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FRI0489 Canakinumab Improves Patient Reported Outcomes in Patients with Periodic Fever Syndromes
  1. H. Lachmann1,
  2. A. Simon2,
  3. J. Anton3,
  4. M. Gattorno4,
  5. I. Kone-Paut5,
  6. S. Ozen6,
  7. J. Frenkel7,
  8. E. Ben-Chetrit8,
  9. H. Hoffman9,
  10. A. Zeft10,
  11. Y. Joubert11,
  12. K. Lheritier11,
  13. A. Speziale11,
  14. G. Junge11,
  15. J. Gregson11,
  16. F. De Benedetti12
  1. 1UK National Amyloidosis Centre, University College London Medical School, London, United Kingdom
  2. 2Radboud University Medical Centre, Nijmegen, Netherlands
  3. 3Hospital Sant Joan de Déu, Barcelona, Spain
  4. 4Pediatric Rheumatology, G. Gaslini Institute, Genoa, Italy
  5. 5Hôpital Kremlin Bicetre, University of Paris SUD, Paris, France
  6. 6Hacettepe University Children's Hospital, Ankara, Turkey
  7. 7University Medical Center Utrecht, Utrecht, Netherlands
  8. 8Rheumatology Unit, Hadassah–Hebrew University Medical Center, Jerusalem, Israel
  9. 9University of California, La Jolla
  10. 10Pediatrics Rheumatology, Cleveland Clinic, Cleveland, United States
  11. 11Novartis Pharma AG, Basel, Switzerland
  12. 12IRCCS Ospedale Pediatrico Bambino Gesú, Rome, Italy

Abstract

Background Periodic Fever Syndromes (PFS) are rare autoinflammatory conditions including Familial Mediterranean Fever (FMF), Hyper-IgD Syndrome/ Mevalonate Kinase Deficiency (HIDS/MKD), and TNF-Receptor Associated Periodic Syndrome (TRAPS).1 It has been shown that colchicine-resistant FMF (crFMF), HIDS/MKD and TRAPS considerably impact physical and emotional aspects of patients' lives.2–4 Open label studies suggested that canakinumab (CAN), a fully human and highly specific anti-IL-1β monoclonal antibody, is efficacious in crFMF, HIDS/MKD and TRAPS.5–7 To date, there is no data showing the effect of CAN on Health-Related Quality of Life (HRQoL) in PFS patients.

Objectives To evaluate the effect of CAN on HRQoL using Child Health Questionnaire – Parent Form 50 (CHQ-PF50) and SF-12 Health Survey (SF-12) in PFS patients.

Methods In a Phase 3 randomised placebo controlled study of CAN in PFS (NCT02059291), SF-12 Physical Component Summary (PCS) and Mental Component Summary (MCS) were assessed in adults. For children (>5–<18 years), CHQ-PF50 Physical (PhS) and Psychosocial (PsS) Summary scores were assessed.

Results 181 patients were randomised to CAN or placebo in 3 cohorts (63 crFMF, 72 MKD/HIDS, 46 TRAPS). 71 adults ≥18 years and 110 children (age range ≥2–<18 years). Patients reported early clinically meaningful improvement in SF-12 PCS scores reported at Week (Wk) 5 which were sustained and increased to a large effect size by Wk 16 for all indications (Table). Similarly, clinically meaningful improvements in SF-12 MCS, CHQ-PF50 PhS and PsS was observed in all indications, with the exception of PsS in HIDS/MKD and TRAPS patients (Table).

Table 1.

Patient reported outcomes

Conclusions Canakinumab showed rapid improvement by Week 5 in patient reported outcomes in adults and children with PFS, which was sustained through Week 16.

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  2. Dandekar P, et al. Pediatr. Rheumatol. 13(S1):P22

  3. Dandekar P, et al. Pediatr. Rheumatol. 13(S1):P23

  4. Dandekar P, et al. Pediatr. Rheumatol. 13(S1):P24

  5. Brik R, et al. Arthritis Rheumatol. 2014;66(11):3241–3

  6. Arostegui, J.I, et al. Arthritis Rheumatol. 2015; 67 (S10)

  7. Gattorno M, et al. Arthritis Rheumatol. 2015; 67 (S10)

  8. User's manual for the SF-12v2 Health Survey, 3rd ed. 2012

  9. Cohen J, et. al. Statistical Power Analysis for the Behavioural Sciences,2nd ed. 1988

Disclosure of Interest H. Lachmann Consultant for: Novartis, SOBI, Takeda, GSK, Speakers bureau: Novartis, SOBI, A. Simon Grant/research support from: CSL Behring, Novartis, Xoma/Servier, J. Anton Grant/research support from: Novartis, Pfizer, Abbvie, Roche, SOBI, Consultant for: Novartis, M. Gattorno Grant/research support from: Novartis, SOBI, Consultant for: Novartis, SOBI, Speakers bureau: Novartis, SOBI, I. Kone-Paut Grant/research support from: SOBI, Roche, Novartis, Consultant for: Novartis, SOBI, Pfizer, Abbvie, Chugai, S. Ozen Consultant for: Novartis, Speakers bureau: SOBI, J. Frenkel Grant/research support from: Novartis, SOBI, E. Ben-Chetrit Consultant for: Novartis, H. Hoffman Grant/research support from: Bristol Myers Squibb, Consultant for: Novartis, Sob Biovitrum, Regeneron, Speakers bureau: Novartis, A. Zeft: None declared, Y. Joubert Employee of: Novartis, K. Lheritier Employee of: Novartis, A. Speziale: None declared, G. Junge Employee of: Novartis, J. Gregson Employee of: Novartis, F. De Benedetti Grant/research support from: Pfizer, Abbvie, Roche, Novartis, Novimmune, BMS

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