HIBA-Institutional Registry of Amyloidosis (RIA-HIBA)

Institutional Registry of Amyloidosis (Hospital Italiano de Buenos Aires)

1. Creating a population-based registry system Amyloidosis prospective epidemiological survey

   • risk factors
   • diagnosis
   • prognosis
   • treatment
   • monitoring
   • survival
2. Describe the occurrence of amyloidosis in the population of HIBA, Hospital Italiano de Buenos Aires.
3. Describe the characteristics of clinical presentation, evolution and predisposing factors of amyloidosis.

Study Overview

• Status: Recruiting
• Conditions: Amyloidosis

Detailed Description

Amyloidosis is a systemic disease that is usually a result of misfolded proteins in the form of amorphous fibrillar material in various tissues and can cause progressive dysfunction of the same. The prevalence of amyloidosis varies depending on the population concerned and the type of amyloid. While prevalence in the general population is unknown, according to estimates by the Mayo Clinic this prevalence is 1 in 90 666% in the U.S. In England this disease generated about 0.0084% (1367 / 16232579) of all hospital visits between April 2008 and April 2009.

The most common clinical manifestations include cardiac disease, renal and liver function, but it may vary widely depending on the type of amyloidosis, the organ infected and extent of the deposits. Amyloid infiltration can produce signs and symptoms that may be very similar to other rheumatic diseases. This may suggest potential clinical polymorphic underdiagnosis due to low clinical suspicion.

The registries are organized systems of systematic data collection of a large number of patients quickly and efficiently on a particular disease at a given time.

The main difficulty of the registries is the guarantee of the quality of their data.

The main objectives of the registry are:

1. Understand risk factors and prognosis.
2. Evaluate the diagnostic and therapeutic comparison with current standards.
3. Advance knowledge of the disease to optimize the assessment, treatment and monitoring of patients.
4. Analyze the effectiveness of new therapies.
5. Studying differences between populations.
6. Quickly estimate the morbidity, mortality and resource utilization associated with a disease entity.
7. Examine the course of a disease
8. Formulate novel hypotheses for further prospective studies. Currently, there are registries for patients with transthyretin amyloidosis (TAHOS), global registry of transplant patients with familial amyloid poly neuropathy. Also, there are indirect registries such as the kidney transplant, heart transplant, among others.

The investigators found no data on the prevalence or incidence, evolution and prognosis of amyloidosis in our country. There are no existing records of national Amyloidosis in Latin America that could describe the behavior of this disease in our environment. Because it is a chronic disease with amyloid infiltration and can produce signs and symptoms that may be very similar to other rheumatic diseases, this clinic potentially poliform, may suggest underestimation of low clinical suspicion. As there is no cure, some patients may persist symptomatic despite adequate therapy, that is why it is important the creation of a monitoring system to generate data on the evolution and prognosis. The data registries can be used to develop new treatment guidelines and recommendations, also to inform and educate physicians on the management of this disease.

The Hospital Italiano de Buenos Aires is a center of high complexity of derivation of this type of pathology and due to the fact that the hospital, that has a private health insurance system (HIBA's health plan [plan de salud, PS], gives the unique opportunity denominators for the generation of a population on their affiliates, therefore the investigators propose to make a Institutional Registry of Amyloidosis.

• Study Type: Observational
• Enrollment (Estimated): 314

Contacts and Locations

• Study Contact: Name: María L Posadas-Martínez, MD, PhD; Phone Number: 9353 +54 11 4959 0200; Email: maria.posadas@hospitalitaliano.org.ar
• Study Contact Backup: Name: Adela Aguirre, MD, PhD; Phone Number: 9353 +54 11 4959 0200; Email: adela.aguirre@hospitalitaliano.org.ar

Study Locations

• Argentina
  • Buenos Aires, Argentina, C1199
    • Recruiting
    • Hospital Italiano de Buenos Aires
    • Contact: Maria L Posadas-Martinez, MD; Phone Number: 9353 54 11 4959 0200; Email: maria.posadas@hospitalitaliano.org.ar

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Adults over 18 years old with diagnosis or suspected amyloidosis of Italiano Hospital of Buenos Aires

Description

Cases of amyloidosis are captured by electronic medical records whenever the physician register amyloidosis as a patient diagnosis, and/or there is amyloidosis in a biopsy specimen and/or requests for the following studies of an adult patient: plasma kappa and lambda light-chain concentrations, the kappa: lambda ratio, transthoracic Doppler echocardiography, or cardiovascular magnetic resonance examination or pyrophosphate scintigraphy From the possible cases included in the IRA, a prospective review of the electronics health records was performed to confirm the presence of amyloidosis

**Inclusion Criteria: 1 AND (2 or 3)

1. Patients over 18 years:
2. Confirmed amyloidosis: Proof of deposit of amyloid pathology by tissue biopsy in abdominal fat, bone marrow, rectum or organ involved (eg, kidney, liver, sural nerve)

3. Clinically compatible case of Amyloidosis :

   • Exclusion Criteria:

Refusal to participate in the study or the informed consent process by the patient or legal representative or refusal to consent to participate in the study in the case of minors.

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Epidemiologic characteristics	Survival anual	From inclusion date (daignosis) until the date of death from any cause or date of last follow up(if not dead) assessed up to 10 years
Epidemiologic characteristics	Organ response	From treatment date until the date of best organ response or documented progression or date of death from any cause, whichever came first, assessed up to 10 years
From treatment to folow up, annualy	Baseline description	at baseline
Treatment patterns	Type of treatment, duration, adverse events, completition	From diagnosis, evaluated after every line of treatment or annualy up to 10 years

Collaborators and Investigators

• Sponsor: Hospital Italiano de Buenos Aires
• Collaborators: Austral University, Argentina; La Fundacion Favaloro para la Investigacion y la Docencia Medica; Centro de Educación Medica e Investigaciones Clínicas Norberto Quirno; Hospital de Clinicas José de San Martín; Instituto Cardiovascular de Buenos Aires; HOSPITAL BRITANICO DE BUENOS AIRES; Hospital de Alta Complejidad en Red; Hospital Privado de Rosario; Sanatorio Anchorena
• Investigators: Principal Investigator: María Lourdes Posadas-Martínez, MD, Hospital Italiano de Buenos Aires, Argentina; Study Chair: Elsa Nucifora, MD, Hospital Italiano de Buenos Aires, Argentina; Study Chair: Diego Hernan Giunta, MD, Hospital Italiano de Buenos Aires, Argentina; Study Chair: Dorotea Fantl, MD, Hospital Italiano de Buenos Aires, Argentina; Study Chair: Gustavo Greloni, MD, Hospital Italiano de Buenos Aires, Argentina; Study Chair: Maria Adela Aguirre, MD, Hospital Italiano de Buenos Aires, Argentina; Study Chair: Cesar Antonio Belziti, MD, Hospital Italiano de Buenos Aires, Argentina; Study Chair: Patricia Sorroche, Hospital Italiano de Buenos Aires, Argentina; Study Chair: Soledad Saez, Hospital Italiano de Buenos Aires, Argentina; Study Chair: Erika Brulc, MD, Hospital Italiano de Buenos Aires, Argentina; Study Chair: Diego Perez de Arenaza, MD, Hospital Italiano de Buenos Aires, Argentina; Study Chair: Eugenia Villanueva, MD, Hospital Italiano de Buenos Aires, Argentina; Study Chair: Marcelina Carretero, MD, Hospital Italiano de Buenos Aires, Argentina; Principal Investigator: Carolina María Petit Cwirko, MD, Clinica Foianini, Santa Cruz, Bolivia; Principal Investigator: Pablo Young, MD, Hospital Británico, Buenos Aires, Argentina; Principal Investigator: Gisela Streitenberger, MD, Hospital de Alta Complejidad El Cruce Nestor Kirchner, Buenos Aires, Argentina; Study Chair: Luciana Leon Cejas, MD, Hospital Británico, Buenos Aires, Argentina; Principal Investigator: Veronica Volberg, MD, Hospital de Clínicas José de San Martin, Buenos Aires, Argentina; Principal Investigator: Sergio Juan Baratta, MD, Hospital Austral, Buenos Aires, Argentina; Principal Investigator: Juan Pablo Costabel, MD, Instituto cardiovascular Buenos Aires, Argentina; Principal Investigator: Carlos Dumont Dunayevich, MD, Hospital Privado de Rosario, Santa Fe, Argentina; Principal Investigator: Alejandro Quiroga, MD, Fundación Favaloro, Buenos Aires, Argentina; Study Chair: Adrian Fernandez, MD, Fundación Favaloro, Buenos Aires, Argentina; Principal Investigator: Josefina Parodi, MD, Anchorena, Buenos Aires, Argentina; Study Director: Franco Leoni, MD, Médica Uruguaya, Uruguay; Study Director: Eduardo Ortuño Campos, MD, Hospital Universitarios Austral, Buenos Aires, Argentina; Principal Investigator: Jorge Thierer, MD, CEMIC, , Buenos Aires, Argentina

Study record dates

Study Major Dates

• Study Start: April 1, 2011
• Primary Completion (Estimated): January 1, 2030
• Study Completion (Estimated): January 1, 2030

Study Registration Dates

• First Submitted: May 2, 2011
• First Submitted That Met QC Criteria: May 3, 2011
• First Posted (Estimated): May 4, 2011

Study Record Updates

• Last Update Posted (Actual): June 7, 2023
• Last Update Submitted That Met QC Criteria: June 5, 2023
• Last Verified: June 1, 2023

More Information

Terms related to this study

• Keywords: Amyloidosis; Known or Suspected
• Additional Relevant MeSH Terms: Metabolic Diseases; Proteostasis Deficiencies; Amyloidosis
• Other Study ID Numbers: 1675

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No