Longitudinal changes in gastrointestinal amyloid deposition before and after patisiran treatment in Hereditary ATTR amyloidosis.

Authors

Shinji Masuko, Mitsuto Sato, Ken Takasone, Akira Matsushima, Shin Yanagisawa, Kazuhiro Oguchi, Yusuke Mochizuki, Yusuke Takahashi, Tsuneaki Yoshinaga, Nagaaki Kato, Yoshiki Sekijima

Published in

Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis. Pages 1-11. Nov 09, 2026. Epub Nov 09, 2026.

Abstract

Whether amyloid deposits regress after RNA interference therapy in hereditary ATTR (ATTRv) amyloidosis remains uncertain.
To quantify gastrointestinal amyloid changes before and after patisiran initiation and evaluate gastric and duodenal trajectories.
Twenty-six patients underwent serial gastroduodenal biopsies from up to 3 years before through 3 years after patisiran initiation. Digitally quantified submucosal Congo red (CR)-positive and transthyretin (TTR)-immunoreactive area fractions were analyzed using piecewise linear mixed-effects models with a knot at treatment initiation; categorical-year and site-stratified analyses were secondary.
Among 102 patient-time-point observations, CR positivity increased before patisiran (odds ratio [OR] per year, 1.87; 95% CI, 1.29-2.70) and decreased afterward (OR/year, 0.72; 95% CI, 0.55-0.94), with a significant slope change (OR ratio, 0.38; 95% CI, 0.22-0.66). At year 3, estimated CR positivity was 0.30%, compared with 1.08% at baseline (OR, 0.27; Holm-adjusted p = 0.0287). The TTR slope change was not significant (ratio, 0.56; p = 0.062). Gastric CR positivity showed a pronounced reversal (pretreatment OR/year, 3.71; post-treatment OR/year, 0.48; slope ratio, 0.13; p < 0.001), whereas the duodenal slope did not change significantly.
Patisiran was associated with regression of CR-positive gastrointestinal amyloid, while TTR-immunostaining and duodenal findings suggested assay- and site-dependent heterogeneity.

PMID:
43002629
Bibliographic data and abstract were imported from PubMed on 10 Nov 2026.

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