EJHaem最新文献

Introduction

Immune thrombocytopenia (ITP) and antiphospholipid syndrome (APS) are distinct autoimmune disorders with clinical intersections. While APS is marked by thrombosis and pregnancy complications, ITP typically presents as isolated low platelet counts with bleeding risk. Thrombocytopenia can also occur in APS, and a subset of ITP patients test positive for antiphospholipid antibodies (aPL).

Discussion

This study examined aPL in 90 ITP patients and compared them to 132 APS patients. Among the ITP group, 18.3% were aPL-positive, with lupus anticoagulant most common; 12% had anti-phosphatidylserine/prothrombin (aPS/PT) antibodies. In the APS cohort, 16% exhibited thrombocytopenia (< 100,000/µL), with a mean platelet count of 44,000/µL, and 71% of these were positive for aPS/PT. Platelet levels varied significantly among ITP aPL-negative, ITP aPL-positive and APS-ITP groups (p < 0.001). aPL positivity was linked to less severe thrombocytopenia but affected treatment approaches, showing differences in first-line (p = 0.034) and second-line (p = 0.025) therapies. Patients with ITP secondary to APS had a higher mean platelet count compared to aPL-positive ITP patients and aPL-negative ITP patients.

Conclusion

Screening for aPL and aPS/PT is vital to identify an ITP subset with milder thrombocytopenia and increased thrombotic risk, and may guide therapeutic decisions such as between thrombopoietin receptor agonists and SYK inhibitor.

Trial Registration

The authors have confirmed clinical trial registration is not needed for this submission.

Background

Hemoglobinopathies, including thalassemia and sickle cell disease (SCD), are increasingly prevalent in Spain due to migration and historical genetic patterns. Despite recent advances in screening and care, regional disparities in diagnosis and treatment persist.

Objectives

To critically assess the current landscape of hemoglobinopathies in Spain, focusing on screening program implementation, healthcare access, therapeutic innovations, and the integration of patient-centered outcomes.

Methods

This review synthesizes data from national registries, recent peer-reviewed publications, and health policy documents, offering a multidisciplinary analysis of clinical, epidemiological, and public health dimensions of hemoglobinopathies in Spain.

Results

Spain has progressively implemented neonatal screening programs for SCD and thalassemias, with substantial regional variation. Innovative treatments, including gene-editing approaches, are being piloted, yet accessibility remains limited by cost and infrastructure. Multinational collaborations and national registries have improved patient monitoring and evidence-based care, although healthcare inequalities persist.

Conclusions

Spain's efforts in diagnosis, treatment, and research of hemoglobinopathies have been significant, but further action is needed to ensure equitable healthcare access, ethical integration of gene therapies, and incorporation of patient-reported outcomes into clinical decision-making.

Trial Registration:

The author has confirmed clinical trial registration is not needed for this submission.

Background

Radiotherapy (RT) is a well-established treatment modality in multiple myeloma (MM), particularly for skeletal-related events. However, its role is likely evolving with the introduction of modern systemic treatments.

Methods

We conducted a retrospective analysis of a large, single-centre MM cohort treated with contemporary protocols to describe current patterns of RT use.

Results

RT continues to offer clinical benefit, with shifting patterns of use observed compared to historical cohorts.

Conclusion

RT remains relevant in the modern MM landscape and may hold increasing value in later treatment stages. We postulate a future role as bridging therapy prior to CAR-T/bispecific antibody treatment.

Trial Registration

The authors have confirmed clinical trial registration is not needed for this submission

Introduction

Veno-occlusive disease (VOD) is a life-threatening complication of haematopoietic stem cell transplantation (HSCT). The diagnosis remains challenging, with under recognition of the initial signs and symptoms potentially resulting in delayed diagnoses. The aim of this Delphi study is to establish a consensus regarding the optimal risk assessment and onward care of patients with VOD.

Methods

The process employed a modified Delphi methodology. A steering group of six VOD experts working in the United Kingdom attended a virtual meeting in September 2023, developed 44 statements for testing. Respondents were offered a four-point Likert scale to indicate their level of agreement with each statement.

Results

A total of 70 responses were received from healthcare providers working in the area of haematology and oncology in the United Kingdom. All statements achieved consensus. Overall, 82% of statements achieved ≥ 90% (n = 36/44), and 18% achieved ≥ 75% agreement (n = 8/44).

Conclusion

This modified Delphi process achieved consensus across all statements, allowing for a set of recommendations to be developed to support a consistent approach across the United Kingdom for the risk assessment and patient monitoring procedures for VOD post-HSCT.

Trial Registration: The authors have confirmed clinical trial registration is not needed for this submission.

Background

Chronic lymphocytic leukemia (CLL) treatment choice remains a challenge in the era of molecular biology and targeted therapy.

Methods

We conducted a bicentric retrospective analysis of the impact of NOTCH1 mutation according to the treatment of CLL patients in real life.

Results

A total of 45 patients with NOTCH1 mutation have been reported, including 15 patients treated with FCR BR or DRC regimen and 18 patients with a BTK inhibitor. NOTCH1 mutation is the most frequently occurring molecular abnormality in CLL and is closely associated with poor prognosis but is not used in treatment guidelines unlike TP53 and IGHV. In our study, progression-free survival was significantly longer in CLL patients with NOTCH1 mutation treated by BTK inhibitors compared to immunochemotherapy.

Conclusion

Routine screening for NOTCH1 mutations could identify patients who may benefit from BTKi treatment. However, the impact of NOTCH1 mutations on combination therapies, such as obinutuzumab-venetoclax or venetoclax-BTKi, is yet to be determined.

The authors have confirmed clinical trial registration is not needed for this submission

Background

Venous thromboembolism (VTE) is a serious complication in cancer patients, with malignancy increasing the risk significantly. Direct oral anticoagulants (DOACs) have emerged as a convenient alternative to traditional therapies, though optimal dosing remains uncertain.

Methods

We performed a systematic review and meta-analysis on three studies. A comprehensive literature search was performed on PubMed, Embase, the Cochrane Library, and ScienceDirect till April 2025. Analysis was carried out on RevMan 5.4. The risk of bias was assessed via RoB 2.0.

Results

A total of three studies with 2416 participants were identified, including 1495 patients in the reduced-dose group and 1232 patients in the full-dose group. No significant difference was observed in recurrent VTE (OR 0.70, 95% CI 0.45–1.09, p = 0.11) or recurrent symptomatic VTE (OR 0.96, 95% CI 0.50–1.84, p = 0.91). However, reduced-dose DOACs were associated with a significantly lower incidence of incidental VTE (OR 0.31, 95% CI 0.14–0.69, p = 0.004). The reduced-dose group also had a lower incidence of CRNMB plus major bleeding (OR 0.69, 95% CI 0.55–0.88, p = 0.002).

Conclusions

In terms of venous thromboembolism, bleeding events, and all-cause mortality, reduced-dose DOACs demonstrated a safety profile that was either superior or comparable to that of full-dose DOACs.

Trial Registration

The authors have confirmed clinical trial registration is not needed for this submission

Introduction

A novel therapy for heavily pretreated triple-class–exposed multiple myeloma (TCE MM) is B-cell maturation antigen (BCMA)-targeted immunotherapy. While the number of TCE+BCMA-exposed patients is growing, real-world data for this group are limited.

Methods

We present real-world data from patients with TCE+BCMA-exposed MM who initiated a subsequent line of therapy (LOT) using a US-based claims database, Komodo's Healthcare Map.

Results

We identified 656 TCE+BCMA-exposed patients; mean age was 66.5 years. Time from MM diagnosis to index was 5.4 years; mean number of prior LOTs was 5.9. The most prevalent prior therapy received within each drug class was daratumumab (98.5%), pomalidomide (86.0%), carfilzomib (85.8%) and belantamab mafodotin (74.5%). A total of 137 different subsequent treatment regimens were observed following TCE+BCMA exposure; the most common regimen was teclistamab (10.4%). The top three targeted agents within the subsequent regimen were carfilzomib (20.2%), pomalidomide (20.1%) and bortezomib (16.6%). Among this TCE+BCMA-exposed population who received subsequent treatment, the median time to next treatment or death was 6.8 (95% CI, 6.1–7.5) months; time to treatment discontinuation or death was 3.5 (95% CI, 3.2–3.7) months.

Conclusion

This first real-world analysis of patients with heavily pretreated TCE+BCMA-exposed MM shows poor clinical outcomes, frequent therapy retreatment and no standard-of-care, highlighting the need for novel treatments.

Clinical Trial Registration

The authors have confirmed clinical trial registration is not needed for this submission.