Bone Marrow Transplantation HLA Haploidentical After a Reduced Intensity Conditioning and Prevention of GvHD Based on Post-transplant Cyclophosphamide Administration in Patients With Severe Sickle Cell Disease

About the study

multicentric interventional biomedical research phase II, prospective, non-randomized
evaluating a haploidentical marrow transplants after reduced-intensity conditioning and
prevention of GvHD based on cyclophosphamide administration post transplantation in patients
with severe sickle cell disease.

Study point of contact

Camille Jung, MD, PhD
[email protected]
Nathalie Dhedin, MD, PhD
+33-1-4238 51 27
[email protected]


7 France sites


13 to 40 Years


Phase 2

Study type








participation requirements


– 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)

participation restrictions


– 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


  • Créteil, France, CHU Henri-Mondor, 94000 [Recruiting]
  • Créteil, France, intercommunal hospital of Créteil, 94000 [Recruiting]
  • Marseille, France, CHU La Timone [Not yet recruiting]
  • Paris, France, Hospital Necker [Recruiting]
  • Paris, France, Hospital Robert-Debré [Recruiting]
  • Paris, France, Saint-Louis hospital [Recruiting]
  • Strasbourg, France, CHU Strasbourg [Recruiting]
Last updated 2021-08-02