Real-life use of tolvaptan in ADPKD: a retrospective analysis of a large Canadian cohort

被引:7
作者
Calvaruso, Luca [1 ,2 ,3 ]
Yau, Kevin [1 ,2 ]
Akbari, Pedram [1 ,2 ]
Nasri, Fatemah [4 ]
Khowaja, Saima [1 ,2 ]
Wang, Bill [5 ]
Haghighi, Amirreza [1 ,2 ,6 ,7 ]
Khalili, Korosh [4 ]
Pei, York [1 ,2 ]
机构
[1] Univ Hlth Network, Dept Med, Div Nephrol, Toronto, ON, Canada
[2] Univ Toronto, Toronto, ON, Canada
[3] IRCCS, Fdn Policlin Univ A Gemelli, UOC Nefrol, Rome, Italy
[4] Univ Toronto, Univ Hlth Network, Dept Med Imaging, Toronto, ON, Canada
[5] Patient Liaison Advisory Grp Int Soc Nephrol, Hong Kong, Peoples R China
[6] Brigham & Womens Hosp, Div Genet, Boston, MA USA
[7] Harvard Med Sch, Boston, MA USA
来源
SCIENTIFIC REPORTS | 2023年 / 13卷 / 01期
基金
加拿大健康研究院;
关键词
POLYCYSTIC KIDNEY-DISEASE; PROGRESSION; DIAGNOSIS; METFORMIN; MODEL;
D O I
10.1038/s41598-023-48638-9
中图分类号
O [数理科学和化学]; P [天文学、地球科学]; Q [生物科学]; N [自然科学总论];
学科分类号
07 ; 0710 ; 09 ;
摘要
Tolvaptan is the first disease-modifying drug proven to slow eGFR decline in high-risk patients with ADPKD. However, barriers from the patient perspective to its use in real-life settings have not been systemically examined in a large cohort. This was a single-center, retrospective study of 523 existing or new patients with ADPKD followed at the Center for Innovative Management of PKD in Toronto, Ontario, between January 1, 2016 to December 31, 2018. All patients underwent clinical assessment including total kidney volume measurements and Mayo Clinic Imaging Class (MCIC). Those who were deemed to be at high risk were offered tolvaptan with their preference (yes or no) and reasons for their choices recorded. Overall, 315/523 (60%) patients had MCIC 1C-1E; however, only 96 (30%) of them were treated with tolvaptan at their last follow-up. Among these high-risk patients, those not treated versus treated with tolvaptan were more likely to have a higher eGFR (82 +/- 26 vs. 61 +/- 27 ml/min/1.73 m2), CKD stages 1-2 (79% vs. 41%), and MCIC 1C (63% vs. 31%). The most common reasons provided for not taking tolvaptan were lifestyle preference related to the aquaretic effect (51%), older age >= 60 (12%), and pregnancy/family planning (6%). In this real-world experience, at least 60% of patients with ADPKD considered to be at high risk for progression to ESKD by imaging were not treated with tolvaptan; most of them had early stages of CKD with well-preserved eGFR and as such, were prime targets for tolvaptan therapy to slow disease progression. Given that the most common reason for tolvaptan refusal was the concern for intolerability of the aquaretic side-effect, strategies to mitigate this may help to reduce this barrier to tolvaptan therapy.
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页数:8
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