Clinical trial NCT04451785: Hereditary Spherocytosis and Vascular Function
Official title: Relationships Between Hemolysis, Erythrosis, Circulating Microparticles and Vascular Function in Patients With Hereditary Spherocytosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Hereditary Spherocytosis
Keywords: Hereditary spherocytosis
hemolysis
microparticles
vascular function
red blood cell
Lead sponsor: Hospices Civils de Lyon
Sponsor class: OTHER
First posted: 2020-06-30
Enrollment: 72
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: BASIC_SCIENCE

Brief summary: Background : Patients with hereditary spherocytosis (HS) are characterized by increased red blood cell (RBC) fragility and a loss of RBC deformability. While the clinical variability of the disease may be heterogenous from one patient to another, some studies reported the occurrence of vascular complications, notably in patients who have been splenectomized.

Purpose : The aim of the study is to test the associations between the degree of vascular dysfunction and the extent of hemolysis, the amount of circulating microparticles, the level of erythrosis and the degree of RBC biophysical alterations.

Abstract : Recent studies reported the occurrence of vascular complications in patients with HS, notably in patients who have previously been splenectomized. However, the exact reasons of these complications are unknown and no study investigated the vascular function in HS patients.

Main objective Highlight the presence of altered vascular function in HS patients and test the relationships with the level of hemolysis and circulating microparticles.

Secondary objectives To evaluate the associations between clinical severity and 1) the level of vascular dysfunction and 2) several biomarkers (hemolysis, hematological parameters, circulating microparticles, erythrosis, RBC biophysical properties).


Eligibility criteria: Inclusion Criteria:

Healthy controls:

* age ≥ 6 years old
* written, informed and signed consent by the healthy volunteer, or by both parents or legal guardian if the healthy volunteer is a minor
* Healthy volunteer affiliated to a social security scheme or assimilated
* Healthy volunteer not subject to any legal protection measure

Patients with hereditary spherocytosis:

* age ≥ 6 years old
* Patient with hereditary or non-splenectomized spherocytosis
* Written, informed and signed consent by the patient, or by at least one of the two parents or legal guardian if the patient is a minor
* Patient affiliated to a social security scheme or assimilated
* Patient not subject to any legal protection measure

Exclusion Criteria:

Healthy controls:

* Pregnant or lactating woman
* Subjects with hereditary spherocytosis or other characterized condition by chronic hemolysis
* Subjects with known pathology affecting the vascular system
* Blood donation (less than a month old)
* Not affiliated to a social security scheme
* Patient participating in another interventional research protocol that may interfere with this protocol (according to the investigator's judgment).

Patients with hereditary spherocytosis:

* Patient who received a blood transfusion in the 3 months preceding
* Pregnant or lactating woman
* Any disease or condition other than hereditary spherocytosis, chronic or not, likely to induce chronic or acute intravascular hemolysis
* Patient participating in another interventional research protocol that may interfere with this protocol (according to the investigator's judgment).

Primary outcomes:
measure: measurement of pulse wave velocity (PWV)
timeFrame: Day 1
Secondary outcomes:
measure: Hemogram
timeFrame: Day 1
measure: Markers of hemolysis
timeFrame: Day 1
measure: Circulating microparticles
timeFrame: Day 1
measure: Markers of erythrosis
timeFrame: Day 1
measure: Blood viscosity
timeFrame: Day 1
measure: Red blood cell deformability
timeFrame: Day 1
measure: Red blood cell aggregation
timeFrame: Day 1
Other outcomes:
Locations:
city: Lyon
country: France
facility: Institut d'Hématologie et Oncologie PEdiatrique (iHOPe)
city: Lyon
country: France
facility: Hôpital Edouard Herriot
Study officials:
References: