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Pediatric Drug Development

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https://www.readbyqxmd.com/read/28540765/an-update-on-pediatric-bronchiectasis
#1
Danielle F Wurzel, Anne B Chang
The prevalence and awareness of bronchiectasis not related to cystic fibrosis (CF) is increasing and it is now recognized as a major cause of respiratory morbidity, mortality and healthcare utilization worldwide. The need to elucidate the early origins of bronchiectasis is increasingly appreciated and has been identified as an important research priority. Current treatments for pediatric bronchiectasis are limited to antimicrobials, airway clearance techniques and vaccination. Several new drugs targeting airway inflammation are currently in development...
May 25, 2017: Expert Review of Respiratory Medicine
https://www.readbyqxmd.com/read/28539444/improved-prefusion-stability-optimized-codon-usage-and-augmented-virion-packaging-enhance-the-immunogenicity-of-respiratory-syncytial-virus-rsv-fusion-protein-in-a-vectored-vaccine-candidate
#2
Bo Liang, Joan O Ngwuta, Sonja Surman, Barbora Kabatova, Xiang Liu, Matthias Lingemann, Xueqiao Liu, Lijuan Yang, Richard Herbert, Joanna Swerczek, Man Chen, Syed M Moin, Azad Kumar, Jason S McLellan, Peter D Kwong, Barney S Graham, Peter L Collins, Shirin Munir
Respiratory syncytial virus (RSV) is the most important viral agent of severe pediatric respiratory tract disease worldwide, but lacks a licensed vaccine or suitable antiviral drug. A live-attenuated chimeric bovine/human parainfluenza virus type-3 (rB/HPIV3) was developed previously as a vector expressing RSV fusion (F) protein to confer bivalent protection against RSV and HPIV3. In a previous clinical trial in virus-naïve children, rB/HPIV3 was well-tolerated but the immunogenicity of wildtype RSV F was unsatisfactory...
May 24, 2017: Journal of Virology
https://www.readbyqxmd.com/read/28532715/caretakers-barriers-to-pediatric-antiretroviral-therapy-adherence-in-vietnam-a-qualitative-and-quantitative-study
#3
C T Tran, T H Pham, K T Tran, T K C Nguyen, M Larsson
BACKGROUND: Poor antiretroviral therapy (ART) adherence leads to drug resistance and treatment failures. The options for second and third line ART regimens, particularly for pediatric patients, are very limited in low and middle-income countries. HIV-infected children are mostly passive drug-takers, thus caretakers play a very important role in assuring ART adherence. Pediatric ART adherence is still a challenging problem in Vietnam since non-adherence is the major risk factor for treatment failure...
June 2017: Applied Nursing Research: ANR
https://www.readbyqxmd.com/read/28528870/recurrent-multifocal-osteomyelitis-in-children-experience-in-a-tertiary-care-center
#4
Ana Belén Ariza Jiménez, Esmeralda Núñez Cuadros, Rocío Galindo Zavala, Leticia Núñez Caro, Gisela Díaz-Cordobés Soriano, Antonio Urda Cardona
INTRODUCTION: Chronic recurrent multifocal osteomyelitis is a rare aseptic bone inflammation that affects pediatric patients. Its management and treatment have not yet been standardized. METHODS: Retrospective, descriptive study of patients under 14 years of age diagnosed with chronic nonbacterial osteomyelitis (CNBO) in a tertiary hospital. We included patients diagnosed over the last 6 years (2010-2015) who met the Jansson criteria. The clinical and radiological characteristics of CNBO were analyzed, as was the outcome after different therapeutic options...
May 18, 2017: Reumatología Clinica
https://www.readbyqxmd.com/read/28526601/model-based-precision-dosing-of-sirolimus-in-pediatric-patients-with-vascular-anomalies
#5
Tomoyuki Mizuno, Chie Emoto, Tsuyoshi Fukuda, Adrienne M Hammill, Denise M Adams, Alexander A Vinks
Sirolimus is the first drug to show efficacy in the treatment of patients with complicated vascular anomalies. The current study expands on the evolution of a PK model-based strategy for the precision dosing of sirolimus as part of prospective concentration controlled clinical trials in pediatric patients with vascular anomalies. Twelve month follow up data collected from 52 pediatric patients participating in the Phase 2 clinical trial were analyzed. Target attainment across the age range of 3weeks to 18years after 2-3months of therapy was 94% (49 out of 52 patients)...
May 16, 2017: European Journal of Pharmaceutical Sciences
https://www.readbyqxmd.com/read/28509784/pharmacotherapy-options-for-pediatric-diabetes
#6
Maria Loredana Marcovecchio, Francesco Chiarelli
PURPOSE OF REVIEW: Type 1 diabetes (T1D) and type 2 diabetes (T2D) are frequent conditions during childhood and adolescence. The present review offers an update on current available treatment strategies for T1D and T2D approved for use in children and adolescents. RECENT FINDINGS: Insulin remains the main and essential therapeutic strategy in youth with T1D. A second generation of insulin analogues is being evaluated and could help in improving glycemic control...
May 13, 2017: Current Opinion in Pediatrics
https://www.readbyqxmd.com/read/28506869/model-based-clinical-dose-optimization-for-phenobarbital-in-neonates-an-illustration-of-the-importance-of-data-sharing-and-external-validation
#7
Swantje Völler, Robert B Flint, Leo M Stolk, Pieter L J Degraeuwe, Sinno H P Simons, Paula Pokorna, David M Burger, Ronald de Groot, Dick Tibboel, Catherijne A J Knibbe
BACKGROUND: Particularly in the pediatric clinical pharmacology field, data-sharing offers the possibility of making the most of all available data. In this study, we utilize previously collected therapeutic drug monitoring (TDM) data of term and preterm newborns to develop a population pharmacokinetic model for phenobarbital. We externally validate the model using prospective phenobarbital data from an ongoing pharmacokinetic study in preterm neonates. METHODS: TDM data from 53 neonates (gestational age (GA): 37 (24-42) weeks, bodyweight: 2...
May 12, 2017: European Journal of Pharmaceutical Sciences
https://www.readbyqxmd.com/read/28506690/romidepsin-induces-caspase-dependent-cell-death-in-human-neuroblastoma-cells
#8
Shane V Hegarty, Katie L Togher, Eimear O'Leary, Franziska Solger, Aideen M Sullivan, Gerard W O'Keeffe
Neuroblastoma is the most common extracranial pediatric solid tumor, arising from the embryonic sympathoadrenal lineage of the neural crest, and is responsible for 15% of childhood cancer deaths. Although survival rates are good for some patients, those children diagnosed with high-risk neuroblastoma have survival rates as low as 35%. Thus, neuroblastoma remains a significant clinical challenge and the development of novel therapeutic strategies is essential. Given that there is widespread epigenetic dysregulation in neuroblastoma, epigenetic pharmacotherapy holds promise as a therapeutic approach...
May 12, 2017: Neuroscience Letters
https://www.readbyqxmd.com/read/28499879/developing-a-flexible-pediatric-dosage-form-for-antiretroviral-therapy-a-fast-dissolving-tablet
#9
Manjari Lal, Manshun Lai, Marcus Estrada, Changcheng Zhu
Current presentations of the anti-HIV drugs lopinavir and ritonavir make appropriate dosing for children difficult. We conducted a feasibility study to develop a formulation for these drugs with child-safe excipients in a flexible dosage form for children across the pediatric age spectrum. The freeze-drying-in-blister approach was used to produce fast-dissolving tablets (FDTs), as these can be dispersed in fluids for easy administration, even to infants, and appropriate portions of the dispersion can be given for different ages/weights...
May 9, 2017: Journal of Pharmaceutical Sciences
https://www.readbyqxmd.com/read/28499018/intracystic-interferon-alpha-in-pediatric-craniopharyngioma-patients-an-international-multicenter-assessment-on-behalf-of-siope-and-ispn
#10
John-Paul Kilday, Massimo Caldarelli, Luca Massimi, Robert Hsin-Hung Chen, Yi Yen Lee, Muh-Lii Liang, Jeanette Parkes, Thuran Naiker, Marie-Lise van Veelen, Erna Michiels, Conor Mallucci, Benedetta Pettorini, Lisethe Meijer, Christian Dorfer, Thomas Czech, Manuel Diezi, Antoinette Yn Schouten-van Meeteren, Stefan Holm, Bengt Gustavsson, Martin Benesch, Hermann L Müller, Anika Hoffmann, Stefan Rutkowski, Joerg Flitsch, Gabriele Escherich, Michael Grotzer, Helen A Spoudeas, Kristian Azquikina, Michael Capra, Rolando Jiménez-Guerra, Patrick MacDonald, Donna L Johnston, Rina Dvir, Shlomi Constantini, Meng-Fai Kuo, Shih-Hung Yang, Ute Bartels
Background: Craniopharyngiomas are frequent hypothalamo-pituitary tumors in children, presenting predominantly as cystic lesions. Morbidity from conventional treatment has focused attention on intracystic drug delivery, hypothesized to cause fewer clinical consequences. However, the efficacy of intracystic therapy remains unclear. We report the retrospective experiences of several global centers using intracystic interferon-alpha. Methods: European Société Internationale d'Oncologie Pédiatrique and International Society for Pediatric Neurosurgery centers were contacted to submit a datasheet capturing pediatric patients with cystic craniopharyngiomas who had received intracystic interferon-alpha...
May 11, 2017: Neuro-oncology
https://www.readbyqxmd.com/read/28498004/a-sequential-implementation-model-for-workforce-development-a-case-study-of-medical-residency-training-for-substance-use-concerns
#11
Patricia S O'Sullivan, Patrick Yuan, Derek D Satre, Maria Wamsley, Jason Satterfield
PROBLEM: In graduate medical education, residency programs are often educationally isolated from each other, with varying needs and patient populations, so strategies are needed when attempting to implement training in evidence-based practices across multiple residencies. INTERVENTION: Using implementation science as a guide, we adapted a community development model to sequentially implement an evidence-based intervention, Screening, Brief Intervention, and Referral to Treatment (SBIRT) for alcohol and drug use problems, across internal medicine, pediatrics, emergency medicine, psychiatry, and obstetrics and gynecology residency programs...
May 12, 2017: Teaching and Learning in Medicine
https://www.readbyqxmd.com/read/28497658/toxic-epidermal-necrolysis-in-a-child-6-months-post-hematopoietic-stem-cell-transplantation
#12
Utako Oba, Hiroshi Yamada, So-Ichi Suenobu, Yusuke Nakamura, Akiko Ito, Yutaka Hatano, Nobuyoshi Itonaga, Kouichi Ohshima, Yuhki Koga, Shouichi Ohga, Kenji Ihara
TEN is a rare and critical disease mostly caused by drugs. It is mediated by activated CD8+ T cells that cause keratinocyte apoptosis with the assistance of cytokines/chemokines. We herein report a pediatric case of TEN after allogeneic HSCT with precursor B-cell acute lymphoblastic leukemia (pre-B-ALL) in second complete remission. Although we did not evaluate the T-cell subpopulation in blood or skin lesion of the patient, an imbalanced immune reconstitution after HSCT might additively contribute to the development of TEN...
May 12, 2017: Pediatric Transplantation
https://www.readbyqxmd.com/read/28495415/rhinovirus-from-bench-to-bedside
#13
REVIEW
Kelvin K W To, Cyril C Y Yip, Kwok-Yung Yuen
Rhinovirus has been neglected in the past because it was generally perceived as a respiratory virus only capable of causing mild common cold. Contemporary epidemiological studies using molecular assays have shown that rhinovirus is frequently detected in adult and pediatric patients with upper or lower respiratory tract infections. Severe pulmonary and extrapulmonary complications are increasingly recognized. Contrary to popular belief, some rhinoviruses can actually replicate well at 37 °C and infect the lower airway in humans...
May 8, 2017: Journal of the Formosan Medical Association, Taiwan Yi Zhi
https://www.readbyqxmd.com/read/28494052/association-of-minimal-residual-disease-with-clinical-outcome-in-pediatric-and-adult-acute-lymphoblastic-leukemia-a-meta-analysis
#14
Donald A Berry, Shouhao Zhou, Howard Higley, Lata Mukundan, Shuangshuang Fu, Gregory H Reaman, Brent L Wood, Gary J Kelloff, J Milburn Jessup, Jerald P Radich
Importance: Minimal residual disease (MRD) refers to the presence of disease in cases deemed to be in complete remission by conventional pathologic analysis. Assessing the association of MRD status following induction therapy in patients with acute lymphoblastic leukemia (ALL) with relapse and mortality may improve the efficiency of clinical trials and accelerate drug development. Objective: To quantify the relationships between event-free survival (EFS) and overall survival (OS) with MRD status in pediatric and adult ALL using publications of clinical trials and other databases...
May 11, 2017: JAMA Oncology
https://www.readbyqxmd.com/read/28490206/pharmacokinetics-pharmacodynamics-and-pharmacogenetics-associated-with-nonsteroidal-anti-inflammatory-drugs-and-opioids-in-pediatric-cancer-patients
#15
Jonathan Constance, Sarah Campbell, Amit A Somani, Venkata Yellepeddi, Katie Owens, Catherine Sherwin
Advancing appropriate and adequate analgesic pharmacotherapy in pediatric patients with cancer is an area of clinical need. Few studies have been performed to evaluate the selection of an analgesic and appropriate dosing corresponding to analgesic effect among pediatric cancer patients. This review describes information related to pharmacokinetic, pharmacodynamic, and pharmacogenomic (when applicable) considerations for analgesics that are commonly used to manage pain experienced by pediatric patients with cancer...
May 11, 2017: Expert Opinion on Drug Metabolism & Toxicology
https://www.readbyqxmd.com/read/28484171/current-diagnosis-and-treatment-for-pediatric-acute-myeloid-leukemia
#16
Norio Shiba
Acute myeloid leukemia (AML) is a complex disease caused by chromosomal aberrations, mutations, epigenetic modifications, and the deregulated expression of genes, leading to increased myeloid cell proliferation and decreased hematopoietic progenitor cell differentiation. Although most of these aberrations are correlated with prognosis, accurate risk stratification remains a challenge even after incorporating these molecular markers. Currently, some genetic mutations that allow risk stratification have been identified in adult AML, including DNMT3A and IDH1/2...
2017: [Rinshō Ketsueki] the Japanese Journal of Clinical Hematology
https://www.readbyqxmd.com/read/28476522/preliminary-study-of-neurodevelopmental-outcomes-and-parenting-stress-in-pediatric-mitochondrial-disease
#17
Soyong Eom, Young-Mock Lee
BACKGROUND: Little is known regarding the neuropsychological profiles of pediatric patients with mitochondrial diseases or their parents, information that is crucial for improving the quality of life (QOL) for both patients and parents. We aimed to delineate neurodevelopment and psychological comorbidity in children with mitochondrial diseases in the preliminary investigation of adequate intervention methods, better prognoses, and improved QOL for both patients and parents. METHODS: Seventy children diagnosed with mitochondrial diseases were neuropsychologically evaluated...
February 2, 2017: Pediatric Neurology
https://www.readbyqxmd.com/read/28471280/bad-trip-due-to-25i-nbome-a-case-report-from-the-eu-project-spice-ii-plus
#18
Maren Hermanns-Clausen, Verena Angerer, Josephine Kithinji, Christina Grumann, Volker Auwärter
OBJECTIVE: The potent hallucinogenic drug 25I-NBOMe has recently emerged on the drug market. We present a case with analytically confirmed 25I-NBOMe intoxication from the prospective study "SPICE II Plus". CASE REPORT: Because of a severe headache a 42-year-old man took one sip of a pediatric analgesic syrup, which had been refilled with a self-made solution of 25I-NBOMe in ethanol. Thirty minutes later restlessness occurred. On arrival in the emergency department mydriasis, strong sweating, disorientation, and agitation were noticed...
May 4, 2017: Clinical Toxicology
https://www.readbyqxmd.com/read/28468529/pharmacogenomics-in-pediatric-acute-lymphoblastic-leukemia-promises-and-limitations
#19
Zeina N Al-Mahayri, George P Patrinos, Bassam R Ali
Despite the significant advances achieved in pediatric acute lymphocytic leukemia (ALL) treatment, adverse side effects of drugs remain a challenging issue. Numerous ALL pharmacogenomic studies have been conducted to elucidate the predisposing genetic factors for their development. Plausible pharmacogenomic data are available for the osteonecrosis associated with glucocorticoids, the neurotoxicity associated with vincristine and the cardiotoxicity related to anthracyclines. However, these data have not been fully translated into the clinic due to several limitations, most importantly the lack of reliable evidence...
May 4, 2017: Pharmacogenomics
https://www.readbyqxmd.com/read/28466670/points-to-consider-in-designing-and-conducting-juvenile-toxicology-studies
#20
Norman N Kim, Robert M Parker, Gerhard F Weinbauer, Amera K Remick, Thomas Steinbach
In support of a clinical trial in the pediatric population, available nonclinical and clinical data provide input on the study design and safety monitoring considerations. When the existing data are lacking to support the safety of the planned pediatric clinical trial, a juvenile animal toxicity study is likely required. Usually a single relevant species, preferably a rodent, is chosen as the species of choice, while a nonrodent species can be appropriate when scientifically justified. Juvenile toxicology studies, in general, are complicated both conceptually and logistically...
January 1, 2017: International Journal of Toxicology
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