British Journal of Haematology最新文献

Waldenström's macroglobulinaemia (WM) is a rare B-cell lymphoproliferative disorder with multiple effective front-line treatment options. However, real-world comparative data on commonly used regimens are limited. We conducted a retrospective cohort study of 348 consecutive, newly diagnosed WM patients treated between 2002 and 2024. Patients received first-line therapy with either bortezomib-dexamethasone-rituximab (BDR, n = 35), Bruton's tyrosine kinase inhibitors (BTKis, n = 57) or dexamethasone-rituximab-cyclophosphamide (DRC, n = 256). BTKi demonstrated the highest MRR (major response rate, ≥PR) (80.7%), followed by DRC (68.4%) and BDR (40.0%) (p < 0.001). The median PFS and OS did not differ significantly among regimens. TTNT seemed to be longer in the BTKi group (log-rank p = 0.025), with a 74% reduced risk of salvage therapy compared to DRC (aHR = 0.26, p = 0.016). Cumulative WM-related mortality at 5 years was lowest in BTKi-treated patients (4.1% vs. 13.0% DRC vs. 17.1% BDR), though differences were not statistically significant. In this single-centre analysis, both BTKi and DRC led to prolonged disease control in the upfront treatment of patients with WM. Extended follow-up and prospective validation are needed to reveal potential long-term survival differences.

BRUIN CLL-321 is the first prospective, randomized study conducted in covalent BTK inhibitor (cBTKi) pretreated chronic lymphocytic leukaemia/small lymphocytic lymphoma (CLL/SLL) patients. In this heavily pretreated population, pirtobrutinib significantly improved progression-free survival (PFS) compared to investigator's choice (IC) of idelalisib/rituximab (IdelaR) or bendamustine/rituximab (BendaR). This report presents results from Chinese patients enrolled in BRUIN CLL-321, who were randomized 1:1 to pirtobrutinib (200 mg once daily) or IC of BendaR (idelalisib is not approved in China). End-points included independent review committee (IRC)-assessed PFS, investigator (INV)-assessed PFS, overall survival (OS), event-free survival (EFS), time to next treatment (TTNT) and safety. Among 40 Chinese patients (pirtobrutinib n = 19; BendaR n = 21), IRC-assessed PFS favoured pirtobrutinib (stratified hazard ratio [HR] = 0.281; 95% confidence interval [CI], 0.070-1.125, nominal p = 0.0554), with median PFS not reached versus 10.6 months with BendaR; INV-assessed PFS supported these findings. TTNT (HR = 0.150; 95% CI, 0.031-0.728) and EFS (HR = 0.322; 95% CI, 0.094-1.101) were also improved. A trend towards OS benefit was observed (HR = 0.343; 95% CI, 0.031-3.787). Pirtobrutinib showed a favourable safety profile, with fewer grade ≥3 treatment-emergent adverse events (36.8% vs. 93.3%) and serious adverse events (15.8% vs. 46.7%). These findings support pirtobrutinib as a clinically active and tolerable option for cBTKi-pretreated Chinese CLL/SLL population.

Commentary on: Schmidt-Barbo et al. High pre-treatment peripheral blood T cell receptor (TCR) clonality as a predictor of prolonged response in immune thrombocytopenia to the British Journal of Haematology. Br J Haematol 2026 (Online ahead of print). doi: 10.1111/bjh.70310 and Jauch et al. Efficacy and safety of eltrombopag in combination with dexamethasone as first-line treatment in adult patients with newly diagnosed immune thrombocytopenia for the British Journal of Haematology. Br J Haematol 2026 (Online ahead of print). doi: 10.1111/bjh.70312.

The prognosis of peripheral T-cell non-Hodgkin lymphomas (PTCL) is dismal, particularly in the relapsed/refractory (r/r) setting, where 3-year overall survival (OS) is 20%-30%. No superior second-line therapy for PTCL has been universally established, and durable remissions rely on consolidation with allogeneic haematopoietic stem cell transplantation (alloHSCT). Enhancing response rates to salvage therapy is, therefore, crucial to increase transplant eligibility. We retrospectively evaluated the efficacy of bendamustine, gemcitabine and vinorelbine combination (BeGeV) in 24 consecutive patients with r/r PTCL treated at our centre since 2017. BeGeV achieved an overall response rate (ORR) of 66% and a complete remission rate (CRR) of 41%. After a median follow-up of 41.8 months, 1-year progression-free survival (PFS) and OS were 37.5% and 58.3% respectively. Outcomes differed by histology: PTCL not otherwise specified (PTCL-NOS) showed inferior responses (ORR 41%, CRR 16%) compared with T-follicular helper lymphomas (PTCL-TFH; ORR 100%, CRR 75%) and systemic anaplastic large cell lymphoma (sALCL; ORR 75%, CRR 50%). Survival analyses confirmed substantial differences across subtypes, with 12-month PFS and OS rates of 8.3% and 41.7% for PTCL-NOS, 50% and 75% for sALCL and 75% and 75% for PTCL-TFH respectively. Despite the limitations of small sample size and retrospective design, this study provides preliminary evidence supporting BeGeV as a potential bridge to alloHSCT in r/r PTCL-TFH and sALCL.

Haematology is at a crossroads, divided between haemato-oncology and the disparate disciplines collectively known as 'non-malignant' haematology. This latter term is a misnomer that devalues a spectrum of complex, life-threatening conditions and contributes to workforce shortages and research inequities. This article argues for the formal adoption of the term Medical Haematology to redefine this domain. We chart its central role across medicine, from guiding anticoagulation, transfusion and thrombosis care across specialties to addressing global health challenges. We highlight its pioneering contributions to molecular medicine and immunotherapy, exemplified by gene therapy for haemophilia and the repurposing of chimeric antigen receptor T cells for autoimmune disease. Finally, we present a forward-looking blueprint involving establishment of 'Blood Teams', revamping educational curricula and championing equity to secure the speciality's future. Embracing Medical Haematology is a strategic imperative to reflect the life-threatening nature of many conditions within the speciality, attract trainees, rebalance research priorities and firmly re-establish haematology's indispensable role at the heart of modern medical practice.

Despite red blood cell (RBC) immunization being a frequent complication of chronic transfusion in myelodysplastic syndromes (MDS), its prognostic significance remains unclear. We analysed 486 transfused patients diagnosed with MDS. Prognostic impact of RBC immunization (allo- or autoantibodies) was evaluated as a time-dependent covariate. Competing risk methods were applied to estimate the cumulative incidence of immunization. Sixty-nine patients (14.2%) developed RBC immunization, most commonly anti-K and anti-E, which was more frequent in patients transfused before MDS diagnosis (subhazard ratio [SHR]: 2.9, 95% confidence interval [CI]: 1.6-5.4; p = 0.001) and Rh(D)-negative blood group (SHR: 1.9, 95% CI: 1.1-3.2; p = 0.026). RBC immunization was associated with a significant and independent reduction in remaining survival (hazard ratio: 11.9, 95% CI: 7.3-19.6; p = 0.001), without differences between auto- and alloantibodies. RBC immunization was followed by increased transfusion intensity, but transfusion requirements also rose in non-immunized patients over time. RBC immunization did not predict progression to acute myeloid leukaemia (AML). A trend towards fewer new antibodies was observed during hypomethylating therapy. RBC immunization is independently associated with reduced survival in transfusion-dependent patients with MDS, irrespective of AML progression. These findings highlight the potential prognostic relevance of RBC antibodies and call for further investigation into the mechanisms linking immunization, transfusion burden and survival outcomes.