Status:
COMPLETED
HLA Haploidentical Bone Marrow Transplant in Patients With Severe Sickle Cell Disease
Lead Sponsor:
Centre Hospitalier Intercommunal Creteil
Collaborating Sponsors:
Keocyt
Association Clinique Thérapeutique Infantile du val de Marne
Conditions:
Sickle Cell Disease
Eligibility:
All Genders
13-40 years
Phase:
PHASE2
Brief Summary
multicentric interventional biomedical research phase II, prospective, non-randomized evaluating a haploidentical marrow transplants after reduced-intensity conditioning and prevention of GvHD based o...
Detailed Description
Sickle cell disease is a severe disease with frequent occurrence of painful crises and progressive installation of a multi organ injuries. Despite the progress in its management, particularly since th...
Eligibility Criteria
Inclusion
- Inclusion criteria recipient:
- Age: 13 years-40 years
- Severe Sickle cell with at least one of the following criteria:
- Stenosing vasculopathy with abnormal MRA despite prolonged transfusion program
- PAH confirmed by right catheterization with mPAP\> 25mmHg
- Systolic ejection fraction \<55% and tricuspid regurgitation speed\> 2.5m /s at distance from an acute episode
- No possibility of blood transfusion or very complicated blood transfusion
- Report albumin / creatinine\> 30 mg / mmol, confirmed 3 times, away at distance from acute episode and persistent despite hydroxyurea or IEC
- GFR \<80ml / min /1.73m2 (CKD-Epi without ethnic criterion)
- Previous history of acute liver sequestration with liver failure
- Acute chest syndrome or vaso-occlusive crises under hydroxyurea
- Complications of sickle cell transfusion imposing an exchange program with no possible withdrawal beyond a period of one year
- Not having geno-identical donor, but a haploidentical major donor (parent, sibling, adult child, or HbAA AS)
- Having red and understood the information letter and signed the informed consent
- Patients affiliated to a social security system (Social Security or Universal Medical Coverage)
- Exclusion Criteria recipient:
- Patient with a geno-identical donor
- Performans status: ECOG\> 1
- lung disease: FEV1 and FVC \<50% predicted,
- score of PAH NYHA≥2
- Liver disease with bilirubin\> 50 .mu.mol / L
- heart failure defined by NYHA≥3 score ejection fraction \<45% or shortening fraction \<24%
- anti HLA alloimmunization against the donor or against red cell antigens of the donor
- Serology or HIV viral load positively
- Patients who for family, social or geographical reasons, do not wish to be regularly monitored in consultation
- severe uncontrolled infection at the time of inclusion or graft
- pregnant woman (positive beta HCG) or during lactation
- incapable adult patient, trust, guardianship, or safeguard justice
- Inclusion criteria donor
- Age\> 18 years and \<60 years
- Viral serologic economy allows the graft
- No contraindication for general anesthesia
- No contraindication the administration of G-CSF (the existence of sickle cell trait is not a contraindication)
- Lack antigens HLA recognized by the recipient antibody
- Hemoglobin S \<50%
- When several donors are compatible: choose according to the ABO recipient: prefer ABO compatibility and major incompatibility and minor incompatibility, and finally major and minor incompatibility.
- Signature of informed consent
- Non-inclusion criteria donors: β HCG positive or known pregnancy
Exclusion
Key Trial Info
Start Date :
August 10 2017
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
April 8 2024
Estimated Enrollment :
25 Patients enrolled
Trial Details
Trial ID
NCT03240731
Start Date
August 10 2017
End Date
April 8 2024
Last Update
September 2 2025
Active Locations (7)
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1
CHU Henri-Mondor
Créteil, France, 94000
2
intercommunal hospital of Créteil
Créteil, France, 94000
3
CHU La Timone
Marseille, France
4
Hospital Necker
Paris, France