Abstract: SA-PO0121
Real-World Experience of Combination Octreotide and Tolvaptan for Symptomatic Polycystic Liver and Kidney Disease
Session Information
- ADPKD and Cystic Kidney Disease - 3
October 24, 2026 | Location: Exhibit Hall A, Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Genetic Diseases of the Kidneys
- 1201 Genetic Diseases of the Kidneys: Cystic (Monogenic)
Authors
- Arriola Montenegro, Jose J., Mayo Clinic Minnesota, Rochester, Minnesota, United States
- Dahlen, Erin, Mayo Clinic Minnesota, Rochester, Minnesota, United States
- Mueller, Theodore Leo, Mayo Clinic Minnesota, Rochester, Minnesota, United States
- Zoghby, Ziad, Mayo Clinic Minnesota, Rochester, Minnesota, United States
- Torres, Vicente E., Mayo Clinic Minnesota, Rochester, Minnesota, United States
- Hogan, Marie C., Mayo Clinic Minnesota, Rochester, Minnesota, United States
Background
Approx. 5–10% ADPKD patients develop symptomatic polycystic liver disease (PLD) with no FDA-approved therapy. KDIGO recommends tolvaptan (T) for rapidly progressive ADPKD (MIC1C–1E) & somatostatin analogues (SSAs) for markedly enlarged polycystic livers with severe volume-related symptoms (1B). A KDIGO practice point notes SSAs can be considered for massively enlarged kidneys when no better options are available. Preclinical data suggest enhanced efficacy of combination therapy through complementary cAMP suppression, yet real-world data on combined use are lacking.
Methods
We identified all patients on concurrent octreotide LAR (OctLAR) & T from T FDA approval (4/ 2018- 5/2026). TLV, TKV, (MRI,CT), eGFR were collected (median;range). Adverse events (AEs), liver function, & discontinuation rates were monitored using an EHR-based application.
Results
Of patients in our therapy monitoring registry, 127 currently receive T & 26 OctLAR; 5 (3.9% of T-treated; 19.2% of OctLAR-treated patients) receive combination therapy, median 48 yrs (range 43–77); 80% F; MIC 1C (n=4), 1D (n=1). Four received OctLAR 40mg q28 days IM; one required dose reduction to 20 mg for gastrointestinal intolerance. T was tolerated without side effects in all. Baseline TLV 3741 mL (2774–4470); TKV 1712 mL (982–5622). Median combination therapy duration was 24 mos (11–39). TLV change ranged from −30.4% to +9.0%; 3/5 patients (60%) achieved TLV reduction (median −2.6%). TKV change ranged from −46.1% to +10.4%; 4/5 (80%) achieved TKV reduction (median −15.3%). Two demonstrated improved eGFR (+1.8 and +2.8 mL/min/yr); 3 declined (−1.3 to −14.5 mL/min/yr). Four underwent 8 adjunctive sclerotherapy sessions treating 37 cysts, which may have contributed to volume reductions independent of pharmacotherapy. No discontinuations or liver function perturbations were observed, notable given T's hepatotoxicity risk and theoretical concern for additive hepatotoxicity with dual therapy.
Conclusion
Combination OctLAR & T was well tolerated. Volume stabilization or reduction was observed in the majority, though the independent contribution of pharmacotherapy vs. adjunctive sclerotherapy cannot be determined in this cohort. These findings support feasibility & tolerability of multimodal management for ADPKD patients with symptomatic PLD. Prospective studies are warranted to delineate the contributions of each therapeutic modality.
Funding
- Private Foundation Support