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Fuchs S.,Ann and oRobert H. Lurie Childrens Hospital of Chicago
Pediatric Clinics of North America

Pediatricians regularly see emergencies in the office, or children that require transfer to an emergency department, or hospitalization. An office self-assessment is the first step in determining how to prepare for an emergency. The use of mock codes and skill drills make office personnel feel less anxious about medical emergencies. Emergency information forms provide valuable, quick information about complex patients for emergency medical services and other physicians caring for patients. Furthermore, disaster planning should be part of an office preparedness plan. © 2013 Elsevier Inc. Source

Smith L.,Center for Pediatric Genomic Medicine | Rhead W.,Childrens Hospital of Wisconsin | Charrow J.,Ann and oRobert H. Lurie Childrens Hospital of Chicago | Shankar S.P.,Emory University | And 9 more authors.
Molecular Genetics and Metabolism

Background: Gaucher Disease type 1 (GD1) often manifests in childhood. Early treatment with enzyme replacement therapy (ERT) may prevent disease complications. We report the assessment of velaglucerase alfa ERT in pediatric GD1 patients who participated in a long-term extension study (HGT-GCB-044, ClinicalTrials.gov Identifier NCT00635427). Methods: Safety and efficacy were evaluated in pediatric patients receiving velaglucerase alfa 30-60 U/kg by intravenous infusion every other week. In addition to key hematological and visceral efficacy assessments, exploratory assessments conducted specifically in pediatric patients included evaluation of height, bone age, bone marrow burden, and Tanner stage of puberty. Results: The study included 24 pediatric patients. Fifteen patients were naïve to ERT on entry into the preceding trials TKT032 (12-month trial) or HGT-GCB-039 (9-month trial): in the preceding trials, ten of these 15 patients received velaglucerase alfa and five patients received imiglucerase ERT. Nine patients in the study were previously treated with imiglucerase for >. 30 months and were switched to velaglucerase alfa in the preceding trial TKT034 (12-month trial). Cumulative ERT exposure in the clinical studies ranged from 2.0 to 5.8 years. Three serious adverse events, including a fatal convulsion, were reported; none were deemed related to velaglucerase alfa. One patient tested positive for anti-velaglucerase alfa antibodies. An efficacy assessment at 24 months showed that velaglucerase alfa had positive effects on primary hematological and visceral parameters in treatment-naïve patients, which were maintained with longer-term treatment. Disease parameters were stable in patients switched from long-term imiglucerase ERT. Exploratory results may suggest benefits of early treatment to enable normal growth in pediatric patients. Conclusion: The safety profile and clinical response seen in pediatric patients are consistent with results reported in adults. © 2016 Shire Development LLC. Source

Pierce M.C.,Ann and oRobert H. Lurie Childrens Hospital of Chicago | Pierce M.C.,Northwestern University | Kaczor K.,Ann and oRobert H. Lurie Childrens Hospital of Chicago | Thompson R.,Richard lica Center For Innovation In Children And Family Services
Pediatric Clinics of North America

The social environment of a child is a key determinant of the child's current and future health. Factors in a child's family environment, both protective and harmful, have a profound impact on a child's long-term health, brain development, and mortality. The social history may be the best all-around tool available for promoting a child's future health and well-being. It is a key first step in identifying social needs of a child and family so that they may benefit from intervention. This article focuses on key social history elements known to increase a child's risk of maltreatment and provides case examples. © 2014 Elsevier Inc. Source

Sharma A.K.,Ann and oRobert H. Lurie Childrens Hospital of Chicago | Sharma A.K.,Northwestern University | Cheng E.Y.,Ann and oRobert H. Lurie Childrens Hospital of Chicago | Cheng E.Y.,Northwestern University
Advanced Drug Delivery Reviews

Regenerative medicine strategies combine various attributes from multiple disciplines including stem cell biology, chemistry, materials science and medicine. The junction at which these disciplines intersect provides a means to address unmet medical needs in an assortment of pathologies with the goal of creating sustainable, functional replacement tissues. Tissue damage caused by trauma for example, requires rapid responses in order to mitigate further tissue deterioration. Cell/scaffold composites have been utilized to initiate and stabilize regenerative responses in vivo with the hope that functional tissue can be attained. Along with the gross reconfiguration of regenerating tissues, small molecules and growth factors also play a pivotal role in tissue regeneration. Several regenerative studies targeting a variety of urological tissues demonstrate the utility of these small molecules or growth factors in an in vivo setting. © 2014 Elsevier B.V. Source

Bury M.I.,Ann and oRobert H. Lurie Childrens Hospital of Chicago | Fuller N.J.,Ann and oRobert H. Lurie Childrens Hospital of Chicago | Meisner J.W.,Northwestern University | Hofer M.D.,Northwestern University | And 12 more authors.

Current attempts at tissue regeneration utilizing synthetic and decellularized biologic-based materials have typically been met in part by innate immune responses in the form of a robust inflammatory reaction at the site of implantation or grafting. This can ultimately lead to tissue fibrosis with direct negative impact on tissue growth, development, and function. In order to temper the innate inflammatory response, anti-inflammatory signals were incorporated through display on self-assembling peptide nanofibers to promote tissue healing and subsequent graft compliance throughout the regenerative process. Utilizing an established urinary bladder augmentation model, the highly pro-inflammatory biologic scaffold (decellularized small intestinal submucosa) was treated with anti-inflammatory peptide amphiphiles (AIF-PAs) or control peptide amphiphiles and used for augmentation. Significant regenerative advantages of the AIF-PAs were observed including potent angiogenic responses, limited tissue collagen accumulation, and the modulation of macrophage and neutrophil responses in regenerated bladder tissue. Upon further characterization, a reduction in the levels of M2 macrophages was observed, but not in M1 macrophages in control groups, while treatment groups exhibited decreased levels of M1 macrophages and stabilized levels of M2 macrophages. Pro-inflammatory cytokine production was decreased while anti-inflammatory cytokines were up-regulated in treatment groups. This resulted in far fewer incidences of tissue granuloma and bladder stone formation. Finally, functional urinary bladder testing revealed greater bladder compliance and similar capacities in groups treated with AIF-PAs. Data demonstrate that AIF-PAs can alleviate galvanic innate immune responses and provide a highly conducive regenerative milieu that may be applicable in a variety of clinical settings. © 2014 Elsevier Ltd. Source

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