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更新于：2025-05-30

Anagrelide Hydrochloride

盐酸阿那格雷

更新于：2025-05-30

概要

基本信息

药物类型

小分子化药

别名

anagrelide、Anagrelide Hydrochloride Hydrate、Anagrelide hydrochloride monohydrate

+ [17]

靶点

PDE3

作用方式

抑制剂

作用机制

PDE3抑制剂(磷酸二酯酶3抑制剂)

治疗领域

血液及淋巴系统疾病其他疾病肿瘤+ [2]

在研适应症

原发性血小板增多症血小板增多症胃肠道间质瘤

非在研适应症-

原研机构

Shire Pharmaceuticals Services Ltd.

在研机构

Takeda Pharmaceuticals U.S.A., Inc.Viatris Ltd. (New Zealand)

Takeda Pharmaceutical Co., Ltd.

+ [2]

非在研机构

Aop Orphan Pharmaceuticals GmbHMylan Pharmaceuticals, Inc.

Shire Plc

权益机构-

最高研发阶段批准上市

首次获批日期

美国 (1997-03-14),

血小板增多症

最高研发阶段(中国)-

特殊审评孤儿药 (美国)

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结构/序列

分子式C10H10Cl3N3O2

InChIKeyYLFXXKJQBOJJIX-UHFFFAOYSA-N

CAS号823178-43-4

关联

项与盐酸阿那格雷相关的临床试验

IRCT20210519051345N60

/ SuspendedN/AIIT

In vivo bioequivalence study of Anagrelide 0.5 mg capsule manufactured by Pars Darou Co. compared with innovator product

开始日期2024-07-05

申办/合作机构

Tabriz University of Medical Sciences

IRCT20130313012810N30

/ RecruitingN/A

Bioequivalence study of Anagrelide 0.5 mg capsule manufactured by Modava Pharmaceutical Company

开始日期2024-06-24

申办/合作机构-

IRCT20210519051345N27

/ RecruitingN/AIIT

Bioequivalence study of Anagrelide 0.5mg capsule manufactured by Ronak Co. compared with innovator product

开始日期2023-03-01

申办/合作机构

Tabriz University of Medical Sciences

100 项与盐酸阿那格雷相关的临床结果

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100 项与盐酸阿那格雷相关的转化医学

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100 项与盐酸阿那格雷相关的专利（医药）

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640

项与盐酸阿那格雷相关的文献（医药）

2025-12-31·DRUG DELIVERY

Pharmacokinetic profile and in vivo anticancer efficacy of anagrelide administered subcutaneously in rodents

Article

作者: Schöffski, Patrick ; Salmikangas, Sami ; De Sutter, Luna ; Merikoski, Nanna ; Wozniak, Agnieszka ; Sihto, Harri ; Duan, Jianmin ; Maksimow, Mikael ; Wyns, Karo ; Böhling, Tom ; Lahtinen, Maria ; Toivanen, Kirsi

Anagrelide (ANA) is a phosphodiesterase 3A (PDE3A) inhibitor, commonly prescribed for essential thrombocythemia. It also functions as a molecular glue, inducing complex formation between PDE3A and Schlafen 12. This association either triggers apoptosis or inhibits proliferation in tumor cells, supporting its use in cancer therapy. Conventionally administered orally, ANA undergoes rapid metabolism and elimination, resulting in a short drug exposure time at the site of action. Here, we explored the pharmacokinetic profile of a subcutaneously (SC) injected ANA formulation in Sprague-Dawley rats by quantifying plasma ANA and metabolite concentrations using liquid-chromatography-tandem mass spectrometry. We further evaluated the in vivo tumor regression efficacy of orally and SC administered ANA in a patient-derived gastrointestinal stromal xenograft mouse model - UZLX-GIST2B - characterized by a KIT exon 9 driver mutation. The SC ANA exhibited extended-release plasma concentration-time profiles compared to intravenous and oral administrations. After a single administration in rats, plasma concentrations of ANA were detected up to 56 days later, and ANA metabolites up to 30 days later. The SC formulation also significantly reduced tumor volumes and demonstrated dose-dependent histological responses, nearly eradicating tumor tissue in 11 days with the highest dose. These findings suggest that the SC slow-release formulation maintains stable drug concentrations during treatment, potentially improving therapeutic efficacy at the target site.

2025-04-01·Hamostaseologie

Thrombosis at Unusual Sites: Focus on Myeloproliferative Neoplasms and Paroxysmal Nocturnal Hemoglobinuria

Review

作者: Panse, Jens ; Koschmieder, Steffen

Abstract:

Patients with thrombosis at an unusual site will need to be explored for rare causes of thrombosis. Two of these rare causes include myeloproliferative neoplasms (MPNs) and paroxysmal nocturnal hemoglobinuria (PNH). It is important not to overlook these causes, since they require specific management, in addition to antithrombotic treatment (anticoagulants, antiplatelet agents). Unusual sites of venous thrombosis include upper extremity veins, splanchnic veins, cerebral veins, and retinal veins, and unusual sites of arterial thrombosis include renal, adrenal, splenic and mesenteric arteries, and intracardiac and aortal locations. Suspicion for MPN and PNH should be raised if there are concomitant abnormalities, such as elevated or decreased blood cell counts or splenomegaly. Diagnosis of MPN and PNH should include JAK2V617F mutational screening as well as flow cytometric assessment of GPI-anchored proteins in the peripheral blood, respectively. Specific treatments for MPN may include phlebotomy or cytoreductive drugs such as hydroxyurea, anagrelide, pegylated interferon-alpha, or Janus kinase inhibitors. Drugs used for PNH treatment include terminal complement inhibitors, such as eculizumab and ravulizumab, as well as proximally acting inhibitors such as pegcetacoplan or iptacopan. Patients with MPN and PNH are at high risk for thrombosis during their entire lifetime and should thus be followed by specialists experienced in the care of these diseases.

2025-02-15·Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics

[Advances in the diagnosis and treatment of thrombocytosis in children].

Review

作者: Xu, Yu-Ting ; Hu, Qun

Thrombocytosis is a common condition in children, classified into primary and secondary types. Secondary thrombocytosis is mainly caused by factors such as infection, anemia, iron deficiency, trauma, or surgical intervention, and it typically occurs without severe thrombosis or bleeding events. Platelet counts can return to normal after control of the primary factors, with favorable clinical outcomes. Primary thrombocytosis is mainly caused by myeloproliferative neoplasms such as polycythemia vera, essential thrombocythemia, and myelofibrosis, often accompanied by gene mutations in hematopoietic cells. In children, clinical manifestations are atypical compared to adults, with few thromboembolic or bleeding events. No special treatment is required for patients who are asymptomatic or have mild symptoms, and it is recommended to regularly monitor platelet counts. Antiplatelet therapy with aspirin can be considered for patients at risk of thrombosis or those with extreme thrombocytosis, and cytoreductive therapy can be performed when necessary, but the toxicities and side effects of drugs should be closely monitored. At present, hydroxyurea, interferon-alpha, and anagrelide are commonly used for cytoreductive therapy. This article provides an overview of the etiology, classification, clinical manifestations, diagnosis, and treatment of childhood thrombocytosis to guide healthcare professionals in treatment decisions.

项与盐酸阿那格雷相关的新闻（医药）

2025-05-20

·Business Wire

PharmaEssentia to Present Phase 3 SURPASS-ET Data in Oral Session at 2025 ASCO Annual Meeting

BURLINGTON, Mass.--(BUSINESS WIRE)--PharmaEssentia USA Corporation, a subsidiary of PharmaEssentia Corporation (TWSE: 6446), a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, today announced it will present results from the Phase 3 SURPASS-ET clinical trial in an oral session at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting, taking place May 30-June 3 in Chicago. SURPASS-ET (NCT04285086) is evaluating ropeginterferon alfa-2b-njft as a second-line treatment for patients with essential thrombocythemia (ET). Earlier this year, PharmaEssentia announced positive topline Phase 3 results, with ropeginterferon alfa-2b-njft demonstrating a significantly higher durable clinical response rate compared to anagrelide (42.9% vs. 6.0%; p=0.0001), along with a favorable safety profile and a greater reduction in JAK2 V617F allelic burden over 12 months. Ropeginterferon alfa-2b-njft is currently FDA-approved and marketed as BESREMi® for the treatment of adults with polycythemia vera (PV). BESREMi® has been recognized by the National Comprehensive Cancer Network® (NCCN®) as a preferred first-line cytoreductive therapy for adults with symptomatic, low-risk PV and the only preferred therapeutic option for both high-risk and low-risk (symptomatic) patients, regardless of treatment history. “Current options for patients with ET are limited. Standard therapies like hydroxyurea have notable drawbacks and do not target the underlying biology of the disease, while anagrelide has been associated with toxicity concerns and limited efficacy,” said Ruben Mesa, M.D., co-principal investigator, presenting author, and President of Atrium Health Levine Cancer Institute, the largest cancer program in the Carolinas which includes the Comprehensive Cancer Center at Wake Forest Baptist. “This marks the first registrational Phase 3 trial of a long-acting interferon in ET, demonstrating not only well-tolerated blood count control but also a measurable reduction in JAK2 mutation allele burden. These findings support further investigation of ropeginterferon as a second-line option for patients with ET who are seeking additional treatment approaches.” “The ASCO meeting is an important opportunity to share detailed findings of our positive data from the SURPASS-ET study with the medical community,” said Albert Qin, M.D., Ph.D., Chief Medical Officer of PharmaEssentia USA. “These data highlight a significant advance in the treatment of essential thrombocythemia and reinforce our commitment to delivering innovative, non-chemotherapy options for patients living with myeloproliferative neoplasms.“ Presentation Details Title: Ropeginterferon alfa-2b versus anagrelide for the treatment of essential thrombocythemia: Topline results of the phase 3 SURPASS-ET trial Abstract Number: 6500 Presenter: Dr. Ruben Mesa Session: Hematologic Malignancies — Leukemia, Myelodysplastic Syndromes, and Allotransplant Date: Monday, June 2, 2025 Time: 3:00 p.m. - 6:00 p.m. CDT About Essential Thrombocythemia Essential thrombocythemia is a chronic, rare blood disorder that is the most common type of myeloproliferative neoplasm. Essential thrombocythemia is most often caused by genetic mutations that cause the bone marrow to produce too many platelets, which can obstruct blood flow and cause a stroke, heart attack or pulmonary embolism. About BESREMi® (ropeginterferon alfa-2b-njft) in polycythemia vera (PV) BESREMi is an innovative monopegylated, long-acting interferon. With its unique pegylation technology, BESREMi has a long duration of activity in the body and is aimed to be administered once every two weeks (or every four weeks with hematological stability for at least one year), allowing flexible dosing that helps meet the individual needs of patients. BESREMi has orphan drug designation for the treatment of polycythemia vera (PV) in adults in the United States. BESREMi has been approved in more than 40 countries, with approval from the European Medicines Agency (EMA) in 2019, by the US Food and Drug Administration (FDA) in 2021, and by the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan in 2023. It was invented by PharmaEssentia and is manufactured in the company’s Taichung plant, which was cGMP certified by TFDA in 2017 and by EMA in January 2018. PharmaEssentia retains full global intellectual property rights for the product in all indications. BESREMi was approved with a boxed warning for risk of serious disorders including aggravation of neuropsychiatric, autoimmune, ischemic and infectious disorders. Please see full Prescribing Information, including Boxed Warning. About PharmaEssentia PharmaEssentia USA Corporation, located in Burlington, Massachusetts, is a subsidiary of PharmaEssentia Corporation (TWSE: 6446). PharmaEssentia Corporation headquartered in Taipei, Taiwan, is a global and rapidly growing biopharmaceutical innovator. Leveraging deep expertise and proven scientific principles, PharmaEssentia aims to deliver effective new biologics for challenging diseases in the areas of hematology, oncology, and immunology with one approved product and a diversifying pipeline. Founded in 2003 by a team of Taiwanese-American executives and renowned scientists from U.S. biotechnology and pharmaceutical companies, today PharmaEssentia is expanding its global presence with operations in the U.S., Japan, China, and Korea, along with a world-class biologics production facility in Taichung, Taiwan. For more information about PharmaEssentia USA, visit the website, LinkedIn or X (formerly Twitter).

上市批准临床3期临床结果孤儿药ASCO会议

2025-05-16

·ioncology

2025 EHA大会摘要公布！30项中国研究入选口头报告，于“时尚之都”米兰奏响中国最强音

编者按第30届欧洲血液学会年会（EHA 2025）将于6月12日至15日在意大利米兰盛大召开。作为全球血液学领域的顶尖学术盛会之一，本届大会将全面聚焦血液学及其各亚专科的最新进展，深入探讨循证诊疗新方法，发布临床与转化研究的重磅成果，呈现创新技术、精准诊断工具与风险评估策略的最新突破。日前，大会官网正式公布了本次年会的入选摘要（不含LBA）。《肿瘤瞭望-血液时讯》第一时间汇总整理了中国学者入选的口头报告名单（如有遗漏，欢迎留言补充），让我们一同见证东方智慧在“时尚之都”米兰的精彩绽放。全体大会摘要（Plenary Abstract Session）01摘要号：S102英文标题：BETTER SAFETY AND EFFICACY WITH ROPEGINTERFERON ALFA-2B OVER ANAGRELIDE AS SECOND-LINE TREATMENT OF ESSENTIAL THROMBOCYTHEMIA IN THE TOPLINE RESULTS OF THE RANDOMIZED PHASE 3 SURPASS-ET TRIAL中文标题：随机Ⅲ期SURPASS-ET试验的初步结果显示，罗培干扰素α-2b相较阿那格雷二线治疗原发性血小板增多症的安全性和疗效更佳类型：口头报告专场名称：全体大会摘要汇报者及单位：Harry Gill，中国香港大学李嘉诚医学院口头报告（Oral Presentations）急性髓系白血病——生物学与转化研究Acute myeloid leukemia - Biology & translational research01摘要号：S134英文标题：DE NOVO DESIGN OF A CHROMATIN READER ZMYND8-TARGETING PEPTIDE PROTAC FOR ACUTE MYELOID LEUKEMIA THERAPY中文标题：靶向染色质识别蛋白ZMYND8的肽类PROTAC的重新设计：用于急性髓系白血病的治疗类型：口头报告专场名称：AML发病机制中的染色质和基因调控(Chromatin and gene regulation in AML pathogenesis)汇报者及单位：Hui Feng，西安交通大学第一附属医院02摘要号：S125英文标题：CELL-INTRINSIC INFLAMMATION SHAPES CELL-STATE HETEROGENEITY AND DRIVES THERAPEUTIC RESISTANCE IN HUMAN ACUTE MYELOID LEUKEMIA中文标题：细胞内在炎症塑造人类急性髓系白血病细胞状态异质性并驱动治疗耐药性类型：口头报告专场名称：AML免疫治疗的新进展（Novel advances in AML immunotherapy）汇报者及单位：Lina Liu，浙江大学医学院附属第一医院骨髓移植中心急性髓系白血病——临床Acute myeloid leukemia - Clinical01摘要号：S143英文标题：LONG-TERM FOLLOW-UP OF PREDOMINANTLY ASIAN PATIENTS WITH RELAPSED/REFRACTORY FLT3-MUTATED ACUTE MYELOID LEUKEMIA TREATED WITH GILTERITINIB VERSUS SALVAGE CHEMOTHERAPY IN THE PHASE 3 COMMODORE TRIAL中文标题：Ⅲ期COMMODRE试验中接受吉瑞替尼与挽救化疗治疗的复发/难治性FLT3突变急性髓系白血病患者（以亚洲患者为主）的长期随访类型：口头报告专场名称：AML的新疗法与FLT3抑制剂（Novel therapies and FLT3 inhibition for AML）汇报者及单位：王建祥，中国医学科学院血液病医院（中国医学科学院血液学研究所）侵袭性非霍奇金淋巴瘤——临床Aggressive Non-Hodgkin lymphoma - Clinical01摘要号：S246英文标题：A PIVOTAL STUDY OF SHR2554, AN ORAL INHIBITOR AGAINST ENHANCER OF ZESTE HOMOLOG 2 (EZH2), IN RELAPSED OR REFRACTORY (R/R) PERIPHERAL T-CELL LYMPHOMA (PTCL)中文标题：SHR2554[一种口服的增强子同源物2（EZH2）抑制剂）]用于复发或难治性（R/R）外周T细胞淋巴瘤（PTCL）的治疗：一项关键研究类型：口头报告专场名称：侵袭性非霍奇金淋巴瘤——临床（前瞻性）[Aggressive Non-Hodgkin lymphoma - Clinical (Prospective)]汇报者及单位：宋玉琴，北京大学肿瘤医院出血性疾病（先天性和获得性）Bleeding disorders (congenital and acquired)01摘要号：S315英文标题：BLASTS- AND MODIFIED EASIX-GUIDED RISK STRATIFICATION FOR CAR-T-ASSOCIATED COAGULOPATHY AND OUTCOMES中文标题：BLASTS和改良EASIX指导的CAR-T相关凝血病及预后的风险分层类型：口头报告专场名称：出血性疾病（先天性和获得性）[Bleeding disorders (congenital and acquired)]汇报者及单位：Yingying Li，华中科技大学同济医学院附属协和医院骨髓衰竭综合征（包括PNH）——临床Bone marrow failure syndromes incl.PNH-Clinical01摘要号：S184英文标题：EFFICACY AND SAFETY OF CYCLOSPORINE PLUS LUSPATERCEPT VERSUS CYCLOSPORINE IN NEWLY DIAGNOSED NON-TRANSFUSION-DEPENDENT NON-SEVERE APLASTIC ANEMIA: A PROSPECTIVE RANDOMIZED TRIAL中文标题：环孢素联合罗特西普 vs. 环孢素治疗新诊断非输血依赖型非重型再生障碍性贫血的疗效和安全性：一项前瞻性随机试验类型：口头报告专场名称：骨髓衰竭综合征(Bone marrow failure syndromes)汇报者及单位：Zhuxin Zhang，中国医学科学院北京协和医院慢性髓系白血病——生物学及转化研究Chronic myeloid leukemia-Biology & translational research01摘要号：S162英文标题：INTEGRATING GENOMIC AND TRANSCRIPTOMIC INSIGHTS FOR PREDICTING RESPONSES AND OUTCOMES IN PATIENTS WITH CML RECEIVING 3RD-GENERATION TKI THERAPY中文标题：整合基因组与转录组见解以预测接受第三代TKI治疗的CML患者的反应和预后类型：口头报告专场名称：CML治疗反应与耐药的生物学(Biology of CML treatment response and resistance)汇报者及单位：张小帅，北京大学人民医院基因治疗、细胞免疫治疗和疫苗接种——生物学和转化研究Gene therapy,cellular immunotherapy and vaccination-Biology & translational research01摘要号：S277英文标题：TYPE I INTERFERON IMPAIRS CAR-T CELL FUNCTION BY DISRUPTING IMMUNE SYNAPSE ASSEMBLY中文标题：I型干扰素通过破坏免疫突触组装削弱CAR-T细胞功能类型：口头报告专场名称：基因治疗、细胞免疫治疗和疫苗接种——生物学和转化研究（Gene therapy, cellular immunotherapy and vaccination - Biology & translational research）汇报者及单位：Meijuan Huang，华中科技大学同济医学院附属同济医院基因治疗、细胞免疫治疗和疫苗接种——临床Gene therapy,cellular immunotherapy and vaccination-Clinical01摘要号：S283英文标题：CTD402: A UNIVERSAL ANTI-CD7 CHIMERIC ANTIGEN RECEPTOR T-CELL THERAPY FOR PATIENTS WITH RELAPSED OR REFRACTORY (R/R) T-CELL ACUTE LYMPHOBLASTIC LEUKEMIA/LYMPHOBLASTIC LYMPHOMA (T-ALL/LBL)中文标题：CTD402：一种针对复发或难治性（R/R）T细胞急性淋巴细胞白血病/淋巴母细胞淋巴瘤（T-ALL/LBL）患者的通用抗CD7嵌合抗原受体T细胞疗法类型：口头报告专场名称：基因治疗、细胞免疫治疗和疫苗接种——临床（Gene therapy, cellular immunotherapy and vaccination - Clinical）汇报者及单位：Jiangtao Ren，Bioheng Therapeutics Co., Ltd,, Nanjing, China造血、干细胞和微环境Hematopoiesis,stem cells and microenvironment01摘要号：S272英文标题：ENHANCE THE THERAPEUTIC EFFICACY OF HUMAN UMBILICAL CORD-DERIVED MESENCHYMAL STEM CELLS IN PREVENTION OF ACUTE GRAFT-VERSUS-HOST DISEASE THROUGH CRISPLD2 MODULATION中文标题：通过调控CRISPLD2增强人脐带间充质干细胞在预防急性移植物抗宿主病中的治疗效果类型：口头报告专场名称：造血干细胞及其微环境(Hematopoietic stem cells and their niche)汇报者及单位：Xu Qing，陆军军医大学第二附属医院（新桥医院）02摘要号：S273英文标题：TGF-ß1-TRIGGERED MALADAPTIVE BONE MARROW ENDOTHELIUM IMPEDES HEMATOPOIETIC RECOVERY中文标题：TGF-β1诱导的骨髓内皮功能失调阻碍造血恢复类型：口头报告专场名称：造血干细胞及其微环境(Hematopoietic stem cells and their niche)汇报者及单位：Zhong-Shi Lyu，北京大学人民医院淋巴瘤生物学与转化研究Lymphoma biology & translational research01摘要号：S251英文标题：MULTI-OMICS DISSECTION OF EBV-DRIVEN HETEROGENEITY, METABOLIC REMODELING, AND TUMOR CELLULAR LANDSCAPE IN ENKTL PROGRESSION中文标题：EB病毒驱动的ENKTL进展中的异质性、代谢重塑及肿瘤细胞图谱的多组学解析类型：口头报告专场名称：淋巴瘤生物学与转化研究(Lymphoma biology & translational research)汇报者及单位：梁金花，南京医科大学第一附属医院（江苏省人民医院）02摘要号：S252英文标题：PTPN2 INHIBITION DISRUPTS MITOCHONDRIAL RENEWAL AND BLOCKS TFRC-MEDIATED PINK1-PRKN-DEPENDENT MITOPHAGY TO EXERT ANTI-TUMOR ACTIVITIES IN ALK-POSITIVE ANAPLASTIC LARGE CELL LYMPHOMA中文标题：PTPN2抑制通过破坏线粒体更新并阻断TFRC介导的PINK1-PRKN依赖性线粒体自噬，在ALK阳性间变性大细胞淋巴瘤中发挥抗肿瘤作用类型：口头报告专场名称：淋巴瘤生物学与转化研究(Lymphoma biology & translational research)汇报者及单位：梁金花，南京医科大学第一附属医院（江苏省人民医院）骨髓增生异常综合征——生物学与转化研究Myelodysplastic syndromes-Biology & translational research01摘要号：S170英文标题：SINGLE-CELL ATLAS OF PEDIATRIC NRAS-MUTANT MYELOID NEOPLASMS REVEALS MYELOID LINEAGE BIAS AND IDENTIFIES POTENTIAL THERAPEUTIC TARGETS中文标题：儿童NRAS突变髓系肿瘤的单细胞图谱揭示髓系谱系偏倚并识别潜在治疗靶点类型：口头报告专场名称：MDS cellular and molecular therapeutic targeting汇报者及单位：Suyu Zong，中国医学科学院血液病医院（中国医学科学院血液学研究所）骨髓瘤及其他单克隆免疫球蛋白病——生物学与转化研究Myeloma and other monoclonal gammopathies -Biology & translational research01摘要号：S188英文标题：ENHANCER EXTRACHROMOSOMAL CIRCULAR DNA ANKRD28 DRIVES DRUG RESISTANCE VIA POU2F2-MEDIATED TRANSCRIPTIONAL REWIRING IN MULTIPLE MYELOMA中文标题：增强子染色体外环状DNA ANKRD28通过POU2F2介导的转录重编程驱动多发性骨髓瘤的药物耐药类型：口头报告专场名称：骨髓瘤及其他单克隆免疫球蛋白病——生物学与转化研究（Myeloma and other monoclonal gammopathies - Biology & translational research）汇报者及单位：Binzhen Chen，上海交通大学医学院附属仁济医院骨髓增生性肿瘤——临床Myeloproliferative neoplasms-Clinical01摘要号：S222英文标题：ROPEGINTERFERON ALFA-2B FOR PRE-FIBROTIC PRIMARY MYELOFIBROSIS AND DIPSS LOW/INTERMEDIATE-RISK MYELOFIBROSIS中文标题：罗培干扰素α-2b治疗纤维化前原发性骨髓纤维化及DIPSS低/中风险骨髓纤维化类型：口头报告专场名称：Innovative treatment approaches in MPN汇报者及单位：Harry Gill，中国香港大学李嘉诚医学院血液学中的新技术、新方法及数字分析工具Novel technologies,techniques and digital analytical tools in hematology01摘要号：S332英文标题：STEM CELL AGING CLOCK: A DEEP LEARNING DRIVEN FRAMEWORK FOR PREDICTING STEM CELL AGE中文标题：干细胞衰老时钟：基于深度学习的干细胞年龄预测框架类型：口头报告专场名称：计算机与人工智能辅助血液学的新进展（New developments in computational and AI-assisted hematology）汇报者及单位：Guanlin Wang，复旦大学代谢与整合生物学研究院血小板疾病Platelet disorders01摘要号：S313英文标题：BARICITINIB CORRECTS AP2-NAK MEDIATED C-MPL ENDOCYTIC DYSREGULATION TO RESTORE MEGAKARYOCYTES TPO-RA SENSITIVITY IN IMMUNE THROMBOCYTOPENIA中文标题：巴瑞替尼纠正AP2-NAK介导的c-MPL内吞调控异常，恢复免疫性血小板减少症中巨核细胞对TPO-RA的敏感性类型：口头报告专场名称：血小板疾病（Platelet disorders）汇报者及单位：安倬玉，北京大学人民医院02摘要号：S318英文标题：TARGETED SUPPRESSION OF EFFECTOR T CELLS BY GPIBα CAAR-TREG CELLS IN REFRACTORY/RELAPSED IMMUNE THROMBOCYTOPENIA中文标题：GPIBα CAAR-Treg细胞对难治/复发性免疫性血小板减少症效应T细胞的靶向抑制类型：口头报告专场名称：出血性疾病（先天性与获得性）[Bleeding disorders (congenital and acquired)]汇报者及单位：Jinhui Shu，华中科技大学同济医学院附属协和医院干细胞移植——临床Stem cell transplantation-Clinical01摘要号：S261英文标题：AUTONOMOUS AI-DRIVEN INTENSIFIED PROPHYLAXIS OF SEVERE ACUTE GRAFT-VERSUS-HOST DISEASE IN HAPLOIDENTICAL TRANSPLANTS: A SINGLE-CENTRE, PROOF-OF-CONCEPT, PHASE 2 TRIAL中文标题：自主人工智能驱动的加强预防严重急性移植物抗宿主病在半相合移植中的应用：一项单中心概念验证Ⅱ期试验类型：口头报告专场名称：干细胞移植——专场2(Stem cell transplantation - Session 2)汇报者及单位：Yigeng Cao，中国医学科学院血液病医院（中国医学科学院血液学研究所）02摘要号：S262英文标题：ANTI-CD25 ANTIBODY AS AN ALTERNATIVE TO METHOTREXATE FOR GRAFT-VERSUS-HOST DISEASE PROPHYLAXIS RESULTED IN BETTER OUTCOMES IN PATIENT OF CHIMERIC ANTIGEN RECEPTOR-T CELL THERAPY BRIDGING TO TRANSPLANT中文标题：抗CD25抗体作为替代甲氨蝶呤的移植物抗宿主病预防方案，在嵌合抗原受体T细胞治疗桥接移植患者中取得了更好的疗效类型：口头报告专场名称：干细胞移植——专场2（Stem cell transplantation - Session 2）汇报者及单位：李智慧，北京高博博仁医院03摘要号：S263英文标题：PHARMACOKINETIC-GUIDED ATG DOSING OPTIMIZES OUTCOMES IN MYELOABLATIVE HAPLO-CORD HCT中文标题：基于药代动力学指导的ATG给药优化改善了清髓性半相合脐带血干细胞移植的治疗效果类型：口头报告专场名称：干细胞移植——专场2（Stem cell transplantation - Session 2）汇报者及单位：Qingya cui,苏州大学附属第一医院04摘要号：S256英文标题：TARGETED DOSING OF ANTI-THYMOCYTE GLOBULIN VERSUS FIXED DOSING STRATEGY IN PATIENTS UNDERGOING UNMANIPULATED HAPLOIDENTICAL HAEMATOPOIETIC STEM-CELL TRANSPLANTATION: A RANDOMIZED, MULTICENTER, PHASE 3 CLINICAL TRIAL中文标题：抗胸腺细胞球蛋白的目标给药与固定给药策略在接受未处理半相合造血干细胞移植患者中的比较：一项随机、多中心、Ⅲ期临床试验类型：口头报告专场名称：干细胞移植——专场1（Stem cell transplantation - Session 1）汇报者及单位：刘代红，解放军总医院第五医学中心05摘要号：S258英文标题：WHO IS THE BEST ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANT DONOR FOR SEVERE APLASTIC ANEMIA? A MULTICENTER RETROSPECTIVE EXPERIENCE FROM CHINA中文标题：重型再生障碍性贫血的最佳异基因造血干细胞移植供者是谁？来自中国的多中心回顾性研究经验类型：口头报告专场名称：干细胞移植——专场1（Stem cell transplantation - Session 1）汇报者及单位：Xiao-Di Ma，北京大学人民医院06摘要号：S259英文标题：COMPARABLE SURVIVAL OUTCOMES IN HLA-MATCHED AND HAPLOIDENTICAL HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR SEVERE APLASTIC ANEMIA PATIENTS AGED 40-50: A CBMTR REGISTRY-BASED PSM ANALYSIS OVER THE LAST DECADE中文标题：接受HLA匹配与半相合造血干细胞移植40-50岁重型再生障碍性贫血患者的生存结局相当：过去十年基于CBMTR登记的PSM分析类型：口头报告专场名称：干细胞移植——专场1（Stem cell transplantation - Session 1）汇报者及单位：许兰平，北京大学人民医院干细胞移植——实验Stem cell transplantation-Experimental01摘要号：S255英文标题：JAK/ROCK INHIBITION WITH ROVADICITINIB SUPPRESSES MURINE AND HUMAN ACUTE GRAFT-VERSUS-HOST DISEASE: THE RESULTS OF PRECLINICAL AND PHASE 1B STUDY中文标题：JAK/ROCK抑制剂罗伐昔替尼抑制小鼠及人类急性移植物抗宿主病：临床前及Ⅰb期研究结果类型：口头报告专场名称：干细胞移植——专场1（Stem cell transplantation - Session 1）汇报者及单位：赵妍敏，浙江大学医学院附属第一医院骨髓移植中心地中海贫血Thalassemias01摘要号：S290英文标题：EFFICACY AND SAFETY OF BRL-101 IN TDT AND SCD中文标题：BRL-101 在输血依赖型地中海贫血（TDT）和镰状细胞病（SCD）中的疗效与安全性类型：口头报告专场名称：地中海贫血和红细胞生成（Thalassemia and erythopoiesis）汇报者及单位：Tan Qian，Shanghai tech immunology department, Shanghai, China02摘要号：S291英文标题：TREATMENT OF PATIENTS WITH SEVERE TRANSFUSION-DEPENDENT ß-THALASSEMIA WITH CS-101, AN AUTOLOGOUS, EX VIVO EDITED, CD34+ HEMATOPOIETIC STEM CELL PRODUCT USING INNOVATIVE TRANSFORMER BASE EDITOR (TBE)中文标题：采用创新型Transformer基础编辑器（TBE）对自体CD34+造血干细胞进行体外编辑制备的CS-101，用于治疗重型输血依赖型β-地中海贫血患者类型：口头报告专场名称：地中海贫血和红细胞生成（Thalassemia and erythopoiesis）汇报者及单位：陈佳，上海科技大学生命科学与技术学院基因编辑中心03摘要号：S292英文标题：FIRST-IN-HUMAN STUDY OF AUTOLOGOUS HBA2-EDITED CD34+ HEMATOPOIETIC STEM AND PROGENITOR CELLS IN ALPHA-THALASSEMIA WITH CONSTANT SPRING MUTATION中文标题：首次人体研究：自体HBA2基因编辑的CD34+造血干/祖细胞用于Constant Spring突变型α-地中海贫血治疗类型：口头报告专场名称：地中海贫血和红细胞生成（Thalassemia and erythopoiesis）汇报者及单位：Hui Xu，Reforgene Medicine, Guangzhou, China（来源：《肿瘤瞭望–血液时讯》编辑部）声明凡署名原创的文章版权属《肿瘤瞭望》所有，欢迎分享、转载。本文仅供医疗卫生专业人士了解最新医药资讯参考使用，不代表本平台观点。该等信息不能以任何方式取代专业的医疗指导，也不应被视为诊疗建议，如果该信息被用于资讯以外的目的，本站及作者不承担相关责任。

蛋白降解靶向嵌合体临床结果

2025-04-25

·ioncology

常春康教授：2025 CSCO恶性血液病指南之MDS和MPN更新解读丨2025 CSCO指南会

点击蓝字关注我们编者按：2025年4月18日-19日，由中国临床肿瘤学会（CSCO）和北京市希思科临床肿瘤学研究基金会联合主办的2025 CSCO指南会在济南山东大厦隆重召开。在本次大会上，上海交通大学附属第六人民医院常春康教授，围绕2025 CSCO恶性血液病指南中关于骨髓增生异常综合征（MDS）和骨髓增殖性肿瘤（MPN）[包括真性红细胞增多症（PV）、原发性血小板增多症（ET）和原发性骨髓纤维化（PMF）]部分的更新内容进行了精彩解读。《肿瘤瞭望-血液时讯》特整理课件内容，以飨读者。骨髓增生异常综合征（MDS）01治疗前评估MDS中染色体异常及其比例a注释更新:a.WHO和ICC都建议形态学未达到发育不良标准，只伴有以上细胞遗传学异常不能作为诊断MDS的确切证据，如果同时伴有持续性血细胞减少，只能考虑拟诊MDS，处理方式与CHIP的突变相同。b.IPSS和IPSS-R分组依赖于细胞遗传学结果，如果不能检测常规核型分析或无可供分析的中期分裂相，最低推荐FISH探针需包括的组合。02惰性髓系造血疾病谱MDS诊断的确立需要鉴别和除外下列因不满足MDS最低诊断标准而衍生的包括意义未明的特发性血细胞减少(ICUS)、意义未明的特发性发育异常(IDUS)、潜质未定的克隆性造血(CHIP)及意义不明的克隆性细胞减少症(CCUS)在内的可能发展为MDS的前驱疾病惰性髓系造血疾病。注释新增:d.因WHO（2022）第五版中将WHO（2016）分类定义的具有MDS定义的细胞遗传学无细胞发育不良异常MDS-U患者的归类为CCUS，但除外其中del（5q）、多重打击TP53突变或-7/del（7q）或复杂核型缺乏发育异常或过量原始细胞的病例被分类为MDS-5q、MDS-BiTP53。故目前的CCUS克隆性特征，要么存在既往的髓系相关体细胞突变（存在于>2%的变异等位基因频率[VAF]），要么具有上述克隆核型异常（存在于≥2个中期分裂相）。03治疗基于PACE、MEDALIST、COMMANDS和MDS-004四项关键研究，罗特西普在治疗低危MDS（LR-MDS）方面实现突破，从二线治疗提升至一线起始治疗。基于艾伏尼布治疗IDH1突变复发/难治性MDS患者的1期子研究结果，艾伏尼布被纳入新版指南推荐。k 维奈克拉是否加入基于患者的状态和耐受性。注释更新：(5) 伊美司他：一种用于静脉注射的13个碱基的寡核苷酸端粒酶抑制剂，起始剂量 7.1mg/kg 静脉注射，上市研究IMerge研究中可能频繁发生短暂性血小板减少和中性粒细胞减少，因此可能导致剂量减少。起始血小板和中性粒细胞水平应为≥75×109/L和≥1.5×109/L。2024年6月6日美国食品药品监督管理局（FDA）批准上市，目前国内尚未上市。（9）小分子靶向药物：一些新数据显示了维奈克拉和IDH1/2抑制剂对患有HMA难治性疾病的高危MDS患者的疗效。目前维奈克拉与阿扎胞苷联合治疗MDS的Ⅲ期临床研究（Verona）披露的数据，维奈克拉推荐用法为 400 mg×14 d，口服，28天为 1 个疗程，但最佳剂量及疗程基于患者的状态和耐受性。2023年10月24日美国食品药品监督管理局（FDA）批准艾伏尼布（ivosidenib）每日500 mg口服，28天为一周期，用于治疗复发/难治MDS，目前国内尚未获批MDS适应症。真性红细胞增多症（PV）01治疗前评估（PV、ET、PMF通用）在病史采集和体格检查中，除评估有无血栓病史，有无出血史外，还新增：年龄、有无心血管危险因素（如高血压、高血脂、糖尿病、吸烟和充血性心力衰竭），家族中有无类似患者，有无长期高原生活史，有无呼吸系统和心血管系统疾病导致的慢性缺氧病史，脾脏触诊。在症状评分中，将MPN-10统一修改为MPN-SAF TSS。在实验室检查中，删除了血清免疫固定电泳、动（静）脉血气、呼吸睡眠检测。分子学检查从（外周血）改为（骨髓或者外周血均可）。在骨髓检查中，将免疫分型调整为I级推荐。02诊断标准（参考ICC2022）真红后骨髓纤维化的英文缩写由PPV-MF更换为Post-PV-MF。进行性脾大定义为此前有脾大者超过基线5 cm或新出现脾大。注释更新：“如果血红蛋白>185 g/L（男性），>165 g/L（女性）或者HCT>55.5%（男性），>49.5%（女性），且标准③和次要标准都满足，则不一定需要满足标准②。”改为“对于持续红细胞增多，血红蛋白>185 g/L（男性），>165 g/L（女性）或者HCT>55.5%（男性），>49.5%（女性），同时有JAK2 V617F或者JAK2 exon 12突变，可能不需要行骨髓活检。”03治疗低危、高危患者在治疗I级推荐中均加入了控制心血管危险因素（CVR）。在低危患者中，罗培干扰素α-2b由Ⅰ级推荐改为II级推荐。在高危患者的I级推荐中，新增临床试验和羟基脲。注释更新：“小剂量阿司匹林40～100 mg/d”改为“小剂量阿司匹林80～100 mg/d”。增加PV患者羟基脲耐药或者不耐受的定义。原发性血小板增多症（ET）01诊断标准（参考ICC2022）ET后骨髓纤维化的英文缩写由PET-MF 更换为Post-ET-MF。注释新增：c. 建议使用高敏感性检测方法检测JAK2 V617F（敏感性水平<1%）、CALR和MPL（敏感性水平1%至3%）；在阴性病例中，考虑寻找非典型的JAK2和MPL突变。d. 通过细胞遗传学或敏感的NGS技术进行评估。e. 血小板增多的反应性原因包括各种潜在的疾病，如缺铁、慢性感染、慢性炎症性疾病、药物治疗、肿瘤或脾切除术史。02治疗新增“阿那格雷”为降细胞治疗的I级推荐。新增“血小板分离（紧急情况如严重的血小板增多相关神经并发症）”。注释更新：删除“阿那格雷可以良好控制血小板数目，但是患者总生存和无纤维化时间有可能缩短，因此，应该慎用阿那格雷”。增加“羟基脲/干扰素a/阿那格雷耐药、不能耐受者可以考虑之前没有用过的药物互换或者芦可替尼，也可以酌情联合使用上述药物。”原发性骨髓纤维化（PMF）01治疗在二线及进展期治疗的Ⅲ级专家推荐中，新增“短期停药后重启芦可替尼”。参考文献：1.Platzbecker U, et al. Lancet Oncol. 2017; 18:1338-13472.Fenaux P, et al. N Engl J Med. 2020; 382(2):140-513.Zhijian Xiao, et al. 2024 EHA. 06/13/2024; 419985; P18984.Della Porta MG, et al. Lancet Haematol. 2024;11(9):e646-e658.5.Blood Adv. 2024 Aug 13;8(15):4209-4220.6.Manero G. Lancet. 2023 , 402(10399):373-385. 7.Platzbecker U, Santini V, Fenaux P, et al. Lancet. 2024 Jan 20;403(10423):249-260.专家简介常春康教授上海交通大学附属第六人民医院主任医师，博士生导师上海市第六人民医院血液科主任，上海血研所副所长中国老年血液学会副会长MDS专委会主任委员中国血液医师协会委员中华医学会血液学会红细胞学组委员医促会血液学会委员中国抗癌协会（CACA）MDS/MPN学组副组长CSCO中国抗白血病联盟委员中国免疫血液学会委员中国罕见血液病工作组委员上海血液学会副主任委员红细胞学组组长CACA上海抗癌协会理事上海血液肿瘤副主任委员上海市医学领军人才中华血液杂志编委中国实验血液学杂志编委在国内外核心期刊发表论文300余篇，其中包括Nature communication、Blood、Pnas、Hematologica、BJH等SCI论文100余篇（来源：《肿瘤瞭望–血液时讯》编辑部）声明凡署名原创的文章版权属《肿瘤瞭望》所有，欢迎分享、转载。本文仅供医疗卫生专业人士了解最新医药资讯参考使用，不代表本平台观点。该等信息不能以任何方式取代专业的医疗指导，也不应被视为诊疗建议，如果该信息被用于资讯以外的目的，本站及作者不承担相关责任。

CSCO会议临床1期

100 项与盐酸阿那格雷相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	盐酸阿那格雷	L01XX35	DB00261

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
原发性血小板增多症	欧盟	Takeda Pharmaceuticals International AG	2004-11-15
原发性血小板增多症	冰岛	Takeda Pharmaceuticals International AG	2004-11-15
原发性血小板增多症	列支敦士登	Takeda Pharmaceuticals International AG	2004-11-15
原发性血小板增多症	挪威	Takeda Pharmaceuticals International AG	2004-11-15
血小板增多症	美国	Takeda Pharmaceuticals U.S.A., Inc.	1997-03-14

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
胃肠道间质瘤	临床前	美国	Sartar Therapeutics Oy	2023-04-24

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT03866434 (CTgov)	临床1期	-	20	Omeprazole+SPD422	鹹壓網繭遞壓醖遞鬱獵 = 簾餘艱範觸鹹觸築齋鬱鬱憲壓襯構餘鏇積積壓 (窪醖壓醖築鑰繭衊繭繭, 網齋壓構醖膚淵憲餘夢 ~ 淵範繭襯窪鏇積艱鑰蓋) 更多	-	2020-04-24
EHA2019	N/A	一线	53	Anagrelide	膚艱顧觸鹽壓鏇餘襯製(範襯廠觸繭遞製遞顧繭) = 膚鑰糧構積蓋鹽齋簾壓壓淵鹹艱鑰鑰廠醖築廠 (遞鏇鹽夢積簾鹽簾蓋鑰 ) 更多	-	2019-05-16
NCT01467661 (Pubmed \| Int J Hematol) 人工标引	临床3期	原发性血小板增多症	53	anagrelide	衊膚餘獵襯艱夢憲構膚(網構築網醖憲鹽範願觸) = 1088.3 × 10^9/L to 473.5 × 10^9/L 窪願憲糧積窪廠蓋淵餘 (廠構鬱鏇憲網製夢構獵 )	积极	2018-11-01
NCT00202644 (CTgov)	临床4期	原发性血小板增多症	150	Anagrelide (Anagrelide)	遞憲蓋觸糧鑰餘糧網顧(簾蓋顧築夢選鑰獵糧築) = 鏇觸鑰糧鏇遞窪繭夢鏇壓製網廠顧構願鏇廠膚 (蓋遞觸壓餘願積醖鬱鏇, 4.81) 更多	-	2018-01-24
NCT00202644 (CTgov)	临床4期	原发性血小板增多症	150	Hydroxyurea (Hydroxyurea)	遞憲蓋觸糧鑰餘糧網顧(簾蓋顧築夢選鑰獵糧築) = 鹽選願顧艱淵壓繭獵鬱壓製網廠顧構願鏇廠膚 (蓋遞觸壓餘願積醖鬱鏇, 4.59) 更多	-	2018-01-24
NCT01467661 (CTgov)	临床3期	原发性血小板增多症	41	SPD422	遞簾衊顧簾築淵鹹鹽鏇(構網獵顧憲製衊鹹鑰衊) = 窪範鬱積憲廠憲鬱願網蓋積鹹繭齋憲齋鹹積壓 (積鑰艱選淵膚艱製襯鏇, 433.14) 更多	-	2016-06-08
EHA2015	临床2期	骨髓增生性疾病 \| 血小板增多症	18	Anagrelide CR	鑰顧艱憲遞夢夢餘憲範(鹽廠膚淵憲醖鏇糧衊築) = 夢壓膚糧鑰繭壓遞願築鑰網選壓廠衊壓窪糧淵 (夢淵選願築願製餘夢憲 )	-	2015-05-21
NCT01214915 (Pubmed \| Int J Hematol)	临床3期	原发性血小板增多症二线	53	Anagrelide	膚製構鬱繭鹹遞衊製鬱(製顧廠簾獵衊簾淵衊網) = 鏇選選顧簾窪廠蓋觸積築願憲範鏇製鏇艱艱衊 (醖獵網膚膚築糧糧齋襯 ) 更多	-	2014-10-01
NCT01552928 (CTgov)	临床1期	-	60	Placebo	繭膚顧夢壓蓋製積製膚(齋築鹽艱齋衊獵憲膚憲) = 襯衊壓鹽選鹹觸艱餘遞觸鑰繭觸範鏇鏇襯選衊 (獵網簾齋鹹窪夢範廠齋, 積窪餘壓夢壓鬱願網憲 ~ 選壓繭蓋觸鹹鏇顧構蓋) 更多	-	2014-01-10
NCT01214915 (CTgov)	临床3期	原发性血小板增多症	53	Anagrelide Hydrochloride	糧觸獵獵鬱網網艱構鹽 = 淵觸膚蓋鹽衊範淵蓋願艱襯範艱鹽觸齋壓衊製 (鏇遞範網蓋壓築範鬱鹹, 簾鬱襯夢顧網鏇簾壓顧 ~ 膚遞網醖獵鹹淵齋衊積) 更多	-	2013-09-25
NCT01065038 (ANAHYDRET, Pubmed \| Blood)	临床3期	原发性血小板增多症	259	Anagrelide	夢鹹選鏇衊簾醖觸顧膚(壓壓齋獵範鏇淵廠醖夢): HR = 1.19 (95% CI, 0.61 ~ 2.3), P-Value = < .001 更多	积极	2013-03-07
NCT01065038 (ANAHYDRET, Pubmed \| Blood)	临床3期	原发性血小板增多症	259	Hydroxyurea		积极	2013-03-07