title: Antithymocyte globulin has limited efficacy and substantial toxicity in unselected anemic patients with myelodysplastic syndrome.
abstract: Antithymocyte globulin (ATG) has recently been popularized as an effective treatment in myelodysplastic syndrome (MDS). We treated 8 anemic MDS patients (refractory anemia [RA] and refractory anemia with excess blasts [RAEB-1]) with ATG (40 mg/kg/d for 4 days) and prednisone in a phase 2 trial. The study was stopped early according to a preset termination rule because of lack of efficacy. There were no salutary responses. Toxicities included serum sickness (in all patients), transient neutropenia and thrombocytopenia, diarrhea, vomiting, and syncope with a generalized seizure. At least 3 patients had the HLA-DR15 (DR2) allele. We conclude that the risk-benefit ratio of ATG in an unselected population of MDS patients may be unfavorable, and more work is needed to define the subset of patients who will respond to ATG before its widespread use can be recommended.
authors.affiliation: Division of Hematology, Department of Internal Medicine, Mayo Clinic, Rochester, MN 55905, USA. steensma.david@mayo.edu
authors.forename: David P
authors.initials: DP
authors.lastname: Steensma
authors.forename: Angela
authors.initials: A
authors.lastname: Dispenzieri
authors.forename: S Breanndan
authors.initials: SB
authors.lastname: Moore
authors.forename: Georgene
authors.initials: G
authors.lastname: Schroeder
authors.forename: Ayalew
authors.initials: A
authors.lastname: Tefferi
mesh_terms: D000368:Aged; D000740:Anemia / Q000150:complications / Q000276:immunology / Q000628:therapy*; D000753:Anemia, Refractory / Q000150:complications / Q000628:therapy; D000754:Anemia, Refractory, with Excess of Blasts / Q000150:complications / Q000628:therapy; D000961:Antilymphocyte Serum / Q000009:adverse effects* / Q000627:therapeutic use*; D005260:Female; D006684:HLA-DR Antigens / Q000032:analysis / Q000235:genetics; D059810:HLA-DR Serological Subtypes; D006650:Histocompatibility Testing; D006801:Humans; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000150:complications / Q000276:immunology / Q000628:therapy*
chemical_list: D000961:Antilymphocyte Serum; D006684:HLA-DR Antigens; D059810:HLA-DR Serological Subtypes; C106559:HLA-DR15 antigen