Kidney Week

Abstract: PO1529

Natural History of Focal Segmental Glomerulosclerosis (FSGS): The UK National RaDaR Idiopathic Nephrotic Syndrome Cohort

Session Information

• Glomerular Diseases: Clinical Features and Outcomes in Nephrotic Syndromes and Complement-Mediated Diseases
  November 04, 2021 | Location: On-Demand, Virtual Only
  Abstract Time: 10:00 AM - 12:00 PM

Category: Glomerular Diseases

• 1203 Glomerular Diseases: Clinical, Outcomes, and Trials

Authors

• Saleem, Moin, University of Bristol, Bristol, Bristol, United Kingdom

Moin Saleem, MD, MBBS, PhD

• Barratt, Jonathan, University of Leicester, Leicester, Leicestershire, United Kingdom

Jonathan Barratt,

• Braddon, Fiona E. M., UK Renal Registry, Bristol, Bristol, United Kingdom

Fiona E. M. Braddon,

• Carroll, Kevin, KLC Statistics Ltd, Cheshire, England, United Kingdom

Kevin Carroll,

• Diva, Ulysses A., Travere Therapeutics Inc, San Diego, California, United States

Ulysses A. Diva,

• Afzal, Maryam, University of Bristol, Bristol, Bristol, United Kingdom

Maryam Afzal,

• Hendry, Bruce M., Travere Therapeutics Inc, San Diego, California, United States

Bruce M. Hendry,

• Mercer, Alex, JAMCO Pharma Consulting, Stockholm, Sweden

Alex Mercer,

• Pitcher, David, UK Renal Registry, Bristol, Bristol, United Kingdom

David Pitcher,

• Steenkamp, Retha D., UK Renal Registry, Bristol, Bristol, United Kingdom

Retha D. Steenkamp,

• Turner, A. Neil, University of Edinburgh, Edinburgh, Edinburgh, United Kingdom

A. Neil Turner,

• Gale, Daniel P., Royal Free Hospital, London, London, United Kingdom

Daniel P. Gale,

Background

Idiopathic FSGS is an important cause of proteinuric renal disease leading to ESKD. Here we describe the natural history of FSGS using the UK National Registry of Rare Kidney Diseases Idiopathic Nephrotic Syndrome (RaDaR-INS) Cohort, including retrospective and prospective data from 3907 patients with nephrotic syndrome (NS) not attributable to glomerulonephritis or systemic disorders recruited from 107 adult and paediatric kidney units across the UK since 2010.

Methods

Participants included those with biopsy-proven or monogenic FSGS and ≥12 mo. observation from baseline. Patients with ESKD (CKD stage 5 or on renal replacement therapy) at or prior to baseline were excluded. Baseline date was defined as first database occurrence of renal biopsy, primary renal diagnosis, NS symptoms, PCR ≥1 g/g, or initiation of immunosuppression. Renal survival was defined as absence of ESKD or death with survival time calculated from baseline to last follow-up.

Results

Of 786 FSGS patients meeting eligibility, median baseline age was 28 yrs (IQR 9-49) with paediatric patients representing 38% of the study population. Median proteinuria at baseline was 5.9 g/g (IQR 3.3-11.0; n=140), while mean eGFR was 163 mL/min/1.73m² (SD 47; n=69) and 71 mL/min/1.73m² (SD 32; n=103) for children and adults, respectively. Median follow-up duration was 9.7 yrs (IQR 5.7-16.1) with ESKD/death events occurring in 46% of patients (1% death). Kaplan-Meier survival curves of children and adults show 50% renal survival probability of 16 years & 12 years, respectively (Figure 1).

Conclusion

The RaDaR-INS Cohort represents a large study population with lengthy follow-up data. These analyses indicate rapid progression and poor outcomes, highlighting a need for effective treatments for patients with FSGS.

Funding

• Commercial Support – Travere Therapeutics