ARP Rheumatology最新文献

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X-linked hypophosphataemia diagnosed in adulthood with coexisting seronegative rheumatoid arthritis: a diagnostic pitfall. 成年并发血清阴性类风湿性关节炎的x连锁低磷血症:一个诊断陷阱
IF 1.3 4区 医学
ARP Rheumatology Pub Date : 2026-07-01
Ana Catarina Moniz, Margarida Patrocínio Gomes, Mariana Emília Santos, Eunice Cacheira, Margarida Mateus, Manuela Costa, Alexandre Sepriano
{"title":"X-linked hypophosphataemia diagnosed in adulthood with coexisting seronegative rheumatoid arthritis: a diagnostic pitfall.","authors":"Ana Catarina Moniz, Margarida Patrocínio Gomes, Mariana Emília Santos, Eunice Cacheira, Margarida Mateus, Manuela Costa, Alexandre Sepriano","doi":"","DOIUrl":"","url":null,"abstract":"<p><strong>Introduction: </strong>X-linked hypophosphataemia (XLH) is the most common inherited cause of renal phosphate wasting, typically diagnosed in childhood but occasionally identified in adulthood.</p><p><strong>Case report: </strong>We report a case of a 45-year-old woman with long-standing lower limb deformities, short stature, and diffuse joint pain. Laboratory evaluation revealed hypophosphataemia due to isolated renal phosphate wasting. Fibroblast growth factor 23 levels were markedly elevated. Imaging showed skeletal deformities and enthesopathic changes consistent with chronic XLH. Genetic testing identified a pathogenic PHEX variant (c.1645+1G>A), confirming the diagnosis. Treatment with oral phosphate and calcitriol resulted in biochemical improvement. During follow-up, the patient developed symmetrical erosive polyarthritis of the hands and wrists, consistent with seronegative rheumatoid arthritis superimposed on the underlying metabolic disorder.</p><p><strong>Discussion: </strong>This case highlights the diagnostic challenge of XLH in adults, where musculoskeletal manifestations can closely mimic inflammatory arthritis. Recognising XLH as the underlying cause of chronic osteomalacia is essential to avoid inappropriate treatment and ensure timely management of coexisting conditions. New-onset erosive arthritis should prompt consideration of a concurrent inflammatory condition rather than attribution to the underlying metabolic disorder.</p>","PeriodicalId":29669,"journal":{"name":"ARP Rheumatology","volume":"5 3","pages":"194-199"},"PeriodicalIF":1.3,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149168189","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
PD-1 inhibitor-associated Sicca syndrome mimicking Sjögren's disease successfully managed without immunotherapy withdrawal. PD-1抑制剂相关的Sicca综合征模仿Sjögren的疾病成功管理没有免疫治疗退出。
IF 1.3 4区 医学
ARP Rheumatology Pub Date : 2026-07-01
Filipa Canhão André, João Teixeira, André Saraiva, João Luís Amaral, Joana Calvão, Inês Coutinho, Margarida Coutinho
{"title":"PD-1 inhibitor-associated Sicca syndrome mimicking Sjögren's disease successfully managed without immunotherapy withdrawal.","authors":"Filipa Canhão André, João Teixeira, André Saraiva, João Luís Amaral, Joana Calvão, Inês Coutinho, Margarida Coutinho","doi":"","DOIUrl":"","url":null,"abstract":"<p><p>Immune checkpoint inhibitors (ICIs) are increasingly associated with a broad spectrum of immune-related adverse events (irAEs), including rheumatic manifestations. We report a case of ICI-associated sicca syndrome (ICI-S) mimicking Sjögren's disease, with a brief literature review. The patient was a 70-year-old man with BRAF-mutated metastatic melanoma who developed inflammatory arthralgias followed by parotid and submandibular enlargement, severe xerostomia, and xerophthalmia, during pembrolizumab therapy. Treatment was withheld and low-dose glucocorticoids (GC) initiated, with rapid improvement of joint symptoms and gland swelling, although marked xerostomia persisted. The diagnostic work-up revealed elevated inflammatory markers and positive antinuclear antibodies (ANA), with negative anti-SSA/SSB. Salivary gland ultrasound showed grade 3 changes (OMERACT) and PET-CT confirmed bilateral sialadenitis. Minor salivary gland biopsy demonstrated T-cell-predominant lymphocytic infiltrate without CD20+ B cells, supporting an ICI-S rather than primary Sjögren's disease. Pembrolizumab rechallenge triggered a flare of sialadenitis and arthritis, controlled with GC reintroduction plus hydroxychloroquine and supportive measures. This case provides educational insights into the diagnostic differentiation between ICI-S and primary Sjögren's disease, supporting early recognition and appropriate management without compromising oncologic treatment.</p>","PeriodicalId":29669,"journal":{"name":"ARP Rheumatology","volume":"5 3","pages":"184-189"},"PeriodicalIF":1.3,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149168564","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Distinct clinical profiles of methotrexate intolerance and hepatotoxicity in psoriatic arthritis. 银屑病关节炎甲氨蝶呤不耐受和肝毒性的独特临床特征。
IF 1.3 4区 医学
ARP Rheumatology Pub Date : 2026-07-01
Cristina Macia-Villa, Mauro Ferre-Sanfrancisco, Ana Maria Ruiz-Bejerano, Jose Luis Morell-Hita
{"title":"Distinct clinical profiles of methotrexate intolerance and hepatotoxicity in psoriatic arthritis.","authors":"Cristina Macia-Villa, Mauro Ferre-Sanfrancisco, Ana Maria Ruiz-Bejerano, Jose Luis Morell-Hita","doi":"","DOIUrl":"","url":null,"abstract":"<p><strong>Introduction: </strong>Methotrexate (MTX) is the most widely used conventional DMARD for psoriatic arthritis (PsA), but intolerance and hepatotoxicity often limit its long-term use. This study aimed to describe the prevalence and clinical correlates of MTX intolerance and hepatotoxicity in PsA patients.</p><p><strong>Methods: </strong>We conducted a retrospective study on adults with PsA who were exposed to MTX. Demographic, clinical, and treatment data were retrieved from electronic medical records. Intolerance was defined as discontinuation or dose reduction due to adverse effects, while hepatotoxicity was defined as discontinuation or dose reduction due to persistent liver enzyme elevation (≥2 times the upper limit of normal). Independent associations were explored using multivariable logistic regression.</p><p><strong>Results: </strong>Among the 341 patients included, MTX intolerance occurred in 26.7% and hepatotoxicity in 10.3%. Bivariate analysis showed that intolerance was associated with female sex, shorter psoriasis-arthritis delay, enthesitis, higher use of csDMARDs and ts/bDMARDs, and lower persistence of TNF and IL-17 inhibitors. No independent predictors of intolerance were identified. Hepatotoxicity was linked to male sex, dactylitis, hypertension, hyperuricemia, and treatment-refractory PsA. In multivariable analysis, hepatotoxicity was independently associated with arterial hypertension (OR 4.40; p 0.007) and treatment-refractory PsA (OR 6.23; p 0.016), and inversely associated with intolerance (OR 0.09; p 0.023).</p><p><strong>Conclusion: </strong>MTX intolerance and hepatotoxicity in PsA were associated with distinct clinical patterns: intolerance predominates in women and is associated with frequent therapeutic modifications, whereas hepatotoxicity is more common in men with metabolic comorbidities and treatment-refractory disease. Recognizing these patterns may help optimize MTX safety, guide therapeutic decisions, and enhance treatment persistence.</p>","PeriodicalId":29669,"journal":{"name":"ARP Rheumatology","volume":"5 3","pages":"145-151"},"PeriodicalIF":1.3,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149170343","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Trends, outcomes, and disparities in hospitalizations among patients with Sjögren's syndrome: a national inpatient sample analysis (2016-2020). Sjögren综合征患者住院的趋势、结果和差异:2016-2020年全国住院患者样本分析
IF 1.3 4区 医学
ARP Rheumatology Pub Date : 2026-07-01
Shaivya Pathak
{"title":"Trends, outcomes, and disparities in hospitalizations among patients with Sjögren's syndrome: a national inpatient sample analysis (2016-2020).","authors":"Shaivya Pathak","doi":"","DOIUrl":"","url":null,"abstract":"<p><strong>Aims: </strong>Sjögren's syndrome (SS) is a systemic autoimmune disease with poorly characterized hospitalization outcomes in the United States. We aimed to characterize hospitalization trends, comorbidity profiles, organ-specific manifestations, predictors of in-hospital mortality, and racial, socioeconomic, and hospital-level disparities among SS-associated hospitalizations using the NIS 2016-2020 dataset.</p><p><strong>Methods: </strong>Retrospective cross-sectional analysis of the NIS (2016-2020). Adults with SS (ICD-10-CM M35.00-M35.0C) were identified across 40 diagnosis fields and matched 3:1 with non-autoimmune controls by age, sex, and year. Survey-weighted analyses included bivariate comparisons, Elixhauser comorbidity profiling, organ-specific phenotyping, nested multivariable logistic regression for mortality predictors, and adjusted disparities analyses using predictive margins.</p><p><strong>Results: </strong>Among 272,630 weighted SS-associated hospitalizations (91.4% female, mean age 64.2 years), extraglandular manifestations were documented in 33.8%. Despite a significantly higher baseline comorbidity burden compared with 817,890 matched control hospitalizations, SS-associated admissions exhibited paradoxically lower crude in-hospital mortality (2.13% vs. 2.59%, p<0.001). However, within the SS cohort, acute complications were associated with substantial mortality risk in the fully adjusted model: independent predictors included mechanical ventilation (OR 21.08), sepsis (OR 2.80), stroke (OR 2.78), malignancy (OR 2.46), and interstitial lung disease (OR 2.10). Significant disparities persisted after multivariable adjustment: Asian/Pacific Islander hospitalizations had higher mortality odds (OR 1.67, p=0.002) and incurred $22,643 more in adjusted charges compared with White hospitalizations. Self-pay or uninsured status was associated with doubled mortality odds (OR 2.01, p<0.001).</p><p><strong>Conclusions: </strong>Enhanced clinical phenotyping underscores that Sjögren's syndrome is a complex, multisystem disease with a substantial inpatient burden. While SS-associated hospitalizations present with high baseline comorbidities, in-hospital mortality is strongly associated with acute complications, particularly respiratory failure, sepsis, stroke, and interstitial lung disease. Future clinical pathways should prioritize vigilant management of these vulnerabilities while addressing the structural and socioeconomic disparities associated with unequal outcomes.</p>","PeriodicalId":29669,"journal":{"name":"ARP Rheumatology","volume":"5 3","pages":"172-183"},"PeriodicalIF":1.3,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149167579","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
When SLE patients have rosacea - key distinguishing features. 当SLE患者有酒糟鼻-关键的区别特征。
IF 1.3 4区 医学
ARP Rheumatology Pub Date : 2026-07-01
Filipa Costa, Gustavo Almeida, Carla Macieira, Paulo Filipe
{"title":"When SLE patients have rosacea - key distinguishing features.","authors":"Filipa Costa, Gustavo Almeida, Carla Macieira, Paulo Filipe","doi":"","DOIUrl":"","url":null,"abstract":"<p><p>Facial erythema associated with antinuclear antibody (ANA) positivity frequently prompts referral to Rheumatology due to suspicion of acute cutaneous lupus erythematosus (ACLE). However, patients with systemic lupus erythematosus (SLE) may present with malar rash that clinically overlaps with rosacea. Because these conditions differ substantially in pathophysiology, prognosis, treatment, and follow-up requirements, distinguishing between them is essential for accurate diagnosis and management. This review summarizes the key clinical, dermoscopic, and histopathological features that aid in differentiating rosacea from ACLE. Correct differentiation between rosacea and ACLE is critical because rosacea is primarily managed with trigger avoidance, topical therapies, and oral antibiotics, whereas ACLE often requires immunosuppressive therapy and long-term systemic monitoring. Misdiagnosis may therefore result in either unnecessary immunosuppression or delayed treatment of systemic autoimmune disease.</p>","PeriodicalId":29669,"journal":{"name":"ARP Rheumatology","volume":"5 3","pages":"213-215"},"PeriodicalIF":1.3,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149169921","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Anifrolumab for refractory autoimmune hemolytic anemia in SPENCD-associated lupus: a case-based review. anfrolumab治疗spencd相关性狼疮难治性自身免疫性溶血性贫血:一项基于病例的回顾
IF 1.3 4区 医学
ARP Rheumatology Pub Date : 2026-07-01
Maria João Cadório, Cátia Duarte
{"title":"Anifrolumab for refractory autoimmune hemolytic anemia in SPENCD-associated lupus: a case-based review.","authors":"Maria João Cadório, Cátia Duarte","doi":"","DOIUrl":"","url":null,"abstract":"<p><p>Spondyloenchondrodysplasia (SPENCD) is a rare monogenic interferonopathy caused by biallelic ACP5 mutations, characterized by skeletal dysplasia, neurological involvement and immune dysregulation frequently manifesting as lupus-like disease. Autoimmune cytopenias, particularly autoimmune hemolytic anemia (AIHA), are among the most severe and treatment-refractory manifestations. Anifrolumab, a monoclonal antibody targeting interferon alpha receptor subunit 1 (IFNAR1), blocks signaling by all type I interferons and is approved for moderate-to-severe systemic lupus erythematosus. We report a 50-year-old man with SPENCD-associated lupus and refractory, transfusion-dependent AIHA who achieved sustained hematologic remission following anifrolumab initiation. Using this case as a framework, we discuss the pathophysiological basis of SPENCD, the spectrum and treatment challenges of its associated autoimmune cytopenias, and the evolving role of targeted therapies in monogenic interferonopathies, with particular emphasis on SPENCD, and summarize the published experience with anifrolumab in these disorders. Collectively, this case and the available literature provide preliminary support for IFNAR1 blockade as a mechanism-based, steroid-sparing strategy for refractory autoimmune cytopenias in SPENCD.</p>","PeriodicalId":29669,"journal":{"name":"ARP Rheumatology","volume":"5 3","pages":"200-207"},"PeriodicalIF":1.3,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149165103","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Manifestations and predictors of vascular involvement in Behçet's disease. behet病血管受累的表现和预测因素。
IF 1.3 4区 医学
ARP Rheumatology Pub Date : 2026-07-01
Roberto Pereira da Costa, Margarida Lucas Rocha, Joana Martins-Martinho, Ana Teodósio Chícharo, Carla Macieira, Cristina Ponte, Nikita Khmelinskii
{"title":"Manifestations and predictors of vascular involvement in Behçet's disease.","authors":"Roberto Pereira da Costa, Margarida Lucas Rocha, Joana Martins-Martinho, Ana Teodósio Chícharo, Carla Macieira, Cristina Ponte, Nikita Khmelinskii","doi":"","DOIUrl":"","url":null,"abstract":"<p><strong>Background: </strong>Behçet´s disease (BD) is a variable vessel vasculitis in which vascular involvement (VBD) occurs in up to 40% of patients and represents a poor prognosis marker. Predictors of VBD have been poorly defined.</p><p><strong>Aims: </strong>We aimed to characterise VBD patients and to identify predictors of this clinical subtype.</p><p><strong>Methods: </strong>We performed a single-centre, retrospective study including patients fulfilling the 2013 International Criteria for BD, registered in the Rheumatic Diseases Portuguese Registry (Reuma.pt) between October 2014 and May 2023. VBD was defined as arterial or venous manifestations attributable to BD and confirmed by imaging. Multivariable logistic regression was used to identify predictors of VBD.</p><p><strong>Results: </strong>A total of 157 BD patients were included, 24 (15%) of whom had vascular involvement. Seven (5%) presented VBD as the inaugural manifestation. The median age at VBD onset was 36.0 years, occurring a median of 8.9 years after BD onset. Venous involvement (20/24, 83%) predominated over arterial (10/24, 42%). Deep vein thrombosis (50%) and superficial thrombophlebitis (42%) were the most common venous events. Arterial manifestations included aneurysms (21%), pulmonary thrombosis (13%) and pseudoaneurysms (8%). Multiple-vessel involvement (54%) and recurrent events (58%) were common. On comparison with patients without VBD, male sex (OR 3.99, 95%CI 1.55-10.28) and ocular involvement at disease onset (OR 3.90, 95%CI 1.29-11.72) emerged as predictors of VBD.</p><p><strong>Conclusion: </strong>In our BD cohort, vascular involvement occurred in 15% of patients, predominantly venous. Male sex and ocular involvement at disease onset were identified as predictors of VBD, suggesting their potential role as clinical markers for identifying high-risk patients.</p>","PeriodicalId":29669,"journal":{"name":"ARP Rheumatology","volume":"5 3","pages":"216-218"},"PeriodicalIF":1.3,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149168316","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
A pediatric case of orbital inflammation and sinusitis- the diagnostic boundary between localized ANCA-associated vasculitis and IgG4-related disease. 眼眶炎症和鼻窦炎的儿童病例-局部anca相关血管炎和igg4相关疾病的诊断界限
IF 1.3 4区 医学
ARP Rheumatology Pub Date : 2026-07-01
Vitória Cadete, Ana Pestana Lopes, Rodrigo Cavalcanti, Manuela Mafra, Rita Luís, Mara Ferreira, Carla Conceição, Inês Soares da Cunha, João Farela Neves, Marta Cabral, Marta Conde
{"title":"A pediatric case of orbital inflammation and sinusitis- the diagnostic boundary between localized ANCA-associated vasculitis and IgG4-related disease.","authors":"Vitória Cadete, Ana Pestana Lopes, Rodrigo Cavalcanti, Manuela Mafra, Rita Luís, Mara Ferreira, Carla Conceição, Inês Soares da Cunha, João Farela Neves, Marta Cabral, Marta Conde","doi":"","DOIUrl":"","url":null,"abstract":"<p><p>ANCA-associated vasculitis (AAV) are characterized by necrotizing inflammation of small- and medium-sized blood vessels, frequently associated with antibodies against myeloperoxidase (MPO) or proteinase 3. In contrast, IgG4-related disease (IgG4-RD) is an immune-mediated fibroinflammatory condition that can affect virtually any organ and is histologically characterized by lymphoplasmacytic infiltrates enriched in IgG4-positive plasma cells. Both conditions may present with localized or systemic disease and share clinical and histopathological features, particularly in the head and neck region, making differential diagnosis challenging. We report the case of an 11-year-old girl who initially presented with bilateral orbital inflammation. Orbital biopsy demonstrated lymphoplasmacytic infiltrates, an elevated IgG4+/IgG+ plasma cell ratio, and obliterative phlebitis, suggestive of IgG4-RD. However, markedly elevated MPO-ANCA levels raised suspicion for AAV. Although glucocorticoid therapy initially achieved clinical improvement, relapses occurred, prompting treatment with rituximab. Subsequently, the patient developed rhinosinusitis, and nasal biopsy revealed lymphoplasmacytic inflammation with multinucleated giant cells, consistent with granulomatosis with polyangiitis (GPA). Treatment was intensified with mycophenolate mofetil and corticosteroids, resulting in remission. This case highlights the diagnostic and therapeutic challenges posed by overlapping features of AAV and IgG4-RD.</p>","PeriodicalId":29669,"journal":{"name":"ARP Rheumatology","volume":"5 3","pages":"208-212"},"PeriodicalIF":1.3,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149169282","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Aortic-renal aneurysm in a patient with VEXAS syndrome treated with Tocilizumab. 托珠单抗治疗的VEXAS综合征患者主动脉-肾动脉瘤
IF 1.3 4区 医学
ARP Rheumatology Pub Date : 2026-07-01
Senol Kobak, Mehmet Hilmi Dogu, Ali Baran Budak, Adem Ucar
{"title":"Aortic-renal aneurysm in a patient with VEXAS syndrome treated with Tocilizumab.","authors":"Senol Kobak, Mehmet Hilmi Dogu, Ali Baran Budak, Adem Ucar","doi":"","DOIUrl":"","url":null,"abstract":"<p><p>VEXAS syndrome is adult onset autoinflammatory disease caused by somatic mutations in the gene UBA1 and characterized with overlapping rheumatologic and hematologic clinical features. The most common clinical features included recurrent fever, artralgia/arthritis, pulmonary involvement, skin lessions, vasculitis, and/or thromboembolic event. Aortic-renal aneurysm in VEXAS syndrome is rare entity and its associated with poor prognosis. Herein we described a 69 year-old male patient with VEXAS syndrome with aortic-renal aneurysm responded well to Tocilizumab treatment.</p>","PeriodicalId":29669,"journal":{"name":"ARP Rheumatology","volume":"5 3","pages":"190-193"},"PeriodicalIF":1.3,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149171983","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Subcutaneous methotrexate for treating rheumatoid arthritis in Portugal: a cost-utility analysis versus current practice. 皮下甲氨蝶呤治疗类风湿性关节炎在葡萄牙:成本效用分析与目前的做法。
IF 1.3 4区 医学
ARP Rheumatology Pub Date : 2026-07-01
Matthew Tucker, Maria João Salvador, Miguel Bernardes, Ana Cristina Van Cauteren Peres Cordeiro, Kristin Grabe-Heyne, Johannes Pöhlmann, Richard F Pollock
{"title":"Subcutaneous methotrexate for treating rheumatoid arthritis in Portugal: a cost-utility analysis versus current practice.","authors":"Matthew Tucker, Maria João Salvador, Miguel Bernardes, Ana Cristina Van Cauteren Peres Cordeiro, Kristin Grabe-Heyne, Johannes Pöhlmann, Richard F Pollock","doi":"","DOIUrl":"","url":null,"abstract":"<p><strong>Aims: </strong>This study evaluated the cost-utility of subcutaneous versus oral methotrexate for rheumatoid arthritis (RA) management in Portugal.</p><p><strong>Methods: </strong>A cost-utility analysis (CUA) was conducted over a lifetime horizon from the Portuguese National Health Service perspective, adapting a model previously published for the United Kingdom. Patients were modelled to transition through lines of therapy including conventional synthetic, followed by biologic and targeted synthetic disease-modifying anti-rheumatic drugs (DMARDs). In the base case, first-line subcutaneous methotrexate was compared with first-line oral methotrexate, while a scenario analysis explored second-line subcutaneous methotrexate use, after failing first-line oral methotrexate. Clinical, quality-of-life, and cost data were sourced from the literature, using Portuguese data where available. Projected costs and benefits were combined in net monetary benefit (NMB) estimates, evaluated against a willingness-to-pay threshold of €50,000 per quality-adjusted life-year (QALY) gained. The robustness of results was explored in sensitivity and scenario analyses.</p><p><strong>Results: </strong>First-line subcutaneous methotrexate was projected to yield an additional 0.25 life-years and 0.55 QALYs, while reducing total costs by €11,108 over patients' lifetimes, relative to first-line oral methotrexate. Using subcutaneous methotrexate after first-line oral methotrexate was associated with gains in life expectancy (+0.54 life-years) and quality-adjusted life expectancy (+1.19 QALYs), while also reducing projected costs (-€25,892). In both analyses, subcutaneous methotrexate was therefore associated with improved effectiveness and reduced total costs. The corresponding NMBs were €38,821 and €85,485, respectively. Health gains resulted from more patient time spent on active treatment, with improved disease control, and consequently better health-related quality of life and reduced mortality risk. Improved disease control also implied reduced hospitalization costs, with costs further reduced due to delaying, or avoiding, costly biologics, which offset higher subcutaneous methotrexate acquisition costs. Sensitivity and scenario analyses confirmed base case results, including for different later-line biologic treatments, clinical response data, and time horizons.</p><p><strong>Conclusions: </strong>This CUA evaluated the value-for money of subcutaneous versus oral methotrexate for RA management in Portugal and found that subcutaneous methotrexate is likely a cost-effective strategy, with the potential to reduce healthcare expenditure and improve patient outcomes. These findings support consideration of wider adoption of subcutaneous methotrexate within Portuguese clinical practice.</p>","PeriodicalId":29669,"journal":{"name":"ARP Rheumatology","volume":"5 3","pages":"152-171"},"PeriodicalIF":1.3,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149165132","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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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