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  • Hemophagocytic Lymphohistiocytosis in an Adolescent with NLRP12‐Related Autoinflammatory Disorder - A Case Report
    Publication . Hormigo, I; Valente Pinto, M; Cordeiro, AI; Henriques, C; Martins, C; Parente Freixo, J; Conde, M; Gouveia, C; Farela Neves, J
  • Case Report: Varicella Associated Neuropsychiatric Syndrome (VANS) in Two Pediatric Cases
    Publication . Dahiya, D; Matos, CM; Lim, M; Madureira, I; Duarte, S; Byrne, S; Rossor, T
    Background: Viral or bacterial infections can trigger auto-immune inflammatory reactions and conditions in children. Self-reactivity arises due to similarities in molecular structures between pathogenic microorganisms and regular body structures with consequent immune-cross reactions. Reactivation of latent Varicella Zoster Virus (VZV) infections can cause neurological sequalae, including cerebellitis, post-herpetic neuralgias, meningo/encephalitis, vasculopathy and myelopathy. We propose a syndrome caused by auto-immune reactivity triggered by molecular mimicry between VZV and the brain, culminating in a post-infectious psychiatric syndrome with childhood VZV infections. Case presentation: Two individuals, a 6-year-old male and 10-year-old female developed a neuro-psychiatric syndrome 3-6 weeks following a confirmed VZV infection with intrathecal oligoclonal bands. The 6-year-old male presented with a myasthenic syndrome, behavior deterioration and regression in school, he was poorly responsive to IVIG and risperidone, however had a pronounced response to steroid treatment. The 10-year-old female presented with marked insomnia, agitation, and behavioral regression as well as mild bradykinesia. A trial of neuroleptics and sedatives resulted in a mild unsustained reduction in psychomotor agitation and IVIG was also unsuccessful, however the patient was very responsive to steroid therapy. Conclusion: Psychiatric syndromes with evidence of intrathecal inflammation temporally related to VZV infections that are responsive to immune modulation have not been described before. Here we report two cases demonstrating neuro-psychiatric symptoms following VZV infection, with evidence of persistent CNS inflammation following the resolution of infection, and response to immune modulation.
  • A Novel Variant of DeSanto-Shinawi Syndrome with Joint Manifestations
    Publication . Branco, J; Amorim, M; Conde, M
    variable degrees of developmental delay and intellectual disability that were recently delineated as DeSanto- Shinawi syndrome (OMIM 616708). We describe a patient with DeSanto-Shinawi syndrome caused by a novel frameshift variant in WAC gene (NM_016628.4(WAC):c.1689del (p.Phe563Leufs*6)). As noted in cases previously reported, our patient phenotype included facial dysmorphism, intellectual disability, behavioral problems, feeding difficulties, hirsutism, constipation and astigmatism. She also had limited range of motion of joints since birth and Juvenile Idiopathic Arthritis diagnosed at eleven years old. Although in the last years some additional features were reported in DeSanto-Shinawi syndrome, joint manifestations have not been previously described. As limited range of motion of joints was reported since birth with no correlation with arthritis onset, it could be a new clinical feature. Polyarthritis in this patient can be only a coincidence, since there is a first degree relative with psoriasis, or might be related to WAC mutation. Indeed, WAC encodes a protein that plays a vital role in autophagy. It has already been demonstrated that WAC haploinsufficiency leads to increased autophagy and, according to different authors, increased autophagy may display a pathogenic role in several autoimmune disorders such as Rheumatoid Arthritis and Juvenile Idiopathic Arthritis. Thus, WAC haploinsufficiency may have contributed to autoimmune disorder in this patient.
  • Association of Body Mass Index with Juvenile Idiopathic Arthritis Disease Activity: a Portuguese and Brazilian Collaborative Analysis
    Publication . Neto, A; Mourão, AF; Oliveira-Ramos, F; Campanilho-Marques, R; Estanqueiro, P; Salgado, M; Guedes, M; Piotto, D; Emi Aikawa, N; Melo Gomes, J; Cabral, M; Conde, M; Figueira, R; Santos, MJ; Fonseca, JE; Terreri, MT; Canhão, H
    Objective: To investigate the relationship between body mass index (BMI) and disease activity in patients with Juvenile Idiopathic Arthritis (JIA). Methods: Patients with JIA, aged ≤18 years, registered at the Rheumatic Diseases Portuguese Register (Reuma.pt) in Portugal and Brazil were included. Ageand sex-specific BMI percentiles were calculated based on WHO growth standard charts and categorized into underweight (P<3), normal weight (3≤P≤85), overweight (8597). Disease activity was assessed by Juvenile Arthritis Disease Activity Score (JADAS-27). Uni- and multivariable analyses were performed. Results: A total of 275 patients were included. The prevalence of underweight, normal weight, overweight and obesity was 6.9%, 67.3%, 15.3% and 10.5%, respectively. Underweight patients had significantly higher number of active joints (p<0.001), patient’s/parent’s global assessment of disease activity (PGA) (p=0.020), physician’s global assessment of disease activity (PhGA) (p<0.001), erythrocyte sedimentation rate (ESR) (p=0.032) and overall higher JADAS-27 (p<0.001), compared to patients with normal weight, overweight and obesity. In the multivariable regression, normal weight (B=-9.43, p<0.01), overweight (B=-9.30, p=0.01) and obesity (B=-9.12, p=0.01) were significantly associated with lower disease activity compared to underweight, when adjusted for age, gender, country, ethnicity, JIA category and therapies used. The diagnosis of RF- (B=3.65, p=0.006) or RF+ polyarticular JIA (B=5.29, p=0.024), the absence of DMARD therapy (B=5.54, p<0.001) and the use of oral GC (B=4.98, p=0.002) were also associated with higher JADAS-27. Conclusion: We found an independent association between underweight and higher disease activity in patients with JIA. Further studies are needed to understand the underlying mechanisms of this association.
  • Sarcoidose ou Tuberculose? Dificuldades no Diagnóstico
    Publication . Guerra, AC; Ramos, M; Candeias, F; Brito, MJ
    A sarcoidose é uma doença multissistémica rara, caracterizada por granulomas não caseosos. A clínica e os testes diagnósticos são pouco sensíveis e específicos, dificultando o diagnóstico diferencial, particularmente com a tuberculose. Relata-se o caso clínico de um rapaz de 17 anos com nódulos cutâneos dolorosos, astenia, hipersudorese e uveíte recorrente com dois anos de evolução. Apresentava prova tuberculínica e interferon gamma release assay positivos, anemia e velocidade de sedimentação elevada, hipergamaglobulinemia, lisozima e enzima de conversão de angiotensina elevadas. A tomografia computorizada torácica mostrava opacidades micronodulares centrilobulares, gânglios mediastinicos e hilares calcificados; a broncofibroscopia e o teste de difusão de monóxido de carbono foram normais. A biópsia de nódulo cutâneo revelou paniculite septolobular. A cintigrafia das glândulas lacrimal e salivar sugeriu sarcoidose. Não se podendo excluir tuberculose concomitante, iniciou antibacilares. Dois meses depois, foram associados prednisolona e metotrexato. Seis meses depois verificou-se remissão dos sintomas e normalização dos exames laboratoriais. Este caso mostra as dificuldades no diagnóstico de sarcoidose e tuberculose na ausência dos achados microbiológicos e histológicos típicos e a possibilidade da coexistência destas entidades.
  • 2016 Update of the Portuguese Recommendations for the Use of Biological Therapies in Children and Adolescents with Juvenile Idiopathic Arthritis
    Publication . Santos, MJ; Conde, M; Mourão, AF; Ramos, FO; Cabral, M; Brito, I; Ramos, MP; Marques, RC; Gomes, SM; Guedes, M; Gonçalves, MJ; Estanqueiro, P; Zilhão, C; Rodrigues, M; Henriques, C; Salgado, M; Canhão, H; Fonseca, JE; Gomes, JM
    To provide evidence-based guidance for the rational and safe prescription of biological therapies in children and adolescents with juvenile idiopathic arthritis (JIAs) considering the latest available evidence and the new licensed biologics. Rheumatologists and Pediatricians with expertise in Pediatric Rheumatology updated the recommendations endorsed by the Portuguese Society of Rheumatology and the Portuguese Society of Pediatrics based on published evidence and expert opinion. The level of agreement with final propositions was voted using an online survey. RESULTS: In total, 20 recommendations to guide the use of biological therapy in children and adolescents with JIAs are issued, comprising 4 general principles and 16 specific recommendations. A consensus was achieved regarding the eligibility and response criteria, maintenance of biological therapy, and procedures in case of non-response, for each JIA category. Specific recommendations concerning safety procedures were also updated. These recommendations take into account the specificities of each JIA category and are intended to continuously improve the management of JIA patients.
  • Manifestações Neurológicas nas Imunodeficiências Primárias
    Publication . Oliveira, M; Cordeiro, AI; Conde, M; Lopes Silva, R; Neves, C; Farela Neves, J
    As imunodeficiências primárias são um grupo heterogéneo de doenças individualmente raras. A sua associação a manifestações neurológicas não é rara, sendo os mecanismos fisiopatológicos implicados distintos consoante a patologia em causa. As manifestações neurológicas podem ser a característica predominante da doença ou estar presentes de forma ligeira e/ou inconstante. O correto reconhecimento desta associação pode permitir um diagnóstico atempado destas doenças e o desenvolvimento de estratégias preventivas e terapêuticas. Os autores fazem uma revisão e sistematização das imunodeficiências primárias em que se verifica esta relação, sumariando as manifestações neurológicas e imunes de cada uma das patologias.
  • Recommendations for Vaccination in Adult Patients with Systemic Inflammatory Rheumatic Diseases from the Portuguese Society of Rheumatology
    Publication . Cordeiro, I; Duarte, AC; Ferreira, J; Gonçalves, MJ; Meirinhos, T; Rocha, T; Romão, V; Sousa, S; Guedes, M; Conde, M; Abreu, C; Aleixo, MJ; Santos, MJ
    Serious infections are a major cause of morbidity and mortality in systemic inflammatory rheumatic disease (SIRD) patients. Although vaccination may prevent numerous infections, vaccination uptake rates are low in this group of patients. OBJECTIVES: To develop evidence-based recommendations for vaccination in SIRD patients. METHODS: We searched MEDLINE (until 31 October 2014) and EMBASE (until 14 December 2014) databases, as well as the ACR and EULAR congress abstracts (2011-2014). Patients with any systemic inflammatory rheumatic disease were included and all vaccines were considered. Any safety and efficacy outcomes were admitted. Search results were submitted to title and abstract selection, followed by detailed review of suitable studies. Data were subsequently pooled according to the type of vaccine and the SIRD considered. Results were presented and discussed by a multidisciplinary panel and systematic literature review (SLR)-derived recommendations were voted according to the Delphi method. The level of agreement among rheumatologists was assessed using an online survey. RESULTS: Eight general and seven vaccine-specific recommendations were formulated. Briefly, immunization status should routinely be assessed in all SIRD patients. The National Vaccination Program should be followed and some additional vaccines are recommended. To maximize the efficacy of vaccination, vaccines should preferably be administered 4 weeks before starting immunosuppression or, if possible when disease activity is controlled. Non-live vaccines are safe in SIRD, including immunosuppressed patients. The safety of live attenuated vaccines in immunosuppressed patients deserves further ascertainment, but might be considered in particular situations. DISCUSSION: The present recommendations combine scientific evidence with the multidisciplinary expertise of our taskforce panel and attained desirable agreement among Portuguese rheumatologists. Vaccination recommendations need to be updated on a regular basis, as more scientific data regarding vaccination efficacy and safety, emergent infectious threats, new vaccines as well as new immunomodulatory therapies become available.