Recombinant humanized anti-IL-2 receptor antibody (daclizumab) produces responses in patients with moderate aplastic anemia

Blood. 2003 Nov 15;102(10):3584-6. doi: 10.1182/blood-2003-04-1032. Epub 2003 Jul 24.

Abstract

In contrast to severe aplastic anemia (sAA), the appropriate management of patients with moderate pancytopenia is unclear. In this study, we examined the efficacy of a humanized monoclonal antibody recognizing interleukin-2 receptor (daclizumab), which has proven to be a successful immunosuppressive agent in solid organ and bone marrow transplantation. We treated 17 patients with moderate aplastic anemia (mAA) with 1 mg/kg every 2 weeks for 3 months. mAA was defined as depression of 2 of the 3 blood counts: absolute neutrophil count 1200/mm3 or less, platelet count 70,000/mm3 or less, hemoglobin level 8.5 g/dL or lower, and absolute reticulocyte count 60,000/mm3 or less. The primary end point of our protocol was a hematologic response in at least one affected peripheral blood value. Daclizumab had little toxicity. Six of the 16 (38%) evaluable patients responded to treatment. Two patients with previously chronic disease showed complete return of normal counts, which were sustained for more than 2 years following treatment. Four patients had single-lineage responses. Two previously transfusion-dependent patients became transfusion independent; one patient with many neutropenia-related infections had a normal neutrophil count following treatment. Daclizumab appears safe; its efficacy in this pilot protocol suggests that expanded study of this monoclonal antibody in immune-mediated bone marrow failure syndrome is warranted.

Publication types

  • Clinical Trial

MeSH terms

  • Adolescent
  • Adult
  • Anemia, Aplastic / drug therapy*
  • Antibodies, Monoclonal / administration & dosage
  • Antibodies, Monoclonal / pharmacology*
  • Antibodies, Monoclonal / therapeutic use
  • Antibodies, Monoclonal, Humanized
  • Blood Cell Count
  • Child
  • Daclizumab
  • Female
  • Humans
  • Immunoglobulin G / administration & dosage
  • Immunoglobulin G / pharmacology*
  • Immunoglobulin G / therapeutic use
  • Male
  • Middle Aged
  • Pilot Projects
  • Receptors, Interleukin-2 / immunology
  • Recombinant Proteins / pharmacology
  • Recombinant Proteins / therapeutic use
  • Time Factors
  • Treatment Outcome

Substances

  • Antibodies, Monoclonal
  • Antibodies, Monoclonal, Humanized
  • Immunoglobulin G
  • Receptors, Interleukin-2
  • Recombinant Proteins
  • Daclizumab