Special Issue "Pediatric Diseases"

A special issue of Diseases (ISSN 2079-9721).

Deadline for manuscript submissions: closed (30 June 2018)

Special Issue Editor

Guest Editor
Assoc. Prof. Mark A. Brown

College of Veterinary Medicine and Biomedical Sciences, Colorado State University,1052 Campus Delivery,Fort Collins, CO 80523-1052, USA
Website | E-Mail
Interests: oncology; drug development; chromatin modifications; education

Special Issue Information

Dear Colleagues,

There is a growing interest in the re-emergence of diseases that impact pediatric demographics. While major strides have been made in the field of childhood cancers, there are still more questions than answers. In addition, the resistance to childhood vaccinations fueled by misinformation has allowed infectious diseases to resurface in developed nations. Meanwhile, climate change and other destabilizing factors are shifting vector populations and driving the emergence of new disease. In this special issue of Diseases, we invite papers related to detection, prevention, diagnosis and treatment of human pediatric diseases. Papers related to molecular mechanisms, epidemiology,immunology, and public health initiatives associated with pediatric diseases are also encouraged.

Dr. Mark A. Brown
Guest Editor

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All papers will be peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Diseases is an international peer-reviewed open access quarterly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 350 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

Keywords

  • pediatric medicine
  • oncology
  • infectious disease
  • vaccines
  • molecular mechanisms
  • immunology
  • public health

Published Papers (9 papers)

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Research

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Open AccessFeature PaperCommunication Facilitating a More Efficient Commercial Review Process for Pediatric Drugs and Biologics
Received: 7 November 2017 / Revised: 14 December 2017 / Accepted: 19 December 2017 / Published: 22 December 2017
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Abstract
Over the past two decades, the biopharmaceutical industry has seen unprecedented expansion and innovation in concert with significant technological advancements. While the industry has experienced marked growth, the regulatory system in the United States still operates at a capacity much lower than the
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Over the past two decades, the biopharmaceutical industry has seen unprecedented expansion and innovation in concert with significant technological advancements. While the industry has experienced marked growth, the regulatory system in the United States still operates at a capacity much lower than the influx of new drug and biologic candidates. As a result, it has become standard for months or even years of waiting for commercial approval by the U.S. Food and Drug Administration. These regulatory delays have generated a system that stifles growth and innovation due to the exorbitant costs associated with awaiting approval from the nation’s sole regulatory agency. The recent re-emergence of diseases that impact pediatric demographics represents one particularly acute reason for developing a regulatory system that facilitates a more efficient commercial review process. Herein, we present a range of initiatives that could represent early steps toward alleviating the delays in approving life-saving therapeutics. Full article
(This article belongs to the Special Issue Pediatric Diseases)
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Open AccessFeature PaperArticle Having a Child Diagnosed with Cancer: Raising the Challenges Encountered by the Caregivers at the Pediatric Oncology Ward in Egypt
Received: 20 October 2017 / Revised: 25 November 2017 / Accepted: 14 December 2017 / Published: 19 December 2017
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Abstract
Having a child diagnosed with a life-threatening illness, and undergoing a severe treatment regimen, is a massive challenge for many caregivers, not the least of who are those with low socioeconomic status and living in a society where deeply rooted cultural and societal
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Having a child diagnosed with a life-threatening illness, and undergoing a severe treatment regimen, is a massive challenge for many caregivers, not the least of who are those with low socioeconomic status and living in a society where deeply rooted cultural and societal misconceptions are immensely noticeable. The aim of the study is to raise the great concerns experienced by the caregivers at the pediatric oncology ward in Egypt. The study is comprised of 24 caregivers of children with cancer undergoing treatment at the inpatient ward. Semi-structured interviews and participant observations were used as a means of data collection. Numerous concerns are addressed in this study which are all related to fear of the illness and guilty feelings of having caused the child this illness. The fears and concerns addressed in this paper seem to obstruct the caregivers’ overall psychosocial wellbeing, which is known to have multiple effects on the child’s overall wellbeing. Thus, it is very important to take into consideration caregivers in the child’s cancer treatment. Full article
(This article belongs to the Special Issue Pediatric Diseases)
Open AccessArticle Competitive Promoter-Associated Matrix Attachment Region Binding of the Arid3a and Cux1 Transcription Factors
Received: 15 November 2017 / Revised: 5 December 2017 / Accepted: 5 December 2017 / Published: 10 December 2017
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Abstract
Arid3a/Bright/Dril1 is a B cell-specific transactivator that regulates immunoglobulin heavy chain (IgH) gene transcription by binding promoter and enhancer-associated matrix attachment regions (MARs) within the IgH gene locus. Promoter MAR-mediated Arid3a transactivation is antagonized by direct competition of MAR binding by Cux1/CDP—a ubiquitously
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Arid3a/Bright/Dril1 is a B cell-specific transactivator that regulates immunoglobulin heavy chain (IgH) gene transcription by binding promoter and enhancer-associated matrix attachment regions (MARs) within the IgH gene locus. Promoter MAR-mediated Arid3a transactivation is antagonized by direct competition of MAR binding by Cux1/CDP—a ubiquitously expressed repressor originally termed NF-μNR. We report that the NF-μNR complex includes Arid3a in B cells but not in non-B cells through mobility shift assays. The binding activity of NF-μNR and Arid3a in B cells is reciprocally altered during the cell division cycle and by the B cell mitogen lipopolysaccharide LPS. LPS treatment had no effect on Arid3a localization but increased its total abundance within the nucleus and cytoplasm. We show that this increased level of Arid3a is capable of displacing Cux from the MARs to facilitate IgH gene transcription. Finally, we showed that the MARs (termed Bf150 and Tx125) associated with the VH1 rearranged variable region expressed in the S107 murine plasmacytoma, can repress reporter gene transcription in non-B cells and that they can relieve the repression mediated by Eμ enhancer in B cells. These results have significant implications for early human development and demonstrate that MARs in IgH locus, NF-µNR and Arid3a regulate IgH gene expression in a concerted fashion. This paves the way for future studies examining the misregulation of this pathway in pediatric disease. Full article
(This article belongs to the Special Issue Pediatric Diseases)
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Review

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Open AccessReview Treatment of Major Depressive Disorder in Pediatric Populations
Received: 30 April 2018 / Revised: 28 May 2018 / Accepted: 31 May 2018 / Published: 4 June 2018
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Abstract
Major depressive disorder (MDD) is a severe illness that afflicts about 16.6% of people over their lifetime. MDD is highly correlated with suicidality, and often first presents in adolescence. Unfortunately, many pediatric patients suffering from MDD go undiagnosed, and current evidence-based treatment options
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Major depressive disorder (MDD) is a severe illness that afflicts about 16.6% of people over their lifetime. MDD is highly correlated with suicidality, and often first presents in adolescence. Unfortunately, many pediatric patients suffering from MDD go undiagnosed, and current evidence-based treatment options in the U.S. are limited to psychotherapy and two selective serotonin reuptake inhibitors approved by the United States Food and Drug Administration. Molecular mechanisms have been shown to play a role in MDD pathogenesis, progression, and response to medication, yet few studies have explored the role of these pathways in pediatric MDD. In this review, we outline the gravity and importance of MDD in pediatric patients, some challenges in diagnosis and treatment, current treatments available for pediatric patients, and research to investigate differences between pediatric and adult MDD. We hope that this review will provide an outline of the current understanding and treatment of MDD in pediatric patients, and provide thoughtful insights for future work that could advance our understanding of MDD in pediatric populations, and also identify new therapeutic strategies. Full article
(This article belongs to the Special Issue Pediatric Diseases)
Open AccessReview Zebrafish Models of Rare Hereditary Pediatric Diseases
Received: 27 April 2018 / Revised: 17 May 2018 / Accepted: 19 May 2018 / Published: 22 May 2018
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Abstract
Recent advances in sequencing technologies have made it significantly easier to find the genetic roots of rare hereditary pediatric diseases. These novel methods are not panaceas, however, and they often give ambiguous results, highlighting multiple possible causative mutations in affected patients. Furthermore, even
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Recent advances in sequencing technologies have made it significantly easier to find the genetic roots of rare hereditary pediatric diseases. These novel methods are not panaceas, however, and they often give ambiguous results, highlighting multiple possible causative mutations in affected patients. Furthermore, even when the mapping results are unambiguous, the affected gene might be of unknown function. In these cases, understanding how a particular genotype can result in a phenotype also needs carefully designed experimental work. Model organism genetics can offer a straightforward experimental setup for hypothesis testing. Containing orthologs for over 80% of the genes involved in human diseases, zebrafish (Danio rerio) has emerged as one of the top disease models over the past decade. A plethora of genetic tools makes it easy to create mutations in almost any gene of the zebrafish genome and these mutant strains can be used in high-throughput preclinical screens for active molecules. As this small vertebrate species offers several other advantages as well, its popularity in biomedical research is bound to increase, with “aquarium to bedside” drug development pipelines taking a more prevalent role in the near future. Full article
(This article belongs to the Special Issue Pediatric Diseases)
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Open AccessReview Revisiting CD28 Superagonist TGN1412 as Potential Therapeutic for Pediatric B Cell Leukemia: A Review
Received: 29 April 2018 / Revised: 17 May 2018 / Accepted: 18 May 2018 / Published: 19 May 2018
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Abstract
Pediatric acute lymphoblastic leukemia (ALL) represents the most common pediatric cancer diagnosis, with numbers rising gradually every year. This paper proposes a novel therapeutic agent for pediatric ALL on the basis of a failed clinical drug trial in 2006. TGN1412 was a promising
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Pediatric acute lymphoblastic leukemia (ALL) represents the most common pediatric cancer diagnosis, with numbers rising gradually every year. This paper proposes a novel therapeutic agent for pediatric ALL on the basis of a failed clinical drug trial in 2006. TGN1412 was a promising therapeutic agent that yielded outstanding results in both in vitro studies and animal trials. It is a CD28 superagonist monoclonal antibody that activates T regulatory (TReg) cells in the absence of costimulation of the T cell receptor (TCR) by an antigen-presenting cell. This drug was intended as a solution to T cell deficient diseases such as B cell leukemia and autoimmune diseases such as rheumatoid arthritis. When phase I clinical trials were conducted, all volunteers that received the drug experienced severe cytokine release syndrome (CRS) and faced multiple-organ failure within hours. TGN1412 was reassessed and re-entered clinical trials as a therapeutic for rheumatoid arthritis. A new assay was developed to better quantify T cell response, and volunteers in this trial experienced no pro-inflammatory cytokine release. This essay analyzes how misinformation contributed to the failure of TGN1412 in clinical trials and how revisiting this therapeutic could yield a novel treatment for pediatric B cell leukemia. Full article
(This article belongs to the Special Issue Pediatric Diseases)
Open AccessFeature PaperReview Familial Screening for Left-Sided Congenital Heart Disease: What Is the Evidence? What Is the Cost?
Received: 7 November 2017 / Revised: 3 December 2017 / Accepted: 5 December 2017 / Published: 8 December 2017
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Abstract
Since the American Heart Association’s recommendation for familial screening of adults with congenital heart disease for bicuspid aortic valve, similar recommendations for other left-sided heart defects, such as hypoplastic left heart syndrome (HLHS), have been proposed. However, defining at-risk populations for these heart
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Since the American Heart Association’s recommendation for familial screening of adults with congenital heart disease for bicuspid aortic valve, similar recommendations for other left-sided heart defects, such as hypoplastic left heart syndrome (HLHS), have been proposed. However, defining at-risk populations for these heart defects based on genetics is less straightforward due to the wide variability of inheritance patterns and non-genetic influences such as environmental and lifestyle factors. We discuss whether there is sufficient evidence to standardize echocardiographic screening for first-degree relatives of children diagnosed with HLHS. Due to variations in the inclusion of cardiac anomalies linked to HLHS and the identification of asymptomatic individuals with cardiac malformations, published studies are open to interpretation. We conclude that familial aggregation of obstructive left-sided congenital heart lesions in families with history of HLHS is not supported and recommend that additional screening should adopt a more conservative definition of what truly constitutes this heart defect. More thorough consideration is needed before embracing familial screening recommendations of families of patients with HLHS, since this could inflict serious costs on healthcare infrastructure and further burden affected families both emotionally and financially. Full article
(This article belongs to the Special Issue Pediatric Diseases)

Other

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Open AccessFeature PaperComment Risk Evaluation Requires an Independent Mind
Received: 13 November 2017 / Revised: 21 November 2017 / Accepted: 21 November 2017 / Published: 24 November 2017
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Abstract
Biomedical research pertaining to pathologies observed in adolescents can involve areas where patients can expect no immediate benefits. Here, Congress stipulates that local review boards are restricted to approving procedures posing no greater than minimal risk (45 CFR 46.404). An evaluation of risk
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Biomedical research pertaining to pathologies observed in adolescents can involve areas where patients can expect no immediate benefits. Here, Congress stipulates that local review boards are restricted to approving procedures posing no greater than minimal risk (45 CFR 46.404). An evaluation of risk embraces the current state of the art with regard to the safety and efficacy of procedures. A tendency of biomedical scholars to cite highly cited documents can introduce a bias in an argumentation in favor or against a given recommendation in the context that bias in citations can be correlated with an imprudent use of funds for research. We use choice examples to highlight the necessity of approaching any scholarly task with an independent mind. Full article
(This article belongs to the Special Issue Pediatric Diseases)
Open AccessCommentary The Evolution of Pediatric Disease—A Moving Target in Public Health
Received: 28 August 2017 / Accepted: 28 August 2017 / Published: 31 August 2017
Cited by 1 | PDF Full-text (745 KB) | HTML Full-text | XML Full-text
Abstract
There is a growing threat in the re-emergence of diseases that impact pediatric demographics. While major strides have been made in the field of childhood cancers, there are still more questions than answers. In addition, public resistance to recommended practices related to childhood
[...] Read more.
There is a growing threat in the re-emergence of diseases that impact pediatric demographics. While major strides have been made in the field of childhood cancers, there are still more questions than answers. In addition, public resistance to recommended practices related to childhood vaccinations fueled by misinformation has allowed infectious diseases to resurface in developed nations. Meanwhile, climate change and other destabilizing factors are shifting vector populations and driving the emergence of new diseases. Herein we call upon the community of human health researchers to confront the evolving specter of pediatric disease. Full article
(This article belongs to the Special Issue Pediatric Diseases)
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