αß T-cell depleted haploidentical stem cell transplantation for pediatric and young adult patients with transfusion-dependent thalassemia - Takeaways - MDSpire

αß T-cell depleted haploidentical stem cell transplantation for pediatric and young adult patients with transfusion-dependent thalassemia

  • By

  • Katharina Kleinschmidt

  • Gina Penkivech

  • Anja Troeger

  • Juergen Foell

  • Tarek Hanafee-Alali

  • Stefanie Leszczak

  • Marcus Jakob

  • Sonja Kramer

  • Silke Kietz

  • Petra Hoffmann

  • Claudia Behrendt-Böhm

  • Carina Kaess

  • Andreas Brosig

  • Robert Offner

  • Daniel Wolff

  • Selim Corbacioglu

  • March 18, 2025

  • 0 min

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  • 1

    Transfusion-dependent beta-thalassemia (TDT) requires curative allogenic hematopoietic stem cell transplantation (HSCT) due to complications from iron overload.

  • 2

    Matched donor HSCT is the standard treatment for TDT, but donor availability is limited, especially in ethnic minorities.

  • 3

    Haploidentical HSCT (haplo-HSCT) provides an alternative, with two regimens: ex vivo T-cell depletion and in vivo T-cell depletion using post-transplantation cyclophosphamide.

  • 4

    In a study of 20 TDT patients, T-haplo-HSCT showed overall survival rates of 84-90%, indicating its feasibility and efficacy.

  • 5

    The conditioning regimen for haplo-HSCT included thiotepa, fludarabine, and treosulfan, with no pretransplant pharmacologic immunosuppression applied.

Original Source(s)

αß T-cell depleted haploidentical stem cell transplantation for pediatric and young adult patients with transfusion-dependent thalassemia

  • by Katharina Kleinschmidt, Gina Penkivech, Anja Troeger, Juergen Foell, Tarek Hanafee-Alali, Stefanie Leszczak, Marcus Jakob, Sonja Kramer, Silke Kietz, Petra Hoffmann, Claudia Behrendt-Böhm, Carina Kaess, Andreas Brosig, Robert Offner, Daniel Wolff, Selim Corbacioglu

  • March 18, 2025

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