Table 1 (abstract P160). Baseline characteristics and interim analysis data of patients with HIDS

Number of patients (consecutive patient number #1-8) in disease remission (physician’s assessment)

4 (#1,3,5,6)

4 (#1,2,5,6)

3 (#2,5,7)

Patient assessment of current disease activity (d) and fatigue (f); 0–10, median (min; max)

d: 0 (0; 7)

f: 2.5 (0; 7)

d: 0,0 (0; 8)

f: 1.0 (0; 4)

d: 0,0 (0; 0)

f: 1.0 (0; 2)

Number (%) of patients with days absent from work/school within the last 6 months

2 (25)

0 (0)

1 (25)

CRP / SAA, median (mg/dl)

0.2 / 0.6

0.3 / 0.8

2.1 / 0.5

Mutation(s), disease activity, CRP/SAA value and dosage per patient

Mutation

Disease activity (absent, mild/moderate, severe)

CRP / SAA

(mg/dl)

Dosing

mg (mg/kg)*, per 4 weeks

Patient #1

n.a.

Absent

0.1 / 0.5

300

Patient #2

V203A / I268T

mild / moderate

89.0 / 0.7

150

Patient #3 (MKD)

V377I / E284Kfs*17

mild / moderate

0.0 / 0.3

102 (6.2 mg/kg)

Patient #4

V377I / E93fs*38

mild / moderate

0.4 / 2.6

65 (5.6 mg/kg)

Patient #5

G212R / G212R

Absent

1.1 / -

150 (7.7 mg/kg)

Patient #6

V377I / V377I

absent

0.1 / 0.3

60 (1.6 mg/kg)

Patient #7

V357I

Absent

0.1 / 3.6

100 (4.4 mg/kg)

Patient #8

V377I / I268T

mild / moderate

0.5 / 0.7

150