Journal of clinical haematology最新文献

Analysis of Updates in Multiple Myeloma Treatment and Management 多发性骨髓瘤治疗与管理的最新进展分析

Journal of clinical haematology Pub Date : 2023-10-04 DOI: 10.33696/haematology.4.055

Maria Jacqueline Nieto, Aryles Hedjar, Margaret Locke, Jessica Caro, Muhammad Wasif Saif

{"title":"Analysis of Updates in Multiple Myeloma Treatment and Management","authors":"Maria Jacqueline Nieto, Aryles Hedjar, Margaret Locke, Jessica Caro, Muhammad Wasif Saif","doi":"10.33696/haematology.4.055","DOIUrl":"https://doi.org/10.33696/haematology.4.055","url":null,"abstract":"Introduction: During the past two decades, new therapeutic agents have greatly improved the treatment landscape in multiple myeloma (MM). Treatments such as proteasome inhibitors, immunomodulatory agents, targeted monoclonal antibody therapy, and chimeric antigen receptor (CAR) T-cell therapy have improved outcomes with less toxicity. Advances in laboratory testing have accompanied this change, performing faster and more accurate assessments of treatment response. Despite these advances, however, disparities in MM outcomes persist. Objective: The purpose of this study was to review epidemiological trends in MM over the past two decades and to identify disparities that may impact MM identification and survival. Methods: Retrospective analysis was conducted on adult patients diagnosed with MM between the years 2000-2019 using the November 2021 Surveillance, Epidemiology, and End Results (SEER) program database. Joinpoint models were used to calculate annual percent changes (APCs) and average annual percent change (AAPC). Results: There were a total of 111,328 diagnoses of MM extracted from the SEER database. Most patients were male (55.17%) and white (76.7%). Age-adjusted rate analysis found a significantly higher incidence among black patients compared to white patients. The APC between 2000-2015 was 1.46, and the APC between 2015-2019 was -1.34. Relative survival also increased from 2000 to 2014. The 5-year cancer survival in MM also increased at an average of 1.8% for every year after diagnosis. The annual probability of MM-related mortality at the 1-year mark also decreased from 28.5% in 2000 to 16.7% in 2018. Conclusion: Novel advances in MM therapeutic agents and diagnostic testing have paved the way for significant improvements in patient survival outcomes. Disparities persist along racial lines. Further research is needed to evaluate responses to specific MM treatment in the age of newly developed targeted therapies to overcome these disparities.","PeriodicalId":87297,"journal":{"name":"Journal of clinical haematology","volume":"20 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-10-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135592159","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Molecular Features Associated with Response to Enasidenib Plus Azacitidine in Newly Diagnosed IDH2-Mutated Acute Myeloid Leukemia 新诊断的IDH2突变急性髓细胞白血病对依那西替尼加阿扎胞苷反应的分子特征

Journal of clinical haematology Pub Date : 2023-08-28 DOI: 10.33696/haematology.4.054

A. Risueño, W. See, T. Prebet, C. Dinardo, H. Döhner, E. Stein, A. Fathi, P. Vyas, L. Quek, A. Gandhi, Maroof Hasan

{"title":"Molecular Features Associated with Response to Enasidenib Plus Azacitidine in Newly Diagnosed IDH2-Mutated Acute Myeloid Leukemia","authors":"A. Risueño, W. See, T. Prebet, C. Dinardo, H. Döhner, E. Stein, A. Fathi, P. Vyas, L. Quek, A. Gandhi, Maroof Hasan","doi":"10.33696/haematology.4.054","DOIUrl":"https://doi.org/10.33696/haematology.4.054","url":null,"abstract":"IDH2 gene mutations, typically at residues R140 and R172, occur in 8–19% of patients with acute myeloid leukemia (AML). These mutations induce production of 2-hydroxyglutarate (2-HG), an oncometabolite that causes DNA and histone hypermethylation, and subsequent blockade of hematopoietic cell differentiation. In a phase 1b/2 trial (NCT02677922), combination therapy with azacitidine + enasidenib significantly improved overall response rate compared with azacitidine only therapy (74% vs 36%; P<0.001) in patients with newly diagnosed IDH2-mutated AML not eligible for intensive chemotherapy. We investigated the association between molecular features and clinical outcomes from that trial. In all, 101 patients were randomized to enasidenib + azacitidine (n=68) or azacitidine only (n=33); 74% of patients had IDH2-R140. Baseline 2-HG levels and IDH2 variant allele frequency (VAF) were similar between treatment arms and IDH2 variants and were not significantly different between clinical response categories. Significant 2-HG and IDH2 VAF reductions from baseline were observed with combination therapy compared with azacitidine only. Molecular profiling revealed SRSF2 preferentially co-mutated with IDH2-R140, and DNMT3A co-mutated with IDH2-R172. IDH2 VAF was reduced to <1% in 50% of patients who achieved CR with combination therapy (18/36) and azacitidine only (2/4). VAFs of genes in the DNA methylation, receptor-tyrosine-kinase, and RAS canonical pathways were reduced in patients achieving CR. Of note, combination therapy improved event-free survival in patients with RAS-pathway mutations, which have been associated with resistance to enasidenib monotherapy.","PeriodicalId":87297,"journal":{"name":"Journal of clinical haematology","volume":" ","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-08-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"42855899","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

A Rare Blood Malignancy in a Genetic Hematological Disorder: Polycythemia Vera (PV) in Sickle Cell Disease (SCD) 遗传性血液病中一种罕见的血液恶性肿瘤:镰状细胞病(SCD)中的真性红细胞增多症(PV)

Journal of clinical haematology Pub Date : 2023-08-12 DOI: 10.33696/haematology.4.053

C. L. Edwards, Sharena Scott, M. Boggan, J. Meek, W. J. Bryson, Alexandria McDougald, Milo Broadnax, C. Barker, Jessica Miller, J. Sollers, Tanisha I. Burford, J. Livingston, E. Whitworth, G. Byrd, Kelvin Williams, Sherry C. Eaton, Malik Muhammad, George Cliette, Dana Jones, Brianna Downey, Hilary T. Dietahin, Merrell Turner, Roland Thorpe, Keith Whitfield, Debra O. Parker, E. Robinson, M. Wood, Kenyon Railey, Shiv Sudhakar, Wandy Morel Cubilete, N. Shah

{"title":"A Rare Blood Malignancy in a Genetic Hematological Disorder: Polycythemia Vera (PV) in Sickle Cell Disease (SCD)","authors":"C. L. Edwards, Sharena Scott, M. Boggan, J. Meek, W. J. Bryson, Alexandria McDougald, Milo Broadnax, C. Barker, Jessica Miller, J. Sollers, Tanisha I. Burford, J. Livingston, E. Whitworth, G. Byrd, Kelvin Williams, Sherry C. Eaton, Malik Muhammad, George Cliette, Dana Jones, Brianna Downey, Hilary T. Dietahin, Merrell Turner, Roland Thorpe, Keith Whitfield, Debra O. Parker, E. Robinson, M. Wood, Kenyon Railey, Shiv Sudhakar, Wandy Morel Cubilete, N. Shah","doi":"10.33696/haematology.4.053","DOIUrl":"https://doi.org/10.33696/haematology.4.053","url":null,"abstract":"Objective: To delineate the etiology, symptomatology, and treatment of Polycythemia Vera in adults with Sickle Cell Disease. The current review contains a review of the 4 case reports that we found on the topic. To our knowledge, no other case reports exist. \u0000Methods: We reviewed the scientific literature to discover case reports that included the topic of PV. We noted consistencies in presentation, evaluation, treatment, and clinical outcomes.\u0000Results: We reviewed 4 case reports and a limited number of clinical papers on PV in SCD. We found and reported on consistencies in clinical presentation and the diversity of treatments. We reported hematological, bone marrow, and radiographic findings.\u0000Conclusions: There is great variability in the evaluation and treatment of cases of PV in SCD. We advocate for more research and deconstructing the complicated relationship between these two comorbid disorders.","PeriodicalId":87297,"journal":{"name":"Journal of clinical haematology","volume":" ","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-08-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"45559843","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Thrombotic Events and Risk Factors for Thrombosis in Polycythemia in Senegal, West Africa 西非塞内加尔红细胞增多症的血栓事件和血栓形成的危险因素

Journal of clinical haematology Pub Date : 2023-06-05 DOI: 10.33696/haematology.4.052

M. Seck, E. S. Bousso, S. A. Touré, A. Sall, Maryama Ndao, S. Guèye, B. Faye, A. Diallo, Mohamed Keita, A. Touré, S. Diop

{"title":"Thrombotic Events and Risk Factors for Thrombosis in Polycythemia in Senegal, West Africa","authors":"M. Seck, E. S. Bousso, S. A. Touré, A. Sall, Maryama Ndao, S. Guèye, B. Faye, A. Diallo, Mohamed Keita, A. Touré, S. Diop","doi":"10.33696/haematology.4.052","DOIUrl":"https://doi.org/10.33696/haematology.4.052","url":null,"abstract":"Objectives: We aim to identify thrombotic events and risk factors for thrombosis (RFT) comparing polycythemia vera (PV) and secondary polycythemia (SP) patients.\u0000\u0000Methods: We carried out a retrospective study of a cohort of 59 patients with PV (n=34) and SP (n=25) followed for a period of 14 years. Variables studied were the frequency and type of thrombosis, sociodemographic, clinical, and biological RFT. Statistical analysis was performed using SPSS software version 18. Multivariate analysis was performed to identify RFT.\u0000\u0000Results: Mean age in PV was 53 years (33 - 79) and 44.5 years (5 - 78) in SP (p=0.74). The sex ratio in PV was 1.12 and 2.57 in PS (p=0.52). Sixteen patients had thrombotic events (27.1%) including 12 PV (35.3%) versus 4 SP (16%) (p=0.09). Twenty-two thrombotic events were identified; 14 (63.7%) of arterial thrombosis and 8 (36.3%) of venous thrombosis. There were 18 thrombotic events (81.8%) in PV versus 4 (18.2%) in SP (p=0.02). Arterial thrombosis was more frequent in PV (55.6%). Thrombosis events were more frequent in female patients (59.1%). RFT in PV were age (40-59 years), hematocrit >45% and thrombocytosis ≥ 600 G/L and in SP, RFT were hematocrit >45% and hypercholesterolemia. After multivariate analysis, hematocrit >45% was the only RFT independently associated with thrombosis occurrence.\u0000\u0000Conclusion: We show through this study, a frequent occurrence of arterial thrombosis in polycythemia. Main RFT is the high hematocrit level (>45%). We insist on the interest of therapeutic bloodletting, sometimes associated with cytoreductive treatment in order to maintain a hematocrit level <45%.","PeriodicalId":87297,"journal":{"name":"Journal of clinical haematology","volume":"1 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-06-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41888619","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Targeting ANP32A Is a Novel Strategy Against Leukemia 靶向ANP32A是治疗白血病的新策略

Journal of clinical haematology Pub Date : 2022-12-13 DOI: 10.33696/haematology.3.047

引用次数: 1

Safety and Tolerability of Nilotinib in Patients with Chronic Myeloid Leukemia during Routine Clinical Practice: Results from the ERASER Study from Greece 尼洛替尼在慢性髓系白血病患者常规临床实践中的安全性和耐受性:来自希腊ERASER研究的结果

Journal of clinical haematology Pub Date : 2022-12-13 DOI: 10.33696/haematology.3.051

A. Symeonidis, A. Anagnostopoulos, M. Ximeri, G. Kaiafa, E. Kapsali, N. Viniou, T. Marinakis, D. Karakasis, V. Pappa, G. Vassilopoulos, D. Margaritis, Maria Tabitsika, M. Dimou

{"title":"Safety and Tolerability of Nilotinib in Patients with Chronic Myeloid Leukemia during Routine Clinical Practice: Results from the ERASER Study from Greece","authors":"A. Symeonidis, A. Anagnostopoulos, M. Ximeri, G. Kaiafa, E. Kapsali, N. Viniou, T. Marinakis, D. Karakasis, V. Pappa, G. Vassilopoulos, D. Margaritis, Maria Tabitsika, M. Dimou","doi":"10.33696/haematology.3.051","DOIUrl":"https://doi.org/10.33696/haematology.3.051","url":null,"abstract":"Regional real-world evidence on the safety and efficacy of tyrosine kinase inhibitors in patients with chronic myeloid leukemia (CML) is limited. This multicenter, observational, prospective study, ERASER, evaluated the safety and tolerability of nilotinib in routine clinical practice in Greece.\u0000\u0000Adult patients with newly diagnosed BCR/ABL+ chronic phase (CP) CML and those with CP CML, resistant/intolerant to prior therapy were included in this study and followed up for 36 months. Nilotinib 300 mg/400 mg twice daily was prescribed, with appropriate dose adjustment by the investigator.\u0000\u0000The analysis population (57 patients; median age, 55 years) remained in the study for a median of 34 months. Overall, 44 (77.2%) and 13 (22.8%) patients received nilotinib as first-line treatment and owing to resistance/intolerance to prior therapy, respectively. The most common adverse events (AEs) were thrombocytopenia in 8 (14%), neutropenia in 6 (10.5%), and blood bilirubin increased/hyperbilirubinemia in 10 (17.5%) patients. Permanent treatment discontinuation, including deaths and progression, occurred in 13 (22.8%) patients. Of 52 patients with available molecular response (MR), 30 achieved MR4.5 by end of the study.\u0000\u0000The study affirms the long-term safety of nilotinib in real-world setting in Greece, in patients with newly diagnosed CML, and in those with resistance/intolerance to prior therapy.","PeriodicalId":87297,"journal":{"name":"Journal of clinical haematology","volume":" ","pages":""},"PeriodicalIF":0.0,"publicationDate":"2022-12-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"42256951","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Making a Rationale Decision About -Uses of FFP: A Study at the Blood Bank of Tertiary Care Hospital of Southern Rajasthan 对FFP的使用做出合理决策：在拉贾斯坦邦南部三级护理医院血库进行的研究

Journal of clinical haematology Pub Date : 2022-12-13 DOI: 10.33696/haematology.3.046

引用次数: 0

Concomitant Langerhans Cell Histiocytosis and Chronic Myelomonocytic Leukaemia Responding to 5-azacitidine 5-氮杂胞苷引起的Langerhans细胞组织细胞增多症和慢性粒细胞白血病

Journal of clinical haematology Pub Date : 2022-12-13 DOI: 10.33696/haematology.3.050

引用次数: 0

B-cell Acute Lymphoblastic Leukemia with a Normal Platelet Count, Presenting as a Limp in a 2-year-old Child 2岁儿童b细胞急性淋巴细胞白血病伴正常血小板计数，表现为跛行

Journal of clinical haematology Pub Date : 2022-12-13 DOI: 10.33696/haematology.3.048

引用次数: 0

Multiple Myeloma with Neutrophilia: Two Etiologic Pathways for a Rare Presentation of a Common Diagnosis 多发性骨髓瘤伴中性粒细胞增多症：罕见常见诊断的两种病因途径

Journal of clinical haematology Pub Date : 2022-12-13 DOI: 10.33696/haematology.3.049

引用次数: 0