Journal of autoimmunity最新文献

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A framework for exclusion of alternative diagnoses in sarcoidosis 排除肉样瘤病替代诊断的框架。
IF 7.9 1区 医学
Journal of autoimmunity Pub Date : 2024-12-01 DOI: 10.1016/j.jaut.2024.103288
Logan J. Harper , Carol F. Farver , Ruchi Yadav , Daniel A. Culver
{"title":"A framework for exclusion of alternative diagnoses in sarcoidosis","authors":"Logan J. Harper ,&nbsp;Carol F. Farver ,&nbsp;Ruchi Yadav ,&nbsp;Daniel A. Culver","doi":"10.1016/j.jaut.2024.103288","DOIUrl":"10.1016/j.jaut.2024.103288","url":null,"abstract":"<div><div>Sarcoidosis is a multisystem granulomatous syndrome that arises from a persistent immune response to a triggering antigen(s). There is no “gold standard” test or algorithm for the diagnosis of sarcoidosis, making the diagnosis one of exclusion. The presentation of the disease varies substantially between individuals, in both the number of organs involved, and the manifestations seen in individual organs. These qualities dictate that health care providers diagnosing sarcoidosis must consider a wide range of possible alternative diagnoses, from across a range of presentations and medical specialties (infectious, inflammatory, cardiac, neurologic). Current guideline-based diagnosis of sarcoidosis recommends fulfillment of three criteria: 1) compatible clinical presentation and/or imaging 2) demonstration of granulomatous inflammation by biopsy (when possible) and, 3) exclusion of alternative causes, but do not provide guidance on standardized strategies for exclusion of alternative diagnoses. In this review, we provide a summary of the most common differential diagnoses for sarcoidosis involvement of lung, eye, skin, central nervous system, heart, liver, and kidney. We then propose a framework for testing to exclude alternative diagnoses based on pretest probability of sarcoidosis, defined as high (typical findings with sarcoidosis involvement confirmed in another organ), moderate (typical findings in a single organ), or low (atypical/findings suggesting of an alternative diagnosis). This work highlights the need for informed and careful exclusion of alternative diagnoses in sarcoidosis.</div></div>","PeriodicalId":15245,"journal":{"name":"Journal of autoimmunity","volume":"149 ","pages":"Article 103288"},"PeriodicalIF":7.9,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141859866","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Genetic predisposition to sarcoidosis 结节病的遗传易感性。
IF 7.9 1区 医学
Journal of autoimmunity Pub Date : 2024-12-01 DOI: 10.1016/j.jaut.2023.103122
Shu-Yi Liao , Tasha Fingerlin , Lisa Maier
{"title":"Genetic predisposition to sarcoidosis","authors":"Shu-Yi Liao ,&nbsp;Tasha Fingerlin ,&nbsp;Lisa Maier","doi":"10.1016/j.jaut.2023.103122","DOIUrl":"10.1016/j.jaut.2023.103122","url":null,"abstract":"<div><div>Sarcoidosis is a complex systemic disease with clinical heterogeneity based on varying phenotypes and natural history. The detailed etiology of sarcoidosis remains unknown, but genetic predisposition as well as environmental exposures play a significant role in disease pathogenesis. We performed a comprehensive review of germline genetic (DNA) and transcriptomic (RNA) studies of sarcoidosis, including both previous studies and more recent findings. In this review, we provide an assessment of the following: genetic variants in sarcoidosis susceptibility and phenotypes, ancestry- and sex-specific genetic variants in sarcoidosis, shared genetic architecture between sarcoidosis and other diseases, and gene-environment interactions in sarcoidosis. We also highlight the unmet needs in sarcoidosis genetic studies, including the pressing requirement to include diverse populations and have consistent definitions of phenotypes in the sarcoidosis research community to help advance the application of genetic predisposition to sarcoidosis disease risk and manifestations.</div></div>","PeriodicalId":15245,"journal":{"name":"Journal of autoimmunity","volume":"149 ","pages":"Article 103122"},"PeriodicalIF":7.9,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"49677872","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Pulmonary sarcoidosis: A comprehensive review: Past to present 肺结节病:一篇综合综述:从过去到现在。
IF 7.9 1区 医学
Journal of autoimmunity Pub Date : 2024-12-01 DOI: 10.1016/j.jaut.2023.103107
John A. Belperio , Michael C. Fishbein , Fereidoun Abtin , Jessica Channick , Shailesh A. Balasubramanian , Joseph P. Lynch III
{"title":"Pulmonary sarcoidosis: A comprehensive review: Past to present","authors":"John A. Belperio ,&nbsp;Michael C. Fishbein ,&nbsp;Fereidoun Abtin ,&nbsp;Jessica Channick ,&nbsp;Shailesh A. Balasubramanian ,&nbsp;Joseph P. Lynch III","doi":"10.1016/j.jaut.2023.103107","DOIUrl":"10.1016/j.jaut.2023.103107","url":null,"abstract":"&lt;div&gt;&lt;div&gt;Sarcoidosis is a sterile non-necrotizing granulomatous disease without known causes that can involve multiple organs with a predilection for the lung and thoracic lymph nodes. Worldwide it is estimated to affect 2–160/100,000 people and has a mortality rate over 5 years of approximately 7%. For sarcoidosis patients, the cause of death is due to sarcoid in 60% of the cases, of which up to 80% are from advanced cardiopulmonary failure (pulmonary hypertension and respiratory microbial infections) in all races except in Japan were greater than 70% of the sarcoidosis deaths are due to cardiac sarcoidosis.&lt;/div&gt;&lt;div&gt;Scadding stages for pulmonary sarcoidosis associates with clinical outcomes. Stages I and II have radiographic remission in approximately 30%–80% of cases. Stage III only has a 10%–40% chance of resolution, while stage IV has no change of resolution. Up to 40% of pulmonary sarcoidosis patients progress to stage IV disease with lung parenchyma fibroplasia, bronchiectasis with hilar retraction and fibrocystic disease. These patients are at highest risk for the development of precapillary pulmonary hypertension, which may occur in up to 70% of these patients. Sarcoid patients with pre-capillary pulmonary hypertension can respond to targeted pulmonary arterial hypertension medications. Stage IV fibrocytic sarcoidosis with significant pulmonary physiologic impairment, &gt;20% fibrosis on HRCT or pre-capillary pulmonary hypertension have the highest risk of mortality, which can be &gt;40% at 5-years.&lt;/div&gt;&lt;div&gt;First line treatment for patients who are symptomatic (cough and dyspnea) with parenchymal infiltrates and abnormal pulmonary function testing (PFT) is oral glucocorticoids, such as prednisone with a typical starting dose of 20–40 mg daily for 2 weeks to 2 months. Prednisone can be tapered over 6–18 months if symptoms, spirometry, PFTs, and radiographs improve. Prolonged prednisone may be required to stabilize disease. Patients requiring prolonged prednisone ≥10 mg/day or those with adverse effects due to glucocorticoids may be prescribed second and third line treatements. Second and third line treatments include immunosuppressive agents (e.g., methotrexate and azathioprine) and anti-tumor necrosis factor (TNF) medication; respectively. Effective treatments for advanced fibrocystic pulmonary disease are being explored. Despite different treatments, relapse rates range from 13% to 75% depending on the stage of sarcoid, number of organs involved, socioeconomic status, and geography.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Conclusion&lt;/h3&gt;&lt;div&gt;The mortality rate for sarcoidosis over a 5 year follow up is approximately 7%. Unfortunately, 10%–40% of patients with sarcoidosis develop progressive pulmonary disease, and &gt;60% of deaths resulting from sarcoidosis are due to advance cardiopulmonary disease. Oral glucocorticoids are the first line treatment, while methotrexate and azathioprine are considered second and anti-TNF agents are third line treatments","PeriodicalId":15245,"journal":{"name":"Journal of autoimmunity","volume":"149 ","pages":"Article 103107"},"PeriodicalIF":7.9,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"49677873","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Quality of life in sarcoidosis 结节病患者的生活质量。
IF 7.9 1区 医学
Journal of autoimmunity Pub Date : 2024-12-01 DOI: 10.1016/j.jaut.2023.103123
Catharina C. Moor , Ogugua Ndili Obi , Vivienne Kahlmann , Katharina Buschulte , Marlies S. Wijsenbeek
{"title":"Quality of life in sarcoidosis","authors":"Catharina C. Moor ,&nbsp;Ogugua Ndili Obi ,&nbsp;Vivienne Kahlmann ,&nbsp;Katharina Buschulte ,&nbsp;Marlies S. Wijsenbeek","doi":"10.1016/j.jaut.2023.103123","DOIUrl":"10.1016/j.jaut.2023.103123","url":null,"abstract":"<div><div>Having sarcoidosis often has a major impact on quality of life of patients and their families. Improving quality of life is prioritized as most important treatment aim by many patients with sarcoidosis, but current evidence and treatment options are limited. In this narrative review, we describe the impact of sarcoidosis on various aspects of daily life, evaluate determinants of health-related quality of life (HRQoL), and provide an overview of the different patient-reported outcome measures to assess HRQoL in sarcoidosis. Moreover, we review the current evidence for pharmacological and non-pharmacological interventions to improve quality of life for people with sarcoidosis.</div></div>","PeriodicalId":15245,"journal":{"name":"Journal of autoimmunity","volume":"149 ","pages":"Article 103123"},"PeriodicalIF":7.9,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41182636","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Kidney manifestations of sarcoidosis 肉样瘤病的肾脏表现。
IF 7.9 1区 医学
Journal of autoimmunity Pub Date : 2024-12-01 DOI: 10.1016/j.jaut.2024.103207
Francesco Bonella , Adriane DM Vorselaars , Benjamin Wilde
{"title":"Kidney manifestations of sarcoidosis","authors":"Francesco Bonella ,&nbsp;Adriane DM Vorselaars ,&nbsp;Benjamin Wilde","doi":"10.1016/j.jaut.2024.103207","DOIUrl":"10.1016/j.jaut.2024.103207","url":null,"abstract":"<div><div>Renal involvement is a clinically relevant organ manifestation of sarcoidosis, leading to increased morbidity and complications. Although the exact incidence remains unknown, renal disease is likely to occur in up to one third of all sarcoidosis patients. Every patient with newly diagnosed sarcoidosis should receive a renal work-up and screening for disrupted calcium metabolism.</div><div>Amid various forms of glomerulonephritis, granulomatous interstitial nephritis is the most common one, but it rarely leads to renal impairment. Histologically, granulomas can be absent. Nephrocalcinosis and nephrolithiasis are frequent forms when hypercalcaemia or hypercalciuria occur. Drugs used for treatment of systemic sarcoidosis can also cause renal damage.</div><div>Due to its high heterogeneity, renal sarcoidosis can be difficult to treat. Glucocorticoids and various immunosuppressive treatments have been proven to be effective based on case series, but clinical trials are lacking. A treatment guideline for renal sarcoidosis is urgently needed.</div><div>In this review article, we present an overview of the different forms of renal sarcoidosis and the diagnostic steps to confirm renal involvement; in addition, we provide insights on the management and available treatments. A better understanding regarding the pathogenesis of sarcoidosis is the key for the development of more specific, targeted therapies.</div></div>","PeriodicalId":15245,"journal":{"name":"Journal of autoimmunity","volume":"149 ","pages":"Article 103207"},"PeriodicalIF":7.9,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140193881","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
The immunopathogenesis of sarcoidosis 肉样瘤病的免疫发病机制。
IF 7.9 1区 医学
Journal of autoimmunity Pub Date : 2024-12-01 DOI: 10.1016/j.jaut.2024.103247
Jelle Miedema , Francesco Cinetto , Anna Smed-Sörensen , Paolo Spagnolo
{"title":"The immunopathogenesis of sarcoidosis","authors":"Jelle Miedema ,&nbsp;Francesco Cinetto ,&nbsp;Anna Smed-Sörensen ,&nbsp;Paolo Spagnolo","doi":"10.1016/j.jaut.2024.103247","DOIUrl":"10.1016/j.jaut.2024.103247","url":null,"abstract":"<div><div>Sarcoidosis is a granulomatous multiorgan disease, thought to result from exposure to yet unidentified antigens in genetically susceptible individuals. The exaggerated inflammatory response that leads to granuloma formation is highly complex and involves the innate and adaptive immune system. Consecutive immunological studies using advanced technology have increased our understanding of aberrantly activated immune cells, mediators and pathways that influence the formation, maintenance and resolution of granulomas. Over the years, it has become increasingly clear that disease immunopathogenesis can only be understood if the clinical heterogeneity of sarcoidosis is taken into consideration, along with the distribution of immune cells in peripheral blood and involved organs. Most studies offer an immunological snapshot during disease course, while the cellular composition of both the circulation and tissue microenvironment may change over time. Despite these challenges, novel insights on the role of the immune system are continuously published, thus bringing the field forward.</div><div>This review highlights current knowledge on the innate and adaptive immune responses involved in sarcoidosis pathogenesis, as well as the pathways involved in non-resolving disease and fibrosis development. Additionally, we describe proposed immunological mechanisms responsible for drug-induced sarcoid like reactions. Although many aspects of disease immunopathogenesis remain to be unraveled, the identification of crucial immune reactions in sarcoidosis may help identify new treatment targets. We therefore also discuss potential therapies and future strategies based on the latest immunological findings.</div></div>","PeriodicalId":15245,"journal":{"name":"Journal of autoimmunity","volume":"149 ","pages":"Article 103247"},"PeriodicalIF":7.9,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140908762","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Unraveling the immunomodulatory impact of hydroxychloroquine on peripheral T cells using single-cell RNA sequencing. 利用单细胞 RNA 测序揭示羟氯喹对外周 T 细胞免疫调节的影响
IF 7.9 1区 医学
Journal of autoimmunity Pub Date : 2024-12-01 Epub Date: 2024-10-13 DOI: 10.1016/j.jaut.2024.103324
Huizhong Long, Luis Espinosa, Amr H Sawalha
{"title":"Unraveling the immunomodulatory impact of hydroxychloroquine on peripheral T cells using single-cell RNA sequencing.","authors":"Huizhong Long, Luis Espinosa, Amr H Sawalha","doi":"10.1016/j.jaut.2024.103324","DOIUrl":"10.1016/j.jaut.2024.103324","url":null,"abstract":"<p><p>Hydroxychloroquine (HCQ) is widely used in the treatment of a variety of autoimmune diseases. However, the mechanisms responsible for the immunomodulatory properties of HCQ in T cells remain unclear. Here we used single-cell RNA-sequencing to examine the effect of HCQ on T cells following in vitro stimulation. HCQ treatment led to a reduction in effector CD4<sup>+</sup> T cells and upregulation of inhibitory genes including CTLA4 and TNFAIP3 in effector and naive CD4<sup>+</sup> T cells, respectively. HCQ induced a significant expansion of effector CD8<sup>+</sup> T cells, and significantly upregulated key cytotoxicity genes including GZMA, GZMB, GZMH, KLRD1, NKG7, and PRF1, as well as IFNG expression. Furthermore, HCQ treatment led to a reduction in the CD38<sup>+</sup> CD8<sup>+</sup> T cell subset, which is characterized by defective cytotoxicity and thought to both play a pathogenic role and increase susceptibility to infections in autoimmunity. We analyzed single-cell RNA-sequencing data in effector CD8<sup>+</sup> T cells from lupus patients with or without HCQ treatment and confirmed upregulation of key cytotoxicity genes in patients receiving HCQ. In conclusion, this work provides additional insights into the immunomodulatory effects of HCQ and indicates that HCQ improves T cell cytotoxicity, which could explain a previously suggested protective effect of HCQ against infections in patients with autoimmune diseases.</p>","PeriodicalId":15245,"journal":{"name":"Journal of autoimmunity","volume":"149 ","pages":"103324"},"PeriodicalIF":7.9,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142466404","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Sarcoidosis and its relation to other immune-mediated diseases: Epidemiological insights 结节病及其与其他免疫介导疾病的关系:流行病学见解。
IF 7.9 1区 医学
Journal of autoimmunity Pub Date : 2024-12-01 DOI: 10.1016/j.jaut.2023.103127
Elizabeth V. Arkema , Marios Rossides , Yvette C. Cozier
{"title":"Sarcoidosis and its relation to other immune-mediated diseases: Epidemiological insights","authors":"Elizabeth V. Arkema ,&nbsp;Marios Rossides ,&nbsp;Yvette C. Cozier","doi":"10.1016/j.jaut.2023.103127","DOIUrl":"10.1016/j.jaut.2023.103127","url":null,"abstract":"<div><div>Several epidemiological studies show a co-occurrence of sarcoidosis with other immune-mediated diseases (IMD). There are many similarities between sarcoidosis and IMDs in their geographical distribution and risk factors. Understanding these similarities and identifying the differences can help us to better understand sarcoidosis and put it into context with other IMDs. In this review, we present the current knowledge about the overlap between sarcoidosis and other IMDs derived from epidemiological studies. Epidemiologic methods utilize study design and statistical analysis to describe the patterns in data and, ideally, identify causal relationships between an exposure and a health outcome. We discuss how study design and analysis may affect the interpretation of epidemiological studies on this topic and highlight some theories that attempt to explain the relation between sarcoidosis and other IMDs.</div></div>","PeriodicalId":15245,"journal":{"name":"Journal of autoimmunity","volume":"149 ","pages":"Article 103127"},"PeriodicalIF":7.9,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41202114","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Application of laboratory models for sarcoidosis research 应用实验室模型进行肉样瘤病研究。
IF 7.9 1区 医学
Journal of autoimmunity Pub Date : 2024-12-01 DOI: 10.1016/j.jaut.2024.103184
Maneesh Bhargava , Elliott D. Crouser
{"title":"Application of laboratory models for sarcoidosis research","authors":"Maneesh Bhargava ,&nbsp;Elliott D. Crouser","doi":"10.1016/j.jaut.2024.103184","DOIUrl":"10.1016/j.jaut.2024.103184","url":null,"abstract":"<div><div>This manuscript will review the implications and applications of sarcoidosis models towards advancing our understanding of sarcoidosis disease mechanisms, identification of biomarkers, and preclinical testing of novel therapies. Emerging disease models and innovative research tools will also be considered.</div></div>","PeriodicalId":15245,"journal":{"name":"Journal of autoimmunity","volume":"149 ","pages":"Article 103184"},"PeriodicalIF":7.9,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140039492","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Developmental drugs for sarcoidosis 开发治疗肉样瘤病的药物。
IF 7.9 1区 医学
Journal of autoimmunity Pub Date : 2024-12-01 DOI: 10.1016/j.jaut.2024.103179
Ogugua Ndili Obi , Lesley Ann Saketkoo , Lisa A. Maier , Robert P. Baughman
{"title":"Developmental drugs for sarcoidosis","authors":"Ogugua Ndili Obi ,&nbsp;Lesley Ann Saketkoo ,&nbsp;Lisa A. Maier ,&nbsp;Robert P. Baughman","doi":"10.1016/j.jaut.2024.103179","DOIUrl":"10.1016/j.jaut.2024.103179","url":null,"abstract":"<div><div><span>Sarcoidosis<span> is a multi-organ granulomatous </span></span>inflammatory disease<span> of unknown etiology. Over 50% of patients will require treatment at some point in their disease and 10%–30% will develop a chronic progressive disease with pulmonary fibrosis leading to significant morbidity and mortality.</span></div><div><span>Recently published guidelines recommend immunosuppressive therapy for sarcoidosis patients at risk of increased disease-related morbidity and mortality, and in whom disease has negatively impacted quality of life. </span>Prednisone the currently recommended first line therapy is associated with significant toxicity however none of the other guideline recommended steroid sparing therapy is approved by regulatory agencies for use in sarcoidosis, and data in support of their use is weak. For patients with severe refractory disease requiring prolonged therapy, treatment options are limited.</div><div>The need for expanding treatment options in sarcoidosis has been emphasized. Well conducted large, randomized trials evaluating currently available therapeutic options as well as novel pathways for targeting disease are necessary to better guide treatment decisions. These trials will not be without significant challenges. Sarcoidosis is a rare disease<span> with heterogenous presentation and variable progression and clinical outcome. There are no universally agreed upon biomarkers of disease activity and measurement of outcomes is confounded by the need to balance patient centric measures and objective measures of disease activity.</span></div><div>Our paper provides an update on developmental drugs in sarcoidosis and outlines several novel pathways that may be targeted for future drug development<span>. Currently available trials are highlighted and ongoing challenges to drug development and clinical trial design are briefly discussed.</span></div></div>","PeriodicalId":15245,"journal":{"name":"Journal of autoimmunity","volume":"149 ","pages":"Article 103179"},"PeriodicalIF":7.9,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140318366","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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