Kidney Week

Abstract: TH-PO1191

Comparison of Patients with Primary Hyperoxaluria 1 and Late-Stage CKD from the BONAPH1DE Registry and ILLUMINATE-C Lumasiran Trial

Session Information

• Late-Breaking Research Posters
  November 06, 2025 | Location: Exhibit Hall, Convention Center
  Abstract Time: 10:00 AM - 12:00 PM

Category: CKD (Non-Dialysis)

• 2302 CKD (Non-Dialysis): Clinical, Outcomes, and Trials

Authors

• Lieske, John C., Mayo Clinic, Rochester, Minnesota, United States

John C. Lieske, MD, FASN

• Baum, Michelle Amy, Boston Children's Hospital, Boston, Massachusetts, United States

Michelle Amy Baum, MD

• Benshalom, Efrat, Shaare Zedek Medical Center, Jerusalem, Israel

Efrat Benshalom, MD

• Belostotsky, Vladimir, McMaster University, Hamilton, Ontario, Canada

Vladimir Belostotsky, MD, PhD

• Knauf, Felix, Charité Universitätsmedizin Berlin, Berlin, Germany

Felix Knauf, MD

• Sellier-Leclerc, Anne-Laure All, Hôpital Femme Mère Enfant en Centre d’Investigation Clinique INSERM, Hospices Civils de Lyon, ERKnet, Bron, France

Anne-Laure All Sellier-Leclerc, MD

• Du, Weiming, Alnylam Pharmaceuticals, Cambridge, Massachusetts, United States

Weiming Du, MS

• Callanan, Mary, Alnylam Pharmaceuticals, Cambridge, Massachusetts, United States

Mary Callanan

• Kauf, Teresa, Alnylam Pharmaceuticals, Cambridge, Massachusetts, United States

Teresa Kauf, PhD

• Murphy, Michael Desmond, Alnylam Pharmaceuticals, Cambridge, Massachusetts, United States

Michael Desmond Murphy, PharmD, RPh

• Groothoff, Jaap, Emma Children's Hospital, Amsterdam, Netherlands

Jaap Groothoff, MD, PhD

Background

Patients (pts) with primary hyperoxaluria type 1 (PH1) and late-stage chronic kidney disease (CKD) from the real-world observational BONAPH1DE study (BPH1; NCT04982393) and Phase 3 ILLUMINATE-C trial (ILLUM-C; NCT04152200) were reviewed to assess if pt characteristics and outcomes in these real-world and clinical studies are concordant.

Methods

BPH1 (N=174; all ages; any stage CKD) included 19 lumasiran ever-treated pts on dialysis and 11 not on dialysis with BL eGFR ≤45 mL/min/1.73m². ILLUM-C (N=21; all ages; late-stage CKD) included 15 pts on dialysis and 6 not on dialysis with BL eGFR ≤45; all initiated lumasiran. Outcomes after isolated kidney transplant (iKT) in a subset of 7 BPH1 pts (lumasiran-treated at iKT; ≥1 year follow-up; ≥1 post-iKT POx and eGFR value) were compared with 5 similar ILLUM-C pts.

Results

In BPH1, pts not on dialysis had a mean (SD) BL eGFR of 18.9 (15.5) and BL POx of 110.5 (108.0) µmol/L; those on dialysis had a mean (SD) BL POx of 185.4 (348.6). Before lumasiran treatment, ILLUM-C pts with BL eGFR ≤45 and not on dialysis had a mean (SD) BL eGFR of 19.8 (9.6) and BL POx of 64.7 (41.3); those on dialysis had a mean (SD) BL POx of 108.4 (29.5; Figure 1). Overall, among lumasiran-treated pts in both studies who underwent iKT, eGFR increased and POx declined post-iKT. In ILLUM-C, POx was already reduced from BL at the time of iKT (19.2-64.5) and declined further post-iKT.

Conclusion

BPH1 and ILLUM-C data showed comparable POx reduction, eGFR improvement, and iKT outcomes with lumasiran treatment in both real-world and clinical trial studies.

Funding

• Commercial Support – Alnylam Pharmaceuticals

Digital Object Identifier (DOI)

• doi: 10.1681/ASN.2025b26anqr1