Abstract: PUB176
Improvement in Metabolic Control in Patients with Distal Renal Tubular Acidosis (dRTA) After Switching from Standard of Care to ADV7103
Session Information
Category: Genetic Diseases of the Kidneys
- 1002 Genetic Diseases of the Kidneys: Non-Cystic
Authors
- Panzarino, Valerie Marie, University of South Florida, Tampa, Florida, United States
- Ogg, Carol, Advicenne Pharmaceuticals, Paris, France
- Manso-Silvan, Maria Asuncion, Advicenne Pharmaceuticals, Paris, France
- Dixon, Bradley P., University of Colorado Denver School of Medicine, Aurora, Colorado, United States
Background
Patients with dRTA present with hyperchloremic metabolic acidosis leading to poor growth as well as compromised bone and kidney health. Current standard of care (SoC) requires a multiple dose regimen with reported poor adherence which may render metabolic control difficult. We describe three patients switched from SoC to a prolonged-release galenic formulation of potassium bicarbonate and potassium citrate with twice daily dosing (ADV7103).
Methods
Three patients with clinically confirmed dRTA (18Y-M, 22Y-F, 16.5Y-F) enrolled in the ARENA2 Phase 3 study (NCT03644706) prior to COVID-19 interruption. Plasma bicarbonate and potassium levels were measured >12 months pre-study initiation on SoC, baseline screening, and 3, 6, & 12 months after participants were switched to ADV7103. Adverse events, palatability, patient satisfaction and adherence rates (estimated proportion of treatment effectively taken based on study drug retrieval) were also reported.
Results
All three patients demonstrated low plasma bicarbonate levels with SoC (< 22 mmol/L) 12 months prior to baseline screening. All patients receiving ADV7103 recorded normal plasma bicarbonate from 3 to 12 months after enrolment. Plasma potassium levels (3.0, 4.3 and 2.8 mmol/L respectively at baseline) were normal for the duration of the trial on ADV7103. One patient reported dyspepsia at 8 months on ADV7103 but resolved with symptomatic treatment. Palatability and patient satisfaction were greatly improved switching from SoC to ADV7103. Adherence rates by 12 months improved from average (50-75%) or good (75-90%) to excellent (>90%) for 2 patients and improved from poor (<50%) to good (75-90%) for one patient. One patient, an adolescent, reported a tremendous positive impact on growth velocity and lifestyle and is reluctant to return to SoC.
Conclusion
All patients maintained normal levels of plasma bicarbonate and potassium after switching from SoC to twice daily dosing with ADV7103. All three patients demonstrated better adherence with ADV7103 as compared to SoC. This small cohort of patients reported improved palatability and satisfaction with ADV7103 treatment as reasons for improved medication adherence and this increased adherence is believed to have had a direct impact on the overall metabolic control achieved throughout the study.
Funding
- Commercial Support – Advicenne Pharmaceuticals