Repository logo

SNF - Serviço de Neurofisiologia

Permanent URI for this community

http://hdl.handle.net/10400.16/60

Browse

Browsing SNF - Serviço de Neurofisiologia by Author "Ajroud-Driss, Senda"

Now showing 1 - 1 of 1
  • Quality of life outcomes in APOLLO, the phase 3 trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis
    Publication . Obici, Laura; Berk, John L; González-Duarte, Alejandra; Coelho, Teresa; Gillmore, Julian; Schmidt, Hartmut H-J; Schilling, Matthias; Yamashita, Taro; Labeyrie, Céline; Brannagan, Thomas H; Ajroud-Driss, Senda; Gorevic, Peter; Kristen, Arnt V; Franklin, Jaclyn; Chen, Jihong; Sweetser, Marianne T; Wang, Jing Jing; Adams, David
    Introduction: 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.
    2020-09Journal article Open access Show more