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@ARTICLE{Kwiatkowski:307629,
      author       = {J. L. Kwiatkowski and A. A. Thompson and J. Schneiderman
                      and I. Thuret and A. Kulozik$^*$ and E. Yannaki and M.
                      Cavazzana and S. Hongeng and T. S. Olson and M. G. Sauer and
                      A. J. Thrasher and A. Lal and J. E. Rasko and J. B. Kunz and
                      M. A. Kinney and A. Chawla and S. Ali and G. Tao and H.
                      Thakar and C. Paramore and N. Witthuhn and M. C. Walters and
                      F. Locatelli},
      title        = {{L}ong-term efficacy and safety results of betibeglogene
                      autotemcel gene therapy for transfusion-dependent
                      β-thalassemia.},
      journal      = {Blood},
      volume       = {nn},
      issn         = {0006-4971},
      address      = {Washington, DC},
      publisher    = {American Society of Hematology},
      reportid     = {DKFZ-2026-00101},
      pages        = {nn},
      year         = {2026},
      note         = {epub},
      abstract     = {Betibeglogene autotemcel (beti-cel) gene therapy for
                      transfusion-dependent β-thalassemia (TDT) involves
                      autologous transplantation of hematopoietic stem and
                      progenitor cells transduced with a modified β-globin gene
                      to produce functional adult hemoglobin (HbAT87Q).
                      Sixty-three participants with TDT (median [range] age: 17
                      [4-35] years) received beti-cel in phase 1/2 (n = 22) or
                      phase 3 (n = 41) studies and enrolled in the long-term
                      follow-up LTF-303 study (clinicaltrials.gov/NCT02633943;
                      median [range] follow-up: 5.9 [2.9-10.1] years).
                      Manufacturing refinements in phase 3 increased transduction
                      efficiency, resulting in higher drug product vector copy
                      number and HbAT87Q levels, which translated into higher
                      hemoglobin and transfusion independence (TI) rates compared
                      with phase 1/2. TI was achieved by $68.2\%$ (15/22) of phase
                      1/2 participants (median weighted average Hb during TI, 10.2
                      g/dL) and $90.2\%$ (37/41) of phase 3 participants (median,
                      11.2 g/dL) and was sustained through last follow-up.
                      Treatment efficacy was similar across ages and TDT
                      genotypes. Among participants achieving TI, $73\%$ (38/52)
                      had discontinued iron chelation at last follow-up, with no
                      increase in liver iron concentration. Markers of ineffective
                      erythropoiesis, including serum transferrin receptor and
                      erythropoietin, improved with restoration of iron
                      homeostasis. Health-related quality-of-life assessment
                      scores showed durable improvements. No malignancies,
                      insertional oncogenesis, or vector-derived
                      replication-competent lentivirus were reported. These
                      findings establish beti-cel as a durable, one-time therapy
                      that achieves TI, restores iron balance, and improves
                      quality of life, offering a potentially curative treatment
                      option for people with TDT.},
      cin          = {A400},
      ddc          = {610},
      cid          = {I:(DE-He78)A400-20160331},
      pnm          = {311 - Zellbiologie und Tumorbiologie (POF4-311)},
      pid          = {G:(DE-HGF)POF4-311},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:41525466},
      doi          = {10.1182/blood.2025029196},
      url          = {https://inrepo02.dkfz.de/record/307629},
}