Formulation and Clinical Evaluation of Sodium Benzoate Oral Solution for the Treatment of Urea Cycle Disorders in Pediatric Patients.

BACKGROUND: Sodium benzoate, a common food preservative, is used in the treatment of patients with urea cycle disorders (UCDs) as it stimulates ammonia removal by a non-urea cycle-based pathway. Despite its use in the clinical routine, no commercially available oral formulations currently exist. Liquid formulation is normally well accepted in pediatric age and allows precise dosage according to the children's needs. AIMS: (1) To prepare an oral sodium benzoate solution in different tastes and determine its stability, palatability, and tolerability and (2) to describe the long-term follow-up of two pediatric patients with UCDs treated with our formulation. METHODS: We prepared five oral solutions of sodium benzoate (200 mg/ml) by adding different flavoring agents. We measured drug concentration in the samples by high-performance liquid chromatography (HPLC). We evaluated palatability and tolerability with adult volunteers. Long-term drug compliance and metabolic control were appraised in two pediatric patients. RESULTS: All the oral solutions remained stable at room temperature along the 96-day test period, and they were well tolerated. The mint-flavored solution resulted the most palatable and preferred by adult volunteers. We report good drug compliance and good metabolic outcomes for both pediatric patients during the entire follow-up. CONCLUSIONS: Our study highlighted the stability and tolerability of flavored sodium benzoate oral solutions. These solutions were well accepted during a long-term follow-up and guaranteed a good metabolic control. Since taste attributes are critical to ensure acceptable medication adherence in the pediatric age, flavored liquid formulations of sodium benzoate may be an efficient strategy to achieve therapeutic outcomes in UCD pediatric patients.

Infantile cerebellar thrombosis: a case of lupus anticoagulants?

Lupus Anticoagulants (LA) pathology is a disease frequently associated with venous and/or arterial thrombosis. The authors describe a 6 year-old boy complaining cephalea and emesis, followed by lethargy, in whom antiphospholipids antibodies were detected. Brain magnetic resonance scans showed lesions of the cerebellum. The characteristics of LA disease are discussed.

[Hospital experience in active immunoprophylaxis against HBV]. / Esperienza ospedaliera di immunoprofilassi attiva anti HBV.

According to the Ministry of Health, at the paediatric section of Central Hospital of Rovigo, a vaccination campaign against HBV was undertaken in the year 1985 with the main purpose to reduce the sickness-rate and the mortality by HBV. Two vaccination patterns have been applied and they show to be efficient. 46 risk-subjects divided into two groups were vaccinated: group A: newborn, sons of women HBc Ag positive; group B: children living together with carriers or subjects sick of hepatitis B. The group A subjects had undergone to passive immunoprophylaxis at birth and to a vaccination cycle. The group B subjects only vaccination. The research of antibody titres produced by the vaccination demonstrated in the majority of cases greater than 121 IU/L. Only about 25% of vaccinated subjects showed a feeble movement of transaminases. The children's parents have cooperated very well.

[2 cases of Potter's syndrome]. / Due casi di sindrome di Potter.

In this report, we describe two patients with Potter's syndrome classical signs and take the opportunity to discuss about the etiopathogenetic hypothesis concerning various phenotypical expressions of that syndrome.

Serum glutamic-oxaloacetic transaminase and glutamic-pyruvic transaminase activity in premature and full-term asphyxiated newborns.

The serum activity of SGOT and SGPT is one of the more specific parameters of liver cell injury both in adults and in the pediatric age-group. The determination of serum transaminase activity could offer a routine and rapid laboratory test for establishing the presence of hepatic cellular damage following intrauterine or perinatal asphyxia. In fact, it appears that there is a correlation between hypoxia and the increase in serum activity of transaminases in full-term and premature asphyxiated newborns. However, this increase is reversible up to the 30th day of life. The behavior of transaminase enzymatic activity in premature asphyxiated newborns compared to full-term asphyxiated newborns suggests a higher resistance of membranes to hypoxic-ischemic injuries and a lower enzymatic pool of cellular metabolism in premature newborns. Therefore, knowledge of the behavior of SGOT and SGPT activity may have important implications in the diagnosis and early treatment of perinatal asphyxia.

[Treatment of diaphragmatic paralysis in the newborn infant]. / Il trattamento della paralisi diaframmatica del neonato.

The paralysis of the diaphragm in the newborn is a rare pathological event. The "paradoxical movement" of the affected emidiaphragm can sometimes determine a important respiratory insufficiency. Medical treatment involves supplying oxygen, CPAP by means of a nasal cannula or mechanical ventilation with PEEP. Surgical plication of the affected emidiaphragm is recommended when a regular diaphragmatic function is not restored at 5-6 weeks of age and in the presence of serious respiratory insufficiency. Three cases are reported in this article which needed different therapeutical approaches.

[Development of the sympatoadrenergic function in the fetus and the newborn infant]. / Sviluppo della funzione simpatoadrenergica nel feto e nel neonato.

An analysis of the available data concerning the events bringing to the completion of the adrenergic function in early life shows the existence of an early response making the detection of MOPEG-SO4 levels in the urine--the foetus and in the new-born. This response isn't the same in the adult and in the infant as in the latter the number of receptors is lower, the prevalent mediator is NA and the receptorial subtype is almost entirely beta. The effector system, on the contrary, is as fully developed in the infant as it's in the adult. Maybe it will be possible in the future to evaluate--through non-invasive methods such as the detection of MOPEG-SO4 levels in the urine--the degree of development of the central simpatoadrenergic system in order to exactly work out perinatal asphyxia and brains congenital injuries.