title: First-line immunosuppressive treatment in children with aplastic anemia: rabbit antithymocyte globulin.
abstract: Immunosuppressive therapy is the treatment of choice in children with acquired severe aplastic anemia (AA) and no HLA-matched family donor. The paper presents results of a multicenter study of 63 children with AA treated with rabbit antithymocyte globulin (r-ATG) and cyclosporine A as the first line treatment in the years 1996-2012. Therapeutic effects were evaluated at Days 112, 180, and 360. At Day 112, remission was achieved in 28 out of the 63 patients (44.4 %), complete remission in 10 patients (15.9 %), and partial remission in 18 (28.5 %). At Day 180, 31 patients (49.2 %) were in remission including 15 cases in complete (23.8 %), and 16 cases in partial remission (25.4 %). One year after therapy onset, 34 patients (64.9 %) were in remission including 24 patients (38.0 %) in complete and 10 (15.9 %) in partial remission. Relapse occurred in 4 patients, from 8 months up to 2 years and 2 months after remission. One child, 5 years after remission, was diagnosed with paroxysmal nocturnal hemoglobinuria. The estimated 10-year overall survival rate and 10-year event-free survival rate were 67 % and 57 %, respectively.
authors.affiliation: Department of Pediatric, Hematology and Oncology, Medical University of Warsaw, 24 Marszalkowska St., Warsaw, 00-576, Poland, katarzyna.pawelec@litewska.edu.pl.
authors.forename: K
authors.initials: K
authors.lastname: Pawelec
authors.forename: M
authors.initials: M
authors.lastname: Salamonowicz
authors.forename: A
authors.initials: A
authors.lastname: Panasiuk
authors.forename: U
authors.initials: U
authors.lastname: Demkow
authors.forename: J
authors.initials: J
authors.lastname: Kowalczyk
authors.forename: W
authors.initials: W
authors.lastname: Balwierz
authors.forename: E
authors.initials: E
authors.lastname: Zaleska-Czepko
authors.forename: A
authors.initials: A
authors.lastname: Chybicka
authors.forename: K
authors.initials: K
authors.lastname: Szmyd
authors.forename: T
authors.initials: T
authors.lastname: Szczepanski
authors.forename: H
authors.initials: H
authors.lastname: Bubala
authors.forename: M
authors.initials: M
authors.lastname: Wysocki
authors.forename: A
authors.initials: A
authors.lastname: Kurylak
authors.forename: J
authors.initials: J
authors.lastname: Wachowiak
authors.forename: D
authors.initials: D
authors.lastname: Szpecht
authors.forename: W
authors.initials: W
authors.lastname: Młynarski
authors.forename: M
authors.initials: M
authors.lastname: Bulas
authors.forename: M
authors.initials: M
authors.lastname: Krawczuk-Rybak
authors.forename: E
authors.initials: E
authors.lastname: Leszczynska
authors.forename: T
authors.initials: T
authors.lastname: Urasinski
authors.forename: J
authors.initials: J
authors.lastname: Peregud-Pogorzelski
authors.forename: A
authors.initials: A
authors.lastname: Balcerska
authors.forename: B
authors.initials: B
authors.lastname: Kaczorowska-Hac
authors.forename: M
authors.initials: M
authors.lastname: Matysiak
mesh_terms: D000293:Adolescent; D000741:Anemia, Aplastic / Q000276:immunology / Q000401:mortality / Q000628:therapy*; D000818:Animals; D000961:Antilymphocyte Serum / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D007165:Immunosuppression Therapy / Q000379:methods*; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D007223:Infant; D008297:Male; D011817:Rabbits; D012189:Retrospective Studies; D016896:Treatment Outcome
chemical_list: D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents