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

IF 3.8 2区医学 Q1 HEMATOLOGY British Journal of Haematology Pub Date : 2024-11-17 DOI:10.1111/bjh.19843

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

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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.

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英国和爱尔兰儿科急性早幼粒细胞白血病的实际经验。

由 t(15;17)(q24;q21)易位定义的急性早幼粒细胞白血病（APL）占儿科急性髓性白血病病例的 5%-10%。全反式维甲酸（ATRA）和三氧化二砷（ATO）是主要的治疗方法，但获得 ATO 的途径各不相同。我们对 2014 年至 2021 年间确诊的 50 例英国儿科 APL 患者的治疗、并发症和存活率进行了评估。所有患者均接受了ATRA治疗，大多数患者接受了ATO治疗。高危患者的无事件生存率较低（85% 对 100%，p = 0.03），诊断时未接受 ATO 治疗的患者的无事件生存率也较低。所有复发患者均可通过 ATO 得到救治。解决ATO的可用性和持续供资问题对于确保及时治疗和改善预后至关重要。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

British Journal of Haematology 医学-血液学

CiteScore

8.60

自引率

4.60%

发文量

565

审稿时长

1 months

期刊介绍： The British Journal of Haematology publishes original research papers in clinical, laboratory and experimental haematology. The Journal also features annotations, reviews, short reports, images in haematology and Letters to the Editor.