title: Antithymocyte globulin for treatment of the bone marrow failure associated with myelodysplastic syndromes.
abstract: BACKGROUND
  Almost half of the deaths that result from myelodysplastic syndromes are due to cytopenia associated with bone marrow failure. Treatment is mostly supportive care.
  
  
  OBJECTIVE
  To determine whether treatment with antithymocyte globulin improves cytopenia and reverses dependence on red blood cell transfusions in patients with myelodysplastic syndromes.
  
  
  METHODS
  Single-treatment, prospective study.
  
  
  METHODS
  Tertiary referral center.
  
  
  METHODS
  61 patients with myelodysplastic syndromes.
  
  
  METHODS
  Antithymocyte globulin, 40 mg/kg of body weight, given daily for 4 days.
  
  
  METHODS
  Evaluation of bone marrow, blood counts, transfusions, progression, and survival for a median of 30 months (range, 1 to 88 months).
  
  
  RESULTS
  Within 8 months of treatment, 21 of 61 patients (34%) no longer required red blood cell transfusions. This independence from transfusions was maintained in 17 responders (81%) for a median of 36 months (range, 3 to 72 months). Ten of 21 patients (47.5%) with severe thrombocytopenia had sustained platelet count increases, and 6 of 11 patients (55%) with severe neutropenia had sustained neutrophil counts of greater than 1 x 10(9) cells/L. Characteristics favorable for response were younger patient age (P = 0.005) and lower platelet counts (P = 0.038). One of the 21 responders (5%) and 22 of the 40 nonresponders (55%) died before the end of the study (P = 0.008). One of the 21 responders (5%) and 13 of the 40 nonresponders (33%) had disease progression (P = 0.086).
  
  
  CONCLUSIONS
  Although this study was a nonrandomized, single-treatment study, 34% of patients treated with antithymocyte globulin became transfusion independent. Response was associated with a statistically significant longer survival and an almost significant decreased time to disease progression. Treatment with antithymocyte globulin did not seem to be detrimental because historical overall median survival times were similar to those of nonresponders.
authors.affiliation: National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, Maryland 70892, USA. jmolldre@mdanderson.org
authors.forename: Jeffrey J
authors.initials: JJ
authors.lastname: Molldrem
authors.forename: Eric
authors.initials: E
authors.lastname: Leifer
authors.forename: Erkut
authors.initials: E
authors.lastname: Bahceci
authors.forename: Yogen
authors.initials: Y
authors.lastname: Saunthararajah
authors.forename: Mary
authors.initials: M
authors.lastname: Rivera
authors.forename: Cynthia
authors.initials: C
authors.lastname: Dunbar
authors.forename: Johnson
authors.initials: J
authors.lastname: Liu
authors.forename: Riotoro
authors.initials: R
authors.lastname: Nakamura
authors.forename: Neal S
authors.initials: NS
authors.lastname: Young
authors.forename: A John
authors.initials: AJ
authors.lastname: Barrett
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000753:Anemia, Refractory / Q000188:drug therapy / Q000209:etiology; D000961:Antilymphocyte Serum / Q000009:adverse effects / Q000627:therapeutic use*; D001772:Blood Cell Count; D001853:Bone Marrow / Q000503:physiopathology*; D018450:Disease Progression; D017707:Erythrocyte Transfusion; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000009:adverse effects / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000188:drug therapy* / Q000503:physiopathology*; D009503:Neutropenia / Q000188:drug therapy / Q000209:etiology; D011446:Prospective Studies; D012044:Regression Analysis; D016019:Survival Analysis; D013921:Thrombocytopenia / Q000188:drug therapy / Q000209:etiology
chemical_list: D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents