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https://www.readbyqxmd.com/read/30452422/phase-1-study-of-edasalonexent-cat-1004-an-oral-nf-%C3%AE%C2%BAb-inhibitor-in-pediatric-patients-with-duchenne-muscular-dystrophy
#1
Erika Finanger, Krista Vandenborne, Richard S Finkel, H Lee Sweeney, Gihan Tennekoon, Sabrina Yum, Maria Mancini, Pradeep Bista, Andrew Nichols, Hanlan Liu, Angelika Fretzen, Joanne M Donovan
BACKGROUND: Edasalonexent is an orally administered small molecule designed to inhibit NF-κB, which is activated from infancy in Duchenne muscular dystrophy and is central to causing muscle damage and preventing muscle regeneration. OBJECTIVE: Evaluate the safety, tolerability, pharmacokinetics and exploratory pharmacodynamics of three doses of edasalonexent in ambulatory males ≥4 to <8 years of age with genetically confirmed Duchenne muscular dystrophy. METHODS: This was a 1-week, open-label, multiple-dose study with 3 sequential ascending doses (33, 67 and 100 mg/kg/day) of edasalonexent administered under different dietary conditions to 17 males with a mean age of 5...
November 15, 2018: Journal of Neuromuscular Diseases
https://www.readbyqxmd.com/read/30446895/effectiveness-of-antibiotic-therapy-in-pediatric-patients-with-cat-scratch-disease
#2
Loujain A Shorbatli, Katalin I Koranyi, Milap C Nahata
Background Appropriate antibiotic treatment of cat scratch disease (CSD) in pediatrics is not well established. Objective The purpose of this study was to evaluate the response rates of antibiotic therapy in pediatrics with CSD. Methods The electronic medical records of a cohort of pediatric patients with confirmed diagnosis of CSD (2006-2016) were reviewed, retrospectively. Data collection included patient demographics, clinical and laboratory results, antibiotic treatment and follow-up evaluations. Results One hundred and seventy-five patients (aged 7...
November 16, 2018: International Journal of Clinical Pharmacy
https://www.readbyqxmd.com/read/30368034/the-effect-of-faculty-coaching-on-resident-attitudes-confidence-and-patient-rated-communication-a-multi-institution-study
#3
Caroline E Rassbach, Alyssa L Bogetz, Nicola Orlov, Alisa McQueen, Vasudha Bhavaraju, David Mahoney, Collin Leibold, Rebecca L Blankenburg
OBJECTIVE: Despite a national focus on physician-patient communication, there is a paucity of literature on how patient and family feedback (PFF) can be used as a tool to help residents learn communication skills. The purpose of this study was to assess the effect of coaching on residents' attitudes towards PFF, self-confidence in communication, and patient-rated communication skills. METHODS: This was an IRB-approved, randomized-controlled trial with pediatric residents at three institutions in 2015-2016...
October 24, 2018: Academic Pediatrics
https://www.readbyqxmd.com/read/30345955/assessment-of-the-location-of-the-mandibular-lingula-in-pediatric-patients-using-cone-beam-computed-tomography-images
#4
Johan K M Aps, Leslie Yuan Gazdeck, Travis Nelson, Rebecca L Slayton, JoAnna M Scott
Purpose: The purpose of this study was to determine the position of the mandibular lingula in a sample of cone beam computed tomography (CBCT) images taken on pediatric patients. Methods: A sample of 280 outpatient CBCT (i-CAT) scans (153 males and 127 females) were divided into three age groups: six to nine years (n=103), 10 to 13 years (n=103), and 14 to 18 years (n=74). The position of the lingula was determined relative to the anterior and posterior border of the ramus, mandibular notch, inferior border of the mandible, and mandibular plane...
May 15, 2018: Journal of Dentistry for Children
https://www.readbyqxmd.com/read/30275747/qualitative-analysis-of-the-psychosocial-adaptation-process-in-children-with-chronic-kidney-disease-toward-effective-support-during-transition-from-childhood-to-adulthood
#5
Haruka Aoto, Hikaru Nakatani, Shunsuke Kanayama, Shin-Ichi Okada, Mika Fukada, Keiichi Hanaki
Background: Pediatric chronic renal disease only shows abnormal values in a urinalysis in the initial stage, and subjective signs and symptoms are rare. If adolescents with chronic renal disease face a disease crisis combined with the usual developmental crisis, this may cause psychosocial maladaptation. We analyzed psychosocial adaptation in Japanese children with chronic renal disease in order to identify factors influencing healthy adaptation. Methods: Ten children and adult patients with chronic kidney disease attending Tottori University Hospital, Japan in 2016 participated in a semi-structured interview (a modified version of the grounded theory approach) comprising questions about episodes since disease onset and thoughts/feelings at onset...
September 2018: Yonago Acta Medica
https://www.readbyqxmd.com/read/30232023/the-impact-of-social-deprivation-on-pediatric-promis-health-scores-after-upper-extremity-fracture
#6
Ugochi C Okoroafor, William Gerull, Melissa Wright, Jason Guattery, Brinkley Sandvall, Ryan P Calfee
PURPOSE: Although social deprivation is acknowledged to influence physical and mental health in adults, it is unclear if and how social deprivation influences perceived health in children. This study was conducted to evaluate the impact of social deprivation on Patient-Reported Outcomes Measurement Information System (PROMIS) scores in children presenting for treatment of upper extremity fractures. METHODS: This cross-sectional evaluation analyzed data from 975 new pediatric patients (8-17 years old) with upper extremity fractures presenting to a tertiary orthopedic center between June 1, 2016, and June 1, 2017...
October 2018: Journal of Hand Surgery
https://www.readbyqxmd.com/read/30138013/results-from-the-child-adolescent-anxiety-multimodal-longitudinal-study-camels-functional-outcomes
#7
RANDOMIZED CONTROLLED TRIAL
Anna J Swan, Philip C Kendall, Thomas Olino, Golda Ginsburg, Courtney Keeton, Scott Compton, John Piacentini, Tara Peris, Dara Sakolsky, Boris Birmaher, Anne Marie Albano
OBJECTIVE: To report functional outcomes from the multisite Child/Adolescent Anxiety Multimodal Extended Long-term Study (CAMELS), which examined the impact of youth anxiety treatment (cognitive-behavioral therapy [CBT], coping cat; Sertraline, SRT; COMB [CBT + SRT]; pill placebo) on (a) global and (b) domain-specific functioning assessed an average of 3.1 times, 3- to 12-years postrandomization (first assessment = mean 6.5 years postrandomization). METHOD: Three-hundred and 19 of 488 families from the Child/Adolescent Anxiety Multimodal Study (CAMS; Walkup et al...
September 2018: Journal of Consulting and Clinical Psychology
https://www.readbyqxmd.com/read/30130519/measuring-the-reliability-and-construct-validity-of-the-pediatric-evaluation-of-disability-inventory-computer-adaptive-test-pedi-cat-in-children-with-cerebral-palsy
#8
Benjamin J Shore, Benjamin G Allar, Patricia E Miller, Travis H Matheney, Brian D Snyder, Maria Fragala Pinkham
OBJECTIVE: The purpose of this study was to (1) investigate the construct validity and (2) test-retest reliability of the Pediatric Evaluation of Disability Inventory - Computer Adaptive Test (PEDI-CAT) in children with cerebral palsy (CP). DESIGN: A prospective convenience cross-sectional sample SETTING: Multidisciplinary CP clinic in a tertiary level pediatric children's hospital PARTICIPANTS: One hundred and one, English and Spanish speaking school aged children with a diagnosis of CP, stratified by Gross Motor Function Classification System level, who presented to our multidisciplinary clinic...
August 18, 2018: Archives of Physical Medicine and Rehabilitation
https://www.readbyqxmd.com/read/30102478/allergen-variability-and-house-dust-mite-sensitivity-in-pre-school-children-with-allergic-complaints
#9
Selçuk Yazıcı, Soner Güneş, Melike Kurtuluş-Çokboz, Özlem Kemer, Gülce Baranlı, Sezin Aşık-Akman, Demet Can
Yazıcı S, Güneş S, Kurtuluş-Çokboz M, Kemer Ö, Baranlı G, Aşık-Akman S, Can D. Allergen variability and house dust mite sensitivity in pre-school children with allergic complaints. Turk J Pediatr 2018; 60: 41-49. The increase in the prevalence of allergic diseases in pre-school children who are often at home may be due to an increase in house dust mite sensitivity, which is rarely expected in this age group. In our study, it was aimed to investigate allergen sensitivities, especially house dust mite sensitivity in pre-school children with allergic disease complaints by skin prick test (SPT)...
2018: Turkish Journal of Pediatrics
https://www.readbyqxmd.com/read/30092382/measuring-the-involvement-in-family-life-of-children-with-autism-spectrum-disorder-a-dbpnet-study
#10
Justin Schwartz, Noelle Huntington, Marisa Toomey, Michele Laverdiere, Katherine Bevans, Nathan Blum, Carolyn Bridgemohan
BACKGROUND: Children with Autism Spectrum Disorder (ASD) have social and communication deficits that impair their involvement in family life. No measures of child involvement in the family have been validated for the ASD population. AIM: To evaluate the validity of a measure of Family Involvement (FI) of children ages 5-12 with ASD. METHOD: Parents of children ages 5-12 with ASD (n = 114) completed FI items from the PROMIS® pediatric Family Relationships item bank in computerized adaptive testing (CAT) format, as well as measures of ASD symptom burden, parenting stress, and parental depression...
August 6, 2018: Research in Developmental Disabilities
https://www.readbyqxmd.com/read/30081662/performance-of-pediatric-promis-cats-in-children-with-upper-extremity-fractures
#11
William D Gerull, Ugochi C Okoroafor, Jason Guattery, Charles A Goldfarb, Lindley B Wall, Ryan P Calfee
BACKGROUND: This study was designed to quantify the performance of the pediatric Patient-Reported Outcome Measurement Information System (PROMIS) when delivered as part of routine care to children with upper extremity (UE) fractures. METHODS: This cross-sectional study analyzed 964 new pediatric patients presenting with an UE fracture. All patients completed PROMIS computer adaptive tests for pain interference, peer relationships, UE function, and mobility domains at clinic registration...
August 6, 2018: Hand: Official Journal of the American Association for Hand Surgery
https://www.readbyqxmd.com/read/29999144/-beta-hcg-producing-thymic-teratoma-an-uncommon-cause-of-peripheral-precocious-puberty
#12
Claudia Lozano C, Marcela Molina P
INTRODUCTION: Among the causes of peripheral precocious puberty in men are the beta-human cho rionic gonadotropin (Β-HCG)-secreting tumors, such as hepatoblastomas, dysgerminomas, chorio carcinomas, and immature teratomas. In pediatrics, the mediastinal teratomas are rare, representing the 7-10% of extragonadal teratomas. OBJECTIVE: To describe the case of a patient with peripheral precocious puberty due to a Β-HCG -secreting thymic teratoma. CLINICAL CASE: A seven-years-old schoolboy presents a three-months history of voice changes, gynecomastia, pubic hair appearance, and increased genital volume...
June 2018: Revista Chilena de Pediatría
https://www.readbyqxmd.com/read/29987743/a-conceptual-model-of-angelman-syndrome-and-review-of-relevant-clinical-outcomes-assessments-coas
#13
Joseph C Grieco, Beverly Romero, Emuella Flood, Raquel Cabo, Jeannie Visootsak
BACKGROUND: Angelman syndrome (AS) is a rare, neurological genetic disorder for which no clinical outcomes assessments (COAs) or conceptual models (CM) have been developed. OBJECTIVE: This study aimed to identify symptoms and impacts relevant and important in this patient population and develop a conceptual model of AS, and to evaluate the content validity of selected COA instruments with potential for inclusion in clinical studies of AS to capture treatment benefit...
July 10, 2018: Patient
https://www.readbyqxmd.com/read/29981855/provision-of-parent-feedback-via-the-communication-assessment-tool-does-it-improve-resident-communication-skills
#14
Dominick DeBlasio, Francis J Real, Nicholas J Ollberding, Melissa D Klein
OBJECTIVE: To determine the impact of a curriculum that included parent feedback, via the Communication Assessment Tool (CAT), on resident communication skills. METHODS: In a prospective, controlled study, categorical pediatric residents in continuity clinic were divided into control and intervention groups based on clinic day. Parent feedback was obtained for all residents at the beginning and end of the year using the CAT, a validated survey to assess physician communication...
July 5, 2018: Academic Pediatrics
https://www.readbyqxmd.com/read/29943851/pediatric-pet-consort-dermatitis-allergic-contact-dermatitis-from-transfer-of-bronopol-from-a-pet-cat
#15
Dathan Hamann, Alyson Ridpath, Esteban Fernandez Faith
Consort dermatitis refers to an allergic contact dermatitis caused by transfer from an intimate contact to a sensitized patient. Although close contact with other humans most commonly provokes consort dermatitis, pets have been the source in a minority of cases. We present a unique case of transfer dermatitis from a patient's cat litter to her forearms. Pediatric dermatologists should be aware of the possibility of consort or "transfer" allergic contact dermatitis from pets.
September 2018: Pediatric Dermatology
https://www.readbyqxmd.com/read/29935115/thyroid-storm-after-choking
#16
Ana L Creo, Bryan C Cannon, Siobhan T Pittock
BACKGROUND: A thyroid storm (TS) is a rare, but life-threatening condition in hyperthyroid patients. Mortality in adult TS patients may be as high as 10%. Typically, a trigger precipitates the storm in hyperthyroid patients. CASE PRESENTATION: We report the case of an adolescent girl with untreated hyperthyroidism who developed fulminant TS after a significant choking episode. Initially, she was found to have neck swelling and tachycardia leading providers to suspect infection...
August 28, 2018: Journal of Pediatric Endocrinology & Metabolism: JPEM
https://www.readbyqxmd.com/read/29895324/toxocariasis-a-silent-threat-with-a-progressive-public-health-impact
#17
REVIEW
Jia Chen, Quan Liu, Guo-Hua Liu, Wen-Bin Zheng, Sung-Jong Hong, Hiromu Sugiyama, Xing-Quan Zhu, Hany M Elsheikha
BACKGROUND: Toxocariasis is a neglected parasitic zoonosis that afflicts millions of the pediatric and adolescent populations worldwide, especially in impoverished communities. This disease is caused by infection with the larvae of Toxocara canis and T. cati, the most ubiquitous intestinal nematode parasite in dogs and cats, respectively. In this article, recent advances in the epidemiology, clinical presentation, diagnosis and pharmacotherapies that have been used in the treatment of toxocariasis are reviewed...
June 13, 2018: Infectious Diseases of Poverty
https://www.readbyqxmd.com/read/29808150/osteomyelitis-in-cat-scratch-disease-a-never-ending-dilemma-a-case-report-and-literature-review
#18
D Donà, L Nai Fovino, E Mozzo, G Cabrelle, G Bordin, R Lundin, C Giaquinto, T Zangardi, O Rampon
Background: We performed a review of published case studies of osteomyelitis associated with cat-scratch disease to consolidate existing information on clinical presentation, diagnostic tools, therapy, and outcome, as well as presenting a case of disseminated cat-scratch disease in a 12-year-old female with skull osteomyelitis and spleen involvement. Methods: A search for articles indexed in PubMed, Embase, and Google Scholar was performed with the search terms " Bartonella ," "bone," "osteomyelitis," "osteolytic," and "cat-scratch disease" limited to the immunocompetent pediatric population and articles in English...
2018: Case Reports in Pediatrics
https://www.readbyqxmd.com/read/29799351/bartonella-henselae-infection-presenting-as-cervical-spine-osteomyelitis-case-report
#19
Syed Hassan A Akbari, Christine E Averill, Jarod L Roland, Rachel Orscheln, Jennifer Strahle
Bartonella henselae is a gram-negative bacillus implicated in cat-scratch disease. Cat-scratch disease is usually self-limiting and results in local lymphadenopathy. In rare circumstances, patients may develop endocarditis, neuroretinitis, or osteomyelitis. Osteomyelitis of the cervical spine is exceedingly rare, especially in the pediatric population, and to date there have been only 4 previously reported cases of cervical spine osteomyelitis caused by B. henselae, all of which were treated surgically. In this article, the authors report the case of a 7-year-old boy who presented with neck swelling and was found to have a C2-4 paravertebral B...
August 2018: Journal of Neurosurgery. Pediatrics
https://www.readbyqxmd.com/read/29771638/the-first-report-evaluating-the-post-exposure-rabies-prophylaxis-in-children-exposed-to-animals-in-the-lublin-province-eastern-poland-in-2010-2016-a-retrospective-study
#20
Joanna Krzowska-Firych, Ewelina Mazurek, Barbara Hasiec, Krzysztof Tomasiewicz
INTRODUCTION: Rabies continues to be one of the most important viral diseases and remains a significant threat to public health across the globe. The World Health Organization (WHO) has determined that most rabies cases occur in children. Dog bites on humans are a major public-health problem. Poland has not achieved rabies free status yet. Post-exposure prophylaxis (PEP) in humans can effectively prevent death after exposure to a potentially infected animal. THE AIM: The main objective of this study was to estimate the frequency of PEP among children exposed to animals, who had attended consultations at the Department of Infectious Diseases for Children in the Jan Boży Hospital in Lublin...
May 17, 2018: Human Vaccines & Immunotherapeutics
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