Prospective study of rabbit antithymocyte globulin and cyclosporine for aplastic anemia from the EBMT Severe Aplastic Anaemia Working Party

Judith C. Marsh, Andrea Bacigalupo, Hubert Schrezenmeier, Andre Tichelli, Antonio M. Risitano, Jakob R. Passweg, Sally B. Killick, Alan J. Warren, Theodora Foukaneli, Mahmoud Aljurf, H. A. Al-Zahrani, Philip Schafhausen, Alexander Roth, Anke Franzke, Tim H. Brummendorf, Carlo Dufour, Rosi Oneto, Philip Sedgwick, Alain Barrois, Shahram KordastiModupe O. Elebute, Ghulam J. Mufti, Gerard Socie

Research output: Contribution to journalArticle

Abstract

Rabbit antithymocyte globulin (rATG; thymoglobulin, Genzyme) in combination with cyclosporine, as first-line immunosuppressive therapy, was evaluated prospectively in a multicenter, European, phase 2 pilot study, in 35 patients with aplastic anemia. Results were compared with 105 age- and disease severity-matched patients from the European Blood and Marrow Transplant registry, treated with horse ATG (hATG; lymphoglobulin) and cyclosporine. The primary end point was response at 6 months. At 3 months, no patients had achieved a complete response to rATG. Partial response occurred in 11 (34%). At 6 months, complete response rate was 3% and partial response rate 37%. There were 10 deaths after rATG (28.5%) and 1 after subsequent HSCT. Infections were the main cause of death in 9 of 10 patients. The best response rate was 60% for rATG and 67% for hATG. For rATG, overall survival at 2 years was 68%, compared with 86% for hATG (P = .009). Transplant-free survival was 52% for rATG and 76% for hATG (P = .002). On multivariate analysis, rATG (hazard ratio = 3.9, P = .003) and age more than 37 years (hazard ratio = 4.7, P = .0008) were independent adverse risk factors for survival. This study was registered at www.clinicaltrials.gov as NCT00471848.

Original languageEnglish
Pages (from-to)5391-5396
Number of pages6
JournalBlood
Volume119
Issue number23
DOIs
Publication statusPublished - Jun 7 2012

ASJC Scopus subject areas

  • Hematology
  • Biochemistry
  • Cell Biology
  • Immunology

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    Marsh, J. C., Bacigalupo, A., Schrezenmeier, H., Tichelli, A., Risitano, A. M., Passweg, J. R., Killick, S. B., Warren, A. J., Foukaneli, T., Aljurf, M., Al-Zahrani, H. A., Schafhausen, P., Roth, A., Franzke, A., Brummendorf, T. H., Dufour, C., Oneto, R., Sedgwick, P., Barrois, A., ... Socie, G. (2012). Prospective study of rabbit antithymocyte globulin and cyclosporine for aplastic anemia from the EBMT Severe Aplastic Anaemia Working Party. Blood, 119(23), 5391-5396. https://doi.org/10.1182/blood-2012-02-407684