First-line immunosuppressive treatment in children with aplastic anemia: rabbit antithymocyte globulin

Adv Exp Med Biol. 2015:836:55-62. doi: 10.1007/5584_2014_38.

Abstract

Immunosuppressive therapy is the treatment of choice in children with acquired severe aplastic anemia (AA) and no HLA-matched family donor. The paper presents results of a multicenter study of 63 children with AA treated with rabbit antithymocyte globulin (r-ATG) and cyclosporine A as the first line treatment in the years 1996-2012. Therapeutic effects were evaluated at Days 112, 180, and 360. At Day 112, remission was achieved in 28 out of the 63 patients (44.4 %), complete remission in 10 patients (15.9 %), and partial remission in 18 (28.5 %). At Day 180, 31 patients (49.2 %) were in remission including 15 cases in complete (23.8 %), and 16 cases in partial remission (25.4 %). One year after therapy onset, 34 patients (64.9 %) were in remission including 24 patients (38.0 %) in complete and 10 (15.9 %) in partial remission. Relapse occurred in 4 patients, from 8 months up to 2 years and 2 months after remission. One child, 5 years after remission, was diagnosed with paroxysmal nocturnal hemoglobinuria. The estimated 10-year overall survival rate and 10-year event-free survival rate were 67 % and 57 %, respectively.

MeSH terms

  • Adolescent
  • Anemia, Aplastic / immunology
  • Anemia, Aplastic / mortality
  • Anemia, Aplastic / therapy*
  • Animals
  • Antilymphocyte Serum / therapeutic use*
  • Child
  • Child, Preschool
  • Female
  • Humans
  • Immunosuppression Therapy / methods*
  • Immunosuppressive Agents / therapeutic use*
  • Infant
  • Male
  • Rabbits
  • Retrospective Studies
  • Treatment Outcome

Substances

  • Antilymphocyte Serum
  • Immunosuppressive Agents