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Table 1 Characteristics of patients with MLD included in this analysis

From: Insights into the natural history of metachromatic leukodystrophy from interviews with caregivers

Characteristic Late-infantile MLD (n = 16) Juvenile MLD (n = 16)
Sex, n (%)
 Male 5 (31.3) 8 (50.0)
 Female 11 (68.8) 8 (50.0)
Age at interview, yearsa   
 Mean (SD) 7.6 (4.6) 20.7 (8.1)
 Range 4.1–21.7 8.8–37.2
Age at first symptom, years
 Mean (SD) 1.5 (0.4) 8.7 (3.6)
 Range 1.0–2.4 4.0–14.8
Age at diagnosis, years
 Mean (SD) 2.6 (1.7) 11.6 (5.5)
 Range 0.4–8.6 3.1–21.6
Time between first symptom and diagnosis, yearsb (n = 15) (n = 14)
 Mean 1.2 3.7
 Range 0.3–7.1 0.2–6.8
Treatments, n (%)
 Any transplant 3c (18.8) 9 (56.3)
  BMT 1 (6.3) 7 (43.8)
  SCT 2 (12.5) 2 (12.5)
  Cord blood 1 (6.3) 0 (0.0)
 G-tube fitted 15 (93.8) 9 (56.3)
  1. BMT bone marrow transplant, G-tube gastrostomy tube, MLD metachromatic leukodystrophy, SCT stem cell transplant, SD standard deviation
  2. aFor patients who had died, age at interview was defined as age at death
  3. bThree patients (one with late-infantile MLD; two with juvenile MLD) were diagnosed prior to symptom onset following diagnosis of an older sibling, and therefore were not included here
  4. cOne patient received both BMT and SCT