Table 3 Summary of CTCAE case reports and number of the drug-related deaths

IV^b Ribavirin [32]

Severe Adenovirus Disease

5

5

Severe Adenovirus Disease (n = 3, 60.0%)

–

Nivolumab [34]

Pediatric glioblastoma

1

5

Malignant cerebral edema and herniation (n = 1, 100%)

1

Miransertib [35]

Pik3ca-related overgrowth syndrome

2

1

Hyperlipidaemia (n = 1, 50.0%)

–

Loose stools (n = 1, 50.0%)

Nirogacestat [36]

Desmoid tumors

1

2

Diarrhea (n = 1, 100.0%)

–

AL101 [40]

Desmoid tumors

1

1

Fatigue (n = 1, 100.0%)

–

2

Nausea (n = 1, 100.0%)

–

Weight decreased (n = 1,100.0%)

3

Diarrhea (n = 1,100.0%)

–

Lorlatinib [44]

Inflammatory myofibroblastic tumour

1

1

Oral stomatitis, insomnia, paronychia (n = 1,100.0%) each

2

Hyperlipidaemia (n = 1,100.0%)

Defibrotide [45]

Hepatic veno-occlusive disease

19

1–2

3–4

Nausea (n = 6, 31.6%)

–

Transient mild systolic hypotension (n = 5, 26.3%)

Fever (n = 5,26.3%)

Abdominal cramping (n = 3, 15.8%)

Vasomotor symptoms (n = 1, 5.3%)

Sepsis (n = 7, 36.8%)

–

Pulmonary edema (n = 5, 26.3%)

Cytomegalovirus infection (n = 2, 10.5%)

Hypotension (n = 2, 10.5%)

Respiratory failure (n = 2, 10.5%)

Diarrhea, bronchospasm, alveolar hemorrhage, renal failure, gastrointestinal bleeding, supraventricular arrhythmia, and encephalopathy (n = 1,5.3%) each

Bedaquiline [46]

Multidrug-resistant tuberculosis

82

5

Respiratory failure (n = 3, 3.6%)

Cardiopulmonary failure (n = 2, 2.4%)

Peripheral oedema, Nephrotic syndrome, Tuberculosis, Committed suicide, Tuberculosis disease progression, and Myocardial infarction (n = 1, 1.2%) each

2

Gemtuzumab

Ozogamicin [47]

AML^c, high-risk myelodysplastic syndrome, or acute promyelocytic leukemia

331

3–4

5

Anemia (n = 142, 42.9%)

WBC^d count decrease (n = 120, 36.3%)

Platelet count decrease (n = 118, 35.6%)

Febrile neutropenia (n = 120, 36.3%)

Disease progression (n = 48, 14.5%)

Lymphocyte count decrease (n = 81, 24.5%)

Hypokalemia (n = 66, 19.9%)

Neutrophil count decrease (n = 79, 23.9%)

Lung infection (n = 13, 3.9%)

–

Disease progression (n = 46, 13.9%)

AML (n = 16, 4.8%)

Sepsis (n = 7, 2.1%)

Respiratory failure (n = 5,1.5%)

Intracranial hemorrhage (n = 1, 0.3%)

Pulmonary hemorrhage, respiratory distress,

sepsis, and multiple organ dysfunction

syndrome (n = 2, 0.6%) each

15

Ropeginterferon

Alpha-2b (Besremi) [50]

[Ph (−)]^e myeloproliferative neoplasms

9

1

Anemia, thrombocytopenia, depression, and bone pain (n = 1, 11.1%) each

Mucositis (n = 2, 22.2%)

Dizziness (n = 2, 22.2%)

Alopecia (n = 3, 33.3%)

–

2

Anemia, leukopenia, and neuropathy (n = 2, 22.2%) each

Headache, insomnia, and transaminitis (n = 1, 11.1%) each

–

3

Anemia, leukopenia, thrombocytopenia, and transaminitis (n = 1, 11.1%) each

–

4

Anemia (n = 1, 11.1%)

–

Aflibercept [51]

Colorectal cancer

5

1

Alopecia, hypertension, dysphonia, epistaxis conjunctival toxicity, and acute neurotoxicity (n = 1, 20.0%) each

Diarrhoea (n = 2, 40.0%)

–

2

Proteinuria, palmar plantar erythrodysesthesia, alopecia, fatigue, nausea, diarrhoea, neutropenia,respiratory infection, and mucositis (n = 1, 20.0%) each

Hypertension (n = 2, 40.0%)

Hyporexia (n = 2, 40.0%)

–

–

3

Neutropenia, hypertension, and fatigue (n = 1, 20.0%) each

Hydralazine and

Valproate

(TRANSKRIP™) [52]

Myelodysplastic syndrome

14

1–2

Distal tremor (n = 9, 64.3%)

Edema (n = 8, 57.1%)

Nausea (n = 7, 50.0%)

Drowsiness (n = 6, 42.9%)

Headache (n = 5, 35.7%)

Skin rash (n = 1, 7.1%)

–

Letermovir [59]

CMV^f infection

80

5

Graft-versus-host disease (n = 5, 6.2%)

CMV infection (n = 2, 2.5%)

Adenovirus colitis, influenza infection, leukemia relapse, cardiac arrest, and adenovirus pneumonia (n = 1, 1.2%) each

1

Gemtuzumab

Ozogamicin [62]

AML

24

3–4

Neutropenia and/or thrombocytopenia (n = 24, 100%)

–

5

Veno-occlusive disease (n = 1, 4.2%)

Septic shock (n = 1, 4.2%)

–

Abiraterone

Acetate [63]

Prostate cancer

368

3–4

Pain (n = 23, 6.3%)

Nausea (n = 5, 1.4%)

Vomiting (n = 2, 0.5%)

Fatigue (n = 25, 6.8%)

Peripheral edema (n = 3, 0.8%)

Cardiac disorders (n = 3, 0.8%)

Anemia (n = 51, 13.9%)

Neutropenia (n = 7, 1.9%)

Thrombocytopenia (n = 15, 4.1%)

Liver enzymes (n = 13, 3.5%)

Hypokalemia (n = 27, 7.3%)

Hypertension (n = 13, 3.5%)

–

Avapritinib [65]

Gastrointestinal stromal tumour

1

2

Bilateral lower limb oedema (n = 1, 100.0%)

–

Imatinib [58]

Chordoma

48

 ≤ 2

Oedema (n = 22, 45.8%)

Gastritis (n = 1, 2.1%)

–

 ≥ 3

Oedema (n = 2, 4.2%)

Nausea (n = 4, 8.4%)

Neutropenia (n = 1, 2.1%)

Ulixertinib [66]

BRAF D594G cutaneous melanoma

1

1

2

Mucositis, rash, and diarrhea (n = 1, 100.0%) each

–

Intermittent visual floaters (n = 1, 100.0%)

–

Avapritinib [70]

Mucosal melanoma and thymic carcinoma

4

2

Vasculitis, uveitis, anaemia, and thrombocytopaenia (n = 1, 25.0%) each

–