Cyclosporine

Provides SGX945 Seven Years of U.S. Market Exclusivity Upon FDA Approval PRINCETON, N.J., Aug. 18, 2025 /PRNewswire/ -- Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, announced today that the Office of Orphan Products Development of the United States (U.S.) Food and Drug Administration (FDA) has granted orphan drug designation to dusquetide, the active ingredient in SGX945, for "treatment of Behçet's Disease" following review of recent Phase 2a clinical results demonstrating biological efficacy and safety in patients with Behçet's Disease. The U.S. Orphan Drug Act is intended to assist and encourage companies to develop safe and effective therapies for the treatment of rare diseases and disorders, defined as one that affects fewer than 200,000 people in the U.S. In addition to providing a seven-year term of market exclusivity upon final FDA approval, orphan drug designation also positions Soligenix to be able to leverage a wide range of financial and regulatory benefits, including government grants for conducting clinical trials, waiver of expensive FDA user fees for the potential submission of a New Drug Application (NDA), and certain tax credits. "Behçet's Disease is an area of unmet medical need, with up to 18,000 people in the U.S., 50,000 in Europe, 350,000 people in Turkey and as many as 1 million people worldwide affected by this incurable disease. Given the clinically meaningful improvements seen in a Phase 2 proof-of-concept study in patients with oral aphthous ulcers due to Behçet's Disease, we are hopeful dusquetide will have a role to play in helping underserved patients suffering from this difficult to treat and chronic auto-immune disease," stated Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. "The FDA's decision to grant orphan drug designation to the SGX945 program signifies an important step for Soligenix as we continue to advance the program and adds significantly to the existing intellectual property estate surrounding this novel technology." About Dusquetide Dusquetide, the active ingredient in SGX945 (Behçet's Disease) and SGX942 (oral mucositis), is an innate defense regulator (IDR), a new class of short, synthetic peptides. It has a novel mechanism of action whereby it modulates the body's reaction to both injury and infection towards an anti-inflammatory, anti-infective, and tissue healing response. IDRs have no direct antibiotic activity but, by modulating the host's innate immune system responses, increase survival after infections caused by a broad range of Gram-negative and Gram-positive bacterial pathogens. Dusquetide also accelerates resolution of tissue damage following exposure to a variety of agents including bacterial pathogens, trauma, and chemo- and/or radiation therapy. Preclinical efficacy and safety have been demonstrated in numerous animal disease models including mucositis, colitis, macrophage activation syndrome as well as bacterial infections. In addition, potential anti-tumor activity has been demonstrated in multiple in vitro and in vivo xenograft studies. Dusquetide has demonstrated safety and tolerability in a Phase 1 clinical study in 84 healthy human volunteers. In Phase 2 and 3 clinical studies with dusquetide in over 350 subjects with oral mucositis due to chemoradiation therapy for head and neck cancer, positive efficacy results were demonstrated, including potential long-term ancillary benefits. Dusquetide has also demonstrated biological efficacy and safety in a Phase 2a pilot study in 8 patients with Behçet's Disease. The Phase 2a study was an open-label study designed to be highly comparable (e.g., study endpoints, inclusion-exclusion criteria) to the published Phase 3 study which was used to support marketing approval of apremilast (Otezla®) for oral ulcers in Behçet's disease. The primary endpoint in the Phase 3 apremilast study was the area under the curve (AUC) of the mean number of ulcers versus time. Using this same endpoint after 4 weeks of treatment, the SGX945 treated group had a 40% improvement relative to the placebo group from the Phase 3 apremilast study, whereas apremilast had a 37% improvement relative to placebo. This improvement was sustained throughout the 4-week follow-up after treatment with SGX945, with 32% improvement evaluated at Week 8 despite treatment having stopped at Week 4. In contrast, apremilast, which was continuously administered through Week 12, had a 41% improvement at Week 8. One patient began the study with a punctuated skin ulcer and this also resolved during the 4-week treatment with SGX945. Skin ulcers are generally considered very difficult to resolve and usually require protracted treatment. Notably, some patients also explicitly reported experiencing less ulcers and pain during the 4-week follow-up period, as also reflected in the numerical analysis. SGX945 was well-tolerated with no treatment-related adverse events. Common adverse events for apremilast included diarrhea (41% of patients), nausea (19% of patients) and headache (14% of patients), none of which were observed with SGX945. Soligenix has a strong intellectual property position in the IDR technology platform, including composition of matter for dusquetide and related analogs. Dusquetide was developed pursuant to discoveries made by Professors B. Brett Finlay, PhD and Robert Hancock, PhD of the University of British Columbia, Canada. Dusquetide has been awarded Fast-Track designation for the treatment of oral lesions of Behçet's Disease and Orphan Drug designation for the treatment of Behçet's Disease by the FDA. About Behçet's Disease Behçet's Disease is commonly known as an inflammatory disorder of the blood vessels (vasculitis). Often first diagnosed in young adults, its effects and severity will wax and wane over time. Major signs and symptoms usually include mouth sores (approximately 95% of patients), skin rashes and lesions (approximately 50% of patients), genital sores (approximately 50% of patients), leg ulcers (approximately 40% of patients) and eye inflammation (approximately 15% of patients). It is a painful disease, directly impacting the patient's quality of life and ability to productively engage in life activities, including work. Behçet's Disease is thought to be an auto-immune disease with both genetic and environmental factors. It is most common along the "Silk Road" in the Middle East and East Asia, including Turkey, Iran, Japan and China. There are approximately 18,000 known cases of Behçet's Disease in the U.S. and over 50,000 in Europe. There are as many as 1,000,000 people worldwide living with Behçet's Disease. There is no cure for Behçet's Disease, rather treatments are prescribed to manage symptoms. Treatments may include both maintenance therapies and those specifically addressing flares (e.g., mouth ulcers, genital ulcers and leg ulcers). Corticosteroids are generally applied topically to sores and as eyedrops and may also be given systemically to reduce inflammation. Although used frequently, they have limited efficacy over the long-term and have significant side effects that become more concerning with more chronic use. Genital ulcers are often associated with significant genital scarring while leg ulcers can result in a post-thrombotic syndrome. Other treatments for Behçet's Disease flares involve suppressing the immune system with drugs (e.g., cyclosporine or cyclophosphamide). These drugs come with a higher risk of infection, liver and kidney problems, low blood counts and high blood pressure. Finally, anti-inflammatory drugs are also used, including anti-TNF medications. The only approved drug in Behçet's Disease is apremilast, which is used as a maintenance therapy to prevent formation of oral ulcers. Unfortunately, apremilast must be used continuously to be effective and is associated with both high cost and side effects including diarrhea, nausea, upper respiratory tract infection and headache. About Soligenix Soligenix is a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need. Our Specialized BioTherapeutics business segment is developing and moving toward potential commercialization of HyBryte™ (SGX301 or synthetic hypericin sodium) as a novel photodynamic therapy utilizing safe visible light for the treatment of cutaneous T-cell lymphoma (CTCL). With successful completion of the second Phase 3 study, regulatory approvals will be sought to support potential commercialization worldwide. Development programs in this business segment also include expansion of synthetic hypericin (SGX302) into psoriasis, our first-in-class innate defense regulator (IDR) technology, dusquetide (SGX942) for the treatment of inflammatory diseases, including oral mucositis in head and neck cancer, and (SGX945) in Behçet's Disease. Our Public Health Solutions business segment includes development programs for RiVax®, our ricin toxin vaccine candidate, as well as our vaccine programs targeting filoviruses (such as Marburg and Ebola) and CiVax™, our vaccine candidate for the prevention of COVID-19 (caused by SARS-CoV-2). The development of our vaccine programs incorporates the use of our proprietary heat stabilization platform technology, known as ThermoVax®. To date, this business segment has been supported with government grant and contract funding from the National Institute of Allergy and Infectious Diseases (NIAID), the Defense Threat Reduction Agency (DTRA) and the Biomedical Advanced Research and Development Authority (BARDA). For further information regarding Soligenix, Inc., please visit the Company's website at and follow us on LinkedIn and Twitter at @Soligenix_Inc. This press release may contain forward-looking statements that reflect Soligenix's current expectations about its future results, performance, prospects and opportunities, including but not limited to, potential market sizes, patient populations, clinical trial enrollment, the expected timing for closing the offering described herein and the intended use of proceeds therefrom. Statements that are not historical facts, such as "anticipates," "estimates," "believes," "hopes," "intends," "plans," "expects," "goal," "may," "suggest," "will," "potential," or similar expressions, are forward-looking statements. These statements are subject to a number of risks, uncertainties and other factors that could cause actual events or results in future periods to differ materially from what is expressed in, or implied by, these statements, and include the expected amount and use of proceeds from the offering and the expected closing date of the offering. Soligenix cannot assure you that it will be able to successfully develop, achieve regulatory approval for or commercialize products based on its technologies, particularly in light of the significant uncertainty inherent in developing therapeutics and vaccines against bioterror threats, conducting preclinical and clinical trials of therapeutics and vaccines, obtaining regulatory approvals and manufacturing therapeutics and vaccines, that product development and commercialization efforts will not be reduced or discontinued due to difficulties or delays in clinical trials or due to lack of progress or positive results from research and development efforts, that it will be able to successfully obtain any further funding to support product development and commercialization efforts, including grants and awards, maintain its existing grants which are subject to performance requirements, enter into any biodefense procurement contracts with the U.S. Government or other countries, that it will be able to compete with larger and better financed competitors in the biotechnology industry, that changes in health care practice, third party reimbursement limitations and Federal and/or state health care reform initiatives will not negatively affect its business, or that the U.S. Congress may not pass any legislation that would provide additional funding for the Project BioShield program. In addition, there can be no assurance as to the timing or success of any of its clinical/preclinical trials. Despite the statistically significant result achieved in the first HyBryte™ (SGX301) Phase 3 clinical trial for the treatment of cutaneous T-cell lymphoma, there can be no assurance that the second HyBryte™ (SGX301) Phase 3 clinical trial will be successful or that a marketing authorization from the FDA or EMA will be granted. Additionally, although the EMA has agreed to the key design components of the second HyBryte™ (SGX301) Phase 3 clinical trial, no assurance can be given that the Company will be able to modify the development path to adequately address the FDA's concerns or that the FDA will not require a longer duration comparative study. Notwithstanding the result in the first HyBryte™ (SGX301) Phase 3 clinical trial for the treatment of cutaneous T-cell lymphoma and the Phase 2a clinical trial of SGX302 for the treatment of psoriasis, there can be no assurance as to the timing or success of the clinical trials of SGX302 for the treatment of psoriasis. Additionally, despite the biologic activity observed in aphthous ulcers induced by chemotherapy and radiation, there can be no assurance as to the timing or success of the clinical trials of SGX945 for the treatment of Behçet's Disease. Further, there can be no assurance that RiVax® will qualify for a biodefense Priority Review Voucher (PRV) or that the prior sales of PRVs will be indicative of any potential sales price for a PRV for RiVax®. Also, no assurance can be provided that the Company will receive or continue to receive non-dilutive government funding from grants and contracts that have been or may be awarded or for which the Company will apply in the future. These and other risk factors are described from time to time in filings with the Securities and Exchange Commission (the "SEC"), including, but not limited to, Soligenix's reports on Forms 10-Q and 10-K. Unless required by law, Soligenix assumes no obligation to update or revise any forward-looking statements as a result of new information or future events. SOURCE SOLIGENIX, INC. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

大颗粒淋巴细胞白血病（LGLL）是一种慢性克隆性T或NK细胞疾病，可导致显著的血细胞减少。不同的表型和基因型与不同的临床病程相关。LGLL可与多种其他疾病相关，包括感染、其他恶性肿瘤和自身免疫性疾病。随着对疾病发病机制的深入了解，新型疗法如JAK抑制剂展现出希望。越来越多的不符合大颗粒淋巴细胞白血病诊断标准的T细胞克隆被发现，这增加了诊断的复杂性，尤其在它们与血细胞减少相关时。英国癌症研究所的Dima El-Sharkawi教授与Claire Dearden教授指出，鉴于目前对意义未明T细胞克隆（T-cell clone of uncertain significance, TCUS）的了解甚少，因此建议以监测意义未明单克隆B淋巴细胞增多症（monoclonal B lymphocytosis, MBL）患者的类似方式进行监测。 01 引言 大颗粒淋巴细胞白血病（LGLL）定义为外周血大颗粒淋巴细胞数量持续（>6个月）克隆性增加，通常高于2×109/L，且无明确病因。大多数病例（90%）为T细胞起源（T-LGLL），通常表现为细胞毒性T细胞表型（CD8+）。其余病例来源于NK细胞，导致慢性NK细胞淋巴组织增殖性疾病（chronic lymphoproliferative disorder of NK-cells, CLPD-NK或如WHO所定义的NK-LGLL）。更罕见的亚型包括CD4阳性表型或表达TCR γ-δ受体者。 LGLL通常在对淋巴细胞增多伴或不伴血细胞减少的检查中被诊断，但也越来越多地在仅有血细胞减少而无淋巴细胞增多的患者中被发现。应确认T细胞克隆性以排除反应性原因并确诊LGLL。然而，检测极微小T细胞克隆的技术能力对当前的数值阈值和定义提出了挑战。克隆大小<0.5×109/L的病例，现在称为意义未明T细胞克隆（T-cell clones of uncertain significance, T-CUS），类似于意义未明单克隆丙种球蛋白病（monoclonal gammopathy of uncertain significance, MGUS）或意义未明单克隆B淋巴细胞增多症（monoclonal B-cell lymphocytosis, MBL）。 02 LGLL发病机制 LGLL被描述为处于恶性、自身免疫以及对癌症或感染免疫应答的交叉点。“恶性细胞”被认为起源于正常终末效应记忆T细胞对应物，其处于慢性抗原刺激之下。由于持续的刺激及LGL细胞内的分子改变，导致寡克隆进而单克隆扩增。最常见的诱因并不明显。然而，感染因子如肝炎病毒、EB病毒、HIV或HTLV2与LGLL有关。在一例病例中，治疗感染因子导致LGLL病情改善。其他癌症也可能触发这种免疫应答。据报道LGLL与许多其他癌症共存，包括浆细胞病和淋巴瘤。此外，癌症治疗可触发免疫应答，随后发展成LGLL。这已在异基因干细胞移植后、近来在CAR-T细胞治疗后、以及与慢性粒细胞白血病（CML）的达沙替尼治疗相关中被观察到。在这些病例中，与患有相同疾病但无LGL细胞群的患者相比，LGLL的存在通常与更好的预后相关。 LGLL患者存在促炎症环境，Th17:Treg比例改变，中间型和非经典单核细胞增多，这可能通过增加IL6等细胞因子来维持LGL克隆的持续存在。在细胞内水平，JAK-STAT、PDGF和ERK1通路的组成性激活也导致增殖和逃避凋亡。后者部分可能归因于复发性STAT3突变，但在未出现这些突变的患者中也可见到，其提出的替代机制是通过miRNA。LGL细胞上存在Fas过表达，同时胞内Fas配体增加，这被认为是LGL细胞逃避凋亡能力的假设机制，也是其他谱系血细胞减少的原因。 03 临床特征 LGLL主要是一种惰性疾病，症状与血细胞减少或相关疾病有关。诊断时的中位年龄为60多岁，无性别或种族倾向。约80%的患者存在中性粒细胞减少，这是发病和死亡的主要原因。贫血和血小板减少分别发生在大约40%和20%的患者中。显著的淋巴细胞增多并不常见。肝脾肿大、B症状或乏力可以是其特征。约30%的患者可能患有“无症状性LGLL”，无血细胞减少或相关症状。 一旦诊断确立，记录血常规参数、患者经历的感染次数及类型、以及任何输血需求非常重要。这为确定治疗指征和后续监测提供了基线。纯红细胞再生障碍（PRCA）和自身免疫性溶血性贫血（AIHA）都曾在LGLL中被描述，在AIHA患者中也检测到了B细胞克隆。溶血性贫血在CLPD-NK中似乎更为常见。最后，脾脏大小可通过临床检查或超声测量。虽然大多数病例不需要标准的断层扫描成像，但少数LGLL患者具有更“淋巴瘤样”的分布，伴有器官或淋巴结受累，在这些病例中，CT分期可能是有帮助的基线评估。 04 LGLL的分类与诊断 国际共识分类（ICC）保留了与WHO第4版相同的命名，即T细胞大颗粒淋巴细胞白血病和慢性NK细胞淋巴组织增殖性疾病（CLPD-NK）的临时条目，承认在大多数中心的常规临床实践中无法证明NK细胞的克隆性。相比之下，最新的WHO分类已将后者更名为NK-LGLL。WHO的LGLL诊断标准要求证实循环细胞毒性T细胞或NK细胞增多，通常>2×109/L，但在特定情况下可以较低（0.5–2×109/L=低计数型LGLL）。这种克隆扩增必须持续存在>6个月，具有典型的LGLL表型并证明克隆性。复发性突变的存在和证实骨髓受累被接受为诊断标准。 对于白细胞计数低的患者，推荐进行骨髓检查，不仅是为了能够更全面地表征T细胞克隆，也是为了排除其他血液系统疾病，如骨髓增生异常综合征（MDS）、再生障碍性贫血或阵发性睡眠性血红蛋白尿症（PNH）。 形态学 所有表型（αβ、γδ和NK）LGL的形态学，无论是反应性的还是恶性的，都是无法区分的。LGL是大细胞，具有成熟的卵圆形或肾形细胞核，丰富的淡蓝色胞浆内含嗜天青颗粒，在光镜下可见。若行骨髓检查，这些细胞具有特征性的线性窦内分布，CD8阳性，表达细胞毒性标志物如TIA-1、穿孔素和颗粒酶B。 免疫表型 最常见LGLL亚型的表型为CD2、CD3、CD8、CD57和CD16阳性，CD56表达不定。少数患者会具有CD4阳性或γδ T细胞。 克隆性 克隆性通过分子分析确认，通常通过TCR γ和/或β链的PCR检测。近来，二代测序（NGS）因其更高的敏感性和覆盖范围、能在单次检测中分析多种分子异常的能力，开始取代传统技术。然而，并非所有实验室都能便捷地获得该技术。免疫表型分型方法更快、更经济。 流式细胞术分析TRBC1和TRBC2可作为分子分析的替代方法。其缺点在于该方法无法检测罕见的γδ亚型，也不能用于确认后续检测中是否存在同一克隆。 NK细胞的克隆性无法被证明，但其杀伤细胞免疫球蛋白样受体（killer-cell immunoglobulin-like receptor, KIR）家族表达失调，表现为KIR受体受限或完全缺失（可通过流式细胞术检测CD158a、b和e）。CD94/NKG2A复合物的表达也可用作克隆性的替代指标。 突变分析 已鉴定出STAT3、STAT5b和TET2的复发性突变，这些突变可能有助于诊断，同时也为了解疾病的发病机制及靶向治疗的潜力提供见解。超过一半患者可见STAT3突变，并与中性粒细胞减少、贫血及较差的预后相关。STAT5b突变较少见（2%），在CD4阳性LGLL中似乎更常见。也有报道存在TNFAIP3、FLT3、BCL11B、PTPN23、CCL22以及DNMT3A、IDH1、IDH2、JAK1和SETD2的突变。 05 相关疾病 LGLL与许多疾病相关，包括感染如慢性EB病毒感染；自身免疫病如类风湿关节炎和干燥综合征；包涵体肌炎；肺动脉高压；其他恶性肿瘤，包括血液系统和非血液系统肿瘤，以及药物和其他治疗。这可能反映了其他疾病可能与LGLL有共同的易感性，或伴随疾病提供了导致LGLL发展的抗原刺激，或者反之，LGLL驱动了其他疾病。即使在无LGLL诊断的情况下，也在PRCA和Felty综合征患者的CD8阳性T细胞中鉴定出了STAT3突变。 在诊断时，应评估潜在的自身免疫病和基础感染，并行蛋白电泳以排除浆细胞病。其他检查主要根据症状驱动。 06 鉴别诊断 反应性大颗粒淋巴细胞增多 在原发免疫应答中，T细胞扩增通常是多克隆的，尽管在某些情况下也可能是寡克隆甚至单克隆的。形态学上LGLs看起来相同。然而，反应性T细胞扩增通常是短暂的、自限性的，并且通常与可识别的触发事件相关，如病毒感染、脾切除术或干细胞移植。这与LGLL中持续的淋巴细胞增多形成对比，后者可能没有明显的诱发原因。 意义未明T细胞克隆（T-CUS） 意义未明T细胞克隆（T-CUS）这一术语已使用近三十年，指的是存在具有与LGLL相同免疫表型和分子特征的T细胞克隆。其免疫表型是可变的，据报道与LGLL相比，可能存在CD2和CD3表达降低及CD7表达增高的趋势。该克隆持续存在但低于LGLL的诊断阈值（<0.5×109/L）。在克隆大小上，T-CUS与LGLL可能存在重叠，因此仅凭淋巴细胞计数的任意截断值可能不足以区分两者。对于存在T细胞克隆且伴血细胞减少的患者，骨髓检查将有指征用于进一步调查。T-CUS被视为一种癌前病变，类似于MBL、MGUS和CHIP（克隆性造血不确定潜能）。健康个体中此类偶发性T细胞扩增，尤其是年长者，发生率可能高达1%–2%，与MBL相当。 目前缺乏T-CUS的可靠纵向数据。因此，它们进展为有意义克隆的频率远低于其B细胞对应物的情况。 其他T细胞恶性肿瘤 为区分T-LGLL与其他T细胞恶性肿瘤，包括T幼淋巴细胞白血病（T-cell prolymphocytic leukemia, TPLL）、成人T细胞白血病/淋巴瘤（adult T-cell leukemia/lymphoma, ATLL）、Sézary综合征（Sézary syndrome, SS）和外周T细胞淋巴瘤（peripheral T-cell lymphoma, PTCL），整合所有临床和实验室信息（外周血形态学、免疫学及遗传学标志物）至关重要。仅依赖一到两个参数可能导致误诊。 侵袭性NK细胞白血病具有非常不同的患者人口学特征、临床表现和预后。 07 治疗与预后 总体生存率良好，大多数系列研究报道5年总生存率（OS）为90%。临床与生物学相关性尚未完全明确。虽然诊断上关注T细胞或NK细胞群本身，但在管理方面，治疗的临床指征应决定最佳治疗选择以及由谁来监督治疗。 例如，在类风湿关节炎合并LGLL的患者中，如果治疗的主要原因是关节炎，那么风湿科医生应主要负责管理疾病，使用类风湿关节炎的标准治疗。这有时也能改善LGLL。然而，在某些情况下情况相反也成立，例如肺动脉高压在LGLL治疗后得到改善。 在临床试验之外，管理应基于临床需求驱动，大多数病例的需求是改善血细胞减少。部分无症状患者不需要治疗，可通过主动监测进行管理。约50%–69%的患者在某个时间点需要治疗。治疗通常适用于有症状的血细胞减少。 多年来，标准治疗一直是免疫抑制治疗，大多数临床医生仍将每周使用甲氨蝶呤、小剂量环磷酰胺和环孢素作为第一至第三线治疗。根据回顾性研究（总结于表2），这些药物的缓解率为30%–75%。使用这些药物的中位起效时间为3～4个月，因此不应因缺乏疗效而过早终止治疗。 直至近期，这些药物之间尚无头对头比较研究，但一项法国前瞻性随机研究报道（2022年），服用环磷酰胺（100mg）的患者缓解率显著高于接受甲氨蝶呤的患者。大约150名患者被随机分配到两组中一组，环磷酰胺组在4个月时有73%的患者获得缓解，而对照组的缓解率仅为32%。这表明应考虑将小剂量环磷酰胺作为初始治疗（若无禁忌证）。潜在的缺点在于环磷酰胺是以固定疗程使用，最多达一年，以尽量减少继发性恶性肿瘤的风险。出于类似原因，在较年轻患者中，它也可能会是一个不太受欢迎的选择。 在许多患者中，血细胞减少对这些标准免疫抑制剂无应答，或应答随时间消失。大量其他药物已被研究过，通常仅以单例病例报告或小型回顾性病例系列形式。 JAK抑制剂近来已被证实在此疾病中具有活性。卢索替尼（ruxolitinib）在23例患者中进行了研究，在报告时可评估的20例患者中，总缓解率（overall response rate, ORR）为55%，STAT3突变的存在与缓解相关。一项“真实世界”数据系列的报告中报告了类似的良好结局。 其他靶点专注于抑制促炎细胞因子如IL15或IL6，相关试验正在进行并显示出初步希望。其他正在研究的疗法包括达雷妥尤单抗（daratumumab）、度维利塞（duvelisib）、阿扎胞苷（azacitidine）、STAT3降解剂和抗CD94单克隆抗体（用于NK-LGLL）。 最近成立了一个由对LGLL感兴趣的临床医生组成的国际联盟，旨在制定关于该疾病的诊断、疗效评估和管理的共识指南。这将对促进LGLL临床试验活动的进一步发展，以及为评估在研治疗药物的疗效建立统一标准具有极其重要的意义。 总 结 LGLL需要被考虑作为血细胞减少的一个病因，尤其是在轻度至中度淋巴细胞增多的背景下。为做出诊断，必须证明存在持续存在的、具有LGL表型的克隆性T或NK细胞群。应考虑筛查相关的疾病，如自身免疫病或浆细胞病，以及针对其他潜在关联进行症状导向的检查。如果存在临床表现，特别是有症状的血细胞减少，则需要治疗。目前治疗领域中，免疫抑制疗法依然是治疗的基石，尽管新药（尤其是JAK抑制剂）展现出显著前景。 （来源：《肿瘤瞭望–血液时讯》编辑部） 声 明 凡署名原创的文章版权属《肿瘤瞭望》所有，欢迎分享、转载。本文仅供医疗卫生专业人士了解最新医药资讯参考使用，不代表本平台观点。该等信息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，如果该信息被用于资讯以外的目的，本站及作者不承担相关责任。