Outcome of aplastic anaemia in children. A study by the severe aplastic anaemia and paediatric disease working parties of the European group blood and bone marrow transplant

<jats:title>Summary</jats:title><jats:p>This study analysed the outcome of 563 Aplastic Anaemia (<jats:styled-content style="fixed-case">AA</jats:styled-content>) children aged 0–12 years reported to the Severe Aplastic Anaemia Working Party database of the European Society for Blood and Marrow Transplantation, according to treatment received. Overall survival (<jats:styled-content style="fixed-case">OS</jats:styled-content>) after upfront human leucocyte antigen‐matched family donor (<jats:styled-content style="fixed-case">MFD</jats:styled-content>) haematopoietic stem cell transplantation (<jats:styled-content style="fixed-case">HSCT</jats:styled-content>) or immunosuppressive treatment (<jats:styled-content style="fixed-case">IST</jats:styled-content>) was 91% vs. 87% (<jats:italic>P</jats:italic> 0·18). Event‐free survival (<jats:styled-content style="fixed-case">EFS</jats:styled-content>) after upfront <jats:styled-content style="fixed-case">MFD HSCT</jats:styled-content> or <jats:styled-content style="fixed-case">IST</jats:styled-content> was 87% vs. 33% (<jats:italic>P</jats:italic> 0·001). Ninety‐one of 167 patients (55%) failed front‐line <jats:styled-content style="fixed-case">IST</jats:styled-content> and underwent rescue <jats:styled-content style="fixed-case">HSCT</jats:styled-content>. The <jats:styled-content style="fixed-case">OS</jats:styled-content> of this rescue group was 83% compared with 91% for upfront <jats:styled-content style="fixed-case">MFD HSCT</jats:styled-content> patients and 97% for those who did not fail <jats:styled-content style="fixed-case">IST</jats:styled-content> up‐front (<jats:italic>P</jats:italic> 0·017). Rejection was 2% for <jats:styled-content style="fixed-case">MFD HSCT</jats:styled-content> and <jats:styled-content style="fixed-case">HSCT</jats:styled-content> post‐<jats:styled-content style="fixed-case">IST</jats:styled-content> failure (<jats:italic>P</jats:italic> 0·73). Acute graft‐<jats:italic>versus</jats:italic>‐host disease (<jats:styled-content style="fixed-case">GVHD</jats:styled-content>) grade <jats:styled-content style="fixed-case">II</jats:styled-content>‐<jats:styled-content style="fixed-case">IV</jats:styled-content> was 8% in <jats:styled-content style="fixed-case">MFD</jats:styled-content> graft vs. 25% for <jats:styled-content style="fixed-case">HSCT</jats:styled-content> post‐<jats:styled-content style="fixed-case">IST</jats:styled-content> failure (<jats:italic>P</jats:italic> < 0·0001). Chronic <jats:styled-content style="fixed-case">GVHD</jats:styled-content> was 6% in <jats:styled-content style="fixed-case">MFD HSCT</jats:styled-content> vs. 20% in <jats:styled-content style="fixed-case">HSCT</jats:styled-content> post‐<jats:styled-content style="fixed-case">IST</jats:styled-content> failure (<jats:italic>P</jats:italic> < 0·0001). <jats:styled-content style="fixed-case">MFD HSCT</jats:styled-content> is an excellent therapy for children with <jats:styled-content style="fixed-case">AA</jats:styled-content>. <jats:styled-content style="fixed-case">IST</jats:styled-content> has a high failure rate, but remains a reasonable first‐line choice if <jats:styled-content style="fixed-case">MFD HSCT</jats:styled-content> is not available because high <jats:styled-content style="fixed-case">OS</jats:styled-content> enables access to <jats:styled-content style="fixed-case">HSCT</jats:styled-content>, which is a very good rescue option.</jats:p>