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Quality of life outcomes in APOLLO, the phase 3 trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis

2020-09Journal article Open access
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dc.contributor.authorObici, Laura
dc.contributor.authorBerk, John L
dc.contributor.authorGonzález-Duarte, Alejandra
dc.contributor.authorCoelho, Teresa
dc.contributor.authorGillmore, Julian
dc.contributor.authorSchmidt, Hartmut H-J
dc.contributor.authorSchilling, Matthias
dc.contributor.authorYamashita, Taro
dc.contributor.authorLabeyrie, Céline
dc.contributor.authorBrannagan, Thomas H
dc.contributor.authorAjroud-Driss, Senda
dc.contributor.authorGorevic, Peter
dc.contributor.authorKristen, Arnt V
dc.contributor.authorFranklin, Jaclyn
dc.contributor.authorChen, Jihong
dc.contributor.authorSweetser, Marianne T
dc.contributor.authorWang, Jing Jing
dc.contributor.authorAdams, David
dc.date.accessioned2021-07-02T18:29:17Z
dc.date.available2021-07-02T18:29:17Z
dc.date.issued2020-09
dc.description.abstractIntroduction: Hereditary transthyretin-mediated (hATTR) amyloidosis is a rare, fatal, multisystem disease leading to deteriorating quality of life (QOL). The impact of patisiran on QOL in patients with hATTR amyloidosis with polyneuropathy from the phase 3 APOLLO study (NCT01960348) is evaluated.Methods: Patients received either patisiran 0.3 mg/kg (n = 148) or placebo (n = 77) intravenously once every three weeks for 18 months. Multiple measures were used to assess varying aspects of QOL.Results: At 18 months, compared with placebo, patisiran improved Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QOL-DN) score; (least squares [LS] mean difference: -21.1; p = 1.10 × 10-10; improved across all domains), EuroQoL 5-dimensions 5-levels (LS mean difference: 0.2; p = 1.4 × 10-12), EuroQoL-visual analog scale (LS mean difference: 9.5; p=.0004), Rasch-built Overall Disability Scale (LS mean difference: 9.0; p = 4.07 × 10-16) and Composite Autonomic Symptom Score-31(COMPASS-31; LS mean difference: -7.5; p=.0008). Placebo-treated patients experienced rapid QOL deterioration; treatment effects for patisiran were observed as early as 9 months. At 18 months, patisiran improved Norfolk QOL-DN total score and three individual domains as well as COMPASS-31 total scores relative to baseline. Consistent benefits were also observed in the cardiac subpopulation.Conclusion: The benefits of patisiran across all QOL measures and the rapid deterioration observed with placebo, highlight the urgency in early treatment for patients with hATTR amyloidosis with polyneuropathy.pt_PT
dc.description.sponsorshipThis study was funded by Alnylam Pharmaceuticalspt_PT
dc.description.versioninfo:eu-repo/semantics/publishedVersionpt_PT
dc.identifier.citationObici L, Berk JL, González-Duarte A, Coelho T, Gillmore J, Schmidt HH, Schilling M, Yamashita T, Labeyrie C, Brannagan TH 3rd, Ajroud-Driss S, Gorevic P, Kristen AV, Franklin J, Chen J, Sweetser MT, Wang JJ, Adams D. Quality of life outcomes in APOLLO, the phase 3 trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis. Amyloid. 2020 Sep;27(3):153-162. doi: 10.1080/13506129.2020.1730790. Epub 2020 Mar 4. PMID: 32131641.pt_PT
dc.identifier.doi10.1080/13506129.2020.1730790pt_PT
dc.identifier.issn1350-6129
dc.identifier.urihttp://hdl.handle.net/10400.16/2481
dc.language.isoengpt_PT
dc.peerreviewedyespt_PT
dc.publisherTaylor & Francispt_PT
dc.relation.publisherversionhttps://www.tandfonline.com/doi/full/10.1080/13506129.2020.1730790?scroll=top&needAccess=truept_PT
dc.rights.urihttp://creativecommons.org/licenses/by-nc-nd/4.0/pt_PT
dc.subjectAPOLLOpt_PT
dc.subjectNorfolk QOL-DNpt_PT
dc.subjecthereditary transthyretin-mediated amyloidosispt_PT
dc.subjectpatisiranpt_PT
dc.subjectquality of lifept_PT
dc.titleQuality of life outcomes in APOLLO, the phase 3 trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosispt_PT
dc.typejournal article
dspace.entity.typePublication
oaire.citation.conferencePlaceEnglandpt_PT
oaire.citation.endPage162pt_PT
oaire.citation.issue3pt_PT
oaire.citation.startPage153pt_PT
oaire.citation.titleAmyloidpt_PT
oaire.citation.volume27pt_PT
person.familyNameCoelho
person.givenNameTeresa
person.identifier.ciencia-idC71E-3343-F445
rcaap.rightsopenAccesspt_PT
rcaap.typearticlept_PT
relation.isAuthorOfPublicationca69c0a3-d48a-4b92-bbf7-c17288d609b7
relation.isAuthorOfPublication.latestForDiscoveryca69c0a3-d48a-4b92-bbf7-c17288d609b7

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