British Journal of Haematology最新文献

Teenage and young adult (TYA) patients undergoing allogeneic stem cell transplant have distinct psychosocial needs, yet they are poorly represented in research and their outcomes are not well understood. This study uses prospectively collected data from the British Society of Blood and Marrow Transplantation and Cellular Therapy (BSBMTCT) registry to explore UK transplant practice and outcomes for TYA patients (aged 16-24) in this healthcare setting, alongside children (aged 1-15) and adults (aged 25-39), transplanted for acute leukaemia (including lymphoblastic, acute lymphoblastic leukaemia [ALL], and myeloid, acute myeloid leukaemia [AML]). Nine hundred and forty TYA patients, transplanted between 1999 and 2018, are included, representing 87% of all UK activity during the study period. On adjusted analyses, overall survival after transplant for ALL worsened from children, through TYA, to adults; survival for patients with AML was similar across age groups. Non-relapse mortality was not significantly worse in TYA patients compared with children (p = 0.117 in ALL, p = 0.379 in AML). The risk of chronic graft-versus-host disease (GvHD) was strongly correlated with age, with rates in the TYA group much closer to those seen in adults. While a graft-versus-leukaemia effect may be suppressing relapse, the high rate of GvHD represents an unmet need in this group, who are at a crucial juncture in their personal, educational and social development.

Family history of haematological malignancy (FHHM) reflects inherited susceptibility to non-Hodgkin lymphoma (NHL), but its prognostic significance remains poorly understood. We used a cohort study of 1994 NHL patients to evaluate associations between FHHM and clinical outcomes across lymphoma subtypes. We estimated associations, adjusted for sex and lymphoma-specific prognostic index, of self-reported FHHM in a first-degree relative with overall survival (OS), event-free survival (EFS) and lymphoma-specific survival (LSS) using multivariable Cox regression models and with failure to achieve EFS at 24 months (EFS24) using logistic regression. FHHM prevalence was 12.8%, and the median follow-up among survivors was 12.0 years. FHHM was not associated with OS. In subtype-specific analyses, FHHM was associated with inferior LSS (hazard ratio [HR] = 1.98, 95% confidence interval [CI] 1.11-3.53) in follicular lymphoma (FL); this association and the association with EFS24 failure strengthened among those treated with front-line immunochemotherapy (EFS24 failure odds ratio = 2.57, 95% CI 1.08-5.99; LSS HR = 2.68, 95% CI 1.09-6.58). In mantle cell lymphoma, FHHM was associated with inferior EFS (HR = 1.70, 95% CI 1.02-2.85). Interaction testing did not demonstrate significant heterogeneity of FHHM effects by lymphoma subtype. FHHM may be associated with adverse outcomes in select NHL groups, particularly FL. These findings warrant further investigation of germline and environmental factors potentially contributing to more aggressive FLs.

Despite several studies reporting the effectiveness of dapsone in immune thrombocytopenic purpura (ITP), information on its mechanism of action, dose-response relationship and response predictors remains limited. To address this knowledge gap, we analysed 433 healthcare visit data of 58 ITP patients (29 males and 29 females; 39 adults and 19 paediatric) treated with dapsone (mean: 1.19 mg/kg/day) after 0-4 prior treatments. Among them, 14 had newly diagnosed, 23 had persistent and 21 had chronic ITP. The median ages for paediatric and adult patients were 6.4 and 40.9 years respectively. Median response time, response duration and follow-up period were 40, 151 and 142 days respectively. The overall response rate was 58.6%, of which 94.1% of patients responded within 6 months and 44.8% (n = 26) maintained a response at the last follow-up. Mediation analysis showed that the association between dapsone and responses was mediated via haemoglobin reduction. Response rates were 5.38 times higher in patients who experienced a reduction in haemoglobin than in those who did not experience a reduction in haemoglobin (p = 0.008). Age, sex, previous treatment numbers and response, ITP types, baseline platelets and concurrent treatments were not associated with a response. Two patients needed dapsone discontinuation due to adverse events.