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- Open Access
Long-term efficacy of IL-1 blockers in PAPA patients
© Finetti et al. 2015
Published: 28 September 2015
PAPA syndrome (Pyogenic Arthritis, Pyoderma gangrenosum, and Acne) is an ultra-rare autosomal dominant, auto-inflammatory disease associated to mutations in the PSTPIP1/CD2BP1 gene. The therapeutic approach during recurrences consists of steroids, while no agreement exists on the chronic management. Evidences on the use of biologics are anecdotal and variable results have been reported.
To evaluate the long-term response to treatment with IL1 antagonist in six patients affected by PAPA syndrome.
Six patients (M:F=3:3; 4 pediatric, 1 young adult and 1 adult, mean age 18 years, range 3-50) affected by PAPA syndrome were enrolled and treated with IL1 blockers (5 patients Anakinra, 1 patient Anakinra followed by Canakinumab). Three patients were already treated with anti-TNFα monoclonal antibodies without benefit. Data were collected retrospectively (mean follow-up 26 months, range 4-38). The frequency of articular and cutaneous flares in the 24 months before starting therapy where compared to those occurred during anti-IL1 regimen. Acute phase reactants (ESR, CRP, SAA) were assessed at the last visit before the study enrolment and at last follow-up.
Manifestations in the 24 months before treatment
N± of flares during follow-up
Treatment (dose, duration)
2 flares of pyogenic arthritis
5 pyoderma gangrenosum
3 flares of pyogenic arthritis
1 sterile osteomyelitis
1 palpebral edema
7 flares of pyogenic arthritis (polyarticular)
3 (mild) articular flares
(38 months) and low dose steroid
Pyogenic muscular abscess
1 persistent muscular abscess
1 cutaneous abscess
3 pyoderma gangrenosum
1 pyoderma gangrenosum (resolved after Canakinumab)
Acne and furunculosis
6 articular flares
The long-term use of IL1 blockers is associated to satisfactory and persistent control of clinical manifestations and laboratory findings in PAPA syndrome.
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