Description Module
The Description Module contains narrative descriptions of the clinical trial, including a brief summary and detailed description. These descriptions provide important information about the study's purpose, methodology, and key details in language accessible to both researchers and the general public.
Description Module path is as follows:
Study -> Protocol Section -> Description Module
Description Module
Ignite Creation Date:
2025-12-24 @ 10:02 PM
Ignite Modification Date:
2025-12-24 @ 10:02 PM
NCT ID:
NCT05053932
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
Study:
NCT05053932
Study Brief:
Protocol Section:
NCT05053932