Children's Memorial Hospital

About: Children's Memorial Hospital is a based out in . It is known for research contribution in the topics: Population & Transplantation. The organization has 5652 authors who have published 8967 publications receiving 283837 citations.

Unique aspects of human newborn cerebral metabolism evaluated with phosphorus nuclear magnetic resonance spectroscopy.

Abstract: In vivo phosphorus nuclear magnetic resonance spectroscopy (31P NMR) was used to evaluate the pattern of phosphate compounds in seven newborn babies (mean gestational age, 32 weeks; birth weight, 1,430 gm; age, 37 days) with a history of perinatal asphyxia. Spectra were collected in a 1.9 Tesla superconductive magnet with surface coil techniques. The spectra had characteristic peaks for phosphorylated monoesters (PME), inorganic phosphate (Pi), phosphodiesters (PD), phosphocreatine (PCr), and ATP. In contrast to cortical spectra from mature animals, these newborn infant 31P NMR spectra were dominated by a large PME peak and had small PCr, Pi, and PD peaks. Intracellular pH, as measured from the chemical shift of the Pi peak relative to the PCr peak, was 7.1 +/- 0.1 (SD). We studied one infant postmortem, and a large PME peak was present in his spectrum. The presence of PME 3 hours after death strongly suggests that it is not a sugar phosphate. In NMR spectroscopy, compounds are identified by their chemical shift relative to a known standard (PCr); the chemical shift of the PME peak was 6.5 ppm, suggesting that it is a mixture of phosphoryl ethanolamine and phosphoryl choline. The PCr/Pi ratio (1.3 +/- 0.7) and the PCr/ATP ratio (0.7 +/- 0.4) were lower in these babies than in mature animals (greater than 2 and greater than 1.4, respectively); the PME/PD ratio (1.2 +/- 0.6), however, was much greater in the infants (mature animals, less than 0.2). These findings suggest that there are unique aspects of human newborn cerebral metabolites and bioenergetic reserve.

The effect of enzyme replacement therapy on bone crisis and bone pain in patients with type 1 Gaucher disease.

Abstract: The effect of enzyme replacement therapy (ERT) on bone crisis and bone pain was investigated in patients with Gaucher disease (GD) type 1 followed over 4 years. Data from the International Collaborative Gaucher Group Gaucher Registry were used. Only patients with bone crisis and/or bone pain data for 1 year prior to ERT, and for each of 3 years after the start of ERT, were included. Bone crises were reported in 17% of patients during the year before starting ERT. The frequencies of bone crises decreased to 5%, <1% and 3% for 1, 2, and 3 years after initiation of treatment, respectively (p < 0.0001). Bone pain followed a similar pattern of response. Bone pain was reported in 49% of patients the year before treatment and decreased to 30% in the first year, 29% in the second year, and 30% in the third year of ERT (p < 0.0001). ERT is associated with a reduction in bone crisis and bone pain in patients with GD type 1 . This study shows that significant improvements in symptoms of skeletal disease are achievable clinical outcomes and treatment goals in GD type 1.

Correction of extrahepatic portal vein thrombosis by the mesenteric to left portal vein bypass.

Abstract: OBJECTIVE The goal of this study was to determine the effectiveness of mesenteric vein to left portal vein bypass operation (MLPVB) in correcting extrahepatic portal vein thrombosis (EHPVT) in children. The treatment of idiopathic EHPVT has been primarily palliative, whereas MLPVB restores hepatic portal flow in patients with EHPVT. METHODS Thirty-four children with symptomatic EHPVT underwent surgery with intent to perform MLPVB and were followed for up to 7 years. MLPVB was successful in 31 patients (91%), all of whom maintain patent vein grafts and have symptomatic relief of EHPVT in follow-up. All patients had complete relief from gastrointestinal bleeding. Patients with hypersplenism had significant increases in platelet and leukocyte counts and reduction in spleen size. Superior mesenteric vein flow increased from 119 +/- 66 mL/min before bypass to 447 +/- 225 mL/min (P < 0.0001) after surgery. Postoperative blood flow in the bypass graft expressed as a fraction of calculated ideal portal flow for size correlated inversely with age (P < 0.001). Left-portal vein diameter increased from 2.6 +/- 1.6 mm to 7.3 +/- 2.4 mm 2 years after surgery (P < 0.002). Liver volume increased from 703 +/- 349 cm3 to 799 +/- 351 cm3 1 week after surgery (P < 0.001). Prothrombin time improved to normal in all patients 1 year after surgery. CONCLUSIONS MLPVB provides excellent relief of symptoms in children with idiopathic EHPVT and results in liver growth and normalization of coagulation parameters. This surgery is corrective and should be done at as early an age as possible.