Determinants of Subtype-Selectivity of the Anthelmintic Paraherquamide A on Nicotinic Acetylcholine Receptors.

The anthelmintic paraherquamide A acts selectively on the nematode L-type nicotinic acetylcholine receptors (nAChRs), but the mechanism of its selectivity is unknown. This study targeted the basis of paraherquamide A selectivity by determining an X-ray crystal structure of the acetylcholine binding protein (AChBP), a surrogate nAChR ligand-binding domain, complexed with the compound and by measuring its actions on wild-type and mutant nematodes and functionally expressed nAChRs. Paraherquamide A showed a higher efficacy for the levamisole-sensitive [L-type (UNC-38/UNC-29/UNC-63/LEV-1/LEV-8)] nAChR than the nicotine-sensitive [N-type (ACR-16)] nAChR, a result consistent with in vivo studies on wild-type worms and worms with mutations in subunits of these two classes of receptors.

Infantile Kaposiform hemangioendothelioma in a female patient complicated with severe obstructed jaundice: a case report.

Background: Kaposiform hemangioendothelioma (KHE) is a rare locally aggressive vascular neoplasm that occurs mainly in the pediatric population. KHE usually originates just underneath the skin and affects deeper tissues through infiltrative growth; however, visceral tissue involvement is quite rare.

Case Presentation: An 8-month-old girl with jaundice and acholic stool was referred to our hospital for further evaluation of a hepatoduodenal ligament tumor.

Bacterial meningitis due to the group in children with cerebrospinal fluid leak.

The group constitutes a part of the oral flora in humans and has been reported to cause infective endocarditis, brain abscesses, sepsis, pneumonia, and peritonitis. However, the group rarely causes meningitis in children. We experienced a case of bacterial meningitis due to the group in a 14-year-old girl with Gorham-Stout disease undergoing treatment with sirolimus for skull base osteolysis and cerebrospinal fluid (CSF) leak.

Neurogenic bladder as a lurking complication in Moebius syndrome.

Moebius syndrome (MBS) is a congenital disorder characterized by facial and abducens palsy, sometimes accompanied with other cranial nerve palsies and comorbid conditions. Anatomical anomalies of the brainstem are assumed to be major etiologies of MBS. Its phenotypic presentation can be variable.

Challenge for management without tracheostomy tube after laryngo-tracheal separation in children with neurological disorders.

Objectives: The present study analyzed surgical outcomes of laryngotracheal separation (LTS) in children with neurological disorders. The purpose of this study was to investigate respiratory impairment and severe complications after LTS in children, and identify the possibility of permanent tracheostomy without a tracheostomy tube as the safest respiratory management method.

Methods: Twenty-eight patients (male:female = 16:12) with neurological disorders (6 months to 32 years) who underwent LTS between January 2012 and April 2018 were reviewed.

A case of neonatal cytomegalovirus infection with severe thrombocytopenia that was successfully managed with empiric antiviral therapy.

Antiviral therapy against cytomegalovirus (CMV) infection is indicated for symptomatic infection in the fetus and premature neonates. In mature neonates, the benefit of antiviral therapy for severe CMV infection remains controversial. Additionally, when diagnosing symptomatic CMV disease occurring during the early neonatal period, it is difficult to differentiate between congenital and acquired infections.

Low-dose l-isoproterenol versus salbutamol in hospitalized pediatric patients with severe acute exacerbation of asthma: A double-blind, randomized controlled trial.

Background: Although the guidelines in most countries do not recommend continuous inhalation of l-isoproterenol to treat pediatric patients with acute severe exacerbation of asthma, lower dose of l-isoproterenol has been widely used in Japan. To determine whether the efficacy of low-dose l-isoproterenol was superior to that of salbutamol, we conducted a double-blind, randomized controlled trial.

Methods: Hospitalized patients aged 1-17 years were eligible if they had severe asthma exacerbation defined by the modified pulmonary index score (MPIS).

Efficacy and safety of sirolimus treatment for intractable lymphatic anomalies: A study protocol for an open-label, single-arm, multicenter, prospective study (SILA).

Introduction: Lymphatic anomalies (LAs) refer to a group of diseases involving systemic dysplasia of lymphatic vessels. These lesions are classified as cystic lymphatic malformation (macrocystic, microcystic or mixed), generalized lymphatic anomaly, and Gorham-Stout disease. LAs occur mainly in childhood, and present with various symptoms including chronic airway problems, recurrent infection, and organ disorders.

Clinical Utility of the Modified Pulmonary Index Score as an Objective Assessment Tool for Acute Asthma Exacerbation in Children.

Introduction: The Modified Pulmonary Index Score (MPIS) was developed as an objective assessment tool for acute asthma exacerbation in children. Although it is considered reliable, there are no known studies of its clinical utility. The objective of this study was to evaluate the validity of the MPIS for children with acute asthma in a clinical setting.

Independent risk factors for recurrence of apparent life-threatening events in infants.

Unlabelled: The risk factors for recurrent apparent life-threatening event (ALTE) are unclear although the risk of recurrent ALTE is an important consideration for the management of ALTE patients. This study aimed to identify the risk factors for recurrent ALTE. We conducted a secondary analysis of the data from a single center retrospective cohort study in Japan conducted from March 2002 to January 2012, which included children diagnosed with ALTE at a pediatric emergency department (ED) in Tokyo.

Severe and Rapid Progression in Very Early-Onset Chronic Granulomatous Disease-Associated Colitis.

Purpose: Chronic granulomatous disease (CGD) is a primary immunodeficiency disease that leads to recurrent infection and hyper-inflammation, occasionally represented by CGD-associated colitis (CGD colitis). Although clinical symptoms of CGD colitis mimic those of ulcerative colitis (UC), there is no reliable standard measurement of disease activity or standard therapeutic strategy for CGD colitis. Here, we examined the clinical manifestation of CGD colitis based on severity using a noninvasive measure of disease activity, the Pediatric Ulcerative Colitis Activity Index (PUCAI), which has been validated and widely used for pediatric UC.

Modified pulmonary index score was sufficiently reliable to assess the severity of acute asthma exacerbations in children.

Background: The Modified Pulmonary Index Score (MPIS) was developed as an indicator of the severity of acute asthma in children. The objective of this study is to evaluate the reliability and validity of the MPIS for children with acute asthma, including those five years or younger in age.

Methods: We evaluated the inter-rater reliability and internal consistency of the MPIS by having at least two trained physicians and a nurse-each of whom was blinded to the others' scores-simultaneously examine inpatients with asthma exacerbation and rate them according to the MPIS.

Interstitial lung disease with multiple microgranulomas in chronic granulomatous disease.

Background: Chronic granulomatous disease (CGD) is a primary immunodeficiency disease that is characterized by susceptibility to bacterial and fungal infections. CGD patients also suffer from immune regulatory disorders, such as CGD-associated bowel inflammation with granuloma, which could be caused by excessive inflammation without demonstrable infection.

Purpose: We investigated the clinical manifestation of interstitial lung disease (ILD) resulting from excessive inflammation in X-linked CGD patients.

Cricopharyngeal achalasia treated with myectomy and post-operative high-resolution manometry.

Cricopharyngeal achalasia is an uncommon cause of dysphagia in neonates or children. A nine-year-old female patient was referred to us with a long history of dysphagia, recurrent pulmonary infection and growth stunting. A gastrostomy was introduced to improve her nutritional condition and to minimize potential inflammation in the pharynx.