Pediatric Rheumatology最新文献

{"title":"Diagnosis, management, and monitoring of interleukin-1 mediated diseases in Central and Eastern Europe: real-world data.","authors":"Marija Jelusic, Mario Sestan, Natasa Toplak, Constantin Tamas, Jelena Vojinovic, Zbigniew Zuber, Beata Wolska-Kusnierz, Mihaela Sparchez, Milos Jesenak, Skirmante Rusoniene, Valda Stanevica, Pavla Dolezalova, Liora Harel, Yosef Uziel, Marco Gattorno","doi":"10.1186/s12969-025-01105-3","DOIUrl":"10.1186/s12969-025-01105-3","url":null,"abstract":"<p><strong>Background: </strong>Global healthcare disparities, stemming from organizational differences in healthcare systems, lead to variable availability and funding, resulting in a gap between recommended and implemented practices for interleukin (IL)-1-mediated autoinflammatory diseases. We aimed to assess diagnostic, treatment and follow-up options for these diseases in Central and Eastern European countries, comparing them with the 2021 recommendations of the European Alliance of Associations for Rheumatology (EULAR)/American College of Rheumatology (ACR).</p><p><strong>Methods: </strong>In 2023, a structured collaborative effort was organized with representatives from 10 Central and Eastern European countries to address autoinflammatory diseases. The discussion focused on potential strategies to achieve the goals mentioned above.</p><p><strong>Results: </strong>Almost all the participating countries have specialized centers for the diagnosis and treatment of autoinflammatory diseases and the care is provided either by rheumatologists and/or clinical immunologists. Genetic testing is available in all countries, but there is variation in the types of tests offered. Massive parallel sequencing panels for autoinflammatory diseases are available in all countries, with waiting periods for results ranging from 3 to 6 months in most cases. The availability of disease-specific laboratory assessments, such as S100 proteins, is limited. IL-1 inhibitors are available in all countries, but there are differences in practices regarding the licensing and reimbursement of anakinra and canakinumab based on specific indications or diagnoses. The age at which the transition process begins varies, but in most countries, it typically starts around the age of 18 or beyond and in majority of the participating countries there is no structured transition program.</p><p><strong>Conclusions: </strong>Adherence to the 2021 EULAR/ACR recommendations for IL-1-mediated autoinflammatory diseases is achievable in Central and Eastern European countries. Determining the prevalence and incidence of these diseases in this region remains a persistent challenge for future research efforts, with the overarching goal of identifying new patients with autoinflammatory diseases.</p>","PeriodicalId":54630,"journal":{"name":"Pediatric Rheumatology","volume":"23 1","pages":"56"},"PeriodicalIF":2.8,"publicationDate":"2025-05-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12100914/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144128748","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Nailfold videocapillaroscopy in patients with deficiency of adenosine deaminase 2 (DADA2): a case-control study.","authors":"Pietro Francesco Bica, Caterina Matucci-Cerinic, Elvis Hysa, Andrea Cere, Silvia Rosina, Stefano Volpi, Roberta Caorsi, Alberto Sulli, Sabrina Paolino, Vanessa Smith, Maurizio Cutolo, Marco Gattorno","doi":"10.1186/s12969-025-01104-4","DOIUrl":"10.1186/s12969-025-01104-4","url":null,"abstract":"<p><strong>Background: </strong>Deficiency of adenosine deaminase 2 (DADA2) is a rare monogenic autoinflammatory disease mainly characterized by the presence of systemic inflammation and vascular manifestations such as vasculitis and early-onset stroke. Raynaud's phenomenon (RP) can occur in up to 22% of DADA2 patients. The aim of this work was to investigate the microvascular status of DADA2 patients by the mean of nailfold videocapillaroscopy (NVC) comparing them with adequate healthy controls (HC) and primary RP patients.</p><p><strong>Findings: </strong>NVC data of 9 DADA2 patients (mean age 18 ± 6 y) followed at the Children Gaslini Institute were retrospectively retrieved and compared to age and sex cross matched 11 HCs and 7 with primary RP patients. The NVC parameters were classified according to the EULAR SG Fast Track Algorithm and distinguished between scleroderma-pattern (giant capillaries and/or loss of capillaries combined with abnormally shaped capillaries) and non-scleroderma patterns (non-specific NVC alterations). In all DADA2 patients, NVC showed the presence of non-specific alterations (capillaries with dilations in 100% of cases, abnormal shapes in 23% and microhaemorrhages in 11% of patients). The capillary density was normal and no scleroderma pattern was found. Between DADA2, RP patients and HC, no significant differences in the rate of each microvascular finding were detected (p values NS).</p><p><strong>Conclusions: </strong>This is the first report on NVC in DADA2 patients. Only non-specific abnormalities were found, characterized mainly by capillaries' dilations, but in the absence of giant capillaries. However, larger studies are needed to definitively disclose the microvascular status in DADA2 disease.</p>","PeriodicalId":54630,"journal":{"name":"Pediatric Rheumatology","volume":"23 1","pages":"58"},"PeriodicalIF":2.8,"publicationDate":"2025-05-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12100850/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144133119","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Insights from the 2024 pediatric rheumatology basic/translational years in review.","authors":"Jessica L Turnier, Scott W Canna","doi":"10.1186/s12969-025-01102-6","DOIUrl":"10.1186/s12969-025-01102-6","url":null,"abstract":"<p><strong>Background: </strong>Advances in Pediatric Rheumatology are driven by mechanistic insights from basic and translational science. We have selected and reviewed the most impactful basic/translational science from our \"Year in Review (YIR)\" presentations from the 2024 Pediatric Rheumatology European Society and American College of Rheumatology Convergence meetings (September and November 2024, respectively).</p><p><strong>Main body: </strong>We drew from fundamental immunology, human genetics, animal models, and computational & \"omic\" manuscripts published in the year preceding these meetings. Avoiding overlap with other topics presented in this \"Perspectives\" series, summarized herein are the major themes we gleaned from that process. These include (1) innovative concepts and tools to study immune health, (2) new mechanistic insights into pediatric rheumatic diseases and (3) novel therapeutic targets and treatment approaches in rheumatic disease.</p><p><strong>Conclusions: </strong>As part of a living relationship with the basic/translational literature that shapes our field and practice, we hope readers will be inspired to delve more deeply into the topics and manuscripts highlighted in this YIR summary.</p>","PeriodicalId":54630,"journal":{"name":"Pediatric Rheumatology","volume":"23 1","pages":"57"},"PeriodicalIF":2.8,"publicationDate":"2025-05-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12100868/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144132891","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Bayesian trial of adalimumab versus secukinumab for children with juvenile idiopathic arthritis associated uveitis or chronic anterior uveitis.","authors":"Athimalaipet V Ramanan, Andrew D Dick, Thomas Jaki, Gianmarco Caruso, David S Robertson, Ashley P Jones, Ben Hardwick, Sian Drake, Balini Balasubramaniam, Coziana Ciurtin, Ivan Foeldvari, Elke O Kreps, Alice Leahy, Kristina May, Pierre Quartier, Matthieu P Robert, Gabriele Simonini, Catherine Guly, Michael W Beresford","doi":"10.1186/s12969-025-01107-1","DOIUrl":"10.1186/s12969-025-01107-1","url":null,"abstract":"<p><strong>Background: </strong>Juvenile idiopathic arthritis (JIA)-associated uveitis and chronic anterior uveitis in children may result in permanent sight loss. Currently, the only licensed and approved treatment for JIA-uveitis is adalimumab. However, even in patients where adalimumab may be initially effective, therapeutic response may subside for example, due to neutralising drug antibodies. Further treatment options are necessary to prevent sight loss in children with uveitis. Interleukin 17 is elevated in uveitis. Inhibition of interleukin 17 ameliorates inflammation in mouse models of uveitis. Secukinumab, an antibody which neutralizes interleukin 17 A, has been shown to be partially effective in adult uveitis. The objective of the Bayesian consensus meeting was to quantify prior expert opinion about the potential utility of secukinumab in treatment of uveitis in JIA.</p><p><strong>Methods: </strong>Nine international experts in paediatric rheumatology, paediatric ophthalmology and/or paediatric uveitis took part in a structured Bayesian prior elicitation meeting.</p><p><strong>Results: </strong>The final consensus was that adalimumab is expected to yield a higher response rate than secukinumab (mean 0.67 vs. 0.55). The uncertainty in the response rate on secukinumab is somewhat larger than for adalimumab. The equivalent sample size for the prior distribution of adalimumab is 15.7 and 13.1 for secukinumab. The decisions based on the combined evidence would still be driven by the trial data, yet substantial enhancement of the power of the study can be expected by adding information from the equivalent of almost 30 patients.</p><p><strong>Conclusions: </strong>The Bayesian analysis adds substantial enhancement of the power of the study and supports a head-to-head trial of adalimumab and secukinumab for JIA-associated uveitis and chronic anterior uveitis.</p><p><strong>Trial registration: </strong>ISRCTN 12,427,150 Registration date 14/02/2023. EudraCT 2022-003068-26 Registration date 07/09/2022.</p>","PeriodicalId":54630,"journal":{"name":"Pediatric Rheumatology","volume":"23 1","pages":"55"},"PeriodicalIF":2.8,"publicationDate":"2025-05-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12090588/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144103233","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A mixed-method systematic review evaluating interventions in paediatric rheumatology to address caregiver support and well-being.","authors":"K Kupiec, A Najafi, J Ledochowski, H Chesters, P Livermore","doi":"10.1186/s12969-025-01090-7","DOIUrl":"10.1186/s12969-025-01090-7","url":null,"abstract":"<p><strong>Background: </strong>Paediatric rheumatology conditions are chronic autoimmune and auto-inflammatory conditions that affect predominantly the musculoskeletal systems of children and young people (CYP). About 6-7 million children are estimated to be affected worldwide. Having a rheumatological condition affects CYP as well as the wider family. Negative outcomes on psycho-social well-being, quality of life, family relationships and family functioning are commonly observed. The current review addresses interventions for caregivers of CYP with paediatric rheumatological conditions.</p><p><strong>Methods: </strong>The Preferred Reporting Items for Systematic Reviews and Meta-analyses (PRISMA) 2020 statement guided this review. Eligibility criteria were pre-defined and registered in the International Prospective Register of Systematic Reviews (PROSPERO). Articles were included if they (1) were targeted at caregivers of CYP with rheumatological conditions, and (2) included an intervention to improve well-being of caregivers.</p><p><strong>Results: </strong>Of 1065 identified studies, 15 studies were included in the final review. A mixed-method systematic review was conducted, and included literature was assessed using the Mixed-Method Appraisal Tool (MMAT). Quantitative, qualitative and mixed methods studies, as well as review articles and abstracts investigating the effectiveness of caregiver interventions were evaluated. A third of the identified literature did not report on outcome measures. A narrative synthesis was employed to appraise interventions tailored at caregivers.</p><p><strong>Conclusions: </strong>Despite evidence suggesting that a family approach is needed to support caregivers and CYP with rheumatological conditions as well as the wider family to improve health outcomes for the child, increase family functioning, reduce family conflict, and increase psycho-social well-being, only a small number of caregiver interventions have been carried out to date. The review highlights the need for caregiver interventions to be appraised to better understand what interventions yield results that lead to better quality of life for families who are caring for a child with a chronic rheumatological condition.</p><p><strong>Trial registration: </strong>PROSPERO CRD42024524570.</p>","PeriodicalId":54630,"journal":{"name":"Pediatric Rheumatology","volume":"23 1","pages":"54"},"PeriodicalIF":2.8,"publicationDate":"2025-05-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12082927/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144086960","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Application of the Scleroderma Clinical Trials Consortium Damage Index in patients with juvenile systemic sclerosis.","authors":"Caihui Zhang, Changyan Wang, Sihao Gao, Mingsheng Ma, Wei Wang, Tianyu Zhang, Yu Zhang, Xiaoyan Tang, Zhuo Li, Zhixing Sun, Lin Wang, Hongzhong Jin, Xiaofeng Zeng, Hongmei Song","doi":"10.1186/s12969-025-01110-6","DOIUrl":"https://doi.org/10.1186/s12969-025-01110-6","url":null,"abstract":"<p><strong>Background: </strong>Juvenile systemic sclerosis (jSSc) can lead to permanent and irreversible anatomical or physiological dysfunction. The Scleroderma Clinical Trials Consortium-Damage Index (SCTC-DI), which has been employed and validated in adult patients, can quantify organ damage and predict mortality and morbidity. However, its application in paediatric patients remains unexplored.</p><p><strong>Methods: </strong>Clinical data, laboratory results, and prognostic information were collected for patients with jSSc at Peking Union Medical College Hospital (PUMCH) from January 2012 and January 2024. Differences between the SCTC-DI and the juvenile systemic sclerosis severity score (J4S) were recorded and compared. Furthermore, we compared the SCTC-DI between jSSc and adult systemic sclerosis (SSc) patients.</p><p><strong>Results: </strong>A total of 64 jSSc patients were included. Facet joint contractures, fingertip ulcers and interstitial lung disease are common manifestations. Compared with adult SSc patients, jSSc patients had a lower incidence of gastrointestinal and urinary system involvement. The baseline J4S levels were significantly correlated with SCTC-DI levels at follow-up. A higher baseline SCTC-DI score was associated with a greater progression of organ damage (P = 0.001).</p><p><strong>Conclusion: </strong>There are differences in clinical presentations between adult SSc patients and jSSc patients. The SCTC-DI can be applied to JSSc patients, and it is recommended that JSSc patients undergo regular evaluations of the J4S as well as the SCTC-DI.</p>","PeriodicalId":54630,"journal":{"name":"Pediatric Rheumatology","volume":"23 1","pages":"53"},"PeriodicalIF":2.8,"publicationDate":"2025-05-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12079982/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144082100","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"International multidisciplinary consensus on the definition and clinical approach for monogenic inflammatory immune dysregulation disorders.","authors":"Hend M Alkwai, Ibrahim A Almaghlouth, Leonardo Oliveira Mendonça, Shuayb Elkhalifa, Hassan Abolhassani, Suliman Aljumaah, Hamoud Al-Mousa, Mohammed F Alosaimi, Alhanouf AlSaleem, Tadej Avcin, Winnie Ky Chan, Graciela Espada, Marie-Louise Frémond, Ahmet Gül, Djohra Hadef, Nasim Movahedi, Helmut Wittkowski, Sulaiman M Al-Mayouf","doi":"10.1186/s12969-025-01109-z","DOIUrl":"10.1186/s12969-025-01109-z","url":null,"abstract":"<p><strong>Objective: </strong>To achieve consensus on the definition and clinical approach of Monogenic Inflammatory Immune Dysregulation Disorders (MIIDDs), a collective term for rare conditions marked by inflammation, immune dysregulation, and infection susceptibility. These consensus guidelines specifically apply to pathogenic (or likely pathogenic) gene mutations affecting both innate and adaptive immunity, excluding variants of unknown significance (VUS).</p><p><strong>Methods: </strong>A multi-step, evidence-based, multidisciplinary consensus process was employed, consisting of: (1) a systematic literature review across four electronic databases (Cochrane Library, Web of Science, Scopus, and MEDLINE via PubMed), updated through December 31, 2024; (2) a pre-Delphi electronic survey completed by 95 international adult and pediatric immunologists and rheumatologists; and (3) a modified online Delphi process with an international multidisciplinary expert panel, where statements were iteratively analyzed and refined until achieving consensus (≥ 80% agreement among panelists).</p><p><strong>Results: </strong>Fifteen experts from 12 countries participated in two rounds of the Delphi process, resulting in the development of eight overarching principles and 10 consensus statements. These were categorized into five domains: (1) definitions and conceptual framework, (2) diagnostic and monitoring considerations, (3) treatment and therapeutic strategies, (4) multidisciplinary and collaborative care, and (5) patient education and support.</p><p><strong>Conclusion: </strong>This consensus defines MIIDDs and provides a structured clinical framework to streamline research efforts and improve patient outcomes.</p>","PeriodicalId":54630,"journal":{"name":"Pediatric Rheumatology","volume":"23 1","pages":"51"},"PeriodicalIF":2.8,"publicationDate":"2025-05-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12079908/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144082105","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Investigating the association between SARS-CoV-2 infection, COVID-19 vaccination, and autoimmune diseases in a pediatric population: a comprehensive analysis.","authors":"Cynthia Freiberg, Arad Dotan, Dana Arnheim, Yonatan Butbul Aviel","doi":"10.1186/s12969-025-01093-4","DOIUrl":"https://doi.org/10.1186/s12969-025-01093-4","url":null,"abstract":"<p><strong>Background: </strong>During the COVID-19 pandemic there were reports of an increased association between COVID 19 and various autoimmune diseases (AID) in adults. This study aims to investigate the incidence of AIDs in children before and during the pandemic and explores potential links to SARS-CoV-2 vaccination.</p><p><strong>Methods: </strong>We analyzed 493,705 anonymized medical records from Maccabi Healthcare Services, Israel's second-largest healthcare provider, to study AID incidence during 2014-2022. The study period was divided into three phases: two pre-pandemic phases of equal duration (A and B) and a pandemic phase (C).</p><p><strong>Results: </strong>Of 4,596 (0.9%) patients diagnosed with an AID in the cohort, incidence rates were 0.9% for Group A (2014-2016), 1.0% for Group B (2017-2019), and 0.9% for Group C (2020-2022) (p = 0.13). Logistic regression showed no significant differences in overall autoimmune disease incidence between the pre-COVID and COVID periods. Notably, specific conditions like celiac disease showed reduced incidence in Group A (OR 0.8309, p = 0.0071) while arthritis was significantly more common in Groups A and B. Additionally, COVID-19 diagnosis was not significantly associated with increased autoimmune disease risk (HR 1.092, p = 0.491); however, receiving at least one COVID vaccine was linked to higher risk (HR 1.2323, p = 0.0033).</p><p><strong>Conclusion: </strong>Our findings suggest that the overall incidence of new-onset autoimmune diseases in children remained relatively stable during the COVID-19 pandemic. The study indicates a potential association between COVID-19 vaccination and an increased risk of developing autoimmune diseases, necessitating further research to elucidate long-term effects in the pediatric population.</p>","PeriodicalId":54630,"journal":{"name":"Pediatric Rheumatology","volume":"23 1","pages":"52"},"PeriodicalIF":2.8,"publicationDate":"2025-05-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12080261/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144082108","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Chronic non-bacterial osteomyelitis in children- five-year standardized follow-up of a prospective observational cohort in the pre-biological era.","authors":"Christine Hofmann, Annette Holl-Wieden, Christiane Reiser, Meinrad Beer, Peter Raab, Henner Morbach, Hermann J Girschick","doi":"10.1186/s12969-025-01106-2","DOIUrl":"10.1186/s12969-025-01106-2","url":null,"abstract":"<p><strong>Background: </strong>This prospective, long-term observational study, initiated in 2002, aimed to characterize clinical and laboratory data, whole body MRI detected lesions, and treatment responses in 37 juvenile patients with chronic non-bacterial osteomyelitis at a time when biological DMARDs were not yet standard therapy.</p><p><strong>Methods: </strong>Patients were assessed at baseline and at 1 (without MRI), 3, 6, 12, 18, 24, 36, 48, 60 months. All patients received naproxen as first-line therapy. Clinical management allowed for escalation to sulfasalazine, pamidronate, and glucocorticoids as needed. Treatment response was evaluated using the pedCNO disease activity score (30/50/70/90% improvement). Further composite numeric disease activity (DA) scores- the CARRA CDAS and a new MRI DAS - were applied.</p><p><strong>Results: </strong>The mean age at disease onset was 10.8 years, with a diagnostic delay of 5.8 months. Naproxen was the initial treatment in all patients. Second-line therapy was initiated in 10 patients due to inadequate improvement in physician global assessment of disease activity, patient-reported overall wellbeing or MRI lesions. Escalated therapies included sulfasalazine (n = 10), bisphosphonates (n = 1), methotrexate (n = 1), and short- (< 4 wks) or long-term oral glucocorticoids (n = 5 and n = 3, respectively). The mean number of clinical lesions decreased from 2.1 to 0.4 at 12 months and reached 0.15 at 60 months. MRI-detected lesions declined from 5.0 to 2.25 at 12 months and to 1.1 at 60 months.</p><p><strong>Conclusion: </strong>Most children experienced favourable long-term outcomes. Clinical improvement occurred more rapidly than radiologic resolution. Patients with insufficient response to NSAIDs should be considered for a treat-to-target approach, including the use of conventional and biologic DMARDs.</p><p><strong>Trial registration: </strong>A trial registration EUDRA CT was not available at the time the study was started. Informed consent was given by all parents.</p>","PeriodicalId":54630,"journal":{"name":"Pediatric Rheumatology","volume":"23 1","pages":"50"},"PeriodicalIF":2.8,"publicationDate":"2025-05-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12076821/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143995452","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Effect of additional intravenous immunoglobulin for infliximab-refractory Kawasaki disease: a cohort study.","authors":"Satoki Hatano, Hiro Nakao, Hiroshi Masuda, Hiroshi Ono, Mitsuru Kubota, Akira Ishiguro","doi":"10.1186/s12969-025-01108-0","DOIUrl":"10.1186/s12969-025-01108-0","url":null,"abstract":"<p><strong>Background: </strong>Infliximab (IFX) is a reliable choice of treatment for intravenous immunoglobulin (IVIG)-resistant Kawasaki disease (KD) patients. Nationwide surveys in Japan demonstrated that additional treatment was still required for 20-30% of patients after IFX infusion. Additional IVIG was selected for 70% as the treatment for KD refractory to IFX. This study aimed to describe the therapeutic effect of IVIG after IFX for patients with KD refractory to IFX.</p><p><strong>Methods: </strong>A cohort study was conducted at a single center involving patients treated with additional IVIG for KD refractory to IFX between January 2016 and March 2023 (IVIG-after-IFX group). Additionally, KD patients resistant to the initial IVIG and who received a second IVIG in 2016 were included as a comparison group (second-IVIG group). We employed descriptive statistics and survival analysis to describe their clinical course information, including the time from initiation of the treatment to resolution of fever and the appearance of coronary artery lesions (CALs).</p><p><strong>Results: </strong>The analysis included 27 cases in the IVIG-after-IFX group. The additional IVIG-after-IFX was initiated on a median 11 days of illness (range 8-29). The median time until fever resolution was 1.0 day in the IVIG-after-IFX group and 1.0 day in the second-IVIG group (P = 0.783, HR 1.00; 95% CI 0.58-1.70). The fever resolved within 2.0 days after the initiation of the therapy in 78% (21/27) in the IVIG-after-IFX group and 68% (26/38) in the second-IVIG group. CALs were identified in 26% (7/27) before initiating IVIG-after-IFX, and 7% (2/27) showed new CALs after IVIG after IFX. Persistent CALs were observed in 19% (5/27) after 12 months after diagnosis.</p><p><strong>Conclusions: </strong>Additional IVIG for IFX-refractory KD may have a therapeutic effect comparable to that of the second IVIG for IVIG-resistant KD and be a hopeful therapeutic option for IFX-refractory KD. Treatment of IFX-refractory KD remains a challenge for us and requires further exploration, particularly regarding CAL prevention.</p>","PeriodicalId":54630,"journal":{"name":"Pediatric Rheumatology","volume":"23 1","pages":"49"},"PeriodicalIF":2.8,"publicationDate":"2025-05-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12076848/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144046274","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}