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. 2025 Jan;206(1):204-208.
doi: 10.1111/bjh.19843. Epub 2024 Nov 17.

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

Aditi Vedi  1   2 , Sarah Maria Leiter  1 , Irum Latif Memon  3 , Arunthethy Mahendrayogam  1 , Elsje Van Rijswijk  4 , Urmila Uparkar  4 , Geoff Shenton  5 , Amelie Trinquand  6 , Katherine Clesham  7 , Vanessa McLelland  8 , Helen Campbell  9 , Katharine Patrick  10 , Lyndsey Thompson  11 , Susan Baird  12 , Philip Connor  13 , Donna Lancaster  4 , Beki James  14
Affiliations

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

Aditi Vedi et al. Br J Haematol. 2025 Jan.

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.

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Conflict of interest statement

No conflicts of interest declared.

Figures

FIGURE 1
FIGURE 1
Treatment combinations, funding source for ATO and survival of paediatric APL in the United Kingdom, 2014–2021. (A) Bar chart representing treatment combinations used to treat non‐HR and HR patients at diagnosis in this cohort. (B) Bar chart representing source of funding for ATO for SR and HR patients. p‐values calculated using Fisher's exact test. (C) Kaplan–Meier survival curves for patients by risk group in this cohort. (Left) Overall survival, (Right) Progression‐free survival. ATO, arsenic trioxide; ATRA, all‐trans retinoic acid; HR, high risk; Ida, Idarubicin; SR, standard risk.
See this image and copyright information in PMC

References

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