Long-term Comparative Cerebrovascular Outcome After Transplantation vs Standard Care in Sickle Cell Anemia

NCT05053932 | Completed

• 1 other identifier: DREPAGREFFE2
• Study Type: observational
• Target: 67
• Locations: 1 country
• Sites: 14

Brief Summary

The purpose of the present observational study is to remotely reevaluate the cohort of 67 sickle cell patients with transcranial Doppler-detected cerebral vasculopathy included in the national "Sickle Cell Transplant" protocol and whose 1- and 3-year results were published in JAMA (Journal of the American Medical Association) in 2019 and in BHJ in 2020.

Conditions

Sickle Cell Disease; Cerebral Ischemia; Stenosis

Keywords

transplantation

Outcome Measures

Primary Outcomes (2)

• Long term evolution (at 9-10 years) of Ischemic lesion on brain magnetic resonance imaging 10-year measurement of ischemic lesion on magnetic resonance imaging

  The MRI-scores, ranging from 0 (best outcome) to 10 (worst outcome), are obtained by adding up the ischemic lesion scores from the left and right sides, i.e., 3 for territorial or 2 for border zone (cortical and subcortical), 1 for white matter and 1 for basal ganglia infarcts and 0 if absent on each side.

  within 6 months of inclusion

• Long term evolution (at 9-10 years) of arterial stenosis on cerebral and cervical magnetic resonance angiography

  The MRA stenosis-scores, ranging from 0 (best outcome) to 32 (worst outcome) are defined as the weighted sums over the 8 assessed cerebral arteries, as 0 if no stenosis, 1 if mild stenosis (25-49%), 2 if moderate stenosis (50-74%), 3 if severe stenosis (75-99%), and 4 if occlusion.

  within 6 months of inclusion

Secondary Outcomes (3)

• Long term evolution (at 9-10 years) of cognitive performance

  within 6 months of inclusion

• Long term evolution (at 9-10 years) of quality of life

  within 6 months of inclusion

• Evolution at 9-10 years of factors of hypoxia and oxidative stress

  within 6 months of inclusion

Study Arms (1)

Patients included in Drepagreffe 1 study (NCT01340404)

biological collection

Other: blood collection

Interventions

blood collection OTHER

blood collection

Patients included in Drepagreffe 1 study (NCT01340404)

Eligibility Criteria

• Age: 13 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Population concerned 67 patients included in 2011-2013 in the DREPAGREFFE protocol \[NCT 01340404\]

You may qualify if:

• Patient of legal age or minor who participated in the DREPAGREFFE research protocol \[NCT 01340404\] between December 2010 and June 2013,
• Having read and understood the information letter

You may not qualify if:

• Refusal to participate
• Patient deceased

Sponsors & Collaborators

• Centre Hospitalier Intercommunal Creteil: lead
• Centre National de la Recherche Scientifique, France: collaborator
• Etablissement Français du Sang: collaborator

Study Sites (14)

Hôpital Robert Debré AP-HP

Paris, IDF, 75019, France

Location

Groupe hospitalier Pellegrin

Bordeaux, 33000, France

Location

Centre Hospitalier Intercommunal de Créteil

Créteil, 94000, France

Location

Hôpital Henri Monr - APHP

Créteil, 94000, France

Location

CHU de la Guadeloupe

La Guadeloupe, 97110, France

Location

Hôpital Bicêtre AP-HP

Le Kremlin-Bicêtre, 94270, France

Location

IHOPe

Lyon, 69008, France

Location

CHU de Lyon

Lyon, 69737, France

Location

Hôpital de la Timone

Marseille, 13005, France

Location

CHU de Montpellier

Montpellier, 34295, France

Location

Hôpital Necker - AP-HP

Paris, 75015, France

Location

CH de Pau

Pau, 64000, France

Location

CHU de Rennes

Rennes, 35033, France

Location

Hôpital Hautepierre

Strasbourg, 67200, France

Location

Biospecimen

Retention: SAMPLES WITH DNA

* One blood sample for the checking of anti-erythroid alloimmunisation and exposition of phosphatidyl-serine. * DNA and plasma will be frozen, stored for hypoxia-factors/angiogenesis and nitroso-redox stress analysis

MeSH Terms

Conditions

Anemia, Sickle Cell; Brain Ischemia; Constriction, Pathologic

Interventions

Blood Specimen Collection

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Cerebrovascular Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Vascular Diseases; Cardiovascular Diseases; Pathological Conditions, Anatomical; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Specimen Handling; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Punctures; Surgical Procedures, Operative; Investigative Techniques

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 12, 2021
• First Posted: September 23, 2021
• Study Start: October 7, 2022
• Primary Completion: January 5, 2025
• Study Completion: January 31, 2025
• Last Updated: June 15, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share

Locations

FR (14)