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

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

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  • Medientyp: E-Artikel
  • Titel: Prospective study of rabbit antithymocyte globulin and cyclosporine for aplastic anemia from the EBMT Severe Aplastic Anaemia Working Party
  • Beteiligte: Marsh, Judith C.; Bacigalupo, Andrea; Schrezenmeier, Hubert; Tichelli, Andre; Risitano, Antonio M.; Passweg, Jakob R.; Killick, Sally B.; Warren, Alan J.; Foukaneli, Theodora; Aljurf, Mahmoud; Al-Zahrani, H. A.; Schafhausen, Philip; Roth, Alexander; Franzke, Anke; Brummendorf, Tim H.; Dufour, Carlo; Oneto, Rosi; Sedgwick, Philip; Barrois, Alain; Kordasti, Shahram; Elebute, Modupe O.; Mufti, Ghulam J.; Socie, Gerard
  • Erschienen: American Society of Hematology, 2012
  • Erschienen in: Blood, 119 (2012) 23, Seite 5391-5396
  • Sprache: Englisch
  • DOI: 10.1182/blood-2012-02-407684
  • ISSN: 0006-4971; 1528-0020
  • Entstehung:
  • Anmerkungen:
  • Beschreibung: 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.
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