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Kidney Week

Abstract: TH-PO848

Sotradecol Foam Sclerotherapy for Treatment of Symptomatic Cysts in ADPKD and Autosomal Dominant Polycystic Liver Disease

Session Information

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.

EndpointBaseline
Mean (SD)
Change from Baseline to Month 6p-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