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Long-term Comparative Cerebrovascular Outcome After Transplantation vs Standard Care in Sickle Cell Anemia (DREPAGREFFE2)

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ClinicalTrials.gov Identifier: NCT05053932
Recruitment Status : Recruiting
First Posted : September 23, 2021
Last Update Posted : November 22, 2022
Sponsor:
Collaborators:
Centre National de la Recherche Scientifique, France
Etablissement Français du Sang
Information provided by (Responsible Party):
Centre Hospitalier Intercommunal Creteil

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.

Condition or disease Intervention/treatment
Sickle Cell Disease Cerebral Ischemia Stenosis Other: blood collection

Detailed Description:
The present observational study has the objective to reevaluate at distance the cohort of 67 children with sickle cell anemia enrolled in the "Drepagreffe"trial because of cerebral vasculopathy detected by transcranial Doppler. Results at 1 and 3 years were reported in JAMA in 2019 in BHJ in 2020. This trial was the first worldwide prospective study comparing transplantation to standard care in sickle cell disease. Velocities were highly significantly more reduced with a higher proportion of patients with normalized velocities and better quality of life after transplantation than on standard care. Despite a trend to a better ischemic lesions outcome at 3 years, the difference was not significant and cognitive performances were not different between both groups. The biologic study only assessed at enrollment and 1-year showed lower levels of Ang-2 and HGF (hepatocyte growth factor) after transplant and a significant and independent association between Doppler normalization probability with low Ang-2 and BDNF (brain-derived neurotrophic factor) levels.The aim of the present study is to reassess at 9-10 years this cohort with grants allowing to reevaluate cognitive functioning and hypoxia/angiogenic factors not realized in the systematic cohort follow-up

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Study Type : Observational
Estimated Enrollment : 67 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Long-term Comparative Cerebrovascular Outcome After Transplantation vs Standard Care in Children With Sickle Cell Anemia ( DREPAGREFFE-2)
Actual Study Start Date : October 7, 2022
Estimated Primary Completion Date : June 2024
Estimated Study Completion Date : June 2025

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Anemia

Group/Cohort Intervention/treatment
Patients included in Drepagreffe 1 study (NCT01340404)
biological collection
Other: blood collection
blood collection




Primary Outcome Measures :
  1. 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 [ Time Frame: within 6 months of inclusion ]
    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.

  2. Long term evolution (at 9-10 years) of arterial stenosis on cerebral and cervical magnetic resonance angiography [ Time Frame: within 6 months of inclusion ]
    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.


Secondary Outcome Measures :
  1. Long term evolution (at 9-10 years) of cognitive performance [ Time Frame: within 6 months of inclusion ]
    Full Scale Intelligence Quotient (40= worst outcome, 160= best outcome) is measured by Wechsler Intelligence Scale for Children -Fourth Edition (WISC-4) for children 7-16 years of age and by WAIS-3 (Weschler Adult Intelligence Scale-3) for patients older than 16 years

  2. Long term evolution (at 9-10 years) of quality of life [ Time Frame: within 6 months of inclusion ]
    Quality of life assessment is collected using the French version of the Pediatric Quality of Life Inventory Generic Core Scale (PedsQLTM 4.0 generic core scales) (physical, emotional, social, school items) (0= worst outcome, 100= best outcome) via self-report and parent proxy-report

  3. Evolution at 9-10 years of factors of hypoxia and oxidative stress [ Time Frame: within 6 months of inclusion ]
    Assessment on plasma Phosphatidyl-serine expression VEGF, Angiopoietin-1 (Ang-1) and Angiopoietin-2 (Ang-2),EPO, HIF-1, BDNF, PDGF-AA


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


Information from the National Library of Medicine

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Ages Eligible for Study:   13 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Population concerned 67 patients included in 2011-2013 in the DREPAGREFFE protocol [NCT 01340404]
Criteria

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • Refusal to participate
  • Patient deceased

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05053932


Contacts
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Contact: Camille JUNG, MD 01 57 02 22 68 ext +33 camille.jung@chicreteil.fr

Locations
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France
Centre Hospitalier Intercommunal de Créteil Recruiting
Créteil, France, 94000
Contact: Françoise BERNAUDIN, MD       francoise.bernaudin@chicreteil.fr   
Principal Investigator: Francoise BERNAUDIN, MD         
Principal Investigator: Corinne GUITTON, MD         
Principal Investigator: Marie PETRAS, MD         
Principal Investigator: Suzanne VERLHAC, MD         
Principal Investigator: Marianne De MONTALEMBERT, MD         
Principal Investigator: Alexandra GAUTHIER, MD         
Principal Investigator: Isabelle THURET, MD         
Principal Investigator: Catherine PAILLARD, PhD         
Sponsors and Collaborators
Centre Hospitalier Intercommunal Creteil
Centre National de la Recherche Scientifique, France
Etablissement Français du Sang
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Responsible Party: Centre Hospitalier Intercommunal Creteil
ClinicalTrials.gov Identifier: NCT05053932    
Other Study ID Numbers: DREPAGREFFE2
First Posted: September 23, 2021    Key Record Dates
Last Update Posted: November 22, 2022
Last Verified: November 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Centre Hospitalier Intercommunal Creteil:
transplantation
Additional relevant MeSH terms:
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Brain Ischemia
Cerebral Infarction
Anemia, Sickle Cell
Ischemia
Anemia
Hematologic Diseases
Pathologic Processes
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hemoglobinopathies
Genetic Diseases, Inborn
Cerebrovascular Disorders
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Vascular Diseases
Cardiovascular Diseases
Brain Infarction
Stroke
Infarction
Necrosis