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

作者：Judith C. W. Marsh, Andrea Paolo Bacigalupo, Hubert Schrezenmeier, André Tichelli, Antonio Maria Risitano, Jakob Robert Passweg, Sally B. Killick, Alan John Warren, Theodora Foukaneli, Mahmoud Deeb Aljurf, Hazzaa Abdulla Alzahrani, Philip Schafhausen, Alexander Röth, Anke Franzke, Tim H. Brümmendorf, Carlo Dufour, Rosi Oneto, Philip M. Sedgwick, Alain Barrois, Shahram Y. Kordasti, Modupe O. Elebute, Ghulam J. Mufti, Gèrard Socié · 发表于：Blood · 年份：2012 · DOI：10.1182/blood-2012-02-407684 · 被引用次数：183 · 研究领域：Hematopoietic Stem Cell Transplantation、Transplantation: Methods and Outcomes、Reproductive Biology and Fertility

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.