New transplant approach offers hope for severe sickle cell disease

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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