Multiparametric MRI at the time of HSCT provides complementary prognostic and longitudinal information in juvenile metachromatic leukodystrophy. Baseline microstructural and metabolic abnormalities are associated with long-term clinical trajectories, whereas longitudinal disease evolution after HSCT is more consistently captured by volumetric and lesion-based MRI markers. These findings support the use of advanced MRI biomarkers for early risk stratification and subsequent monitoring and warrant further evaluation, including in gene therapy-treated cohorts.
Strölin et al. (Tue,) studied this question.