Investigating red blood cell deformability of sickle cell patients who started therapy.

Recruiting

Conditions: hereditary hemoglobinopathy
sickle cell disease
10018902
10005330

Registration Number: NL-OMON54704

Lead Sponsor: niversitair Medisch Centrum Utrecht

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Recruiting

Sex: Not specified

Target Recruitment: 344

Inclusion Criteria

• No blood transfusion within the past 2 months (only in case of hydrea therapy
and newborns)
• Diagnosed with sickle cell disease by electrophoresis or HPLC.
• Starting with Hydrea therapy or getting blood transfusion or HSCT or gene
therapy, or newborn with SCD, or in steady state with treatment or without
treatment. When included in baseline cohort: no treatment, or on chronic blood
transfusion or steady state under hydroxyurea.
• Adults patients or parents/legal guardians (and child depending on age) must
give informed consent

Exclusion Criteria

• Blood transfusion within past 2 months (not a criteria in patients who are
treated with blood transfusion)
• Body weight below 10 kg (not a criteria in newborns)
• Age <1 year (not a criteria in newborns)

Study & Design

Study Type: Observational invasive

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Investigating changes in red blood cell deformability, before and during<br /><br>treatment with hydroxyurea, or before and after blood transfusion, or during<br /><br>the first 9 months of life, as measured with the hyperoxia-hypoxia Lorrca<br /><br>module in SCD patients, patients with SCD and HbC disease, and patients with<br /><br>SCD and thalassemia.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>1. To assess changes in RBC deformability measured with other Lorrca modules<br /><br>during 6 months of HU treatment, or just before and after blood transfusion,<br /><br>before and after HSCT or gene therapy, or during the first 9 months of life.<br /><br>2. To explore the association between ektacytometry measurements at different<br /><br>time points with clinical symptoms and signs, haematological parameters and<br /><br>oxidative stress markers. </p><br>