Pub Date : 2025-02-13DOI: 10.1093/rheumatology/keaf095
Simonetta R G van Griethuysen, Quirine A Dumoulin, Elise van Mulligen, Annette H M van der Helm-van Mil
Objectives: Negative illness perceptions(IPs) are associated with poorer disease-outcomes in rheumatoid arthritis(RA). Unfortunately, IPs are generally stable in established RA. We hypothesized that IPs, especially in the cognitive domain, are modifiable in arthralgia-at-risk of RA. We aimed to study if receiving DMARD-treatment, or the offer of DMARD-treatment associates with more positive IPs in patients with clinically-suspect-arthralgia(CSA).
Methods: The population studied were CSA-patients to which a wait-and-see approach was adopted without offering DMARD-treatment, or patients were offered DMARD-treatment via the TREAT EARLIER-trial and subsequently randomized to receive methotrexate or placebo. IP was assessed using the Brief-Illness-Perception-Questionnaire(BIPQ), covering cognitive, emotional and comprehensibility domains. The effect of DMARD-treatment on IPs over time was studied by comparing the 2-year-course of BIPQs of patients receiving methotrexate or placebo. The effect of offering DMARD-treatment was examined by comparing the BIPQs of CSA-patients in the trial with those undergoing a 'wait-and-see'-policy.
Results: In total 375 CSA-patients were studied, of which 236 of the TREAT EARLIER-trial and 139 with a wait-and-see approach. Patients who received treatment showed sustained improvements in IPs over time compared with placebo in four cognitive domains: experience of physical complaints(p= 0.040), the illness' influence on life (p= 0.001), treatment-effectiveness(p= 0.041) and disease-duration (p= 0.045). Comparison at baseline showed that CSA-patients to whom treatment was offered had more confidence in treatment (p< 0.001) and tented to have a deeper understanding of their disease (p= 0.054).
Conclusion: Both the prospect of and DMARD-treatment itself improved IPs in CSA, mainly in cognitive domains. These data suggest CSA as a suitable time period for influencing IPs', which may provide possibilities to improve disease outcomes in patients developing RA.
{"title":"Can treatment expectations or treatment itself in patients with arthralgia suspicious for progression to rheumatoid arthritis improve illness perceptions?","authors":"Simonetta R G van Griethuysen, Quirine A Dumoulin, Elise van Mulligen, Annette H M van der Helm-van Mil","doi":"10.1093/rheumatology/keaf095","DOIUrl":"https://doi.org/10.1093/rheumatology/keaf095","url":null,"abstract":"<p><strong>Objectives: </strong>Negative illness perceptions(IPs) are associated with poorer disease-outcomes in rheumatoid arthritis(RA). Unfortunately, IPs are generally stable in established RA. We hypothesized that IPs, especially in the cognitive domain, are modifiable in arthralgia-at-risk of RA. We aimed to study if receiving DMARD-treatment, or the offer of DMARD-treatment associates with more positive IPs in patients with clinically-suspect-arthralgia(CSA).</p><p><strong>Methods: </strong>The population studied were CSA-patients to which a wait-and-see approach was adopted without offering DMARD-treatment, or patients were offered DMARD-treatment via the TREAT EARLIER-trial and subsequently randomized to receive methotrexate or placebo. IP was assessed using the Brief-Illness-Perception-Questionnaire(BIPQ), covering cognitive, emotional and comprehensibility domains. The effect of DMARD-treatment on IPs over time was studied by comparing the 2-year-course of BIPQs of patients receiving methotrexate or placebo. The effect of offering DMARD-treatment was examined by comparing the BIPQs of CSA-patients in the trial with those undergoing a 'wait-and-see'-policy.</p><p><strong>Results: </strong>In total 375 CSA-patients were studied, of which 236 of the TREAT EARLIER-trial and 139 with a wait-and-see approach. Patients who received treatment showed sustained improvements in IPs over time compared with placebo in four cognitive domains: experience of physical complaints(p= 0.040), the illness' influence on life (p= 0.001), treatment-effectiveness(p= 0.041) and disease-duration (p= 0.045). Comparison at baseline showed that CSA-patients to whom treatment was offered had more confidence in treatment (p< 0.001) and tented to have a deeper understanding of their disease (p= 0.054).</p><p><strong>Conclusion: </strong>Both the prospect of and DMARD-treatment itself improved IPs in CSA, mainly in cognitive domains. These data suggest CSA as a suitable time period for influencing IPs', which may provide possibilities to improve disease outcomes in patients developing RA.</p>","PeriodicalId":21255,"journal":{"name":"Rheumatology","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2025-02-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143410403","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-02-13DOI: 10.1093/rheumatology/keaf099
Karim Dorgham, Maude Calixte, Omaira Da Mata Jardin, Zahir Amoura, Guy Gorochov, Alexis Mathian
{"title":"Comment On: Association of serum interferon alpha-2a levels with disease severity and prognosis in systemic sclerosis.","authors":"Karim Dorgham, Maude Calixte, Omaira Da Mata Jardin, Zahir Amoura, Guy Gorochov, Alexis Mathian","doi":"10.1093/rheumatology/keaf099","DOIUrl":"https://doi.org/10.1093/rheumatology/keaf099","url":null,"abstract":"","PeriodicalId":21255,"journal":{"name":"Rheumatology","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2025-02-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143410405","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-02-12DOI: 10.1093/rheumatology/keaf096
Xavier Sendaydiego, Laura S Gold, Maureen Dubreuil, James S Andrews, Pankti Reid, David F L Liew, Radjiv Goulabchand, Grant C Hughes, Jeffrey A Sparks, Jeffrey G Jarvik, Siddharth Singh, Jean W Liew, Namrata Singh
Objectives To assess the comparative safety of tumor necrosis factor inhibitor (TNFi), non-TNFi, and Janus kinase inhibitor (JAKi) biologic or targeted synthetic disease-modifying anti-rheumatic drugs (b/tsDMARD) in patients with rheumatoid arthritis (RA) for the risk of major adverse cardiovascular events (MACE) using US administrative claims data. Methods We performed a cohort study using MerativeTM Marketscan® Research Databases (2012–2021) of individuals aged 18–64 years with RA initiating b/tsDMARD treatment. We used Cox proportional hazard models to estimate hazard ratios (HR) and 95% CI for developing MACE within 2 years of b/tsDMARD initiation, adjusting for potential confounders. Results We included a total of 34 375 treatment exposures: 71% TNFi, 10% JAKi, 8% abatacept, 5% rituximab, and 5% IL-6i. Most individuals were female (77–84%) with a median (interquartile range) of 50 (42, 56) years. Rituximab had the highest incidence rate of MACE (196/10 000 person-years; 95% CI 126, 291), followed by IL-6i (111/10 000 person-years; 95% CI 57, 193). Multivariable analyses showed non-statistically significantly higher MACE risk with rituximab (HR 1.5; 95% CI 0.9, 2.4) and IL-6i (HR 1.3; 95% CI 0.7, 2.4) exposures but no increased risk with JAKi relative to TNFi use. Conclusion In this large nationwide study, rituximab and IL-6i users had numerically higher, but not statistically significant, MACE risk. Our data support the safety of b/tsDMARD use for RA treatment. This study was limited by short follow-up time and confounding by indication; further studies that can overcome these limitations are needed.
{"title":"Comparative safety of biologic and targeted synthetic disease-modifying anti-rheumatic drugs for cardiovascular outcomes in rheumatoid arthritis","authors":"Xavier Sendaydiego, Laura S Gold, Maureen Dubreuil, James S Andrews, Pankti Reid, David F L Liew, Radjiv Goulabchand, Grant C Hughes, Jeffrey A Sparks, Jeffrey G Jarvik, Siddharth Singh, Jean W Liew, Namrata Singh","doi":"10.1093/rheumatology/keaf096","DOIUrl":"https://doi.org/10.1093/rheumatology/keaf096","url":null,"abstract":"Objectives To assess the comparative safety of tumor necrosis factor inhibitor (TNFi), non-TNFi, and Janus kinase inhibitor (JAKi) biologic or targeted synthetic disease-modifying anti-rheumatic drugs (b/tsDMARD) in patients with rheumatoid arthritis (RA) for the risk of major adverse cardiovascular events (MACE) using US administrative claims data. Methods We performed a cohort study using MerativeTM Marketscan® Research Databases (2012–2021) of individuals aged 18–64 years with RA initiating b/tsDMARD treatment. We used Cox proportional hazard models to estimate hazard ratios (HR) and 95% CI for developing MACE within 2 years of b/tsDMARD initiation, adjusting for potential confounders. Results We included a total of 34 375 treatment exposures: 71% TNFi, 10% JAKi, 8% abatacept, 5% rituximab, and 5% IL-6i. Most individuals were female (77–84%) with a median (interquartile range) of 50 (42, 56) years. Rituximab had the highest incidence rate of MACE (196/10 000 person-years; 95% CI 126, 291), followed by IL-6i (111/10 000 person-years; 95% CI 57, 193). Multivariable analyses showed non-statistically significantly higher MACE risk with rituximab (HR 1.5; 95% CI 0.9, 2.4) and IL-6i (HR 1.3; 95% CI 0.7, 2.4) exposures but no increased risk with JAKi relative to TNFi use. Conclusion In this large nationwide study, rituximab and IL-6i users had numerically higher, but not statistically significant, MACE risk. Our data support the safety of b/tsDMARD use for RA treatment. This study was limited by short follow-up time and confounding by indication; further studies that can overcome these limitations are needed.","PeriodicalId":21255,"journal":{"name":"Rheumatology","volume":"208 1","pages":""},"PeriodicalIF":5.5,"publicationDate":"2025-02-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143393457","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-02-12DOI: 10.1093/rheumatology/keaf094
Paula García-Escudero, Marta López-Gómez, Berta Magallares López, Alicia García Dorta, Beatriz Frade-Sosa, Meritxell Sallés Lizarzaburu, Íñigo Rúa-Figueroa, Dolly Viviana Fiallo, Francisco Javier Toyos Sáenz de Miera, Rafael Benito Melero-Gonzalez, Diego Dios Santos, José Alberto Miranda, Clara García Belando, Giuliano Boselli, Alina Lucica Boteanu, Cristina Corrales Selaya, Cristiana Sieiro Santos, Elvira Díez Álvarez, Judit Font, Elena Riera Alonso, Ernesto Trallero Araguás, Eugenia Enríquez Merayo, María Rodriguez-Laguna, Irene Monjo, Ignacio Vázquez Gómez, Paloma Vela-Casasempere, Carolina Merino, Marta Ibáñez Martínez, José Ángel Hernández Beriain, Alberto Ruiz-Román, Jaime Calvo-Alén
Objectives To describe the clinical spectrum of VEXAS syndrome in patients managed by rheumatology units and analyze genotype-phenotype correlations. Methods A multicentre, cross-sectional, retrospective study was conducted across 126 Spanish hospitals. Patients with VEXAS syndrome diagnosed between December 2020 and January 2024 were included. Demographic data, clinical manifestations, laboratory findings, genetic analyses, treatments, and outcomes were collected from medical records. Results Thirty-nine male patients were included (mean age at diagnosis: 72.78 years). Common manifestations were cutaneous lesions (87.18%), polyarthritis (82.05%) and fever (79.49%). Renal involvement was observed in 20.51% of patients. Genetic testing confirmed UBA1 mutations in all cases: 18 M41L, 14 M41T, 6 M41V, and 1 novel mutation of unknown significance at site c.209T>A. The M41V mutation was significantly associated with renal involvement, while M41T was linked to deep vein thrombosis and thrombocytopenia. Glucocorticoids were used in all patients, with improved response rates post-diagnosis (55.26% vs 97.14%) probably influenced by an increase in administered doses. IL-6 inhibitors and JAK inhibitors showed promising response rates (75% and 76.92%, respectively). Conclusions This study provides insights into the clinical spectrum of VEXAS syndrome in rheumatology settings, highlighting a higher prevalence of joint symptoms and renal involvement than previously reported. Genotype-phenotype correlations were observed, with M41V significantly associated with renal involvement, and M41T linked to deep vein thrombosis and thrombocytopenia. A new, presumably causative variant of VEXAS syndrome at site c.209T>A was described. These findings contribute to the growing understanding of VEXAS syndrome and may inform future diagnostic and treatment strategies.
{"title":"VEXAS syndrome through a rheumatologist's lens: insights from a Spanish national cohort","authors":"Paula García-Escudero, Marta López-Gómez, Berta Magallares López, Alicia García Dorta, Beatriz Frade-Sosa, Meritxell Sallés Lizarzaburu, Íñigo Rúa-Figueroa, Dolly Viviana Fiallo, Francisco Javier Toyos Sáenz de Miera, Rafael Benito Melero-Gonzalez, Diego Dios Santos, José Alberto Miranda, Clara García Belando, Giuliano Boselli, Alina Lucica Boteanu, Cristina Corrales Selaya, Cristiana Sieiro Santos, Elvira Díez Álvarez, Judit Font, Elena Riera Alonso, Ernesto Trallero Araguás, Eugenia Enríquez Merayo, María Rodriguez-Laguna, Irene Monjo, Ignacio Vázquez Gómez, Paloma Vela-Casasempere, Carolina Merino, Marta Ibáñez Martínez, José Ángel Hernández Beriain, Alberto Ruiz-Román, Jaime Calvo-Alén","doi":"10.1093/rheumatology/keaf094","DOIUrl":"https://doi.org/10.1093/rheumatology/keaf094","url":null,"abstract":"Objectives To describe the clinical spectrum of VEXAS syndrome in patients managed by rheumatology units and analyze genotype-phenotype correlations. Methods A multicentre, cross-sectional, retrospective study was conducted across 126 Spanish hospitals. Patients with VEXAS syndrome diagnosed between December 2020 and January 2024 were included. Demographic data, clinical manifestations, laboratory findings, genetic analyses, treatments, and outcomes were collected from medical records. Results Thirty-nine male patients were included (mean age at diagnosis: 72.78 years). Common manifestations were cutaneous lesions (87.18%), polyarthritis (82.05%) and fever (79.49%). Renal involvement was observed in 20.51% of patients. Genetic testing confirmed UBA1 mutations in all cases: 18 M41L, 14 M41T, 6 M41V, and 1 novel mutation of unknown significance at site c.209T&gt;A. The M41V mutation was significantly associated with renal involvement, while M41T was linked to deep vein thrombosis and thrombocytopenia. Glucocorticoids were used in all patients, with improved response rates post-diagnosis (55.26% vs 97.14%) probably influenced by an increase in administered doses. IL-6 inhibitors and JAK inhibitors showed promising response rates (75% and 76.92%, respectively). Conclusions This study provides insights into the clinical spectrum of VEXAS syndrome in rheumatology settings, highlighting a higher prevalence of joint symptoms and renal involvement than previously reported. Genotype-phenotype correlations were observed, with M41V significantly associated with renal involvement, and M41T linked to deep vein thrombosis and thrombocytopenia. A new, presumably causative variant of VEXAS syndrome at site c.209T&gt;A was described. These findings contribute to the growing understanding of VEXAS syndrome and may inform future diagnostic and treatment strategies.","PeriodicalId":21255,"journal":{"name":"Rheumatology","volume":"15 1","pages":""},"PeriodicalIF":5.5,"publicationDate":"2025-02-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143401255","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Objectives Kashin-Beck disease (KBD) is a form of osteoarthropathy that affects the skeletal and joint systems of children and adolescents. Insulin-like Growth Factor Binding Protein 5 (IGFBP5) plays an important role in bone growth and development. This study aimed to investigate the role of IGBFP5 in regulating the function and differentiation of chondrocytes in KBD. Methods The mRNA and protein expressions of IGFBP5, IGF-1 and IGF-1R were detected by RT-qPCR and western blot assays. Commercial kits were performed to measure the mitochondrial ROS content, calcium loading and ATP synthesis in chondrocytes. MTT assay was used to detect the cell viability of chondrocytes. Co-IP and pull-down assays were conducted to verify the binding activity of IGFBP5 to IGF-1R. The rat KBD model was constructed by a low selenium diet and T-2 toxin. Results The expression of IGFBP5 was upregulated in KBD patient and rat tissues. Further studies showed that interfering with IGFBP5 effectively inhibited T-2-induced chondrocyte damage and mitochondrial stress. IGFBP5 depressed the interaction between IGF-1 and IGF-1R, thereby affecting the regulation of IGF-1/IGF-1R signalling in the repair of chondrocytes. In addition, the fibrous differentiation of cartilage progenitor cells (CPCs) and the activity and migration of CPCs induced by T-2 stimulation were suppressed under IGFBP5 silence treatment. Conclusion IGFBP5 was upregulated during the pathological progression of KBD, and IGFBP5 competitively bound with IGF-1R to impede the interactions between IGF-1 and IGF-1R. Knockdown of IGFBP5 inhibited fibrotic differentiation and ameliorated reduction of CPC function in KBD model.
{"title":"IGFBP5 regulates fibrocartilage differentiation and cartilage injury induced by T-2 toxin via blocking IGF-1/IGF-1R signaling","authors":"Xiaoqing Wang, Yinxia Wang, Pengzhen Lei, Xiaodong Qu, Rui Qi, Duanmingyu Chen, Yanhai Chang","doi":"10.1093/rheumatology/keaf084","DOIUrl":"https://doi.org/10.1093/rheumatology/keaf084","url":null,"abstract":"Objectives Kashin-Beck disease (KBD) is a form of osteoarthropathy that affects the skeletal and joint systems of children and adolescents. Insulin-like Growth Factor Binding Protein 5 (IGFBP5) plays an important role in bone growth and development. This study aimed to investigate the role of IGBFP5 in regulating the function and differentiation of chondrocytes in KBD. Methods The mRNA and protein expressions of IGFBP5, IGF-1 and IGF-1R were detected by RT-qPCR and western blot assays. Commercial kits were performed to measure the mitochondrial ROS content, calcium loading and ATP synthesis in chondrocytes. MTT assay was used to detect the cell viability of chondrocytes. Co-IP and pull-down assays were conducted to verify the binding activity of IGFBP5 to IGF-1R. The rat KBD model was constructed by a low selenium diet and T-2 toxin. Results The expression of IGFBP5 was upregulated in KBD patient and rat tissues. Further studies showed that interfering with IGFBP5 effectively inhibited T-2-induced chondrocyte damage and mitochondrial stress. IGFBP5 depressed the interaction between IGF-1 and IGF-1R, thereby affecting the regulation of IGF-1/IGF-1R signalling in the repair of chondrocytes. In addition, the fibrous differentiation of cartilage progenitor cells (CPCs) and the activity and migration of CPCs induced by T-2 stimulation were suppressed under IGFBP5 silence treatment. Conclusion IGFBP5 was upregulated during the pathological progression of KBD, and IGFBP5 competitively bound with IGF-1R to impede the interactions between IGF-1 and IGF-1R. Knockdown of IGFBP5 inhibited fibrotic differentiation and ameliorated reduction of CPC function in KBD model.","PeriodicalId":21255,"journal":{"name":"Rheumatology","volume":"6 1","pages":""},"PeriodicalIF":5.5,"publicationDate":"2025-02-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143393461","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-02-11DOI: 10.1093/rheumatology/keaf088
Hirotaka Yamamoto, Yoshinori Taniguchi, Shigeyoshi Tsuji, Philip S Helliwell, Mitsumasa Kishimoto
{"title":"Efficacy of IL-23 inhibitors in axial involvements of chronic non-bacterial osteitis with palmoplantar pustulosis: a case series.","authors":"Hirotaka Yamamoto, Yoshinori Taniguchi, Shigeyoshi Tsuji, Philip S Helliwell, Mitsumasa Kishimoto","doi":"10.1093/rheumatology/keaf088","DOIUrl":"https://doi.org/10.1093/rheumatology/keaf088","url":null,"abstract":"","PeriodicalId":21255,"journal":{"name":"Rheumatology","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2025-02-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143399890","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Objectives: Corticosteroids are used to abort disease flares in PFAPA syndrome. We aimed to obtain a global overview of physicians' corticosteroid usage strategies and analyze the data in the literature regarding corticosteroid use in PFAPA syndrome.
Methods: The JIR-CliPS PFAPA questionnaire included nine questions on corticosteroid use in addition to the demographic data questions. The survey was distributed via e-mail to potential respondents. The MEDLINE/PubMed and Scopus databases were searched systematically to extract the data regarding corticosteroid use in PFAPA syndrome.
Results: From 47 countries, 144 physicians (F/M = 2.6; 67.4% pediatric rheumatologists) answered the survey. Most respondents (n = 133; 92.4%) prescribe corticosteroids in PFAPA flares. The most frequently prescribed corticosteroid was prednisolone (63.2%). The definition of response to corticosteroid was indicated as "response within 12 h" by the highest number of respondents (n = 61; 42.4%). When corticosteroids cause an increase in attack frequency, most (57.9%) consider another treatment if this causes a decrease in quality of life. Forty-four (30.6%) respondents were "routinely" prescribing corticosteroids to PFAPA patients, and this practice was more frequent among more experienced physicians (p< 0.001).We identified 46 articles in the literature describing 4564 PFAPA patients treated with corticosteroids. Prednisone was the most frequently preferred corticosteroid (48.2%). Response to corticosteroids was around 95%, although an increase in attack frequency was noted in almost 35% of the patients.
Conclusion: Physicians frequently use corticosteroids for PFAPA in their routine clinical practice. Regarding treatment modification, the quality of life was a prominent consideration for physicians.
{"title":"Corticosteroid use in PFAPA syndrome: clinical practice data from the JIR-CliPS Survey Study and a comprehensive literature review.","authors":"Ezgi Deniz Batu, Seher Sener, Mariana Rodrigues, Caroline Vinit, Francois Hofer, Katerina Laskari, Ricardo Craveiro Costa, Margarida Santos Faria, Gulcan Ozomay Baykal, Oksana Boyarchuk, Olivier Gilliaux, Konstantinos Pateras, Hafize Emine Sonmez, Natasa Toplak, Marco Gattorno, Michaël Hofer","doi":"10.1093/rheumatology/keaf036","DOIUrl":"https://doi.org/10.1093/rheumatology/keaf036","url":null,"abstract":"<p><strong>Objectives: </strong>Corticosteroids are used to abort disease flares in PFAPA syndrome. We aimed to obtain a global overview of physicians' corticosteroid usage strategies and analyze the data in the literature regarding corticosteroid use in PFAPA syndrome.</p><p><strong>Methods: </strong>The JIR-CliPS PFAPA questionnaire included nine questions on corticosteroid use in addition to the demographic data questions. The survey was distributed via e-mail to potential respondents. The MEDLINE/PubMed and Scopus databases were searched systematically to extract the data regarding corticosteroid use in PFAPA syndrome.</p><p><strong>Results: </strong>From 47 countries, 144 physicians (F/M = 2.6; 67.4% pediatric rheumatologists) answered the survey. Most respondents (n = 133; 92.4%) prescribe corticosteroids in PFAPA flares. The most frequently prescribed corticosteroid was prednisolone (63.2%). The definition of response to corticosteroid was indicated as \"response within 12 h\" by the highest number of respondents (n = 61; 42.4%). When corticosteroids cause an increase in attack frequency, most (57.9%) consider another treatment if this causes a decrease in quality of life. Forty-four (30.6%) respondents were \"routinely\" prescribing corticosteroids to PFAPA patients, and this practice was more frequent among more experienced physicians (p< 0.001).We identified 46 articles in the literature describing 4564 PFAPA patients treated with corticosteroids. Prednisone was the most frequently preferred corticosteroid (48.2%). Response to corticosteroids was around 95%, although an increase in attack frequency was noted in almost 35% of the patients.</p><p><strong>Conclusion: </strong>Physicians frequently use corticosteroids for PFAPA in their routine clinical practice. Regarding treatment modification, the quality of life was a prominent consideration for physicians.</p>","PeriodicalId":21255,"journal":{"name":"Rheumatology","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2025-02-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143383027","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}