Clinical trial NCT05053932: Long-term Comparative Cerebrovascular Outcome After Transplantation vs Standard Care in Sickle Cell Anemia
Official title: Long-term Comparative Cerebrovascular Outcome After Transplantation vs Standard Care in Children With Sickle Cell Anemia ( DREPAGREFFE-2)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Sickle Cell Disease
Cerebral Ischemia
Stenosis
Keywords: transplantation
Lead sponsor: Centre Hospitalier Intercommunal Creteil
Sponsor class: OTHER
Collaborators: name: Centre National de la Recherche Scientifique, France
name: Etablissement Français du Sang
First posted: 2021-09-23
Enrollment: 67
Sex: ALL
Minimum age (years): 13
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Population concerned 67 patients included in 2011-2013 in the DREPAGREFFE protocol \[NCT 01340404\]

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.

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

Eligibility 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

Primary outcomes:
measure: 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
timeFrame: within 6 months of inclusion
measure: Long term evolution (at 9-10 years) of arterial stenosis on cerebral and cervical magnetic resonance angiography
timeFrame: within 6 months of inclusion
Secondary outcomes:
measure: Long term evolution (at 9-10 years) of cognitive performance
timeFrame: within 6 months of inclusion
measure: Long term evolution (at 9-10 years) of quality of life
timeFrame: within 6 months of inclusion
measure: Evolution at 9-10 years of factors of hypoxia and oxidative stress
timeFrame: within 6 months of inclusion
Other outcomes:
Locations:
city: Paris
country: France
facility: Hôpital Robert Debré AP-HP
state: IDF
city: Bordeaux
country: France
facility: Groupe hospitalier Pellegrin
city: Créteil
country: France
facility: Centre Hospitalier Intercommunal de Créteil
city: Créteil
country: France
facility: Hôpital Henri Monr - APHP
city: La Guadeloupe
country: France
facility: CHU de la Guadeloupe
city: Le Kremlin-Bicêtre
country: France
facility: Hôpital Bicêtre AP-HP
city: Lyon
country: France
facility: IHOPe
city: Lyon
country: France
facility: CHU de Lyon
city: Marseille
country: France
facility: Hôpital de la Timone
city: Montpellier
country: France
facility: CHU de Montpellier
city: Paris
country: France
facility: Hôpital Necker - AP-HP
city: PAU
country: France
facility: CH de Pau
city: Rennes
country: France
facility: CHU de Rennes
city: Strasbourg
country: France
facility: Hôpital Hautepierre
Study officials:
References: