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

ABSTRACT:

Objective

The 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).

Methods

Inherited 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.

Results

The 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 discrimina

SUBMITTER: Johnson NE 

PROVIDER: S-EPMC6168418 | biostudies-literature | 2018 Aug

REPOSITORIES: biostudies-literature