National Heart Lung and Blood Instititute, NIH, Bethesda, MD 20892, USA.
Haematologica. 2010 Mar;95(3):382-7. doi: 10.3324/haematol.2009.013557.
Pure red cell aplasia and moderate aplastic anemia are marrow failure states with an immune pathogenesis. Previously, we described short-term improvements in blood counts in two pilot studies treating moderate aplastic anemia (mAA) and pure red cell aplasia (PRCA) patients with daclizumab, a humanized monoclonal antibody to the interleukin-2 receptor; we now report our long-term experience with a larger cohort of patients.
After a median follow-up period of 4.8 years, 19 of 45 (42%) evaluable mAA patients and 10 of 26 (38%) patients with PRCA responded by three months and 2 additional mAA patients responded by six months following administration of the drug.
Seven of 28 (25%) mAA patients achieved long-term packed red blood cell PRBC transfusion independence, and all PRCA responders achieved long-term transfusion PRBC transfusion independence.
Red cell transfusion-independence prior to treatment in mAA patients predicted response. The only significant adverse treatment-related events were transient rashes and arthralgias. Daclizumab is safe and effective, and produces lengthy remissions in patients with PRCA and mAA.
纯红细胞再生障碍和中度再生障碍性贫血是骨髓衰竭状态，具有免疫发病机制。此前，我们描述了在使用达珠单抗治疗中度再生障碍性贫血（mAA）和纯红细胞再生障碍性贫血（PRCA）患者的两项试点研究中，计数短暂改善的情况；现在我们报告了更大患者队列的长期经验。
在中位随访期为 4.8 年后，在接受药物治疗后三个月，19 名可评估的 mAA 患者中有 19 名（42%）和 26 名 PRCA 患者中有 10 名（38%）有反应，另外 2 名 mAA 患者在六个月时有反应。
28 名 mAA 患者中有 7 名（25%）实现了长期的红细胞 PRBC 输血独立性，所有 PRCA 患者均实现了长期的输血 PRBC 输血独立性。
mAA 患者在治疗前的红细胞输血独立性预测了反应。唯一显著的不良治疗相关事件是短暂的皮疹和关节痛。达珠单抗安全有效，可使 PRCA 和 mAA 患者产生长时间缓解。
