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

Vedi, A.; Leiter, SM.; Memon, IL.; Mahendrayogam, A.; Van Rijswijk, E.; Uparkar, U.; Shenton, G.; Trinquand, A.; Clesham, K.; McLelland, V.; Campbell, H.; Patrick, K.; Thompson, L.; Baird, S.; Connor, P.; Lancaster, D.; James, B.

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

All Authors

Vedi, A.

Leiter, SM.

Memon, IL.

Mahendrayogam, A.

Van Rijswijk, E.

Uparkar, U.

Shenton, G.

Trinquand, A.

Clesham, K.

McLelland, V.

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LTHT Author

James, Beki

LTHT Department

Leeds Children's Hospital

Publication Date

2025

Item Type

Journal Article

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.