Extrapolation : Experience gained from original biologics

Altres ajuts: Acord transformatiu CRUE-CSICBiologicals undergo modifications throughout their commercial lifecycle. Major changes can unintentionally magnify their inherent physicochemical variability. Although trials comparing the pre- and the post-change versions have been requested occasionally, analytical comparison is the most sensitive approach to anticipating clinical equivalence. Therefore, it may be concluded, by means of 'extrapolation', that non-identical versions of a given biologic will behave equally in all indications. Despite the lessons learned with original biologics, there are still controversies around the approval of biosimilars through extrapolation. Here, a comprehensive analysis of scattered information allows for an account of cases of original biologic versions approved in some indications with no patient trials involved. Healthcare professionals can be reassured that inasmuch as extrapolation has proven valid for new versions of original biologics, the same holds for biosimilars

Análisis Costo-Beneficio de la Fluoración del Agua Potable en Niños de 12 años de la Región del Biobío, Chile.

Fluoridation has been shown to be an effective measure against caries in children. The present study evaluates the cost-benefit of the fluoridated water program for the reduction of dental caries in 12-year-old children in the Biobío Region, the only region in Chile that has not implemented this program. An economic cost-benefit evaluation was carried out, comparing two alternative interventions: non-fluoridated drinking water versus fluoridated drinking water. The prevalence of caries, direct and indirect costs of the treatments, the cost of implementing the programs and the benefits of both interventions were estimated. From this study it is concluded that the savings in oral health costs in 12-year-old children when using fluoridating drinking water in the Biobío region is significantly higher than the cost involved in implementing the water fluoridation program, resulting in total savings for the Chilean state of $129,861,645 (USD$ 152,833) as well as an estimated reduction of 15% in the history of caries in the study population.Se ha demostrado que la fluoración es una medida efectiva contra disminución de la caries en la población infantil. La presente investigación buscó evaluar cual es el costo-beneficio del programa del agua fluorada para la disminución de caries dental en niños de 12 años de la Región del Biobío, única región de Chile que no adhiere a este programa. Se realizó una evaluación económica de costo-beneficio, comparando dos intervenciones alternativas: agua potable no fluorada versus agua potable fluorada. Para tal fin se estimó la prevalencia de caries, costos directos e indirectos de los tratamientos, el costo de implementación de los programas y el beneficio de ambas intervenciones . De este estudio se concluye que el ahorro en costos de salud bucal en niños de 12 años al fluorar el agua potable en la región del Biobío, es significativamente mayor al costo que implica la implementación del programa de fluoración de aguas, lográndose un ahorro total para el Estado de $129.861.645 (USD$152.833) así como una estimación de reducción del 15% en la historia de caries en la población de estudio

Genetic and metabolic urgencies in the neonatal intensive care unit: maple syrup urine disease

La enfermedad de la orina con olor a jarabe de arce (EOJA) es un trastorno hereditario del metabolismo de aminoácidos ramificados causada por mutaciones en el complejo mitocondrial Deshidrogenasa de Cetoácidos de Cadena Ramificada (BCKDH). La disfunción de este complejo genera la acumulación tóxica de aminoácidos de cadena ramificada (BCAA), lo que conlleva un especial impacto en el sistema nervioso central. La acumulación de sustratos provoca encefalopatía aguda neonatal severa, rápidamente progresiva y catastrófica si no se instaura a tiempo el tratamiento. Esta entidad tiene un manejo médico específico en fase aguda y un tratamiento a largo plazo para evitar secuelas y mortalidad. De ahí la importancia de tenerla en cuenta como diagnóstico diferencial. Se presenta a continuación un caso colombiano de un paciente con EOJA de presentación clásica neonatal con desenlace fatal, como ejemplo de urgencia metabólica y diagnóstico diferencial en un neonato encefalopático.Maple syrup urine disease (MSUD) is a hereditary disorder of branched chain amino/keto acid metabolism, caused by a decreased activity of the branched-chain alpha- ketoacid dehydrogenase complex (BCKAD), which leads to abnormal elevated plasma concentrations of branched-chain amino acids (BCAAs) clinically manifested as a heavy burden for Central Nervous system. The toxic accumulation of substrates promotes the development of a severe and rapidly progressive neonatal encephalopathy if treatment is not immediately given. This disorder has a specific medical management in acute phase in order to minimize mortality and morbidity. For all those reasons, it is important to include the MSUD as a possible diagnosis in a encephalopathic newborn. We present a colombian newborn with classical MSUD with fatal outcome as an example of metabolic emergency and a differential diagnosis in the encephalopathic newborn