This phase I/II first study of recombinant human GAA derived from CHO cells showed that rhGAA is capable of improving cardiac and skeletal muscle functions in infantile GSD-II patients. Further study will be needed to assess the overall potential of this therapy.

This phase I/II first study of recombinant human GAA derived from CHO cells showed that rhGAA is capable of improving cardiac and skeletal muscle functions in infantile GSD-II patients. Further study will be needed to assess the overall potential of this therapy.

ABSTRACT. Purpose. The prevalence of infective endocarditis (IE) among children with Staphylococcus aureus bacteremia (SAB) is unknown. The objective of this study was to determine prospectively the prevalence of IE among pediatric patients with SAB in a large tertiary care center.Methods. Between July 1998 and June 2001, all children who developed SAB whose parent/guardian signed informed consent underwent echocardiography. Clinical and follow-up results were collected prospectively. Endocarditis was classified according to the modified Duke criteria.Results. Fifty-one children developed SAB during the study interval. Definite (6 patients [11.8%]) or possible (4 patients [7.8%]) IE was present in 10 of 51 (20%) children with SAB. Most children (73%) developed bacteremia as a consequence of an infected intravascular device. Children with underlying congenital heart disease had a significantly higher prevalence of definite or possible IE, compared with those with structurally normal hearts (53% vs 3%). All patients with definite IE had multiple positive blood cultures. Mortality was high among patients with and without IE (40% vs 12%).Conclusions. In this study, the prevalence of definite IE among children with SAB was ϳ12% and was frequently associated with congenital heart disease and multiple positive blood cultures. The mortality for children with SAB and definite or possible S aureus IE is high. Pediatrics 2005;115:e15-e19. URL: www.pediatrics.org/cgi/

No abstract

No abstract

Patients with obstructed total anomalous pulmonary venous connection (TAPVC) usually present critically ill and continue to be extremely challenging with presurgical stabilization. We present an extra corporeal membrane oxygenation (ECMO)-dependent neonate with obstructed TAPVC that was successfully palliated with transvenous stent placement in the obstructed vertical vein.

No abstract

ObjectiveThis study compares the total hospital cost (HC) for one-stage versus "two-stage" repair of tetralogy of Fallot (TOF) in infants younger than 1 year of age. Summary Background DataTotal (one-stage) correction of TOF is now being performed with excellent results in infancy. Alternatively, a two-stage approach, with palliation of infants in the first year of life, followed by complete repair at a later time can be used. In some institutions, the two-stage approach is standard practice for infants younger than 1 year of age or is used selectively in patients with an anomalous coronary artery across the right ventricular outflow tract (RVOT), "small pulmonary arteries," multiple congenital anomalies, critical illnesses (Cl), which increase the risk of bypass (e.g., sepsis or DIC), or severe hypercyanotic spells (HS) at the time of presentation. The cost implications of these two approaches are unknown. MethodsThe authors reviewed 22 patients younger than 1 year of age who underwent repair of TOF at their institution between 1993 and 1995. Eighteen patients had one-stage (10) repair (mean age, 3.4 ± 3.1 months; range, 3 days-9 months) and 4 patients were treated by a staged approach with initial palliation (1.6 ± 0.4 month; range, 1.5-2 months) followed by later repair (14.75 ± 1.5 months; range, 13-16 months). The reasons for palliation were severe HS at time of presentation (two patients), anomalous coronary artery (one patient) and Cl (one patient). In the 18 patients undergoing 10 repair, 3 (16.6%) presented with HS, 6 (33.3%) had a transanular repair, and 6 (33.3%) were able to be repaired through an entirely transatrial approach (youngest patient, 1.5 months). The HC (1996 dollars) and hospital length of stay (LOS; days) were evaluated for all patients. The HCs were calculated using transition 1, which is a cost accounting system used by our medical center since July 1992. Transition provides complete data on all direct and indirect hospital-based, nonprofessional costs. 779