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Pediatric Drug Development

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https://www.readbyqxmd.com/read/29147075/pediatric-ocular-nanomedicines-challenges-and-opportunities
#1
Natasha D Sheybani, Hu Yang
The eye is a highly complex, yet readily accessible organ within the human body. As such, the eye is an appealing candidate target for a vast array of drug therapies. Despite advances in ocular drug therapy research, the focus on pediatric ocular drug delivery continues to be highly underrepresented due to the limited number of degenerative ocular diseases with childhood onset. In this review, we explore more deeply the reasons underlying the disparity between ocular therapies available for children and for adults by highlighting diseases that most commonly afflict children (with focus on the anterior eye) and existing prognoses, recent developments in ocular drug delivery systems and nanomedicines for children, and barriers to use for pediatric patients...
September 2017: Chinese Chemical Letters Zhongguo Hua Xue Kuai Bao
https://www.readbyqxmd.com/read/29143423/treatment-and-biology-of-pediatric-acute-lymphoblastic-leukemia
#2
REVIEW
Motohiro Kato, Atsushi Manabe
Acute lymphoblastic leukemia (ALL) is the most common pediatric malignancy. In the past ALL was intractable but now the survival probability is as high as 80-90%. Improved supportive care, treatment stratification based on relapse risk, biological features of leukemic cells, and optimization of treatment regimens by nationwide and international collaboration have contributed to this dramatic improvement. While including traditional risk factors (e.g., age and leukocyte count at diagnosis), the treatment has been modified based on biological characteristics (aneuploidy and translocation) and treatment response (assessed by minimal residual disease)...
November 16, 2017: Pediatrics International: Official Journal of the Japan Pediatric Society
https://www.readbyqxmd.com/read/29140495/traditional-and-complementary-medicine-in-pediatric-oncology-and-low-middle-income-countries-recommendations-from-the-international-society-of-pediatric-oncology-siop-t-cm-collaborative
#3
Elena J Ladas, Stacey Marjerrison, Brijesh Arora, Peter B Hesseling, Roberta Ortiz, Federico Antillon, Shalini Jatia, Glenn M Afungchwi
Significant strides have been made in the treatment of childhood cancer. Improvements in survival have led to increased attention toward supportive care indications; including the use of traditional and complementary medicine (T&CM). The use of T&CM among children and adolescents with cancer is well documented in both high-income countries (HICs) and low-middle income countries (LMICs). A higher incidence of the use of T&CM has been reported among children undergoing treatment in LMICs, which has elevated concerns related to drug interactions, adherence to therapy, and treatment-related toxicities...
November 1, 2017: Journal of the National Cancer Institute. Monographs
https://www.readbyqxmd.com/read/29138650/evaluation-of-antibiotic-self-medication-among-outpatients-of-the-state-university-hospital-of-port-au-prince-haiti-a-cross-sectional-study
#4
Kenny Moise, Joseph Junior Bernard, Jean Hugues Henrys
In Haiti, where all drugs are available over the counter, self-medication with antibiotics appears as a common practice. Inappropriate use of beta-lactams and macrolides is likely to contribute to the development of antimicrobial resistance. This study aimed to (i) assess the extent of self-medication with antibiotics, (ii) explore the contributing factors (age, gender and educational background) and (iii) identify specific antibiotic drug classes used among patients attending the outpatient clinic of the State University Hospital of Port-au-Prince...
2017: Pan African Medical Journal
https://www.readbyqxmd.com/read/29119912/the-glutamate-and-the-immune-systems-new-targets-for-the-pharmacological-treatment-of-ocd
#5
Donatella Marazziti, Umberto Albert, Federico Mucci, Armando Piccinni
BACKGROUND: In the last decades the pharmacological treatment of obsessive-compulsive disorder (OCD) has been significantly promoted by the effectiveness of selective serotonin (5-HT) reuptake inhibitors (SSRIs) and the subsequent development of the 5-HT hypothesis of OCD. However, since a large majority of patients (between 40% and 60 %) do not respond to SSRIs or strategies based on the modulation of the 5-HT system, it is now essential to search for other possible therapeutic targets...
November 8, 2017: Current Medicinal Chemistry
https://www.readbyqxmd.com/read/29116003/protocols-on-classification-monitoring-and-therapy-in-children-s-rheumatology-pro-kind-results-of-the-working-group-polyarticular-juvenile-idiopathic-arthritis
#6
REVIEW
Gerd Horneff, Ariane Klein, Gerd Ganser, Michaela Sailer-Höck, Annette Günther, Ivan Foeldvari, Frank Weller-Heinemann
OBJECTIVE: Several effective pharmacologic treatment options for polyarticual juvenile idiopathic arthritis (JIA) have emerged but initial treatment is heterogeneous in Germany. Therefore, the German Society of Pediatric Rheumatolgy has established a commission to develop consensus "Protocols on classification, monitoring and therapy in children's rheumatology (PRO-KIND)" to harmonize diagnostic and treatment approaches for new-onset JIA in Germany. METHODS: A set of definitions for in- and exclusion, diagnostic workup, parameters for the evaluation of disease activity criteria, therapeutic options, medication dosing, monitoring recommendations, targets, definitions of a therapy failure and four therapeutic algorithms developed by a working group were agreed by web based survey to which all members of the GKJR have been invited...
November 7, 2017: Pediatric Rheumatology Online Journal
https://www.readbyqxmd.com/read/29115708/cyp3a5-genotype-and-its-impact-on-vincristine-pharmacokinetics-and-development-of-neuropathy-in-kenyan-children-with-cancer
#7
Jodi L Skiles, ChienWei Chiang, Claire H Li, Steve Martin, Ellen L Smith, Gilbert Olbara, David R Jones, Terry A Vik, Saskia Mostert, Floor Abbink, Gertjan J Kaspers, Lang Li, Festus Njuguna, Tammy J Sajdyk, Jamie L Renbarger
BACKGROUND: Vincristine (VCR) is a critical part of treatment in pediatric malignancies and is associated with dose-dependent peripheral neuropathy (vincristine-induced peripheral neuropathy [VIPN]). Our previous findings show VCR metabolism is regulated by the CYP3A5 gene. Individuals who are low CYP3A5 expressers metabolize VCR slower and experience more severe VIPN as compared to high expressers. Preliminary observations suggest that Caucasians experience more severe VIPN as compared to nonCaucasians...
November 8, 2017: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/29113971/correction-to-2017-acc-aap-aha-health-policy-statement-on-opportunities-and-challenges-in-pediatric-drug-development-learning-from-sildenafil
#8
(no author information available yet)
No abstract text is available yet for this article.
November 2017: Circulation. Cardiovascular Quality and Outcomes
https://www.readbyqxmd.com/read/29113313/whole-exome-analysis-in-osteosarcoma-to-identify-a-personalized-therapy
#9
Caterina Chiappetta, Massimiliano Mancini, Francesca Lessi, Paolo Aretini, Veronica De Gregorio, Chiara Puggioni, Raffaella Carletti, Vincenzo Petrozza, Prospero Civita, Sara Franceschi, Antonio G Naccarato, Carlo Della Rocca, Chiara M Mazzanti, Claudio Di Cristofano
Osteosarcoma is the most common pediatric primary non-hematopoietic bone tumor. Survival of these young patients is related to the response to chemotherapy and development of metastases. Despite many advances in cancer research, chemotherapy regimens for osteosarcoma are still based on non-selective cytotoxic drugs. It is essential to investigate new specific molecular therapies for osteosarcoma to increase the survival rate of these patients. We performed exomic sequence analyses of 8 diagnostic biopsies of patients with conventional high grade osteosarcoma to advance our understanding of their genetic underpinnings and to correlate the genetic alteration with the clinical and pathological features of each patient to identify a personalized therapy...
October 6, 2017: Oncotarget
https://www.readbyqxmd.com/read/29112802/consensus-treatment-plans-for-chronic-nonbacterial-osteomyelitis-refractory-to-nonsteroidal-anti-inflammatory-drugs-and-or-with-active-spinal-lesions
#10
Yongdong Zhao, Eveline Y Wu, Melissa S Oliver, Ashley M Cooper, Matthew L Basiaga, Sheetal S Vora, Tzielan C Lee, Emily Fox, Gil Amarilyo, Sara M Stern, Jeffrey A Dvergsten, Kathleen A Haines, Kelly A Rouster-Stevens, Karen B Onel, Julie Cherian, Jonathan S Hausmann, Paivi Miettunen, Tania Cellucci, Farzana Nuruzzaman, Angela Taneja, Karyl S Barron, Matthew C Hollander, Sivia K Lapidus, Suzanne C Li, Seza Ozen, Hermann Girschick, Ronald M Laxer, Fatma Dedeoglu, Christian M Hedrich, Polly J Ferguson
OBJECTIVE: To develop standardized treatment regimens for chronic nonbacterial osteomyelitis (CNO), also known as chronic recurrent multifocal osteomyelitis (CRMO) to enable comparative effectiveness treatment studies. METHODS: Virtual and face-to-face discussions and meetings were held within the CNO subgroup of the Childhood Arthritis and Rheumatology Research Alliance (CARRA). A literature search was conducted, and CARRA membership was surveyed to evaluate available treatment data and identify current treatment practices...
November 7, 2017: Arthritis Care & Research
https://www.readbyqxmd.com/read/29109579/program-to-manage-new-and-expensive-drugs-in-pediatrics-profile-of-a-new-drug-policy-and-a-12-month-descriptive-study
#11
Jennifer Corny, Camille Cotteret, Élaine Pelletier, Philippe Ovetchkine, Jean-François Bussières
Background: With growing financial pressure and the range of new and expensive drugs, hospital administrators, clinicians, and pharmacy directors are facing tough decisions on how to manage drug budgets. At a Canadian mother-child hospital, a policy for new and expensive drugs was developed, with the goal of managing their use and costs. Objectives: To describe the development and implementation of a policy for new and expensive drugs in a mother-child teaching hospital and to describe the profile of requests for these therapies over a 12-month period...
September 2017: Canadian Journal of Hospital Pharmacy
https://www.readbyqxmd.com/read/29107733/determination-of-the-r-and-s-enantiomers-of-vigabatrin-in-human-plasma-by-ultra-high-performance-liquid-chromatography-and-tandem-mass-spectrometry
#12
Paul Duhamel, Marwa Ounissi, Thomas Le Saux, Hugues Bienayme, Catherine Chiron, Vincent Jullien
An analytical method was developed for the quantification in plasma of the R and S enantiomers of vigabatrin (VGB), a drug used for the treatment of some refractory pediatric epileptic syndromes. After adding 50μL of the internal standard, which consisted of a 15mg/L solution of deuterated racemic VGB, and 100μL of water to 100μL of plasma samples, a protein precipitation was performed by adding 600μL of methanol. The supernatant was evaporated to dryness under a stream of nitrogen and the dry residue was reconstituted with 500μL of water...
October 20, 2017: Journal of Chromatography. B, Analytical Technologies in the Biomedical and Life Sciences
https://www.readbyqxmd.com/read/29103429/immunotherapeutics-in-pediatric-autoimmune-central-nervous-system-disease-agents-and-mechanisms
#13
Margherita Nosadini, Stefano Sartori, Suvasini Sharma, Russell C Dale
Beyond the major advances produced by careful clinical-radiological phenotyping and biomarker development in autoimmune central nervous system disorders, a comprehensive knowledge of the range of available immune therapies and a deeper understanding of their action should benefit therapeutic decision-making. This review discusses the agents used in neuroimmunology and their mechanisms of action. First-line treatments typically include corticosteroids, intravenous immunoglobulin, and plasmapheresis, while for severe disease second-line "induction" agents such as rituximab or cyclophosphamide are used...
August 2017: Seminars in Pediatric Neurology
https://www.readbyqxmd.com/read/29103180/safety-of-weekly-adalimumab-in-the-treatment-of-juvenile-idiopathic-arthritis-and-pediatric-chronic-uveitis
#14
Colleen K Correll, Danielle R Bullock, Rachel M Cafferty, Richard K Vehe
Weekly adalimumab dosing is used to treat juvenile idiopathic arthritis (JIA), uveitis, and other pediatric rheumatic diseases, but the safety of such dosing has not previously been studied. A retrospective chart review was conducted to assess the safety of weekly adalimumab. Demographic and clinical data were collected. Basic descriptive analysis was performed to assess for adverse events from weekly adalimumab. Sixty-nine patients at the University of Minnesota or Gillette Children's Hospital were identified as treated with weekly adalimumab...
November 4, 2017: Clinical Rheumatology
https://www.readbyqxmd.com/read/29099871/the-role-of-h19-a-long-non-coding-rna-in-mouse-liver-postnatal-maturation
#15
Chad Pope, Stephanie C Piekos, Liming Chen, Shashank Mishra, Xiao-Bo Zhong
H19 RNA is highly expressed at early postnatal ages and precipitously decreases at a specific time corresponding with increases in expression of genes important for mature liver function, such as drug metabolizing enzymes. H19's role in the regulation of liver maturation is currently unknown. Using an H19 knockout mouse model to determine the role of H19 in liver development, we quantified gene expression for insulin growth factor signaling, Wnt signaling, key cytochrome P450 (P450) enzymes known to change as the liver develops, and fetal and adult plasma protein produced in liver...
2017: PloS One
https://www.readbyqxmd.com/read/29097609/recent-advances-of-cell-cycle-inhibitor-therapies-for-pediatric-cancer
#16
REVIEW
Christopher C Mills, E A Kolb, Valerie B Sampson
This review describes the pivotal roles of cell-cycle and checkpoint regulators and discusses development of specific cell-cycle inhibitors for therapeutic use for pediatric cancer. The mechanism of action as well as the safety and tolerability of drugs in pediatric patients, including compounds that target CDK4/CDK6 (palbociclib, ribociclib, and abemaciclib), aurora kinases (AT9283 and MLN8237), Wee1 kinase (MK-1775), KSP (ispinesib), and tubulin (taxanes, vinca alkaloids), are presented. The design of mechanism-based combinations that exploit the cross-talk of signals activated by cell-cycle arrest, as well as pediatric-focused drug development, are critical for the advancement of drugs for rare childhood diseases...
November 2, 2017: Cancer Research
https://www.readbyqxmd.com/read/29097019/secondary-intracranial-hypertension-in-pediatric-patients-with-leukemia
#17
Miguel Ángel Fernández-García, Verónica Cantarín-Extremera, Maitane Andión-Catalán, Anna Duat-Rodríguez, Saioa Jiménez-Echevarría, Ignacio Bermejo-Arnedo, Montesclaros Hortigüela-Saeta, Saray Rekarte-García, Lara Babín-López, David Ruano Domínguez
BACKGROUND: We investigated the clinical characteristics of a pediatric population with hemato-oncological disease and intracranial hypertension, analyze the therapeutic response and outcome, and compare its characteristics with respect to a control group with idiopathic intracranial hypertension. METHODS: We retrospectively analyzed patients with hemato-oncological disease and secondary intracranial hypertension in our center during the past five years. We compared these individuals with a historical cohort with idiopathic intracranial hypertension from our institution (control group)...
September 1, 2017: Pediatric Neurology
https://www.readbyqxmd.com/read/29093098/identification-of-a-critical-and-conformational-neutralizing-epitope-in-human-adenovirus-type-4-hexon
#18
Xingui Tian, Hongling Qiu, Zhichao Zhou, Shouli Wang, Ye Fan, Xiao Li, Ruiai Chu, Haitao Li, Rong Zhou, Hui Wang
Human adenovirus type 4 (HAdV-4) is an epidemic virus that contributes to serious acute respiratory disease (ARD) in both pediatric and adult patients. However, no licensed drug or vaccine is currently available to the civilian population. The identification of the neutralizing epitopes of HAdV-4 should allow the development of a novel antiviral vaccine and novel gene transfer vector, and an effective neutralizing monoclonal antibody (MAb) will be useful in developing appropriate antiviral drugs. In this study, the MAb MN4b is reported to show strong neutralizing activity against HAdV-4...
November 1, 2017: Journal of Virology
https://www.readbyqxmd.com/read/29090524/outcome-of-pediatric-patients-with-acute-lymphoblastic-leukemia-lymphoblastic-lymphoma-with-hypersensitivity-to-pegaspargase-treated-with-pegylated-erwinia-asparaginase-pegcrisantaspase-a-report-from-the-children-s-oncology-group
#19
Rachel E Rau, ZoAnn Dreyer, Mi Rim Choi, Wei Liang, Roman Skowronski, Krishna P Allamneni, Meenakshi Devidas, Elizabeth A Raetz, Peter C Adamson, Susan M Blaney, Mignon L Loh, Stephen P Hunger
BACKGROUND: Erwinia asparaginase is a Food and Drug Administration approved agent for the treatment of acute lymphoblastic leukemia (ALL) for patients who develop hypersensitivity to Escherichia coli derived asparaginases. Erwinia asparaginase is efficacious, but has a short half-life, requiring six doses to replace one dose of the most commonly used first-line asparaginase, pegaspargase, a polyethylene glycol (PEG) conjugated E. coli asparaginase. Pegcristantaspase, a recombinant PEGylated Erwinia asparaginase with improved pharmacokinetics, was developed for patients with hypersensitivity to pegaspargase...
November 1, 2017: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/29089643/targeting-acute-myeloid-leukemia-by-drug-induced-c-myb-degradation
#20
V Walf-Vorderwülbecke, K Pearce, T Brooks, M Hubank, M M van den Heuvel-Eibrink, C M Zwaan, S Adams, D Edwards, J Bartram, S Samarasinghe, P Ancliff, A Khwaja, N Goulden, G Williams, J de Boer, O Williams
Despite advances in our understanding of the molecular basis for particular subtypes of acute myeloid leukemia (AML), effective therapy remains a challenge for many individuals suffering from this disease. A significant proportion of both pediatric and adult AML patients cannot be cured and since the upper limits of chemotherapy intensification have been reached, there is an urgent need for novel therapeutic approaches. The transcription factor c-MYB has been shown to play a central role in the development and progression of AML driven by several different oncogenes, including mixed lineage leukemia (MLL)-fusion genes...
November 1, 2017: Leukemia: Official Journal of the Leukemia Society of America, Leukemia Research Fund, U.K
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