib","year":"2018","journal-title":"J. Am. Soc. Nephrol."},{"key":"ref_16","unstructured":"(2025, October 01). Available online: https:\/\/www.gov.pl\/web\/zdrowie\/obwieszczenie-ministra-zdrowia-z-dnia-21-pazdziernika-2021-r-w-sprawie-wykazu-refundowanych-lekow-srodkow-spozywczych-specjalnego-przeznaczenia-zywieniowego-oraz-wyrobow-medycznych-na-1-listopada-2021-r."},{"key":"ref_17","doi-asserted-by":"crossref","first-page":"113","DOI":"10.5603\/rdatf.98440","article-title":"Principles of using tolvaptan in the treatment of patients with autosomal dominant polycystic kidney disease (ADPKD). Recommendations of the Working Group of the Polish Socirty of Nephrology","volume":"16","author":"Jankowska","year":"2023","journal-title":"Ren. Dis. Transplant. Forum."},{"key":"ref_18","doi-asserted-by":"crossref","first-page":"1702","DOI":"10.34067\/KID.0000000000000302","article-title":"Comparing Effects of Tolvaptan and Instruction to Increase Water Consumption in ADPKD: Post Hoc Analysis of TEMPO 3:4","volume":"4","author":"Gobburu","year":"2023","journal-title":"Kidney360"},{"key":"ref_19","doi-asserted-by":"crossref","first-page":"562","DOI":"10.1093\/ndt\/gfab278","article-title":"Vaptans or voluntary increased hydration to protect the kidney: How do they compare?","volume":"38","author":"Bankir","year":"2023","journal-title":"Nephrol. Dial. Transplant."},{"key":"ref_20","doi-asserted-by":"crossref","first-page":"1008","DOI":"10.1093\/ndt\/gfad232","article-title":"Immediate drop of urine osmolality upon tolvaptan initiation predicts impact on renal prognosis in patients with ADPKD","volume":"39","author":"Akihisa","year":"2024","journal-title":"Nephrol. Dial. Transplant."},{"key":"ref_21","doi-asserted-by":"crossref","first-page":"1148","DOI":"10.34067\/KID.0007342020","article-title":"Dose-Dependent Effect of Tolvaptan on Renal Prognosis in Patients with Autosomal Dominant Polycystic Kidney Disease","volume":"2","author":"Akihisa","year":"2021","journal-title":"Kidney360"},{"key":"ref_22","doi-asserted-by":"crossref","first-page":"1522","DOI":"10.34067\/KID.0000000816","article-title":"Real-World Tolvaptan Use in Autosomal Dominant Polycystic Kidney Disease: Insights from Two US Medical Centers","volume":"6","author":"Rao","year":"2025","journal-title":"Kidney360"},{"key":"ref_23","doi-asserted-by":"crossref","first-page":"2953","DOI":"10.1016\/j.ekir.2025.06.054","article-title":"Cardiovascular Complications in ADPKD","volume":"10","author":"Ebrahimi","year":"2025","journal-title":"Kidney Int. Rep."},{"key":"ref_24","doi-asserted-by":"crossref","unstructured":"Chebib, F.T., Zhou, X., Garbinsky, D., Davenport, E., Nunna, S., Oberdhan, D., and Fernandes, A. (2023). Tolvaptan and Kidney Function Decline in Older Individuals With Autosomal Dominant Polycystic Kidney Disease: A Pooled Analysis of Randomized Clinical Trials and Observational Studies. Kidney Med., 5.","DOI":"10.1016\/j.xkme.2023.100639"},{"key":"ref_25","doi-asserted-by":"crossref","first-page":"908","DOI":"10.2215\/CJN.16871020","article-title":"Overweight and Obesity and Progression of ADPKD","volume":"16","author":"Nowak","year":"2021","journal-title":"Clin. J. Am. Soc. Nephrol."},{"key":"ref_26","doi-asserted-by":"crossref","first-page":"2171","DOI":"10.1016\/j.ekir.2021.05.037","article-title":"Tolvaptan in ADPKD Patients With Very Low Kidney Function","volume":"6","author":"Torres","year":"2021","journal-title":"Kidney Int. Rep."},{"key":"ref_27","doi-asserted-by":"crossref","first-page":"1864","DOI":"10.1016\/j.ekir.2025.03.020","article-title":"Kidney Function Trajectories with Tolvaptan in ADPKD Patients with CKD-G5","volume":"10","author":"Sekine","year":"2025","journal-title":"Kidney Int. Rep."},{"key":"ref_28","doi-asserted-by":"crossref","first-page":"522","DOI":"10.34067\/KID.0000000000000404","article-title":"Initial eGFR Changes Predict Response to Tolvaptan in ADPKD","volume":"5","author":"Mochizuki","year":"2024","journal-title":"Kidney360"},{"key":"ref_29","doi-asserted-by":"crossref","first-page":"345","DOI":"10.1007\/s10157-012-0706-3","article-title":"Clinical characteristics and predictors of progression of chronic kidney disease in autosomal dominant polycystic kidney disease: A single center experience","volume":"17","author":"Ozkok","year":"2013","journal-title":"Clin. Exp. Nephrol."},{"key":"ref_30","doi-asserted-by":"crossref","first-page":"1887","DOI":"10.1093\/ndt\/gfv422","article-title":"Albuminuria and tolvaptan in autosomal-dominant polycystic kidney disease: Results of the TEMPO 3:4 Trial","volume":"31","author":"Gansevoort","year":"2016","journal-title":"Nephrol. Dial. Transplant."},{"key":"ref_31","doi-asserted-by":"crossref","first-page":"39","DOI":"10.1007\/s11892-021-01442-z","article-title":"Renal Protection with SGLT2 Inhibitors: Effects in Acute and Chronic Kidney Disease","volume":"22","author":"Bailey","year":"2022","journal-title":"Curr. Diabetes Rep."},{"key":"ref_32","doi-asserted-by":"crossref","first-page":"419","DOI":"10.1007\/s13730-024-00859-1","article-title":"Additional renoprotective effect of the SGLT2 inhibitor dapagliflozin in a patient with ADPKD receiving tolvaptan treatment","volume":"13","author":"Minatoguchi","year":"2024","journal-title":"CEN Case Rep."},{"key":"ref_33","doi-asserted-by":"crossref","first-page":"500","DOI":"10.1007\/s13730-025-00990-7","article-title":"Renoprotective effects of combination therapy with tolvaptan and dapagliflozin in autosomal dominant polycystic kidney disease: A four-case series","volume":"14","author":"Nishida","year":"2025","journal-title":"CEN Case Rep."},{"key":"ref_34","doi-asserted-by":"crossref","first-page":"745","DOI":"10.1159\/000509817","article-title":"Genetics May Predict Effectiveness of Tolvaptan in Autosomal Dominant Polycystic Kidney Disease","volume":"51","author":"Sekine","year":"2020","journal-title":"Am. J. Nephrol."}],"container-title":["Medicina"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.mdpi.com\/1648-9144\/62\/1\/194\/pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,1,22]],"date-time":"2026-01-22T05:17:54Z","timestamp":1769059074000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.mdpi.com\/1648-9144\/62\/1\/194"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,1,16]]},"references-count":34,"journal-issue":{"issue":"1","published-online":{"date-parts":[[2026,1]]}},"alternative-id":["medicina62010194"],"URL":"https:\/\/doi.org\/10.3390\/medicina62010194","relation":{},"ISSN":["1648-9144"],"issn-type":[{"value":"1648-9144","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,1,16]]}}}