The congenital diaphragmatic hernia composite prognostic index correlates with survival in left-sided congenital diaphragmatic hernia.

Purpose: We developed the congenital diaphragmatic hernia congenital prognostic index (CDH-CPI) to incorporate all known prognostic variables into a single composite index to improve prognostic accuracy. The purpose of this study is to examine the ability of the CDH-CPI to predict survival in patients with left-sided congenital diaphragmatic hernia and to determine if the index has a stronger correlation with survival than each of the individual components.

Methods: A retrospective review of patients with left-sided congenital diaphragmatic hernia between 2004 and 2010 was conducted.

Patient-derived olfactory mucosa cells but not lung or skin fibroblasts mediate axonal regeneration of retinal ganglion neurons.

Although human olfactory mucosa derived cells (OMC) have been used in animal models and clinical trials with CNS repair purposes, the exact identity of these cells in culture with respect to their tissue of origin is not fully understood and their neuroregenerative capacity in vitro has not yet been demonstrated. In this study we have compared human OMC with human ensheathing glia from olfactory bulb (OB) and human fibroblasts from skin and lung. Our results indicate that these different cultured cell types exhibit considerable overlap of antigenic markers such that it is presently not possible to distinguish them immunocytochemically.

Tissue-engineered provisional matrix as a novel approach to enhance diabetic wound healing.

Inherent pathologies associated with diabetic wound microenvironment including increased proteolysis, inflammatory dysregulation, and impaired neovascularization prevent timely resolution of chronic diabetic ulcers. It is hypothesized that augmentation of local wound microenvironment with a stable provisional matrix formed by proteolysis-resistant angiogenic peptide nanofibers (NFs) will create permissive environment for attenuated inflammation, enhanced neovascularization, and improved diabetic wound healing. Using murine excisional wound healing models, full-thickness dorsal skin wounds were treated with either NFs or control solutions (phosphate buffered saline; hyaluronic acid) and analyzed for morphology, inflammatory response, neovascularization, and biomechanical properties.

Prenatal imaging of amniotic band sequence: utility and role of fetal MRI as an adjunct to prenatal US.

Background: Amniotic band sequence and its US manifestations have been well-described. There is little information, however, regarding the accuracy and utility of fetal MRI.

Objective: To describe the MRI findings in amniotic band sequence and to compare the diagnostic accuracy of MRI and US.

Barriers to diagnosis of a rare neurological disorder in China--lived experiences of Rett syndrome families.

Rett syndrome is a rare neurological disorder affecting girls and usually caused by a mutation on the MECP2 gene. It is estimated that approximately 1,000 girls are born every year in China with Rett syndrome but far fewer have received a diagnosis. Fourteen of 74 Chinese families known to the International Rett Syndrome Phenotype Database participated in this qualitative study.

Stimulation of inflammatory gene expression in human preadipocytes by macrophage-conditioned medium: upregulation of IL-6 production by macrophage-derived IL-1β.

The aim of this study was to examine the effects of macrophage secretions on global gene expression in human preadipocytes using microarrays. Preadipocytes were cultured with unconditioned or conditioned medium from U937 macrophages, and gene expression examined with Agilent arrays (43,000 probes). 472 transcripts were differentially regulated (>2-fold difference; P<0.

Prevalence and progression of recipient-twin cardiomyopathy in early-stage twin-twin transfusion syndrome.

Objective: The management of twin-twin transfusion syndrome (TTTS) in its early stages (Quintero Stages I and II) is controversial. We describe the prevalence, severity, incidence and rate of progression of recipient-twin cardiomyopathy in Stages I and II TTTS.

Methods: Among 451 cases of TTTS evaluated between 2004 and 2009, 123 (27.

The anatomy of failures following the ligation of intersphincteric tract technique for anal fistula: a review of 93 patients over 4 years.

Background: Although the ligation of intersphincteric tract technique is a promising sphincter-preserving option in managing anal fistulas, failures are still seen.

Objective: This study aimed to illustrate the patterns of failures and recurrences following the ligation of intersphincteric tract procedure for anal fistulas.

Design: This study is a retrospective review.

A culture model for neurite regeneration of human spinal cord neurons.

Effective therapeutic interventions for injuries of the central nervous system such as spinal cord injury are still unavailable, having a great impact on the quality of life of victims and their families, as well as high costs in medical care. Animal models of spinal cord injury are costly, time-consuming and labor-intensive, making them unsuitable for screening large numbers of experimental conditions. Thus, culture models that recapitulate key aspects of neuronal changes in central nervous system injuries are needed to gain further understanding of the pathological and regenerative mechanisms involved, as well as to accelerate the screening of potential therapeutic agents.

Multicystic dysplastic kidney disease presenting with a single large cyst in a fetus-anatomical basis and radiological aspects.

Multicystic dysplastic kidney (MCDK) is a congenital maldevelopment in which the renal cortex is characteristically replaced by numerous cysts of multiple sizes. MCDK presenting as a single predominant large cyst in morphology is less common. We report on the prenatal imaging findings and perinatal management of a fetus with MCDK unusually presenting as a single predominant large cyst, erroneously interpreted as a severe fetal hydronephrosis.

Pneumococcal pneumonia complicating purulent pericarditis in a previously healthy girl: a rare yet possible fatal complication in the antibiotic era.

Purulent pericarditis is an extremely rare complication of invasive Streptococcus pneumoniae infection among children in the antibiotic era, and its mortality remains high if left untreated. This report involves a 4½-year-old girl who presented to our emergency department with productive cough, shortness of breath, and left-sided chest pain with a diagnosis of pneumococcal pneumonia. She subsequently developed life-threatening conditions including bilateral empyema with respiratory failure, purulent pericarditis, and multiple organ failure leading to death.

Prenatal and postnatal urologic complications of sacrococcygeal teratomas.

Purpose: Urologic complications are an underappreciated sequelae of the mass effect of sacrococcygeal teratoma (SCT) and its resection. The goal of this study was to evaluate the incidence and severity of urologic complications in patients with SCT.

Methods: A retrospective review of patients with a prenatal diagnosis of SCT and postnatal referral for SCT at a single institution during a 5-year period (2004-2009) was performed.

Late gestation fetal magnetic resonance imaging-derived total lung volume predicts postnatal survival and need for extracorporeal membrane oxygenation support in isolated congenital diaphragmatic hernia.

Purpose: Magnetic resonance imaging (MRI) has been used as an imaging modality to assess pulmonary hypoplasia in congenital diaphragmatic hernias (CDHs). The objective of this study was to determine if there is a correlation between late gestational fetal MRI-derived total lung volumes (TLVs) and CDH outcomes.

Methods: From 2006 to 2009, 44 patients met criteria of an isolated CDH with a late gestational MRI evaluation.

Expression of plasminogen activator inhibitor-1 by olfactory ensheathing glia promotes axonal regeneration.

Olfactory ensheathing glia (OEG) cells are known to facilitate repair following axotomy of adult neurons, although the molecular mechanisms involved are not fully understood. We previously identified plasminogen activator inhibitor-1 (PAI-1), proteinase-activated receptor-1 (PAR-1), and thrombomodulin (TM) as candidates to regulate rat OEG-dependent axonal regeneration. In this study, we have validated the involvement of these proteins in promoting axonal regeneration by immortalized human OEGs.

Infectious delivery and long-term persistence of transgene expression in the brain by a 135-kb iBAC-FXN genomic DNA expression vector.

Novel gene-based therapies for disease will depend in many cases on long-term persistent transgene expression. To develop gene therapy strategies for Friedreich's ataxia (FRDA), we have examined the persistence of transgene expression in the brain in vivo provided by the entire 135 kb FXN genomic DNA locus delivered as an infectious bacterial artificial chromosome (iBAC) herpes simplex virus type 1 (HSV-1)-based vector injected in the adult mouse cerebellum. We constructed genomic DNA-reporter fusion vectors carrying a complete 135 kb FXN genomic locus with an insertion of the Escherichia coli lacZ gene at the ATG start codon (iBAC-FXN-lacZ).

Chemotherapy/chemoradiation in anal cancer: a systematic review.

Introduction: Results from recent phase III trials in anal cancer failed to show any benefit for neoadjuvant chemotherapy (NACT) with cisplatin, or cisplatin-based consolidation chemotherapy compared to chemoradiation alone for loco-regional control, disease-free survival (DFS) and overall survival (OS).

Aims: This systematic review examines evidence for efficacy and toxicity of chemotherapy and chemoradiotherapy in anal cancer.

Results: In total, for chemoradiation, 103 retrospective/observational studies, four phase I/II studies, 16 phase II prospective studies, two randomised phase II studies, and six phase III trials of chemoradiation in anal cancer were identified.

A new roadmap for biopharmaceutical drug product development: Integrating development, validation, and quality by design.

Quality by design (QbD) is a science- and risk-based approach to drug product development. Although pharmaceutical companies have historically used many of the same principles during development, this knowledge was not always formally captured or proactively submitted to regulators. In recent years, the US Food and Drug Administration has also recognized the need for more controls in the drug manufacturing processes, especially for biological therapeutics, and it has recently launched an initiative for Pharmaceutical Quality for the 21st Century to modernize pharmaceutical manufacturing and improve product quality.

Anal cancer: an examination of radiotherapy strategies.

The Radiation Therapy Oncology Group 9811, ACCORD-03, and ACT II Phase III trials in anal cancer showed no benefit for cisplatin-based induction and maintenance chemotherapy, or radiation dose-escalation >59 Gy. This review examines the efficacy and toxicity of chemoradiation (CRT) in anal cancer, and discusses potential alternative radiotherapy strategies. The evidence for the review was compiled from randomized and nonrandomized trials of radiation therapy and CRT.

Fetal MRI clues to diagnose cloacal malformations.

Background: Prenatal US detection of cloacal malformations is challenging and rarely confirms this diagnosis.

Objective: To define the prenatal MRI findings in cloacal malformations.

Materials And Methods: We performed a retrospective study of patients with cloacal malformations who had pre- and post-natal assessment at our institution.

Safety and reactogenicity of DTPa-HBV-IPV/Hib and DTPa-IPV/I-Hib vaccines in a post-marketing surveillance setting.

Combination vaccines have been shown to improve the timeliness of vaccination and vaccine coverage. Safety and reactogenicity of combined diphtheria-tetanus-acellular pertussis-inactivated poliovirus and Haemophilus influenzae type b vaccine (DTPa-IPV/Hib, Infanrix IPV+Hib, GlaxoSmithKline Biologicals) was assessed in two clinical studies. In Study A, 2,590 subjects received DTPa-IPV/Hib at 3, 4 and 5 months of age with a booster at 18 months.

Submucosal gland development in the human fetal trachea xenograft model: implications for fetal gene therapy.

Background/purpose: Our previous work in a human-fetal trachea xenograft model suggests potential benefits of treating cystic fibrosis in utero. The target for postnatal gene therapy in cystic fibrosis is tracheal submucosal glands (SMGs). The aim of this study was to determine if SMG development in our model recapitulates normal trachea development and its validity for studying fetal gene transfer.

MRI findings in multifetal pregnancies complicated by twin reversed arterial perfusion sequence (TRAP).

Background: Twin reversed arterial perfusion sequence (TRAP) is a rare complication in multifetal monochorionic pregnancies in which a normal "pump" twin provides circulation to an abnormal acardiac co-twin, resulting in high-output cardiac dysfunction in the pump twin.

Objective: To define fetal MRI findings of TRAP sequence.

Materials And Methods: Fetal MR images were retrospectively reviewed in 35 pregnancies complicated by TRAP sequence.