A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Elritercept (KER-050) for the Treatment of Transfusion-Dependent Anemia in Adult Participants With Very Low-, Low-, or Intermediate-Risk Myelodysplastic Syndromes (MDS) (RENEW)

KER-050-D301 is a Phase 3, double-blind, randomized, placebo-controlled study to evaluate the efficacy and safety of KER-050 versus placebo in adult participants with transfusion-dependent anemia with very low, low, or intermediate risk MDS, or more recently defined as myelodysplastic neoplasms, with or without ring sideroblasts. The study is divided into the Screening Period, Treatment Period, Safety Follow-Up Period and Long-term Follow-up Period.

开始日期2024-12-31

申办/合作机构

Keros Therapeutics, Inc.

NCT06525948 / Not yet recruiting临床4期IIT

Efficacy and Safety of rhTPO in Combination With Cyclosporine Versus Cyclosporine Alone in the Treatment of Transfusion-dependent Non-severe Aplastic Anaemia (TD-NSAA)

Investigating the efficacy and safety of rhTPO in combination with cyclosporine versus cyclosporine alone for the treatment of TD-NSAA

开始日期2024-07-30

申办/合作机构

北京协和医院

NCT06414031 / Recruiting临床3期IIT

Tranexamic Acid for Reduction of Intra- and Postoperative Transfusion Requirements in Elective Abdominal Surgery: Randomized Controlled Trial

The goal of this clinical trial is to learn if the administration of tranexamic acid can reduce the necessity of blood transfusions in adult patients undergoing major abdominal surgery. . It will also inform about safety of tranexamic acid in this setting.
The main question it aims to answer is: Does tranexamic acid lower the probability of receiving at least one blood transfusion during or after surgery?
Participants will compare tranexamic acid o a placebo (a look-alike substance that contains no drug) to see if tranexamic acid works to reduce the necessity of a blood transfusion.

开始日期2024-06-17

申办/合作机构

Martin-Luther-Universität Halle-Wittenberg

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100 项与输血依赖性贫血相关的临床结果

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100 项与输血依赖性贫血相关的转化医学

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0 项与输血依赖性贫血相关的专利（医药）

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499

项与输血依赖性贫血相关的文献（医药）

2024-10-24·Blood

Proposals for revised International Working Group–European LeukemiaNet criteria for anemia response in myelofibrosis

Article

作者: Hexner, Elizabeth O. ; Vannucchi, Alessandro M. ; Guglielmelli, Paola ; Gotlib, Jason ; Hernandez-Boluda, Juan-Carlos ; Kuykendall, Andrew T. ; Pardanani, Animesh ; Mesa, Ruben ; Döhner, Konstanze ; Barbui, Tiziano ; Sirhan, Shireen ; Odenike, Olatoyosi ; Hobbs, Gabriela S. ; Vachhani, Pankit J. ; Pemmaraju, Naveen ; Tefferi, Ayalew ; Szuber, Natasha ; Kiladjian, Jean-Jacques ; Loscocco, Giuseppe G. ; Mora, Barbara ; Masarova, Lucia ; Gisslinger, Heinz ; Rambaldi, Alessandro ; Oh, Stephen T. ; Garcia, Jacqueline S. ; Kroger, Nicolaus ; Bose, Prithviraj ; Koschmieder, Steffen ; Mascarenhas, John ; Patel, Anand ; Passamonti, Francesco ; Harrison, Claire ; Talpaz, Moshe ; Gupta, Vikas ; Rampal, Raajit ; Gangat, Naseema ; Ellis, Martin ; Barosi, Giovanni

AbstractWith emerging new drugs in myelofibrosis (MF), a robust and harmonized framework for defining the severity of anemia and response to treatment will enhance clinical investigation and facilitate interstudy comparisons. Accordingly, the lead authors on the 2013 edition of the International Working Group–European LeukemiaNet (IWG-ELN) response criteria in MF were summoned to revise their document with the intent to (1) account for gender-specific differences in determining hemoglobin levels for eligibility criteria; (2) revise the definition of transfusion-dependent anemia (TDA) based on current restrictive transfusion practices; and (3) provide a structurally simple and easy to apply response criteria that are sensitive enough to detect efficacy signals (minor response) and also account for major responses. The initial draft of the 2024 IWG-ELN proposed criteria was subsequently circulated around a wider group of international experts and their feedback incorporated. The proposed articles include new definitions for TDA (≥3 units in the 12 weeks before study enrollment) and hemoglobin thresholds for eligibility criteria (<10 g/dL for women and <11 g/dL for men). The revised document also provides separate (TDA vs non-TDA) and graded (major vs minor response) response criteria while preserving the requirement for a 12-week period of screening and observation on treatment.

2024-09-16·Cureus

β-Thalassemia Major Complicated by Acute Myeloid Leukemia

Article

作者: Poombal, Fnu ; Poombal, FNU ; Suhail, Maymoona ; Faisal, Alizah ; Khalid, Mehreen

This report describes the rare co-occurrence of acute myeloid leukemia (AML) French-American-British type M2 in a 4.5-year-old boy with previously diagnosed thalassemia major, an inherited hemoglobinopathy, typically presenting with severe, transfusion-dependent anemia. Chronic transfusions, though lifesaving, can lead to iron overload, which may generate free radicals and potentially contribute to malignancy. Our case highlights the importance of close monitoring for secondary malignancies in thalassemia patients. Our patient born to consanguineous parents, presented with persistent fever, abdominal pain, and splenomegaly. Hematological investigations revealed severe cytopenias (low blood cell counts) and many immature blood cells (blasts). Bone marrow examination confirmed AML M2, characterized by an overabundance of myeloid blasts. Despite the initiation of myeloid leukemia-directed aggressive chemotherapy, the patient, unfortunately, succumbed to the disease within a month of diagnosis.

2024-09-01·The Lancet Haematology

Low dose lenalidomide versus placebo in non-transfusion dependent patients with low risk, del(5q) myelodysplastic syndromes (SintraREV): a randomised, double-blind, phase 3 trial

Article

作者: Fenaux, Pierre ; Amigo, María Luz ; Del Rey González, Mónica ; Arar, Ali ; Xicoy, Blanca ; González, Teresa ; Lumbreras, Eva ; Sánchez-García, Joaquín ; Hernández-Rivas, Jose-Ángel ; Bernal, Teresa ; Hernández-Rivas, Jesús María ; Thepot, Sylvain ; Díez-Campelo, María ; Preudhomme, Claude ; Guerci-Bresler, Agnès ; Bargay, Joan ; Fenwarth, Laurene ; López-Cadenas, Félix ; Slama, Bohrane ; Coll Jordà, Rosa ; Toribio, Sofía ; Del Cañizo, Consuelo ; Götze, Katharina S ; Platzbecker, Uwe

BACKGROUNDLenalidomide is the standard of care for patients who are transfusion dependent with chromosome 5q deletion (del[5q]) myelodysplastic syndromes. In the SintraREV trial, we aimed to investigate whether an early intervention of low lenalidomide doses for 2 years could delay transfusion dependency in patients with anaemia who were not transfusion dependent.METHODSThis randomised, double-blind, phase 3 trial, was conducted at 22 sites (University Hospitals) in Spain, France, and Germany. Eligible patients were aged 18 years or older diagnosed with low-risk or intermediate-1-risk del(5q) myelodysplastic syndromes with non-transfusion-dependent anaemia (according to the IPSS), were erythropoietin-stimulating agents naive, and had an ECOG performance status of 2 or less. Patients were randomly assigned (2:1) by means of a telephone system to receive lenalidomide 5 mg daily in 28-day cycles versus placebo for 2 years. The primary endpoint was time to transfusion dependency based on blinded independent central review. Analysis were by intent-to-treat (ITT) and evaluable population. Safety analyses included all participants who received at least one dose of treatment. This trial is registered with ClinicalTrials.gov (NCT01243476) and EudraCT (2009-013619-36) and is complete.FINDINGSBetween Feb 15, 2010, and Feb 21, 2018, 61 patients were randomly assigned to receive lenalidomide (n=40; two did not receive treatment) or placebo (n=21). The median age was 72·2 (IQR 65·4-81·9) years, 50 (82%) patients were female, and 11 (18%) were male. The median follow-up time was 60·6 (IQR 32·1-73·9) months. Regarding primary endpoint, median time to transfusion dependency was not reached (95% CI not applicable) in the lenalidomide group versus 11·6 months (95% CI 0·00-30·11) in the placebo group (p=0·0027). Lenalidomide significantly reduced the risk of transfusion dependency by 69·8% (hazard ratio 0·302, 95% CI 0·132-0·692; p=0·0046). The most frequent treatment-related adverse event was neutropenia, occurring in 24 (63%) of 38 patients in the lenalidomide group (grade 3 and 4 in 17 [45%] patients and one [3%], respectively) and in four (19%) of 21 patients in the placebo group (grade 3 in one [5%] patient). Thrombocytopenia was detected in seven (18%) of 38 patients receiving lenalidomide (grade 3 in two [5%] patients). Regarding the non-haematological toxicity, skin disorders (rash nine [23%] of 38 patients) were the most frequently described toxicities among patients receiving lenalidomide, being grade 3 in one (3%) of 38 patients. 19 serious adverse events were reported in 13 patients, 18 in the lenalidomide group and one in the placebo group, five of which were potentially related to the study drug. No treatment-related deaths were identified.INTERPRETATIONAn early approach with low doses of lenalidomide across two years delays the time to transfusion dependency and improves the rate and quality of the responses, with a manageable safety profile in patients who are non-transfusion dependent with del(5q) low-risk myelodysplastic syndromes.FUNDINGBristol Myers Squibb.

113

项与输血依赖性贫血相关的新闻（医药）

2024-10-15

·同写意

基因疗法第一网红，翻盘越来越难了

本届大会以“共建创新生态共赢产业未来”为主题，聚焦生物医药出海与国际化创新、创新药投融资、医疗器械等产业发展风向标，打造“高端化、国际化、专业化、立体化”的年度盛会。提到基因疗法，蓝鸟生物是绕不开的一个存在。 1992年，两位麻省理工学院的研究人员创立了蓝鸟生物的前身Genetix Pharmaceuticals。经过近20年的坚持，蓝鸟生物在基因疗法的努力方显成效。 2010年，《nature》发布了一项临床试验结果，在蓝鸟生物Lentiglobin基因疗法的临床试验中，一位患有重型β地中海贫血的患者成功摆脱输血治疗。要知道，此前对于地中海贫血患者只有终身输血这一治疗方案，而蓝鸟生物则为地中海贫血患者带来新的希望。蓝鸟生物也由此一战成名，获得了众多投资人的青睐。2013年6月，蓝鸟生物登陆纳斯达克。此后，蓝鸟生物一路向上，不仅收获了众多荣誉，2017年被评为全球最聪明的50家公司之一，2018年被评为最有可能被收购的十大并购标的之一；公司股价更是水涨船高，2018年时市值最高曾逼近300亿美金。然而，命运总是充满波折与意想不到。2020年，蓝鸟生物首款基因疗法产品获批，这本该是其高光时刻的开始，没想到却成了其过山车式命运的最高点。自此之后，蓝鸟生物的一路向下，如今市值已不足1亿美金。更糟糕的是，当前公司负面不断，产品安全性问题争议持续，蓝鸟生物的处境变得越来越危险…… 1 7名患者患癌风波日前，发表在《新英格兰医学杂志》上的一项研究，让蓝鸟生物陷入了风波。该研究是针对基因疗法Skysona治疗脑肾上腺脑白质营养不良2-3期研究（ALD-102和 ALD-104）和一项正在进行的随访研究（LTF-304）数据。结果不容乐观：67名儿童患者中，7 名接受Skysona治疗的儿童患上了血癌。6例被诊断为骨髓增生异常综合征（MDS），在Skysona治疗后 14 至 92 个月内出现；另一名患者在57个月时被诊断出患有急性髓性白血病（AML）。目前，6 例MDS患者接受了干细胞移植治疗，1 例患者因移植物抗宿主病而死亡。AML患者仍然活着，并且对移植表现出良好的反应。导致7名儿童患癌的根源，是Skysona。 Skysona作为一种基因疗法，通过将ABCD1基因的功能性拷贝递送到患者的造血干细胞中来发挥作用，从而促进能够表达功能性ALDP蛋白的成熟单核细胞的产生。但其存在风险，Skysona疗法中的慢病毒载体整合到原癌基因中，会诱发血液瘤。Skysona的标签上带有血液系统恶性肿瘤的黑框警告，包括MDS等“危及生命的病例”。而上述7 名接受Skysona 治疗的儿童患上了血癌，大致原因也是如此。据研究人员称，新出现的血癌与与癌症相关基因中的克隆向量插入有关，同时也与某些感兴趣位点的体细胞突变累积有关，包括KRAS、WT1和CDKN2A基因中的突变。虽然患癌风波将会如何影响蓝鸟还不得而知，但这无疑再次为我们敲响了安全性警钟。 2 安全、安全、安全安全问题一直是悬在基因疗法之上的达摩克里斯之剑。 1999年，一名18岁少年在接受腺病毒基因疗法后发生严重免疫反应死亡，这是第一例因基因疗法而死亡的患者。这一事件，也使得FDA收紧了对于基因疗法的临床审批，基因疗法因此沉寂了十余年之久。直到2017年12月，美国首个基因疗法获批上市，由Spark Therapeutics开发的Luxturna基因疗法，用于治疗眼科疾病。基因疗法得以再度活跃。不过，安全问题并未随着基因疗法获批上市而消失。相反，安全问题如同一颗定时炸弹，仍然威胁着基因疗法的发展。 2021年2月，蓝鸟基因疗法bb1111对镰状细胞贫血患者的1/2期临床试验中，产生了两个疑似血液肿瘤病例，一位受试者被诊断为急性髓性白血病，另一名患者发生了骨髓增生异常综合征。因为可能存在的致癌风险，FDA叫停了蓝鸟生物bb1111的临床试验。与此同时，已经在欧洲获批上市的Zynteglo，因为与其使用了相同的慢病毒粒载体，存在潜在的安全问题，也被蓝鸟生物暂停销售。经过5个月的评判，最终证明治疗收益大于风险，Zynteglo得以重新开始销售，而bb1111临床试验也得以重启。但就在半年后，bb1111的临床试验再次遇到问题。 2021年，一名青少年患者在接受bb1111 治疗后出现了持续性非输血依赖性贫血，18岁以下镰状细胞病患者的临床研究进被部分搁置。虽然相比30年前，基因疗法的安全性有了很大的提升，但目前基因疗法仍然难言彻底安全，而这也绝非蓝鸟生物一家之忧。 2020年8月，被安斯泰来30亿美元收购的基因疗法公司Audentes，其核心产品AT132在临床试验中出现了三位患者死亡。 2020年10月，一名患者在接受Lysogene AAV基因治疗后去世。如今，Skysona的致癌风波再次发酵，告诉我们基因疗法的发展仍然如履薄冰，一不小心就会因为安全问题再次坠入深渊。 3 当然，尽管蓝鸟生物这一基因疗法的先驱坠落，但这并不会影响基因疗法仍将持续向上的事实。仅放眼国内市场，随着资金的涌入，也已经出现了一批基因疗法的新兴玩家，如博雅辑因、合生基因、纽福斯、中因科技等。其中，纽福斯的AAV基因治疗药物NR082和中因科技的基因治疗药物ZVS101e，都获得了FDA的孤儿药认证。不过，对于国内玩家来说，蓝鸟生物的经历在前，安全问题同样也是部分国内玩家，需要面临的挑战。蓝鸟生物从风光无限到无奈坠落，也不过三年多的光景。这背后，也映射了市场对于基因疗法的态度变化，从未知的担心，再到充满期待的追捧，再到如今归于平静。不少人已经意识到，虽然长远来看，基因疗法仍然具有无限的潜力、广阔的发展空间，但是短期来看，基因疗法针对适应症多为罕见病，仍属于一个小众治疗手段。基因疗法中像Zolgensma一样的只是少数，大多数都如同蓝鸟生物一样，深陷亏损的泥潭之中。从全球视角来看，基因疗法的安全性和商业化问题仍然还未完全找到答案。而在这之前，基因疗法仍是一个充满无限可能但又危险重重的地方。同写意媒体矩阵，欢迎关注↓↓↓

基因疗法并购临床结果

2024-10-15

·BioSpace

Geron to Announce Third Quarter 2024 Financial Results on November 7, 2024

FOSTER CITY, Calif.--(BUSINESS WIRE)--Geron Corporation (Nasdaq: GERN), a commercial-stage biopharmaceutical company, today announced that it will release its third quarter 2024 financial results and business highlights before the market opens on Thursday, November 7, 2024 via press release, which will be available on the Company’s website at . Geron will host a conference call to discuss the financial results as well as business highlights at 8:00 a.m. ET the same day. A live webcast of the conference call and related presentation will be available on the Company’s website at . An archive of the webcast will be available on the Company’s website for 30 days. Participants may access the webcast by registering online using the following link, . About Geron Geron is a commercial-stage biopharmaceutical company aiming to change lives by changing the course of blood cancer. Our first-in-class telomerase inhibitor RYTELO™ (imetelstat) is approved in the United States for the treatment of certain adult patients with lower-risk myelodysplastic syndromes (LR-MDS) with transfusion dependent anemia. We are also conducting a pivotal Phase 3 clinical trial of imetelstat in JAK-inhibitor relapsed/refractory myelofibrosis (R/R MF), as well as studies in other hematologic malignancies. Inhibiting telomerase activity, which is increased in malignant stem and progenitor cells in the bone marrow, aims to potentially reduce proliferation and induce death of malignant cells. To learn more, visit or follow us on LinkedIn . Contacts Aron Feingold Vice President, Investor Relations and Corporate Communications Kristen Kelleher Associate Director, Investor Relations and Corporate Communications investor@geron.com media@geron.com

上市批准临床3期财报

2024-10-11

·药精通Bio

2024 FDA已批准34款新药！其中Q3新增13款！

OTC2024类器官前沿应用与3D细胞培养论坛圆满落幕，点击图片可查看会后报告，咨询OTC2025类器官论坛请联系：王晨 180 1628 8769 🧴Berdazimer （Zelsuvmi），一种用于治疗局部传染性软疣的一氧化氮（NO）释放剂。💉头孢吡肟/恩美唑巴坦（Exblifep），一种包含头孢吡肟和恩美唑巴坦的固定剂量组合，用于治疗尿路感染。 💉LetibotulinumtoxinA （Letybo），一种纯化的 A 型肉毒杆菌神经毒素蛋白复合物，用于改善眉间纹的外观。💉替雷利珠单抗（Tivembra）是一种 IgG4 变体 PD-1 mAb，适用于治疗不可切除、局部晚期或转移性食管鳞状细胞癌。💊Resmetirom （Rezdiffra），一种甲状腺激素受体 β （NR1A2）激动剂用于治疗非肝硬化性非酒精性脂肪性肝炎。 💊Aprocitentan （Tryvio），一种用于治疗高血压的内皮素受体拮抗剂。💊Givinostat （Duvyzat），一种组蛋白脱乙酰酶抑制剂，用于治疗杜氏肌营养不良症。💉 Sotatercept （Winrevair），一种用于治疗肺动脉高压的激活素信号抑制剂。💊 Vadadustat （Vafseo），一种口服缺氧诱导因子脯氨酰羟化酶抑制剂，用于治疗成人与 CKD 相关的症状性贫血。💊 Danicopan （Voydeya），补体因子 D 抑制剂，用于治疗 PNH 患者的血管外溶血。 💉 Ceftobiprole medocaril （Zevtera）是一种广谱头孢菌素抗菌剂，适用于某些类型的肺炎、菌血症和皮肤感染。💉 Pegulicianine （Lumisight），一种用于乳腺癌荧光成像的光学成像剂。💉 Nogapendekin alfa inbakicept （Anktiva）是一种白细胞介素 15 受体激动剂与卡介苗（BCG）的组合，用于治疗膀胱癌。 💊 Tovorafenib （Ojemda），一种 II 型 RAF 激酶抑制剂，用于治疗具有 BRAF 基因突变的小儿低级别胶质瘤。💊 Mavorixafor （Xolremdi），一种用于治疗 WHIM 综合征的 CXCR4 拮抗剂。💉 Tarlatamab （Imdelltra）是一种靶向 DLL3 和 CD3 的双特异性单克隆抗体和 T 细胞接合器，用于治疗广泛期小细胞肺癌。💉 Imetelstat （Rytelo），一种寡核苷酸端粒酶抑制剂，用于治疗骨髓增生异常综合征伴输血依赖性贫血。 💊 Elafibranor （Iqirvo），一种双重 PPARα/δ 激动剂，用于治疗原发性胆汁性胆管炎。🧴Sofpironium （Sofdra），一种用于治疗多汗症的 M3 毒蕈碱受体抑制剂。💉 Crovalimab （Piasky），一种用于治疗阵发性睡眠性血红蛋白尿症的补体 C5 抑制性 mAb。🫁 Ensifentrine，一种用于治疗 COPD 的双重 PDE3/4 抑制剂。💉Donanemab-azbt，一样β淀粉样蛋白（Aβ）定向抗体，用于治疗阿尔兹海默病。💊Deuruxolitinib，一种JAK1和JAK2抑制剂，用于治疗严重斑秃。💊Vorasidenib，一种 IDH1 和 IDH2 抑制剂，用于治疗具有易感 IDH1 或 IDH2 突变的 2 级星形细胞瘤或少突胶质细胞瘤。💉Palopegteriparatide，一种用于治疗甲状旁腺功能亢进症的甲状旁腺激素类似物。💉Nemolizumab，一种用于治疗结节性痒疹的 IL-31 受体 α 拮抗剂。 💊Seladelpar 是一种 PPAR-δ 激动剂，用于单独治疗原发性胆汁性胆管炎或与熊去氧胆酸联合治疗。💉Axatilimab 是一种 CSF-1R mAb，用于降低慢性移植物抗宿主病患者的致病性巨噬细胞活性。💊Lazertinib，一种 EGFRm 激酶抑制剂，与 amivantamab 联合用于治疗具有 EGFR 突变的非小细胞肺癌。💉Lebrikizumab，一种抗 IL-13 的 IgG4 mAb，用于治疗中度至重度特应性皮炎。💊Arimoclomol 是一种热休克蛋白共诱导剂，用于与 miglustat 联合治疗 C 型尼曼-匹克病的神经系统症状。💊L-乙酰亮氨酸，亮氨酸的一种改良形式，用于治疗 C 型尼曼-匹克病患者的神经系统症状。💊Xanomeline 和 trospium chloride，联合双重 M 1 - 和 M 4 - 选择性毒蕈碱受体激动剂和毒蕈碱受体拮抗剂，用于治疗精神分裂症。 💉Flurpiridaz F-18，一种用于评估心肌缺血和梗死的放射性诊断药物Picture from Chris De Savi 文章来源：早研早聊参考素材：素材来源官方媒体/网络新闻 END 免责声明：本文仅作知识交流与分享及科普目的，不涉及商业宣传，不作为相关医疗指导或用药建议。文章如有侵权请联系删除。 OTC2024类器官前沿应用与3D细胞培养论坛圆满落幕，点击图片可查看会后报告，咨询OTC2025类器官论坛请联系：王晨 180 1628 8769

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