Prevalence of Pulmonary Hypertension (PAH) in Patients With Thalassemia

NCT01496963 | Completed

• 1 other identifier: PAH2010
• Study Type: observational
• Target: 1,500
• Locations: 1 country
• Sites: 8

Brief Summary

This is a multicenter observational case-control analysis lasting 12 months aimed at determining the prevalence of pulmonary hypertension (PAH) in patients with Thalassemia Major and Intermedia. The patients will be followed, treated and examined according to the best standard clinical practice dictated by the Italian Society for the study of Hemoglobinopathies (SITE), Thalassemia International Federation (TIF)and the Task Force for Diagnosis and Treatment of Pulmonary Hypertension of European Society of Cardiology (ESC); European Respiratory Society (ERS); International Society of Heart and Lung Transplantation (ISHLT) guidelines.

Conditions

Thalassemia Major; Thalassemia Intermedia; Pulmonary Arterial Hypertension

Keywords

Thalassemia; Pulmonary Arterial Hypertension

Outcome Measures

Primary Outcomes (1)

• Determination of the prevalence

  Determination of the prevalence, defined as the total number of cases in the population, divided by the number of individuals in the population.

  12 months

Secondary Outcomes (1)

• Critical evaluation of current diagnostic criteria

  12 months

Study Arms (3)

group a)

Patients with pulmonary artery pressure (PAP) assessed (by echocardiogram) \<36 mmHg or a tricuspid regurgitant jet velocity (TG) \<3 m / sec and data on PAP and mean left ventricular ejection fraction (LVEF) \> 50%

Other: Physician standard-of-care according to ESC/ERS Guidelines

group b)

Patients with: PAP estimated (by echocardiography)\> 40 mmHg or TG\> 3.2 m / sec and LVEF\> 50% As indicated by the Guidelines, patients b) with increased PAP (TG\> 3.2 m / sec or\> 40 mm Hg) will be further studied using RHC and vasoreactivity testing. Angio CAT, 6MWT and BNP.

Other: Physician standard-of-care according to ESC/ERS Guidelines

group c)

patients with PAP estimated (by echocardiography) in the range of values \> 3 m / sec (TG) and \<3.2 m / sec or\> 36 mm Hg and \<40 mmHg and LVEF\> 50%

Other: Physician standard-of-care according to ESC/ERS Guidelines

Interventions

Physician standard-of-care according to ESC/ERS Guidelines OTHER

Physician standard-of-care

group a); group b); group c)

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with Thalassemia Major and Intermedia studied by echocardiography in the six months prior to the beginning of the study

You may qualify if:

• Patients with Thalassemia Major Patients or Intermediate referring to Centres using Web-Thal medical record (a clinical data sheet used for congenital anemias. Info: www.thalassemia.it)

You may not qualify if:

• Patients who are considered potentially unreliable and/or not cooperative

Sponsors & Collaborators

• Ente Ospedaliero Ospedali Galliera: lead

Study Sites (8)

SCDU Microcitemie-Pediatria A.O. Universitaria S.Luigi Gonzaga di Orbassano

Orbassano, Turin, 10043, Italy

Location

Divisione di Ematologia Ospedale Perrino

Brindisi, 72100, Italy

Location

Clinica pediatrica Ospedale Microcitemico

Cagliari, 09123, Italy

Location

DH Microcitemia dell'adulto Ospedale Microcitemico

Cagliari, 09123, Italy

Location

Centro della Microcitemia e delle Anemie Congenite -Ematologia e Cardiologia E.O. Ospedali Galliera

Genoa, 16128, Italy

Location

Centro Anemie Congenite Università di Milano IRCCS Ospedale Maggiore Policlinico

Milan, 20162, Italy

Location

Dipartimento di pediatria "F.Fede" A.O. Universitaria FedericoII di Napoli

Napoli, 80131, Italy

Location

U.O.C- Cardiologia Ospedale San Francesco

Nuoro, 8100, Italy

Location

Related Publications (2)

• Task Force for Diagnosis and Treatment of Pulmonary Hypertension of European Society of Cardiology (ESC); European Respiratory Society (ERS); International Society of Heart and Lung Transplantation (ISHLT); Galie N, Hoeper MM, Humbert M, Torbicki A, Vachiery JL, Barbera JA, Beghetti M, Corris P, Gaine S, Gibbs JS, Gomez-Sanchez MA, Jondeau G, Klepetko W, Opitz C, Peacock A, Rubin L, Zellweger M, Simonneau G. Guidelines for the diagnosis and treatment of pulmonary hypertension. Eur Respir J. 2009 Dec;34(6):1219-63. doi: 10.1183/09031936.00139009. Epub 2009 Sep 12. No abstract available.

  PMID: 19749199 BACKGROUND

• Derchi G, Galanello R, Bina P, Cappellini MD, Piga A, Lai ME, Quarta A, Casu G, Perrotta S, Pinto V, Musallam KM, Forni GL; Webthal Pulmonary Arterial Hypertension Group*. Prevalence and risk factors for pulmonary arterial hypertension in a large group of beta-thalassemia patients using right heart catheterization: a Webthal study. Circulation. 2014 Jan 21;129(3):338-45. doi: 10.1161/CIRCULATIONAHA.113.002124. Epub 2013 Sep 30.

  PMID: 24081970 DERIVED

MeSH Terms

Conditions

beta-Thalassemia; Pulmonary Arterial Hypertension; Thalassemia

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hypertension, Pulmonary; Lung Diseases; Respiratory Tract Diseases

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator and Chief of Centre for Microcythemia an Congenital Anemias - Hematology

Study Record Dates

• First Submitted: December 14, 2011
• First Posted: December 22, 2011
• Study Start: January 1, 2012
• Primary Completion: December 1, 2021
• Study Completion: December 1, 2021
• Last Updated: August 4, 2022

Record last verified: 2022-08

Locations

IT (8)