Skip to main content
. Author manuscript; available in PMC: 2014 Sep 8.
Published in final edited form as: Neuropediatrics. 2014 Jan 23;45(3):169–174. doi: 10.1055/s-0033-1364179

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.