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Table 1 Baseline demographics and disease characteristics (intent-to-treat population)

From: Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study

Parameter 0.01 mg/kg 0.05 mg/kg 0.15 mg/kg 0.3 mg/kg 0.3 mg/kg All patients
Q4W Q4W Q4W Q4W Q3W (n = 29)
(n = 4) (n = 3) (n = 3) (n = 7) (n = 12)
Sex, n (%)       
 Male 3 (75.0) 3 (100.0) 2 (66.7) 3 (42.9) 9 (75.0) 20 (69.0)
 Female 1 (25.0) 0 1 (33.3) 4 (57.1) 3 (25.0) 9 (31.0)
Age, years       
 Mean (SD) 65.8 (8.96) 55.7 (24.83) 41.7 (2.52) 58.7 (16.07) 53.8 (15.6) 55.6 (15.61)
Race, n (%)       
 White/Caucasian 4 (100.0) 3 (100.0) 3 (100.0) 7 (100.0) 12 (100.0) 29 (100.0)
Country       
 Portugal 0 1 (33.3) 3 (100.0) 2 (28.6) 3 (25.0) 9 (31.0)
 Sweden 2 (50.0) 1 (33.3) 0 2 (28.6) 1 (8.33) 6 (20.7)
 France 2 (50.0) 1 (33.3) 0 2 (28.6) 3 (25.0) 8 (27.6)
 Brazil 0 0 0 0 1 (8.33) 1 (3.4)
 Germany 0 0 0 0 1 (8.33) 1 (3.4)
 Spain 0 0 0 0 3 (25.0) 3 (10.3)
 United States 0 0 0 1 (14.3) 0 1 (3.4)
TTR genotype, n (%)       
 Val30Met 2 (50.0) 2 (66.7) 3 (100.0) 6 (85.7) 9 (75.0) 22 (75.9)
 Othera 2 (50.0) 1 (33.3) 0 1 (14.3) 3 (25.0) 7 (24.1)
Mean (SD) serum TTR at baseline, μg/mL 272.9 (98.96) 226.5 (12.67) 276.1 (7.65) 242.6 (38.30) 235.5 (44.45) 245.64 (49.44)
FAP stageb       
 1 25 (86.2)
 2 4 (13.8)
Prior exposure to ALN-TTR01c       
 Yes 2 (50.0) 0 2 (66.7) 3 (42.9) 2 (16.67) 9 (31.0)
 No 2 (50.0) 3 (100.0) 1 (33.3) 4 (57.1) 10 (83.33) 20 (69.0)
Concurrent TTR stabilizer use       
 Diflunisal 2 (50.0) 1 (33.3) 0 3 (42.9) 1 (33.3) 7 (24.1)
 Tafamidis 0 1 (33.3) 2 (66.7) 4 (57.1) 7 (66.7) 14 (48.3)
  1. aNon-Val30Met mutations: Arg45Thr (n = 1), Phe46Leu (n = 1), Ser77Tyr (n = 2), Ser77Phe (n = 2), Tyr116Ser (n = 1)
  2. bFAP stage: 1 = unimpaired ambulation, mostly mild neuropathy in lower limbs; 2 = assistance with ambulation required, mostly moderate neuropathy with progression to lower limbs, upper limbs, and trunk; 3 = wheelchair-bound or bedridden, severe neuropathy of all limbs
  3. cALN-TTR01 was the first-generation siRNA-LNP used in phase I studies in patients with ATTR
  4. ATTR TTR amyloid fibrils, FAP familial amyloid polyneuropathy; Q3W every 3 weeks; Q4W every 4 weeks; SD standard deviation; siRNA-LNP small interfering RNA-lipid nanoparticles; TTR transthyretin