Molldrem J J, Caples M, Mavroudis D, Plante M, Young N S, Barrett A J
Clinical Hematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, Maryland, USA.
Br J Haematol. 1997 Dec;99(3):699-705. doi: 10.1046/j.1365-2141.1997.4423249.x.
Twenty-five transfusion-dependent myelodysplastic syndrome (MDS) patients (with < 20% blasts) were treated in a phase II study with antithymocyte globulin (ATG) at 40 mg/kg/d for four doses and then followed with blood counts every 2 weeks and clinic visits every 3 months, for a median of 14 months (range 1-38 months). 11 (44%) patients responded and became transfusion-independent after ATG, including three complete responses, six partial responses, and two minimal responses. Responses were observed in 9/14 patients (64%) with refractory anaemia (RA) and 2/6 patients (33%) with refractory anaemia with excess blasts (RAEB). Median response duration was 10 months (range 3-38 months). The Kaplan-Meier estimate of overall survival was 84% at 38 months, with one early death due to pneumonia and two deaths from disease progression to leukaemia. Side-effects consisted mainly of mild serum sickness in all patients. A single course of ATG restored haemopoiesis in some patients with MDS and was well tolerated.
25例依赖输血的骨髓增生异常综合征（MDS）患者（原始细胞<20%）在一项II期研究中接受抗胸腺细胞球蛋白（ATG）治疗，剂量为40mg/kg/d，共4剂，随后每2周进行血常规检查，每3个月进行门诊随访，中位随访时间为14个月（范围1 - 38个月）。11例（44%）患者在接受ATG治疗后有反应并不再依赖输血，包括3例完全缓解、6例部分缓解和2例微小缓解。14例难治性贫血（RA）患者中有9例（64%）出现反应，6例伴有过多原始细胞的难治性贫血（RAEB）患者中有2例（33%）出现反应。中位反应持续时间为10个月（范围3 - 38个月）。38个月时的Kaplan-Meier总生存率估计为84%，1例因肺炎早期死亡，2例因疾病进展为白血病死亡。副作用主要为所有患者均出现的轻度血清病。单疗程ATG使部分MDS患者恢复了造血功能，且耐受性良好。
