Introduction
Patients and methods
Study design and treatment
Patients
Treatment
Pediatric patients with solid tumors or brain tumors
Adult malignant lymphoma
Assessments
Pharmacokinetic assessment
Analytical methods for assessing plasma concentration
Safety
Efficacy
Statistical analysis
Results
Patient demographics
Pediatric solid tumor or pediatric brain tumor N = 9 | Adult malignant lymphoma N = 10 | |
---|---|---|
Sex | ||
Female, n (%) | 4 (44.4) | 4 (40.0) |
Male, n (%) | 5 (55.6) | 6 (60.0) |
Age, years | ||
Mean (SD) | 7.0 (5.9) | 53.7 (11.6) |
Median (min, max) | 5.0 (2, 16) | 54.5 (35, 68) |
Height, cm, mean (SD) | 114.3 (34.4) | 164.7 (10.2) |
Weight, kg, mean (SD) | 23.0 (14.4) | 60.0 (12.1) |
Body surface area,a m2, mean (SD) | 0.85 (0.40) | 1.65 (0.20) |
ECOG performance status,bn (%) | ||
0 | 4 (44.4) | 7 (70.0) |
1 | 5 (55.6) | 3 (30.0) |
Primary malignancy, n (%) | ||
Pediatric solid tumor | 4 (44.4) | 0 |
Pediatric brain tumor | 5 (55.6) | 0 |
Malignant lymphoma | 0 | 10 (100.0) |
Relapse, n (%) | 2 (22.2) | 6 (60.0) |
Number of previous HSCTs, n (%) | ||
0 | 6 (66.7) | 10 (100.0) |
1 | 3 (33.3) | 0 |
Patients with complicationsc | 6 (66.7) | 9 (90.0) |
Pharmacokinetics
Safety
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) |