Rabbit antithymocyte globulin and cyclosporine as first-line therapy for children with acquired aplastic anemia

摘要

Horse antithymocyte globulin (hATG) and cyclosporine have been used as standard therapy for children with acquired aplastic anemia (AA) for whom an HLA-matched family donor is unavailable. However, in 2009, hATG (lymphoglobulin; Genzyme) was withdrawn and replaced by rabbit ATG (rATG; thymoglobulin; Genzyme) in Japan. Many other countries in Europe and Asia are facing the same situation.1 Marsh et al recently reported outcomes for 35 adult patients with AA who were treated with rATG and cyclosporine as a first-line therapy.2 Although the hematologic response rate was 40% at 6 months, several patients subsequently achieved late responses. The best response rate was 60% compared with 67% in a matched-pair control group of 105 patients treated with hATG. The overall and transplantation-free survival rates appeared to be significantly inferior with rATG compared with hATG at 68% versus 86% (P = .009) and 52% versus 76% (P = .002), respectively. These results are comparable to those from a prospective randomized study reported by Scheinberg et al comparing hATG and rATG.3 Both studies showed the superiority of hATG over rATG.2,3