Survey of quality of life, phenotypic expression, and response to treatment in Krabbe leukodystrophy.

ABSTRACT:

Objectives

To develop a quality of life (QOL) survey for Krabbe disease (KD), and to thereby improve understanding of its phenotypic expression and response to treatment.

Methods

The survey, the Leukodystrophy Quality of Life Assessment (LQLA) and the Vineland Adaptive Behavior Scales were co-administered to 33 patients or their caretakers. These included the phenotypes of early infantile KD (EIKD; 0-6 months old at onset), late infantile cases (LIKD; 7-12 months old at onset), and cases that emerged after 12 months old, late onset (LOKD). The sample included cases with and without stem cell transplantation (SCT). Reliability and concurrent validity were assessed for overall and subscale scores. Analysis of variance tested differences in QOL between phenotypes and transplant

SUBMITTER: Langan TJ 

PROVIDER: S-EPMC6498827 | biostudies-literature | 2019 May

REPOSITORIES: biostudies-literature