OBJECTIVE:To develop evidence-based Pan American League of Associations for Rheumatology (PANLAR) recommendations for the treatment of oligoarthritis category in juvenile idiopathic arthritis (oligo-JIA) patients. METHODS:A panel of pediatric rheumatologists from Latin-America (LATAM) generated clinically meaningful questions related to the treatment of oligo-JIA patients, using Population, Intervention, Comparator, and Outcome (PICO) format. Following Grading of Recommendations Assessment, Development, and Evaluation (GRADE) methodology, a team of methodologists conducted a systematic literature review, extracted, summarized intervention effect estimates, and graded the evidence quality. LATAM pediatric rheumatology experts' panel voted each PICO question, which required a minimum agreement of 70% among the voting members and developed recommendations. RESULTS:Seven recommendations and 4 expert opinion were developed. Short-term course of NSAID and intra-articular corticosteroids was recommended as initial therapy in those with minimal disease activity with no risk factors for poor prognosis. The initiation of a nbDMARD was recommended in patients with high disease activity and risk factors for poor prognosis. For patients who achieve inactive disease state, treatment with DMARD should be maintained for a minimum of 12 months after remission. We proposed sulfasalazine or leflunomide in patients with methotrexate intolerance, or in the presence of contraindication or unavailability of bDMARD. For oligo-JIA patients with high disease activity or uveitis, anti-TNF agents are recommended as the first choice among bDMARD. Regular physical activity was also recommended for these patients. CONCLUSIONS:The first PANLAR oligo-JIA treatment guidelines provide evidence-based guidance for health care providers, treating patients with oligo-JIA in LATAM.

OBJECTIVES:The objective of this study was to develop and test a composite DAS for JDM solely based on parent-reported outcomes (PROs). METHODS:The parent JDM Activity Index (ParJDMAI) includes the following four PROs: (1) global assessment of disease activity; (2) assessment of fatigue; (3) assessment of muscle disease activity; (4) assessment of skin disease activity. Each item is weighted on a 0-10 scale, which yields a total score ranging from 0 to 40. Validation procedures were conducted on a multicentre sample of 254 patients with JDM according to the OMERACT filter for outcome measures in rheumatology. RESULTS:The ParJDMAI is short and simple, and is quick, taking <10 minutes to complete and score, which makes it practical for use in standard clinical care. In validation analyses, the tool demonstrated good construct validity, satisfactory internal consistency (Cronbach's alpha 0.86) and structure (with unambiguous identification of one factor with eigenvalue 2.9 on exploratory factor analysis), strong ability to discriminate between physician-estimated disease activity states (P < 0.001), and fair responsiveness to clinically important change over time (standardized response mean 0.8 among patients judged as improved by the parents). The ParJDMAI correlated strongly with physician-centred measures of disease activity. CONCLUSION:The ParJDMAI was found to be feasible and to possess good measurement properties. It also proved to potentially serve as a surrogate for physicians' evaluations. Completion of the ParJDMAI through digital technologies may provide a reliable and pragmatic approach for building a remote patient-monitoring program.

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). 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

To develop evidence-based treatment guidelines for non-systemic polyarticular-juvenile idiopathic arthritis (poly-JIA) in Latin America, endorsed by the Pan-American League of Associations for Rheumatology (PANLAR), a panel of paediatric rheumatologists from Latin America formulated clinically relevant questions regarding polyarthritis treatment, using the Population, Intervention, Comparator, and Outcome (PICO) format. Following the Grading of Recommendations Assessment, Development, and Evaluation methodology, a team of methodologists conducted a systematic literature review, extracted and summarised intervention effect estimates, and assessed the quality of evidence. The panel of paediatric rheumatologists voted on each PICO question and formulated recommendations, requiring a consensus of at least 70% amongst the voting members. Eight recommendations and one expert opinion statement were developed. For newly diagnosed poly-JIA or those with minimal disease activity, the use of non-steroidal anti-inflammatory drugs as adjuvant therapy, along with a non-biological disease-modifying antirheumatic drug (nbDMARD) is recommended. For children and young people achieving an inactive disease state, continuation of nbDMARD treatment for at least 12 months post-remission is advised. In cases of methotrexate intolerance, contraindications, limited availability, or non-response, leflunomide could be used as an alternative. For children and young people with high disease activity or poor prognostic factors, the addition of a biological disease-modifying antirheumatic drug (bDMARD) is recommended. Triple therapy with methotrexate, sulfasalazine, and hydroxychloroquine can be considered when bDMARDs are not available. Low-dose, short-term corticosteroid use is also recommended. The first PANLAR poly-JIA treatment guidelines offer evidence-based recommendations to support health-care providers in the management of poly-JIA in Latin America.

We aimed to develop evidence-based Pan American League of Associations for Rheumatology recommendations for the pharmacological treatment of systemic juvenile idiopathic arthritis (sJIA). An expert panel of paediatric rheumatologists from Latin America generated clinically meaningful research questions structured using the Population, Intervention, Comparator and Outcome (PICO) format, adhering to Grading of Recommendations Assessment, Development, and Evaluation methodology. A team of methodologists conducted a systematic literature review, extracted and summarized intervention effect estimates and graded the evidence quality. The JIA expert panel voted on each research question structured using the PICO format, requiring a minimum agreement of 70% among the voting members to formulate recommendations. Four evidence-based recommendations were developed, addressing the two most common phenotypes of sJIA: with predominantly systemic features and with predominantly active synovitis. The optimal therapeutic approach emphasizes the early initiation of IL-1 or IL-6 pathway inhibition, coupled with a short-course corticosteroid regimen. For sJIA patients with predominantly systemic features and high disease activity, high-dose i.v. methylprednisolone ('pulse therapy') is advised. These recommendations highlight the importance of limiting glucocorticoid therapy to the lowest effective dose for the shortest possible duration, with gradual tapering and discontinuation within a maximum period of 6 months. These recommendations provide guidance on strategies for the use of pharmacological agents for sJIA patients.

BACKGROUND:After introducing IL-1/IL-6 inhibitors, some patients with Still and Still-like disease developed unusual, often fatal, pulmonary disease. This complication was associated with scoring as DReSS (drug reaction with eosinophilia and systemic symptoms) implicating these inhibitors, although DReSS can be difficult to recognize in the setting of systemic inflammatory disease. OBJECTIVE:To facilitate recognition of IL-1/IL-6 inhibitor-DReSS in systemic inflammatory illnesses (Still/Still-like) by looking at timing and reaction-associated features. We evaluated outcomes of stopping or not stopping IL-1/IL-6 inhibitors after DReSS reaction began. METHODS:In an international study collaborating primarily with pediatric specialists, we characterized features of 89 drug-reaction cases versus 773 drug-exposed controls and compared outcomes of 52 cases stopping IL-1/IL-6 inhibitors with 37 cases not stopping these drugs. RESULTS:Before the reaction began, drug-reaction cases and controls were clinically comparable, except for younger disease-onset age for reaction cases with preexisting cardiothoracic comorbidities. After the reaction began, increased rates of pulmonary complications and macrophage activation syndrome differentiated drug-reaction cases from drug-tolerant controls (P = 4.7 × 10-35 and P = 1.1 × 10-24, respectively). The initial DReSS feature was typically reported 2 to 8 weeks after initiating IL-1/IL-6 inhibition. In drug-reaction cases stopping versus not stopping IL-1/IL-6-inhibitor treatment, reaction-related features were indistinguishable, including pulmonary complication rates (75% [39 of 52] vs 76% [28 of 37]). Those stopping subsequently required fewer medications for treatment of systemic inflammation, had decreased rates of macrophage activation syndrome, and improved survival (P = .005, multivariate regression). Resolution of pulmonary complications occurred in 67% (26 of 39) of drug-reaction cases who stopped and in none who continued inhibitors. CONCLUSIONS:In systemic inflammatory illnesses, recognition of IL-1/IL-6-inhibitor-associated reactions followed by avoidance of IL-1/IL-6 inhibitors significantly improved outcomes.

Objectives:Our aim was to describe the epidemiology and outcomes of multisystem inflammatory syndrome in children (MIS-C) in Latin America. Methods:We conducted an observational, retrospective, and prospective multicenter study that gathered information from 84 participating centers across 16 Latin American countries between August 1, 2020 and June 30, 2022. Results:Of the 1239 reported children with MIS-C, 84.18% were previously healthy. The most frequent clinical manifestation in our studied population was abdominal pain (N = 804, 64.9%), followed by conjunctival injection (N = 784, 63.3%). The median duration of fever at the time of hospital admission was 5 days and a significant number of subjects required admission to an intensive care unit (N = 589, 47.5%). Most of the subjects (N = 1096, 88.7%) were treated with intravenous immunoglobulin, whereas 76.7% (N = 947) were treated with steroids, of whom 10.6% (N = 100) did not receive intravenous immunoglobulin. The death rate attributed to MIS-C was 4.88%, with a rate of 3.39% for those initially diagnosed with MIS-C and 8.85% for those whose admission diagnosis was not MIS-C (P <0.001, odds ratio 2.76, 95% confidence interval 1.6-4.6). Conclusions:One of the most significant findings from our study was the death rate, especially in those not initially diagnosed with MIS-C, in whom the rate was higher. This highlights the importance of increasing awareness and making an earlier diagnosis of MIS-C in Latin America.

Background/PurposeAdequate transition from pediatric to adult care is associated with better adherence to treatment and better outcomes in pediatric patients with chronic diseases. There are little data on transition programs, outcomes, use of transition guidelines, and available tools in pediatric rheumatology centers from Latin America (LATAM). In this study, we described the characteristics of transition programs from 3 pediatric rheumatology centers. We also introduced results of the first survey examining the transition experience in countries from LATAM.MethodsThe experience and implementation process of transition programs from 3 pediatric rheumatology centers are described. A survey based on a questionnaire created by Chira et al (J Rheumatol. 2014;41:768-779) from the Childhood Arthritis and Rheumatology Research Alliance was also administrated to pediatric rheumatology centers from LATAM.ResultsA total of 49 (68%) pediatric rheumatologists answered the survey. Most centers do not have an official and written transition program and reported a need for more tools and resources in their services to facilitate the transition experience.ConclusionsTransition guidelines culturally tailored to developing countries are needed in LATAM.

Background The term juvenile idiopathic arthritis (JIA) represents a heterogeneous group of disorders, all manifesting joint inflammation, but with different clinical phenotypes, disease course, and outcomes [1,2]. In Argentina, information about patients with JIA is scarce. In our country, different factors such as the diversity of sociodemographic conditions and the disparities in the access to health care, could influence the JIA subtypes prevalence and prognosis of these patients. Objectives Describe the sociodemographic and clinical characteristics of patients with different subtypes of JIA. Methods Descriptive, retrospective, chart review and multicenter study. Patients were included with a diagnosis of JIA, according to ILAR criteria (2001) and onset of symptoms ≤ 16 years, diagnosis within the last 3 years and with at least 12 months of follow-up since diagnosis by the same physician or treating team. Sociodemographic variables, clinical variables prior to diagnosis by the pediatric rheumatologist, time to specialist consultation, treatments prior to diagnosis and variables at the time of diagnosis by the specialist were recorded: JIA subtype; serological, articular, ocular, and systemic manifestations; associated complications and treatments given. Results 320 patients from 17 specialized care centers in Argentina (11 public and 6 private) were included. The mean age at symptom onset was 6.9 years (SD 4.09) and 65.6% of the patients were girls. 94.4% of the patients were in school according to their chronological age. 37.2% and 36.9% of the patients belonged to lower-middle socio-economic strata respectively as measured by the Graffar scale. 96.9% had urban residence and the place of residence in decreasing order were: Province of Buenos Aires, Gran Buenos Aires, CABA, Córdoba, Santa Fe, Jujuy; Salta, Tucumán, Entre Ríos, Catamarca, Chubut, Río Negro, Santiago del Estero, San Juan, Formosa, Chaco, La Pampa, La Rioja, Mendoza, Neuquén and Santa Cruz. 49.1% had private medical insurance and 41.6% public. The mean education (in years) of the parents was 13.6 years (SD 4.16). 54.5% of the cases the first professional visited was the orthopedist. 57.% of patients had received previous treatment: 90.9% non-steroidal anti-inflammatory drugs (NSAIDs) and 5.11% corticosteroids. At diagnosis, JIA subtypes were represented by persistent oligoarthritis (37.8%); polyarthritis FR -(20%); systemic (15%); polyarthritis FR +(12.1%); enthesis-related (6.8%); extended oligoarthritis (2.5%); psoriatic (1.8%) and undifferentiated (0.3%). The median delay by subtype was persistent oligoarthritis (3.00[2.00,4.50]); polyarthritis FR -(3.00[2.00,6.00]); systemic (1.00[100,2.00]); polyarthritis FR +(3. 00 [2.00,6.00]); enthesis-related (7.00 [3.25,12.0]); extended oligoarthritis (3.5 [1.00,4.25]); psoriatic (11.5 [4.75,13.8]) and undifferentiated (1.00 [1.00,1.00]). Articular and serological manifestations were the most frequent. Ocular involvement (12.5%) was more frequent in the oligoarticular subtype. Macrophage activation syndrome was observed in the systemic subtype (2%) and FR - polyarthritis (1.5%). The most used treatments were NSAIDs, cDMARDs, with methotrexate being the first choice. Patients belonging to the systemic (18.7%) and FR+ polyarthritis (5.0%) subtypes were treated with cDMARDs (Anti TNF and IL6 inhibitors). Conclusion This is the first national multicenter study of patients with JIA, and the results provide data that should be prioritized, such as shortening the time to specialist consultation. The comprehensive approach to children with JIA should be coordinated by pediatric rheumatologists and supported by a multi- and interdisciplinary team. Reference [1]Garay S., et.al. Rheumatol Int 2018 Apr;38(Suppl 1):51-58. 2- Ravelli A, et.al. Lancet 2007; 369: 767–78. Acknowledgements: NIL. Disclosure of Interests Daniela Vidal Grant/research support from: Novartis Argentina, Judith Giuponni Grant/research support from: Novartis Argentina, Lorena Guerini Grant/research support from: Novartis Argentina, Romina Cecilia Larroulet Grant/research support from: Novartis Argentina, María Susana Galindo Grant/research support from: Novartis Argentina, Javier Farfan Grant/research support from: Novartis Argentina, María Arispe Grant/research support from: Novartis Argentina, Iris Vilca Grant/research support from: Novartis Argentina, Lorena Franco Grant/research support from: Novartis Argentina, Verónica Ochoa y Gomez Grant/research support from: Novartis Argentina, María Marcantoni Grant/research support from: Novartis Argentina, Vanessa Deves Grant/research support from: Novartis Argentina, Fernando Zunino Pradier Grant/research support from: Novartis Argentina, Maria Elena Rama Grant/research support from: Novartis Argentina, Gabriela Yesuron Grant/research support from: Novartis Argentina, Carolina Torres Walsh Grant/research support from: Novartis Argentina, Ingrid Pintos Grant/research support from: Novartis Argentina, Vanesa Duarte Employee of: Novartis Argentina, Graciela Espada Grant/research support from: Novartis Argentina.

Objetivo. Proporcionar un marco para profesionales de la salud que tratan a pacientes pediatricos bajo terapia con glucocorticoides (GC) y desarrollar recomendaciones para la prevencion y el tratamiento de la osteoporosis inducida por GC en la poblacion pediatrica. Metodos. Un panel de expertos en enfermedades oseas y pediatricas genero una serie de preguntas PICO que abordan aspectos relacionados con la prevencion y el tratamiento de osteoporosis en pacientes bajo tratamiento con GC. Siguiendo la metodologia GRADE, se realizo una revision sistematica de la literatura, se resumieron las estimaciones del efecto y se califico la calidad de la evidencia. Luego se procedio a la votacion y a la formulacion de las recomendaciones. Resultados. Se desarrollaron 7 recomendaciones y 6 principios generales para osteoporosis inducida por GC en poblacion pediatrica. Conclusion. Estas recomendaciones proporcionan orientacion para los medicos que deben tomar decisiones en pacientes pediatricos bajo tratamiento con GC.

Objective. To provide a framework for healthcare professionals managing pediatric patients who are on active glucocorticoid (GC) therapy and to develop recommendations for the prevention and treatment of GC-induced osteoporosis in the pediatric population. Methods. A panel of experts on bone and pediatric diseases developed a series of PICO questions that address issues related to the prevention and treatment of osteoporosis in patients on GC therapy. In accordance with the GRADE approach, we conducted a systematic review of the literature, summarized effect estimations, and classified the quality of the evidence. Then, voting and the formulation of recommendations followed.Results. Seven recommendations and six general principles were developed for GC-induced osteoporosis in the pediatric population. Conclusion. These recommendations provide guidance for clinicians who must make decisions concerning pediatric patients undergoing treatment with GC.

Background Multisystem Inflammatory Syndrome in Children (MIS-C) is one of the most feared complications following SARS-CoV2 infection in children and adolescents. Few multinational multicenter studies from Latin America have been published. Objectives To describe the clinical presentation, management, and outcomes of MIS-C in Latin America. Methods Observational, prospective and retrospective, multicenter study to gather information from 84 participating centers across 16 Latin American countries, between August January 1, 2020 and June 30, 2022. Results Of the 1,239 reported cases of MIS-C, 84.2% were previously healthy. The most frequent clinical manifestation in our studied population was abdominal pain (N=804, 64.9%), followed by conjunctival injection (N=784, 63.3%). The median days of fever at the time of hospital admission was 5 and a significant number of subjects required admission to an intensive care unit (N=589, 47.8%). A total of 538 (47.2%) patients had an abnormal initial echocardiogram. Most of the subjects (N= 1,096, 88.7%) were treated with intravenous immunoglobulin (IVIG), while 76.7% (N= 947) were treated with steroids, of which 10.6% (N= 100) did not receive IVIG. The death rate attributed to MIS-C was 4.88%, with a rate of 3.39% for those initially diagnosed with MIS-C and 8.85% for those whose admission diagnosis was not MIS-C (P= 0.00001). Conclusion One of the most significant findings from our study was the death rate, especially in those not initially diagnosed with MIS-C, in whom it was higher. This highlights the importance of increasing awareness and making an earlier diagnosis of MIS-C in Latin America. REFERENCES: NIL. Acknowledgements: NIL. Disclosure of Interests None Declared.

Considering the advances made on mucopolysaccharidosis type I after the consensus study published by a group of experts in Argentina in 2008, recommendations about genetic testing, cardiological follow-up, airway care, hearing impairment detection, spinal and neurological conditions, as well as current treatments, were reviewed. Emphasis was placed on the need for early diagnosis and treatment, as well as an interdisciplinary follow-up.

Here we describe the current challenges of mucopolysaccharidosis type I: the need for an adequate classification, establishing its relationship to therapeutic indications; an early diagnosis, from neonatal screening, its advantages and barriers, to clinical suspicion of severe and attenuated forms; spinal and eye disease care, from diagnosis to follow-up and treatment; allergic reactions caused by enzyme replacement therapy, their diagnosis and treatment. And lastly, transition to adult care.

Objective. To develop and test shortened versions of the Manual Muscle Test-8 (MMT-8) in juvenile dermatomyositis (JDM). Methods. Construction of reduced tools was based on a retrospective analysis of individual scores of MMT-8 muscle groups in 3 multinational datasets. The 4 and 6 most frequently impaired muscle groups were included in MMT-4 and MMT-6, respectively. Metrologic properties of reduced tools were assessed by evaluating construct validity, internal consistency, discriminant ability, and responsiveness to change. Results. Neck flexors, hip extensors, hip abductors, and shoulder abductors were included in MMT-4, whereas MMT-6 also included elbow flexors and hip flexors. Both shortened tools revealed strong correlations with MMT-8 and other muscle strength measures. Correlations with other JDM outcome measures were in line with predictions. Internal consistency was good (0.88–0.96) for both MMT-4 and MMT-6. Both reduced tools showed strong ability to discriminate between disease activity states, assessed by the caring physician or a parent (P < 0.001), and between patients whose parents were satisfied or not satisfied with illness course (P < 0.001). Responsiveness to change (assessed by both standardized response mean and relative efficiency) of MMT-4 and, to a lesser degree, MMT-6, was slightly superior to that of MMT-8. Conclusion. Overall, the metrologic performance of MMT-4 and MMT-6 was comparable to that of the other established muscle strength tools, which indicates that they may be suitable for use in clinical practice and research, including clinical trials. The measurement properties of these tools should be further tested in other patient populations and evaluated prospectively.

Considering the advances made on mucopolysaccharidosis type I after theconsensus study published by a group of experts in Argentina in 2008, recommendations about genetic testing, cardiological follow-up, airway care, hearing impairment detection, spinal and neurological conditions, as well as current treatments, were reviewed. Emphasis was placed on the need for early diagnosis and treatment, as well as an interdisciplinary follow-up.

Here we describe the current challenges of mucopolysaccharidosis type I: the need for an adequate classification, establishing its relationship to therapeutic indications; an early diagnosis, from neonatal screening, its advantages and barriers, to clinical suspicion of severe and attenuated forms; spinal and eye disease care, from diagnosis to follow-up and treatment; allergic reactions caused by enzyme replacement therapy, their diagnosis and treatment. And lastly, transition to adult care.Se describen como desafíos actuales en mucopolisacaridosis I la necesidad de una clasificación adecuada, vinculándola a las indicaciones terapéuticas; el diagnóstico temprano desde la pesquisa neonatal, sus ventajas y dificultades hasta la sospecha clínica de las formas grave y atenuada; el cuidado de la patología espinal y oftalmológica, desde el diagnóstico, el seguimiento y el tratamiento; las reacciones alérgicas por terapia de reemplazo enzimático, su diagnóstico y tratamiento. Por último, la transición hacia el cuidado adulto.

Objective To evaluate changes in health-related quality of life (HRQoL) and disability in children with systemic juvenile idiopathic arthritis (JIA) or polyarticular JIA treated with tocilizumab. Methods Secondary analyses of two double-blind, placebo-controlled trials of intravenous tocilizumab in children with active systemic JIA or polyarticular JIA were conducted. Patient-reported outcomes of disability (Childhood Health Assessment Questionnaire [C-HAQ]), HRQoL (Child Health Questionnaire Parent Form 50 [CHQ-P50], health concepts, physical summary score [CHQ-P50-PhS], psychosocial summary score [CHQ-P50-PsS]), pain, and well-being (100-mm visual analog scale [VAS]) were measured at weeks 0 and 12 for systemic JIA, weeks 16 and 40 for polyarticular JIA, and week 104 for both JIA subgroups. Results The trial included 112 patients with systemic JIA and 188 patients with polyarticular JIA. In patients with polyarticular JIA, the mean +/- SD C-HAQ score decreased from 1.39 +/- 0.74 at baseline to 0.67 +/- 0.65 at week 16 (P < 0.001). In patients with systemic JIA, the mean +/- SD CHQ-P50-PhS improved more with tocilizumab therapy than with placebo at week 12 (7.3 +/- 10.2 versus 2.4 +/- 10.6) (P < 0.05). Almost all mean CHQ-P50 health concept scores, CHQ-P50-PsS, and CHQ-P50-PhS improved (P <= 0.002) by week 104 for patients with systemic JIA. Patients with polyarticular JIA and patients with systemic JIA showed significant reductions in disability (mean +/- SD C-HAQ scores of -1.09 +/- 0.71 and -1.17 +/- 0.80, respectively), improvements in well-being (mean +/- SD well-being VAS scores of -43.76 +/- 26.61 and -51.53 +/- 23.57, respectively), and decreases in pain (mean +/- SD pain VAS scores of -41.56 +/- 31.06 and -51.26 +/- 26.79, respectively) (P < 0.001); in patients with polyarticular JIA and patients with systemic JIA who were treated with tocilizumab, 92.9% of polyarticular JIA patients and 96.8% of systemic JIA patients reported no more than minimal pain (a score of <= 35 mm on the VAS) at week 104. Conclusion Tocilizumab treatment was associated with significantly reduced disability and pain and improved HRQoL in patients with systemic JIA and polyarticular JIA.