Analysis of Interindividual Lesion Progression Variability in Adult Cerebral Adrenoleukodystrophy

Cerebral adrenoleukodystrophy (cALD), the most severe clinical phenotype of X-linked adrenoleukodystrophy (X-ALD), is characterized by rapidly progressive, inflammatory destruction of the cerebral white matter. In adult patients, therapeutic options are limited to hematopoietic stem cell transplantation, which carries substantial treatment-related risks, necessitating careful and individualized clinical decision-making. Monitoring the progression of characteristic MRI changes provides a promising approach, both for guiding treatment and for serving as an outcome measure in interventional trials. Although the natural history of these MRI changes has been well described in childhood cALD, analogous data in adult patients remain scarce.