Study of 'Vascular Competence' Profile and Endothelial Activation in the Hemolytic Uremic Syndrome in Children and Adults (SHU)

The Hemolytic Uremic Syndrome (HUS) is a rare thrombotic microangiopathy (TMA), affecting both children and adults. HUS is characterized by the abnormal occurrence of diffuse thrombosis in the microcirculation resulting in the occurrence of ischemic events affecting especially the kidneys and is associated with hemolytic anemia. One of the major problems encountered in the management of HUS is the absence of reliable marker of treatment response or relapse; conventional hematological markers being too insensitive to judge therapeutic efficacy or identify early relapse. Data from the literature suggest that the endothelial cell is a major target of this syndrome. Our hypothesis is that an initial micro-endothelial activation plays a critical role in the initiation and / or relapse of the disease.The main objective of this study is to define a "vascular competence" profile in a population of patients with typical or atypical HUS; both in the acute phase and in remission of the disease.

Study Overview

• Status: Completed
• Conditions: Hemolytic Uremic Syndrome
• Intervention / Treatment: Biological: Extra blood draw samples

Detailed Description

The Hemolytic Uremic Syndrome (HUS) is a rare thrombotic microangiopathy (TMA), affecting both children and adults. HUS is characterized by the abnormal occurrence of diffuse thrombosis in the microcirculation resulting in the occurrence of ischemic events affecting especially the kidneys and is associated with hemolytic anemia.Its prognosis is severe, with a mortality of 1% in children, 10% in adults and the occurrence of renal failure in 50% of cases.In its typical form, HUS occurs in the aftermath of a diarrheic intestinal infection by bacteria which produce a Shiga toxin. In its unusual shape, which affects both children and adults, there are genetic abnormalities alternate way of regulating complement proteins explaining frequent relapses.One of the major problems encountered in the management of HUS is the absence of reliable marker of treatment response or relapse; conventional hematological markers being too insensitive to judge therapeutic efficacy or identify early relapse. Data from the literature suggest that the endothelial cell is a major target of this syndrome. Our hypothesis is that an initial micro-endothelial activation plays a critical role in the initiation and / or relapse of the disease through the sudden release of high molecular weight ultralarge von Willebrand factor (UL-vWHf) and procoagulant endothelial microparticles.The main objective of this study is to define a "vascular competence" profile in a population of patients with typical or atypical HUS; both in the acute phase and in remission of the disease.

• Study Type: Interventional
• Enrollment (Actual): 19
• Phase: Not Applicable

Contacts and Locations

Study Locations

• France
  • Marseille, France
    • Assistance Publique Hopitaux de Marseille

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Patients with a clinical diagnosis of typical or atypical HUS in acute phase

Exclusion Criteria:

• Patient with positive serological screening test for infection with the HIV.
• Patients with a history of cancer.
• Patients who have undergone organ transplantation or bone marrow.
• Patient with a vivid picture of autoimmune thrombotic thrombocytopenic purpura

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Diagnostic
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: patients with HUS	Biological: Extra blood draw samples

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
number of circulating endothelial cells (CECs) and endothelial progenitor cells (EPCs)	36 months
number of endothelial progenitor cells (EPCs)	36 months

Collaborators and Investigators

• Sponsor: Assistance Publique Hopitaux De Marseille
• Investigators: Study Director: Urielle DESALBRES, Assistance Publique Hopitaux de Marseille

Study record dates

Study Major Dates

• Study Start (Actual): February 11, 2013
• Primary Completion (Actual): April 7, 2017
• Study Completion (Actual): July 27, 2023

Study Registration Dates

• First Submitted: September 8, 2016
• First Submitted That Met QC Criteria: September 13, 2016
• First Posted (Estimated): September 19, 2016

Study Record Updates

• Last Update Posted (Actual): July 28, 2023
• Last Update Submitted That Met QC Criteria: July 27, 2023
• Last Verified: July 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Kidney Diseases; Urologic Diseases; Disease; Hematologic Diseases; Anemia; Thrombocytopenia; Blood Platelet Disorders; Anemia, Hemolytic; Thrombotic Microangiopathies; Uremia; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Syndrome; Hemolysis; Hemolytic-Uremic Syndrome
• Other Study ID Numbers: 2012-03; 2012-A00217-36 (Other Identifier: ANSM)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO