British Journal of Haematology最新文献

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.

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.

Multirefractory immune thrombocytopenia (ITP) is difficult to manage. The simultaneous administration of an immunosuppressive/immunomodulatory drug (IS/IM) and a thrombopoietin receptor agonist (TPO-RA) is increasingly used. We present the experience of the Spanish ITP Group in this retrospective, observational study. Ninety-seven patients with ≥3 failed treatment lines were administered combination therapy (CT) with an IS/IM and a TPO-RA between January 2021 and December 2023. The IS/IMs were steroids, purine synthesis inhibitors and fostamatinib. The TPO-RAs were romiplostim, eltrombopag and avatrombopag. Seventy-five (77.3%) patients responded to treatment in a median (interquartile range [IQR]) time of 14 (7-52) days. An inverse correlation between previous failures and response was observed (Rho = -0.290, p = 0.009). After 840 (448-1054) days, relapse was reported in 35 (46.7%) of 75 responders (time to relapse: 197 [47-402] days). Relapse was more frequent in chronic ITP patients. Twenty-one patients received a second CT after failure of the first one. Eleven were administered a third CT for the same reason. Response and relapse were similar to those observed with the first CT. Three thromboembolic events and 14 infections that required hospitalization were reported, none fatal. The combination of IS/IMs and TPO-RAs arises as an alternative option of treatment in multirefractory ITP.

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.