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Wahn U.,Charité - Medical University of Berlin | Ploszczuk A.,University of Bialystok | Adelt T.,Pediatric Center | Sandner B.,Pediatric Center | And 3 more authors.
Journal of Allergy and Clinical Immunology | Year: 2012

Background: Sublingual allergen-specific immunotherapy is a viable alternative to subcutaneous immunotherapy particularly attractive for use in children. Objective: This study investigated efficacy and safety of high-dose sublingual immunotherapy (SLIT) in children allergic to grass pollen in a randomized, double-blind, placebo-controlled trial. Methods: After a baseline seasonal observation, 207 children aged 4 to 12 years with grass pollen-allergic rhinitis/ rhinoconjunctivitis with/without bronchial asthma (Global Initiative for Asthma I/II) received either high-dose grass pollen SLIT or placebo daily for 1 pre-/co-seasonal period. The primary end point was the change of the area under the curve of the symptom-medication score (SMS) from the baseline season to the first season after start of treatment. Secondary outcomes were well days, responders, immunologic changes, and safety. Results: Mean changes in the area under the curve of the SMS from the baseline to the first grass pollen season after the start of treatment were -212.5 for the active group and -97.8 for the placebo group (P =.0040). Rhinoconjunctivitis SMS (P =.0020) and separated symptom and medication scores were also statistically different between the 2 groups (P = .0121 and P = .0226, respectively). The number of well days and the percentage of responders were greater in the active group. Changes in allergen-specific IgE and IgG levels indicated a significant immunologic effect. The treatment was well tolerated, and no serious treatment-related events were reported. Conclusions: This study confirmed that this SLIT preparation significantly reduced symptoms and medication use in children with grass pollen-allergic rhinoconjunctivitis. The preparation showed significant effects on allergen-specific antibodies, was well tolerated, and appeared to be a valid therapeutic option in children allergic to grass pollen. This trial was registered at www.clinicaltrials.gov as NCT00841256. (J Allergy Clin Immunol 2012;130:886-93.) © 2012 American Academy of Allergy, Asthma & Immunology.


Charvet L.E.,Lourie Center for Pediatric | O'Donnell E.H.,Massachusetts General Hospital | Belman A.L.,Lourie Center for Pediatric | Chitnis T.,Massachusetts General Hospital | And 7 more authors.
Multiple Sclerosis Journal | Year: 2014

Background: Approximately one-third of those with pediatric-onset multiple sclerosis (MS) experience cognitive impairment. Less is known concerning their change in cognitive functioning over time. Objective: Changes in cognitive function over time were measured in the largest pediatric cohort to date through the US Network of Pediatric MS Centers. Methods: A total of 67 individuals with pediatric MS (n=62) or clinically isolated syndrome (CIS, n=5), ranging from 8-17 years of age (mean age±standard deviation (SD)=14.37±2.02) completed initial and follow-up neuropsychological testing after an average of 1.64±0.63 years apart. The nine tests administered measure general intellect, attention and working memory, verbal memory, visuomotor integration, language, and executive functioning. Results: Rate of impairment (having one-third or more scores in the impaired range) was 37% at baseline and 33% at follow-up. Tests commonly impaired were measures of visuomotor integration, speeded processing, and attention. Most tested did not decline over two years. There was no clear pattern of change on any specific measure. Conclusion: Findings suggest that, over short timeframes, stable or even improved performances on measures of cognitive ability can occur. Pediatric MS may instead prevent expected age-related cognitive gains. © The Author(s) 2014.


Chabas D.,University of California at San Francisco | Ness J.,University of Alabama at Birmingham | Belman A.,National United University | Yeh E.A.,Jacobs Neurological Institute | And 10 more authors.
Neurology | Year: 2010

BACKGROUND: The clinical and MRI presentation differs between earlier-and later-onset pediatric multiple sclerosis (MS), whereas the effect of age on the CSF inflammatory profile is unknown and may contribute to delayed diagnosis. OBJECTIVES: To compare the CSF cellular and immunoglobulin G (IgG) profiles between earlier-and later-onset pediatric MS. METHODS: We queried the databases of 6 pediatric MS centers for earlier-onset (onset <11 years) and later-onset (â‰111 and <18 years) patients with MS or clinically isolated syndrome who underwent CSF analysis within the first 3 months of presentation (observational study). We compared CSF white blood cell (WBC) differential count, IgG index, and IgG oligoclonal bands between age groups. RESULTS: We identified 40 earlier-onset (mean age at onset = 7.2 ± 2.7 years, 60% females) and 67 later-onset pediatric MS patients (15.1 ± 1.7 years, 63% females). Although WBC count tended to be higher in earlier-onset patients (median = 9/mm [0-343] vs 6 [0-140], p = 0.15), they had a lower proportion of lymphocytes (70% [0-100] vs 93% [0-100] of WBCs, p = 0.0085; difference = +3% per 1-year increase of age, p = 0.0011) and higher proportion of neutrophils than later-onset patients (0.5% [0-75] vs 0% [0-50] of WBCs, p = 0.16; difference =-1% per 1-year increase of age, p = 0.033). In earlier-onset disease, fewer patients had an elevated IgG index than in the later-onset group (35% vs 68% of patients, p = 0.031). CONCLUSION: Age modifies the CSF profile at pediatric multiple sclerosis (MS) onset, which may mislead the diagnosis. Our findings suggest an activation of the innate rather than the adaptive immune system in the earlier stages of MS or an immature immune response. Copyright © 2010 by AAN Enterprises, Inc.


Bruni M.,Silver Creek Preschool | Cameron D.,University of Toronto | Dua S.,COTA Health | Noy S.,Pediatric Center
Physical and Occupational Therapy in Pediatrics | Year: 2010

Investigators have identified delays and differences in cognitive, language, motor, and sensory development in children with Down syndrome (DS). The purpose of this study was to determine the parent-reported frequency of sensory processing issues in children with DS aged 3-10 years, and the parent-reported functional impact of those sensory issues. Parents completed the short sensory profile (SSP) and a parent questionnaire (PQ). SSP results revealed a total score definite difference rate of 49%. Highest rates of probable and definite difference were in the low energy/weak, underresponsive/seeks sensation, and auditory filtering subsections of the SSP. Themes were generated from responses on the PQ regarding the functional impact of sensory differences on occupational performance in their children with DS, and related strategies currently used by parents. Findings from the study provide information to parents and health care professionals regarding sensory processing patterns in children with DS, and provide foundational data for future research. © 2010 Informa Healthcare USA, Inc.


Neri G.,Shodair Childrens Hospital | Neri G.,Instituto Of Genetica Umana | Martini-Neri M.E.,Shodair Childrens Hospital | Martini-Neri M.E.,Instituto Of Genetica Umana | And 2 more authors.
American Journal of Medical Genetics, Part A | Year: 2013

We describe a familial syndrome of renal dysplasia, Wilms tumor, hyperplasia of the endocrine pancreas, fetal gigantism, multiple congenital anomalies and mental retardation. This condition was previously described by Perlman et al. [1973, 1975] and we propose to call it the "Perlman syndrome." It appears to be transmitted as an autosomal recessive trait. The possible relationships between dysplasia, neoplasia and malformation are discussed. © 2013 Wiley Periodicals, Inc.


Landmann E.,Justus Liebig University | Kollerits B.,Innsbruck Medical University | Kreuder J.G.,Justus Liebig University | Blum W.F.,Eli Lilly and Company | And 2 more authors.
Hormone Research in Paediatrics | Year: 2012

Background/Aims: In postnatal life, polymorphisms in the promoter region of IGFBP3 were associated with insulin-like growth factor binding protein (IGFBP)-3 plasma levels. Whether these associations exist in utero has not been studied yet. Polymorphisms in the IGF1 promoter (polymorphic CA-repeat) and the insulin gene variable number tandem repeats locus (INS VNTR) are further polymorphisms of interest, because associations with birth weight have been reported. We aimed to investigate associations between polymorphisms in the promoter regions of IGF1 (wild type 192 bp), IGFBP3 (rs2854744; rs13241830), and INS VNTR (rs689) with cord plasma levels of IGF-I, IGF-II, and IGFBP-3. Methods: We measured IGF-I, IGF-II, and IGFBP-3 concentrations in cord blood from 677 neonates and genotyped the selected polymorphisms. Results: Carriers of the minor allele of both polymorphisms in the IGFBP3 gene had, on average, 4-5% lower IGFBP-3 levels per copy of the respective minor allele (p = 0.002 and p = 0.028) when compared to wild type carriers. The IGF1 promoter and the INS VNTR polymorphisms were not associated with IGF-I, IGF-II, or IGFBP-3 levels. Conclusions: Our data show associations of cord plasma IGFBP-3 levels and the IGFBP3 gene variants but not of IGF1 promoter and INS VNTR polymorphisms with IGF-I, IGF-II, or IGFBP-3 levels in utero. Copyright © 2012 S. Karger AG, Basel.


Mentessidou A.,Pediatric Center | Mirilas P.,Pediatric Center
Journal of Pediatric Surgery | Year: 2015

Little is known about the possibility that ruptured appendicitis may produce a false sonographic appearance of intussusception. We present here a case of a periappendiceal phlegmon mimicking ileocolic intussusception on ultrasound in a 3.5-year-old girl and provide a surgico-anatomic explanation on the basis of the intraoperative findings for the false sonographic image. CT imaging was used to make the diagnosis. Intraoperatively, it was revealed that the cecum and sigmoid, which were adherent to each other with pseudomembranes, formed an intestinal mass around the appendix. Accordingly, the appendicolith at the center of the phlegmon was responsible for the central echogenicity, and the surrounding cecum and sigmoid for the external hypoechoic and hyperechoic rings of the target-sign appearing mass on the preoperative ultrasound. Such an understanding of the etiology of the false sonographic image may help to increase awareness and avoid misdiagnosis. © 2015 Elsevier Inc.


PubMed | Pediatric Center
Type: Journal Article | Journal: Seminars in pediatric neurology | Year: 2011

This is a proposal for suggested physician competencies in the diagnosis and treatment of children and adolescent patients with headaches. Headaches are common in childhood and occur in up to 10% of school-aged children and over 20% of female adolescents. Therefore, the appropriate recognition, evaluation, and treatment should be a high priority of training and maintenance of certification programs.


PubMed | Pediatric Center
Type: Case Reports | Journal: Journal of pediatric surgery | Year: 2015

Little is known about the possibility that ruptured appendicitis may produce a false sonographic appearance of intussusception. We present here a case of a periappendiceal phlegmon mimicking ileocolic intussusception on ultrasound in a 3.5-year-old girl and provide a surgico-anatomic explanation on the basis of the intraoperative findings for the false sonographic image. CT imaging was used to make the diagnosis. Intraoperatively, it was revealed that the cecum and sigmoid, which were adherent to each other with pseudomembranes, formed an intestinal mass around the appendix. Accordingly, the appendicolith at the center of the phlegmon was responsible for the central echogenicity, and the surrounding cecum and sigmoid for the external hypoechoic and hyperechoic rings of the target-sign appearing mass on the preoperative ultrasound. Such an understanding of the etiology of the false sonographic image may help to increase awareness and avoid misdiagnosis.


News Article | February 28, 2017
Site: www.businesswire.com

WILMINGTON, N.C.--(BUSINESS WIRE)--Pharmaceutical Product Development, LLC (PPD) has established a Rare Disease and Pediatric Center of Excellence to oversee all rare disease and pediatric-related drug development activities. This new center of excellence provides pharmaceutical and biotechnology clients with a dedicated team of professionals focused on the design and execution of clinical trials that address the unique strategic, operational, medical and scientific challenges of clinical studies in rare disease and pediatric patient populations. PPD announced the new center of excellence on Rare Disease Day, which is observed on the last day of February each year to raise awareness of rare diseases. A disease is designated as rare in the United States when it affects fewer than 200,000 people, in Europe when it affects no more than five in 10,000 people and in Japan when it affects fewer than 50,000 people. In the U.S. and Europe alone, as many as 60 million individuals are affected by one of the more than 6,000 identified rare diseases. “Our new center of excellence builds on PPD’s longstanding expertise and experience to address the special drug development considerations of rare diseases and pediatrics,” said Karen Kaucic, M.D., PPD senior vice president and head of the center of excellence. “With a great majority of rare diseases touching children, we believe our approach will enable us to be a more connected and active partner. Our clients can depend on us for the latest trial design innovations, the ability to navigate complex trial logistics and our keen understanding of customized patient access strategies in low-prevalence, widely dispersed patient populations. We felt today was an appropriate time to introduce this new venture, as we join with the drug development community in recognizing Rare Disease Day.” Dr. Kaucic will leverage her leadership experience in rare diseases and pediatrics to drive the implementation of PPD’s center of excellence, leading a dedicated team with a wealth of expertise in rare disease trial design and execution in such disciplines as product development, clinical operations, commercial strategy and early engagement. The PPD team members in the center of excellence are seasoned pharmaceutical and clinical research professionals who have hands-on experience with rare diseases and pediatrics. The team is focused on addressing the specific challenges presented by the small, widely dispersed populations of rare disease and pediatric patients. This group of functional experts has been assembled to support end-to-end clinical trial solutions in such areas as feasibility strategy, clinical information, biostatistics, pharmacovigilance, lab operations and business analytics. PPD’s particular expertise in rare diseases stems from having conducted more than 220 studies with more than 50,000 patients over the past five years. PPD also is a leading clinical researcher in pediatric drug development, having conducted more than 400 clinical trials during the past five years, enrolling more than 97,000 patients across a wide range of therapeutic indications, including infectious diseases, respiratory, cardiology, oncology and immunology. PPD leverages a pediatric investigator network that further enhances its pediatric clinical development capabilities by providing clients with faster trial startup, more predictable patient enrollment and higher-quality data. For both rare disease and pediatric trials, PPD has deployed a purpose-built framework that ensures the application of best practices in study development and enables the company to develop a customized approach to address the unique needs of these trials. PPD is a leading global contract research organization providing comprehensive, integrated drug development, laboratory and lifecycle management services. Our clients and partners include pharmaceutical, biotechnology, medical device, academic and government organizations. With offices in 47 countries and more than 18,500 professionals worldwide, PPD applies innovative technologies, therapeutic expertise and a firm commitment to quality to help clients and partners bend the cost and time curve of drug development to deliver life-changing therapies that improve health. For more information, visit www.ppdi.com. Except for historical information, all of the statements, expectations and assumptions, including statements, expectations and assumptions about PPD’s Rare Disease and Pediatric Center of Excellence contained in this news release are forward-looking statements that involve a number of risks and uncertainties. Although PPD attempts to be accurate in making these forward-looking statements, it is possible that future circumstances might differ from the assumptions on which such statements are based and could cause actual results to differ materially from the forward-looking statements. Other important factors that could cause future results to differ materially include the following: risks associated with and dependence on strategic relationships; the ability to attract, integrate, retain and train key personnel; competition in the outsourcing industry; rapid technological advances that make our services or capabilities less competitive; compliance with drug development regulations; changes in the regulation of the drug development process; PPD’s ability to win new business; overall global economic conditions; economic conditions, research and development spending, and outsourcing trends in the pharmaceutical, biotechnology and government-sponsored research sectors; consolidation in the pharmaceutical and biotechnology industries; loss, delay or modification of large contracts; higher-than-expected cancellation rates; the rate of conversion of backlog into revenue; actual operating performance; risks associated with acquisitions and investments; and the ability to control SG&A spending. PPD assumes no obligation and expressly disclaims any duty to update these forward-looking statements in the future, except as required by applicable law. These forward-looking statements should not be relied upon as representing PPD’s estimates or views as of any date subsequent to the date hereof.

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