Clinical Decision-Making About Immunosuppressive Treatment in Focal Segmental Glomerulosclerosis

Abstract

Rationale & Objective

Focal segmental glomerulosclerosis (FSGS) is a heterogeneous disorder with a high risk of progression to kidney failure. There are no approved therapies for FSGS, and futility of treatment is poorly defined. The Cure Glomerulonephropathy (CureGN) study offers the opportunity to describe the characteristics of participants who started immunosuppressive therapy (IST), never received IST, or in whom this treatment was discontinued.

Study Design

An observational cohort.

Settings & Participants

Participants enrolled in CureGN with FSGS and surveyed nephrologists.

Interventions

The clinical and laboratory data from participants with FSGS who were enrolled in the CureGN observational cohort were reviewed to define features associated with withholding initial IST or terminating ongoing IST. Nephrologists were surveyed about what factors would influence their decision to prescribe or withdraw IST in patients with FSGS.

Outcomes

(1) Identify factors associated with IST initiation and discontinuation in individuals with FSGS; and (2) Identify clinical and laboratory features nephrologists consider when they recommend against the use of IST at diagnosis (initiation of care) and during the course of disease.

Results

Based on quantitative findings from the CureGN cohort and survey responses from practicing nephrologists, a low estimated glomerular filtration rate at presentation, significant glomerulosclerosis, and interstitial fibrosis and tubular atrophy on kidney biopsy make initiation of IST less likely.

Limitations

Heterogeneous nature of the cohort and an inability to divide the patients into KDIGO subgroups of FSGS. Rationale for decision to stop or defer treatment was not available. More surveys were completed by pediatric providers, and the majority were completed by academic practitioners.

Conclusions

The factors that impact decisions about IST initiation and discontinuation were consistent among pediatric and internal medicine nephrologists, namely advanced scarring and lower estimated glomerular filtration rate. We suggest that this information should be incorporated into patient management guidelines and clinical trial design.

Plain Language Summary

Patients with focal segmental glomerulosclerosis (FSGS) are at high risk for progression to kidney failure and there are no approved therapies. This study from the CureGN consortium described clinical situations in which immunosuppressive therapy (IST) was started, not started, or discontinued in participants with FSGS. Furthermore, we surveyed nephrologists to better understand factors that influence the management of patients with FSGS. Participants in the CureGN cohort with a lower estimated glomerular filtration rate and advanced scarring on kidney biopsy were less likely to be started on IST and have this treatment discontinued, which was similar to what surveyed providers did in practice. Understanding the characteristics of these patients may help further develop management guidelines and assist in clinical trial design.