Sickle cell disease (SCD) is due to the mutation of haemoglobin (Hb), from HbA to HbS and characterised by recurrent vaso-occlusive crises (VOC), which can progress to acute chest syndrome (ACS), a leading cause of death in adults with SCD. Hypoxia is a key modifiable factor in the polymerisation of HbS and the pathogenesis of VOC. High-flow nasal oxygen (HFNO) delivers humidified gas at high oxygen concentrations and flow rates: the former may reverse sickling (metabolic effect) to accelerate VOC resolution and prevent ACS, while the latter may reduce the risk of ACS by mitigating hypercapnia and generating positive airway pressure that limits hypoventilation and atelectasis (pulmonary effect). The study hypothesises that HFNO is a safe and effective strategy for treating VOC and preventing secondary ACS, and will assess this using a multi-arm multi-stage (MAMS) trial design.

This is a prospective, multicentre, randomised, open-label controlled trial following an MAMS design with three phases and four arms: one control (low-flow oxygen) and three HFNO intervention arms with varying fraction of inspired oxygen levels (low, intermediate, high). The pilot stage will assess safety and feasibility, using the rate of cardiac and neurological events as the primary endpoint. In the activity stage, arms demonstrating acceptable safety will be compared for efficacy based on the rate of VOC resolution without complications by day 5, allowing selection of the most promising arm. The final efficacy stage will compare the selected HFNO strategy to control, with prevention of secondary ACS by day 14 as the primary endpoint. The study aims to enrol up to 350 VOC episodes in total.

The study has been granted ethical approval (CPP SUD MEDITERRANEE IV). Following the provision of informed consent, patients will be included in the study. The results will be submitted for publication in peer-reviewed journals.

NCT03976180.

Although flow cytometric analysis of peripheral blood neutrophil myeloperoxidase expression can accurately rule out myelodysplastic neoplasms (MDS), it lacks reliability and efficiency due to the practical limitations of laboratory-developed liquid reagent-based assays. This study aimed to quantify the agreement and comparative discriminatory accuracy between a single-use flow cytometric lyophilised reagent tube (BD Lyotube Stain 468) and its laboratory-developed liquid reagent counterpart.

Cross-sectional diagnostic accuracy study of two index tests against a reference diagnosis.

A university hospital in France.

Consecutive adult patients with an indication for bone marrow aspiration due to suspected MDS and unexplained peripheral blood cytopenia.

MDS confirmed by cytomorphological evaluation of the bone marrow aspirate performed in duplicate by experienced haematopathologists blinded to the index test.

Of 103 participants enrolled between July 2020 and August 2021, 37 had MDS (prevalence, 36%). The median intra-individual robust coefficient of variation (RCV) for myeloperoxidase expression was 30.9% using the BD Lyotube Stain 468 and 31.2% using the laboratory-developed liquid reagent assay, with an intraclass correlation coefficient of 0.94 (95% CI 0.91 to 0.96). The areas under the receiver operating characteristic curves were 0.83 (95% CI 0.74 to 0.90) and 0.82 (95% CI 0.73 to 0.89), respectively. Using a prespecified threshold of 30.0%, the corresponding sensitivity estimates were 89% (95% CI 75% to 97%) and 95% (95% CI 82% to 99%).

BD Lyotube Stain 468 performs as well as its laboratory-developed liquid reagent counterpart for the quantification of myeloperoxidase expression by peripheral blood neutrophils. It may obviate the need for invasive bone marrow aspiration in up to 40% of patients with suspected MDS.

NCT04399018.

This study aimed to develop and validate a machine-learning (ML) model to predict iron deficiency without anaemia (IDWA) using routinely collected electronic health record (EHR) data. The primary hypothesis was that an ML model could achieve better accuracy in identifying low ferritin levels (

A retrospective cohort study.

Data were derived from secondary and tertiary care facilities within the eight-hospital Mount Sinai Health System, an urban academic health system.

The study included 211 486 adult patients (aged ≥18 years) with normal haemoglobin levels (≥130 g/L for men and ≥120 g/L for women) and recorded ferritin measurements.

The primary outcome was the prediction of low ferritin levels (

Data from 211 486 Mount Sinai Health System patients with normal haemoglobin levels and ferritin testing were analysed. The model used demographic data, blood count indices and chemistry results to identify low ferritin levels (

Of the 211 486 patients analysed, 19.56% (n=41 368) of the patients had low ferritin levels. In the low ferritin group, the mean age was 41.28 years with 89.64% females. In contrast, the normal ferritin group had a mean age of 50.14 years with 62.02% females. The model achieved an area under the curve (AUC) of 0.814. At a sensitivity threshold of 70%, the model had a specificity of 75.85%, with a positive predictive value of 37.6% and a negative predictive value of 92.41%. The model outperformed an alternative model based only on complete blood count indices (AUC 0.814 vs 0.741). Subgroup analysis showed that model accuracy varied by sex and age, with lower performance in premenopausal women (AUC 0.736) compared with postmenopausal women (AUC 0.793) and men (AUC of 0.832 in those under 60 years and 0.806 in those aged 60 and above).

The ML model provides an effective approach to screening for IDWA using readily available EHR data. Implementing this tool in clinical settings may facilitate early diagnosis of IDWA.

Sickle cell disease (SCD) is associated with significant mortality and morbidity, especially in low- and middle-income countries.

We determined the indications for hospitalisation and predictors of 30-day re-admission among patients with SCD in Northern and Central Uganda.

Retrospective chart review.

Mulago National Referral Hospital in Kampala, St. Mary’s Hospital Lacor in Gulu and Gulu Regional Referral Hospital in Gulu, Uganda.

Patients with confirmed SCD admitted between January 2020 and January 2025 were included.

Primary outcome: indication for hospitalisation. Secondary outcomes: rate and predictors of 30-day hospital re-admission. Socio-demographic, clinical history and hospitalisation data were extracted using a pretested data extraction tool.

We enrolled 505 patients, accounting for 714 hospital admissions, with a mean age of 8.1±6.2 years. Most participants (n=489, 96.8%) had less than four admissions per year, with a median of 1 admission (IQR: 0–2). The most common indications for hospitalisation were infection (n=375, 52.5%), painful crisis (n=366, 51.3%) and anaemia (n=186, 26.1%). Malaria was the most prevalent infection (n=244, 65%). The median length of hospital stay was 4 days (IQR: 3–6), with a 30-day re-admission rate of 6.9% (n=49). Admission with painful crisis (adjusted OR (AOR): 0.45, 95% CI: 0.23 to 0.89, p=0.021), receiving a blood product (AOR: 0.32, 95% CI: 0.16 to 0.66, p=0.002) and having four or more admissions per year (AOR: 0.84, 95% CI: 0.04 to 0.17, p

Infections, especially malaria, and painful crises were the leading causes of hospitalisation among Ugandan patients with SCD. Frequent admissions, painful crises and blood transfusions were associated with lower 30-day re-admission risk. There is an urgent need to strengthen malaria prevention strategies and optimise access to disease-modifying therapy, such as hydroxyurea, to improve patient outcomes.

Cardiac surgery remains a high-risk procedure for bleeding despite advances in patient blood management. Conventional centrifugation-based autotransfusion devices primarily recover red blood cells, losing platelets and coagulation factors. The SAME autotransfusion device (i-SEP, Nantes, France) introduces an innovative filtration-based approach, recovering erythrocytes, leucocytes and platelets to enhance perioperative haemostasis. The main objective is to determine whether the filtration-based SAME device reduces significant perioperative bleeding compared with the centrifugation-based system in high-risk cardiac surgery patients.

The Centrifugation-based vs filtration-based intraOperative cell saLvage on qualiTy of peRioperAtive haemostasis iN cardiac surgEry (COLTRANE) trial is a multicentre, parallel-group, single-blinded, superiority-randomised clinical trial. Conducted over 19 months in 10 French hospitals, the study will target patients at high risk of bleeding undergoing on-pump cardiac surgery via sternotomy. A total of 570 patients (285 per group) are required to achieve 80% statistical power for detecting clinically significant differences. Eligible patients will be randomised to either a centrifugation-based or filtration-based autotransfusion group. Both groups will follow standardised perioperative and cardiopulmonary bypass management, with the devices used only intraoperatively. The primary outcome is the proportion of patients with clinically significant perioperative bleeding defined as classes 2 to 4 of the Universal Definition of Perioperative Bleeding. The secondary outcomes include device efficiency and safety, perioperative haemostasis, lengths of intensive care unit and hospital stays, early postoperative morbidity and 30-day all-cause mortality. Ancillary studies will be performed to evaluate cell recovery and washing performance, the viscoelastic properties of retransfused blood (Quantra Qplus; Stago, Asnières-sur-Seine, France), and the effect of salvaged leucocytes on postoperative inflammation and immune function.

This trial has received a favourable opinion from the Committee for the Protection of Persons and authorisation from the French authorities (Comité de protection des personnes Nord Ouest, IDRCB: 2023-A02566-39). Protocol V.1.1 was approved on 22 January 2024. The trial is registered on ClinicalTrials.gov (NCT06425614). The findings will be disseminated through oral communications at national and international scientific meetings and peer-reviewed journal publications. Individual participant data will be made available on reasonable request to qualified researchers, following review and approval by the study sponsor and ethics committee.

ClinicalTrials.gov, NCT06425614.

To investigate anxiety levels, as well as attitudes and practices toward anxiety in patients undergoing haemodialysis.

Cross-sectional study conducted from May to August 2023.

This study was conducted from May to August 2023 at multiple haemodialysis units in Qingdao, China.

Adult patients who regularly undergo haemodialysis were invited to participate.

NA.

The participants’ anxiety, attitudes and practices related to their condition were measured by a self-designed questionnaire. The demographic characteristics were also collected as exposure variables.

A total of 535 patients participated, with a mean age of 55.50±14.30 years; 314 were male. The average duration of haemodialysis was 4.69±3.80 years. The patients had a mean anxiety score of 59.71±7.59. Attitude and practice scores averaged 17.66±4.88 (range: 6–30) and 32.12±6.28 (range: 8–40), respectively. Multivariate linear regression showed that a monthly household income of ¥5001–10 000 (β=1.2, p=0.006) and anxiety score (β=0.215, p

Patients undergoing haemodialysis exhibited high anxiety levels alongside proactive practices and negative attitudes. Anxiety was positively associated with attitudes but negatively with practices, indicating that proactive practices may alleviate anxiety. Targeted mental health strategies are needed to enhance the quality of life for haemodialysis patients.

To develop and validate a prediction model of Philadelphia chromosome-positive acute lymphoblastic leukaemia (Ph+ALL).

A single-centre retrospective study.

This study analysed 471 newly diagnosed patients with ALL at the Second Affiliated Hospital of Army Medical University from January 2014 to December 2023.

Clinical and laboratory parameters were collected, and the important characteristic parameters were selected using BorutaShap. Multiple machine learning (ML) models were constructed and optimised by using the active learning (AL) algorithm. Performance was evaluated using the area under the curve (AUC), comprehensive indicators and decision curve analysis. The interpretability of the model was evaluated by using SHapley Additive Interpretation (SHAP), and external validation was conducted on an independent test cohort.

10 parameters were selected to construct multiple ML models. The CatBoost model integrated with an AL algorithm (CatBoost-AL) was found to be the most effective model for predicting Ph+ALL within the validation data set. This model achieved an AUC of 0.797 (95% CI 0.710 to 0.884), along with sensitivity, specificity and F1 score of 0.667, 0.864 and 0.777, respectively. The prediction performance of CatBoost-AL was further validated with an external testing set, where it maintained a strong AUC of 0.794 (95% CI 0.707 to 0.881). Using SHAP for global interpretability analysis, age, monocyte count, -glutamyl transferase, neutrophil count and alanine aminotransferase were identified as crucial parameters that significantly influence the diagnostic accuracy of CatBoost-AL.

An interpretable ML model and online prediction tool were developed to determine whether newly diagnosed patients with ALL are Ph+ALL. The key parameters identified by the optimal model provided a further understanding of Ph+ALL characteristics and were valuable for accurate diagnosis and treatment of Ph+ALL.

This study aims to evaluate the prevalence of nutritional anaemia and identify its associated factors among pregnant women in the Sibu Sire District, Western Ethiopia.

A facility-based cross-sectional study.

Public health centres in the Sibu Sire District, Western Ethiopia.

A total of 422 pregnant women attending antenatal care services between February and July 2023.

The primary outcome was the prevalence of nutritional anaemia, determined by haemoglobin concentration. Secondary outcomes included the relationship between nutritional anaemia and factors such as household size, maternal nutritional status (mid-upper arm circumference (MUAC)) and dietary diversity.

Nutritional anaemia was prevalent in 45.7% of the study participants. Multivariable logistic regression analysis revealed significant associations between anaemia and household size (adjusted OR (AOR) = 1.43; 95% CI 1.05 to 1.94), poor maternal nutritional status (MUAC

Nutritional anaemia remains a significant public health issue among pregnant women in the Sibu Sire District, affecting nearly half of the study population. The findings underscore the critical influence of household size, maternal nutritional status and dietary diversity on anaemia risk during pregnancy. Addressing these modifiable factors through comprehensive antenatal nutrition programmes, community-based education and targeted interventions may improve maternal and fetal health outcomes in resource-limited settings.

To investigate the knowledge of stroke and the attitudes towards stroke and prehospital delay among patients who had an acute ischaemic stroke (AIS) and their family members.

This cross-sectional study was conducted through a self-designed questionnaire.

The study took place in a Grade-A tertiary hospital in Zhejiang Province, China, between July 2023 and November 2023.

A total of 521 valid questionnaires were collected from 367 patients who had an AIS and 154 family members.

Participants provided demographic information and answered questions related to stroke knowledge, attitudes towards stroke and prehospital delay.

The primary outcome measures included scores on stroke knowledge, attitudes towards stroke and attitudes towards prehospital delay. Secondary outcomes focused on identifying correlations and independent factors influencing prehospital delay.

The average scores for patients were stroke knowledge 8.74±6.16 (range: 0–24), stroke attitude 23.52±2.73 (range: 7–35) and prehospital delay attitude 38.65±7.68 (range: 10–50). Family members scored 12.66±6.85, 23.60±2.57 and 40.02±7.45, respectively. Significant correlations were found between stroke knowledge and attitude (r=0.2262, p

Both patients who had an AIS and their family members demonstrated insufficient knowledge and moderate attitudes towards stroke and prehospital delay, which were associated with extended prehospital delay. Educational interventions are necessary to enhance stroke knowledge. Targeted stroke awareness programmes and rapid response training could help improve early recognition and timely medical intervention, reducing prehospital delay and improving patient outcomes.

Haematological cancers are common in the UK, with a variety of morphologies. Stem cell transplants and chimeric antigen receptor (CAR) T-cell therapies provide significant options for hard to treat haematological cancers, although with difficult to predict outcomes. Research into the determinates of treatment efficacy, and access to treatments, is key to ensuring equal benefit across patients and patient safety. With this, there are concerns about the small representation of minority groups in related research. We aim to report on the current knowledge to guide future research.

A variety of databases will be searched for literature on UK minority ethnic populations receiving haematopoietic stem cell transplant or CAR T-cell therapy. Searches will be restricted to the year 2011 or later. Many outcomes will be analysed, covering the patient care pathway for those of the target population, although with a focus on follow-up after therapy. Plans have been made to conduct narrative synthesis, with meta-analysis where applicable.

Ethical approval is not required for this study. Outputs will be published in an appropriate journal and discussed with the wider National Institute for Health and Care Research Blood and Transplant Research Unit in Precision Transplant and Cellular Therapeutics (BTRU) group. Discussions will also be undertaken with the BTRU patient partners group.

The current diagnostic pathway for patients with a suspected inherited bleeding disorder is long, costly, resource intensive, emotionally draining for patients and often futile, as half of patients will remain without a diagnosis and be labelled ‘bleeding disorder of unknown cause’. Advances in understanding the genetic basis of the inherited bleeding disorders, coupled with both increasing infrastructure for genetic/genomic testing and decreasing costs, have increased the feasibility of introducing genomic testing into the clinical diagnostic pathway as a potential solution to improve the care of these patients. Yet, there remain evidence gaps on the optimal integration of genomic analysis into the diagnostic pathway.

Using a multicentre randomised-controlled trial design, we will evaluate an early genomic testing strategy for the diagnosis of newly referred patients with a suspected inherited bleeding disorder. Eligible participants will be randomised to early genomic testing diagnostic pathway (intervention) or standard diagnostic pathway (control) and will be followed for a 12-month period. Patients in the control group who remain undiagnosed at study end will be offered identical early genomic testing to ensure equitable access to the intervention. The study will follow a parallel fixed design with waitlist control group and a 1:1 allocation ratio. The study will be conducted at three tertiary care centres in Ontario, Canada, with a target sample size of 212 participants. Clinical utility will be evaluated via the primary outcome of diagnostic yield, as well as the secondary outcome of time to diagnosis. Additional secondary outcomes will allow for assessment of patient impact via health-related quality of life and patient burden measures, as well as evaluation of economic impact through a cost-effectiveness analysis and budget impact analysis.

This investigator-initiated study was approved by the Queen’s University Health Sciences and Affiliated Teaching Hospitals Research Ethics Board through Clinical Trials Ontario (CTO-4909). Participant informed consent/assent is required. Findings will be disseminated through academic publications.

ClinicalTrials.gov, NCT06736158.