Results of Immunosupresive Therapy in Children with Aquired Severe Aplastic Anaemia (SAA). Report Polish Pediatric Hematology Group

Abstract Introduction: Bone marrow transplantation (BMT) from HLA identical family donors is the treatment chosen, according indication of Polish Pediatric Hematology Group and Working Party SAA of the EBMT, 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 116 children (45 girls, 71 boys) aged 2–18 years in ten pediatric hematological center in Poland between 1993–2007 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 90 patients G-CSF or GM-CSF was additionally administered. Hematological response was evaluated on day 112 and 180 of therapy. Results and conclusion: On day 112 remission was obtained in 61children from 116, complete remission (CR) in 47 (40,5%), partial remission (PR) in 24(20,8%). There was no response (NR) to treatment in 45 (38,7). On day 180 remission was obtained in 88 from 116 patients (75,8%), CR in 57 patients (49,1%), PR in 31 (26,7%), NR in 29 patients (24,2%). Period of observation was from 8 months to 14 years. During this time relapse occurred in 32 patients (27.5%). Patients with NR and relapse were treated second course of IST or BMT from matched bone marrow donors. Unrelated BMT was performed in 18 patients with NR or relapsed after IST. We observed 27 deaths (8 early during the first 3 months of IST) in all group. During the 14 year of observation we notice one case with late clonally complication (PNH), one case of transformation to AML and one to MDS. Probably of 14year survival was 76,7% in our group. Our results are effective and compared to other hematological centers.