Clinical development strategy for the evaluation of the efficacy and safety of SURFACEN
Abstract
Background: the first report of the efficacy of an exogenous pulmonary surfactant in the treatment of the membrane hyaline illness dates back to 1980. From this finding, different groups of investigators worldwide dedicate their efforts to the development of pulmonary surfactants either natural or synthetic.
Objective: to develop a strategy for the evaluation of the efficacy and safety of Surfacen® in the treatment of respiratory distress syndrome that would allow its registration and introduction to normal practice.
Methods: to demonstrate the efficacy and safety of Surfacen® in the treatment of RDS in preterm infants, clinical trials and the pharmaco-vigilance were conducted. In other indications such as acute RDS in children and adults; clinical trials (phase II, III and IV) were conducted. In all the trials, the methodology established for each phase was followed.
Results: Surfacen® demonstrated its efficacy by improving oxygenation, ventilation variables, radiographic ones, clinical evolution and contributed to reduce mortality in preterm infants, with a safety profile similar to the rest of commercially available pulmonary surfactants internationally. Surfacen® therapy proved to be effective in improving oxygenation, ventilation variables, radiographic, clinical evolution, contributed to reduce mortality in children, (the latter not in adults) and proved to be well tolerated and safe to notify a reduced number of adverse events related to its administration. The days of stay in the intensive care units and days of mechanical ventilation were not modified with the treatment scheme evaluated.
Conclusions: Surfacen® was registered for the therapeutic indications studied and introduced in routine clinical practice contributing to reduce mortality in children.
DeCS: PULMONARY SURFACTANTS/administration & dosage; PULMONARY SURFACTANTS/therapeutic use; RESPIRATORY DISTRESS SYNDROME, NEWBORN/drug therapy; INFANT, PREMATURE; CLINICAL TRIAL.
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References
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