Ectopic platelet-delivered factor (F) VIII for the treatment of Hemophilia A: Plasma and platelet FVIII, is it all the same?

Hemophilia A is the most common inherited bleeding diathesis and is due to a deficiency of functional coagulation factor (F) VIII. Most patients have a severe deficiency and require a program of prophylactic plus acute infusions of recombinant FVIII to prevent significant joint and other target organ damage. One of the greatest challenges remaining in the care of these patients is that one fifth to third of the patients develop inhibitors to the infused proteins.

Preschool neurobehavioral outcome following fetal myelomeningocele surgery.

Objective: It was the aim of this study to assess the prevalence of preschool neurobehavioral problems in children following fetal myelomeningocele (fMMC) surgery.

Methods: Prior to the Management of Myelomeningocele Study, 30 fMMC patients underwent a standardized neurodevelopmental examination at 5 years of age. The prevalence of behavioral problems was assessed by the Child Behavior Check List (CBCL), which includes a total problem score and 2 broad-band indices (internalizing and externalizing problems).

Social isolation in adolescence alters behaviors in the forced swim and sucrose preference tests in female but not in male rats.

Social interactions in rodents are rewarding and motivating and social isolation is aversive. Accumulating evidence suggests that disruption of the social environment in adolescence has long-term effects on social interactions, on anxiety-like behavior and on stress reactivity. In previous work we showed that adolescent isolation produced increased reactivity to acute and to repeated stress in female rats, whereas lower corticosterone responses to acute stress and decreased anxiety-related behavior were noted in isolated males.

Fetal surgery for myelomeningocele: patient selection, perioperative management and outcomes.

Myelomeningocele (MMC), one of the most common congenital malformations, can result in severe lifelong disabilities,including paraplegia, hydrocephalus, Chiari II malformation(CM-II), incontinence, sexual dysfunction, skeletal deformations and mental impairment. MMC was the first nonlethal anomaly to be treated by fetal surgery. Experimental and clinical evidence suggests that the primary cause of the neurologic deficit associated with MMC is not simply incomplete neurulation but rather chronic mechanical injury and amniotic fluid-induced chemical trauma that progressively damages the exposed neural tissue during gestation.

Cytokine-induced osteopoietic differentiation of transplanted marrow cells.

Transplantation of whole bone marrow (BMT) leads to engraftment of both osteoprogenitor cells and hematopoietic cells; however, the robust osteopoietic chimerism seen early after BMT decreases with time. Using our established murine model, we demonstrate that a post-BMT regimen of either granulocyte-colony stimulating factor, growth hormone, parathyroid hormone, or stem cell factor each stimulates greater donor osteoblast chimerism at 4 months posttransplantation than saline-treated controls and approximates the robust osteopoietic chimerism seen early after BMT; however, only growth hormone led to significantly more donor-derived osteocytes than controls. Importantly, there were no adverse hematologic consequences of the different treatments.

The impact of certificate of need programs on neonatal intensive care units.

Objective: To determine the impact of state certificate of need programs (CON) on the number of hospitals with neonatal intensive care units (NICU) and the number of NICU beds.

Study Design: The presence of a CON program was verified from each state's department of health. Multivariable regression models determined the association between the absence of a CON program and each outcome after controlling for socioeconomic and demographic differences between states.

Doppler tissue imaging in children following cardiac transplantation: a comparison to catheter derived hemodynamics.

  Previous studies have demonstrated a correlation between E:E(a) and ventricular filling pressure in adults after heart transplantation. We sought to determine if E:E(a) correlates with filling pressure after heart transplantation in children. A prospective analysis of children who have undergone heart transplantation was performed.

Clinical utility and validity of the Functional Disability Inventory among a multicenter sample of youth with chronic pain.

The Functional Disability Inventory (FDI) is a well-established and commonly used measure of physical functioning and disability in youth with chronic pain. Further validation of the measure has been called for, in particular, examination of the clinical utility and factor structure of the measure. To address this need, we utilized a large multicenter dataset of pediatric patients with chronic pain who had completed the FDI and other measures assessing pain and emotional functioning.

Amniotic fluid levels of glial fibrillary acidic protein in fetal rats with retinoic acid induced myelomeningocele: a potential marker for spinal cord injury.

Objective: The objective of this study was to determine whether amniotic fluid levels of glial acidic fibrillary protein (GFAP) would reflect myelomeningocele-related neurodegeneration in the rat model of retinoic acid-induced myelomeningocele, which is a model that is very similar to human myelomeningocele and develops the entire spectrum of disease severity including features of the Chiari II malformation.

Study Design: Time-dated (embryonic day 10) pregnant Sprague-Dawley rats were gavage fed 60 mg/kg/bodyweight retinoic acid that had been dissolved in olive oil or olive oil alone. Myelomeningocele, retinoic acid-exposed no myelomeningocele, and control fetuses were harvested at specific time points throughout gestation.

Clinical indications for repeat MRI in children with acute hematogenous osteomyelitis.

Background: At our center and at others, some children with acute hematogenous osteomyelitis (AHO) are evaluated with multiple magnetic resonance imagings (MRIs) during their treatment. Do these serial MRI studies have a role in the management of AHO? We examine several clinical indications for ordering a repeat MRI and whether the imaging study resulted in a change in management.

Methods: A total of 59 children (60 cases) with AHO were imaged with more than 1 MRI.

The relationship of seizure activity and chronic epilepsy in early infancy and short-term neurodevelopmental outcome following fetal myelomeningocele closure.

We explored the relationship between seizure activity (SA) and/or chronic epilepsy (CE) and short-term neurodevelopmental outcomes following fetal myelomeningocele (fMMC) surgery. Retrospective databases and a parental questionnaire focusing on common complications of hindbrain herniation associated with MMC were used to determine the incidence of seizures following fMMC surgery. The Bayley Scales of Infant Development II was used to evaluate the neurocognitive outcomes.

Toward a definition of "fresh" whole blood: an in vitro characterization of coagulation properties in refrigerated whole blood for transfusion.

Background: The hemostatic property of "fresh" whole blood (WB) has been observed in military application and cardiac surgery and is associated with reduced blood loss, transfusion requirements, and donor exposures. The time from donation to transfusion defining "fresh" has not been systematically studied. We undertook an in vitro study of coagulation properties of refrigerated WB stored for 31 days.

Initial validation of a knowledge-based measure of social information processing and anger management.

Over the past fifteen years many schools have utilized aggression prevention programs. Despite these apparent advances, many programs are not examined systematically to determine the areas in which they are most effective. One reason for this is that many programs, especially those in urban under-resourced areas, do not utilize outcome measures that are sensitive to the needs of ethnic minority students.

Osteopoietic engraftment after bone marrow transplantation: effect of inbred strain of mice.

Objective: Transplantable osteoprogenitors, as well as hematopoietic progenitors, reside in bone marrow. We previously reported the first clinical trial of bone marrow transplantation (BMT) for a genetic disorder of bone, osteogenesis imperfecta. Although the patients demonstrated striking clinical benefits after transplantation, measured osteopoietic engraftment was low and did not seem to be durable.

Prospective, interdisciplinary follow-up of children with prenatally diagnosed giant omphalocele: short-term neurodevelopmental outcome.

Purpose: The objective of this study is to determine the short-term neurodevelopmental outcome in infants with giant omphalocele (GO).

Materials And Methods: Between January 2002 and December 2007, 31 consecutive infants with GO received prenatal and postnatal care at our institution. Overall survival was 81% (25/31).

Preschool neurodevelopmental outcome of children following fetal myelomeningocele closure.

Objective: We sought to investigate the preschool neurodevelopmental outcomes of children following fetal myelomeningocele (fMMC) surgery.

Study Design: Prior to the Management of Myelomeningocele Study trial, 54 children underwent fMMC closure at our institution. Thirty (56%) returned at 5 years of age for standardized neurocognitive examination.

Epidemiology of surgical admissions to a children's disability hospital in Nepal.

Background: The goal of this retrospective review was to characterize the spectrum of surgical admissions to a tertiary hospital specializing in musculoskeletal conditions in children and adolescents in Nepal.

Methods: We reviewed hospital records from 1996 to 2006 to categorize all major surgical procedures. Additional information collected included diagnosis, patient age, gender, and home district.

The Impact of Community Violence Exposure on Anxiety in Children of Mothers with Depression.

The dual risk of exposure to community violence and maternal depression has potentially negative effects on children. This study examined whether social skills served as a protective factor for the impact of community violence exposure on anxiety within children of African American mothers with depression. Multiple regressions showed that community violence exposure was associated with less anxious coping but more physical symptoms.

The natural history of patients treated for TWIST1-confirmed Saethre-Chotzen syndrome.

Background: Patients with Saethre-Chotzen syndrome have a heterogeneous phenotype. The purpose of this study was to use the genotypic diagnosis of the authors' series of patients with TWIST1-confirmed Saethre-Chotzen syndrome to describe their natural history and long-term surgical outcomes.

Methods: A retrospective chart review was performed on patients treated at The Children's Hospital of Philadelphia with TWIST1-confirmed Saethre-Chotzen syndrome (n = 22) over 23 years (1985 to 2008).

An initial evaluation of a culturally adapted social problem-solving and relational aggression prevention program for urban African-American relationally aggressive girls.

Recent research demonstrating that relational aggression is associated with peer relationship difficulties, internalizing and externalizing behaviors, social processing deficits, and possibly later mental health disorders among girls has emphasized the need to address the unique expression of aggression among females. Despite these findings, almost all aggression interventions have been directed toward physically aggressive boys. In the current article, the authors describe the acceptability and initial effectiveness of a culturally adapted social problem-solving/social skills intervention for inner-city 3rd- to 5th-grade urban, African American, relationally aggressive girls called the Friend to Friend Program.

Parent-child collaborative decision making for the management of chronic illness: a qualitative analysis.

Parent-child collaborative decision making (CDM) is a potentially important precursor to full decision-making independence and may be particularly significant for the management of childhood chronic illnesses. The primary aim of this qualitative study was to explore the concept of CDM from the perspective of children and parents. Children (ages 8-19 years) with asthma, type 1 diabetes, or cystic fibrosis and parents of children with these illnesses participated in focus groups and individual interviews.

Reversal of hindbrain herniation after maternal-fetal surgery for myelomeningocele subsequently impacts on brain stem function.

The aim of our study was to delineate whether the reversal of hindbrain herniation (HH) following fetal myelomeningocele (fMMC) closure subsequently reduces the incidence and severity of HH-associated brainstem dysfunction (BSD). Prior to the NIH-sponsored Management of Myelomeningocele Study (MOMS) trial, 54 children underwent fMMC closure at our institution. Forty-eight (89%) families participated in a structured survey focusing on HH-associated BSD (e.

Open fetal surgery for life-threatening fetal anomalies.

After more than two decades of experimental and clinical work, fetal surgery is an accepted treatment option for highly selected fetuses with life-threatening anomalies. Fetal lung masses associated with hydrops are usually fatal. These lesions can be resected in utero if they are predominantly solid or multicystic.

Fetal myelomeningocele: natural history, pathophysiology, and in-utero intervention.

Myelomeningocele (MMC) is a common birth defect that is associated with significant lifelong morbidity. Little progress has been made in the postnatal surgical management of the child with spina bifida. Postnatal surgery is aimed at covering the exposed spinal cord, preventing infection, and treating hydrocephalus with a ventricular shunt.

Enteropeptidase, a type II transmembrane serine protease.

Enteropeptidase, a type II transmembrane serine protease, is localized to the brush border of the duodenal and jejunal mucosa. It is synthesized as a zymogen (proenteropeptidase) that requires activation by another protease, either trypsin or possibly duodenase. Active enteropeptidase then converts the pancreatic precursor, trypsinogen, to trypsin by cleavage of the specific trypsinogen activation peptide, Asp-Asp-Asp-Asp-Lys- Ile that is highly conserved in vertebrates.