Epidemiology and Pathophysiological Mechanisms of HTAP in SS and SC Children in Martinique and Guadeloupe.

NCT03368261 | Completed DMC

• 1 other identifier: RBM-PAP-2009/69
• Study Type: interventional
• Target: 185
• Locations: 1 country
• Sites: 1

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 patient's 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.

Conditions

Sickle Cell Disease

Keywords

Pulmonary arterial hypertension; SCD; Epidemiology

Outcome Measures

Primary Outcomes (1)

• incidence of pulmonary arterial hypertension

  The primary outcome of the present study is to estimate the incidence of pulmonary arterial hypertension documented by the presence of tricuspid regurgitation jet velocity of at least 2.5 ms-1 by Doppler echocardiographic assessment.

  Through study completion, an average of 5 years

Secondary Outcomes (1)

• The association between PAH risk and specific SCD complications

  Through study completion, an average of 5 years

Study Arms (1)

HTAP/ clinical complications in the sickle cell disease

OTHER

Supply epidemiological data on this detected HTAP, and allow the characterization of the clinico-biological paintings and the mortality which are associated to them.

Other: HTAP/ clinical complications in the sickle cell disease

Interventions

HTAP/ clinical complications in the sickle cell disease OTHER

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.

HTAP/ clinical complications in the sickle cell disease

Eligibility Criteria

• Age: 8 Years - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• children aged between 8 to 16 years old,
• SS homozygote or SC compound heterozygote, followed by the sickle cell centers of Guadeloupe and Martinique or by the pediatric department of the university hospital of Fort-de-France, identified by the systematic neonatal screening programs performed in Guadeloupe and Martinique or by other labeled centers, registered in the French medical social security national program, and for which the parental and the old children consent has been obtained.

You may not qualify if:

• other hemoglobinopathies, chronic transfusion therapy programs or treatments which affected the expression of the biomarkers studied (unless hydroxyurea treatment),
• recent blood transfusion or phlebotomies (less than 3 months);
• patients not at steady state,
• pregnancy or breast feeding,
• refusal of parental and old children consent.

Sponsors & Collaborators

• Centre Hospitalier Universitaire de la Guadeloupe: lead

Study Sites (1)

Hospital University Center of Martinique

Fort-de-France, 97261, Martinique

Location

MeSH Terms

Conditions

Anemia, Sickle Cell; Pulmonary Arterial Hypertension

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 Officials

• Maryse ETIENNE-JULAN, Doctor specializing in SCD

  Hospital University Center of Pointe-à-Pitre

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 1, 2017
• First Posted: December 11, 2017
• Study Start: January 11, 2010
• Primary Completion: October 2, 2013
• Study Completion: October 17, 2016
• Last Updated: December 11, 2017

Record last verified: 2017-12

Locations

MA (1)