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Experience with stem cell collection for children with Transfusion-dependent thalassemia undergoing gene therapy

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TL;DR

This study reviews stem cell collection in 13 children with transfusion-dependent thalassemia undergoing gene therapy, highlighting that most achieved adequate CD34+ cell yields through multiple collections without adverse reactions, and emphasizing the need to improve mobilization regimens and perform saturated transfusions prior to collection.

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To explore the characteristics and strategies of stem cell collection in children with Transfusion-dependent thalassemia (TDT) undergoing gene therapy. To summarize the challenges encountered and the countermeasures taken during stem cell collection in 13 TDT children undergoing gene therapy at the Department of Pediatrics, 923rd Hospital of the Joint Logistics Support Force of PLA between August 2020 and January 2024. Among the 13 TDT children, one required 1 collection, nine required 2 collections, one required 3 collections, and one required 4 collections. Adequate cells were collected in 12 cases, while 1 case had failed to obtain sufficient CD34+ cells despite multiple rounds of collection. The stem cell collection process was smooth in all 13 cases, with no adverse reactions. The stem cell mobilization regimen for TDT children needs to be enhanced, and saturated blood transfusion should be performed prior to stem cell collection.

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