Rabbit antithymocyte globulin (r-ATG) plus cyclosporine and granulocyte colony stimulating factor is an effective treatment for aplastic anaemia patients unresponsive to a first course of intensive immunosuppressive therapy. Gruppo Italiano Trapianto di Midollo Osseo (GITMO)

Br J Haematol. 1999 Nov;107(2):330-4. doi: 10.1046/j.1365-2141.1999.01693.x.


About 30% of patients with severe aplastic anaemia (SAA) unresponsive to one course of immunosuppressive (IS) therapy with antithymocyte or antilymphocyte globulin can achieve complete or partial remission after a second IS treatment. Among various second-line treatments, rabbit ATG (r-ATG) could represent a safe and effective alternative to horse ALG (h-ALG). In a multicentre study, 30 patients with SAA (17 males and 13 females, median age 21 years, range 2-67) not responding to a first course with h-ALG plus cyclosporin (CyA) and granulocyte colony stimulating factor (G-CSF), were given a second course using r-ATG (3.5 mg/kg/d for 5 d), CyA (5 mg/kg orally from day 1 to 180) and G-CSF (5 microg/kg subcutaneously from day 1 to 90). The median interval between first and second treatment was 151 d (range 58-361 d). No relevant side-effects were observed, but one patient died early during treatment because of sepsis. Overall response, defined as transfusion independence, was achieved in 23/30 (77%) patients after a median time of 95 d (range 14-377). Nine patients (30%) achieved complete remission (neutrophils >/=2.0 x 109/l, haemoglobin >/=11 g/dl and platelets >/=100 x 109/l). The overall survival rate was 93% with a median follow-up of 914 d (range 121-2278). So far, no patient has relapsed. Female gender was significantly associated with a poorer likelihood to respond (P = 0.0006). These data suggest that r-ATG is a safe and effective alternative to h-ALG for SAA patients unresponsive to first-line IS treatment.

Publication types

  • Clinical Trial
  • Multicenter Study

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Anemia, Aplastic / mortality
  • Anemia, Aplastic / therapy*
  • Animals
  • Antilymphocyte Serum / therapeutic use*
  • Child
  • Child, Preschool
  • Cyclosporine / therapeutic use*
  • Drug Therapy, Combination
  • Female
  • Granulocyte Colony-Stimulating Factor / therapeutic use*
  • Humans
  • Immunosuppressive Agents / therapeutic use*
  • Infant
  • Male
  • Middle Aged
  • Rabbits
  • Survival Rate
  • Treatment Failure
  • Treatment Outcome


  • Antilymphocyte Serum
  • Immunosuppressive Agents
  • Granulocyte Colony-Stimulating Factor
  • Cyclosporine