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Original research
Ref Number: APCP-OP-9

Stem Cell Mobilisation and Collection Strategies for Autologous Gene Editing in Transfusion-Dependent β-Thalassaemia

Lin Yu¹, Zifeng Li¹
1- Children's Hospital of Fudan University, China
Corresponding Author: Lin Yu (wingyl0971@163.com)
Abstract Body
Introduction: Autologous gene editing offers a potential cure for transfusion-dependent β- thalassaemia (TDT), provided sufficient CD34+ haematopoietic stem/progenitor cells (HSPCs) can be safely mobilised and collected. TDT patients often present with factors affecting mobilisation efficiency , including iron overload and splenectomy . Objective: To evaluate the feasibility , safety , and efficacy of CD34+ cell mobilisation and collection in TDT patients prior to gene editing therapy . Methods: TDT patients underwent mobilisation with G-CSF ± plerixafor. Apheresis was initiated when peripheral blood CD34+ count reached threshold. Collection outcomes, adverse events, and impact of splenectomy were analysed. Results: Fifty-four percent of patients achieved target CD34 + cell yield. One patient required a second mobilisation to reach target. Splenectomised patients showed higher peak CD34+ counts but required greater leucocyte expansion. Median CD34+ yield was 16.82×10/kg after mean processed blood volume of 7823 mL. All patients required central venous access; no catheter-related infections or thrombotic events occurred. Mild to moderate citrate reactions were common but manageable with calcium supplementation. Grade ≥2 thrombocytopenia occurred in 5.26% of patients without bleeding events. Conclusion: Autologous CD34 + cell mobilisation and collection are feasible and safe in TDT. Splenectomy may enhance mobilisation but necessitates intensified regimens. Second mobilisation is an effective rescue strategy . Standardised supportive care and vascular access management are essential for successful integration into gene editing pathways.
Keywords: Stem cell mobilisation; Gene editing; Transfusion-dependent β-thalassaemia
Presentation Type: Original research
Disclaimer: The Views and opinions expressed in the articles are of the authors and not of the journal.
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