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Table 1 Patient disposition, eliglustat exposure, and adverse event (AE) frequency in the pooled eliglustat clinical trial dataset

From: Long-term adverse event profile from four completed trials of oral eliglustat in adults with Gaucher disease type 1

 

TOTAL

Phase 2 Untreated

ENGAGE Untreated

ENCORE ERT Switch

EDGE Mostly ERT Switch

Eliglustat-treated patients

393

26

40

157

170

Patient-years of treatment exposure

1400

169

154

511

566

Mean duration of treatment (years)

3.6

6.5

3.9

3.3

3.3

Remained in trial until completion or availability of commercial drug, n (%)

319 (81.2)

19 (73.1)

34 (85.0)

129 (82.2)

137 (80.6)

Switched to commercial eliglustat, n

80

0

7

52

21

Previously treated with enzyme replacement therapy, n (%)

305 (77.6)

0 (0)

0 (0)

157 (100.0)

148 (87.1)

Active withdrawals, n (%)

74 (18.8)

7 (26.9)

6 (15.0)

28 (17.8)

33 (19.4)

 Due to any AEa

25 (6.4)

3 (11.5)

0 (0)

12 (7.6)

10 (5.9)

 Wished to withdraw

25 (6.4)

1 (3.8)

5 (12.5)

8 (5.1)

11 (6.5)

 Due to pregnancy

15 (3.8)

3 (11.5)

1 (2.5)

4 (2.5)

7 (4.1)

 Due to noncompliance

3 (0.8)

0 (0)

0 (0)

0 (0)

3 (1.8)

 Lost to follow-up

3 (0.8)

0 (0)

0 (0)

1 (0.6)

2 (1.2)

 Other

3 (0.8)

0 (0)

0 (0)

3 (1.9)

0 (0)

Any patient with an AE, n (%)

373 (94.9)

26 (100.0)

36 (90.0)

147 (93.6)

164 (96.5)

Number of events, n

4814

348

559

2153

1754

 Mild, n (%)

3551 (73.7)

251 (72.1)

448 (80.1)

1590 (73.9)

1262 (71.9)

 Moderate, n (%)

1117 (23.2)

91 (26.1)

107 (19.1)

501 (23.2)

418 (23.8)

 Severe, n (%)

146 (3.0)

6 (1.7)

4 (0.7)

62 (2.9)

74 (4.2)

Any patient with treatment-relatedb AE, n (%)

196 (49.9)

10 (38.5)

22 (55.0)

83 (52.9)

81 (47.6)

Number of treatment-related AEs, n (% of AEs)

682 (14.2)

20 (5.7)

94 (16.8)

314 (14.6)

254 (14.5)

Any patient with serious AE, n (%)

77 (19.6)

5 (19.2)

5 (12.5)

27 (17.2)

40 (23.5)

Number of serious AEs, n

103

8

7

28

60

 Mild, n (%)

17 (17)

3 (38)

4 (57)

3 (11)

7 (12)

 Moderate, n (%)

37 (36)

1 (13)

3 (43)

9 (32)

24 (40)

 Severe, n (%)

49 (48)

4 (50)

0 (0)

16 (57)

29 (48)

Any patient with treatment-related serious AE, n (%)

8 (2.0)

1 (3.8)

1 (2.5)

2 (1.3)

4 (2.4)

Deaths, n (%)

2 (0.5)

0 (0)

0 (0)

0 (0)

2 (1.2)

  1. aIn 9 patients (2.3% of total patients), one or more of the adverse events leading to withdrawal was reported as related to eliglustat treatment. See Additional file 1: Table S1 for further details on all adverse events leading to study discontinuation
  2. bRelatedness of the adverse event to eliglustat was determined by the investigator