Kidney Week

Abstract: INFO12-SA

Framework for Clinical and Regulatory Pathways for Sickle Cell Kidney Disease: A Kidney Health Initiative (KHI) Sponsored Project

Session Information

• Informational Posters - 3
  October 26, 2024 | Location: Exhibit Hall, Convention Center
  Abstract Time: 10:00 AM - 12:00 PM

Category: Diversity and Equity in Kidney Health

• No subcategory defined

Authors

• Saraf, Santosh, University of Illinois Chicago, Chicago, Illinois, United States

Santosh Saraf, MD

• Campbell, Kirk N., Icahn School of Medicine at Mount Sinai, New York, New York, United States

Kirk N. Campbell, MD, FASN

• Derebail, Vimal K., The University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, United States

Vimal K. Derebail, MD, MPH, FASN

• Lebensburger, Jeffrey, The University of Alabama at Birmingham, Birmingham, Alabama, United States

Jeffrey Lebensburger, DO, MPH

• Ataga, Kenneth I., The University of Tennessee Health Science Center College of Medicine, Memphis, Tennessee, United States

Kenneth I. Ataga, MBBS

• Romero, Alain J., Health Care Consultant, DEL MAR, California, United States

Alain J. Romero, MD, PharmD, PhD

Description

Background: Sickle Cell Kidney Disease (SCKD) is defined as “a group of renal complications" in patients with SCD¹ with varying prevalence and progression based on age and genotype. The trajectory of kidney function in SCD is confounded by a hyperfiltration phase followed by progressive decline in glomerular filtration. Persistent albuminuria, although increasing with age, is not always confirmed on repeat testing². SCKD ultimately leads to irreversible kidney damage with a mortality rate of 10.5%, rising to 45% for those over 60^3,4. We cannot reliably predict which patients will develop SCKD and no approved therapies exist.⁵ The absence of clear endpoints to guide RCTs and the exclusion of SCKD⁶ patients challenges.
Objective: to develop a framework for SCKD management focusing on: 1. Characterizing the natural history of the disease, 2. Identifying patients at risk of developing SCKD 3. Providing a sensible roadmap for standardized clinical trials and regulatory pathways for drug development in SCKD.
Methods: In collaboration with ASH Research collaborative and ASN, 3 working groups (WG) will cooperate to fulfill these objectives. It will include a systematic literature review and targeted data analysis using 2 large SCD datasets. WG1 will address the natural history of SCKD and the relationship between risks, surrogates and outcomes. WG2 will identify appropriate clinical trial endpoints, metrics, and effect sizes. WG3 will recommend trial populations based on biomarkers, risk scores and physiopathology.
Outcome: This project will protocolize SCKD care. By synchronizing efforts of experts across patient, nephrology, hematology, industry, and regulatory bodies, the project aims to improve SCKD management while proposing regulatory pathways for new or existing therapeutic interventions.

1.Aeddula et al StatPearls Sept 4 2023; 2. Ataga et al Nature 2022; 3.Serjeant et al 2009; Platt et al 1994; 5. Niss O et al Blood Adv 2020;

Funding

• Kidney Health Initiative