Real-world use of venetoclax in the treatment of paediatric and teenage/young adult haematological malignancies
Wilson, Abbie Moussa, Ahmed Trinquand, Amélie Malone, Andrea Tewari, Sanjay Calvert, Richard Patrick, Katharine Nicholson, Emma Smith, Katy Grandage, Victoria
Wilson, Abbie
Moussa, Ahmed
Trinquand, Amélie
Malone, Andrea
Tewari, Sanjay
Calvert, Richard
Patrick, Katharine
Nicholson, Emma
Smith, Katy
Grandage, Victoria
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2024-09-26
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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 <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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Wilson A, Moussa A, Trinquand A, Malone A, Tewari S, Calvert R, Patrick K, Nicholson E, Smith K, Grandage V, Baird S, George L, Qureshi A, Borg A, Gibson B, Patel P, Bartram J, Samrin L, O'Connor D. Real-world use of venetoclax in the treatment of paediatric and teenage/young adult haematological malignancies. Br J Haematol. 2024 Dec;205(6):2355-2362. doi: 10.1111/bjh.19791. Epub 2024 Sep 26.
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