About exagamglogene autotemcel
Autologous gene-edited stem cell therapy
Used for
- Treats sickle cell disease with recurrent vaso-occlusive crises in eligible patients
- Treats transfusion-dependent beta thalassemia in eligible patients
Usual adult dose
One intravenous infusion of the patient’s modified stem cells; dose is based on body weight and cell count.
Usual dose in children
For eligible patients aged 12 years and older: one intravenous infusion; dose is based on body weight and cell count.
Common side effects
- Low blood-cell counts and increased infection risk
- Mouth sores, nausea, vomiting, and abdominal pain
- Headache, fever, and fatigue
- Infusion reactions
Important warnings
- Requires myeloablative chemotherapy before infusion, which can cause severe complications
- May cause prolonged low blood counts, infections, or bleeding
- Fertility may be reduced; discuss fertility preservation before treatment
- Long-term monitoring is needed; effects on future cancers are not fully known
Pregnancy
Treatment is not recommended during pregnancy. Chemotherapy conditioning can harm an unborn baby; pregnancy testing and contraception are required around treatment.
Breastfeeding
Breastfeeding is not recommended during treatment and for at least one week after the last conditioning chemotherapy dose.
Interactions
- Conditioning chemotherapy and medicines that affect blood counts can increase toxicity
- Live vaccines should be avoided during immune recovery
AI-generated from general medical knowledge, not from this product's official leaflet. It may be incomplete or out of date. Not medical advice — ask a pharmacist or doctor before making any decision.