Yamazaki, Mai and Kawano, Haruna and Miyoshi, Miho and Kimura, Tomoki and Takahashi, Keiji and Muto, Satoru and Horie, Shigeo (2024) Long-Term Effects of Tolvaptan in Autosomal Dominant Polycystic Kidney Disease: Predictors of Treatment Response and Safety over 6 Years of Continuous Therapy. International Journal of Molecular Sciences, 25 (4). p. 2088. ISSN 1422-0067
![[thumbnail of ijms-25-02088.pdf]](http://research.send4journal.com/style/images/fileicons/text.png)
ijms-25-02088.pdf - Published Version
Download (1MB)
Abstract
Long-Term Effects of Tolvaptan in Autosomal Dominant Polycystic Kidney Disease: Predictors of Treatment Response and Safety over 6 Years of Continuous Therapy Mai Yamazaki Department of Urology, Graduate School of Medicine, Juntendo University, Tokyo 113-8431, Japan Haruna Kawano Department of Urology, Graduate School of Medicine, Juntendo University, Tokyo 113-8431, Japan Department of Advanced Informatics for Genetic Diseases, Graduate School of Medicine, Juntendo University, Tokyo 113-8431, Japan Miho Miyoshi Department of Urology, Graduate School of Medicine, Juntendo University, Tokyo 113-8431, Japan Tomoki Kimura Department of Urology, Graduate School of Medicine, Juntendo University, Tokyo 113-8431, Japan http://orcid.org/0009-0004-7434-8015 Keiji Takahashi Department of Urology, Graduate School of Medicine, Juntendo University, Tokyo 113-8431, Japan Satoru Muto Department of Advanced Informatics for Genetic Diseases, Graduate School of Medicine, Juntendo University, Tokyo 113-8431, Japan Department of Urology, Juntendo University Nerima Hospital, Tokyo 177-8521, Japan Shigeo Horie Department of Urology, Graduate School of Medicine, Juntendo University, Tokyo 113-8431, Japan Department of Advanced Informatics for Genetic Diseases, Graduate School of Medicine, Juntendo University, Tokyo 113-8431, Japan
Tolvaptan, an oral vasopressin V2 receptor antagonist, reduces renal volume expansion and loss of renal function in patients with autosomal dominant polycystic kidney disease (ADPKD). Data for predictive factors indicating patients more likely to benefit from long-term tolvaptan are lacking. Data were retrospectively collected from 55 patients on tolvaptan for 6 years. Changes in renal function, progression of renal dysfunction (estimated glomerular filtration rate [eGFR], 1-year change in eGFR [ΔeGFR/year]), and renal volume (total kidney volume [TKV], percentage 1-year change in TKV [ΔTKV%/year]) were evaluated at 3-years pre-tolvaptan, at baseline, and at 6 years. In 76.4% of patients, ΔeGFR/year improved at 6 years. The average 6-year ΔeGFR/year (range) minus baseline ΔeGFR/year: 3.024 (−8.77–20.58 mL/min/1.73 m2). The increase in TKV was reduced for the first 3 years. A higher BMI was associated with less of an improvement in ΔeGFR (p = 0.027), and family history was associated with more of an improvement in ΔeGFR (p = 0.044). Hypernatremia was generally mild; 3 patients had moderate-to-severe hyponatremia due to prolonged, excessive water intake in response to water diuresis—a side effect of tolvaptan. Family history of ADPKD and baseline BMI were contributing factors for ΔeGFR/year improvement on tolvaptan. Hyponatremia should be monitored with long-term tolvaptan administration.
02 08 2024 2088 ijms25042088 https://creativecommons.org/licenses/by/4.0/ 10.3390/ijms25042088 https://www.mdpi.com/1422-0067/25/4/2088 https://www.mdpi.com/1422-0067/25/4/2088/pdf Alam Autosomal dominant polycystic kidney disease Lancet 2019 10.1016/S0140-6736(18)32782-X 393 919 Gabow Autosomal Dominant Polycystic Kidney Disease N. Engl. J. Med. 1993 10.1056/NEJM199307293290508 329 332 Grantham Volume Progression in Polycystic Kidney Disease N. Engl. J. Med. 2006 10.1056/NEJMoa054341 354 2122 Grantham Volume progression in autosomal dominant polycystic kidney disease: The major factor determining clinical outcomes Clin. J. Am. Soc. Nephrol. 2006 10.2215/CJN.00330705 1 148 Torres Tolvaptan in Patients with Autosomal Dominant Polycystic Kidney Disease N. Engl. J. Med. 2012 10.1056/NEJMoa1205511 367 2407 Torres Tolvaptan in Later-Stage Autosomal Dominant Polycystic Kidney Disease N. Engl. J. Med. 2017 10.1056/NEJMoa1710030 377 1930 Zhou Pooled Data Analysis of the Long-Term Treatment Effects of Tolvaptan in ADPKD Kidney Int. Rep. 2022 10.1016/j.ekir.2022.02.009 7 1037 Estilo Evaluating the impact of a Risk Evaluation and Mitigation Strategy with tolvaptan to monitor liver safety in patients with autosomal dominant polycystic kidney disease Clin. Kidney J. 2022 10.1093/ckj/sfac076 15 1553 Edwards Long-Term Administration of Tolvaptan in Autosomal Dominant Polycystic Kidney Disease Clin. J. Am. Soc. Nephrol. 2018 10.2215/CJN.01520218 13 1153 Torres Multicenter Study of Long-Term Safety of Tolvaptan in Later-Stage Autosomal Dominant Polycystic Kidney Disease Clin. J. Am. Soc. Nephrol. 2020 10.2215/CJN.10250620 16 48 Torres Tolvaptan in ADPKD Patients with Very Low Kidney Function Kidney Int. Rep. 2021 10.1016/j.ekir.2021.05.037 6 2171 Muto The effect of tolvaptan on autosomal dominant polycystic kidney disease patients: A subgroup analysis of the Japanese patient subset from TEMPO 3:4 trial Clin. Exp. Nephrol. 2015 10.1007/s10157-015-1086-2 19 867 Torres Rationale and Design of a Clinical Trial Investigating Tolvaptan Safety and Efficacy in Autosomal Dominant Polycystic Kidney Disease Am. J. Nephrol. 2017 10.1159/000456087 45 257 Torres Multicenter, open-label, extension trial to evaluate the long-term efficacy and safety of early versus delayed treatment with tolvaptan in autosomal dominant polycystic kidney disease: The TEMPO 4:4 Trial Nephrol. Dial. Transplant. 2017 10.1093/ndt/gfx043 33 477 Irazabal Imaging classification of autosomal dominant polycystic kidney disease: A simple model for selecting patients for clinical trials J. Am. Soc. Nephrol. 2015 10.1681/ASN.2013101138 26 160 Messchendorp 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 Nephrol. Dial. Transplant. 2022 10.1093/ndt/gfab312 37 825 10.3390/biom13071020 Kimura, T., Kawano, H., Muto, S., Muramoto, N., Takano, T., Lu, Y., Eguchi, H., Wada, H., Okazaki, Y., and Ide, H. (2023). PKD1 Mutation Is a Biomarker for Autosomal Dominant Polycystic Kidney Disease. Biomolecules, 13. Gall The PROPKD Score: A New Algorithm to Predict Renal Survival in Autosomal Dominant Polycystic Kidney Disease J. Am. Soc. Nephrol. 2016 10.1681/ASN.2015010016 27 942 Spithoven Analysis of data from the ERA-EDTA Registry indicates that conventional treatments for chronic kidney disease do not reduce the need for renal replacement therapy in autosomal dominant polycystic kidney disease Kidney Int. 2014 10.1038/ki.2014.120 86 1244 Chebib Assessing Risk of Rapid Progression in Autosomal Dominant Polycystic Kidney Disease and Special Considerations for Disease-Modifying Therapy Am. J. Kidney Dis. 2021 10.1053/j.ajkd.2020.12.020 78 282 Choi Trends in the incidence and prevalence of end-stage renal disease with hemodialysis in entire Korean population: A nationwide population-based study Medicine 2021 10.1097/MD.0000000000025293 100 e25293 Fernando Incidence and survival of end-stage kidney disease due to polycystic kidney disease in Australia and New Zealand (1963–2014) Popul. Health Metr. 2017 10.1186/s12963-017-0123-7 15 7 Oh Clinical Characteristics of Rapid Progression in Asia-Pacific Patients With ADPKD Kidney Int. Rep. 2023 10.1016/j.ekir.2023.06.018 8 1801 Naranjo Comparative analysis of tools to predict rapid progression in autosomal dominant polycystic kidney disease Clin. Kidney J. 2021 10.1093/ckj/sfab293 15 912 Nishio A digest from evidence-based Clinical Practice Guideline for Polycystic Kidney Disease 2020 Clin. Exp. Nephrol. 2021 10.1007/s10157-021-02097-6 25 1292 10.1371/journal.pone.0190430 Solazzo, A., Testa, F., Giovanella, S., Busutti, M., Furci, L., Carrera, P., Ferrari, M., Ligabue, G., Mori, G., and Leonelli, M. (2018). The prevalence of autosomal dominant polycystic kidney disease (ADPKD): A meta-analysis of European literature and prevalence evaluation in the Italian province of Modena suggest that ADPKD is a rare and underdiagnosed condition. PLoS ONE, 13. Gimpel International consensus statement on the diagnosis and management of autosomal dominant polycystic kidney disease in children and young people Nat. Rev. Nephrol. 2019 10.1038/s41581-019-0155-2 15 713 Iseki Body mass index and the risk of development of end-stage renal disease in a screened cohort Kidney Int. 2004 10.1111/j.1523-1755.2004.00582.x 65 1870 Wang Association between obesity and kidney disease: A systematic review and meta-analysis Kidney Int. 2008 10.1038/sj.ki.5002586 73 19 Rowe Defective glucose metabolism in polycystic kidney disease identifies a new therapeutic strategy Nat. Med. 2013 10.1038/nm.3092 19 488 10.1371/journal.pone.0146654 Riwanto, M., Kapoor, S., Rodriguez, D., Edenhofer, I., Segerer, S., and Wüthrich, R.P. (2016). Inhibition of Aerobic Glycolysis Attenuates Disease Progression in Polycystic Kidney Disease. PLoS ONE, 11. Warner Food Restriction Ameliorates the Development of Polycystic Kidney Disease J. Am. Soc. Nephrol. 2015 10.1681/ASN.2015020132 27 1437 Kipp A mild reduction of food intake slows disease progression in an orthologous mouse model of polycystic kidney disease Am. J. Physiol. Renal Physiol. 2016 10.1152/ajprenal.00551.2015 310 F726 Schrier Blood Pressure in Early Autosomal Dominant Polycystic Kidney Disease N. Engl. J. Med. 2014 10.1056/NEJMoa1402685 371 2255 Nowak Overweight and Obesity Are Predictors of Progression in Early Autosomal Dominant Polycystic Kidney Disease J. Am. Soc. Nephrol. 2018 10.1681/ASN.2017070819 29 571 Nowak Overweight and Obesity and Progression of ADPKD Clin. J. Am. Soc. Nephrol. 2021 10.2215/CJN.16871020 16 908 Kashyap Metalloproteinase PAPP-A regulation of IGF-1 contributes to polycystic kidney disease pathogenesis JCI Insight 2020 10.1172/jci.insight.135700 5 e135700 Watkins Clinical Pattern of Tolvaptan-Associated Liver Injury in Subjects with Autosomal Dominant Polycystic Kidney Disease: Analysis of Clinical Trials Database Drug Saf. 2015 10.1007/s40264-015-0327-3 38 1103 Endo Role of Liver Transplantation in Tolvaptan-Associated Acute Liver Failure Kidney Int. Rep. 2019 10.1016/j.ekir.2019.09.002 4 1653 Combs Dysnatremias in Patients with Kidney Disease Am. J. Kidney Dis. 2014 10.1053/j.ajkd.2013.09.017 63 294 Sterns Treatment Guidelines for Hyponatremia: Stay the Course Clin. J. Am. Soc. Nephrol. 2023 10.2215/CJN.0000000000000244 19 129 Tucker Diagnosis and Management of Hyponatremia: A Review JAMA 2022 10.1001/jama.2022.11176 328 280 Matsuo Revised Equations for Estimated GFR From Serum Creatinine in Japan Am. J. Kidney Dis. 2009 10.1053/j.ajkd.2008.12.034 53 982 Kanda Investigation of the freely available easy-to-use software ‘EZR’ for medical statistics Bone Marrow Transplant. 2013 10.1038/bmt.2012.244 48 452
Item Type: | Article |
---|---|
Subjects: | EP Archives > Multidisciplinary |
Depositing User: | Managing Editor |
Date Deposited: | 09 Feb 2024 06:25 |
Last Modified: | 09 Feb 2024 06:25 |
URI: | http://research.send4journal.com/id/eprint/3731 |