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OBJECTIVES: To compare the clinical and laboratory features of pediatric systemic sclerosis sine scleroderma (ssJSSc) with adult-onset ssSSc. METHODS: Demographic, clinical and laboratory data of ssJSSc, retrospectively retrieved from our hospital medical records, case reports from the literature and from the PRES JSSc registry, were compared with the Padua cohort of adult patients with ssSSc. Patients were defined as having ssSSc if they never had skin involvement but all the following features: (I) Raynaud's Phenomenon (RP) and/or digital vasculopathy, (II) positive antinuclear antibodies (ANA), (III) internal organs involvement typical of scleroderma, (IV) no other defined connective tissue diseases. RESULTS: Eighteen juvenile and 38 adult-onset ssSSc patients, mean disease duration 5.8 and 9.7 years, respectively, entered the study. The frequency of females affected was significantly lower in ssJSSc (38.9% vs 89.5%, p < 0.0001). When compared to adults, ssJSSc displayed less SSc-specific capillaroscopy abnormalities (68.8% vs 94.7%, p = 0.02) while significantly higher vascular (digital pitting scars, ulcers 35.3% vs 10.5%, p = 0.042), respiratory (50.0% vs 23.7%, p = 0.02) and cardiac involvement (50.0% vs 2.6%, p < 0.0001). The outcome was significantly worse in ssJSSc as six patients (33%) died (n = 3) or reached an end-stage organ failure (n = 3) in comparison to only two deaths (5.3%) in the adult cohort. Anti-centromere antibodies were significantly lower in children (20.0% vs 68.4%, p = 0.001) while no difference was noted for other SSc-specific autoantibodies. CONCLUSION: Compared to adults where ssSSc generally has an indolent course, children present with aggressive disease that heralds a worse prognosis characterized by high cardiorespiratory morbidity and mortality.Key Indexing Terms: scleroderma, juvenile systemic sclerosis, outcome, heart, pulmonary arterial.
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OBJECTIVE: The main aim of this study was to define the best treatment option for multisystem inflammatory syndrome in children (MIS-C) and to analyse the role of anakinra. METHODS: This is a multicentre retrospective cohort study. Patients were treated according to the attending physician's decision. The patients were divided into four groups on the basis of the first treatment at time of admittance: (i) IVIG, (ii) IVIG and methylprednisolone (≤2 mg/kg/day), (iii) IVIG with high-dose methylprednisolone (>2 mg/kg/day) and (iv) anakinra with or without IVIG and/or methylprednisolone. Primary outcomes were defined as the presence of at least one of the following features: death, the failure of initial treatment, meaning the need for additional treatment for clinical worsening and cardiac involvement at the end of follow-up. RESULTS: Two hundred thirty-nine patients were recruited. At univariate analysis, persistent heart involvement at discharge was more frequent in those not receiving anakinra as initial treatment (3/21 vs 66/189; P = 0.047). After comparisons between the four treatment regimens, adjusting for the propensity score, we observed that early treatment with anakinra was associated with a lower probability of developing persistent heart disease at the end of follow-up (odds ratio: 0.6; 95% CI: 0.4-1.0). CONCLUSION: We report that early treatment with anakinra is safe and very effective in patients with severe MIS-C. In addition, our study suggests that early treatment with anakinra is the most favourable option for patients with a higher risk of developing a severe disease outcome.
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COVID-19/complicações , Imunoglobulinas Intravenosas , Proteína Antagonista do Receptor de Interleucina 1 , Síndrome de Resposta Inflamatória Sistêmica , Criança , Humanos , Proteína Antagonista do Receptor de Interleucina 1/uso terapêutico , Estudos Retrospectivos , Gravidade do Paciente , MetilprednisolonaRESUMO
OBJECTIVE: Juvenile systemic sclerosis (JSSc) is a rare condition in childhood and its variety with no skin involvement, sine scleroderma (ssJSSc), is anecdotal. We report the first case series of patients with ssJSSc. METHODS: Demographic, clinical and laboratory data of patients with JSSc followed at our centre were retrospectively collected. Patients with no skin involvement but with all of the features RP, positive ANA, intestinal dysmotility and/or interstitial lung disease (ILD) or pulmonary arterial hypertension (PAH) and/or cardiac or renal involvement typical of scleroderma were defined as having ssJSSc and compared with those with classic JSSc (cJSSc). RESULTS: Among 52 JSSc patients seen in 20 years, five (9.6%) presented with ssJSSc. Their clinical features and those of the only two patients reported in the literature so far were compared with classic JSSc with available complete data. Six patients had cardiac involvement as presenting feature, three primary cardiomyopathy, three secondary to PAH. Two patients died after a brief disease course and one rapidly underwent heart transplantation. In comparison with cJSSc, ssJSSc showed a significantly longer diagnostic delay (20.1 vs 8.3 months, P = 0.017), higher frequency of cardiac involvement (85.7 vs 15.6%, P = 0.001) and worse outcome, intended as mortality or end-stage organ failure rates (42.9% vs 6.2%, P < 0.001). CONCLUSION: Cardiac involvement represents the most important characteristic of ssJSSc and carries a high morbidity and mortality rate. The longer delay in diagnosis underlines the need for a comprehensive rheumatological work-up in patients with isolated cardiomyopathy or PAH/ILD.
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Doenças Pulmonares Intersticiais , Escleroderma Sistêmico , Criança , Diagnóstico Tardio , Humanos , Doenças Pulmonares Intersticiais/complicações , Estudos Retrospectivos , Esclerodermia Localizada , Escleroderma Sistêmico/complicações , Escleroderma Sistêmico/diagnósticoRESUMO
OBJECTIVE: Cardiac involvement is the most important cause of mortality in juvenile systemic sclerosis (JSSc). Recent reports in adult patients underline that traditional techniques of imaging are inadequate to assess the subclinical cardiac involvement, while speckle tracking echocardiography (STE) is able to identify ventricular dysfunctions in the early stages. The aim of our study was to assess the role of STE in JSSc. METHODS: Demographic, clinical and laboratory data were collected from patients with JSSc. Cardiac investigations performed at baseline (T0) and 18 (T18) and 36 months (T36) follow-up included electrocardiography, conventional echocardiography with measurement of the ejection fraction (EF) and STE with assessment of left and right ventricular global longitudinal strain (LV-GLS and RV-GLS). Cardiac parameters have been compared with demographic characteristics and disease severity, assessed by the Juvenile Systemic Sclerosis Severity Score (J4S). RESULTS: A total of 18 patients, 12 (67%) females, entered the study. At T0, electrocardiography was abnormal in three patients, EF was reduced in one, LV-GLS was abnormal in three (16.7%) and RV-GLS was abnormal in five (27.8%). At T18, EF remained stable while at T36 the result decreased in seven of nine patients. At the same time, LV-GLS also worsened (from -21.6% to -18.2%, P = 0.01). LV-GLS and RV-GLS at baseline showed a significant correlation with J4S (P = 0.012 and P = 0.02, respectively). CONCLUSION: STE is more sensitive than standard echocardiography to identify cardiac involvement in JSSc. Over time, we observed a gradual worsening of LV-GLS, a sign of left ventricular dysfunction, that anticipated by several months the decrease of EF.
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Ecocardiografia/métodos , Escleroderma Sistêmico/complicações , Disfunção Ventricular/etiologia , Adolescente , Criança , Pré-Escolar , Diagnóstico Precoce , Feminino , Humanos , Masculino , Estudos Retrospectivos , Escleroderma Sistêmico/diagnóstico por imagem , Disfunção Ventricular/diagnóstico por imagem , Adulto JovemRESUMO
OBJECTIVES: To investigate safety and efficacy of MMF in patients with severe or MTX-refractory juvenile localized scleroderma. METHODS: Consecutive juvenile localized scleroderma patients undergoing systemic treatment were included in a retrospective longitudinal study. Patients treated with MMF because they were refractory or intolerant to MTX (MMF-group) were compared with responders to MTX (MTX-group). Disease activity was assessed by Localized Scleroderma Cutaneous Assessment Tool and thermography. Disease course was established on the number of relapses and treatment changes. Relapse-free survival was examined by Kaplan-Meier analysis. RESULTS: MMF and MTX groups included 22 and 47 patients, respectively. No significant difference in demographics, follow-up duration and treatment before diagnosis was observed between groups. The most represented clinical subtypes in the MMF-group were pansclerotic morphea and mixed subtype (P = 0.008 and P = 0.029, respectively), and linear scleroderma of the face in the MTX-group (P = 0.048). MMF was started because of MTX resistance (18 patients), relapse during MTX tapering/withdrawal (3 patients) and anaphylaxis to MTX (1 patient). After mean 9.4 years of follow-up, 90.9% of patients on MMF and 100% of those on MTX had inactive disease. No significant difference in relapse-free survival between the groups was found (P = 0.066, log-rank test), although MMF likely induced more persistent remission. MMF was well tolerated and combination of MMF and MTX did not increase its efficacy. CONCLUSION: The present study adds strong evidence on the efficacy and tolerance of MMF in severe and/or MTX-refractory juvenile localized scleroderma. Further controlled studies are needed to prove its efficacy as first line treatment.
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Antirreumáticos/uso terapêutico , Metotrexato/uso terapêutico , Ácido Micofenólico/uso terapêutico , Esclerodermia Localizada/tratamento farmacológico , Criança , Feminino , Humanos , Estimativa de Kaplan-Meier , Estudos Longitudinais , Masculino , Estudos Retrospectivos , Esclerodermia Localizada/diagnóstico , Esclerodermia Localizada/patologia , Termografia , Falha de Tratamento , Resultado do TratamentoRESUMO
Juvenile osteoperiostites (JOP) are a group of inflammatory bone diseases whose differential diagnosis is often difficult. The main conditions are acute osteomyelitis (AOM), chronic non-bacterial osteomyelitis (CNO) and the Goldbloom syndrome (GS). The study was aimed to develop an algorithm to enable an early diagnosis of JOP. Clinical records of patients with AOM, CNO and GS, followed at our Center over the past 10 years, were reviewed. Twelve additional patients with GS were selected from PubMed/MEDLINE literature search. Data collected included demographics, clinical manifestations, laboratory and instrumental investigations at disease onset. The association between categorical variables was investigated, and the segmentation of patients with different diagnoses was analyzed through a classification tree model (CTREE package) in order to build up a diagnostic algorithm. Ninety-two patients (33 CNO, 44 AOM, 15 GS) entered the study. Among 30 variables considered at onset, nine (age at onset, fever, weight loss, symmetry, focality, functional limitation, anemia, elevated ESR, CRP) resulted statistically significant in differentiating the three clinical entities from each other and were chosen to build up a decisional tree. Three variables, symmetry of bone involvement, presence of fever and age at disease onset, resulted significant to discriminate each of the three diseases from the others. The performance of the diagnostic algorithm was validated by comparing the diagnoses provided by the model with the real diagnoses and showed 85.9% accuracy.Conclusion: We propose a diagnostic algorithm, based on simple clinical data, which can help guide a prompt and appropriate diagnosis of JOP. What is Known: ⢠Juvenile osteoperiostitis (JOP) are a group of inflammatory bone diseases followed by various pediatric specialists. ⢠The distinction between these conditions is not easy as clinical and laboratory features often overlap. What is New: ⢠We propose a diagnostic algorithm, based on clinical data of real patients, with high degree accuracy. ⢠This instrument can help guide the prompt and appropriate diagnosis of JOP.
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Osteomielite , Algoritmos , Osso e Ossos , Criança , Diagnóstico Diferencial , Humanos , Osteomielite/diagnóstico , SíndromeRESUMO
OBJECTIVE: Juvenile systemic sclerosis (JSSc) with rapidly progressive course is a life-threatening condition associated with a poor prognosis. Recently, rituximab (RTX) has been shown to be a promising treatment for adult patients with SSc. We present a series of four patients with rapidly progressive JSSc successfully treated with RTX. METHODS: Clinical, laboratory and functional parameters were collected from four patients with rapidly progressive JSSc treated with RTX for at least 1 year. All patients underwent four yearly courses of i.v. RTX 375 mg/m2 on day 0 and 14, at 3-month intervals. Low dose oral prednisone and MMF were also administered. Data were recorded at baseline and every 6 months and included pulmonary and myocardial function parameters, muscular, vascular and skin changes. The Juvenile Systemic Sclerosis Severity Score (J4S) estimated the overall disease severity over time. RESULTS: Four patients (three males, one female), aged 8-17 years, entered the study. Three patients presented with prevalent cardiac involvement, one with severe pulmonary involvement. After 1 year of RTX treatment, all patients showed significant improvement of J4S, Raynaud's phenomenon and cutaneous involvement. Among those with prevalent cardiac involvement, two showed an improvement of the myocardial function (left ventricular ejection fraction [EF] +37% and +19%, respectively) and in the third arrhythmias disappeared. The patient with severe pulmonary involvement showed a significant improvement of the respiratory function (forced vital capacity +46%, forced expiratory volume in 1 s +33%, diffusing capacity of the lung for carbon monoxide [DLCO] +30%). No major side effects were reported. CONCLUSIONS: Our data suggest that a combination of RTX and MMF is effective in arresting the rapid progression of JSSc.
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Antirreumáticos/uso terapêutico , Rituximab/uso terapêutico , Escleroderma Sistêmico/tratamento farmacológico , Adolescente , Criança , Feminino , Humanos , MasculinoRESUMO
Pansclerotic morphoea (PM) is a subtype of juvenile localised scleroderma characterised by severe course with generalised full-thickness skin involvement and possible growth and functional impairment. PM treatment comprises a combination of immunosuppressive agents such as corticosteroids, methotrexate, mycophenolate mofetil, PUVA and antithymocyte globulin and biological agents used in off-label. A possible role of IL-6 in the regulation of firoblast differentiation and stimulation of collagen synthesis has been suggested and in patients with systemic sclerosis (SSc) the treatment with tocilizumab (TCZ) was associated to improvement of skin thickness and joint motion. We describe the first two cases of children with PM refractory to different immunosuppressive agents in which the use of TCZ reduced disease activity and stopped disease progression. Therefore, we suggest that an earlier use of this agent in such severe cases could be considered before irreversible sclerosis and tissue damage occurs.Juvenile localised scleroderma (JLS) comprises a group of autoimmune fibrosing conditions involving skin and subcutaneous tissues following an initial inflammatory reaction. Pansclerotic morphoea (PM), an extremely rare and severe subtype of JLS, is characterised by generalised full-thickness skin involvement that may extend over deeper tissues and bone with subsequent growth disturbance and disabling outcome. We describe the first two children with PM refractory to immunosuppressive treatments in which the off-label use of tocilizumab (TCZ), fully humanised anti IL-6R antibody, allowed to control the inflammation and stopped the extension of the disease.
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Anticorpos Monoclonais Humanizados/uso terapêutico , Esclerodermia Localizada/tratamento farmacológico , Adolescente , Pré-Escolar , Feminino , Humanos , Imunossupressores/uso terapêutico , Masculino , Receptores de Interleucina-6/antagonistas & inibidoresRESUMO
BACKGROUND AND OBJECTIVES: mRNA vaccines elicit a durable humoral response to SARS-CoV-2 in adults, whereas evidence in children is scarce. This study aimed to assess the early and long-term immune response to the mRNA vaccine in children with or without previous SARS-CoV-2 infection. METHODS: In a multicentre prospective observational study, we profiled the immune response to the Pfizer BioNTech (BNT162b2) vaccine in 5-11-year-old children attending the University Pediatric Hospital of Padua and Bambino-Gesù Hospital in Rome (Italy) from December-2021 to February-2023. Blood samples were collected pre-, 1-, and 6-months after vaccination. Neutralizing antibodies (NAbs) and anti-spike-receptor-binding-domain (anti-S-RBD) IgG titers were analyzed through Plaque Reduction Neutralization Test (PRNT) and chemiluminescent immune-enzymatic assay (CLIA), respectively. Immune cell phenotypes were analyzed by flow cytometry. RESULTS: Sixty children (26 [43 %] female, median age = 8 years [IQR = 7-10.7]) were enrolled in the study, including 46 children with a laboratory-confirmed previous COVID-19 (SARS-CoV-2-recovered) and 14 SARS-CoV-2-naïve participants defined as the absence of antigen-specific antibodies before vaccination. SARS-CoV-2-recovered participants recorded higher anti-S-RBD IgG and Wild-type and Omicron BA.2 NAbs titers than SARS-CoV-2-naïve participants at both 1- and 6-months after vaccination. Antibody titers correlated with T (Tregs) and B (Bregs) regulatory cell frequencies in SARS-CoV-2-recovered children. Both SARS-CoV-2-recovered and SARS-CoV-2-naïve participants decreased antibody titers by approximately 100 to 250 % from 1 to 6 months. While children with immunocompromising underlying conditions developed immune responses comparable to those of healthy children, solid organ transplant recipients exhibited lower levels of NAbs and anti-S-RBD IgG titers, as well as reduced frequencies of Tregs and Bregs. CONCLUSIONS: mRNA vaccination triggered a higher production of specific anti-SARS-CoV-2 antibodies along with increased levels of regulatory cells in children with previous SARS-CoV-2 infection up to the following 6 months. These findings provide insights into boosting pre-existing immunity.
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COVID-19 , SARS-CoV-2 , Adulto , Humanos , Feminino , Criança , Pré-Escolar , Masculino , COVID-19/prevenção & controle , Vacinas de mRNA , Vacina BNT162 , Anticorpos Neutralizantes , Anticorpos Antivirais , Hospitais Pediátricos , Imunidade , Imunoglobulina G , VacinaçãoAssuntos
Acne Vulgar/diagnóstico , Acne Vulgar/fisiopatologia , Antibacterianos , Artrite Infecciosa/diagnóstico , Artrite Infecciosa/fisiopatologia , Resistência a Medicamentos , Erisipela/tratamento farmacológico , Osteonecrose/tratamento farmacológico , Pioderma Gangrenoso/diagnóstico , Pioderma Gangrenoso/fisiopatologia , Pré-Escolar , Humanos , Masculino , Síndrome , Resultado do TratamentoRESUMO
OBJECTIVE: To evaluate the efficacy of levofolinic acid (LVF) administered 48 h before methotrexate (MTX) in reducing gastrointestinal side effects without interference with drug efficacy. METHODS: A prospective observational study was performed including patients with Juvenile Idiopathic Arthritis (JIA) reporting significant gastrointestinal discomfort after MTX despite taking a dose of LVF 48 h after MTX. Patients with anticipatory symptoms were excluded. A LVF supplemental dose was added 48 h before MTX and patients were followed every 3-4 months. At each visit data on gastrointestinal symptoms, disease activity (JADAS, ESR, CRP values) and treatment changes were collected. Friedman test for repeated measures analyzed differences between these variables over time. RESULTS: Twenty-one patients were recruited and followed for at least 12 months. All patients received MTX subcutaneously (mean 9.54 mg/m2) and LVF 48 h before and after MTX (mean 6.5 mg/dose), 7 received a biological agent too. Complete remission of gastrointestinal side effects was reported in 61.9% of study patients at first visit (T1) and increased over time (85.7%, 95.2%, 85.7% and 100% at T2, T3, T4, T5, respectively). MTX efficacy was maintained as showed by significant reduction of JADAS and CRP (p = 0.006 and 0.008) from T1 to T4 and it was withdrawn for remission in 7/21. CONCLUSIONS: LVF given 48 h before MTX significantly reduced gastrointestinal side effects and did not reduce drug's efficacy. Our results suggest that this strategy may improve compliance and quality of life in patients with JIA and other rheumatic diseases treated with MTX.
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Antirreumáticos , Artrite Juvenil , Metotrexato , Humanos , Antirreumáticos/efeitos adversos , Artrite Juvenil/tratamento farmacológico , Metotrexato/efeitos adversos , Qualidade de Vida , Resultado do TratamentoRESUMO
Background: Due to the growing evidence of the efficacy of intravenous (IV) cefazolin with an early switch to oral cefalexin in uncomplicated pediatric osteomyelitis (OM) and septic arthritis (SA) in children, we changed our guidelines for empiric antibiotic therapy in these conditions. This study aims at evaluating the impact of the guidelines' implementation in reducing broad-spectrum antibiotic prescriptions, duration of IV antibiotic treatment and hospital stay, treatment failure and recurrence. Materials and methods: This is a retrospective, observational, quasi-experimental study. The four years pre-intervention were compared to the six years, ten months post-intervention (January 2012, through December 2015; January 2016, through October 31st, 2022). All patients aged 3 months to 18 years with OM or SA were evaluated for inclusion. Each population was divided into three groups: pre-intervention, post-intervention not following the guidelines, and post-intervention following the guidelines. Differences in antibiotic prescriptions such as Days of Therapy (DOT), activity spectrum and Length of Therapy (LOT), length of hospital stay (LOS), broad-spectrum antibiotics duration (bsDOT), treatment failure and relapse at six months were analyzed as outcomes. Results: Of 87 included patients, 48 were diagnosed with OM (8 pre-intervention, 9 post-intervention not following the guidelines and 31 post-intervention following the guidelines) and 39 with SA (9 pre-intervention, 12 post-intervention not following the guidelines and 18 post-intervention following the guidelines). In OM patients, IV DOT, DOT/LOT ratio, and bsDOT were significantly lower in the guidelines group, with also the lowest proportion of patients discharged on IV treatment. Notably, significantly fewer cases required surgery in the post-intervention groups. Considering SA, LOS, IV DOT, DOT/LOT ratio, and bsDOT were significantly lower in the guidelines group. The treatment failure rate was comparable among all groups for both OM and SA. There were no relapse cases. The overall adherence was between 72 and 100%. Conclusions: The implementation of guidelines was effective in decreasing the extensive use of broad-spectrum antibiotics and combination therapy for both OM and SA. Our results show the applicability, safety, and efficacy of a narrow-spectrum IV empirical antibiotic regimen with cefazolin, followed by oral monotherapy with first/second-generation cephalosporins, which was non-inferior to broad-spectrum regimens.
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Kidney involvement has been poorly investigated in SARS-CoV-2 Multisystem Inflammatory Syndrome in Children (MIS-C). To analyze the spectrum of renal involvement in MIS-C, we performed a single-center retrospective observational study including all MIS-C patients diagnosed at our Pediatric Department between April 2020 and May 2022. Demographic, clinical, pediatric intensive care unit (PICU) admission's need and laboratory data were collected at onset and after 6 months. Among 55 MIS-C patients enrolled in the study, kidney involvement was present in 20 (36.4%): 13 with acute kidney injury (AKI) and 7 with isolated tubular dysfunction (TD). In eight patients, concomitant AKI and TD was present (AKI-TD). AKI patients needed higher levels of intensive care (PICU: 61.5%, p < 0.001; inotropes: 46.2%, p = 0.002; second-line immuno-therapy: 53.8%, p < 0.001) and showed lower levels of HCO3- (p = 0.012), higher inflammatory markers [neutrophils (p = 0.092), PCT (p = 0.04), IL-6 (p = 0.007)] as compared to no-AKI. TD markers showed that isolated TD presented higher levels of HCO3- and lower inflammatory markers than AKI-TD. Our results indicate a combination of both pre-renal and inflammatory damage in the pathogenesis of kidney injury in MIS-C syndrome. We highlight, for the first time, the presence of tubular involvement in MIS-C, providing new insights in the evaluation of kidney involvement and its management in this condition.
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BACKGROUND: Currently, monoarticular Juvenile Idiopathic Arthritis (monoJIA) is included in the ILAR classification as oligoarticular subtype although various aspects, from clinical practice, suggest it as a separate entity. OBJECTIVES: To describe the clinical characteristics of persistent monoJIA. METHODS: Patients with oligoJIA and with at least two years follow-up entered the study. Those with monoarticular onset and persistent monoarticular course were compared with those with oligoJIA. Variables considered were: sex, age at onset, presence of benign joint hypermobility (BJH), ANA, uveitis, therapy and outcome. Patients who had not undergone clinical follow-up for more than 12 months were contacted by structured telephone interview. RESULTS: Of 347 patients with oligoJIA, 196 with monoarticular onset entered the study and 118 (60.2%), identified as persistent monoJIA, were compared with 229 oligoJIA. The mean follow-up was 11.4 years. The switch from monoarticular onset to oligoarticular course of 78 patients (38.8%) occurred by the first three years from onset. In comparison with oligoJIA, the most significant features of monoJIA were later age at onset (6.1 vs. 4.7 years), lower female prevalence (70.3 vs. 83.4%), higher frequency of BJH (61.9 vs. 46.3%), lower frequency of uveitis (14.4 vs. 34.1%) and ANA+ (68.6 vs. 89.5%) and better long-term outcome. CONCLUSIONS: MonoJIA, defined as persistent arthritis of unknown origin of a single joint for at least three years, seems to be a separate clinical entity from oligoJIA. This evidence may be taken into consideration for its possible inclusion into the new classification criteria for JIA and open new therapeutic perspectives.
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Artrite Juvenil , Uveíte , Humanos , Feminino , Artrite Juvenil/diagnóstico , Artrite Juvenil/epidemiologia , Uveíte/diagnóstico , Uveíte/epidemiologia , Uveíte/etiologia , Estudos RetrospectivosRESUMO
Background: Euthyroid sick syndrome (ESS) is characterized by low serum levels of free triiodothyronine (fT3) with normal or low levels of thyroid stimulating hormone (TSH) and free thyroxine (fT4) and is reported in different acute clinical situations, such as sepsis, diabetic ketoacidosis and after cardiac surgery. Our aim was to evaluate the predicting role of ESS for disease severity in patients with Multisystem Inflammatory Syndrome in children (MIS-C). Methods: A single-centre observational study on consecutive patients with MIS-C. Before treatment clinical, and laboratory data were collected and, in a subset of patients, thyroid function tests were repeated 4 weeks later. Variables distribution was analyzed by Mann-Whitney U-test and correlations between different parameters were calculated by Spearman's Rho coefficient. Results: Forty-two patients were included and 36 (85.7%) presented ESS. fT3 values were significantly lower in patients requiring intensive care, a strong direct correlation was shown between fT3 and Hb, platelet count and ejection fraction values. A significant inverse correlation was retrieved between fT3 levels and C-reactive protein, brain natriuretic peptide, IL-2 soluble receptor and S-100 protein. Subjects with severe myocardial depression (EF < 45%) had lower fT3 values than subjects with higher EF. The thyroid function tests spontaneously normalized in all subjects who repeated measurement 4 weeks after admission. Conclusion: ESS is a frequent and transient condition in acute phase of MIS-C. A severe reduction of fT3 must be considered as important prognostic factor for severe disease course, with subsequent relevant clinical impact in the management of these patients.
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Síndrome de Vazamento Capilar/etiologia , Dermatomiosite/complicações , Adolescente , Síndrome de Vazamento Capilar/diagnóstico , Síndrome de Vazamento Capilar/tratamento farmacológico , Criança , Pré-Escolar , Dermatomiosite/diagnóstico , Dermatomiosite/tratamento farmacológico , Edema/etiologia , Glucocorticoides/uso terapêutico , Humanos , Imunoglobulinas Intravenosas/uso terapêutico , MasculinoRESUMO
BACKGROUND: Limited joint mobility (LJM), previously known as cheiroarthropathy, refers to the presence of reduced extension at the finger joints in people with diabetes and may be associated with scleroderma-like syndromes such as diabetic sclerodactyly. While scleroderma-like syndromes and LJM have been observed in patients with long-term diabetes and associated complications, the coexistence of diabetes with Juvenile systemic sclerosis (jSSc) is rarely described. CASE PRESENTATION: We describe the case of a 14-year-old boy with long-lasting type 1 diabetes (T1D) and suspected LJM associated with Raynaud phenomenon, sclerodactyly and tapering of the fingertips. A comprehensive work-up showed positive autoantibodies (ANA, anti-Ro-52, anti-Mi-2b), abnormal nailfold capillaroscopy with a scleroderma pattern, interstitial lung disease and cardiac involvement. The overall clinical picture was consistent with the diagnosis of jSSc. CONCLUSIONS: LJM can be the initial sign of underlying systemic sclerosis. Nailfold capillaroscopy may help differentiate jSSc from classical LJM in pediatric patients with T1D and finger contractures or skin induration of no clear origin. This case report provides a starting point for a novel hypothesis regarding the pathogenesis of jSSc. The association between T1D and jSSc may be more than a coincidence and could suggest a relationship between glucose metabolism, fibrosis and microangiopathy.
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Diabetes Mellitus Tipo 1 , Escleroderma Sistêmico , Adolescente , Criança , Diabetes Mellitus Tipo 1/complicações , Humanos , Masculino , Angioscopia Microscópica , Esclerodermia Localizada , Escleroderma Sistêmico/complicações , Escleroderma Sistêmico/diagnósticoRESUMO
Multisystem inflammatory syndrome in children (MIS-C) is a serious condition triggered by SARS-COV-2 infection, characterized by persistent fever, multiorgan dysfunction, and increased inflammatory markers. It requires hospitalization and prompt treatment, with nearly 60% of the cases needing intensive care and 2% fatality rate. A wide spectrum of clinical characteristics and therapeutic approaches has been reported in MIS-C. We describe a series of four patients with MIS-C, defined according to the current case definitions, with a self-limiting course and no need for immunomodulatory treatment ("self-limiting MIS-C"). Few data about self-limiting MIS-C are available to date and no information on medium- and long-term outcome of this subset of patients has been reported. Although limited in size, our experience provides new insights into the MIS-C syndrome, highlighting an underestimated aspect of the disease that may have significant therapeutic implications.
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Background: Eligibility criteria for blood donation require hemoglobin levels of ≥12.5 g/dL for women and ≥13.5 g/dL for men, and a platelet count of ≥180 × 109/L. Screening methods before donation should ensure high accuracy, precision, and ease in operation. We assessed the performance, precision, and repeatability of the Horiba Micros ES 60 (Horiba) compared to the Beckman Coulter DXH 800. Methods: Performance was compared by testing samples for each of the 11 devices across 6 sites in the Transfusion Service of Friuli Venezia Giulia Region, Italy. We measured precision by calculating the coefficient of variation (CV), concordance with ρ-Pearson's correlation coefficient, and accuracy with F-tests. The intra-assay agreement was examined in the 11 devices, and repeatability was performed by using CV and the Kruskal−Wallis test. Results: The precision of Horiba was acceptable. Overall, ρ-Pearson's coefficients indicated a strong correlation and positive relationship between all variables. The Bland−Altman plots showed that most of the differences lay within the limits of agreement. All CV were below the reference threshold for all the parameters. Finally, the Kruskal−Wallis test reported non-significant statistical differences for all parameters, except platelet count (p < 0.000). Conclusions: Horiba is adequate for routine pre-donation screening. The intra-assay agreement further demonstrates the accuracy of the device.
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OBJECTIVE: To study disease course and long-term outcome in children with linear scleroderma (SSc) treated with methotrexate (MTX) since diagnosis. METHODS: The present study was retrospective and cross-sectional and included consecutive children with linear SSc who were treated with MTX for >1 year and were followed up for at least 2 years. Disease course was analyzed by the number of relapses and treatment changes. Relapse-free survival was examined by Kaplan-Meier analysis, comparing patients with linear SSc and those with other juvenile localized scleroderma (JLS) disease subtypes. Disease activity and damage were assessed by the Localized Scleroderma Cutaneous Assessment Tool and thermography. RESULTS: Fifty patients with a mean follow-up duration of 7.8 years and a mean MTX treatment duration of 3.1 years were included. Sixteen percent of patients did not respond to the first course of MTX, and 16% had at least 1 flare. Complete remission was observed in 18.2% of patients who were followed up for 2-5 years, in 80.0% of patients followed up for 10 years, and in 87.5% of patients followed up for >10 years. No significant difference in relapse-free survival between patients with linear SSc and in 17 patients with other JLS disease subtypes was observed. Tissue damage was mild in 42% of patients, moderate in 32%, and severe in 26%. The correlations between severity of tissue damage and linear SSc subtype, disease duration, relapses, and remission were not significant. The relationships between treatment duration and disease relapses (P < 0.05) and severity of tissue damage (P < 0.005) were significant. CONCLUSION: Most patients with linear SSc who are treated with MTX achieve complete and long-lasting remission. Overall aesthetic and functional sequelae are moderate, most likely because tissue damage is established early and treatment likely stabilizes the damage. Early diagnosis and MTX treatment, as well as long-term monitoring, are crucial to improve outcome and promptly identify flares.