scholarly journals Cellular Therapy for the Treatment of Paediatric Respiratory Disease

2021 ◽  
Vol 22 (16) ◽  
pp. 8906 ◽  
Author(s):  
Laura C. Brennan ◽  
Andrew O’Sullivan ◽  
Ronan MacLoughlin

Respiratory disease is the leading cause of death in children under the age of 5 years old. Currently available treatments for paediatric respiratory diseases including bronchopulmonary dysplasia, asthma, cystic fibrosis and interstitial lung disease may ameliorate symptoms but do not offer a cure. Cellular therapy may offer a potential cure for these diseases, preventing disease progression into adulthood. Induced pluripotent stem cells, mesenchymal stromal cells and their secretome have shown great potential in preclinical models of lung disease, targeting the major pathological features of the disease. Current research and clinical trials are focused on the adult population. For cellular therapies to progress from preclinical studies to use in the clinic, optimal cell type dosage and delivery methods need to be established and confirmed. Direct delivery of these therapies to the lung as aerosols would allow for lower doses with a higher target efficiency whilst avoiding potential effect of systemic delivery. There is a clear need for research to progress into the clinic for the treatment of paediatric respiratory disease. Whilst research in the adult population forms a basis for the paediatric population, varying disease pathology and anatomical differences in paediatric patients means a paediatric-centric approach must be taken.

2021 ◽  
Vol 22 (1) ◽  
Author(s):  
Tak Kyu Oh ◽  
Hyoung-Won Cho ◽  
Hun-Taek Lee ◽  
In-Ae Song

Abstract Background Quality of life following extracorporeal membrane oxygenation (ECMO) therapy is an important health issue. We aimed to describe the characteristics of patients who developed chronic respiratory disease (CRD) following ECMO therapy, and investigate the association between newly diagnosed post-ECMO CRDs and 5-year all-cause mortality among ECMO survivors. Methods We analyzed data from the National Health Insurance Service in South Korea. All adult patients who underwent ECMO therapy in the intensive care unit between 2006 and 2014 were included. ECMO survivors were defined as those who survived for 365 days after ECMO therapy. Chronic obstructive pulmonary disease (COPD), asthma, interstitial lung disease, lung cancer, lung disease due to external agents, obstructive sleep apnea, and lung tuberculosis were considered as CRDs. Results A total of 3055 ECMO survivors were included, and 345 (11.3%) were newly diagnosed with CRDs 365 days after ECMO therapy. The prevalence of asthma was the highest at 6.1% (185). In the multivariate logistic regression, ECMO survivors who underwent ECMO therapy for acute respiratory distress syndrome (ARDS) or respiratory failure had a 2.00-fold increase in post-ECMO CRD (95% confidence interval [CI]: 1.39 to 2.89; P < 0.001). In the multivariate Cox regression, newly diagnosed post-ECMO CRD was associated with a 1.47-fold (95% CI: 1.17 to 1.86; P = 0.001) higher 5-year all-cause mortality. Conclusions At 12 months after ECMO therapy, 11.3% of ECMO survivors were newly diagnosed with CRDs. Patients who underwent ECMO therapy for ARDS or respiratory failure were associated with a higher incidence of newly diagnosed post-ECMO CRD compared to those who underwent ECMO for other causes. Additionally, post-ECMO CRDs were associated with a higher 5-year all-cause mortality. Our results suggest that ECMO survivors with newly diagnosed post-ECMO CRD might be a high-risk group requiring dedicated interventions.


Obesity Facts ◽  
2021 ◽  
pp. 1-9
Author(s):  
Claudia Pileggi ◽  
Rosa Papadopoli ◽  
Caterina De Sarro ◽  
Carmelo Giuseppe Angelo Nobile ◽  
Maria Pavia

<b><i>Introduction:</i></b> Several studies in the adult population have shown that obesity is an independent risk factor for elevated intraocular pressure (IOP), whereas data in the paediatric population are sparse and controversial. The purpose of the present study is to investigate the relationship between body mass index (BMI), blood pressure (BP), and IOP in healthy school children. <b><i>Methods:</i></b> The survey was conducted among a random sample of 8-year-old Italian students. Data were collected on their health status and behaviours related to obesity (physical activity, food and drinking habits, etc.). Physical examinations, conducted at school, included measurements of height, weight, BP, and IOP. <b><i>Results:</i></b> Five hundred and seventy-six subjects were recruited (92.8% response rate); 42.4% were overweight or obese, 58.9% consumed inadequate daily servings of fruit and vegetables, and 87.5% were involved in sedentary activities. Elevated BP/hypertension (HTN) affected 3.6% and high IOP was revealed in 12.5% of the children. In the multivariate analysis, elevated BP/HTN was the only significant determinant of ocular HTN (OR 5.36, 95% CI 1.95–14.73, <i>p</i> = 0.001). <b><i>Conclusions:</i></b> Our results show that high IOP affects 12.5% of 8-year-old school children and appears to be associated with high BP related to a high BMI.


2012 ◽  
Vol 36 (4) ◽  
pp. 137-139 ◽  
Author(s):  
Nuno Rodrigues Silva ◽  
Ângela Venâncio

SummaryDespite extensive use of valproate in neurology and psychiatry, valproate-induced encephalopathy is a rarely reported complication. Although initially reported in the paediatric population, case reports are growing in the adult population.Clinicians need to be aware of this potentially life-threatening complication. We report a case in a 37-year-old woman with bipolar I disorder with previously uncomplicated valproate use, who developed encephalopathy when valproate was restarted a few years later. The patient has provided consent for publication.


Thorax ◽  
2017 ◽  
Vol 73 (6) ◽  
pp. 575-577 ◽  
Author(s):  
Alan Lunt ◽  
Lucy Mortimer ◽  
David Rees ◽  
Sue Height ◽  
Swee Lay Thein ◽  
...  

To detect and characterise different phenotypes of respiratory disease in children and young adults with sickle cell disease (SCD), 11 lung function and haematological biomarkers were analysed using k-means cluster analysis in a cohort of 114 subjects with SCD aged between 5 and 27 years. Three clusters were detected: cluster 1 had elevated pulmonary capillary blood volume, mixed obstructive/restrictive lung disease, hypoxia and moderately severe anaemia; cluster 2 were older patients with restrictive lung disease; and cluster 3 were younger patients with obstructive lung disease, elevated serum lactate dehydrogenase and bronchodilator reversibility. These results may inform more personalised management strategies to improve outcomes.


Discoveries ◽  
2020 ◽  
Vol 8 (4) ◽  
pp. e119
Author(s):  
Shah Newaz Ahmed ◽  
◽  
Ratinder Jhaj ◽  
Ritendra Patidar ◽  
Mahendra Dangi ◽  
...  

Background: The Anatomical Therapeutic Chemical Classification / Defined Daily Dose (ATC/DDD) system recommended by World Health Organization is accepted worldwide as the standard method of quantification of drug consumption. However, owing to individual variation in body weight, the ATC/DDD system cannot be used for comparison across paediatric population. Objective: This study aimed to develop a novel metric system for standard quantification of antibiotic consumption in paediatric population. Method: The standard unit of drug quantification in adult population is DDD/100 patient days (PD). We conceived a new unit of DDD/1000 kg-days (KD) where KD is the product of the body weight and length of hospital stay of an individual patient. We simulated the quantification and comparison of drugs in a computer model of five virtual paediatric hospitals (H1 to H5, n=100, 200, 100, 100, 100 respectively). We re-applied the metric system on two, real world, hospital-based, time cohorts (TC) (TC18, n=38 and TC19, n=47) of 2 weeks each, in two consecutive years. Results: The body weights (mean±SD) in H1-H5 were 5.7±3.0, 5.7±2.8, 25.3±8.5, 20.6±11.7 and 19.8±11.4 kg, respectively. The antibiotic consumption in terms of DDD/100 PD and DDD/1000 KD in the five hospitals was 1.26, 1.20, 5.52, 4.41 and 2.00, and 2.24, 2.14, 2.22, 2.17 and 1.06 respectively. In TC18 and TC19, the mean body weight, DDD/100 PD and DDD/1000 KD were 12.24±13.17, 30.93, 20.34 and 19.51±12.28, 11.99, 6.23, respectively. Conclusion: DDD/1000 kg-days is a potential standard unit for drug quantification in paediatric population independent of weight distribution and size of the study sample. The universal application and comparison across diverse samples can generate useful information for resource allocation, anti-microbial stewardship, disease burden and drug use, and can help in taking policy decisions to improve healthcare delivery in the paediatric population.


2021 ◽  
Vol 14 (8) ◽  
pp. e243826
Author(s):  
Omar Nasher ◽  
Naved Alizai

Enterocolic lymphocytic phlebitis (ELP) is a rare pathology, usually seen in the adult population. The condition affects the venous vasculature of the intestine sparing the arteries and lymphatics. It is commonly identified histologically after surgical resection making the preoperative diagnosis a clinical challenge. As the condition can lead to intestinal ischaemia and necrosis, surgical resection of the affected segment is the treatment of choice. We report the case of a 9-year-old girl who presented with a 2-day history of lower abdominal pain and anorexia. The initial diagnosis of appendicitis was made clinically and the patient underwent a diagnostic laparoscopy . The appendix was macroscopically normal and there were no other obvious intra-abdominal pathologies to account for her presenting complaints . The appendix was removed, as per the preoperative discussion and consent from parents. She recovered well and was discharged home the following day. The histological examination of the appendix demonstrated ELP. We describe this rare clinical entity in a child especially in view of the scarce published literature in the paediatric population.


2002 ◽  
Vol 30 (3) ◽  
pp. 275-282 ◽  
Author(s):  
L. Silvestri ◽  
R. E. Sarginson ◽  
J. Hughes ◽  
M. Milanese ◽  
D. Gregori ◽  
...  

A prospective observational cohort study was undertaken with two endpoints: (1) to compare the time cut-off of 48h and the carrier state criterion for classifying lower airway infections in adult and paediatric long-term ventilated patients, and (2) to evaluate the potential of optimized time cut-offs for characterizing imported and ICU-acquired lower airway infections. All patients admitted to the general and paediatric intensive care units and expected to require mechanical ventilation for a period 3 days were enrolled. Surveillance cultures of throat and rectum were obtained on admission and thereafter twice weekly to distinguish micro-organisms that were imported into the unit from those acquired during the stay on the unit. A total of 130 adults and 400 children were studied. In the adult population, 70% of lower airway infections were classified as ICU-acquired by the 48 h cut-off and 48% by the criterion of carriage; on the paediatric ICU the percentages were 65% and 20%, respectively. To separate imported from ICU-acquired infections, eight days was optimal in the adult population and 10 days in the paediatric population. Sensitivity, specificity, positive predictive value and negative predictive value for a time cut-off of eight days for adults were 86, 77, 80, 83%, respectively, and using 10 days for children were 87, 62, 90, 56%, respectively. The use of the 48 h cut-off rule classifies patients as having nosocomial pneumonia, when in fact the infections are commonly caused by microorganisms carried in by the patients. In contrast, using the carriage method, the proportion of lung infections due to nosocomial bacteria was relatively small and was a late phenomenon. Although in prolonging the time cut-off the difference between the two types of classification was shorter, time cut-offs were still found to be unreliable for distinguishing imported from unit-acquired lower airway infections.


2020 ◽  
Vol 13 (10) ◽  
pp. e235662
Author(s):  
Gustavo M Cortez ◽  
Andre Monteiro ◽  
Benjamin Ludwig ◽  
Ricardo Hanel

Pilocytic astrocytomas comprise the most common central nervous system tumour during childhood and have an excellent response to surgical treatment in this population. The tumour incidence decreases with age, whereas more aggressive behaviour tends to increase. Haemorrhage as a presenting feature of pilocytic astrocytomas is a rare phenomenon, especially in the adult population. We present a case of a 55-year-old patient with progressive headaches and dizziness. MRI confirmed a sellar and predominantly retrochiasmal suprasellar lesion with heterogeneous signal, enhancement and blood products. Management via transsphenoidal approach was performed, and histopathology revealed the unexpected diagnosis of haemorrhagic pilocytic astrocytoma. Haemorrhagic pilocytic astrocytoma is an infrequent entity in the adult population and it is essential to recognise the peculiarities regarding diagnostic evaluation and management, which differ from the paediatric population. During adulthood, this tumour carries an overall unfavourable prognosis, with higher rates of progression and recurrence.


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