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Patients aged less than 3 years with acute myeloid leukaemia characterize a molecularly and clinically distinct subgroup
Author(s) -
Hara Yusuke,
Shiba Norio,
Yamato Genki,
Ohki Kentaro,
Tabuchi Ken,
Sotomatsu Manabu,
Tomizawa Daisuke,
Kinoshita Akitoshi,
Arakawa Hirokazu,
Saito Akiko M.,
Kiyokawa Nobutaka,
Tawa Akio,
Horibe Keizo,
Taga Takashi,
Adachi Souichi,
Taki Tomohiko,
Hayashi Yasuhide
Publication year - 2020
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/bjh.16203
Subject(s) - medicine , cumulative incidence , incidence (geometry) , subgroup analysis , oncology , overall survival , gastroenterology , cohort , confidence interval , physics , optics
Summary Although infants (age <1 year) with acute myeloid leukaemia (AML) have unique characteristics and are vulnerable to chemotherapy, children aged 1–2 years with AML may have characteristics similar to that of infants. Thus, we analysed 723 paediatric AML patients treated on the Japanese AML99 and AML‐05 trials to identify characteristics of younger children. We identified patients aged <3 years (the younger group) as a distinct subgroup. KMT2A ‐rearrangement ( KMT2A ‐R), CBFA2T3‐GLIS2 , CBFB‐MYH11 and NUP98‐KDM5A were frequently found in the younger group. Prognostic analyses revealed poor 5‐year overall survival (OS), event‐free survival (EFS) and cumulative incidence of relapse (CIR) in patients with CBFA2T3‐GLIS2 (42%, 17% and 83%, respectively) and those with NUP98‐KDM5A (33%, 17% and 83%, respectively). Additionally, we identified KMT2A ‐R and CBFB‐MYH11 as age‐specific prognostic markers. Regarding KMT2A ‐R, the younger group had significantly better OS, EFS and CIR than the older group (aged 3 to <18 years) ( P = 0·023, 0·011 and <0·001, respectively). Conversely, concerning CBFB‐MYH11 , the younger group had significantly poor EFS and CIR than the older group (each P < 0·001), suggesting that certain molecular markers are linked to different prognoses according to age. Therefore, we characterized patients <3 years as a distinct subgroup of paediatric AML.