Evolution of the Florida Pediatric Bone Marrow Transplant and Cell Therapy Consortium (FPBCC): A Statewide Initiative Toward Improving Transplant Outcomes.

Background: Florida Pediatric Bone Marrow Transplant and Cell Therapy Consortium (FPBCC) was formed in 2018 by five pediatric transplant programs in Florida. The key objectives of the consortium are to improve outcomes for children undergoing HSCT through collaboration among centers, data sharing, implementation of best practices, QI projects, and prospective clinical trials. The first step in that process was to analyze HSCT outcomes from all participating centers and identify areas for improvement.

Will my Child Walk Funny? The Rotational Profile of Infants and Children With Classic Bladder Exstrophy.

Background: Bladder exstrophy is a rare, complex disorder that involves the urinary, reproductive, gastrointestinal, and musculoskeletal systems and is marked by an incomplete lower abdominal wall, leaving the bladder exposed to the outside world. Skeletal findings in this condition include externally rotated innominate bones. While the resulting acetabular retroversion might be expected to cause out-toeing, there is no published data on the gait parameters or rotational profile of children with classic bladder exstrophy (CBE) in isolation.

Long-term safety of dexamethasone sodium phosphate encapsulated in autologous erythrocytes in pediatric patients with ataxia telangiectasia.

Background: Dexamethasone sodium phosphate (DSP) encapsulated in autologous erythrocytes (EryDex) was developed as an alternative to standard glucocorticoids in an effort to eliminate chronic steroid toxicity while preserving efficacy. The primary objective of this report is to describe the safety of long-term use of EryDex in treatment of pediatric patients with ataxia telangiectasia.

Methods: This is a post-hoc analysis of patients treated with EryDex for a minimum of 24 months in two prospective clinical trials.

State adoption of paid sick leave and cardiovascular disease mortality among adults in the United States, 2008-2019.

Cardiovascular diseases (CVD) are the leading cause of death in the United States and may be prevented through improved working conditions. The United States does not guarantee paid sick leave (PSL) at the federal level. We used quasi-experimental event study methods to examine the relationship between state-level PSL policies and county-level CVD mortality among working-age adults aged 15 to 64 over time (2008 to 2019).

A case-control study and molecular epidemiology of yersiniosis in Aotearoa New Zealand.

The objective of this study was to determine risk factors and sources attributed to yersiniosis in Aotearoa New Zealand (NZ). A risk factor questionnaire was administered to 247 notified yersiniosis cases and 258 control participants from the Canterbury and/or Wellington regions of NZ. sp.

Avatrombopag for severe refractory thrombocytopenia in a pediatric patient with ALL following allogeneic hematopoietic stem cell transplantation: A case report.

Patients who receive allogeneic hematopoietic stem cell transplantation (alloHSCT) are at risk for developing persistent thrombocytopenia. Here, we describe treatment with avatrombopag, a thrombopoietin receptor agonist, in a pediatric patient with chronic, severe, transfusion-dependent thrombocytopenia (<10 × 10/µL) post-alloHSCT that was persistent despite treatment with romiplostim, another thrombopoietin receptor agonist. Following the granting of a compassionate use investigational new drug authorization, avatrombopag treatment was initiated, and the patient's platelet count increased.

Who learns more: the impact of dual-player and single-player modes in a serious game on dental students' factual knowledge.

Background: The use of serious games in medical education provides a bridge between rapidly developing technology and traditional health-care teaching. Building on a promising web-based serious game for reviewing and acquiring factual knowledge in dental education, the present study investigated the benefits of a dual-player mode and various game options for enhancing knowledge gain and study motivation.

Methods: Before the intervention, students' dental knowledge and game experience were assessed using a pre-knowledge test and questionnaire-based self-assessment.

Safety and efficacy of intra-erythrocyte dexamethasone sodium phosphate in children with ataxia telangiectasia (ATTeST): a multicentre, randomised, double-blind, placebo-controlled phase 3 trial.

Background: Ataxia telangiectasia is a multisystem disorder with progressive neurodegeneration. Corticosteroids can improve neurological functioning in patients with the disorder but adrenal suppression and symptom recurrence on treatment discontinuation has limited their use, prompting the development of novel steroid delivery systems. The aim of the ATTeST study was to evaluate the efficacy and safety of intra-erythrocyte delivery of dexamethasone sodium phosphate compared with placebo in children with ataxia telangiectasia.

Radiographic calibration for pubic diastasis assessment in bladder exstrophy-epispadias complex: a phantom study.

Background: The assessment of pubic diastasis is important for the surgical planning of patients with bladder exstrophy-epispadias complex. Understanding how the diastasis changes during surgical follow-up may help predict patient morbidity. Radiography can follow diastasis but may be affected by patient and technical imaging factors including body size, imaging protocol, and equipment.

The role of donor type and pre-transplant immunosuppression on outcomes of hematopoietic stem cell transplantation in children and young adults with severe aplastic anemia.

Background: The goal of this study was to assess the effect of donor type and pre-transplant immunotherapy (IST) on outcomes of hematopoietic stem cell transplantation (HSCT) for children and young adults with severe aplastic anemia (SAA).

Methods: This retrospective, multi-center study included 52 SAA patients, treated in 5 pediatric transplant programs in Florida, who received HSCT between 2010 and 2020 as the first- or second-line treatment.

Results: The median age at HSCT for all 52 patients was 15 years (range 1-25).

An international learning collaborative phase 2 trial for haploidentical bone marrow transplant in sickle cell disease.

In the setting of a learning collaborative, we conducted an international multicenter phase 2 clinical trial testing the hypothesis that nonmyeloablative-related haploidentical bone marrow transplant (BMT) with thiotepa and posttransplant cyclophosphamide (PTCy) will result in 2-year event-free survival (no graft failure or death) of at least 80%. A total of 70 participants were evaluable based on the conditioning protocol. Graft failure occurred in 8 of 70 (11.

The cellular representation of temperature across the somatosensory thalamus.

Although distinct thalamic nuclei encode sensory information for almost all sensory modalities, the existence of a thalamic representation of temperature with a role in thermal perception remains unclear. To address this, we performed high-density electrophysiological recordings across the entire forelimb somatosensory thalamus in awake mice, and identified an anterior and a posterior representation of temperature that spans three thalamic nuclei. We found that these parallel representations show fundamental differences in the cellular encoding of temperature which reflects their cortical output targets.

New Classification for Pediatric Proximal Fifth Metatarsal Fractures.

Proximal fifth metatarsal fractures are the most common foot fractures in children. Attempts to classify these injuries are misapplied and inadequately predict outcomes. This is the first study to identify factors associated with healing in pediatric fifth metatarsal fractures.

The Economic Effect of Peer Recovery Support Services: A Review of the Literature.

Peer Recovery Support Services (PRSS) have the potential to be an economically valuable intervention. To investigate this potential, we conducted a scoping review to summarize existing research on the economic impact of PRSS. We searched relevant electronic databases for peer-reviewed articles and grey literature between January 2000 and February 2023 that examined an economic outcome related to PRSS.

Caring for a Relative With Dementia in Long-Term Care During the COVID-19 Pandemic: A Prospective Longitudinal Study.

Background And Objectives: The coronavirus disease 2019 (COVID-19) pandemic introduced unprecedented threats and disruptions for caregivers of people with dementia living in residential long-term care (LTC) facilities. Qualitative and cross-sectional studies have reported substantial negative effects of the pandemic on dementia caregivers' well-being, but little to no prospective research has examined the impact of COVID-19 on caregiver well-being using pre-pandemic assessments. The present study used longitudinal data from an ongoing randomized controlled trial of a psychosocial intervention to support family caregivers whose relatives had entered LTC.

Feasibility study of busulfan, fludarabine, and thiotepa conditioning regimen for allogeneic hematopoietic stemcell transplantationfor children and young adults with nonmalignant disorders.

Background: Hematopoietic stem cell transplant (HSCT) is the only curative treatment for several pediatric non-malignant disorders. A widely used conditioning backbone is busulfan, fludarabine, and rabbit anti-thymocyte globulin (rATG). Thiotepa has improved engraftment when added to this regimen, however the minimum effective dose (MED) of thiotepa to achieve engraftment while minimizing toxicities has not been well established.

A near-miss diagnosis of necrotizing breast fasciitis complicated by atrial fibrillation secondary to septic focus: a case report and brief review of literature.

Necrotizing fasciitis of the breast is a rare, life-threatening entity characterized by a rapidly aggressive infection of the soft tissue. There are few literature reports on necrotizing fasciitis at the level of the breast tissue as the most common locations are within the abdominal wall or extremities, but this entity can lead to sepsis and systemic multiorgan failure if not adequately managed. Here, we report a case that highlights the course of a 68-year-old African American female with a past medical history of hypertension, hyperlipidemia, and poorly controlled diabetes mellitus, who presented with the complaint of a painful right breast abscess with intermittent, purulent drainage.

Polygenic Pharmacogenomic Markers as Predictors of Toxicity Phenotypes in the Treatment of Acute Lymphoblastic Leukemia: A Single-Center Study.

Purpose: Acute lymphoblastic leukemia (ALL) is the most prevalent cause of childhood cancer and requires a long course of therapy consisting of three primary phases with interval intensification blocks. Although these phases are necessary to achieve remission, the primary chemotherapeutic agents have potentially serious toxicities, which may lead to delays or discontinuations of therapy. The purpose of this study was to perform a comprehensive pharmacogenomic evaluation of common antileukemic agents and develop a polygenic toxicity risk score predictive of the most common toxicities observed during ALL treatment.

The impact of primary and secondary processing steps on Campylobacter concentrations on chicken carcasses and portions.

Campylobacteriosis is the most commonly notified foodborne disease in New Zealand and poultry meat is the major source for human infection. Carcasses and portions were sampled from key points along primary and secondary processing chains of three New Zealand poultry processors to determine the impact of processing steps on Campylobacter concentrations. Primary processing reduced Campylobacter concentrations on carcasses by almost 6-log; the biggest reduction was achieved by the spinchill, followed by the scald step.

Prospective Validation and Refinement of a Population Pharmacokinetic Model of Fludarabine in Children and Young Adults Undergoing Hematopoietic Cell Transplantation.

Fludarabine is a nucleoside analog with antileukemic and immunosuppressive activity commonly used in allogeneic hematopoietic cell transplantation (HCT). Several fludarabine population pharmacokinetic (popPK) and pharmacodynamic models have been published enabling the movement towards precision dosing of fludarabine in pediatric HCT; however, developed models have not been validated in a prospective cohort of patients. In this multicenter pharmacokinetic study, fludarabine plasma concentrations were collected via a sparse-sampling strategy.

Asymmetrical lineage introgression and recombination in populations of Aspergillus flavus: Implications for biological control.

Aspergillus flavus is an agriculturally important fungus that causes ear rot of maize and produces aflatoxins, of which B1 is the most carcinogenic naturally-produced compound. In the US, the management of aflatoxins includes the deployment of biological control agents that comprise two nonaflatoxigenic A. flavus strains, either Afla-Guard (member of lineage IB) or AF36 (lineage IC).

Can Technology-Based Social Memory Aids Improve Social Engagement? Perceptions of a Novel Memory Aid for Persons With Memory Concerns.

Social withdrawal and isolation are frequently experienced among people with cognitive impairment, Alzheimer's disease, and Alzheimer's disease related dementias. Few assistive technologies exist to support persons with memory concerns' (PWMC) continuing social engagement. This study aimed to understand PWMC and family caregivers' initial perspectives on the feasibility and utility of a wearable technology-based social memory aid.

Retrospective Comparison of Patients ≥ 80 Years With Atrial Fibrillation Prescribed Either an FDA-Approved Reduced or Full Dose Direct-Acting Oral Anticoagulant.

Direct-acting oral anticoagulants (DOACs) represent the standard for preventing stroke and systemic embolization (SSE) in patients with atrial fibrillation (AF). There is limited information for patients ≥ 80 years. We report a retrospective analysis of AF patients ≥ 80 years prescribed either a US Food and Drug Administration (FDA)-approved reduced (n = 514) or full dose (n = 199) DOAC (Dabigatran, Rivaroxaban, or Apixaban) between January 1st, 2011 (first DOAC commercially available) and May 31st, 2017.

A long-acting formulation of rifabutin is effective for prevention and treatment of Mycobacterium tuberculosis.

Tuberculosis (TB) is a communicable disease caused by Mycobacterium tuberculosis (Mtb) and is a major cause of morbidity and mortality. Successful treatment requires strict adherence to drug regimens for prolonged periods of time. Long-acting (LA) delivery systems have the potential to improve adherence.