Klin Onkol 2003; 16(Suppl 2003): 119-121.
Summary: Megatherapy with autologous hematopoietic stem cell transplantation (AHSCT) is now an established treatment modality in oncology. The European register has more than 10 000 entries in recent years. During the 1980s and early 1990s highdose chemotherapy (HDC) with AHSCT has become a treatment choice for children with high-risk or relapsed malignancies. During the period from 1992 to 2001 we transplanted 215 children and adolescents with poor prognosis cancer. There were 57 children with neuroblastoma, 54 with Ewing’s sarcoma, 47 with malignant lymphomas, 20 with advanced germ cell tumors, 25 with soft tissue sarcoma, 6 with recurrent medulloblastoma, 4 with nephroblastoma and two with relapsed osteosarcoma. A total of 46% of patients are alive in complete remission. The most favourable outcome had children with extracranial germ cell tumors (75%), Hodgkin’s lymphoma (65%) and Ewing’s sarcoma (48%). Peritransplant mortality was 8%. Our experience confirms that megatherapy with AHSCT is effective therapeutic modality for selected children with high-risk chemosensitive solid tumors. Unfortunately, relapse of the original malignancy remains the main problem after transplantation. Future studies will focus on developing novel drug combinations, and post-transplant immunotherapy. Advances in cytokine therapy, tumor specific vaccines, and gene therapy may decrease recurrence of the malignancy after HDC. Many important questions about the optimal usage of megatherapy and bone marrow transplantation in pediatric solid tumors remain unanswered and more effort is needed in order to include larger numbers of children in important phase III clinical trials.
