Paediatric-onset Evans syndrome: Breaking away from refractory immune thrombocytopenia

Br J Haematol. 2023 Oct;203(1):28-35. doi: 10.1111/bjh.19073.

Abstract

Since its first description by Evans in 1951, this syndrome has been linked to chronic immune thrombocytopenia with the concurrent or delayed onset of autoimmune haemolytic anaemia or neutropenia. For decades, the evolution of Evans syndrome (ES) has carried a poor prognosis and often resulted in chronic steroid exposure, multiple immune suppressing medications directed against T or B lymphocytes, and splenectomy. This paper presents a new view of ES based on recent advances in genomics which begin to classify patients based on their underlying molecular variants in previously described primary immune disorders. This has opened up new avenues of targeted therapy or bone marrow transplant at rather than broad long-term immune suppression or splenectomy. Importantly, recent studies of the full lifespan of ES suggest that at least 80% of those paediatric patients will progress to various clinical or biological immunopathological manifestations with age despite the resolution of their cytopenias. Those patients merit long-term follow-up and monitoring in dedicated transition programs to improve outcome at the adult age.

Keywords: AIHA; Evans syndrome; ITP; paediatric.

Publication types

  • Review
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Anemia, Hemolytic, Autoimmune* / diagnosis
  • Anemia, Hemolytic, Autoimmune* / therapy
  • Child
  • Humans
  • Neutropenia*
  • Purpura, Thrombocytopenic, Idiopathic* / diagnosis
  • Purpura, Thrombocytopenic, Idiopathic* / therapy
  • Thrombocytopenia* / etiology
  • Thrombocytopenia* / therapy

Supplementary concepts

  • Evans Syndrome