Table 2.
Serious Adverse Events Following Gene Therapya
| Event | Study Phase | Duration, Days | Outcome | Maximum Toxicity Grade |
|---|---|---|---|---|
| Neutropeniab | 3-month follow-up | 29 | resolved | grade 4 |
| Pneumonia | 3-month follow-up | 46 | resolved | grade 4 |
| Autoimmune thrombocytopenia | 3-month follow-up | 12 | resolved | grade 3 |
| Device-related infection | 3-month follow-up | 12 | resolved | grade 3 |
| Device-related infection | 3-month follow-up | 16 | resolved | grade 3 |
| Epstein-Barr virus infection | 3-month follow-up | 9 | resolved | grade 3 |
| Hypertension | 3-month follow-up | 75 | resolved | grade 3 |
| Pancytopenia | 3-month follow-up | 22 | resolved | grade 3 |
| Sepsis (Staphylococcal) | 3-year follow-up | 9 | resolved | grade 4 |
| Aplastic anemia | 3-year follow-up | 120 | resolved | grade 3 |
| Autoimmune hepatitis | 3-year follow-up | 14 | resolved | grade 3 |
| Autoimmune thrombocytopenia | 3-year follow-up | 16 | resolved | grade 3 |
| Device-related infection | 3-year follow-up | 4 | resolved | grade 3 |
| Device-related infection | 3-year follow-up | 14 | resolved | grade 3 |
| Device-related infection | 3-year follow-up | 11 | resolved | grade 3 |
| Device-related infection | 3-year follow-up | 10 | resolved | grade 3 |
| Device-related infection | 3-year follow-up | 17 | resolved | grade 3 |
| Device-related infection | 3-year follow-up | 9 | resolved | grade 3 |
| Diarrhea | 3-year follow-up | 9 | resolved | grade 3 |
| Fever | 3-year follow-up | 4 | resolved | grade 3 |
| Fibrolipoma72 | 3-year follow-up | 4 | resolved | grade 3 |
| Gastroenteritis | 3-year follow-up | 6 | resolved | grade 3 |
| Gastroenteritis | 3-year follow-up | 7 | resolved | grade 3 |
| Guillain-Barré syndrome | 3-year follow-up | 4 | resolved | grade 3 |
| Meningitis | 3-year follow-up | 17 | resolved | grade 3 |
| Neutropenia | 3-month follow-up | 66 | resolved | grade 3 |
| Respiratory tract infection | 3-year follow-up | 3 | resolved | grade 3 |
| Upper respiratory tract infection (bacterial) | 3-year follow-up | 12 | resolved | grade 3 |
| Urinary tract infection | 3-year follow-up | 10 | resolved | grade 3 |
| Varicella | 3-year follow-up | 6 | resolved | grade 3 |
| Gastroenteritis | 3-year follow-up | 28 | resolved | grade 2 |
| Autoimmune aplastic anemia | 7-year follow-up | 10 | resolved | grade 3 |
| Pneumonia | 7-year follow-up | 10 | resolved | grade 3 |
| Pyoderma | 7-year follow-up | 20 | resolved | grade 3 |
| Varicella | 7-year follow-up | 8 | resolved | grade 3 |
| Fever | 7-year follow-up | 2 | resolved | grade 2 |
| Thermal burn | 7-year follow-up | 110 | resolved with sequelae | grade 2 |
| Pneumonia | ≥8-year follow-up | 8 | resolved | grade 3 |
| Urinary tract infection | ≥8-year follow-up | 4 | resolved | grade 2 |
a
Adapted from Cicalese et al.19
b
Identified as an event potentially related to autoimmunity (verbatim term: prolonged neutropenia [anti-neutrophils antibodies positive]).