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Development of clinical paroxysmal nocturnal haemoglobinuria in children with aplastic anaemia
Author(s) -
Narita Atsushi,
Muramatsu Hideki,
Okuno Yusuke,
Sekiya Yuko,
Suzuki Kyogo,
Hamada Motoharu,
Kataoka Shinsuke,
Ichikawa Daisuke,
Taniguchi Rieko,
Murakami Norihiro,
Kojima Daiei,
Nishikawa Eri,
Kawashima Nozomu,
Nishio Nobuhiro,
Hama Asahito,
Takahashi Yoshiyuki,
Kojima Seiji
Publication year - 2017
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.14790
Subject(s) - medicine , aplastic anemia , paroxysmal nocturnal hemoglobinuria , pediatrics , immunology , bone marrow
Summary The clinical significance of paroxysmal nocturnal haemoglobinuria ( PNH ) in children with aplastic anaemia ( AA ) remains unclear. We retrospectively studied 57 children with AA between 1992 and 2010. During the follow‐up, five patients developed clinical PNH , in whom somatic PIGA mutations were detected by targeted sequencing. The 10‐year probability of clinical PNH development was 10·2% (95% confidence interval, 3·6–20·7%). Furthermore, the detection of minor PNH clones by flow cytometry at AA diagnosis was a risk factor for the subsequent development of clinical PNH . These patients with PNH clones at AA diagnosis should undergo periodic monitoring for potential clinical PNH development.