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Comparative Health Status and Quality of Life of Patients With Sickle Cell Disease (SCD) Who Underwent Matched-sibling Hematopoietic Stem Cell Transplantation Versus Non Transplanted SCD Case-control Patients
Sponsor: Assistance Publique - Hôpitaux de Paris
Summary
The long term burden of morbidity and mortality in the natural history of sickle cell disease has not been compared up to date to the risks and mortality of a curative option like bone marrow transplantation in severe sickle-cell disease patients. Given this lack of data, primary-care Sickle Cell Disease (SCD) physicians and transplant physicians are prevented from a factual debate over the benefit/risk ratio for each patient and refining indications of transplant in patients. Therefore, the present study seeks to describe and compare the very long-term outcomes after either Human Leukocyte Antigen (HLA) -matched sibling transplantation (study arm) and "non-transplant care" for severe sickle cell disease SCA patients in order to yield robust comparative data regarding both arms. The main objective is to assess the benefit of Hematopoietic stem cell transplantation (HSCT) regarding quality of life compared to standard care after 10 years, in patients with severe Sickle Cell Disease (SCD).
Key Details
Gender
All
Age Range
15 Years - 80 Years
Study Type
INTERVENTIONAL
Enrollment
220
Start Date
2024-05-01
Completion Date
2027-05-01
Last Updated
2024-04-19
Healthy Volunteers
No
Conditions
Interventions
Spermogram
Spermogram will be proposed to men
Hospital Anxiety and Depression Scale (HADS)
Anxiety and depression will be evaluated 10 years after HSCT
SF36 Quality of life questionnaire
Quality of life will be evaluated 10 years after HSCT
Psychologist interview
During a follow-up visit
Optional sera banking
One in the study
Optional DNA banking
Once in the study