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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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Authors

VDVismay DeshaniFAFarhan AnjumSSSrividhya Senthil

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Overview

This report reveals successful leukapheresis in seven patients with transfusion-dependent β-thalassemia, indicating effective gene therapy potential.

Key Points

  • To evaluate the real-world application and outcomes of apheresis in patients with transfusion-dependent β-thalassemia undergoing gene therapy.
  • Evaluated seven patients for hematopoietic stem cell mobilization using plerixafor and G-CSF followed by leukapheresis.
  • Collected and analyzed data on patient characteristics, apheresis parameters, and complications retrospectively.
  • Administered myeloablative busulfan conditioning chemotherapy to prepare for reinfusion.
  • Six out of seven patients achieved adequate CD34⁺ stem cell collection with a single apheresis cycle.
  • Patients showed varying WBC counts and CD34⁺ cell doses collected, with two achieving gene-edited cell doses exceeding the target threshold after processing.
  • The first patient underwent successful gene therapy and was discharged within four weeks, demonstrating effective engraftment.

Cite This Study

Deshani et al. (2025) studied this question.

synapsesocial.com/papers/69362f5a4fa91c937236db39https://doi.org/10.1182/blood-2025-5964
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Evaluating the efficacy and safety of gene therapy in transfusion-dependent β-thalassemia: A focus on hemolysis improvement and clonal hematopoiesis monitoring2025 · 1 citations
  2. 2Highly efficient collection and manufacture of autologous HSC gene therapy cell product for patients with sickle cell disease using a lentiviral vector containing a shmir targeting BCL11a2025 · 1 citations
  3. 3Rapid, efficient and durable fetal hemoglobin production following CS-101 treatment in transfusion-dependent β-thalassemia participants: An autologous, ex vivo edited CD34+ stem cell product using the innovative transformer base editor (tBE)2025 · 2 citations
  4. 4All 19 subjects with transfusion-dependent β-thalassemia achieved transfusion independence after treatment with RM-001 (autologous HBG1/2 promoter-modified CD34+ hematopoietic stem and progenitor cells)2025
  5. 5Successful gene therapy for transfusion-dependent α-thalassemia: a case report2026