OH Epic formulary, available via Therapy Plan (treatment plan for mobilization and chemotherapy conditioning),

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• Indication: treatment of patients aged 12 years and older with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs)
• Service line: Hematology
• Formulary location: Inpatient (NOMH)
• Service location: Inpatient (NOMH)
• Patient population: adults, pediatrics aged ≥ 12 yrs
• Prior authorization required: Yes –via single case agreement
• Restriction/criteria of use (all will apply unless specified):
  • Authorization for treatment will be subject to single case agreement confirmation
  • Prescribed by or in consultation with a Hematology specialist
  • Diagnosed with sickle cell disease with recurrent vaso-occlusive crises (VOCs)
  • Initial criteria
    • Confirmation that hematopoietic stem cell (HSC) transplantation is appropriate for the patient before mobilization, apheresis and myeloablative conditioning are initiated.
    • Considered use of prophylaxis therapy for seizures prior to initiating myeloablative conditioning
    • Screen patients for HIV-1, HIV-2, HBV, HCV, and any other infectious agents in accordance with local guidelines before collection of cells for manufacturing. Casgevy should not be used in patients with active HIV-1, HIV-2, HBV or HCV.
    • No history of hypersensitivity to dimethyl sulfoxide (DMSO) or dextran 40
    • No history of receiving other gene therapies [[e.g., Lyfgenia (lovotibeglogene autotemcel), Zynteglo (betibeglogene autotemcel), etc.]
    • Will not receive concomitant therapy with any of the following:
      • Iron chelators for 7-days prior to mobilization and 6 months post- treatment (3-months post-treatment for non-myelosuppressive iron chelators)
      • Disease-modifying agents (e.g., hydroxyurea, voxelotor, or crizanlizumab) for at least 8-weeks prior to mobilization and conditioning
  • Sickle cell disease
    • Symptomatic disease despite treatment with hydroxyurea at any point in the past OR add-on therapy (e.g., crizanlizumab, voxelotor, etc.) OR has experienced
      intolerance
    • Experienced two or more vaso-occlusive event/crises (VOE/VOC) in the previous year
    • Patient will be transfused prior to apheresis to a total Hb ≤ 11 g/dL and a HbS level <30 %. Patient will be transfused at least 8 weeks prior to initiation of myeloablative conditioning.
    • Will not receive granulocyte-colon stimulating factor (G-CSF) for the mobilization of hematopoietic stem cells (HSC)

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