Cytotoxic effects of sodium phenylbutyrate on human neuroblastoma cell lines.

Sodium phenylbutyrate (NaPB) is used in urea cycle disorders. We screened 6 neuroblastoma cell lines for in vitro potency of NaPB as an antiproliferative agent, evaluated multiple dosing schedules, and assessed its activity in combination with clinically active agents for neuroblastoma. We determined that NaPB achieves a 30-80% growth inhibition at 5 mM. Repeated dosing and prolonged drug exposure enhanced the cytotoxic effect. NaPB had additive cytotoxic effects when administered with vincristine; however, NaPB did not affect the activity of etoposide, adriamycin, 4-hydroxycyclophosphamide or cisplatinum. These results suggest that NaPB is an active agent against neuroblastoma and could be combined with vincristine in novel chemotherapy regimens.

Successful treatment of life-threatening acute chest syndrome of sickle cell disease with venovenous extracorporeal membrane oxygenation.

PURPOSE: We describe a pediatric patient with sickle cell disease and life-threatening acute chest syndrome who was successfully treated with venovenous extracorporeal membrane oxygenation (ECMO). PATIENT AND METHODS: An 8-year-old boy with sickle cell disease presented with vaso-occlusive crisis, which progressed to fulminant acute chest syndrome requiring a partial exchange transfusion and mechanical ventilation. Despite very high ventilator settings and significant barotrauma, hypoxia persisted and circulatory failure occurred. He was then successfully treated with venovenous ECMO for 11 days. One month after decannulation he had a seizure associated with abnormalities on magnetic resonance images (MRIs). His disease has been managed with a chronic transfusion program since then. Follow-up after 5 years reveals normal pulmonary function tests, a normal magnetic resonance angiogram (MRA), and above-average cognitive skills. CONCLUSION: This is the first report of a pediatric patient with acute chest syndrome successfully managed with venovenous ECMO. His course was complicated by a seizure associated with MRI abnormalities, although the outcome has been excellent. This case suggests that treatment with venovenous ECMO should be strongly considered for sickle cell patients with life-threatening acute chest syndrome, despite maximal conventional support.