Medicines For Children Research Articles

Emerging Therapies: Communication, Consent, and Collaboration in Research-Based Treatment.

Emerging Therapies: Communication, Consent, and Collaboration in Research-Based Treatment.

DEVELOPMENT OF A KIND OF FLAVORING TABLETS FOR CHILDREN'S USE

Objective : In this study, based on children's medication preferences, Synsepalum dulcificum and Siraitia grosvenorii as raw material were used to develop a flavoring medicine for children. Methods : Synsepalum dulcificum and Siraitia grosvenorii as the raw material were applied to extract the Synsepalum dulcificum powder and Siraitia grosvenorii powder respectively, according to the orthogonal test design; the optimal proportion of flavoring powder was obtained. Low-substituted hydroxypropyl cellulose(L-HPC), polyvinylpolypyrrolidone(PVPP), sodium carboxymethyl starch(CMS-Na）, microcrystalline cellulose(MCC) were taken, according to the orthogonal test design the optimum proportion of auxiliary materials, then the flavoring tablets was prepared according to the preparation process. Results : Through orthogonal test design, the optimum ratio of Synsepalum dulcificum: Siraitia grosvenorii was 1:3, and mixing them up in proportion; the optimum ratio of L-HPC: PVPP: CMS-Na: MCC was 10: 8: 3: 6, and mix them up in proportion. Equal amounts of sugar and salt were obtained by mass ratio of 1 to 1. Mixing 1/3 amount of flavoring powder with 3/4 amount of auxiliary materials, using 20% ethanol as a wetting agent, using the remaining sugar as a filler, and granulating. After the granules were dried and whole grain, add the remaining auxiliary materials and magnesium stearate were added, then the flavoring tablets were prepared. Conclusion : The effects of this formula were prominent, which could effectively intervene the taste of children's medicine; solve the practical problems concerning taking medications in children. Keywords : Synsepalum dulcificum; Siraitia grosvenorii; drug use in children; flavoring tablets

The Collaborative African Genomics Network (CAfGEN): Applying Genomic technologies to probe host factors important to the progression of HIV and HIV-tuberculosis infection in sub-Saharan Africa.

Background: The Human Heredity and Health in Africa consortium (H3Africa) was conceived to facilitate the application of genomics technologies to improve health across Africa. Here, we describe how the Collaborative African Genomics Network (CAfGEN) of the H3Africa consortium is using genomics to probe host genetic factors important to the progression of HIV and HIV-tuberculosis (TB) coinfection in sub-Saharan Africa. Methods: CAfGEN is an H3Africa collaborative centre comprising expertise from the University of Botswana; Makerere University; Baylor College of Medicine Children's Clinical Centers of Excellence (COEs) in Botswana, Uganda, and Swaziland; as well as Baylor College of Medicine, Texas. The COEs provide clinical expertise for community engagement, participant recruitment and sample collection while the three University settings facilitate processing and management of genomic samples and provide infrastructure and training opportunities to sustain genomics research. Results: The project has focused on utilizing whole-exome sequencing to identify genetic variants contributing to extreme HIV disease progression phenotypes in children, as well as RNA sequencing and integrated genomics to identify host genetic factors associated with TB disease progression among HIV-positive children. These cohorts, developed using the COEs' electronic medical records, are exceptionally well-phenotyped and present an unprecedented opportunity to assess genetic factors in individuals whose HIV was acquired by a different route than their adult counterparts in the context of a unique clinical course and disease pathophysiology. Conclusions: Our approach offers the prospect of developing a critical mass of well-trained, highly-skilled, continent-based African genomic scientists. To ensure long term genomics research sustainability in Africa, CAfGEN contributes to a wide range of genomics capacity and infrastructure development on the continent, has laid a foundation for genomics graduate programs at its institutions, and continues to actively promote genomics research through innovative forms of community engagement brokered by partnerships with governments and academia to support genomics policy formulation.

Chapter 1. The Historical Roots of Paediatric Gastroenterology, Hepatology, and Nutrition.

The last 50 years have seen the establishment of paediatric gastroenterology, hepatology, and nutrition (PGHAN) as a well-recognised and thriving clinical specialty throughout most of Europe, and further afield. This has happened, in part, through the existence of the European Society for Paediatric Gastroenterology and Nutrition (ESPGHAN) as a forum for those interested in this branch of children's medicine. To illustrate the pan-European roots of PGHAN, some key scientific and medical events, discoveries, and inventions relevant to 3 common clinical problems-diarrhoea, jaundice, and infant-feeding-have been chosen to survey the historical development of the ways in which each was understood and treated within the changing thinking and practice of past times. Together they are used to trace the prehistory of ESPGHAN and provide a background against which to explain the genesis of the Society and how its spheres of clinical and scientific interest came to be defined.

Повышение качества медицинской помощи детям на основе развития отечественного фармацевтического рынка детских лекарственных средств

The main trends in the health status and physical development of the Russian children's population determine the need to further improve the quality of medical care for children, one of the mechanisms of which is the improvement of pharmaceutical assistance and the expansion of the nomenclature of domestic children's drugs. In this study, we present an analysis of the target segment assortment of the Russian pharmaceutical market - children's medicines. The analysis is carried out with a breakdown into ATC-groups, countries of manufacturers and dosage forms. Import dependence has been established for individual ATC-groups, which affects the quality and availability of medical and pharmaceutical assistance for children.

Safety and Efficacy of Off-label and Unlicensed Medicines in Children

BackgroundThe aim of this study was to explore the use of off-label/unlicensed drugs to confirm the safety and efficacy of their prescription in children in Korea.MethodsIn this retrospective study, we analyzed data of patients who received any of the 32 drugs between January–December 2014 in tertiary hospitals in Korea, including demographics, diagnoses, reasons for the medication, administration route, and details of adverse drug reactions. Additionally, the mortality in the cohort was assessed. The primary outcomes were efficacy and safety, including mortality, of these drugs in pediatric patients. The secondary outcomes were the current statuses of the use of off-label/unlicensed drugs in two centers.ResultsTotally, 5,130 prescriptions were found in 2,779 patients. Age (73.5%) and indication (11.7%) were the most frequent reasons for prescriptions being off-labeled/unlicensed. Approximately 88% of the prescriptions were effective, and 19% of the patients developed adverse drug reactions. The number of prescriptions was significantly higher in children with adverse drug reactions than it was in those without (2.8 vs. 1.5; P < 0.001). The number of prescribed off-label/unlicensed medicines and age at prescription were independently associated with adverse drug events (odds ratio, 1.55 and 1.1; P < 0.001 and 0.034, respectively).ConclusionChildren are still prescribed medicines that are not authorized in terms of age, weight, indications, or routes of administration. Therefore, many old products require re-assessment of authorization. More prospective clinical studies should be performed to confirm the efficacy and safety of drugs in the pediatric population.

The Use of Complementary and Alternative Medicine in Children with Type 1 Diabetes Mellitus

The use of complementary and alternative medicine has been increasing in recent years. Complementary and alternative medicine in pediatrics are generally used in children with chronic illness/disabilities. Type 1 Diabetes Mellitus is also one of these chronic diseases. In this study, information on the use of complementary and alternative medicine in children with Type 1 Diabetes Mellitus was presented using scientific evidences. An literature search using the key words, “complementary”, “alternative”, “complementary and alternative medicine”, “integrative medicine”, “children with Type 1 Diabetes Mellitus” was performed by internet search in November-December, 2017 using Google Scholar, Pubmed, Cochrane Library, Science Direct, Web of Science. Researches between 2007-2017 have been included. Descriptive, cross-sectional, randomized controlled, double blind placebo controlled between 2007-2017; child and parent based studies have been examined and 13 studies have been included in the study. Although the studies on the use of complementary and alternative medicine in children with Type 1 Diabetes Mellitus are limited, their use varies between 18% and 56%, their use is affected by sociodemographic characteristics of the children and their parents. It is observed that the most used complementary and alternative medicine in children with Type 1 Diabetes Mellitus are herbal therapies, vitamins/minerals, nutritional/dietary supplements, prayer/spirutual practices, homeopathy and acupuncture. Studies have shown in children that these treatments are not particularly effective at HbA1c levels, that there is limited evidence of benefit-injury assessment, and that children with Type 1 Diabetes Mellitus require further evidence-based clinical trials.

Precision Medicine in Children and Young Adults with Hematologic Malignancies and Blood Disorders: The Columbia University Experience.

The advent of comprehensive genomic profiling has markedly advanced the understanding of the biology of pediatric hematological malignancies, however, its application to clinical care is still unclear. We present our experience integrating genomic data into the clinical management of children with high-risk hematologic malignancies and blood disorders and describe the broad impact that genomic profiling has in multiple aspects of patient care. The Precision in Pediatric Sequencing Program at Columbia University Medical Center instituted prospective clinical next-generation sequencing (NGS) for high-risk malignancies and blood disorders. Testing included cancer whole exome sequencing (WES) of matched tumor-normal samples or targeted sequencing of 467 cancer-associated genes, when sample adequacy was a concern, and tumor transcriptome (RNA-seq). A multidisciplinary molecular tumor board conducted interpretation of results and final tiered reports were transmitted to the electronic medical record according to patient preferences. Sixty-nine samples from 56 patients with high-risk hematologic malignancies and blood disorders were sequenced. Patients carried diagnoses of myeloid malignancy (n = 25), lymphoid malignancy (n = 25), or histiocytic disorder (n = 6). Six patients had only constitutional WES, performed for a suspicion of an inherited predisposition for their disease. For the remaining 50 patients, tumor was sequenced with matched normal tissue when available. The mean number of somatic variants per sample was low across the different disease categories (2.85 variants/sample). Interestingly, a gene fusion was identified by RNA-seq in 58% of samples who had adequate RNA available for testing. Molecular profiling of tumor tissue led to clinically impactful findings in 90% of patients. Forty patients (80%) had at least one targetable gene variant or fusion identified in their tumor tissue; however, only seven received targeted therapy. Importantly, NGS findings contributed to the refinement of diagnosis and prognosis for 34% of patients. Known or likely pathogenic germline alterations were discovered in 24% of patients involving cancer predisposition genes in 12% of cases. Incorporating whole exome and transcriptome profiling of tumor and normal tissue into clinical practice is feasible, and the value that comprehensive testing provides extends beyond the ability to target-specific mutations.

Improving the Study of New Medicines for Children With Rare Diseases

Improving the Study of New Medicines for Children With Rare Diseases

An audit on parental attitudes towards medicines used in children

IntroductionThere is growing concerns regarding the use of medicines in children. Therefore, many strategies were implemented to improve such use and make safe and effective medicines more available for children. Enhancing interaction between parents and health care providers plays an important role in insuring the proper use of medicines. In order to optimize such interaction, the attitudes, beliefs and practices of parents toward medicine use in children need to be explored. MethodsA validated questionnaire was administered via face to face interviews to 1000 parents attending pediatric outpatient clinics with their children in order to investigate their attitudes toward children's medicines. ResultsThe majority of respondents (83.2%) were mothers. The majority participants (84.4%) agreed that medicines are necessary in treating illnesses, 80% of the parents had worries about the side effects and interactions of medicines, and 60% of the parents said they try to avoid giving medicines to their children. Moreover, parents in this study varied considerably in their views toward prescription and over-the- counter medicines. More than half of the participants (55.2%) declared that doctors in Jordan prescribe antibiotics to children too easily. ConclusionParticipants had positive attitudes toward the necessity of medicines for ill children. However, a considerable proportion of the parents had negative attitudes toward children's medicines with respect to their side effects and interactions, their capability of disturbing the body's own capability of healing illnesses, their unnatural characteristic and other aspects related to medicines.

Preliminary Research on Syndrome Types of Chinese Medicine in Children with Primary Nephrotic Syndrome.

To provide an objective reference for the syndrome types of Chinese medicine (CM) associated with pediatric primary nephrotic syndrome (PNS). A cross-sectional study was performed. Data on clinical symptoms, CM syndrome types, biochemical indices, and medications used were collected from 98 children with PNS. Then, the correlation between CM syndromes and biochemical indices, as well as medications used, was analyzed. The four most common symptoms in children with PNS were brown urine, red tongue, excessive sweating, and swelling of the face and limbs. The syndromes of qi deficiency of Fei (Lung) and Shen (Kidney) (FSQD) and yin deficiency of Gan (Liver) and Shen (GSYD) were the most common main CM syndrome types. FSQD syndrome score correlated significantly with the total cholesterol level, urine protein/creatinine ratio, and urine IgG and albumin levels (P<0.01 or P<0.05). The use of maintenance glucocorticoids combined with immunosuppressive agents correlated with FSQD syndrome, and the use of maintenance glucocorticoids alone correlated with GSYD syndrome (P<0.05). Two of the most common CM syndrome types were FSQD and GSYD syndromes. FSQD syndrome may be caused by some factors related to lipid levels, protein loss, and the use of immunosuppressive agents. The use of maintenance glucocorticoids may cause GSYD syndrome.

Survey on the Use and Perception of Health Functional Foods and Herbal Medicine for Children in Local Day Care Center - Focus on Wonju and Chungju Region -

Survey on the Use and Perception of Health Functional Foods and Herbal Medicine for Children in Local Day Care Center - Focus on Wonju and Chungju Region -

Promoting innovation and access to quality, safe, efficacious, and affordable medicines for children: A China approach on the 69th World Health Assembly.

This study is to provide reference for the proper understanding of the content of "Promoting Innovation and Access to Quality, Safe, Efficacious, and Affordable Medicines for Children" resolution of 69th World Health Assembly (WHA), and facilitate the policy making and implication of the Resolution. Through descriptive analysis, the author introduce the proposal background and approving process of the resolution, and interpret the resolution content. The approval of "Promoting Innovation and Access to Quality, Safe, Efficacious, and Affordable Medicines for Children" at WHA represents China's international discourse right on improving the basic medical service among children, the most vulnerable population; highlights China's responsibility and magnitude of a great nation. It is an effective promotion for China's new medical reform and the implication of the Millennium Development Goals (MDGs), as well as a great contribution to the global health of children from China.

Essential Medicines for Children.

The World Health Organization (WHO) defines Essential Medicines as those that satisfy the priority healthcare needs of the population. The right to essential medicines has been considered an important component of the right to health. In the name of equity, children should also have access to appropriate, available, affordable, and quality essential medicines they need, but children's essential medicines are too often missing.

Blastocoele re-expansion time in vitrified-warmed cycles is a strong predictor of clinical pregnancy outcome.

To assess the predictive value of blastocoele re-expansion time in clinical pregnancy outcome in vitrified-warmed cycles. Data on 468 single vitrified-thawed blastocyst transfer cycles (in patients aged <38 years) carried out from January 2012 through December 2012, at the Reproductive Medicine Center, Maternal and Child Health Hospital of Guangxi Zhuang Autonomous Region, were analyzed. Vitrified-warmed blastocysts were divided into three groups according to blastocoele re-expansion time: group A, <1 h; group B, 1-2 h; and group C, >2 h, and the clinical pregnancy outcomes (i.e. live birth rate, miscarriage rate and occurrence of singleton pregnancies) compared between the groups. Significant differences were observed in the implantation/clinical pregnancy rate between groups A, B and C (70.10%, 51.76% and 28.74%, respectively, P < 0.01). There was a significant linear decline in this rate with increasing blastocyst re-expansion time. The rate of miscarriage also tended to increase with increasing blastocyst re-expansion time, but the difference was not statistically significant (P > 0.05). Of the pregnant patients, no significant difference was observed in the rates of monozygotic twins and ectopic pregnancy between the three groups. For the newborns, similar live birth, low-birthweight and premature delivery rates were observed between the groups. Timing of blastocoele re-expansion in vitrified-warmed cycles is a strong predictor of clinical pregnancy outcome. The faster the re-expansion of the blastocoele, the higher the developmental potential of the blastocysts.

Approval status and evidence for WHO essential medicines for children in the United States, United Kingdom, and Japan: a cross-sectional study

BackgroundThe WHO Model List of Essential Medicines for Children (EMLc) covers medicines for globally high-burden diseases. Regulatory approval in high-income countries ensures evidence and dosage form but usually focuses on diseases common in those countries and not in low- and middle-income countries.MethodsThis cross-sectional study assessed supporting evidence for the 346 medicines in the 5th WHO EMLc and their approval data from the United States, United Kingdom, and Japan.ResultsOf the 346 EMLc medicines, 307 were approved in one or more of the three countries, 278 of which had supporting evidence of efficacy. The percentage of medicines approved in one or more of the three countries was lowest for antiparasitics (60%) whereas 100% for medicines for cancers and musculoskeletal and respiratory conditions were approved. Five of the 30 EMLc antineoplastics had no supporting paediatric evidence. Of the 39 EMLc medicines unapproved in all three countries, 26 were indicated for neglected infectious diseases (NIDs). Ten of the 26 had supporting paediatric evidence. Seventeen of the 39 unapproved medicines had no paediatric dosage form available, and all 17 were indicated for NIDs.ConclusionsMost EMLc medicines for diseases common in the three countries had supporting evidence, which was closely associated with approval, whereas a substantial number of medicines for NIDs were unapproved in the three countries, regardless of whether they had supporting evidence. Because of the limited contribution to the EMLc from high income countries, appropriate incentive mechanisms for pharmaceutical companies are required to make paediatric development for NIDs feasible and effective.

Storage that stands out

The Medpac is a bright orange, durable bag, neatly designed for storing children's medicine. Sal McKeown discovers how it can help pre-schools be super organised when it comes to their health and safety procedures.

POSTERS (1) P1.1 - Availability of Information on use of Medicines in Children in Reference Books

POSTERS (1) P1.1 - Availability of Information on use of Medicines in Children in Reference Books

Children's medicine: What do consumers really want to know?

This study explored consumer knowledge gaps and concerns regarding medication use in children. Calls concerning or made by people under 18 to the pharmacist-operated, national consumer medicines call centre National Prescribing Service Medicines Line (2002 to June 2010) were analysed. Calls were classified and narratively explored by age group: <1, 1-4, 5-14 and 15-17 years. Consumer Medication Information (CMI) and evidence-based resources were examined to determine information concordance for common questions. There were 14 753 paediatric-related calls (mean age 4.1 years). Callers were predominantly female (91.5%), mean age was 35.8 years. Most (89.4%) phoned for a child and 2.2% for themselves. Main enquiry types were: lactation (22.1%), treatment/prophylaxis (11.1%), dose (10.2%), adverse reaction (10.0%), interaction (8.4%) and vaccination (8.4%). However, the primary enquiry differed by age group: lactation (<1 year), dosing (1-4, 5-14 years) and interactions (15-17 years). Global concerns were medication safety, with breastfeeding for infants <1 year and age/weight dose clarification for children 1-4 and 5-14 years. In contrast, interaction questions from adolescents concerned nervous system medicines, cold/flu products, contraceptives and recreational drugs. While paracetamol was the primary medication of interest across age groups, the remaining 'top two' differed significantly. Inconsistencies identified between CMI and evidence-based resources contribute to consumer uncertainty. Care givers and older children use a call centre to address their information needs about frequently used and highly accessible medicines, and their concerns vary across paediatric age groups. Inconsistent information provided by CMI on medication use in children contributes to consumer uncertainty and help-seeking behaviour.

European Paediatric Formulation Initiative (EuPFI)-Formulating Ideas for Better Medicines for Children.

The European Paediatric Formulation Initiative (EuPFI), founded in 2007, aims to promote and facilitate the preparation of better and safe medicines for children through linking research and information dissemination. It brings together the capabilities of the industry, academics, hospitals, and regulators within a common platform in order to scope the solid understanding of the major issues, which will underpin the progress towards the future of paediatric medicines we want.The EuPFI was formed in parallel to the adoption of regulations within the EU and USA and has served as a community that drives research and dissemination through publications and the organisation of annual conferences. The membership and reach of this group have grown since its inception in 2007 and continue to develop and evolve to meet the continuing needs and ambitions of research into and development of age appropriate medicines. Five diverse workstreams (age-appropriate medicines, Biopharmaceutics, Administration Devices, Excipients and Taste Assessment & Taste Masking (TATM)) direct specific workpackages on behalf of the EuPFI. Furthermore, EuPFI interacts with multiple diverse professional groups across the globe to ensure efficient working in the area of paediatric medicines. Strong commitment and active involvement of all EuPFI stakeholders have proved to be vital to effectively address knowledge gaps related to paediatric medicines, discuss potential areas for further research and identify issues that need more attention and analysis in the future.