Table 4.

Comparative measures and outcome in early-stage versus late-stage leukodystrophies

Country (publication year)	Target population (numbers of patients)	Discerning measure	Outcome measure	Outcome in advanced patients	Outcome in early patients
Germany (Baumann et al, 2003)	ALD (n = 12)	MRI Score (Loes) ≤ 9 vs. > 9	Clinical reporting	3 deteriorated, 1 stable	3 deteriorated, 5 stable
United States (Beam et al, 2007)	ALD (n = 12)	MRI Score (Loes) ≤ 9 vs. > 9	Event-free survival	3 long-term survival (> 800 d), 2 early death (< 200 d)	5 long-term survival (> 800 d), 2 early death (< 200 d)
United States (Escolar et al, 2005)	GLD (n = 25)	Neurodevelopmental, MRI, and neurophysiology assessment	Clinical reporting	Minimal neurologic improvement	Progressive central myelination, appropriate cognitive function
United States (Mahmood et al, 2007)	ALD (n = 19)	Compares early transplant to no transplant	5-year survival	51%	61%
United States (Miller et al, 2011)	ALD (n = 60)	MRI Score (Loes) < 10 vs. ≥ 10	5-year survival	60%	89%
International (Peters et al, 2004)	ALD (n = 94)	MRI Score (Loes) ≤ 9 vs. > 9	5-year survival	45%	92%
International (Shapiro et al, 2000)	ALD (n = 12)	MRI Score (Loes) ≤ 9 vs. > 9	Cognitive evaluation	2 deteriorated, 1 stabilized	5 improved, 3 stable, 1 deteriorated
Canada (Siddiqi et al, 2006)	GLD (n = 12)	age at transplant (first month vs. later in first year)	Nerve conduction velocities	Motor conduction velocity improved by 3 m/sec	Motor conduction velocity improved by 13 m/sec

Abbreviations: ALD, adrenoleukodystrophy; GLD, globoid leukodystrophy; MLD, metachromatic leukodystrophy; MRI, magnetic resonance imaging.