14800-DvRappard

118 Chapter 7 The small number of patients for each of the outcome groups is a limitation of this study, although the total patient number, given the fact that the study was performed in a single center on the same scanner, is rather large for a rare disorder. In addition, age distribution differed between groups, a fact which can hardly be prevented, because treatment eligibility and success are related to age of onset. Finally, the inevitable selection bias of this retrospective study results in an eventual poor outcome for all non-transplanted patients. Despite these difficulties, this study shows that quantitativeMRSI has high discriminative power regarding clinical outcome in MLD already at diagnosis with NAA as the most important parameter. Severely abnormal metabolite concentrations at baseline indicate low probability of good outcome. Therefore, results are helpful in deciding whether HCT will be beneficial, certainly for patients with borderline neurological and cognitive examination.