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
TRANSPLANTORN2
1 other identifier
interventional
220
0 countries
N/A
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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for not_applicable
Started May 2024
Typical duration for not_applicable
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
April 2, 2024
CompletedFirst Posted
Study publicly available on registry
April 8, 2024
CompletedStudy Start
First participant enrolled
May 1, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 1, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
May 1, 2027
April 19, 2024
December 1, 2023
3 years
April 2, 2024
April 17, 2024
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
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.
10 years after HSCT
Secondary Outcomes (3)
Evaluation of gonadal function
10 years after HSCT
Evaluation of quality of life
10 years after HSCT
Proportion of patients with anxiety and depression
10 years after HSCT
Study Arms (2)
Patients transplanted from allogeneic HLA-compatible sibling donor or from sibling cord blood unit
OTHERControlled patients - not transplanted
OTHERInterventions
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
Eligibility Criteria
You may qualify if:
- 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:
- Gender
- 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
- +1 more criteria
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- not applicable
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- OTHER
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 2, 2024
First Posted
April 8, 2024
Study Start
May 1, 2024
Primary Completion (Estimated)
May 1, 2027
Study Completion (Estimated)
May 1, 2027
Last Updated
April 19, 2024
Record last verified: 2023-12