Abstract: FR-PO0109
Real-World Treatment Patterns Among Patients with APOL1-Mediated Kidney Disease (AMKD) in the United States
Session Information
- Hereditary Glomerular and Tubulointerstitial Kidney Diseases
October 23, 2026 | Location: Exhibit Hall A, Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Genetic Diseases of the Kidneys
- 1202 Genetic Diseases of the Kidneys: Non-Cystic (Complex and Non-Cystic Monogenic)
Authors
- Shah, Ankit, Vertex Pharmaceuticals Incorporated, Boston, Massachusetts, United States
- Voegel, Arthur, Analysis Group, New York, New York, United States
- Salgaonkar, Paresh, Vertex Pharmaceuticals Incorporated, Boston, Massachusetts, United States
- Christensen, Dana, Analysis Group, New York, New York, United States
- Clark, Dinah, Natera Inc, Austin, Texas, United States
- Barber, Beth, Vertex Pharmaceuticals Incorporated, Boston, Massachusetts, United States
Background
AMKD is a rapidly progressing chronic kidney disease (CKD) driven by the presence of two toxic gain-of-function variants in the APOL1 gene. There are no approved treatments specifically for patients with AMKD and little is known about how these patients are managed in routine clinical practice. This study aimed to characterize real-world treatment patterns in AMKD patients in the US.
Methods
A cross-sectional study among US patients with genetic confirmation of AMKD (age 10 and above) identified between March 2025 and Feb 2026 was conducted using Natera’s RenasightIQ Clinicogenomic Database and NorstellaLinQ Real-World Data. Patients were excluded if they had evidence of diabetes, ADPKD or IgAN anytime prior or during the study period. Baseline demographics, comorbidities, and eGFR values in the observation period were summarized descriptively. The proportion of patients with ≥ 1 administration or fill of any of the following treatments was reported: Renin-angiotensin-aldosterone system inhibitors (RAASi), SGLT2 inhibitors (SGLT2i), GLP-1 agonists, calcium channel blockers, and beta blockers.
Results
1,332 patients met the eligibility criteria. The mean age of patients was 45.6 years (SD: 15.4 years) and 54.2% were male. Hypertension (88.9%), hyperlipidemia (42.3%), and chronic pulmonary diseases (23.1%) were the most frequently reported comorbidities. Almost half of the patients (45.7%) were in CKD Stages 4 or 5. Sixty-three percent of patients had eGFR lab values during the study period, and the average value was 38 ml/min/1.73m2 (SD: 29). 35.7% of AMKD patients were treated with RAASi, 35.0% with calcium channel blockers, 27.9% with beta blockers, 10.5% with SGLT2i, and 4.4% with GLP-1 agonists.
Conclusion
Despite currently available background CKD treatments, nearly half of patients with AMKD had progressed to CKD stages 4–5 by a mean age of 45 years. Variable treatment utilization and substantial residual disease burden underscore the significant unmet need for targeted therapies that address the underlying disease pathophysiology.
Funding
- Commercial Support – Vertex Pharmaceuticals Incorporated