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Table 2 Serum TTR knockdown by dose group

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

Dose group (mg/kg)

Dose 1

Dose 2

Maximum TTR KD (%)

TTR KD at Nadir (Mean % ± SD)

Maximum TTR KD (%)

TTR KD at Nadir (Mean % ± SD)

0.01 Q4W (n = 4a)

37.8

22.1 ± 12.5

34.4

32.9 ± 2.3

0.05 Q4W (n = 3)

58.0

48.4 ± 16.2

58.5

46.9 ± 15.0

0.15 Q4W (n = 3)

81.7

74.5 ± 6.8***

86.0

77.0 ± 7.8

0.3 Q4W (n = 7b)

87.5

82.9 ± 5.4***

90.8

85.7 ± 9.6***

0.3 Q3W (n = 12c)

94.2

83.8 ± 5.1***

96.0

86.7 ± 7.0***

  1. p values from ANCOVA models including baseline TTR as covariate and dose groups as factor; models significant at p < 0.001 for Dose 1, p < 0.001 for Dose 2
  2. ***p < 0.001 vs. 0.01 mg/kg group
  3. aIncludes first-dose data from additional patient prior to protocol amendment
  4. bExcludes post-day 28 data from patient who experienced drug extravasation during second infusion
  5. cOne patient discontinued the study before second dose of patisiran
  6. ANCOVA analysis of variance; KD knockdown; Q3W every 3 weeks; Q4W every 4 weeks; SD standard deviation; TTR transthyretin
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172