Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| Centre National de la Recherche Scientifique, France | OTHER |
| Etablissement Français du Sang | OTHER |
Not provided
Not provided
Not provided
Not provided
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.
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
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Patients included in Drepagreffe 1 study (NCT01340404) | biological collection |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| blood collection | Other | blood collection |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Long term evolution (at 9-10 years) of cognitive performance | 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 | within 6 months of inclusion |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Population concerned 67 patients included in 2011-2013 in the DREPAGREFFE protocol [NCT 01340404]
Not provided
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hôpital Robert Debré AP-HP | Paris | IDF | 75019 | France | ||
| Groupe hospitalier Pellegrin |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Long term evolution (at 9-10 years) of quality of life | 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 | within 6 months of inclusion |
| Evolution at 9-10 years of factors of hypoxia and oxidative stress | Assessment on plasma Phosphatidyl-serine expression VEGF, Angiopoietin-1 (Ang-1) and Angiopoietin-2 (Ang-2),EPO, HIF-1, BDNF, PDGF-AA | within 6 months of inclusion |
| Bordeaux |
| 33000 |
| France |
| Centre Hospitalier Intercommunal de Créteil | Créteil | 94000 | France |
| Hôpital Henri Monr - APHP | Créteil | 94000 | France |
| CHU de la Guadeloupe | La Guadeloupe | 97110 | France |
| Hôpital Bicêtre AP-HP | Le Kremlin-Bicêtre | 94270 | France |
| IHOPe | Lyon | 69008 | France |
| CHU de Lyon | Lyon | 69737 | France |
| Hôpital de la Timone | Marseille | 13005 | France |
| CHU de Montpellier | Montpellier | 34295 | France |
| Hôpital Necker - AP-HP | Paris | 75015 | France |
| CH de Pau | Pau | 64000 | France |
| CHU de Rennes | Rennes | 35033 | France |
| Hôpital Hautepierre | Strasbourg | 67200 | France |
| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| D002545 | Brain Ischemia |
| D003251 | Constriction, Pathologic |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D002561 | Cerebrovascular Disorders |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D020763 | Pathological Conditions, Anatomical |
| D013568 | Pathological Conditions, Signs and Symptoms |
Not provided
Not provided
| ID | Term |
|---|---|
| D001800 | Blood Specimen Collection |
| ID | Term |
|---|---|
| D013048 | Specimen Handling |
| D019411 | Clinical Laboratory Techniques |
| D019937 | Diagnostic Techniques and Procedures |
| D003933 | Diagnosis |
| D011677 | Punctures |
| D013514 | Surgical Procedures, Operative |
| D008919 | Investigative Techniques |
Not provided
Not provided