Search Medical Journals & Research Articles

[Clinical Picture] Coexisting genetic abnormalities and thrombocytopenia

A 7-month-old boy presented to Osaka City General Hospital, Osaka, Japan, with persistent thrombocytopenia since birth. Despite treatments including intravenous immunoglobulin, prednisolone, romiplostim, and rituximab (details provided in the appendix p 1), platelet counts remained at approximately 20–30 × 109 platelets per L (normal range 158–348 × 109 platelets per L). A blood transfusion was administered when the platelet count fell below 5 × 109 platelets per L. After platelet transfusions, the platelet count could be maintained for several days.

[Viewpoint] Sustainable anticoagulation for climate resilient care

This Viewpoint issues a multidisciplinary call to embed sustainability into anticoagulation care, aligning clinical excellence with climate responsibility. Anticoagulation management—including drug manufacturing, laboratory diagnostics, therapeutic monitoring, and long-term follow-up—contributes substantially to health-care-associated carbon emissions. We propose a systems-level decarbonisation strategy, anchored in life cycle assessment methodology, to quantify emissions across anticoagulation care and identify high-impact intervention points.

[Review] Von Willebrand factor and von Willebrand disease in ageing: mechanisms, evolving phenotypes, and clinical implications

The global population is ageing and this demographic shift has profound effects on haemostasis, notably a progressive tilt towards a hypercoagulable state. A major age-associated change in haemostasis is the increase in von Willebrand factor (VWF), a plasma glycoprotein essential for primary and secondary haemostasis. VWF deficiency causes von Willebrand disease, which is the most common inherited bleeding disorder and affects approximately 1% of the population. Conversely, elevated VWF concentrations are linked to increased thrombotic risk; VWF concentrations increase with age by approximately 10–15 IU/dL per decade.

[Articles] Patient-reported outcomes with belantamab mafodotin, pomalidomide, and dexamethasone versus bortezomib, pomalidomide, and dexamethasone in patients with relapsed or refractory multiple myeloma (DREAMM-8): a phase 3, open-label, randomised controlled trial

Patients with relapsed or refractory multiple myeloma treated with belantamab mafodotin, pomalidomide, and dexamethasone or bortezomib, pomalidomide, and dexamethasone reported stable HRQOL. Self-reported ocular adverse events were generally manageable and minimally bothersome within the first year of treatment. These findings indicate that belantamab mafodotin, pomalidomide, and dexamethasone is well tolerated, with little detriment to HRQOL, supporting its use in relapsed or refractory multiple myeloma.

[In Focus] Perspectives of potential haematopoietic stem-cell donors on education TikToks

TikTok is a rapidly growing social media platform on which users develop, interact with, or share short vertical videos (TikToks). Although TikToks have immense potential to be harnessed to affect health knowledge, attitudes, and behaviours, little research guides TikToks’ potential public health applications. Since 2020, we have led the development and use of a large TikTok library to engage diverse young adults to haematopoietic stem-cell donation. This library, including hundreds of TikToks starring diverse performers, has generated hundreds of thousands of views and engagements across social media platforms.

[In Focus] Incorporating national disease burden in GBD estimates of haemoglobinopathies in Italy

The Global Burden of Disease (GBD) study represents an incredible source of information to quantify and understand the magnitude and impact of all conditions, including common and rare diseases, on patients’ quality of life, mortality, and morbidity. Emerging evidence suggests that detailed national studies are needed to inform, calibrate, and validate GBD estimates. This need is also supported by action on local GBD network, such as the Italian GBD network, cross-talking with the global GBD network.

[Comment] Cytokine-engineered CAR T-cell therapy in relapsed or refractory B-cell acute lymphoblastic leukaemia

Anti-CD19 CAR T-cell therapy has established a new standard of care for children and adults with relapsed or refractory B-cell acute lymphoblastic leukaemia (r/r B-ALL), culminating in the regulatory approval of three products associated with high rates of remission.1–3 However, the promise of currently available commercial CAR T-cell agents has been constrained by the loss of CAR persistence, resulting in suboptimal durability and high rates of CD19-positive relapse. Innovative approaches to circumvent this limitation include the development of armoured CAR T cells, in which the T cells are engineered to secrete immunomodulatory cytokines.

[Comment] Can ropeginterferon SURPASS resistance in essential thrombocythaemia?

Essential thrombocythaemia is a clonal myeloproliferative neoplasm associated with a lifelong risk of arterial and venous thrombosis, haemorrhage, and disease progression to secondary myelofibrosis and acute leukaemia.1 Current management strategies are primarily guided by the estimated risk of thrombotic events, presence of extreme leukocytosis or thrombocytosis, and disease-related symptoms. Hydroxyurea remains the most widely used first-line cytoreductive therapy for essential thrombocythaemia.

[Editorial] New hope, old challenges in myelodysplastic syndromes

Myelodysplastic syndromes are a complex and heterogenous group of clonal myeloid neoplasms, characterised by ineffective haematopoiesis, diverse cytopenias, and the risk of progression to acute myeloid leukaemia. Since being identified as a distinct disease in the late 1970s, the journey towards understanding, classifying, and treating myelodysplastic syndromes has been arduous, marked by major advances, yet still hampered by persistent challenges. Today patients with myelodysplastic syndromes continue to face low long-term survival, with a 5-year overall survival rate of approximately 37%, and no curative treatments outside haematopoietic stem-cell transplantation.

[Clinical Picture] Primary thyroid diffuse large B-cell lymphoma in a teenager

A 17-year-old boy with no personal or family history presented at St Louis Hospital in Paris in October, 2024 with a thyroid goitre that progressed over several weeks, rapidly causing dysphonia. A CT showed invasion of the thyroid gland by a lobulated structure infiltrating both lobes and the isthmus, measured at 75 × 46 mm (figure A). Biological investigations showed no abnormalities (normal complete blood count, and normal thyroid-stimulating hormone, thyroglobulin, and calcitonin levels). Thyroid fine needle aspiration disclosed the presence of large cells with dispersed chromatin (figure B).

[Review] Prevention and management of infectious diseases in pregnant women with haematological malignancies

The incidence of haematological malignancies during pregnancy ranges from 4·0 to 15·8 cases per 100 000 pregnancies, with Hodgkin lymphoma, acute leukaemia, and aggressive B-cell non-Hodgkin lymphoma being the most frequent subtypes. Although survival rates are similar to those in patients who are not pregnant with similar disease profiles, pregnant women face higher risks of maternal morbidity, along with adverse obstetric and neonatal outcomes. Their management, therefore, requires a carefully balanced approach that minimises obstetric risks and ensures effective oncological control.

[Series] Emerging therapeutic strategies for mature T-cell and natural killer-cell lymphomas

Mature T-cell and natural killer-cell neoplasms are a heterogenous group of uncommon lymphomas. Conventional therapy with mainly cytotoxic chemotherapy for this subgroup is suboptimal, and the treatment outcome is unsatisfactory. With the advances in the understanding of disease biology, considerable progress has been made in recent years. Monoclonal antibodies or antibody–drug conjugates targeting T-cell lymphoma surface antigens have been approved, including brentuximab vedotin, for the treatment of CD30-positive nodal and cutaneous T-cell lymphoma, and mogamulizumab, for mycosis fungoides and adult T-cell leukaemia/lymphoma.

[Articles] Incidence, mortality, and survival associated with acute leukaemia subtypes by age group in China: a population-based cancer registry analysis and cohort study

This comprehensive nationwide study of acute leukaemia incidence, mortality and survival outcomes across China establishes age-specific epidemiological benchmarks, enabling ongoing risk factor monitoring, while supporting expanded transplantation access for eligible patients and highlighting the urgent need for novel, less toxic therapies for older patients who bear a disproportionately higher disease burden.

[Articles] Indicators of inequity in research and funding for sickle cell disease, cystic fibrosis and haemophilia: a descriptive comparative study

The comparative evidence provided support the view that sickle cell disease is neglected compared with cystic fibrosis and haemophilia in relation to research and funding in the UK. The highest value for each indicator, often observed for cystic fibrosis, provides a benchmark target for the other two conditions. More dedicated research funding would likely have a ripple effect on other indicators, which could guide public health policies and have a major effect on the quality of life of people living with sickle cell disease.

[Articles] Rituximab, bendamustine, and cytarabine followed by venetoclax in older patients with high-risk mantle cell lymphoma (FIL_V-RBAC): a multicentre, single-arm, phase 2 study

To our knowledge, this is the first prospective study to stratify patients with mantle cell lymphoma to different treatments according to their risk profile. Our results suggest that the addition of fixed-duration venetoclax improves the performance of RBAC in patients with a high-risk disease profile. Our findings point to the importance of identifying patients with high-risk disease at initial diagnosis.