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Thalassaemia: advocacy, resilience, and adaptability. 地中海贫血:宣传、恢复力和适应性。
IF 17.7 1区 医学 Q1 HEMATOLOGY Pub Date : 2026-01-01 DOI: 10.1016/S2352-3026(25)00346-1
The Lancet Haematology
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引用次数: 0
Correction to Lancet Haematol 2025; 12: e978-85. 《柳叶刀血液学杂志2025》修正;12: e978 - 85。
IF 17.7 1区 医学 Q1 HEMATOLOGY Pub Date : 2026-01-01 DOI: 10.1016/S2352-3026(25)00345-X
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引用次数: 0
Iberdomide plus low-dose cyclophosphamide and dexamethasone in patients with relapsed and refractory multiple myeloma (the ICON study): a multicentre, single-arm, phase 2 trial. 伊伯度胺联合低剂量环磷酰胺和地塞米松治疗复发和难治性多发性骨髓瘤(ICON研究):一项多中心、单臂、2期试验。
IF 17.7 1区 医学 Q1 HEMATOLOGY Pub Date : 2026-01-01 DOI: 10.1016/S2352-3026(25)00298-4
Charlotte L B M Korst, Wouter Plattel, Elizabeth A de Kort, Febe Smits, Alexandra J Croockewit, Mark-David Levin, Matthijs Westerman, Okke de Weerdt, Inger S Nijhof, Jurgen Wegman, Nina Smit, Christie P M Verkleij, Tuna Mutis, Kazem Nasserinejad, Ramses Kerstiens, Marjolein van der Klift, Laurens E Franssen, Maaike E M de Ruijter, Kaz Groen, Ellen van der Spek, Wilfried W H Roeloffzen, Sonja Zweegman, Niels W C J van de Donk

Background: Iberdomide is an oral cereblon E3 ligase modulator with higher affinity to cereblon than immunomodulatory drugs, leading to improved direct anti-myeloma activity and enhanced immunostimulatory effects. We aimed to evaluate the safety and activity of iberdomide plus low-dose cyclophosphamide and dexamethasone (IberCd) in patients with relapsed and refractory multiple myeloma.

Methods: The ICON study is a prospective, single-arm, phase 2, open-label study conducted in eight hospitals in the Netherlands. We enrolled patients (aged ≥18 years) with relapsed and refractory multiple myeloma (lenalidomide-refractory) who had received two to four previous lines of therapy and had a WHO performance status of 0-2. Patients were treated with oral iberdomide (1·6 mg/day on days 1-21 of each 28-day cycle), oral low-dose cyclophosphamide (50 mg/day on days 1-28), and oral dexamethasone (40 mg [20 mg in patients age >75 years] once a week) until progression. All patients received thrombosis prophylaxis daily: 80 mg oral aspirin or 100 mg oral carbasalate calcium. Patients with a previous history of venous thromboembolism received only low molecular-weight heparin by subcutaneous injection. The primary endpoint was progression-free survival, defined as time from the start of treatment to the date of progression or death. Activity and safety were assessed in all patients who started treatment. This trial was registered at ClinicalTrials.gov (NCT04392037) and is ongoing.

Findings: Between Feb 17, 2021, and July 7, 2023, 61 patients were enrolled and received IberCd treatment (29 [48%] were female and 32 [52%] patients were male). The median number of previous lines of therapy was 3 (range 2-5); 52 (85%) patients were triple-class exposed and 27 (44%) had triple-class refractory disease. 50 patients discontinued study treatment, 39 of whom due to progressive disease, but all 61 patients were included in the main analysis population. After a median follow-up of 25·4 months (IQR 19·7-31·6), the median progression-free survival was 17·6 months (one-sided 95% CI 16·6-19·9). In all 61 patients, the most common grade 3-4 adverse events were neutropenia (34 [56%] patients) and infections (21 [34%] patients). Treatment-related serious adverse events were reported in 25 (41%) patients, with infections being the most common (34 [71%] of 48 serious adverse events). Treatment-related death occurred in one (2%) patient due to COVID-19.

Interpretation: IberCd is an all-oral and active combination for patients with relapsed and refractory multiple myeloma that showed clinically meaningful activity. This regimen offers a valuable treatment option for patients who have received two to four previous lines of therapy and compares favourably with other available treatments.

Funding: Bristol Myers Squibb.

背景:Iberdomide是一种口服小脑E3连接酶调节剂,与免疫调节药物相比,其对小脑的亲和力更高,可提高直接抗骨髓瘤活性,增强免疫刺激作用。我们的目的是评估伊伯度胺联合低剂量环磷酰胺和地塞米松(IberCd)治疗复发和难治性多发性骨髓瘤患者的安全性和活性。方法:ICON研究是一项前瞻性、单臂、2期、开放标签研究,在荷兰的8家医院进行。我们招募了复发和难治性多发性骨髓瘤(来那度胺难治性)患者(年龄≥18岁),这些患者之前接受过2至4条治疗线,who表现状态为0-2。患者接受口服伊伯度胺(1·6 mg/天,每28天1-21天)、口服低剂量环磷酰胺(50 mg/天,1-28天)和口服地塞米松(40 mg[年龄在50 - 75岁的患者20 mg],每周一次)治疗,直至病情进展。所有患者每天接受血栓预防:口服阿司匹林80毫克或口服碳酸盐钙100毫克。有静脉血栓栓塞史的患者只接受低分子肝素皮下注射。主要终点是无进展生存期,定义为从治疗开始到进展或死亡日期的时间。对所有开始治疗的患者进行活性和安全性评估。该试验已在ClinicalTrials.gov注册(NCT04392037),并正在进行中。结果:在2021年2月17日至2023年7月7日期间,61例患者入组并接受了IberCd治疗(29例[48%]为女性,32例[52%]为男性)。既往治疗线数中位数为3(范围2-5);52例(85%)患者为三级暴露,27例(44%)患者为三级难治性疾病。50例患者停止了研究治疗,其中39例因疾病进展,但所有61例患者均纳入主要分析人群。中位随访为25.4个月(IQR 19.7 - 31.6),中位无进展生存期为17.6个月(单侧95% CI 16.6 - 19.9)。在所有61例患者中,最常见的3-4级不良事件是中性粒细胞减少(34例[56%])和感染(21例[34%])。25例(41%)患者报告了与治疗相关的严重不良事件,其中感染最为常见(48例严重不良事件中34例[71%])。1例(2%)患者因COVID-19发生治疗相关死亡。解释:IberCd是一种用于复发和难治性多发性骨髓瘤患者的全口服和活性组合药物,具有临床意义。该方案为接受过2 - 4次治疗的患者提供了一个有价值的治疗选择,并且与其他可用的治疗方法相比具有优势。融资:Bristol Myers Squibb。
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引用次数: 0
Lessons from both sides of the bedrail. 从床轨两边吸取教训。
IF 17.7 1区 医学 Q1 HEMATOLOGY Pub Date : 2026-01-01 DOI: 10.1016/S2352-3026(25)00285-6
Andreas Seas
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引用次数: 0
Patient-reported outcomes with belantamab mafodotin, pomalidomide, and dexamethasone versus bortezomib, pomalidomide, and dexamethasone in patients with relapsed or refractory multiple myeloma (DREAMM-8): a phase 3, open-label, randomised controlled trial. 患者报告的复发或难治性多发性骨髓瘤患者使用贝兰他单、马弗多汀、泊马度胺和地塞米松与硼替佐米、泊马度胺和地塞米松的结果(dreams -8):一项3期、开放标签、随机对照试验。
IF 17.7 1区 医学 Q1 HEMATOLOGY Pub Date : 2025-11-01 DOI: 10.1016/S2352-3026(25)00256-X
Meletios A Dimopoulos, Meral Beksac, Ludek Pour, Sosana Delimpasi, Vladimir Vorobyev, Hang Quach, Ivan Spicka, Jakub Radocha, Paweł Robak, Kihyun Kim, Michele Cavo, Kazuhito Suzuki, Jodie Wilkes, Simon McNamara, Amy Phillips-Jones, Kristin Morris, Farrah Pompilus, Molly Purser, Neal Sule, Brandon Kremer, Angely Loubert, Laurine Bunod, Manal M'Hari, Xiaoou L Zhou, Giulia Fulci, María-Victoria Mateos, Suzanne Trudel
<p><strong>Background: </strong>In the DREAMM-8 trial, belantamab mafodotin, pomalidomide, and dexamethasone demonstrated a statistically significant reduction in the risk of progression or death compared with bortezomib, pomalidomide, and dexamethasone in lenalidomide-exposed patients with relapsed or refractory multiple myeloma. We present patient-reported outcomes from this trial.</p><p><strong>Methods: </strong>This phase 3, open-label, randomised controlled trial was done in 95 sites in 18 countries. Eligible patients were adults aged 18 years or older with relapsed or refractory multiple myeloma per International Myeloma Working Group criteria, an Eastern Cooperative Oncology Group performance status of 0-2, and previous treatment with at least one line of therapy that included lenalidomide. Patients were randomly assigned (1:1) by a central interactive response technology system to receive 28-day cycles of intravenous belantamab mafodotin (2·5 mg/kg on day 1 of cycle 1 and 1·9 mg/kg on day 1 of cycle 2 onward) combined with oral pomalidomide (4 mg on days 1 to 21) and oral dexamethasone (40 mg on days 1, 8, 15, and 22; belantamab mafodotin group) or 21-day cycles of subcutaneous bortezomib (1·3 mg/m<sup>2</sup> on days 1, 4, 8, and 11 of cycles 1-8 and days 1 and 8 of cycle 9 onward) combined with pomalidomide and dexamethasone at the same doses and schedules as the belantamab mafodotin group; bortezomib group). Treatment continued until the occurrence of progressive disease, unacceptable adverse effects, withdrawal of consent, or death (whichever occurred first). Secondary patient-reported outcome endpoints were change from baseline in health-related quality of life (HRQOL), measured by the European Organisation for Research and Treatment of Cancer (EORTC) QLQ-C30, EORTC QLQ-MY20, and Patient-Reported Outcomes Common Terminology Criteria for Adverse Events (PRO-CTCAE). Ocular Surface Disease Index and Functional Assessment of Chronic Illness Therapy-Item GP5 results were assessed similarly as exploratory endpoints. EORTC QLQ-C30 and EORTC QLQ-MY20 disease symptom domain results were analysed in the intent-to-treat population, and PRO-CTCAE results were analysed in the safety population (patients who received at least one dose of study treatment). For all patient-reported outcome assessments, proportions included the number and percentage of patients with available data, and changes from baseline were summarised as means with 95% CIs at each timepoint. This trial was registered with ClinicalTrials.gov, NCT04484623, and is ongoing.</p><p><strong>Findings: </strong>Between Oct 12, 2020, and Dec 26, 2022, 382 patients were assessed for eligibility. 80 patients were excluded and 302 patients were enrolled and randomly assigned to either the belantamab mafodotin group (n=155) or bortezomib group (n=147). The median age in the whole population was 66·1 years (SD 9·31). 181 (60%) of 302 patients were male and 260 (86%) were White. At the primary
背景:在dream -8试验中,在来那度胺暴露的复发或难治性多发性骨髓瘤患者中,与硼替佐米、泊马度胺和地塞米松相比,贝兰他单抗马福多汀、泊马度胺和地塞米松相比,在进展或死亡风险方面具有统计学显著性降低。我们介绍了该试验中患者报告的结果。方法:这项3期、开放标签、随机对照试验在18个国家的95个地点进行。符合条件的患者是18岁或以上的复发或难治性多发性骨髓瘤,符合国际骨髓瘤工作组的标准,东部肿瘤合作组织的表现状态为0-2,既往至少接受过包括来那度胺在内的一线治疗。患者由中心交互反应技术系统随机分配(1:1),接受28天周期的静脉注射贝兰他单-马福多汀(第1周期第1天2.5 mg/kg,第2周期第1天1.9 mg/kg),联合口服泊马度胺(第1至21天4 mg)和口服地塞米松(第1、8、15和22天40 mg;或21天周期的皮下硼替佐米(1 -8周期的第1、4、8和11天以及第9周期的第1和8天)联合泊马度胺和地塞米松,剂量和时间表与贝兰他单抗马福多汀组相同;bortezomib集团)。治疗持续到疾病进展、不可接受的不良反应、撤回同意或死亡(以先发生者为准)。患者报告的次要终点是健康相关生活质量(HRQOL)的基线变化,由欧洲癌症研究和治疗组织(EORTC) QLQ-C30、EORTC QLQ-MY20和患者报告的不良事件通用术语标准(PRO-CTCAE)测量。眼表疾病指数和慢性疾病治疗的功能评估项目GP5结果同样作为探索性终点进行评估。在意向治疗人群中分析EORTC QLQ-C30和EORTC QLQ-MY20疾病症状域结果,在安全人群(接受至少一剂研究治疗的患者)中分析PRO-CTCAE结果。对于所有患者报告的结果评估,比例包括可获得数据的患者的数量和百分比,从基线的变化总结为每个时间点95% ci的平均值。该试验已在ClinicalTrials.gov注册,编号NCT04484623,目前正在进行中。研究结果:在2020年10月12日至2022年12月26日期间,对382名患者进行了资格评估。80例患者被排除,302例患者被纳入,并随机分配到贝兰他单抗-马弗多汀组(n=155)或硼替佐米组(n=147)。总体年龄中位数为66.1岁(SD 9.31)。302例患者中男性181例(60%),白人260例(86%)。在初步分析数据截止日期(2024年1月29日),中位随访时间为21.8个月(IQR 13.2 - 27.6)。对于治疗期间所有患者报告的结果评估,第一年内大多数就诊的依从性至少为90%。随着时间的推移,两个治疗组的EORTC QLQ-C30和QLQ-MY20结构域评分从基线的变化保持稳定,在大多数就诊时,贝兰他单马弗多汀组患者比硼替佐米组患者出现有意义的改善(≥10分)的比例始终更高。在治疗的第一年,两组的副作用都是最小的。视力模糊是最常报道的严重或非常严重的不良事件(贝兰他单马弗多汀组146例患者中63例[43%],硼替佐米组139例患者中13例[9%]),其次是疲劳(贝兰他单马弗多汀组56例[38%],硼替佐米组49例[35%])。其他症状性不良事件组间无明显差异。解释:复发或难治性多发性骨髓瘤患者接受贝兰他单马弗多汀、泊马度胺和地塞米松或硼替佐米、泊马度胺和地塞米松治疗后HRQOL稳定。自我报告的眼部不良事件通常是可控的,并且在治疗的第一年内困扰最小。这些研究结果表明,贝兰他单抗马福多汀、泊马度胺和地塞米松耐受性良好,对HRQOL几乎没有损害,支持其用于复发或难治性多发性骨髓瘤。资金:葛兰素史克。
{"title":"Patient-reported outcomes with belantamab mafodotin, pomalidomide, and dexamethasone versus bortezomib, pomalidomide, and dexamethasone in patients with relapsed or refractory multiple myeloma (DREAMM-8): a phase 3, open-label, randomised controlled trial.","authors":"Meletios A Dimopoulos, Meral Beksac, Ludek Pour, Sosana Delimpasi, Vladimir Vorobyev, Hang Quach, Ivan Spicka, Jakub Radocha, Paweł Robak, Kihyun Kim, Michele Cavo, Kazuhito Suzuki, Jodie Wilkes, Simon McNamara, Amy Phillips-Jones, Kristin Morris, Farrah Pompilus, Molly Purser, Neal Sule, Brandon Kremer, Angely Loubert, Laurine Bunod, Manal M'Hari, Xiaoou L Zhou, Giulia Fulci, María-Victoria Mateos, Suzanne Trudel","doi":"10.1016/S2352-3026(25)00256-X","DOIUrl":"10.1016/S2352-3026(25)00256-X","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;In the DREAMM-8 trial, belantamab mafodotin, pomalidomide, and dexamethasone demonstrated a statistically significant reduction in the risk of progression or death compared with bortezomib, pomalidomide, and dexamethasone in lenalidomide-exposed patients with relapsed or refractory multiple myeloma. We present patient-reported outcomes from this trial.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;This phase 3, open-label, randomised controlled trial was done in 95 sites in 18 countries. Eligible patients were adults aged 18 years or older with relapsed or refractory multiple myeloma per International Myeloma Working Group criteria, an Eastern Cooperative Oncology Group performance status of 0-2, and previous treatment with at least one line of therapy that included lenalidomide. Patients were randomly assigned (1:1) by a central interactive response technology system to receive 28-day cycles of intravenous belantamab mafodotin (2·5 mg/kg on day 1 of cycle 1 and 1·9 mg/kg on day 1 of cycle 2 onward) combined with oral pomalidomide (4 mg on days 1 to 21) and oral dexamethasone (40 mg on days 1, 8, 15, and 22; belantamab mafodotin group) or 21-day cycles of subcutaneous bortezomib (1·3 mg/m&lt;sup&gt;2&lt;/sup&gt; on days 1, 4, 8, and 11 of cycles 1-8 and days 1 and 8 of cycle 9 onward) combined with pomalidomide and dexamethasone at the same doses and schedules as the belantamab mafodotin group; bortezomib group). Treatment continued until the occurrence of progressive disease, unacceptable adverse effects, withdrawal of consent, or death (whichever occurred first). Secondary patient-reported outcome endpoints were change from baseline in health-related quality of life (HRQOL), measured by the European Organisation for Research and Treatment of Cancer (EORTC) QLQ-C30, EORTC QLQ-MY20, and Patient-Reported Outcomes Common Terminology Criteria for Adverse Events (PRO-CTCAE). Ocular Surface Disease Index and Functional Assessment of Chronic Illness Therapy-Item GP5 results were assessed similarly as exploratory endpoints. EORTC QLQ-C30 and EORTC QLQ-MY20 disease symptom domain results were analysed in the intent-to-treat population, and PRO-CTCAE results were analysed in the safety population (patients who received at least one dose of study treatment). For all patient-reported outcome assessments, proportions included the number and percentage of patients with available data, and changes from baseline were summarised as means with 95% CIs at each timepoint. This trial was registered with ClinicalTrials.gov, NCT04484623, and is ongoing.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Findings: &lt;/strong&gt;Between Oct 12, 2020, and Dec 26, 2022, 382 patients were assessed for eligibility. 80 patients were excluded and 302 patients were enrolled and randomly assigned to either the belantamab mafodotin group (n=155) or bortezomib group (n=147). The median age in the whole population was 66·1 years (SD 9·31). 181 (60%) of 302 patients were male and 260 (86%) were White. At the primary ","PeriodicalId":48726,"journal":{"name":"Lancet Haematology","volume":"12 11","pages":"e876-e886"},"PeriodicalIF":17.7,"publicationDate":"2025-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145453813","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"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
Can ropeginterferon SURPASS resistance in essential thrombocythaemia? 在原发性血小板血症中,聚乙二醇干扰素能超越耐药性吗?
IF 17.7 1区 医学 Q1 HEMATOLOGY Pub Date : 2025-11-01 DOI: 10.1016/S2352-3026(25)00283-2
Marko Lucijanic, Ivan Krecak
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引用次数: 0
New hope, old challenges in myelodysplastic syndromes. 骨髓增生异常综合征的新希望和老挑战。
IF 17.7 1区 医学 Q1 HEMATOLOGY Pub Date : 2025-11-01 Epub Date: 2025-10-24 DOI: 10.1016/S2352-3026(25)00292-3
The Lancet Haematology
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引用次数: 0
Ropeginterferon alfa-2b in hydroxyurea-intolerant or hydroxyurea-refractory essential thrombocythaemia (SURPASS ET): a multicentre, open-label, randomised, active-controlled, phase 3 study. 羟脲不耐受或羟脲难治性原发性血小板血症(transcend ET):一项多中心、开放标签、随机、活性对照的3期研究。
IF 17.7 1区 医学 Q1 HEMATOLOGY Pub Date : 2025-11-01 DOI: 10.1016/S2352-3026(25)00264-9
Ruben Mesa, Harinder Gill, Lei Zhang, Jie Jin, Keita Kirito, Norio Komatsu, Albert Qin, Zhijian Xiao, Tsewang Tashi, Kazuya Shimoda, Kohshi Ohishi, Suning Chen, Xuelan Zuo, Shuichi Shirane, Yu Hu, Sujiang Zhang, Yi Wang, Katsuto Takenaka, Michiko Ichii, Na Xu, Lee-Yung Shih, Ken-Hong Lim, Sung-Eun Lee, Sung Hwa Bae, Winnie Z Y Teo, Dawn Maze, Stephen T Oh, Prithviraj Bose, Toshiaki Sato, Oleh Zagrijtschuk, Sheena Lin, Weichung Joe Shih, John Mascarenhas, Lucia Masarova
<p><strong>Background: </strong>The initial therapy for high-risk essential thrombocythaemia is usually hydroxyurea, but about a third of patients develop intolerance or resistance. A standard second-line agent has been anagrelide. Ropeginterferon alfa-2b, a new-generation interferon-based therapy, is approved for polycythaemia vera. We aimed to assess efficacy and safety of ropeginterferon alfa-2b compared with anagrelide in patients with essential thrombocythaemia with leukocytosis who are intolerant or resistant to hydroxyurea.</p><p><strong>Methods: </strong>The SURPASS ET open-label, randomised, active-controlled, phase 3 trial was done at 55 clinical sites across China, Japan, Taiwan, Hong Kong, South Korea, the USA, Singapore, and Canada and enrolled patients aged 18 years and older with high-risk (age >60 years with JAK2 Val617Phe or a history of disease-related thrombosis or haemorrhage), hydroxyurea-intolerant or hydroxyurea-resistant essential thrombocythaemia and white blood cell (WBC) count greater than 10 × 10<sup>9</sup> cells/L. Patients were randomly assigned (1:1) to ropeginterferon alfa-2b or anagrelide, stratified by platelet count, symptom score, and country. Ropeginterferon alfa-2b was subcutaneously dosed every 2 weeks, initially at 250 μg, then titrated to 350 μg at week 2, and to 500 μg from week 4 onward. Anagrelide was orally dosed according to the US Food and Drug Administration-approved prescribing information. The primary endpoint was the rate of response at months 9 and 12, as per modified European LeukemiaNet (ELN) criteria. The main planned analysis for the study was done in the intention-to-treat population. The trial is registered at ClinicalTrials.gov, NCT04285086 and is completed, and an extension study for collecting long-term data is ongoing.</p><p><strong>Findings: </strong>Between Aug 25, 2020, and Nov 12, 2024, 245 patients were screened and 174 were randomly assigned (91 to ropeginterferon alfa-2b and 83 to anagrelide). The median follow-up was 12·5 months (IQR 11·5-12·9). At baseline, 47 (52%) of 91 participants in the ropeginterferon alfa-2b group and 44 (53%) of 83 participants in the anagrelide group were female. 167 (96%) of 174 participants were Asian and seven (4%) were White. The trial met its primary endpoint, with 39 (43%) of 91 participants in the ropeginterferon alfa-2b group showing durable modified ELN criteria responses at months 9 and 12, compared with five (6%) of 83 participants in the anagrelide group. This difference (36·5%, 95% CI 25·4-47·7) was significant (p=0·0001). Grade 3 or worse treatment-emergent adverse events occurred in 27 (34%) of 80 patients in the anagrelide group and 21 (23%) of 91 patients in the ropeginterferon alfa-2b group. In the ropeginterferon alfa-2b group, the most common grade 3 or worse adverse events were infections and infestations, occurring eight (9%) of 91 patients, compared with five (6%) of 80 patients in the anagrelide group. In the anagrelide group,
背景:高危原发性血小板血症的初始治疗通常是羟基脲,但约三分之一的患者出现不耐受或耐药。标准的二线药物是阿纳格雷德。Ropeginterferon alfa-2b是一种新一代干扰素为基础的治疗方法,已被批准用于真性红细胞增多症。我们的目的是评估ropeg干扰素α -2b与阿纳格列特在对羟基脲不耐受或耐药的原发性血小板血症伴白细胞增多症患者中的疗效和安全性。方法:超越等非盲、随机,active-controlled, 3期临床试验是在55个临床基地在中国,日本,台湾、香港、韩国、美国、新加坡、加拿大和登记患者18岁以上高危(> 60岁JAK2 Val617Phe或疾病血栓或出血)的历史,hydroxyurea-intolerant或hydroxyurea-resistant基本thrombocythaemia和白细胞(WBC)计数大于10×109细胞/ L。根据血小板计数、症状评分和国家,将患者随机(1:1)分配到ropeginterferon α -2b或anagrelide组。Ropeginterferon α -2b每2周皮下给药,初始剂量为250 μg,第2周滴注至350 μg,第4周滴注至500 μg。阿那格列特是根据美国食品和药物管理局批准的处方信息口服的。主要终点是第9个月和第12个月的缓解率,根据修改的欧洲白血病网(ELN)标准。该研究的主要计划分析是在意向治疗人群中进行的。该试验已在ClinicalTrials.gov注册,编号NCT04285086,并已完成,一项收集长期数据的扩展研究正在进行中。结果:在2020年8月25日至2024年11月12日期间,筛选了245名患者,并随机分配了174名患者(91名接受ropeginterferon α -2b治疗,83名接受阿纳格列酯治疗)。中位随访时间为12.5个月(IQR为11.5 ~ 12.9)。基线时,ropeg干扰素α -2b组91名参与者中有47名(52%)是女性,阿纳格雷德组83名参与者中有44名(53%)是女性。174名参与者中有167名(96%)是亚洲人,7名(4%)是白人。该试验达到了主要终点,ropeg干扰素α -2b组91名参与者中有39名(43%)在第9个月和第12个月表现出持久的改良ELN标准反应,而阿纳格雷德组83名参与者中有5名(6%)。这一差异(36.5%,95% CI 25.4 - 47.7)具有显著性(p= 0.0001)。阿纳格列特组80例患者中有27例(34%)发生了3级或更严重的治疗不良事件,ropeg干扰素α -2b组91例患者中有21例(23%)发生了3级或更严重的治疗不良事件。在ropeg干扰素α -2b组中,最常见的3级或更严重的不良事件是感染和感染,91例患者中发生8例(9%),而阿纳格列德组80例患者中发生5例(6%)。在阿纳格雷德组中,最常见的3级或更严重的不良事件是神经系统紊乱,80例患者中有6例(8%)发生,而使用ropeg干扰素α -2b的91例患者中有1例(1%)发生。阿纳格雷德组80名受试者中有24名(30%)发生严重不良事件,ropeg干扰素α -2b组91名受试者中有13名(14%)发生严重不良事件。最常见的严重不良事件是脑梗死,阿纳格列特组80例患者中有4例(5%)发生脑梗死,而ropeginterferon α -2b组未出现脑梗死。两个研究组均无治疗相关死亡病例。解释:我们的研究结果表明,ropeg干扰素α -2b可被视为原发性血小板血症和白细胞增多症患者的二线治疗选择。资金:PharmaEssentia。
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引用次数: 0
Perspectives of potential haematopoietic stem-cell donors on education TikToks. 教育tiktok上潜在造血干细胞捐赠者的观点。
IF 17.7 1区 医学 Q1 HEMATOLOGY Pub Date : 2025-11-01 DOI: 10.1016/S2352-3026(25)00287-X
Yin Cathy Jian, Brady Park, Shangari Vijenthira, Warren B Fingrut
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引用次数: 0
Coexisting genetic abnormalities and thrombocytopenia. 同时存在遗传异常和血小板减少症。
IF 17.7 1区 医学 Q1 HEMATOLOGY Pub Date : 2025-11-01 DOI: 10.1016/S2352-3026(25)00255-8
Atsushi Sakamoto, Toru Uchiyama, Kazuki Tanimura, Hiroyuki Fujisaki, Akihiro Iguchi, Tadashi Kaname, Akira Ishiguro
{"title":"Coexisting genetic abnormalities and thrombocytopenia.","authors":"Atsushi Sakamoto, Toru Uchiyama, Kazuki Tanimura, Hiroyuki Fujisaki, Akihiro Iguchi, Tadashi Kaname, Akira Ishiguro","doi":"10.1016/S2352-3026(25)00255-8","DOIUrl":"https://doi.org/10.1016/S2352-3026(25)00255-8","url":null,"abstract":"","PeriodicalId":48726,"journal":{"name":"Lancet Haematology","volume":"12 11","pages":"e926"},"PeriodicalIF":17.7,"publicationDate":"2025-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145453762","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"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
期刊
Lancet Haematology
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