Assessment of respiratory involvement in children with mucoplysaccharidosis using pulmonary function tests

Mucopolysaccharidosis [MPS] are classified into seven clinical types based on eleven known lysosomal enzyme deficiencies of glycosaminoglycan [GAG] metabolism. Respiratory involvement seen in most MPS types includes recurrent respiratory infections, upper and lower airway obstruction, tracheomalacia, restrictive lung disease, and sleep disturbances. To delineate the pattern of respiratory compromise and pulmonary function abnormalities in MPS patients. This is a cross section observational study conducted on 30 patients recruited from the Neurometabolic Clinic, Children's Hospital, Cairo University over a period of 18 months. All patients were screened first by the quantitative determination of GAGs in urine, and diagnosis was confirmed by unidimensional electrophoresis for GAGs in urine and/or specific enzymatic assay in blood leucocytes. Infant pulmonary functions [IPFT] were done in twenty-two patients [< 3 years of age], while 8 cases performed impulse oscillometry [IOS] test [3-6 years of age]. Ages at diagnosis ranged from 1 to 9 years with a median of 2.3 years. Male to female ratio was 4:1. Consanguinity was observed in 53.3% whereas similar family condition was present in 40% of cases. Lumbar kyphosis was detected in 60% of cases, while scoliosis was detected in 46.7%. Results of pulmonary functions were mainly obstructive in 20 [66.6%] cases; however, combined obstructive and restrictive were detected in only 6 [20%] of cases. Data showed no association between the presence of scoliosis or the presence of organomegaly and the pattern of pulmonary function abnormalities. Evaluation and follow up of patients with MPS using pulmonary function tests are essential to detect early involvement of respiratory system and hence start treatment for respiratory complications early in the course of the disease

Empirical antibiotic treatment and the risk of necrotizing enterocolitis and death in very low birth weight neonates

Antibiotics are one of the most overused drugs in the neonatal unit. Our objective was to assess associations between the duration of the initial antibiotic course and subsequent necrotizing enterocolitis [NEC] and/or death in very low birth weight [VLBW] neonates with sterile initial postnatal culture results. A retrospective cohort analysis of VLBW neonates admitted to a tertiary center during the period from 1 January 2008 to 31 December 2009. The study included VLBW neonates who had been inborn and admitted to the neonatal intensive care unit within the first 24 hours after birth. We used descriptive statistics to characterize the study population, and multivariate analyses to evaluate association between therapy duration, prolonged empirical therapy, and subsequent NEC and/or death. Of 328 VLBW neonates admitted to our center, 207 [63%] survived >5 days and received initial empirical antibiotic treatment for >/= 5 days. The median duration of initial empirical antibiotic therapy was 7 days [range 5-10 days]. Those neonates were more likely to be of younger gestational age, lower birth weight, and to have lower Apgar scores [P<.001, .001 and .017, respectively]. Each empirical treatment day was associated with increased odds of death [OR 1.45, CI 1.24- 1.69], NEC [OR 1.32, CI 1.05-1.65], and the composite measure of NEC or death [OR 2.13, CI 1.55-2.93]. The use of prolonged initial empirical antibiotic therapy in VLBW neonates with initial sterile culture results may be associated with an increased risk of NEC or death and should be used with caution