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Table 1 Patients’ characteristics and baseline values of clinical outcome measures at start of ERT

From: Effect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: an open-label single-center study

Characteristic

Total study population (N = 69)

Patients with pre-ERT and ERT follow-up (N = 49)

P-value*

Age at start of ERT in years – median (range)

52.1 (26.2 – 76.3)

50.1 (26.2 – 74.0)

0.83

Age at onset of symptoms in years – median (range)

30.8 (1.4 – 62.0)

32.0 (1.4 – 62.0)

0.63

Age at diagnosis in years – median (range)

39.6 (1.4 – 63.8)

40.9 (1.4 – 63.0)

0.97

Duration of disease at start of ERT in years – median (range)

9.3 (0.2 – 31.2)

8.3 (0.5 – 31.2)

0.74

MRC sumscore at start of ERT in percentage – median (range)

77.7 (48.3 – 92.3)

79.2 (60.8 – 92.3)

0.52

HHD sumscore at start of ERT in percentage – median (range)

69.3 (25.9 – 94.1)

69.3 (25.9 – 94.1)

0.97

QMFT score at start of ERT in percentage – median (range)

55.6 (10.0 – 89.1)

59.5 (21.7 – 89.1)

0.42

Time of mechanical ventilation at start of ERT per day in hours – median (range)

11.5 (0.0 –24.0)

10.5 (0.0 –24.0)

0.81

FVC in upright position at start of ERT in percentage – median (range)

68.3 (11.3 – 106.9)

71.9 (26.5 – 106.9)

0.43

FVC in supine position at start of ERT in percentage – median (range)

46.8 (23.0 – 99.0)

52.4 (23.0 – 99.0)

0.71

Gender – No. of patients (%)

  

0.43

 - Male

36 (52)

21 (43)

 

 - Female

33 (48)

28 (57)

 

Wheelchair at start of ERT – No. of patients (%)

   

 - No wheelchair use

42 (61)

33 (67)

0.22

 - Partial wheelchair use

8 (12)

8 (16)

 

 - Permanent wheelchair use

19 (28)

8 (16)

 

Mechanical ventilation at start of ERT – No. of patients (%)

   

 - No mechanical ventilation

44 (64)

36 (74)

0.72

 - Non-invasive mechanical ventilation

19 (28)

10 (20)

 

 - Invasive mechanical ventilation

6 (9)

3 (6)

 
  1. ERT = Enzyme Replacement Therapy; MRC sumscore = Medical Research Council sumscore; HHD sumscore = Hand-Held Dynamometry sumscore; QMFT score = Quick Motor Function Test score; FVC = Forced Vital Capacity; N/No. = Number of patients.
  2. * Differences between the total group (N = 69) and the group with pre-ERT and ERT follow-up (N = 49) were calculated using chi-square tests for the categorical variables, and using Mann–Whitney tests for continuous variables.