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Table 2 Patient demographics and characteristics

From: Safety analysis of self-administered enzyme replacement therapy using data from the Fabry Outcome and Gaucher Outcome Surveys

 

Agalsidase alfa

Velaglucerase alfa

Self-administered infusion (n = 202)

HCP-supported infusion (n = 2233)a

Self-administered infusion (n = 30)

HCP-supported infusion (n = 784)a

Sex, n (%)

 Male

97 (48.0)

1006 (45.1)

16 (53.3)

346 (44.1)

 Female

105 (52.0)

1227 (54.9)

14 (46.7)

438 (55.9)

Country, n (%)

 Israel

0

6 (0.3)

11 (36.7)

292 (37.2)

 Netherlands

55 (27.2)

10 (0.4)

0

0

 UK

98 (48.5)

248 (11.1)

19 (63.3)

63 (8.0)

 USA

0

12 (0.5)

0

266 (33.9)

 Other

49 (24.3)b

1957 (87.6)c

0

163 (20.8)d

Age at FOS/GOS entry

 n

202

2233

30

784

 Mean (SD)

39.7 (16.7)

42.1 (16.8)

41.7 (17.7)

40.2 (20.6)

 Median (range)

40.3 (2.0, 74.9)

43.5 (0.5, 83.5)

40.5 (10.0, 68.6)

38.7 (0, 89.3)

Age at diagnosis

 n (missing)

189 (13)

2128 (105)

25 (5)

728 (56)

 Mean (SD)

31.3 (17.5)

35.9 (18.0)

9.1 (13.5)

20.4 (17.2)

 Median (range)

29.0 (0, 67)

37.0 (0, 80)

4.3 (0, 64)

16.8 (0, 85)

Age at first ERT, years

 n

202

2233

30

784

 Mean (SD)

39.0 (16.8)

41.5 (16.7)

38.4 (16.7)

36.9 (19.8)

 Median (range)

39.9 (2.5, 78.7)

43.1 (2.5, 81.5)

37.3 (8.9, 66.3)

35.7 (0.1, 85.7)

Mean exposure to ERT, years; mean (SD)

10.8 (5.17) 0.9–21.5

8.2 (5.6) 0.0–21.0

9.0 (2.91) 1.1–11.7

7.1 (3.91) 0.0–17.2

Mean time from ERT start to self-administered infusion start, years; mean (SD), range

3.0 (3.53) 0.0–16.4

–

0.8 (2.51) 0.0–10.1

–

Mean age at first self-administered infusion, years; mean (SD), range

42.0 (16.59) 9.7–78.7

–

40.3 (16.42) 9.3–68.2

–

Mean number of self-administered infusions per patient; mean (SD), range

92 (114.69) 0.07–462.55

–

210 (94.69) 0.07–305.38

–

Mean duration of self-administered infusions, years; mean (SD), range

3.5 (4.41) 0.0–17.8

–

8.0 (3.64) 0.0–11.7

–

  1. ERT, enzyme replacement therapy; FOS, Fabry Outcomes Survey; GOS, Gaucher Outcomes Survey; HCP, health care provider; SD, standard deviation
  2. aThe HCP-supported populations do not include patients who transitioned to self-administration after initiating ERT infusions in a clinic/hospital setting
  3. bArgentina (n = 2), Australia (n = 1), Canada (n = 9), Czech Republic (n = 1), Denmark (n = 1), France (n = 6), Germany (n = 20), Italy (n = 3), Slovenia (n = 1), Spain (n = 2), Sweden (n = 1), Switzerland (n = 2)
  4. cArgentina (n = 76), Austria (n = 33), Australia (n = 41), Belgium (n = 24), Brazil (n = 32), Canada (n = 162), Czech Republic (n = 48), Denmark (n = 3), Finland (n = 33), France (n = 100), Germany (n = 417), Hungary (n = 10), Italy (n = 171), Japan (n = 462), Portugal (n = 12), Russia (n = 14), Slovenia (n = 9), South Korea (n = 4), Spain (n = 102), Sweden (n = 14), Switzerland (n = 57), Taiwan (n = 133)
  5. dAlbania (n = 6), Argentina (n = 19), Austria (n = 4), Brazil (n = 1), Canada (n = 15), France (n = 24), Germany (n = 21), Italy (n = 2), Paraguay (n = 19), Poland (n = 7), Russia (n = 25), Spain (n = 20)
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172