High-dose multiagent chemotherapy followed by autologous marrow rescue was used in the treatment of 13 patients with Stage III or IV childhood tumors. Encouraging results are being obtained in abdominal lymphoma (1/3 complete remissions (CR); rhabdomyosarcoma (2/4 CR); and retinoblastoma (1/2 CR). In neuroblastoma, the results are disappointing, with only one of four patients in CR; this patient developed a lymphoma associated with Epstein-Barr virus infection. Marrow reconstitution was obtained in 11 patients, with recovery of neutrophils to greater than 0.5 x 10(9)/liter between six and 30 days and platelet recovery to greater than 50 x 10(9)/liter between seven and 38 days. Investigations on the numbers of cells or committed granulocyte precursors ()CFU-c's) infused and parameters of hematologic recovery show poor correlation and suggest that a more accurate and reliable assay for the predictability of cryopreserved marrow to reconstitute marrow function within a reasonable time is necessary. Nonhematologic toxicities of high-dose multiagent chemotherapy are the principal dose-limiting factors.
Toogood, I. R. G., Ellis, W. M., and Ekert, H. (1979) Aust. Paediatr. J., 15, 91–95. Prognostic criteria, treatment and survival in disseminated histiocytosis X. Twenty‐five children with disseminated histiocytosis X, diagnosed between 1969–75, were clinically grouped into those without organ dysfunction (Group I) and those with organ dysfunction (Group II). They were treated with either oral chlorambucil (CBL) or combination chemotherapy with vinblastine and other agents. Children less than three years of age at the commencement of treatment had Group II disease more frequently (p = 0.02), and children with Group I disease had significantly longer survival (p = 0.04). Oral histiocytosis X was present in 10 children and is frequently associated with diabetes insipidus (p<0.001). Initial response to chemotherapy did not predict prognosis (p = 0.38). Treatment with CBL alone was effective in all children with Group I disease over the age of three years at the onset of symptoms. However, combination chemotherapy appeared to be necessary in children with Group II disease from the time of diagnosis. and in children with Group I disease whose symptoms occur before the age of three years. Chemotherapy is associated with minimal toxicity and has resulted in a survival rate of 80% with a 37‐month median follow‐up of survivors (range 2–118 months).
Intensive chemotherapy followed by infusion of cryopreserved autologous bone marrow (ABMR) was used in the treatment of 22 children with advanced tumours. In nine this was their initial therapy; in eight it was used after partial or complete remission had been achieved with standard therapy; and in five, after relapse had occurred. Recovery of marrow function occurred in 20 patients with a mean time of 13.2 and 18.2 days to recovery of neutrophils p0.5 x 1 09/1) in newly diagnosed and previously treated patients respectively. Platelet count recovery to >50 x 109/1 occurred in a mean time of 13.4 days in newly diagnosed and 20.4 days in previously treated patients.
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