Hematology最新文献

Background: Proline-rich transmembrane protein 4 (PRRT4) has been infrequently studied, with limited literature suggesting its potential as a prognostic marker for gastric cancer. This study aims to investigate the prognostic value of the PRRT4 gene in pan-cancer.

Methods: We acquired and analyzed data from several platforms, including The Cancer Genome Atlas (TCGA), Genotype Tissue Expression Project (GTEx), Cancer Cell Line Encyclopedia (CCLE), cBioPortal, HPA, and TIMER 2.0. In addition, we have further analyzed the data using multivariate analyzes and RT-qPCR. In vitro experiments were performed to detect the proliferation and apoptosis of AML cells before and after PRRT4 knockdown.

Results: PRRT4 exhibited low expression in 10 types of cancers and high expression in 3 types, and this expression was significantly correlated with tumor stage, age, and gender across various cancer types. PRRT4, identified as a potential independent prognostic factor for overall survival (OS) in several cancers including LAML, PAAD, SKCM, STAD, THYM, and UVM, and exhibited a high frequency of mutation in UCEC. Moreover, PRRT4 was found to be correlated with DNA methylation and immune infiltration in various cancers. Ultimately, in the multivariate analysis model, PRRT4 was discerned as an independent prognostic biomarker for AML, predicated on the statistics based from our institution. After PRRT4 knockdown, the proliferation ability of THP1 cells was significantly enhanced, and the apoptosis ratio was significantly decreased.

Conclusion: PRRT4 may serve as a potential therapeutic target and prognostic marker for various malignancies.

Objectives: Whether intermediate-dose tertiary prophylaxis can improve quality of life and psychological health in adults with severe/moderate hemophilia A has not been determined. This research aims to explore the impact of intermediate-dose tertiary prophylaxis with recombinant human FVIII (rhFVIII) on quality of life, anxiety and depression in such individuals transitioned from on-demand treatment.

Methods: This retrospective analysis collected data from July 2019 to July 2022. Haemophilia Quality of Life Questionnaire for Adults (HAEMO-QoL-A), Patient Health Questionnaire-9 (PHQ-9), and Generalized Anxiety Disorder-7 (GAD-7) were compared before and after prophylaxis. Additionally, functional independence and joint status were analyzed at the end of the 3-year prophylaxis period, as well as their correlations with HAEMO-QoL-A, PHQ-9, and GAD-7.

Results: The HAEMO-QoL-A total score decreased after prophylaxis (pre-prophylaxis: 88.90 ± 33.38 vs. post-prophylaxis: 79.59 ± 22.89) (P = 0.016). The mean PHQ-9 scores before and after prophylaxis were 4.44 ± 4.63 and 5.56 ± 4.30 (P = 0.058), respectively, while the mean GAD-7 scores were 3.15 ± 3.84 and 4.44 ± 3.99 (P = 0.016). Significant correlations were observed for HAEMO-QoL-A with functional independence (P < 0.001), mood and emotions with age (P = 0.032), PHQ-9 scores with knee joint rehabilitation scores (P = 0.047), and GAD-7 scores with treatment experience and ankle joint rehabilitation scores (P = 0.029, P = 0.039).

Conclusion: Intermediate-dose tertiary prophylaxis with rhFVIII can improve quality of life but not relieve anxiety and depression in adults with severe/moderate hemophilia A. Better functional independence correlates with improved quality of life. Gait and age also influence the quality of life to some extent. We need to undertake anxiety and depression screening and provide psychological treatment when necessary.

Objectives: Immune thrombocytopaenia (ITP) is a rare autoimmune disorder characterized by low platelet count and increased risk of bleeding. This study aimed to be the first publication to characterize the economic burden of bleeding events in patients with ITP in the UK.

Methods: We performed a microcosting analysis to estimate the costs associated with bleeding events in patients with ITP. Healthcare resources utilized in the management of bleeds of different severity were costed using well-established UK cost sources. The results were validated through semi-structured interviews with clinical experts.

Results: The severity of bleeding events was classified into four categories, ranging from bleeding managed at home, through mild bleeding managed in the outpatient or day case setting, to serious and life-threatening bleeding events requiring inpatient admission. Total medical costs per event ranged from £2,930 for managing a mild bleeding event, through £16,711 for a serious bleeding event to £32,461 for a life-threatening event. The major cost driver for mild and serious events were intravenous immunoglobulin (IVIg) costs, amounting to £1,614 and £8,071 for the two severity categories, respectively. For life-threatening events, the costs of intensive care unit stay (£9,089) exceeded those of IVIg (£8,071).

Conclusion: Real-world costs of managing bleeding in patients with ITP in the UK are substantial and greater than costs set only based on the UK NHS Tariff. Mitigating the risk of bleeding in patients with ITP is likely to yield not only clinical advantages for patients but also offer substantial cost savings to the health system.

Objectives: To summarize available data and contribute to a broader understanding of the global incidence and prevalence of acquired factor X deficiency.

Methods: A comprehensive review of English-language publications from PubMed and Embase was conducted. The majority of publications on acquired factor X deficiency were associated with light-chain (AL) amyloidosis. Therefore, this review is structured to assess publications reporting on (1) acquired factor X deficiency associated with AL amyloidosis or (2) acquired factor X deficiency associated with other causes.

Results: The literature includes case reports, case-series, and limited population-based reports of the epidemiology of acquired factor X deficiency. Though no definitive global incidence or prevalence estimates for AL-amyloidosis-associated acquired factor X deficiency were identified, the finding that roughly 6-14% of patients with AL amyloidosis have factor X activity levels below 45-50% of normal highlights the rarity of acquired factor X deficiency associated with AL-amyloidosis. Indeed, AL amyloidosis itself is a rare disorder with an estimated annual incidence of ∼10 cases per million population. Only case reports were available to inform the epidemiology of acquired factor X deficiency not associated with AL amyloidosis. We identified 35 cases from 29 papers published from around the globe. At least 25 of those patients experienced a bleeding event, with factor X activity levels ranging from <1% to 39%.

Conclusion: More population-based data are needed to understand the epidemiology of acquired factor X deficiency; however, the limited data seem to indicate this condition is quite rare. The variation across papers in thresholds used to define deficiency highlights the need for a standardized definition to better inform drug development, resource allocation, and regulatory decision-making.

Background: Childhood leukaemia remains a major global health challenge and its impact varies significantly by region. Understanding the patterns of incidence, mortality, prevalence, and disability-adjusted life years (DALYs) is crucial for crafting effective public health initiatives and enhancing care outcomes, especially in regions with constrained resources.

Methods: This study evaluates the worldwide, regional, and country-specific effects of childhood leukemia between 1990 and 2021, leveraging data from the Global Burden of Disease (GBD) initiative. It delves into trends related to illness, death rates, prevalence, and DALYs across various income brackets and geographic areas, offering a comprehensive analysis of the disease's impact over three decades.

Results: Childhood leukemia rates have declined globally over the last 30 years, especially in high-income countries due to medical advancements. However, low- and middle-income nations face persistent challenges, as limited healthcare infrastructure, delayed diagnosis, and treatment accessibility contribute to a disproportionate burden. Across all regions, male children consistently bear a higher burden compared to females. Forecasts indicate the worldwide burden will decrease with enhanced treatment, yet disparities between affluent and poorer regions will persist.

Conclusions: While global trends indicate a reduction in the childhood leukemia burden, regional disparities remain. To further reduce the global burden, we must strengthen health systems, improve early detection and ensure access to treatment. To promote more equitable outcomes across regions, public health policies must focus on these areas.

Objective: This systematic review aims to evaluate the extensive application of platelet-rich plasma (PRP) as a novel autologous cell therapy across various clinical disciplines in recent years.

Method: A comprehensive search was conducted across PubMed, Scopus, Embase, and the Cochrane Library from 2009 to 2024, resulting in the identification of 153 research articles. The inclusion criteria were focused on studies that reported on the molecular mechanisms of action and clinical efficacy. The data were synthesized through qualitative narrative analysis and thematic classification.

Result: Key active components found in PRP, including platelet-derived growth factor, transforming growth factor-β, and vascular endothelial growth factor, exhibit regulatory effects on fibroblasts, endothelial cells, and immune cells. These components facilitate cell proliferation and migration via signaling pathways such as PI3 K/Akt and MAPK/ERK. Notably, differences exist between the use of PRP alone versus its application in combination therapies for conditions such as bone and ligament repair, female infertility, alopecia, facial rejuvenation, diabetic foot ulcers, and fistulas. Factors such as PRP storage methods, white blood cell concentration levels, platelet concentration variations, and whether it is activated ex vivo can significantly influence therapeutic outcomes.

Conclusion: PRP represents a highly promising autologous treatment modality. Standardizing protocols for PRP preparation alongside disease-specific treatment strategies will enhance precision in clinical applications.

Purpose: The aim of this study was to assess the predictive role of FOXO1 expression changes in determining the response to hypomethylation therapy in patients with high-risk myelodysplastic syndrome (MDS).

Methods: FOXO1 mRNA levels were measured using real-time PCR in 62 newly diagnosed MDS patients undergoing decitabine treatment. The study analyzed the relationship between FOXO1 expression and clinical indicators, treatment outcomes, and prognosis.

Results: Responders exhibited significantly elevated FOXO1 levels, which were associated with improved peripheral blood counts, decreased bone marrow blasts, and enhanced T-cell immunity and polarization. Increased FOXO1 expression after four cycles of decitabine was associated with a more favorable treatment response. Univariate and multivariate Cox analyses revealed that elevated FOXO1 expression was linked to prolonged overall survival and leukemia-free survival.

Conclusions: Elevated FOXO1 expression in high-risk MDS patients undergoing decitabine treatment enhances patient prognosis and survival.

Background: Primary immune thrombocytopenia (ITP) is an autoimmune hemorrhagic disorder characterized by low platelet counts. Currently, the epidemiology of ITP in China remains poorly understood.

Methods: A cross-sectional study was conducted to analyze data from newly diagnosed ITP patients in Yunnan Province from 2022 to 2023 to assess the epidemiological characteristics of ITP. Enzyme linked immunosorbent assay (ELISA) were conducted to detect plasma cytokine levels in ITP patients and healthy controls from two different altitude regions.

Results: This study reports for the first time an average incidence rate of 6.75 per 100,000 population for ITP in Yunnan Province, China. There was a significant difference in incidence rates between males and females, at 5.9 and 7.59, respectively. Infants under the age of 4 were identified as having the highest risk of ITP. After age 70, the incidence in males began to exceed that in females. This is different from previous reports in Japan, Europe and the United States. Additionally, there was no significant difference in the incidence of ITP based on altitude; however, mid-latitude regions had a higher concentration of ITP patients. Cytokine analysis was performed in 88 individuals, revealing significantly elevated Hypoxia-inducible factor alpha (HIF-1α) and Interleukin (IL-4) levels in ITP patients from high-altitude regions.

Conclusion: This study offers the first insights into the epidemiological characteristics of ITP in Yunnan Province and underscores the impact of altitude on cytokine variations in ITP patients. These findings offer valuable guidance for personalized treatment strategies and the formulation of public health policies for ITP.

Background: CD19-targeted chimeric antigen receptor T-cell (CAR-T) therapy has transformed treatment for relapsed/refractory large B-cell lymphoma, offering promising remission rates. However, it carries significant infectious risks, necessitating revaccination for infection prevention.

Methods: This single-center quality improvement study aimed to (1) assess revaccination uptake and barriers in CAR-T recipients through interviews and focus groups, and (2) use qualitative insights to develop an educational handout to improve revaccination adherence. The study was conducted at Juravinski Hospital and Cancer Centre, Hamilton, Ontario, and enrolled 22 patients who received CAR-T between January 2020 and June 2023. Participants completed a survey evaluating their revaccination experience, which informed the development of a patient-facing educational handout. A focus group of survey participants then reviewed the handout and offered feedback on its clarity and usefulness.

Results: Only 50% of participants proceeded with revaccination. Key barriers included limited awareness among primary care providers (PCPs), poor communication between hematologists and PCPs, and logistical difficulties. The focus group highlighted gaps in understanding post-CAR-T immunization needs and emphasized the importance of patient and physician education. Participants supported the creation of a concise handout outlining the revaccination schedule and clarified the PCP's role in coordination.

Conclusion: This study underscores the need for improved communication across care providers and accessible patient education to support post-CAR-T revaccination. Future efforts should focus on implementing and evaluating targeted educational tools to enhance vaccine uptake in this high-risk population..

Background: Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an effective, and in many cases, the only treatment for curing malignant blood diseases. However, post-transplant relapse is the primary factor affecting survival rates.

Methods: We conducted a retrospective analysis of 123 patients with acute leukemia undergoing allo-HSCT at Jiangsu Provincial People's Hospital from August 2017 to June 2023 to discuss the risk factors for relapse in acute leukemia (AL) patients after allo-HSCT.

Results: Among the 123 patients, 41experienced relapse, with 30 cases of relapse within one year. Multivariable analysis showed that pre-transplant MRD (+) and NR (HR 2.32, 95%CI 1.18-4.54, P = 0.014), post-transplant MRD (+) (HR 2.13, 95%CI 1.04-4.36, P = 0.016), cGVHD (-) (HR 0.48, 95%CI 0.23-0.97, P = 0.041) and age < 40 (HR 0.44, 95%CI 0.23-0.83, P = 0.011) were independent risk factors for relapse after allo-HSCT.

Conclusions: Data from our center indicated that pre-transplant MRD (+) and NR, post-transplant MRD (+), cGVHD (-) and age < 40 are independent risk factors affecting relapse after allo-HSCT.