Assessing psychosocial risk factors in children with Sickle Cell Disease.

Background: Individuals with Sickle Cell Disease (SCD) are a minoritized and marginalized community that have disparate health outcomes as a result of systemic racism and disease-related stigma. The purpose of this study was to determine the psychosocial risk factors for families caring for children with SCD at a pediatric SCD center through use of the Psychosocial Assessment Tool (PAT), a validated caregiver-report screener.

Methods: The PAT was administered annually during routine clinical visits and scored by the SCD Social Worker to provide tailored resources to families.

Impact of Neighborhood Disadvantage on Preventive and Acute Care Utilization in Sickle Cell Disease.

Background: Individuals with sickle cell disease (SCD) experience significant healthcare disparities; however, there is little known on the impact of psychosocial stressors and neighborhood disadvantage on preventive and acute care utilization in this population.

Procedure: A retrospective data collection was performed for all patients cared for at a comprehensive pediatric sickle cell center in the Midwest who had also completed the Psychosocial Assessment Tool (PAT), a validated caregiver-reported measurement of family psychosocial risk, from September 2021 through December 2022. Patient age, payor, primary language, frequency of acute and missed preventive care visits, as well as Area Deprivation Index (ADI) and Childhood Opportunity Index (COI) scores were collected for 256 patients.

Navigating pharmacophore space to identify activity discontinuities: A case study with BCR-ABL.

The exploration of chemical space is a fundamental aspect of chemoinformatics, particularly when one explores a large compound data set to relate chemical structures with molecular properties. In this study, we extend our previous work on chemical space visualization at the pharmacophoric level. Instead of using conventional binary classification of affinity (active vs inactive), we introduce a refined approach that categorizes compounds into four distinct classes based on their activity levels: super active, very active, active, and inactive.

Common mental health conditions and considerations in pediatric chronic kidney disease.

The mental health of youth with chronic kidney disease (CKD) has been increasingly recognized as an area of clinical need. The development of mental health concerns is influenced by a range of physiological, psychological, and environmental factors. Some of these factors are common across child development, but some are more unique to youth with CKD.

Towards a partial order graph for interactive pharmacophore exploration: extraction of pharmacophores activity delta.

This paper presents a novel approach called Pharmacophore Activity Delta for extracting outstanding pharmacophores from a chemogenomic dataset, with a specific focus on a kinase target known as BCR-ABL. The method involves constructing a Hasse diagram, referred to as the pharmacophore network, by utilizing the subgraph partial order as an initial step, leading to the identification of pharmacophores for further evaluation. A pharmacophore is classified as a 'Pharmacophore Activity Delta' if its capability to effectively discriminate between active vs inactive molecules significantly deviates (by at least δ standard deviations) from the mean capability of its related pharmacophores.

Understanding caregiver burden with accessing sickle cell care in the Midwest and their perspective on telemedicine.

Background: Survival for children with sickle cell disease (SCD) has improved significantly. However, patients with SCD still encounter several impediments to accessing adequate healthcare. Rural and medically underserved areas, such as parts of the Midwest, can exacerbate these barriers, separating children with SCD from subspecialists even further.

Psychosocial supports within pediatric nephrology practices: A pediatric nephrology research consortium survey.

Background: The landscape of available psychosocial services within pediatric nephrology care is poorly characterized. However, the effects of kidney disease on emotional health and health-related quality of life are well documented, as is the impact of social determinants of health on kidney disease outcomes. The objectives of this study were to assess pediatric nephrologists' perceptions of available psychosocial services and to elucidate inequities in access to psychosocial care.

MBNL-dependent impaired development within the neuromuscular system in myotonic dystrophy type 1.

Aims: Myotonic dystrophy type I (DM1) is one of the most frequent muscular dystrophies in adults. Although DM1 has long been considered mainly a muscle disorder, growing evidence suggests the involvement of peripheral nerves in the pathogenicity of DM1 raising the question of whether motoneurons (MNs) actively contribute to neuromuscular defects in DM1.

Methods: By using micropatterned 96-well plates as a coculture platform, we generated a functional neuromuscular model combining DM1 and muscleblind protein (MBNL) knock-out human-induced pluripotent stem cells-derived MNs and human healthy skeletal muscle cells.

New Pharmacophore Fingerprints and Weight-matrix Learning for Virtual Screening. Application to Bcr-Abl Data.

In this work, we propose to analyze the potential of a new type of pharmacophoric descriptors coupled to a novel feature transformation technique, called Weight-Matrix Learning (WML, based on a feed-forward neural network). The application concerns virtual screening on a tyrosine kinase named BCR-ABL. First, the compounds were described using three different families of descriptors: our new pharmacophoric descriptors, and two circular fingerprints, ECFP4 and FCFP4.

Caregiver experiences with accessing sickle cell care and the use of telemedicine.

Background: Sickle cell disease (SCD) is associated with a wide range of complications. However, a multitude of barriers prevent SCD patients from receiving adequate healthcare, including difficulties with transportation and lack of provider knowledge about disease sequelae. Importantly, studies have demonstrated the benefits of telemedicine in addressing barriers to healthcare.

Deciphering a Pharmacophore Network: A Case Study Using BCR-ABL Data.

This paper introduces a general method that can be used to create groups of pharmacophores to support their further in-depth analysis. A BCR-ABL molecular dataset was used to calculate graph edit distances between pharmacophores and led to their organization into a novel pharmacophore network. The application of a graph layout algorithm allowed us to discriminate between the pharmacophores associated with active compounds and those associated with inactive compounds.

Optogenetically controlled human functional motor endplate for testing botulinum neurotoxins.

Background: The lack of physiologically relevant and predictive cell-based assays is one of the major obstacles for testing and developing botulinum neurotoxins (BoNTs) therapeutics. Human-induced pluripotent stem cells (hiPSCs)-derivatives now offer the opportunity to improve the relevance of cellular models and thus the translational value of preclinical data.

Methods: We investigated the potential of hiPSC-derived motor neurons (hMNs) optical stimulation combined with calcium imaging in cocultured muscle cells activity to investigate BoNT-sensitivity of an in vitro model of human muscle-nerve system.

hiPSC-Derived Neurons Provide a Robust and Physiologically Relevant Platform to Test Botulinum Neurotoxins.

Botulinum neurotoxins (BoNTs) are zinc metalloproteases that block neurotransmitter release at the neuromuscular junction (NMJ). Their high affinity for motor neurons combined with a high potency have made them extremely effective drugs for the treatment of a variety of neurological diseases as well as for aesthetic applications. Current assays used for testing and developing BoNT therapeutics include primary rodent cells and immortalized cell lines.

The role of executive functioning, healthcare management, and self-efficacy in college students' health-related quality of life.

Examine how executive functioning (EF), healthcare management, and self-efficacy relate to college students' health-related quality of life (HRQOL). : Undergraduates completed questionnaires at baseline (Time 1;  = 387) and 18-24 months later (Time 2;  = 102). Participants reported on their EF and healthcare management skills at Time 1 and self-efficacy and mental and physical HRQOL at Time 2.

The role of executive functioning in health self-management and the transition to adult health care among college students.

To examine the role of executive functioning (EF) in health self-management and the transition to adult health care among college students. A total of 378 undergraduate students from a public university participated in the study. Participants reported on EF, health self-management skills, and their readiness to transition to adult care.

Sleep quality is associated with psychosocial functioning and health-related quality of life in pediatric transplant recipients.

This study examined patient-reported sleep quality in a single-center cross-sectional sample of adolescents with solid organ transplants and evaluated associations between sleep quality, psychosocial functioning (ie, depression/anxiety symptoms), and HRQOL. Health disparities associated with minority race/ethnicity and socioeconomic variables were also examined. Sixty-nine adolescents (M = 16.

A survey of pharmacists' perceptions of the adequacy of their training for addressing mental health-related medication issues.

Introduction: The purpose of this study was to conduct a survey of North Carolina pharmacists' perceptions of their pharmacy training in mental health-related medication issues and how this influenced their perceived ability to address these issues in the provision of pharmaceutical care to their patients.

Methods: A survey consisting of 17 questions was developed and emailed to licensed pharmacists in North Carolina. Surveys that were returned were analyzed to see if conclusions could be made regarding the pharmacists' perceptions about their mental health-related medication training and its influence on their practice.

Effect of Antidepressant Switching vs Augmentation on Remission Among Patients With Major Depressive Disorder Unresponsive to Antidepressant Treatment: The VAST-D Randomized Clinical Trial.

Importance: Less than one-third of patients with major depressive disorder (MDD) achieve remission with their first antidepressant.

Objective: To determine the relative effectiveness and safety of 3 common alternate treatments for MDD.

Design, Setting, And Participants: From December 2012 to May 2015, 1522 patients at 35 US Veterans Health Administration medical centers who were diagnosed with nonpsychotic MDD, unresponsive to at least 1 antidepressant course meeting minimal standards for treatment dose and duration, participated in the study.

Different Demands, Same Goal: Promoting Transition Readiness in Adolescents and Young Adults With and Without Medical Conditions.

Purpose: This study aimed to examine differences in transition readiness, self-involvement and parental involvement in completing medical tasks, and general self-efficacy between a sample of older adolescents and young adults (AYAs) with medical conditions and a sample of healthy peers. Relations among these variables were also examined.

Methods: The sample included 494 AYAs (mean age = 19.

Changes in Socio-Emotional and Behavioral Functioning After Attending a Camp for Children with Tourette Syndrome: A Preliminary Investigation.

Children and adolescents with Tourette syndrome are at risk for social, emotional, and behavioral difficulties that may negatively influence feelings of self-competence and attitudes toward having this diagnosis. Attending a camp for children with Tourette syndrome may facilitate improvements in overall socio-emotional and behavioral functioning and self-perception. The current study obtained data from 37 campers (76 % male, 24 % female) and 47 caregivers of campers to investigate changes in children's emotional and behavioral functioning, self-perception (i.

Executive Functioning, Barriers to Adherence, and Nonadherence in Adolescent and Young Adult Transplant Recipients.

OBJECTIVE : To evaluate levels of executive functioning in a sample of adolescent and young adult (AYA) transplant recipients, and to examine executive functioning in association with barriers to adherence and medication nonadherence.  METHOD : In all, 41 caregivers and 39 AYAs were administered self- and proxy-report measures.  RESULTS : AYA transplant recipients have significant impairments in executive functioning abilities.

The Role of Self-Competence in Health-Related Quality of Life and Behavioral Functioning of Children with Tourette Syndrome.

Objective: To evaluate self-competence, health-related quality of life (HRQOL), and emotional/behavioral functioning in children with Tourette syndrome (TS) compared to normative data and to examine self-competence as a potential protective factor against poorer HRQOL and emotional/behavioral outcomes in this population.

Method: Thirty-nine children between the ages of 8 and 17 years and 72 caregivers participated in this study. Participants completed measures of children's HRQOL, emotional/behavioral functioning, and self-competence.

Health-related quality of life and psychosocial functioning in children with Tourette syndrome: parent-child agreement and comparison to healthy norms.

This study aimed to evaluate the degree of agreement between parent proxy- and child self-report on measures of child psychosocial functioning and health-related quality of life in children with Tourette syndrome. Participants included 28 children with Tourette syndrome and their parents. All participants provided ratings of children's level of quality of life and psychosocial functioning.