The Charcot-Marie-Tooth Health Index: Evaluation of a Patient-Reported Outcome

被引:31
作者
Johnson, Nicholas E. [1 ]
Heatwole, Chad [2 ]
Creigh, Peter [2 ]
McDermott, Michael P. [2 ,3 ]
Dilek, Nuran [2 ]
Hung, Man [4 ]
Bounsanga, Jerry [4 ]
Tang, Wan [5 ]
Shy, Michael E. [6 ]
Herrmann, David N. [2 ]
机构
[1] Virginia Commonwealth Univ, Dept Neurol, Sanger Hall,1101 E Marshall St,Box 980599, Richmond, VA 23298 USA
[2] Univ Rochester, Dept Neurol, Rochester, NY USA
[3] Univ Rochester, Dept Biostat & Computat Biol, Rochester, NY USA
[4] Univ Utah, Dept Orthoped, Salt Lake City, UT USA
[5] Tulane Univ, Dept Global Biostat & Data Sci, New Orleans, LA 70118 USA
[6] Univ Iowa, Dept Neurol, Iowa City, IA 52242 USA
关键词
QUALITY-OF-LIFE; MYOTONIC-DYSTROPHY; DISEASE; NEUROPATHIES; RELIABILITY; PERSPECTIVE; VALIDATION;
D O I
10.1002/ana.25282
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
ObjectiveThe development of a disease-specific patient-reported outcome for Charcot-Marie-Tooth disease is an important step in the preparation for therapeutic trials. This study describes the development of the Charcot-Marie-Tooth Health Index (CMTHI). MethodsInherited Neuropathy Consortium Contact Registry participants were queried on the symptoms that most impacted their lives. The CMTHI was developed based on these responses. Factor analysis, assessment of test-retest reliability, known group validity, and patient interviews were utilized to refine the instrument. ResultsThe final CMTHI contains 18 themes that capture Charcot-Marie-Tooth disease (CMT) burden. The CMTHI has a high internal consistency and test-retest reliability. The CMTHI was able to discriminate between patient groups expected to have different disease burden. The CMTHI was able to discriminate levels of disability as measured by the CMT examination score and the mobility-Disability Severity Index. InterpretationThe CMTHI represents a valid and reliable outcome to assess patient-reported disease burden in CMT. Ann Neurol 2018;84:225-233
引用
收藏
页码:225 / 233
页数:9
相关论文
共 21 条
  • [11] Johnson Nicholas E, 2013, J Clin Neuromuscul Dis, V15, P19, DOI 10.1097/CND.0b013e31829e22e3
  • [12] Reliability of the CMT neuropathy score (second version) in Charcot-Marie-Tooth disease
    Murphy, Sinead M.
    Herrmann, David N.
    McDermott, Michael P.
    Scherer, Steven S.
    Shy, Michael E.
    Reilly, Mary M.
    Pareyson, Davide
    [J]. JOURNAL OF THE PERIPHERAL NERVOUS SYSTEM, 2011, 16 (03) : 191 - 198
  • [13] Correlation between clinical/neurophysiological findings and quality of life in Charcot-Marie-Tooth type 1A
    Padua, Luca
    Shy, Michael E.
    Aprile, Irene
    Cavallaro, Tiziana
    Pareyson, Davide
    Quattrone, Aldo
    Rizzuto, Nicolo
    Vita, Giuseppe
    Tonali, Pietro
    Schenone, Angelo
    [J]. JOURNAL OF THE PERIPHERAL NERVOUS SYSTEM, 2008, 13 (01) : 64 - 70
  • [14] Natural history of Charcot-Marie-Tooth 2: 2-year follow-up of muscle strength, walking ability and quality of life
    Padua, Luca
    Pareyson, D.
    Aprile, I.
    Cavallaro, T.
    Quattrone, D. A.
    Rizzuto, N.
    Vita, G.
    Tonali, P.
    Schenone, A.
    [J]. NEUROLOGICAL SCIENCES, 2010, 31 (02) : 175 - 178
  • [15] GENERIC AND DISEASE-SPECIFIC MEASURES IN ASSESSING HEALTH-STATUS AND QUALITY OF LIFE
    PATRICK, DL
    DEYO, RA
    [J]. MEDICAL CARE, 1989, 27 (03) : S217 - S232
  • [16] Patient-reported outcomes to support medical product labeling claims: FDA perspective
    Patrick, Donald L.
    Burke, Laurie B.
    Powers, John H.
    Scott, Jane A.
    Rock, Edwin P.
    Dawisha, Sahar
    O'Neill, Robert
    Kennedy, Dianne L.
    [J]. VALUE IN HEALTH, 2007, 10 : S125 - S137
  • [17] Defining disability: development and validation of a mobility-Disability Severity Index (mDSI) in Charcot-Marie-tooth disease
    Ramchandren, Sindhu
    Shy, Michael
    Feldman, Eva
    Carlos, Ruth
    Siskind, Carly
    [J]. JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY, 2015, 86 (06) : 635 - 639
  • [18] Factors that influence health-related quality of life in Australian adults with Charcot-Marie-Tooth disease
    Redmond, Anthony C.
    Burns, Joshua
    Ouvrier, Robert A.
    [J]. NEUROMUSCULAR DISORDERS, 2008, 18 (08) : 619 - 625
  • [19] Diagnosis and new treatments in genetic neuropathies
    Reilly, M. M.
    Shy, M. E.
    [J]. JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY, 2009, 80 (12) : 1304 - 1314
  • [20] The Myotonic Dystrophy Health Index: Italian validation of a disease-specific outcome measure
    Sansone, Valeria A.
    Lizio, Andrea
    Greco, Lucia
    Gragnano, Gaia
    Zanolini, Alice
    Gualandris, Marco
    Iatomasi, Marino
    Heatwole, Chad
    [J]. NEUROMUSCULAR DISORDERS, 2017, 27 (11) : 1047 - 1053