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Paediatric Drugs

Oliver Semler, Mirko Rehberg, Nava Mehdiani, Miriam Jackels, Heike Hoyer-Kuhn
Increasing knowledge in the field of rare diseases has led to new therapeutic approaches in the last decade. Treatment strategies have been developed after elucidation of the underlying genetic alterations and pathophysiology of certain diseases (e.g., in osteogenesis imperfecta, achondroplasia, hypophosphatemic rickets, hypophosphatasia and fibrodysplasia ossificans progressiva). Most of the drugs developed are specifically designed agents interacting with the disease-specific cascade of enzymes and proteins involved...
April 3, 2019: Paediatric Drugs
Haya S Raef, Alison M Friedmann, Elena B Hawryluk
Although melanoma is a rare diagnosis in the pediatric population, advances in the management of adults with melanoma offer the prospect of promising therapeutic options for children. At this time, medical management is not considered curative but may reduce the risk of recurrence or prolong survival. Surgical management remains the mainstay of treatment. Medical therapy of pediatric melanoma is not thought to have a role for in situ, early-stage, or localized disease, but adjuvant therapy may have a role in improving the prognosis of patients with positive sentinel lymph node biopsy (SLNB), spread beyond the regional lymph node basin, metastatic disease, or recurrent disease...
March 28, 2019: Paediatric Drugs
Julie C Fitzgerald, Nicole R Zane, Adam S Himebauch, Michael D Reedy, Kevin J Downes, Alexis A Topjian, Susan L Furth, Neal J Thomas, Marc H Scheetz, Athena F Zuppa
BACKGROUND: Acute kidney injury (AKI) commonly occurs after cardiac arrest. Those subsequently treated with vancomycin are at additional risk for drug-induced kidney injury. OBJECTIVE: We aimed to determine whether opportunities exist for improved drug monitoring after cardiac arrest. METHODS: This was a retrospective cohort study of children aged 30 days-17 years treated after cardiac arrest in an intensive care unit from January 2010 to September 2014 who received vancomycin within 24 h of arrest...
March 12, 2019: Paediatric Drugs
Doralina L Anghelescu, Jessica Michala Tesney
Neuropathic pain in pediatric oncology can be caused by distinct lesions or disease processes affecting the somatosensory system, including chemotherapy-related neuronal injury, solid tumor-related involvement of neural structures, post-surgical neuropathic pain-including phantom limb pain and pain after limb-sparing surgery-and the complex circumstances of neuropathic pain at the end of life. Treatment algorithms reflect the general treatment principles applied for adult neuropathic pain, but the dose regimens applied in children are modest and rarely escalated to the maximum doses to optimize analgesic efficacy...
February 14, 2019: Paediatric Drugs
Sudha Kilaru Kessler, Emily McGinnis
Childhood absence epilepsy (CAE) is a common pediatric epilepsy syndrome with distinct seizure semiology, electroencephalography (EEG) features, and treatment. A diagnosis of CAE can be obtained during an office visit with a careful history, physical exam including prolonged hyperventilation, and a routine EEG. The treatment of choice for CAE with absence seizures only is ethosuximide. Valproic acid and lamotrigine are also effective treatments for many patients, but when compared to ethosuximide, valproic acid has more adverse effects and lamotrigine is less effective...
February 8, 2019: Paediatric Drugs
Radna Minou Oostveen, Kathy Pritchard-Jones
Although differences exist in treatment and risk-stratification strategies for children with Wilms tumor (WT) between the European [International Society of Paediatric Oncology (SIOP)] and American [Children's Oncology Group (COG)] study groups, outcomes are very similar, with an overall survival of > 85%. Future strategies aim to de-intensify treatment and reduce toxicity for children with a low risk of relapse and intensify treatment for children with high-risk disease. For metastatic WT, response of lung nodules to chemotherapy is used as a marker to modify treatment intensity...
February 2019: Paediatric Drugs
Jose Carlos Flores-González, Ana Estalella-Mendoza, Patricia Rodríguez-Campoy, Mónica Saldaña-Valderas, Alfonso M Lechuga-Sancho
BACKGROUND: Upper gastrointestinal endoscopies (UGEs) performed under ketamine sedation may increase the risk of respiratory adverse events (RAEs) due to pharyngeal stimulation. Topical lidocaine prevents general anesthesia-induced laryngospasm. OBJECTIVE: Our objective was to determine whether topical lidocaine may reduce the incidence of RAEs induced by pharyngeal stimulation in UGEs performed on children sedated with ketamine. METHODS: We conducted a single-center prospective study...
February 2019: Paediatric Drugs
Sanford Singer, Jamie Koenekoop, Jonathan Meddings, Julie Powell, Anne Desroches, Ernest G Seidman
BACKGROUND: Atopic dermatitis (AD) is a chronic inflammatory skin disease that affects both patients and their families. Current therapies often alleviate symptoms but do not prevent or eradicate the disease. OBJECTIVES: Our objective was to determine whether pancreatic enzyme supplementation is an effective and safe treatment in refractory pediatric AD associated with food allergies. METHODS: We conducted an open-label pilot study using a case-control design...
December 17, 2018: Paediatric Drugs
Jordan T Jones, Nasreen Talib, Daniel Lovell, Mara L Becker
BACKGROUND: Arthropathy of Down syndrome (DA) is largely under-recognized, with an average 2-year delay in diagnosis. Most patients present with polyarthritis, and treatment has historically been challenging. OBJECTIVES: Our objective was to investigate the clinical features and treatment of DA in the largest cohort reported to date. METHODS: In a retrospective chart review at two tertiary care hospitals, International Classification of Diseases, ninth revision, clinical modification (ICD-9-CM) codes for Down syndrome (DS) and juvenile idiopathic arthritis (JIA), between 1 January 1995 and 31 December 2015, were identified and charts reviewed...
December 13, 2018: Paediatric Drugs
Emma H McCafferty, Sohita Dhillon, Emma D Deeks
Chronic myeloid leukemia (CML) is a rare hematopoietic stem cell disease that is typically characterized by the abnormal BCR-ABL1 fusion gene on the Philadelphia (Ph) chromosome in neoplastic cells. Dasatinib (Sprycel® ) is an orally administered, small molecule tyrosine kinase inhibitor indicated for the treatment of certain hematological malignancies, including Ph-positive CML in the chronic phase (Ph+ CML-CP) in adult and pediatric patients. In open-label phase 1 and phase 2 clinical trials, dasatinib produced early and durable target responses (i...
December 2018: Paediatric Drugs
Masato Takeuchi, Shuichi Ito, Masaki Nakamura, Koji Kawakami
BACKGROUND: We sought to quantify the degree of anemia after high-dose intravenous immunoglobulin (IVIG) therapy in patients with Kawasaki disease (KD) by assessing hemoglobin (Hb) dynamics and determining the risk of transfusion. METHODS: We analyzed data from a database containing inpatient data collected from 230 hospitals in Japan. In addition to administrative records, this database included laboratory results for some patients. We searched for individuals aged ≤ 18 years with a diagnosis of KD (International Statistical Classification of Diseases and Related Health Problems, 10th revision, code M30...
December 2018: Paediatric Drugs
R Dobra, C Edmondson, D Hughes, I Martin, J C Davies
Cystic fibrosis is the most common inherited condition in the Caucasian population and is associated with significantly reduced life expectancy. Recent advances in treatment have focussed on addressing the underlying cause of the condition, the defective production, expression and function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Several drugs with different modes of action have produced promising results in clinical trials, and some have been incorporated into routine clinical care for specific patients in many countries worldwide...
December 2018: Paediatric Drugs
Steven L Ciciora, Frederick W Woodley
Gastroesophageal reflux (GER) is the retrograde movement of gastric (and sometimes duodenal) contents into the esophagus. While the majority of GER is physiologic, for patients, it can be associated with symptoms. While some symptoms are merely bothersome (crying), others can be life threatening (cough, gagging, choking). The main driver of GER in infants is the frequent feedings that produce increased intra-abdominal pressure, which is known to trigger transient relaxations of the lower esophageal sphincter...
December 2018: Paediatric Drugs
Julie Park, Urmi Das, Mohammed Didi, Renuka Ramakrishnan, Matthew Peak, Paul Newland, Joanne Blair
BACKGROUND: Hydrocortisone is the preferred treatment for adrenal insufficiency in childhood. A small minority of children experience low cortisol concentrations and symptoms of cortisol insufficiency, poorly responsive to modifications in dosing. We speculated that treatment with modified-release hydrocortisone Plenadren® may be beneficial in these selected patients. OBJECTIVE: The aim of this article was to report cortisol profiles during treatment with standard formulation hydrocortisone and Plenadren, and growth and weight gain during treatment with Plenadren in selected children with adrenal insufficiency...
December 2018: Paediatric Drugs
Kazeem A Oshikoya, Gerold T Wharton, Debbie Avant, Sara L Van Driest, Norman E Fenn, Allison Lardieri, Edwin Doe, Beena G Sood, Carol Taketomo, Phuong Lieu, Lilly Yen, Ann W McMahon
OBJECTIVES: Half of prescription drugs commonly given to children lack product labeling on pediatric safety, efficacy, and dosing. Two drugs most widely used off-label in pediatrics are azithromycin and fentanyl. We sought to determine the risk of serious adverse events (SAEs) when oral azithromycin or intravenous/intramuscular fentanyl are used off-label compared to on-label in pediatric intensive care units (ICUs). STUDY DESIGN: Six pediatric hospitals participated in a retrospective chart review of patients administered oral azithromycin (n = 241) or intravenous/intramuscular fentanyl (n = 367) between January 5, 2013 and December 26, 2014...
November 28, 2018: Paediatric Drugs
Stuart M MacLeod
No abstract text is available yet for this article.
October 2018: Paediatric Drugs
Rolf C R Ljung
Regular prophylactic treatment with factor VIII (FVIII) and factor IX (FIX) concentrates in hemophilia A and B, respectively, is introduced in early infancy and has resulted in dramatic improvement of the conditions. Recombinant FVIII and FIX concentrates have been available for > 25 years and have been modified and refined through the years; however, unfortunately frequent intravenous administrations are still necessary. The half-lives of these products have now been extended (EHL) by fusion with albumin, the Fc-portion of IgG, or by being PEGylated...
October 2018: Paediatric Drugs
Valentina Shakhnovich, P Brian Smith, Jeffrey T Guptill, Laura P James, David N Collier, Huali Wu, Chad E Livingston, Jian Zhao, Gregory L Kearns, Michael Cohen-Wolkowiez
BACKGROUND AND AIMS: Pharmacokinetic data for proton pump inhibitors (PPIs), acid-suppression drugs commonly prescribed to children, are lacking for obese children who are at greatest risk for acid-related disease. In a recent multi-center investigation, we demonstrated decreased, total body weight adjusted, apparent clearance (CL/F) of the PPI pantoprazole for obese children compared with their non-obese peers. Subsequently, we developed a population-based pharmacokinetic (PopPK) model to characterize pantoprazole disposition and evaluated appropriate pantoprazole dosing strategies for obese pediatric patients, using simulation...
October 2018: Paediatric Drugs
Ozkan Ilhan, Meltem Bor, Senem Alkan Ozdemir, Sinem Akbay, Esra Arun Ozer
BACKGROUND: Given the severity and high mortality of multidrug-resistant Gram-negative bacilli (MDR-GNB) infections, the use of colistin will increase in patients with MDR-GNB infection. OBJECTIVE: This study aims to assess the efficacy and safety of intravenous colistin in very low birth weight (VLBW; birth weight < 1500 g) preterm infants. METHODS: We retrospectively analyzed the medical records of patients who received colistin between June 2016 and December 2017...
October 2018: Paediatric Drugs
Catherine Habashy, Erin Springer, Elizabeth A Hall, Doralina L Anghelescu
Methadone is a synthetic opioid with unique pharmacodynamic and pharmacokinetic properties. It is effective in treating both nociceptive and neuropathic pain, which commonly co-exist in children with cancer. Upon reviewing the literature describing the use of methadone in pediatric oncology patients, publications are limited in number and low in quality of evidence; nevertheless, there is support for the safety and efficacy of methadone in treating pain in children with cancer, particularly when pain is refractory to conventional treatment...
October 2018: Paediatric Drugs
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