Answers are grounded in this paper and cite their sources.
This is the largest series of patients with CSF1R-related leukoencephalopathy receiving HSCT. We conclude 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.
No takes yet. Share an insight, caveat, or question.
Tipton et al. (2021) studied this question.
Synapse has enriched 10 related papers. Consider them for comparative context: