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Table 4 Summary of organ volume by MN at baseline and follow-up, all patients

From: Safety and effectiveness of taliglucerase alfa in patients with Gaucher disease: an interim analysis of real-world data from a multinational drug registry (TALIAS)

Organ

Visit (statistics)

Treatment-naïve group (n = 8)

Prior ERT group (n = 93)

Prior ERT and SRT group (n = 5)a

Total patients (N = 106)

Liver, median (range)

Baseline

n = 6

n = 68

n = 4

n = 78

   Absolute values

1.4 (0.3–2.1)

2.4 (0.1–10.9)

0.7 (0.6–0.8)

2.2 (0.1–10.9)

Year 1 (month 12)

n = 2b

n = 40

n = 0

n = 42

   Absolute values

0.9 (0.6, 1.2)

2.4 (0.5–6.2)

2.3 (0.5–6.2)

   Change from baselinec

0.1 (− 0.1, 0.4)

0.0 (− 5.3–1.0)

0.0 (− 5.3–1.0)

   % change from baselinec

63.4 (− 5.3, 132.1)

1.5 (− 50.9–877.5)

1.5 (− 50.9–877.5)

Year 2 (month 24)

n = 0

n = 29

n = 0

n = 29

   Absolute values

2.7 (0.6–4.7)

 

2.7 (0.6–4.7)

   Change from baselinec

− 0.1 (− 6.6–2.3)

− 0.1 (− 6.6–2.3)

   % change from baselinec

− 3.0 (− 60.0–159.3)

− 3.0 (− 60.0–159.3)

Spleen, median (range)

Baseline

n = 4

n = 53

n = 2

n = 59

   Absolute values

9.5 (6.8–18.1)

4.9 (0.6–121.5)

1.8 (1.6, 2.0)

5.3 (0.6–121.5)

Year 1 (month 12)

n = 2b

n = 33

n = 0

n = 35

   Absolute values

4.5 (1.9, 7.0)

4.9 (1.7–120.0)

4.9 (1.7–120.0)

   Change from baselinec

− 9.7 (− 11.1, − 8.3)

0.0 (− 82.5–42.3)

− 0.2 (− 82.5–42.3)

   % change from baselinec

− 71.1 (− 81.0, − 61.3)

− 0.9 (− 67.9–863.0)

− 6.5 (− 81.0–863.0)

Year 2 (month 24)

n = 0

n = 24

n = 0

n = 24

   Absolute values

4.2 (0.8–23.4)

 

4.2 (0.8–23.4)

   Change from baselinec

− 1.0 (− 98.1–4.2)

− 1.0 (− 98.1–4.2)

   % change from baselinec

− 25.0 (− 80.7–114.8)

− 25.0 (− 80.7–114.8)

  1. ERT enzyme replacement therapy; MN, multiples of normal; N number of patients in each group; n number of patients contributing to summary statistics for each visit; SD standard deviation; SRT substrate reduction therapy
  2. aOne patient was reported under the “Prior ERT and SRT” group due to erroneous recording of some of his ERT treatments as SRTs in the study database
  3. bThe individual values were listed for n = 2
  4. cSummaries for change and percent change from baseline were calculated from the pair of observations from patients who completed both post-baseline and baseline measurement