Table 1.
Characteristic | Value |
---|---|
Patients | |
No. enrolled* | 17 |
Age at enrollment (yr) | |
Median | 6 |
Range | 4–13 |
Loes score† | |
Median | 2.0 |
Range | 1.0–7.5 |
Score on neurologic function scale‡ | |
Median | 0 |
Range | 0 |
Time from consent to infusion of drug product (days) | |
Median | 67.0 |
Range | 58.0–89.0 |
Drug product | |
Vector copy number (vector copies/diploid genome) | |
Median | 1.0 |
Range | 0.5–2.5 |
Dose (CD34+ cells/kilogram of body weight) | |
Median | 10,500,000 |
Range | 6,000,000–19,400,000 |
All the patients were male.
Loes scores range from 0 to 34, with higher scores indicating an increased extent of lesions on magnetic resonance imaging.
Scores on the cerebral adrenoleukodystrophy–specific neurologic function scale range from 0 to 25, with higher scores indicating more severe deficits. The scale is used to evaluate the severity of gross neurologic dysfunction through an assessment for 15 disabilities across multiple domains; a score of 0 indicates the absence of clinical signs of cerebral disease.