title: Rabbit antithymocyte globulin (r-ATG) plus cyclosporine and granulocyte colony stimulating factor is an effective treatment for aplastic anaemia patients unresponsive to a first course of intensive immunosuppressive therapy. Gruppo Italiano Trapianto di Midollo Osseo (GITMO)
abstract: About 30% of patients with severe aplastic anaemia (SAA) unresponsive to one course of immunosuppressive (IS) therapy with antithymocyte or antilymphocyte globulin can achieve complete or partial remission after a second IS treatment. Among various second-line treatments, rabbit ATG (r-ATG) could represent a safe and effective alternative to horse ALG (h-ALG). In a multicentre study, 30 patients with SAA (17 males and 13 females, median age 21 years, range 2-67) not responding to a first course with h-ALG plus cyclosporin (CyA) and granulocyte colony stimulating factor (G-CSF), were given a second course using r-ATG (3.5 mg/kg/d for 5 d), CyA (5 mg/kg orally from day 1 to 180) and G-CSF (5 microg/kg subcutaneously from day 1 to 90). The median interval between first and second treatment was 151 d (range 58-361 d). No relevant side-effects were observed, but one patient died early during treatment because of sepsis. Overall response, defined as transfusion independence, was achieved in 23/30 (77%) patients after a median time of 95 d (range 14-377). Nine patients (30%) achieved complete remission (neutrophils >/=2.0 x 109/l, haemoglobin >/=11 g/dl and platelets >/=100 x 109/l). The overall survival rate was 93% with a median follow-up of 914 d (range 121-2278). So far, no patient has relapsed. Female gender was significantly associated with a poorer likelihood to respond (P = 0.0006). These data suggest that r-ATG is a safe and effective alternative to h-ALG for SAA patients unresponsive to first-line IS treatment.
authors.affiliation: Haematology Department, San Bortolo Hospital, Vicenza, Italy. dibona@hemato.ven.it
authors.forename: E
authors.initials: E
authors.lastname: Di Bona
authors.forename: F
authors.initials: F
authors.lastname: Rodeghiero
authors.forename: B
authors.initials: B
authors.lastname: Bruno
authors.forename: A
authors.initials: A
authors.lastname: Gabbas
authors.forename: P
authors.initials: P
authors.lastname: Foa
authors.forename: A
authors.initials: A
authors.lastname: Locasciulli
authors.forename: C
authors.initials: C
authors.lastname: Rosanelli
authors.forename: L
authors.initials: L
authors.lastname: Camba
authors.forename: P
authors.initials: P
authors.lastname: Saracco
authors.forename: A
authors.initials: A
authors.lastname: Lippi
authors.forename: A P
authors.initials: AP
authors.lastname: Iori
authors.forename: F
authors.initials: F
authors.lastname: Porta
authors.forename: G
authors.initials: G
authors.lastname: De Rossi
authors.forename: B
authors.initials: B
authors.lastname: Comotti
authors.forename: P
authors.initials: P
authors.lastname: Iacopino
authors.forename: C
authors.initials: C
authors.lastname: Dufour
authors.forename: A
authors.initials: A
authors.lastname: Bacigalupo
authors.forename: V
authors.initials: V
authors.lastname: De Rossi
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000741:Anemia, Aplastic / Q000401:mortality / Q000628:therapy*; D000818:Animals; D000961:Antilymphocyte Serum / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D016572:Cyclosporine / Q000627:therapeutic use*; D004359:Drug Therapy, Combination; D005260:Female; D016179:Granulocyte Colony-Stimulating Factor / Q000627:therapeutic use*; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D007223:Infant; D008297:Male; D008875:Middle Aged; D011817:Rabbits; D015996:Survival Rate; D017211:Treatment Failure; D016896:Treatment Outcome
chemical_list: D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016179:Granulocyte Colony-Stimulating Factor; D016572:Cyclosporine