Use of Brain MRI in Cerebral Adrenoleukodystrophy: International Recommendations for Screening, Monitoring, and Research

This study reports outcomes of hematopoietic stem cell transplantation (HSCT) in an international cohort of patients with CSF1R-related disorder, a rare adult-onset leukoencephalopathy characterized by progressive neurodegeneration. The results suggest that HSCT may stabilize or slow disease progression in selected patients, particularly when performed at an early stage, although responses are variable. The study highlights the importance of careful patient selection, timing of intervention, and multidisciplinary evaluation, while also emphasizing the need for further data to better define efficacy and long-term outcomes.