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Persistence of tolvaptan medication for autosomal dominant polycystic kidney disease: A retrospective cohort study using Shizuoka Kokuho Database

Autosomal dominant polycystic kidney disease (ADPKD) is a rare hereditary disease leading to end-stage renal failure in approximately half of patients by seventy years of age. It is important to continuously take tolvaptan to control disease progression. However, adherence to tolvaptan in a real-wor...

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Autores principales: Saito, Ryuta, Yamamoto, Hiroyuki, Ichihara, Nao, Kumamaru, Hiraku, Nishimura, Shiori, Shimada, Koki, Mori, Kiyoshi, Miyachi, Yoshiki, Miyata, Hiroaki
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Lippincott Williams & Wilkins 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9542978/
https://www.ncbi.nlm.nih.gov/pubmed/36221375
http://dx.doi.org/10.1097/MD.0000000000030923
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author Saito, Ryuta
Yamamoto, Hiroyuki
Ichihara, Nao
Kumamaru, Hiraku
Nishimura, Shiori
Shimada, Koki
Mori, Kiyoshi
Miyachi, Yoshiki
Miyata, Hiroaki
author_facet Saito, Ryuta
Yamamoto, Hiroyuki
Ichihara, Nao
Kumamaru, Hiraku
Nishimura, Shiori
Shimada, Koki
Mori, Kiyoshi
Miyachi, Yoshiki
Miyata, Hiroaki
author_sort Saito, Ryuta
collection PubMed
description Autosomal dominant polycystic kidney disease (ADPKD) is a rare hereditary disease leading to end-stage renal failure in approximately half of patients by seventy years of age. It is important to continuously take tolvaptan to control disease progression. However, adherence to tolvaptan in a real-world setting, rather than randomized controlled trials (RCTs), has not been sufficiently reported. We aimed to investigate tolvaptan persistence among patients with ADPKD using a large claims database. Using the Shizuoka Kokuho Database, we identified patients diagnosed with ADPKD who were prescribed tolvaptan from March 2014–September 2018 in Japan. The persistence rate of tolvaptan medication was estimated by Kaplan–Meier analysis, and patient background factors associated with treatment discontinuation were exploratively evaluated with log-rank tests. We identified 1714 eligible patients with ADPKD, and among them, 25 patients used tolvaptan medication. We followed up these patients, whose median treatment duration was 21 months. The persistence rates at 12, 24, and 36 months were estimated to be 70.8% (95% confidence interval: 48.2–93.4), 46.5% (23.2–66.9), and 38.7% (16.4–60.8), respectively. In the exploratory analysis, there were no factors that were obviously associated with tolvaptan discontinuation. The persistence rate of tolvaptan in patients with ADPKD in a real-world setting may be lower than that in previous RCTs. Our innovative method, particularly in Japan, to analyze adherence using large claims data should change the way clinical epidemiological research and health policies of rare diseases are designed in the future.
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spelling pubmed-95429782022-10-11 Persistence of tolvaptan medication for autosomal dominant polycystic kidney disease: A retrospective cohort study using Shizuoka Kokuho Database Saito, Ryuta Yamamoto, Hiroyuki Ichihara, Nao Kumamaru, Hiraku Nishimura, Shiori Shimada, Koki Mori, Kiyoshi Miyachi, Yoshiki Miyata, Hiroaki Medicine (Baltimore) 4400 Autosomal dominant polycystic kidney disease (ADPKD) is a rare hereditary disease leading to end-stage renal failure in approximately half of patients by seventy years of age. It is important to continuously take tolvaptan to control disease progression. However, adherence to tolvaptan in a real-world setting, rather than randomized controlled trials (RCTs), has not been sufficiently reported. We aimed to investigate tolvaptan persistence among patients with ADPKD using a large claims database. Using the Shizuoka Kokuho Database, we identified patients diagnosed with ADPKD who were prescribed tolvaptan from March 2014–September 2018 in Japan. The persistence rate of tolvaptan medication was estimated by Kaplan–Meier analysis, and patient background factors associated with treatment discontinuation were exploratively evaluated with log-rank tests. We identified 1714 eligible patients with ADPKD, and among them, 25 patients used tolvaptan medication. We followed up these patients, whose median treatment duration was 21 months. The persistence rates at 12, 24, and 36 months were estimated to be 70.8% (95% confidence interval: 48.2–93.4), 46.5% (23.2–66.9), and 38.7% (16.4–60.8), respectively. In the exploratory analysis, there were no factors that were obviously associated with tolvaptan discontinuation. The persistence rate of tolvaptan in patients with ADPKD in a real-world setting may be lower than that in previous RCTs. Our innovative method, particularly in Japan, to analyze adherence using large claims data should change the way clinical epidemiological research and health policies of rare diseases are designed in the future. Lippincott Williams & Wilkins 2022-10-07 /pmc/articles/PMC9542978/ /pubmed/36221375 http://dx.doi.org/10.1097/MD.0000000000030923 Text en Copyright © 2022 the Author(s). Published by Wolters Kluwer Health, Inc. https://creativecommons.org/licenses/by-nc/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution-Non Commercial License 4.0 (CCBY-NC) (https://creativecommons.org/licenses/by-nc/4.0/) , where it is permissible to download, share, remix, transform, and buildup the work provided it is properly cited. The work cannot be used commercially without permission from the journal.
spellingShingle 4400
Saito, Ryuta
Yamamoto, Hiroyuki
Ichihara, Nao
Kumamaru, Hiraku
Nishimura, Shiori
Shimada, Koki
Mori, Kiyoshi
Miyachi, Yoshiki
Miyata, Hiroaki
Persistence of tolvaptan medication for autosomal dominant polycystic kidney disease: A retrospective cohort study using Shizuoka Kokuho Database
title Persistence of tolvaptan medication for autosomal dominant polycystic kidney disease: A retrospective cohort study using Shizuoka Kokuho Database
title_full Persistence of tolvaptan medication for autosomal dominant polycystic kidney disease: A retrospective cohort study using Shizuoka Kokuho Database
title_fullStr Persistence of tolvaptan medication for autosomal dominant polycystic kidney disease: A retrospective cohort study using Shizuoka Kokuho Database
title_full_unstemmed Persistence of tolvaptan medication for autosomal dominant polycystic kidney disease: A retrospective cohort study using Shizuoka Kokuho Database
title_short Persistence of tolvaptan medication for autosomal dominant polycystic kidney disease: A retrospective cohort study using Shizuoka Kokuho Database
title_sort persistence of tolvaptan medication for autosomal dominant polycystic kidney disease: a retrospective cohort study using shizuoka kokuho database
topic 4400
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9542978/
https://www.ncbi.nlm.nih.gov/pubmed/36221375
http://dx.doi.org/10.1097/MD.0000000000030923
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