The incidence of acquired aplastic anaemia (AA) peaks in adolescents and young adults (AYA). Although age has been associated with response after immunosuppressive therapy (IST), few data exist about the specific outcome of AYA. We retrospectively compared the outcome of 29 children (aged <15 years), 32 AYA (15-25 years) and 23 adults (>25 years) with AA treated front-line with IST in Saint-Louis Hospital. The cumulative incidence of response was lower in adults compared with AYA (subdistribution hazard ratio [SHR] = 0·38, 95% confidence interval [CI] [0·96-1·00], P = 0·008), but no difference was observed between children and AYA (SHR = 0·84, 95% CI [0·96-1·00], P = 0·56), with a 6 months cumulative incidence of partial response of 44·8% in children, 62·5% in AYA and 21·7% in adults. The 5-year failure-free survival was 48·4%, without impact of age, with a 5-year relapse rate of 20·7%. With a median follow-up of 5·4 years, the 5-year overall survival was 86·5%, without significant difference between children and AYA overall survival (hazard ratio [HR] 1·51, 95% CI [0·25-9·02], P = 0·66), while adults displayed poorer survival than AYA (HR 4·98, 95% CI [1·00-24·73], P = 0·049). This study confirms that age is a prognostic factor in AA patients treated with IST. However, AYA patients have a similar outcome to children in terms of response rate and survival.
Keywords: adolescents and young adults; antithymocyte globulin; aplastic anaemia; ciclosporin; immunosuppressive therapy.
© 2018 British Society for Haematology and John Wiley & Sons Ltd.