ArticleNeurology. Clinical practice2023
Friedreich's Ataxia-Health Index: Development and Validation of a Novel Disease-Specific Patient-Reported Outcome Measure.
Open access · greenAbstract read
In one paragraph
Article in Neurology. Clinical practice, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.
0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed
1.0field-weighted citation impact, top 26% of its field
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
8 citing papers in PubMed, 7 citations in OpenAlex.
- Development and Validation of a Novel, Disease-Specific, Caregiver-Reported Outcome Measure: The Duchenne Muscular Dystrophy Caregiver Reported-Health Index (DMDCR-HI).Muscle & nerve · 2026Article
- Development and Validation of the Inclusion Body Myositis-Health Index: A Disease-Specific Patient-Reported Outcome Measure for Use in Clinical Trials and Drug-Labeling Claims.Journal of clinical neuromuscular disease · 2026Article
- A Patient-Reported Outcome Measure of Communication Difficulties in Friedreich Ataxia: COMATAX.Cerebellum (London, England) · 2026Article
- The Parkinson's Disease-Health Index: A novel, disease-specific patient-reported outcome measure.Journal of Parkinson's disease · 2026Article
- Exploring US patient and caregiver perspectives on burden associated with Friedreich Ataxia.Journal of patient-reported outcomes · 2026Article
- A global perspective on research advances and future challenges in Friedreich ataxia.Nature reviews. Neurology · 2025Review
- Clinical and cognitive assessment in Friedreich ataxia clinical trials: a review.Frontiers in neurology · 2025Review
- Friedreich Ataxia Caregiver-Reported Health Index: Development of a Novel, Disease-Specific Caregiver-Reported Outcome Measure.Neurology. Clinical practice · 2024Article
Corrections and comments
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Authors and funding
18 authors at 1 institution in 1 country.
Jamison SeaburyCenter for Health + Technology (CHeT) (JS, SR, AV, JW, CE, BC, EW, CZ, CH); Department of Neurology and Biostatistics (ND), University of Rochester; Pittsford Sutherland High School (JH), NY; Loyola University Chicago Stritch School of Medicine (DA), IL; PepGen Inc. (JL), Boston, MA; Children's Hospital of Philadelphia (CHOP) (DRL, CP, MW), PA; University of Florida College of Medicine (SHS), Gainesville; Friedreich's Ataxia Research Alliance (FARA) (SW), Downingtown, PA; and Department of Neurology (CH), University of Rochester, NY.
Spencer RoseroCenter for Health + Technology (CHeT) (JS, SR, AV, JW, CE, BC, EW, CZ, CH); Department of Neurology and Biostatistics (ND), University of Rochester; Pittsford Sutherland High School (JH), NY; Loyola University Chicago Stritch School of Medicine (DA), IL; PepGen Inc. (JL), Boston, MA; Children's Hospital of Philadelphia (CHOP) (DRL, CP, MW), PA; University of Florida College of Medicine (SHS), Gainesville; Friedreich's Ataxia Research Alliance (FARA) (SW), Downingtown, PA; and Department of Neurology (CH), University of Rochester, NY.
Anika VarmaCenter for Health + Technology (CHeT) (JS, SR, AV, JW, CE, BC, EW, CZ, CH); Department of Neurology and Biostatistics (ND), University of Rochester; Pittsford Sutherland High School (JH), NY; Loyola University Chicago Stritch School of Medicine (DA), IL; PepGen Inc. (JL), Boston, MA; Children's Hospital of Philadelphia (CHOP) (DRL, CP, MW), PA; University of Florida College of Medicine (SHS), Gainesville; Friedreich's Ataxia Research Alliance (FARA) (SW), Downingtown, PA; and Department of Neurology (CH), University of Rochester, NY.
Jennifer WeinsteinCenter for Health + Technology (CHeT) (JS, SR, AV, JW, CE, BC, EW, CZ, CH); Department of Neurology and Biostatistics (ND), University of Rochester; Pittsford Sutherland High School (JH), NY; Loyola University Chicago Stritch School of Medicine (DA), IL; PepGen Inc. (JL), Boston, MA; Children's Hospital of Philadelphia (CHOP) (DRL, CP, MW), PA; University of Florida College of Medicine (SHS), Gainesville; Friedreich's Ataxia Research Alliance (FARA) (SW), Downingtown, PA; and Department of Neurology (CH), University of Rochester, NY.
Charlotte EngebrechtCenter for Health + Technology (CHeT) (JS, SR, AV, JW, CE, BC, EW, CZ, CH); Department of Neurology and Biostatistics (ND), University of Rochester; Pittsford Sutherland High School (JH), NY; Loyola University Chicago Stritch School of Medicine (DA), IL; PepGen Inc. (JL), Boston, MA; Children's Hospital of Philadelphia (CHOP) (DRL, CP, MW), PA; University of Florida College of Medicine (SHS), Gainesville; Friedreich's Ataxia Research Alliance (FARA) (SW), Downingtown, PA; and Department of Neurology (CH), University of Rochester, NY.
Nuran DilekCenter for Health + Technology (CHeT) (JS, SR, AV, JW, CE, BC, EW, CZ, CH); Department of Neurology and Biostatistics (ND), University of Rochester; Pittsford Sutherland High School (JH), NY; Loyola University Chicago Stritch School of Medicine (DA), IL; PepGen Inc. (JL), Boston, MA; Children's Hospital of Philadelphia (CHOP) (DRL, CP, MW), PA; University of Florida College of Medicine (SHS), Gainesville; Friedreich's Ataxia Research Alliance (FARA) (SW), Downingtown, PA; and Department of Neurology (CH), University of Rochester, NY.
John HeatwoleCenter for Health + Technology (CHeT) (JS, SR, AV, JW, CE, BC, EW, CZ, CH); Department of Neurology and Biostatistics (ND), University of Rochester; Pittsford Sutherland High School (JH), NY; Loyola University Chicago Stritch School of Medicine (DA), IL; PepGen Inc. (JL), Boston, MA; Children's Hospital of Philadelphia (CHOP) (DRL, CP, MW), PA; University of Florida College of Medicine (SHS), Gainesville; Friedreich's Ataxia Research Alliance (FARA) (SW), Downingtown, PA; and Department of Neurology (CH), University of Rochester, NY.
Danae AlexandrouCenter for Health + Technology (CHeT) (JS, SR, AV, JW, CE, BC, EW, CZ, CH); Department of Neurology and Biostatistics (ND), University of Rochester; Pittsford Sutherland High School (JH), NY; Loyola University Chicago Stritch School of Medicine (DA), IL; PepGen Inc. (JL), Boston, MA; Children's Hospital of Philadelphia (CHOP) (DRL, CP, MW), PA; University of Florida College of Medicine (SHS), Gainesville; Friedreich's Ataxia Research Alliance (FARA) (SW), Downingtown, PA; and Department of Neurology (CH), University of Rochester, NY.
Brittany CohenCenter for Health + Technology (CHeT) (JS, SR, AV, JW, CE, BC, EW, CZ, CH); Department of Neurology and Biostatistics (ND), University of Rochester; Pittsford Sutherland High School (JH), NY; Loyola University Chicago Stritch School of Medicine (DA), IL; PepGen Inc. (JL), Boston, MA; Children's Hospital of Philadelphia (CHOP) (DRL, CP, MW), PA; University of Florida College of Medicine (SHS), Gainesville; Friedreich's Ataxia Research Alliance (FARA) (SW), Downingtown, PA; and Department of Neurology (CH), University of Rochester, NY.
Jane LarkindaleCenter for Health + Technology (CHeT) (JS, SR, AV, JW, CE, BC, EW, CZ, CH); Department of Neurology and Biostatistics (ND), University of Rochester; Pittsford Sutherland High School (JH), NY; Loyola University Chicago Stritch School of Medicine (DA), IL; PepGen Inc. (JL), Boston, MA; Children's Hospital of Philadelphia (CHOP) (DRL, CP, MW), PA; University of Florida College of Medicine (SHS), Gainesville; Friedreich's Ataxia Research Alliance (FARA) (SW), Downingtown, PA; and Department of Neurology (CH), University of Rochester, NY.
David R LynchCenter for Health + Technology (CHeT) (JS, SR, AV, JW, CE, BC, EW, CZ, CH); Department of Neurology and Biostatistics (ND), University of Rochester; Pittsford Sutherland High School (JH), NY; Loyola University Chicago Stritch School of Medicine (DA), IL; PepGen Inc. (JL), Boston, MA; Children's Hospital of Philadelphia (CHOP) (DRL, CP, MW), PA; University of Florida College of Medicine (SHS), Gainesville; Friedreich's Ataxia Research Alliance (FARA) (SW), Downingtown, PA; and Department of Neurology (CH), University of Rochester, NY.ORCID https://orcid.org/0000-0001-7168-214X
Courtney ParkCenter for Health + Technology (CHeT) (JS, SR, AV, JW, CE, BC, EW, CZ, CH); Department of Neurology and Biostatistics (ND), University of Rochester; Pittsford Sutherland High School (JH), NY; Loyola University Chicago Stritch School of Medicine (DA), IL; PepGen Inc. (JL), Boston, MA; Children's Hospital of Philadelphia (CHOP) (DRL, CP, MW), PA; University of Florida College of Medicine (SHS), Gainesville; Friedreich's Ataxia Research Alliance (FARA) (SW), Downingtown, PA; and Department of Neurology (CH), University of Rochester, NY.
Sub H SubramonyCenter for Health + Technology (CHeT) (JS, SR, AV, JW, CE, BC, EW, CZ, CH); Department of Neurology and Biostatistics (ND), University of Rochester; Pittsford Sutherland High School (JH), NY; Loyola University Chicago Stritch School of Medicine (DA), IL; PepGen Inc. (JL), Boston, MA; Children's Hospital of Philadelphia (CHOP) (DRL, CP, MW), PA; University of Florida College of Medicine (SHS), Gainesville; Friedreich's Ataxia Research Alliance (FARA) (SW), Downingtown, PA; and Department of Neurology (CH), University of Rochester, NY.ORCID https://orcid.org/0000-0002-8052-9240
Ellen WagnerCenter for Health + Technology (CHeT) (JS, SR, AV, JW, CE, BC, EW, CZ, CH); Department of Neurology and Biostatistics (ND), University of Rochester; Pittsford Sutherland High School (JH), NY; Loyola University Chicago Stritch School of Medicine (DA), IL; PepGen Inc. (JL), Boston, MA; Children's Hospital of Philadelphia (CHOP) (DRL, CP, MW), PA; University of Florida College of Medicine (SHS), Gainesville; Friedreich's Ataxia Research Alliance (FARA) (SW), Downingtown, PA; and Department of Neurology (CH), University of Rochester, NY.
Susan WaltherCenter for Health + Technology (CHeT) (JS, SR, AV, JW, CE, BC, EW, CZ, CH); Department of Neurology and Biostatistics (ND), University of Rochester; Pittsford Sutherland High School (JH), NY; Loyola University Chicago Stritch School of Medicine (DA), IL; PepGen Inc. (JL), Boston, MA; Children's Hospital of Philadelphia (CHOP) (DRL, CP, MW), PA; University of Florida College of Medicine (SHS), Gainesville; Friedreich's Ataxia Research Alliance (FARA) (SW), Downingtown, PA; and Department of Neurology (CH), University of Rochester, NY.
McKenzie WellsCenter for Health + Technology (CHeT) (JS, SR, AV, JW, CE, BC, EW, CZ, CH); Department of Neurology and Biostatistics (ND), University of Rochester; Pittsford Sutherland High School (JH), NY; Loyola University Chicago Stritch School of Medicine (DA), IL; PepGen Inc. (JL), Boston, MA; Children's Hospital of Philadelphia (CHOP) (DRL, CP, MW), PA; University of Florida College of Medicine (SHS), Gainesville; Friedreich's Ataxia Research Alliance (FARA) (SW), Downingtown, PA; and Department of Neurology (CH), University of Rochester, NY.
Christine ZizziCenter for Health + Technology (CHeT) (JS, SR, AV, JW, CE, BC, EW, CZ, CH); Department of Neurology and Biostatistics (ND), University of Rochester; Pittsford Sutherland High School (JH), NY; Loyola University Chicago Stritch School of Medicine (DA), IL; PepGen Inc. (JL), Boston, MA; Children's Hospital of Philadelphia (CHOP) (DRL, CP, MW), PA; University of Florida College of Medicine (SHS), Gainesville; Friedreich's Ataxia Research Alliance (FARA) (SW), Downingtown, PA; and Department of Neurology (CH), University of Rochester, NY.
Chad HeatwoleCenter for Health + Technology (CHeT) (JS, SR, AV, JW, CE, BC, EW, CZ, CH); Department of Neurology and Biostatistics (ND), University of Rochester; Pittsford Sutherland High School (JH), NY; Loyola University Chicago Stritch School of Medicine (DA), IL; PepGen Inc. (JL), Boston, MA; Children's Hospital of Philadelphia (CHOP) (DRL, CP, MW), PA; University of Florida College of Medicine (SHS), Gainesville; Friedreich's Ataxia Research Alliance (FARA) (SW), Downingtown, PA; and Department of Neurology (CH), University of Rochester, NY.
Children's Hospital of Philadelphia · US
Funding
No grant is acknowledged in the PubMed record.
Abstract
Background and Objectives: To develop a valid, disease-specific, patient-reported outcome (PRO) measure for adolescents and adults with Friedreich ataxia (FA) for use in therapeutic trials. Methods: We conducted semistructured qualitative interviews and a national cross-sectional study of individuals with FA to determine the most prevalent and burdensome symptoms and symptomatic themes to this population. These symptoms and symptomatic themes were included as questions in the first version of the Friedreich's Ataxia-Health Index (FA-HI). We subsequently used factor analysis, beta interviews with 17 individuals with FA, and test-retest reliability assessments with 20 individuals with FA to evaluate, refine, and optimize the FA-HI. Finally, we determined the capability of the FA-HI to differentiate between subgroups of FA participants with varying levels of disease severity. Results: Participants with FA identified 18 symptomatic themes of importance to be included as subscales in the FA-HI. The FA-HI demonstrates high internal consistency and test-retest reliability, and it was identified by participants as highly relevant, comprehensive, and easy to complete. FA-HI total and subscale scores statistically differentiated between subgroups of participants with varying levels of disease burden. Discussion: Initial evaluation of the FA-HI supports its validity and reliability as a PRO for assessing how individuals with FA feel and function.
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