High dose melphalan and autologous bone marrow transplantation for solid tumours of childhood

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Abstract
Between October, 1977 and December, 1984, 61 children aged 5 months to 18 years (median 6.5) with solid tumours have been treated with high dose melphalan (HDM) 100-240 mg/m2 with autologous non-cryo-preserved bone marrow "rescue" to increase the rate of haematological recovery. Of these 61, 60 were evaluable on December 31st 1984: one patient was lost to follow up. Thirty-two patients received HDM and marrow autograft after conventional chemotherapy with or without radiotherapy (16 neuroblastoma, 6 rhabdomyosarcoma, 9 Ewings, 1 osteosarcoma) while 28 patients received this treatment after relapse following conventional chemotherapy (7 neuroblastoma, 9 rhabdomyosarcoma, 6 Wilms, 2 Ewings, 1 histiocytosis X, 1 carcinoma of the ovary, 1 yolk sac tumour, and 1 nasopharyngeal carcinoma). The median duration of leucopenia was 11 days (range 5-36 days). The other immediate toxicities due to melphalan were severe vomiting and nausea, mucositis in most patients had convulsions in 6. Of the 32 patients treated in first remission. 9 are now disease-free at 1 to 82 months. Of the 28 patients treated after relapse, 3 are alive without disease at 6-86 months. HDM produces responses in patients with disease resistant to other agents, particularly with Wilms'' tumours and rhabdomyosarcoma, and occasionally these responses are durable. It seems to be less effective for children with Stage IV neuroblastoma.