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http://hdl.handle.net/2445/138257
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DC Field | Value | Language |
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dc.contributor.author | Campistol Plana, Josep M. | - |
dc.contributor.author | González Duarte, Alejandra | - |
dc.contributor.author | Suhr, Ole B. | - |
dc.contributor.author | Goyal, Sunita | - |
dc.contributor.author | Gandhi, Pritesh J. | - |
dc.contributor.author | Polydefkis, Michael | - |
dc.contributor.author | Sekijima, Yoshiki | - |
dc.contributor.author | O'Riordan, William D. | - |
dc.contributor.author | Yang, Chih-Chao | - |
dc.contributor.author | Ueda, Mitsuharu | - |
dc.contributor.author | Kristen, Arnt V. | - |
dc.contributor.author | Coelho, Teresa | - |
dc.contributor.author | Berk, John L. | - |
dc.contributor.author | Lin, Kon Ping | - |
dc.contributor.author | Vita, Giuseppe | - |
dc.contributor.author | Attarian, Shahram | - |
dc.contributor.author | Planté Bordeneuve, Violaine | - |
dc.contributor.author | Mezei, Michelle M. | - |
dc.contributor.author | Buades, Juan | - |
dc.contributor.author | Brannagan, Thomas H. | - |
dc.contributor.author | Kim, Byoung J. | - |
dc.contributor.author | Oh, Jeeyoung | - |
dc.contributor.author | Parman, Yesim | - |
dc.contributor.author | Hawkins, Philip N. | - |
dc.contributor.author | Solomon, Scott D. | - |
dc.contributor.author | Dyck, Peter J. | - |
dc.contributor.author | Chen, Jihong | - |
dc.contributor.author | Strahs, Andrew L. | - |
dc.contributor.author | Nochur, Saraswathy V. | - |
dc.contributor.author | Sweetser, Marianne T. | - |
dc.contributor.author | Garg, Pushkal P. | - |
dc.contributor.author | Vaishnaw, Akshay K. | - |
dc.contributor.author | Gollob, Jared A. | - |
dc.date.accessioned | 2019-07-25T09:12:30Z | - |
dc.date.available | 2019-07-25T09:12:30Z | - |
dc.date.issued | 2018-07-05 | - |
dc.identifier.issn | 0028-4793 | - |
dc.identifier.uri | http://hdl.handle.net/2445/138257 | - |
dc.description.abstract | 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]×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. | - |
dc.format.extent | 11 p. | - |
dc.format.mimetype | application/pdf | - |
dc.language.iso | eng | - |
dc.publisher | Massachusetts Medical Society | - |
dc.relation.isformatof | Reproducció del document publicat a: https://doi.org/10.1056/NEJMoa1716153 | - |
dc.relation.ispartof | New England Journal of Medicine, 2018, vol. 379, num. 1, p. 11-21 | - |
dc.relation.uri | https://doi.org/10.1056/NEJMoa1716153 | - |
dc.rights | (c) Massachusetts Medical Society, 2018 | - |
dc.source | Articles publicats en revistes (Medicina) | - |
dc.subject.classification | Amiloïdosi | - |
dc.subject.classification | RNA | - |
dc.subject.classification | Assaigs clínics | - |
dc.subject.other | Amyloidosis | - |
dc.subject.other | RNA | - |
dc.subject.other | Clinical trials | - |
dc.title | Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis | - |
dc.type | info:eu-repo/semantics/article | - |
dc.type | info:eu-repo/semantics/publishedVersion | - |
dc.identifier.idgrec | 681162 | - |
dc.date.updated | 2019-07-25T09:12:30Z | - |
dc.rights.accessRights | info:eu-repo/semantics/openAccess | - |
Appears in Collections: | Articles publicats en revistes (Medicina) |
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File | Description | Size | Format | |
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681162.pdf | 366.83 kB | Adobe PDF | View/Open |
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