Long-term follow-up of patients with moderate aplastic anemia and pure red cell aplasia treated with daclizumab. | Aplastic Anemia and MDS International Foundation

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

Journal Title: 
Haematologica
Author(s): 
Sloand EM, Olnes MJ, Weinstein B, Wu C, Maciejewski J, Scheinberg P, Young NS
Primary Author: 
Sloand EM
Original Publication Date: 
Monday, March 1, 2010

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 mAA 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.

Bone Marrow Diseases: