RARE DISEASE
Takayasu arteritis
Takayasu arteritis
Takayasu arteritis
Drug discovery
2
drugs
With orphan designations
Overview
Takayasu arteritis (TAK) is a rare, chronic large-vessel vasculitis characterized by autoimmune-mediated inflammation of the aorta and its primary branches, leading to stenosis, occlusion, or aneurysm formation [1][6][12]. It predominantly affects females under 40, with a 9:1 female-to-male ratio, and has higher prevalence in Asian populations [6][12][15]. Diagnosis relies on imaging (angiography, MRI) due to nonspecific early symptoms like fever and fatigue, followed by vascular complications such as hypertension, stroke, and limb ischemia [1][3][12].
Burden
Morbidity: Hypertension (56%), renal artery stenosis (28%), aortic insufficiency (19%), and stroke risk [4][7][9].
Healthcare utilization: 7.35-day average hospitalization (2.1 days longer than non-TAK patients) and increased costs [4][9][16].
Chronicity: Relapse rates exceed 40%, necessitating long-term immunosuppression and monitoring [8][11][18].
Therapies
First-line: High-dose corticosteroids (e.g., prednisone) for inflammation control [3][8][11].
Steroid-sparing agents: Methotrexate, azathioprine, or mycophenolate mofetil; biologics (tocilizumab, TNF inhibitors) for refractory disease [8][11][18].
Surgical interventions: Revascularization or endovascular procedures for critical stenosis/aneurysms [3][9][18].
Categories: rare circulatory system diseases, rare renal diseases, rare systemic and rheumatological diseases, rare systemic or rheumatologic diseases of childhood, rare transplant-related disorders
Research Papers
1,312 drug discovery papers about Takayasu arteritis, with 1 first-in-class and 1 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
1,312 drug discovery papers about Takayasu arteritis, with 1 first-in-class and 1 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
categories:
Small molecules
2026-08-10 | Bilateral Ocular ischemia in a patient with Takayasu arteritis.
To describe a rare case of bilateral ocular ischemia as the initial presentation of Takayasu arteritis and highlight the role of early recognition and treatment in preventing irreversible vision loss. A woman in her 50s presented with a gradual, progressive diminution of vision, initially in the left eye and later involving the right. Comprehensive ophthalmic evaluation, systemic examination, fundus fluorescein angiography, and CT angiography of the head and neck were performed. Laboratory investigations, including ESR, CRP, and ANA, were obtained. The American College of Rheumatology (ACR) criteria were applied to support the diagnosis. The patient demonstrated severe ocular ischemic features, including attenuated retinal vessels, disc neovascularization, and intumescent cataract in the left eye. Systemic examination revealed feeble brachial and radial pulses with a significant inter-arm blood pressure difference. CT angiography showed narrowing of the right common carotid artery and non-opacification of both subclavian arteries. Laboratory findings revealed elevated ESR and CRP, with a positive ANA. Based on clinical, radiologic, and laboratory features, a diagnosis of bilateral ocular ischemia secondary to Takayasu arteritis was made. The patient was treated with oral corticosteroids (60 mg/day, tapered) and azathioprine. At 4 weeks, visual acuity improved to 6/36 in the right eye, while no improvement was noted in the left eye. Our case highlights bilateral ocular ischemia as an uncommon but vision-threatening manifestation of Takayasu arteritis. Ocular findings such as retinal vascular attenuation, disc neovascularization, delayed choroidal filling, and peripheral capillary non-perfusion may precede the diagnosis of systemic vasculitis. Recognition of associated systemic signs, including pulse deficits and inter-arm blood pressure discrepancies, facilitated timely diagnosis. Prompt initiation of corticosteroid and immunosuppressive therapy led to visual improvement in the right eye, underscoring the importance of early diagnosis and multidisciplinary management to prevent irreversible visual loss and systemic vascular complications. Bilateral ocular ischemia may be the first manifestation of Takayasu arteritis. This case underscores the importance of a high index of suspicion in patients with unexplained ischemic ocular features. Early systemic evaluation and prompt initiation of immunosuppressive therapy are essential to preserve vision and prevent further vascular complications.
2026-08-06 | Misty Mesentery as an Initial Imaging Finding Suggestive of Large Vessel Vasculitis.
Large vessel vasculitis (LVV) refers to vasculitis predominantly affecting large arteries, including the aorta and its major branches. It encompasses conditions such as giant cell arteritis and Takayasu arteritis, as well as unclassified or idiopathic forms. Imaging techniques, including computed tomography (CT), are essential for detecting large vessel involvement, particularly when biopsy findings are negative. We report the case of a 56-year-old man admitted with fever, headache, and upper abdominal pain lasting 2 weeks. On examination, he had scalp tenderness and upper abdominal tenderness without temporal artery enlargement. Laboratory tests revealed elevated C-reactive protein (CRP: 19.75 mg/dL) and normal IgG4 (45 mg/dL). Initial CT demonstrated increased attenuation of mesenteric fat (Figure 1), suggestive of misty mesentery. Antibiotic therapy for presumed infectious mesenteritis was ineffective, and CRP increased further to 30.20 mg/dL. Contrast-enhanced CT showed thickening of the aortic wall (Figure 2). Based on fever, headache, systemic inflammation, and aortic wall thickening, LVV was considered the most plausible diagnosis. Cite this article as: Noda S, Kondo S. Misty mesentery as an initial imaging finding suggestive of large vessel vasculitis. Eur J Rheumatol. 13(1), 0084, doi: 10.5152/eurjrheum.2026.25084.
2026-08-04 | Age-associated B cells contribute to inflammation via antibody-independent mechanisms in Takayasu's arteritis.
Although Takayasu's arteritis (TAK) is not a prototypical autoantibody-mediated disease, accumulating evidence suggests that B cells are involved. This study aimed to investigate the pathway of B-cell activation and its contributions to TAK pathogenesis. Histological analysis of paravascular lymph nodes and affected arteries was conducted to investigate B-cell activation pathways in TAK. Bulk RNA-seq, single-cell RNA-seq (scRNA-seq), flow cytometry, and in vitro experiments were performed to characterize the composition, transcriptomic features and functional profiles of B cells. The numeric and phenotypic alterations induced by TNF and JAK inhibition were assessed both in vitro and in 4 patients with TAK. Histological (n=5), flow cytometric (n=125) and bulk RNA-seq (n=12) analyses indicated the presence of extrafollicular response and upregulated age-associated B cell (ABC) production in TAK, along with cross-dataset transcriptomic differences between B cells from patients with TAK and systemic lupus erythematosus (SLE). Cross-dataset scRNA-seq analysis and in vitro experiments (n=5) demonstrated that ABC differentiation in TAK was largely uncoupled from antibody-secreting cell (ASC) generation, unlike that in SLE. Functional experiments showed that ABCs exhibited proinflammatory properties, including proinflammatory cytokine production, and that CD11c+ B cells, which contain the ABC compartment, promoted Th17 cell differentiation (n=6). In vitro, tofacitinib was more effective than adalimumab at reducing ABC proportions and attenuating their proinflammatory phenotype (n=15), consistent with trends in the exploratory clinical follow-up (n=4). ABCs promote inflammation through proinflammatory functions independent of ASC differentiation in TAK. JAK inhibition exerts distinct suppressive effects on ABCs compared with anti-TNF therapy.
2026-08-01 | TAKAYASU ARTERITIS PRESENTING AS MULTIFOCAL ACUTE INFARCTS IN A YOUNG FEMALE: A CASE REPORT
Takayasu arteritis (TA) is a rare, chronic granulomatous large-vessel vasculitis that involves the aorta and its major branches and predominantly affects young women. We report a 30-year-old woman with a six-month history of right upper-limb claudication who presented with sudden-onset altered sensorium following a one-day febrile illness. Clinical examination revealed bilateral carotid bruits with absent right upper-limb pulses and preserved lower-limb blood pressure. MRI brain demonstrated acute infarcts in the left fronto-temporal region and right caudate nucleus, and CT aortogram showed circumferential wall thickening with significant luminal narrowing of both common carotid arteries, confirming the diagnosis of Takayasu arteritis. The patient was managed with high-dose oral corticosteroids and antiplatelet therapy, with endovascular stenting of the right common carotid artery planned. This case highlights the importance of considering TA in young women presenting with stroke and limb claudication, and the need for early immunosuppressive therapy to prevent irreversible vascular damage.
2026-08-01 | Biological therapy in Takayasu arteritis. Results from the REVAS registry.
Biological therapy in patients with Takayasu arteritis is frequently used, but the factors associated with its use have not been determined. The present study aims to describe and identify predictors of the need for biological therapy in this vasculitis. We conducted a retrospective, multicentre, descriptive study including all patients with Takayasu arteritis enrolled in the REVAS registry from Spain. We performed a descriptive and a multivariate logistic regression analysis to identify variables associated with the need of biological therapy requirement. The study included 77 patients, 65 (84.4 %) women, with a median follow up of 9.9 (interquartile range 3.5-21.5) years. Sixty-three (81.8 %) participants received glucocorticoids and 45 (58.4 %) needed immunosuppressive therapy. Twenty-three patients had received biological therapy, 20 of whom were diagnosed after 2006. Biological therapy included infliximab (n = 3), adalimumab (n = 9) and tocilizumab (n = 18). Patients requiring biological therapy had a higher proportion of systemic symptoms (fever and/or constitutional syndrome) at baseline (70.0 %vs. 28.6 %, p = 0.005), received glucocorticoids more frequently (100.0 %vs. 78.6 %, p = 0.03) and required a greater number of immunosuppressants (2 (1-2) vs. 1 (0-1), p = 0.03) during follow-up. On multivariate logistic regression, the presence of systemic symptoms was associated with the need for biological therapy requirement (odds radio: 3.9 (1.02-14.8), p = 0.047). Conclusion: to our knowledge, this is the first study that evaluates variables associated with the need for biological therapy requirement in Takayasu arteritis. The presence of systemic symptoms at presentation was independently associated with the need for biological therapy, highlighting the importance of a thorough clinical evaluation.
2026-08-10 | Bilateral Ocular ischemia in a patient with Takayasu arteritis.
To describe a rare case of bilateral ocular ischemia as the initial presentation of Takayasu arteritis and highlight the role of early recognition and treatment in preventing irreversible vision loss. A woman in her 50s presented with a gradual, progressive diminution of vision, initially in the left eye and later involving the right. Comprehensive ophthalmic evaluation, systemic examination, fundus fluorescein angiography, and CT angiography of the head and neck were performed. Laboratory investigations, including ESR, CRP, and ANA, were obtained. The American College of Rheumatology (ACR) criteria were applied to support the diagnosis. The patient demonstrated severe ocular ischemic features, including attenuated retinal vessels, disc neovascularization, and intumescent cataract in the left eye. Systemic examination revealed feeble brachial and radial pulses with a significant inter-arm blood pressure difference. CT angiography showed narrowing of the right common carotid artery and non-opacification of both subclavian arteries. Laboratory findings revealed elevated ESR and CRP, with a positive ANA. Based on clinical, radiologic, and laboratory features, a diagnosis of bilateral ocular ischemia secondary to Takayasu arteritis was made. The patient was treated with oral corticosteroids (60 mg/day, tapered) and azathioprine. At 4 weeks, visual acuity improved to 6/36 in the right eye, while no improvement was noted in the left eye. Our case highlights bilateral ocular ischemia as an uncommon but vision-threatening manifestation of Takayasu arteritis. Ocular findings such as retinal vascular attenuation, disc neovascularization, delayed choroidal filling, and peripheral capillary non-perfusion may precede the diagnosis of systemic vasculitis. Recognition of associated systemic signs, including pulse deficits and inter-arm blood pressure discrepancies, facilitated timely diagnosis. Prompt initiation of corticosteroid and immunosuppressive therapy led to visual improvement in the right eye, underscoring the importance of early diagnosis and multidisciplinary management to prevent irreversible visual loss and systemic vascular complications. Bilateral ocular ischemia may be the first manifestation of Takayasu arteritis. This case underscores the importance of a high index of suspicion in patients with unexplained ischemic ocular features. Early systemic evaluation and prompt initiation of immunosuppressive therapy are essential to preserve vision and prevent further vascular complications.
2026-08-06 | Misty Mesentery as an Initial Imaging Finding Suggestive of Large Vessel Vasculitis.
Large vessel vasculitis (LVV) refers to vasculitis predominantly affecting large arteries, including the aorta and its major branches. It encompasses conditions such as giant cell arteritis and Takayasu arteritis, as well as unclassified or idiopathic forms. Imaging techniques, including computed tomography (CT), are essential for detecting large vessel involvement, particularly when biopsy findings are negative. We report the case of a 56-year-old man admitted with fever, headache, and upper abdominal pain lasting 2 weeks. On examination, he had scalp tenderness and upper abdominal tenderness without temporal artery enlargement. Laboratory tests revealed elevated C-reactive protein (CRP: 19.75 mg/dL) and normal IgG4 (45 mg/dL). Initial CT demonstrated increased attenuation of mesenteric fat (Figure 1), suggestive of misty mesentery. Antibiotic therapy for presumed infectious mesenteritis was ineffective, and CRP increased further to 30.20 mg/dL. Contrast-enhanced CT showed thickening of the aortic wall (Figure 2). Based on fever, headache, systemic inflammation, and aortic wall thickening, LVV was considered the most plausible diagnosis. Cite this article as: Noda S, Kondo S. Misty mesentery as an initial imaging finding suggestive of large vessel vasculitis. Eur J Rheumatol. 13(1), 0084, doi: 10.5152/eurjrheum.2026.25084.
2026-08-04 | Age-associated B cells contribute to inflammation via antibody-independent mechanisms in Takayasu's arteritis.
Although Takayasu's arteritis (TAK) is not a prototypical autoantibody-mediated disease, accumulating evidence suggests that B cells are involved. This study aimed to investigate the pathway of B-cell activation and its contributions to TAK pathogenesis. Histological analysis of paravascular lymph nodes and affected arteries was conducted to investigate B-cell activation pathways in TAK. Bulk RNA-seq, single-cell RNA-seq (scRNA-seq), flow cytometry, and in vitro experiments were performed to characterize the composition, transcriptomic features and functional profiles of B cells. The numeric and phenotypic alterations induced by TNF and JAK inhibition were assessed both in vitro and in 4 patients with TAK. Histological (n=5), flow cytometric (n=125) and bulk RNA-seq (n=12) analyses indicated the presence of extrafollicular response and upregulated age-associated B cell (ABC) production in TAK, along with cross-dataset transcriptomic differences between B cells from patients with TAK and systemic lupus erythematosus (SLE). Cross-dataset scRNA-seq analysis and in vitro experiments (n=5) demonstrated that ABC differentiation in TAK was largely uncoupled from antibody-secreting cell (ASC) generation, unlike that in SLE. Functional experiments showed that ABCs exhibited proinflammatory properties, including proinflammatory cytokine production, and that CD11c+ B cells, which contain the ABC compartment, promoted Th17 cell differentiation (n=6). In vitro, tofacitinib was more effective than adalimumab at reducing ABC proportions and attenuating their proinflammatory phenotype (n=15), consistent with trends in the exploratory clinical follow-up (n=4). ABCs promote inflammation through proinflammatory functions independent of ASC differentiation in TAK. JAK inhibition exerts distinct suppressive effects on ABCs compared with anti-TNF therapy.
2026-08-01 | TAKAYASU ARTERITIS PRESENTING AS MULTIFOCAL ACUTE INFARCTS IN A YOUNG FEMALE: A CASE REPORT
Takayasu arteritis (TA) is a rare, chronic granulomatous large-vessel vasculitis that involves the aorta and its major branches and predominantly affects young women. We report a 30-year-old woman with a six-month history of right upper-limb claudication who presented with sudden-onset altered sensorium following a one-day febrile illness. Clinical examination revealed bilateral carotid bruits with absent right upper-limb pulses and preserved lower-limb blood pressure. MRI brain demonstrated acute infarcts in the left fronto-temporal region and right caudate nucleus, and CT aortogram showed circumferential wall thickening with significant luminal narrowing of both common carotid arteries, confirming the diagnosis of Takayasu arteritis. The patient was managed with high-dose oral corticosteroids and antiplatelet therapy, with endovascular stenting of the right common carotid artery planned. This case highlights the importance of considering TA in young women presenting with stroke and limb claudication, and the need for early immunosuppressive therapy to prevent irreversible vascular damage.
2026-08-01 | Biological therapy in Takayasu arteritis. Results from the REVAS registry.
Biological therapy in patients with Takayasu arteritis is frequently used, but the factors associated with its use have not been determined. The present study aims to describe and identify predictors of the need for biological therapy in this vasculitis. We conducted a retrospective, multicentre, descriptive study including all patients with Takayasu arteritis enrolled in the REVAS registry from Spain. We performed a descriptive and a multivariate logistic regression analysis to identify variables associated with the need of biological therapy requirement. The study included 77 patients, 65 (84.4 %) women, with a median follow up of 9.9 (interquartile range 3.5-21.5) years. Sixty-three (81.8 %) participants received glucocorticoids and 45 (58.4 %) needed immunosuppressive therapy. Twenty-three patients had received biological therapy, 20 of whom were diagnosed after 2006. Biological therapy included infliximab (n = 3), adalimumab (n = 9) and tocilizumab (n = 18). Patients requiring biological therapy had a higher proportion of systemic symptoms (fever and/or constitutional syndrome) at baseline (70.0 %vs. 28.6 %, p = 0.005), received glucocorticoids more frequently (100.0 %vs. 78.6 %, p = 0.03) and required a greater number of immunosuppressants (2 (1-2) vs. 1 (0-1), p = 0.03) during follow-up. On multivariate logistic regression, the presence of systemic symptoms was associated with the need for biological therapy requirement (odds radio: 3.9 (1.02-14.8), p = 0.047). Conclusion: to our knowledge, this is the first study that evaluates variables associated with the need for biological therapy requirement in Takayasu arteritis. The presence of systemic symptoms at presentation was independently associated with the need for biological therapy, highlighting the importance of a thorough clinical evaluation.
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Drug Discovery Landscape
2 orphan drug designations for Takayasu arteritis.
2 orphan drug designations for Takayasu arteritis.
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
tocilizumab | antibodies | FDA | 2025-12-05 | — | ODDIFACT SAS |
adalimumab | antibodies | FDA | 2025-03-13 | — | ODDIFACT SAS |
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