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Thanks to the synergistic action of a combined treatment of physical and instrumental therapy, despite the rare pathology and complex disability, the patient had important benefits in terms of performance and independence in daily activity.

Congenital pulmonary airway malformation (CPAM) is the most common prenatally-diagnosed lung malformation. This lesion, classified as macrocystic or microcystic, can lead to significant fetal compromise. Management options include observation, maternal antenatal steroid administration, and fetal surgical intervention. Current evidence suggests that microcystic (but not macrocystic) lesions and those with a cyst volume ratio (CVR) >1.6 are responsive to steroid therapy. The objective of this study was to identify patterns of prenatal steroid administration for the management of CPAMs and to identify characteristics of CPAMs prompting steroid administration.

An 18-question survey was distributed to obstetricians from the Pregnancy-Related Care Research Network (PRCRN) and the North American Fetal Therapy Network (NAFTNet), from January to April 2019, to capture antenatal steroid prescribing patterns.

Response rates were 28.3% (138/487) for PRCRN and 63.3% (19/30) for NAFTNet. Among PRCRN members, 16.8% administered prenatal steroids, with most (77.2%) doing so for both microcystic and macrocystic CPAMs; corresponding percentages for NAFTNet members were 90.9% and 52.6%. Two thirds (65.6%) of obstetricians who administer steroids do so for a CVR > 1.6, without evidence of mediastinal shift or hydrops fetalis.

There is a lack of consensus among obstetricians as to the CPAM characteristics that should prompt administration of prenatal steroids. Many surveyed obstetricians do not use cyst type or CVR to guide decision-making regarding steroid therapy.

There is a lack of consensus among obstetricians as to the CPAM characteristics that should prompt administration of prenatal steroids. Many surveyed obstetricians do not use cyst type or CVR to guide decision-making regarding steroid therapy.

The purpose of this study was to compare the outcomes of infants with giant omphalocele (GO) born in two different epochs over two decades at a single institution. Specifically, it examined whether the utilization of selective pulmonary vasodilators and extracorporeal membrane oxygenator (ECMO) in the management of pulmonary hypertension in the second epoch were associated with improved outcomes.

The medical records of all patients diagnosed with GO at a large children's hospital from January 1, 1996 to December 31, 2016 were reviewed and divided into two epochs. Patients were classified as having an isolated GO or GO with minor or major associated anomalies. GO was defined as a defect more than or equal to 5 cm in size and/or liver in the sac.

During the study period, 59 infants with GO were identified. The duration of invasive mechanical ventilation was significantly shorter among the survivors from the second epoch (p = 0.03), with none greater than seven days. There were no significant differences in the outcomes of survival to NICU discharge and length of stay (LOS) between infants in the two epochs.

Infants with GO who required invasive mechanical ventilation for more than seven days did not survive in the second epoch. Survival did not improve with uses of selective pulmonary vasodilators and ECMO. This information could be shared with families during prenatal and postnatal counselling to facilitate informed decision making regarding goals of care.

Infants with GO who required invasive mechanical ventilation for more than seven days did not survive in the second epoch. Survival did not improve with uses of selective pulmonary vasodilators and ECMO. This information could be shared with families during prenatal and postnatal counselling to facilitate informed decision making regarding goals of care.

Children born prematurely (<37 gestational weeks) are at risk for a variety of adverse medical events. They may experience ischemic and/or hemorrhagic events leading to negative neural sequelae. They are also exposed to repeated stressful experiences as part of life-saving care within the neonatal intensive care unit (NICU). These experiences have been associated with methylation of SLC6A4, a gene which codes for serotonin transport proteins, and is associated with anxiety, depression, and increased incidence of autism spectrum disorders.The purpose of this study was to examine the effects of altered serotonin levels on behavioral and neuroanatomical outcomes in a neonatal rodent model with or without exposure to hypoxic-ischemic (HI) injury.

Wistar rat pups were randomly assigned to either HI injury or sham groups. Pups within each group were treated with a chronic SSRI (Citalopram HBr) to simulate the effects of SLC6A4 methylation, or saline (NS). Subjects were assessed on behavioral tasks and neuropathologic indices.

HI injured subjects performed poorly on behavioral tasks. SSRI subjects did not display significantly greater anxiety. HI + SSRI subjects learned faster than HI+NS. Histologically, SSRI subjects had predominantly larger brain volumes than NS.

SSRI treated subjects without injury showed patterns of increased anxiety, consistent with theories of SLC6A4 methylation. The paradoxical trend to improved cognition in HI+SSRI subjects relative to HI alone, may reflect an unexpected SSRI neuroprotective effect in the presence of injury, and may be related to serotonin-induced neurogenesis.

SSRI treated subjects without injury showed patterns of increased anxiety, consistent with theories of SLC6A4 methylation. The paradoxical trend to improved cognition in HI+SSRI subjects relative to HI alone, may reflect an unexpected SSRI neuroprotective effect in the presence of injury, and may be related to serotonin-induced neurogenesis.

Late-onset neonatal sepsis (LONS) detection is problematic as no single examinations (blood culture, c-reactive protein (CRP), procalcitonin (PCT)) are reliable. Toll-like receptors (TLRs), which detect the presence of pathogen-associated molecular patterns is a promising novel biomarker, but less studied in LONS. This study aimed to determine neutrophils and monocytes TLR2 and TLR4 expression in LONS and their diagnostic value.

A cross-sectional study conducted in May and June 2017 involving 52 neonates with clinical late-onset (>72 hours of age) sepsis. We examine complete blood count, I/T ratio, CRP, PCT, as well as TLR2 and TLR4 expression to compared with blood culture as the gold standard. We classified cases into proven or unproven sepsis.

The incidence of LONS was 32.6% in the subjects. The expression of TLR2 was low in LONS, while TLR4 was high. TLR4 neutrophil expression has 88.2% sensitivity, 20% specificity, 34.9% positive predictive value (PPV), 77.8% negative predictive value (NPV), and an AUC of 0.541. TLR4 monocyte expression has 92.1% sensitivity, 11.4% specificity, 34% PPV, 80% NPV, and an AUC of 0.528. The AUC of CRP is increased from 0.608 to 0.843 after combination with TLR4, comparable with CRP + PCT (AUC 0.829).

The increase in TLR4 expression has good sensitivity but low specificity. TLR4 expression, in combination with CRP, could become a reliable biomarker for the diagnosis of LONS.

The increase in TLR4 expression has good sensitivity but low specificity. TLR4 expression, in combination with CRP, could become a reliable biomarker for the diagnosis of LONS.

Infection with Helicobacter pylori seems overrepresented in Parkinson's disease. Clinical observations suggest a suboptimal treatment effect of levodopa in Helicobacter positive patients.

Describe and explain the connection between a Helicobacter pylori infection of the upper gut and changes in pharmacokinetics of oral levodopa treatment in Parkinson's disease.

PubMed, Google Scholar, and Cross Reference search was done using the key words and combined searches Bioavailability, drug metabolism, dyskinesia, Helicobacter, L-dopa, levodopa, motor control, pharmacodynamics, pharmacokinetics, prevalence, unified Parkinson's disease rating scale.

The prevalence of Helicobacter pylori in Parkinson's disease patients is reported to be about 1.6-fold higher than in a control population in some studies. Helicobacter has therefore been assumed to be linked to Parkinson's disease, but the mechanism is unclear. As regards symptoms and treatment, patients with Parkinson's disease on levodopa therapy and with Helicobacter pylori infection display worse motor control than those without Helicobacter infection. Eradication of the infection improves levodopa response in Parkinson's disease, likely as a consequence of an increased oral pre-systemic bioavailability of levodopa, likely to be explained by reduced Helicobacter-dependent levodopa consumption in the stomach. In addition, small intestinal bacterial overgrowth may also have an impact on the therapeutic setting for levodopa treatment but is less well established.

Eradication of Helicobacter pylori improves levodopa bioavailability resulting in improved motor control. Eradication of Helicobacter should be considered in patients with poor symptomatic control and considerable motor fluctuations.

Eradication of Helicobacter pylori improves levodopa bioavailability resulting in improved motor control. Deferiprone Eradication of Helicobacter should be considered in patients with poor symptomatic control and considerable motor fluctuations.

ND0612 is a continuous, subcutaneous levodopa/carbidopa delivery system in development for patients with Parkinson's disease (PD) experiencing motor fluctuationsObjectiveEvaluate the efficacy and safety of two ND0612 dosing regimens in patients with PD.

This was a 28-day open-label study (NCT02577523) in PD patients with ≥2.5 hours/day of OFF time despite optimized treatment. Patients were randomized to treatment with either a 24-hour infusion (levodopa/carbidopa dose of 720/90 mg) or a 14-hour 'waking-day' infusion (levodopa/carbidopa dose of 538/68 mg plus a morning oral dose of 150/15 mg). Supplemental oral doses of levodopa were permitted for patients in both groups if required. In-clinic assessments of OFF time (primary endpoint) and ON time with or without dyskinesia were determined by a blinded rater over 8 hours (normalized to 16 hours).

A total of 38 patients were randomized and 33 (87%) completed the study. Compared to baseline, OFF time for the overall population was reduced by a least squares (LS) mean[95% CI] of 2.0[- 3.3, - 0.7] hours (p = 0.003). ON time with no/mild dyskinesia (no troublesome dyskinesia) was increased from baseline by a LS mean of 3.3[2.0, 4.6] hours (p < 0.0001), and ON time with moderate/severe dyskinesia was reduced by a LS mean of 1.2[- 1.8, - 0.5] hours (p≤0.001). Reduction in OFF time was larger in the 24-hour group (- 2.8[- 4.6, - 0.9] hours; p = 0.004) than in the 14-hour group (- 1.3[- 3.1, 0.5] hours; p = 0.16). Complete resolution of OFF time was observed in 42% (n = 8) of patients in the 24-hour group. Infusion site reactions were the most common adverse event.

This study demonstrates the feasibility and safety of continuous subcutaneous delivery of levodopa as a treatment for PD and provides preliminary evidence of efficacy.

This study demonstrates the feasibility and safety of continuous subcutaneous delivery of levodopa as a treatment for PD and provides preliminary evidence of efficacy.