Klin Onkol 1997; 10(5): 148-151.
Despite the improved prognosis of patients with localized rhabdomyosarcoma (RMS), patients with primary metastatic or recurrent disease have a very poor prognosis. Before introduction of high-dose therapy, 5 year survival rates were between 18-30%. With the main goal to improve survival in high risk rhabdomyosarcoma patients, many centers performed megatherapy using different types of hematopoietic rescue (predominantly autologous bone marrow or peripheral blood progenitor cell.) We report retrospective analysis of the results of treatment of 14 children with primary metastatic (3pts), unfavourable (4 pts), or relapsed RMS (7) who were given megatherapy and autologous hematopoietic stem cell rescue (AHSCR). Six were in 1 st complete or partial remission, seven in 2 nd complete or partial remission at the time of megatherapy, and one child was transplanted with progressive disease. Conditioning consisted mostly of melphalan, carboplatin, etoposide (total body irradiation, 13 pts grafted). There was one toxic, transplant-related death. Five of fourteen (35,7%) patients are alive, event-free survivals with a median follow up 3,2 years. Eight patients relapsed and died. The average time from AHSCT to relapse was 9 months (4-24). Patients who had undergone megatherapy in first remission did better (EFS 67%) than those transplanted in second or third remission (EFS 14%). The role of megatherapy followed by autologous hematopoietic stem cell rescue remain unclear regardless of the slightly better looking result. Further investigation of AHSCT for sarcoma patients should be performed as part of controlled, prospective, randomised multicentric trials.
