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Table 1 Patients’ characteristics

From: Childhood cerebral adrenoleukodystrophy (CCALD) in France: epidemiology, natural history, and burden of disease - A population-based study

Characteristics

LEUKOFRANCE CCALD cohort (N = 52)

Age (y)

median (Q1-Q3)

7.0 (6.0–9.0)

 min-max

 

0.5–13.0

Age classes (y)

  

 < 3 years old

N (%)

2 (3.8)

 [3–5[ years old

2 (3.8)

 [5–8[ years old

27 (51.9)

 [8–12[ years old

17 (32.7)

 [12–15[ years old

4 (7.7)

 [15–18[ years old

0 (0.0)

ALD family history

 

 yes

N (%)

15 (29.4)

 no

36 (70.6)

Incident cases

  

 2009

N

2

 2010

3

 2011

5

 2012

2

 2013

4

 2014

2

 2015

6

 2016

3

 2017

4

 2018

4

 10-year study period

N (%)

35 (67.3)

Prevalent cases

N (%)

17 (32.7)

Inclusion status

  

Familial screen cases

N (%)

14 (26.9)

Index cases

N (%)

38 (73.1)

Hematopoietic stem cell transplant (HSCT) status

  

Patients with allogenic HSCT

N (%)

19 (36.5)

Patients without allogenic HSCT

N (%)

29 (55.8)

Patients with gene and auto HSCT therapy

N (%)

3 (5.8)

Missing value

N (%)

1 (1.9)

  1. ALD: adrenoleukodystrophy; CCALD: childhood cerebral ALD