TREATMENT PATHWAY

Treatment with ZYNTEGLO is managed through
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 donor1

  • COLLECTION1
    (Approximately 2 months prior to ZYNTEGLO infusion)

    • Cell collection is required for treatment, since ZYNTEGLO uses the patient's own HSCs
    • 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 ≥1.5×106 CD34+ cells/kg (if collected by apheresis) or >1.0×108 TNC/kg (if collected by bone marrow harvest) is required

  • 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

  • CONDITIONING1
    (At least 6 days before ZYNTEGLO infusion)
    In clinical trials, a 4-day regimen of busulfan was the only myeloablative conditioning tested with ZYNTEGLO

    • 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 at least 30 days prior to mobilisation and during myeloablative conditioning
    • Iron chelation should be stopped at least 7 days prior to myeloablative conditioning
    • Prophylaxis for hepatic veno-occlusive disease (VOD) is recommended
    • Prophylaxis for seizures should be considered

  • 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

  • FOLLOW-UP CARE AND REGISTRY1
    (Registry 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
    • 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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Learn more about ZYNTEGLO
Qualified Treatment Centres