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Table 1 Patient baseline demographics and disease characteristics

From: A phase II, open-label, extension study of long-term patisiran treatment in patients with hereditary transthyretin-mediated (hATTR) amyloidosis

Characteristic Total population (n = 27)
Age, years, median (range) 64 (29 to 77)
Sex, n
 Male 18 (67%)
 Female 9 (33%)
Genotype
 Val30Met 20 (74%)
 Othera 7 (26%)
FAP stage
 1 24 (89%)
 2 3 (11%)
PND score
 I 15 (56%)
 II 9 (33%)
 IIIa 2 (7%)
 IIIb 1 (4%)
On TTR stabilizer at study entry
 Tafamidis 13 (48%)
 Diflunisal 7 (26%)
 None 7 (26%)
mNIS+7 (max. impairment: 304) 53.0 (2.0 to 122.5)
NIS (max. impairment: 244) 34.8 (4.0 to 93.4)
mBMI, (kg/m2 × g/L) 1030.5 (728.6 to 1379.6)
EQ-5D (max. impairment: 0) 0.8 (0.3 to 1.0)
Cardiac subgroup 11 (41%)
 Val30Met/non-Val30Metb, n 8/3
 NT-proBNP (pg/mL)c 809.8 (105.0 to 2070.0)
 Troponin I (ng/mL)d 0.14 (0.03 to 0.69)
 LV wall thickness (cm) 1.6 (1.3 to 1.9)
  1. Abbreviations: EQ-5D EuroQoL 5-dimensions questionnaire, FAP familial amyloid polyneuropathy, LV left ventricular, mBMI modified body mass index, mNIS+7 modified Neuropathy Impairment Score + 7, NIS Neuropathy Impairment Score, NT-proBNP N-terminal pro-brain natriuretic peptide, PND polyneuropathy disability, QOL quality of life, TTR transthyretin. All data are mean (range) or n (%), unless otherwise stated
  2. aNon-Val30Met mutations: Ser77Tyr (n = 2), Ser77Phe (n = 2), Tyr116Ser (n = 1), Phe64Leu (n = 1), Arg54Thr (n = 1)
  3. bNon-Val30Met mutations in cardiac subgroup: Arg54Thr (n = 1), Ser77Phe (n = 1), Ser77Tyr (n = 1)
  4. cNT-proBNP normal range is: ≤ 97 pg/mL (age 18–45); ≤ 121 pg/mL (age 45–55); ≤ 198 pg/mL (age 55–65); ≤ 285 pg/mL (age 65–75); ≤ 526 pg/mL (age ≥ 75)
  5. dTroponin I normal range is: < 0.03 ng/mL