New Hemolysis Parameters in Sickle Cell Disease (HEMODREP)

Evaluation de la capacité Physique Sous Maximale au Moyen du Test de Marche de 6 Minutes Des Enfants et Adultes Jeunes Suivis Pour drépanocytose et Nouveaux paramètres d'hémolyse : Vers Une prédiction Des Complications Vasculaires ?

The 6-minute walk (6MWT) test is used in adults and children affected by a wide range of chronic diseases to evaluate their sub-maximal exercise capacity. It reflects the global response of various physiological systems (respiratory, cardio-vascular, neurologic, metabolic and musculosquelettic) in a situation simulating a daily life activity. In children with sickle cell disease, the 6MWT is correlated with a low level of hemoglobin, a low level of fetal hemoglobin and low red cell deformability. Our team previously reported that in a population of children with sickle cell disease, highly treated with hydroxyurea, the sole factor which was independently linked to the 6MWT was the presence of silence infarct.

As the cardio-vascular and cerebro-vascular injury in sickle cell disease are directly correlated with hemolysis, the investigators aim to evaluate a) the clinical relevance of endothelial and inflammation parameters and new hemolysis markers and b) if the presence of silent infarct and the 6MWT are correlated with this biological markers.

This cross-sectional study will include sickle cell disease patients regularly followed for more than 5 years at Hôpital Universitaire des Enfants Reine Fabiola, Centre Hospitalier Universitaire (CHU)-Brugmann, Centre Hospitalier Etterbeek-Ixelles, CHU Saint-Pierre, Cliniques Universitaires Saint-Luc (Bruxelles, Belgium). Inclusion criteria are: sickle cell disease (SS, Sbeta°, SC, Sbeta+), age range : 6 to 25 years, signed informed consent. Exclusion criteria are: transplanted patients, inability to perform the 6MWT (severe cognitive disability, femoral osteonecrosis with functional impairment), hospitalization and/ or transfusion in the last 3 months for acute event.

Demographic data and clinical data will be retrospectively recorded. Blood test and 6MWT will be performed in steady state. Studied analysis will be: coagulation factors, free hemoglobin, Pro-B type natriuretic peptide (Pro-BNP), High sensitivity C reactive protein (HS-CRP), Intercellular Adhesion Molecule (ICAM), Vascular Cell Adhesion Molecule (VCAM) and Selectins.

With this study, the investigators expect to validate new predictive markers for cardio-vascular or cerebrovascular injury and to identify patients at high risk to develop these complications.

Study Overview

• Status: Unknown
• Conditions: Sickle Cell Disease

• Study Type: Observational
• Enrollment (Anticipated): 150

Contacts and Locations

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years to 25 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Children and young adults with Sickle Cell Disease

Description

Inclusion Criteria:

• Sickle Cell Disease (HbSS, S beta°, S beta+, SC)
• Regular follow-up from more than 5 years
• Written informed consent

Exclusion Criteria:

• Transplanted patients
• Hospitalisation for an acute event within 3 months
• Acute transfusion within 3 months
• Unable to performed 6-minute walk test

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Cross-Sectional

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Correlation between new biological data and clinical phenotype	The primary outcome of this study is to validate the relationship between these new biological markers and the clinical phenotype.	2 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Correlation between new biological data and 6-minute walk test	The secondary outcome is to confirm the correlation between the 6-minute walk test and the presence of silent infarct and to explore the relationship between these new markers and the 6-minute walk test.	2 years

Collaborators and Investigators

• Sponsor: Queen Fabiola Children's University Hospital

Study record dates

Study Major Dates

• Study Start (Anticipated): September 1, 2019
• Primary Completion (Anticipated): September 1, 2021
• Study Completion (Anticipated): March 1, 2022

Study Registration Dates

• First Submitted: January 24, 2017
• First Submitted That Met QC Criteria: January 31, 2017
• First Posted (Estimate): February 2, 2017

Study Record Updates

• Last Update Posted (Actual): February 12, 2019
• Last Update Submitted That Met QC Criteria: February 11, 2019
• Last Verified: February 1, 2019

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Anemia, Sickle Cell
• Other Study ID Numbers: P2016/ONCO-HEMATO/HEMODREP

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Undecided