TREATMENT PATHWAY
Treatment with ZYNTEGLO is managed through
a highly coordinated process
a highly coordinated process
By using a patient’s own cells, ZYNTEGLO gives patients the potential to achieve transfusion independence without the need for a donor.1
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COLLECTION1
Approximately 2 months prior to ZYNTEGLO infusion- Cell collection is required for treatment, since ZYNTEGLO uses the patient's own haematopoietic stem cells
- Includes mobilisation followed by apheresis
- Mobilisation and apheresis may be repeated to ensure collection of sufficient stem cells for treatment
- A back-up collection of CD34+ stem cells of at least 1.5×106 CD34+ cells/kg (if collected by apheresis) or >1.0×108 TNC/kg (if collected by bone marrow harvest) is required
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MANUFACTURING1
- After cell collection, the patient’s cells are shipped to the licensed manufacturing facility, where they are genetically modified with functional copies of βA-T87Q-globin gene in order to manufacture ZYNTEGLO
- ZYNTEGLO is then cryopreserved and, after passing quality release testing, stored until ready to be shipped to the ZYNTEGLO Qualified Treatment Centre
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CONDITIONING1
At least 6 days before ZYNTEGLO infusion- The treating physician should confirm that HSC transplantation is appropriate for the patient before myeloablative conditioning is initiated
- Full myeloablative conditioning (a chemotherapy given over 4 days) must be administered before infusion of ZYNTEGLO
- It is recommended that patients maintain Hb ≥11 g/dL for 30 days prior to myeloablative conditioning
- Iron chelation should be stopped at least 7 days prior to myeloablative conditioning
- Prophylaxis for hepatic veno-occlusive disease (VOD) is recommended
- Depending on the myeloablative conditioning agent administered, prophylaxis for seizures should be considered
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INFUSION AND MONITORING1
In the hospital setting- ZYNTEGLO is administered to the patient via intravenous infusion at a Qualified Treatment Centre
- Per the clinical judgement of the treating physician, the patient will remain in the hospital until they are ready to be discharged
- NOTE: Restarting iron chelation after ZYNTEGLO infusion may be necessary and should be based on clinical practice; phlebotomy can be used in lieu of iron chelation, when appropriate
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REGISTRY1
For up to 15 years following administration of ZYNTEGLO- Every patient receiving ZYNTEGLO should be informed about and encouraged to enrol in the product registry for up to 15 years
- The registry collects data on safety and efficacy of treatment
