newer therapeutic modalities in pediatrics

BACKGROUND: Despite advancement in therapies, overall survival rates for relapsed pediatric sarcomas are dismal. Newer therapies are needed to effectively salvage these patients. Oncolytic viruses (such as reovirus) and other genetically altered viruses (such as herpes simplex viruses and adenoviruses) have shown efficacy in a variety of solid tumors including sarcomas. Reolysin is an unmodified oncolytic reovirus that selectively replicates in Ras-activated cancer cells while not causing any significant human illness in its wild form...

Arteriovenous malformation (AVM) is the most common cause of spontaneous intraparenchymal hemorrhage in children, excluding hemorrhages of prematurity and early infancy. Because most children diagnosed with an AVM undergo initial treatment emergently, the natural history of AVMs in the pediatric population is not well understood. Most pediatric AVMs do not come to clinical attention unless they hemorrhage. Therefore, their optimal management remains controversial. Children with intracranial AVMs represent a special challenge in that they harbor unacceptable lifelong risks of hemorrhage and potential neurologic deficits...

Many childhood pancreatic disorders are rare, although they can represent significant and potentially severe disease. The spectrum of disease is very broad, ranging from the complex and bizarre congenital anomalies to the more typical acquired causes (e.g., drug-induced pancreatitis or trauma injury). Genetics appears to play a major role in many childhood pancreas diseases, unlike adults where alcohol is a major factor. Nevertheless, there are similarities, and most of the disorders discussed here can be found in both the pediatric and adult age groups...

PURPOSE OF REVIEW: Myocarditis is an uncommon pediatric illness, and it is frequently missed by medical personnel. It often masquerades as more common pediatric illnesses such as respiratory distress or gastrointestinal disease. Given that myocarditis accounts for 12% of sudden cardiac death among adolescents and young adults, the suspicion of this illness in the differential diagnosis of children presenting with nonspecific symptomatology and disease progression can be lifesaving. RECENT FINDINGS: Historically, the diagnosis of myocarditis required endomyocardial biopsy...

Neuroblastoma, the most common extracranial pediatric solid tumor remains a clinical enigma with outcomes ranging from cure in >90% of patients with locoregional tumors with little to no cytotoxic therapy, to <30% for those >18months of age at diagnosis with metastatic disease despite aggressive multimodality therapy. Age, stage and amplification of the MYCN oncogene are the most validated prognostic markers. Recent research has shed light on the biology of neuroblastoma allowing more accurate stratification of patients which has permitted reducing or withholding cytotoxic therapy without affecting outcome for low-risk patients...

BACKGROUND: Pathologic diagnosis and therapeutic interventions on the small bowel have been difficult and challenging for gastroenterologists. In the last few years, significant advances have been made in this direction. New diagnostic and therapeutic modalities for visualizing the small bowel have been introduced. Furthermore, increased indications for small-bowel imaging and therapeutics have been recognized. However, the currently available methods have limitations, and development of newer, rapid, minimally invasive, safe, and readily available techniques is needed...

The Ewing sarcoma family of tumors (ESFT) is one of the most common groups of malignancies arising in children, adolescents, and young adults up to approximately 25 years of age. It comprises Ewing sarcoma arising from bone and extraosseous Ewing sarcoma arising from soft tissues (which includes peripheral neuroectodermal tumors and Askin tumor arising from the chest wall). Ewing sarcoma is treated successfully in many cases by a combination of chemotherapy, surgery, and radiotherapy. A number of prognostic factors have been identified that can be used to stratify patients according to the risk of relapse, allowing optimization of treatment...

Rhabdomyosarcoma is the most common sarcoma of childhood. Fortunately, the goal of cure is realistic for the majority of patients with localized tumors. However, management of these patients remains challenging. The fact that the tumor arises in a wide variety of primary sites, some of which are associated with specific patterns of local invasion, regional lymph node spread, and therapeutic response, requires physicians to be familiar with site-specific staging and treatment details. In addition, rhabdomyosarcoma requires multimodality therapy that can be associated with significant acute toxicities and long-term effects, particularly when administered to young children...

OBJECTIVE: Atopic dermatitis (AD) is a common, chronic, inflammatory skin disease that can significantly reduce the quality of life of not only patients but also entire families. This review will focus on the currently available non-pharmacologic and pharmacologic treatments for the control and management of AD. RESEARCH DESIGN AND METHODS: A review of English-language articles from January 1953 to May 2006 was performed within the MEDLINE database. Search terms included, but were not limited to, atopic dermatitis, topical corticosteroids, and topical calcineurin inhibitors...

Children with epilepsy, particularly infants, differ from adults not only in the clinical manifestations of their seizures but also in the presence of unique electroencephalographic patterns, etiologies, and response to antiepileptic drugs (AEDs). There is a growing list of newer AEDs and nonpharmacologic therapies available to manage childhood epilepsy. These newer AEDs may not be overall more efficacious than the older drugs, but they do appear to be safer, better tolerated, and to have fewer drug-drug interactions...

Pediatric endoscopy has evolved from a purely observational modality into one with the potential for many therapeutic applications. Common therapeutic uses of endoscopy in children now include treatment of variceal bleeds and foreign body retrieval and newer procedures such as endoluminal gastroplication and endoscopic pyloromyotomy. Continuing research in pediatric endoscopy will allow pediatric gastroenterologists to perfect existing interventional endoscopic techniques and to learn to perform new ones.

PURPOSE OF REVIEW: To summarize and contextualize current concepts in the incidence, diagnosis, management and long-term sequelae of extraesophageal reflux disease in children. RECENT FINDINGS: Extraesophageal reflux disease is a different disease entity from gastroesophageal reflux disease. The two diseases have a common etiology, refluxate causing mucosal damage, but the extent and location of the damage varies considerably depending on the underlying mucosal characteristics...

Strategies for the treatment of childhood cancer have changed considerably during the last 50 years and have led to dramatic improvements in long-term survival. Despite these accomplishments, CNS tumors remain the leading cause of death in pediatric oncology. Astrocytic tumors form the most common histologic group among childhood brain tumors. They are a heterogeneous group that from a practical therapeutic point of view can be subdivided into low-grade astrocytomas (LGA), optic pathway gliomas (OPG), high-grade astrocytomas (HGA), and brainstem gliomas (BSG)...

Central nervous system (CNS) cancers are the second most frequent malignancy (and the most common solid tumor) in childhood. In recent years, significant advances in surgery, radiotherapy, and chemotherapy have improved survival in children with these tumors. However, a significant proportion of patients with CNS tumors suffer progressive disease despite such treatment. Advances in the understanding of the nature of the blood-brain/tumor barrier, chemotherapy resistance, tumor biology, and the role of angiogenesis in tumor progression and metastases have led to the advent of newer therapeutic strategies that circumvent these obstacles or target specific receptors that control signal transduction and/or angiogenesis in tumor cells...

Lumps and bumps of the scalp are a common presenting complaint in children and often pose a diagnostic dilemma. These lesions can be difficult to image, with evaluation confounded by their small size. However, accuracy in diagnosis is critical because the diagnostic and therapeutic implications can vary significantly. The clinical examination can be helpful in developing the differential diagnosis and the imaging strategy. Often, however, a single imaging study is insufficient, and the radiologist finds it necessary to image with more than one modality to correctly diagnose a lesion and provide adequate information for the surgeon...

PURPOSE OF REVIEW: Although heartburn and regurgitation are common manifestations of gastroesophageal reflux disease (GERD), otolaryngologic or respiratory symptoms may be the only indication of GERD. This review focuses on the recent developments in the pathophysiology, diagnosis, and treatment of GERD and their implications in pediatrics. RECENT FINDINGS: Newer diagnostic modalities include intraesophageal impedance and Bravo wireless pH monitoring. Impedance technology measures both acid and non-acid reflux, whereas the Bravo capsule allows prolonged pH monitoring under more physiologic conditions...

Since its inception in 1929, cardiac catheterization has undergone many changes. In the last two decades we have seen an evolution in cardiac catheterization from a diagnostic (anatomic and physiologic) to a therapeutic modality. This article highlights some of the more common and newer interventional procedures now performed.

A significant improvement has been noticed over the last 20 years in children in whom shock syndrome has developed. This has been attained through the application of technological advances in respiratory, cardiovascular, renal, nutritional support and improved antibacterial and antifungal therapy, but mostly through a better understanding of the physiology of shock. Newer concepts of the pathophysiology of sepsis and septic shock are presented, with clinical definitions referring to the pediatric patient. Innovative therapeutic modalities designed to modulate the systemic inflammatory response triggered by bacterial infection are discussed...

As more children are now being cured of cancer, increasing attention needs to be focused on the long-term sequelae of cancer therapy, principally late-appearing second cancers and damage to organ systems. To this end, the authors have established an oncology follow-up clinic specifically designed to examine former pediatric cancer patients who are likely to be cured. Patients receive a physical examination and appropriate organ function studies based upon initial diagnosis, pre-existing conditions, treatment received, and any complications experienced during the course of therapy...

Newer techniques of radiation therapy seek to improve the therapeutic ratio in childhood brain tumors by adding potentially more effective treatment in ways that will limit radiation toxicities. In tumors marked primarily by local disease progression, the use of interstitial brain implantation "up front" or radiosurgery deserves systematic exploration. Whether local "boost therapy" with either of the latter techniques will add substantially to disease control and preserve neurologic function remains to be established both in malignant CNS tumors and in low grade gliomas...