ABSTRACT Background This cross-sectional study assessed health-related quality of life (HRQOL) outcomes and the factors affecting HRQOL in Chinese patients with β-thalassemia major (β-TM) receiving transfusion and iron chelation therapy. Research design and methods The Pediatric Quality of Life Inventory (PedsQL)4.0 Generic Core Scales were administered to 198 patients and 431 heathy controls aged 4–18 years old. The relationship of sociodemographic and clinical factors with HRQOLscores were analyzed using association and linear regression analyses. Results Compared with healthy peers, patients exhibited significantly lower PedsQL scores across all domains (p < 0.001). Patients with Body Mass Index (BMI) below the 50th percentile (the median BMI for Chinese children and adolescents) exhibited poorer HRQOL scores than those with BMI ator above this threshold in physical functioning (p = 0.034). Iron chelation therapy and pre-transfusion hemoglobin levels exceeding 7.5 g/dL emerged as significant positive predictors of total summary score, while the presence of two comorbidities was identified as significant negative predictors (p = 0.037, p < 0.001 and p = 0.001, respectively). Conclusions Standardized treatment involving blood transfusion and iron chelation therapy in β-TM patients requires further enhancement. Additionally, improving nutritional status and preventing and treating comorbidities may benefit patients’ HRQOL.

Abstract This study aimed to identify key determinants influencing red blood cell (RBC) transfusion efficacy in very low birth weight (VLBW) infants and develop a clinically applicable predictive model. We conducted a retrospective analysis of 993 eligible very low birth weight (VLBW) infants who received leukoreduced suspended RBC transfusions in the Neonatal Intensive Care Unit of Women and Children’s Hospital of Ningbo University between January 2020 and December 2023. Comprehensive data collection encompassed infant characteristics, clinical parameters, and donor information.Using logistic regression analysis, we identified five independent predictors for ineffective RBC transfusion: birth weight, postnatal age at transfusion, weight during transfusion, transfusion volume, and pre-transfusion hemoglobin levels. These variables were incorporated into a nomogram prediction model developed using R software. The model demonstrated strong predictive performance, with calibration curves showing excellent agreement between predicted and observed outcomes (AUC = 0.799). Our findings provide valuable insights into transfusion efficacy in VLBW infants and offer a practical tool for clinical decision-making.The validated nomogram enables individualized risk assessment, facilitating precision transfusion practices and potentially improving outcomes for this vulnerable patient population. This study represents a significant step toward optimizing transfusion strategies in neonatal care.

To evaluate red blood cell (RBC) transfusion practices in a pediatric intensive care unit (PICU) and identify factors predicting transfusion needs. A retrospective observational study was conducted on children (0-18 years) admitted to a tertiary academic PICU from January 2015 to December 2021. Demographic data, clinical parameters, and transfusion events were analyzed using Chi-square tests, logistic regression, and Kaplan-Meier estimation. Of 2,011 patients, 27.7% ( n = 558) received at least one RBC transfusion, with a median pre-transfusion hemoglobin level of 8.9 g/dL [IQR: 8.1-9.6]. Anemia was present in 47.59% ( n = 957) at admission, with 48.17% ( n = 461) requiring transfusion. Younger age (0-2 years: OR 2.22, 95% CI 1.9-2.61) and lower weight-for-age Z (WFA-Z) scores (severely underweight: OR 1.64, 95% CI 1.2-2.24) were associated with increased transfusion likelihood. Multiple transfusions were linked to higher mortality risk (OR 15.16, 95% CI 8.0-28.73, P < 0.001). Transfusion decisions in the PICU appeared to be influenced not only by hemoglobin values but also by age-related physiology, nutritional status, and baseline anemia. Anemia at discharge remained common, underscoring the need for structured post-discharge follow-up and prospective studies to refine individualized transfusion decision frameworks.

Reducing inequalities among patients with inherited, chronic, and complex disorders requiring lifelong care remains a global public health priority. Despite medical advances, inequalities in healthcare access and quality persist for patients with β-thalassaemia. This study examined the associations between socio-economic indicators at the individual and country levels and clinical outcomes in β-thalassaemia patients using data from the International Thalassaemia Collaborative Assessment Patient Survey. Data from 971 patients across 45 countries were analysed. Individual-level socio-economic status (SES) was assessed through patients' educational attainment and employment status, while country-level SES was represented by the Human Development Index and the World Bank Income classification. Clinical outcomes included pre-transfusion haemoglobin levels, serum ferritin concentration, cardiac T2* score, and liver iron concentration. Inequalities were quantified using the Slope Index of Inequality, representing absolute differences in outcomes between patients of lowest and highest SES. All SES indicators were positively associated with better clinical outcomes. Higher education and full-time employment corresponded to better levels of haemoglobin, ferritin, cardiac, and liver iron measures. Similarly, patients living in higher-income countries had better clinical measures compared to those living in lower-income countries. These findings indicate that socio-economic advantage is linked with improved disease management and monitoring quality. Socio-economic determinants influence the clinical outcomes of transfusion-dependent β-thalassaemia patients, which in turn affect their social integration and quality of life. Reducing these disparities requires investment in equitable healthcare systems, multidisciplinary care, and disease-specific public health policies.

Background/Objectives: Transfusion-dependent β-thalassaemia (TDT) is a lifelong condition requiring coordinated multidisciplinary care. In France, where the disease is rare, transition from pediatric to adult care remains poorly structured, potentially compromising adherence and long-term outcomes. Methods: This national retrospective study evaluated current transition practices and their clinical impact among young adults with TDT. Patients aged 20-25 years in December 2022 were identified from the national NaThalY registry. Those diagnosed and managed in France before age 15 were included. Clinical data were collected for the two years preceding and following transition. Transition practices were assessed using a standardized questionnaire sent to pediatric centers. Results: Thirty-four patients were included (mean transition age: 19 years). The rate of response to the questionnaire was 90.5%, with feedback from 19 centers. Only one-third of centers offered joint pediatric-adult consultations, and one-quarter provided transition-focused education. No written transition protocols were reported. Mean pre-transfusion hemoglobin levels were significantly lower after the transition (8.5 vs. 8.0 g/dL; p = 0.01). Ferritin levels showed a non-significant increase, with no statistically significant changes observed in hepatic or cardiac iron concentrations. Conclusions: This study demonstrates marked heterogeneity and limited formalization of transition practices in France. Development of structured, standardized transition pathways is urgently needed to ensure continuity of care and optimal disease management in adults with TDT.

Pediatric transfusion practices beyond critical care: Evidence for the need of standardized guidelines and patient blood management.

Chimeric antigen receptor T-cell (CAR-T) therapy has significantly improved overall survival (OS) in relapsed or refractory large B-cell lymphomas (R/R LBCL). However, factors associated with outcomes of CAR-T cell therapy in patients with R/R LBCL have not been fully elucidated. And limited evidence supports the use of early endpoints to evaluate the efficacy and long-term survival. Progression-free survival (PFS) at 6 months (PFS6) was defined as being alive and free of relapse or progression within 6 months of CAR-T cell infusion. We aimed to assessed OS stratified by PFS6 by analyzing data from 71 R/R LBCL patients treated with CAR-T therapy across 2 hospitals. Subsequent OS was defined from the time of PFS6 or progression within 6 months to death. Among them, 58% reached PFS6. Compared with patients failed to achieve PFS6, 1-year OS was 91.3% vs. 40.2% and 2-year OS was 91.3% vs. 32.1%, respectively. Patients achieving PFS6 had excellent outcome, whereas patients exhibiting earlier progression had a poor survival. Key predictors of PFS failure included older age (> 60) (P = 0.040, OR:3.40, 95%CI:1.06–10.93), lower pretransfusion hemoglobin level (P = 0.019 OR:0.27, 95%CI:0.09–0.81), and higher IFN-ʏ level (P = 0.022, OR:2.00, 95% CI:1.66–4.08). This insight could aid in risk stratification and support the use of PFS6 as a surrogate endpoint in clinical trials.

This article aims to analyze current red blood cell (RBC) transfusion practices and evaluate the efficacy and safety of different transfusion thresholds in patients with pediatric acute leukemia (AL) at a single center. Clinical data from 220 children with AL who received RBC transfusions at the Third Xiangya Hospital of Central South University between January 1 and December 31, 2023, were retrospectively collected. Patients were divided into three groups based on pre-transfusion hemoglobin (Hb) levels: a low Hb group (≤ 60 g/L, n = 43), a medium Hb group (61–70 g/L, n = 137), and a high Hb group (&gt; 70 g/L, n = 40). Basic clinical characteristics, Hb increment (ΔHb), anemia symptom improvement rate, and incidence of transfusion-related adverse reactions were compared among the groups. The median pre-transfusion Hb threshold was approximately 66 g/L across all subgroups based on age, disease subtype, presence of co-existing infection, or anemia symptoms, with no statistically significant differences observed among the groups (P &gt; 0.05). No significant differences were identified among the three groups regarding transfusion dose or ΔHb (median: 15 g/L, 16 g/L, and 17 g/L, respectively; P = 0.581). Among the 55 children with pre-transfusion anemia symptoms, symptom improvement rates also showed no significant differences among the three groups (P &gt; 0.05). The overall incidence of transfusion-related adverse reactions was 0.91% (2/220), with no differences among the groups. The results suggest that RBC transfusions effectively increase Hb levels and alleviate symptoms across different threshold groups, with a good safety profile. No clear association was observed between transfusion thresholds and efficacy or safety.

Biomarkers of ineffective erythropoiesis in patients with transfusion- dependent thalassemia

Early efficacy and safety of luspatercept in myelofibrosis-associated anemia: A clinical study with biomarker exploration

BackgroundThere is a knowledge gap in the outpatient transfusion practice of red blood cells (RBC) according to optimal trigger, dose, and interval as well as which patients are in need of outpatient transfusion.Study Design and MethodsThis single‐center retrospective analysis investigates RBC transfusion practices in 804 patients with 5591 visits.ResultsBlood disorders and solid tumors were the primary indications for transfusion, accounting for 82% of RBC use. Median (interquartile range) pretransfusion hemoglobin (Hb) level was 7.5 (6.9;8.1)g/dL. Linear regression models revealed that Hb increased by 1.04 g/dL per unit transfused and identified the concomitant transfusion of platelets, male sex, higher height, weight, and lower pre‐transfusion Hb level as negative factors for the increase in Hb. Factors that correlate with Hb decline after transfusion include lower reticulocytes, male sex, height, and concomitant platelet transfusion. A longer interval to next transfusion was associated with higher reticulocyte counts, higher pre‐transfusion Hb levels and number of units transfused. According to our models, the potential for reducing the use of RBC through single‐unit transfusions decreases with increasing height, concomitant platelet transfusions, and lower pretransfusion Hb levels and is going along with a shorter interval to next transfusion. Nonsevere adverse reactions occurred in ≤0.24% of transfusions.ConclusionThese results highlighted the effect of patient age, sex, Hb, height, and weight on outpatient RBC transfusion. These variables have to be considered in clinical practice and trials evaluating patient outcomes.

Purpose: This study evaluates the efficacy of a patient blood management (PBM) initiative in reducing unnecessary red blood cell (RBC) transfusions at a New York City community teaching hospital over eight years (2013-2020). Methods: A retrospective analysis of RBC transfusion data was performed, covering the period from 2013 to 2020. Findings: Post-PBM implementation, notable advancements were recorded annually. Mean pretransfusion hemoglobin (Hgb) levels decreased from 7.26 g/dL in 2013 to 6.68 g/dL in 2020. Annual RBC transfusion units decreased, with units transfused at Hgb ≥ 7 g/dL falling from 1210 (58.7%) in 2013 to 377 (20.0%) in 2020, a drop of 39%. Two-unit RBC orders at Hgb ≥ 7 g/dL declined from 65 in 2013 to 10 in 2020. Estimated cost savings from 2014 to 2020 totaled US Dollar (USD) 2.2 million. Conclusions: The PBM program significantly curtailed unnecessary RBC transfusions and optimized transfusion practices, demonstrating that resource-light, evidence-based strategies can yield substantial clinical and economic benefits.

Transfusion-dependent thalassemia is a major public health concern in Nepal, with limited access to comprehensive care and paucity of national data. The main objectives of this study were to explore clinico-epidemiological profile, management practices and complications among pediatric transfusion-dependent thalassemia patients at a tertiary pediatric hospital in Nepal. This is a retrospective observational study conducted at the Thalassemia Day Care Unit, Kanti Children's Hospital, Kathmandu. Transfusion-dependent thalassemia cases registered from January 2020 to December 2024, aged less than 15 years were included. Data on demographics, clinical features, transfusion and chelation profiles, complications, and nutritional status were acquired from hospital registry and analyzed using descriptive statistics. Out of 187 patients, 121 (64.71%) were males and 156 (83.42%) had β-thalassemia major. Mean age at last follow-up was 7.52±3.68 years. 83 (44.38%) were Janajatis, particularly Tharus 40 (21.39%). Median age at diagnosis was 10 months (IQR 6-22 months), with mean pre-transfusion hemoglobin level 8.86 g/dL, and average transfusion period 2.64±0.83 weeks. Among 167 (89.30%) patients receiving chelation, 37 (19.87%) had good compliance. Endocrinopathies, hepatic dysfunction and cardiac complications were observed in 104 (55.62%), 65 (34.76%) and 2 (1.07%) patients respectively. Serum ferritin levels were above 2500 ng/mL in 92 (49.19%) patients. Regular follow up was done by 56 (29.95%) patients. β-thalassemia major was the most common type of transfusion dependent thalassemia in our study. There was male predominance, and over half of the patients had iron overload and some form of complications of thalassemia. Regular follow up and compliance to chelation therapy has to be focused for better care of transfusion dependent thalassemia patients.

Blood transfusions in the emergency department: A descriptive analysis and referral patterns at a non-trauma center.

Background: Thalassemia major, a severe form of thalassemia, is marked by inherited defects in haemoglobin (Hb) production, leading to anemia and potential growth retardation without regular blood transfusions. Objective: This study evaluated the growth patterns and the association between mean serum ferritin levels and growth in children with transfusion-dependent thalassemia major. Methods: The Shri Maharaja Gulab Singh Hospital, GMC Jammu, hosted this potential cohort study for duration of 1 year, from November 1, 2021, to October 31, 2022. A total of 125 transfusion-dependent thalassemia major patients aged 1–16 years were included. Anthropometric measurements (height, weight, and head circumference) were taken at baseline and every 3 months, compared with reference growth standards. Patients were grouped based on mean Hb levels before transfusion: &lt;8 g/dL and ≥8 g/dL. Data on leukocyte count, platelet count, serum ferritin, and other biochemical parameters were collected. SPSS software was used to carry out the statistical analysis. Quantitative data were presented as means ± standard deviation or median with interquartile range, while categorical variables were shown as percentages. The Kolmogorov–Smirnov test was used to verify normality. In addition to Chi-square or Fisher’s exact tests for categorical variables, parametric (independent t -test and analysis of variance) and nonparametric (Mann–Whitney test) tests were employed. P &lt;0.05 was regarded as statistically significant. Results: Among patients, 25 had short stature, while 72 (65.6%) had heights between the 3 rd and 50 th percentiles. Twenty patients (16%) were severely underweight. Higher proportions of patients aged 11–16 years had mean pretransfusion Hb &lt;8 g/dL, with short stature more common in this group. Mean serum ferritin in patients with short stature was significantly higher (6432.32 ng/mL, P = 0.0004). Conclusion: Growth failure in transfusion-dependent thalassemia major worsens with age, especially in adolescents. Elevated serum ferritin levels negatively impact growth, highlighting the need for monitoring iron overload.

Background Thalassaemia is a significant public health concern in Southeast Asia, yet little is known about health risk behaviours in adolescents with this condition. This study aimed to compare health risk behaviours between adolescents with thalassaemia and healthy controls, and to identify associated clinical and sociodemographic factors. Methods This cross-sectional study included 60 adolescents with thalassaemia (aged 10–18 years) and 60 age- and sex-matched healthy controls in Thailand. Health risk behaviours were assessed using the validated Thai Youth Risk Behaviour Survey. Clinical data were extracted from medical records. Multivariable logistic regression was used to identify factors associated with risk behaviours. Results Adolescents with thalassaemia reported higher rates of non-seatbelt use (25.0% vs. 1.7%, p < 0.001) and mental health problems (13.3% vs. 0.8%, p = 0.027) compared to controls. However, they exhibited lower rates of inadequate exercise (70.0% vs. 86.7%, p = 0.045) and excessive screen time (66.7% vs. 96.7%, p < 0.001). In the thalassaemia group, older age (aOR = 2.78, 95% CI: 1.09–7.09), female gender (aOR = 39.73, 95% CI: 0.89–1770.49) and lower pre-transfusion haemoglobin levels (aOR = 0.09, 95% CI: 0.01–0.64) were associated with increased odds of mental health problems. Higher pre-transfusion haemoglobin levels were associated with lower odds of violent behaviour (aOR = 0.44, 95% CI: 0.21–0.92). Conclusions Adolescents with thalassaemia exhibit distinct patterns of health risk behaviours, influenced by age, gender and disease severity. These findings underscore the need for integrated physical and psychosocial care, highlighting the importance of tailored health education and interventions for this vulnerable group.

Abstract Aim It is recognised that transfusing outside of ‘routine’ hours is less safe and should be avoided unless clinically essential. This audit evaluates out-of-hours red cell concentrate (RCC) transfusions by indication. Method We identified prospectively RCC transfusions occurring out of hours in our institution over a one-month period. Indications were examined against NICE blood transfusion guidelines. Based on these guidelines and advice from our local haemovigilance officer we defined five criteria for an appropriate out of hours transfusion. Transfusions fulfilling any one of these criteria were defined as appropriate. Results Following withdrawals, data on 51 units of RCC transfused out of hours were included in the analysis of which 70.6% were defined as appropriate as per our criteria. Transfusion recipients were male in 54.9% of cases. The mean pre-transfusion haemoglobin (Hb) level was 7.6 m/dL. The mean post-transfusion Hb was 8.4 m/dL. Incidence of active haemorrhage was 31.4%. Incidence of symptomatic anaemia was 51%. The haemoglobin level was appropriately low in 29.4% of cases. 3.9% of the transfusions were given in the context of perioperative haemorrhage. A further 3.9% were given for low haemoglobin pre-operatively. There were no recorded transfusion complications. Conclusions Although the majority of transfusions were clinically indicated, 29.4% of out-of-hours RCC transfusions were not indicated under our criteria. We recommend the implementation of a verbal proforma prior to a re-audit, with a view to improving patient safety and compliance. Guidelines outlining appropriate requests should be developed and reasons for delays in the transfusion process be explored.

Background: Thalassemia is a common hereditary blood disorder in Bangladesh, requiring regular blood transfusions and iron chelation therapy to manage its complications and improve life quality. Objective: The study aimed to evaluate the pretransfusion hemoglobin levels, the pattern of iron chelation therapy, and the associated complications in transfusion-dependent thalassemia patients. Methods: This hospital-based cross-sectional descriptive study was conducted over six months (November 2016 to April 2017) at Rajshahi Medical College Hospital, with 75 transfusion-dependent thalassemia patients. Data were collected through structured questionnaires from patients' parents, and statistical analysis was performed using SPSS (version 16) to assess means, standard deviations, and p-values. Results: The average age of the patients was 7.7±2.3 years, with 57.33% in the 5-10 years age group. The first blood transfusion occurred at 2.5 years. The mean number of blood transfusions was 52.48±23.90 units. Serum ferritin levels were elevated (mean 3638.74±2567.79 ng/ml), and pretransfusion hemoglobin was low (7.2±1.56 gm/dl). Only 9.33% received regular chelation therapy, and 74.67% did not use any form of chelation. Statistical analysis revealed that patients with facial dysmorphism had significantly lower hemoglobin (6.32 gm/dl, p=0.03) and higher serum ferritin (4774.46 ng/ml in stunted patients). Patients with &gt;50 transfusions had a higher serum ferritin level. Additionally, a longer duration of chelation therapy showed a lower serum ferritin level. Conclusion: This study indicates that thalassemia patients often receive transfusions irregularly and exhibit delayed chelation therapy initiation. Most patients fail to adhere to regular chelation despite elevated serum ferritin levels.

Background: Beta thalassemia major is a genetic blood disorder inherited by parents thalassemia traits (carrier) characterized by abnormal hemoglobin formation. Abnormalities during the process of hemoglobin formation cause gene changes or mutations that cause symptoms of severe anemia which has a long-term clinical impact on patients. High performance liquid chromatography can be used to determine the description of abnormal hemoglobin analysis, thus suspected diagnosis of thalassemia β major can be established by the clinician as a definitive diagnosis to enhance a more comprehensive diagnosis. Primary theraphy of beta thalassemia major is blood transfusion to support the quality of life regularly throughout the patient's life, transfusion of patients requires measurements such as pre-transfusion hemoglobin levels. Objective: This study aims to determine the description of the results of the analysis of hemoglobin patients with suspected thalassemia beta major at RSUP Dr. M. Djamil Padang. Methods: This study used a cross sectional design. The sampling technique uses total sampling with a total sample of 19 people from 1-35 years old. Patient data was from the suspected diagnosis of medical records established by clinicians at Dr. M. Djamil Hospital Padang. This data was analyzed univariately. Results: The results of this study indicated is mostly female with an average age of 13,74 years (SD ±12.1). This study resulted in a low pretransfusion hemoglobin average of 6.20 g/dL. The results of hemoglobin analysis for beta thalassemia major with average HbA (48,1%), HbA2 (27,3%) and HbF (24,5%). Conclusion: The results of the examination hemoglobin analysis on the HPLC method had results that did not compatible predictions of the suspected diagnosis of beta thalassemia major from the clinician

Background: β-Thalassemia is a hereditary hemoglobin disorder characterized by chronic hemolytic anemia and increased transfusion dependency. Splenectomy is often performed in patients with massive splenomegaly, hypersplenism, or excessive transfusion requirements to reduce hemolysis and improve hematological parameters. However, its impact on cardiac function and post-splenectomy complications remains a subject of concern. This study aims to evaluate the hematological, echocardiographic, and cardiovascular changes before and after splenectomy in β-thalassemia patients. Methods: A longitudinal descriptive observational study was conducted at the Department of Paediatric Surgery, Bangladesh Shishu Hospital &amp; Institute, Dhaka, Bangladesh from January 2017 to April 2018. A total of 40 β-thalassemia patients undergoing splenectomy were included. Hematological parameters (hemoglobin levels, transfusion requirements, and serum ferritin) and echocardiographic parameters (left ventricular diameters, mass, and cardiac output) were assessed pre- and post-splenectomy. Data were analyzed using SPSS version X, with paired t-tests applied for statistical comparisons. Results: Post-splenectomy, there was a significant increase in pre-transfusion hemoglobin levels (4.19 ± 0.92 g/dL to 8.66 ± 0.73 g/dL, p &lt; 0.001) and a marked reduction in transfusion needs (14.30 ± 3.18 to 2.15 ± 1.46 units per six months, p &lt; 0.001). Serum ferritin levels increased non-significantly (p = 0.085). Cardiac output decreased significantly (5.85 ± 1.72 L/min to 4.99 ± 1.64 L/min, p = 0.020), while left ventricular parameters remained unchanged. Thrombocytosis developed in 75% of patients, with 15% requiring aspirin therapy due to extreme thrombocytosis. Malaria was the most common post-splenectomy infection (18.75%), but no cases of overwhelming post-splenectomy infection (OPSI) were recorded. Conclusion: Splenectomy significantly reduces transfusion requirements and improves hemoglobin levels in β-thal