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Abstract: TH-PO617

Design and Rationale for PERFORM Patient Registry™: An Observational Cohort of Patients Utilizing TARPEYO® for Immunoglobulin A Nephropathy (IgAN) in the United States

Session Information

Category: Glomerular Diseases

  • 1402 Glomerular Diseases: Clinical, Outcomes, and Trials

Authors

  • Patel, Mit M., Calliditas NA Enterprises Inc., New York, New York, United States
  • Zhao, Danni, IQVIA Inc., Durham, North Carolina, United States
  • Lin, Nancy D., IQVIA Inc., Durham, North Carolina, United States
  • Bonzani, Ian C., IQVIA Inc., Durham, North Carolina, United States
  • Koshkina, Elena, IQVIA Inc., Durham, North Carolina, United States
  • Brooks, Warren P., Calliditas NA Enterprises Inc., New York, New York, United States
Background

Immunoglobulin A Nephropathy (IgAN) is a rare autoimmune disease characterized by the deposition of galactose-deficient IgA1 in the mesangial area of the glomeruli. Up to 50% of patients with IgAN are at risk of developing ESRD within ten to twenty years of diagnosis with poor survival outcomes. TARPEYO® (budesonide) was the first US FDA approved treatment (accelerated approval granted in December 2021) to reduce proteinuria in adults with primary IgAN at risk of rapid disease progression, generally a urine protein:creatinine ratio (UPCR) ≥1.5 g/g. Given the rarity and progressiveness of IgAN, an understanding of real-world treatment patterns and patient outcomes is warranted. The PERFORM Patient RegistryTM uses a novel direct-to-patient approach to capture electronic health data and patient-reported data in real-world patients with IgAN who are utilizing TARPEYO.

Methods

Adult participants (≥ 18 years) with at least one dispensed prescription of TARPEYO will self- enroll in the registry online via the IQVIA Integrated Health Platform study application. This approach includes participant consent to sharing of their electronic health data (electronic medical records [EMR], TARPEYO dispensing claims) and collection of electronic participant-reported data (including the participant reported outcome [ePRO] of EQ-5D-5L). Participants will be followed up for a minimum of 36 months, until their withdrawal of consent or the end of the registry, whichever occurs first.

Results

This study will describe data obtained via the online study application for eligible participants. Additional exploratory analyses include characterization of the IgAN-treated participant population in terms of demographic and clinical characteristics, patterns of TARPEYO treatment utilization and clinical management of the disease over time.

Conclusion

This is the first direct to patient real-world registry of patients with IgAN utilizing TARPEYO. Patient recruitment via Version 1.0 of the registry began in December 2022 and the full registry Version 2.0 is expected to launch in May 2023.

Funding

  • Commercial Support – Calliditas NA Enterprises Inc.