Charcot-Marie-Tooth Index

About

Brief Description The CMT-Health Index (CMT-HI) is a disease-specific, patient-reported, outcome measure designed to reliably and responsively measure therapeutic benefit in Charcot-Marie-Tooth disease (CMT) clinical trials. The CMT-HI may also be used to measure overall patient health in a clinical setting. It covers the 18 areas of greatest importance to the adult CMT population and has been designed to satisfy recommendations from the FDA for use in drug labeling claims. Applications The CMT-HI is capable of both classifying patients with inherited neuropathies and measuring clinically-relevant change in disease state during therapeutic trials Advantages The CMT-HI has been designed to meet all FDA guidance criteria as an instrument to support drug labeling claims. The instrument has been developed using input from over 400 CMT patients, neuropathy experts, and psychometric specialists.  It is composed of 18 CMT-specific subscales and can be completed by patients in ~15 minutes.   The instrument is scored to provide individual scores for each subscale and a total CMT-HI score based on a weighted composite of the subscales.  Individual subscales within the instrument can be used individually to measure a patient’s perspective on their own: 1) Mobility and ambulation; 2) Foot and ankle strength; 3) Balance; 4) Ability to due activities; 5) Hand and finger function; 6) Numbness; 7) Pain; 8) Fatigue; 9) Daytime sleepiness; 10) Emotional state; 11) Performance in social situations; 12) Cognitive function; 13) Heartburn; 14) Lower gastrointestinal function; 15) Hearing; 16) Breathing; 17) Ability to swallow; and 18) Shoulder and arm function.   All subscales have been previously identified and validated by CMT patients as being clinically important in their daily lives.