Pacritinib

Press releases News Image gallery YouTube Subscribe Sobi® (STO: SOBI), today announced its participation at the 65th American Society of Hematology (ASH) Annual Meeting, taking place on 6 – 9 December in Orlando, Florida. At this year’s meeting, Sobi will showcase its commitment to advancing care in haematology with 19 scientific abstracts, including two oral presentations. These feature the most recent clinical data and insights from completed and ongoing studies across Sobi’s innovative portfolio including data from efanesoctocog alfa, pegcetacoplan, avatrombopag, emapalumab, and pacritinib. These presentations underscore Sobi’s mission to deliver life-changing therapies for patients with rare and severe blood disorders. “We will present evidence at ASH from a post hoc analysis demonstrating that pacritinib reduces or stabilises spleen size, improves haematologic parameters, and lessens myelofibrosis symptoms in patients with highrisk disease. These findings matter because they address key drivers of morbidity which affect daytoday functioning and quality of life. The ASH meeting also offers an opportunity to discuss the latest research on emapalumab, efanesoctocog alfa, pegcetacoplan, avatrombopag, as well as loncastuximab tesirine,” said Lydia Abad-Franch, MD, Head of R&D and Medical Affairs, and Chief Medical Officer at Sobi. Summary of full Sobi data to be presented at ASH 2025: Efanesoctocog alfa Clinical outcomes up to four years of once-weekly Efanesoctocog Alfa Prophylaxis in previously treated adults, adolescents, and children with severe Haemophilia A: Interim analysis of the Phase 3 XTEND-ed long-term extension study. Oral Presentation Session Name: 322. Haemophilia A and B: Clinical and epidemiological: Innovations shaping the future of Haemophilia care Date: 7 December 2025 Time: 12:00 PM - 1:30 PM ET Presentation Time: 1:00 PM - 1:15 PM ET Room: OCCC – W304EFGH Publication Number: 539 Understanding unmet needs for people with Haemophilia A receiving factor and non-factor treatments. Poster Presentation Session Name: Poster Session I Date: 6 December 2025 Time: 5:30 PM - 7:30 PM ET Room: OCCC - West Halls B3-B4 Publication Number: 2679 Real-world experience of Efanesoctocog Alfa in Haemophilia A patients in the US: A retrospective analysis. Poster Presentation Session Name: Poster Session I Date: 6 December 2025 Time: 5:30 PM - 7:30 PM ET Room: OCCC - West Halls B3-B4 Publication Number: 1286 Patient characteristics, treatment patterns, and bleeding in people with Haemophilia A without inhibitors initiating Efanesoctocog alfa in the US: An administrative claims analysis. Poster Presentation Session Name: Poster Session I Date: 6 December 2025 Time: 5:30 PM - 7:30 PM ET Room: OCCC - West Halls B3-B4 Publication Number: 1290 Quality of life and functional improvements with Efanesoctocog alfa in patients with moderate to severe Haemophilia A: A real-world survey. Poster Presentation Session name: Poster Session III Date: 8 December 2025 Time: 6:00 PM - 8:00 PM ET Room: OCCC - West Halls B3-B4 Publication Number: 4846 Pegcetacoplan Consistent benefits of Pegcetacoplan treatment in PNH patients with and without a history of Aplastic Anaemia in real world: Analysis of the ongoing COMPLETE Phase 4 observational study. Poster Presentation Session Name: 101. Red Cells and Erythropoiesis, Excluding Iron: Poster I Date: 6 December 2025 Time: 5:30 PM - 7:30 PM ET Room: OCCC - West Halls B3-B4 Early results from the ongoing Pegcetacoplan Silo of the International Paroxysmal Nocturnal Haemoglobinuria Interest Group Registry. Poster Presentation Session Name: 508. Bone Marrow Failure: Acquired: Poster II Date: 7 December 2025 Time: 6:00:00 PM – 6:00:00 PM ET Location: OCCC - West Halls B3-B4 Real-world clinical characteristics and treatment outcomes in PNH patients prescribed Pegcetacoplan: Insights of complement inhibitor-experienced and -naïve patients across Europe, the United States and Canada. Poster Presentation Session Name: 508. Bone Marrow Failure: Acquired: Poster III Date: 8 December 2025 Time: 6:00 PM - 8:00 PM ET Room: OCCC - West Halls B3-B4 Pegcetacoplan - Publication Only Abstracts Real-world effectiveness of Pegcetacoplan in Paroxysmal Nocturnal Haemoglobinuria: A systematic review of clinical and patient-reported outcomes. Publication only - published online on 3 November 2025, at 9:00 AM ET Low risk for Meningococcal and encapsulated bacteria infections with systemically administered Pegcetacoplan in Paroxysmal Nocturnal Haemoglobinuria and C3 Glomerulopathies. Overview of treatment advances with complement Inhibitors in patients with Paroxysmal Nocturnal Haemoglobinuria. Optimising PNH treatment with the complement inhibitor Pegcetacoplan: A case report. User experience with Pegcetacoplan on-body Injector in patients with Paroxysmal Nocturnal Hemoglobinuria. Avatrombopag Real-world treatment patterns and outcomes among patients with immune thrombocytopenia (ITP) who switched treatment from Eltrombopag or Romiplostim to Avatrombopag in the United States: Results from the real-AVA 3.5 study. Poster Presentation Session Name: 905. Outcomes research: Non-malignant conditions excluding Hemoglobinopathies: Poster I Date: 6 December 2025 Time: 5:30 PM - 7:30 PM ET Room: OCCC - West Halls B3-B4 Real-world safety and efficacy of Avatrombopag in adults with Immune Thrombocytopenia: A systematic review and meta-analysis. Global Abstract Session Name: 311. Disorders of platelet number or function: Clinical and epidemiological: Poster I Date: 6 December 2025 Time: 5:30 PM – 7:30 PM ET Room: OCCC – West Halls B3-B4 Patient-reported outcomes of Avatrombopag for Chronic Immune Thrombocytopenia: Interim analysis of the Phase 4 ADOPT Study. Poster Presentation Session Name: 905. Outcomes research: Non-malignant conditions excluding Hemoglobinopathies: Poster III Date: 8 December 2025 Time: 6:00 PM - 8:00 PM ET Room: OCCC - West Halls B3-B4 Emapalumab Use of Emapalumab is associated with rapid and sustained benefits in pHLH subgroups, including CNS involvement and previously untreated patients: Pooled analysis of prospective trials NI-0501-04, NI-050105 and NI-050109. Poster Presentation Session Name: 201. Granulocytes, Monocytes, and Macrophages: Poster II Date: 7 December 2025 Time: 6:00 PM - 8:00 PM ET Room: OCCC - West Halls B3-B4 Emapalumab induces rapid, durable responses and reliable bridging to curative HSCT in patients with primary HLH: Pooled analysis of prospective trials NI-0501-04, NI-0501-05 and NI-0501-09. Poster Presentation Session Name: 201. Granulocytes, Monocytes, and Macrophages: Poster III Date: 8 December 2025 Time: 6:00 PM - 8:00 PM ET Room: OCCC - West Halls B3-B4 Pacritinib Real-world treatment patterns and outcomes in patients with myelofibrosis who presented with thrombocytopenia and anaemia at initiation of Pacritinib treatment. Oral Presentation Session Name: 908. Outcomes Research: Myeloid Malignancies: Real-World Experiences Session date: 7 December 2025 Session time: 4:30 PM - 6:00 PM ET Presentation time: 5:30 PM - 5:45 PM Room: OCCC - W414CD Pacritinib in patients with high-risk myelofibrosis: Outcomes from post-hoc analyses of two Phase 3 studies. Poster Presentation Session Name: 634. Myeloproliferative Syndromes: Clinical and epidemiological: Poster I Date: 6 December 2025 Time: 5:30 PM - 7:30 PM ET Room: OCCC - West Halls B3-B4 Real-world treatment patterns and clinical outcomes in patients with Myelofibrosis treated with Pacritinib (PAC): Results from the MY-PAC Study. Poster Presentation Session Name: 908. Outcomes research: Myeloid Malignancies: Poster II Date: 7 December 2025 Time: 6:00 PM - 8:00 PM ET Room: OCCC - West Halls B3-B4 PROSPERA (ABNL-MARRO 002): A randomised Phase 2 study of Pacritinib vs. Hydroxyurea in patients with Advanced Proliferative Chronic Myelomonocytic Leukaemia (CMML) Poster Presentation Session Name: 637. Myelodysplastic Syndromes: Clinical and epidemiological: Poster II Date: 7 December 2025 Time: 6:00 PM - 8:00 PM ET Room: OCCC - West Halls B3-B4 Treatment patterns and outcomes in patients with myelofibrosis treated with Pacritinib following a switch from Ruxolitinib: The MY-PAC Study. Session Name: 634. Myeloproliferative Syndromes: Clinical and epidemiological: Poster III Date: 8 December 2025 Time: 6:00 PM - 8:00 PM ET Room: OCCC - West Halls B3-B4 Incidence, prevalence, and clinical outcomes of Myelofibrosis with and without Cytopenia in the United States. About ALTUVOCT ® (efanesoctocog alfa) ALTUVOCT® (efanesoctocog alfa) is indicated for the treatment and prophylaxis of bleeding in patients with haemophilia A (HA). ALTUVOCT can be used for all age groups and any disease severity. About the Sobi and Sanofi Collaboration Sobi and Sanofi collaborate on the development and commercialisation of ALTUVOCT® (efanesoctocog alfa), or ALTUVIIIO™ in the US. Sobi has final development and commercialisation rights in the Sobi territory (essentially Europe, North Africa, Russia, and most Middle Eastern markets). Sanofi has final development and commercialisation rights in North America and all other regions in the world excluding the Sobi territory. About Aspaveli®/Empaveli® (pegcetacoplan) Aspaveli/Empaveli (pegcetacoplan) is a targeted C3 and C3b inhibitor designed to regulate excessive activation of the complement cascade, part of the body’s immune system, which can lead to the onset and progression of many serious diseases. Aspaveli/Empaveli is approved for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH) in the US, European Union, and other countries globally, and for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in the United States. It is under regulatory review for C3G and primary IC-MPGN in the European Union and other countries globally. About the Sobi and Apellis Collaboration Apellis and Sobi have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-U.S. commercialisation rights for systemic pegcetacoplan, and its opt-in rights for future development programs are unchanged, exercisable at any time prior to commercialisation. Apellis has exclusive U.S. commercialisation rights for systemic pegcetacoplan and worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy. 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 for the treatment of severe thrombocytopenia in adult patients with chronic liver disease (CLD) scheduled to undergo an invasive procedure. Doptelet is also approved for the treatment of chronic ITP in pediatric patients. About Gamifant® (emapalumab-lzsg) Gamifant is an anti-IFNγ antibody that binds free and receptor-bound IFNγ, which when secreted in an uncontrolled manner can cause hyperinflammation. Gamifant is indicated for intravenous infusion over one hour and is FDA approved for the treatment of primary hemophagocytic lymphohistiocytosis (HLH) and macrophage activation syndrome (MAS) in Still’s disease. About Vonjo® (pacritinib) Vonjo (pacritinib) is a kinase inhibitor indicated in the United States for the treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 10⁹/L. This indication is approved under accelerated approval based on spleen volume reduction. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials. Vonjo is also being investigated for other rare hematologic conditions, including VEXAS syndrome. 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. To stay up to date with our development, please subscribe to our press releases. Sign up We are a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases. 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Haematology Haemophilia Immune thrombocytopenia (ITP) Paroxysmal nocturnal haemoglobinuria (PNH) Diffuse large B-cell lymphoma (DLBCL) Haemophilia Immune thrombocytopenia (ITP) Paroxysmal nocturnal haemoglobinuria (PNH) Diffuse large B-cell lymphoma (DLBCL) Immunology Familial Mediterranean fever (FMF) Cryopyrin-associated periodic syndromes (CAPS) Haemophagocytic lymphohistiocytosis (HLH) Respiratory syncytial virus (RSV) Still’s disease Familial Mediterranean fever (FMF) Cryopyrin-associated periodic syndromes (CAPS) Haemophagocytic lymphohistiocytosis (HLH) Respiratory syncytial virus (RSV) Still’s disease Specialty Care Pipeline Congresses Research & development Investigator sponsored studies Managed Access Programmes Clinical studies and data privacy Investigator sponsored studies Managed Access Programmes Clinical studies and data privacy Medical Grant We contribute to societies by improving access to treatment of rare diseases. 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Our values Meet our employees Open positions About At a glance Focus on rare diseases Business strategy Partnership Our commitment Connect Disease awareness Patients as equal contributors Access programmes Clinical Trials Humanitarian programmes Nurture Capability building Trusted education source Patient Engagement in acquisitions Continuous and transparent communication Collaborate Define and deliver on shared objectives Informed decision making (IDM) Patient Engagement and experience data Innovate Innovate disease awareness campaigns Value added services History Board of directors Executive committee Corporate Governance General Meetings Nomination committee Committees Remuneration Auditors Articles of association Therapeutic areas Haematology Haemophilia Immune thrombocytopenia (ITP) Paroxysmal nocturnal haemoglobinuria (PNH) Diffuse large B-cell lymphoma (DLBCL) Immunology Familial Mediterranean fever (FMF) Cryopyrin-associated periodic syndromes (CAPS) Haemophagocytic 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Focus on rare diseases Business strategy Partnership Our commitment Connect Disease awareness Patients as equal contributors Access programmes Clinical Trials Humanitarian programmes Nurture Capability building Trusted education source Patient Engagement in acquisitions Continuous and transparent communication Collaborate Define and deliver on shared objectives Informed decision making (IDM) Patient Engagement and experience data Innovate Innovate disease awareness campaigns Value added services History Board of directors Executive committee Corporate Governance General Meetings Nomination committee Committees Remuneration Auditors Articles of association Partnership Connect Disease awareness Patients as equal contributors Access programmes Clinical Trials Humanitarian programmes Nurture Capability building Trusted education source Patient Engagement in acquisitions Continuous and transparent communication Collaborate Define and deliver on shared objectives Informed decision making (IDM) Patient Engagement and experience data Innovate Innovate disease awareness campaigns Value added services Disease awareness Patients as equal contributors Access programmes Clinical Trials Humanitarian programmes Capability building Trusted education source Patient Engagement in acquisitions Continuous and transparent communication Define and deliver on shared objectives Informed decision making (IDM) Patient Engagement and experience data Innovate disease awareness campaigns Value added services General Meetings Nomination committee Committees Remuneration Auditors Articles of association Haematology Haemophilia Immune thrombocytopenia (ITP) Paroxysmal nocturnal haemoglobinuria (PNH) Diffuse large B-cell lymphoma (DLBCL) Immunology Familial Mediterranean fever (FMF) Cryopyrin-associated periodic syndromes (CAPS) Haemophagocytic lymphohistiocytosis (HLH) Respiratory syncytial virus (RSV) Still’s disease Specialty Care Pipeline Congresses Research & development Investigator sponsored studies Managed Access Programmes Clinical studies and data privacy Medical Grant Haemophilia Immune thrombocytopenia (ITP) Paroxysmal nocturnal haemoglobinuria (PNH) Diffuse large B-cell lymphoma (DLBCL) Familial Mediterranean fever (FMF) Cryopyrin-associated periodic syndromes (CAPS) Haemophagocytic lymphohistiocytosis (HLH) Respiratory syncytial virus (RSV) Still’s disease Investigator sponsored studies Managed Access Programmes Clinical studies and data privacy Sustainability performance Key data Sustainability governance Code of Conduct Policies Governance Commitment to patients Access to treatment Patient centricity Humanitarian aid Act responsibly Caring for employees Environmental footprint Responsible sourcing Compliance Transparency Disclosure of Payments to Healthcare Trade association memberships Supporting patient organisations Key data Code of Conduct Policies Governance Access to treatment Patient centricity Humanitarian aid Caring for employees Environmental 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2025年11月6日至7日，第17届骨髓增殖性肿瘤国际大会（MPN 2025）在美国纽约布鲁克林成功举办。作为全球骨髓增殖性肿瘤（MPN）领域的年度学术盛会，本次会议汇聚了来自48个国家的约1100名参会者，会议以“推动MPN诊疗创新与全球协作”为核心，围绕MPN临床治疗突破、基础探索及新药研发展开深入研讨，为全球MPN领域的科研与临床实践提供了关键参考。中国学者团队在 哈尔滨血液病肿瘤研究所 马军教授 率领下参会，积极推动中国融入MPN国际协作网络，参与全球大规模临床试验，致力于通过国际合作实现我国MPN领域的突破与新药研发创新。 MPN 2025 一、骨髓纤维化（MF）：联合治疗突破与预后评估完善 （一）、药物治疗与疗效评估 MF的治疗以缓解症状、改善造血功能、控制脾脏肿大为核心，本次大会多项研究聚焦现有药物优化与新药研发。momelotinib的亚组分析（102#）评估了其对未接受过JAK抑制剂的贫血型MF患者（基线血红蛋白momelotinib组脾脏体积缩小≥35%（SVR35）率为31%；但基线血小板momelotinib组SVR35率为46%；momelotinib组27%的患者同时实现SVR35和无输血依赖（TI）；且 TI是momelotinib治疗患者总生存期（OS）的最强预测因子（HR=0.19，95% CI 0.06-0.65）。 另一项事后分析（103#）整合SIMPLIFY-1和MOMENTUM研究数据，显示：momelotinib治疗MF合并中重度贫血患者时，基线时中度贫血（血红蛋白8-10g/dL的比例（SIMPLIFY-1 中 69%、MOMENTUM 中 47%）高于重度贫血患者（SIMPLIFY-1 中50%、MOMENTUM中24%）；无论贫血严重程度，血红蛋白>10 g/dL的患者OS均更长，强调了早期干预贫血的重要性。 pacritinib的真实世界研究（108#）纳入35例初始血小板≥50×10⁹/L的MF患者，中位随访8个月发现：80%的患者仍在接受治疗；66.7%的脾脏肿大患者实现脾脏大小降低；血小板计数中位增加15%，17.4%的基线血小板 联合治疗方面，IMproveMF 研究（114#）评估了imetelstat联合芦可替尼治疗中高危MF 患者的Ⅰ/ⅠB期结果：19例患者接受imetelstat4.4-8.9 mg/kg治疗，血浆暴露量呈剂量依赖性；3/4级血细胞减少发生率低，无剂量限制性毒性；8.9 mg/kg剂量组71% 的患者人端粒酶逆转录酶（hTERT）表达降低≥50%，45%的患者驱动基因突变VAF降低≥20%；21%的患者实现症状应答，11%实现SVR35，为联合治疗提供了安全性与活性数据。 POIESIS 研究（105#）是一项Ⅲ期随机双盲研究，评估navtemadlin（MDM2抑制剂）联合芦可替尼治疗对芦可替尼应答不佳的MF患者，目前在23个国家254个中心招募，有望为应答不佳患者提供新策略。 塞利尼索（selinexor，XPO1抑制剂）的Ⅱ期研究（106#）显示：其单药治疗既往接受JAK抑制剂的MF患者时，33%实现SVR35，29%实现总症状评分降低≥50%（TSS50），且能稳定血红蛋白、减少输血，安全性良好；体外研究（107#）证实塞利尼索与JAK抑制剂协同抑制IL-6、TNFα等促炎细胞因子，为联合治疗提供机制支持。 （二）、临床特征与预后评估 早期MF的干预价值得到重视。一项回顾性研究（131#）纳入92例早期MF患者（纤维化前期或低危/中危-1 MF），46例接受IFN-α治疗，结果显示：IFN-α组总缓解率70%，中位OS（18年）显著长于对照组（14年，P ESC-B与Triage-4评分的预后价值研究（138#）纳入52例MF患者，发现：纤维化MPN 患者输血需求（38% vs 3%）和高症状负担（43% vs 19%）发生率显著高于非纤维化患者；ESC-B 评分（高症状负担或输血需求）鉴别纤维化与非纤维化MPN的相对风险为8.9（P=0.001）；Triage-4评分≥2分的患者中71%为纤维化MPN，ROC分析显示ESC-B和Triage-4的AUC分别为0.73和0.69，为早期识别纤维化提供了简单工具。 不同种族MF患者的预后研究（139#）纳入460例患者（白人65%、黑人22.2%），结果显示：年龄≥65岁是OS的独立不良因素（HR=1.93，P=0.028）；外周血原始细胞比例每增加1%，白血病转化风险增加37%（HR=1.37，P=0.005）；种族与OS无显著关联，为不同种族患者的预后评估提供参考。 （三）、基础研究与机制探索 单细胞多组学研究（132#）揭示了MPN患者对去甲基化药物（HMAs）的耐药机制：复发时未成熟造血干细胞比例增加，出现7q拷贝数异常克隆，且所有克隆均上调CYP1B1；体外模拟显示CYP1B1可结合磷酸化HMAs，可能通过代谢药物导致耐药，为逆转耐药提供靶点。 HMGA1 染色质调节因子的研究（129#）显示：其在JAK2-V617F MPN中过表达，驱动巨核细胞扩增、纤维化；Hmga1单等位基因缺失可减少JAK2-V617F小鼠造血干细胞异常扩增，预防纤维化；且HMGA1可诱导IFN-α信号，增强JAK2V617F造血干细胞对IFN-α的敏感性，为靶向HMGA1提供理论基础。 铁缺乏与MPN血栓风险的研究发现：铁缺乏通过增强缺氧诱导因子（HIF）活性，上调促血栓基因（SELP、F3）、下调抗血栓因子KLF2，增加PV/ET患者血栓风险；铁替代治疗可逆转这些变化，为血栓预防提供新策略。 二、真性红细胞增多症（PV）：治疗革新与疾病管理精细化 （一）、临床治疗与疾病管理 PV的治疗核心目标是控制红细胞压积（HCT） 放血治疗作为PV的基础治疗手段，其疗效与安全性仍需优化。一项基于美国 MarketScan数据库的研究（112#）纳入8482例仅接受放血治疗的PV患者，平均随访3.7年发现：90.6%的患者存在HCT控制不佳（部分或全部 HCT 测量值≥45%），62.7%的患者HCT≥50%；14.4%的患者发生血栓事件，9.1%发展为缺铁性贫血。频繁放血（6个月内≥3次或12个月内≥5次）的患者缺铁性贫血发生率（10.4%）显著高于非频繁放血患者（8.1%），但两组血栓风险无显著差异，提示单纯放血难以实现理想的HCT控制，且易导致铁缺乏，需联合其他治疗手段。 另一项针对高/低风险PV患者的真实世界研究（113#）纳入11311例患者（高风险51.8%、低风险48.2%），结果显示：即使接受当前标准治疗（放血+/-减细胞治疗），82.0%的高风险患者和90.3%的低风险患者仍存在HCT控制不佳；高风险患者血栓事件发生率（21.3%）、疾病进展率（6.1%）和缺铁性贫血发生率（11.0%）均显著高于低风险患者；发生血栓的患者12个月内全因医疗费用较高（高风险68332美元、低风险77867美元），进一步证实了PV现有治疗的局限性。 新药研发方面，rusfertide的Ⅲ期VERIFY研究（119#）为PV治疗带来突破。该研究纳入293例需频繁放血的PV患者，随机分配至rusfertide组（n=147）或安慰剂组（n=146）。结果显示：20-32周时，rusfertide组76.9%的患者达到临床应答（无需放血、未接受放血且完成32周治疗），显著高于安慰剂组（32.9%，P 罗培干扰素α-2b（ropeginterferon alfa-2b）的给药方案优化研究（124#）通过跨研究分析5项前瞻性试验数据，比较快速滴定（起始250μg，第2周350μg，第4周 500μg）与缓慢滴定（起始 100μg，每2周增50μg至500μg）方案的疗效。结果显示：0-32周内，快速滴定组中国患者平均年放血率0.67±2.35，韩国患者95%实现无放血；而缓慢滴定组欧洲患者平均年放血率3.81±4.33，仅29.3%实现无放血。维持期（20-32周）快速滴定组仍保持优势，且安全性与缓慢滴定组相当，为临床给药方案优化提供了依据。 （二）、疾病特征与预后因素 PV患者的铁缺乏问题及肾脏损伤风险也受到关注。REVEAL研究的事后分析（126#）纳入2510例PV患者，纵向监测铁缺乏标志物发现：57%的患者在研究期间出现铁缺乏，31% 的患者基线即存在铁缺乏；接受年度放血≥1次的患者铁缺乏发生率（77%）显著高于未放血患者（44%）。放血治疗给患者带来显著负担，每次放血平均耗时3-4小时（含交通、治疗及误工时间），提示需在放血与铁状态监测间寻找平衡。 肾脏损伤是PV的潜在并发症，一项结合REVEAL研究与动物模型的研究（127#）发现：24.5%的PV患者存在估算肾小球滤过率（eGFR）快速下降（>3mL/min/1.7m²/年）；血栓史、高风险PV（年龄≥60岁和/或血栓史）、基线乳酸脱氢酶（LDH）升高是eGFR快速下降的风险因素；eGFR快速下降的患者疾病进展率（14.4%）显著高于eGFR稳定患者（5.9%）。JAK2-V617F转基因小鼠模型显示，JAK2-V617F突变可导致肾小球系膜扩张和肾小管损伤，而 IFN-α治疗可减轻肾小管损伤，为临床干预提供了实验依据。 （三）、基础研究与机制探索 铁代谢异常是PV的关键机制之一。DISC-3405是一种靶向TMPRSS6的重组人源化抗体，可通过抑制TMPRSS6增加铁调素水平，减少铁吸收。其Ⅰ期研究（116#）在健康志愿者中显示：单剂量和多剂量给药均安全耐受，无严重不良事件；多剂量给药后，铁调素水平较基线增加100%-400%，血清铁降低50%-80%，血红蛋白和HCT下降5%-10%。目前，评估 DISC-3405在PV患者中疗效的Ⅱ期研究（NCT06985147）正在招募，有望为PV治疗提供新靶点。 另一项铁脉冲研究（115#）评估了DISC-3405对口服铁吸收的影响，结果显示：治疗后 1天口服铁吸收抑制率> 90%，15天后仍维持> 60%的抑制率，证实其通过诱导铁调素阻断肠道铁吸收的机制，为HCT控制提供理论支持。 三、原发性血小板增多症（ET）：传统治疗再评估与新药探索 （一）、临床治疗与疗效评估 ET的治疗目标是控制血小板计数、预防血栓/出血，本次大会多项研究聚焦新药与现有药物优化。bomedemstat，LSD1抑制剂的Ⅲ期Shorespan-007研究正在招募未接受减细胞治疗的ET患者，计划纳入300例，比较其与羟基脲的疗效，主要终点为52周持久临床血液学应答，有望为ET提供新选择。 羟基脲的回顾性研究纳入634例一线羟基脲治疗的ET患者，中位随访36个月发现：30.8%的患者发生并发症（血栓24.4%、出血3.9%、进展5.4%）；46.7%的患者需治疗调整（中断35.0%、停药18.1%）；12个月时30.6%的患者血小板≥600×10⁹/L，51.3%发生血细胞减少，提示羟基脲疗效有限，需新药补充。 INCA033989（突变钙网蛋白特异性单抗）的Ⅰ期研究（125#）纳入49例CALR突变、既往治疗耐药/不耐受的ET患者，结果显示：无剂量限制性毒性，96%的患者仍在治疗；83% 实现血液学应答（完全应答74%）；89%的患者CALR VAF降低，47%降低≥20%，为CALR突变ET患者提供精准治疗选择。 （二）、基础研究与机制探索 家族性MPN的研究（140#）报道一个家系：患者（38岁）存在JAK2- V617F（VAF 1%）和PAX5变异（VAF 51.9%），其父（71岁）存在CALR突变（VAF 55.9%）和PAX5变异（VAF 49.4%），诊断为MF；提示家族性MPN可能存在不同驱动突变，PAX5变异可能为遗传易感因素，强调家族性MPN患者基因筛查的重要性。 四、慢性髓系白血病（CML）研究 儿童CML研究（141#）纳入88例CML-CP患者，发现：3个月时BCR-ABL1≤10%的患者12个月MMR率（80.8% vs 5.6%）和24个月DMR率（70.4% vs 42.2%）显著更高；骨髓纤维化与MMR发生率降低相关，提示早期 BCR-ABL1动态可预测儿童CML应答。 GeneXpert在塞内加尔儿童CML中的应用研究（143#）纳入49例疑似患者，39例（79.59%）确诊，20例随访中1例实现稳定MMR，但因仅检测 p210 转录本可能遗漏儿童常见的p190，需结合其他检测。 五、特殊人群MPN研究进展 妊娠管理研究（130#）纳入110例MPN患者的150次妊娠，结果显示：ET患者妊娠结局最佳（无并发症23.7%、流产15.7%、活产80.2%），PV/PMF患者并发症更多；治疗以IFN-α联合低分子肝素/阿司匹林为主，为MPN患者妊娠管理提供参考。 青少年和年轻成人（AYA）MPN研究（135#）纳入40例13-39岁患者（ET 52.5%、PV 27.5%），发现：70%偶然确诊，60%有心血管危险因素，25%为高风险；45%接受罗培干扰素治疗，10 例实现完全应答，症状负担低（MPN-SAF平均6.7分），提示AYA患者需重视心血管风险，罗培干扰素疗效良好。 六、MPN评估与检测技术 MPN患者（因未满足需求）评估（101#）纳入627例受访者，发现：仅10%参与临床试验，71%未被告知试验选项；疲劳（71%）、瘙痒（55%）是常见症状，72%未听说MPN 10 症状追踪工具；患者治疗优先级为延缓进展（80%）、改善生活质量（52%），但仅54%的患者治疗目标被考虑，提示需加强医患沟通与患者教育。 JAK2-V617F检测技术研究（136#）开发超选择性PCR方法，以mRNA为靶点，兼具高灵敏度与特异性，成本低、速度快，适用于临床诊断与动态监测。IFN应答性别二态性研究（137#）显示：雌性JAK2V617F小鼠对IFN-α的造血干细胞抑制作用更强，可能与女性免疫系统IFN应答更强相关，为个体化治疗提供依据。 七、总结与展望 MPN 2025大会全面展示了MPN领域的进展，MF治疗中momelotinib、pacritinib及联合治疗探索，PV中rusfertide、罗培干扰素方案优化，ET中INCA033989等新药研发，均为临床提供新选择；基础研究揭示了HMGA1、CYP1B1 等新靶点；特殊人群管理研究为 AYA、妊娠患者提供参考。 中国学者积极参与国际协作，推动我国MPN临床试验与新药研发，优化诊疗策略。随着机制研究深入与新药发展，MPN患者的治疗前景将更广阔，有望实现疾病有效控制，改善患者生存质量。 编辑：Cherry 审核：马军教授 排版：Baa 执行：Cole 医脉通是专业的在线医生平台，“感知世界医学脉搏，助力中国临床决策”是平台的使命。医脉通旗下拥有「临床指南」「用药参考」「医学文献王」「医知源」「e研通」「e脉播」等系列产品，全面满足医学工作者临床决策、获取新知及提升科研效率等方面的需求。 本平台旨在为医疗卫生专业人士传递更多医学信息。本平台发布的内容，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。如该等信息被用于了解医学信息以外的目的，本平台不承担相关责任。本平台对发布的内容，并不代表同意其描述和观点。若涉及版权问题，烦请权利人与我们联系，我们将尽快处理。