Treatment of severe aplastic anemia with antilymphocyte globulin, cyclosporine and two different granulocyte colony-stimulating factor regimens: A GITMO prospective randomized study

Anna Locasciulli, Barbara Bruno, Alessandro Rambaldi, Paola Saracco, Carlo Dufour, Carlo Finelli, Simona Sica, Stefania Varotto, William Arcese, Franco Locatelli, Davide Soligo, Andrea Bacigalupo

Research output: Contribution to journalArticlepeer-review

Abstract

Background and Objectives. In a previous study we showed that patients with severe aplastic anemia (SAA) treated with anti-lymphocyte globulin (ALG), cyclosporin (CyA) and granulocyte colony-stimulating factor (G-CSF) 5 μg/kg/day had an encouraging outcome. However, failure to respond, delayed responses, partial responses, relapses and early deaths remain signifcant problems. The aim of the present study was to test whether a higher dose of G-CSF (10 μg/kg/day) would reduce these complications. Design and Methods. This was a multicenter prospective trial in 77 SAA patients treated with horse ALG (15 mg/kg/day day 1-5) and CyA (5 mg/kg/day day 1-180). Patients were randomized to receive G-CSF 5 μg/kg/day (n=38, group A) or 10 μg/kg/day (n=39, group B) from day +1 to day +30. All patients then received G-CSF 5 μg/kg/day from day +31 to day +90. The primary end point of this study was response at day +120. Secondary end points were early deaths, blood counts at day +120, and survival. Results. At day +120 responses were classified as absent, partial, and complete in 12, 22, and 4 patients in group A and in 23, 7, and 9 patients in group B (p=0.001). At last follow-up these figures were respectively 9, 12, and 17 vs 19, 2, and 18 (p=0.004). Thirteen patients (5 in group A and 8 in group B) died before day 120 (p=0.3). Median peripheral blood counts on day 120 were comparable in the two groups: Hb 10.5 and 9.5 g/dL in group A and B, respectively (p=0.6), Neutrophil counts were 2.4 vs 1.9×109/L in groups A and B (p=0.4) and platelet counts were, respectively, 42 vs 36×109/L (p=0.3). The actuarial survival at 4 years is 72% in group A and 67% in group B (p=0.3). Interpretation and Conclusions. Increasing the dose of G-CSF does not appear to reduce early deaths, does not improve peripheral blood counts nor survival, and may reduce the response rate in patients with SAA receiving ALG and CyA.

Original languageEnglish
Pages (from-to)1054-1061
Number of pages8
JournalHaematologica
Volume89
Issue number9
Publication statusPublished - Sep 2004

Keywords

  • Acquired aplastic anemia
  • G-CSF dose
  • Immunosuppression

ASJC Scopus subject areas

  • Hematology

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