Dose
|
mg/kg; cells; vector particles…
|
250 mg/m2
|
10 mg/kg
|
≥8 × 10^6 cells
|
Schedule
|
Daily; 4× weekly w/2 wk holidays…
|
Weekly until disease progression
|
3× @ 2-wk intervals; 4-wk intervals after
|
|
Administration/target
|
Infusion; oral; intratumoral…
|
Intravenous infusion
|
Intravenous infusion
|
Intravenous infusion
|
Co-intervention
|
Radiotherapy; immunosuppresion…
|
|
Standard of care
|
Non-myeloablative conditioning
|
Risk mitigation
|
Cardiac monitoring; liver enzymes…
|
Serum electrolytes monitoring
|
Monitor patients for hypersensitivity
|
|
Timing delivery
|
Symptom onset; 12 h after event…
|
After irinotecan or oxaliplatin failure
|
|
3 weeks after stopping PEG-ADA
|
Population dimensions
|
Diagnostic criteria
|
Marker positivity; diagnostic score…
|
KRAS mutation in codon 12 or 13
|
α-nuclear Ab titer ≥ 1:80 and/or α-dsDNA
|
|
Contraindications
|
Concurrent infection; clothing disorder…
|
|
Prior anaphylaxis to belimumab
|
Neutralizing Abs to vector (AAV8)
|
Indications
|
Disease; injury; at-risk population…
|
Metastatic colorectal cancer
|
Active systemic lupus erythematosus (SLE)
|
ADA-SCID
|
Age
|
|
Adults
|
Non-geriatric adults
|
Children
|
Outcome dimensions
|
Endpoint
|
Change on scale; time to event…
|
Survival
|
SLE responder index
|
Immune system reconstitution
|
Duration |
Short term; ‘long term’ remission… |
|
1 year |
Permanent |