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
Nathalie Dhedin, MD, PhD
+33-1-4238 51 27


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 2021-11-23