Expert Review of Hematology最新文献_第2页

Evaluating quality-of-life outcomes during decitabine therapy in older adults with acute myeloid leukemia: overview of reported studies and assessment instruments. 评估老年急性髓性白血病患者地西他滨治疗期间的生活质量：已报道的研究和评估工具概述

IF 2.1 4区医学

Expert Review of Hematology Pub Date : 2025-10-04 DOI: 10.1080/17474086.2025.2570333

Pasquale Niscola

引用次数: 0

Somatic variants and frequencies of familial myeloma germline predisposition genes among patients within the CoMMpass dataset. compass数据集中患者家族性骨髓瘤种系易感性基因的体细胞变异和频率。

IF 2.1 4区医学

Expert Review of Hematology Pub Date : 2025-10-03 DOI: 10.1080/17474086.2025.2567299

Erman Akkus, Timur Tuncalı, Hasan Yalim Akin, Meral Beksaç

{"title":"Somatic variants and frequencies of familial myeloma germline predisposition genes among patients within the CoMMpass dataset.","authors":"Erman Akkus, Timur Tuncalı, Hasan Yalim Akin, Meral Beksaç","doi":"10.1080/17474086.2025.2567299","DOIUrl":"10.1080/17474086.2025.2567299","url":null,"abstract":"Background: Genetic factors associated with familial multiple myeloma (MM) have been studied, yet the somatic engagement of germline predisposition genes remains underexplored. This study aims to systematically analyze somatic variants in previously reported germline familial myeloma predisposition genes.Research design and methods: A systematic literature search identified 179 genes associated with familial MM. Somatic variants and associated demographic data from the Multiple Myeloma Research Foundation (MMRF) CoMMpass Study, which includes non-selected myeloma patients (5.3% with a first-degree family history of hematological malignancy) were analyzed.Results: 1863 somatic variants across the 179 predisposition genes were detected, with substitutions being the most common variant type (95.7%) and missense variants the most frequent consequence (40.6%). Notably mutated genes with pathogenic potential included DIS3, LRP1B, EP300, SAMHD1, ARID1A, DNAH2, MUC17, BIRC6, MYH14, DSP, and DCHS1. Pathogenic variants did not show significant demographic associations. Moreover, variant types, consequences, and associated demographics revealed similar rates in young myeloma patients (≤50 years) and patients with a first-degree family history of hematological malignancy.Conclusions: This study highlights a significant rate of pathogenic somatic variants in germline predisposition genes of familial myeloma, suggesting candidate genes to be investigated in myelomagenesis.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-10"},"PeriodicalIF":2.1,"publicationDate":"2025-10-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145148530","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}

引用次数: 0

Safety and efficacy of non-first-line drugs in the treatment of immune thrombocytopenia: a systematic review and network meta-analysis. 非一线药物治疗免疫性血小板减少症的安全性和有效性：系统评价和网络荟萃分析。

IF 2.1 4区医学

Expert Review of Hematology Pub Date : 2025-10-03 DOI: 10.1080/17474086.2025.2568131

Xin Zhou, Mengran Li, Xiaohui Sui, Ai Li, Xiaoyan Li, Ningning Shan

{"title":"Safety and efficacy of non-first-line drugs in the treatment of immune thrombocytopenia: a systematic review and network meta-analysis.","authors":"Xin Zhou, Mengran Li, Xiaohui Sui, Ai Li, Xiaoyan Li, Ningning Shan","doi":"10.1080/17474086.2025.2568131","DOIUrl":"10.1080/17474086.2025.2568131","url":null,"abstract":"Background: This systematic review and network meta-analysis(NMA) comprehensively evaluated the efficacy and safety of non-first-line drugs in the treatment of adult patients with immune thrombocytopenia (ITP).Researchdesign and methods: PubMed, Web of Science, Embase, and the Cochrane Library were systematically searched. Randomized controlled trials (RCTs) investigating Count data were extracted in the form of event occurrences/non-occurrences. NMA was carried out via R.Results: 29 RCTs were encompassed. In contrast to placebo, the avatrombopag 20 mg group demonstrated the highest PR (RR = 12.23, 95% CrI: 5.48-33.72). The combination of eltrombopag and danazol exhibited the lowest incidence of bleeding events (RR = 0.31, 95% CrI: 0.16-0.57), while the avatrombopag 5 mg group had the lowest incidence of SAEs (RR = 0.44, 95% CrI: 0.22-0.84). The comprehensive evaluation suggested that romiplostim, initiated at a dose of 1 μg/kg within a dose-adjustment regimen, may confer one of the most favorable benefit - risk profiles, with a relatively high PR (SUCRA = 75.1%) and a low incidence of bleeding events (SUCRA = 54.8%).Conclusions: When initiating therapy with romiplostim at a dose of 1 μg/kg and subsequently titrating the dosage according to the patient's platelet response, romiplostim may represent one of the most effective therapeutic options.Registration: The study protocol for this systematic review was registered in the International Prospective Registry of Systematic Reviews (PROSPERO) database (http://www.crd.york.ac.uk/prospero/), and it was allocated the PROSPERO identification number.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-13"},"PeriodicalIF":2.1,"publicationDate":"2025-10-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145185140","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}

引用次数: 0

Current experiences with teclistamab in patients with multiple myeloma and renal impairment. 目前在多发性骨髓瘤和肾损害患者中使用teclistamab的经验。

IF 2.1 4区医学

Expert Review of Hematology Pub Date : 2025-10-02 DOI: 10.1080/17474086.2025.2567296

Meletios A Dimopoulos, Yael C Cohen, Aurore Perrot, Matthew J Pianko, Edward A Faber, Nelson Leung, María-Victoria Mateos, Ajay K Nooka

{"title":"Current experiences with teclistamab in patients with multiple myeloma and renal impairment.","authors":"Meletios A Dimopoulos, Yael C Cohen, Aurore Perrot, Matthew J Pianko, Edward A Faber, Nelson Leung, María-Victoria Mateos, Ajay K Nooka","doi":"10.1080/17474086.2025.2567296","DOIUrl":"10.1080/17474086.2025.2567296","url":null,"abstract":"Introduction: Renal impairment (RI), defined as a creatinine clearance of < 40 mL/min, affects up to half of patients with multiple myeloma (MM). Patients with MM and RI historically had poorer outcomes, likely due to the limited access to novel treatments available through clinical trials. Strict eligibility criteria for MM clinical trials often exclude patients with RI, necessitating reliance on patient data acquired from real-world (RW) clinical practice to guide therapeutic decisions. Therefore, there is a need for RW data and expert recommendations to guide treatment strategies for patients with MM and RI.Areas covered: Teclistamab treatment and pharmacokinetics in patients with mild-to-moderate RI, including the first report of a RI patient subgroup from the MajesTEC-1 study, as well as published RW experiences of teclistamab, primarily in patients with moderate-to-severe RI.Expert opinion: Current guidelines, available data, and our clinical experience broadly support the feasibility and potential benefit of teclistamab for patients with MM and RI, including those on dialysis, providing appropriate precautions are taken. This expert opinion offers recommendations for optimizing the management of patients with MM and RI treated with teclistamab. Additional RW data will further inform the safety and efficacy profile of teclistamab in this patient population.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-8"},"PeriodicalIF":2.1,"publicationDate":"2025-10-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145191519","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}

引用次数: 0

Transforming hemophilia care: emerging therapeutic innovations and challenges. 转变血友病护理：新兴治疗创新和挑战。

IF 2.1 4区医学

Expert Review of Hematology Pub Date : 2025-10-01 Epub Date: 2025-06-30 DOI: 10.1080/17474086.2025.2527344

Kun Huang

{"title":"Transforming hemophilia care: emerging therapeutic innovations and challenges.","authors":"Kun Huang","doi":"10.1080/17474086.2025.2527344","DOIUrl":"10.1080/17474086.2025.2527344","url":null,"abstract":"Introduction: Hemophilia A and B are lifelong bleeding disorders traditionally managed with factor replacement therapy. While effective, this approach is limited by frequent infusions, inhibitor development, and high treatment costs, underscoring the need for innovative therapies that improve patient outcomes and quality of life.Areas covered: This review explores recent advances in hemophilia management, focusing on extended half-life factor concentrates, non-factor therapies, such as bispecific antibodies and rebalancing agents, and the emergence of gene therapy as a potential functional cure. English-language studies on hemophilia therapies were searched in PubMed, Embase, Web of Science, and ClinicalTrials.gov (Jan 2014 to Apr 2025). A comprehensive literature review was conducted, covering pivotal clinical trials, real-world data, and translational research addressing both efficacy and safety considerations.Expert opinion: Innovative therapies are transforming hemophilia care, offering simplified administration, superior bleed prevention, and new hope for patients with inhibitors. Gene therapies mark a milestone, but present challenges related to durability, immune response, and cost. Personalized treatment strategies, integrating patient-specific factors and shared decision-making, are essential to optimizing outcomes. Continued research, long-term surveillance, and efforts to improve global access will define the next era of hemophilia management, moving closer to a future where patients can lead lives free from the burden of bleeding.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"785-802"},"PeriodicalIF":2.1,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144527121","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}

引用次数: 0

t(4;14), and revised myeloma comorbidity index as good predictors of survival for multiple myeloma. T（4;14）和修订后的骨髓瘤合并症指数作为多发性骨髓瘤生存的良好预测指标。

IF 2.1 4区医学

Expert Review of Hematology Pub Date : 2025-10-01 Epub Date: 2025-07-14 DOI: 10.1080/17474086.2025.2534706

Aline G Ramirez-Alvarado, Susana Gabriela Gonzalez-Prieto, Yael Solis-Aranda, Ana Paulina Rivera-Espinoza, Jaime Garcia-Chavez, Laura Arcelia Montiel-Cervantes, Jorge Vela-Ojeda

{"title":"t(4;14), and revised myeloma comorbidity index as good predictors of survival for multiple myeloma.","authors":"Aline G Ramirez-Alvarado, Susana Gabriela Gonzalez-Prieto, Yael Solis-Aranda, Ana Paulina Rivera-Espinoza, Jaime Garcia-Chavez, Laura Arcelia Montiel-Cervantes, Jorge Vela-Ojeda","doi":"10.1080/17474086.2025.2534706","DOIUrl":"10.1080/17474086.2025.2534706","url":null,"abstract":"Background: Cytogenetic tests are essential prognostic indicators for patients diagnosed with multiple myeloma (MM). The study aimed to evaluate the prevalence of cytogenetic abnormalities and their prognostic significance in patients with newly diagnosed MM.Research design and methods: A cohort study involving 88 cases. Malignant plasma cells were isolated, and interphase fluorescent in situ hybridization was performed on bone marrow samples.Results: The gain of 1q was observed in 17 (19%) patients, followed by t(4;14) in 10 (11.5%), and the 17p or P53 mutation was found in only 6 (7%) patients. Three patients (3.5%) exhibited t(14;16). Amplification of 1q was not detected in any of the samples. The presence of t(4;14), anemia, renal disease, a revised myeloma comorbidity index (R-MCI) of 7-9, and a lack of treatment response were associated with poor progression-free survival. Additionally, t(4;14), anemia, elevated LDH, an R-MCI of 7-9, and absence of maintenance treatment correlated with decreased overall survival. In the Cox regression analysis, the presence of t(4;14) and an R-MCI of 7-9 were the most significant factors predicting worse outcomes.Conclusions: The t(4;14) and RMCI are reliable predictors of poor survival in newly diagnosed MM patients.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"847-854"},"PeriodicalIF":2.1,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144616861","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}

引用次数: 0

Mendelian randomization combined with transcriptomic data reveals LINC00652 as a protective factor against the risk of acute lymphoblastic leukemia. 孟德尔随机化结合转录组学数据显示，LINC00652是对抗急性淋巴细胞白血病风险的保护因子。

IF 2.1 4区医学

Expert Review of Hematology Pub Date : 2025-10-01 Epub Date: 2025-07-10 DOI: 10.1080/17474086.2025.2528886

Haiyan Qi, Jianping Lan, Wensong Wang, Xiaogang Wang

{"title":"Mendelian randomization combined with transcriptomic data reveals LINC00652 as a protective factor against the risk of acute lymphoblastic leukemia.","authors":"Haiyan Qi, Jianping Lan, Wensong Wang, Xiaogang Wang","doi":"10.1080/17474086.2025.2528886","DOIUrl":"10.1080/17474086.2025.2528886","url":null,"abstract":"Background: LINC00652 plays pivotal roles in acute lymphoblastic leukemia (ALL) a heterogeneous hematological malignancy. Here, we investigated the potential regulatory mechanisms of LINC00652 in ALL.Research design and methods: Genome-wide association study data for LINC00652 were obtained for Mendelian randomization and integrated with RNA sequencing data, Bioinformatics analyses, including weighted gene co-expression network analysis (WGCNA), Spearman or Pearson correlation analysis, function, immune microenvironment, subcellular localization, and competing endogenous RNA (ceRNA) network construction, were employed explore the potential mechanisms of LINC00652 in ALL.Results: LINC00652 exhibited no significant causal relationship with ALL but was identified as a protective factor (p > 0.05). LINC00652 levels were significantly decreased in patients with ALL, while WGCNA demonstrated the significance of black and blue modules for ALL. Seventy-nine messenger RNA (mRNAs) (SYT7) significantly correlated with LINC00652 (p > 0.05) and were enriched in the Janus kinase/signal transducer and activator of transcription (STAT), interleukin 2-STAT5, apoptosis, and adipogenesis pathways. Twenty-eight immune cells infiltrated the ALL, with immature B-cells and gamma delta T-cells significantly associated with LINC00652 expression (p < 0.05). LINC00652 was predominantly cytoplasmic, and a ceRNA network involving LINC00652, 36 microRNAs, and 18 mRNAs were proposed.Conclusions: LINC00652 May protect against ALL and influence its pathogenesis through ceRNA mechanisms.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"867-880"},"PeriodicalIF":2.1,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144539710","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}

引用次数: 0

Non-MPL-W515K/L mutations in myeloproliferative neoplasms: Insights from two case reports and a review of the literature. 骨髓增殖性肿瘤中的非MPL-W515K/L突变：来自两个病例报告和文献综述的见解

IF 2.1 4区医学

Expert Review of Hematology Pub Date : 2025-10-01 Epub Date: 2025-07-04 DOI: 10.1080/17474086.2025.2527345

Ane Sofie Tønne Nesse, Hilde Kollsete Gjelberg, Miriam Sandnes, Rakel Brendsdal Forthun, Håkon Reikvam

{"title":"Non-MPL-W515K/L mutations in myeloproliferative neoplasms: Insights from two case reports and a review of the literature.","authors":"Ane Sofie Tønne Nesse, Hilde Kollsete Gjelberg, Miriam Sandnes, Rakel Brendsdal Forthun, Håkon Reikvam","doi":"10.1080/17474086.2025.2527345","DOIUrl":"10.1080/17474086.2025.2527345","url":null,"abstract":"Background: Philadelphia chromosome-negative myeloproliferative neoplasms (MPNs) result from clonal proliferation of hematopoietic stem cells, and include polycythemia vera (PV), essential thrombocythemia (ET), and primary myelofibrosis (PMF). Key driver mutations in the JAK2, CALR, and MPL genes are important for diagnosis and differentiation of triple-negative cases. The MPL gene, particularly exon 10, harbors mutation hotspots influencing pathogenesis and prognosis.Research design and methods: This study presents two cases of atypical MPL mutations in MPN-patients and investigates the prevalence of non-canonical MPL mutations in the literature.Results: We report two MPN cases with non-canonical MPL mutations (S204P and W515R) detected by next-generation sequencing. We also conducted a systematic review of the PubMed database, identifying 67 cases of non-W515L/K MPL mutations. A total of 84 mutations were identified, comprised of 30 unique non-canonical mutations. W515R/S/A were the most frequent (32%), followed by V501A/M (15%) and S505N/C (13%). About 58% of patients had ET, 25% PMF and 13% post-ET/PV MF. Most mutations (69%) occurred in exon 10. About 26% harbored concurrent JAK2, CALR and MPL mutations.Conclusions: Our findings highlight the importance of non-canonical mutations in diagnosis of MPN to prevent misclassification and improve patient management. Understanding these mutations may lead to more tailored treatments and better outcomes in MPN patients.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"855-865"},"PeriodicalIF":2.1,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144527119","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}

引用次数: 0

Proactive measures to reduce maternal mortality and post-partum hemorrhage in sub-Saharan Africa: the hematology perspective. 减少撒哈拉以南非洲孕产妇死亡率和产后出血的积极措施：血液学观点。

IF 2.1 4区医学

Expert Review of Hematology Pub Date : 2025-10-01 Epub Date: 2025-07-01 DOI: 10.1080/17474086.2025.2527348

Bilgimol Chumappumkal Joseph, Esther Chioma Emenalom, Patrícia Silva, Annette von Drygalski

引用次数: 0

How will genomic testing impact a clinician's choice for managing chronic myeloid leukemia? 基因组检测将如何影响临床医生治疗慢性髓性白血病的选择？

IF 2.1 4区医学

Expert Review of Hematology Pub Date : 2025-10-01 Epub Date: 2025-07-16 DOI: 10.1080/17474086.2025.2533235

Jessica Sia, Naranie Shanmuganathan

{"title":"How will genomic testing impact a clinician's choice for managing chronic myeloid leukemia?","authors":"Jessica Sia, Naranie Shanmuganathan","doi":"10.1080/17474086.2025.2533235","DOIUrl":"10.1080/17474086.2025.2533235","url":null,"abstract":"Introduction: Chronic myeloid leukemia (CML) is a disease characterized by a single diagnostic molecular abnormality and epitomizes a genetically based diagnosis and management. Targeted therapy with tyrosine kinase inhibitors has revolutionized treatment and prognosis of this disease; however, a proportion of patients will experience resistance to therapy and progress to blast phase, the terminal phase of this disease. Emerging genomic profiling in CML reveals a genetically heterogenous landscape which may permit further risk stratification, personalization of treatment, and the potential for drug development.This review encompasses original literature exploring the genomic profile of CML and the potential impact of additional genomic abnormalities on prognosis and treatment response.Expert opinion: Somatic variants in cancer-associated genes, particularly ASXL1, are of prognostic and potential therapeutic significance in CML. Up-front next-generation sequencing is therefore useful when available in all patients with newly diagnosed CML and repeat testing should be considered at timepoints of suboptimal treatment response, TKI resistance, and progression. Gene rearrangements and fusions are an emerging class of mutation that may confer adverse prognostic risk and hence use of a robust testing method that enables detection of such abnormalities is desirable.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"777-783"},"PeriodicalIF":2.1,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144599892","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}

引用次数: 0