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Abstract: PO1197

Foam Sclerotherapy for Directed Treatment of Symptomatic Cysts in Autosomal Dominant Polycystic Kidney and Liver Disease

Session Information

Category: Genetic Diseases of the Kidneys

  • 1001 Genetic Diseases of the Kidneys: Cystic

Authors

  • Hogan, Marie C., Mayo Foundation for Medical Education and Research, Rochester, Minnesota, United States
  • Martin, William P., University College Dublin, Dublin, Ireland
  • Bendel, Emily, Mayo Foundation for Medical Education and Research, Rochester, Minnesota, United States
  • Mara, Kristin C., Mayo Foundation for Medical Education and Research, Rochester, Minnesota, United States
  • Gregory, Adriana, Mayo Foundation for Medical Education and Research, Rochester, Minnesota, United States
  • Chong, Grace Yun, Mayo Foundation for Medical Education and Research, Rochester, Minnesota, United States
  • Torres, Vicente E., Mayo Foundation for Medical Education and Research, Rochester, Minnesota, United States
  • Neidert, Newton, Mayo Foundation for Medical Education and Research, Rochester, Minnesota, United States
Background

Patients frequently describe mass symptoms & reduced quality of life (QoL) that correlate with large (≥5cm diameter) organ cysts in ADPKD/ADPLD. Since 1/18/2017, we have studied the safety & impact of cyst drainage followed by sotradecol foam sclerotherapy (SFS) to treat symptomatic, large cysts.

Methods

In this single-center, single-arm, prospective observational study, ADPKD and ADPLD patients with compressive symptoms due to liver or kidney cysts are referred for SFS. Small volumes (20cc max) of 3% sotradecol sclerosant admixed with air are injected (fluoroscopy-guided under local anesthesia) to ablate the epithelial cyst lining. QoL measures using polycystic liver disease QoL tool (PLD-Q) & organ volumes (planimetry using CT/MR) are recorded at baseline & 12+ 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

We performed 148 SFS procedures among 68 cases (mean age 55yr 77% Female, mean 2.2 procedures per pt): 53 (77.9%) with ADPKD/ADPLD, 5 (7.4%) with ADPLD, & 10 (14.7%) with cystic disease NOS. PLD-Q scores improved by 7.7 (IQR 0.1, 24.8) (p=0.012) at mo 12. A subgroup (13 patients) (mean age 54.7yr, 69% F) have undergone multiple sequential SFS procedures: 10 (77%) with ADPKD/ADPLD, 2 (15%) ADPLD, & 1 (8%) cystic disease NOS. Among 5 with multiple kidney procedures, median kidney volume decreased by 135mL/yr. Among 5 with multiple liver procedures, median liver volume decreased by 114mL/yr. SFS was well tolerated with low complication rates.

Conclusion

SFS directed at large, symptomatic liver and kidney cysts was well tolerated, improved QoL at 12 months, & decreased early satiety, SOB, pain & fullness. Furthermore, multiple sequential SFS procedures are feasible, efficacious, and have an acceptable safety profile.

ADPKD Case Who Underwent 5 sequential SFS Procedures

Funding

  • NIDDK Support