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).
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
OTHER
Masking
NONE
Enrollment
220
Spermogram will be proposed to men
Anxiety and depression will be evaluated 10 years after HSCT
Quality of life will be evaluated 10 years after HSCT
During a follow-up visit
One in the study
Once in the study
Evaluation of quality of life assessed by SF36
It will be assessed by the Short-form 36 (SF36) scale. The Short Form (36) Health Survey is a 36-item measure if health status. The score obtained varies between 0 and 100. The higher the score the less disability. Ware JE, Sherbourne CD. The Medical Outcomes Study 36-item short-form health survey (SF-36): I. Conceptual framework and item selection. Med Care 1992;30:473-83.
Time frame: 10 years after HSCT
Evaluation of gonadal function
Spermogram in men (proposed not requested), Luteinising Hormone (LH), Follicle-Stimulating Hormone (FSH), estrogen, Anti-Müllerian Hormone (AMH) in women, testosterone in men, Amenorrhea in women.
Time frame: 10 years after HSCT
Evaluation of quality of life
Questionnaire including data about employment, education level, social financial support.
Time frame: 10 years after HSCT
Proportion of patients with anxiety and depression
Anxiety and depression levels will be assessed using the Hamilton Anxiety Depression scale : The HAD scale is a self-assessment scale for detecting states of depression and anxiety in the setting of an hospital medical outpatient clinic. HADS is a self-administered scale of 14 items which assessed levels of depression and anxiety, divided into 2 subscales of 7 items (Anxiety or HADS-A, Depression or HADS-D). Each item is scored on a scale of 0 to 3. A score is generated for each of the two sub-scales (sum of the 7 items, ranging from 0 to 21). Limit scores, for each of the scores, distinguish: non-cases or asymptomatic ones (score ≤ 7); probable or borderline cases (score 8-10); clearly or clinically symptomatic cases (score ≥ 11)
Time frame: 10 years after HSCT
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