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Friedreich Ataxia Caregiver-Reported Health Index: Development of a Novel, Disease-Specific Caregiver-Reported
Jamison Seabury1, Anika Varma1, Jennifer Weinstein1
1Center for Health + Technology (JS, AV, JW, SJR, CE, SK, CZ, ESW, BLC, CRH); Department of Neurology (CZ, ND, CRH), University of Rochester, NY; Loyola University Chicago Stritch School of Medicine (DA), Maywood, IL; Cornell University (JMH), Ithaca, NY; Department of Neurology (DRL); Children's Hospital of Philadelphia (CHOP) (CCP, MW), PA; and Department of Neurology (SHS), University of Florida College of Medicine, Gainsville.
Background And Objectives:
The Friedreich ataxia (FRDA) scientific community needs access to patient-centered outcome measures that satisfy regulatory guidelines and are capable of tracking clinically meaningful changes in FRDA disease burden. The objective of this research was to develop a novel, disease-specific caregiver-reported outcome measure for use in FRDA research and clinical care.
Methods:
In prior work, we conducted qualitative interviews and a cross-sectional study of FRDA caregivers and patients to determine the symptoms of greatest importance to individuals with FRDA. We designed the Friedreich Ataxia Caregiver-Reported Health Index (FACR-HI) to serially measure the symptoms of greatest importance to patients and utilized factor analysis, beta testing, reliability testing, and cross-sectional subgroup analysis to further evaluate and optimize this disease-specific outcome measure.
Results:
The FACR-HI was designed to measure total disease burden and disease burden in 18 symptomatic domains. The FACR-HI total score demonstrated high internal consistency (Cronbach's α = 0.98) and test-retest reliability (intraclass correlation coefficient = 0.96). Beta interview participants found the FACR-HI to be highly relevant, comprehensive, and easy to use. FACR-HI total and subscale scores were associated with functional staging for ataxia scores and speech impairment.
Discussion:
Initial evaluation of the FACR-HI supports its content validity, test-retest reliability, and construct validity as a caregiver-reported outcome measure for assessing how pediatric individuals with FRDA feel and function. The FACR-HI provides a potential mechanism to quantify changes in multifactorial FRDA disease burden during future clinical trials.
