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Abstract: SA-PO495

Patient-Reported Disease Burden and Urinary Impact in Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Session Information

  • ADPKD: Clinical Studies
    October 27, 2018 | Location: Exhibit Hall, San Diego Convention Center
    Abstract Time: 10:00 AM - 12:00 PM

Category: Genetic Diseases of the Kidney

  • 1001 Genetic Diseases of the Kidney: Cystic


  • Oberdhan, Dorothee, Otsuka, Rockville, Maryland, United States
  • Sanon, Myrlene, Otsuka, Rockville, Maryland, United States
  • Agarwal, Indra, Otsuka, Rockville, Maryland, United States
  • Czerwiec, Frank S., Otsuka, Rockville, Maryland, United States
  • Perrone, Ronald D., Tufts Medical Center, Boston, Massachusetts, United States

ADPKD is an inherited disease leading to kidney enlargement, worsening of kidney function, and quality of life impacts. ADPKD patients experience symptoms consistent with urine concentration deficits, such as polyuria, nocturia, urinary frequency and urgency. Combined with tolvaptan (TLV) therapy those symptoms may be accentuated.


In a randomized, placebo-controlled, double-blind clinical trial (NCT01451827) urinary burden, and health-related quality of life (HRQoL) were assessed over 8 weeks. Urinary burden was measured by number of voids and 24h urine volume. HRQoL measures included the ADPKD Impact Scale (ADPKD-IS), ADPKD Urinary Impact Scale (ADPKD-UIS), and SF-12v2 Physical and Mental Component Summary (PCS and MCS).


A total of 177 subjects enrolled in the clinical trial. 134 subjects were randomized to various doses of TLV in several formulations (50-120mg/day, pooled) and 42 subjects to placebo (PLC). Mean number of voids were similar for TLV and PLC subjects at baseline (daytime: 6.4 vs 6.2; nighttime: 1.5 vs 1.6) but increased in the TLV group by Week 8 (daytime: 10.1 vs 6.9, p=0.0002; nighttime: 2.4 vs 1.5, p≤0.00001). Mean 24h urine volume at baseline was similar between TLV and PLC subjects (2.3L vs 2.4L) but increased in the TLV group through Week 8 (6.1L vs 2.9L, p<0.0001). Compliance was 98% for TLV vs 97% for PLC.


Baseline urinary burden is low with subjects reporting only minimal bother/impact during daytime and slightly higher burden associated with nocturia. With tolvaptan treatment, the urine volume doubled and number of voids increased by over 50% with intermediate increases in patient-reported urinary burden during daytime and nighttime. Overall HRQoL showed no noticeable change during the study duration even with increased urinary burden.

Mean HRQoL
BaselineWeek 8BaselineWeek 8


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