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Real-World Study of US Adults with Paroxysmal Nocturnal Hemoglobinuria Treated with Pegcetacoplan. 美国成人阵发性夜间血红蛋白尿症患者接受培基他克普兰治疗的真实世界研究。
IF 1.1 Q4 HEMATOLOGY Pub Date : 2024-10-29 DOI: 10.3390/hematolrep16040065
Brian Mulherin, Apeksha Shenoy, Lily Arnett, Weiqi Jiao, Joseph Guarinoni, Sujata Sarda, Jinny Min, David Dingli

Background: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, life-threatening disease characterized by complement-mediated hemolysis. OPERA is the first US longitudinal real-world study on C3 inhibitor therapy, known as pegcetacoplan. Methods: OPERA enrolled US patients with PNH, age ≥18, who were prescribed pegcetacoplan, and data were collected from routine care. Hemoglobin was reported by patients during regular follow-up (censored from transfusions). The Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue (0-52 score) and Patient-Reported Outcomes Measurement Information System scale for Cognitive Function Abilities (PROMIS-CF; 23.27-67.09 t-score) were completed electronically (low score = negative outcome). Patients self-reported incidence of healthcare resource utilization (HCRU). Results: By January 2024, 70 patients (mean age 44.6 years; 57.1% female) reported up to 9 months of pegcetacoplan treatment, with a median [IQR] follow-up of 6.6 [3.8] months. The latest reported hemoglobin levels improved by a mean (SD) of 2.6 (1.9) g/dL from baseline. At 3, 6 and 9 months, patients reported clinically meaningful improvements (≥5 points) in FACIT-F (53.3-69.0%) and (≥2 points) PROMIS-CF (46.7-55.2%). Patients reported a <10% incidence rate per person month of all HCRU events. Conclusions: This first longitudinal real-world US study indicates a positive trend in Hb, fatigue, and cognition with limited HCRU during pegcetacoplan treatment in adults with PNH.

背景:阵发性夜间血红蛋白尿症(PNH)是一种罕见的获得性危及生命的疾病,以补体介导的溶血为特征。OPERA 是美国第一项关于 C3 抑制剂疗法(即培西他克普兰)的纵向真实世界研究。研究方法OPERA 在美国招募了年龄≥18 岁的 PNH 患者,这些患者接受了培高氯普兰治疗,数据从常规护理中收集。血红蛋白由患者在定期随访时报告(从输血中剔除)。慢性病治疗功能评估(FACIT)-疲劳(0-52 分)和患者报告结果测量信息系统认知功能能力量表(PROMIS-CF;23.27-67.09 t-score)均以电子方式完成(低分 = 阴性结果)。患者自我报告了医疗资源利用率(HCRU)。结果:截至 2024 年 1 月,70 名患者(平均年龄 44.6 岁;57.1% 为女性)报告接受了长达 9 个月的培加氯普兰治疗,随访中位数 [IQR] 为 6.6 [3.8] 个月。最新报告的血红蛋白水平与基线相比平均(标度)提高了 2.6 (1.9) g/dL。在 3 个月、6 个月和 9 个月时,患者报告 FACIT-F(53.3%-69.0%)和 PROMIS-CF (46.7%-55.2%)有临床意义的改善(≥5 分)。患者报告结论:这项首次美国真实世界纵向研究表明,在培加氯普兰治疗成人 PNH 期间,患者的血红蛋白、疲劳和认知能力呈积极趋势,但 HCRU 有限。
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引用次数: 0
Alternative Splicing: A Potential Therapeutic Target in Hematological Malignancies. 替代剪接:血液恶性肿瘤的潜在治疗靶点
IF 1.1 Q4 HEMATOLOGY Pub Date : 2024-10-29 DOI: 10.3390/hematolrep16040066
Gazmend Temaj, Silvia Chichiarelli, Sarmistha Saha, Pelin Telkoparan-Akillilar, Nexhibe Nuhii, Rifat Hadziselimovic, Luciano Saso

Leukemia represents the most prevalent malignancy in children, constituting 30% of childhood cancer cases, with acute lymphoblastic leukemia (ALL) being particularly heterogeneous. This paper explores the role of alternative splicing in leukemia, highlighting its significance in cancer development and progression. Aberrant splicing is often driven by mutations in splicing-factor genes, which can lead to the production of variant proteins that contribute to oncogenesis. The spliceosome, a complex of small nuclear RNAs and proteins, facilitates RNA splicing, a process critical for generating diverse mRNA and protein products from single genes. Mutations in splicing factors, such as U2AF1, SF3B1, SRSF2, ZRSR2, and HNRNPH1, are frequently observed across various hematological malignancies and are associated with poor prognosis and treatment resistance. This research underscores the necessity of understanding the mechanisms of RNA splicing dysregulation in order to develop targeted therapies to correct these aberrant processes, thereby improving outcomes for patients with leukemia and related disorders.

白血病是儿童最常见的恶性肿瘤,占儿童癌症病例的 30%,其中急性淋巴细胞白血病(ALL)尤其具有异质性。本文探讨了替代剪接在白血病中的作用,强调了它在癌症发生和发展过程中的重要意义。剪接异常通常是由剪接因子基因突变驱动的,这会导致产生有助于致癌的变异蛋白。剪接体是由核小 RNA 和蛋白质组成的复合体,它促进了 RNA 的剪接,这一过程对于从单个基因产生多样化的 mRNA 和蛋白质产物至关重要。U2AF1、SF3B1、SRSF2、ZRSR2 和 HNRNPH1 等剪接因子的突变在各种血液恶性肿瘤中经常出现,并与预后不良和耐药性有关。这项研究强调了了解 RNA 剪接失调机制的必要性,以便开发出纠正这些异常过程的靶向疗法,从而改善白血病及相关疾病患者的预后。
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引用次数: 0
Gene Therapy: A Revolutionary Step in Treating Thalassemia. 基因疗法:治疗地中海贫血症的革命性一步。
IF 1.1 Q4 HEMATOLOGY Pub Date : 2024-10-21 DOI: 10.3390/hematolrep16040064
Jhancy Malay, Rasha Aziz Attia Salama, Ghania Shehzad Alam Qureshi, Ali Raafat Ali Ahmed Ammar, Gayatri Janardhan, Maryam Safdar, Hesham Amin Hamdy Elshamy

Beta thalassemia is an inherited blood disorder that results in inefficient erythropoiesis due to genetic mutation that leads to the reduction or absence of the hemoglobin beta-globulin protein. Approximately 8.5% of UAE residents suffer from β-thalassemia, a significant health and financial problem. The treatment options available for β-Thalassemia major are limited and associated with a wide range of complications. β-thalassemia gene therapy is emerging as a potential novel treatment option that eliminates the complications caused by the current long-term treatment modalities and the associated economic burden. This paper reviews the scientific literature related to emerging gene therapy for β-Thalassemia by analyzing all the articles published from January 2010 to December 2023 in the English language on Databases like PubMed, Scopus, ProQuest, and CINAHL. The use of gene therapy has demonstrated promising outcomes for a permanent cure of β-Thalassemia. To conclude, gene therapy is an innovative solution. It demonstrates a promising future, but does come with its own setbacks and is something that must be tackled in order to revolutionize it in the medical world. FDA-approved ZYNTEGLO is a potentially one-time curative treatment for β-Thalassemia. Although cutting-edge, its use is limited because of the high cost-a price of USD 2.8 million per patient.

β地中海贫血症是一种遗传性血液疾病,由于基因突变导致血红蛋白β-球蛋白蛋白减少或缺失,从而导致红细胞生成效率低下。大约 8.5% 的阿联酋居民患有 β-地中海贫血症,这是一个严重的健康和经济问题。重型β-地中海贫血症的治疗方案有限,且伴有多种并发症。β-地中海贫血基因疗法正在成为一种潜在的新型治疗方案,它可以消除目前长期治疗方法所引起的并发症和相关的经济负担。本文通过分析 2010 年 1 月至 2023 年 12 月期间在 PubMed、Scopus、ProQuest 和 CINAHL 等数据库中发表的所有英文文章,对有关新出现的β-地中海贫血基因疗法的科学文献进行了综述。基因疗法的使用为永久治愈β-地中海贫血症带来了希望。总之,基因疗法是一种创新的解决方案。它展示了一个充满希望的未来,但也有其自身的挫折,必须加以解决,才能在医学界掀起一场革命。美国食品和药物管理局批准的 ZYNTEGLO 有可能一次性治愈 β-地中海贫血症。虽然它是最先进的治疗方法,但由于成本高昂,每名患者的治疗费用高达 280 万美元,因此其使用受到了限制。
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引用次数: 0
Pancytopenia Related to Splenic Angiosarcoma: A Case Report and Literature Review. 与脾脏血管肉瘤相关的全血细胞减少症:病例报告和文献综述。
IF 1.1 Q4 HEMATOLOGY Pub Date : 2024-10-18 DOI: 10.3390/hematolrep16040063
Jakub Misiak, Bernard Sokołowski, Norbert Skrobisz, Mateusz Matczak, Marcin Braun

Background: Angiosarcomas are highly aggressive malignancies with endothelial differentiation, presenting considerable challenges in oncology, especially when arising in rare locations such as the spleen. These tumors predominantly affect adults and are commonly found in the skin, breast, liver, or soft tissues, with more unusual occurrences in other organs. Angiosarcomas have a high propensity for metastasis, typically spreading to the liver, lungs, lymph nodes, and gastrointestinal tract. Splenic angiosarcoma, with fewer than 300 documented cases, is an especially rare and complex form of this malignancy.

Case presentation: This report details a case of splenic angiosarcoma in a 45-year-old male, where bone marrow metastases were the first clinical presentation, initially mimicking myelodysplastic syndrome (MDS) due to persistent pancytopenia.

Conclusions: The eventual identification of the splenic origin underscores the diagnostic difficulties and clinical challenges inherent in managing such atypical and rare presentations.

背景:血管肉瘤是具有内皮分化的高度侵袭性恶性肿瘤,给肿瘤学带来了相当大的挑战,尤其是发生在脾脏等罕见部位的血管肉瘤。这类肿瘤主要影响成年人,常见于皮肤、乳腺、肝脏或软组织,在其他器官的发生率较低。血管肉瘤有很高的转移倾向,通常会扩散到肝、肺、淋巴结和胃肠道。脾血管肉瘤记录在案的病例不到 300 例,是这种恶性肿瘤中尤为罕见和复杂的一种:本报告详细介绍了一例脾血管肉瘤病例,患者为 45 岁男性,骨髓转移是第一临床表现,最初因持续泛发性骨髓增生异常综合征(MDS)而模仿骨髓转移:结论:脾脏来源的最终确定凸显了在处理此类非典型和罕见病例时固有的诊断困难和临床挑战。
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引用次数: 0
Trabecular Attenuation of L1 in Adult Patients with Multiple Myeloma: An Observational Study on Low-Dose CT Images. 多发性骨髓瘤成人患者 L1 小梁衰减:低剂量 CT 图像观察研究
IF 1.1 Q4 HEMATOLOGY Pub Date : 2024-10-17 DOI: 10.3390/hematolrep16040061
Carlo Augusto Mallio, Valeria Tomarchio, Francesco Pulcini, Edoardo Verducci, Caterina Bernetti, Maria Antonietta Tafuri, Federico Greco, Luigi Rigacci, Bruno Beomonte Zobel, Ombretta Annibali

Background: The aim of this study was to evaluate the impact of trabecular attenuation of the L1 vertebral body in low-dose CT in adult patients with multiple myeloma (MM), smoldering multiple myeloma (SMM), and monoclonal gammopathy of undetermined significance (MGUS).

Materials and methods: The study population consisted of 22 patients with MGUS and 51 consecutive patients with newly diagnosed MM (SMM, n = 21; symptomatic MM, n = 36). CT scans were conducted using a 128-slice CT scanner (Somatom go.Top, Siemens, Munich, Germany). Low-dose whole-body CT scans were performed at a single time point for each patient. Trabecular bone density values were obtained by defining regions of interest on non-contrast images at the level of L1 vertebra. A threshold of p = 0.05 was applied to determine statistical significance.

Results: The median Hounsfield unit (HU) value in patients with MGUS, SMM, and MM was 148 HU (range 81-190), 130 HU (range 93-193), and 92 HU (range 26-190), respectively, with a statistically significant difference between the groups (p = 0.0015). Patients with HU values ≤ 92 had lower progression-free survival with statistically significant differences compared to the group with HU values > 92 (p < 0.0499).

Conclusions: This is the earliest evidence of the importance of evaluating L1 attenuation values in low-dose CT images in patients with MGUS, SMM, and MM. Further prospective studies could contribute to reinforcing these results and exploring the clinical applicability and generalization of L1 attenuation values in low-dose whole-body CT scans in routine clinical practice.

研究背景本研究旨在评估多发性骨髓瘤(MM)、烟雾型多发性骨髓瘤(SMM)和意义未定的单克隆丙种球蛋白病(MGUS)成年患者低剂量 CT 中 L1 椎体小梁衰减的影响:研究对象包括22名MGUS患者和51名新近确诊的MM患者(SMM,21人;无症状MM,36人)。CT扫描使用128排CT扫描仪(德国慕尼黑西门子公司Somatom go.Top)进行。每位患者在一个时间点进行低剂量全身 CT 扫描。通过在L1椎体水平的非对比图像上定义感兴趣区,获得骨小梁密度值。采用 p = 0.05 的阈值来确定统计学意义:结果:MGUS、SMM 和 MM 患者的中位 Hounsfield 单位(HU)值分别为 148 HU(范围 81-190)、130 HU(范围 93-193)和 92 HU(范围 26-190),组间差异有统计学意义(p = 0.0015)。HU值≤92的患者无进展生存期较低,与HU值>92的组相比,差异有统计学意义(p < 0.0499):这是低剂量CT图像中L1衰减值评估对MGUS、SMM和MM患者重要性的最早证据。进一步的前瞻性研究有助于巩固这些结果,并探索低剂量全身CT扫描中L1衰减值在常规临床实践中的临床适用性和普遍性。
{"title":"Trabecular Attenuation of L1 in Adult Patients with Multiple Myeloma: An Observational Study on Low-Dose CT Images.","authors":"Carlo Augusto Mallio, Valeria Tomarchio, Francesco Pulcini, Edoardo Verducci, Caterina Bernetti, Maria Antonietta Tafuri, Federico Greco, Luigi Rigacci, Bruno Beomonte Zobel, Ombretta Annibali","doi":"10.3390/hematolrep16040061","DOIUrl":"https://doi.org/10.3390/hematolrep16040061","url":null,"abstract":"<p><strong>Background: </strong>The aim of this study was to evaluate the impact of trabecular attenuation of the L1 vertebral body in low-dose CT in adult patients with multiple myeloma (MM), smoldering multiple myeloma (SMM), and monoclonal gammopathy of undetermined significance (MGUS).</p><p><strong>Materials and methods: </strong>The study population consisted of 22 patients with MGUS and 51 consecutive patients with newly diagnosed MM (SMM, <i>n</i> = 21; symptomatic MM, <i>n</i> = 36). CT scans were conducted using a 128-slice CT scanner (Somatom go.Top, Siemens, Munich, Germany). Low-dose whole-body CT scans were performed at a single time point for each patient. Trabecular bone density values were obtained by defining regions of interest on non-contrast images at the level of L1 vertebra. A threshold of <i>p</i> = 0.05 was applied to determine statistical significance.</p><p><strong>Results: </strong>The median Hounsfield unit (HU) value in patients with MGUS, SMM, and MM was 148 HU (range 81-190), 130 HU (range 93-193), and 92 HU (range 26-190), respectively, with a statistically significant difference between the groups (<i>p</i> = 0.0015). Patients with HU values ≤ 92 had lower progression-free survival with statistically significant differences compared to the group with HU values > 92 (<i>p</i> < 0.0499).</p><p><strong>Conclusions: </strong>This is the earliest evidence of the importance of evaluating L1 attenuation values in low-dose CT images in patients with MGUS, SMM, and MM. Further prospective studies could contribute to reinforcing these results and exploring the clinical applicability and generalization of L1 attenuation values in low-dose whole-body CT scans in routine clinical practice.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"16 4","pages":"624-635"},"PeriodicalIF":1.1,"publicationDate":"2024-10-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11503432/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142499163","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Outcome of Allogeneic Hematopoietic Stem Cell Transplantation in Adult Patients with Acute Lymphoblastic Leukemia: Results of a Single-Center Study. 急性淋巴细胞白血病成人患者异基因造血干细胞移植的结果:单中心研究结果。
IF 1.1 Q4 HEMATOLOGY Pub Date : 2024-10-17 DOI: 10.3390/hematolrep16040062
Davide Stella, Jessica Gill, Roberto Passera, Sofia Zompi, Chiara Maria Dellacasa, Ernesta Audisio, Marco Cerrano, Irene Dogliotti, Michele Dicataldo, Carolina Secreto, Benedetto Bruno, Roberto Freilone, Alessandro Busca, Luisa Giaccone

Background: Despite the adoption of pediatric-like chemotherapy protocols, the introduction of new immunotherapies and a better understanding of the oncogenic landscape, the outcome for adult patients with acute lymphoblastic leukemia (ALL) remain substantially dismal. The aim of the present study was to evaluate the outcome in terms of survival in a cohort of adult patients with ALL who received allogeneic hematopoietic stem cell transplantation (alloSCT) between 2013 and 2023.

Methods: This was a single-center observational retrospective study including all consecutive adult patients with ALL who received an alloSCT between April 2013 and April 2023 at the Stem Cell Transplant Center AOU Città della Salute e della Scienza of Torino. The primary endpoints were overall survival (OS), graft-versus-host disease (GVHD) Relapse-Free Survival (GRFS), Leukemia-Free Survival (LFS) and cumulative incidence (CI) of Non-Relapse Mortality (NRM).

Results: The 4-year OS and LFS were 63.4% and 48.1%, respectively, and the 1-year GRFS was 42.9%. The 1-year CI of bloodstream infections (BSI), invasive fungal infections and NRM were 38%, 7% and 18.4%, respectively. Multivariate analysis showed that the use of total body irradiation (TBI), a time interval from diagnosis to alloSCT 7 months and female gender were factors significantly associated with better OS. Relapse of the underlying malignancy and BSI were the main causes of death.

Conclusion: Our study suggests that alloSCT from a matched sibling donor (MSD) and alternative donors may be considered an effective tool for patients with ALL achieving a CR.

背景:尽管采用了类似儿科的化疗方案,引入了新的免疫疗法,并对致癌物质有了更深入的了解,但急性淋巴细胞白血病(ALL)成人患者的治疗效果仍然不容乐观。本研究旨在评估2013年至2023年间接受异基因造血干细胞移植(alloSCT)的成年ALL患者的生存结果:这是一项单中心观察性回顾研究,包括2013年4月至2023年4月期间在都灵AOU Città della Salute e della Scienza干细胞移植中心接受异体造血干细胞移植的所有连续成年ALL患者。主要终点为总生存期(OS)、移植物抗宿主病(GVHD)无复发生存期(GRFS)、无白血病生存期(LFS)和非复发死亡率(NRM)累积发生率(CI):结果:4年OS和LFS分别为63.4%和48.1%,1年GRFS为42.9%。1年血流感染(BSI)、侵袭性真菌感染和非复发死亡率的CI分别为38%、7%和18.4%。多变量分析表明,全身照射(TBI)的使用、从诊断到异体移植的时间间隔为7个月和女性性别是与较好的OS显著相关的因素。基础恶性肿瘤复发和BSI是死亡的主要原因:我们的研究表明,对于达到CR的ALL患者来说,配型同胞供体(MSD)和替代供体的异体干细胞移植可被视为一种有效的手段。
{"title":"Outcome of Allogeneic Hematopoietic Stem Cell Transplantation in Adult Patients with Acute Lymphoblastic Leukemia: Results of a Single-Center Study.","authors":"Davide Stella, Jessica Gill, Roberto Passera, Sofia Zompi, Chiara Maria Dellacasa, Ernesta Audisio, Marco Cerrano, Irene Dogliotti, Michele Dicataldo, Carolina Secreto, Benedetto Bruno, Roberto Freilone, Alessandro Busca, Luisa Giaccone","doi":"10.3390/hematolrep16040062","DOIUrl":"https://doi.org/10.3390/hematolrep16040062","url":null,"abstract":"<p><strong>Background: </strong>Despite the adoption of pediatric-like chemotherapy protocols, the introduction of new immunotherapies and a better understanding of the oncogenic landscape, the outcome for adult patients with acute lymphoblastic leukemia (ALL) remain substantially dismal. The aim of the present study was to evaluate the outcome in terms of survival in a cohort of adult patients with ALL who received allogeneic hematopoietic stem cell transplantation (alloSCT) between 2013 and 2023.</p><p><strong>Methods: </strong>This was a single-center observational retrospective study including all consecutive adult patients with ALL who received an alloSCT between April 2013 and April 2023 at the Stem Cell Transplant Center AOU Città della Salute e della Scienza of Torino. The primary endpoints were overall survival (OS), graft-versus-host disease (GVHD) Relapse-Free Survival (GRFS), Leukemia-Free Survival (LFS) and cumulative incidence (CI) of Non-Relapse Mortality (NRM).</p><p><strong>Results: </strong>The 4-year OS and LFS were 63.4% and 48.1%, respectively, and the 1-year GRFS was 42.9%. The 1-year CI of bloodstream infections (BSI), invasive fungal infections and NRM were 38%, 7% and 18.4%, respectively. Multivariate analysis showed that the use of total body irradiation (TBI), a time interval from diagnosis to alloSCT 7 months and female gender were factors significantly associated with better OS. Relapse of the underlying malignancy and BSI were the main causes of death.</p><p><strong>Conclusion: </strong>Our study suggests that alloSCT from a matched sibling donor (MSD) and alternative donors may be considered an effective tool for patients with ALL achieving a CR.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"16 4","pages":"636-647"},"PeriodicalIF":1.1,"publicationDate":"2024-10-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11503301/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142499159","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Utilizing Clinical Transformation Criteria for Prognostic Stratification in Follicular Lymphoma Prior to Initial Immunochemotherapy. 在初始免疫化疗前利用临床转化标准对滤泡性淋巴瘤进行预后分层
IF 1.1 Q4 HEMATOLOGY Pub Date : 2024-10-04 DOI: 10.3390/hematolrep16040060
Yoshikazu Hori, Hiroki Hosoi, Takayuki Hiroi, Ke Wan, Shogo Murata, Masaya Morimoto, Toshiki Mushino, Akinori Nishikawa, Takashi Sonoki

Background: Although the prognosis of follicular lymphoma (FL) has improved, some patients experience early disease progression, including progression of disease within 24 months (POD24). Histological transformation is a critical event in FL. However, the heterogeneity of FL tumors makes it challenging to diagnose transformation accurately. We retrospectively applied the clinical transformation criteria used for FL transformation assessments at relapse or disease progression to conduct transformation assessments before the initial immunochemotherapy.

Methods: Sixty-six FL patients who first received immunochemotherapy between January 2009 and February 2023 at our institution were selected. Twenty-three were clinical-transformation-positive (CLT+).

Results: The progression-free survival (PFS) rate of the CLT+ patients was significantly lower than that of the clinical-transformation-negative (CLT-) patients. In the POD24 assessment subgroup, the CLT+ patients had a higher incidence of POD24 than the CLT- patients. There was no significant difference in PFS between the patients treated with CHOP-like regimens and those treated with bendamustine regimens. In the CHOP-like group, the CLT+ patients exhibited significantly lower PFS than the CLT- patients. In the bendamustine group, the clinical transformation did not affect PFS.

Conclusion: Clinical transformation criteria may be useful for the prognostic stratification of FL prior to immunochemotherapy. Additionally, they may serve as predictors of POD24.

背景:尽管滤泡性淋巴瘤(FL)的预后有所改善,但仍有一些患者会出现早期疾病进展,包括 24 个月内疾病进展(POD24)。组织学转化是 FL 的关键事件。然而,FL 肿瘤的异质性使得准确诊断转化具有挑战性。我们回顾性地将复发或疾病进展时用于FL转化评估的临床转化标准应用于首次免疫化疗前的转化评估:方法:选取 2009 年 1 月至 2023 年 2 月期间在我院首次接受免疫化疗的 66 例 FL 患者。23例为临床转化阳性(CLT+):结果:CLT+患者的无进展生存期(PFS)明显低于临床转化阴性(CLT-)患者。在POD24评估亚组中,CLT+患者的POD24发生率高于CLT-患者。接受CHOP类方案治疗的患者与接受苯达莫司汀方案治疗的患者在PFS方面没有明显差异。在CHOP类药物组中,CLT+患者的PFS明显低于CLT-患者。在苯达莫司汀组,临床转化并不影响PFS:临床转化标准可能有助于在免疫化疗前对 FL 进行预后分层。此外,它们还可作为 POD24 的预测指标。
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引用次数: 0
Comprehensive Genomic Profiling in Non-Myeloid Hematologic Malignancies Identifies Variants That Can Alter Clinical Practice. 非髓性血液恶性肿瘤的全面基因组分析发现了可改变临床实践的变异。
IF 1.1 Q4 HEMATOLOGY Pub Date : 2024-09-30 DOI: 10.3390/hematolrep16040059
Chenyu Lin, Katherine I Zhou, Michelle F Green, Bennett A Caughey, John H Strickler, Michael B Datto, Matthew S McKinney

Background: Comprehensive genomic profiling (CGP) is frequently adopted to direct the clinical care of myeloid neoplasms and solid tumors, but its utility in the care of lymphoid and histiocytic cancers is less well defined.

Methods: In this study, we aimed to evaluate the frequency at which mutations identified by CGP altered management in non-myeloid hematologic malignancies. We retrospectively examined the CGP results of 105 samples from 101 patients with non-myeloid hematologic malignancies treated at an academic medical center who had CGP testing between 2014 and 2021.

Results: CGP revealed one or more pathogenic or likely pathogenic variant in 92 (88%) of samples and 73 (72%) of tested patients had one or more mutations with diagnostic, prognostic, or therapeutic significance. The identification of a resistance variant resulted in the suspension of the active treatment or affected subsequent treatment choice in 9 (69%) out of 13 patients. However, the presence of a therapy sensitizing variant only led to consideration of a biomarker-directed therapy in 6 (10%) out of 61 patients.

Conclusions: Overall, CGP of non-myeloid hematologic malignancies identified clinically significant variants in 72% of patients and resulted in a change in management in 22% of patients.

背景:全面基因组分析(CGP)经常被用于指导髓系肿瘤和实体瘤的临床治疗,但其在淋巴细胞和组织细胞癌症治疗中的作用还不太明确:在这项研究中,我们旨在评估 CGP 发现的突变改变非骨髓性血液恶性肿瘤治疗的频率。我们回顾性研究了一个学术医疗中心在2014年至2021年期间对101名接受CGP检测的非骨髓性血液恶性肿瘤患者的105份样本的CGP结果:92份样本(88%)的CGP发现了一种或多种致病或可能致病的变异,73份样本(72%)的受检患者出现了一种或多种具有诊断、预后或治疗意义的突变。在 13 名患者中,有 9 人(69%)因耐药变异的鉴定而暂停了积极治疗或影响了后续治疗的选择。然而,在61名患者中,只有6名(10%)患者因出现治疗敏感变异而考虑采用生物标记物导向疗法:总的来说,非骨髓性血液恶性肿瘤CGP在72%的患者中发现了具有临床意义的变异体,并导致22%的患者改变了治疗方案。
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引用次数: 0
The Real-World Outcomes of Relapsed/Refractory Multiple Myeloma Treated with Elotuzumab, Pomalidomide, and Dexamethasone. 埃洛珠单抗、泊马度胺和地塞米松治疗复发性/难治性多发性骨髓瘤的实际效果
IF 1.1 Q4 HEMATOLOGY Pub Date : 2024-09-30 DOI: 10.3390/hematolrep16040058
Hitomi Nakayama, Yoshinobu Aisa, Chisako Ito, Aki Sakurai, Tomonori Nakazato

Introduction: A combination of elotuzumab, pomalidomide, and dexamethasone (EPd) was approved for the treatment of relapsed/refractory multiple myeloma (RRMM) following the ELOQUENT-3 phase II clinical trial. However, the clinical experience with this therapy is still limited. In this retrospective study, we analyzed the efficacy and safety of EPd in a real-world cohort of RRMM patients. Patients and Methods: The medical records of 22 patients who received EPd for RRMM at Yokohama Municipal Citizen's Hospital (Japan) between January 2020 and July 2021 were reviewed. Results: The median age of our cohort was 73.5 years. The overall response rate was 55%. With a median follow-up of 20.2 months, the median progression-free survival (PFS) was 9.1 months (95% confidence interval [CI], 2.5-23.0 months). The median PFS was shorter in patients with a poor performance status (PS) than in those with favorable PS (2.5 vs. 10.8 months; p < 0.01). Patients with prior daratumumab had significantly shorter PFS than those without prior daratumumab (2.1 vs. 23.0 months; p < 0.01). Additionally, patients with prior pomalidomide had significantly shorter PFS (1.7 vs. 10.3 months; p < 0.01). In the multivariate analysis, poor PS (hazard ratio [HR] = 4.1, 95% CI: 1.1-15.6; p = 0.04) and prior exposure to daratumumab (HR = 3.8, 95% CI: 1.1-13.8; p = 0.04) remained significantly associated with shorter PFS. Conclusions: The results of our study suggest that EPd is an active and well-tolerated regimen in RRMM, even in real-world patients. Furthermore, EPd may be useful, especially in daratumumab-naïve patients.

简介在ELOQUENT-3 II期临床试验后,埃洛珠单抗、泊马度胺和地塞米松(EPd)联合疗法被批准用于治疗复发/难治性多发性骨髓瘤(RRMM)。然而,这种疗法的临床经验仍然有限。在这项回顾性研究中,我们分析了EPd在RRMM患者队列中的疗效和安全性。患者和方法:回顾2020年1月至2021年7月期间在横滨市民医院(日本)接受EPd治疗RRMM的22名患者的病历。结果组群的中位年龄为 73.5 岁。总体应答率为 55%。中位随访时间为 20.2 个月,中位无进展生存期(PFS)为 9.1 个月(95% 置信区间 [CI],2.5-23.0 个月)。表现状态(PS)较差的患者的中位无进展生存期比表现良好的患者短(2.5 个月对 10.8 个月;P < 0.01)。既往使用过达拉曲单抗的患者的中位生存期明显短于未使用过达拉曲单抗的患者(2.1个月对23.0个月;P<0.01)。此外,既往使用过泊马度胺的患者的 PFS 明显更短(1.7 个月 vs. 10.3 个月;p < 0.01)。在多变量分析中,不良PS(危险比[HR] = 4.1,95% CI:1.1-15.6;p = 0.04)和既往使用过达拉曲单抗(HR = 3.8,95% CI:1.1-13.8;p = 0.04)仍与较短的PFS显著相关。结论我们的研究结果表明,EPd是治疗RRMM的一种积极且耐受性良好的方案,即使在现实世界的患者中也是如此。此外,EPd可能是有用的,尤其是在达拉单抗无效的患者中。
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引用次数: 0
Two Cases of Immune Thrombocytopenia (ITP) Related to Viral Vector Vaccination ChAdOx1-S (AstraZeneca) and a Good Response after Thrombopoietin Receptor Agonist (TPO-RA) Therapy. 两例与病毒载体疫苗 ChAdOx1-S (AstraZeneca) 相关的免疫性血小板减少症 (ITP) 病例及接受促血小板生成素受体激动剂 (TPO-RA) 治疗后的良好反应。
IF 1.1 Q4 HEMATOLOGY Pub Date : 2024-09-27 DOI: 10.3390/hematolrep16040057
Konstantina Salveridou, Theodoros Tzamalis, Maika Klaiber-Hakimi, Sabine Haase, Stefanie Gröpper, Aristoteles Giagounidis

Background: In 2019, a new coronavirus disease emerged in Wuhan, China, known as SARS-CoV-2, severe acute respiratory syndrome coronavirus 2, and caused an ongoing pandemic. Symptomatology of the syndrome is variable, with complications extending to hematopoiesis and hemostasis. Approximately a year after onset of the virus, four vaccination formulas became available to the public, based on a viral vector or mRNA technology. These vaccine formulas have been hampered with hematological complications, like vaccine-induced immune thrombotic thrombocytopenia (VITT) and vaccine-related ITP (immune thrombocytopenic purpura). ITP is a disease with autoimmune pathogenesis characterized by antibody production against platelets and an increased hemorrhagic risk. A decent number of cases have been referred to as possible adverse effects of COVID-19 vaccinations.

Case presentation: in this case report, we present two cases of newly diagnosed ITP after vaccination with ChAdOx1-S (AstraZeneca), with a good response to treatment with thrombopoietin-receptor agonists (TPO-RAs).

Discussion: we observed an absence of response after corticosteroids and IVIG therapy and a positive therapeutic outcome on TPO-RA.

Conclusions: in the ongoing pandemic, there is an urgent need to create therapeutic guidelines for vaccination-related clinical entities and to clarify indications for the vaccination of patients with pre-existing hematological diseases.

背景:2019年,中国武汉出现了一种新型冠状病毒疾病,被称为SARS-CoV-2,即严重急性呼吸系统综合征冠状病毒2型,并引发了持续的大流行。该综合征的症状多种多样,并发症涉及造血和止血。病毒出现约一年后,公众可获得四种基于病毒载体或 mRNA 技术的疫苗配方。这些疫苗配方都受到血液并发症的影响,如疫苗诱发的免疫性血小板减少症(VITT)和疫苗相关的 ITP(免疫性血小板减少性紫癜)。免疫性血小板减少性紫癜是一种具有自身免疫发病机制的疾病,其特点是产生针对血小板的抗体并增加出血风险。病例介绍:在本病例报告中,我们介绍了两例接种 ChAdOx1-S(阿斯利康)疫苗后新诊断的 ITP 病例,患者对血小板生成素受体激动剂(TPO-RA)治疗反应良好。讨论:我们观察到皮质类固醇和 IVIG 治疗后无反应,TPO-RA 治疗效果良好。结论:在当前的大流行病中,迫切需要为疫苗接种相关的临床实体制定治疗指南,并明确已有血液病患者的疫苗接种适应症。
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引用次数: 0
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Hematology Reports
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