Increasing Access to Undesignated Asthma Medication in Illinois.

Asthma is common among the pediatric population. Exacerbations of this chronic condition can lead to emergency department visits and hospitalizations, resulting in time away from school. Children spend the majority of their day at school, where they may need access to quick relief medication to treat respiratory distress.

Safety, Tolerability, and EEG-Based Target Engagement of STP1 (PDE3,4 Inhibitor and NKCC1 Antagonist) in a Randomized Clinical Trial in a Subgroup of Patients with ASD.

This study aimed to evaluate the safety and tolerability of STP1, a combination of ibudilast and bumetanide, tailored for the treatment of a clinically and biologically defined subgroup of patients with Autism Spectrum Disorder (ASD), namely ASD Phenotype 1 (ASD-Phen1). We conducted a randomized, double-blind, placebo-controlled, parallel-group phase 1b study with two 14-day treatment phases (registered at clinicaltrials.gov as NCT04644003).

"I'd like more options!": Interviews to explore young people and family decision-making needs for pain management in juvenile idiopathic arthritis.

Background: Juvenile idiopathic arthritis (JIA) is a common pediatric rheumatic condition and is associated with symptoms such as joint pain that can negatively impact health-related quality of life. To effectively manage pain in JIA, young people, their families, and health care providers (HCPs) should be supported to discuss pain management options and make a shared decision. However, pain is often under-recognized, and pain management discussions are not optimal.

Main Principles of Vitrectomy Using Intraocular Tamponades - A Basic Course in Surgery.

This article is intended to clearly present the basic principles for the use of intraocular tamponades in vitreous/retinal surgery in the event of retinal detachment and other pathologies using additional video footage. It examines the various gases, silicone oils and perfluorocarbon liquids with their indications, administration and in particular intraoperative handling including pitfalls and complications. Characteristic animations show the principles of use in surgery in a comprehensible way.

Characterization of Plasma Protein Alterations in Pregnant and Postpartum Individuals Living With HIV to Support Physiologically-Based Pharmacokinetic Model Development.

Alterations in plasma protein concentrations in pregnant and postpartum individuals can influence antiretroviral (ARV) pharmacokinetics. Physiologically-based pharmacokinetic (PBPK) models can serve to inform drug dosing decisions in understudied populations. However, development of such models requires quantitative physiological information (e.

CT Assessment of Intraorbital Cable Movement of Electronic Subretinal Prosthesis in Three Different Surgical Approaches.

Purpose: Electronic retinal implants restore some visual perception in patients blind from retinitis pigmentosa. Eye movements cause mechanical stress in intraorbital power supply cables leading to cable breaks. By using computer tomography (CT) scans at the extreme positions of the four cardinal gaze directions, this study determined in vivo, which of three surgical routing techniques results in minimal bending radius variation and favors durability.

Harnessing formulation and clinical pharmacology knowledge for efficient pediatric drug development: Overview and discussions from M-CERSI pediatric formulation workshop 2019.

A pediatric formulation workshop entitled "Pediatric Formulations: Challenges of Today and Strategies for Tomorrow" was held to advance pediatric drug product development efforts in both pre-competitive and competitive environments. The workshop had four main sessions discussing key considerations of Formulation, Analytical, Clinical and Regulatory. This paper focuses on the clinical session of the workshop.

PERSEUS 24-month analysis: a prospective non-interventional study to assess the effectiveness of intravitreal aflibercept in routine clinical practice in Germany in patients with neovascular age-related macular degeneration.

Purpose: To evaluate the real-world effectiveness of intravitreal aflibercept injections in Germany in patients with neovascular age-related macular degeneration over 24 months.

Methods: PERSEUS was a prospective, non-interventional cohort study. The primary endpoint was the mean change in visual acuity (VA) from baseline.

Importance of continuous treatment with intravitreal aflibercept injections in patients with neovascular age-related macular degeneration-12-month post hoc analysis of the PERSEUS real-world evidence study.

Purpose: To investigate the influence of treatment regularity with intravitreal aflibercept injections (IVT-AFL injections) on visual acuity (VA) outcomes in patients with neovascular age-related macular degeneration (nAMD) enrolled in the PERSEUS trial who received at least 7 IVT-AFL injections during the first year.

Methods: This was a post hoc analysis of the PERSEUS trial, a prospective, non-interventional, multicenter cohort study, and included 370 patients with nAMD who had received ≥ 7 IVT-AFL injections during year 1. In addition to the prespecified subgroups of treatment-naïve and previously treated patients, results were compared between patients with regular (n = 209) and irregular (n = 161) treatment.

Restriction of eye motility in patients with RETINA IMPLANT Alpha AMS.

Purpose: To evaluate the motility of the eye in patients with the RETINA IMPLANT Alpha AMS.

Methods: Eye motility was determined in eight gaze directions in ten blind retinitis pigmentosa patients, who had received the RETINA IMPLANT Alpha AMS, before implantation of the subretinal implant and at six time-points up to one year after.

Results: The analysis of eye motility showed a restriction in the upgaze and gaze to the temporal side directly after surgery in eight of the nine patients included.

Concentration distribution of more than 100 drugs and metabolites in forensic hair samples.

Drug testing in hair can complement conventional blood and urine analysis as it enlarges the window of detection and may allow a differentiation of heavy from moderate or rare use. Databases of drug concentrations in biological matrices are a valuable support in interpreting analytical results. In forensic toxicology, several databases exist especially for blood/serum samples.

Exposure-Response Assessment in Pediatric Drug Development Studies Submitted to the US Food and Drug Administration.

Exposure-response (E-R) modeling provides a quantitative tool to leverage adult data to support pediatric trial design and drug approval. The pediatric E-R studies submitted to US Food and Drug Administration (FDA) between 2007 and 2018 were surveyed in the context of various types of trial designs supporting drug approval in the pediatric population. The applications of E-R evaluation in pediatric drug development programs are mainly focused on three areas: (i) supporting pediatric extrapolation when the E-R relationships are similar between the pediatric and adult populations; (ii) dose selection to balance the risk-benefit profile based on the change in efficacy and safety response with different exposure levels; and (iii) approval of a new formulation, new dosing regimen, or new route of administration, where E-R evaluation helps quantify the change in clinical response between the old and new strategies.

Extrapolation of Adult Efficacy to Pediatric Patients With Chemotherapy-Induced Nausea and Vomiting.

Chemotherapy-induced nausea and vomiting (CINV) is a common treatment-related adverse event that negatively impacts the quality of life of cancer patients. During pediatric drug development, extrapolation of efficacy from adult to pediatric populations is a pathway that can minimize the exposure of children to unnecessary clinical trials, improve efficiency, and increase the likelihood of success in obtaining a pediatric indication. The acceptability of the use of extrapolation depends on a series of evidence-based assumptions regarding the similarity of disease, response to intervention, and exposure-response relationships between adult and pediatric patients.

Aflibercept for Patients with Neovascular Age-Related Macular Degeneration in Routine Clinical Practice in Germany: Twelve-Month Outcomes of PERSEUS.

Purpose: To explore real-world effectiveness of intravitreal aflibercept injection (IAI) for neovascular age-related macular degeneration (nAMD) in Germany.

Design: A 24-month, prospective, noninterventional, noncontrolled, multicenter observational cohort study.

Participants: Patients (n = 848) with nAMD treated with IAI.

Determination of hydroxy metabolites of cocaine from hair samples and comparison with street cocaine samples.

Drugs which are commonly smoked or sniffed (e.g. cocaine), can contaminate hair through smoke or dust; therefore testing for metabolites, especially hydroxy metabolites, is highly recommended.

Implantation, removal and replacement of subretinal electronic implants for restoration of vision in patients with retinitis pigmentosa.

Purpose Of Review: The purpose of this review is to provide an update on the efforts to restore vision through subretinal implants in patients with degenerative retinal diseases. In addition to the current technique and its latest improvements, it will focus on the surgical technique of implantation as well as explantation and reimplantation.

Recent Findings: The durability of the current subretinal implant RETINA IMPLANT Alpha AMS has increased substantially compared with the predecessor model RETINA IMPLANT Alpha IMS.

Optical Coherence Tomography in Patients With the Subretinal Implant Retina Implant Alpha IMS.

Background And Objective: The aim of this study was to assess changes in retinal structure and thickness after subretinal implantation of the Retina Implant Alpha IMS (Retina Implant AG, Reutlingen, Germany).

Patients And Methods: Spectral-domain optical coherence tomography (SD-OCT) imaging was performed to assess the structure and thickness of the retina anterior to the microphotodiode array preoperatively, within 6 weeks and 6 months ± 1 month after implantation. Thickness measurements were performed using the distance tool of the built-in software.

[Time Course of Changes in Visual Acuity After a Single Injection of Aflibercept or Ranibizumab in Neovascular Age-related Macular Degeneration - Analysis of Aggregated Real Life Data].

Treatment of neovascular age-related macular degeneration (nvAMD) under real life conditions may differ from controlled prospective trials with respect to the number of injections and long term preservation of visual acuity. In many instances, intervals for controls and re-injection cannot be maintained as frequently as required. This case series examines actual intervals for control and re-injection visits, in order to estimate how prolonged intervals have an impact on momentary visual acuity and how long term visual outcome is affected.

[Real-life Data on the Treatment of Diabetic Macular Oedema in Germany].

Controlled prospective clinical trials on the treatment of diabetic macular oedema (DME) using anti-VEGF compounds show very good results in visual acuity gain over several years. To date, only limited data are available from comparable studies under real-life settings in clinical routine. However, real-life data from other indications for anti-VEGF treatment suggest that, in clinical routine, gain in visual acuity is less pronounced and cannot be maintained over a longer period of time, which is related to the significantly lower number of injections administered in clinical routine.

[The Conundrum of Relationships in the Multivariate Dataset of nvAMD Treatment].

The evaluation of relationships in clinical case series is complicated by statistical pitfalls, one of which is spurious correlation. In this case, one or more potential factors are actually not related to a target measure, yet it may be wrongly inferred that they are. This may be due to either coincidence or the presence of a one or more confounding unregarded factors.

Development of Drug Therapies for Newborns and Children: The Scientific and Regulatory Imperatives.

Pediatric legislation has generated information about the efficacy, safety, and dosing of more than 600 products in children. Extrapolation of adult efficacy data has been an integral part of pediatric drug development. Advances in our understanding of physiology and pharmacology have improved the approach to pediatric dose selection.

[Real-life Evidence on Treatment Outcomes of Neovascular Age-related Macular Degeneration in Germany].

Treatment of neovascular age-related macular degeneration (AMD) with anti-vascular endothelial growth factor agents has been improved since its introduction in Germany. Several observational studies have demonstrated an increased frequency of injections in parallel with improved visual outcomes during the first year of treatment over the last 7 years. These results are reflected in several case series.

Determination of hydroxy metabolites of cocaine in hair samples for proof of consumption.

Although hair is widely used to identify drug use, there is a risk of false positives due to environmental contamination. This especially applies to cocaine (COC). Several strategies such as detection of norcocaine (NCOC) or cocaethylene, metabolite concentration ratios or intricate washing procedures have been proposed to differentiate actual use from contamination.