Clinical trial NCT01347047: HIBA-Institutional Registry of Amyloidosis
Official title: Institutional Registry of Amyloidosis (Hospital Italiano de Buenos Aires)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Amyloidosis
Keywords: Amyloidosis
Known or Suspected
Lead sponsor: Hospital Italiano de Buenos Aires
Sponsor class: OTHER
Collaborators: name: Instituto Cardiovascular de Buenos Aires
name: Austral University, Argentina
name: Hospital de Alta Complejidad en Red
name: Hospital de Clinicas José de San Martín
name: HOSPITAL BRITANICO DE BUENOS AIRES
name: Hospital Privado de Rosario
name: Centro de Educación Medica e Investigaciones Clínicas Norberto Quirno
name: La Fundacion Favaloro para la Investigacion y la Docencia Medica
name: Sanatorio Anchorena
First posted: 2011-05-04
Enrollment: 314
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: ECOLOGIC_OR_COMMUNITY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Adults over 18 years old with diagnosis or suspected amyloidosis of Italiano Hospital of Buenos Aires

Brief summary: 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.

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Epidemiologic characteristics
timeFrame: 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
measure: Epidemiologic characteristics
timeFrame: 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
measure: From treatment to folow up, annualy
timeFrame: at baseline
measure: Treatment patterns
timeFrame: From diagnosis, evaluated after every line of treatment or annualy up to 10 years
Secondary outcomes:
Other outcomes:
Locations:
city: Buenos Aires
country: Argentina
facility: Hospital Italiano de Buenos Aires
Study officials:
affiliation: Hospital Italiano de Buenos Aires, Argentina
name: María Lourdes Posadas-Martínez, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Hospital Italiano de Buenos Aires, Argentina
name: Elsa Nucifora, MD
role: STUDY_CHAIR
affiliation: Hospital Italiano de Buenos Aires, Argentina
name: Diego Hernan Giunta, MD
role: STUDY_CHAIR
affiliation: Hospital Italiano de Buenos Aires, Argentina
name: Dorotea Fantl, MD
role: STUDY_CHAIR
affiliation: Hospital Italiano de Buenos Aires, Argentina
name: Gustavo Greloni, MD
role: STUDY_CHAIR
affiliation: Hospital Italiano de Buenos Aires, Argentina
name: Maria Adela Aguirre, MD
role: STUDY_CHAIR
affiliation: Hospital Italiano de Buenos Aires, Argentina
name: Cesar Antonio Belziti, MD
role: STUDY_CHAIR
affiliation: Hospital Italiano de Buenos Aires, Argentina
name: Patricia Sorroche
role: STUDY_CHAIR
affiliation: Hospital Italiano de Buenos Aires, Argentina
name: Soledad Saez
role: STUDY_CHAIR
affiliation: Hospital Italiano de Buenos Aires, Argentina
name: Erika Brulc, MD
role: STUDY_CHAIR
affiliation: Hospital Italiano de Buenos Aires, Argentina
name: Diego Perez de Arenaza, MD
role: STUDY_CHAIR
affiliation: Hospital Italiano de Buenos Aires, Argentina
name: Eugenia Villanueva, MD
role: STUDY_CHAIR
affiliation: Hospital Italiano de Buenos Aires, Argentina
name: Marcelina Carretero, MD
role: STUDY_CHAIR
affiliation: Clinica Foianini, Santa Cruz, Bolivia
name: Carolina María Petit Cwirko, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Hospital Británico, Buenos Aires, Argentina
name: Pablo Young, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Hospital de Alta Complejidad El Cruce Nestor Kirchner, Buenos Aires, Argentina
name: Gisela Streitenberger, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Hospital Británico, Buenos Aires, Argentina
name: Luciana Leon Cejas, MD
role: STUDY_CHAIR
affiliation: Hospital de Clínicas José de San Martin, Buenos Aires, Argentina
name: Veronica Volberg, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Hospital Austral, Buenos Aires, Argentina
name: Sergio Juan Baratta, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Instituto cardiovascular Buenos Aires, Argentina
name: Juan Pablo Costabel, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Hospital Privado de Rosario, Santa Fe, Argentina
name: Carlos Dumont Dunayevich, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Fundación Favaloro, Buenos Aires, Argentina
name: Alejandro Quiroga, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Fundación Favaloro, Buenos Aires, Argentina
name: Adrian Fernandez, MD
role: STUDY_CHAIR
affiliation: Anchorena, Buenos Aires, Argentina
name: Josefina Parodi, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Médica Uruguaya, Uruguay
name: Franco Leoni, MD
role: STUDY_DIRECTOR
affiliation: Hospital Universitarios Austral, Buenos Aires, Argentina
name: Eduardo Ortuño Campos, MD
role: STUDY_DIRECTOR
affiliation: CEMIC, , Buenos Aires, Argentina
name: Jorge Thierer, MD
role: PRINCIPAL_INVESTIGATOR
References: