@article{f3e0f87ce9d54672ba6da6125f259be7,
title = "Indirect treatment comparison of the efficacy of patisiran and inotersen for hereditary transthyretin-mediated amyloidosis with polyneuropathy",
abstract = "Introduction: Patisiran and inotersen are two therapies approved for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis with polyneuropathy, a rapidly progressive disease with a substantial clinical burden. This analysis indirectly compares the efficacy of patisiran and inotersen on neuropathy and quality of life (QOL). Methods: Published results from the NEURO-TTR study of inotersen and individual patient data from the APOLLO study of patisiran were used. Indirect comparisons were conducted for 15-month changes in neuropathy and QOL endpoints: modified Neuropathy Impairment Score +7 (mNIS+7Ionis,), Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QOL-DN) questionnaire, body mass index (BMI), and Polyneuropathy Disability (PND) score. Analyses were conducted under different assumptions about the impact of missing data and to adjust for baseline differences between studies. Results: Patisiran showed significantly greater treatment effects than inotersen for mNIS+7Ionis (mean difference: −12.3 [95% confidence interval: −21.4, −3.3]), Norfolk QOL-DN (−11.3 [−19.8, −2.9]), and BMI (1.0 [0.4, 1.7]). The proportion of patients with improvement or no change from baseline on PND score was higher for patisiran-treated patients (odds ratio: 8.9 [4.6, 17.5]). Results were consistent and robust across analyses and methods. Conclusions: Patisiran demonstrated greater treatment effects on neuropathy and QOL than inotersen in patients with hATTR amyloidosis with polyneuropathy.",
keywords = "Hereditary transthyretin-mediated amyloidosis, indirect comparison, inotersen, neuropathy, patisiran, quality of life",
author = "Peter Gorevic and Jaclyn Franklin and Jihong Chen and Gautam Sajeev and Wang, {Jessie C.H.} and Hollis Lin",
note = "Funding Information: This work was funded by Alnylam Pharmaceuticals. Medical writing assistance was provided by Shelley A. Batts, PhD, an employee of Analysis Group, Inc. Funding for this service was provided by the sponsor, Alnylam Pharmaceuticals. The authors would like to thank T Brannagan of Columbia University Medical Center for their assistance with research and the analysis and L Obici from the Amyloidosis Research and Treatment Center for their insightful discussion along with the development of the original abstract and poster presentation. Funding Information: Medical writing assistance was provided by Shelley A. Batts, PhD, an employee of Analysis Group, Inc. Funding for this service was provided by the sponsor, Alnylam Pharmaceuticals. The authors would like to thank T Brannagan of Columbia University Medical Center for their assistance with research and the analysis and L Obici from the Amyloidosis Research and Treatment Center for their insightful discussion along with the development of the original abstract and poster presentation. Funding Information: P Gorevic received grant support from Alnylam Pharmaceuticals, Ionis, and Pfizer for the conducting of phase 3 studies on patisiran, inotersen, and tafamimdis. H Lin is an employee of Alnylam Pharmaceuticals and holds stocks/options. J Franklin is a former employee of Alnylam Pharmaceuticals and was employed during the development of the manuscript by them. J Franklin also holds stocks and options with them. Furthermore, she is currently employed as a Program Manager at Voyager Therapeutics. J Chen is an employee of Alnylam Pharmaceuticals and holds stocks/options. G Sajeev is an employee of Analysis Group Inc, which has received consulting fees from Alnylam Pharmacetucals. JCH Wang is also an employee of Analysis Group Inc. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed. Publisher Copyright: {\textcopyright} 2020 The Author(s). Published by Informa UK Limited, trading as Taylor & Francis Group.",
year = "2021",
doi = "10.1080/14656566.2020.1811850",
language = "English",
volume = "22",
pages = "121--129",
journal = "Expert Opinion on Pharmacotherapy",
issn = "1465-6566",
publisher = "Taylor and Francis Ltd.",
number = "1",
}