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Patisiran, an RNAi ...
Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis
- Article/chapterEnglish2018
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Massachusetts Medical Society,2018
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Numbers
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LIBRIS-ID:oai:DiVA.org:umu-150728
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https://urn.kb.se/resolve?urn=urn:nbn:se:umu:diva-150728URI
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https://doi.org/10.1056/NEJMoa1716153DOI
Supplementary language notes
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Language:English
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Summary in:English
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Subject category:ref swepub-contenttype
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Subject category:art swepub-publicationtype
Notes
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BACKGROUND: Patisiran, an investigational RNA interference therapeutic agent, specifically inhibits hepatic synthesis of transthyretin.METHODS: In this phase 3 trial, we randomly assigned patients with hereditary transthyretin amyloidosis with polyneuropathy, in a 2:1 ratio, to receive intravenous patisiran (0.3 mg per kilogram of body weight) or placebo once every 3 weeks. The primary end point was the change from baseline in the modified Neuropathy Impairment Score+7 (mNIS+7; range, 0 to 304, with higher scores indicating more impairment) at 18 months. Other assessments included the Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QOL-DN) questionnaire (range, -4 to 136, with higher scores indicating worse quality of life), 10-m walk test (with gait speed measured in meters per second), and modified body-mass index (modified BMI, defined as [weight in kilograms divided by square of height in meters] x albumin level in grams per liter; lower values indicated worse nutritional status).RESULTS: A total of 225 patients underwent randomization (148 to the patisiran group and 77 to the placebo group). The mean (+/- SD) mNIS+7 at baseline was 80.9 +/- 41.5 in the patisiran group and 74.6 +/- 37.0 in the placebo group; the least-squares mean (+/- SE) change from baseline was -6.0 +/- 1.7 versus 28.0 +/- 2.6 (difference, -34.0 points; P<0.001) at 18 months. The mean (+/- SD) baseline Norfolk QOL-DN score was 59.6 +/- 28.2 in the patisiran group and 55.5 +/- 24.3 in the placebo group; the least-squares mean (+/- SE) change from baseline was -6.7 +/- 1.8 versus 14.4 +/- 2.7 (difference, -21.1 points; P<0.001) at 18 months. Patisiran also showed an effect on gait speed and modified BMI. At 18 months, the least-squares mean change from baseline in gait speed was 0.08 +/- 0.02 m per second with patisiran versus -0.24 +/- 0.04 m per second with placebo (difference, 0.31 m per second; P<0.001), and the least-squares mean change from baseline in the modified BMI was -3.7 +/- 9.6 versus -119.4 +/- 14.5 (difference, 115.7; P<0.001). Approximately 20% of the patients who received patisiran and 10% of those who received placebo had mild or moderate infusion-related reactions; the overall incidence and types of adverse events were similar in the two groups.CONCLUSIONS: In this trial, patisiran improved multiple clinical manifestations of hereditary transthyretin amyloidosis.
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Added entries (persons, corporate bodies, meetings, titles ...)
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Gonzalez-Duarte, A.
(author)
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O'Riordan, W. D.
(author)
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Yang, C. -C
(author)
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Ueda, M.
(author)
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Kristen, A. V.
(author)
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Tournev, I.
(author)
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Schmidt, H. H.
(author)
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Coelho, T.
(author)
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Berk, J. L.
(author)
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Lin, K. -P
(author)
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Vita, G.
(author)
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Attarian, S.
(author)
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Plante-Bordeneuve, V.
(author)
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Mezei, M. M.
(author)
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Campistol, J. M.
(author)
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Buades, J.
(author)
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Brannagan, T. H. , I I I
(author)
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Kim, B. J.
(author)
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Oh, J.
(author)
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Parman, Y.
(author)
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Sekijima, Y.
(author)
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Hawkins, P. N.
(author)
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Solomon, S. D.
(author)
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Polydefkis, M.
(author)
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Dyck, P. J.
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Gandhi, P. J.
(author)
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Goyal, S.
(author)
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Chen, J.
(author)
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Strahs, A. L.
(author)
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Nochur, S. V.
(author)
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Sweetser, M. T.
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Garg, P. P.
(author)
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Vaishnaw, A. K.
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Gollob, J. A.
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Suhr, Ole B.Umeå universitet,Medicin(Swepub:umu)olsu0002
(author)
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Umeå universitetMedicin
(creator_code:org_t)
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In:New England Journal of Medicine: Massachusetts Medical Society379:1, s. 11-210028-47931533-4406
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Adams, D.
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Gonzalez-Duarte, ...
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O'Riordan, W. D.
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Yang, C. -C
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Ueda, M.
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Kristen, A. V.
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Tournev, I.
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Schmidt, H. H.
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Coelho, T.
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Berk, J. L.
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Lin, K. -P
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Vita, G.
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Attarian, S.
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Plante-Bordeneuv ...
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Mezei, M. M.
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Campistol, J. M.
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Buades, J.
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Brannagan, T. H. ...
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Kim, B. J.
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Oh, J.
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Parman, Y.
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Sekijima, Y.
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Hawkins, P. N.
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Solomon, S. D.
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Polydefkis, M.
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Dyck, P. J.
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Gandhi, P. J.
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Goyal, S.
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Chen, J.
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Strahs, A. L.
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Nochur, S. V.
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Sweetser, M. T.
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Garg, P. P.
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Vaishnaw, A. K.
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Gollob, J. A.
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Suhr, Ole B.
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Umeå University