Pediatric ALL

In Ghana, and many other low and middle income countries in Africa, manual vital signs monitoring is the prevalent mode of vital signs monitoring because continuous bedside monitors are non-functional. This lack of continuous vital signs monitoring may result in missed opportunities to catch physiologic deterioration. The investigators propose to develop a dashboard that is based on the Garmin Venu 3, a consumer wearable device that reliably measures heart rate, SPO2, and respiratory rate, as an alternative to bedside monitors in hospitals in Ghana.

This is a Phase I clinical study evaluating the safety and maximum tolerated dose of a novel CAR T-cell product: allogeneic memory (CD45RA- negative) T-cells expressing a CD19-specific CAR 41BBz (CD19-CAR.CD45RA- negative T-cells) for the treatment of patients ≤ 21 years old with relapsed and/ or refractory CD19-positive leukemia. Primary Objective To determine the maximum tolerated dose (MTD) and characterize the safety profile and dose-limiting toxicities (DLTs) of treatment with allogeneic CD19-CAR.CD45RA-negative T-cells in pediatric, adolescent and young adult patients ≤ 21 years of age, with relapsed and/or refractory CD19-positive leukemia. Secondary Objectives * To evaluate the anti-leukemic activity of allogeneic CD19-CAR.CD45RA-negative T-cells. * To determine rates and severity of graft-versus-host-disease (GVHD) after treatment with allogeneic CD19-CAR.CD45RA-negative T-cells. Exploratory Objectives * To study the expansion, persistence and phenotype of allogeneic CD19-CAR.CD45RA-negative T-cells. * To characterize the cytokine profile in the peripheral blood and CSF after treatment with allogeneic CD19-CAR.CD45RA-negative T-cells. * To assess whether allogeneic CD19-CAR.CD45RA-negative T-cells acquire functional versus exhaustion-associated epigenetic programs. * To determine immune reconstitution post treatment, and the clonal structure and endogenous repertoire of allogeneic CD19-CAR.CD45RA-negative T-cells and relate inferred specificity to CAR response profiles. * To characterize incidence and mechanisms of relapse post-therapy with allogeneic CD19-CAR.CD45RA-negative T-cells.

The goal of this clinical trial is to test the extent to which different treatment components work to improve pediatric post-traumatic stress disorder (PTSD). It will also provide evidence for how these components work. The main research questions are: What are the effects of different components used to treat PTSD? What do these components change to produce benefits in PTSD? Researchers will: Compare components to a psychological placebo to estimate their effects and measure how they work Examine how components work alone and in conjunction with other components Participants will: Receive different combinations of components and placebo Attend weekly treatment sessions Provide information to evaluate changes in PTSD

There are many barriers to heart-healthy lifestyles in pediatric patients with acquired and congenital heart disease. Investigators want to further understand how participants heart and skeletal muscles work together during exercise and evaluate the impact on cardiac function. To do this, the investigators will use magnetic resonance imaging (MRI) to scan the heart and skeletal muscles during exercises to assess blood flow, oxygenation and function.

This is a prospective, monocentric diagnostic study aiming to evaluate whether a sedation score obtained by a per-procedural electroencephalogram (EEG), the PSI score, could identify patients for whom a 2 μg/kg dose of DEX would not be sufficient for the successful performance of cerebral NMRI, and who could therefore benefit from a higher dosage of DEX (4 μg/kg).

Heart transplantation is the long-term treatment for children and adults with advanced heart failure. Post-transplant outcomes have improved over time, such that 50% of pediatric heart transplant recipients (HTR) remain alive with a need for re-transplantation 17-years following the initial transplant. With improved short- and medium-term outcomes, focus has shifted towards optimizing long-term survival and reducing transplant-associated morbidities. This includes strategies aimed at optimizing cardiorespiratory fitness and physical activity levels. Pediatric and adult HTRs have reduced exercise capacity compared with the general population. Previous groups have shown gradual improvements in heart rate response to exercise and exercise capacity in pediatric HTRs. However, after an initial improvement, exercise capacity appears to plateau, or even decline in pediatric HTRs, and remains sub-optimal compared with the general population. Most exercise interventions in HTRs to date have focused on moderate-intensity continuous exercise (MICE), with some resistance components incorporated. More recently, high-intensity interval training (HIIT), consisting of short, intense bursts of exercise with rest periods, has been explored in the adult HTR population, with findings to date suggesting that it may yield greater improvements in cardiorespiratory fitness compared with MICE. Exercise interventions, particularly HIIT interventions, have consistently shown clinically important improvements in exercise capacity in adult HTRs that are linked with improved long-term post-transplant outcomes and well-being. Unfortunately, trials of exercise interventions in pediatric HTRs remain lacking. This study team is proposing an assessment of the feasibility of a home-based HIIT exercise program using a novel telemedicine-enable video game linked customizable cycle ergometer (MedBIKE™).

The objective of this project is to collect baseline data from a normative population of 20 children that gender and age matches the population of patients expected to be treated by Mary Bridge Children's Research \& Movement Lab.

The present trial conducted to demonstrate the administration of sugammadex as reversal of neuromuscular blockade agent reduces the incidence of emergence agitation (EA) comparing to neostigmine

Congenital heart disease (CHD), the most common birth defect, is present in nearly 1% of the population. CHD patients are associated with intense resource utilization and premature death in adulthood. The risk of premature death is linked with reduced exercise capacity, a finding consistently noted in youth with CHD. Reduced exercise capacity in this population has also been associated with reduce physical activity and health-related quality of life. Cardiac rehabilitation (CR) in adults with acquired heart disease is an established secondary prevention strategy that improves exercise capacity. The investigators propose a prospective clinical trial of a home-based high intensity interval training (HIIT) program using a novel telemedicine-equipped video game-linked cycle ergometer (MedBIKE™) for 10 to 18 year olds with repaired moderate-complex CHD. The pilot study with the MedBIKE has shown promising results. The investigators now seek to study the efficacy of this program in a broader CHD population.

This is a prospective study of children \<21 years of age with the goal of characterizing the PK of drugs administered per standard of care as prescribed by the treating caregiver.

This is a study comparing 3 years of retrospective data (pre-implementation) to 2 years of prospective data after the implementation of a pediatric version of Electronic Cardiac Arrest Risk Triage (pediatric eCART), a clinical decision support (CDS) tool that uses electronic health records (EHR) to identify patients with high risk for life threatening outcomes. Up to 30,000 encounters with pediatric patients will be assessed. Acceptability of the pediatric eCART intervention will also be measured from pediatric nurse clinicians.

The goal of this Long-Term Follow-Up Trial is to assess long-term safety and efficacy of Miltenyi CAR T treatment.

This study aims to use functional near-infrared spectroscopy (fNIRS) to identify and quantify pain biomarkers during burn dressing changes and pain relief induced by virtual reality (VR).

Implant devices are important tools - their use is essential across a number of orthopaedic indications, including hip conditions, trauma and limb deformity. Given the vital role fixation devices play in maintaining alignment, promoting healthy bone healing and preventing joint degeneration, it is essential to understand the expected lifetime outcomes of these implants, and evaluate their safety and efficacy. Prospective implant efficacy and safety registries are needed to support this endeavour, especially considering new regulatory requirements from the European Union Medical Devices Regulation (EU MDR) in relation to post-market clinical follow-up (PMCF).

Early childhood detection of motor delays or impairments provides the opportunity for early treatment which improves health outcomes. This study will use state of the art sensors combined with machine learning algorithms to develop objective, accurate, easy-to-use tools for the early scoring of deficits and lays the foundation for the early prediction of physical disability.

Retrospective study for development of imaging-genetics (brain imaging/genome big data) models and algorithms that are clinically explainable and have high predictive performance in brain research on pediatric developmental disorders

This multisite study seeks to understand whether and how pulse oximeter accuracy varies across children with different skin colors in real world clinical settings. Many factors can affect how well pulse oximeters work; for example, movement and even fingernail polish. Some studies in adults show that skin color may also affect the pulse oximeter reading. In this study, we will explore pulse oximeter accuracy in children of all skin colors who are undergoing cardiac catheterizations. The study will address limitations of prior work by objectively measuring skin color across multiple dimensions of color and comparing the the pulse oximeter reading to the "gold standard" blood sample measurement.

This study will demonstrate safety in pediatric patients with Parenteral Nutrition-Associated Cholestasis treated with Omegaven®, which is indicated as a source of calories and fatty acids in this patient population

This study will be a prospective, single-center, randomized controlled trial in a tertiary pediatric emergency department with two parallel groups of voluntary pediatric physicians and nurses. The impact of a mHealth supportive tool will be compared with conventional communication methods on situational awareness, leadership, team communication effectiveness and performance during standardized, simulation-based, pediatric in-hospital cardiac arrest scenario using a high-fidelity manikin. Thirty-six participants will be randomized (1:1). The primary endpoint is the situational awareness score measured with the situation awareness global assessment technique (SAGAT) instrument.

The Health Advocate for Liver Transplant (HEAL-Tx) Pilot is a nonrandomized, open-label intervention pilot of a health advocate intervention aimed to assess feasibility and acceptability of integrating a Health Advocate onto the transplant team. Across studies, health advocate roles vary, and can include coordinating medical care treatment, facilitating financial assistance (e.g., taxi vouchers), and connecting patients to community resources, which can improve self-management, mitigate social risks, and lead to better communication between the healthcare system and the family. In this pilot, the investigators will adapt this intervention for pediatric liver transplant patients and measure acceptability and feasibility according to RE-AIM.

This is a multicenter study to test a decision-making support process for families and clinicians facing decisions about chronic home ventilation for a child. The investigators hypothesize that the intervention will increase family preparedness for decision-making and will improve clinician-family shared-decision making. Half of families will be assigned to "usual care" arm and half to the "intervention" arm. Intervention families will view the study website with study staff and will answer questions related to website content. All families will be interviewed and surveyed at 1, 6 and 12 months after enrollment. Each family will designate 1-2 physician involved in the decision about home ventilation; each physician will be interviewed and surveyed at 1 month.

The goal of this clinical trial\] is to evaluate the efficacy and safety of the ultrasound-guided "Modified Dynamic Needle Tip Positioning Short-Axis, Out-Of-Plane" (MDNTP-SAOP) technique compared to the "Conventional, Short-Axis, Out-Of-Plane" (C-SAOP) technique for femoral arterial cannulation performed by anesthesia residents in children. The main question it aims to answer is: What is the time required for attempted femoral arterial cannulation by anesthesia residents at the first puncture site? Researchers will compare the efficacy and safety of MDNTP-SAOP versus C-SAOP methods for femoral arterial cannulation performed by anesthesia residents in infants and children undergoing cardiac surgery. Participants will be randomized to either the MDNTP-SAOP or the C-SAOP group. Intraoperatively, time taken for attempted cannulation by the resident at the first site of femoral arterial puncture, number of attempts at arterial cannulation, and number of cannulae required for successful cannula insertion will be recorded. Adverse events will be monitored on postoperative days 1 and 3. The insertion site will be examined for thrombosis, hematoma, infection, or limb ischemia distal to the insertion site

This randomized controlled experimental study aims to evaluate the effectiveness of guided imagery and virtual reality (VR) in reducing pain and fear in children during blood draw procedures. The sample will consist of children aged 8-14 who meet the inclusion criteria and present to the Koç Hospital Pediatric Clinic Blood Drawing Room. After informing the children and their parents about the study, written and verbal consents will be obtained. Participants will then be randomly assigned into one of three groups-two experimental (guided imagery and VR) and one control-using a random number list generated via Excel. Pre-procedure: Prior to the intervention, children and parents will be informed using consent forms. Demographic and baseline data will be collected using the Child Patient Introductory Information Form. Pain will be assessed with the Faces Pain Scale-Revised (FPS-R) by the child and the Visual Analog Scale (VAS) by the parent and observer. Fear will be evaluated using the Children's Fear Scale (CFS), self-rated by the child and assessed by the parent/observer. A pulse oximeter will be attached to measure baseline pulse and oxygen saturation (SpO2) levels starting 1 minute before the procedure. Guided Imagery Group: Children will listen to an audio recording titled "A Walk in the Forest" using headphones. They will be instructed to close their eyes and imagine the scene described, focusing on the sound to facilitate distraction. The audio will start 1 minute prior to the procedure. Virtual Reality Group: Children in this group will wear VR glasses and watch an immersive AI-generated video of "A Walk in the Forest." This aims to provide a more interactive distraction during the blood draw. Control Group: Children will undergo routine blood draw procedures with no additional distraction techniques. The procedure will be explained as usual. Procedure: A trained nurse will perform all blood draws. During the procedure, SpO2 and pulse data will be recorded. Post-procedure, these will also be documented immediately and 1 minute after the intervention. Post-procedure: After the procedure, FPS-R and CFS will be used again by children to assess pain and fear. Parents and observers will complete VAS and CFS to evaluate observed pain and fear. Headphones or VR glasses will be removed following the final measurement.

The Health Advocate for Liver Transplant (HEAL-Tx) Transition of Care Pilot is a nonrandomized, open-label intervention pilot of a health advocate intervention aimed to assess feasibility and acceptability of integrating a Health Advocate onto the transplant team to help adolescents transition their care to adult transplant teams. Across studies, health advocate roles vary, and can include coordinating medical care treatment, facilitating financial assistance (e.g., taxi vouchers), and connecting patients to community resources, which can improve self-management, mitigate social risks, and lead to better communication between the healthcare system and the family. In this pilot, the investigators will adapt this intervention for adolescent/young adult liver transplant patients and measure acceptability and feasibility according to RE-AIM.

Umbilical venous catheters (UVC) are typically places with poor guidance and some radiological confirmation. Misplacement of the catheter could lead to its placement in other unintended anatomical areas such as the liver or the spleen, which could be detrimental in critically-ill infants. Our study aims at using a more non-invasive means of placing and continuously monitoring catheter placement using superficial electrocardiogram (ECG) tracings.