This paper reports late effects and health status of 198 children who had cancer or leukemia diagnosed under 2 years of age and their therapies electively withdrawn. This series (92 neuroblastoma [NBL], 57 Wilms' tumor [WT], 46 acute lymphoblastic leukemia [ALL], and 3 non-Hodgkin's lymphoma) was followed for 1-12 years after discontinuation of therapy. Thirty-three children were diagnosed before 1973, 92 between 1973 and 1977, and 73 after 1977 in 16 Italian Pediatric Oncology Centers. As of December 1983, 176 children were reported to be alive and without evidence of primary cancer by physicians responsible for their care. One child died from a second primary tumor, two from late recurrences of the primary cancer, and three from other causes; eight were alive with evidence of primary cancer; and eight were lost to follow-up. Kyphoscoliosis was found in 22 children and other musculoskeletal anomalies in 8. Neurological sequelae were observed in 8 out 35 children with ALL treated with radiotherapy (RT) and intrathecal methotrexate. All but one were in continuous complete remission when they developed seizures (three cases), leukoencephalopathy (three cases), or intracerebral calcifications (two cases). One child had cardiomyopathy and subsequently died from cardiac failure: he had received doxorubicin (400 mg/m2) and mediastinal RT (13 Gy) for NBL. Growth impairments were observed in children with NBL and WT.

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A total of 290 children off therapy after acute lymphoblastic leukemia, in continuous complete remission for at least 2 years, were evaluated for height at the onset of the disease and at the most recent clinical visit (median time after suspension of treatment 4 years 4 months, range 2 years-11 years 3 months). All patients had been treated with multidrug schedules; intrathecal drugs had been given to 84% of the patients for prevention of CNS involvement, associated with radiotherapy. The height percentiles at the most recent examination were shifted downward significantly compared with the expected pattern (p less than 0.001). The effect on stature was much more marked in girls, with a reduction of height percentiles at most recent examination from expected and from diagnosis; in males there was a reduction from diagnosis to latest follow-up, but the values were within the limits of normal. The short stature was mostly observed in pubertal girls and in patients who had undergone radiotherapy.