Development of clinical paroxysmal nocturnal haemoglobinuria in children with aplastic anaemia. (23rd June 2017)

• Record Type: Journal Article
• Authors: 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
• Is Part Of: British journal of haematology. Volume 178:Number 6(2017)
• Page Start: 954
• Page End: 958
• Publication Date: 2017-06-23
• Subjects: paroxysmal nocturnal haemoglobinuria -- aplastic anaemia -- PIGA -- Children; Hematology -- Periodicals; Blood -- Diseases -- Periodicals; 616.15
• DOI: 10.1111/bjh.14790
• Languages: English
• ISSNs: 0007-1048

Abstract

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.