First-Line Immunosuppressive Treatment in Children with Aplastic Anemia: Rabbit Antithymocyte Globulin.

Advances in experimental medicine and biology

PubMedID: 25310948

Pawelec K, Salamonowicz M, Panasiuk A, Demkow U, Kowalczyk J, Balwierz W, Zaleska-Czepko E, Chybicka A, Szmyd K, Szczepanski T, Bubala H, Wysocki M, Kurylak A, Wachowiak J, Szpecht D, Mlynarski W, Bulas M, Krawczuk-Rybak M, Leszczynska E, Urasinski T, Peregud-Pogorzelski J, Balcerska A, Kaczorowska-Hac B, Matysiak M. First-Line Immunosuppressive Treatment in Children with Aplastic Anemia: Rabbit Antithymocyte Globulin. Adv Exp Med Biol. 2014;.
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.