Acta Haematologica最新文献

{"title":"Characterization of Ferroptosis-Related Genes in Aplastic Anaemia: An Integrated Analysis of Bulk and Single-Cell RNA Sequencing Data.","authors":"Chuyun Shen, Fengming Wang","doi":"10.1159/000543656","DOIUrl":"10.1159/000543656","url":null,"abstract":"<p><strong>Introduction: </strong>Ferroptosis offers novel perspectives for treating multiple blood-related diseases, yet its role in aplastic anaemia (AA) is rare. This study aimed to explore key ferroptosis-related genes (FRGs) in AA using bulk and single-cell RNA sequencing (scRNA-seq) data.</p><p><strong>Methods: </strong>scRNA-seq and bulk RNA-seq data, along with FRG lists, were obtained from public databases. Differentially expressed FRGs (DEFRGs) between AA and control samples were identified, followed by functional enrichment and protein-protein interaction analyses. Single-cell analyses were conducted to reveal main cell types in samples and DEFRGs activity in each cell was assessed. Moreover, DEGs between AA and control samples at the cellular level were explored, followed by integration with DEFRGs to determine common key genes. KEGG pathway analysis of these genes was performed at the cellular level. Immune infiltration analysis was used to evaluate the relationship between key genes and immune cells.</p><p><strong>Results: </strong>A total of 38 DEFRGs were identified, enriched in pathways such as the intrinsic apoptotic signalling pathway. scRNA-seq analysis identified seven cell types, with elevated DEFRGs activity in platelets and stromal cells. Key genes DDIT4 and NCF2, identified through integrated analysis, were involved in autophagy, mTOR signalling, and osteoclast differentiation pathways. Moreover, their expressions were positively correlated with activated dendritic cells in AA samples.</p><p><strong>Conclusion: </strong>Our findings highlight the roles of DDIT4 and NCF2, in AA progression, providing potential insights for further mechanistic exploration of AA.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-12"},"PeriodicalIF":1.7,"publicationDate":"2025-02-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143405126","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Inflammatory Factors and Immune Cells in Relation to Multiple Myeloma.","authors":"Meng-Jun Huang, Qing-Yi Zeng, Dan Chen, Chun-Xia Yang, Ying Yang, Man Zhou, Fen-Li Zhang, Qiu-Han Bian, Xiao-Yan Yang","doi":"10.1159/000543429","DOIUrl":"10.1159/000543429","url":null,"abstract":"<p><strong>Introduction: </strong>Many reports indicate that the occurrence of multiple myeloma (MM) is closely related to inflammation and immunity. Although the survival rates have been gradually improving in recent years, the cure rate is still not optimistic enough. Therefore, it is necessary to continue exploring the causes of MM.</p><p><strong>Methods: </strong>This study utilizes Mendelian randomization (MR) analysis to establish the connection between inflammatory factors, immune cells, and the occurrence of MM.</p><p><strong>Results: </strong>In MR studies, a significant correlation was observed between interleukin-1 receptor antagonist (IL-1Ra), tumor necrosis factor receptor 1 (TNFR1), memory B-cell percentage of B cells (memory B-cell %B cells), and immunoglobulin D-positive, CD24-negative percentage B cells (IgD+ CD24- %B cells) with the onset of MM. In particular, IgD+ CD24- %B cells showed a statistically significant inverse relationship with the development of MM (p < 0.05, OR <1), whereas IL-1Ra, TNFR1, and memory B-cell %B cells displayed a positive association with the onset of MM (p < 0.05, OR >1). These findings contribute valuable insights to the understanding of the pathogenesis of MM.</p><p><strong>Conclusion: </strong>This study emphasizes the significant role of inflammatory factors and immune cells in multiple myeloma (MM) progression. IL-1Ra, TNFR1, and memory B-cell percentages are identified as risk factors, while IgD+ CD24- %B cells may protect against progression, suggesting new immunomodulatory treatment strategies. However, research on IgD+ CD24- %B cells and MM is limited, necessitating future studies to clarify their mechanisms and effects on the tumor microenvironment. There is also an urgent need for clinical trials to assess therapies targeting these cells, as well as long-term follow-ups to understand their dynamic changes in relation to disease progression. Further investigation using animal models is warranted to validate their functional role in MM development.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-9"},"PeriodicalIF":1.7,"publicationDate":"2025-02-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143397586","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Prognostic Factors, FLT-3 Mutations, and Treatment Outcomes with Pediatric-Inspired Protocols in Adolescent and Young Adults and Adult Patients with Acute Lymphoblastic Leukemia.","authors":"Uriel Oanunu, Noa Gross Even-Zohar, Shlomzion Aumann, Vladimir Vainstein, Alexander Gural, Moshe E Gatt, Arnon Haran, Boaz Nachmias","doi":"10.1159/000543861","DOIUrl":"10.1159/000543861","url":null,"abstract":"<p><strong>Introduction: </strong>The treatment protocols of adolescent and young adult (AYA) patients with acute lymphoblastic leukemia (ALL) have evolved, with the advent of pediatric-based regimens, measurable residual disease monitoring, and mutation analysis. Among the latter, previous reports have identified FLT-3 mutations in up to 5% of pediatric patients; however, the full clinical significance of these mutations in the non-pediatric population is still uncertain.</p><p><strong>Methods: </strong>Our cohort includes AYA patients with ALL treated with the NY-II and BFM protocols at different time periods, allowing analysis of prognostic factors and survival outcomes. Additionally, we analyzed DNA samples for FLT-3 mutations, focusing on the potential prognostic implications and treatment responses within our cohort.</p><p><strong>Results: </strong>No significant differences were found in overall survival or progression-free survival between the two treatment protocols. However, a higher rate of hematopoietic stem-cell transplantation was noted in the NY-II patients. Older age and high WBC count at presentation were identified as adverse prognostic factors using multivariate analysis. FLT-3 mutations were identified in 4 patients (5%) of the cohort, with only 1 patient having FLT-3 internal tandem duplication mutation and 3 patients having FLT-3-tyrosine kinase domain mutations.</p><p><strong>Conclusions: </strong>The low rate and variability of FLT-3 mutations in an Israeli cohort precludes broad conclusions regarding their prognostic significance. In our cohort, age and WBC count but not treatment protocol or FLT-3 mutations influenced survival.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-11"},"PeriodicalIF":1.7,"publicationDate":"2025-01-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143063090","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"DRESS Is Not a Rare Complication during the Initial Treatment of Newly Diagnosed Multiple Myeloma: The Experience of Two Medical Institutes.","authors":"Jiao Yang, Qingqing Hu, Jiaqing Lu, Jian Wang, Die Wu, Xiaoming Fei, Lixia Wang, Xianqiu Yu, Yu Tang","doi":"10.1159/000543779","DOIUrl":"10.1159/000543779","url":null,"abstract":"<p><strong>Introduction: </strong>Drug reaction with eosinophilia and systemic symptoms (DRESS) is a severe hypersensitivity reaction rarely documented in patients with multiple myeloma (MM).</p><p><strong>Methods: </strong>In our retrospective study of 108 newly diagnosed MM (NDMM) patients from January 2021 to October 2023, we identified 4 cases of DRESS. The clinical characteristics such as clinical manifestations, laboratory results, treatment, and outcome were analyzed.</p><p><strong>Results: </strong>These patients presented with fever, persistent and recurrent, along with a widespread red rash characterized by diffuse erythematous macules or papules accompanied by intense itching, desquamation, and other dermatological manifestations. Multiorgan involvement was common, including hepatic impairment, acute kidney injury, and type I respiratory failure, alongside pleural effusion and multiple lymphadenopathy. Laboratory findings revealed elevated eosinophil counts, often exceeding 1.5 × 109/L, abnormal liver function tests, acute kidney injury, and inflammatory markers. Anti-MM treatment was promptly suspended, and all patients received corticosteroid therapy. Outcomes varied, with 1 patient succumbing to myeloma progression, another to multiorgan failure, while the remaining 2 patients survived.</p><p><strong>Conclusion: </strong>In NDMM patients undergoing induction therapy, occurrences of DRESS are infrequent but noteworthy, with an incidence higher than observed in the general population. It presents with significant morbidity and mortality, highlighting the crucial need for early recognition and management.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-9"},"PeriodicalIF":1.7,"publicationDate":"2025-01-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143050982","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Prognostic Survival Model Based on Endocrine-Related Gene Expression in Acute Myelogenous Leukemia.","authors":"Weiran Lv, Yun Wang, Fang Hu, Hanying Huang, Yingying Cui, Yuanbin Song, Lezong Chen, Bingyi Wu, Yang Liang","doi":"10.1159/000543272","DOIUrl":"10.1159/000543272","url":null,"abstract":"<p><strong>Introduction: </strong>Accurate prediction of survival in patients with acute myelogenous leukemia (AML) is challenging. Therefore, we developed a predictive survival model using endocrine-related gene expression to identify an endocrine signature for accurate stratification of AML prognosis.</p><p><strong>Methods: </strong>RNA matrices and clinical data for AML were downloaded from a training dataset (Gene Expression Omnibus) and two validation datasets (the Cancer Genome Atlas and Therapeutically Applicable Research to Generate Effective Treatments).</p><p><strong>Results: </strong>In relation to the survival outcome, a risk model was constructed by incorporating seven endocrine-related genes. The model exhibited favorable predictive efficacy in estimating 5-year survival rates, as demonstrated by both the training and validation cohorts. Multivariable analysis revealed that the endocrine signature demonstrated autonomous prognostic significance in the aforementioned cohorts. Prediction accuracy for 5-year overall survival increased using a nomogram combining endocrine risk score and classical prognostic factors compared with using classical prognostic factors alone. The model predictions were confirmed using AML cell lines.</p><p><strong>Conclusion: </strong>The endocrine-related prognostic model established in this study improves AML survival prediction accuracy.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-15"},"PeriodicalIF":1.7,"publicationDate":"2025-01-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142982325","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-World Experience in the Management of Chronic Myeloid Leukemia Patients Focused on Tyrosine Kinase Inhibitors Intolerance and Health-Related Quality of Life.","authors":"Hee Jeong Cho, Dong Won Baek, Juhyung Kim, Young Eun Jang, Yunji Lee, Joon Ho Moon, Sang Kyun Sohn","doi":"10.1159/000542562","DOIUrl":"10.1159/000542562","url":null,"abstract":"<p><strong>Introduction: </strong>This study aimed to analyze the survival outcomes and adverse events (AEs) associated with the long-term use of tyrosine kinase inhibitors (TKIs) and to assess health-related quality of life (HRQoL) in patients with chronic myeloid leukemia (CML).</p><p><strong>Methods: </strong>Medical records of 345 patients with CML treated with at least one type of TKI were retrospectively reviewed.</p><p><strong>Results: </strong>No significant differences in survival were observed based on the number of different TKIs the patients received (p = 0.301) or the sequence of TKIs used (p = 0.770). Among 182 patients treated with nilotinib, 25 experienced cardiovascular events (CVEs). After 10 years of nilotinib treatment, CVEs occurred in 55.2% of patients with ≥2 vascular risk factors. Pleural effusion was observed in 27 of 78 dasatinib-treated patients. In terms of HRQoL, patients treated with nilotinib generally reported higher satisfaction levels than did those treated with imatinib or dasatinib. When stratified by age or duration of TKI treatment, patients aged <60 years or those with a treatment duration of ≥1 year exhibited better satisfaction levels.</p><p><strong>Conclusion: </strong>Survival outcomes were not affected by history of TKI treatment. Nilotinib is favorable for HRQoL but increases the risk of serious CVEs in patients with vascular risk factors.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-14"},"PeriodicalIF":1.7,"publicationDate":"2025-01-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142969164","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Direct STAT3 and STAT5 Inhibition Overcomes Treatment Resistance in a Murine-Derived in vitro Model of Acute Lymphoblastic Leukaemia Driven by ETV6::JAK2.","authors":"Jane Frances Thompson, Randall Grose, David Yeung, Deborah L White","doi":"10.1159/000543428","DOIUrl":"https://doi.org/10.1159/000543428","url":null,"abstract":"<p><strong>Introduction: </strong>ETV6::JAK2 is a fusion known to drive acute lymphoblastic leukaemia (ALL) in the presence of other genomic lesions which define the JAK/STAT class of Philadelphia chromosome-like acute lymphoblastic leukaemia (Ph-like ALL). Ph-like ALL comprises approximately 15% of ALL. Patients with mutations or gene fusions signalling through the JAK/STAT pathway have particularly poor prognosis. Emerging treatments targeting JAK2 fusions and mutations are promising, and phase 3 clinical trials are in progress. However, with widespread use of JAK2 inhibitors, it is important to anticipate and manage resistance mechanisms. The JAK2 p.G993A mutation confers resistance in vitro, even to high-dose JAK2 inhibitors such as ruxolitinib. We postulated that direct inhibition of STAT3 and STAT5, downstream from JAK2, may overcome resistance.</p><p><strong>Methods: </strong>Murine-derived IL-3-dependent Ba/F3 cells were transfected with ETV6::JAK2 containing a p.G993A mutation for this study. These cells were confirmed to demonstrate IL-3 independence and ruxolitinib resistance prior to use in experiments. An inhibitor-response assay was conducted using differing concentrations of SH-4-54 and pimozide (STAT3/5 inhibitors) applied to ETV6::JAK2 p.G993A cells and two control cell lines.</p><p><strong>Result: </strong>SH-4-54 and pimozide were effective against ETV6::JAK2 p.G993A cells with median lethal doses (LD50) of 296 n<sc>M</sc> for SH-4-54 and 455 n<sc>M</sc> for pimozide. Both drugs demonstrated a lesser effect on empty vector Ba/F3 cells, with an LD50 of 371 n<sc>M</sc> for SH-4-54 and 596 n<sc>M</sc> for pimozide. Neither drug demonstrated significant effect on non-JAK/STAT-activated KG-1a myeloid cells at doses near the LD50.</p><p><strong>Conclusion: </strong>SH-4-54 and pimozide both overcame treatment resistance in our in vitro model of JAK/STAT-driven Ph-like ALL with a mutation conferring JAK2 inhibitor resistance. While SH-4-54 demonstrates greater potency than pimozide, pimozide may be a more promising option given its demonstrated safety profile in humans. Direct STAT3 and STAT5 inhibition may be an effective approach for overcoming inevitable JAK2 inhibitor resistance-conferring mutations in patients with the poor prognostic subtype of JAK/STAT class Ph-like ALL.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-5"},"PeriodicalIF":1.7,"publicationDate":"2025-01-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143381415","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Initial Characterization and Outcome Assessment of Anal Lymphomas in a Large-Size Contemporary Cohort: A Population-Based SEER Database Study (2000-2022).","authors":"Pierre Loap, Youlia Kirova","doi":"10.1159/000541595","DOIUrl":"10.1159/000541595","url":null,"abstract":"<p><strong>Introduction: </strong>Anal lymphoma (AL) is a rare presentation of extranodal lymphomas, characterized by occurrence in the anal area and largely understudied due to its infrequency. This study aimed to address gaps in knowledge about AL's demographic and clinical profiles, treatments, and survival outcomes, leveraging data from the SEER program.</p><p><strong>Methods: </strong>We conducted a retrospective analysis of 79 AL cases identified in the SEER database from 2000 to 2022; 36 stage I AL cases were identified and defined as localized primary anal lymphoma (L-PAL). Data on demographics, tumor specifics, treatment modalities, and survival were analyzed using the Kaplan-Meier method and Cox proportional hazards models.</p><p><strong>Results: </strong>The majority of AL cases were diffuse large B-cell lymphoma (70.9%). Other notable subtypes included anaplastic T-cell lymphoma, marginal zone lymphoma, B-cell non-Hodgkin lymphoma, Burkitt lymphoma/leukemia (each accounting for 6.3%), followed by follicular lymphoma and mantle-cell lymphoma (each at 1.3%). AL primarily affected younger males (median age 50), with a significant majority being Caucasian. Initial stages (I and II) were more commonly observed, and treatments varied, with chemotherapy being most prevalent (67.1%), followed by radiation (30.4%) and surgery (30.4%). The 5- and 10-year overall survival (OS) rates were 59.4% and 44.1%, respectively, while the corresponding cancer-specific survival (CSS) rates were 67.9% and 58.0%, respectively. Age was a significant prognostic factor for OS but not for CSS. Radiotherapy tended to improve CSS in the AL population.</p><p><strong>Conclusion: </strong>This research corresponds to the first in-depth analysis of AL, highlighting its distinct demographic patterns, clinical features, and responses to various treatments, distinguishing it from other types of anal cancers. Our results underscore the importance of developing specialized diagnostic and treatment strategies. To enhance our understanding and management of this uncommon form of lymphoma, future studies should aim for broader and more collaborative international research efforts.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-7"},"PeriodicalIF":1.7,"publicationDate":"2025-01-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142930391","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Role of Autologous Transplant in Newly Diagnosed Multiple Myeloma Patients Treated with Novel Triplets: A Systematic Review and Meta-Analysis.","authors":"Irina Amitai, Ronit Gurion, Pia Raanani, Iuliana Vaxman, Moshe Yeshurun, Hila Magen, Anat Gafter-Gvili, Liat Shargian","doi":"10.1159/000540232","DOIUrl":"10.1159/000540232","url":null,"abstract":"<p><strong>Introduction: </strong>High-dose therapy with melphalan followed by autologous stem cell transplant in the upfront setting (upfront ASCT) has significantly improved clinical outcomes of myeloma patients and become the standard of care for the past 30 years. However, with the advent of modern induction therapy, the role of upfront ASCT approach has been called into question. Several prospective studies have examined whether continuing with triplet therapy as consolidation with optional ASCT at relapse (triplet-alone) could result in comparable outcomes.</p><p><strong>Methods: </strong>This was a systematic review and meta-analysis of randomized controlled trials comparing upfront ASCT versus triplet-alone approach among myeloma patients treated with triplet therapy, which included two novel agents and a corticosteroid, as induction. Cochrane Library, PubMed and conference proceedings were searched. Primary outcome was overall survival (OS). Secondary outcomes included progression-free survival (PFS), safety, and second primary malignancies (SPM). Subgroup analysis was conducted for high-risk cytogenetics.</p><p><strong>Results: </strong>Our search yielded three trials, conducted between 2010-2018, including 1,737 patients. Two trials evaluated bortezomib plus lenalidomide (VRd) induction and the third study tested carfilzomib plus lenalidomide (KRd) induction. Maintenance was given in all trials to both arms. There was no difference in OS between the arms; the pooled OS in all patients and those with high-risk cytogenetics was hazard ratio (HR) 1.03 (95% CI, 0.85-1.26; I2 = 0%; 1,737 patients, 3 trials) and 0.85 (95% CI, 0.59-1.23; I2 = 0%; 222 patients, 2 trials), respectively. The pooled PFS for upfront ASCT versus triplet-alone was significantly improved in all the patients and in the high-risk cytogenetics subgroup, HR 0.67 (95% CI 0.59-0.76; I2 = 0%; 1,737 patients, 3 trials) and HR 0.59 (95% CI: 0.44-0.7; I2 = 0%; 306 patients, 3 trials), respectively. The risk of any grade 3-4 adverse events was higher in the upfront ASCT arm versus triplet-alone approach (relative risk = 1.17 [95% CI, 1.12-1.23; 1,737 patients]). The risk of secondary malignancies was reported in all three trials and was comparable between both arms. Two trials reported on secondary myeloid neoplasms, which were significantly higher among upfront ASCT arm versus triplet-alone approach, OR 9.7 (1.8-52.25, I2 = 0%, 1,422 patients).</p><p><strong>Conclusion: </strong>Although upfront ASCT approach, in the era of triplet therapy, resulted in a significantly longer PFS among all patients, this did not translate into a survival benefit, regardless of cytogenetic risk. Upfront ASCT was associated with an increased rate of secondary myeloid neoplasms. In the current plethora of innovative therapies, the role of upfront ASCT is debatable.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"468-476"},"PeriodicalIF":1.1,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142278646","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Evaluating the Economic Burden of Acute Myeloid Leukemia in Canada.","authors":"Jean Lachaine, Catherine Beauchemin, Fatéma Dodat, Yunghan Au, William K Evans, Brian Leber, Kristjan Paulson, Andre Schuh, John Storring","doi":"10.1159/000537725","DOIUrl":"10.1159/000537725","url":null,"abstract":"<p><strong>Introduction: </strong>Acute myeloid leukemia (AML) represents a significant burden for patients and their families, and to the healthcare system. This study estimated the total cost of illness associated with newly diagnosed AML patients in Canada.</p><p><strong>Methods: </strong>The economic burden of AML was estimated using an incidence-based model, analyzing different types of AML cases in Canada. Direct and indirect costs were calculated using scientific literature and Canadian clinical experts' inputs. Patients were categorized depending on their eligibility for intensive chemotherapy (fit and unfit patients) as well as according to age and cytogenetic markers.</p><p><strong>Results: </strong>The total average cost of AML per patient is estimated to be CAD 178,073 with a cost of CAD 210,983 and CAD 145,163 for fit and unfit patients, respectively. The costs related to treatment represent half of the total average cost (52%), followed by hematopoietic stem cell transplant (23%), best supportive care (16%), productivity loss (6%), and wastage (4%).</p><p><strong>Conclusion: </strong>For patients with AML, the costs associated with fit patients are higher than unfit patients. Hospitalization and best supportive care costs are key cost drivers for the total costs of fit and unfit patients, respectively. This study highlights that AML is associated with a significant economic burden in Canada.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"8-21"},"PeriodicalIF":1.7,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139929471","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}