Monocentric experience of venetoclax‐based regimen in paediatric refractory and relapsed <scp>AML</scp>/<scp>MDS</scp>
BCL-2 inhibitor venetoclax demonstrates promising efficacy in paediatric relapsed/refractory acute myeloid leukaemia (r/r AML). This retrospective analysis evaluated 12 patients treated with venetoclax-based regimens under compassionate use for r/r myeloid malignancies. The overall response rate (ORR) was 41.6%, with complete response (CR) achieved in 33% of patients. Three patients successfully underwent allogeneic haematopoietic scell transplantation (HSCT) after venetoclax bridging therapy. Venetoclax demonstrated a favourable safety profile with manageable side effects. These findings suggest venetoclax's potential as a valuable therapeutic option for paediatric r/r AML, particularly for heavily pretreated patients. Further investigation in larger multicentre trials is warranted to refine treatment strategy.
Authors
- Benoît Brethon (ORCID: https://orcid.org/0000-0002-0663-3429)
- André Baruchel (ORCID: https://orcid.org/0000-0003-0120-6089)
- Julie Roupret‐Serzec
- Jean‐Hugues Dalle (ORCID: https://orcid.org/0000-0002-8406-0793)
- Lauriane Goldwirt (ORCID: https://orcid.org/0000-0001-9741-803X)
- Marion Strullu (ORCID: https://orcid.org/0000-0002-9229-0150)
- Marie-Émilie Dourthe (ORCID: https://orcid.org/0000-0002-8326-5973)
- Mathieu Simonin (ORCID: https://orcid.org/0000-0002-0448-8811)
- Charlotte Calvo (ORCID: https://orcid.org/0000-0002-6628-6342)
- Sophie Cousson
Institutions
- Université Paris Cité (FR)
- Sorbonne Université (FR)
- Hôpital Armand-Trousseau (FR)
- Sorbonne Paris Cité (FR)
- Assistance Publique – Hôpitaux de Paris (FR)
Publication Details
- Journal
- British Journal of Haematology
- Published
- 2024-10-24
- DOI
- https://doi.org/10.1111/bjh.19849
- Citations
- 1
- Primary Topic
- Acute Myeloid Leukemia Research
- Type
- article
- Field-Weighted Citation Impact
- 0.45