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Table 1 Baseline demographics at time of treatment access request for the total population who received ≥ 1 lanadelumab dose

From: Long-term prophylaxis with lanadelumab for HAE: authorization for temporary use in France

Patient characteristic

n = 77

Median (range) age, y

42.4 (11.7–78.9)

Age, y, n (%)

 < 18

6 (7.8)

 18–64

63 (81.8)

 ≥ 65

8 (10.4)

Female, n (%)

53 (68.8)

Weight (kg)

 Mean (SD)

75.8 (20.6)

Age at diagnosis, years

 Median (range)

10.0 (1–48)

HAE type

 1

68 (88.3)

 2

7 (9.1)

 HAE nC1-INHa

1 (1.3)

 Acquired angioedema

1 (1.3)

Positive family history of HAE, n (%)

52 (67.5%)

Proportion of laryngeal attacks for the 3 most severe attacks in the 6 months prior to ATU entry, n (%)

 Number of severe attacks

149

 Proportion of pharyngo-laryngeal attacks

12 (8.1)

Number of HAE attacks in the 6 months prior to ATU entry, median (range) (n = 70)

13.5 (1–99)

Exposure to LTP prior to ATU entry, n (%)b

72 (93.5)

Ongoing LTP prior to ATU entry, n (%)

60 (77.9)

 Single oral agentc

20 (26.0)

 Single C1-INH agentd

23 (29.9)

 Combination of LTP agents

17 (22.1)

Monthly attack rate 6 months prior to ATU entry for patients with ongoing LTP, mean (SD)

n = 52

2.53 (2.67)

Number of attacks in the 6 months prior to ATU entry by ongoing LTP mean (SD)

 No LTP

n = 13

20.0 (8.8)

 Single oral LTPs

n = 18

12.9 (8.7)

 Single C1-INH LTPs

n = 17

17.4 (13.3)

 LTP combinations

n = 17

15.4 (23.4)

  1. ATU authorization for temporary use, C1-INH C1 inhibitor, HAE hereditary angioedema, IV intravenous, LTP long-term prophylaxis, HAE nC1-INH HAE with normal C1-INH levels, pdC1-INH plasma-derived C1-INH, SD standard deviation
  2. aOne patient with HAE with normal C1-INH levels (HAE Type III) was erroneously granted treatment access
  3. bRefers to exposure at any time during the patient’s life
  4. cOral agents included danazol (n = 11), tranexamic acid (n = 3), progestins (n = 5), and rituximab (n = 1 [the nominative ATU patient with acquired angioedema]); 3 patients were receiving C1-INH in combination with danazol
  5. dIV pdC1-INH concentrate, fixed dose (n = 11); IV pdC1-INH concentrate, weight based (n = 4); IV recombinant C1-INH concentrate (n = 8)