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

Eros Di Bona, F. Rodeghiero, B. Bruno, A. Gabbas, P. Foa, A. Locasciulli, C. Rosanelli, L. Camba, P. Saracco, A. Lippi, A. P. Iori, F. Porta, V. De Rossi, B. Comotti, P. Iacopino, C. Dufour, A. Bacigalupo

Research output: Contribution to journalArticlepeer-review

Abstract

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 5d), CyA (5mg/kg orally from day 1 to 180) and G-CSF (5 μg/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 10 9/l, haemoglobin ≥11 g/dl and platelets ≥100x10 9/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.

Original languageEnglish
Pages (from-to)330-334
Number of pages5
JournalBritish Journal of Haematology
Volume107
Issue number2
DOIs
Publication statusPublished - 1999

Keywords

  • Aplastic anaemia
  • Cyclosporin
  • Horse antilymphocyte globulin
  • Immunosuppressive therapy
  • Rabbit antithymocyte globulin

ASJC Scopus subject areas

  • Hematology

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