The Myotonic Dystrophy Health Index Short Form (MDHI-SF) is a brief survey designed to efficiently measure the overall level of disease burden in a patient with myotonic dystrophy type-1. It covers the 17 areas of greatest importance to the adult myotonic dystrophy population. This instrument was developed and validated through the outcome measures laboratory at the University of Rochester and is based on original questions developed for adult myotonic dystrophy patients (also see UR Tech Id: 6-2189 “The Myotonic Dystrophy Health Index (MDHI)” for a more complete description).
Applications
This disease-specific patient-reported outcome measure is designed to reliably and responsively measure therapeutic benefits in myotonic dystrophy clinical trials. It may also be used to measure overall patient health in a clinical setting.
Advantages
This instrument has been thoroughly validated and designed to be used as a drug labeling tool (via FDA criteria). It can measure overall burden of disease and the level of disease in 17 separate areas of myotonic dystrophy subhealth. This version of the MDHI can be completed rapidly by patients in ~1 to 2 minutes.
This MDHI is a validated instrument capable of measuring clinically meaningful changes in patient health during clinical trials. As a disease-specific instrument, this outcome measure focuses on the symptoms that are most important to myotonic dystrophy patients while excluding questions that are less relevant. The content validity, face validity, construct validity, convergent validity, test retest reliability, and responsiveness of the questions in this instrument have been previously evaluated in preparation for the instrument’s use in drug labeling applications.