Kidney Week

Abstract: SA-OR73

Comparative Effectiveness of Immunosuppressive Medications in Nephrotic Syndrome: A Cure Glomerulonephropathy (CureGN) Study Report

Session Information

• Pediatric Nephrology: Insights and Innovations
  October 26, 2024 | Location: Room 23, Convention Center
  Abstract Time: 04:30 PM - 04:40 PM

Category: Pediatric Nephrology

• 1900 Pediatric Nephrology

Authors

• Robinson, Cal, The Hospital for Sick Children, Toronto, Ontario, Canada

Cal Robinson, MBChB

• Canetta, Pietro A., Columbia University Irving Medical Center, New York, New York, United States

Pietro A. Canetta, MD, MS

• Cara-Fuentes, Gabriel M., Nationwide Children's Hospital, Columbus, Ohio, United States

Gabriel M. Cara-Fuentes, MD, PhD

• Greenbaum, Larry A., Emory University, Atlanta, Georgia, United States

Larry A. Greenbaum, MD, PhD

• Helmuth, Margaret, University of Michigan, Ann Arbor, Michigan, United States

Margaret Helmuth, MA

• Hladunewich, Michelle A., University of Toronto, Toronto, Ontario, Canada

Michelle A. Hladunewich, MD, MS, FASN

• Kidd, Jason M., Virginia Commonwealth University, Richmond, Virginia, United States

Jason M. Kidd, MD, FASN

• Larkina, Maria, University of Michigan, Ann Arbor, Michigan, United States

Maria Larkina, MS

• Reidy, Kimberly J., Children's Hospital at Montefiore, New York, New York, United States

Kimberly J. Reidy, MD

• Parekh, Rulan S., Women's College Hospital, Toronto, Ontario, Canada

Rulan S. Parekh, MD, MS, FASN

Background

Rituximab is increasingly used to treat frequently-relapsing nephrotic syndrome in children and young adults. Yet, the real-world comparative effectiveness of immunosuppressive medications is unclear.

Methods

Using target trial methods, we emulated a multi-center, open-label, pragmatic randomized controlled trial with CureGN data. We included children and young adults (<40 years) diagnosed with minimal change disease (MCD) or focal segmental glomerulosclerosis (FSGS) that had prior complete remission and initiated rituximab, mycophenolate mofetil (MMF), or calcineurin inhibitors (CNI). Randomization was emulated by propensity score overlap weighting. The primary outcome was time-to-relapse using weighted Cox proportional hazards models.

Results

Of 221 eligible CureGN participants, 111 initiated rituximab, 46 MMF, and 64 CNI. Baseline characteristics were balanced after propensity score weighting. Mean age was 13.3 years and 80% had prior steroid-sparing drug use. During median 4.3-year (IQR 1.9-6.1) follow-up, relapse occurred in 56%, 46%, and 58% after rituximab, MMF, and CNI use, respectively. There was no difference in relapses after rituximab vs. MMF or CNI (weighted HR 1.16, 95%CI 0.74-1.81). There were also no differences in relapse rates, kidney function decline, or adverse events.

Conclusion

There was no difference in relapse risk after rituximab vs. MMF or CNI among children and young adults with difficult-to-treat nephrotic syndrome. Therapeutic selection should be a shared decision, considering medication-specific side-effects, costs, access, duration, and patient adherence.

Figure. Weighted relapse-free survival after rituximab vs. MMF or CNI use

Funding

• Government Support – Non-U.S.