How I manage Evans Syndrome and AIHA cases in children

Br J Haematol. 2016 Feb;172(4):524-34. doi: 10.1111/bjh.13866. Epub 2015 Dec 2.


The management of Evans Syndrome in children is challenging due to the lack of evidence-based data on treatment. Steroids, the first-choice therapy, are successful in about 80% of cases. For children who are resistant, relapse or become steroid-dependent, rituximab is considered a valid second-line treatment, with the exception of those with an underlying diagnosis of autoimmune lymphoproliferative syndrome who may benefit from other options such as mycophenolate mofetil and sirolimus. Better knowledge of the immunological mechanisms underlying cytopenias and the availability of new immunosuppressive drugs can be helpful in the choice of more targeted therapies that would enable the reduction of the use of long-term steroid administration or other more aggressive options, such as splenectomy or stem cell transplantation. This manuscript provides an overview of the pathogenic background of the disease, and suggests a clinical approach to diagnosis and treatment with a particular focus on the management of relapsing/resistant disease.

Keywords: Evans syndrome; autoimmune haemolytic anaemia; autoimmune lymphoproliferative syndrome; children; immune thrombocytopenia; immunosuppression.

Publication types

  • Review

MeSH terms

  • Alemtuzumab
  • Anemia, Hemolytic, Autoimmune / diagnosis
  • Anemia, Hemolytic, Autoimmune / therapy*
  • Antibodies, Monoclonal, Humanized / therapeutic use
  • Bortezomib / therapeutic use
  • Child
  • Cyclophosphamide / therapeutic use
  • Cyclosporine / therapeutic use
  • Drug Resistance
  • Erythrocyte Transfusion / methods
  • Humans
  • Immunoglobulins, Intravenous / therapeutic use
  • Immunologic Factors / therapeutic use*
  • Immunosuppressive Agents / therapeutic use
  • Mycophenolic Acid / analogs & derivatives
  • Mycophenolic Acid / therapeutic use
  • Recurrence
  • Rituximab / therapeutic use
  • Sirolimus / therapeutic use
  • Splenectomy / methods
  • Stem Cell Transplantation / methods
  • Steroids / therapeutic use
  • Thrombocytopenia / diagnosis
  • Thrombocytopenia / therapy*


  • Antibodies, Monoclonal, Humanized
  • Immunoglobulins, Intravenous
  • Immunologic Factors
  • Immunosuppressive Agents
  • Steroids
  • Alemtuzumab
  • Rituximab
  • Bortezomib
  • Cyclosporine
  • Cyclophosphamide
  • Mycophenolic Acid
  • Sirolimus

Supplementary concepts

  • Evans Syndrome