Real‐world use of venetoclax in the treatment of paediatric and teenage/young adult haematological malignancies

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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Publication Details

Journal
British Journal of Haematology
Published
2024-09-26
DOI
https://doi.org/10.1111/bjh.19791
Citations
4
Primary Topic
Acute Myeloid Leukemia Research
Type
article
Field-Weighted Citation Impact
1.82
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article

Real‐world use of venetoclax in the treatment of paediatric and teenage/young adult haematological malignancies

Victoria Grandage, Jack Luke Bartram, Amélie Trinquand, Katy Smith et al.
4 citations
British Journal of Haematology
Acute Myeloid Leukemia Research
1.82
article

Real‐world use of venetoclax in the treatment of paediatric and teenage/young adult haematological malignancies

Victoria Grandage, Jack Luke Bartram, Amélie Trinquand, Katy Smith, Abbie Wilson, Amrana Qureshi, Pritesh Patel, Albert Borg, David O’Connor, Emma Nicholson, Lamia Samrin, Ahmed Moussa, Sanjay Tewari, Katharine Patrick, Susan Baird, Brenda Gibson, Richard J. Calvert, Lindsay George, Andrea Malone
article en
4 citations

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).

British Journal of HaematologyVol. 205(6)
University College Hospital (GB), University Hospitals Birmingham NHS Foundation Trust (GB), Royal Marsden NHS Foundation Trust (GB), Institute of Cancer Research (GB), Great Ormond Street Hospital (GB), Royal Hospital for Children (GB), Children's Health Ireland at Crumlin (IE), Oxford University Hospitals NHS Trust (GB), Sheffield Children's Hospital (GB), University College London (GB)
Good health and well-being
Openalex Percentile: Top 14%
Acute Myeloid Leukemia Research
1.82
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