Long-Term Follow-up of Subjects With Transfusion-Dependant β-Thalassemia Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector

Beta-thalassaemia

The number of subjects with malignancies, The number of subjects with immune-related AEs (e.g., autoimmune disorders, GVHD, opportunistic infections, HIV), The number of subjects with new or worsening hematologic disorders, The number of subjects with new or worsening neurologic disorders

βA-T87Q-globin expression in peripheral blood over time post-drug product infusion through last follow-up, including Year 5, Year 10, and Year 15, Proportion of subjects who meet the definition of transfusion independence (TI), defined as a weighted average Hb ≥ 9 g/dL without any pRBC transfusions for a continuous period of ≥ 12 months at any time after drug product infusion in parent study and/or Study LTF-303, Proportion of subjects who meet the definition of TI at yearly timepoints including Year 5, Year 10, and Year 15 post-drug product infusion, and at last follow-up, Characterization of TI: Time from drug product infusion to achievement of TI (in parent study or Study LTF-303); Duration of TI; Weighted average Hb during T, Characterization of transfusion reduction (TR): Reduction in annualized pRBC transfusion volume (mL/kg/year) from 6 months post-drug product infusion (parent study) through last followup of at least 50%, 60%, 75%, 90%, or 100% as compared to the annualized pRBC transfusion volume during the 2 years prior to parent study enrollment, Characterization of transfusion reduction (TR):Annualized pRBC transfusion volume (mL/kg/year) and frequency (number/year) from 6 months post-drug product infusion (parent study) through last follow-up as compared to the annualized pRBC transfusion requirements during the 2 years prior to parent study enrollment, Characterization of transfusion reduction (TR): Time from drug product infusion to last pRBC transfusion (in parent study or Study LTF-303), Characterization of transfusion reduction (TR): Time from last pRBC transfusion (in parent study or Study LTF-303) to last follow-up, Weighted average nadir Hb from 6 months post-drug product infusion (parent study) through last follow-up as compared to the weighted average nadir Hb during the 2 years prior to parent study enrollment, Unsupported total Hb levels over time through last follow-up, including Year 5, Year 10, and Year 15, Unsupported total Hb levels ≥ 10 g/dL, ≥ 11 g/dL, ≥ 12 g/dL, ≥ 13 g/dL, and ≥ 14 g/dL over time through last follow-up, including Year 5, Year 10, and Year 15, Iron burden over time and change from parent study baseline in iron burden at yearly timepoints through last follow-up, as measured by: Liver iron content by magnetic resonance imaging (MRI)/Superconducting Quantum Interference Device (SQUID) as available ; Cardiac T2* by MRI as available ; Serum ferritin, Chelation therapy use, including time from last use of chelation therapy to last follow-up and absence of chelation use for at least 6 months post-drug product infusion in parent study and/or Study LTF-303, Therapeutic phlebotomy use, including annualized frequency, Measures of dyserythropoiesis over time as compared to parent study baseline, assessed by the following parameters: Reticulocytes ; Nucleated RBCs, Health-related quality of life (HRQoL) over time as compared to parent study baseline, using the following validated tools as available and as appropriate: Pediatric Quality of Life Inventory (PedsQL) ; EuroQol-5D (EQ-5D; youth version, EQ-5D-Y) ; Short Form-36 (SF-36) v2 ; Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT)

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