Omalizumab

小李，28岁，因腹胀加重半年就诊。最初他以为是吃得多导致的“发胖”，尝试节食后腹部却越发突出，同时伴有疲乏无力、反复腹泻。他的面色苍白，皮肤略显蜡黄。多次外院检查均未发现明确原因，被建议“调整饮食，多锻炼”。最终，小李到当地三甲医院血液科就诊。查体发现，他的脾脏显著肿大，触及肋缘以下6厘米，且胸骨轻叩有压痛。血液检测提示白细胞、血小板显著减少，骨髓穿刺显示肥大细胞显著增多。进一步基因检测发现KIT D816V突变，最终确诊为一种由肥大细胞异常增殖引起的罕见疾病：系统性肥大增多症（SM）。什么是SM？肥大细胞增多症是异常肥大细胞在一个或多个组织器官中浸润导致的一种罕见的异质性肿瘤。根据第五版世界卫生组织（WHO）分类标准，肥大细胞增多症可分为三个疾病亚型——皮肤型肥大细胞增多症（CM）、系统性肥大增多症（SM）和肥大细胞肉瘤（MCS）。系统性肥大增多症（SM）是侵袭性肥大细胞在真皮组织以外的一个或多个组织、器官或系统的克隆性增殖，并释放大量的血管活性介质，引起多器官功能障碍，常见的受累部位包括骨髓、皮肤、脾脏、淋巴结、肝脏和（或）单核-巨噬细胞系统，约占肥大细胞增多症的10%。临床表现包括全身症状、肥大细胞活化症状（介质导致的症状）、相关疾病症状以及体征：（1）全身症状：发热、疲劳、体重减轻、盗汗等。（2）肥大细胞活化症状：①过敏反应；②乏力；③眩晕、晕厥/昏迷；④皮肤：面部、颈部和胸部皮肤潮红，瘙痒伴或不伴皮疹，荨麻疹伴或不伴血管性水肿；⑤胃肠：胃部不适、腹泻、恶心、呕吐、腹痛、腹胀、胃食管反流病；⑥神经精神：头痛、脑雾、认知障碍、焦虑、抑郁、记忆力减退、注意力不集中；⑦心血管：心率快、胸痛、低血压、高血压、血压不稳定；⑧呼吸：喘息和气短；⑨骨骼肌肉：骨骼肌肉疼痛、骨质硬化、骨质减少、骨质疏松；⑩鼻喉：鼻痒、鼻塞、喉咙发痒和肿胀。（3）相关疾病症状：部分患者存在血液系统疾病相关症状。（4）体征：肝肿大、脾肿大和淋巴结肿大等。分型SM可分为骨髓肥大细胞增多症（BMM）、ISM、冒烟型系统性肥大细胞增多症（SSM）、ASM、SM-AHN和MCL六个亚型。诊断SM需满足主要标准和1项次要标准或同时满足≥3项次要标准。治疗系统性肥大增多症（SM）的治疗以缓解肥大细胞活化症状、降低疾病负担为目标，具体措施包括以下几方面：1.抗介质治疗： 避免诱因，局部应用润肤霜或色甘酸钠，严重症状时使用抗组胺药、抗白三烯药物或皮质类固醇激素。2.预防性治疗： 针对慢性介质相关症状可逐步应用酮替芬、H1和H2受体阻断药、白三烯受体拮抗剂及质子泵抑制剂等。严重患者可能需要奥马珠单抗或糖皮质激素。3.急性过敏反应的处理： 系统性荨麻疹或过敏性休克需立即肌注肾上腺素，并根据情况重复注射，必要时静脉给药。4.骨质疏松治疗： 可根据骨病严重程度使用双膦酸盐或其他骨代谢相关药物。5.传统治疗： 羟基脲和干扰素-α可用于控制症状和减少肥大细胞负担，克拉屈滨对严重病例疗效显著。6.靶向治疗： 小分子激酶抑制剂（如阿伐替尼、米哚妥林）通过抑制KIT突变改善病情，特别适用于晚期SM患者。异基因造血干细胞移植：对于进展型SM（AdvSM）患者，allo-HSCT是唯一潜在的治愈手段，但需严格评估适应症。参考资料：1.  中华医学会血液学分会实验诊断学组，中国肥大细胞增多症协作网络. 成人系统性肥大细胞增多症诊断与治疗中国指南（2022年版）. 中华血液学杂志，2022，43(12):969-978. DOI:10.3760/cma.j.issn.0253-2727.2022.12.0012.罗睿恒，王二华，李昕,等. 急性髓系白血病合并系统性肥大细胞增多症1例. 中国临床案例成果数据库，2022，04(01):E07211-E07211. DOI:10.3760/cma.j.cmcr.2022.e07211来源 | 梅斯医学编辑 | wanny神经系统罕见病交流群↓点击下方“阅读原文”，下载梅斯医学APP吧！

CSU treatment evolves beyond anti-IgE biologics as novel drugs, biosimilars, and oral therapies reshape the competitive landscape Xolair leads but faces biosimilar pressure; Dupilumab, remibrutinib, and novel drugs like Barzolvolimab aim to reshape treatment with unique mechanisms.” — DataM Intelligence AUSTIN, TX, UNITED STATES, May 28, 2025 / EINPresswire.com / -- The chronic spontaneous urticaria (CSU) space is witnessing a pivotal transformation. Long recognized as a frustrating and poorly understood skin condition, CSU is now at the center of a therapeutic renaissance, fueled by cutting-edge biologics, advanced small molecules, and a sharpened industry focus on targeting non-responders and refractory cases. CSU is characterized by the sudden, recurrent appearance of hives, angioedema, or both, persisting for six weeks or more-often without any identifiable external trigger. Affecting nearly 1% of the global population, it tends to strike individuals in their prime years, most commonly between the ages of 20 and 40. While some experience symptoms for just a few years, others endure fluctuating episodes over a decade or more. Women are disproportionately affected, with incidence rates nearly double that of men. Book You CI Consultation Call for Free: https://www.datamintelligence.com/strategic-insights/ci/chronic-spontaneous-urticaria-csu This previously underserved market is now drawing heightened interest from global pharmaceutical leaders, driven by significant unmet needs and a growing patient base. Traditional antihistamines and the widely used anti-IgE biologic, omalizumab (Xolair), have been foundational treatments-but their limitations are increasingly apparent. A substantial subset of patients remain symptomatic despite these therapies, highlighting the urgent demand for more personalized, effective, and convenient options. A Therapeutic Landscape in Motion Approved therapies for CSU have evolved beyond antihistamines and Xolair, which remains the gold standard due to its robust real-world data and clinician trust. However, the competitive pressure is intensifying. A new wave of therapies is being designed to fill critical gaps, offering hope to patients who have historically had few alternatives. Sanofi and Regeneron’s Dupixent (dupilumab), already a blockbuster in other atopic diseases, is now being positioned for biologic-refractory CSU. With its mechanism targeting IL-4 and IL-13, it has demonstrated promise in addressing type 2 inflammation that plays a role in certain CSU patients. Meanwhile, Novartis is preparing to introduce Remibrutinib, a BTK inhibitor that represents a new class of oral small molecules in CSU. Designed specifically for antihistamine-refractory cases, this candidate could be a game-changer for patients seeking alternatives to injectables. And it doesn't stop there. Biotech innovators are developing therapies that target other key pathways in CSU pathogenesis, such as mast cell activation, JAK1/TYK2 inhibition, and the KIT receptor. Candidates like Barzolvolimab (Celldex), TLL-018 (Hangzhou Highlightll), and CMAB007 (Taizhou Mabtech) bring novel mechanisms of action that aim to redefine how CSU is managed across varying patient profiles. Biosimilars: Disruption with Access and Affordability The rise of biosimilars in the anti-IgE space is reshaping market dynamics. Players like Celltrion (OMLYCLO), Kashiv BioSciences (ADL-018), and Teva (TEV-45779) are introducing cost-effective versions of omalizumab, promising broader patient access without compromising efficacy. These products, many in advanced stages of regulatory approval, are likely to erode market share from branded biologics and increase pricing pressure across the board. This biosimilar wave is not merely a cost play. With proven interchangeability, favorable safety profiles, and growing physician confidence, they are expected to become integral components of treatment protocols, especially in cost-sensitive markets. Strategic Differentiation: The New Standard for Emerging Therapies Emerging CSU therapies must meet a higher bar than ever before. Speed of action, durability of response, convenience, and safety have become non-negotiable for success. According to analysts tracking the market, the most competitive agents will be those that can: • Deliver rapid relief (within 1–2 weeks) • Sustain remission over six months or more • Offer convenient oral or monthly dosing • Demonstrate efficacy in biologic non-responders • Maintain a clean safety profile with minimal immunosuppression • Target novel mechanisms beyond the conventional IgE and IL-4/13 pathways • Additionally, the rise of companion diagnostics and biomarker-driven personalization is expected to further optimize outcomes. By identifying likely responders in advance, developers can strengthen their value proposition to both clinicians and payers, accelerating adoption. Ask for Free Sample of Chronic Spontaneous Urticaria (CSU) Competitive Intelligence : https://www.datamintelligence.com/strategic-insights/sample/chronic-spontaneous-urticaria-csu The Competitive Pulse While Xolair continues to dominate with over a decade of physician loyalty and strong efficacy data, it now faces formidable competition. Dupixent and Remibrutinib are positioning themselves as high-efficacy alternatives, especially for patients not well controlled with current options. Meanwhile, first-in-class agents like Barzolvolimab are targeting mast cell pathways with the potential to establish entirely new treatment paradigms. Companies across North America, Europe, and Asia are racing to secure regulatory approvals, with several candidates in pre-registration phases in major markets like the U.S., EU, China, and the UK. This global expansion is expected to significantly widen the therapeutic footprint of CSU treatments over the next three years. Looking Ahead: Innovation Meets Opportunity The CSU market is entering a golden age of therapeutic innovation. As more targeted and differentiated agents reach commercialization, the outlook for patients is becoming increasingly hopeful. What was once a chronic, often invisible struggle is now drawing the attention of top-tier drug developers, regulatory bodies, and clinicians alike. The convergence of scientific discovery, commercial opportunity, and patient need is setting the stage for a transformative era in CSU management. Whether through best-in-class biologics, next-generation small molecules, or cost-effective biosimilars, the market is well-positioned for sustained growth and disruption. Final Word Chronic Spontaneous Urticaria is no longer an orphaned disease in the shadows. As the market evolves beyond anti-IgE monoclonals, new entrants with novel mechanisms and compelling value propositions are unlocking the next growth frontier. For stakeholders across the pharmaceutical value chain-developers, investors, clinicians, and patients-the CSU space has never been more dynamic or full of promise. About DataM Intelligence: DataM Intelligence 4Market Research LLP is a leading provider of Competitive Intelligence (CI) services, empowering life sciences and healthcare companies with real-time, actionable insights. We specialize in tracking competitor strategies, pipeline developments, clinical trials, regulatory updates (FDA/EMA), and market trends across key therapy areas and indications. Our offerings include CI insights, commercial analytics, opportunity analysis, KOL overviews, pricing and reimbursement strategies, social media listening, brand/LCM strategies, and market entry assessments. We also offer in-house tools, clinical trial databases, newsletters, and conference coverage to support your strategic decision-making. With deep industry expertise and a commitment to timely intelligence, we help you stay ahead in a fast-evolving global market. Read our Related CI Reports: 1. Frontotemporal Dementia | Competitive Intelligence 2. Obstructive Sleep Apnea | Competitive Intelligence Sai Kiran DataM Intelligence 4market Research LLP +1 877-441-4866 email us here Visit us on social media: LinkedIn X Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.