title: Comparison of horse and rabbit antithymocyte globulin in immunosuppressive therapy for refractory cytopenia of childhood.
abstract: Refractory cytopenia of childhood is the most common subtype of myelodysplastic syndrome in children. In this study, we compared the outcome of immunosuppressive therapy using horse antithymocyte globulin (n=46) with that using rabbit antithymocyte globulin (n=49) in 95 patients with refractory cytopenia of childhood and hypocellular bone marrow. The response rate at 6 months was 74% for horse antithymocyte globulin and 53% for rabbit antithymocyte globulin (P=0.04). The inferior response in the rabbit antithymocyte globulin group resulted in lower 4-year transplantation-free (69% versus 46%; P=0.003) and failure-free (58% versus 48%; P=0.04) survival rates in this group compared with those in the horse antithymocyte globulin group. However, because of successful second-line hematopoietic stem cell transplantation, overall survival was comparable between groups (91% versus 85%; P=ns). The cumulative incidence of relapse (15% versus 9%; P=ns) and clonal evolution (12% versus 4%; P=ns) at 4 years was comparable between groups. Our results suggest that the outcome of immunosuppressive therapy with rabbit antithymocyte globulin is inferior to that of horse antithymocyte globulin. Although immunosuppressive therapy is an effective therapy in selected patients with refractory cytopenia of childhood, the long-term risk of relapse or clonal evolution remains. (ClinicalTrial.gov identifiers: NCT00662090).
authors.affiliation: ayami.yoshimi@uniklinik-freiburg.de.
authors.forename: Ayami
authors.initials: A
authors.lastname: Yoshimi
authors.forename: Marry M
authors.initials: MM
authors.lastname: van den Heuvel-Eibrink
authors.forename: Irith
authors.initials: I
authors.lastname: Baumann
authors.forename: Stephan
authors.initials: S
authors.lastname: Schwarz
authors.forename: Ingrid
authors.initials: I
authors.lastname: Simonitsch-Klupp
authors.forename: Pascale
authors.initials: P
authors.lastname: de Paepe
authors.forename: Vit
authors.initials: V
authors.lastname: Campr
authors.forename: Gitte Birk
authors.initials: GB
authors.lastname: Kerndrup
authors.forename: Maureen
authors.initials: M
authors.lastname: O'Sullivan
authors.forename: Rita
authors.initials: R
authors.lastname: Devito
authors.forename: Roos
authors.initials: R
authors.lastname: Leguit
authors.forename: Miguel
authors.initials: M
authors.lastname: Hernandez
authors.forename: Michael
authors.initials: M
authors.lastname: Dworzak
authors.forename: Barbara
authors.initials: B
authors.lastname: de Moerloose
authors.forename: Jan
authors.initials: J
authors.lastname: Stary
authors.forename: Henrik
authors.initials: H
authors.lastname: Hasle
authors.forename: Owen P
authors.initials: OP
authors.lastname: Smith
authors.forename: Marco
authors.initials: M
authors.lastname: Zecca
authors.forename: Albert
authors.initials: A
authors.lastname: Catala
authors.forename: Markus
authors.initials: M
authors.lastname: Schmugge
authors.forename: Franco
authors.initials: F
authors.lastname: Locatelli
authors.forename: Monika
authors.initials: M
authors.lastname: Führer
authors.forename: Alexandra
authors.initials: A
authors.lastname: Fischer
authors.forename: Anne
authors.initials: A
authors.lastname: Guderle
authors.forename: Peter
authors.initials: P
authors.lastname: Nöllke
authors.forename: Brigitte
authors.initials: B
authors.lastname: Strahm
authors.forename: Charlotte M
authors.initials: CM
authors.lastname: Niemeyer
mesh_terms: D000293:Adolescent; D000818:Animals; D000961:Antilymphocyte Serum / Q000008:administration & dosage / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D005260:Female; D006736:Horses; D006801:Humans; D007165:Immunosuppression Therapy; D007166:Immunosuppressive Agents / Q000008:administration & dosage / Q000627:therapeutic use*; D007223:Infant; D008297:Male; D010198:Pancytopenia / Q000175:diagnosis / Q000188:drug therapy*; D011817:Rabbits; D012008:Recurrence; D012307:Risk Factors; D016896:Treatment Outcome
chemical_list: D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents