British Journal of Haematology最新文献

In adults with classic Hodgkin lymphoma (cHL), metabolic tumour volume (MTV) is valuable for risk stratification. This systematic review explored the role of volumetric parameters in children, adolescents and young adults (CAYA) with cHL. This systematic review was performed according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) method. Five databases were searched on 16 October 2024. Eligible studies were peer-reviewed manuscripts, written in English, published since database inception that included patients ≤21 years of age with a diagnosis of cHL treated with chemotherapy ± radiotherapy with a baseline ¹⁸F-Fluorodeoxyglucose (F-FDG) PET/CT scan for MTV quantification. The search strategy identified 3669 studies, of which 35 were eligible for inclusion. Fifteen studies (43%) evaluated a cohort exclusively ≤21 years of age. All eight studies that explored the association of baseline PET parameters with known risk factors identified a significant correlation. Nine of 12 papers (75%) demonstrated a statistically significant correlation with disease response to therapy on PET/CT. Fifteen of 20 papers (75%) demonstrated an independent statistically significant correlation with clinical outcomes. In the CAYAHL population, volumetric PET parameters are valuable for risk stratification despite heterogeneity in results. Future harmonization of the segmentation approach will facilitate incorporation of MTV into clinical practice.

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.

Hepatitis-associated aplastic anaemia is a rare entity that can sometimes be life-threatening due to its rapid progression to liver failure, necessitating an urgent liver transplantation (LT). Treating severe aplastic anaemia following an LT can be very challenging. While immunosuppressive therapy (IST) has been reported to have some success, the vulnerable state of these patients in addition to the time taken for IST to work makes this a less suitable option for the majority of patients. Haematopoietic stem cell transplantation (HSCT) with matched related donors has been reported as a curative option; there has been less success using alternate donors. Here, we present our experience of predominantly alternate donor HSCT following liver transplantation in an extremely high-risk group of 10 children. Overall survival was 90% at a median of 38 months post-LT. Surviving children have normal blood counts, normal liver function and performance status. No liver-related complications or significant graft versus host disease were encountered. Multiple infective and non-infective post-HSCT complications were successfully treated with excellent multidisciplinary input. Upfront HSCT, even with alternate donors, can be lifesaving if performed in a timely manner, following close liaison between liver transplantation and HSCT teams and with the appropriate multidisciplinary support.

Identifying chronic myeloid leukaemia (CML) patients at risk of therapeutic switch remains debated. We analysed the cumulative risk of treatment change in the CML Italian network prospective cohort based on first-line tyrosine kinase inhibitors and patient characteristics. Sub-hazard ratios (sHRs) were estimated using Fine and Gray multivariable models. Among 1662 patients, initial treatment consisted of imatinib for 840 (50.5%), nilotinib for 490 (29.5%) and dasatinib for 332 (20.0%). Subsequently, 492 patients (29.6%) required second-line therapy, with 232 (47.1%) switching due to resistance and 176 (35.8%) due to intolerance. At 2 years, the risk of resistance was 18.3% for imatinib, 8.4% for dasatinib (sHR = 0.32; 95% confidence interval 0.21-0.49) and 6.8% for nilotinib (0.29; 0.19-0.42). The risk of switching increased in intermediate (1.95; 1.40-2.72) and high Eutos long term survival (ELTS) risk (3.19; 2.10-4.83) but was reduced with older age (0.97 per year; p < 0.0001). Intolerance at 2 years was 8.5% for imatinib, 12.4% for dasatinib (2.55; 1.73-3.75) and 5.2% for nilotinib (1.04; 0.65-1.65). Switching to a third-line therapy at 3 years was 8% for imatinib, 5% for dasatinib and 4% for nilotinib. The results showed that the time to first treatment switch for resistance is shorter for younger patients, for imatinib and for intermediate/high ELTS risks. The risk of switching for intolerance is higher for patients initially treated with dasatinib.

Sickle cell disease (SCD) and its treatments may lead to gonadal dysfunction. Limited research has examined how these effects translate into actual fertility outcomes and family-building perspectives. We aimed to determine the frequency of infertility and to examine family-building goals, knowledge and concerns among 91 adults with SCD from The Ohio State University Wexner Medical Center SCD clinic. Participants completed surveys capturing their demographic and medical information. Fertility status and family-building perspectives were measured using modified versions of surveys used in fertility-related literature in other populations. Descriptive statistics summarized demographics, frequency of infertility and family-building perspectives. Most participants expressed a desire for children. Approximately half met the clinical definition of infertility at some point, but only a few who met this definition had knowledge of it. Lastly, most reported low to moderate fertility and reproductive concerns potentially due to lack of awareness about their infertility status. These findings underscore the need to increase infertility education and counselling for individuals with SCD.

Excisional lymph node biopsy remains the gold standard for lymphoma diagnosis, yet the optimal timing is often overlooked. In this study, two cases illustrating delays caused by reliance on imaging and fine-needle aspiration, emphasizing histopathology and clinical suspicion for timely diagnosis, were reported.

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.