Successful treatment of severe refractory autoimmune hemolytic anemia after hematopoietic stem cell transplant with abatacept

Transfusion. 2018 Sep;58(9):2122-2127. doi: 10.1111/trf.14907. Epub 2018 Sep 4.

Abstract

Background: After hematopoietic stem cell transplantation (HSCT) autoimmune hemolytic anemia (AIHA) is a known and fairly common complication. It is often refractory to conventional therapies including corticosteroids, intravenous immunoglobulin, splenectomy, and the more recently described use of monoclonal antibodies. The high morbidity associated with these severe persistent cases elucidates the gaps in alternative therapies available for treatment.

Study design and methods: We described the successful use of abatacept for severe refractory AIHA after HSCT in three patients.

Results: Three pediatric patients with refractory AIHA after allogeneic stem cell transplantation were observed to be unresponsive to multitude immunosuppressive therapies, resulting in persistent transfusion dependency. Treatment with abatacept, a fusion protein that inhibits T-cell activation by binding to CD80/CD86 on antigen-presenting cells (APCs), thus blocking the required CD28 interaction between APCs and T cells, resulted in the resolution of hemolysis.

Conclusion: Abatacept may provide significant clinical benefit in the management of AIHA after HSCT.

Publication types

  • Case Reports

MeSH terms

  • Abatacept / therapeutic use*
  • Adolescent
  • Anemia, Hemolytic, Autoimmune / drug therapy*
  • Anemia, Hemolytic, Autoimmune / etiology
  • Anemia, Sickle Cell / therapy
  • Bacteremia / complications
  • Blood Grouping and Crossmatching
  • Child
  • Child, Preschool
  • Drug Resistance
  • Drug Substitution
  • Female
  • Guanine Nucleotide Exchange Factors / deficiency
  • Hematopoietic Stem Cell Transplantation / adverse effects*
  • Humans
  • Immunosuppressive Agents / therapeutic use*
  • Job Syndrome / complications
  • Lymphohistiocytosis, Hemophagocytic / therapy
  • Male
  • Methicillin-Resistant Staphylococcus aureus
  • Pneumonia, Pneumocystis / complications
  • Remission Induction
  • Retrospective Studies
  • Staphylococcal Infections / complications
  • Virus Diseases / complications

Substances

  • DOCK8 protein, human
  • Guanine Nucleotide Exchange Factors
  • Immunosuppressive Agents
  • Abatacept