ACR Meeting Abstracts

ACR Meeting Abstracts

  • Home
  • Meetings Archive
    • ACR Convergence 2021
    • ACR Convergence 2020
    • 2020 ACR/ARP PRSYM
    • 2019 ACR/ARP Annual Meeting
    • 2018 ACR/ARHP Annual Meeting
    • 2017 ACR/ARHP Annual Meeting
    • 2017 ACR/ARHP PRSYM
    • 2016-2009 Meetings
    • Download Abstracts
  • Keyword Index
  • Advanced Search
  • Your Favorites
    • Favorites
    • Login
    • View and print all favorites
    • Clear all your favorites
  • Meeting Resource Center

Abstract Number: 2031

Safety and Efficacy of Anakinra in Patients with Deficiency of Interleukin-1 Receptor Antagonist

Gina A. Montealegre1, Adriana Almeida de Jesus2, Dawn C. Chapelle3, Paul Dancey4, Joost Frenkel5, Annet van Royen-Kerkhoff6, Ronit Herzog7, Giovanna Ciocca8, Rafael F. Rivas-Chacon8, Ann M. Reed9, Nicole Plass3, Ivona Aksentijevich10, Polly J. Ferguson11, Suvimol C. Hill12, Edward Cowen13 and Raphaela T. Goldbach-Mansky3, 1NIAMS, Bethesda, MD, 2Translational Autoinflammatory Disease Section, National Institute of Arthritis and Musculoskeletal and Skin Diseases, NIH, Bethesda, MD, 3Translational Autoinflammatory Diseases Section, National Institute of Arthritis and Musculoskeletal and Skin Diseases, NIH, Bethesda, MD, 4Health Science Centre, Memorial University of Newfoundland, St Johns, Canada, 5Wilhelmina Childrens Hospital, University of Utrecht, Utrecht, Netherlands, 6University of Utrecht, Utrecht, Netherlands, 7Cornell University, New York, NY, 8Miami Children's Hospital, Miami, FL, 9Rheumatology, Mayo Clinic, Rochester, MN, 10Inflammatory Disease Section, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, 11Dept of Pediatrics--Rheum, University of Iowa Carver College of Medicine, Iowa City, IA, 12Radiology and Imaging Sciences, NIH Clinical Center, Bethesda, MD, 13Dermatology Consultation Servce, National Cancer Institute, National Institutes of Health, Bethesda, MD

Meeting: 2012 ACR/ARHP Annual Meeting

Keywords: Anakinra, interleukins (IL), osteomyelitis, pediatric rheumatology and treatment

  • Tweet
  • Email
  • Print
Session Information

Session Title: Pediatric Rheumatology - Clinical and Therapeutic Aspects: Juvenile Idiopathic Arthritis and Other Pediatric Rheumatic Diseases

Session Type: Abstract Submissions (ACR)

Background/Purpose:

Deficiency of interleukin-1 receptor antagonist (DIRA) is a neonatal-onset autoinflammatory syndrome caused by mutations in IL1RN gene and clinically characterized by a perinatal onset of pustular dermatosis, aseptic multifocal osteomyelitis and marked elevation of acute phase reactants. Individual reports have been shown that these patients present a prompt response to the recombinant human IL-1 receptor antagonist (IL1Ra) anakinra. However, long-term efficacy and safety of anakinra treatment in DIRA patients have not been assessed. Thus, the objectives of this study were to assess the clinical and laboratory findings of patients with DIRA followed at the NIH and enrolled in a natural history study to assess long-term outcomes in autoinflammatory syndromes patients.

Methods:

Eight patients with a genetically confirmed diagnosis of DIRA were followed longitudinally. Demographic, clinical and laboratory findings and response to treatment variables were collected for each patient at the enrollment and subsequent clinical visits.  Statistical analyses were performed using unpaired t- test with Welch’s correction.

Results:

Four (50%) patients were female. Age at disease onset ranged from 1 to 15 days of life and age at anakinra starting was 11 (2 – 114) months. Five patients had been receiving anakinra 4 to 54 months prior to the study enrollment. Before IL1Ra was started, 5 patients presented with intermittent fever; all 8 patients had mild to severe pustular rashes and 5 had nail abnormalities. Various degrees of multifocal osteomyelitis were observed in all patients and 3 had odontoid non-fusion. Unspecific lung disease was observed in 3 patients, 2 patients presented thrombotic events and 2 had vasculitis. Laboratory findings prior to anakinra showed mild to severe anemia and increased white blood cell (WBC) count in all 8 patients and thrombocytosis in 5. C-reactive protein (CRP) and erythrocyte sedimentation rate (ESR) were elevated in all 8 patients. At the last follow-up visit (6 to 48 month), time on anakinra was 35 (20 – 102) months and anakinra doses ranged from 2.1 to 5.1 (median: 3.5) mg/kg/day. At that time, none of the patients had developed new osteomyelitis lesions, nor pustular skin manifestations and CRP and ESR were 0.21 (0.016 – 0.579) mg/dL and 7 (3 – 13) mm/h, respectively. All patients fulfilled criteria for inflammatory and clinical remission, except for one patient that had CRP of 0.579mg/dL. While acute phase reactants had almost normalized at the time of the first NIH visit, a decrease in the platelets (502 ± 180.0 vs. 273 ± 32.3 x 103cells/mm3, p=0.016) and in the WBC (15.96 ± 6.56 vs. 8.44 ± 1.98 x 103cells/mm3, p=0.023) was seen and took longer to normalize. Three serious adverse events were observed: rash and pneumonia in one patient and subarachnoid bleed in one patient. Anakinra was considered well tolerated and an increase of opportunistic or recurrent infections was not observed within the enrolled patients. 

Conclusion: DIRA patients on treatment with anakinra for 20-102 months presented with a sustained clinical and laboratory response and no patient developed novel inflammatory lesions while on treatment. Anakinra is well tolerated and none of the patients has discontinued therapy.


Disclosure:

G. A. Montealegre,
None;

A. Almeida de Jesus,
None;

D. C. Chapelle,
None;

P. Dancey,
None;

J. Frenkel,
None;

A. van Royen-Kerkhoff,
None;

R. Herzog,
None;

G. Ciocca,
None;

R. F. Rivas-Chacon,
None;

A. M. Reed,
None;

N. Plass,
None;

I. Aksentijevich,
None;

P. J. Ferguson,
None;

S. C. Hill,
None;

E. Cowen,
None;

R. T. Goldbach-Mansky,
None.

  • Tweet
  • Email
  • Print

« Back to 2012 ACR/ARHP Annual Meeting

ACR Meeting Abstracts - https://acrabstracts.org/abstract/safety-and-efficacy-of-anakinra-in-patients-with-deficiency-of-interleukin-1-receptor-antagonist/

Advanced Search

Your Favorites

You can save and print a list of your favorite abstracts during your browser session by clicking the “Favorite” button at the bottom of any abstract. View your favorites »

ACR Pediatric Rheumatology Symposium 2020

© COPYRIGHT 2022 AMERICAN COLLEGE OF RHEUMATOLOGY

Wiley

  • Home
  • Meetings Archive
  • Advanced Search
  • Meeting Resource Center
  • Online Journal
  • Privacy Policy
  • Permissions Policies