Demographics, clinical characteristics, and real-world treatment patterns among patients with beta-thalassemia: a retrospective medical record abstraction study.

Background: Beta-thalassemias (BTs) are characterized by deficient or absent synthesis of the beta-globin subunit, leading to anemia. Patient characteristics and treatment patterns in these patients may vary.

Objective: This retrospective study evaluated demographics, clinical characteristics, and treatment patterns in patients with transfusion-dependent BT (TDT) and non-transfusion-dependent BT (NTDT).

Identifying thresholds for meaningful improvements in NTDT-PRO scores to support conclusions about treatment benefit in clinical studies of patients with non-transfusion-dependent beta-thalassaemia: analysis of pooled data from a phase 2, double-blind, placebo-controlled, randomised trial.

Objectives: To estimate thresholds for defining meaningful within-patient improvement from baseline to weeks 13-24 and interpreting meaningfulness of between-group difference for the non-transfusion-dependent beta-thalassaemia patient-reported outcome (NTDT-PRO) tiredness/weakness (T/W) and shortness of breath (SoB) scores. A secondary objective was to determine the symptom severity threshold for the NTDT-PRO T/W domain to identify patients with symptomatic T/W.

Design: Pooled blinded data from the phase 2, double-blind, placebo-controlled, randomised BEYOND trial in NTDT (NCT03342404) were used.

Treatment characteristics and outcomes in lower-risk, non-del(5q) myelodysplastic syndromes: findings from a medical record review in the USA, Canada and Europe.

To assess treatment patterns and outcomes in patients with non-del(5q) lower-risk myelodysplastic syndromes. Patient medical records were reviewed in the USA, Canada (CAN), UK and the EU. Analysis included 119 patients in the USA/CAN (median age, 61.

A Systematic Literature Review of Predictors of Erythropoiesis-Stimulating Agent Failure in Lower-Risk Myelodysplastic Syndromes.

Erythropoiesis-stimulating agents (ESAs) are the first-line treatment option for anemia in patients with lower-risk myelodysplastic syndromes (LR-MDS). A systematic literature review was conducted to identify evidence of the association between prognostic factors and ESA response/failure in LR-MDS. MEDLINE, Embase, and relevant conferences were searched systematically for studies assessing the association between prognostic factors and ESA response/failure in adult patients.

The Role of Adenovirus in Hepatitis of Unknown Etiology Among Children in Turkey, July 2022 to January 2023.

Background: In the first half of 2022, the increase in cases of severe acute hepatitis in children with no epidemiological link and unknown cause has aroused concern worldwide. In this study, we aimed to determine the frequency of adenovirus in children with hepatitis of unknown origin in Turkey.

Methods: In this study, which was conducted with the participation of 16 centers from Turkey, between July 2022 and January 2023, 36 pediatric patients under the age of 18 who met the definition of acute hepatitis were included.

Evaluation of nutritional status in pediatric intensive care unit patients: the results of a multicenter, prospective study in Turkey.

Introduction: Malnutrition is defined as a pathological condition arising from deficient or imbalanced intake of nutritional elements. Factors such as increasing metabolic demands during the disease course in the hospitalized patients and inadequate calorie intake increase the risk of malnutrition. The aim of the present study is to evaluate nutritional status of patients admitted to pediatric intensive care units (PICU) in Turkey, examine the effect of nutrition on the treatment process and draw attention to the need for regulating nutritional support of patients while continuing existing therapies.

Improved benefit of continuing luspatercept therapy: sub-analysis of patients with lower-risk MDS in the MEDALIST study.

Red blood cell transfusion independence (RBC-TI) is an important goal in treating lower-risk myelodysplastic syndromes with ring sideroblasts. In the phase 3 MEDALIST study, RBC-TI of ≥ 8 weeks was achieved by significantly more luspatercept- versus placebo-treated patients in the first 24 weeks of treatment. In this post hoc analysis, we evaluated RBC transfusion units and visits based on patients' baseline transfusion burden level and the clinical benefit of luspatercept treatment beyond week 25 in initial luspatercept nonresponders (patients who did not achieve RBC-TI ≥ 8 weeks by week 25) but continued luspatercept up to 144 weeks.

All-star approach to a small medical imaging dataset: combined deep, transfer, and classical machine learning approaches for the determination of radial head fractures.

Background: Radial head fractures are often evaluated in emergency departments and can easily be missed. Automated or semi-automated detection methods that help physicians may be valuable regarding the high miss rate.

Purpose: To evaluate the accuracy of combined deep, transfer, and classical machine learning approaches on a small dataset for determination of radial head fractures.

Luspatercept for the treatment of anaemia in non-transfusion-dependent β-thalassaemia (BEYOND): a phase 2, randomised, double-blind, multicentre, placebo-controlled trial.

Background: In patients with non-transfusion-dependent β-thalassaemia, haemoglobin concentrations lower than 10 g/dL are associated with a higher risk of morbidity, mortality, and impaired quality of life. No drugs are specifically approved for anaemia management in patients with non-transfusion-dependent β-thalassaemia, other than transfusion therapy administered infrequently in accordance with patients' needs. We assessed the efficacy and safety of luspatercept versus placebo in patients with non-transfusion-dependent β-thalassaemia.

Relationship between Serum Ferritin and Outcomes in β-Thalassemia: A Systematic Literature Review.

Among the difficulties of living with β-thalassemia, patients frequently require blood transfusions and experience iron overload. As serum ferritin (SF) provides an indication of potential iron overload, we conducted a systematic literature review (SLR) to assess whether SF levels are associated with clinical and economic burden and patient-reported outcomes (PROs). The SLR was conducted on 23 April 2020 and followed by analysis of the literature.

Evaluation of nutritional status and related factors in children with cystic fibrosis.

Background: This study aimed to evaluate the nutritional status and body composition in children with cystic fibrosis (CF), in accordance with the new nutritional targets defined by European Society for Clinical Nutrition and Metabolism (ESPEN), the European Society for Paediatric Gastroenterology, Hepatology and Nutrition (ESPGHAN) and the European Cystic Fibrosis Society (ECFS) 2016.

Methods: In this cross-sectional study, data were collected prospectively in a single centre. A record was made for a total of 95 patients with CF of clinical data.

A Systematic Literature Review of the Relationship between Serum Ferritin and Outcomes in Myelodysplastic Syndromes.

Anemia is the most common form of cytopenia in patients with myelodysplastic syndromes (MDS), who require chronic red blood cell transfusions and may present high serum ferritin (SF) levels as a result of iron overload. To better understand the potential effects of high SF levels, we conducted a systematic literature review (SLR) to identify evidence on the relationship between SF levels and clinical, economic, or humanistic outcomes in adult patients with MDS. Of 267 references identified, 21 were included.

Development and content validation of the Satisfaction and Experience Questionnaire for Granulocyte Colony-Stimulating Factor (SEQ-G-CSF).

Background: Several options for granulocyte colony-stimulating factor (G-CSF) prophylaxis of chemotherapy-induced febrile neutropenia are available to patients worldwide. We have developed a novel patient-reported outcome measure, the Satisfaction and Experience Questionnaire for G-CSF (SEQ-G-CSF), to help understand patients' perspectives of and satisfaction with different G-CSF options.

Results: Three oncology nurses and 40 adult oncology patients in the United States were enrolled and participated in focus group discussions to develop and refine the SEQ-G-CSF.

Racial/ethnic differences in treatment quality among youth with primary care provider-initiated versus mental health specialist-initiated care for major depressive disorders.

Objectives: To compare the racial/ethnic differences in treatment quality among youth with primary care provider-initiated versus mental health specialist-initiated care for major depressive disorders (MDD).

Methods: A retrospective cohort study was conducted using the 2005-2007 Medicaid claims data from Texas. Youth aged 10-20 during the study period were identified if they had two consecutive MDD diagnoses and received either medications for MDD or psychotherapy.

Relationship of mesenteric panniculitis with visceral and subcutaneous adipose tissue.

Background/aim: Mesenteric panniculitis (MP) is an idiopathic benign disease characterized by fat necrosis, chronic inflammation, and fibrosis. The relationship between obesity and chronic low-grade inflammation has been reported. This study investigated the relationship of MP diagnosed using multidetector computed tomography (MDCT) with visceral adipose tissue (VAT) and subcutaneous adipose tissue (SAT) areas.

The long-term social value of granulocyte colony-stimulating factors.

Objectives: Febrile neutropenia (FN) is a life-threatening complication of chemotherapy that can lead to hospitalizations, chemotherapy dose reductions or delays, and mortality. Granulocyte colony-stimulating factor (G-CSF) prophylaxis reduces the incidence of FN, enabling patients to undergo and remain on myelosuppressive chemotherapy. We estimate the benefits of continuing current G-CSF use patterns and an alternative that aligns prophylactic G-CSF use with guideline recommendations.

The Effect of Enteral Nutrition Support Rich in TGF-β in the Treatment of Inflammatory Bowel Disease in Childhood.

Malnutrition is a major complication of inflammatory bowel disease (IBD). Our aim of the study was to examine the effects of Modulen IBD supplementation, which was administered to IBD patients without limiting their daily diet in addition to medical treatment, on the clinical, laboratory, anthropometric values, and disease activities of these patients. Seventy three children with IBD were evaluated retrospectively.

Clinical effectiveness and safety of erythropoietin-stimulating agents for the treatment of low- and intermediate-1-risk myelodysplastic syndrome: a systematic literature review.

Many patients with lower-risk myelodysplastic syndrome (MDS) experience anaemia, which has negative consequences. Erythropoiesis-stimulating agents (ESAs) and their biosimilars are used to treat anaemia in MDS and, currently, epoetin alfa and darbepoetin alfa are commonly used and recommended by clinical guidelines. To better understand the evidence available on the use of ESAs for anaemia in lower-risk MDS, we conducted a systematic literature review to identify randomized and nonrandomized prospective studies reporting on clinical efficacy/effectiveness, patient-reported quality of life (QoL), and safety.

Racial/ethnic differences in the treatment of adolescent major depressive disorders (MDD) across healthcare providers participating in the medicaid program.

Background: To examine whether racial/ethnic differences in receipt of MDD treatment could be explained by the specialty of provider diagnosing the adolescent.

Method: Adolescents (10-20 years-old) with ≥2 MDD diagnoses were identified using 2005-2007 Medicaid data from Texas. Patients were categorized based on the types of provider who gave the initial MDD diagnosis (psychiatrist (PSY-I), social worker/psychologist (SWP-I), and primary care physician (PCP-I)).

Ingestion of Fireworks: Rare Cause of Poisoning in Children.

Background: Mistaken ingestion of all manner of toxic matter is common in childhood, but poisoning with fireworks and matchsticks is rare. Fireworks usually contain 10% yellow phosphorus and 50% potassium chlorate. Potassium chlorate is an extremely reactive and toxic agent that is used in fireworks and matchstick heads.