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((Myelodysplastic Syndrome) OR (MD)) AND Pedia*

Giulia M Benedetti Md, Faye S Silverstein Md
Targeted temperature management encompasses a range of clinical interventions to regulate systemic temperature, and includes both induction of varying degrees of hypothermia and fever prevention ("targeted normothermia"). Targeted temperature management plays a key role in the contemporary management of critically ill neonates and children with acute brain injury. Yet, many unanswered questions remain regarding optimal temperature management in pediatric neurocritical care. The introduction highlights experimental studies that have evaluated the neuroprotective efficacy of therapeutic hypothermia and explored possible mechanisms of action in several brain injury models...
July 21, 2018: Pediatric Neurology
Kenichi Yoshida
Recent research reveals novel insights into the pathogenesis of childhood myelodysplastic syndromes (MDS) in addition to that of juvenile myelomonocytic leukemia (JMML). In pediatric MDS, the genetic characteristics of which have been barely elucidated previously, germline mutations, particularly those in GATA2, SAMD9, and SAML9L, have been frequently identified, indicating the importance of germline predisposition in childhood MDS compared with adult MDS. In JMML, in addition to the known Ras-pathway mutations, novel secondary mutations and causative fusion genes have been reported...
2018: [Rinshō Ketsueki] the Japanese Journal of Clinical Hematology
Erin E Doherty, Erica K Schallert, Alexandra M Stevens, Jyotinder N Punia, Jennifer H Foster
We report the case of a 3-year-old girl diagnosed with acute megakaryoblastic leukemia, who presented after >1 year of bilateral leg pain. At times the pain was severe enough to prevent ambulation, prompting visits to her primary care provider. However, it was not until acute respiratory failure occurred with subsequent hospitalization in the pediatric intensive care unit that severe anemia and thrombocytopenia were discovered and the diagnosis of acute myeloid leukemia was made. Bilateral lower extremity swelling was noted on admission and radiographs showed diffusely abnormal appearance of the long bones of her lower extremities with periosteal reaction and echogenic debris in the subperiosteal space, thought to represent leukemic cells...
October 9, 2018: Journal of Pediatric Hematology/oncology
Esther Mueller-Godeffroy, Reinhard Vonthein, Carmen Ludwig-Seibold, Bettina Heidtmann, Claudia Boettcher, Miriam Kramer, Nicole Hessler, Doerte Hilgard, Eggert Lilienthal, Andreas Ziegler, Verena M Wagner
OBJECTIVE: Continuous subcutaneous insulin infusion (CSII) is on the rise among pediatric patients with type 1 diabetes mellitus. Metabolic effects alone cannot explain this rising popularity. From the patient's perspective, the main benefits of CSII may be found in subjective psychosocial health outcomes (patient-reported outcomes [PRO]). SUBJECTS AND METHODS: In a multicenter open randomized controlled trial, children and adolescents aged 6 to16 years currently treated with multiple daily injections (MDI) were randomized 1:1, stratified by center, to either starting with CSII immediately after the baseline interview or to continuing MDI while waiting 6 months for transmission to CSII...
October 10, 2018: Pediatric Diabetes
Eliana B Saltzman, Schneider K Rancy, Darryl B Sneag, Joseph H Feinberg Md, Dale J Lange, Scott W Wolfe
BACKGROUND: Acute flaccid myelitis is associated with enterovirus D68 induced inflammation and destruction of cervical anterior horn cells. To date, no medical intervention has altered the disease course. METHODS: We report two pediatric patients who were treated with nerve transfer in three limbs with sustained upper extremity neuropathy. Postoperative outcomes included muscle strength, graded on the British Medical Research Council (BMRC) scale, range of motion, and electromyography...
August 20, 2018: Pediatric Neurology
Xiaoxi Liu, Anne Smits, Yuhuan Wang, Marleen Renard, Stephanie Wead, Richard J Kagan, Daniel P Healy, Pieter De Cock, Karel Allegaert, Catherine Sherwin
BACKGROUND: Amikacin is widely used to treat severe Gram-negative bacterial infections. Its peak concentration in plasma is associated with treatment efficacy. Amikacin pharmacokinetics (PK) is influenced by disease conditions, in addition to other patient characteristics. In this retrospective study, we evaluated the impact of clinical characteristics and disease condition on amikacin PK in children with burn injuries and those with cancer. METHODS: Amikacin PK data from 66 children with burn injuries and 112 children with cancer were analyzed...
October 3, 2018: Therapeutic Drug Monitoring
Sameer K Singh, David Gu, Robson Capasso, Stanley Liu, Christopher J Gouveia
OBJECTIVES/HYPOTHESIS: Examine US and international clinical trials in obstructive sleep apnea (OSA) to characterize researchers involved, interventions being studied, and opportunities for future investigation. STUDY DESIGN: Retrospective database review. METHODS: The information from was used to assess OSA clinical trials between 1999 and 2017. Information was gathered on principle investigator (PI) demographics, interventions studied, study funding source, and regional distribution of research institutions...
October 3, 2018: Laryngoscope
Ying Wang, Yongsheng Zhang, Wenting Lu, Liying Wang
Background: Mycoplasma pneumoniae pneumonia (MPP) is one of the most common forms of community-acquired pneumonia in children. The objective of this study was to explore potential changes in levels of serum tumor necrosis factor- α (TNF- α ) and interferon- γ (IFN- γ ) associated with pediatric MPP. Methods: This protocol has been registered (PROSPERO 2017: CRD42017077979). A literature search was performed in October 2017 using PubMed, Embase, the Cochrane Library, and other Chinese medical databases to identify studies...
2018: Canadian Respiratory Journal: Journal of the Canadian Thoracic Society
Kayla Md Cornett, Elizabeth Wojciechowski, Amy D Sman, Terri Walker, Manoj P Menezes, Paula Bray, Mark Halaki, Joshua Burns
INTRODUCTION: Biomarkers of disease severity in Charcot-Marie-Tooth disease (CMT) are required to evaluate early responses to treatment. The study aimed to evaluate the relationship between muscle volume and intramuscular fat accumulation by magnetic resonance imaging (MRI) and weakness, disability and impaired gait in affected children and adolescents. METHODS: 55 participants underwent MRI of the anterior compartment of the lower leg. Muscle and fat volumes were calculated...
September 28, 2018: Muscle & Nerve
Molly M Lamb, Bo Cai, Julie Royer, Shree Pandya, Aida Soim, Rodolfo Valdez, Carolyn DiGuiseppi, Katherine James, Nedra Whitehead, Holly Peay, Swamy Y Venkatesh, Dennis Matthews
To describe the long-term effect of steroid treatment on weight in nonambulatory males with Duchenne Muscular Dystrophy (DMD), we identified 392 males age 7-29 years with 4,512 weights collected after ambulation loss (176 steroid-naïve and 216 treated with steroids ≥6 months) from the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet). Comparisons were made between the weight growth curves for steroid-naïve males with DMD, steroid-treated males with DMD, and the US pediatric male population...
September 26, 2018: American Journal of Medical Genetics. Part A
Li Jiang, Wei Sun, Kai Zhang, Bin Zhou, Xiangqing Kong
Sildenafil is a pulmonary artery hypertension (PH)-targeted drug that finds an increased indiscriminate use in children with PH secondary to congenital heart disease (CHD).We performed a meta-analysis to evaluate the effects of sildenafil on pediatric patients with PH secondary to CHD during perioperative period.PubMed, EMBASE, the Cochrane Library, and the Google Scholar were searched up to May 2016 for randomized controlled trials (RCTs) assessing the perioperative treatment of sildenafil in pediatric patients with PH secondary to CHD...
September 25, 2018: International Heart Journal
Eleonora Napoli, Andrea Schneider, Randi Hagerman, Gyu Song, Sarah Wong, Flora Tassone, Cecilia Giulivi
Mitochondrial dysfunction (MD) has been identified in lymphocytes, fibroblasts and brain samples from adults carrying a 55-200 CGG expansion in the fragile X mental retardation 1 ( FMR1 ) gene (premutation; PM); however, limited data are available on the bioenergetics of pediatric carriers. Here we discuss a case report of three PM carriers: two monozygotic twins (aged 8 years) harboring an FMR1 allele with 150-180 CGG repeats, with no cognitive or intellectual issues but diagnosed with depression, mood instability and ADHD, and their mother (asymptomatic carrier with 78 CGG repeats)...
2018: Frontiers in Genetics
Avrey Thau, Maureen Lloyd, Sharon Freedman, Allen Beck, Alana Grajewski, Alex V Levin
PURPOSE OF REVIEW: The Childhood Glaucoma Research Network (CGRN) has created a new classification system for childhood glaucoma that has become the first International Consensus Classification. The purpose of this review is to present this classification system and share its use to date. RECENT FINDINGS: The diagnoses of the classification system include glaucoma and glaucoma suspect. The primary glaucomas include: primary congenital glaucoma and juvenile open-angle glaucoma...
September 2018: Current Opinion in Ophthalmology
James Reinecke, Stefanie Lowas, Jessica Snowden, Kari Neemann
BACKGROUND: Frequent surveillance of bacterial pathogens responsible for microbiologically defined-blood stream infections (MD-BSI), and their respective antibiotic susceptibilities is central to tailoring empiric antibiotic therapy in febrile neutropenia (FN) episodes in pediatric patients with leukemia. The safety of deescalating antibiotic therapy in pediatric patients with leukemia and neutropenia is incompletely understood. METHODS: A retrospective chart review of 194 FN episodes occurred between the years of 2013 and 2016 in 67 patients with leukemia...
August 8, 2018: Journal of Pediatric Hematology/oncology
Gil Gutvirtz, Tamar Wainstock, Eyal Sheiner, Daniella Landau, Alon Slutzky, Asnat Walfisch
Children born at early term (37 0/7 to 38 6/7 weeks' gestation) are at an increased risk for long-term respiratory, cardiovascular, neurological, and developmental morbidities as compared with children born at full term (39 0/7 to 40 6/7 weeks' gestation). In this population-based cohort analysis, we sought to evaluate the long-term hematological morbidity of early-term born children. The cohort consisted of 223,242 term singleton deliveries. Hospitalizations of the offspring up to 18 years of age involving hematological morbidity were evaluated, including hereditary and acquired anemias, immunodeficiency disorders, coagulation disorders, white blood cell disorders, cytopenias, polycythemia, and myelodysplastic syndrome...
August 7, 2018: European Journal of Pediatrics
Kris M Mahadeo, Sajad J Khazal, Hisham Abdel-Azim, Julie C Fitzgerald, Agne Taraseviciute, Catherine M Bollard, Priti Tewari, Christine Duncan, Chani Traube, David McCall, Marie E Steiner, Ira M Cheifetz, Leslie E Lehmann, Rodrigo Mejia, John M Slopis, Rajinder Bajwa, Partow Kebriaei, Paul L Martin, Jerelyn Moffet, Jennifer McArthur, Demetrios Petropoulos, Joan O'Hanlon Curry, Sarah Featherston, Jessica Foglesong, Basirat Shoberu, Alison Gulbis, Maria E Mireles, Lisa Hafemeister, Cathy Nguyen, Neena Kapoor, Katayoun Rezvani, Sattva S Neelapu, Elizabeth J Shpall
In 2017, an autologous chimeric antigen receptor (CAR) T cell therapy indicated for children and young adults with relapsed and/or refractory CD19+ acute lymphoblastic leukaemia became the first gene therapy to be approved in the USA. This innovative form of cellular immunotherapy has been associated with remarkable response rates but is also associated with unique and often severe toxicities, which can lead to rapid cardiorespiratory and/or neurological deterioration. Multidisciplinary medical vigilance and the requisite health-care infrastructure are imperative to ensuring optimal patient outcomes, especially as these therapies transition from research protocols to standard care...
August 6, 2018: Nature Reviews. Clinical Oncology
Pierre Antherieu, R Levy, T De Saint Denis, L Lohkamp, G Paternoster, F Di Rocco, N Boddaert, M Zerah
PURPOSE: Diffusion tensor imaging (DTI) allows studying the micro and macro architecture. One of the major challenges in dysraphism is to know the morphologic organization of the spinal cord. In a preliminary work, spinal lipoma was chosen for analyzing the micro-architecture parameters and fiber morphology of the spinal cord by DTI with tractography. METHODS: Twelve patients (0-8 years) related to spinal lipomas treated between May 2017 and March 2018 were included...
August 2, 2018: Child's Nervous System: ChNS: Official Journal of the International Society for Pediatric Neurosurgery
W David Lohr, Liza Creel, Yana Feygin, Michelle Stevenson, Michael J Smith, John Myers, Charles Woods, Gil Liu, Deborah Winders Davis
BACKGROUND: The rates of mental health diagnoses in children have increased. Children in poverty have the highest rates. The use of psychotropic medication for children has been increasing, which is concerning because of the unknown long-term effects and the increased burden on the health care system. The state of Kentucky ranks among the highest in the United States for children with mental health problems, children living in poverty, and children receiving psychotropic medication. OBJECTIVE: To examine recent trends and determinants of interclass psychotropic polypharmacy (PP) use for children and youth receiving Medicaid to inform intervention development...
August 2018: Journal of Managed Care & Specialty Pharmacy
Marcin W Wlodarski, Sushree S Sahoo, Charlotte M Niemeyer
Myelodysplastic syndromes (MDS) in children and adolescents are a rare heterogeneous group of clonal stem cell disorders. Complete or partial loss of chromosome 7 constitutes the most common cytogenetic abnormality encountered in any type of childhood MDS, is associated with more advanced disease, and usually requires a timely allogeneic stem cell transplantation. This article provides insights into the current understanding of the genotype, phenotype, and clonal evolution patterns in pediatric MDS associated with loss of chromosome 7...
August 2018: Hematology/oncology Clinics of North America
Kelly M Jones, Gina O'Grady, Miriam J Rodrigues, Anna Ranta, Richard H Roxburgh, Donald R Love, Alice Theadom
BACKGROUND: Genetic muscle disorders, including muscular dystrophies, congenital myopathies, and ion channel muscle diseases can be associated with significant disability. OBJECTIVE: This study aimed to explore child and parent perspectives of the impact of living with a genetic muscle disorder. METHODS: Eighty-three children (<16 years) with a clinical or molecular diagnosis were identified as part of a national prevalence study. Parents' experiences and needs were assessed using a study-specific questionnaire...
2018: Journal of Neuromuscular Diseases
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