Ignite Creation Date:
2024-05-06 @ 12:06 AM
Last Modification Date:
2024-10-26 @ 10:45 AM
Study NCT ID:
NCT01496963
Status:
COMPLETED
Last Update Posted:
2022-08-04
First Post:
2011-12-14
Brief Title:
Prevalence of Pulmonary Hypertension PAH in Patients With Thalassemia
Sponsor:
Ente Ospedaliero Ospedali Galliera
Organization:
Ente Ospedaliero Ospedali Galliera
Study Overview
Official Title:
Observational Multicenter Study Lasting 12 Months to Determine the Prevalence of Pulmonary Hypertension PAH in Patients With Thalassemia Major and Intermedia and Verify the Suitability of Common Diagnostic Criteria in This Population
Status:
COMPLETED
Status Verified Date:
2022-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
PAH2010
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 TIFand 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
Detailed Description:
The most recent International Classification of pulmonary arterial hypertension PHA include Hemoglobinopathies in Class I At present there is no determination of the prevalence of this disease in a large population of thalassemic patients followed in a uniform way The diagnostic criteria used for the normal population may not be suitable for a population such as thalassemia patients who present features like chronic anemia iron overload liver disease endocrine disorders etc
The criteria used to define the disease PHA will be those ones dictated by the above mentioned guidelines
Primary Objective of the study is the determination of the prevalence and severity of PHA in thalassemia syndromes recently introduced in Class I of the Classification of PHA
Secondary objectives are
Critical evaluation of current diagnostic criteria derived from those applied to the general population taking into account the peculiarities of the observed disease in the thalassemic population
Evaluation of sensitivity and specificity of echocardiogram versus right cardiac catheterization RHC
Evaluation of the correlation between
echocardiography and RHC resistance and heart rate determined both by RHC and echocardiography Determination of the patients resulted vasoreactive during RHC Validation of sensitivity and specificity sensspec of 6 minutes walking test 6MWT and brain natriuretic peptide BNP in Thalassemia Major TM and Intermediate TI with reference to specific pulmonary hypertensive disease
In order to achieve the objectives of the study observed patients will be divided into group according to the following the criteria
group a
Pulmonary artery pressure PAP assessed by echocardiogram 36 mmHg or a tricuspid regurgitant jet velocity TG 3 msec and data on PAP and mean left ventricular ejection fraction LVEF 50 group b
PAP estimated by echocardiography 40 mmHg or TG 32 m sec and LVEF 50
As indicated by the Guidelines patients b with increased PAP TG 32 m sec or 40 mm Hg will be further studied using RHC and vasoreactivity testing Angio CAT 6MWT and BNP
group c - PAP estimated by echocardiography in the range of values 3 msec TG and 32 m sec or 36 mm Hg and 40 mmHg and LVEF 50 Each case included both in group b and c will be paired with two controls included in the group a to make the groups more comparable
The group a will serve as control group to compare the diagnostic methods evaluated as per protocol
In order to divide the patients into the three groups specified above first it will be evaluated PAP LVEF and TG assessed by echocardiographic examination performed in the six months prior to the beginning of the study thalassemic patients have to perform echocardiography once a year to monitor cardiac function according to the guidelines
Patients belonging to groups a b and c will perform the following assessments
- Clinical cardiac evaluation according to the New York Heart Association NYHA functional class I to IV
As per the International Guidelines patients belonging to groups b and c have clinical indications to undergo Two-dimensional echocardiography-Doppler Duplex Scanner PW and color flow CW to determine the following parameters
End-systolic volume and left ventricular end diastolic indexed according to body surface
Percentage change of right ventricular areas area diastolicsystolic area expressed as a percentage
Tricuspid lateral annulus excursion Longitudinal TAPS
Eccentricity Index EI
TG
Pulmonary resistance
Moreover patients belonging to group b according to the International Guidelines have clinical indication to undergo RHC diagnostic evaluation to rule out the presence of associated diseases and to measure
Pulmonary pressures
Mean atrial pressure
Pulmonary resistance
Cardiac rate
Vasoreactivity test
To compare the data derived from the RHC and the echocardiography the following echocardiographic parameters will be further evaluated
Right atrial pressure Cardiac output Pulmonary wedge pressure
The criteria used to define the disease PHA will be those ones dictated by the above mentioned guidelines
Primary Objective of the study is the determination of the prevalence and severity of PHA in thalassemia syndromes recently introduced in Class I of the Classification of PHA
Secondary objectives are
Critical evaluation of current diagnostic criteria derived from those applied to the general population taking into account the peculiarities of the observed disease in the thalassemic population
Evaluation of sensitivity and specificity of echocardiogram versus right cardiac catheterization RHC
Evaluation of the correlation between
echocardiography and RHC resistance and heart rate determined both by RHC and echocardiography Determination of the patients resulted vasoreactive during RHC Validation of sensitivity and specificity sensspec of 6 minutes walking test 6MWT and brain natriuretic peptide BNP in Thalassemia Major TM and Intermediate TI with reference to specific pulmonary hypertensive disease
In order to achieve the objectives of the study observed patients will be divided into group according to the following the criteria
group a
Pulmonary artery pressure PAP assessed by echocardiogram 36 mmHg or a tricuspid regurgitant jet velocity TG 3 msec and data on PAP and mean left ventricular ejection fraction LVEF 50 group b
PAP estimated by echocardiography 40 mmHg or TG 32 m sec and LVEF 50
As indicated by the Guidelines patients b with increased PAP TG 32 m sec or 40 mm Hg will be further studied using RHC and vasoreactivity testing Angio CAT 6MWT and BNP
group c - PAP estimated by echocardiography in the range of values 3 msec TG and 32 m sec or 36 mm Hg and 40 mmHg and LVEF 50 Each case included both in group b and c will be paired with two controls included in the group a to make the groups more comparable
The group a will serve as control group to compare the diagnostic methods evaluated as per protocol
In order to divide the patients into the three groups specified above first it will be evaluated PAP LVEF and TG assessed by echocardiographic examination performed in the six months prior to the beginning of the study thalassemic patients have to perform echocardiography once a year to monitor cardiac function according to the guidelines
Patients belonging to groups a b and c will perform the following assessments
- Clinical cardiac evaluation according to the New York Heart Association NYHA functional class I to IV
As per the International Guidelines patients belonging to groups b and c have clinical indications to undergo Two-dimensional echocardiography-Doppler Duplex Scanner PW and color flow CW to determine the following parameters
End-systolic volume and left ventricular end diastolic indexed according to body surface
Percentage change of right ventricular areas area diastolicsystolic area expressed as a percentage
Tricuspid lateral annulus excursion Longitudinal TAPS
Eccentricity Index EI
TG
Pulmonary resistance
Moreover patients belonging to group b according to the International Guidelines have clinical indication to undergo RHC diagnostic evaluation to rule out the presence of associated diseases and to measure
Pulmonary pressures
Mean atrial pressure
Pulmonary resistance
Cardiac rate
Vasoreactivity test
To compare the data derived from the RHC and the echocardiography the following echocardiographic parameters will be further evaluated
Right atrial pressure Cardiac output Pulmonary wedge pressure
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None