Table 2

Patient demographics and disease characteristics stratified by Loes score at the time of HCT

CharacteristicLoes score < 10Loes score ≥ 10P
Age at diagnosis, y   < .01 
    Median (range) 5.2 (0-14.3) 8.4 (2.5-22.3)  
Time from ALD diagnosis*to HCT, y   < .01 
    Median (range) 1.4 (0-10.1) 0.2 (0-10.3)  
Cerebral disease at HCT   .66 
    Predominant frontal 3 (10%) 5 (17%)  
    Predominant parieto-occipital 25 (83%) 22 (73%)  
    Mixed 2 (7%) 3 (10%)  
Cerebral disease at diagnosis*   .06 
    Yes 16 (53%) 21 (70%)  
    No 12 (40%) 5 (17%)  
    Unknown 2 (7%) 4 (13%)  
NFS at HCT   < .01 
    0 20 (67%) 3 (10%)  
    1 9 (30%) 8 (27%)  
    ≥ 2 1 (3%) 19 (63%)  
Neuropsychometric indices, median (range)    
    Verbal IQ at HCT 89 (50-121) 88 (59-117) .33 
    Performance IQ at HCT 96 (61-131) 77 (45-100) < .01 
    Full Scale IQ at HCT 91 (45-124) 75 (51-103) < .01 
Treatment with NAC   .30 
    Yes 15 (50%) 19 (63%)  
    No 15 (50%) 11 (37%)  
CharacteristicLoes score < 10Loes score ≥ 10P
Age at diagnosis, y   < .01 
    Median (range) 5.2 (0-14.3) 8.4 (2.5-22.3)  
Time from ALD diagnosis*to HCT, y   < .01 
    Median (range) 1.4 (0-10.1) 0.2 (0-10.3)  
Cerebral disease at HCT   .66 
    Predominant frontal 3 (10%) 5 (17%)  
    Predominant parieto-occipital 25 (83%) 22 (73%)  
    Mixed 2 (7%) 3 (10%)  
Cerebral disease at diagnosis*   .06 
    Yes 16 (53%) 21 (70%)  
    No 12 (40%) 5 (17%)  
    Unknown 2 (7%) 4 (13%)  
NFS at HCT   < .01 
    0 20 (67%) 3 (10%)  
    1 9 (30%) 8 (27%)  
    ≥ 2 1 (3%) 19 (63%)  
Neuropsychometric indices, median (range)    
    Verbal IQ at HCT 89 (50-121) 88 (59-117) .33 
    Performance IQ at HCT 96 (61-131) 77 (45-100) < .01 
    Full Scale IQ at HCT 91 (45-124) 75 (51-103) < .01 
Treatment with NAC   .30 
    Yes 15 (50%) 19 (63%)  
    No 15 (50%) 11 (37%)  
*

Diagnosis is defined as the first positive plasma VLCFA profile.

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