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NOT YET RECRUITING
NCT06351462
NA

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

View on ClinicalTrials.gov

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

Interventions

OTHER

Spermogram

Spermogram will be proposed to men

OTHER

Hospital Anxiety and Depression Scale (HADS)

Anxiety and depression will be evaluated 10 years after HSCT

OTHER

SF36 Quality of life questionnaire

Quality of life will be evaluated 10 years after HSCT

OTHER

Psychologist interview

During a follow-up visit

OTHER

Optional sera banking

One in the study

OTHER

Optional DNA banking

Once in the study