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

Amyloid : the International Journal of Experimental and Clinical Investigation : the Official Journal of the International Society of Amyloidosis
Laura ObiciDavid Adams

Abstract

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 a...Continue Reading

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Citations

May 8, 2020·Journal of the Peripheral Nervous System : JPNS·Laura Obici, David Adams
Aug 17, 2020·Current Opinion in Neurology·David Adams, Michel Slama
May 14, 2020·Acta Pharmacologica Sinica·Wei-Min YinMin Luo
Sep 15, 2020·Muscle & Nerve·Divpreet KaurPaola Sandroni
Jan 30, 2021·Biochemical Pharmacology·M May ZhangXiao-Bo Zhong
Feb 21, 2021·Heart Failure Reviews·Jose N Nativi-NicolauMathew S Maurer
May 1, 2021·Brain Sciences·Luca GentileAnna Mazzeo
May 8, 2021·Orphanet Journal of Rare Diseases·Thomas H BrannaganFrederick L Ruberg
Aug 2, 2021·Molecular Aspects of Medicine·Manish PandeyGeetanjali Chawla

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