scholarly journals Use of the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) in X-Linked Myotubular Myopathy: Content Validity and Psychometric Performance

2021 ◽  
Vol 8 (1) ◽  
pp. 63-77
Author(s):  
Tina Duong ◽  
Gale Harding ◽  
Sally Mannix ◽  
Cristina Abel ◽  
Dawn Phillips ◽  
...  
Keyword(s):  
Content Validity ◽  
Pediatric Population ◽  
Neuromuscular Disorders ◽  
Delphi Panel ◽  
Study Drug Administration ◽  
Psychometric Performance ◽  
Myotubular Myopathy ◽  
Children's Hospital ◽  
Children’S Hospital ◽  
Neuromuscular Functioning

X-linked myotubular myopathy (XLMTM) is a life-threatening, congenital myopathy characterized by extreme hypotonia, weakness, delayed motor milestones, and respiratory failure, often resulting in pediatric mortality. This study evaluated the content validity and psychometric performance of the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders as a measure of neuromuscular functioning in children with X-linked myotubular myopathy. This study was conducted in two phases. Phase I assessed the content validity of the measure for use in an XLMTM pediatric population through: literature review, clinical expert interviews, caregiver interviews, and a modified-Delphi panel among clinicians. Phase II assessed psychometric performance based on the INCEPTUS observational clinical study and the ASPIRO interventional gene therapy study, including tests of reliability (internal consistency, test-retest, and interrater), validity (construct and criterion), and responsiveness based on observational and interventional clinical trial data analyses. Data established construct validity and reliability of the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders among XLMTM patients before administration of resamirigene bilparvovec, and sensitivity to study drug administration as evidenced by the significant post-administration response in ASPIRO. Findings support the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders as an appropriate neuromuscular functioning assessment in a pediatric X-linked myotubular myopathy patient population.

Passy-Muir Speaking Valve Use in a Children's Hospital: An Interdisciplinary Approach

2008 ◽  
Vol 18 (2) ◽  
pp. 76-86 ◽  
Author(s):  
Lauren Hofmann ◽  
Joseph Bolton ◽  
Susan Ferry
Keyword(s):  
Tracheostomy Tube ◽  
Pediatric Population ◽  
Interdisciplinary Approach ◽  
Extended Period ◽  
Speech Development ◽  
Tube Placement ◽  
Background Information ◽  
Speaking Valve ◽  
Children's Hospital ◽  
Children’S Hospital

Abstract At The Children's Hospital of Philadelphia (CHOP) we treat many children requiring tracheostomy tube placement. With potential for a tracheostomy tube to be in place for an extended period of time, these children may be at risk for long-term disruption to normal speech development. As such, speaking valves that restore more normal phonation are often key tools in the effort to restore speech and promote more typical language development in this population. However, successful use of speaking valves is frequently more challenging with infant and pediatric patients than with adult patients. The purpose of this article is to review background information related to speaking valves, the indications for one-way valve use, criteria for candidacy, and the benefits of using speaking valves in the pediatric population. This review will emphasize the importance of interdisciplinary collaboration from the perspectives of speech-language pathology and respiratory therapy. Along with the background information, we will present current practices and a case study to illustrate a safe and systematic approach to speaking valve implementation based upon our experiences.

scholarly journals 1367. Reduced Ceftaroline Susceptibility Among Invasive MRSA Isolates at a Tertiary Children’s HospitalReduced Ceftaroline Susceptibility Among Invasive MRSA Isolates at a Tertiary Children’s Hospital

2020 ◽  
Vol 7 (Supplement_1) ◽  
pp. S693-S694
Author(s):  
Lauren Sommer ◽  
Jesus G Vallejo ◽  
Kristina G Hulten ◽  
Sheldon L Kaplan ◽  
Jonathon C McNeil
Keyword(s):  
Pediatric Population ◽  
Staphylococcal Infection ◽  
Healthcare Associated Infections ◽  
Accessory Gene ◽  
Children's Hospital ◽  
Children’S Hospital ◽  
Mrsa Infection ◽  
Group Ii ◽  
Healthcare Associated ◽  
Research Grant

Abstract Background The emergence of community-acquired methicillin-resistant Staphylococcus aureus (MRSA) in the late 1990s-early 2000s complicated the empiric management of suspected staphylococcal infection in children. Rising clindamycin resistance rates in many communities adds further to management challenges. Ceftaroline, an anti-MRSA cephalosporin, represents an attractive therapy option. Little data are available, however, regarding the frequency of reduced susceptibility (RS) to ceftaroline among MRSA isolates from a general pediatric population. Methods Isolates were selected from an ongoing S. aureus surveillance study at Texas Children’s Hospital. Invasive MRSA isolates from 2015-2018 were included. Isolates were initially screened for ceftaroline RS with E-test; all isolates with a ceftaroline E-test MIC ≥ 1.5µg/ml underwent ceftaroline broth dilution. Ceftaroline RS was regarded as an MIC ≥ 2 µg/ml; full ceftaroline resistance was defined as an MIC ≥ 8 μg/ml. Accessory gene regulator (agr) groups were characterized by PCR. Results 201 viable isolates were included. The ceftaroline MIC50 and MIC90 were 0.5 and 1 µg/ml, respectively (Figure 1). Six isolates had MIC ≥ 2 µg/ml (2.9%) with two having MIC ≥ 8 µg/ml (0.9%). All ceftaroline RS isolates were from healthcare associated infections. Ceftaroline RS isolates were more often associated with clindamycin-resistance and agr group II (Figure 2). Infections with ceftaroline RS were associated with central venous lines, recent ICU admission, preceding antibiotic exposure (specifically cephalosporins) and prior MRSA infection. One subject with MRSA CLABSI had a ceftaroline susceptible MRSA infection followed < 1 month later by a ceftaroline resistant infection (MIC =32 μg/ml); the isolates were identical by PFGE. Only 3 subjects had previously received ceftaroline, none of which subsequently developed a ceftaroline RS isolate. Figure 1. Ceftaroline MIC Distribution Figure 2. Clinical And Microbiologic Associations with Reduced Ceftaroline Susceptibility Conclusion Ceftaroline RS occurs in 2.9% of invasive MRSA isolates in children and is most prominent among healthcare-associated infections. These isolates were associated with clindamycin resistance and agr group II. While ceftaroline RS is rare among invasive MRSA infections, the lack of preceding ceftaroline exposure is concerning and warrants careful surveillance. Disclosures Sheldon L. Kaplan, MD, Allergan (Research Grant or Support)Pfizer (Grant/Research Support) Jonathon C. McNeil, MD, Allergan (Research Grant or Support, Allergan provided ceftaroline powder for use in studies described in this abstract)

Primary and Metastatic Lung Tumors in the Pediatric Population: A Review and 25-Year Experience at a Large Children's Hospital

2008 ◽  
Vol 132 (7) ◽  
pp. 1079-1103 ◽  
Author(s):  
Megan K. Dishop ◽  
Supriya Kuruvilla
Keyword(s):  
Medical Literature ◽  
Pediatric Population ◽  
Case Series ◽  
Lung Tumors ◽  
Malignant Neoplasms ◽  
Primary Tumors ◽  
Children's Hospital ◽  
Pathologic Features ◽  
Metastatic Lung ◽  
Children’S Hospital

Abstract Context.—Primary lung neoplasms are rare in children, but they comprise a broad and interesting spectrum of lesions, some of which are familiar from other tissue sites, and some of which are unique to the pediatric lung. Objective.—To determine the relative incidence of primary and metastatic lung tumors in children and adolescents through a single-institution case series, to compare these data to reports in the medical literature, to discuss the clinical and pathologic features of primary tumors of the tracheobronchial tree and lung parenchyma in children, and to provide recommendations for handling pediatric lung cysts and tumors. Data Sources.—A 25-year single institutional experience with pediatric lung tumors, based on surgical biopsies and resections at Texas Children's Hospital from June 1982 to May 2007, an additional 40 lung tumors referred in consultation, and a review of the medical literature. Conclusions.—A total of 204 pediatric lung tumors were diagnosed at our institution, including 20 primary benign lesions (9.8%), 14 primary malignant lesions (6.9%), and 170 secondary lung lesions (83.3%). The ratio of primary benign to primary malignant to secondary malignant neoplasms is 1.4:1:11.6. The common types of lung cancer in adults are exceptional occurrences in the pediatric population. The most common primary lung malignancies in children are pleuropulmonary blastoma and carcinoid tumor. Other primary pediatric lung tumors include congenital peribronchial myofibroblastic tumor and other myofibroblastic lesions, sarcomas, carcinoma, and mesothelioma. Children with primary or acquired immunodeficiency are at risk for Epstein-Barr virus–related smooth muscle tumors, lymphoma, and lymphoproliferative disorders. Metastatic lung tumors are relatively common in children and also comprise a spectrum of neoplasia distinct from the adult population.

scholarly journals Mucin 4: A Sensitive Diagnostic Marker for Pediatric Mucoepidermoid CarcinomaNick Shillingford

2020 ◽  
pp. 1-12
Author(s):  
Mackenzie Postel M S ◽  
◽  
Julia T Chu ◽  
Henry H Tran ◽  
Shamlal Mangray ◽  
...  
Keyword(s):  
Los Angeles ◽  
Rhode Island ◽  
Predictive Value ◽  
Pediatric Population ◽  
Children's Hospital ◽  
Morphological Heterogeneity ◽  
Muc4 Expression ◽  
Children’S Hospital ◽  
Expression Score ◽  
Sensitive Marker

Background: Pediatric salivary gland-type neoplasms (SGTNs) pose a significant diagnostic problem due to histo-morphological heterogeneity. Previous reports have shown that Mucin 4 (MUC4) expression is associated with adult mucoepidermoid carcinoma (MEC). We hypothesize that MUC4 is also a sensitive marker for distinguishing MEC from other SGTNs in the pediatric population. Objective: To evaluate MUC4 expression in pediatric SGTNs. Methods: A retrospective review of 74 SGTNs diagnosed between 1993–2015 at Children’s Hospital Los Angeles, Boston Children’s Hospital, and Rhode Island Hospital was performed. H&E sections of 31 MECs were compared to 3 adenoid cystic carcinomas (AdCCs), 6 acinic cell carcinomas (AcCCs), 30 pleomorphic adenomas (PAs), 3 mammary analogue secretory carcinomas (MASCs), and one sialoblastoma (SB). Samples underwent immunohistochemical staining for MUC4, with expression score criteria: 0% positivity = 0, 1-10% = +, 11-50% = ++, 51-90% = +++, >90% = ++++. Results: All MECs were MUC4-positive, with 25 (80.65%) having an expression score ≥ +++. AdCCs and PAs demonstrated no to minimal MUC4-positivity. Subsets of AcCCs and MASCs were unexpectedly MUC4-positive. As a novel marker for pediatric MEC, MUC4’s sensitivity = 100%, specificity = 79.41%, positive predictive value = 75.86%, and negative predictive value = 100%. Conclusion: MUC4 is a sensitive marker for pediatric MEC

scholarly journals Changes in the prevalence of index diseases in pediatric surgery: case analysis of single national children’s hospital

2021 ◽  
Vol 64 (3) ◽  
pp. 232-238
Author(s):  
Soo-Hong Kim ◽  
Yong-Hoon Cho ◽  
Hae Young Kim
Keyword(s):  
Acute Appendicitis ◽  
Pediatric Surgery ◽  
Pediatric Population ◽  
Hypertrophic Pyloric Stenosis ◽  
Relative Number ◽  
Emergency Operation ◽  
Absolute Number ◽  
Live Births ◽  
Children's Hospital ◽  
Children’S Hospital

Pusan National University Children’s Hospital (PNUCH) opened in November 2008 as the second National Children’s Hospital in Korea and celebrated its 10th anniversary in 2018. In the last decade, birth rates and pediatric populations have been declining sharply and has been more serious in Busan, Ulsan, and Gyeongsangnam-do where the PNUCH is located, affecting the prevalence of index diseases. The number of the index diseases was investigated by year according to changes in the population. Patients who underwent surgery at PNUCH for 10 years were included. Changes in pediatric population and the number of live births were analyzed using the data from Statistics Korea. The number of the index diseases was analyzed retrospectively through the review of electronic medical records. Statistical analysis was performed through Poisson regression. The number of live births and pediatric population decreased from 69,528 to 48,600 (30.1%) and 1,880,284 to 1,444,961 (23.2%), respectively. Operations of pediatric surgery decreased by 32.3% compared to that during the peak season, but the prevalence rate ratio of the pediatric population increased to 1.03 (<i>P</i><0.001). The number of emergency operation increased due to the largest surge in acute appendicitis operations. Both esophageal atresia and Hirschsprung disease decreased, while hypertrophic pyloric stenosis significantly increased. The absolute number of pediatric surgery cases decreased. However, considering the changes in pediatric population and the number of live births, the relative number of pediatric surgery cases does not changed. The decrease in the number of each disease would be related to the decrease in pediatric population and the number of live births. We should consider the increase of emergency operations such as acute appendicitis.

scholarly journals The Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND): Test development and reliability

2010 ◽  
Vol 20 (3) ◽  
pp. 155-161 ◽  
Author(s):  
A.M. Glanzman ◽  
E. Mazzone ◽  
M. Main ◽  
M. Pelliccioni ◽  
J. Wood ◽  
...  
Keyword(s):  
Neuromuscular Disorders ◽  
Test Development ◽  
Children's Hospital ◽  
Children’S Hospital

scholarly journals Etiology, Presentation and Management of Liver Abscesses at the Children’s Hospital Lahore

2010 ◽  
Vol 14 (4) ◽  
Author(s):  
Huma Arshad Cheema ◽  
Anjum Saeed
Keyword(s):  
Liver Abscess ◽  
Pediatric Population ◽  
Management Strategies ◽  
Positive Culture ◽  
Diagnostic Tools ◽  
Key Words Liver ◽  
Children's Hospital ◽  
Liver Abscesses ◽  
Satisfactory Outcome ◽  
Children’S Hospital

Background:  Liver abscess is a common problem among children. The purpose of this study is to describe the etiology, different management strategies and outcome of children with liver abscess in Children’s Hospital Lahore. Patients and Methods:  This study included 38 children with liver abscess seen during two-year period, from September 2004 to September 2006 at the Children’s Hospital and Institute of Child Health, Lahore. Diagnosis was made on history, examination and investigations. Ultrasound and CT scan were the main diagnostic tools. Results:  Out of 38 patients, 33 children underwent aspiration and remaining 5 were managed conservatively.  Eight aspirates yielded positive culture for various organisms. Three patients were positive for E.Coli, 2 for Staph Aureus, and 1 each case of Klebsiella and Pseudomonas. One patient had multiple micro-abscesses and was also positive for typhidot IgM. Another patient was positive for acid fast mycobacterium tuberculosis. All patients recovered from their abscesses. There was no mortality reported. Conclusions:  In our pediatric population, liver abscess are mostly caused by pyogenic organisms. Amebic etiology was not proven in any case. Majority of liver abscesses can be satisfactorily treated by aspiration and /or percutaneous image guided drainage with satisfactory outcome. Key Words:  Liver abscess, children.

scholarly journals QOL-41. CARDIAC DYSFUNCTION IN MEDULLOBLASTOMA SURVIVORS TREATED WITH PHOTON IRRADIATION

2020 ◽  
Vol 22 (Supplement_3) ◽  
pp. iii438-iii439
Author(s):  
Chantel Cacciotti ◽  
Christine Chordas ◽  
Kate Valentino ◽  
Rudy Allen ◽  
Peter Manley ◽  
...  
Keyword(s):  
Cardiac Dysfunction ◽  
Pediatric Population ◽  
Retrospective Chart Review ◽  
Cost Effective ◽  
Left Ventricular ◽  
Photon Radiation ◽  
Children's Hospital ◽  
Photon Irradiation ◽  
Craniospinal Radiation ◽  
Children’S Hospital

Abstract BACKGROUND Medulloblastoma is an aggressive central nervous system (CNS) tumor that occurs mostly in the pediatric population. Treatment includes surgical resection, craniospinal radiation (CSI) and chemotherapy. Children who receive standard photon radiation (RT) are at risk for cardiac toxicities. Potential late effects include coronary artery disease, left ventricular scarring and dysfunction, valvular damage and atherosclerosis. Current survivorship guidelines recommend routine ECHO surveillance for these patients but this comes at significant health care costs over a lifetime. We describe the experience of cardiac dysfunction in medulloblastoma survivors in a multi-institution study. METHODS A retrospective chart review of medulloblastoma patients treated between 1980 and 2010 with radiation at Lurie Children’s Hospital and Dana-Farber/ Boston Children’s Hospital who had an echocardiogram done following completion of therapy. RESULTS 168 patients were treated for medulloblastoma during the study time. Of whom, 80 patients had echocardiogram follow up and 76 received photon irradiation. The latter were included in the study. The mean age at CSI was 8.6 years (range 2.9- 20), and mean number of years post RT at echocardiogram 7.4 years (range 2–16). Mean ejection fraction (EF) was 60.03% and shortening fraction (SF) 33.8%. Four patients (5%) had abnormal results, all of which had EF&lt;50%. CONCLUSION Patients who received craniospinal irradiation for medulloblastoma therapy have relatively normal echocardiograms post treatment. Although RT may result in cardiac risks, echocardiograms may not be the most cost effective or efficacious mode to evaluate the risk in these survivors long term.

Assessing Immediate Bed Availability and Barriers to Discharge in a United States Children’s Hospital

2020 ◽  
pp. 1-5
Author(s):  
M. Catherine Burgess ◽  
Debbie DeLorenzo ◽  
Carl Eriksson
Keyword(s):  
United States ◽  
Large Scale ◽  
Pediatric Population ◽  
Hospital Setting ◽  
Hospitalized Patients ◽  
Treatment Needs ◽  
Children's Hospital ◽  
Discharge Plan ◽  
Children’S Hospital ◽  
Total Capacity

ABSTRACT Objectives: The aim of this study was to quantify immediate bed availability (IBA) in a United States children’s hospital and treatment needs of hospitalized patients whose needs could be met outside a traditional hospital setting. Methods: Using a novel tool to capture census, scheduled discharges, and resource needs for hospitalized patients, we surveyed our hospital’s 5 non-neonatal inpatient pediatric units on 4 d over 1 y. Results: Median ward occupancy was 81% (range, 58-79), median intensive care unit occupancy was 80% (range, 7-19), and median IBA was 42% (range, 34-59). A median of 14 patients per day (13% of total capacity) had treatment needs that could be met by providing limited support in a nontraditional setting; the most common reason for requiring ongoing hospitalization in this group of patients was a safe discharge plan. Conclusions: Our median IBA of 42% exceeds federal recommendations, but varies widely between days surveyed. Even on days when IBA percentage is high, our total number of available beds is unlikely to meet pediatric population needs in a large-scale public health emergency.

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