EUR-1008 pancreatic enzyme replacement is safe and effective in patients with cystic fibrosis and pancreatic insufficiency.

BACKGROUND: EUR-1008 (Zenpep [pancrelipase]) is a new, enteric-coated, porcine-derived pancreatic enzyme product (PEP) developed for the treatment of cystic fibrosis (CF) patients with malabsorption associated with exocrine pancreatic insufficiency (EPI). Unlike currently marketed PEPs, EUR-1008 contains the label-claimed lipase content. Safety and efficacy were assessed in younger (<7 years) and older (> or =7 years) CF patients with EPI. METHODS: Two multicenter studies were conducted: a randomized, double-blind, placebo-controlled, crossover trial in patients > or =7 years of age (N=34) and a supplemental, open-label study in children <7 years of age (N=19). Use of any medications altering gastric pH/motility was prohibited during the studies. Outcome measures in the randomized trial included changes in the coefficient of fat absorption (CFA), coefficient of nitrogen absorption (CNA), and signs/symptoms of malabsorption for EUR-1008 vs. placebo. Outcome measures in the supplemental study included safety and response (defined as no steatorrhea and no overt signs/symptoms of malabsorption) to EUR-1008 vs. previous enzyme treatment. RESULTS: In the randomized trial, EUR-1008 treatment compared to placebo resulted in a significantly higher mean CFA (88.3% vs. 62.8%, respectively) and CNA (87.2% vs. 65.7%, respectively) (both p<0.001) and reduced the incidence of malabsorption signs and symptoms in 32 evaluable patients. In the supplemental study, 11 of 19 patients met the criteria for responder with EUR-1008 at the end of the study vs. 10 of 19 patients at screening (previous PEP), and improvements in clinical symptoms were reported with EUR-1008 treatment. EUR-1008 was safe and well tolerated, and no serious drug-related AEs were reported in either study. CONCLUSIONS: EUR-1008 was safe, well tolerated, and effective in CF patients of all ages with EPI-associated malabsorption in two clinical trials. Treatment led to clinically and statistically significant improvements in CFA and CNA in the randomized study, and control of malabsorption and clinical symptoms in both studies.

Diagnosis of interstitial lung disease in children.

Chronic interstitial lung disease (ILD) in infants and children is a challenging diagnostic clinical problem. There are many unresolved and controversial issues in the diagnosis of this heterogeneous group of uncommon disorders in children. Diagnosis requires a high index of suspicion as the initial clinical manifestations are subtle, highly variable and non-specific. There is no consensus for the clinical diagnostic criteria of paediatric ILD. The spectrum of clinical findings is highly variable. The diagnostic evaluation of a child with suspected ILD includes a comprehensive history, physical examination, oxygen saturation (at rest, during exercise or during feeding), a plain chest x ray and a high-resolution thin-cut tomography scan of the chest. Pulmonary function studies can be useful in older children; these typically show a restrictive pattern with a decreased forced expiratory volume in 1 s (FEV(1)), forced vital capacity (FVC) and total lung capacity, but normal FEV(1)/FVC. A systematic approach to diagnosis is useful in the evaluation of an infant or child with suspected chronic ILD. Due to the rarity of most of these disorders, multi-centre collaboration is needed to improve our understanding of this orphan lung disease.

Utilidad de la biopsia muscular en el diagnóstico del síndrome de Reye / Usefulness of muscular biopsy in the diagnosis of Reye's syndrome

Se estudiaron cinco niños con síndrome de Reye: de ellos cuatro lactantes, que cursaron con el padecimiento en su forma grave, con crisis convulsivas tempranas y evolución tórpida, diferente a la descrita para pacientes de mayor edad, lo que dificultó clasificarlos inicialmente en forma adecuada. El tratamiento estuvo dirigido básicamente a mantener las constantes vitales y disminuir el edema cerebral. Un paciente murió y tres quedaron con secuelas neurológicas graves. En todos los casos se efectuó biopsia muscular y hepática, a los cuales se les practicó tinción de rojo oleoso. Al microscopio de luz, se encontró infiltración grasa microvesicular dentro de las células musculares. Ya que los pacientes con sindrome de Reye presentan defectos de la coagulación y la biopsia hepática puede incrementar las complicaciones por sangrado, la biopsia muscular puede ser una alternativa útil para establecer el diagnóstico en pacientes con datos clínicos que sugieran esta entidad. Sin embargo la infiltración grasa del músculo estriado no está establecida como un procedimiento rutinario en el diagnóstico de esta enfermedad

Hiperpigmentacion cutanea difusa de mucosas y fondo de ojo asociada a lupus eritematoso sistemico. / Diffuse cutaneous hyperpigmentation of mucosae and fundus oculi associated with systemic lupus erythematosus

Se presenta el caso de una nina de 10 anos de edad con lupus eritematoso sistemico (LES), en la cual el signo clinico sobresaliente fue la hiperpigmentacion cutanea difusa. Las lesiones palmares que presento asociados al LES son una rareza ya que previamente solo se han descrito cuatro casos en la literatura medica. Por otra parte, la hiperpigmentacion de mucosas y fondo de ojo, tambien presentes en este caso, se han descrito unicamente en pacientes con colagenopatias que han recibido terapia con antipaludicos. En pacientes que presenten hiperpigmentacion cutanea difusa de mucosas y de fondo de ojo, debe considerarse al LES dentro del diagnostico diferencial