Why the study?
The prognosis of adrenoleukodystrophy with neurological involvement is dismal, but allogeneic stem cell transplantation is recognized to stabilize or improve clinical symptoms.
Does allogeneic stem cell transplantation with reduced intensity conditioning improve survival and neurological symptoms in patients with adrenoleukodystrophy?
Population
16 patients with ALD who were symptomatic (n = 14) or presymptomatic (n = 2)
Comparison
Allogeneic stem cell transplantation with reduced intensity conditioning
Design
Single-center consecutive case series
Follow-up
Median of 45 months (range 16–91)
Authors
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Reduced-intensity allogeneic SCT appears feasible in ALD; leaves open durability of neurological benefit and need for controlled trials.
Does allogeneic stem cell transplantation with reduced intensity conditioning improve survival and neurological symptoms in patients with adrenoleukodystrophy?
Allogeneic stem cell transplantation with reduced intensity conditioning is safe and can stabilize neurological symptoms in ALD patients, particularly those without internal capsule involvement.
Kato et al. (2018) studied this question.
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