HLA Haploidentical Bone Marrow Transplant in Patients With Severe Sickle Cell Disease

Other illness

• lung disease: FEV1 and FVC \<50% predicted,
• Serology or HIV viral load positively
• severe uncontrolled infection at the time of inclusion or graft

Lab values

• Liver disease with bilirubin\> 50 .mu.mol / L
• heart failure defined by NYHA≥3 score ejection fraction \<45% or shortening fraction \<24%

Other

• Patient with a geno-identical donor
• Performans status: ECOG\> 1
• score of PAH NYHA≥2
• anti HLA alloimmunization against the donor or against red cell antigens of the donor
• Patients who for family, social or geographical reasons, do not wish to be regularly monitored in consultation
• pregnant woman (positive beta HCG) or during lactation
• incapable adult patient, trust, guardianship, or safeguard justice

• 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)
• 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