Scientific Board Members (as of this date) |
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Teresa Coelho, MD, Chairperson
Unidade Clinica de Paramiloidose Hospital Geral de Santo Antonio
Porto, Portugal
Ole B. Suhr, MD, PhD
Umeå University Hospital
Umeå, Sweden
Yukio Ando, MD, PhD
Kumamoto University
Kumamoto, Japan
Isabel Conceição, MD
Hospital Santa Maria
Lisbon, Portugal
Márcia Waddington-Cruz, MD
Hospital Universitario Clementino Fraga FilhoRua Rodolpho Paulo Rocco
Rio de Janeiro, Brazil
Bo-Göran Ericzon, MD
Karolinska Institutet Karolinska
Stockholm, Sweden
Rodney Falk, MD
Harvard Vanguard Medical Associates, Cardiology
Boston, MA
Shû-ichi Ikeda, MD, PhD
Shinshû University School of Medicine
Matsumoto, Japan
Arnt Kristen, MD
Medical University of Heidelberg
Department of Cardiology, angiology, Respiratory Medicine
Heidelberg, Germany
Ilise Lombardo, MD
Pfizer, Inc.
New York, NY, USA
Mathew Maurer, MD
Columbia University Medical Center
New York Presbyterian Hospital
New York, NY, USA
Anna Mazzeo, MD
U.O. di Neurologia e Malattie Neuromuscolari
AOU Policlinico G. Martino
Messina, Italy
Violaine Planté-Bordeneuve, MD, PhD
Service de Neurologie
CHU Henri Mondor
Créteil, France
Claudio Rapezzi, MD
Instituto di Cardiologia,PAD 21
Universita di Bologna
Bologna, Italy
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Financial support for the THAOS registry has been provided by Pfizer. The THAOS registry is overseen by a scientific board comprised of scientific and clinical experts in the field of amyloid disease, including Pfizer representation. Pfizer is the legal owner of all survey data, and may use the data for purposes including regulatory submissions.
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WELCOME TO THAOS |
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THAOS Summary
The Transthyretin Amyloidoses Outcomes Survey (THAOS) is a longitudinal, observational survey open to all patients with transthyretin-associated amyloidosis (ATTR). The principal aims of the outcome survey are to better understand and characterize the natural history of the disease by studying a large and heterogeneous patient population. Survey data may be used to develop new treatment guidelines and recommendations, and to inform and educate clinicians about the management of this disease.
Background
The hereditary forms of ATTR are autosomal dominantly inherited diseases with variable penetrance. There are over 100 known amyloidogenic mutations of TTR, which result in variable phenotypic expressions that commonly affect the peripheral nerves, heart, kidney or eyes. The disease is progressive and fatal with an average survival between 10 and 15 years. The phenotype varies between different mutations, but also within the same mutation. The most prevalent neuropathic mutation (ATTR Val30Met) gives rise to a peripheral and autonomic neuropathy with an average age at onset that varies between different endemic areas from 37 to 56 years of age. Cardiomyopathy is also associated with this mutation.
The mutations with predominantly heart manifestations include the Danish ATTR Ile111Met mutation and the ATTR V122I mutation, which is present in 3.5% of African-American population. In addition, wild-type (native) TTR can misfold into amyloid fibrils which are deposited in the myocardial interstitium. This condition, known in the literature as senile systemic amyloidosis, primarily affects elderly (>70) men. Regardless of whether the TTR amyloid fibrils are as a result of a mutation, or the aging process, the condition is characterized by a restrictive cardiomyopathy with thickened ventricular walls, diastolic dysfunction and congestive heart failure.
The only currently available disease modifying treatment for TTR-amyloidosis is liver transplantation.
The clinical course of many mutations is scarcely known, and for the more common mutations the clinical presentation is complex and the impact of different organ manifestations on prognosis not determined. Therefore, an international, longitudinal, observational survey open to all patients with TTR-amyloidoses is needed, especially to determine the effect of various treatment regimes.
Objectives
In collaboration with external experts, FoldRx has developed the Transthyretin Amyloidoses Outcomes Survey (THAOS), an international, longitudinal, observational survey open to all patients with transthyretin-associated amyloidoses. The principal aims of the survey are to better understand and characterize the natural history of TTR-associated amyloidoses by studying a large and heterogeneous patient population.
Specifically, the survey objectives are:
- To describe the population of patients affected with TTR amyloidoses.
- To enhance the understanding of disease natural history, including the variability and progression of the hereditary and acquired forms of the disease
- To better understand the genotype – phenotype relationship in hereditary TTR amyloidoses (ATTR)
- To compare the progression of disease and overall survival in patients with ATTR with and without liver transplant
- To foster an international community of medical experts who will develop recommendations on the clinical management of TTR amyloidoses
- To better understand, manage and treat patients with TTR amyloidoses through publication of the survey data
- To evaluate treatment modalities that may benefit patients with TTR amyloidoses
Methods
THAOS is a global, multi-center, longitudinal observational survey open to all patients with TTR-associated amyloidoses, including both inherited and wild-type disease. It is open-ended with a minimum duration of 10 years. Patients will be followed as long as they are able to participate.
It is anticipated that THAOS will enroll several thousand patients across 50 or more survey sites over the 10-year enrollment period. Scientific and clinical disease experts will analyze and interpret these data and provide guidance and oversight of the survey to ensure that its goals are met.
The frequency and type of clinical and laboratory assessments will be performed according to the physician’s standard of care. Physicians are not required to modify their standard assessments or treatment interventions for patients. Patients will continue to receive their current medications and all other standard care for their disease.
THAOS allows for data to be recorded at any time during a patient’s participation in the survey. It is recommended that assessments be recorded in the THAOS database at least annually, or more often as needed. Final data recording will occur when the survey is completed, if the patient dies, or if the patient elects to discontinue the survey.
Data will be entered into a secure, confidential interactive internet-based application that allows for remote data entry through locally secured connections. The THAOS database system is designed to be an intuitive tool for each Investigator to enter and review data for their patients at any time during the survey.
THAOS Management
THAOS combines the experience and infrastructure a pharmaceutical company brings in establishing and maintaining a complicated international registry and the scientific oversight and independence a Scientific Board brings to ensure credible data analysis and publication.
FoldRx will be the legal repository of the THAOS database and is responsible for all operational aspects of the registry. FoldRx will be responsible for site identification, site initiation, site support during patient enrollment, database maintenance and statistical and logistical support to the Scientific Board. The THAOS Scientific Board is comprised of international amyloid experts and will be responsible for the analysis and timely public dissemination of the THAOS data on an on-going basis.
THAOS Timelines and Accomplishments
2007 Achievements:
- THAOS launched
- Initiated patient enrollment and site initiation
- Established Scientific Board
2008
- Increase awareness of THAOS through registry listing
(e.g. www.clinicaltrials.gov), discussion with patient organizations and presentations at scientific meetings
- Expand site participation and patient enrollment
- Establish publication strategy
- Initial data analysis and publication
2009-2017
- Expand site participation and patient enrollment
- Continuous data analysis
- On-going publication and data dissemination
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