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Patient Dies in Beam Trial of Sickle Cell Disease Candidate; Company Cites Conditioning.

Hum Gene Ther

December 2024

Genetic Engineering & Biotechnology News Mary Ann Liebert, Inc., publishers 140 Huguenot Street New Rochelle, NY 10801.

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Background: Peripheral T-cell lymphomas (PTCL) frequently result in relapsed or refractory diseases. Upfront autologous hematopoietic stem cell transplantation (ASCT) using the BEAM (carmustine, etoposide, cytarabine, and melphalan) regimen is recommended. However, relapses are common in PTCL, highlighting a critical need for improved survival outcomes in these patients.

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Article Synopsis
  • Hematopoietic stem cell transplantation (HSCT) is a treatment for blood cancers that can potentially cure patients, but it is linked to increasing cases of cardiac dysfunction over time.
  • A study of 55 HSCT patients found that 27.3% developed asymptomatic cardiac dysfunction one year post-transplant, with some classified as having moderate or mild dysfunction.
  • Key factors associated with increased risk of cardiac dysfunction included previous use of anthracyclines and undergoing the BEAM chemotherapy regimen as part of the conditioning process before HSCT.
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Article Synopsis
  • This study examines how monitoring training load affects wellness and performance in collegiate female gymnasts over one season.
  • The researchers found that training load and its acute to chronic workload ratio (ACWR) fluctuated, with positive correlations between wellness factors like mood and energy and both ACWR and training load.
  • The study proposes a new framework for tracking training load and wellness, suggesting it could benefit gymnasts of all ages and competition levels.
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Background: Transfusion-dependent β-thalassaemia (TDT) is a severe disease, resulting in lifelong blood transfusions, iron overload, and associated complications. Betibeglogene autotemcel (beti-cel) gene therapy uses autologous haematopoietic stem and progenitor cells (HSPCs) transduced with BB305 lentiviral vector to enable transfusion independence.

Methods: HGB-212 was a non-randomised, multicentre, single-arm, open-label, phase 3 study of beti-cel in patients with TDT conducted at eight centres in France, Germany, Greece, Italy, the UK, and the USA.

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