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Novel treatment (new drug/intervention; established drug/procedure in new situation)
CASE REPORT
Severe refractory idiopathic warm autoimmune haemolytic anaemia responsive to complement inhibition with eculizumab
  1. Lucy Neave1,
  2. Andrew J Wilson1,2,
  3. Maxine Lissack2 and
  4. Marie Scully1
  1. 1 Department of Haematology, University College London Hospitals NHS Foundation Trust, London, UK
  2. 2 Department of Haematology, Barnet Hospital, Royal Free London NHS Foundation Trust, Hertfordshire, UK
  1. Correspondence to Marie Scully, m.scully{at}ucl.ac.uk

Abstract

We report a case of severe idiopathic warm autoimmune haemolytic anaemia (wAIHA) which was initially poorly responsive to treatment with corticosteroids, intravenous immunoglobulin, mycophenolate mofetil and rituximab, and required transfusion of more than 30 units of red cells over 12 weeks. Off-label use of the terminal complement pathway inhibitor, eculizumab, led to rapid amelioration of the haemolysis, presumably by the inhibition of an intravascular component, and allowed time for slower acting immunosuppressive agent to take effect. This novel approach warrants further evaluation, given the poor prognosis of multirefractory wAIHA.

  • haematology (drugs and medicines)
  • immunological products and vaccines
  • haematology (incl blood transfusion)
  • drugs and medicines
  • immunology

https://doi.org/10.1136/bcr-2018-226429

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    Footnotes

    • Contributors LN, AJW and MS wrote the manuscript. ML provided clinical data and reviewed the manuscript. LN and AJW contributed equally to the manuscript.

    • Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.

    • Competing interests MS has received honoraria and speaker’s fees from Alexion. LN, AJW and ML have no conflicts of interest to declare.

    • Patient consent Obtained.

    • Provenance and peer review Not commissioned; externally peer reviewed.