Aim of the study: Posttransplant morbidity and clinical outcome in children with advanced neuroblastoma (NBL) who underwent megachemotherapy followed by HSCT were investigated.
Patients and methods: In the study 73 children with advanced NBL treated in four Departments of Paediatric Haematology and Oncology in Lublin, Kraków, Wrocław and Bydgoszcz from 1995 to 2004 were analysed. Median age of children was 4.9 years (range 1.8 to 15). Reinfusion of CD34 cells followed myeloablative chemotherapy with Busulfan / Melfalan in 58 patients; Treosulfan / Melfalan in 2 patients; Melfalan / VP16/ Carbo in 9 patient, Melfalan alone in 3 patients and Thiotepa /CTX/ Carbo in 1 patient. Stem cells from peripheral blood were used in 57 cases, bone marrow in 10 patients, bone marrow and peripheral blood in 6 patients.
Results: 41/73 (56%) children are alive with median follow up 12 months (range 3 to 68 months), 29 children are in complete remission (CR), 12 patients are in partial remission (PR). 32/73 (44%) children died, 26 of them due to progressive disease; six children died due to posttransplantation complications. Overall survival (OS) at median observation time 12 months is 0.65; disease free survival (DFS) is 0.58. Probability of 5-year OS and DFS in the group of children transplanted in first partial/complete remission are 0.42 and 0.4 respectively.
Conclusions: Treatment with megachemotherapy followed by autoHSCT in patients with advanced neuroblastoma has not many adverse effects. Probabilities of 5-year OS and DFS are higher in the group of transplanted children in 1 partial/complete remission than in children transplanted after relapse.