exagamglogene autotemcel: prescribing and clinical use
Focused clinical detail for use alongside current product information, local policy and patient-specific assessment.
exagamglogene autotemcel: clinical details
Prescribing considerations
- Confirm that autologous haematopoietic stem cell transplantation is appropriate and that licensed disease, age, genotype and donor criteria are met.
- Administration requires an authorised centre experienced in beta-haemoglobinopathies and stem cell transplantation.
- Cryopreserve unmodified rescue cells before conditioning.
- Verify patient identity, lot information and completeness of all patient-specific vials.
- Discuss fertility preservation and arrange long-term registry follow-up.
Contraindications and cautions
- Hypersensitivity to exagamglogene autotemcel or its excipients, with consideration of DMSO and dextran 40.
- Contraindications to required mobilisation or myeloablative conditioning medicines.
- Active HIV, hepatitis B or hepatitis C infection.
- Treatment is not recommended after previous autologous or allogeneic haematopoietic stem cell transplantation.
Monitoring
- Screen for relevant transmissible infections before cell collection.
- Monitor for infusion hypersensitivity during and after administration.
- Monitor full blood count, neutrophil and platelet engraftment, bleeding and infection.
- Assess organ function and complications associated with conditioning and transplantation.
- Maintain product, batch and patient traceability and arrange long-term safety follow-up.
Clinical pharmacology
Autologous CD34-positive haematopoietic stem and progenitor cells are edited ex vivo at the erythroid-specific BCL11A enhancer. Reduced BCL11A expression increases gamma-globin and fetal haemoglobin production after successful engraftment.
Formulation and product differences
- Patient-specific cryopreserved dispersion for intravenous infusion, supplied in one or more vials.
- Cell concentration and vial number vary by manufactured batch and patient.
- The cryopreservative contains DMSO and dextran 40; the product also contains sodium.
- It must remain deeply frozen until use and must not be refrozen, mixed with other medicines, altered or irradiated.
exagamglogene autotemcel interactions
The specialist team must review all medicines and vaccines before mobilisation, conditioning and infusion.
Hydroxyurea or hydroxycarbamide
Use around mobilisation and conditioning requires specialist management; experience after infusion is limited.
Voxelotor or crizanlizumab
Use around mobilisation and conditioning requires specialist management because interactions with preparative medicines are unknown.
Iron-chelating medicines
Require specialist management around conditioning and recovery because of interaction and bone-marrow suppression concerns.
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.