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Table 2 Summary of clinical data and outcome measures at baseline

From: Disease progression in women with X-linked adrenoleukodystrophy is slow

 

N

Mean ± SD / median (range) / proportion

Clinical characteristics of the cohort

Follow-up time in years

34

7.8 (6.4–8.7)

Symptomatic (baseline)

65

37 (57%)

Symptomatic (follow-up)

34

27 (79%)

Conversion to symptomatic during follow-up period

34

8 (24%)

Walking (baseline) - unrestricted

62

40 (65%)

- restricted

62

14 (23%)

- with an aid

62

8 (13%)

Change in walking status during follow-up

32

3 (0.1%)

Age in years (baseline)

65

49.2 ± 14.2

- Age youngest symptomatic woman

1

36

- Age oldest asymptomatic woman

1

73

- Age youngest woman with aided walking

1

38

- Age oldest woman with unrestricted walking

1

74

Outcome measures at baseline

EDSS

63

2.5 (0–6)

ALDS

64

89.47 (71.92–89.47)

SF-36: Physical functioning

64

0.22 (−3.69–1.16)

SF-36: Role limitations due to physical problems

64

0.52 (−2.55–1.63)

SF-36: Bodily pain

64

0.12 (− 3.11–1.50)

SF-36: General health perceptions

64

−0.15 (− 3.63–1.85)

SF-36: Vitality

63

−0.14 (− 2.48–1.58)

SF-36: Social functioning

64

0.24 (− 3.02–1.09)

SF-36: Role limitations due to emotional problems

64

0.55 (− 2.77–0.95)

SF-36: Mental health

63

0.33 (−1.79–1.54)

SF-36: Physical component summary

63

49.37 (17.26–62.36)

SF-36: Mental component summary

63

53.89 (35.89–66.30)

  1. The EDSS ranges from 0 (normal) to 10 (death). The ALDS scores are regression coefficients which were linearly transformed for interpretation, ranging from 10 (highest level of disability) to 89.47 (lowest level of disability). SF-36 values were compared with norm values for the Dutch population and corrected for gender and age. The SF-36 subdomain scores are expressed in Z scores, ranging from −4 (lowest quality of life) to + 4 (highest quality of life). The SF-36 summary scores were linearly transformed to a range from 0 (lowest quality of life) to 100 (highest quality of life), with a mean of 50 and standard deviation of 10
  2. ALDS = AMC Linear Disability Scale; EDSS = Expanded Disability Status Scale; SD = standard deviation; SF-36 = Short Form (36) Health Survey