Real-world experience of paediatric acute promyelocytic leukaemia in the United Kingdom and Ireland

Aditi Vedi; Sarah Maria Leiter; Irum Latif Memon; Arunthethy Mahendrayogam; Elsje Van Rijswijk; Urmila Uparkar; Geoff Shenton; Amelie Trinquand; Katherine Clesham; Vanessa McLelland; Helen Campbell; Katharine Patrick; Lyndsey Thompson; Susan Baird; Philip Connor; Donna Lancaster; Beki James

doi:10.1111/bjh.19843

Real-world experience of paediatric acute promyelocytic leukaemia in the United Kingdom and Ireland

Br J Haematol. 2024 Nov 17. doi: 10.1111/bjh.19843. Online ahead of print.

Authors

Aditi Vedi^{1

2}, Sarah Maria Leiter¹, Irum Latif Memon³, Arunthethy Mahendrayogam¹, Elsje Van Rijswijk⁴, Urmila Uparkar⁴, Geoff Shenton⁵, Amelie Trinquand⁶, Katherine Clesham⁷, Vanessa McLelland⁸, Helen Campbell⁹, Katharine Patrick¹⁰, Lyndsey Thompson¹¹, Susan Baird¹², Philip Connor¹³, Donna Lancaster⁴, Beki James¹⁴

Affiliations

¹ Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK.
² Department of Paediatrics, University of Cambridge, Cambridge, UK.
³ Great Ormond Street Hospital, London, UK.
⁴ Royal Marsden Hospital, Surrey, UK.
⁵ Newcastle Hospitals NHS Foundation Trust, Newcastle, UK.
⁶ National Children's Cancer Service, Children's Health Ireland at Crumlin, Dublin, Ireland.
⁷ University College London Hospital, London, UK.
⁸ University Hospitals Bristol NHS Foundation Trust, Bristol, UK.
⁹ Manchester University Hospitals, Manchester, UK.
¹⁰ Sheffield Children's NHS Foundation Trust, Sheffield, UK.
¹¹ Royal Belfast Hospital for Sick Children, Belfast, UK.
¹² Royal Hospital for Children and Young People, Edinburgh, UK.
¹³ Health Care Research Wales, Cardiff, UK.
¹⁴ Leeds Children's Hospital, Leeds, UK.

PMID: 39551717
DOI: 10.1111/bjh.19843

Abstract

Acute promyelocytic leukaemia (APL), defined by the t(15;17)(q24;q21) translocation, accounts for 5%-10% of paediatric acute myeloid leukaemia cases. All-trans retinoic acid (ATRA) and arsenic trioxide (ATO) are key treatments, though ATO access varies. We evaluated treatment, complications and survival in 50 UK paediatric APL patients diagnosed between 2014 and 2021. All patients received ATRA and most received ATO. Event-free survival was lower in high-risk patients (85% vs. 100%, p = 0.03), and those not receiving ATO at diagnosis. All relapsed patients could be salvaged with ATO. Addressing ATO availability and consistent funding is crucial to ensure timely treatment and improve outcomes.

Keywords: APL; Leukaemia; Paediatric; all‐trans retinoic acid; arsenic trioxide.