December 8, 2025Blood

Real-world experience with apheresis for gene therapy in transfusion-dependent β-thalassemia: The largest single-center report

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Observational analysis shows successful stem cell mobilization in transfusion-dependent β-thalassemia, suggesting gene therapy is viable.

Six out of seven patients achieved adequate CD34⁺ stem cell collections after apheresis, and one patient required a repeat cycle.
The total CD34⁺ cell dose of patient 1 was 70.24 × 10⁶/kg, with gene-edited cells comprising 18.5 × 10⁶/kg, both exceeding targets.
Assessment involved leukapheresis with plerixafor and G-CSF to mobilize hematopoietic stem cells from patients.
These findings highlight the potential for gene therapy to provide a curative treatment for β-thalassemia with minimal adverse events.

Deshani et al. (2025) studied this question.

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December 8, 2025Blood

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Authors

Observational analysis shows successful stem cell mobilization in transfusion-dependent β-thalassemia, suggesting gene therapy is viable.

Six out of seven patients achieved adequate CD34⁺ stem cell collections after apheresis, and one patient required a repeat cycle.
The total CD34⁺ cell dose of patient 1 was 70.24 × 10⁶/kg, with gene-edited cells comprising 18.5 × 10⁶/kg, both exceeding targets.
Assessment involved leukapheresis with plerixafor and G-CSF to mobilize hematopoietic stem cells from patients.
These findings highlight the potential for gene therapy to provide a curative treatment for β-thalassemia with minimal adverse events.

Deshani et al. (2025) studied this question.

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