Early engraftment and immune kinetics following allogeneic transplant using a novel reduced-toxicity transplant strategy in children/adolescents with high-risk transfusion-dependent thalassemia: Early results of the ThalFAbS trial.

Background: Allogeneic transplant for patients with transfusion-dependent thalassemia is challenging once there has been iron overload and chronic transfusion support.

Objective(s): A transplant strategy that reduced intensity of the preparative regimen and tailored immunosuppression to both support donor engraftment and prevent GVHD was developed for this population. The combination of a pretransplant immunosuppression phase with reduced dosing of fludarabine/prednisone, treosulfan-based preparative regimen with reduced cyclophosphamide dosing, and introduction of a calcineurin/methotrexate-free GVHD prophylaxis/engraftment supporting regimen with abatacept/sirolimus/ATG was tested.

[Acute blindness in a patient with metformin-associated lactic acidosis].

Lactic acidosis is a rare metabolic complication that can occur in patients with diabetes mellitus type 2 who use metformin. We discuss a 79-year old woman with metformin-associated lactic acidosis (MALA) and acute kidney injury based on gastroenteritis. Patient reported acute blindness which in literature is described as a rare presentation of a metabolic acidosis (regardless of its underlying cause).

User-Centered Design and Usability of Voxe as a Pediatric Electronic Patient-Reported Outcome Measure Platform: Mixed Methods Evaluation Study.

Background: Electronic patient-reported outcome measures (ePROMs) are standardized digital instruments integrated into clinical care to collect subjective data regarding patients' health-related quality of life, functional status, and symptoms. In documenting patient-reported progress, ePROMs can guide treatment decisions and encourage measurement-based care practices. Voxe is a pediatric and user-centered ePROM platform for patients with chronic health conditions.

Understanding the physical literacy development of 8- to 12-year-old children living with chronic medical conditions: A comprehensive, mixed methods inquiry.

Background: Physical literacy is a concept used to describe the combined physical, affective and cognitive capacities facilitating an active lifestyle. Physical activity participation is essential for children living with chronic medical conditions, but knowledge of physical literacy among this group is scarce.

Methods: An explanatory, sequential mixed methods design was used to comprehensively describe the physical literacies of children with chronic medical conditions (CMCs).

Quality of life in childhood immune thrombocytopenia: Revision of the Kids' ITP Tools (KIT).

The Kids ITP Tools (KIT) is a health-related quality of life (HRQoL) questionnaire that evaluates quality of life in children with immune thrombocytopenia (ITP). There are three formats: Child Self-Report, Parent Proxy-Report and Parent Impact-Report. This study aimed to develop a domain structure by grouping-related questions from the questionnaire into domains that independently reflect various aspects of HRQoL.

Childhood autoimmune hemolytic anemia: A scoping review.

Background And Objective: Autoimmune hemolytic anemia (AIHA) is a rare but important cause of morbidity in pediatric hematology patients. Given its rarity, there is little high-quality evidence on which to base the investigation and management of pediatric AIHA. This scoping review aims to summarize the current evidence and highlight key gaps to inform future studies.

Quality of Life in Transfusion-Dependent Thalassemia Patients in Greece Before and During the COVID-19 Pandemic.

Objectives: The peak of the COVID-19 pandemic was a challenging situation for transfusion-dependent thalassemia (TDT) patients. The objectives of this study were to measure the quality of life (QoL) in TDT patients during the COVID-19 lockdown restriction measures, compare the results with the pre-COVID-19 era, and evaluate the influence of sociodemographic and clinical factors on QoL.

Methods: This was a cross-sectional study of 110 consecutively selected adult TDT patients, during the stringent lockdown restriction measures implemented in Greece.

Evaluating changes in functioning and psychological distress in visitors of the @ease youth mental health walk-in centres.

Background: Highly accessible youth initiatives worldwide aim to prevent worsening of mental health problems, but research into outcomes over time is scarce.

Aims: This study aimed to evaluate outcomes and support use in 12- to 15-year-old visitors of the @ease mental health walk-in centres, a Dutch initiative offering free counselling by trained and supervised peers.

Method: Data of 754 visitors, collected 2018-2022, included psychological distress (Clinical Outcomes in Routine Evaluation 10 (CORE-10)), social and occupational functioning (Social and Occupational Functioning Assessment Scale (SOFAS)), school absenteeism and support use, analysed with change indicators (first to last visit), and mixed models (first three visits).

Shared decision-making related to treatment of haemophilia: A scoping review of influential factors and available support tools.

Introduction: Treatment selection in haemophilia is increasingly challenging given evolving therapeutic options and the need for individualization. Shared decision-making (SDM) approaches have recently gained interest, though a synthesis of available studies is lacking.

Aim: A scoping review was conducted to summarize literature reporting on factors impacting treatment SDM in haemophilia and tools or models available to support such decisions.

Synchronous timing of return to breeding sites in a long-distance migratory seabird with ocean-scale variation in migration schedules.

Background: Migratory birds generally have tightly scheduled annual cycles, in which delays can have carry-over effects on the timing of later events, ultimately impacting reproductive output. Whether temporal carry-over effects are more pronounced among migrations over larger distances, with tighter schedules, is a largely unexplored question.

Methods: We tracked individual Arctic Skuas Stercorarius parasiticus, a long-distance migratory seabird, from eight breeding populations between Greenland and Siberia using light-level geolocators.

A Cross-Sectional, Multicentric, Disease-Specific, Health-Related Quality of Life Study in Greek Transfusion Dependent Thalassemia Patients.

The assessment of health-related quality of life (HRQoL) in thalassemia offers a holistic approach to the disease and facilitates better communication between physicians and patients. This study aimed to evaluate the HRQoL of transfusion-dependent thalassemia (TDT) patients in Greece. This was a multicentric, cross-sectional study conducted in 2017 involving 283 adult TDT patients.

Face validity of the Kids' ITP Tools (KIT) in the era of thrombopoietin receptor agonists.

The Kids' ITP Tools (KIT) is a questionnaire to assess quality of life of children with immune thrombocytopenia (ITP). The aim of this study was to update this previously validated tool to align with changes in clinical practice, specifically, treatment with thrombopoietin receptor agonists (TPO-RAs). Children aged 1-18 with ITP and/or their families were recruited to participate in interviews to review the KIT.

Eating Event Recognition Using Accelerometer, Gyroscope, Piezoelectric, and Lung Volume Sensors.

In overcoming the worldwide problem of overweight and obesity, automatic dietary monitoring (ADM) is introduced as support in dieting practises. ADM aims to automatically, continuously, and objectively measure dimensions of food intake in a free-living environment. This could simplify the food registration process, thereby overcoming frequent memory, underestimation, and overestimation problems.

Quick, Effective Screening Tasks Identify Children With Medical Conditions or Disabilities Needing Physical Literacy Support.

Purpose: This study evaluated screening tasks able to identify children with medical conditions or disabilities who may benefit from physical literacy.

Method: Children completed ≤20 screening tasks during their clinic visit and then the Canadian Assessment of Physical Literacy (2nd edition) at a separate visit. Total Canadian Assessment of Physical Literacy scores <30th percentile were categorized as potentially needing physical literacy support.

PROMISE: effect of protein supplementation on fat-free mass preservation after bariatric surgery, a randomized double-blind placebo-controlled trial.

Introduction: Protein malnutrition after bariatric surgery is a severe complication and leads to significant morbidity. Previous studies have shown that protein intake and physical activity are the most important factors in the preservation of fat-free mass during weight loss. Low protein intake is very common in patients undergoing bariatric surgery despite dietary counseling.

Psychometric validation of the Pyruvate Kinase Deficiency Diary and Pyruvate Kinase Deficiency Impact Assessment in adults in the phase 3 ACTIVATE trial.

Background: Pyruvate kinase (PK) deficiency is a rare hereditary disorder characterized by chronic hemolytic anemia and serious sequalae which negatively affect patient quality of life. This study aimed to psychometrically validate the first disease-specific patient-reported outcome (PRO) instruments: the 7-item PK Deficiency Diary (PKDD) and 12-item PK Deficiency Impact Assessment (PKDIA), designed to assess signs, symptoms, and impacts of PK deficiency in patients enrolled in the ACTIVATE global phase 3 study of mitapivat versus placebo (NCT03548220).

Methods: All validation analyses for the PKDD and PKDIA were performed on blinded data, with analyses on item integrity, scoring, reliability, and validity conducted on data from screening and baseline.

Preconception maternal gastric bypass surgery and the impact on fetal growth parameters.

Background: Bariatric surgery is increasingly performed in women of reproductive age. As bariatric surgery will result in postoperative rapid catabolic weight loss which potentially leads to fetal malnutrition and directly related impaired intra-uterine growth, it is advised to postpone pregnancy for at least 12-18 months after surgery.

Objectives: To investigate the consequences of preconception gastric bypass surgery (pGB) on fetal growth parameters and maternal pregnancy outcome.

F-FDG-PET/CT to Detect Pathological Complete Response After Neoadjuvant Treatment in Patients with Cancer of the Esophagus or Gastroesophageal Junction: Accuracy and Long-Term Implications.

Purpose: The curative strategy for patients with esophageal cancer without distant metastases consists of esophagectomy with preceding chemo(radio)therapy (CRT). In 10-40% of patients treated with CRT, no viable tumor is detectable in the resection specimen (pathological complete response (pCR)). This study aims to define the clinical outcomes of patients with a pCR and to assess the accuracy of post-CRT FDG-PET/CT in the detection of a pCR.

Suspension TRAPping Filter (sTRAP) Sample Preparation for Quantitative Proteomics in the Low µg Input Range Using a Plasmid DNA Micro-Spin Column: Analysis of the Hippocampus from the 5xFAD Alzheimer's Disease Mouse Model.

Suspension TRAPping filter (sTRAP) is an attractive sample preparation method for proteomics studies. The sTRAP protocol uses 5% SDS that maximizes protein solubilization. Proteins are trapped on a borosilicate glass membrane filter, where SDS is subsequently removed from the filter.

Development of an Alcohol Refusal Training in Immersive Virtual Reality for Patients With Mild to Borderline Intellectual Disability and Alcohol Use Disorder: Cocreation With Experts in Addiction Care.

Background: People with mild to borderline intellectual disability (MBID; IQ=50-85) are at risk for developing an alcohol use disorder (AUD). One factor contributing to this risk is sensitivity to peer pressure. Hence, tailored trainings are needed to practice alcohol refusal in impacted patients.