Assa A.,Schneider Childrens Hospital |
Assa A.,Ludwig Maximilians University of Munich
Journal of Pediatric Gastroenterology and Nutrition | Year: 2017
BACKGROUND:: Data on the outcomes of children with perianal Crohnʼs disease (pCD) are limited, although its presence is often used for justifying early use of biologics. We aimed to assess whether pCD in children is associated with more severe outcomes as found in adults. METHODS:: Data were extracted from the ImageKids database, a prospective, multicenter, longitudinal cohort study. The study enrolled 246 children at disease onset or thereafter. All patients underwent comprehensive clinical, endoscopic, and radiologic evaluation at enrollment; 98 children had repeat evaluation at 18 months. RESULTS:: Of the 234 included patients [mean age 14.2?±?2.4 years; 131 (56%) males], 57 (24%) had perianal findings while only 21 (9%) had fistulizing perianal disease. Children with pCD had reduced weight and height z-scores compared with non-pCD patients (-0.9 vs. -0.35, p?=?0.03 and -0.68 vs. -0.23, respectively; p?=?0.04), higher weighted pediatric Crohnʼs disease activity index [32 (IQR 16–50) vs. 20 (8–37); p?=?0.004], lower serum albumin (3.6?±?0.7 vs. 4.5?±?0.8, P?=?0.016) and higher magnetic resonance enterography (MRE) global inflammatory score (P?=?0.04). Children with pCD had more rectal (57% vs. 38%, p?=?0.04), and jejunal involvement (31% vs. 11% p?=?0.003) and a higher prevalence of granulomas (64% vs. 23%, p?=?0.0001). MRE-based damage scores did not differ between groups. Patients with skin tags/fissures only, had similar clinical, endoscopic and radiologic characteristics as patients with no perianal findings. CONCLUSION:: Pediatric pCD patients with fistulizing disease have distinct phenotypic features and a predisposition to a greater inflammatory burden. © 2017 by European Society for Pediatric Gastroenterology, Hepatology, and Nutrition and North American Society for Pediatric Gastroenterology,
Barrett D.,Children's Hospital of Philadelphia |
Fish J.D.,Schneider Childrens Hospital |
Grupp S.A.,Children's Hospital of Philadelphia |
Grupp S.A.,University of Pennsylvania
Pediatric Clinics of North America | Year: 2010
Since the 1950s, the overall survival of children with cancer has gone from almost zero to approaching 80%. Although there have been notable successes in treating solid tumors such as Wilms tumor, some childhood solid tumors have continued to elude effective therapy. With the use of megatherapy techniques such as tandem transplantation, dose escalation has been pushed to the edge of dose-limiting toxicities, and any further improvements in event-free survival will have to be achieved through novel therapeutic approaches. This article reviews the status of autologous and allogeneic hematopoietic stem cell transplantation (HSCT) for many pediatric solid tumor types. Most of the clinical experience in transplant for pediatric solid tumors is in the autologous setting, so some general principles of autologous HSCT are reviewed. The article then examines HSCT for diseases such as Hodgkin disease, Ewing sarcoma, and neuroblastoma, and the future of cell-based therapies by considering some experimental approaches to cell therapies. © 2010 Elsevier Inc.
Schneider J.,Childrens Hospital Los Angeles |
Schneider J.,Schneider Childrens Hospital |
Khemani R.,Childrens Hospital Los Angeles |
Khemani R.,University of Southern California |
And 4 more authors.
Critical Care Medicine | Year: 2010
Objective: To evaluate the ability of the RIFLE criteria to characterize acute kidney injury in critically ill children. Design: Retrospective analysis of prospectively collected clinical data. Setting: Multidisciplinary, tertiary care, 20-bed pediatric intensive care unit. Patients: All 3396 admissions between July 2003 and March 2007. Interventions: None. Measurements and Main Results: A RIFLE score was calculated for each patient based on percent change of serum creatinine from baseline (risk = serum creatinine ×1.5; injury = serum creatinine ×2; failure = serum creatinine ×3). Primary outcome measures were mortality and intensive care unit length of stay. Logistic and linear regressions were performed to control for potential confounders and determine the association between RIFLE score and mortality and length of stay, respectively.One hundred ninety-four (5.7%) patients had some degree of acute kidney injury at the time of admission, and 339 (10%) patients had acute kidney injury develop during the pediatric intensive care unit course. Almost half of all patients with acute kidney injury had their maximum RIFLE score within 24 hrs of intensive care unit admission, and approximately 75% achieved their maximum RIFLE score by the seventh intensive care unit day. After regression analysis, any acute kidney injury on admission and any development of or worsening of acute kidney injury during the pediatric intensive care unit stay were independently associated with increased mortality, with the odds of mortality increasing with each grade increase in RIFLE score (p <.01). Patients with acute kidney injury at the time of admission had a length of stay twice that of those with normal renal function, and those who had any acute kidney injury develop during the pediatric intensive care unit course had a four-fold increase in pediatric intensive care unit length of stay. Also, other than being admitted with RIFLE risk score, an independent relationship between any acute kidney injury at the time of pediatric intensive care unit admission, any acute kidney injury present during the pediatric intensive care unit course, or any worsening RIFLE scores during the pediatric intensive care unit course and increased pediatric intensive care unit length of stay were identified after controlling for the same high-risk covariates (p <.01). Conclusions: RIFLE criteria serves well to describe acute kidney injury in critically ill pediatric patients. Copyright © 2010 by the Society of Critical Care Medicine and Lippincott Williams & Wilkins.
Smith L.P.,Schneider Childrens Hospital |
Roy S.,University of Houston
American Journal of Otolaryngology - Head and Neck Medicine and Surgery | Year: 2011
Purpose: The aim of the study was to characterize the causes of operating room (OR) fires in otolaryngology. Materials and methods: A questionnaire was designed to elicit the characteristics of OR fires experienced by otolaryngologists. The survey was advertised to 8523 members of the American Academy of Otolaryngology-Head and Neck Surgery. Results: Three hundred forty-nine questionnaires were completed. Eighty-eight surgeons (25.2%) witnessed at least one OR fire in their career, 10 experienced 2 fires each, and 2 reported 5 fires each. Of 106 reported fires, details were available for 100. The most common ignition sources were an electrosurgical unit (59%), a laser (32%), and a light cord (7%). Twenty-seven percent of fires occurred during endoscopic airway surgery, 24% during oropharyngeal surgery, 23% during cutaneous or transcutaneous surgery of the head and neck, and 18% during tracheostomy; 7% were related to a light cord, and 1% was related to an anesthesia machine. Eighty-one percent of fires occurred while supplemental oxygen was in use. Common fuels included an endotracheal tube (31%), OR drapes/towels (18%), and flash fire (where no substrate burned) (11%). Less common fuels included alcohol-based preparation solution, gauze sponges, patient's hair or skin, electrosurgical unit with retrofitted insulation over the tip, tracheostomy tube, tonsil sponge, suction tubing, a cottonoid pledget, and a red rubber catheter. Conclusions: OR fire may occur in a wide variety of clinical settings; endoscopic airway surgery, oropharyngeal surgery, cutaneous surgery, and tracheostomy present the highest risk for otolaryngologists. Electrosurgical devices and lasers are the most likely to produce ignition. © 2011 Elsevier Inc. All rights reserved.
Wiygul J.B.,Schneider Childrens Hospital |
Palmer L.,Schneider Childrens Hospital
Urology | Year: 2010
Hematuria in children is usually a benign entity, with hypercalciuria being the usual etiology; however, rarer causes also exist, including genitourinary hemangioma. Hemangioma is a benign tumor, which has a tendency to regress spontaneously by the second year of life. Involvement of the urinary tract is rare, and in patients with bladder involvement, an accompanying syndrome such as Sturge-Weber is usually found. However, there are reports of isolated bladder hemangioma in the pediatric population causing isolated gross hematuria. We report a case of bladder hemangioma causing gross painless hematuria in an otherwise healthy 14-year-old boy. © 2010 Elsevier Inc.
Laron Z.,Schneider Childrens Hospital |
Iluz M.,Schneider Childrens Hospital |
Kauli R.,Schneider Childrens Hospital
Growth Hormone and IGF Research | Year: 2012
Background: Head circumference (HC) is a simple and practical measure of brain size, development and longitudinal measurements of the HC in childhood are an index of brain growth. Objective: To determine the effects of long IGF-I deficiency and treatment on HC in patients with Laron syndrome (LS). Patients: 20 untreated adult LS patients, aged 48.4 ± 11.2. years and 13 LS patients treated between ages of 5.6 ± 4 to 11.3 ± 3. years were studied. 15 patients with congenital IGHD treated between age 6.1 ± 4 and 13 ± 4 by hGH served as controls. Methods: HC was expressed as standard deviation (SD) and Ht as SDS. HC was measured and plotted on Nellhaus charts. Linear height (Ht) was measured by a Harpenden Stadiometer. Results: The mean HC deficit of the adult untreated LS males was - 2.9 ± 0.6 SD compared to a Ht deficit of - 7.0 ± 1.7 SDS. The HC of the LS adult females was - 3.6 ± 1 SD compared to a Ht SDS of - 6.9 ± 1.5 (p < 0.001). IGF-I treatment (150-200 μg/kg once daily) increased the HC from - 3.3 ± 0.9 (m ± SD) to normal values (0.87 ± 1.8 SD) (p < 0.001) in 11/13 children. The Ht SDS deficit decreased only by 1.5 SDS. hGH treatment of cIGHD children increased the HC from - 2.0 ± 1.8 to 0.3 ± 1.2 SD and the Ht SDS from - 4.8 ± 1.6 to 1.6 ± 1.0. Conclusions: a)Untreated children and adults with LS and cIGHD have a reduced HC (i.e. brain size). IGF-I treatment of LS children and hGH treatment of IGHD children induced a fast catch-up growth denoting the role of IGF-I on brain growth.b)Comparison between IGF-I and hGH on linear growth stimulation revealed a greater potency of hGH. © 2012 Elsevier Ltd.
Lipton J.M.,Feinstein Institute for Medical Research |
Lipton J.M.,Yeshiva University |
Lipton J.M.,Schneider Childrens Hospital |
Ellis S.R.,University of Louisville
Current Opinion in Pediatrics | Year: 2010
Purpose of Review: Diamond Blackfan anemia (DBA) is an inherited bone marrow failure syndrome characterized by erythroid failure, congenital anomalies and predisposition to cancer. Recently, the notion of DBA as a disorder of ribosome biogenesis has been clarified. Correlations between molecular underpinnings and disease pathophysiology, while elusive, are beginning to emerge. Advances in these areas will be explored in this review. Recent Findings: All known genes mutated in DBA encode ribosomal proteins associated with either the small (RPS) or large (RPL) subunit and in these cases ribosomal protein haploinsufficiency gives rise to the disease. The number of genes affected, their potential interactions with the environment and modifier genes, and the myriad of potential signaling pathways linking abortive ribosome synthesis to cell-cycle regulators may all contribute to disease heterogeneity. Genotype/phenotype relationships emerging over the past year promise to shed light on these complex interrelationships and their role in DBA pathophysiology. Summary: The nosology of DBA has recently expanded to include two distinct disease categories: a classical inherited bone marrow failure syndrome and a 'ribosomopathy'. The description of DBA as a ribosomopathy has provided a context for scientific inquiry analogous to the description of Fanconi anemia as a disorder of DNA repair. © 2010 Wolters Kluwer Health | Lippincott Williams &Wilkins.
Kaye J.D.,Schneider Childrens Hospital |
Palmer L.S.,Schneider Childrens Hospital
Urology | Year: 2010
OBJECTIVES To investigate whether voiding dysfunction (VD) in children with attention deficit hyperactivity disorder (ADHD) could be treated successfully using individualized therapy. We also sought to describe the spectrum of voiding symptoms experienced by children with ADHD. ADHD is diagnosed in 3-5% of children. These children have a greater incidence of VD than non-ADHD controls, and it is less amenable to treatment. METHODS A comprehensive history assessed the nature of the voiding disorder. A physical examination, screening urologic ultrasonography, and urinalysis were routinely performed, with electromyography combined with uroflowmetry performed for certain cases. Treatment was individualized to include behavioral modification, bowel and diet management, biofeedback, pharmacotherapy, and close follow-up. RESULTS A total of 75 children with ADHD were referred because of VD. The 75 children (39 boys and 36 girls) were 5-16 years old. Of the 75 children, 60 were taking medication for ADHD. All the children had daytime wetting (>1/d, 5-7 d/wk) and urgency. In addition, 88% had frequency and 87% had sleep enuresis. Of the 75 families, 56 proceeded with our prescribed program. Of the 56 children, 47 (83.9%) had complete resolution or improvement of the daytime symptoms. Of the 17 patients (30.4%) with complete resolution, 9 responded to behavior modification and anticholinergics and 8 needed biofeedback. Of the 30 patients with a partial response (53.6%), 9 responded to behavior modification alone, 15 to behavior modification and anticholinergics, and 6 required all 3 modalities. Of the 56 children, 9 failed to respond to any of the 3 modalities. CONCLUSIONS Diurnal symptoms constituted the most common referred complaint in children with ADHD. VD can be successfully treated if the treatment is individualized. Multimodal treatment, including behavior modification combined with anticholinergic agents and/or biofeedback, appears to be effective in managing VD in most of these challenging cases. UROLOGY 76: 220-224, 2010. © 2010 Elsevier Inc.
Continuous renal replacement therapy results in respiratory and hemodynamic beneficial effects in pediatric patients with severe systemic inflammatory response syndrome and multiorgan system dysfunction
Naran N.,Schneider Childrens Hospital |
Sagy M.,Schneider Childrens Hospital |
Bock K.R.,Schneider Childrens Hospital
Pediatric Critical Care Medicine | Year: 2010
Background: Proinflammatory mediators have been implicated in the pathogenesis of systemic inflammatory response syndrome and multiorgan system dysfunction. These mediators are of molecular weights that render them amenable to clearance by the hemodiafiltration mode of continuous renal replacement therapy. Objective: To determine whether a period of 48 hrs of continuous renal replacement therapy in patients with multiorgan system dysfunction secondary to systemic inflammatory response syndrome improves their degree of anasarca as well as their cardiovascular and respiratory systems performances. Data Source: Retrospective chart review. Study Design: Charts of patients diagnosed with systemic inflammatory response syndrome, who were mechanically ventilated in the pediatric intensive care unit and at the same time were receiving continuous renal replacement therapy, from 2004 to 2008, were reviewed. Patients with preexisting renal failure and/or received extracorporeal membrane oxygenation were excluded. Changes in the patients' body weights, oxygenation indices, and vasopressor scores were used as markers for responsiveness to continuous renal replacement therapy. Data Analysis And Main Results: Data from twenty-two patients with systemic inflammatory response syndrome and with three to five concomitantly diagnosed organ system dysfunctions, at the time continuous renal replacement therapy was initiated, were analyzed. None of the six patients who had five organ system dysfunctions survived to be discharged from the pediatric intensive care unit. Of the remaining 16 patients with three or four organ system dysfunctions, eight (50%) survived and eight (50%) died. The patients' weight, oxygenation indices, and vasopressor scores did not significantly change with 48 hrs of continuous renal replacement therapy. Conclusions: Mechanically ventilated patients with systemic inflammatory response syndrome and multiorgan system dysfunction demonstrated a precarious and insignificant response to 48 hrs of continuous renal replacement therapy in a hemodiafiltration mode. However, the patients' overall clinical status did not deteriorate during this therapy. More prospective studies are necessary to determine the effectiveness of continuous renal replacement therapy in patients with multiorgan system dysfunction. Copyright © 2010 by the Society of Critical Care Medicine and the World Federation of Pediatric Intensive and Critical Care Societies.
Palmer L.S.,Schneider Childrens Hospital
Current Urology Reports | Year: 2010
Significant progress has been made over the past 15 years regarding the development of bladder control and the evaluation and management of pediatric voiding dysfunction. Incontinence is a problem of significant social consequence that is commonly due to dysfunctional voiding (ie, discoordination between the detrusor and the external sphincter). Biofeedback was introduced in 1979 but was relatively forgotten until the late 1990 s. Nonanimated biofeedback relates sphincter activity to electromyographic activity as reflected as tracing on a computer screen or auditory feedback. This successful modality was exclusively available until recently when animated biofeedback became available, allowing for a more rapid grasp of the physiologic event to be modulated. Biofeedback has been shown to be very effective in children to correct incontinence secondary to dysfunctional voiding, as well as in treating giggle incontinence and to help resolve vesicoureteral reflux. © 2010 Springer Science+Business Media, LLC.