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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