Ilaris NIS in Korea

Adverse events and serious adverse events in hereditary periodic fever syndrome (CAPS, crFMF, TRAPS and HIDS/MKD) and sJIA patients treated with Ilaris.

Defining patients as responders when they are assessed as having achieved complete response at 4 weeks of treatment and reach 16 weeks of treatment without flare for crFMF, TRAPS, HIDS/MKD and sJIA patients or complete response at 8 weeks of treatment and reach 16 weeks of treatment without flare for CAPS patients.

Complete response will be evaluated in patients with data of clinical response as measured by Physician Global Assessment of Disease Activity (PGA) (5-point scale of none, minor, mild, moderate and severe) Complete response is defined as meeting both of the following criteria.

• PGA ≤ minor (PGA < 2) (clinical response)
• CRP ≤10 mg/L (= 1 mg/dL), or reduction by ≥70% from the start of treatment (serological response) Flare is defined as meeting both of the following criteria.
• PGA ≥ mild (PGA ≥ 2) (clinical flare)
• CRP >30 mg/L (= 3 mg/dL) (serological flare)

Participants assessed by physician on Physician's Global Assessment measured on a 5--point scale for disease activity as: 0 = None/absent; 1 = Minor; 2 = Mild; 3 = Moderate; 4 = Severe.

Proportion of patients with a C-reactive protein (CRP) of ≤ 10 mg/L or a ≥ 70% decrease from the start of treatment.

Proportions of patients with serum amyloid A (SAA) normalization defined as SAA ≤ 10 mg/L.

Physician's severity assessment of key disease-specific signs/symptoms uses the following 5- point scale.

0 = none

• = minor
• = mild
• = moderate
• = severe Key signs/symptoms to be assessed differ from disease to disease under study, which are as follows.

CAPS: hearing impairment, visual impairment, renal impairment, and joint dysfunction, skin rash, injection site reaction crFMF: chest pain, abdominal pain, arthralgia/arthritis, skin rash TRAPS: skin rash, musculoskeletal pain, abdominal pain, eye manifestations HIDS (MKD): lymphadenopathy, aphthous ulcers, abdominal pain sJIA: arthritis, generalized lymphadenopathy, rheumatoid rash, hepatosplenomegaly or splenomegaly, serositis

Symptoms that may significantly affect physical functioning and vital prognosis will be evaluated using the following 5-point scale.

0 = none

• = minor
• = mild
• = moderate
• = severe Evaluation of auditory disorder, visual disorder and renal impairment, and status of central nervous system disorder

The CHQ is a generic self-administered instrument designed to capture the physical, emotional, and social components of health status of children. It comprises 15 health concepts (range, 0 to 100) exploring either physical and psychosocial domains. Higher scores in the scales indicate better HRQoL.

The SF-36 health survey is a subjective measure of health-related QoL and consists of 36 questions relating to eight domains: physical functioning, social functioning, physical role functioning, emotional role functioning, vitality, mental health, pain, and general health. Scores of each domain varies from 0 to 100 points, and a higher score represents better QoL

The WPAI-SHP contains six items relating to health problems interfering with work or daily activities over the previous week, the WPASI-SHP yields four scores, absenteeism, presenteeism, work productivity loss, and activity impairment, which can range from 0 (no impact on work/activities) to 100 (total incapacity).