Results of Immunosuppressive Therapy in Children with Acquired Severe Aplastic Anaemia (SAA). Report Polish Paediatric Leukaemia and Lymphoma Study Group.

Introduction: Bone marrow transplantation (BMT) from HLA identical family donors is the treatment chosen for children with (SAA). When no donor is available, combined immunosuppressive therapy (IS) is given. Material and method: SAA was recognised in 85 children (31 girls, 54 boys) aged 2–17.5 years in ten haematological clinic in Poland between 1993–2003 years. All patients received IS according SAA Working Party of the EBMT protocol: ALG or ATG, CsA, prenisolon and in 79 patients G-CSF or GM-CSF was additionally administered. Haematological response was evaluated on day 180 of therapy. Results and conclusion: Complete remission (CR) occurred in 43 patients (40.5%), partial remission (PR) on 22 (25.8%), no response (NR) was obtained in 20 children (23.7%). Period of observation was from 12 months to 10.5 years. During this time relapse occurred in 10 patients (8.5%). Patients with NR and relapse were treated second course of IS or BMT from matched bone marrow donors. Unrelated BMT was performed in 11 patients with NR after IS. We observed 17 deaths (10 early during the first 3 months of IS) in all group. At present 54 children (63.5%) is in first remission (CR or PR) with lasts from 12 months to 10 years. During the 10.5 year of observation we notice one case with late clonal complication (PNH). Transformation to neoplasma was not observed in none of our patients after IS.