BMSEHA15
Haematologica, Vol 95, Issue 3, 382-387 doi:10.3324/haematol.2009.013557
Copyright © 2010 by Ferrata Storti Foundation
This Article
Right arrow Full Text
Right arrow Full Text (PDF)
Right arrow Sloand et al. Supplementary Appendix
Right arrow Alert me when this article is cited
Right arrow Alert me if a correction is posted
Services
Right arrow Email this article to a friend
Right arrow Similar articles in this journal
Right arrow Similar articles in PubMed
Right arrow Alert me to new issues of the journal
Right arrow Download to citation manager
Google Scholar
Right arrow Articles by Sloand, E. M.
Right arrow Articles by Young, N. S.
PubMed
Right arrow PubMed Citation
Right arrow Articles by Sloand, E. M.
Right arrow Articles by Young, N. S.

Aplastic Anemia

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

Elaine M. Sloand, Matthew J. Olnes, Barbara Weinstein, Colin Wu, Jaroslaw Maciejewski, Phillip Scheinberg, Neal S. Young

National Heart Lung and Blood Institute, Bethesda, MD, USA

Correspondence: Elaine M. Sloand, MD, Senior Clinical Investigator, National Heart Lung and Blood Instititute Bethesda, Md 20892 USA., E-mail: sloande{at}nih.gov

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.

Key words: interleukin-2 receptors, immunosuppression, T-regulatory cells, marrow failure.