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Children, Volume 7, Issue 2 (February 2020) – 7 articles

Cover Story (view full-size image): Acute promyelocytic leukemia (APL) is a rare disease accounting for only 5%–10% of pediatric acute myeloid leukemia (AML) and fewer than 1000 cases occur annually in the United States across all age groups. Characterized by t (15; 17), with a resultant PML-RARA gene fusion driving leukemia development, advances in therapy have improved outcomes for APL significantly in the past several decades, now making APL the most curable form of AML in both children and adults. Cure rates in APL are now comparable to pediatric B-lymphoid leukemias. The success of APL treatment is due, in part, to the breadth of understanding of the driver PML-RARA mutation as well as collaborative efforts to quickly introduce and maximize the benefit of new therapies. Here, we review the presentation, clinical features, pathogenesis, and treatment advances in pediatric APL. View this paper.
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15 pages, 2027 KiB  
Article
The Profile, Health Seeking Behavior, Referral Patterns, and Outcome of Outborn Neonates Admitted to a District and Regional Hospital in the Upper West Region of Ghana: A Cross-Sectional Study
by Edem M. A. Tette, Benjamin Demah Nuertey, Emmanuel A. Azusong and Naa Barnabas Gandau
Children 2020, 7(2), 15; https://doi.org/10.3390/children7020015 - 18 Feb 2020
Cited by 7 | Viewed by 5267
Abstract
Neonatal mortality is the major contributor to under-five mortality rates in many low and middle income countries. We examined the health practices, care-seeking behavior, and referral of sick outborn neonates to a district and regional hospital in the Upper West Region of Ghana. [...] Read more.
Neonatal mortality is the major contributor to under-five mortality rates in many low and middle income countries. We examined the health practices, care-seeking behavior, and referral of sick outborn neonates to a district and regional hospital in the Upper West Region of Ghana. The study was a cross-sectional study conducted over an eight (8) month period in 2018. Data were obtained from caregiver interviews and case notes. Altogether, 153 outborn neonates were examined. Inappropriate practices including the use of enemas, cord care with cow dung, and herbal baths were found. Three babies treated this way died. The majority of caregivers sought care at a health facility. However, 67 (44%) sought care only after their babies were ill for ≥7 days, suggesting the influence of a period of confinement on health seeking. More than half, 94 (61.4%), of the facilities visited referred patients to destination hospitals without giving any treatment. Delayed care-seeking was associated with a low birth weight, using home remedies, and a maternal age of ≥30 years. Altogether, 12 neonates (7.8%) died, consisting of three males and nine females (p = 0.018). Socio-cultural factors strongly influence health seeking behavior and the health outcome of neonates in this setting. There appeared to be a limited repertoire of interventions for treating neonatal disease in primary care. Full article
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15 pages, 648 KiB  
Review
Harnessing T Cells to Target Pediatric Acute Myeloid Leukemia: CARs, BiTEs, and Beyond
by Rebecca Epperly, Stephen Gottschalk and Mireya Paulina Velasquez
Children 2020, 7(2), 14; https://doi.org/10.3390/children7020014 - 17 Feb 2020
Cited by 17 | Viewed by 6911
Abstract
Outcomes for pediatric patients with acute myeloid leukemia (AML) remain poor, highlighting the need for improved targeted therapies. Building on the success of CD19-directed immune therapy for acute lymphocytic leukemia (ALL), efforts are ongoing to develop similar strategies for AML. Identifying target antigens [...] Read more.
Outcomes for pediatric patients with acute myeloid leukemia (AML) remain poor, highlighting the need for improved targeted therapies. Building on the success of CD19-directed immune therapy for acute lymphocytic leukemia (ALL), efforts are ongoing to develop similar strategies for AML. Identifying target antigens for AML is challenging because of the high expression overlap in hematopoietic cells and normal tissues. Despite this, CD123 and CD33 antigen targeted therapies, among others, have emerged as promising candidates. In this review we focus on AML-specific T cell engaging bispecific antibodies and chimeric antigen receptor (CAR) T cells. We review antigens being explored for T cell-based immunotherapy in AML, describe the landscape of clinical trials upcoming for bispecific antibodies and CAR T cells, and highlight strategies to overcome additional challenges facing translation of T cell-based immunotherapy for AML. Full article
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13 pages, 236 KiB  
Article
Prevalence and Selected Sociodemographic of Movement Behaviors in Schoolchildren from Low- and Middle-Income Families in Nanjing, China: A Cross-Sectional Questionnaire Survey
by Si-Tong Chen and Jin Yan
Children 2020, 7(2), 13; https://doi.org/10.3390/children7020013 - 13 Feb 2020
Cited by 30 | Viewed by 4223
Abstract
Background: To investigate the prevalence of meeting the 24-h movement guidelines and its sociodemographic correlates in youth from low- and middle-income families (LMIFs) in Nanjing, China. Methods: Cross-sectional data on moderate to vigorous physical activity (MVPA), screen time (ST), and sleep (SLP) were [...] Read more.
Background: To investigate the prevalence of meeting the 24-h movement guidelines and its sociodemographic correlates in youth from low- and middle-income families (LMIFs) in Nanjing, China. Methods: Cross-sectional data on moderate to vigorous physical activity (MVPA), screen time (ST), and sleep (SLP) were collected using the Chinese version of Health Behavior School-Aged Children questionnaire among grade schoolchildren of 4th–12th (mean age 13.6 years). The prevalence of meeting the 24-h movement guidelines was in line with the recommendations of the Canadian 24-h movement guidelines. Generalized linear models were used to explore the relationships of correlates with the prevalence of meeting the movement guidelines. Results: The prevalence of meeting the MVPA, ST, and SLP guidelines and their combination was 9.9%, 65.2%, 37.2%, and 2.9%, respectively. As for the prevalence of meeting the MVPA guidelines, boys, younger schoolchildren, and those living in urban areas had a higher chance of meeting them. With regards to the prevalence of meeting the ST guidelines, girls, urban schoolchildren, and the oldest participants were more likely to meet the guidelines. Residential areas and grades were two correlates of meeting the SLP guidelines as well as 24-h movement guidelines. Conclusions: The majority of schoolchildren from LMIFs in Nanjing, China had unhealthy movement behaviors. This concerning situation was mainly predicted by schoolchildren’s grades, indicating older schoolchildren exhibited lower levels of movement behavior. Improved studies are encouraged to identify the correlates of movement behaviors in schoolchildren, which in turn designs and implements effective interventions. Full article
(This article belongs to the Section Global Pediatric Health)
15 pages, 3983 KiB  
Review
New and Emerging Targeted Therapies for Pediatric Acute Myeloid Leukemia (AML)
by Jing Chen and Chana L. Glasser
Children 2020, 7(2), 12; https://doi.org/10.3390/children7020012 - 10 Feb 2020
Cited by 24 | Viewed by 9375
Abstract
The relapse rate for children with acute myeloid leukemia (AML) remains high despite advancements in risk classification, multi-agent chemotherapy intensification, stem cell transplantation, and supportive care guidelines. Prognosis for this subgroup of children with relapsed/refractory AML remains poor. It is well known that [...] Read more.
The relapse rate for children with acute myeloid leukemia (AML) remains high despite advancements in risk classification, multi-agent chemotherapy intensification, stem cell transplantation, and supportive care guidelines. Prognosis for this subgroup of children with relapsed/refractory AML remains poor. It is well known that the ceiling of chemotherapy intensification has been reached, limited by acute and chronic toxicity, necessitating alternative treatment approaches. In the last several years, our improved understanding of disease biology and critical molecular pathways in AML has yielded a variety of new drugs to target these specific pathways. This review provides a summary of antibody drug conjugates (ADCs), small molecule inhibitors, and tyrosine kinase inhibitors with an emphasis on those that are currently under clinical evaluation or soon to open in early phase trials for children with relapsed/refractory AML. Full article
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11 pages, 243 KiB  
Review
Advances in Pediatric Acute Promyelocytic Leukemia
by Shannon E. Conneely and Alexandra M. Stevens
Children 2020, 7(2), 11; https://doi.org/10.3390/children7020011 - 2 Feb 2020
Cited by 23 | Viewed by 6672
Abstract
Acute promyelocytic leukemia (APL) is a rare disease accounting for only 5%–10% of pediatric acute myeloid leukemia (AML) and fewer than 1000 cases occur annually in the United States across all age groups. Characterized by t (15; 17), with a resultant PML-RARA gene [...] Read more.
Acute promyelocytic leukemia (APL) is a rare disease accounting for only 5%–10% of pediatric acute myeloid leukemia (AML) and fewer than 1000 cases occur annually in the United States across all age groups. Characterized by t (15; 17), with a resultant PML-RARA gene fusion driving leukemia development, advances in therapy have improved outcomes for APL significantly in the past several decades, now making APL the most curable form of AML in both children and adults. Cure rates in APL are now comparable to pediatric B-lymphoid leukemias. The success of APL treatment is due, in part, to the breadth of understanding of the driver PML-RARA mutation as well as collaborative efforts to quickly introduce and maximize the benefit of new therapies. Here, we review the presentation, clinical features, pathogenesis, and treatment advances in pediatric APL. Full article
11 pages, 264 KiB  
Article
Hypovitaminosis D and Cardiometabolic Risk Factors in Adolescents with Severe Obesity
by Teodoro Durá-Travé, Fidel Gallinas-Victoriano, Diego Mauricio Peñafiel-Freire, María Urretavizcaya-Martinez, Paula Moreno-González and María Jesús Chueca-Guindulain
Children 2020, 7(2), 10; https://doi.org/10.3390/children7020010 - 1 Feb 2020
Cited by 9 | Viewed by 4256
Abstract
Background/Objectives. Obesity is associated with cardiometabolic risk factors and with Vitamin D deficiency. The aim of this study was to examine the relationship between 25(OH)D concentrations and cardiometabolic risk factors in adolescents with severe obesity. Subjects/Methods. A cross-sectional clinical assessment (body mass index, [...] Read more.
Background/Objectives. Obesity is associated with cardiometabolic risk factors and with Vitamin D deficiency. The aim of this study was to examine the relationship between 25(OH)D concentrations and cardiometabolic risk factors in adolescents with severe obesity. Subjects/Methods. A cross-sectional clinical assessment (body mass index, fat mass index, fat-free mass index, waist-to-height ratio, and blood pressure) and metabolic study (triglycerides, total cholesterol, HDL-C, LDL-C, glucose, insulin, HOMA-IR, leptin, calcium, phosphorous, calcidiol, and PTH) were carried out in 236 adolescents diagnosed with severe obesity (BMI z-score > 3.0, 99th percentile), aged 10.2–15.8 years. The criteria of the US Endocrine Society were used for the definition of Vitamin D status. Results. Subjects with Vitamin D deficiency had significantly elevated values (p < 0.05) for BMI z-score, waist circumference, waist z-score, body fat percentage, fat mass index, systolic and diastolic blood pressure, total cholesterol, triglycerides, LDL-C, insulin, HOMA-IR, leptin, and PTH than subjects with normal Vitamin D status. There was a significant negative correlation (p < 0.05) of serum 25(OH)D levels with body fat percentage, FMI, systolic BP, total cholesterol, triglyceride, LDL-C, glucose, insulin, HOMA-IR, leptin, and PTH. Conclusions. Low Vitamin D levels in adolescents with severe obesity were significantly associated with some cardiometabolic risk factors, including body mass index, waist circumference, fat mass index, high blood pressure, impaired lipid profile, and insulin resistance. Full article
6 pages, 2853 KiB  
Brief Report
Bowel Dilatation on Initial Plane Abdominal Radiography May Help to Assess the Severity of Necrotizing Enterocolitis in Preterm Infants
by Zlatan Zvizdic, Irmina Sefic Pasic, Amra Dzananovic, Nedzad Rustempasic, Emir Milisic, Asmir Jonuzi and Semir Vranic
Children 2020, 7(2), 9; https://doi.org/10.3390/children7020009 - 23 Jan 2020
Cited by 10 | Viewed by 7204
Abstract
Background: Necrotizing enterocolitis (NEC) is the most common life-threatening gastrointestinal emergency associated with prematurity. Timely diagnosis and adequate treatment are crucial to reduce the morbidity and mortality of the affected infants. The aim of this study was to evaluate the diagnostic yield of [...] Read more.
Background: Necrotizing enterocolitis (NEC) is the most common life-threatening gastrointestinal emergency associated with prematurity. Timely diagnosis and adequate treatment are crucial to reduce the morbidity and mortality of the affected infants. The aim of this study was to evaluate the diagnostic yield of bowel dilatation on plane abdominal radiography (AR) in the early diagnosis and NEC severity in preterm infants. Methods: We retrospectively reviewed initial ARs of 50 preterm infants with NEC ≥ stage II admitted to the neonatal intensive care unit (NICU) in a tertiary-care hospital. The largest bowel loops diameters (AD), the latero-lateral diameters of the peduncle of the first lumbar vertebra (L1), and the distance of the upper edge of the first lumbar vertebra and the lower edge of the second one, including the disc space (L1–L2), were measured. All anteroposterior ARs were done in a supine projection on the day of onset of the initial symptoms of NEC. Results: Preterm infants with surgical NEC showed a statistically significant increase in the AD/L1 ratio (p < 0.001) and AD/L1-L2 ratio (p < 0.001) compared with preterm infants with medical NEC. We found no significant association between the site of the most distended bowel loop and the severity of NEC (p > 0.05). Conclusion: Bowel loop distension on initial AR may serve as an additional diagnostic tool in the early diagnosis and severity of stages II/III NEC. Further prospective clinical studies should validate the results from this study. Full article
(This article belongs to the Section Pediatric Surgery)
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