Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes.

Sporadic inclusion body myositis, a variant of inflammatory myopathy, has features distinct from polymyositis/dermatomyositis. The disease affects men more than women, most commonly after age 50. Clinical features include weakness of the quadriceps, finger flexors, ankle dorsiflexors, and dysphagia.

Modeling functional decline over time in sporadic inclusion body myositis.

Introduction: The ability to individualize recommendations or expectations of disease progression based on a patient's unique characteristics has merit for use in sporadic inclusion body myositis (sIBM).

Methods: Fifty-five subjects with sIBM completed a battery of strength and functional outcomes at 2 study visits. These were used to develop mathematical models of disease progression in patients with sIBM for use in clinical and research settings.

Evaluation of Alternative In Vivo Drug Screening Methodology: A Single Mouse Analysis.

Traditional approaches to evaluating antitumor agents using human tumor xenograft models have generally used cohorts of 8 to 10 mice against a limited panel of tumor models. An alternative approach is to use fewer animals per tumor line, allowing a greater number of models that capture greater molecular/genetic heterogeneity of the cancer type. We retrospectively analyzed 67 agents evaluated by the Pediatric Preclinical Testing Program to determine whether a single mouse, chosen randomly from each group of a study, predicted the median response for groups of mice using 83 xenograft models.

Early Conventional MRI for Prediction of Neurodevelopmental Impairment in Extremely-Low-Birth-Weight Infants.

Background: Extremely-low-birth-weight (ELBW; ≤1,000 g) infants are at high risk for neurodevelopmental impairments. Conventional brain MRI at term-equivalent age is increasingly used for prediction of outcomes. However, optimal prediction models remain to be determined, especially for cognitive outcomes.

Patterns of Fever in Children After Primary Treatment for Kawasaki Disease.

Background: We sought to determine if fever in the early postintravenous immunoglobulin (IVIG) time period (first 36 hours after IVIG completion) for Kawasaki disease, with or without additional infliximab, can predict IVIG resistance and coronary artery abnormalities (CAA).

Methods: Acute Kawasaki disease subjects enrolled in a clinical trial of infliximab plus IVIG (n = 96) versus placebo/IVIG (n = 94) had temperatures recorded every 6 hours after completion of IVIG infusion. Fever was defined as temperature >38.

Reliability and validity of active-seated: An outcome in dystrophinopathy.

Introduction: Traditional upper extremity measures typically focus on distal abilities and do not quantify the unique progression of decline in dystrophinopathy. We designed ACTIVE-seated to meet this need. Our objective was to establish the tool's validity and reliability.

Impact of preoperative location on outcomes in congenital heart surgery.

Background: Little is known about the impact of preoperative location on outcomes in infants undergoing cardiac surgery for congenital heart disease. This study was designed to evaluate the morbidity and mortality among infants who were cared for in a neonatal ICU (NICU) versus dedicated cardiovascular intensive care unit (CVICU) prior to cardiac surgery in a multi-institutional population.

Methods: Data were obtained from a multicenter, administrative, national dataset, Pediatric Health Information System (PHIS).

A randomized, double-blind trial of lisinopril and losartan for the treatment of cardiomyopathy in duchenne muscular dystrophy.

Objectives: This study sought to compare the effectiveness and safety of an angiotensin converting enzyme inhibitor (ACE-I) (lisinopril) vs. an angiotensin receptor blocker (ARB) (losartan) for the treatment of cardiomyopathy (CM) in boys with Duchenne muscular dystrophy (DMD).

Background: Development of CM is universal in boys with DMD.

A validated model for predicting outcome after liver transplantation: implications on transplanting the extremely sick.

Given the organ shortage, there is a need to optimize outcome after liver transplantation (LT). We defined posttransplant hospital length of stay > 60 days (LOS > 60) as a surrogate of suboptimal outcome. In the first phase of the study, a 'Study cohort' (SC) of 643 patients was used to identify risk factors and construct a mathematical model to identify recipients with anticipated inferior results.

Resection of isolated local and metastatic recurrence in periampullary adenocarcinoma.

Background: The majority of patients with periampullary cancer develop local or metastatic recurrence despite successful negative margin resection. Unfortunately, there are no established therapeutic strategies for managing these patients. The literature on the surgical resection of recurrent disease is limited.

Parental ranking of terms describing nonepileptic events.

Paroxysmal nonepileptic events of psychogenic etiology in children and adolescents are common. Patients and their parents are often confused by the terminology used to describe these events. This can lead to frustration and may result in the failure to obtain the necessary nonpharmacologic treatment.

Is juvenile dermatomyositis a different disease in children up to three years of age at onset than in children above three years at onset? A retrospective review of 23 years of a single center's experience.

Background: We tested the hypothesis that the course and outcome of juvenile dermatomyositis (JDM) in children seen at one center with the JDM disease onset at or below three years of age is different from that in the children with disease onset at greater than three years of age.

Methods: Institutional Review Board approval was obtained to retrospectively review the charts of 78 patients from age 0-18 years with JDM seen in the pediatric rheumatology clinic at Nationwide Children's Hospital in Columbus, Ohio over the past 23 years from January 1988. The diagnosis was made by the treating pediatric rheumatologist.

Posttraumatic seizures in children with severe traumatic brain injury.

Purpose: Traumatic brain injury (TBI) remains a leading cause of childhood death and disability worldwide. Seizures are a common complication of TBI and they are particularly common in pediatric populations. The proper management of children sustaining severe TBI is still controversial.

Outcomes of renal transplantation in recipients with peak panel reactive antibody >30% under tacrolimus-based immunosuppression.

Background: Renal transplant recipients with high panel reactive antibodies (PRA) have worse outcomes than those with lower PRA. High PRA re-transplant recipients are thought to have worse outcomes than high PRA first transplant recipients. In this study, we examined outcomes of renal transplantation recipients with a peak PRA >30% and compared the outcomes of first and re-transplanted recipients.

Use of positron emission tomography in the evaluation of diffuse intrinsic brainstem gliomas in children.

Background: Diffuse intrinsic brainstem gliomas (DIBSGs) in children remain difficult tumors to treat and have a very poor prognosis. Intensifying both chemotherapy and radiation programs have been attempted without success. Positron emission tomography (PET) has been used to differentiate benign from malignant tumors and may predict outcome.

Lymphoproliferative disorders and de novo malignancies in intestinal and multivisceral recipients: improved outcomes with new outlooks.

Background: Early experience with intestinal and multivisceral transplantation was plagued with high risk of rejection and posttransplant lymphoproliferative disorders (PTLD). To improve outcome, innovative management and immunosuppressant strategies were sequentially evolved.

Methods: With initiation of the program in 1990, serial monitoring of Epstein-Barr-Viral load was introduced in 1994 with adoption of preemptive antiviral therapy.

Kidney after nonrenal transplantation-the impact of alemtuzumab induction.

Background: Calcineurin inhibitor nephrotoxicity in nonrenal allograft recipients can lead to end-stage renal disease and the need for kidney transplantation. We sought to evaluate the role of alemtuzumab induction in this population.

Patients And Methods: We evaluated 144 patients undergoing kidney transplantation after nonrenal transplantation between May 18, 1998, and October 8, 2007.

Pyridoxal-5'-phosphate deficiency after intestinal and multivisceral transplantation.

Background: Successful intestinal transplantation is measured by the achievement of clinical nutritional autonomy (CNA). However, the ability of the graft to maintain normal micronutrient levels including vitamins has yet to be thoroughly evaluated.

Objective: After an initial clinical observation of isolated cases of pyridoxal-5'-phosphate (PLP) deficiency, this prospective study was designed to address the incidence of, risk factors for, and management of PLP deficiency in adult intestinal transplant recipients.

Liver transplantation for hepatocellular carcinoma: extension of indications based on molecular markers.

Background/aims: Liver transplantation usually cures hepatocellular carcinoma when the Milan selection criteria are applied, whereas there is substantial risk of posttransplant recurrence with tumors beyond these criteria. This study uses molecular data to identify a subgroup of patients who, despite having hepatocellular carcinoma beyond Milan criteria, have favorable outcomes.

Methods: Allelic imbalance of 18 microsatellites was analyzed in 70 consecutive patients (35 within Milan, 35 beyond Milan criteria) transplanted for hepatocellular carcinoma of whom 24 had recurrence and 46 survived at least 5 years recurrence-free.

Fractional allelic imbalance could allow for the development of an equitable transplant selection policy for patients with hepatocellular carcinoma.

Liver transplantation (LT) in the presence of hepatocellular carcinoma (HCC) remains a controversial issue because the current staging systems are not sufficiently predictive of outcomes. Paraffin blocks from 183 patients that underwent LT in the presence of HCC were collected. Molecular analysis was carried out blindly on the native liver specimens in all cases with respect to recurrence outcomes.

A simple prognostic scoring system for patients with unresectable hepatocellular carcinoma treated by chemo-embolization.

Background: Hepatocellular carcinoma (HCC) comprises heterogeneous groups of patients with differing outcomes.

Methods: In order to attempt to identify patient sub-sets, we retrospectively examined the records of 750 patients with biopsy-proven unresectable HCC, who were treated with hepatic artery chemo-embolization and were followed till death. We used the Cox proportional hazard model, as it was neutral with respect to prejudged cut-off between short and long survival.

MELD and prediction of post-liver transplantation survival.

The model for end-stage liver disease (MELD) was developed to predict short-term mortality in patients with cirrhosis. It has since become the standard tool to prioritize patients for liver transplantation. We assessed the value of pretransplant MELD in the prediction of posttransplant survival.

Outcome of 1000 liver cancer patients evaluated at the UPMC Liver Cancer Center.

We evaluated 1000 consecutive patients with liver tumors at the University of Pittsburgh Medical Center (UPMC) Liver Cancer Center over the 4-year period from August 2000 to August 2004. Of the 1000 patients seen, 573 had primary liver cancer and 427 had metastatic cancer to the liver. The mean age of the patients evaluated was 62.

Pretransplant survival is shorter in HIV-positive than HIV-negative subjects with end-stage liver disease.

Despite improved survival after liver transplantation (OLTX) in HIV-positive individuals treated with highly active antiretroviral therapy (HAART), some transplant candidates do not survive to OLTX. To determine if pretransplant outcome is related to severity of liver disease and/or HIV infection, we prospectively evaluated 58 consecutive HIV-positive candidates seen at a single center from 1997-2002. Of the 58, 15 (25.