Abstract: TH-PO848
Sotradecol Foam Sclerotherapy for Treatment of Symptomatic Cysts in ADPKD and Autosomal Dominant Polycystic Liver Disease
Session Information
- Cystic Kidney Diseases: Clinical
November 07, 2019 | Location: Exhibit Hall, Walter E. Washington Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Genetic Diseases of the Kidneys
- 1001 Genetic Diseases of the Kidneys: Cystic
Authors
- Martin, William P., St. Vincent's University Hospital, Dublin 4, Ireland
- Neidert, Newton, Mayo Clinic, Rochester, Minnesota, United States
- Bendel, Emily, Mayo Clinic, Rochester, Minnesota, United States
- Edwards, Marie E., Mayo Clinic, Rochester, Minnesota, United States
- Chebib, Fouad T., Mayo Clinic, Rochester, Minnesota, United States
- Novotny, Paul, Mayo Clinic, Rochester, Minnesota, United States
- Kremers, Walter K., Mayo Clinic, Rochester, Minnesota, United States
- Torres, Vicente E., Mayo Clinic, Rochester, Minnesota, United States
- Hogan, Marie C., Mayo Clinic, Rochester, Minnesota, United States
Background
Patients frequently describe mass symptoms & reduced quality of life (QoL) that correlate with visible large liver/kidney cysts in ADPKD/ ADPLD. Since 1/18/2017 we have used cyst drainage followed by sotradecol foam sclerotherapy (SFS) to treat symptomatic, large (≥5 cm in diameter) cysts. Small volumes (20cc max) of sotradecol sclerosant admixed with air are injected under fluoroscopy to ablate the epithelial cyst lining. We studied its safety & impact on QoL & organ volumes.
Methods
In this single-center, single-arm, prospective observational study, ADPKD and ADPLD patients with compressive symptoms due to dominant (liver or kidney) cysts are referred for SFS with 3% sotradecol performed under local anesthesia. QoL using linear analog scale assessment tool (LASA), SF-12, the polycystic liver disease QoL tool (PLD-Q), equivalent opioid dose (mg/24hr), & organ volumes (planimetry using CT/MR) are recorded at baseline & 6 months post-SFS. Changes over time were tested using Wilcoxon tests and confirmed using repeated measures mixed models. Improvements >0.5 SD were considered clinically meaningful.
Results
45 patients (mean age 55yr, 84% female) are enrolled: 12 (27%) with ADPKD, 31 (69%) with ADPLD, & 2 (4%) with cystic disease NOS. 31 (69%) & 14 (31%) underwent first SFS for symptomatic liver & kidney cysts, respectively. 56 SFS procedures (mean 1.24 per patient) have been performed to treat 68 cysts (mean 1.51 per patient). Total PLD-Q, overall QoL, physical well-being, bodily pain, & vitality improved at month 6 (Table). Non-significant reductions in organ volumes seen at 6 months is likely due to small numbers. Longer term follow-up to 12 months is ongoing.
Conclusion
SFS directed at symptomatic large cysts was well tolerated, improved QoL at 6 months, & decreased early satiety, SOB, pain & fullness. Smaller volume instillations of SFS have replaced alcohol sclerotherapy in our practice and are a safe option for directed therapy of symptomatic large cysts in ADPKD and ADPLD.
| Endpoint | Baseline Mean (SD) | Change from Baseline to Month 6 | p-value (Wilcoxon) | ||
| Liver Volume (mL) (n=9) | 4587.8 (2136.3) | -866.2 (1713.9) | 0.1679 | ||
| Kidney Volume (mL) (n=7) | 2198.9 (3937.3) | -663.6 (951.1) | 0.1144 | ||
| PLD-Q (n=20) | 60.1 (18.3) | -15.5 (19.7) | 0.0023 | ||
| LASA Overall QoL (n=28) | 6.3 (2.3) | 1.1 (2.4) | 0.0258 | ||
| LASA Physical Wellbeing (n=28) | 5.3 (2.1) | 1.3 (2.2) | 0.0053 | ||
| SF-12 Vitality (n=29) | 44.2 (31.7) | 12.9 (31.8) | 0.0369 | ||
| SF-12 Bodily Pain (n=27) | 53.0 (31.2) | 16.7 (27.7) | 0.0044 | ||
| SF-12 General Health (n=29) | 48.6 (26.5) | 10.5 (30.6) | 0.0750 | ||
| Equivalent Opioid Dose (mg/24h) (n=26) | 4.5 (12.1) | -1.5 (6.1) | 0.2196 | ||
Funding
- Private Foundation Support