Table 1 Baselinea characteristics.

From: Assessment of plasma lyso-Gb3 for clinical monitoring of treatment response in migalastat-treated patients with Fabry disease

 

Overall N = 97

ERT-naiveb n = 48

ERT-experienced n = 49

Males n = 37

Females n = 60

Age ≤ 40 years n = 31

Age > 40 years n = 66

Age, years, mean (SD)

46.2 (13.1)

43.0 (11.3)

49.4 (14.1)

45.8 (14.1)

46.4 (12.6)

30.6 (6.8)

53.5 (7.9)

Time since Fabry diagnosis, years, mean (SD)

9.0 (10.5)

6.3 (7.8)

11.6 (12.0)

7.1 (8.9)

10.1 (11.2)

8.4 (9.4)

9.2 (11.0)

Duration of ERT exposure,c years, mean (SD)

3.4 (2.4)

NA

3.4 (2.4)

3.5 (2.3)

3.4 (2.5)

3.7 (3.3)

3.4 (2.1)

Duration of migalastat exposure, years, median (range)

5.1 (0.1–8.5)

6.5 (0.1–8.5)

5.0 (0.1–7.2)

5.6 (0.5–8.4)

5.1 (0.1–8.5)

2.5 (0.1–8.2)

5.6 (0.1–8.5)

KIC Gb3 inclusions, median (range)

0.2 (0.01–5.69)

0.2 (0.01–5.69)

NA

0.6 (0.01–5.69)d

0.2 (0.02–0.60)d

0.2 (0.05–5.69)d

0.1 (0.01–3.05)d

LVMi, g/m2, mean (SD)

93.9 (29.6)

96.5 (32.9)

91.5 (26.3)

113.3 (34.5)

82.0 (17.9)

78.1 (17.6)

101.1 (31.2)

eGFRCKD-EPI, mL/min/1.73 m2, mean (SD)

91.2 (22.5)

93.1 (24.4)

89.3 (20.5)

88.5 (26.3)

92.9 (19.8)

109.8 (18.9)

82.5 (18.4)

24-hour urine protein, mg/24 hours, median (range)

198.0 (0.0–5566.0)

245.0 (44.0–2663.0)

116.0 (0.0–5566.0)

242.0 (0.0–5566.0)

152.0 (0.0–2663.0)

151.0 (0.0–2663.0)

215.0 (0.0–5566.0)

History of prior FACEs,e n (%)

86 (88.7)

45 (93.8)

41 (83.7)

33 (89.2)

53 (88.3)

24 (77.4)

62 (93.9)

Plasma lyso-Gb3,f,g nmol/L

 Median (range)

9.9 (0.8–218.3)

16.8 (1.2–218.3)

6.4 (0.8–59.1)

21.8 (0.8–218.3)

8.2 (0.8–31.7)

13.0 (0.8–135.3)

9.0 (0.8–218.3)

 Mean (SD)

24.1 (39.1)

45.2 (54.2)

10.1 (11.7)

47.7 (55.1)

9.3 (6.7)

30.6 (42.2)

21.2 (37.6)

  1. eGFRCKD-EPI estimated glomerular filtration rate using the Chronic Kidney Disease Epidemiology Collaboration equation, ERT enzyme replacement therapy, FACE Fabry-associated clinical events, Gb3 globotriaosylceramide, KIC kidney interstitial capillary, LVMi left ventricular mass index, lyso-Gb3 globotriaosylsphingosine, NA not applicable, NC not calculated, SD standard deviation.
  2. aBaseline was defined as the beginning of migalastat treatment. For patients in the ERT-migalastat group, this was month 18 of ATTRACT, prior to starting migalastat treatment in the 12-month open-label extension. Age, time since diagnosis, and duration of ERT exposure were recorded at study (FACETS or ATTRACT) enrollment.
  3. bPatients were ERT-naive or had not received ERT for ≥6 months prior to FACETS enrollment.13
  4. cPrior ERT treatment duration was not reported for 4 males and 2 females.
  5. dERT-naive patients only.
  6. eFACEs included renal, cardiac, and cerebrovascular events and death.
  7. fIn ERT-experienced patients, lyso-Gb3 values were measured 2–17 days after the last dose of ERT.
  8. gThe upper limit of the normal reference range for plasma lyso-Gb3 is 1.19 nmol/L.14
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