Bone Marrow Transplant Study on Treatment of CSF1R-Related Leukoencephalopathy

August 11, 2021

This is the largest series of patients with CSF1R-related leukoencephalopathy receiving HSCT. The study concludes that HSCT can stabilize the disease in some patients. Variability in patient responsiveness suggests that measures of disease heterogeneity and severity need to be considered when evaluating a patient’s candidacy for transplant. HSCT appears to be the first disease-modifying therapy for CSF1R-related leukoencephalopathy. This milestone may serve as a foothold toward better understanding the disease’s pathomechanism, thus providing new opportunities for better disease-specific therapies.