HEPATOMEGALY IN NEUROBLASTOMA STAGE 4S - CRITERIA FOR TREATMENT OF THE VULNERABLE NEONATE

Infants with neuroblastoma (NBL) frequently present as stage 4s and ov erall, such patients have a good prognosis. However, not all survive, and neonates with hepatomegaly are particularly at risk. We therefore reviewed our 4s experience, the objective being to identify lethal pat terns of disease progression. The specific aims of this work were (1) to develop a semiquantitative scoring system based on the severity of signs and symptoms that alone or in combination presaged a fatal outco me, and (2) to determine if early intervention could reverse life-thre atening disease. Thirty-five patients were seen over a period of 50 ye ars. The signs and symptoms of organ distress caused by hepatomegaly o ccurred in the lungs, kidneys, gastrointestinal tract (GI), the inferi or vena cava (IVC), and the liver. A scoring scale reflecting organ co mpromise was developed, the scores ranging from 0 (0 compromise) to 10 (all 5 systems showing evidence of impairment). Scores were derived f or 32 of 35 patients; 13 were 4 weeks old or under (neonates) when fir st seen, and 19 were aged 1-12 months (infants). Neonates were more li kely than infants to develop increasing symptomatology (50% versus 25% ) and were more likely to die when a score of 2 or more developed. Non e of the 6 neonates who did so survived despite treatment, compared Wi th three of four infants. Early intervention is recommended: (1) for 4 s neonates who develop a score of 1 and (2) for older infants with a s core greater than or equal to 2. (C) 1996 Wiley-Liss, Inc.