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Prepublished online as a Blood First Edition Paper on July 24, 2003; DOI 10.1182/blood-2003-04-1032.

Submitted April 14, 2003
Accepted June 28, 2003
Recombinant humanized anti-IL-2 receptor antibody (Daclizumab) produces responses in patients with moderate aplastic anemia
Jaroslaw P Maciejewski, Elaine M Sloand*, Olga Nunez, Carol Boss, and Neal S Young
Hematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD, USA
* Corresponding author; email: sloande{at}nih.gov.
In contrast to severe aplastic anemia (sAA), the appropriate management of patients with moderate pancytopenia is unclear. In this study, we examined the efficacy of a humanized monoclonal antibody recognizing interleukin-2 receptor (Daclizumab), which has proven a successful immunosuppressive agent in solid organ and bone marrow transplantation. We treated 17 patients with moderate aplastic anemia (mAA) with 1 mg/kg every 2 weeks for 3 months. Moderate AA was defined as depression of two of the three blood counts: ANC 1200/mm3, platelet count 70,000/mm3, hemoglobin 8.5 g/dl and absolute reticulocyte count 60,000/mm3. The primary endpoint of our protocol was a hematologic response in at least one affected peripheral blood value. Daclizumab had little toxicity. Six of the 16 (38%) evaluable patients responded to treatment. Two patients with previously chronic disease showed complete return of normal counts, which were sustained for more than two years following treatment. Four patients had single lineage responses. Two previously transfusion-dependent patients became transfusion-independent; one patient with many neutropenia-related infections has a normal neutrophil count following treatment. Daclizumab appears safe; its efficacy in this pilot protocol suggests that expanded study of this mAb in immune-mediated bone marrow failure syndrome is warranted.

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