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
Trial Snapshot
- Phase
- Not Applicable
- Status
- Not yet recruiting
- Enrollment
- 220
- Primary Endpoint
- Evaluation of quality of life assessed by SF36
Study Overview
Brief 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).
Study Design
- Study Type
- Interventional
- Allocation
- Non Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Other
- Masking
- None
Eligibility Criteria
- Ages
- 15 Years to 80 Years (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Study population (exposed-patients), all criteria should be fulfilled:
- •Patients alive with Sickle Cell Anemia (SCA, meaning SS and Sbeta0 sickle cell anemia genotype)
- •Patients transplanted from allogeneic HLA-compatible sibling donor or from sibling cord blood unit from the 1st of January 2000 and the 31st of December 2012, whatever the age at transplant
- •Patients having received conditioning regimen containing busulfan 1mg/kg/dose (or equivalent adjusted body-weight dosage according to recommendation) x 16 doses + cyclophosphamide 200mg/kg total dose + anti-thymoglobuline
- •For patient under 18 years at time of enrolment, signed informed consent from both parental representatives
- •For patient aged 18 years old : signed informed consent
- •Having an affiliation to a social security regime
- •Control-population (Non-exposed patients) :
- •For each allografted patient, one non-exposed patient will be matched, based on the following criteria:
- •Age at the date of transplantation of the exposed patient (+/- 1 year)
- •Foetal hemoglobin (HbF) level (+/- 3%) before treatment intensification (defined as the initiation of either hydroxyurea or a transfusion program)
- •Hb level (+/- 0,9 g/dl) before treatment intensification
- •For patient under 18 years at time of enrolment, signed informed consent from both parental representatives
- •For patient above 18 years of age: signed informed consent
- •Having an affiliation of to a social security regime
Exclusion Criteria
- •Study population:
- •Transplantation from donor other than sibling or related cord-blood
- •Conditioning regimen other than busulfan 16mg/kg total dose + cyclophosphamide 200mg/kg total dose + anti-thymoglobuline
- •For both population:
- •Absence of signed informed consent
- •Having any debilitating medical or psychiatric illness, which preclude understanding of the inform consent as well as optimal treatment and follow-up
Arms & Interventions
Patients transplanted from allogeneic HLA-compatible sibling donor or from sibling cord blood unit
Intervention: Spermogram (Other)
Patients transplanted from allogeneic HLA-compatible sibling donor or from sibling cord blood unit
Intervention: Hospital Anxiety and Depression Scale (HADS) (Other)
Patients transplanted from allogeneic HLA-compatible sibling donor or from sibling cord blood unit
Intervention: SF36 Quality of life questionnaire (Other)
Patients transplanted from allogeneic HLA-compatible sibling donor or from sibling cord blood unit
Intervention: Psychologist interview (Other)
Patients transplanted from allogeneic HLA-compatible sibling donor or from sibling cord blood unit
Intervention: Optional sera banking (Other)
Patients transplanted from allogeneic HLA-compatible sibling donor or from sibling cord blood unit
Intervention: Optional DNA banking (Other)
Controlled patients - not transplanted
Intervention: Spermogram (Other)
Controlled patients - not transplanted
Intervention: Hospital Anxiety and Depression Scale (HADS) (Other)
Controlled patients - not transplanted
Intervention: SF36 Quality of life questionnaire (Other)
Controlled patients - not transplanted
Intervention: Psychologist interview (Other)
Controlled patients - not transplanted
Intervention: Optional sera banking (Other)
Controlled patients - not transplanted
Intervention: Optional DNA banking (Other)
Outcomes
Primary Outcomes
Evaluation of quality of life assessed by SF36
Time Frame: 10 years after HSCT
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.
Secondary Outcomes
- Proportion of patients with anxiety and depression(10 years after HSCT)
- Evaluation of gonadal function(10 years after HSCT)
- Evaluation of quality of life(10 years after HSCT)