• 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, Britta Höchsmann, Philip Schafhausen, Alexander Roth, Anke Franzke, Tim H Brummendorf, Carlo Dufour, Rosi Oneto, Philip Sedgwick, Alain Barrois, Shahram Kordasti, Modupe O Elebute, Ghulam J Mufti, Gerard Socie, and European Blood and Marrow Transplant Group Severe Aplastic Anaemia Working Party.
• Department of Haematological Medicine, King's College Hospital/King's College London, London, United Kingdom. judith.marsh@nhs.net
• Blood. 2012 Jun 7; 119 (23): 5391-6.

AbstractRabbit 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.

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