Table 2.
Predefined adverse events excluding stomatitis events (primary endpoint) with an incidence of ≥10% regardless of causality in both arms based on the safety population (n = 155) at 24 weeks
| AEs | Any grade |
High-grade (grade 3-4) |
||||
|---|---|---|---|---|---|---|
| EVE esc n = 79, n (%) | EVE 10mg n = 76, n (%) | P value | EVE esc n = 79, n (%) | EVE 10mg n = 76, n (%) | P value | |
| Summary of all AEs | ||||||
| Any AE | 79 (100) | 76 (100) | n.a. | 37 (46.8) | 33 (43.4) | 0.747 |
| Any haematological AE | 70 (88.6) | 72 (94.7) | 0.247 | 8 (10.1) | 8 (10.5) | >0.99 |
| Any nonhaematological AE | 79 (100) | 76 (100) | n.a. | 35 (44.3) | 29 (38.2) | 0.514 |
| Other AEs | 75 (94.9) | 68 (89.5) | 0.240 | 25 (31.7) | 17 (22.4) | 0.210 |
| At least one SAE | 23 (29.1) | 22 (28.9) | >0.99 | n.a. | n.a. | — |
| AESI (pneumonitis) | 6 (7.6) | 6 (7.9) | >0.99 | 1 (1.3) | 0 (0.0) | >0.99 |
| Predefined AEs | ||||||
| Anaemia | 59 (74.7) | 62 (81.6) | 0.336 | 3 (3.8) | 5 (6.6) | 0.489 |
| Leukopenia | 53 (67.1) | 51 (67.1) | >0.99 | 3 (3.8) | 2 (2.6) | >0.99 |
| Thrombocytopenia | 29 (36.7) | 36 (47.4) | 0.196 | 1 (1.3) | 1 (1.3) | >0.99 |
| Neutropenia | 33 (41.8) | 29 (38.2) | 0.743 | 3 (3.8) | 4 (5.3) | 0.716 |
| Blood AP increased | 41 (51.9) | 36 (47.4) | 0.631 | 2 (2.5) | 1 (1.3) | >0.99 |
| ASAT increased | 63 (79.7) | 55 (72.4) | 0.347 | 7 (8.9) | 2 (2.6) | 0.168 |
| ALAT increased | 53 (67.1) | 41 (53.9) | 0.103 | 4 (5.1) | 1 (1.3) | 0.367 |
| Blood creatinine increased | 24 (30.4) | 31 (40.8) | 0.184 | 0 (0.0) | 0 (0.0) | n.a. |
| Fatigue | 42 (53.2) | 40 (52.6) | >0.99 | 2 (2.5) | 1 (1.3) | >0.99 |
| Diarrhoea | 28 (35.4) | 19 (25.0) | 0.167 | 2 (2.5) | 2 (2.6) | >0.99 |
| Decreased appetite | 22 (27.8) | 16 (21.1) | 0.355 | 1 (1.3) | 2 (2.6) | 0.615 |
| Nausea | 23 (29.1) | 26 (34.2) | 0.604 | 1 (1.3) | 0 (0.0) | >0.99 |
| Cough | 24 (30.4) | 21 (27.6) | 0.727 | 0 (0.0) | 0 (0.0) | n.a. |
| Headache | 24 (30.4) | 17 (22.4) | 0.279 | 0 (0.0) | 1 (1.3) | 0.490 |
| Weight decreased | 16 (20.3) | 22 (28.9) | 0.263 | 0 (0.0) | 0 (0.0) | n.a. |
| Dyspnoea | 16 (20.3) | 23 (30.3) | 0.195 | 1 (1.3) | 2 (2.6) | 0.615 |
| Arthralgia | 18 (22.8) | 22 (28.9) | 0.463 | 0 (0.0) | 0 (0.0) | n.a. |
| Epistaxis | 9 (11.4) | 6 (7.9) | 0.589 | 0 (0.0) | 0 (0.0) | n.a. |
| Vertigo | 11 (13.9) | 8 (10.5) | 0.627 | 0 (0.0) | 0 (0.0) | n.a. |
| Hypertriglyceridemia | 58 (73.4) | 55 (72.4) | >0.99 | 1 (1.3) | 1 (1.3) | >0.99 |
| Hypoglycaemia | 16 (20.3) | 9 (11.8) | 0.192 | 0 (0.0) | 0 (0.0) | n.a. |
| Hyperglycaemia | 45 (57.0) | 46 (60.5) | 0.745 | 4 (5.1) | 7 (9.2) | 0.362 |
| Serum cholesterol increased | 61 (77.2) | 65 (85.5) | 0.219 | 1 (1.3) | 2 (2.6) | 0.615 |
| LDL cholesterol increaseda | 41 (51.9) | 55 (72.4) | 0.013 | n.a. | n.a. | n.a. |
| HDL cholesterol increaseda | 76 (96.2) | 71 (93.4) | 0.489 | n.a. | n.a. | n.a. |
AEs are not mutually exclusive. One patient in the EVE 10mg arm was excluded due to uncompleted safety documentation (missing data) and one patient who was randomised to the EVE esc arm received a full dose of 10 mg everolimus during the escalation phase, and therefore was analysed in the EVE 10mg arm (EVE esc: n = 79 and EVE 10mg n = 76).
AE, adverse event; AESI, adverse event of special interest; ALAT, alanine aminotransferase; AP, alkaline phosphatase; ASAT, aspartate aminotransferase; EVE, everolimus; esc, escalated; HDL, high-density lipoprotein; LDL, low-density lipoprotein; n.a., not applicable; SAE, serious adverse event.
No grading available.