期刊
CLINICAL KIDNEY JOURNAL
卷 11, 期 1, 页码 62-69出版社
OXFORD UNIV PRESS
DOI: 10.1093/ckj/sfx071
关键词
autosomal dominant polycystic kidney disease; patient selection; PROPKD score; rapid progression; tolvaptan; total kidney volume
资金
- Medical Research Council [MR/M012212/1]
- Newcastle Upon Tyne Hospitals NHS Charity
- Northern Counties Kidney Research Fund
- Kidney Research UK
- Nachwuchsgruppen
- NRW program of the Ministry of Science Northrine-Westfalia (MIWF)
- German Kidney Foundation (Deutsche Nierenstiftung)
- Deutsche Forschungsgemeinschaft [MU 3629/2-1]
- Otsuka Pharmaceutical Europe Limited
- Medical Research Council [MR/M012212/1] Funding Source: researchfish
- MRC [MR/M012212/1] Funding Source: UKRI
Background: Autosomal dominant polycystic kidney disease (ADPKD) is the most common inherited kidney disease worldwide. The renal phenotype is characterized by progressive cystic enlargement of the kidneys leading to a decline in renal function, hypertension and often end-stage renal disease (ESRD). Supportive care with blood pressure control and management of pain, urinary infections and renal stone disease has, until recently, been the mainstay of treatment. With the recent approval of tolvaptan for use in ADPKD, the disease progression may now be targeted specifically. Algorithms that guide treatment initiation have been proposed but a more pragmatic and patient-individualized approach is often needed to make decisions regarding therapy. It is highly important to identify ADPKD patients with rapidly progressive disease who are likely to benefit most from this treatment and avoid treatment in patients that are unlikely to reach ESRD. Methods and Results: Here we present a series of cases of ADPKD patients in whom therapy with tolvaptan has been considered and report the rationale for the treatment decisions based on available lifestyle, clinical, biochemical, radiological and genetic data. Conclusions: These cases provide a discussion for the use of tolvaptan in ADPKD within the nephrology clinic and allow insights into the practicalities of using this therapy outside of clinical trials.
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