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Kidney Week

Abstract: TH-PO833

How Well Do Risk Assessment Guidelines Perform for Autosomal Dominant Polycystic Kidney Disease (ADPKD)?

Session Information

Category: Genetic Diseases of the Kidneys

  • 1001 Genetic Diseases of the Kidneys: Cystic

Authors

  • Ahmed, Syed Essam, Toronto General Hospital UHN, Toronto, Ontario, Canada
  • Akbari, Pedram, Toronto General Hospital UHN, Toronto, Ontario, Canada
  • Guiard, Elsa, Toronto General Hospital UHN, Toronto, Ontario, Canada
  • Quist, Crystal F., Toronto General Hospital UHN, Toronto, Ontario, Canada
  • Shi, Beili, Toronto General Hospital UHN, Toronto, Ontario, Canada
  • Iliuta, Ioan-Andrei, University Health Network and University of Toronto, Toronto, Ontario, Canada
  • Nasri, Fatemeh, Toronto General Hospital UHN, Toronto, Ontario, Canada
  • Khalili, Korosh, University Health Network and University of Toronto, Toronto, Ontario, Canada
  • Pei, York P., University Health Network and University of Toronto, Toronto, Ontario, Canada
Background

The approval of Tolvaptan for treatment of ADPKD heralds a new era when mechanism-based therapy is now possible. However, Tolvaptan is an expensive drug that is associated with potentially serious side-effects. Thus, it is currently reserved for patients who are at high-risk for progression. Two sets of risk assessment guidelines for ADPKD are now available based on the consensus of two panels of nephrologists from Canada and Europe. However, how well do these guidelines perform in risk assessment has not been formally assessed.

Methods

We conducted a prospective study in 474 patients with typical imaging pattern of ADPKD by MRI who also had detailed clinical and laboratory data including total kidney volume (TKV). We used age- and height-adjusted TKV to derive Mayo Clinic Imaging Class as a “gold-standard” for risk assessment (i.e. low-risk: 1A-1B; high-risk: 1C-1E). We then applied the revised Canadian guidelines (Can J Kidney Health Dis. 2018; 5:2054358118801589) and European guidelines (NDT 2016; 31: 337-348) to our patient cohort to assess their performance.

Results

Applying the updated Canadian risk assessment algorithm resulted in exclusion of 52% (245/474) of patients in whom 65% were deemed to be low-risk (i.e. MCIC 1A-1B) and 35%, high-risk (MCIC 1C-1E). The resultant cohort (221/503) was enriched with 88% high-risk patients but also included 12% of low-risk patients. The European guidelines provide a 5-step hierarchal algorithm. Applying the first step based on age and CKD stages resulted in exclusion of 74% (351/474) of patients in whom 69% were deemed to be high-risk and 31%, low-risk. The resultant cohort (123/474) was enriched with 72% high-risk patients but also included 28% of low-risk patients. Applying the second step based on rate of eGFR decline resulted in a total exclusion of 93% (440/474) of patients in whom 59% were deemed to be high-risk and 41%, low-risk. The resultant cohort (34/474) was enriched with 82% high-risk patients but also included 18% of low-risk patients.

Conclusion

Risk assessment in ADKPD is an evolving process to be refined by new clinical data and test technologies. Guidelines that enrich “high-risk”, while minimizing “low-risk”, patients, have most clinical utility.

Funding

  • Government Support - Non-U.S.