Improving Scientific Rigor of Renal Clinical Endpoints for Sickle Cell Anemia

University of Alabama at Birmingham120 enrolled

Overview

The investigators will attempt to develop a more accurate equation to estimate eGFR in pediatric and adult sickle cell patients

200 pediatric and 200 adult SCA participants will undergo mGFR using iohexol at baseline and at one year. On the same day, participants will undergo clinical and laboratory variables important for developing an eGFR equation. From baseline data, the investigators will perform a training and validation cohort of biologically relevant predictor variables to develop a novel equation. Then the investigators will compare the novel SCA eGFR equation to the established eGFR equation. From one year data, the investigators will determine the longitudinal concordance between pediatric and adult novel and standard eGFR equations with mGFR and variables that may alter longitudinal inferences. In a subset of 30 adult participants, the investigators will also evaluate the correlation between mGFR using iohexol and a novel measure of mGFR that is more time efficient

Study Type

OBSERVATIONAL

Enrollment

120

Conditions

Sickle Cell Disease Renal Disease Glomerular Disease

Interventions

iohexol GFRDIAGNOSTIC_TEST

We will perform mGFR in 400 SCA patients

Eligibility

Sex: ALLMin age: 5 YearsMax age: 50 YearsHealthy volunteers:

Medical Language ↔ Plain English

Inclusion Criteria: * SCA patients (HbSS and SB0 thalassemia) * Age: 5.0-50.0 at enrollment Exclusion Criteria: * Recent SCA complication associated with hospitalization (within 30 days) or ED visit (within 14 days) * Current AKI defined as \>0.3mg/g increase in SCr from prior visit * Known history of anaphylaxis with contrast agent or known pregnancy

Locations (4)

University of Alabama at Birmingham

Birmingham, Alabama, United States

University of Illinois Chicage

Chicago, Illinois, United States

St Jude Childrens Research Hospital

Memphis, Tennessee, United States

University of Tennessee Health Science Center

Memphis, Tennessee, United States

Outcomes

Primary Outcomes

To develop a novel SCA-specific eGFR equation and compare with existing eGFR equations validated for the general population

The investigators will use biologically relevant variables to develop a new equation for eGFR in pediatric and adult sickle cell patients

Time frame: 5 years

Secondary Outcomes

To determine the longitudinal concordance between pediatric and adult novel and standard eGFR equations with mGFR and variables that may alter longitudinal inferences.

To compare the concordance between eGFR and mGFR from baseline to one year

Time frame: 5 years

To test the correlation of the novel mGFR method using VFI FAST and iohexol mGFR.

To perform a 3 hour mGFR and compare this mGFR to the 6 hour mGFR (iohexol)

Time frame: 5 years

Data from ClinicalTrials.gov

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