Ignite Creation Date:
2024-05-06 @ 10:50 AM
Last Modification Date:
2024-10-26 @ 12:36 PM
Study NCT ID:
NCT03368261
Status:
COMPLETED
Last Update Posted:
2017-12-11
First Post:
2017-12-01
Brief Title:
Epidemiology and Pathophysiological Mechanisms of HTAP in SS and SC Children in Martinique and Guadeloupe
Sponsor:
Centre Hospitalier Universitaire de Pointe-a-Pitre
Organization:
Centre Hospitalier Universitaire de Pointe-a-Pitre
Study Overview
Official Title:
Epidemiology and Pathophysiological Mechanisms of Pulmonary Arterial Hypertension in SS and SC Children in Martinique and GuadeloupeThree Years Follow up of a Cohort of Children Aged From 8 to 16 Years Old
Status:
COMPLETED
Status Verified Date:
2017-12
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:
SAPOTILLE
Brief Summary:
pulmonary arterial hypertension PAH has been reported with a prevalence of approximately 30 in adult sickle cell disease SCD patients with an increased mortality in SCD patients with PAH compared with those without PAH The identification of several hemolysis biomarkers such as lactate dehydrogenase bilirubin reticulocytes or hemoglobin level has clearly documented a link between hemolysis and PAH However other physiopathological mechanisms may be involved to explain PAH in these patients such as pulmonary thromboembolism pulmonary fibrosis or left heart diastolic and or systolic dysfunction
The investigators suggest studying HTAP in patients presenting the most frequent both drepanocytic syndromes SS and SC and homogeneous in their medical coverage and the association between HTAP risk and specific SCD complications
The investigators suggest studying HTAP in patients presenting the most frequent both drepanocytic syndromes SS and SC and homogeneous in their medical coverage and the association between HTAP risk and specific SCD complications
Detailed Description:
The primary aim of this study is to estimate the prevalence and the incidence of PAH in a population of SCD children SS SC with similar medical caring aged from 8 to 16 years old
Unlike the important number of studies in SCD adults very few SCD children studies were performed None of these studies reported the mortality rate associated with PAH in children although the literature reported a decrease of this morbid-mortality comparing different medical caring of the patients The investigators hypothesized that physiopathological mechanisms responsible for PAH had to be different in SCD children compared with adults as most degenerative processes had no time enough to appear during childrens lives At the inclusion in our study the diagnosis of PAH will be performed by transthoracic Doppler-echocardiograms in a group of 306 children aged between 8 to 16 years old with either SS or SC genotype with similar medical caring to avoid a known selection bias These patients will be followed during a 3 years longitudinal period The occurrence of the clinical specific complications associated with SCD acute chest syndrome painful vaso occlusive crisis septicemia and stroke and the observed mortality rate of our children group will be compared in patients groups stratified according to the occurrence of PAH The expression of several molecular and cellular genetic biomarkers potentially associated with this complication will also be studied
Unlike the important number of studies in SCD adults very few SCD children studies were performed None of these studies reported the mortality rate associated with PAH in children although the literature reported a decrease of this morbid-mortality comparing different medical caring of the patients The investigators hypothesized that physiopathological mechanisms responsible for PAH had to be different in SCD children compared with adults as most degenerative processes had no time enough to appear during childrens lives At the inclusion in our study the diagnosis of PAH will be performed by transthoracic Doppler-echocardiograms in a group of 306 children aged between 8 to 16 years old with either SS or SC genotype with similar medical caring to avoid a known selection bias These patients will be followed during a 3 years longitudinal period The occurrence of the clinical specific complications associated with SCD acute chest syndrome painful vaso occlusive crisis septicemia and stroke and the observed mortality rate of our children group will be compared in patients groups stratified according to the occurrence of PAH The expression of several molecular and cellular genetic biomarkers potentially associated with this complication will also be studied
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None