Imunodeficiência primária, hereditária, rara, devido a uma anomalia da proteína da cascata do complemento, caracterizada por suscetibilidade significativamente aumentada a infecções por espécies de Neisseria. Afeta apenas homens, geralmente apresentando doença meningocócica grave ou fulminante.
Introdução
O que você precisa saber de cara
A deficiência de properdina é uma imunodeficiência genética rara que prejudica uma parte do sistema de defesa chamada via alternativa do complemento. Ela atinge apenas homens e provoca uma suscetibilidade muito alta a infecções bacterianas graves causadas por bactérias do gênero Neisseria, como meningites fulminantes. A causa está em alterações genéticas no gene CFP, responsável pela produção da proteína properdina. No Brasil, a doença não conta com protocolo clínico (PCDT) próprio nem remédios específicos dispensados pelo SUS, sendo o cuidado focado na prevenção e combate rápido a infecções.
Imunodeficiência primária, hereditária, rara, devido a uma anomalia da proteína da cascata do complemento, caracterizada por suscetibilidade significativamente aumentada a infecções por espécies de Neisseria. Afeta apenas homens, geralmente apresentando doença meningocócica grave ou fulminante.
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Entender a doença
Do básico ao detalhe, leia no seu ritmo
Preparando trilha educativa...
Sinais e sintomas
O que aparece no corpo e com que frequência cada sintoma acontece
Características mais comuns
Os sintomas variam de pessoa para pessoa. Abaixo estão as 3 características clínicas mais associadas, ordenadas por frequência.
Linha do tempo da pesquisa
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Genética e causas
O que está alterado no DNA e como passa nas famílias
Genes associados
1 gene identificado com associação a esta condição.
Curadoria gene-doença
fontes oficiaisProperdin deficiency
Results in higher susceptibility to bacterial infections; especially to meningococcal infections. Three phenotypes have been reported: complete deficiency (type I), incomplete deficiency (type II), and dysfunction of properdin (type III).
Variantes genéticas (ClinVar)
160 variantes patogênicas registradas no ClinVar.
Classificação de variantes (ClinVar)
Distribuição de 13 variantes classificadas pelo ClinVar.
Vias biológicas (Reactome)
6 vias biológicas associadas aos genes desta condição.
Diagnóstico
Os sinais que médicos procuram e os exames que confirmam
Tratamento e manejo
Remédios, cuidados de apoio e o que precisa acompanhar
Onde tratar no SUS
Hospitais de referência no Brasil e o protocolo oficial do SUS (PCDT)
🇧🇷 Atendimento SUS — Deficiência de properdina
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Dados de DATASUS/CNES, SBGM, ABNeuro e Ministério da Saúde. Sempre confirme a disponibilidade diretamente com o estabelecimento.
Pesquisa ativa
Ensaios clínicos abertos e novidades científicas recentes
Pesquisa e ensaios clínicos
Nenhum ensaio clínico registrado para esta condição.
Publicações mais relevantes
Recurrent Gonococcemia Reveiling X-linked Properdin Deficiency: A Novel Case Report.
We present a unique case involving a patient who was diagnosed with X-linked properdin deficiency after 2 episodes of disseminated gonococcal infections 1 year apart. Although this deficiency is well-documented for its association with meningococcemia, its correlation with disseminated gonococcal infections (DGI) has not been previously reported. Recurrent DGI cases reported in the literature with identified cause are mostly associated with acquired or congenital complement pathway deficiencies. However, properdin deficiency is rarely screened for during a first episode. Our case not only highlights the clinical presentation that should raise suspicion of DGI but also underscores the importance of investigating the alternative complement pathway in such cases. At a time when gonococcal resistance is increasing, it is essential to consider existing strategies for preventing these infections, including vaccinations.
Recurrent meningococcal infections as a sign of inborn error immunity.
Invasive meningococcal diseases (IMD) caused by Neisseria meningitidis are generally rare. They affect mostly selected age categories and risk groups of patients (in terms of age, comorbidities, or applied therapy), and the immune system and its defects may play an important modifying role. Meningococcal infections could be the first and only clinical sign of unrecognised immunodeficiency. IMD are a typical clinical presentation of inborn errors of immunity with low concentrations or dysfunction of the terminal components of complement cascade. Meningitis is present in approximately 40% of the patients with terminal complement components deficiencies and in 6% of the patients with properdin deficiency. Despite evident advances in the understanding of the pathogenesis of meningococcal infections and the mechanisms of immune defence against this pathogen, patients with defects in the alternative or terminal complement pathway are highly predisposed to invasive and recurrent meningococcal infections, usually with a mild course. Therefore, it is recommended that each patient with IMD, especially recurrent, should undergo an immunological examination to rule out complement deficiencies.
Severe Meningococcal Meningitis Revealing a Novel Form of Properdin Deficiency in a Previously Healthy 13-Year-old Child.
A 13-year-old boy was admitted with severe meningococcal meningitis. Immunologic workup revealed a properdin deficiency, and genetic sequencing of CFP identified a novel, private and predicted pathogenic variant in exon 8. The patient received broad immunizations and penicillin prophylaxis. Children with invasive meningococcal disease should be tested for complement deficiency.
Properdin deficiency associated with systemic meningococcal disease due to a novel p.Cys337Arg pathogenic variant.
HBSP improves kidney ischemia-reperfusion injury and promotes repair in properdin deficient mice via enhancing phagocytosis of tubular epithelial cells.
Phagocytosis plays vital roles in injury and repair, while its regulation by properdin and innate repair receptor, a heterodimer receptor of erythropoietin receptor (EPOR)/β common receptor (βcR), in renal ischaemia-reperfusion (IR) remains unclear. Properdin, a pattern recognition molecule, facilitates phagocytosis by opsonizing damaged cells. Our previous study showed that the phagocytic function of tubular epithelial cells isolated from properdin knockout (PKO) mouse kidneys was compromised, with upregulated EPOR in IR kidneys that was further raised by PKO at repair phase. Here, helix B surface peptide (HBSP), derived from EPO only recognizing EPOR/βcR, ameliorated IR-induced functional and structural damage in both PKO and wild-type (WT) mice. In particular, HBSP treatment led to less cell apoptosis and F4/80+ macrophage infiltration in the interstitium of PKO IR kidneys compared to the WT control. In addition, the expression of EPOR/βcR was increased by IR in WT kidneys, and furthered increased in IR PKO kidneys, but greatly reduced by HBSP in the IR kidneys of PKO mice. HBSP also increased PCNA expression in IR kidneys of both genotypes. Moreover, iridium-labelled HBSP (HBSP-Ir) was localized mainly in the tubular epithelia after 17-h renal IR in WT mice. HBSP-Ir also anchored to mouse kidney epithelial (TCMK-1) cells treated by H2O2. Both EPOR and EPOR/βcR were significantly increased by H2O2 treatment, while further increased EPOR was showed in cells transfected with small interfering RNA (siRNA) targeting properdin, but a lower level of EPOR was seen in EPOR siRNA and HBSP-treated cells. The number of early apoptotic cells was increased by EPOR siRNA in H2O2-treated TCMK-1, but markedly reversed by HBSP. The phagocytic function of TCMK-1 cells assessed by uptake fluorescence-labelled E.coli was enhanced by HBSP dose-dependently. Our data demonstrate for the first time that HBSP improves the phagocytic function of tubular epithelial cells and kidney repair post IR injury, via upregulated EPOR/βcR triggered by both IR and properdin deficiency.
Publicações recentes
Recurrent Gonococcemia Reveiling X-linked Properdin Deficiency: A Novel Case Report.
Recurrent meningococcal infections as a sign of inborn error immunity.
Properdin deficiency associated with systemic meningococcal disease due to a novel p.Cys337Arg pathogenic variant.
Severe Meningococcal Meningitis Revealing a Novel Form of Properdin Deficiency in a Previously Healthy 13-Year-old Child.
HBSP improves kidney ischemia-reperfusion injury and promotes repair in properdin deficient mice via enhancing phagocytosis of tubular epithelial cells.
📚 EuropePMC44 artigos no totalmostrando 18
Recurrent Gonococcemia Reveiling X-linked Properdin Deficiency: A Novel Case Report.
Open forum infectious diseasesRecurrent meningococcal infections as a sign of inborn error immunity.
Epidemiologie, mikrobiologie, imunologie : casopis Spolecnosti pro epidemiologii a mikrobiologii Ceske lekarske spolecnosti J.E. PurkyneProperdin deficiency associated with systemic meningococcal disease due to a novel p.Cys337Arg pathogenic variant.
Genes & diseasesSevere Meningococcal Meningitis Revealing a Novel Form of Properdin Deficiency in a Previously Healthy 13-Year-old Child.
The Pediatric infectious disease journalHBSP improves kidney ischemia-reperfusion injury and promotes repair in properdin deficient mice via enhancing phagocytosis of tubular epithelial cells.
Frontiers in immunologyProperdin Deficiency Impairs Phagocytosis and Enhances Injury at Kidney Repair Phase Post Ischemia-Reperfusion.
Frontiers in immunologyNeisseria meningitidis inside neutrophils, revealing properdin deficiency.
International journal of infectious diseases : IJID : official publication of the International Society for Infectious Diseases[Inflammatory myopathy following acute meningoccemia in a properdin-deficient patient: A case report].
La Revue de medecine interne[H Factor Deficiency: A Case with an Atypical Presentation].
Acta medica portuguesaInherited Classical and Alternative Pathway Complement Deficiencies in Children: A Single Center Experience.
Iranian journal of immunology : IJIThe role of properdin in complement-mediated renal diseases: a new player in complement-inhibiting therapy?
Pediatric nephrology (Berlin, Germany)Blocking Properdin Prevents Complement-Mediated Hemolytic Uremic Syndrome and Systemic Thrombophilia.
Journal of the American Society of Nephrology : JASN[Assessment of health information available online regarding meningococcal B vaccine recommendations].
Revista espanola de salud publicaFunctional and structural insight into properdin control of complement alternative pathway amplification.
The EMBO journalTumour cell conditioned medium reveals greater M2 skewing of macrophages in the absence of properdin.
Immunity, inflammation and diseaseC5 inhibition prevents renal failure in a mouse model of lethal C3 glomerulopathy.
Kidney internationalProperdin deficiency protects from 5-fluorouracil-induced small intestinal mucositis in a complement activation-independent, interleukin-10-dependent mechanism.
Clinical and experimental immunologyBackground Paper for the update of meningococcal vaccination recommendations in Germany: use of the serogroup B vaccine in persons at increased risk for meningococcal disease.
Bundesgesundheitsblatt, Gesundheitsforschung, GesundheitsschutzAssociações
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Doença com base genética
Um médico geneticista pode ajudar no diagnóstico de Deficiência de properdina e no aconselhamento genético da família.
Doenças relacionadas
Doenças com sintomas parecidos — ajudam quem ainda está buscando diagnóstico
Perguntas frequentes
O que as famílias mais perguntam sobre esta doença — cada resposta com a fonte de onde saiu
A doença é causada por alterações genéticas hereditárias no gene CFP. Esse gene é responsável pela síntese da properdina, proteína essencial para a via alternativa do sistema complemento.
Referências
Fontes citadas no texto, publicações do grafo e bases de dados usadas neste verbete
5 publicações do grafo RarasNet (PubMed) · 7 bases de dados. Títulos, periódicos e PMIDs vêm direto da fonte, sem intermediação de IA.
Dados compilados pelo RarasNet a partir de fontes abertas (Orphanet, OMIM, MONDO, PubMed/EuropePMC, ClinicalTrials.gov, DATASUS, PCDT/MS). Este conteúdo é informativo e não substitui avaliação médica.
Citar este verbete
Raras. (s.d.). Deficiência de properdina. Em Raras — Enciclopédia de Doenças Raras do Brasil. https://raras.org/doenca/deficiencia-de-properdina
Formato APA. Conteúdo sob CC BY 4.0 — reuso livre com atribuição.
Conteúdo mantido por Agente Raras · Médicos e pesquisadores podem colaborar
Deficiência de properdina
📋 Origem dos dados
Esta página agrega dados de fontes públicas e oficiais. Dados sobre cobertura no SUS (PCDT, CEAF) são verificados ativamente por agente proativo (ver badge no infobox). Demais dados têm atribuição de fonte + data da última sincronização — clique para abrir o original.
- Doença rara (ontologia)
- fonte: Orphanet
- Identificador unificado
- fonte: MONDO
- Genética mendeliana
- fonte: OMIM
- Codificação WHO/SUS
- fonte: WHO ICD-10 / DATASUS
- CID-11 (futuro)
- fonte: WHO ICD-11
- NIH/GARD
- fonte: GARD (NIH)
- Indexação biomédica
- fonte: MeSH (NLM)
- Dado público estruturado
- fonte: Wikidata