We thank all THAOS patients and investigators for their important contributions to this study. We also thank Marcus Vinicius Pinto, Rajiv Mundayat, and Denise Rill for their contributions to earlier versions of this work.
Additional THAOS investigators contributing to this analysis are: Fabio Adrian Barroso: FLENI, Ciudad Autonoma de Buenos Aires, Argentina; Marcelo Rugiero: Hospital Italiano de Buenos Aires, Buenos Aires, Argentina; Johan Van Cleemput: Afdeling Klinische Cardiologie, O&N I, Leuven, Belgium; Ivaylo Tarnev: Alexandrovska University Hospital Clinic of Neurology, Sofia, Bulgaria; Theodoros Kyriakides: Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus; Arnt Kristen: Medical University of Heidelberg, Heidelberg, Germany; Hartmut Schmidt: Universitätsklinikum Münster – Transplant Hepatology, Münster, Germany; Felix Darstein: Johann-Gutenberg-Universität, Mainz, Germany; Burkhard Gess: University Hospital of RWTH Aachen, Aachen, Germany; Josep Maria Campistol Plana: Institut Clinic de Nefrologia i Urologia – ICNU, Hospital Clinic i Provincial de Barcelona, Barcelona, Spain; Juan Gonzalez Moreno: Hospital Son Llatzer, Palma de Mallorca, Spain; Jose Gonzalez Costello: Hospital Universitari de Bellvitge, Barcelona, Spain; Pablo Garcia Pavia: Hospital Universitario Puerta de Hierro, Majadahonda, Spain; Roberto Fernandéz Torrón: Hospital Universitario Donostia, Gipuzkoa – San Sebastian, Spain; Francisco Munoz Beamud: Hospital Juan Ramon Jimenez, Huelva, Spain; Violaine Planté-Bordeneuve, CHU Henri Mondor, Créteil, France; David Adams: CHU de Bicetre, Cedex, France; Olivier Lairez: CHU de Toulouse – Hopital Rangueil, Toulouse Cedex 09, France; Claudio Rapezzi: Comitato Etico Indipendente dell Azienda, Bologna, Italy; Giampaolo Merlini: Centro per lo Studio e la Cura delle Amiloidosi Sistemiche, Pavia, Italy; Marco Luigetti: Fondazione Policlinico Gemelli – Universita Cattolica del Sacro Cuore, Roma, Italy; Yoshiki Sekijima: Shinshu University School of Medicine, Matsumoto, Japan; Taro Yamashita: Kumamoto University, Kumamoto-city, Japan; Sonoko Misawa: Chiba University Hospital, Chiba-shi, Japan; Soon Chai Low: University Malaya Medical Centre, Kuala Lumpur, Malaysia; Hans Nienhuis: University Medical Center Groningen, Groningen, the Netherlands; Teresa Coelho: Unidade Clinica de Paramiloidose-Centro Hospitalar Porto, EPE-Hospital Geral Santo Antonio, Porto, Portugal; Isabel Conceição: Hospital Santa Maria, Lisboa, Portugal; Rayomand Press: Karolinska University Hospital, Huddinge, Stockholm, Sweden; Yesim Parman: Istanbul University, Istanbul Faculty of Medicine, Department of Neurology, Istanbul, Turkey; Mathew Maurer: Columbia University Medical Center, New York, NY, USA; Stephen Gottlieb: University of Maryland, Baltimore, MD, USA; Annabel Wang: University of California, Irvine, Orange, CA, USA; Brian Drachman: University of Pennsylvania – Perelman Center for Advanced Medicine, Philadelphia, PA, USA; Angela Dispenzieri: Mayo Clinic, Rochester, MN, USA; Sasa Zivkovic: University of Pittsburgh Medical Center, Pittsburgh, PA, USA; Daniel Lenihan: Washington University School of Medicine, St. Louis, MO, USA
The THAOS registry and this analysis were sponsored by Pfizer. Medical writing support was provided by Caitlin Watson, PhD, of Engage Scientific Solutions, and funded by Pfizer, who also provided funding for the journal’s Rapid Service Fee.
All named authors meet the International Committee of Medical Journal Editors (ICMJE) criteria for authorship for this article, take responsibility for the integrity of the work as a whole, and have given their approval for this version to be published.
All authors contributed to the design and conduct of the analysis; interpretation of the data; and preparation, review, and approval of the manuscript.
A prior version of the analysis in this manuscript was presented at the 2019 Peripheral Nerve Society (PNS) Annual Meeting, June 22–26, 2019, Genoa, Italy.
Márcia Waddington-Cruz received research funding, consulting fees, and travel support for advisory boards and meetings from FoldRx Pharmaceuticals and Pfizer. Jonas Wixner received consulting fees and travel support for lectures and advisory boards from Pfizer, Akcea Therapeutics, and Alnylam Pharmaceuticals. Yukio Ando declares receipt of consulting fee or honorarium, support for travel to meetings, and provision for writing assistance from Pfizer. Leslie Amass, Doug Chapman, and Jan Kiszko are full-time employees of Pfizer and hold stock and/or stock options with Pfizer.
Compliance with Ethics Guidelines
All THAOS study sites received ethical or institutional review board approval prior to patient enrollment and followed the International Council for Harmonisation Good Pharmacoepidemiology Practice guidelines and the principles of the Declaration of Helsinki. Each patient provided written informed consent.
Pfizer provides secure access to anonymized patient-level data to qualified researchers in response to scientifically valid research proposals. Further details can be found at https://www.pfizer.com/science/clinical-trials/trial-data-and-results.