Etanercept as a salvage treatment for refractory aplastic anemia

Pediatr Blood Cancer. 2009 Apr;52(4):522-5. doi: 10.1002/pbc.21886.

Abstract

About 10-15% of patients with acquired aplastic anemia (AAA) have resistant/recurrent disease not eligible for standard treatment like hematopoietic stem cell transplantation and/or combined immunosuppression. We report a 17-year-old male with an 11 years history of AAA who, after two courses of immunosuppression, was red cell transfusion-dependent, severely thrombocytopenic, refractory to platelet transfusion, had iron overload and post-transfusion HCV infection. This patient achieved transfusion independence from platelets and normalized Hb after treatment with the anti-TNF agent Etanercept. Over a 12 months follow-up he experienced only transient increase of liver transaminases.

Publication types

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

MeSH terms

  • Adolescent
  • Anemia, Aplastic / drug therapy*
  • Anemia, Aplastic / physiopathology
  • Anemia, Refractory / drug therapy*
  • Anemia, Refractory / physiopathology
  • Clinical Trials as Topic
  • Etanercept
  • Hepatitis C / complications
  • Humans
  • Immunoglobulin G / therapeutic use*
  • Immunosuppressive Agents / therapeutic use*
  • Male
  • Receptors, Tumor Necrosis Factor / therapeutic use*
  • Salvage Therapy / methods*

Substances

  • Immunoglobulin G
  • Immunosuppressive Agents
  • Receptors, Tumor Necrosis Factor
  • Etanercept