Wilson, Abbie and Moussa, Ahmed and Trinquand, Amélie and Malone, Andrea and Tewari, Sanjay and Calvert, Richard and Patrick, Katharine and Nicholson, Emma and Smith, Katy and Grandage, Victoria and Baird, Susan and George, Lindsay and Qureshi, Amrana and Borg, Albert and Gibson, Brenda and Patel, Pritesh and Bartram, Jack and Samrin, Lamia and O'Connor, David (2024) Real‐world use of venetoclax in the treatment of paediatric and teenage/young adult haematological malignancies. British Journal of Haematology, 205 (6). pp. 2355-2362. ISSN 0007-1048

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Abstract

Early-phase trials of venetoclax in children and teenagers/young adults with leukaemia have yielded promising results, but there remains a paucity of real-world data. To address this, we report a cohort of 41 children treated with venetoclax for a range of haematological malignancies, demonstrating complete remission in 43.6%, with most achieving minimal residual disease (MRD) negativity. Venetoclax was particularly effective as a bridge to transplant, with bridging successful in 75% of patients. Patients with MRD &lt;1% at initiation of venetoclax were more likely to achieve MRD negativity (81.8% vs. 34.5%, p = 0.007) and had improved overall survival (54.5% vs. 17.9%, p = 0.004).

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