Unified Huntington's disease rating scale for advanced patients: Validation and follow-up study

Authors

  • Katia Youssov MD,

    1. Assistance Publique–Hôpitaux de Paris (AP-HP), Centre de référence Maladie de Huntington, Groupe Hospitalier Universitaire (GHU) Henri Mondor-Albert Chenevier, Créteil, France
    2. INSERM U955, Equipe 01 Neuropsychologie interventionnelle, Créteil, France
    3. Ecole Normale Supérieure, Institut d'Etudes Cognitives, Paris, France
    4. Université Paris Est, Faculté de Médecine, Créteil, France
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  • Guillaume Dolbeau RI,

    1. Assistance Publique–Hôpitaux de Paris (AP-HP), Centre de référence Maladie de Huntington, Groupe Hospitalier Universitaire (GHU) Henri Mondor-Albert Chenevier, Créteil, France
    2. INSERM U955, Equipe 01 Neuropsychologie interventionnelle, Créteil, France
    3. Université Paris Est, Faculté de Médecine, Créteil, France
    4. Unité de recherche Clinique, AP-HP, Hôpital Henri Mondor, Créteil, France
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  • Patrick Maison MD, PhD,

    1. INSERM U955, Equipe 01 Neuropsychologie interventionnelle, Créteil, France
    2. Ecole Normale Supérieure, Institut d'Etudes Cognitives, Paris, France
    3. Université Paris Est, Faculté de Médecine, Créteil, France
    4. Pharmacologie Clinique, AP-HP, Hôpital Henri Mondor, Créteil, France
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  • Marie-Françoise Boissé MSc,

    1. Assistance Publique–Hôpitaux de Paris (AP-HP), Centre de référence Maladie de Huntington, Groupe Hospitalier Universitaire (GHU) Henri Mondor-Albert Chenevier, Créteil, France
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  • Laurent Cleret de Langavant MD, PhD,

    1. Assistance Publique–Hôpitaux de Paris (AP-HP), Centre de référence Maladie de Huntington, Groupe Hospitalier Universitaire (GHU) Henri Mondor-Albert Chenevier, Créteil, France
    2. INSERM U955, Equipe 01 Neuropsychologie interventionnelle, Créteil, France
    3. Ecole Normale Supérieure, Institut d'Etudes Cognitives, Paris, France
    4. Université Paris Est, Faculté de Médecine, Créteil, France
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  • Raymund A.C. Roos MD, PhD,

    1. Department of Neurology, Leiden University Medical Center, Leiden, The Netherlands
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  • Anne-Catherine Bachoud-Lévi MD, PhD

    Corresponding author
    1. Assistance Publique–Hôpitaux de Paris (AP-HP), Centre de référence Maladie de Huntington, Groupe Hospitalier Universitaire (GHU) Henri Mondor-Albert Chenevier, Créteil, France
    2. INSERM U955, Equipe 01 Neuropsychologie interventionnelle, Créteil, France
    3. Ecole Normale Supérieure, Institut d'Etudes Cognitives, Paris, France
    4. Université Paris Est, Faculté de Médecine, Créteil, France
    • Correspondence to: Dr. Anne-Catherine Bachoud-Lévi, Service de neurologie, Hôpital Henri Mondor, 51 av du Mal de Lattre de Tassigny, 94010 Créteil, France; bachoud@gmail.com

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  • Funding agencies: This work was supported by the Centre of reference of Huntington's disease (Ministry of Health, France) and by a Contrat d'interface INSERM (ACBL). Additional support was provided through the NeurATRIS (European Advanced Translational Research Infrastructure in Neurosciences) project, the Groupement d'Intérêt Scientifique (GIS)-Institut des Maladies Rares and the” Biomarker program” AP-HP.

  • Relevant conflicts of interest/financial disclosures: Dr Maison reports support for eusapharma for analysing the RHLF cohort. Dr Roos receives project grants from CHDI and the Gossweiler Foundation. Dr Bachoud-Lévi serves as an expert for the University of Sacramento, and receives institutional support from the Ministry of Health for the management of the Centre of reference for Huntington's disease. She benefits from a “Contrat d'interface” by the INSERM for conducting her research and a contract with CHDI for Registry.

ABSTRACT

The Unified Huntington's Disease Rating Scale (UHDRS) adequately measures decline in patients at early and moderate stages of Huntington's disease (HD). In advanced patients, floor effects hamper the evaluation, thus calling for an adjusted scale. We designed the UHDRS-For Advanced Patients (UHDRS-FAP), in order to improve longitudinal assessment of patients at advanced disease stage. Sixty-nine patients with a Total Functional Capacity (TFC) ≤ 5 were recruited in France and in the Netherlands. Among them, 45 patients were followed longitudinally (mean 1.6 ± 1.2 years) with the UHDRS-FAP; 30 were also assessed with the UHDRS. Cross-sectional analyses evaluated psychometric properties and interrater reliability of the scale. Longitudinal analyses evaluated the sensitivity to decline compared to the UHDRS. Internal consistency was higher for motor and cognitive scores than for somatic and behavioral scores (0.84, 0.91, 0.70, and 0.49, respectively). Interrater reliability was ≥ 0.88 in all scores. The somatic score, specific to the UHDRS-FAP, declined over time, as well as motor and cognitive performance with both scales. Although performance with the 2 scales correlated, the UHDRS-FAP appeared more sensitive to change and was the only scale that detected decline in patients with a TFC ≤ 1. Neither scale detected a significant decline in behavioral scores. The UHDRS-FAP is reliable and more sensitive to change than the original UHDRS for cognitive and motor domains. It offers items relevant for daily care. Behavioral scores tended to decline but this may reflect the decline in the communicative abilities of the patients. © 2013 International Parkinson and Movement Disorder Society

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