RT Journal Article
T1 Patisiran treatment in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy after liver transplantation.
A1 Schmidt, Hartmut H
A1 Wixner, Jonas
A1 Planté-Bordeneuve, Violaine
A1 Muñoz-Beamud, Francisco
A1 Lladó, Laura
A1 Gillmore, Julian D
A1 Mazzeo, Anna
A1 Li, Xingyu
A1 Arum, Seth
A1 Jay, Patrick Y
A1 Adams, David
A1 Patisiran Post-LT Study Group, 
K1 clinical research/practice
K1 clinical trial
K1 liver allograft function/dysfunction
K1 liver transplantation/hepatology
K1 molecular biology: small interfering RNA
K1 neurology
K1 patient survival
K1 pharmacology
AB Hereditary transthyretin-mediated (hATTR) amyloidosis, or ATTRv amyloidosis, is a progressive disease, for which liver transplantation (LT) has been a long-standing treatment. However, disease progression continues post-LT. This Phase 3b, open-label trial evaluated efficacy and safety of patisiran in patients with ATTRv amyloidosis with polyneuropathy progression post-LT. Primary endpoint was median transthyretin (TTR) reduction from baseline. Twenty-three patients received patisiran for 12 months alongside immunosuppression regimens. Patisiran elicited a rapid, sustained TTR reduction (median reduction [Months 6 and 12 average], 91.0%; 95% CI: 86.1%-92.3%); improved neuropathy, quality of life, and autonomic symptoms from baseline to Month 12 (mean change [SEM], Neuropathy Impairment Score, -3.7 [2.7]; Norfolk Quality of Life-Diabetic Neuropathy questionnaire, -6.5 [4.9]; least-squares mean [SEM], Composite Autonomic Symptom Score-31, -5.0 [2.6]); and stabilized disability (Rasch-built Overall Disability Scale) and nutritional status (modified body mass index). Adverse events were mild or moderate; five patients experienced ≥1 serious adverse event. Most patients had normal liver function tests. One patient experienced transplant rejection consistent with inadequate immunosuppression, remained on patisiran, and completed the study. In conclusion, patisiran reduced serum TTR, was well tolerated, and improved or stabilized key disease impairment measures in patients with ATTRv amyloidosis with polyneuropathy progression post-LT (www.clinicaltrials.gov NCT03862807).
YR 2022
FD 2022-03-26
LK http://hdl.handle.net/10668/19889
UL http://hdl.handle.net/10668/19889
LA en
DS RISalud
RD Apr 19, 2025