Long-term follow-up of patients with moderate aplastic anemia and pure red cell aplasia treated with daclizumab

作者：Sloand Elaine M^*; Olnes Matthew J; Weinstein Barbara; Wu Colin; Maciejewski Jaroslaw; Scheinberg Phillip; Young Neal S

来源：Haematologica-The Hematology Journal, 2010, 95(3): 382-387.

DOI：10.3324/haematol.2009.013557

• interleukin-2 receptors
• immunosuppression
• T-regulatory cells
• marrow failure

Background
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.
Design and Methods
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 m-AA patients responded by six months following administration of the drug.
Results
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.
Conclusions
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.

• 出版日期2010-3