An Open Label Study to Investigate the Long-term Safety and Efficacy of Belimumab Administered Subcutaneously in Adults With Systemic Sclerosis Associated Interstitial Lung Disease (SSc-ILD)

This is an open label extension (OLE) study of an ongoing randomized controlled clinical study 218224 (Parent Study). The OLE study will describe how well tolerated belimumab will be long term, and whether it might continue to slow progression of lung function decline, slow overall disease progression and improve quality of life.

开始日期2024-12-12

申办/合作机构

GSK Plc

[+1]

NCT06381453

/ Recruiting临床2期IIT

Belimumab in the Management of Autoimmune Hepatitis: a Multi-centre, Open-label Trial of Add-on Belimumab Therapy to Standard of Care

Background: Autoimmune hepatitis (AIH) is a rare chronic and lifelong liver disease. Untreated, disease progresses to end-stage cirrhosis and the focus of therapy is with immunosuppression. Current therapies are limited, not targeted, and associated with side effects that patients report reduce quality of life. AIH is believed to arise as a consequence of genetic & environmental risks. Disease is characterised by impaired immunoregulation, that favours a chronic and relapsing hepatitis. As well as recognising an important role for cytotoxic T cells and regulatory T cells, it has become apparent that in AIH, as well as other related autoimmune conditions, that B-cells are important. AIH is characterised by a plasma cell rich interface hepatitis and elevated IgG concentrations. Furthermore B-cell lineages interact with regulatory T-cells. Off-label use of Rituximab, an anti-CD20 agent, has been described for patients with AIH. A number of other ways of effectively targeting B-cells in the treatment of related autoimmune diseases have also been developed, but there have been limited studies in people living with autoimmune hepatitis. Belimumab is a human monoclonal antibody that inhibits B-cell activating factor (BAFF), also known as B-lymphocyte stimulator. It is approved in the Canada to treat systemic lupus erythematosus and lupus nephritis. It has not been studied before in AIH, but off-label reports are published. In an open-label clinical trial of people living with autoimmune hepatitis, the investigator will now formally study the effect of adding Belimumab to existing standard of care, with the goal being to evaluate treatment efficacy, the ability to reduce the burden of existing therapies whilst still controlling AIH disease, and to describe the tolerability & safety of Belimumab in people with AIH. Study Design: Open label, multi-centre, Canadian clinical trial. Patient population: Patients with autoimmune hepatitis, excluding patients with decompensated liver disease, who either have active disease despite standard of care (Group A), or who are maintained with disease remission using standard of care therapy (Group B). 48 patients will be recruited. Intervention: Weekly sub-cutaneous Belimumab. Duration: 72 weeks with interim analysis after 24 patients have been treated for 24 weeks; target recruitment 48 patients. Evaluation: Safety, Serum liver tests, quality of life, exploratory immunologic biomarkers, optional liver biopsy or fine needle liver aspirate. Primary end-point: Group A: 50% or more of subjects have an ALT<2x ULN & corticosteroids at a dose of

开始日期2024-12-11

申办/合作机构

University Health Network

[+1]

NCT06572384

/ Recruiting临床3期

A Phase 3, Randomized, Double-Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Belimumab Administered Subcutaneously in Adults With Interstitial Lung Disease (ILD) Associated With Connective Tissue Disease (CTD)

Interstitial lung disease (ILD) is a lung condition resulting in inflammation and stiffening of the lung, often associated with connective tissue diseases (CTDs). ILD causes reduction in lung volume, shortness of breath, cough and fatigue therefore has high impact on quality of life and is also the leading cause of death in participants with these conditions. The study will assess whether treatment of CTD-ILD participants with belimumab in addition to standard therapy will result in the stabilization and/or improvement of lung function and improve symptoms associated with ILD with an acceptable safety profile.

开始日期2024-09-11

申办/合作机构

GSK Plc

100 项与贝利尤单抗相关的临床结果

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100 项与贝利尤单抗相关的转化医学

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100 项与贝利尤单抗相关的专利（医药）

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1,316

项与贝利尤单抗相关的文献（医药）

2025-08-01·SEMINARS IN ARTHRITIS AND RHEUMATISM

Infectious complications of Belimumab with standard care in systemic lupus erythematosus: a systematic review and meta-analysis

Review

作者: Wei, James Cheng-Chung ; Fan, Yongsheng ; Chi, Gerald ; Ma, Chenhang ; Wang, Xinchang ; Xia, Shujun ; Wang, Weijie ; Zhao, Yu

OBJECTIVES:

We aimed to evaluate the risk of infectious complications of Belimumab with standard care in systemic lupus erythematosus (SLE).

METHODS:

We searched PubMed, Web of Science, EMBASE, and Cochrane databases for studies on SLE and Belimumab and evaluated the study quality using the Cochrane Collaboration tool (ROB 2). A random-effect model was employed to analyze the results. To evaluate publication bias, Egger's tests were used. We also performed subgroup analysis based on the Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) and British Isles Lupus Assessment Group(BILAG). The study protocol has been registered in PROSPERO (CRD42023421255).

RESULTS:

Eight studies involving 8545 patients were included, of which four studies had a high attrition bias. The present study suggested that the risks of infectious complications including infection (RR=1.02, 95 %CI=(0.97,1.08), I²=7.7 %) or serious infections (RR=0.94, 95 %CI=(0.77,1.15), I²=0 %), nasopharyngitis(RR=1.02, 95 %CI=(0.79,1.33), I²=36 %), upper respiratory tract infection(RR=0.97, 95 %CI=(0.83,1.14), I²=20.6 %), urinary tract infection(RR=1.09, 95 %CI=(0.91,1.32), I²=0 %), herpes zoster (RR=0.75, 95 %CI=(0.54,1.05), I²=0 %)and influenza(RR=0.98, 95 %CI=(0.69,1.39), I²=0 %) were not significantly increased in patients who received Belimumab with standard care, except for bronchitis (RR=1.51, 95 %CI=(0.98,2.33), I²=23.7 %). Additionally, the subgroup analysis of SLEDAI and the proportion of patients with BILAG 1A/2B did not show any significant impact on infectious complications.

CONCLUSION:

Meta-analysis results demonstrated that intravenous (IV) Belimumab(≤10 mg/kg), along with standard care, did not significantly increase the risk of infectious complications in SLE patients having mild-to-moderate disease activity, except for bronchitis. Baseline disease activity and organ damage did not impact the risk of infectious complications.

2025-07-01·JOINT BONE SPINE

Comparative efficacy and safety of anifrolumab for belimumab-experienced and biologic-naïve patients with systemic lupus erythematosus: Results from the Kyushu Collagen Disease Network for Systemic Lupus Erythematosus (KCDN-SLE) registry

Letter

作者: Maekawa, Makiko ; Ueda, Naoyasu ; Tanaka, Atsushi ; Ayano, Masahiro ; Kushimoto, Kazuo ; Mishima, Koji ; Tsuru, Tomomi ; Doi, Goro ; Akashi, Koichi ; Akahoshi, Mitsuteru ; Ota, Shun-Ichiro ; Nakayama, Tsuyoshi ; Niiro, Hiroaki ; Horiuchi, Takahiko

2025-07-01·AUTOIMMUNITY REVIEWS

Dual B-cell targeting in systemic lupus erythematosus: The role of combined and sequential therapy with rituximab and belimumab

Review

作者: Frade-Sosa, Beatriz ; Gómez-Puerta, José A ; Arnaud, Laurent ; Sarmiento-Monroy, Juan C ; Bruce, Ian N

Systemic lupus erythematosus (SLE) is a complex autoimmune disease characterized by immune dysregulation and autoantibody production. Despite advances in treatment, achieving sustained disease control remains challenging. Rituximab (RTX) and belimumab (BELI) are two B-cell-targeting biologics with complementary mechanisms of action, leading to increasing interest in their combination as a therapeutic strategy for refractory SLE. RTX depletes CD20+ B cells, whereas BELI inhibits B-lymphocyte stimulator (BLyS), reducing the survival of autoreactive B cells. Sequential therapy with these agents may mitigate B-cell repopulation and improve disease control. Recent studies, including SynBioSe and BEAT-LUPUS, suggest that RTX-BELI therapy can reduce autoantibody levels, neutrophil extracellular trap formation, and disease activity, with many patients achieving a lupus low disease activity state (LLDAS). However, the BLISS-BELIEVE and CALIBRATE trials did not demonstrate superiority over monotherapy, highlighting the need to refine patient selection. Combination therapy may be particularly beneficial in lupus nephritis, where BELI delays autoreactive B-cell reconstitution following RTX, potentially prolonging remission. While RTX-BELI therapy is generally well-tolerated, some studies report increased infections, necessitating careful patient monitoring. Lessons from other immune-mediated diseases, including inflammatory bowel disease and rheumatoid arthritis, underscore the potential benefits and risks of dual biologic therapy. Further research, including the ongoing SynBioSe-2 trial, is needed to clarify the optimal use, sequencing, and safety profile of RTX-BELI in SLE. Identifying biomarkers predictive of response may enable personalized treatment approaches, ultimately improving long-term outcomes for patients with refractory SLE.

203

项与贝利尤单抗相关的新闻（医药）

2025-06-20

·FiercePharma

Europe's CHMP gives thumbs up to ExCellThera's blood cancer cell therapy Zemcelpro

Europe’s Committee for Medicinal Products for Human Use has recommended marketing authorization for ExCellThera’s novel cell therapy Zemcelpro in certain blood cancer patients. In a first for the bloc, Europe’s Committee for Medicinal Products for Human Use (CHMP) has recommended marketing authorization for a new type of stem cell transplant therapy for certain blood cancer patients. The marketing endorsement was awarded to ExCellThera’s Zemcelpro and covers a recommended approval in adults with blood cancers that require an allogeneic hematopoietic stem cell transplantation (HSCT) following myeloablative conditioning for whom no other type of suitable donor cells is available.Zemcelpro contains stem cells from a donor’s umbilical cord blood, some of which have been grown and multiplied, the European Medicines Agency (EMA) said in a press release on the decision. By boosting the number of cells, Zemcelpro makes the stem cells from a small cord blood unit more effective, the European regulator explained. For several blood cancers—including leukemias, which are detected in the blood; lymphomas, which are found in the lymph nodes; and myelodysplastic syndrome and myelomas, which are diagnosed in the bone marrow—the only current curative treatment option is allogeneic HSCT. This type of transplant involves using donated stem cells to replace the patient’s bone marrow cells to form new marrow that produces healthy blood cells. The CHMP based its Zemcelpro recommendation on pooled analysis of two single-arm trials that included 25 patients. The studies found that 84% of patients who received Zemcelpro achieved neutrophil engraftment, when donor stem cells establish themselves in the recipient’s bone marrow and produce the white blood cells known as neutrophils within a median span of 20 days.Some 10,000 new blood cancer cases requiring bone marrow transplant crop up in Europe each year, ExCellThera estimates. Many of these patients do not have access to suitable donor cells largely because of the unavailability of matched donors, the company explained. ExCellThera said it expects to gain marketing authorization from the EMA in roughly two months' time. After winning an endorsement from the CHMP, prospective medicines must go before the European Commission for a final approval verdict.ExCellThera also plans to file for approval of Zemcelpro in the U.S., U.K. and other countries. Madrigal’s Rezdiffra among others gaining CHMP nodsThe committee has also given a thumbs up to Madrigal’s Rezdiffra for the treatment of adults with noncirrhotic metabolic dysfunction-associated steatohepatitis (MASH), a disease where fat cells accumulate in the liver causing chronic inflammation. If the drug gains marketing authorization, it would become the first treatment in the EU for the condition. Fifteen months ago, Rezdiffra became the first drug approved for MASH in the U.S. Also making the grade this month was Teva blockbuster Austedo for patients with moderate-to-severe tardive dyskinesia. The FDA approved the treatment in the indication eight years ago. Tardive dyskinesia is an irreversible movement disorder characterized by repetitive and uncontrollable movements of the tongue, lips, face, trunk and extremities and can be caused by medications used to treat mental health or gastrointestinal conditions.Meanwhile, Pfizer spinout SpringWorks Therapeutics has picked up a CHMP nod for Ogsiveo in adults with progressing desmoid tumors, which are marked by noncancerous soft tissue growths in the limbs and abdomen that often cause severe pain and disfigurement. The FDA approved Ogsiveo in the same indication in 2023.Also gaining a positive recommendation from the CHMP was Partner Therapeutics’ Imreplys for the treatment of patients exposed to myelosuppressive doses of radiation with hematopoietic sub-syndrome of acute radiation syndrome. The medicine is known as Leukine in the U.S. where it was approved in 2018. CHMP signs off on label expansions for 6 drugsAside from new approval recommendations, the CHMP has also endorsed labeled expansions for a half-dozen established medicines. The committee gave a thumbs up to Ipsen’s Cabometyx as a second-line treatment for patients with unresectable or metastatic, well differentiated extra-pancreatic (epNET) and pancreatic (pNET) neuroendocrine tumors. The FDA signed off Cabometyx in the indication three months ago. The treatment, which has been on the market since 2016, brought home sales of $1.8 billion last year.Also winning CHMP recommendations were Johnson & Johnson blockbusters Darzalex, for smoldering multiple myeloma, and Imbruvica, for patients with previously treated mantle cell lymphoma who would be eligible for autologous stem cell transplant. AbbVie partners with J&J on Imbruvica.The committee further signed off on expansions for GSK’s lupus treatment Benlysta, Bayer’s prostate cancer drug Nubeqa and Sanofi’s multiple myeloma medicine Sarclisa. CHMP opens re-examination of Lilly’s KisunlaElsewhere, fifteen months after giving a thumbs down to Eli Lilly’s Alzheimer’s disease treatment Kisunla in March of last year, the CHMP will give the medication a second look. The regulator said that it re-opened its examination of Kisunla on June 2. The FDA signed off on Kisunla in July of last year, opening up competition with Eisai and Biogen’s Alzheimer's rival Leqembi.

上市批准细胞疗法临床结果

2025-06-17

·小药说药

自身免疫和炎症性疾病的新治疗策略

-01-引言免疫介导的炎症性疾病(IMID)是一组常见的、临床多样化的疾病，包括类风湿性关节炎(RA)、脊柱关节炎(SpA)、结缔组织疾病、皮肤炎症性疾病（包括银屑病和特应性皮炎）、炎症性肠病(IBD)、哮喘和自身免疫性神经系统疾病如多发性硬化症。1948年首次使用可的松治疗RA是IMID治疗史上最重要的进展之一。在20世纪下半叶，迅速发展的制药工业使几种常规免疫抑制剂得以实现化学合成，如环磷酰胺、甲氨蝶呤、硫唑嘌呤、环孢菌素、他克莫司和霉酚酸酯。随后的研究证实了几种免疫靶点的临床相关性，如IL-1、IL-6、IL-17、IL-23、B淋巴细胞刺激因子（BLyS）和IFN-α受体、细胞表面标记物如CD20和CD52、共刺激分子如细CTLA-4和PD-1，以及信号通路分子如Janus激酶（JAK）和酪氨酸激酶2（Tyk2），一系列不同的靶向治疗和免疫治疗获得了监管部门的批准。然而，并非所有IMID患者对靶向治疗都有反应，许多患者在治疗后有复发。因此，迫切需要在IMID领域积极开发新的治疗策略，如双特异性抗体（BsAbs）、纳米抗体和纳米颗粒、治疗性疫苗、siRNA干扰、自体造血干细胞移植（aHSCT）和CAR-T细胞。尽管这些新的治疗策略中的大多数仍在临床前和临床中进行评估，但它们已经在未来改善IMID患者方面展现出良好的前景。-02-一、靶向治疗的联合应用两个或多个互补途径可以同时或依次靶向，首先诱导自身免疫性疾病缓解，然后恢复免疫耐受状态，防止疾病过程的再激活。例如B细胞活化因子（BAFF）在利妥昔单抗诱导的B细胞清除后表达上调，导致B细胞活化和存活，基于这种机制，一些联合应用已经在不同适应症中进行测试，如系统性红斑狼疮（SLE）、干燥综合征或系统性硬化症。2021年公布的两项III期试验结果表明，在干燥综合征中，belimumab和利妥昔单抗的联合治疗显示，随着时间的推移，EULAR干燥综合征疾病活动指数（ESSDAI）和刺激唾液流有改善的趋势，这一趋势在治疗后持续存在。而在另一项SLE的临床实验中，belimumab和利妥昔单抗的联合应用没有显著提高SLE患者的疾病控制率。此外，在这两项研究中，与单独的belimumab和利妥昔单抗相比，没有发现额外的安全问题。最近，法国发表了一项大型前瞻性多中心队列研究的结果，该研究调查了143名IMID患者的150种靶向治疗组合。患者主要患有炎症性肠病（77.6%）、脊椎关节炎（44%）和RA（9.1%），其中一半患者患有两种或两种以上联合IMID。最常见的靶向治疗组合是TNFi联合Vedolizumab（30%）或Ustekinumab（28.7%）。从结果来看，50%的患者报告的结果得到了显著改善，27%的患者获得了轻度至中度改善。联合治疗期间的严重感染估计为4.51/100每患者年，1年后未中断治疗的维持率估计为74.9%。-03- 二、双特异性抗体在自身免疫性疾病中，BsAbs的治疗作用主要涉及通过靶向表面标记物或中和炎性细胞因子来预防幼稚T细胞与抗原呈递细胞（APC）或B淋巴细胞之间的免疫相互作用。这些靶点集中于B细胞（CD19、FcγRIIb、BAFF、B7RP1、CD32B和CD79B）或APC（CD86）上的表面受体，以及促炎T细胞调节因子（IL-1、IL-4、TNF-α、IL-13和IL-17）。关于IMID，到目前为止，已有十几种BsAb在临床试验中进行了评估。第一种被批准用于治疗IMID的双特异性单克隆抗体是bimekizumab，它于2021年被美国食品药品监督管理局（FDA）批准用于治疗成人中重度斑块状银屑病。Bimekizumab靶向两种细胞因子，IL17A和IL17F，它们参与银屑病的炎症过程。通过阻断IL17A和IL17F，与以前仅针对IL17A的治疗相比更有效。Tibulizumab (LY3090106)是礼来开发的四价双特异性抗体，包含两种独立作用的二价抗体，靶向BAFF和IL17A；AMG570靶向诱导型T细胞共刺激配体（ICOSL）和BAFF；bsHexAb是一种六价双特异性抗体，靶向CD20和CD22；MT-6194是一种同时靶向IL17A和IL6R的BsAb。-04-三、纳米抗体纳米抗体相比单克隆抗体在生产、结构、溶解度以及对pH和温度范围的耐受性方面具有额外的优势，很可能成为IMID靶向治疗的下一个重要领域。到目前为止，已经在临床试验中评估了十多个纳米抗体。Ozoralizumab（ATN-103/TS-152）是一种三价双特异性纳米抗体，可有效中和TNFα并与人血清白蛋白结合以增加其体内半衰期。它包括两个抗TNFα纳米抗体和一个白蛋白结合纳米抗体。最近公布了其在RA中的II/III期和III期临床试验（OHZORA和NATSUZORA研究）的结果。结果显示，与安慰剂组相比，Ozoralizumab达到主要终点（第16周ACR20改善）的患者比例显著增高（79.6% vs 37.3%）。2022年9月，日本批准了Ozoralizumab用于治疗对现有治疗反应不充分的RA患者。 -05-四、造血干细胞移植aHSCT在自身免疫性疾病中的应用在于自身反应性免疫细胞清除后，从aHSC产生新的自我耐受免疫系统。这一程序有可能通过重置免疫系统来改变自身免疫性疾病的进程，建立一个具有恢复免疫检查点的新免疫库。aHSCT在20世纪90年代末首次用于SSc。三项随机对照临床试验的结果显示，aHSCT与第一年治疗相关死亡率的增加有关。然而，aHSCT具有显著的长期无事件生存益处，并显著改善了SSc中的几个器官受累。此外，在最近的研究中，aHSCT在治疗难治性狼疮肾炎（LN）方面似乎是有效和安全的。在该研究中，22名难治性LN患者接受了aHSCT治疗，随访期间没有患者死亡，10年无病生存率为35%，10年后只有10%的患者需要肾脏支持。在另一项研究中，8名难治性SLE患者接受了aHSCT治疗，5例为LN，3例为中枢神经系统受累。八名患者中有五名获得了完全缓解，包括系统性红斑狼疮疾病活动指数（SLEDAI）降至零，四名患者的缓解持续时间中位数为165个月；一名患者在18个月后出现部分缓解，随后复发。此外，两名患有肾炎和潜在合并症的患者因感染和多器官衰竭死亡。-06-五、CAR-T细胞治疗在IMID的治疗应用中，与单克隆抗体相比，CAR-T细胞有特别的优势：因为它们是长寿命细胞，可以增殖、运输到淋巴组织或靶器官，并发展成记忆群体，可以防止致病淋巴细胞的再次出现。因此，CAR-T细胞治疗可能只需要单一剂量就能达到持久的治疗效果。此外，T细胞的其他嵌合修饰也在自身免疫性疾病中进行研究。嵌合自身抗体受体T细胞（CAAR-T）针对细胞表面的抗体，CAR-Treg针对靶细胞中的特异性抗原，利用Tregs的免疫调节功能。最近，CAR-T细胞已被应用于RA、结肠炎、SLE、寻常型天疱疮（PV）、自身免疫性脑脊髓炎和1型糖尿病的临床前和临床研究，为自身免疫性疾病的治疗选择带来了新的希望。2019年，CAR-T细胞在IMID中得到首次临床应用。一名20岁的严重活动性和难治性SLE患者接受了CD19 CAR-T细胞治疗，疾病迅速完全缓解。另一项由五名难治性SLE患者参与的小型研究中，在CD19 CAR-T细胞给药3个月后，SLE得到缓解。即使在B细胞再次出现后，在更长的随访期间仍保持缓解。该治疗耐受性良好，仅出现中度的细胞因子释放综合征。目前，CAR-T细胞治疗正在各种IMID进行11项研究，如SLE、干燥综合征、SSc和自身免疫性溶血性贫血。-07-六、治疗性疫苗另一种针对促炎细胞因子的治疗方法是使用疫苗来产生针对这些细胞因子的内源性自然免疫反应。目前，最领先的策略是基于肽的针对促炎细胞因子的主动疫苗接种，旨在直接中和致病细胞因子。与高免疫原性蛋白，如KLH偶联的靶向细胞因子复合物可以产生靶向该细胞因子的多克隆中和抗体。使用灭活的IFNα2b合成K-IFNα，在28名轻度至中度SLE女性的I/II期剂量递增研究中，K-IFNα显著降低了IFN诱导基因的表达，在另一项I/II期研究也发现了类似的结果。然而，一在项针对活动性SLE和IFNGS阳性患者的IIb期研究，尽管更多的患者达到了狼疮低疾病活动状态（LLDAS），但没有达到其主要终点。目前，K-IFNα在SLE中的开发已经暂停。IMID的治疗性疫苗策略是替代传统治疗的一种有趣的方案，但仍在很大程度上处于早期开发阶段，需要更好地了解IMID的发病机制。克服目前限制的一种方法可能是根据患者个人疾病转录特征选择个性化疫苗。-08-七、RNA干扰RNA干扰（RNAi）的原理是小RNA结合互补mRNA并诱导基因沉默。过去十年来，治疗人类疾病的RNAi药物开发一直在进行中，Patisiran是第一种上市的RNAi药物，靶向肝细胞中甲状腺素运载蛋白（TTR）mRNA的3′-非翻译区，用于治疗成人hATTR淀粉样变的多发性神经病。在自身免疫疾病中，研究人员一直在临床前疾病模型中积极开发RNAi策略。在RA中，几个团队已经证明，靶向TNFα或NF-κB的RNAi改善了关节炎动物模型中的关节炎和结构损伤。另一项研究表明，在红斑狼疮小鼠的NZB/NZW F1模型中，靶向BLyS或IFN调节因子5（IRF5）的siRNA改善了疾病的进程。尽管目前还没有正在进行的研究RNAi在自身免疫性疾病中的临床试验，但在不久的将来，在自身免疫中使用RNAi靶向“不可成药”的分子途径可能会展现出其巨大的潜力。-09-结语新型策略通过精准靶向或多通路干预，有望解决传统治疗应答不足与复发问题，推动IMID进入"异病同治"时代。随着医疗健康领域步入了大数据时代，国家级大型数据库（如中国的CRDC数据库）中的“云病例”和大型生物样本库的关联组装，可使得跨学科的IMID病例能够更加精细的被比较和归类。同时，基于组织活检样本RNA单细胞测序等新兴技术也将进一步揭开IMID背后的复杂炎症机制，促使更多治疗靶点和靶向联合治疗方案的产生，推动临床管理思路的迭代更新，使更多IMID患者受益。参考资料：1.Novel therapeutic strategies for autoimmune and inflammatory rheumatic diseases. Drug Discov Today.2023 May 8;28(7):103612.公众号内回复“ADC”或扫描下方图片中的二维码免费下载《抗体偶联药物：从基础到临床》的PDF格式电子书！公众号已建立“小药说药专业交流群”微信行业交流群以及读者交流群，扫描下方小编二维码加入，入行业群请主动告知姓名、工作单位和职务。

免疫疗法临床3期临床结果疫苗细胞疗法

2025-06-17

·EINPresswire

Pulmonologists and Rheumatologists Struggle to Diagnose and Manage CTD-ILD, According to New Research from Spherix Global Insights

Systemic sclerosis and myositis interstitial lung disease subtypes flagged as most difficult to manage; specialists cite lack of effective treatments and diagnostic clarity EXTON, PA, June 17, 2025 (GLOBE NEWSWIRE) -- According to Spherix Global Insights’ newly published Special Topix™: Interstitial Lung Disease in Rheumatology (US) report, both rheumatologists and pulmonologists report significant challenges in diagnosing and managing connective tissue disease-associated interstitial lung disease (CTD-ILD), with systemic sclerosis (SSc) and myositis (IIM) cited as the most difficult subtypes to treat. The research, based on responses from 138 US specialists (n=68 rheumatologists and 70 pulmonologists), highlights persistent unmet needs, diagnostic ambiguity, and a call for more robust and collaborative management strategies. The findings reflect that undiagnosed and misdiagnosed patients make up a substantial portion of the CTD-ILD population, with nearly one-third of rheumatologists and over half of pulmonologists naming differential diagnosis as a key challenge. Specialists underscore a lack of specific biomarkers, logistical biopsy issues, and overlapping symptoms as primary obstacles. “The most definitive test (biopsy) is invasive. It is difficult to find the clinicians who are willing to do it in a timely manner, and to convince the patient it needs to be done.” – Rheumatologist “Ensuring the correct diagnosis to avoid misdiagnosing the type of ILD or source of ILD. I usually employ the assistance of a rheumatologist.” – Pulmonologist “Often it would be difficult to get patients biopsied for logistical reasons, and often the pathology report would not be precise enough.” – Pulmonologist Even once diagnosed, CTD-ILD patients face a treatment landscape with few options to slow or halt disease progression. When asked about the most difficult aspects of management, specialists consistently pointed to a lack of effective and targeted therapies, with SSc-ILD and myositis-ILD receiving the highest unmet need scores. For SSc-ILD specifically, not only is it associated with the most aggressive progression and worst long-term outcomes, but it also presents a uniquely complex therapeutic challenge. The clinical burden is especially pronounced in patients with diffuse cutaneous systemic sclerosis (dcSSc), where the use of corticosteroids is often limited due to the heightened risk of scleroderma renal crisis. This leaves physicians with a narrow band of anti-fibrotic and immunosuppressive options, many of which are perceived as only modestly effective in controlling ILD progression, let alone reversing it. Even therapies with regulatory approval for SSc-ILD, such as Boehringer Ingelheim’s OFEV (nintedanib), garner only measured enthusiasm from treating physicians. While rheumatologists acknowledge nintedanib’s potential to slow functional decline, they note it does little to address the underlying autoimmune pathology. Similarly, mycophenolate mofetil and rituximab remain staples of clinical practice, yet their real-world performance is viewed as inconsistent and difficult to monitor. Across the board, satisfaction ratings for currently utilized agents fall short of expectations, reinforcing a treatment landscape still dominated by trial-and-error approaches. This disconnect between disease complexity and treatment efficacy is fueling demand for dual-acting agents, therapies that can address both systemic autoimmunity and pulmonary fibrosis. Nowhere is this demand more visible than in systemic lupus erythematosus-associated ILD (SLE-ILD), where rheumatologists express growing interest in GSK’s Benlysta (belimumab). Though not currently approved for ILD, belimumab’s immunologic mechanism and expanded label for lupus nephritis have raised hopes that similar benefit may extend to pulmonary manifestations. Across interviews and survey feedback, physicians articulate a desire for treatments that reduce systemic activity while simultaneously preventing fibrotic progression—without the need for an antifibrotic add-on with limited synergy. Looking ahead, specialists are cautiously optimistic about the pipeline. While interest in agents with antifibrotic and immunomodulatory properties is rising, physicians emphasize that what’s most needed is not just another drug, but a paradigm shift—a treatment model that reflects the multifaceted nature of CTD-ILD. As one physician summarized, the goal is to "treat the patient, not just the lungs." In addition to tracking physician sentiment and practice patterns, the study includes target product profile evaluations for several pipeline therapies: Benlysta (GSK), BMS-986278 (Bristol Myers Squibb), Nerandomilast (Boehringer Ingelheim), and Treprostinil (United Therapeutics). These profiles offer manufacturers timely insights into clinical expectations, adoption potential, and differentiation opportunities in an increasingly complex ILD treatment landscape. Special Topix ™ is an independent service that includes access to a report or series of reports based on current events or topics of interest in specialty markets covered by Spherix. About Spherix Global Insights Spherix is a leading independent market intelligence and advisory firm that delivers commercial value to the global life sciences industry, across the brand lifecycle. The seasoned team of Spherix experts provides an unbiased and holistic view of the landscape within rapidly evolving specialty markets, including dermatology, gastroenterology, rheumatology, nephrology, neurology, ophthalmology, and hematology. Spherix clients stay ahead of the curve with the perspective of the extensive Spherix Physician Community. As a trusted advisor and industry thought leader, Spherix’s unparalleled market insights and advisory services empower clients to make better decisions and unlock opportunities for growth. To learn more about Spherix Global Insights, visit spherixglobalinsights.com or connect through LinkedIn . For more details on Spherix’s primary market research reports and interactive dashboard offerings, visit or register here: https://clientportal.spherixglobalinsights.com NOTICE: All company, brand or product names in this press release are trademarks of their respective holders. The findings and opinions expressed within are based on Spherix Global Insight’s analysis and do not imply a relationship with or endorsement of the companies or brands mentioned in this press release. Andy Stankus, Rheumatology Franchise Head Spherix Global Insights 4848794284 andy.stankus@spherixglobalinsights.com 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.

上市批准

100 项与贝利尤单抗相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D03068	贝利尤单抗	L04AA26	DB08879

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适应症	国家/地区	公司	日期
狼疮性肾炎	欧盟	GlaxoSmithKline (Ireland) Ltd.	2011-07-13
狼疮性肾炎	冰岛	GlaxoSmithKline (Ireland) Ltd.	2011-07-13
狼疮性肾炎	列支敦士登	GlaxoSmithKline (Ireland) Ltd.	2011-07-13
狼疮性肾炎	挪威	GlaxoSmithKline (Ireland) Ltd.	2011-07-13
系统性红斑狼疮	美国	GlaxoSmithKline LLC	2011-03-09

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适应症	最高研发状态	国家/地区	公司	日期
支气管疾病	临床3期	美国	GSK Plc	2024-12-12
支气管疾病	临床3期	中国	GSK Plc	2024-12-12
支气管疾病	临床3期	阿根廷	GSK Plc	2024-12-12
结缔组织病	临床3期	美国	GSK Plc	2024-09-11
结缔组织病	临床3期	中国	GSK Plc	2024-09-11
结缔组织病	临床3期	日本	GSK Plc	2024-09-11
结缔组织病	临床3期	阿根廷	GSK Plc	2024-09-11
结缔组织病	临床3期	澳大利亚	GSK Plc	2024-09-11
结缔组织病	临床3期	比利时	GSK Plc	2024-09-11
结缔组织病	临床3期	巴西	GSK Plc	2024-09-11

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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


EULAR2024	N/A	-	54	Belimumab	餘構壓壓遞齋艱繭夢遞(網淵膚積願築積積淵鬱) = 糧齋壓構網遞鬱襯壓鹹獵鏇築鹹選艱襯襯鑰鹽 (糧膚壓繭廠鏇廠積簾鹽 ) 更多	积极	2024-06-05
EULAR2024	N/A	狼疮性肾炎 \| 系统性红斑狼疮	106	Subcutaneous Belimumab	製壓鏇繭蓋鏇齋範遞醖(窪鏇願遞憲齋膚襯餘選) = PSL reduction ≥5 mg was achievable in active disease and positive C1q-binding immune complex 鹽觸製鹹繭顧選鏇憲艱 (鏇憲製淵選鹹製膚壓壓 ) 更多	积极	2024-06-05
EULAR2024	N/A	系统性红斑狼疮 BAFF cytokine	1,137	Belimumab	壓艱遞襯蓋鏇網艱鹹糧(獵願膚鬱糧膚簾願蓋願) = 獵獵觸繭鹽積積選構醖鏇齋衊顧壓願選簾廠憲 (簾夢淵簾窪願淵觸衊艱 ) 更多	积极	2024-06-05
				Belimumab (Standard of Care)	壓艱遞襯蓋鏇網艱鹹糧(獵願膚鬱糧膚簾願蓋願) = 範衊餘繭顧願積願顧築鏇齋衊顧壓願選簾廠憲 (簾夢淵簾窪願淵觸衊艱 ) 更多
EULAR2024	N/A	红斑狼疮	-	Belimumab	齋鹹壓鹹鏇鏇艱膚鹹製(範鹹廠遞鑰膚夢廠鑰構) = 顧積鑰憲淵範鑰齋構壓構夢築遞衊窪繭鏇積築 (顧醖夢淵築築鬱鬱夢夢 )	积极	2024-06-05
EULAR2024	N/A	红斑狼疮	-	Belimumab	遞範廠鬱鹹襯積顧廠蓋(獵願遞衊鹽襯製醖製觸) = 簾構簾願廠蓋鏇壓構壓壓網餘憲淵網廠築鹽鑰 (艱積醖繭醖積遞憲築餘, 6.3)	-	2024-06-05
				Control (no belimumab exposure)	遞範廠鬱鹹襯積顧廠蓋(獵願遞衊鹽襯製醖製觸) = 鑰艱衊淵餘觸積顧襯夢壓網餘憲淵網廠築鹽鑰 (艱積醖繭醖積遞憲築餘, 6.5)
EULAR2024	N/A	系统性红斑狼疮	-	Belimumab	構衊衊夢廠鹹顧範憲夢(齋夢憲選簾壓鬱鏇簾構): HR = 0.72 (95% CI, 0.62 ~ 0.83), P-Value = <0.0001 更多	-	2024-06-05
				Placebo
EULAR2024	N/A	怀孕	17	Belimumab	鑰夢醖鑰衊窪顧壓願壓(網遞襯積範鹹鬱鑰壓積) = 餘範鑰襯襯築構醖憲觸鏇鏇蓋鹹簾壓淵簾窪淵 (鹹夢壓衊簾願範壓鑰範 ) 更多	积极	2024-06-05
				Non-Belimumab	繭淵夢簾糧齋窪艱範獵(築艱構壓鹹醖衊鏇鑰醖) = 願衊蓋衊襯觸觸觸顧衊蓋鑰鑰獵鹽淵獵繭衊築 (選願積艱顧鹹構網壓顧 ) 更多
EULAR2024	临床3期	系统性红斑狼疮 baseline SLE disease duration \| baseline Systemic Lupus International Collaborating Clinics/American College of Rheumatology Damage Index (SDI) score \| baseline immunosuppressant (IS) use	3,086	Belimumab	壓夢製網願獵製鹽襯範(廠齋選襯簾襯鑰獵襯鹹): OR = 1.7 (95% CI, 1.46 ~ 1.98), P-Value = <0.0001	积极	2024-06-05
				Placebo
EULAR2024	N/A	系统性红斑狼疮维持 anti-ds DNA \| RNP \| Sm ... 更多	69	Belimumab (BE)	襯鏇選齋膚積廠選淵繭(鏇簾夢構窪構鹹鑰廠積) = 齋糧簾鑰願鏇廠製獵醖窪夢網構積膚顧齋艱餘 (淵夢鹹蓋繭艱簾淵鏇範 ) 更多	积极	2024-06-05
NCT04179032 (CTgov)	临床2期	系统性红斑狼疮	25	Belimumab	繭繭範鬱淵範鏇餘蓋壓(壓觸鏇鏇餘鑰鬱廠蓋鑰) = 淵鹹鏇網觸憲範蓋淵艱艱鹹窪構願鏇廠願衊網 (蓋鏇簾鑰衊憲鬱簾艱襯, 齋憲積憲顧餘蓋膚遞淵 ~ 衊積繭構顧遞觸齋構艱) 更多	-	2024-03-26