Disease activity accounts for long-term efficacy of IL-1 blockers in pyogenic sterile arthritis pyoderma gangrenosum and severe acne syndrome.

Objective. To provide a rationale for anti-IL-1 treatment in pyogenic sterile arthritis, pyoderma gangrenosum and acne (PAPA) by defining whether IL-1β secretion is enhanced; requires NLRP3; and correlates with proline-serine-threonine phosphatase-interacting protein 1 mutations, disease activity an...

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Publicado en:Rheumatology Vol. 55; no. 7; pp. 1325 - 1336
Autores principales: Omenetti, Alessia, Carta, Sonia, Caorsi, Roberta, Finetti, Martina, Marotto, Daniela, Lattanzi, Bianca, Jorini, Mauro, Delfino, Laura, Penco, Federica, Picco, Paolo, Buoncompagni, Antonella, Martini, Alberto, Rubartelli, Anna, Gattorno, Marco
Formato: pictorial research tables/charts Journal Article
Publicado: Oxford University Press / USA Jul2016
Acceso en línea:Ver este registro en EBSCOhost
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      dt: Jul2016
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      pub: Oxford University Press / USA
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        116280645
        116280645
        10.1093/rheumatology/kew031
        116280645
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        atl: Disease activity accounts for long-term efficacy of IL-1 blockers in pyogenic sterile arthritis pyoderma gangrenosum and severe acne syndrome.
      aug:
        au:
          Omenetti, Alessia
          Carta, Sonia
          Caorsi, Roberta
          Finetti, Martina
          Marotto, Daniela
          Lattanzi, Bianca
          Jorini, Mauro
          Delfino, Laura
          Penco, Federica
          Picco, Paolo
          Buoncompagni, Antonella
          Martini, Alberto
          Rubartelli, Anna
          Gattorno, Marco
        affil: Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal-Infant Sciences (DINOGMI), University of Genoa, Italy
      sug:
        subj:
          Pyoderma Gangrenosum
          Arthritis
          Acne Vulgaris
          Hereditary Autoinflammatory Diseases Drug Therapy
          Interleukin 1 Immunology
          Interleukin 1 Antagonists and Inhibitors
          Mutation
          Human
          Descriptive Statistics
          Enzyme-Linked Immunosorbent Assay
          Sequence Analysis
          Retrospective Design
          Pedigree
          Polymerase Chain Reaction
          Cytokines
          Mann-Whitney U Test
          Kruskal-Wallis Test
          Post Hoc Analysis
          Funding Source
      ab: Objective. To provide a rationale for anti-IL-1 treatment in pyogenic sterile arthritis, pyoderma gangrenosum and acne (PAPA) by defining whether IL-1β secretion is enhanced; requires NLRP3; and correlates with proline-serine-threonine phosphatase-interacting protein 1 mutations, disease activity and/or the clinical picture in PAPA. Methods. Monocytes were isolated from 13 patients and 35 healthy donors and studied at baseline and following activation. Secretion pattern of IL-1β, IL-1α, IL-1Ra, IL-6, IL-18 and TNF-α was assessed in supernatants by ELISA. The NLRP3 requirement for IL-1β secretion was investigated by silencing technique in PAPA and healthy donor monocytes. Long-term follow-up (mean 26 months, range 4-38) was performed in five patients enrolled in an anti-IL-1 regimen. Results. IL-1β secretion in PAPA is increased, requires NLRP3 and correlates with disease activity. Patients with a history of osteoarticular flares release more IL-1β, IL-6 and TNF-α compared with those with predominant cutaneous recurrences. Monocytes from patients in anti-IL-1 treatment dramatically reduced IL-1β secretion after ex vivo activation, and long-term follow-up demonstrated decreased frequency of flares and normalization of acute phase reactants in all the patients. A straightforward correlation between genotype and IL-1β signalling was not observed suggesting that factors other than mutation itself may play a role in regulating IL-1β secretion and response to treatment in PAPA. Conclusion. PAPA patients with active lesions display increased NLRP3-mediated IL-1β secretion, and long-term efficacy of IL-1 blockade was demonstrated. Even if other mechanisms related to the complex proline-serine-threonine phosphatase-interacting protein 1 protein networking might play additional roles, this study further supports the potential of IL-1 blockade as an effective therapeutic strategy in PAPA syndrome.
      pubtype: Academic Journal
      doctype:
        pictorial
        research
        tables/charts
        Journal Article
      ougenre: Article
    language: English
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