Phase II Open-label Clinical Trial Evaluating Efficacy of Romiplostim Added to Standard of Care for Children and Young Adults With Treatment Naive and Relapsed or Refractory Severe Aplastic Anemia

This Phase II open-label interventional clinical trial aims to evaluate the efficacy of romiplostim, in patients with severe aplastic anemia (SAA), both treatment naïve and relapsed/refractory, in inducing trilineage hematopoiesis in children and young adults.

开始日期2025-07-31

申办/合作机构

University of Iowa

[+2]

NCT07043894

/ Not yet recruiting临床2期IIT

A Prospective, Multicenter Study on the Efficacy and Safety of Romiplostim N01 for Chemotherapy-Induced Thrombocytopenia (CIT) in Children, Adolescents, and Young Adults (CAYA) With Hematological and Solid Tumors

The goal of this clinical trial is to evaluate the efficacy and safety of romiplostim N01 in treating chemotherapy-induced thrombocytopenia (CIT) in children and adolescents/young adults (CAYA; aged 6-24 years) with hematologic malignancies or solid tumors. The main questions it aims to answer are:
* What proportion of participants achieve platelet count recovery (≥100×10⁹/L or an increase of ≥30×10⁹/L from baseline) within 3 weeks of romiplostim N01 treatment?
* What is the safety profile of romiplostim N01 in this population, including the incidence and severity of adverse events (especially bleeding and thrombosis)? This is a single-arm study (no comparison group). Researchers will assess the outcomes against predefined efficacy thresholds and historical data (e.g., a 60.7% response rate reported for another TPO-RA, hetrombopag).
Participants will:
* Receive weekly subcutaneous injections of romiplostim N01 (starting dose: 2 µg/kg).
* Have their romiplostim dose adjusted weekly based on platelet counts (increase by 1-2 µg/kg if platelets <99×10⁹/L, maximum dose 10 µg/kg, stop when target recovery is met).
* Undergo frequent monitoring, including blood tests (especially platelet counts), vital signs, physical exams, and assessment for adverse events and bleeding throughout the treatment and follow-up period.

开始日期2025-07-01

申办/合作机构

中国医学科学院肿瘤医院

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项与罗米司亭相关的文献（医药）

2025-12-31·ANNALS OF MEDICINE

Effective treatment of refractory aplastic anemia with romiplostim after failure of multiple thrombopoietin receptor agonists: a single-center retrospective study

Article

作者: Chen, Miao ; Liu, Ziwei ; Han, Bing ; Yang, Chen ; Lin, Xijuan ; Zhuang, Junling

BACKGROUND:

Romiplostim (ROM), a second-generation thrombopoietin receptor agonist (TPO-RA), has shown promising results in patients with refractory aplastic anemia (AA); however, its optimal dosage, efficacy, and safety in patients with refractory AA who experienced treatment failure with immunosuppressive therapy (IST) and other types of TPO-RAs remain unclear. In the present study, we aimed to retrospectively assess the efficacy and safety of high-dose ROM in these patients.

PATIENTS AND METHODS:

Patients who received ROM consecutively for at least 3 months between 8 April 2023 and 23 October 2023, and were followed up for at least 6 months after therapy were analyzed. All enrolled patients had failed to respond and stopped the previous treatments for at least 3 months before receiving ROM.

RESULTS:

Eleven patients were followed up for at least 6 months; all experienced treatment failure with IST and at least two types of other TPO-RAs. They had a median age of 54 years (range: 27-77 years), and eight (72.7%) were women. The patients' initial and maximal ROM dose was 20 μg/kg per week. At a median follow-up of 8 months (range, 6-8 months), 72.7% (8/11) of the patients reached the response criteria at a median of 1 month (range: 1-3 months). Of these patients, 54.5% (6/11) met the criteria for a complete response at a median of 2.5 months (range: 1-3 months), and 27.3% (3/11) achieved a trilineage response. No severe ROM-related adverse events that led to treatment discontinuation or dosage reduction were observed. Notably, 12.5% (1/8) of the responders relapsed at 5 months after stopping ROM for 2 months.

CONCLUSION:

High-dose ROM with an initial dosage of 20 μg/kg per week is effective and safe for patients with refractory AA who experienced treatment failure with IST and multiple oral TPO-RAs, exerting a rapid response.

2025-12-31·Hematology

Sustained response off treatment after fostamatinib in refractory immune thrombocytopenia: A series of four case reports

Article

作者: Ghanima, Waleed ; Rackwitz, Stefan ; Lucas Boronat, Francisco Javier ; Carrai, Valentina

INTRODUCTION:

A goal of most primary immune thrombocytopenia (ITP) treatments is reducing or discontinuing treatment while maintaining a response including an absence of bleeding events. We present four cases describing treatment with the spleen tyrosine kinase (SYK) inhibitor, fostamatinib, that showed sustained response off treatment (SROT).

CASE PRESENTATIONS:

Case 1 was a 66-year-old male with chronic ITP. He was pre-treated with prednisone and rituximab before being in the FIT-2 clinical trial (placebo). He received fostamatinib in the FIT-3 open-label extension for seven weeks and maintained SROT for 2.5 years. Case 2 was a 54-year-old female patient with chronic, highly refractory ITP. SROT was achieved after 6 months of fostamatinib and was maintained for more than 16 months (in remission to date). Case 3 was a 60-year-old male with chronic ITP. He was successfully treated with cycles of corticosteroids for six years prior to fostamatinib. He was treated with fostamatinib plus prednisone for approximately two months. SROT was observed in this patient for one year. Case 4 was a 67-year-old male with persistent ITP. Before fostamatinib, he was unresponsive to high-dose dexamethasone, IVIG, eltrombopag and romiplostim. After 11 months of fostamatinib, his dose was tapered for three months and ultimately discontinued. SROT was observed for more than ten months (in remission to date).

DISCUSSION:

These cases emphasize that SROT is achievable with fostamatinib in complex ITP cases unresponsive to multiple previous therapies. Additional research is needed to identify the magnitude of the underlying mechanisms, and the clinical factors associated with, and potentially predictive of, SROT.

2025-12-31·PLATELETS

A case with a novel GATA1 variant mimicking immune thrombocytopenia attacks

Article

作者: Aktekin, Elif Habibe ; Ceylaner, Serdar ; Erbay, Ayşe ; Beşen, Şeyda ; Yazıcı, Nalan

One of the leading thrombocytopenia occasions in childhood is immune thrombocytopenia (ITP). Small number of these patients do not respond to treatment methods known to be effective for ITP such as corticosteroids, intravenous immunoglobulin and thrombopoietin receptor agonists (eltrombopag, romiplostim, etc.) and have serious bleeding symptoms. We present a case in which a molecular genetic analysis was performed due to challenging responses to medical treatment for severe systemic bleeding symptoms which were similar to ITP. A hemizygous variant was detected in the GATA1 gene and a homozygous variant in the ATP2A1 gene for Brody myopathy. Eltrombopag, romiplostim, IVIG and corticosteroid were applied to resistant thrombocytopenia of the patient without knowing the cause. Patient had response in platelet counts in some occasions. This situation attracted our attention after the detection of this GATA variant in the etiopathogenesis of thrombocytopenia. Further investigations should be performed for much more rare causes of thrombocytopenia in patients with refractory thrombocytopenia attacks.

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2025-06-12

·sobi.com

Sobi share new clinical data across multiple hematologic diseases at EHA 2025

Sobi® (STO: SOBI) will present data at the 30th EHA (European Haematology Association) hybrid congress, in Milan, Italy (12-15 June). The congress will feature the latest advances in the treatment of diffuse large B-cell lymphoma (DLBCL), immune thrombocytopenia (ITP), myelofibrosis, paroxysmal nocturnal haemoglobinuria (PNH), and primary hemophagocytic lymphohistiocytosis (pHLH). An extensive programme of poster presentations will showcase Sobi’s commitment to helping patients with rare diseases by advancing treatment options. In addition, Sobi will host several scientific symposiums at the congress including: 1.Advancing Therapeutic Knowledge in Paroxysmal Nocturnal Haemoglobinuria: Reshaping disease management to unlock new norms, Thursday, 12 June, 10:00am - 11:30am CEST, at Amber Hall 3 & 4. 2. Boosting Platelets: Expert Approaches to Adult Immune Thrombocytopenia (ITP), Saturday 14 June, 8.00 am - 9.30 am CEST, at Amber Hall 7 & 8. 3. Dissecting Treatment Sequencing in relapsed/refractory DLBCL, from laboratory to real life. Saturday, 14 June, 8:00 am – 9:30 am, CEST at Coral Hall 1. “The breadth of data that we share at this year’s EHA congress demonstrates Sobi’s comprehensive approach to addressing rare conditions in haematology. We are proud to contribute to advancing the science in several indications from early clinical phases in diffuse large B-cell lymphoma to clinical and real-world evidence in myelofibrosis, primary hemophagocytic lymphohistiocytosis, immune thrombocytopenia and paroxysmal nocturnal haemoglobinuria,” said Lydia Abad-Franch, MD, Head of R&D and Medical Affairs, and Chief Medical Officer at Sobi. Key data to be presented at EHA 2025 DLBCL PS1911: Initial Results From LOTIS-7: A Phase 1b Study of Loncastuximab Tesirine Plus Glofitamab in Patients With Relapsed/Refractory (R/R) Diffuse Large BCell Lymphoma (DLBCL) Presenting Author: Juan Pablo Alderuccio Poster presentation Session title: Poster Session 2Session date: Saturday, 14 June Session time: 18:30-19:30 CEST Location: Poster Hall PS1957: Updated Safety Run-in Results from LOTIS-5: A Phase 3, Randomized Trial of Loncastuximab Tesirine with Rituximab Versus Immunochemotherapy in Patients With R/R DLBCL Presenting Author: Carmelo Carlo-Stella Immune Thrombocytopenia (ITP) PF1236: Platelet Response to Avatrombopag Among Patients with Primary Immune Thrombocytopenia Who Switched from Eltrombopag or Romiplostim: the REAL-AVA 2.0 Real-World Study Presenting Author: Shruti Chaturvedi Poster presentation Session title: Poster Session 1Session date: Friday, 13 June Session time: 18:30 - 19:30 CEST Location: Poster Hall PF1239: Durability of Response to Avatrombopag Among Patients with Primary Immune Thrombocytopenia: The REAL-AVA 2.0 Real-World Study Presenting Author: Srikanth Nagalla PF1251: Clinically Meaningful Response to Avatrombopag: A Phase 3B Trial for Treatment of Children with ITP Presenting Author: Rachael F. Grace PS2231: Effectiveness and safety of avatrombopag for the treatment of adults with newly diagnosed, persistent, or chronic immune thrombocytopenia: Interim results from the phase 4 ADOPT study Presenting Author: Waleed Ghanima Poster presentation Session title: Poster Session 2Session date: Saturday, 14 June Session time: 18:30 - 19:30 CEST Location: Poster Hall PS2234: Efficacy and safety of avatrombopag for the treatment of pediatric immune thrombocytopenia in the open-label extension of a phase 3, randomized, double-blind, placebo-controlled trial Presenting Author: Rachael F. Grace PS2239: Real-World Treatment Patterns & Clinical Outcomes in Thrombopoietin Receptor Agonist Naive Patients with Immune Thrombocytopenia Treated with Avatrombopag: Interim Results from the REAL-AVA 3.0 Study Presenting Author: Sandhya Panch PS2242: Effectiveness and safety of avatrombopag for treatment of immune thrombocytopenia in older patients and those with comorbidities or prior TPO-RA exposure: Interim results from the phase 4 ADOPT study Presenting Author: María Eva Mingot-Castellano PS2244: Response to Avatrombopag Among Patients with Chronic and Persistent Primary Immune Thrombocytopenia: the REAL-AVA 2.0 Real-World Study Presenting Author: M Y Levy PS2250: Evaluation of Efficacy and Safety of Avatrombopag in Children with Immune Thrombocytopenia based on Disease Duration: Results from the Avatrombopag Phase 3-b Pediatric Trial Presenting Author: Rachael F. Grace PB3676: Baseline Correlates with Durability of Avatrombopag Response: A Phase 3B Trial for Treatment of Children with ITP Publication Only Published on May 14 at 15:30 CEST PB3684: Consistent Response to Avatrombopag across Various Baseline Characteristics: Results from the Phase 3-b Trial for the Treatment of Children with Immune Thrombocytopenia Myelofibrosis PF849: Hematologic improvement experienced by pacritinib-treated patients with myelofibrosis in real-world clinical settings Presenting Author: Michael Marrone Poster presentation Session title: Poster Session 1Session date: Friday, 13 June Session time: 18:30 - 19:30 CEST Location: Poster Hall PF1242: Efficacy of pacritinib vs momelotinib in patients with thrombocytopenic MF: a matched adjusted indicated treatment comparison Presenting Author: Koo Wilson PF1306: Transfusion-related cost and time burden offsets in patients with myelofibrosis treated with pacritinib compared to best available therapy based on PERSIST-2 trial Presenting Author: Abiola Oladapo PS1827: Real-world effectiveness of pacritinib in patients with myelofibrosis who have concurrent thrombocytopenia and anemia Presenting Author: Raajit Rampal Poster presentation Session title: Poster Session 2 Session date: Saturday, 14 June Session time: 18:30-19:30 CEST Location: Poster Hall PS1842: Real-World Treatment Patterns and Clinical Outcomes in Patients with Myelofibrosis Treated with Pacritinib (PAC) with platelets ≥50 x109/L at PAC initiation: Interim results from the MY-PAC Study Presenting Author: Doug Tremblay PS2295: Economic Burden of Cytopenia in Patients with Myelofibrosis: Analysis of a US National Administrative Claims Database Presenting Author: Lucia Marasova PB3079: Cytopenia is associated with real-world disease progression and diminished survival in patients with myelofibrosis: Analysis of a US national administrative claims database Publication Only Published on May 14 at 15:30 CEST Paroxysmal Nocturnal Hemoglobinuria PF672: Early response in complement inhibitor naïve patients with paroxysmal nocturnal hemoglobinuria treated with pegcetacoplan in the Phase 3 PRINCE trial Presenting Author: Austin Kulasekararaj Poster presentation Session title: Poster Session 1Session date: Friday, 13 June Session time: 18:30 - 19:30 CEST Location: Poster Hall PF676: Interim analysis of the ongoing COMPLETE study on the real-world effectiveness of pegcetacoplan in patients with paroxysmal nocturnal hemoglobinuria (PNH) Presenting Author: Regis Peffault de Latour PS1662: A benefit assessment of pegcetacoplan dose increase in the Phase 3 PEGASUS trial of PNH patients with difficult-to-control disease Presenting Author: Morag Griffin Poster presentation Session title: Poster Session 2Session date: Saturday, 14 June Session time: 18:30 - 19:30 CEST Location: Poster Hall PS1665: Benefit of pegcetacoplan in patients with paroxysmal nocturnal hemoglobinuria irrespective of baseline transfusion status Presenting Author: Britta Höchsmann Primary Hemophagocytic Lymphohistiocytosis (pHLH) PF1036: Emapalumab in patients with primary hemophagocytic lymphohistiocytosis: Efficacy and safety outcomes from a multinational, open-label, single-arm study Presenting Author: Franco Locatelli Poster presentation Session title: Poster Session 1Session date: Friday, 13 June Session time: 18:30 - 19:30 CEST Location: Poster Hall About pegcetacoplan in rare diseases Pegcetacoplan is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, a part of the body’s immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is under investigation for rare diseases across haematology and nephrology. Pegcetacoplan is approved for the treatment of paroxysmal nocturnal haemoglobinuria (PNH) as EMPAVELI®/Aspaveli® in the United States, European Union, and other countries globally About Doptelet® (avatrombopag) Doptelet® (avatrombopag) is indicated for the treatment of primary chronic immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments, and a treatment of severe thrombocytopenia in adult patients with chronic liver disease (CLD) who are scheduled to undergo an invasive procedure. About Zynlonta® (loncastuximab tesirine)Zynlonta® (loncastuximab tesirine) is a CD19-directed antibody drug conjugate (ADC). Zynlonta as monotherapy is indicated for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and high-grade B-cell lymphoma (HGBL), after two or more lines of systemic therapy. About Sobi® Sobi is a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases. Sobi has approximately 1,900 employees across Europe, North America, the Middle East, Asia and Australia. In 2024, revenue amounted to SEK 26 billion. Sobi’s share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn. Contacts For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. We provide access to innovative treatments that transform life for people. Our therapies are concentrated within the areas of Haematology, Immunology and Specialty Care. We contribute to societies by improving access to treatment of rare diseases. Every day, we work actively to find better ways to understand and meet patient needs. Find out more about our business and financial performance. Here we present current and past media releases, images and videos.

临床3期免疫疗法临床结果上市批准临床1期

2025-06-02

·Pharmaceutical Technology

ASCO25: Amgen’s Nplate proves effective in GI cancer-linked thrombocytopaenia

A Phase III trial with Amgen’s Nplate met its endpoint for efficacy in treating chemotherapy-associated thrombocytopaenia for GI cancer patients. Photo by Bloomberg/Bloomberg via Getty Images Amgen’s Nplate (romiplostim) has demonstrated efficacy in treating thrombocytopaenia among gastrointestinal (GI) cancer patients, as Phase III RECITE results are presented at ASCO 2025. In the Phase III RECITE trial (NCT03362177), Nplate limited the modification of immunosuppressive therapies while improving platelet response compared to placebo, as per the abstract unveiled at the 2025 American Society of Clinical Oncology (ASCO) conference in Chicago, Illinois. The meeting is taking place from 30 May to 3 June. The RECITE trial enrolled 165 patients from 14 countries with colorectal, gastroesophageal, or pancreatic cancer who received oxaliplatin-based chemotherapy, resulting in persistent thrombocytopaenia. Participants were randomised 2:1 to be administered Nplate or placebo for three chemotherapy cycles; intravenous Nplate started at 2 μg/kg and was raised weekly by 1 μg/kg up to 10 μg/kg. The study found 84% of Nplate-treated patients achieved the trial’s primary endpoint, which evaluated the need for dose modification of myeloimmunosuppressive agents, compared to 36% of those given a placebo. Nplate was well tolerated, eliciting treatment-related adverse events (TEAEs) among 12% of patients versus 7% on placebo, but no TEAEs considered serious or leading to discontinuation were observed in either group. Nplate was first approved by the US Food and Drug Administration (FDA) in August 2008 to treat thrombocytopaenia in adults with chronic immune thrombocytopaenic purpura (ITP). It was later approved in December 2018 for paediatric patients with IPT unresponsive to other treatments. Then, in October 2019, the drug’s adult indication was expanded to cover newly diagnosed ITP patients who were also unresponsive to alternative treatments. GlobalData predicts Nplate will generate total global sales of $1.5bn in 2025. Amgen is scheduled to lose its exclusive US rights to the drug’s formulation on 12 February 2028. Annual sales are projected to fall to $1.1bn by 2031. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence GlobalData is the parent company of Pharmaceutical Technology . The therapy also significantly increased patients’ platelet nadirs, reaching a median low of 87×10 9 /L versus 58×10 9 /L for placebo. Nplate- and placebo-treated patients achieved platelet responses at 97% and 77%, respectively, with respective median times to first response of 1.1 weeks and 2.1 weeks. The abstract authors described these results as indicative of Nplate as a “potentially practice-changing” therapy for treating thrombocytopaenia, a routine and serious condition met by patients on chemotherapy. Chemotherapy-induced thrombocytopaenia is a common side effect of chemotherapeutic regimens, which lower the platelet count in the blood, weaken blood clotting, and raise the risk of internal bleeding. Pharmaceutical Technology Excellence Awards - Have you nominated? Nominations are now open for the prestigious Pharmaceutical Technology Excellence Awards - one of the industry's most recognised programmes celebrating innovation, leadership, and impact . This is your chance to showcase your achievements, highlight industry advancements , and gain global recognition . Don't miss the opportunity to be honoured among the best - submit your nomination today! Nominate Now

临床结果临床3期上市批准免疫疗法ASCO会议

2025-05-30

·ETPharma

Amgen blood platelet booster helps patients who need chemotherapy

London: Amgen's Nplate , which treats low blood platelet counts caused by an autoimmune condition, was highly effective at preventing a similar common and serious complication of powerful chemotherapy cancer drugs, researchers reported ahead of the American Society of Clinical Oncology meeting in Chicago. The 165 patients in the pivotal trial had either colorectal, gastroesophageal, or pancreatic cancer. They were all receiving toxic cancer drugs that caused them to have chemotherapy-induced thrombocytopenia - an abnormally low blood platelet count - after only a single session. A low platelet level "puts patients with cancer at a high risk of severe or potentially fatal internal bleeding," said study leader Dr. Hanny Al-Samkari of the Massachusetts General Hospital in Boston. It also "impacts the ability to treat patients with the indicated chemotherapy given as it should be, at full dose and on time," he added. Trial participants received either a weekly subcutaneous injection of Nplate, known chemically as romiplostim, or a placebo. Nplate acts by stimulating the bone marrow to increase platelet production. Of those in the Nplate group, 84% did not need any reductions of their subsequent chemotherapy doses due to a low platelet count, the researchers found. That was true for only 36% of patients in the placebo group. After taking individual risk factors into account, patients receiving Nplate were more than 10 times more likely than patients in the placebo group to continue their cancer treatments without a dose reduction. "Reduced chemotherapy intensity has been clearly shown to worsen survival of patients... reducing the likelihood of cure in curable patients and shortening the lives of incurable patients," Al-Samkari said. There are no FDA-approved drugs to treat chemotherapy-induced platelet loss, he noted. "These results are therefore potentially practice-changing for a common serious condition encountered routinely in clinical practice worldwide that prevents delivery of on-time, full-dose anti-cancer therapy," Al-Samkari said. AI IMPROVES PATIENT SELECTION FOR HER2 CANCER DRUGS Artificial intelligence can improve a doctor's accuracy at identifying breast cancer patients who could benefit from treatment with drugs that target the HER2 protein on cancer cells, researchers found in a study that will be presented at the ASCO meeting. These drugs, including AstraZeneca's Enhertu, are beneficial even when patients' levels of the protein are extremely low. However, low or ultra-low levels can be hard to detect, leaving some patients misclassified as HER2-negative. About 50% to 60% of breast cancer patients likely fall into the low-HER2 category, according to the U.S. National Cancer Institute. Willingness to use Enhertu to treat breast cancer has far outpaced the ability to determine with certainty which patients might benefit, experts have said. Funded by AstraZeneca, researchers worked with MindPeak to develop a training program to help pathologists assess HER2 levels in breast cancer. They recruited 105 pathologists from 10 countries to take the course and to analyze tumor samples from 20 patients, both with and without AI assistance. The pathologists' accuracy at determining HER2 clinical categories - null, ultra-low, low, or positive - improved from 66.7% without AI to 88.5% with AI. Misclassification of HER2-ultralow cases as HER2-negative occurred in 29.5% of readings without AI but decreased to 4.0% with AI assistance. "Our study provides the first multinational evidence that artificial intelligence can help enhance accuracy" in determining HER2 clinical categories, "potentially closing critical diagnostic gaps and enabling more patients to have access to new therapies, said Dr. Marina De Brot of the A.C. Camargo Cancer Center in Sao Paulo, Brazil, who led the study. "Until recently, most of these patients would have not been offered these options," she said. EXTRA PRE-TREATMENT MAY IMPROVE ESOPHAGEAL CANCER SURGERY OUTCOMES A cancer immunotherapy drug from China's BeiGene used in inoperable esophageal cancer may also change the way some operable esophagus tumors are treated, a mid-stage trial suggests. When patients with newly diagnosed esophageal cancer that has begun to grow beyond its original location have completed their standard pre-operative chemotherapy, a second course of treatment that includes BeiGene's Tevimbra given before surgery can significantly improve their outcomes, researchers reported in advance of ASCO 2025. Researchers recruited 87 such patients whose tumors were no longer seen on imaging exams after chemotherapy with paclitaxel and carboplatin, plus radiation. Ordinarily, the next step in their care would be surgery to remove part or all of the esophagus, at which point pathologists would examine the excised tissue to see if any cancer cells remained. Cancer cells lingering after surgery contribute to the more than 50% recurrence rate in these patients, the researchers said. Half the patients in the study received two additional treatments with chemotherapy and Tevimbra, while the rest went directly to surgery. When pathologists examined the tissues removed during surgery, 40.7% of Tevimbra-treated patients were free of any lingering cancer cells, compared with 18.1% of the control group. After accounting for individual risk factors, patients in the Tevimbra group were nearly three times more likely to have what is known as a pathological complete response. One year later, 95.6% of patients in the Tevimbra group were alive without their disease worsening, known as progression-free survival, compared with 77.3% of patients who did not receive the extra treatment before surgery. That difference was not deemed to be statistically significant, however. "This pioneering study demonstrates that sequential chemo-immunotherapy... significantly improves pathological complete response rates by more than two-fold and shows promising progression-free survival trends with manageable toxicity," the researchers said. They called for larger late-stage trials to confirm the results that could "redefine the standard-of-care." (Reporting by Nancy Lapid; editing by Bill Berkrot )

临床结果ASCO会议免疫疗法临床研究

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KEGG	Wiki	ATC	Drug Bank
D08990	罗米司亭	B02BX04	DB05332

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适应症	国家/地区	公司	日期
急性放射综合征	美国	Amgen, Inc.	2021-01-28
再生障碍性贫血	韩国	Kyowa Kirin Korea Co., Ltd.	2011-06-01
慢性特发性血小板减少性紫癜	日本	Kyowa Kirin Co., Ltd.	2011-01-21
免疫性血小板减少症	澳大利亚	Amgen Australia Pty Ltd.	2008-08-08
血小板减少症	澳大利亚	Amgen Australia Pty Ltd.	2008-08-08

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适应症	最高研发状态	国家/地区	公司	日期
乳腺癌	临床3期	美国	Amgen, Inc.	2020-02-26
乳腺癌	临床3期	阿根廷	Amgen, Inc.	2020-02-26
乳腺癌	临床3期	奥地利	Amgen, Inc.	2020-02-26
乳腺癌	临床3期	巴西	Amgen, Inc.	2020-02-26
乳腺癌	临床3期	保加利亚	Amgen, Inc.	2020-02-26
乳腺癌	临床3期	智利	Amgen, Inc.	2020-02-26
乳腺癌	临床3期	哥伦比亚	Amgen, Inc.	2020-02-26
乳腺癌	临床3期	希腊	Amgen, Inc.	2020-02-26
乳腺癌	临床3期	匈牙利	Amgen, Inc.	2020-02-26
乳腺癌	临床3期	墨西哥	Amgen, Inc.	2020-02-26

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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


NCT03362177 (ASCO2025) 人工标引	临床3期	血小板减少症	165	Romiplostim	願構範鑰衊遞廠願糧網(淵築膚網簾鏇鏇鹹夢簾) = 鬱鹹餘選憲構艱壓醖齋鏇衊構製簾衊鹹觸鏇廠 (衊膚顧鏇簾積觸鑰糧範 ) 达到更多	积极	2025-05-30
NCT03362177 (ASCO2025) 人工标引	临床3期	血小板减少症	165	Placebo	願構範鑰衊遞廠願糧網(淵築膚網簾鏇鏇鹹夢簾) = 願憲鏇願醖鏇選窪鬱淵鏇衊構製簾衊鹹觸鏇廠 (衊膚顧鏇簾積觸鑰糧範 ) 达到更多	积极	2025-05-30
PB3673 (EHA2025)	N/A	血小板减少症	168	Romiplostim N01	衊網鹽壓壓餘淵鬱壓範(夢網願願齋製觸網餘網) = 齋範積簾顧築鏇餘窪繭獵觸鏇齋觸鏇簾構鹽衊 (夢製鬱構製淵廠餘醖鬱 )	积极	2025-05-14
PB3680 (EHA2025)	N/A	免疫性血小板减少症二线	52	Romiplostim	衊窪觸壓遞襯襯廠襯鏇(範製襯獵構範繭壓鬱製) = 憲築夢選構觸艱鑰鏇襯積築鑰餘簾鏇選鑰艱築 (襯築餘製廠鑰顧窪壓淵 ) 更多	积极	2025-05-14
NCT02052882 (CTgov)	临床2期	血小板减少症	60	romiplostim	繭遞蓋顧簾積鏇鏇鬱獵 = 選繭願襯膚選顧繭構艱顧襯繭壓蓋鏇繭繭鑰夢 (鏇顧範夢鬱襯鬱糧築獵, 鬱築選鬱鹹築蓋網簾餘 ~ 蓋襯衊壓獵網鬱遞鏇衊) 更多	-	2024-12-27
ASH2024	N/A	难治性重度再生障碍性贫血	-	Romiplostim 20 μg/kg per week	醖夢夢簾獵鏇觸遞積鏇(壓簾簾鹽壓網衊糧廠窪) = 艱簾鬱鬱糧淵顧壓鹽鏇壓蓋鹽觸壓醖選鏇壓鏇 (範築襯製憲鏇壓製壓選 ) 更多	-	2024-12-09
ASH2024	N/A	免疫性血小板减少症	-	Eltrombopag	簾壓餘顧網範衊淵餘顧(顧蓋壓製簾遞壓夢積襯) = 鑰廠餘鹹網繭簾選壓鏇網蓋餘鑰觸獵鑰遞網積 (簾鹽網鑰顧襯遞糧範襯 ) 更多	-	2024-12-08
ASH2024	N/A	免疫性血小板减少症	-	Romiplostim	簾壓餘顧網範衊淵餘顧(顧蓋壓製簾遞壓夢積襯) = 衊憲鏇膚廠廠築鏇鏇顧網蓋餘鑰觸獵鑰遞網積 (簾鹽網鑰顧襯遞糧範襯 ) 更多	-	2024-12-08
ASH2024	N/A	免疫性血小板减少症	-	Eltrombopag	鹹餘憲觸簾齋觸鹽鹹鏇(遞鏇壓憲鬱網襯獵繭繭) = 襯鹹鏇鑰鏇鹽艱醖積獵襯網齋範餘憲壓艱網糧 (鑰遞構夢獵壓膚廠簾齋 ) 更多	-	2024-12-08
ASH2024	N/A	免疫性血小板减少症	-	Romiplostim	鹹餘憲觸簾齋觸鹽鹹鏇(遞鏇壓憲鬱網襯獵繭繭) = 網夢廠築膚衊廠窪壓夢襯網齋範餘憲壓艱網糧 (鑰遞構夢獵壓膚廠簾齋 ) 更多	-	2024-12-08
ASH2024	N/A	免疫性血小板减少症	-	Romiplostim < 3 mcg/kg	鹹遞構夢艱選窪壓夢衊(鬱鹽築鹹淵構構衊膚蓋) = Thrombosis after romiplostim is rare in the pediatric population. Both cases of thrombosis in this cohort were attributable to other secondary causes. 夢襯網淵夢糧鹹淵廠簾 (蓋選構窪願鬱襯廠積襯 )	-	2024-12-07
ASH2024	N/A	免疫性血小板减少症	-	Romiplostim ≥ 3 mcg/kg		-	2024-12-07
ASCO2024	N/A	实体瘤	-	Romiplostim	窪選範繭餘簾選繭繭襯(衊簾鏇築淵範顧鹽糧衊) = 齋構製製廠壓網壓製選築繭醖鹹窪鏇衊選繭鹽 (膚繭憲築構築網餘襯窪, 72.47 ~ 87.48)	积极	2024-05-24
CARMEN-France (EHA2024)	N/A	-	1,751	Romiplostim	艱膚鑰築願製鏇簾鬱積(築憲鹽壓壓淵築鹹網鹹) = 鏇選艱膚網窪獵蓋餘獵鏇膚選積餘襯構繭繭鬱 (顧構窪衊積糧糧築醖獵 ) 更多	积极	2024-05-14