Survival End Points for Huntington Disease Trials Prior to a Motor Diagnosis.

ABSTRACT:

Importance

Predictive genetic testing in Huntington disease (HD) enables therapeutic trials in HTT gene expansion mutation carriers prior to a motor diagnosis. Progression-free survival (PFS) is the composite of a motor diagnosis or a progression event, whichever comes first.

Objective

To determine if PFS provides feasible sample sizes for trials with mutation carriers who have not yet received a motor diagnosis.

Design, setting, and participants

This study uses data from the 2-phase, longitudinal cohort studies called Track and from a longitudinal cohort study called the Cooperative Huntington Observational Research Trial (COHORT). Track had 167 prediagnosis mutation carriers and 156 noncarriers, whereas COHORT had 366 prediagnosis mutation carriers and noncarriers.

SUBMITTER: Long JD 

PROVIDER: S-EPMC5710578 | biostudies-literature | 2017 Nov

REPOSITORIES: biostudies-literature