British Journal of Haematology最新文献

More than half of patients with relapsed/refractory (r/r) large B-cell lymphoma (LBCL) experience progression after chimeric antigen receptor (CAR) T-cell therapy, and subsequent treatment options remain limited with poor outcomes. Despite the need for effective therapies in this setting, post-CAR T clinical trial enrolment is low. We conducted a single-centre study of patients with r/r LBCL who progressed after CAR T-cell therapy between January 2018 and September 2023 to describe the practice patterns and identify factors associated with clinical trial participation. Patient, disease and clinical characteristics were analysed across screening, enrolment and treatment phases. Among 166 patients who progressed after CAR T-cell therapy, 39% were screened, 23% enrolled and 22% ultimately received trial treatment. High-risk clinical features, including eastern cooperative oncology group (ECOG) performance status ≥2, stage IV disease, high-risk International Prognostic Index, incomplete response to CAR T-cell therapy and severe cytokine release syndrome, were associated with non-participation. Using the eligibility criteria of pivotal trials that led to FDA approval of novel agents, only 14%-36% of patients who had relapsed disease after CAR T-cell therapy were eligible for these trials. The study highlights the unmet need to develop trials that accommodate high-risk populations to reduce barriers to trial participation following CAR T-cell therapy failure.

Stroke in sickle cell disease (SCD) has been well characterized in children, but data in adults remain insufficient, particularly regarding long-term functional consequences. The objective was to determine lifetime prevalence of symptomatic stroke, followed by characterization of stroke type, aetiology, treatments and functional status at last follow-up. We retrospectively reviewed adults (≥18 years) with any SCD phenotype followed at a tertiary centre from 2011 to 2023. Functional status was assessed using the Montreal Cognitive Assessment (MoCA) and Modified Rankin Scale (mRS). Among 454 adults with all major phenotypes of SCD, median age was 32 years [range 18-79] and 261 (57.5%) were women. At last follow-up, 21 individuals (4.6%) had a confirmed history of symptomatic stroke (median age at stroke onset of 42 years [range 4-68]), including 14 (3.1%) with cerebral infarction (median age 32 years [4-68]) and 7 (1.5%) with intracranial haemorrhage (median age 45 years [20-65]). Stroke was associated with marked long-term impairment, represented by lower MoCA (20.6 (±1.3) vs. 26.1 (±0.2), p < 0.001) and higher mRS (2 [1-3] vs. 0 [0-1], p < 0.001). These findings fill a critical evidence gap and underscore the urgency of targeted prevention and intervention strategies in this high-risk population.

Warm autoimmune haemolytic anaemia (wAIHA) is an acquired autoimmune disorder caused by autoantibodies-primarily immunoglobulin G (IgG)-that bind to red blood cells and trigger haemolysis. It can be primary or secondary to associated conditions. This nutshell review summarizes pathophysiology, diagnostic workup, prognosis and treatment options.

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.

Few studies have compared the prevalence of transfusion reactions (TRs) in paediatric and adult recipients, and none have been conducted in Canada. We carried out a retrospective cross-sectional study of TRs reported in a Canadian haemovigilance system (i.e. Québec's haemovigilance system [QHS]) from 1 January 2005 to 31 December 2022. QHS is a province-wide voluntary haemovigilance system that records TRs among blood recipients. Overall, 5 372 752 transfusions were recorded; 23 946 reactions deemed definitely, probably or possibly associated with transfusion were analysed (paediatric recipients = 1951 [8.2%]; adults = 21 995 [91.9%]). Paediatric recipients experienced higher rates (95% confidence interval) of reported TRs than adults (97.05 [92.79-101.45] vs. 42.53 [41.97-43.10] per 10 000 transfusions). This difference was predominantly driven by children (age: ≥12 months to <18 years), platelets, red blood cells and minor allergic reactions (all p < 0.0001). Similar results were obtained for nearly all individual TRs except transfusion-associated circulatory overload, whose rates were lower among paediatric recipients (p < 0.0001). However, paediatric age subgroups exhibited divergent patterns: recipients aged <1 month had lower rates than adults, while those aged ≥1 to <12 months and ≥12 months to <18 years had higher rates (all p < 0.0001). The aforementioned differences were consistently observed throughout the 18-year period, with only minor year-to-year variations in subgroups and for individual TRs. We conclude that paediatric recipients consistently exhibited higher rates of reported TRs than adult recipients.