title: Long-term follow-up of patients with moderate aplastic anemia and pure red cell aplasia treated with daclizumab.
abstract: 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.
  
  
  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.
authors.affiliation: National Heart Lung and Blood Instititute, NIH, Bethesda, MD 20892, USA. sloande@nih.gov
authors.forename: Elaine M
authors.initials: EM
authors.lastname: Sloand
authors.forename: Matthew J
authors.initials: MJ
authors.lastname: Olnes
authors.forename: Barbara
authors.initials: B
authors.lastname: Weinstein
authors.forename: Colin
authors.initials: C
authors.lastname: Wu
authors.forename: Jaroslaw
authors.initials: J
authors.lastname: Maciejewski
authors.forename: Phillip
authors.initials: P
authors.lastname: Scheinberg
authors.forename: Neal S
authors.initials: NS
authors.lastname: Young
mesh_terms: D000741:Anemia, Aplastic / Q000188:drug therapy*; D000911:Antibodies, Monoclonal / Q000627:therapeutic use*; D061067:Antibodies, Monoclonal, Humanized; D000077561:Daclizumab; D005500:Follow-Up Studies; D006801:Humans; D007074:Immunoglobulin G / Q000627:therapeutic use*; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D053645:Interleukin-2 Receptor alpha Subunit / Q000037:antagonists & inhibitors; D012010:Red-Cell Aplasia, Pure / Q000188:drug therapy*; D012449:Safety; D015996:Survival Rate; D013997:Time Factors; D016896:Treatment Outcome
chemical_list: D000911:Antibodies, Monoclonal; D061067:Antibodies, Monoclonal, Humanized; C508017:IL2RA protein, human; D007074:Immunoglobulin G; D007166:Immunosuppressive Agents; D053645:Interleukin-2 Receptor alpha Subunit; D000077561:Daclizumab