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Table 3 Summary of hemoglobin concentration and platelet count 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)

Endpoint

Visit (statistics)

Treatment-naïve group (n = 8)

Prior ERT group (n = 93)

Prior ERT and SRT group (n = 5)a

Total patients (N = 106)

Hemoglobin, mean (SD), g/dL

Baseline

n = 8

n = 83

n = 4

n = 95

   Absolute values

11.6 (1.4)

13.3 (1.7)

13.8 (1.5)

13.2 (1.7)

Year 1 (month 12)

n = 3

n = 54

n = 1

n = 58

   Absolute values

13.0 (0.8)

13.1 (1.5)

14.4

13.2 (1.5)

   Change from baselineb

1.2 (1.3)

0 (0.8)

− 0.7

0 (0.9)

   % change from baselineb

10.5 (11.3)

0.1 (6.7)

− 4.6

0.5 (7.2)

Year 2 (month 24)

n = 0

n = 44

n = 0

n = 44

   Absolute values

13.4 (1.3)

13.4 (1.3)

   Change from baselineb

0.1 (0.9)

0.1 (0.9)

   % change from baseline

0.7 (7.3)

0.7 (7.3)

Platelet count without splenectomy, mean (SD), 103/μL

Baseline

n = 5

n = 66

n = 2

n = 73

   Absolute values

128.8 (30.3)

169.2 (77.0)

202.5 (21.9)

167.3 (74.5)

Year 1 (month 12)

n = 3

n = 41

n = 1

n = 45

   Absolute values

137.7 (35.4)

168.0 (52.4)

187.0

166.4 (51.2)

   Change from baselineb

17.0 (18.2)

7.9 (42.5)

− 31.0

7.6 (41.2)

   % change from baselineb

14.3 (14.4)

10.7 (32.4)

− 14.2

10.4 (31.2)

Year 2 (month 24)

n = 0

n = 36

n = 0

n = 36

   Absolute values

193.7 (80.0)

193.7 (80.0)

   Change from baselineb

22.1 (38.1)

22.1 (38.1)

   % change from baselineb

20.0 (35.1)

20.0 (35.1)

Platelet count with splenectomy, mean (SD), 103/μL

Baseline

n = 3

n = 16

n = 0

n = 19

   Absolute values

183.7 (28.3)

250.0 (100.2)

239.7 (95.3)

Year 1 (month 12)

n = 0

n = 9

n = 0

n = 9

   Absolute values

235.1 (87.8)

235.1 (87.8)

   Change from baselineb

− 7.0 (60.7)

− 7.0 (60.7)

   % change from baselineb

1.7 (25.2)

1.7 (25.2)

Year 2 (month 24)

n = 0

n = 7

n = 0

n = 7

   Absolute values

281.1 (89.2)

281.1 (89.2)

   Change from baselineb

− 2.1 (68.1)

− 2.1 (68.1)

   % change from baselineb

1.0 (27.8)

1.0 (27.8)

  1. 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
  2. bSummaries for change and percent change from baseline were calculated from the pair of observations from patients who completed both post-baseline and baseline measurement