Results of Immunosuppressive Therapy in Children with Severe Aplastic Anaemia (SAA). Report Polish Pediatric Hematology Group (Warszawa, Lublin, Krakow, Wroclaw, Zabrze, Bydgoszcz, Poznan, Lodz, Bialystok, Szczecin, Gdansk).

Introduction: Bone marrow transplantation (BMT) from HLA identical family donors is the treatment chosen, according indication of Polish Pediatric Hematology Group, for children with severe aplastic anemia (SAA). When no donor is available, combined immunosuppressive therapy (IST) is given.

Material and method: SAA was recognized in 105 children (42 girls, 73 boys) aged 2–18 years in eleven pediatric hematological center in Poland between 1993–2006 years. All patients received IST according Polish Pediatric Hematology and Working Party SAA of the EBMT protocol: antithymocyte globulin horse or rabbit, cyclosporine A (CSA), prenisolon and in 79 patients G-CSF or GM-CSF was additionally administered. Hematological response was evaluated on day 84 and 180 of therapy.

Results and conclusion: On day 180 remission was obtained in 80 from 105 patients (76.1%), complete remission (CR) in 53 patients (51.5%), partial remission (PR) in 27 (24.7%), There was no response to treatment (NR) in 25 patients (23.8%). Period of observation was from 10 months to 13 years. During this time relapse occurred in 28 patients (25.7%). Patients with NR and relapse were treated second course of IST or BMT from matched bone marrow donors. Unrelated BMT was performed in 15 patients with NR after IST. We observed 22 deaths (8 early during the first 3 months of IST) in all group. During the 13 year of observation we notice one case with late clonally complication (PNH), one case of transformation to AML and one to MDS. Probably of 13 year survival was 75.2% in our group. Our results are effective and compared to other hematological centers.