Blood outgrowth endothelial cells as a patient-derived ex vivo model system to study degranulation mechanisms in storage pool disease

Completed

• Conditions: platelet function disorder; storage pool disease; 10035534; 10005330

• Registration Number: NL-OMON42746
• Lead Sponsor: niversitair Medisch Centrum Utrecht

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 23 April 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 10

Inclusion Criteria

- Age >= 18 years
- Diagnosed with storage pool disease using standard luminoaggregometry

Exclusion Criteria

- Inability to give informed consent
- Received a bone marrow or stem cell transplantation

Study & Design

• Study Type: Observational invasive
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Secretory responses of SPD platelets and BOECs as compared to healthy platelets<br /><br>and BOECs.<br /><br>Defects or abnormalities in Weibel-Palade bodies (WPB) biogenesis of SPD BOECs<br /><br>as compared to healthy BOECs.<br /><br>Alterations of SPD whole platelet and BOEC proteomes as compared to already<br /><br>established healthy platelet and BOEC proteomes</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Mepacrine uptake and release of platelets of patients with storage pool disease<br /><br>as compared to healthy platelets<br /><br>Diagnostic utility of mepacrine uptake and release as compared to ATP/ADP<br /><br>ratio, electron microscopy, fluorescence microscopy and measurement of dense<br /><br>granule markers after activation using flow cytometry</p><br>