Patients with nephroblastoma have a high risk of relapse if they present with stage IV and anaplastic histology or with extraregional lymph node involvement; at time of relapse, bad prognosis is heralded by recurrence in an irradiated region, second or subsequent relapse, early first relapse (6 months after nephrectomy), relapse with adverse histology, relapse in two or more organ systems and lymph node or bone metastases at relapse. For such patients, an attempt to increase survival by high-dose chemotherapy seems to be justified. In 8 children aged 7.3 years (3.8-14.7) treated within the German Nephroblastoma Study SIOP9/GPOH, high dose chemotherapy with autologous hematopoietic rescue was instituted for the following reasons: stage IV with anaplastic histology and extra-regional lymph nodes (1), second or subsequent pulmonary relapse (3), early relapse with diffuse pleural (1) or skeletal (1) dissemination, recurrence in irradiated area (1) and lung metastases after early local relapse (1). At megatherapy, the patients were in first (1), second (2), third (2) and fourth complete (2) or in partial remission (1). The high dose regimen consisted of carboplatin, etoposide and melphalan. Treatment related toxicity, all non-lethal, included acute but reversible renal failure (1), esophagitis with need of parenteral nutrition for 6 months (1), cardiomyopathy (3; chronic in 1). After a follow-up of 2.1 years (0.5-3.7), 6/8 patients survive in complete remission; for 5 of them who are in remission since > 18 months a recurrence is unlikely. Both children with local relapse died after a further recurrence 3 and 8 months after high dose treatment.
Conclusion: High dose consolidation with ASCR seems to effectively ameliorate the prognosis of patients with high risk nephroblastoma.