MedPath

Central Nervous System Vascular Changes in Adult Sickle Cell Disease and the Effect of Treatment With Simvastatin

Phase 1
Conditions
Stroke
Sickle Cell Disease
Interventions
Registration Number
NCT03599609
Lead Sponsor
University of Campinas, Brazil
Brief Summary

Stroke is a frequent complication of sickle cell disease (SCD), with varying levels of central nervous system (CNS) involvement. The summation of several ischemic events, even when silent, can lead to devastating consequences, from reduced academic performance to physical dependence. Despite knowledge that brain flow velocities evaluated by Doppler ultrasound identify pediatric SCD patients at a greater stroke risk (Adams et al, NEJM 1998; 339:5-11), this method is not able to predict the occurrence of strokes in adults. There is also no consensus on the management of adult patients in relation to primary and secondary prevention. The aim of this study is to evaluate the effects of the administration of Simvastatin on CNS structural and functional vascular changes in 30 adult patients with SCD (SS and Sβ), above 35 years of age, observed through Magnetic Resonance Imaging (MRI). The data on the effect of simvastatin on disease manifestations is quite scarce, however this drug reportedly significantly reduces plasma concentrations of adhesion molecules and inflammatory markers, such as E-selectin, VEGF, CRP and IL-6 (Hoppe et al, BJH 2011; 153:655-663; Hoppe et al, BJH 2017;177:620-629). Thus, in addition to the search for early diagnostic markers and risk stratification for primary or recurrent stroke, we will also compare CNS images before and 12 months after the administration of Simvastatin. The drug alter stroke recurrence rates in the general adult population, but their effects on vascular changes in patients with SCD have not yet been adequately elucidated. This is particularly important because these are low cost drugs which present good tolerability, and could be part of the therapeutic arsenal of SCD, even in low income settings. Concomitantly with the CNS evaluation, this study also intends to investigate molecular pathways that may be affected by the drugs. We will evaluate microvesicle release patterns, as well as the content of microRNAs possibly involved in the occurrence of stroke, in addition to metabolomic studies and plasma cytokine profile.

Detailed Description

Not available

Recruitment & Eligibility

Status
UNKNOWN
Sex
All
Target Recruitment
28
Inclusion Criteria
  • Diagnosis of Sickle Cell Disease
Exclusion Criteria
  • Previous stroke
  • Some relevant concomitant clinical condition (cancer, AIDS, inflammatory / autoimmune diseases, etc.).
  • Pregnancy
  • Individuals considered to be vulnerable (minors,institutionalized individuals, patients with a history of psychiatric illness with cognitive impairment or incapacity)

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
TreatmentSimvastatin 40mgTreatment: Simvastatin 40mg/day
Primary Outcome Measures
NameTimeMethod
Stroke prevention5 years

Number of patients in the cohort presenting srtoke or silent infarction detected at MRI

Secondary Outcome Measures
NameTimeMethod
Improvement of hemodynamic parameters in MRI: endothelial shear stress1 year

Evaluation of changes in endothelial shear stress (Pa) in the Circle of Willis

Improvement of hemodynamic parameters in MRI:velocity1 year

Evaluation of changes in vessel segment-averaged velocity (cm/s) in the Circle of Willis

Improvement of hemodynamic parameters in MRI: lumen area1 year

Evaluation of changes in vessel lumen area (mm2) in the Circle of Willis

Improvement of hemodynamic parameters in MRI: flow1 year

Evaluation of changes in flow (ml/s) in the Circle of Willis

Trial Locations

Locations (1)

Hematology and Transfusion Medicine Center

🇧🇷

Campinas, São Paulo, Brazil

© Copyright 2025. All Rights Reserved by MedPath