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pediatric therapeutics

Constanze Hantel, Igor Shapiro, Giada Poli, Costanza Chiapponi, Martin Bidlingmaier, Martin Reincke, Michaela Luconi, Sara Jung, Felix Beuschlein
In recent years it has been recognized that clinical translation of novel therapeutic strategies for patients with adrenocortical carcinoma (ACC) often fails. These disappointing results indicate that the currently utilized tumor models only poorly reflect relevant pathophysiology and, thereby, do not predict clinical applicability of novel pharmacological approaches. However, also the development of new preclinical ACC models has remained a challenge with only one human cell line (NCI-H295R) and one recently established human pediatric xenograft model (SJ-ACC3) being available for this highly heterogeneous malignancy...
October 15, 2016: Oncotarget
Seung Man Cho, Byung-Ho Choe
This review article was requested by editor-in-chief of this journal as 'pediatric CHB treatment' for the upcoming special issue. The main objective of chronic hepatitis B (CHB) treatment is diminishing the risk of complications related to chronic liver disease. In Asia, there are already some reports about hepatocellular carcinoma (HCC) in hepatitis B virus (HBV) infected children. The key points of treatment in children with CHB infections are selection of which patients to treat and conformation of the optimal therapy time that would reduce viral resistance...
September 2016: Annals of Translational Medicine
Osman Yeşilbaş, Hasan Serdar Kıhtır, Hamdi Murat Yıldırım, Nevin Hatipoğlu, Esra Şevketoğlu
BACKGROUND: Leptospirosis is a zoonotic infectious disease caused by pathogenic spirochetes of the genus Leptospira. Although it is usually asymptomatic and self-limited, severe potentially fatal illness accompanied by multi-organ failure may occur. CASE REPORT: Here we report an unusual case of severe leptospirosis successfully treated with continuous venovenous hemofiltration (CVVHF) and therapeutic plasma exchange (TPE). The patient presented with pericardial tamponade, renal failure and macrophage activation syndrome, and later suffered prolonged jaundice and sclerosing cholangitis during hospitalization in the pediatric intensive care unit (PICU)...
September 2016: Balkan Medical Journal
Katherine A Dunn, Jessica Moore-Connors, Brad MacIntyre, Andrew Stadnyk, Nikhil A Thomas, Angela Noble, Gamal Mahdi, Mohsin Rashid, Anthony R Otley, Joseph P Bielawski, Johan Van Limbergen
BACKGROUND: Exclusive enteral nutrition (EEN) is a first-line therapy in pediatric Crohn's disease (CD) thought to induce remission through changes in the gut microbiome. With microbiome assessment largely focused on microbial taxonomy and diversity, it remains unclear to what extent EEN induces functional changes that thereby contribute to its therapeutic effect. METHODS: Fecal samples were collected from 15 pediatric CD patients prior to and after EEN treatment, as well as from 5 healthy controls...
November 2016: Inflammatory Bowel Diseases
Felipe P Andrade, Roberto Montoro, Renan Oliveira, Gabriela Loures, Luana Flessak, Roberta Gross, Camille Donnabella, Andrea Puchnick, Lisa Suzuki, Rodrigo Regacini
OBJECTIVES: 1) To verify clinical signs correlated with appropriate cranial computed tomography scan indications and changes in the therapeutic approach in pediatric minor head trauma scenarios. 2) To estimate the radiation exposure of computed tomography scans with low dose protocols in the context of trauma and the additional associated risk. METHODS: Investigators reviewed the medical records of all children with minor head trauma, which was defined as a Glasgow coma scale ≥13 at the time of admission to the emergency room, who underwent computed tomography scans during the years of 2013 and 2014...
October 1, 2016: Clinics
Jesus Duque-Afonso, Chiou-Hong Lin, Kyuho Han, Michael C Wei, Jue Feng, Jason Kurzer, Corina Schneidawind, Stephen H K Wong, Michael C Bassik, Michael L Cleary
There is limited understanding of how signaling pathways are altered by oncogenic fusion transcription factors that drive leukemogenesis. To address this, we interrogated activated signaling pathways in a comparative analysis of mouse and human leukemias expressing the fusion protein E2A-PBX1, which is present in 5-7% of pediatric and 50% of pre-B-cell receptor (preBCR+) acute lymphocytic leukemia (ALL). In this study, we describe remodeling of signaling networks by E2A-PBX1 in pre-B-ALL which result in hyperactivation of the key oncogenic effector enzyme PLCγ2...
October 7, 2016: Cancer Research
Sumit Mehndiratta
Phenytoin is a commonly used antiepileptic medication in the pediatric age group, but it has a narrow therapeutic range. Various adverse effects have been reported commonly. We report a relatively rare case of encephalopathy in a child from overdose of injectable phenytoin due to ignorance of the previous treatment. Scrutiny of medical records and history is of utmost importance while administering such medications.
July 2016: Indian Journal of Pharmacology
Laura L Bio, Brandon J Patterson, Sanchita Sen, Angela L Bingham, Jane F Bowen, Benjamin Ereshefsky, Laura A Siemianowski
Objective. To identify the temporal effect and factors associated with student pharmacist self-initiation of interventions during acute patient care advanced pharmacy practice experiences (APPE). Methods. During the APPE, student pharmacists at an academic medical center recorded their therapeutic interventions and who initiated the intervention throughout clinical rotations. At the end of the APPE student pharmacists completed a demographic survey. Results. Sixty-two student pharmacists were included. Factors associated with lower rates of self-initiated interventions were infectious diseases and pediatrics APPEs and an intention to pursue a postgraduate residency...
September 25, 2016: American Journal of Pharmaceutical Education
Takaki Emura, Kenji Hosoda, Shota Harai, Noboru Oyachi, Takeyuki Suzuki, Ken Takada, Koji Kobayashi, Hisatake Ikeda
BACKGROUND: Massive gastrointestinal bleeding in children, mostly caused by esophageal varices secondary to chronic liver disease, is uncommon. Dieulafoy lesion in the gastrointestinal tract is a rare but important cause of gastrointestinal bleeding; massive bleeding from this lesion can be fatal unless adequate treatment is promptly initiated. We report a case of gastric Dieulafoy lesion in a 2-year old successfully treated with endoscopic hemoclipping. CASE PRESENTATION: A 2-year-old Japanese boy was admitted to our department with sudden massive hematemesis...
October 19, 2016: Journal of Medical Case Reports
Chih-Cheng Luo, Kuang-Fu Cheng, Chen-Sheng Huang, Hung-Chieh Lo, Sheng-Mao Wu, Hung-Chang Huang, Wen-Kuei Chien, Ray-Jade Chen
BACKGROUND: In this study, we studied the therapeutic effectiveness of percutaneous drainage with antibiotics and the need for an interval appendectomy for treating appendiceal abscess in children with a research-oriented dataset released by the Bureau of National Health Insurance in Taiwan through the Collaboration Center for Health Information Application (CCHIA). METHODS: We identified 1225 patients under 18 years of age who had non-surgical treatment for an appendiceal abscess between 2007 and 2012 in a Taiwan CCHIA dataset...
October 18, 2016: BMC Surgery
Toshinori Sakai, Fumitake Tezuka, Kazuta Yamashita, Yoichiro Takata, Kosaku Higashino, Akihiro Nagamachi, Koichi Sairyo
STUDY DESIGN: A retrospective review of prospectively collected data. OBJECTIVE: To investigate recent outcomes of conservative treatment for bony healing in pediatric patients with lumbar spondylolysis (LS) and to identify the problems that need to be resolved. SUMMARY OF BACKGROUND DATA: Several diagnostic and therapeutic techniques for LS have been developed recently, leading to better outcomes for bony healing. METHODS: Overall, 63 consecutive pediatric patients (53 boys and 10 girls) with LS (average age: 13...
October 17, 2016: Spine
Isabella Guzzo, Federica Morolli, Francesca Diomedi Camassei, Antonina Piazza, Elvira Poggi, Luca Dello Strologo
BACKGROUND: Several cases of severe antibody-mediated rejection (AMR) secondary to antibodies against the angiotensin II type 1 receptor (AT1R-Ab) have been described with variable outcome. CASE-DIAGNOSIS/TREATMENT: We report the case of a 13-year-old boy whose first kidney transplant failed due to steroid-resistant acute cellular rejection, with the subsequent development of sensitization. He received a second kidney transplant which was complicated by early humoral rejection, with weakly positive staining for the complement degradation product C4d...
October 17, 2016: Pediatric Nephrology: Journal of the International Pediatric Nephrology Association
Linda Aurpibul, Sirinya Teerananchai, Wasana Prasitsuebsai, Tavitiya Sudjaritruk, Pope Kosalaraksa, Nia Kurniati, Khanh Huu Truong, Viet Chau Do, Lam Van Nguyen, Kulkanya Chokephaibulkit, Thida Singtoroj, Stephen J Kerr
BACKGROUND: Failure rates of second-line boosted protease inhibitor antiretroviral therapy regimens in children rise over time. Therapeutic drug monitoring (TDM) can contribute to assessments of adherence. The authors assessed the performance characteristics of the US DHHS-recommended lopinavir (LPV) concentration of 1.0 mg/L for predicting virologic failure (VF) and intermediate-to-high level LPV resistance in Asian children. MATERIALS AND METHODS: LPV concentration, HIV RNA level, and adherence data from study participants in Indonesia, Thailand, and Vietnam receiving second-line LPV-based ART and followed for ≥24 weeks were analyzed...
August 1, 2016: Therapeutic Drug Monitoring
Kirsten C Odegard, Robert Vincent, Rahul G Baijal, Suanne M Daves, Robert G Gray, Alexander J Javois, Barry A Love, Philip Moore, David Nykanen, Lori Q Riegger, Scott G Walker, Elizabeth C Wilson
Current practice of sedation and anesthesia for patients undergoing pediatric and congenital cardiac catheterization laboratory (PCCCL) procedures is known to vary among institutions, a multi-society expert panel with representatives from the Congenital Heart Disease Council of the Society for Cardiovascular Angiography and Interventions, the Society for Pediatric Anesthesia and the Congenital Cardiac Anesthesia Society was convened to evaluate the types of sedation and personnel necessary for procedures performed in the PCCCL...
October 3, 2016: Anesthesia and Analgesia
Maxime Luu, Marc Bardou, Philippe Bonniaud, Françoise Goirand
Omalizumab is a subcutaneously administrated monoclonal anti-IgE antibody indicated in adults, adolescents and children 6 years of age and older with moderate to severe allergic asthma uncontrolled by conventional pharmacological treatments and sensitization to at least one perennial allergen. Area covered: This drug evaluation summarizes published data on pharmacokinetic and pharmacodynamic properties of omalizumab, on clinical efficacy and safety, including real-world evidence, and provides a medico-economic evaluation of the drug...
October 17, 2016: Expert Opinion on Drug Metabolism & Toxicology
Saul Castaneda, Esbeydy Garcia, Hermelinda De la Cruz, Oscar Ramirez, Samuel Melendez, Jose Sanchez-Palacio
BACKGROUND: Infantile hemangiomas are the most common childhood vascular tumors, occurring in 10 % of children aged less than 1 year. Propranolol, a β-adrenergic blocker mainly indicated for hypertension, has proven effective in treating these types of tumors. OBJECTIVE: To evaluate the efficacy and adverse effects of propranolol in Mexican pediatric patients diagnosed with infantile hemangioma, treated with an extemporaneously compounded solution of propranolol...
March 2016: Drugs—Real World Outcomes
Donald Nuss, Robert J Obermeyer, Robert E Kelly
Historically, pectus excavatum (PE) was reported to be congenital, but in our experience only 22% are noticed in the first decade of life. Thus far, genetic studies support an autosomal recessive heritability, which coincides with only 40% of our patients having some positive family history, but is also contradictory given a constant sex ratio of 4:1 in favor of males. This inconsistency may be explained by the effect of more than one pectus disease-associated allele. Once the deformity is noticed, it tends to progress slowly until puberty, when rapid progression is often seen...
September 2016: Annals of Cardiothoracic Surgery
A Lemoine, F Baudin, R Vialle, E Grimprel
INTRODUCTION: The prognosis of osteoarticular infections has improved over the past 20 years but it still remains potentially severe. The treatment of these infections has been simplified and shortened. In 2008, the Pediatric Infectious Disease Group (GPIP) established new therapeutic guidelines in order to standardize treatment in France. The aim of this study is to analyze practices in a Parisian hospital and assess the efficacy of this treatment in short and medium terms. MATERIALS AND METHODS: This retrospective study focused on patients older than 3 months, without comorbidities, who were hospitalized for an acute osteoarticular infection in 2012 at Trousseau Hospital (Paris), with a follow-up of at least 4 weeks...
October 10, 2016: Archives de Pédiatrie: Organe Officiel de la Sociéte Française de Pédiatrie
Xiaoli Wu, Xuefeng Feng, Xiaoqing Zhao, Futian Ma, Na Liu, Hongming Guo, Chaonan Li, Huan Du, Baoxi Zhang
BACKGROUND/AIMS: Acute and chronic leukemia are severe malignant cancers worldwide, and can occur in pediatric patients. Since bone marrow cell transplantation is seriously limited by the availability of the immune-paired donor sources, the therapy for pediatric leukemia (PL) remains challenging. Autophagy is essential for the regulation of cell survival in the harsh environment. However, the role of autophagy in the survival of PL cells under the oxidative stress, e.g. chemotherapy, remain ill-defined...
October 17, 2016: Cellular Physiology and Biochemistry
Mehmet Bosnak, Seher Erdogan, E Habibe Aktekin, Ali Bay
We report two children who were diagnosed as having primary hemophagocytic syndrome and who successfully underwent therapeutic plasma exchange (TPE). The first patient was a 6-month-old girl diagnosed with HLH who was admitted to the pediatric intensive care unit. The patient's clinical condition worsened on the 9th day of the HLH-2004 treatment protocol. Her ferritin level was found 50.000 ng/mL, and TPE was performed for 9 sessions, after which her clinical condition and laboratory findings improved. The patient is still on the HLH-2004 protocol and waits for a suitable stem cell transplantation donor...
September 28, 2016: Transfusion and Apheresis Science
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