Table 2.
TEAEs relateda to thiotepa occurring in ≥ 2 patients in either target disease group by system organ class and preferred term (safety analysis population)
| System organ class Preferred term, n (%) |
Pediatric solid tumor or pediatric brain tumor N = 9 |
Adult malignant lymphoma N = 10 |
Total N = 19 |
|---|---|---|---|
| Blood and lymphatic system disorders | 5 (55.6) | 10 (100.0) | 15 (78.9) |
| Febrile neutropenia | 5 (55.6) | 10 (100.0) | 15 (78.9) |
| Cardiac disorders | 3 (33.3) | 0 | 3 (15.8) |
| Pericardial effusion | 2 (22.2) | 0 | 2 (10.5) |
| Gastrointestinal disorders | 9 (100.0) | 10 (100.0) | 19 (100.0) |
| Diarrhea | 8 (88.9) | 8 (80.0) | 16 (84.2) |
| Nausea | 6 (66.7) | 10 (100.0) | 16 (84.2) |
| Stomatitis | 9 (100.0) | 5 (50.0) | 14 (73.7) |
| Vomiting | 9 (100.0) | 4 (40.0) | 13 (78.9) |
| Oral disorder | 0 | 4 (40.0) | 4 (21.1) |
| Abdominal pain | 2 (22.2) | 0 | 2 (10.5) |
| Anal inflammation | 2 (22.2) | 0 | 2 (10.5) |
| Enterocolitis | 2 (22.2) | 0 | 2 (10.5) |
| Proctalgia | 0 | 2 (20.0) | 2 (10.5) |
| General disorders and administration site conditions | 6 (66.7) | 2 (20.0) | 8 (42.1) |
| Malaise | 3 (33.3) | 1 (10.0) | 4 (21.1) |
| Face edema | 3 (33.3) | 0 | 3 (15.8) |
| Pyrexia | 2 (22.2) | 1 (10.0) | 3 (15.8) |
| Edema peripheral | 2 (22.2) | 0 | 2 (10.5) |
| Infections and infestations | 3 (33.3) | 1 (10.0) | 4 (21.1) |
| Bacterial infection | 3 (33.3) | 0 | 3 (15.8) |
| Investigations | 7 (77.8) | 7 (70.0) | 14 (73.7) |
| ALT increased | 6 (66.7) | 6 (60.0) | 12 (63.2) |
| AST increased | 6 (66.7) | 4 (40.0) | 10 (52.6) |
| GGT increased | 3 (33.3) | 4 (40.0) | 7 (36.8) |
| Blood alkaline phosphatase increased | 0 | 2 (20.0) | 2 (10.5) |
| Metabolism and nutrition disorders | 9 (100.0) | 8 (80.0) | 17 (89.5) |
| Decreased appetite | 7 (77.8) | 7 (70.0) | 14 (73.7) |
| Hypoalbuminemia | 6 (66.7) | 0 | 6 (31.6) |
| Hypokalemia | 1 (11.1) | 2 (20.0) | 3 (15.8) |
| Hypocalcemia | 2 (22.2) | 0 | 2 (10.5) |
| Nervous system disorders | 5 (55.6) | 6 (60.0) | 11 (57.9) |
| Dysgeusia | 2 (22.2) | 6 (60.0) | 8 (42.1) |
| Psychiatric disorders | 2 (22.2) | 1 (10.0) | 3 (15.8) |
| Anxiety | 2 (22.2) | 0 | 2 (10.5) |
| Renal and urinary disorders | 4 (44.4) | 0 | 4 (21.1) |
| Acute kidney injury | 2 (22.2) | 0 | 2 (10.5) |
| Respiratory, thoracic and mediastinal disorders | 3 (33.3) | 1 (10.0) | 4 (21.1) |
| Pleural effusion | 2 (22.2) | 0 | 2 (10.5) |
| Pulmonary edema | 2 (22.2) | 0 | 2 (10.5) |
| Skin and subcutaneous tissue disorders | 8 (88.9) | 4 (40.0) | 12 (63.2) |
| Alopecia | 1 (11.1) | 3 (30.0) | 4 (21.1) |
| Rash maculopapular | 2 (22.2) | 2 (20.0) | 4 (21.1) |
| Skin hyperpigmentation | 4 (44.4) | 0 | 4 (21.1) |
| Dry skin | 2 (22.2) | 0 | 2 (10.5) |
| Vascular disorders | 2 (22.2) | 0 | 2 (10.5) |
| Capillary leak syndrome | 1 (11.1) | 0 | 1 (5.3) |
| Hypertension | 1 (11.1) | 0 | 1 (5.3) |
TEAEs were defined as AEs which started on the day of or any day after treatment initiation with thiotepa, up to Day 28 post-HSCT. All AEs were coded using MedDRA dictionary version 19.1. Multiple reports of AEs that map to a common System Organ Class and Preferred Term counted only once for each patient
AE adverse event, ALT alanine aminotransferase, AST aspartate aminotransferase, GTT γ-glutamyltransferase, MedDRA Medical Dictionary for Regulatory Activities, TEAE treatment-emergent AE
aTEAEs with a causal relationship determined by the investigator as ‘possibly’, ‘probably’, or ‘definitely’ related to thiotepa